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\n[\/et_pb_sidebar][\/et_pb_column][et_pb_column type=\u201d3_4\u2033 specialty_columns=\u201d3\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|phone\u201d disabled_on=\u201don|on|\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d|35px||0px\u201d custom_padding_tablet=\u201d|35px||35px||true\u201d custom_padding_phone=\u201d\u201d animation_direction=\u201dtop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dEnsaios cl\u00ednicos de medicamentos Progeria: hist\u00f3rico\u201d _builder_version=\u201d4.27.2\u2033 header_4_line_height=\u201d1.2em\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d cabe\u00e7alho_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_2_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_3_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d caixa_sombra_horizontal_tablet=\u201d0px\u201d caixa_sombra_vertical_tablet=\u201d0px\u201d caixa_sombra_desfoque_tablet=\u201d40px\u201d caixa_sombra_espalhada_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/HayleyMichielAmber-Aug09.jpg\")
Os ensaios cl\u00ednicos de medicamentos para Progeria s\u00e3o a melhor esperan\u00e7a para crian\u00e7as com Progeria, testando tratamentos potenciais que podem permitir que vivam vidas mais longas e saud\u00e1veis. Esses ensaios s\u00e3o o \u00e1pice de anos de pesquisa focada em qual medicamento ou combina\u00e7\u00e3o de medicamentos tratar\u00e1 e curar\u00e1 as crian\u00e7as.<\/p>\n
Desde 1999, quando fundamos a PRF e n\u00e3o havia recursos para essas crian\u00e7as, n\u00f3s voamos da obscuridade total, para a descoberta de genes, para os primeiros ensaios cl\u00ednicos de Progeria, para o primeiro tratamento aprovado pela FDA, chamado lonafarnib \u2013 tudo em um ritmo praticamente in\u00e9dito na comunidade cient\u00edfica. E enquanto ajudamos esse punhado de crian\u00e7as, a conex\u00e3o da Progeria com doen\u00e7as card\u00edacas comuns e envelhecimento tem implica\u00e7\u00f5es tremendas para todos n\u00f3s.<\/p>\n
[\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dO que vem por a\u00ed para tratamentos e cura? \u201d _builder_version=\u201d4.27.2\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d custom_margin=\u201d|||15px|false|false\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n
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[\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 preenchimento_personalizado=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dIniciando um novo ensaio cl\u00ednico de medicamentos\u201d _builder_version=\u201d4.27.2\u2033 header_4_line_height=\u201d1.2em\u201d vertical_offset_tablet=\u201d0\u2033 horizontal_offset_tablet=\u201d0\u2033 custom_margin=\u201d||25px\u201d hover_enabled=\u201d0\u2033 header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 texto_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d texto_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d texto_texto_sombra_for\u00e7a_desfocada_tablet=\u201d1px\u201d link_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d link_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d link_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d ul_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d ul_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d ul_texto_sombra_comprimento_for\u00e7a_desfocada_tablet=\u201d1px\u201d ol_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d ol_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d ol_texto_sombra_comprimento_for\u00e7a_desfocada_tablet=\u201d1px\u201d cita\u00e7\u00e3o_texto_sombra_horizontal_comprimento_tablet=\u201d0px\u201d cita\u00e7\u00e3o_texto_sombra_vertical_comprimento_tablet=\u201d0px\u201d cita\u00e7\u00e3o_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_texto_sombra_horizontal_comprimento_tablet=\u201d0px\u201d cabe\u00e7alho_texto_sombra_vertical_comprimento_tablet=\u201d0px\u201d cabe\u00e7alho_texto_sombra_blur_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_2_texto_sombra_horizontal_comprimento_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_vertical_comprimento_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_blur_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_3_texto_sombra_horizontal_comprimento_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_vertical_comprimento_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_blur_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_for\u00e7a_desfoque_tablet=\u201d1px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_for\u00e7a_desfoque_tablet=\u201d1px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_for\u00e7a_desfoque_tablet=\u201d1px\u201d caixa_sombra_horizontal_tablet=\u201d0px\u201d caixa_sombra_vertical_tablet=\u201d0px\u201d caixa_sombra_desfoque_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d sticky_enabled=\u201d0\u2033]<\/p>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/11\/Bastianforweb-449x1024.png\")
<\/strong><\/em><\/strong><\/h4>\n<\/em><\/strong><\/p>\n A PRF, em colabora\u00e7\u00e3o com o patrocinador do estudo sediado na Coreia, PRG Science & Technology (PRG S&T), iniciou a inscri\u00e7\u00e3o para um novo ensaio cl\u00ednico com um medicamento chamado Progerinina. Evid\u00eancias laboratoriais mostram que esse medicamento, quando tomado em combina\u00e7\u00e3o com lonafarnibe, pode ser mais eficaz do que lonafarnibe sozinho. A PRF financiou o trabalho laboratorial que levou \u00e0 forma\u00e7\u00e3o da PRG S&T e seu desenvolvimento da Progerinina. O trabalho pr\u00e9-teste come\u00e7ou no Hospital Infantil de Boston, na expectativa de trazer crian\u00e7as de todo o mundo para se inscreverem neste ensaio nos pr\u00f3ximos meses. Estamos super animados em come\u00e7ar um novo ensaio com um medicamento t\u00e3o promissor e ansiosos para compartilhar mais detalhes com voc\u00ea assim que estiverem dispon\u00edveis.<\/p>\n Entre em contato com Shelby Phillips para saber mais sobre o julgamento. A cofundadora e diretora executiva da PRF, Audrey Gordon, fecha o acordo em junho com o Dr. Bum-Joon Park da PRG S&T para prosseguir com o trabalho pr\u00e9-julgamento.<\/em><\/p>\n <\/em><\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dTerapia de RNA: Estudo de viabilidade de administra\u00e7\u00e3o de medicamentos conclu\u00eddo!\u201d _builder_version=\u201d4.27.2\u2033 header_4_line_height=\u201d1.2em\u201d vertical_offset_tablet=\u201d0\u2033 horizontal_offset_tablet=\u201d0\u2033 custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d cabe\u00e7alho_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_2_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_3_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d caixa_sombra_horizontal_tablet=\u201d0px\u201d caixa_sombra_vertical_tablet=\u201d0px\u201d caixa_sombra_desfoque_tablet=\u201d40px\u201d caixa_sombra_espalhada_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n A PRF deu os primeiros passos envolvendo pacientes em dire\u00e7\u00e3o a um ensaio cl\u00ednico em terapia de RNA \u2013 MUITO emocionante!<\/p>\n Contexto: Em janeiro de 2021, relatamos descobertas revolucion\u00e1rias em terap\u00eauticas de RNA<\/strong>, onde esta terapia inibiu a produ\u00e7\u00e3o de RNA que codifica a prote\u00edna causadora da doen\u00e7a Progeria, progerina<\/strong>. O estudo*, liderado pelo Dr. Francis Collins, Conselheiro Cient\u00edfico da Casa Branca e ex-diretor do Instituto Nacional de Sa\u00fade (NIH), revelou que camundongos com Progeria tratados com um medicamento chamado SRP-2001 reduziu a express\u00e3o prejudicial do mRNA e da prote\u00edna da progerina nos vasos sangu\u00edneos<\/strong>, assim como em outros tecidos. Os vasos sangu\u00edneos eram mais fortes e os ratos mostraram uma aumento da sobreviv\u00eancia de mais de 60%<\/strong> comparado a ratos n\u00e3o tratados. Assim, o trabalho com esta terapia promissora continuou, e n\u00f3s tomamos a\u00a0pr\u00f3ximo passo <\/strong>com um Estudo de viabilidade <\/strong>do seguinte modo:<\/p>\n Normalmente, os terap\u00eauticos de RNA s\u00e3o l\u00edquidos que s\u00e3o injetados intravenosamente (diretamente na veia). No entanto, aqueles com Progeria n\u00e3o seriam capazes de tolerar a administra\u00e7\u00e3o intravenosa da dosagem di\u00e1ria necess\u00e1ria. Assim, a PRF desenvolveu um sistema de administra\u00e7\u00e3o subcut\u00e2nea<\/strong> onde o l\u00edquido pode ser injetado com uma pequena agulha sob a pele. Um estudo de 6 meses no BCH foi conclu\u00eddo para determinar a viabilidade desta abordagem de entrega para aqueles com Prog\u00e9ria<\/strong>. A equipe testou se a administra\u00e7\u00e3o de uma solu\u00e7\u00e3o salina pode ser confortavelmente injetada por via subcut\u00e2nea e qual sistema de inje\u00e7\u00e3o e local seriam mais confort\u00e1veis. Uma vez que os dados deste estudo sejam analisados, estaremos um passo mais perto de um ensaio cl\u00ednico em terapia gen\u00e9tica<\/strong>!<\/p>\n *<\/strong>Erdos, MR, Cabral, WA, Tavarez, UL et al. Uma abordagem terap\u00eautica antisense direcionada para a s\u00edndrome de progeria de Hutchinson\u2013Gilford. NatM\u00e9dico<\/em> (2021).<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dO que vem por a\u00ed para tratamentos e cura? \u201d _builder_version=\u201d4.27.0\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d custom_margin=\u201d|||15px|false|false\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dO que est\u00e1 acontecendo HOJE com os ensaios cl\u00ednicos da Progeria? \u201d _builder_version=\u201d4.27.0\u2033 header_4_line_height=\u201d1.2em\u201d vertical_offset_tablet=\u201d0\u2033 horizontal_offset_tablet=\u201d0\u2033 custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d cabe\u00e7alho_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_2_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_2_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_3_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_3_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_4_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_5_texto_sombra_desfoque_for\u00e7a_tablet=\u201d1px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_horizontal_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d0px\u201d cabe\u00e7alho_6_texto_sombra_comprimento_vertical_tablet=\u201d1px\u201d caixa_sombra_horizontal_tablet=\u201d0px\u201d caixa_sombra_vertical_tablet=\u201d0px\u201d caixa_sombra_desfoque_tablet=\u201d40px\u201d caixa_sombra_espalhada_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n O ensaio mais recente envolveu 2 medicamentos: lonafarnibe<\/strong>\u00a0e um novo medicamento, everolimo<\/strong>. A fase 1, para determinar a dosagem segura e apropriada de everolimus, come\u00e7ou em abril de 2016 e foi conclu\u00edda com sucesso em junho de 2017. A fase 2, que testou a efic\u00e1cia da combina\u00e7\u00e3o de dois medicamentos, come\u00e7ou em julho de 2017 e foi conclu\u00edda em abril de 2022. Sessenta crian\u00e7as de 27 pa\u00edses foram inscritas nesta fase de dois medicamentos!<\/p>\n Everolimus \u00e9 uma forma do medicamento rapamicina; everolimus poderia ser administrado mais facilmente a crian\u00e7as com Progeria porque requer menos coletas de sangue para medir os n\u00edveis do medicamento. Enquanto lonafarnib pode bloquear o desenvolvimento da progerina t\u00f3xica, a rapamicina parece permitir que as c\u00e9lulas limpem a progerina mais rapidamente. Assim, com a rapamicina mirando uma via diferente da lonafarnib, a combina\u00e7\u00e3o pode provar ser um "golpe duplo" para a Progeria - esperan\u00e7osamente um tratamento melhor do que lonafarnib por si s\u00f3.<\/p>\n Agora entramos em um per\u00edodo de an\u00e1lise de dados e esperamos eventualmente publicar os resultados em um peri\u00f3dico cient\u00edfico revisado por pares. Enquanto isso, os participantes do teste foram transferidos para a extens\u00e3o de monoterapia do teste ou para outro programa de acesso. Por qualquer um dos caminhos, os participantes continuam a receber lonafarnib, o padr\u00e3o atual de tratamento.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dHist\u00f3rico de testes em resumo\u201d _builder_version=\u201d4.27.0\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d custom_margin=\u201d|||15px|false|false\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|phone\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.22.1\u2033 custom_margin=\u201d0px||||false|false\u201d custom_padding=\u201d|35px|0px|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dAt\u00e9 o momento, a PRF financiou e coordenou tr\u00eas ensaios cl\u00ednicos\u201d _builder_version=\u201d4.27.0\u2033 text_font=\u201d||||||||\u201d header_4_font=\u201d||||||||\u201d header_4_line_height=\u201d1.2em\u201d custom_padding=\u201d||0px|||\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dno|telefone\u201d global_colors_info=\u201d{}\u201d]<\/p>\n At\u00e9 o momento, a PRF financiou e co-coordenou quatro ensaios cl\u00ednicos<\/strong>. A PRF \u00e9 e sempre foi respons\u00e1vel por todas as despesas do teste, incluindo testes, viagens, alimenta\u00e7\u00e3o, hospedagem, tradutores e equipe. Cada novo teste \u00e9 mais caro que o anterior, pois mais crian\u00e7as se inscrevem para ter uma chance de vidas mais longas e saud\u00e1veis.<\/p>\n [\/et_pb_text][et_pb_button button_url=\u201dhttps:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\u201d url_new_window=\u201don\u201d button_text=\u201dPor favor, ajude-nos a financiar esses testes vitais. Doe hoje\u201d admin_label=\u201dDoe\u201d _builder_version=\u201d4.22.1\u2033 background_layout=\u201ddark\u201d custom_margin=\u201d20px||25px\u201d animation_style=\u201dslide\u201d animation_direction=\u201dleft\u201d animation_intensity_slide=\u201d25%\u201d global_colors_info=\u201d{}\u201d] #1 envolveu um \u00fanico medicamento, lonafarnib, come\u00e7ou em 2007 e provou ser bem-sucedido. Leia tudo sobre a descoberta hist\u00f3rica do tratamento aqui.<\/a><\/p>\n #2, a parte de Viabilidade do \u201cTriple Trial\u201d envolveu 3 medicamentos: lonafarnib, pravastatina e zoledronato. Este \u201cmini trial\u201d de fase 1 de 1 m\u00eas ocorreu em mar\u00e7o de 2009 para determinar se adicionar mais 2 medicamentos ao regime de lonafarnib era seguro para prosseguir com uma popula\u00e7\u00e3o maior (o que era).<\/p>\n #3, o \u201cTriple Trial,\u201d foi uma continua\u00e7\u00e3o do Feasibility trial, e foi um estudo de fase 2, que come\u00e7ou em agosto de 2009. Seu protocolo mudou ao longo de cinco anos, voltando para apenas lonafarnib e reabrindo a inscri\u00e7\u00e3o para que mais crian\u00e7as pudessem participar. Leia mais aqui<\/a>.<\/p>\n #4 \u00e9 o ensaio de dois f\u00e1rmacos, lonafarnib e everolimus. A fase 1, para determinar a dosagem segura e apropriada de everolimus, come\u00e7ou em abril de 2016 e foi conclu\u00edda com sucesso em junho de 2017. A fase 2, que testou a efic\u00e1cia da combina\u00e7\u00e3o de dois f\u00e1rmacos, come\u00e7ou em julho de 2017 e foi conclu\u00edda em abril de 2022. A extens\u00e3o de monoterapia deste ensaio continua hoje.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][et_pb_column_inner tipo=\u201d1_2\u2033 tipo_de_coluna_de_especialidade_salva=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 preenchimento_personalizado=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201dno|desktop\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/02\/Ana-Clara-trial-page.jpg\u201d align=\u201dcenter\u201d align_tablet=\u201dcenter\u201d align_phone=\u201d\u201d align_last_edited=\u201dno|desktop\u201d admin_label=\u201dliv\u201d _builder_version=\u201d4.22.1\u2033 border_width_all=\u201d4px\u201d border_color_all=\u201d#392887\u2033 global_colors_info=\u201d{}\u201d] Em 2006, os pesquisadores identificaram um potencial tratamento medicamentoso para crian\u00e7as com Prog\u00e9ria,\u00a0chamados FTIs.<\/a>\u00a0Pela primeira vez, t\u00ednhamos diante de n\u00f3s um poss\u00edvel tratamento para crian\u00e7as com Prog\u00e9ria.\u00a0<\/b>Tempos emocionantes! O teste cl\u00ednico do medicamento Progeria come\u00e7ou em 7 de maio de 2007 com duas crian\u00e7as \u2013 Meghan e Megan \u2013 chegando ao Hospital Infantil de Boston em Boston, MA para sua primeira de sete visitas ao longo de um per\u00edodo de 2 anos. Nesta primeira visita, elas fizeram testes extensivos e suas primeiras doses do medicamento. Uma m\u00e9dia de duas fam\u00edlias viajou para Boston a cada semana depois disso, at\u00e9 dezembro de 2009, seguido por um per\u00edodo de tempo em que a equipe do teste analisou os muitos milhares de elementos de dados (cada crian\u00e7a passou por mais de 100 testes por visita!) e buscou a publica\u00e7\u00e3o dos resultados.<\/p>\n <\/p>\n [\/et_pb_text][et_pb_testimonial author=\u201dFrancis Collins, MD, PhD\u201d job_title=\u201dDiretor do Instituto Nacional de Pesquisa do Genoma Humano que mapeou o genoma humano, palestrante de workshop e co-descobridor do gene Progeria.\u201d quote_icon_color=\u201d#00b2e2\u2033 portrait_width=\u201d140px\u201d portrait_height=\u201d140px\u201d _builder_version=\u201d4.16\u2033 background_color=\u201d#f7f7f7\u2033 custom_margin_tablet=\u201d\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 custom_css_testimonial_author=\u201dcolor: #00b2e2;||tamanho da fonte: 19px !important;||espessura da fonte: 700;\u201d custom_css_testimonial_meta=\u201destilo da fonte: it\u00e1lico;||espessura da fonte: 700;\u201d box_shadow_style_image=\u201dpreset3\u2033 bloqueado=\u201ddesligado\u201d global_colors_info=\u201d{}\u201d]<\/p>\n \u201cN\u00e3o conhe\u00e7o nenhuma outra doen\u00e7a gen\u00e9tica rara que tenha passado da descoberta gen\u00e9tica para o ensaio cl\u00ednico em menos de quatro anos \u2013 um testemunho fenomenal do trabalho \u00e1rduo da The Progeria Research Foundation.\u201d<\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201d1_2,1_2\u2033 custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dfalse\u201d padding_left_right_link_2=\u201dfalse\u201d _builder_version=\u201d4.16\u2033 custom_margin=\u201d||25px\u201d custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201d1_2\u2033 tipo_de_coluna_de_especialidade_salva=\u201d3_4\u2033 _vers\u00e3o_do_construtor=\u201d4.16\u2033 preenchimento_personalizado=\u201d|20px||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201dno|telefone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/11\/redoof2007photocollage.png\u201d title_text=\u201dredoof2007photocollage\u201d align_tablet=\u201dcenter\u201d align_phone=\u201d\u201d align_last_edited=\u201dno|\u00e1rea de trabalho\u201d admin_label=\u201dcolagem\u201d _builder_version=\u201d4.27.0\u2033 custom_margin=\u201d|-50px|||false|false\u201d global_colors_info=\u201d{}\u201d] <\/p>\n Vinte e oito (28) crian\u00e7as de dezesseis pa\u00edses participaram, com idades entre 3 e 15 anos. As crian\u00e7as retornavam ao Hospital Infantil de Boston a cada quatro meses, para testes e para receber novos suprimentos de medicamentos, e ficavam em Boston por 4 a 8 dias em cada visita. Enquanto estavam em casa, seus m\u00e9dicos mantinham uma vigil\u00e2ncia rigorosa sobre as crian\u00e7as e enviavam relat\u00f3rios peri\u00f3dicos de sa\u00fade para a equipe de pesquisa de Boston. Durante a dura\u00e7\u00e3o do teste, uma m\u00e9dia de 2 crian\u00e7as por semana viajavam para Boston para participar.<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d tipo_de_coluna_de_especialidade_salva=\u201d3_4\u2033 _vers\u00e3o_do_construtor=\u201d4.16\u2033 preenchimento_personalizado=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dQuem, Onde, Quando, Como e Quanto\u2026\u201d _builder_version=\u201d4.27.0\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dOs tr\u00eas primeiros ensaios cl\u00ednicos foram liderados por Mark Kieran MD\u201d _builder_version=\u201d4.27.0\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d custom_margin_tablet=\u201d35px||\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201dno|desktop\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dno|telefone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Os tr\u00eas primeiros ensaios cl\u00ednicos foram liderados por Mark Kieran MD, PhD,\u00a0<\/strong>Diretor, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute e Children's Hospital Boston; Professor Assistente, Departamentos de Pediatria e Hematologia\/Oncologia, Harvard Medical School. O Dr. Kieran \u00e9 um oncologista pedi\u00e1trico com ampla experi\u00eancia com o medicamento em estudo (farnesiltransferase, ou FTI) em crian\u00e7as. Em 2017, ele deixou seu cargo na Dana Farber para trabalhar no setor privado. Os copresidentes foram Monica Kleinman, MD, Diretora da Unidade de Terapia Intensiva M\u00e9dico-Cir\u00fargica, Associada S\u00eanior em Medicina de Cuidados Cr\u00edticos no BCH, Professora Assistente na Harvard Medical School; e Leslie Gordon, MD, PhD, Diretora M\u00e9dica da PRF, Palestrante no BCH e Harvard Medical School, Professora de Pediatria no Hasbro Children's Hospital e Brown University em Providence, RI. O Dr. Kleinman assumiu o papel principal de Pesquisador Principal.<\/p>\n Os ensaios cl\u00ednicos s\u00e3o um esfor\u00e7o colaborativo, envolvendo m\u00e9dicos do Hospital Infantil de Boston, do Instituto de C\u00e2ncer Dana-Farber e do Hospital Brigham and Women's,<\/strong>\u00a0todas as institui\u00e7\u00f5es da Universidade de Harvard. Al\u00e9m disso, m\u00e9dicos e cientistas da The Warren Alpert Medical School da Brown University e do NIH ajudaram a tornar este primeiro e os outros testes um sucesso.<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dComo chegamos a esse ponto?\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Em 2003, a equipe de pesquisa colaborativa da Progeria Research Foundation\u00a0\u00a0descobriu o gene Progeria<\/a>.<\/a> Esta descoberta n\u00e3o s\u00f3 levou a uma maior compreens\u00e3o da Progeria, mas os cientistas agora sabem que estudar a Progeria pode nos ajudar a aprender mais sobre doen\u00e7as card\u00edacas e o processo normal de envelhecimento que afeta a todos n\u00f3s. Desde a descoberta do gene, o apoio de pesquisadores, cl\u00ednicos, fam\u00edlias de crian\u00e7as com Progeria e pessoas como VOC\u00ca nos levaram a outra encruzilhada na busca por um tratamento. Os pesquisadores come\u00e7aram um estudo intenso desta prote\u00edna inimiga chamada\u00a0progerina<\/em>, e em 2006 eles identificaram um potencial tratamento medicamentoso para crian\u00e7as com Prog\u00e9ria, chamado inibidores de farnesiltransferase (FTIs), e conduziram estudos no laborat\u00f3rio que apoiaram um teste humano com o medicamento. O FTI escolhido foi inicialmente fornecido pela Merck e chamado lonafarnib<\/em>.\u00a0Clique aqui<\/a>\u00a0para mais detalhes sobre a pesquisa.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dPor que os pesquisadores pensaram que esse medicamento funcionaria na Prog\u00e9ria?\u201d _builder_version=\u201d4.16\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n C\u00e9lula normal, c\u00e9lula de Prog\u00e9ria, c\u00e9lula de Prog\u00e9ria ap\u00f3s tratamento com FTI.<\/p><\/div>\n A prote\u00edna que acreditamos ser respons\u00e1vel pela Progeria \u00e9 chamada progerina. Para bloquear a fun\u00e7\u00e3o celular normal e causar Progeria, uma mol\u00e9cula chamada \u201cgrupo farnesil\u201d deve ser anexada \u00e0 prote\u00edna progerina. Os FTIs agem bloqueando (inibindo) a liga\u00e7\u00e3o do grupo farnesil \u00e0 progerina. Ent\u00e3o, se o medicamento FTI pode bloquear essa liga\u00e7\u00e3o do grupo farnesil em crian\u00e7as com Progeria, ent\u00e3o a progerina pode ser \u201cparalisada\u201d e a Progeria melhorada.\u00a0\u00a0Clique aqui<\/a>\u00a0para mais informa\u00e7\u00f5es sobre FTIs.<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dComo a PRF financiou o teste?\u201d _builder_version=\u201d4.16\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Gra\u00e7as ao apoio de milhares, conseguimos levantar todos os fundos necess\u00e1rios para cobrir os custos do teste. Nossa sincera gratid\u00e3o vai para todos que contribu\u00edram com seu \u201ctempo, talentos e tesouro\u201d para tornar essa conquista incr\u00edvel poss\u00edvel e, claro, para todas as fam\u00edlias corajosas que participaram.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|35px|35.2344px|35px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dO lonafarnib FTI agora \u00e9 um tratamento comprovado para Prog\u00e9ria.\u201d _builder_version=\u201d4.22.1\u2033 text_font=\u201d||||||||\u201d header_font=\u201d||||||||\u201d header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201dno|telefone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201d2012 2014\u2033 _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d39px||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dno|telefone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Em 2012, os resultados do estudo foram publicados, demonstrando que cada crian\u00e7a experimentou melhora em uma ou mais \u00e1reas, incluindo o sistema cardiovascular vital. Em maio de 2014, um estudo mostrou que um ou mais de 3 medicamentos \u2013 incluindo lonafarnib \u2013 sendo testados em ensaios cl\u00ednicos financiados pela PRF prolongaram a vida \u00fatil; n\u00e3o estava claro qual medicamento teve esse impacto positivo de mudan\u00e7a de vida. No entanto, em abril de 2018, um estudo publicado em O Jornal da Associa\u00e7\u00e3o M\u00e9dica Americana (JAMA)<\/em> relataram que o lonafarnib sozinho prolongou a sobreviv\u00eancia de crian\u00e7as com Prog\u00e9ria em pelo menos 1,6 anos. Clique aqui<\/a>\u00a0<\/a>para detalhes sobre o estudo hist\u00f3rico de descoberta do tratamento de 2012,\u00a0aqui\u00a0<\/a>para obter detalhes sobre as conclus\u00f5es de 2014 e\u00a0aqui<\/a>\u00a0para detalhes sobre o estudo de 2018.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_testimonial portrait_url=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cam-and-DadMarch09.jpg\u201d quote_icon_color=\u201d#00b2e2\u2033 portrait_width=\u201d140px\u201d portrait_height=\u201d140px\u201d _builder_version=\u201d4.16\u2033 background_color=\u201d#f7f7f7\u2033 custom_margin_tablet=\u201d\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 custom_css_testimonial_author=\u201dcor: #00b2e2;||tamanho da fonte: 19px !importante;||peso da fonte: 700;\u201d custom_css_testimonial_meta=\u201destilo da fonte: it\u00e1lico;||peso da fonte: 700;\u201d box_shadow_style_image=\u201dpreset3\u2033 bloqueado=\u201ddesligado\u201d global_colors_info=\u201d{}\u201d]<\/p>\n \u201cTODOS foram t\u00e3o maravilhosos. Para n\u00f3s, voc\u00eas s\u00e3o TODOS ENVIADOS DE DEUS e APRECIAMOS tudo o que voc\u00eas fazem por esses anjinhos. Nossa fam\u00edlia est\u00e1 t\u00e3o emocionada com a anima\u00e7\u00e3o e todos os tipos de emo\u00e7\u00f5es com a viagem de Adalia para Boston neste fim de semana, que n\u00e3o consigo nem come\u00e7ar a digitar as palavras de como estamos nos sentindo.\u201d<\/em><\/p>\n \u201cEste novo medicamento para Zach nos d\u00e1 uma esperan\u00e7a renovada de que seu cora\u00e7\u00e3o ficar\u00e1 mais forte, seu sorriso ficar\u00e1 mais brilhante e sua vida ser\u00e1 mais longa. Este novo teste de medicamento \u00e9 uma resposta \u00e0s nossas ora\u00e7\u00f5es. Obrigado a todos os envolvidos com a PRF que fizeram isso acontecer... os m\u00e9dicos, os pesquisadores e a equipe. Voc\u00eas s\u00e3o nossos her\u00f3is!\u201d<\/em><\/p>\n \u201cEm nome de Cam e da nossa fam\u00edlia, muito obrigado a todos da PRF por tudo o que fizeram! Estar\u00edamos perdidos em um mundo de confus\u00e3o e tristeza sem voc\u00eas. Em vez disso, vivemos em um mundo de esperan\u00e7a e prop\u00f3sito. Obrigado de novo e de novo! Com muito amor e respeito.\u201d\u00a0<\/em><\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_margin=\u201d39px||\u201d preenchimento_personalizado=\u201d0|35px|0|0px|falso|falso\u201d preenchimento_personalizado_tablet=\u201d0px|35px||35px\u201d preenchimento_personalizado_phone=\u201d0px||0px\u201d dire\u00e7\u00e3o_anima\u00e7\u00e3o=\u201ddireita\u201d informa\u00e7\u00f5es_de_cores_global=\u201d{}\u201d][et_pb_column_inner type=\u201dindefinido\u201d tipo_de_coluna_de_especialidade_salva=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 preenchimento_personalizado=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/trophies-3-kids.jpg\u201d align=\u201dcenter\u201d align_tablet=\u201dcenter\u201d align_phone=\u201d\u201d align_last_edited=\u201don|desktop\u201d admin_label=\u201dimagem aloha kids\u201d _builder_version=\u201d4.22.1\u2033 animation_style=\u201dfade\u201d animation_direction=\u201dbottom\u201d global_colors_info=\u201d{}\u201d] <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|30px|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201dno|telefone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dResumo, etc.\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d39px||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dno|telefone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Pesquisadores identificaram dois medicamentos adicionais que, quando usados em combina\u00e7\u00e3o com o medicamento FTI atual sendo testado (lonafarnib), podem fornecer um tratamento ainda mais eficaz para crian\u00e7as com Progeria do que os FTIs sozinhos. Pravastatina e zoledronato foram adicionados ao tratamento atual lonafarnib. Este teste muito maior incluiu 45 crian\u00e7as de 24 pa\u00edses diferentes!<\/p>\n Todos os tr\u00eas medicamentos t\u00eam como alvo diferentes pontos ao longo do caminho que leva \u00e0 produ\u00e7\u00e3o da progerina causadora da doen\u00e7a. Em empolgantes estudos laboratoriais apresentados pelo Dr. Carlos Lopez-Otin da Espanha no Workshop Cient\u00edfico da Progeria Research Foundation de 2007, os dois novos medicamentos melhoraram a doen\u00e7a em c\u00e9lulas de Progeria e prolongaram a vida \u00fatil em modelos de camundongos de Progeria.<\/p>\n Se os tr\u00eas medicamentos administrados neste teste puderem bloquear efetivamente essa liga\u00e7\u00e3o do grupo farnesil, ent\u00e3o a progerina pode ser \u201cparalisada\u201d e a Progeria pode ser melhorada ainda mais do que com o lonafarnib sozinho. A esperan\u00e7a \u00e9 que os medicamentos funcionem como parceiros, para se complementarem, de modo que a prote\u00edna progerina seja mais afetada pela combina\u00e7\u00e3o dos tr\u00eas medicamentos.<\/p>\n A equipe conduziu um mini-teste para 5 crian\u00e7as com Prog\u00e9ria. O curto teste de \u201cviabilidade\u201d de um m\u00eas perguntou se a combina\u00e7\u00e3o de tr\u00eas medicamentos seria bem tolerada, antes de embarcar em um teste internacional maior. Os efeitos colaterais foram aceit\u00e1veis, e a equipe seguiu em frente para o teste de efic\u00e1cia maior.\u00a0\u00a0<\/strong><\/p>\n 45 crian\u00e7as inscritas neste ensaio, de 24 pa\u00edses, falando 17 idiomas. Isso inclui crian\u00e7as que participaram do ensaio somente FTI, os participantes do ensaio de viabilidade e outras crian\u00e7as que eram muito jovens para participar do primeiro ensaio ou crian\u00e7as que descobrimos durante o primeiro ensaio cl\u00ednico (ap\u00f3s o t\u00e9rmino da inscri\u00e7\u00e3o). As crian\u00e7as inscritas no ensaio somente FTI tiveram a oportunidade de se inscrever no ensaio triplo quando participaram de sua \u00faltima visita para o ensaio atual. Isso permitiu que essas crian\u00e7as continuassem tomando FTI sem nenhuma dose esquecida.<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dMedicamentos em teste em resumo\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d39px||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Pravastatina<\/strong>\u00a0(comercializado como Pravachol ou Selektine) \u00e9 um membro da classe de medicamentos das estatinas. \u00c9 geralmente usado para reduzir o colesterol e prevenir doen\u00e7as cardiovasculares.<\/p>\n \u00c1cido zoledr\u00f4nico\u00a0<\/strong>\u00e9 um\u00a0bisfosfonato<\/strong>, geralmente usado como um medicamento \u00f3sseo para melhorar a osteoporose e para prevenir fraturas esquel\u00e9ticas em pessoas que sofrem de alguns tipos de c\u00e2ncer.<\/p>\n Lonafarnibe <\/strong>\u00e9 um\u00a0FTI\u00a0<\/strong>(Inibidor da farnesiltransferase), um medicamento que pode reverter uma anormalidade nas c\u00e9lulas de Progeria em laborat\u00f3rio e melhorou a doen\u00e7a em camundongos com Progeria. Em julho um\u00a0estudar\u00a0<\/a>** foi publicado que n\u00e3o mostrou nenhuma melhora significativa encontrada al\u00e9m da terapia \u00fanica com lonafarnibe. **Gordon, et. al., Ensaio cl\u00ednico de inibidores de farnesila\u00e7\u00e3o de prote\u00ednas lonafarnib, pravastatina e \u00e1cido zoledr\u00f4nico em crian\u00e7as com s\u00edndrome de Hutchinson-Gilford Progeria, Circula\u00e7\u00e3o<\/em>, 10.1161\/CIRCULA\u00c7\u00c3OAHA.116.022188<\/strong><\/p>\n No entanto, o \u201cTriple Trial\u201d foi estendido al\u00e9m do seu prazo original de 2-3 anos e expandido para incluir at\u00e9 80 crian\u00e7as, para que cada crian\u00e7a pudesse ter acesso apenas ao lonafarnib porque sabemos que ele est\u00e1 ajudando as crian\u00e7as. Normalmente, os ensaios cl\u00ednicos seguem seu curso e os pacientes s\u00e3o retirados de todos os medicamentos at\u00e9 a aprova\u00e7\u00e3o do FDA; isso pode levar anos. O PRF garantiu que as crian\u00e7as continuassem a tomar o \u00fanico tratamento conhecido, enquanto elas e seus parceiros de pesquisa continuam explorando op\u00e7\u00f5es de tratamento adicionais (como o everolimus que est\u00e1 sendo testado atualmente).<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dEverolimus\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Everolimus \u00e9 uma forma do medicamento rapamicina; everolimus poderia ser administrado mais facilmente a crian\u00e7as com Progeria porque requer menos coletas de sangue para medir os n\u00edveis do medicamento. Enquanto lonafarnib pode bloquear o desenvolvimento da progerina t\u00f3xica, a rapamicina parece permitir que as c\u00e9lulas limpem a progerina mais rapidamente. Assim, com a rapamicina mirando uma via diferente da lonafarnib, a combina\u00e7\u00e3o pode provar ser um "golpe duplo" para a Progeria - esperan\u00e7osamente um tratamento melhor do que lonafarnib por si s\u00f3.<\/p>\n Rapamicina<\/em> \u00e9 um medicamento aprovado pela FDA que j\u00e1 demonstrou prolongar a vida de modelos de camundongos sem Progeria. Um estudo* realizado por pesquisadores do NIH em Bethesda, MD e do Massachusetts General Hospital em Boston demonstra que a rapamicina diminui a quantidade da prote\u00edna progerina causadora da doen\u00e7a em 50%, melhora o formato nuclear anormal e prolonga a vida \u00fatil das c\u00e9lulas de Progeria em laborat\u00f3rio.<\/p>\n A rapamicina \u00e9 conhecida por suas propriedades antienvelhecimento em camundongos. Essas descobertas s\u00e3o parte de uma lista crescente de estudos que ajudam a validar a teoria de que encontrar a cura para a Progeria tamb\u00e9m pode beneficiar toda a popula\u00e7\u00e3o idosa.<\/p>\n \u00a0* K. Cao, J. J. Graziotto, C. D. Blair, J. R. Mazzulli, M. R. Erdos, D. Krainc, F. S. Collins, \u201cRapamycin Reverses Cellular Fen\u00f3tipos e Aumenta a Elimina\u00e7\u00e3o de Prote\u00ednas Mutantes em C\u00e9lulas da S\u00edndrome de Hutchinson-Gilford Progeria.\u201d Sci. Transl. Med. 3, 89ra58 (2011).<\/p>\n A Progeria Research Foundation forneceu c\u00e9lulas para este projeto a partir do\u00a0Banco de c\u00e9lulas e tecidos PRF<\/a>\u00a0e ajudou a financiar a pesquisa por meio de nosso\u00a0programa de bolsas<\/a>\u00a0\u2013 mais uma prova de que os programas relacionados \u00e0 pesquisa da PRF s\u00e3o essenciais para os avan\u00e7os em dire\u00e7\u00e3o \u00e0 cura.<\/p>\n Este teste de 2 medicamentos foi um esfor\u00e7o colaborativo que se baseou no conhecimento obtido nos testes cl\u00ednicos anteriores da PRF. As crian\u00e7as foram vistas virtualmente pela mesma equipe de m\u00e9dicos do Boston Children's Hospital e do Brigham and Women's Hospital, todos os quais agora t\u00eam experi\u00eancia mundialmente reconhecida em Progeria, bem como nos medicamentos envolvidos.<\/p>\n Sessenta crian\u00e7as de 27 pa\u00edses foram inscritas nesta fase de dois medicamentos. Dados da parte de dois medicamentos do teste est\u00e3o sendo analisados e os resultados est\u00e3o sendo formulados e escritos para publica\u00e7\u00e3o em um peri\u00f3dico cient\u00edfico revisado por pares.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|30px|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px|2px|35px|false|false\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d border_width_top=\u201d10px\u201d border_color_top=\u201d#8fd2ed\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#00b2e2\u2033 global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dMiss\u00e3o\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d25px||25px\u201d custom_padding=\u201d||\u201d animation_direction=\u201ddireita\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dno|telefone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Nosso trabalho com terapias gen\u00e9ticas est\u00e1 avan\u00e7ando a todo vapor! Estudos de terapia de RNA e edi\u00e7\u00e3o de genes de DNA <\/strong>mostraram uma vasta melhora na expectativa de vida dos camundongos Progeria. <\/strong>A PRF continua a investir fundos substanciais no seu desenvolvimento, com a esperan\u00e7a de que estes esfor\u00e7os de investiga\u00e7\u00e3o levar a ensaios cl\u00ednicos e, finalmente, \u00e0 cura.<\/strong><\/p>\n Estas terapias de ponta t\u00eam enorme potencial<\/em>! Com a sua ajuda, a PRF pode continuar avan\u00e7ando o mais r\u00e1pido poss\u00edvel em dire\u00e7\u00e3o aos tratamentos mais eficazes e \u00e0 cura.<\/p>\n [\/et_pb_text][et_pb_button button_url=\u201dhttps:\/\/www.progeriaresearch.org\/lonafarnib-map-program\/\u201d button_text=\u201dPacientes, cuidadores e m\u00e9dicos: Clique aqui para obter detalhes.\u201d admin_label=\u201dBot\u00e3o do PDF Lonafarnib\u201d _builder_version=\u201d4.22.1\u2033 background_layout=\u201descuro\u201d custom_margin=\u201d10px||10px||false|false\u201d z_index_tablet=\u201d500\u2033 button_text_shadow_horizontal_length_tablet=\u201d0px\u201d button_text_shadow_vertical_length_tablet=\u201d0px\u201d button_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d] [\/et_pb_cta][\/et_pb_column][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_cta button_url=\u201dhttps:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\u201d button_text=\u201dDoe agora\u201d admin_label=\u201dJuntos, encontraremos a cura!\u201d module_class=\u201dsign-btn\u201d _builder_version=\u201d4.16\u2033 header_font_size=\u201d25px\u201d background_color=\u201d#29327a\u201d animation_style=\u201dslide\u201d animation_direction=\u201dleft\u201d animation_intensity_slide=\u201d25%\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d body_font_size_tablet=\u201d\u201d body_font_size_phone=\u201d\u201d body_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 border_radii=\u201don|25px|25px|25px|25px\u201d global_colors_info=\u201d{}\u201d button_bg_color__hover_enabled=\u201don\u201d bot\u00e3o_bg_color__hover=\u201d#8fd2ed\u201d bot\u00e3o_border_color__hover_enabled=\u201don\u201d]<\/p>\n [\/et_pb_cta][\/et_pb_column][et_pb_column tipo=\u201d1_2\u2033 _builder_version=\u201d4.16\u2033 preenchimento_personalizado=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2024\/08\/2024-strip-footer-strip-copy.png\u201d title_text=\u201d2024 c\u00f3pia de tira de rodap\u00e9\u201d _builder_version=\u201d4.27.0\u2033 _module_preset=\u201ddefault\u201d custom_margin=\u201d35px||||false|false\u201d global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":" [et_pb_section fb_built=\u201d1\u2033 fullwidth=\u201don\u201d disabled_on=\u201doff|off|off\u201d _builder_version=\u201d4.16\u2033 border_width_bottom=\u201d55px\u201d border_color_bottom=\u201d#29327a\u201d 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[\u2026]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"\t\t\t\t[vc_row][vc_column][vc_custom_heading text=\"Clinical Trials\" font_container=\"tag:h1|text_align:center\" use_theme_fonts=\"yes\"][vc_column_text]From total obscurity in 1999 to first-ever clinical drug trials starting in 2007, PRF is leading the charge in the quest to discover treatments and the cure.\r\n The FTI lonafarnib is now a proven treatment for Progeria. In 2012 the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.\u00a0 And in May 2014, further study revealed lonafarnib (and possibly the other 2 drugs tested in the Triple Trial \u2013 see below) increases estimated lifespan by at least 1.6 years (time will tell if that number increases \u2013 it just hasn\u2019t been long enough to determine.)\u00a0 Click here<\/a> for details on the 2012 study, and here<\/a> for details on the 2014 findings.<\/p>\r\n<\/a>\r\nAlways Moving Forward: The Progeria Triple Drug Trial Begins August 2009<\/strong>\r\n\r\nWho Enrolled in the Triple Drug Trial?<\/b>\r\n\r\nThe Progeria Research Foundation and Boston Children\u2019s Hospital once again partnered to conduct a new clinical trial for children with Progeria. This much larger trial initially included 45 children from 24 different countries!\r\n\r\n\u00a0\r\n\r\n[caption id=\"\" align=\"alignright\" width=\"200\"] \u201cOn behalf of Cam and our family, thank you all at PRF so much for all you have done! We would have been lost in a world of confusion and grief without you. Instead, we live in a world of hope and purpose. Thank you again and again! With much love and respect,\u201d\u00a0<\/em><\/p>\r\nThere were changes to the Triple Trial protocol.<\/b>\r\n\r\n[caption id=\"\" align=\"alignright\" width=\"150\"]Iniciando um novo ensaio cl\u00ednico de medicamentos: as inscri\u00e7\u00f5es para o ensaio com progerinina j\u00e1 come\u00e7aram!<\/em><\/strong><\/h4>\n
E-mail: sphillips@progeriaresearch.org<\/a>
WhatsApp, Telegrama, WeChat: 1-978-876-2407
Telefone do escrit\u00f3rio: 978-548-5308<\/p>\nTerapia de RNA: Administra\u00e7\u00e3o de Medicamentos<\/strong> Estudo de viabilidade conclu\u00eddo!<\/strong><\/h4>\n
O que est\u00e1 acontecendo HOJE com os ensaios cl\u00ednicos da Prog\u00e9ria?<\/strong><\/h2>\n
Hist\u00f3rico de julgamentos em resumo<\/b><\/h2>\n
\n[\/et_pb_button][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201d1_2,1_2\u2033 custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201d1_2\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dDetalhes sobre os testes anteriores\u201d _builder_version=\u201d4.27.0\u2033 global_colors_info=\u201d{}\u201d]<\/p>\nDetalhes sobre os ensaios anteriores<\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_margin=\u201d||25px\u201d custom_padding=\u201d|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d35px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201d7 de maio de 2007: O in\u00edcio do primeiro ensaio cl\u00ednico do medicamento Progeria marca um momento hist\u00f3rico na hist\u00f3ria da pesquisa sobre Progeria!\u201d _builder_version=\u201d4.27.2\u2033 z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n7 de maio de 2007: O in\u00edcio do primeiro ensaio cl\u00ednico do medicamento Progeria marca um momento hist\u00f3rico na hist\u00f3ria da pesquisa sobre Progeria!<\/strong><\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=\u201d1_2\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|desktop\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dVinte e oito (28) crian\u00e7as de dezesseis pa\u00edses participaram\u201d _builder_version=\u201d4.27.0\u2033 custom_margin=\u201d|||-30px|false|false\u201d custom_margin_tablet=\u201d35px||\u201d margem_personalizada_telefone=\u201d\u201d margem_personalizada_\u00faltima_edi\u00e7\u00e3o=\u201dno|desktop\u201d global_colors_info=\u201d{}\u201d]<\/p>\nQuem, onde, quando, como e quanto\u2026<\/strong><\/h2>\n
Como chegamos a esse ponto?<\/b><\/h4>\n
Por que os pesquisadores pensaram que esse medicamento funcionaria na Prog\u00e9ria?<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/FTI-3-cell-image-crpd-300x71.jpg\")
Como a PRF financiou o teste?
<\/strong><\/h4>\nO FTI lonafarnib \u00e9 agora um tratamento comprovado para a Progeria<\/strong>.<\/h2>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 module_id=\u201dthreeCT\u201d _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dO teste do medicamento triplo da Prog\u00e9ria come\u00e7a em agosto de 2009\u2033 _builder_version=\u201d4.22.1\u2033 text_font=\u201d||||||||\u201d header_font=\u201d||||||||\u201d header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d global_colors_info=\u201d{}\u201d]<\/p>\nSempre avan\u00e7ando: o teste triplo de medicamentos para progeria come\u00e7a em agosto de 2009<\/strong><\/h2>\n
Resumo:<\/strong><\/h4>\n
Estrat\u00e9gia:<\/strong><\/h4>\n
Meta:<\/strong><\/h4>\n
O teste de viabilidade:<\/strong><\/h4>\n
O ensaio de efic\u00e1cia:<\/strong><\/h4>\n
Medicamentos experimentais em resumo<\/strong><\/h4>\n
Todos os tr\u00eas medicamentos bloqueiam a produ\u00e7\u00e3o da mol\u00e9cula farnesil, necess\u00e1ria para que a progerina crie a doen\u00e7a na Prog\u00e9ria.
<\/em>
*<\/em>\u00a0\u201c<\/strong>Tratamento combinado com estatinas e aminobifosfonatos prolonga a longevidade em um modelo murino de envelhecimento humano prematuro"<\/strong>, de Ignacio Varela, Sandrine Pereira, Alejandro P. Ugalde, Claire L. Navarro, Mar\u00c4\u00b1a F. Suarez, Pierre Cau, Juan Cadinanos, Fernando G. Osorio, Nicolas Foray, Juan Cobo, Felix de Carlos, Nicolas Levy, Jose Deputados Freije e Carlos Lopez-Otan. Medicina Natural, 2008. 14(7): p. 767-72.<\/p>\nA adi\u00e7\u00e3o de um mais novo<\/strong> Medicamento: Everolimus<\/strong><\/h4>\n
A ci\u00eancia por tr\u00e1s da adi\u00e7\u00e3o deste segundo medicamento<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cao-with-caption.jpg\")
<\/em><\/p>\nNossa busca pela cura continua\u2026<\/strong><\/h4>\n
\n[\/et_pb_button][\/et_pb_column_inner][\/et_pb_row_inner][\/et_pb_column][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 module_class=\u201drodap\u00e9\u201d _builder_version=\u201d4.21.0\u2033 background_color=\u201d#29327a\u201d custom_margin=\u201d-2px|||||\u201d custom_padding=\u201d0|0px|0|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d12px\u201d border_color_top=\u201d#00b2e2\u2033 global_module=\u201d133\u2033 locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_row_inner column_structure=\u201d1_3,1_3,1_3\u2033 disabled_on=\u201don|on|on\u201d admin_label=\u201dLinha\u201d _builder_version=\u201d4.21.0\u2033 _module_preset=\u201ddefault\u201d min_height=\u201d277px\u201d custom_margin=\u201d|20px|30px||false|false\u201d custom_padding=\u201d0px|30px|0px||false|false\u201d disabled=\u201don\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201d1_3\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.21.0\u2033 _module_preset=\u201dpadr\u00e3o\u201d global_colors_info=\u201d{}\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/08\/Startbanner-1.png\u201d title_text=\u201dStartbanner\u201d url=\u201dhttps:\/\/www.gravoc.com\/\u201d align=\u201dright\u201d _builder_version=\u201d4.21.0\u2033 _module_preset=\u201dpadr\u00e3o\u201d custom_margin=\u201d|-50px||20px|false|false\u201d global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner tipo=\u201d1_3\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.21.0\u2033 _module_preset=\u201dpadr\u00e3o\u201d global_colors_info=\u201d{}\u201d][\/et_pb_column_inner][et_pb_column_inner tipo=\u201d1_3\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.21.0\u2033 _module_preset=\u201dpadr\u00e3o\u201d global_colors_info=\u201d{}\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/07\/RFRFinish.png\u201d title_text=\u201dRFRFinish\u201d _builder_version=\u201d4.21.0\u2033 _module_preset=\u201ddefault\u201d custom_margin=\u201d|20px||-50px|false|false\u201d global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row column_structure=\u201d1_4,1_4,1_2\u2033 make_equal=\u201don\u201d module_class=\u201d et_pb_row_fullwidth\u201d _builder_version=\u201d4.16\u2033 width=\u201d89%\u201d width_tablet=\u201d80%\u201d width_phone=\u201d\u201d width_last_edited=\u201dem|\u00e1rea de trabalho\u201d max_width=\u201d89%\u201d max_width_tablet=\u201d80%\u201d max_width_phone=\u201d\u201d max_width_last_edited=\u201dem|\u00e1rea de trabalho\u201d z_index_tablet=\u201d500\u2033 make_fullwidth=\u201dem\u201d width_unit=\u201ddesligado\u201d custom_width_percent=\u201d100%\u201d global_colors_info=\u201d{}\u201d][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_cta button_url=\u201dhttps:\/\/www.progeriaresearch.org\/newsletter-signup\/\u201d button_text=\u201dInscreva-se agora\u201d admin_label=\u201dInscreva-se para receber o boletim informativo\u201d module_class=\u201dsign-btn\u201d _builder_version=\u201d4.16\u2033 header_font_size=\u201d25px\u201d background_color=\u201d#29327a\u201d animation_style=\u201dslide\u201d animation_direction=\u201dleft\u201d animation_intensity_slide=\u201d25%\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 border_radii=\u201dligado|25px|25px|25px|25px\u201d global_colors_info=\u201d{}\u201d button_bg_color__hover_enabled=\u201dligado\u201d button_bg_color__hover=\u201d#8fd2ed\u201d button_border_color__hover_enabled=\u201dligado\u201d]<\/p>\nInscrever-se<\/h2>\n
para o nosso<\/h2>\n
Boletim informativo!<\/h2>\n
Juntos, n\u00f3s<\/h2>\n
VAI<\/em><\/h2>\n
encontre a cura!<\/h2>\n
Trials history at-a-glance:<\/strong><\/h4>\r\nIn just 17 years, since we founded PRF and there were no resources for these children, we have soared from total obscurity, to gene finding, to the first clinical trials in Progeria, to a first-ever treatment \u2013 all at a pace virtually unheard of in the scientific community. And while helping this handful of children, the connection of Progeria to common heart disease and aging has tremendous implications for us all.\r\n\r\nTo date, PRF has funded and co-coordinated four clinical trials. PRF is and always has been responsible for\u00a0all trial expenses, including testing, travel, food, lodging, translators and staff.\u00a0\u00a0 Each new trial is more expensive than the last, as more children enroll for a chance at longer, healthier lives. Please help us fund these vital trials \u2013 DONATE TODAY!<\/a>\r\n\r\n#1<\/strong> involved a single drug lonafarnib, began in 2007, and proved successful;\u00a0<\/a>\r\n\r\n#2<\/strong> was a 1-month, phase 1 \u201cmini trial\u201d in March 2009 to determine if adding 2 more drugs to the lonafarnib regimen (pravastatin and zoledronate) was safe to move forward with a larger population (which it was);<\/a>\r\n\r\n#3<\/strong>, the \u201cTriple Trial\u201d involved the 3 drugs described above (lonafarnib, pravastatin and zoledronate) and began August 2009.\u00a0 Its protocol changed over the course of 5 years, switching back to just lonafarnib and re-opening enrollment so more children could participate; and<\/a>\r\n\r\n#4<\/strong> is a 2-drug trial involving the treatment lonafarnib and a new drug everolimus.\u00a0 It began in April 2016 and is ongoing.\u00a0 Phase 1, to determine the dosage of everolimus, was successfully completed in June 2017. Phase 2, which will test the effectiveness of the 2-drug combination, began in July 2017.\u00a0 As of February 2018, 41 children from 18 countries have enrolled.\u00a0<\/a>\r\n\r\nClick here to learn more about the current clinical trial.<\/a>\r\n\r\nRead all about the historic treatment discovery here,\u00a0<\/a>and below is the detailed history of these 4 trials.[\/vc_column_text][vc_column_text]<\/a>May 7, 2007:\u00a0The Start of the First-Ever Progeria Clinical Drug Trial Marks Historic Moment in Progeria Research History!<\/strong>\r\n
![](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/2-megans-frnt-cover.jpg\")
\r\n\r\nIn 2006, researchers identified\u00a0a potential\u00a0drug treatment\u00a0for children with\u00a0Progeria,\u00a0called FTIs.<\/a>\u00a0For the first time, we had in front of us a possible treatment for children with Progeria.\u00a0<\/b>Exciting times! \u00a0The Progeria clinical drug trial began on May 7th, 2007 with two children \u2013 Meghan and Megan - arriving at Boston Children\u2019s Hospital in Boston, MA for their first of seven visits over a 2-year period. At this first visit, they were given extensive tests and their first doses of the drug. An average of two families traveled to Boston each week thereafter, through December 2009, followed by a period of time in which the trial team analyzed the many thousands of data elements (each child underwent over 100 tests per visit!)and sought publication of the results.\r\n\r\n\u201cI know of no other rare genetic disease that has gone from gene discovery to clinical trial in under four years - a phenomenal testament to the hard work of The Progeria Research Foundation.\u201d \u00a0Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.<\/i>\r\n\r\nTwenty-eight (28) children from sixteen countries participated, ages 3 to 15 years.\u00a0Children returned to Children\u2019s Hospital Boston every four months, for testing and to receive new drug supply, and stayed in Boston for 4-8 days each visit. \u00a0While at home, their doctors kept a close watch over the children and submitted periodic health reports to the Boston research team.\u00a0 For the duration of the trial, an average of 2 children per week traveled to Boston to participate.\r\n![\"Children](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/Trial-kids-words-flags.jpg\")
<\/p>\r\nThe Progeria Clinical Drug Trial:\u00a0 Who, Where, When, How and How Much\u2026<\/strong>\r\n\r\nThe first three clinical trials were led by Mark Kieran MD, PhD, <\/b>Director, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute and Children\u2019s Hospital Boston; Assistant Professor, Departments of Pediatrics and Hematology\/Oncology, Harvard Medical School.\u00a0Dr. Kieran is a pediatric oncologist with extensive experience with the drug under study (farnesyltransferase, or FTI) in children. Co-Chairs were Monica Kleinman, MD, Director of Medical-Surgical Intensive Care Unit, Sr. Associate in Critical Care Medicine at BCH, Assistant Prof. at Harvard Medical School; and\u00a0 Leslie Gordon, MD, PhD, Medical Director of PRF, Lecturer at BCH and Harvard Medical School, Associate Prof. of Pediatrics at Hasbro Children's Hospital and Brown University in Providence, RI.\u00a0 All three continue to lead the current trial, with Dr. Kleinman assuming the lead role of Principal Investigator.\u00a0The clinical trials are a collaborative effort, involving physicians at Boston Children\u2019s Hospital, Dana-Farber Cancer Institute, and Brigham and Women\u2019s Hospital,<\/b> all Harvard University institutions.\u00a0In addition, physicians and scientists from The Warren Alpert Medical School at Brown University and NIH helped to make this first and the other trials a success.\r\n\r\nHow did we get to this point?<\/b>\r\n\r\nIn 2003, The Progeria Research Foundation\u2019s collaborative research team\u00a0 discovered the Progeria gene<\/a>.<\/a>\u00a0\u00a0This discovery not only led to further understanding of Progeria, but scientists now know that studying Progeria can help us learn more about heart disease and the normal aging process that affects us all. \u00a0 Since the\u00a0gene discovery, the support of researchers, clinicians, families of children with Progeria and people like\u00a0YOU brought us to another crossroads in the search for a treatment. Researchers began an intense study of this enemy protein called progerin<\/i>, and in 2006 they identified\u00a0a potential\u00a0drug treatment\u00a0for children with\u00a0Progeria,\u00a0called farnesyltransferase inhibitors (FTIs), and conducted studies in the lab that supported a human trial with the drug. The FTI chosen is supplied by Merck and called lonafarnib<\/i>. \u00a0Click here<\/a>\u00a0for more details on the research.\r\n\r\nWhy did researchers think this drug would work in Progeria?<\/b>\r\n\r\nThe protein that we believe is responsible for Progeria is called progerin.\u00a0In order to block normal cell function and cause Progeria, a molecule called a \u201cfarnesyl group\u201d must be attached to the progerin protein. FTIs act by blocking (inhibiting) the attachment of the farnesyl group onto progerin. \u00a0So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be \u201cparalyzed\u201d and Progeria improved.\u00a0Click here<\/a> for more information on FTIs.\r\n\r\n[caption id=\"\" align=\"aligncenter\" width=\"375\"]![\"Normal](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/FTI-3-cell-image-crpd.jpg\")
Normal cell, Progeria cell, Progeria cell after being treated with FTI.<\/strong><\/span>[\/caption]\r\n\r\nHow did PRF fund the trial? <\/b>\r\n\r\nThanks to the support of thousands, we were able to raise all the funds necessary to cover the trial costs, including clinical testing, translators, staff, travel, food and lodging for the 28 families from 16 countries who came to Boston every four months for two years.\u00a0 Our heartfelt gratitude goes out to everyone who contributed their \u201ctime, talents and treasure\u201d to make this incredible achievement possible, and of course to all the courageous families who participated.\r\n\r\n[caption id=\"\" align=\"aligncenter\" width=\"300\"]![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/trophies-3-kids.jpg\")
All of the children received trophies at their final, 2-year visit for the first-ever Progeria clinical drug trial. Here, Mateo, Milagros and Jesper are thrilled to get their awards<\/span>.<\/strong><\/span>[\/caption]\r\n\r\n\u00a0\r\n![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/HayleyMichielAmber-Aug09.jpg\")
Hayley from England and Michiel from Belgium are all smiles as they pose with their trophies for completing the first-ever Progeria clinical drug trial in August, 2009. They also, along with Michiel\u2019s sister Amber (right), completed their first visit for the triple drug trial that week.<\/strong><\/span>[\/caption]\r\n\r\n\u00a0\r\n\r\nSummary:<\/b>\u00a0Researchers identified two additional drugs that, when used in combination with the current FTI drug being tested (lonafarnib), may provide an even more effective treatment for children with Progeria than FTI\u2019s alone. Pravastatin and zoledronate were added to the current treatment lonafarnib.\r\n\r\nStrategy:<\/i><\/b> All three drugs target different points along the pathway leading to production of the disease-causing progerin. In exciting laboratory studies presented by Dr. Carlos Lopez-Otin of Spain at the 2007 Progeria Research Foundation Scientific Workshop, the two new drugs improved disease in Progeria cells and extended lifespan in mouse models of Progeria.\r\n\r\nGoal:<\/i><\/b> If the three drugs administered in this trial can effectively block this farnesyl group attachment, then progerin may be \u201cparalyzed\u201d and Progeria may be improved even more than it is with the lonafarnib alone. The hope is that the drugs will work as partners, to complement each other so that the progerin protein is affected more by combining the three drugs.\r\n\r\nThe Feasibility Trial:<\/b> The team conducted a mini-trial for 5 children with Progeria. The short, one-month \u201cfeasibility\u201d trial asked whether the three-drug combination would be well-tolerated, prior to embarking on a larger international trial. Side effects were acceptable, and the team moved ahead to the larger efficacy trial. \u00a0<\/b>\r\n\r\nThe Efficacy Trial:<\/b>\u00a045 children enrolled in this trial, from 24 different countries, speaking 17 different languages. This includes children that participated in the FTI-only trial, the 5 participants in the feasibility trial, and other children that were either too young to participate in the first trial or children that we discovered during the first clinical trial (after enrollment had ended). Children enrolled in the FTI-only trial had the opportunity to enroll in the triple trial when they participated in their last visit for the current trial. This allowed those children to continue taking FTI without any missed doses.\r\n\r\nThe Treatment\/Progeria Relationship <\/b>\r\n<\/b>How did we get from gene discovery to drug therapy for children with Progeria? Finding the gene for Progeria was the key. This gene is called LMNA, and it normally encodes a protein called prelamin A (this protein is further processed and becomes lamin A). Children with Progeria have a mutation in LMNA which leads to the production of an abnormal form of prelamin A called \u201cprogerin.\u201d Many years\u2019 worth of basic research on prelamin A and lamin A gave us the ability to understand that the drugs administered in this trial may affect disease in Progeria. Over the past six years, research has focused on systematically testing these drugs on Progeria cells and Progeria mice.\r\n\r\nThe Clinical Trial Team<\/b>\r\nSince the first trial began in May 2007, a 28-member team has treated children with Progeria from around the globe. Members of the team have expertise not only in Progeria, but also in the drugs administered.\r\n\r\nTrial Medications at a Glance<\/b>\r\nPravastatin<\/b> (marketed as Pravachol or Selektine) is a member of the drug class of statins. It is usually used for lowering cholesterol and preventing cardiovascular disease.\r\nZoledronic acid <\/b>is a bisphosphonate<\/b>, usually used as a bone drug for improving osteoporosis, and to prevent skeletal fractures in people suffering from some forms of cancer.\r\nLonafarnib<\/b> is an FTI <\/b>(Farnesyltransferase inhibitor), a drug that can reverse an abnormality in Progeria cells in the laboratory, and has improved disease in Progeria mice.\r\n\r\nAll 3 drugs block the production of the farnesyl molecule that is needed for progerin to create disease in Progeria. <\/i>\r\n<\/i>\r\n*<\/i><\/b> \u201c<\/b>Combined treatment with statins and aminobisphosphonates extends longevity in a mouse model of human premature aging\u201d<\/b>, by Ignacio Varela, Sandrine Pereira, Alejandro P. Ugalde, Claire L. Navarro, Mar\u00c4\u00b1a F. Suarez, Pierre Cau, Juan Cadinanos, Fernando G. Osorio, Nicolas Foray, Juan Cobo, Felix de Carlos, Nicolas Levy, Jose MP Freije and Carlos Lopez-Ot\u00c4\u00b1n. Nature Medicine, 2008. 14(7): p. 767-72.\r\n<\/a>\r\nThe new generation of children PRF is helping<\/b>\r\n![](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/AdaliaJAN09.jpg\")
\r\n\r\nIn March 2009, five children, ages 2-3, participated in the one-month feasibility study to determine if the side effects of the three drugs taken together were tolerable. The results were positive, paving the way for the full Triple Drug Trial to (initially) enroll up to 45 children with Progeria. Hats off to these amazing families! Here is what some of them had to say:\r\n<\/b>\r\n<\/b>\u201cEVERYONE has been so wonderful. To us you are ALL GOD SENT and we APPRECIATE all that you do for these little angels. Our family is so overwhelmed with excitement and all sorts of emotions with Adalia\u2019s trip to Boston this weekend, I can\u2019t even begin to type the words of how we are feeling.\"<\/em>\r\n\r\n\u201cThis new medication for Zach gives us a renewed hope that his heart will be stronger, his smile will be brighter and his life will be longer. This new drug trial is an answer to our prayers. Thank you to everyone involved with PRF who made this happen...the doctors, the researchers and the staff. You are our heroes!\u201d<\/em>\r\n![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/Cam-and-DadMarch09.jpg\")
Cam and his dad learn how to mix the FTI drug with a sweetener.<\/strong><\/span>[\/caption]\r\n\r\nThe \u201cTriple Trial\u201d was extended beyond its original 2-3-year timeframe, and expanded to include up to 80 children, so that every child could have access to treatment that may give them longer and healthier lives. However, during this last trial phase, all children are taking lonafarnib alone while the trial team analyzes the many thousands of data elements (each child underwent over 100 tests per visit!) on the three-drug therapy phase of the trial.\u00a0\u00a0 The triple therapy trial was pre-designed to include lonafarnib, pravastatin, and zoledronate.\u00a0 The hope is that adding two additional drugs to lonafarnib will boost the beneficial effects of the single drug therapy.\u00a0 While we know that lonafarnib is helping the children, it is still too early to tell if the other two are boosting the benefit.\u00a0 Usually, clinical trials run their course and the patients are taken off all the drugs until the data is analyzed to see if it helps; this could take years.\u00a0 PRF and the Boston Children\u2019s Hospital team made the important decision to add additional time of lonafarnib treatment<\/b> while the trial team explores other potential benefits and tracks any long-term side effects of lonafarnib, including lifespan. While the children continue to take the one known treatment, PRF and its research partners continue exploring additional treatment options - and have found one that they began testing in April 2016! Thus this last, lonafarnib only phase of the \u201cTriple Trial\u201d is overlapping with PRF\u2019s fourth trial, and all known children living with Progeria today have the opportunity to participate in a clinical trial.<\/a>\r\n\r\nWe are very happy with the visit\u2026To know that we will have a better quality of life for my baby makes me feel amazing, as my wife says he is a \"Warrior of Life \".<\/i>\r\n\r\nNew Drug, New Hope for Children with Progeria: Phase 1, 2-Drug Trial Began April 2016<\/strong>\r\n\r\nPRF is thrilled to announce that we are now funding and co-coordinating a new clinical trial, which will assess a two-drug combination of lonafarnib plus everolimus.\u00a0 Everolimus is a form of the drug rapamycin, <\/b>but everolimus can be more easily given to the children with Progeria because it requires fewer blood draws to measure drug levels.\u00a0 While lonafarnib may block progerin from developing, rapamycin appears to allow cells to more rapidly clear out the toxic progerin.\u00a0 Thus with rapamycin targeting a different pathway than lonafarnib, the combination may prove to be a \u201cone-two punch\u201d to Progeria - hopefully a better treatment than lonafarnib on its own.\r\n\r\nRapamycin<\/em> is an FDA-approved drug that has previously been shown to extend the lives of non-Progeria mouse models.\u00a0 A study* by researchers at the NIH in Bethesda, MD and Massachusetts General Hospital in Boston demonstrates that rapamycin decreases the amount of the disease-causing protein progerin by 50%, improves the abnormal nuclear shape, and extends the lifespan of Progeria cells in the laboratory.\r\n\r\nRapamycin is known for its anti-aging properties in mice. These findings are part of a growing list of studies that help to validate the theory that finding the cure for Progeria may also benefit the entire aging population.\r\n\r\n* K.\u00a0 Cao, J.\u00a0 J.\u00a0 Graziotto, C.\u00a0 D.\u00a0 Blair, J.\u00a0 R.\u00a0 Mazzulli, M.\u00a0 R.\u00a0 Erdos, D.\u00a0 Krainc, F.\u00a0 S.\u00a0 Collins, \u201cRapamycin Reverses\r\n\r\nCellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Syndrome Cells.\u201d Sci.\u00a0 Transl.\u00a0 Med.\u00a0 <\/em>3<\/strong>, 89ra58 (2011).\r\n\r\nPhase 1, to determine the dosage of everolimus, began in April 2016 and was successfully completed in June 2017, within the timeline and under the projected budget. The 17 children that participated in Phase 1 have been moved into Phase 2, which will test the effectiveness of the 2-drug combination. Enrollment of additional children began in July 2017. 2017 was a busy year, with 39 children from 18 countries traveling to Boston. Enrollment of additional children will continue through July 2018, and those already enrolled will be returning for their second visit. Together, this Phase 1-2 treatment trial may enroll up to 80 children, and take an estimated 3.5-4 years to complete, at a cost of $2.5 million dollars.\r\n\r\n![](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/Cao-rapamycin-image.jpg\")
The Progeria Research Foundation provided cells for this project from the PRF Cell & Tissue Bank<\/a> and helped fund the research through our grants program<\/a> \u2013 more proof that PRF\u2019s research-related programs are essential to advancements toward the cure.\r\n\r\nThis new trial is a collaborative effort that will build upon the knowledge gained from the previous Progeria trials. The children will be seen by virtually the same team of physicians from Boston Children\u2019s Hospital, Dana-Farber Cancer Institute and Brigham and Women\u2019s Hospital, all of whom now have world-renowned expertise in Progeria as well as the drugs involved.\r\n\r\nAs we begin this new chapter, PRF is and always has been responsible for\u00a0all trial expenses, including testing, travel, food, lodging, translators and staff. Each new trial is more expensive than the last, as more children enroll for a chance at longer, healthier lives. \u00a0 Please help us fund these vital trials \u2013 DONATE TODAY!<\/a>\r\n\r\n[caption id=\"attachment_3791\" align=\"aligncenter\" width=\"250\"]![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/02\/Ana-Clara-trial-page.jpg\")
8-year-old Ana Clara shares a special moment with her mother while taking a break from testing at Boston Children\u2019s Hospital. She recently enrolled in phase 2 of the Progeria clinical trial. Travel from her remote town in Brazil included a 10-hour bus ride to the airport.<\/span><\/strong><\/span>[\/caption]\r\n\r\n[\/vc_column_text][\/vc_column][\/vc_row][vc_row][vc_column][\/vc_column][\/vc_row][vc_row][vc_column][vc_column_text]\r\n\r\n[\/vc_column_text][\/vc_column][\/vc_row]\t\t","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-772","page","type-page","status-publish","hentry"],"yoast_head":"\n