[et_pb_section fb_built=”1″ fullwidth=”on” disabled_on=”off|off|off” _builder_version=”4.16″ border_width_bottom=”55px” border_color_bottom=”#29327a” locked=”off” global_colors_info=”{}”][et_pb_fullwidth_header _builder_version=”4.16″ title_font_size=”55px” background_color=”#29327a” background_image=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/09\/Kids-on-Bridge.png” custom_padding=”11.5vw||11.5vw||true” custom_padding_tablet=”” custom_padding_phone=”|56px||” custom_padding_last_edited=”on|desktop” title_font_size_tablet=”45px” title_font_size_phone=”40px” title_font_size_last_edited=”on|phone” z_index_tablet=”500″ custom_css_main_element=”background-position: center 18% !important;” title_text_shadow_horizontal_length_tablet=”0px” title_text_shadow_vertical_length_tablet=”0px” title_text_shadow_blur_strength_tablet=”1px” content_text_shadow_horizontal_length_tablet=”0px” content_text_shadow_vertical_length_tablet=”0px” content_text_shadow_blur_strength_tablet=”1px” subhead_text_shadow_horizontal_length_tablet=”0px” subhead_text_shadow_vertical_length_tablet=”0px” subhead_text_shadow_blur_strength_tablet=”1px” content_link_text_shadow_horizontal_length_tablet=”0px” content_link_text_shadow_vertical_length_tablet=”0px” content_link_text_shadow_blur_strength_tablet=”1px” content_ul_text_shadow_horizontal_length_tablet=”0px” content_ul_text_shadow_vertical_length_tablet=”0px” content_ul_text_shadow_blur_strength_tablet=”1px” content_ol_text_shadow_horizontal_length_tablet=”0px” content_ol_text_shadow_vertical_length_tablet=”0px” content_ol_text_shadow_blur_strength_tablet=”1px” content_quote_text_shadow_horizontal_length_tablet=”0px” content_quote_text_shadow_vertical_length_tablet=”0px” content_quote_text_shadow_blur_strength_tablet=”1px” button_one_text_shadow_horizontal_length_tablet=”0px” button_one_text_shadow_vertical_length_tablet=”0px” button_one_text_shadow_blur_strength_tablet=”1px” button_two_text_shadow_horizontal_length_tablet=”0px” button_two_text_shadow_vertical_length_tablet=”0px” button_two_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” box_shadow_horizontal_image_tablet=”0px” box_shadow_vertical_image_tablet=”0px” box_shadow_blur_image_tablet=”40px” box_shadow_spread_image_tablet=”0px” box_shadow_horizontal_button_one_tablet=”0px” box_shadow_vertical_button_one_tablet=”0px” box_shadow_blur_button_one_tablet=”40px” box_shadow_spread_button_one_tablet=”0px” box_shadow_horizontal_button_two_tablet=”0px” box_shadow_vertical_button_two_tablet=”0px” box_shadow_blur_button_two_tablet=”40px” box_shadow_spread_button_two_tablet=”0px” text_shadow_horizontal_length_tablet=”0px” text_shadow_vertical_length_tablet=”0px” text_shadow_blur_strength_tablet=”1px” global_colors_info=”{}”]<\/p>\n
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[\/et_pb_fullwidth_header][\/et_pb_section][et_pb_section fb_built=”1″ use_custom_gutter=”on” gutter_width=”1″ specialty=”on” padding_left_1=”35px” padding_left_2=”35px” padding_2_tablet=”|||0px” padding_2_phone=”|||0px” padding_2_last_edited=”on|desktop” module_class_1=”sidebar-secondary-nav” module_class=”handprint-bg” _builder_version=”4.16″ background_image=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/blue-handprint-only.png” parallax=”on” parallax_method=”off” inner_width=”100%” inner_max_width=”100%” custom_padding=”0|0px|54px|0px|false|false” z_index_tablet=”500″ border_width_top=”10px” border_color_top=”#8fd2ed” use_custom_width=”on” width_unit=”off” custom_width_percent=”100%” global_colors_info=”{}”][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_sidebar area=”et_pb_widget_area_1″ disabled_on=”on|on|off” module_class=”subpage-sidebars” _builder_version=”4.16″ animation_style=”fade” z_index_tablet=”500″ border_width_right=”5px” locked=”off” global_colors_info=”{}”]
\n[\/et_pb_sidebar][\/et_pb_column][et_pb_column type=”3_4″ specialty_columns=”3″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|phone” disabled_on=”on|on|” _builder_version=”4.16″ custom_padding=”|35px||0px” custom_padding_tablet=”|35px||35px||true” custom_padding_phone=”” animation_direction=”top” z_index_tablet=”500″ global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Progeria Clinical Drug Trials: Background” _builder_version=”4.27.2″ header_4_line_height=”1.2em” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ text_text_shadow_horizontal_length_tablet=”0px” text_text_shadow_vertical_length_tablet=”0px” text_text_shadow_blur_strength_tablet=”1px” link_text_shadow_horizontal_length_tablet=”0px” link_text_shadow_vertical_length_tablet=”0px” link_text_shadow_blur_strength_tablet=”1px” ul_text_shadow_horizontal_length_tablet=”0px” ul_text_shadow_vertical_length_tablet=”0px” ul_text_shadow_blur_strength_tablet=”1px” ol_text_shadow_horizontal_length_tablet=”0px” ol_text_shadow_vertical_length_tablet=”0px” ol_text_shadow_blur_strength_tablet=”1px” quote_text_shadow_horizontal_length_tablet=”0px” quote_text_shadow_vertical_length_tablet=”0px” quote_text_shadow_blur_strength_tablet=”1px” header_text_shadow_horizontal_length_tablet=”0px” header_text_shadow_vertical_length_tablet=”0px” header_text_shadow_blur_strength_tablet=”1px” header_2_text_shadow_horizontal_length_tablet=”0px” header_2_text_shadow_vertical_length_tablet=”0px” header_2_text_shadow_blur_strength_tablet=”1px” header_3_text_shadow_horizontal_length_tablet=”0px” header_3_text_shadow_vertical_length_tablet=”0px” header_3_text_shadow_blur_strength_tablet=”1px” header_4_text_shadow_horizontal_length_tablet=”0px” header_4_text_shadow_vertical_length_tablet=”0px” header_4_text_shadow_blur_strength_tablet=”1px” header_5_text_shadow_horizontal_length_tablet=”0px” header_5_text_shadow_vertical_length_tablet=”0px” header_5_text_shadow_blur_strength_tablet=”1px” header_6_text_shadow_horizontal_length_tablet=”0px” header_6_text_shadow_vertical_length_tablet=”0px” header_6_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”]<\/p>\n
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Progeria clinical drug trials are the best hope for children with Progeria, testing potential treatments that may enable them to live longer, healthier lives. These trials are the culmination of years of research focused on what drug or combination of drugs will treat and cure the children.<\/p>\n
Since 1999 when we founded PRF and there were no resources for these children, we have soared from total obscurity, to gene finding, to the first Progeria clinical trials, to the first-ever FDA-approved treatment, called lonafarnib \u2013 all at a pace virtually unheard of in the scientific community. And while helping this handful of children, Progeria\u2019s connection to common heart disease and aging has tremendous implications for us all.<\/p>\n
[\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”What\u2019s Next for Treatments and Cure? ” _builder_version=”4.27.2″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” custom_margin=”|||15px|false|false” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n
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<\/strong><\/em><\/strong><\/h4>\n<\/em><\/strong><\/p>\n PRF, in collaboration with Korean-based study sponsor PRG Science & Technology (PRG S&T), has started enrollment for a brand-new clinical trial with a drug called Progerinin. Laboratory evidence shows that this drug, when taken in combination with lonafarnib, may be more effective than lonafarnib alone. PRF funded the laboratory work that led to the formation of PRG S&T and its development of Progerinin. Pre-trial work has begun at Boston Children\u2019s Hospital, in anticipation of bringing children from all over the world to enroll for this trial in the coming months. We\u2019re super excited about starting a new trial with such a promising drug, and look forward to sharing more details with you as they become available.<\/p>\n Please contact Shelby Phillips\u00a0to learn more about the trial. PRF co-founder and Executive Director Audrey Gordon seals the deal in June with Dr. Bum-Joon Park of PRG S&T to move forward with pre-trial work.<\/em><\/p>\n <\/em><\/p>\n [\/et_pb_text][et_pb_text admin_label=”RNA Therapy: Drug Administration Feasibility Study is Complete!” _builder_version=”4.27.2″ header_4_line_height=”1.2em” vertical_offset_tablet=”0″ horizontal_offset_tablet=”0″ custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ text_text_shadow_horizontal_length_tablet=”0px” text_text_shadow_vertical_length_tablet=”0px” text_text_shadow_blur_strength_tablet=”1px” link_text_shadow_horizontal_length_tablet=”0px” link_text_shadow_vertical_length_tablet=”0px” link_text_shadow_blur_strength_tablet=”1px” ul_text_shadow_horizontal_length_tablet=”0px” ul_text_shadow_vertical_length_tablet=”0px” ul_text_shadow_blur_strength_tablet=”1px” ol_text_shadow_horizontal_length_tablet=”0px” ol_text_shadow_vertical_length_tablet=”0px” ol_text_shadow_blur_strength_tablet=”1px” quote_text_shadow_horizontal_length_tablet=”0px” quote_text_shadow_vertical_length_tablet=”0px” quote_text_shadow_blur_strength_tablet=”1px” header_text_shadow_horizontal_length_tablet=”0px” header_text_shadow_vertical_length_tablet=”0px” header_text_shadow_blur_strength_tablet=”1px” header_2_text_shadow_horizontal_length_tablet=”0px” header_2_text_shadow_vertical_length_tablet=”0px” header_2_text_shadow_blur_strength_tablet=”1px” header_3_text_shadow_horizontal_length_tablet=”0px” header_3_text_shadow_vertical_length_tablet=”0px” header_3_text_shadow_blur_strength_tablet=”1px” header_4_text_shadow_horizontal_length_tablet=”0px” header_4_text_shadow_vertical_length_tablet=”0px” header_4_text_shadow_blur_strength_tablet=”1px” header_5_text_shadow_horizontal_length_tablet=”0px” header_5_text_shadow_vertical_length_tablet=”0px” header_5_text_shadow_blur_strength_tablet=”1px” header_6_text_shadow_horizontal_length_tablet=”0px” header_6_text_shadow_vertical_length_tablet=”0px” header_6_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”]<\/p>\n PRF has taken the first patient-involved steps toward a clinical trial in RNA therapy \u2013 SO exciting!<\/p>\n Background: In January 2021, we reported breakthrough findings in RNA therapeutics<\/strong>, wherein this therapy inhibited production of RNA coding for the Progeria disease-causing protein, progerin<\/strong>. The study*, led by Dr. Francis Collins, White House Science Advisor and former Director of the National Institutes of Health (NIH), revealed that Progeria mice treated with a drug named SRP-2001 reduced the harmful progerin mRNA and protein expression in blood vessels<\/strong>, as well as in other tissues. The blood vessels were stronger, and the mice showed an increased survival of over 60%<\/strong> compared to untreated mice. Thus the work with this promising therapy continued, and we have taken the\u00a0next step <\/strong>with a Feasibility Study <\/strong>as follows:<\/p>\n Typically, RNA therapeutics are liquids that are injected intravenously (directly into the vein). However, those with Progeria would not be able to tolerate intravenous delivery of the required daily dosage. Thus, PRF developed a subcutaneous delivery system<\/strong> whereby the liquid can be injected with a small needle under the skin. A 6-month study at BCH was completed to determine the feasibility of this delivery approach for those with Progeria<\/strong>. The team tested whether administration of a saline solution can comfortably be injected subcutaneously and which injection system and site would be most comfortable. Once data from this study are analyzed, we will be one step closer to a clinical trial in genetic therapy<\/strong>!<\/p>\n *<\/strong>Erdos, M.R., Cabral, W.A., Tavarez, U.L. et al. A targeted antisense therapeutic approach for Hutchinson\u2013Gilford progeria syndrome. Nat Med<\/em> (2021).<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”What\u2019s Next for Treatments and Cure? ” _builder_version=”4.27.0″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” custom_margin=”|||15px|false|false” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”What\u2019s Happening TODAY With the Progeria Clinical Trials? ” _builder_version=”4.27.0″ header_4_line_height=”1.2em” vertical_offset_tablet=”0″ horizontal_offset_tablet=”0″ custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ text_text_shadow_horizontal_length_tablet=”0px” text_text_shadow_vertical_length_tablet=”0px” text_text_shadow_blur_strength_tablet=”1px” link_text_shadow_horizontal_length_tablet=”0px” link_text_shadow_vertical_length_tablet=”0px” link_text_shadow_blur_strength_tablet=”1px” ul_text_shadow_horizontal_length_tablet=”0px” ul_text_shadow_vertical_length_tablet=”0px” ul_text_shadow_blur_strength_tablet=”1px” ol_text_shadow_horizontal_length_tablet=”0px” ol_text_shadow_vertical_length_tablet=”0px” ol_text_shadow_blur_strength_tablet=”1px” quote_text_shadow_horizontal_length_tablet=”0px” quote_text_shadow_vertical_length_tablet=”0px” quote_text_shadow_blur_strength_tablet=”1px” header_text_shadow_horizontal_length_tablet=”0px” header_text_shadow_vertical_length_tablet=”0px” header_text_shadow_blur_strength_tablet=”1px” header_2_text_shadow_horizontal_length_tablet=”0px” header_2_text_shadow_vertical_length_tablet=”0px” header_2_text_shadow_blur_strength_tablet=”1px” header_3_text_shadow_horizontal_length_tablet=”0px” header_3_text_shadow_vertical_length_tablet=”0px” header_3_text_shadow_blur_strength_tablet=”1px” header_4_text_shadow_horizontal_length_tablet=”0px” header_4_text_shadow_vertical_length_tablet=”0px” header_4_text_shadow_blur_strength_tablet=”1px” header_5_text_shadow_horizontal_length_tablet=”0px” header_5_text_shadow_vertical_length_tablet=”0px” header_5_text_shadow_blur_strength_tablet=”1px” header_6_text_shadow_horizontal_length_tablet=”0px” header_6_text_shadow_vertical_length_tablet=”0px” header_6_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”]<\/p>\n <\/p>\n The most recent trial involved 2 drugs: lonafarnib<\/strong>\u00a0and a new drug, everolimus<\/strong>. Phase 1, to determine the safe and appropriate dosage of everolimus, began in April 2016 and was successfully completed in June 2017. Phase 2, which tested the effectiveness of the 2-drug combination, began in July 2017 and was completed in April 2022. Sixty children from 27 countries were enrolled in this two-drug phase!<\/p>\n Everolimus is a form of the drug rapamycin; everolimus could be more easily given to children with Progeria because it requires fewer blood draws to measure drug levels. While lonafarnib may block the toxic progerin from developing, rapamycin appears to allow cells to more rapidly clear out progerin. Thus with rapamycin targeting a different pathway than lonafarnib, the combination may prove to be a \u201cone-two punch\u201d to Progeria \u2013 hopefully a better treatment than lonafarnib on its own.<\/p>\n We have now entered a period of data analysis and expect to eventually publish results in a peer-reviewed, scientific journal. In the meantime, trial participants have either rolled over into the monotherapy extension of the trial, or into another access program. Through either pathway, participants continue to be supplied with lonafarnib, the current standard of care.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Trials History at a Glance” _builder_version=”4.27.0″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” custom_margin=”|||15px|false|false” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|phone” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.22.1″ custom_margin=”0px||||false|false” custom_padding=”|35px|0px|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”To date, PRF has funded and co-coordinated three clinical trials” _builder_version=”4.27.0″ text_font=”||||||||” header_4_font=”||||||||” header_4_line_height=”1.2em” custom_padding=”||0px|||” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” global_colors_info=”{}”]<\/p>\n To date, PRF has funded and co-coordinated four clinical trials<\/strong>. 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.<\/p>\n [\/et_pb_text][et_pb_button button_url=”https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR” url_new_window=”on” button_text=”Please help us fund these vital trials. Donate Today” admin_label=”Donate” _builder_version=”4.22.1″ background_layout=”dark” custom_margin=”20px||25px” animation_style=”slide” animation_direction=”left” animation_intensity_slide=”25%” global_colors_info=”{}”] #1 involved a single drug, lonafarnib, began in 2007, and proved successful. Read all about the historic treatment discovery here.<\/a><\/p>\n #2, the Feasibility portion of the \u201cTriple Trial\u201d involved 3 drugs: lonafarnib, pravastatin, and zoledronate. This 1-month, phase 1 \u201cmini trial\u201d took place in March 2009 to determine if adding 2 more drugs to the lonafarnib regimen was safe to move forward with a larger population (which it was).<\/p>\n #3, the \u201cTriple Trial,\u201d was a continuation of the Feasibility trial, and was a phase 2 study, which began in August 2009. Its protocol changed over the course of five years, switching back to just lonafarnib and re-opening enrollment so more children could participate. Read more here<\/a>.<\/p>\n #4 is the two-drug, lonafarnib and everolimus trial. Phase 1, to determine the safe and appropriate dosage of everolimus, began in April 2016 and was successfully completed in June 2017. Phase 2, which tested the effectiveness of the 2-drug combination, began in July 2017 and was completed in April 2022. The monotherapy extension of this trial continues today.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|desktop” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/02\/Ana-Clara-trial-page.jpg” align=”center” align_tablet=”center” align_phone=”” align_last_edited=”on|desktop” admin_label=”liv” _builder_version=”4.22.1″ border_width_all=”4px” border_color_all=”#392887″ global_colors_info=”{}”] In 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 \u2013 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.<\/p>\n <\/p>\n [\/et_pb_text][et_pb_testimonial author=”Francis Collins, MD, PhD” job_title=”Director of the National Human Genome Research Institutethat mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.” quote_icon_color=”#00b2e2″ portrait_width=”140px” portrait_height=”140px” _builder_version=”4.16″ background_color=”#f7f7f7″ custom_margin_tablet=”” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” z_index_tablet=”500″ custom_css_testimonial_author=”color: #00b2e2;||font-size: 19px !important;||font-weight: 700;” custom_css_testimonial_meta=”font-style: italic;||font-weight: 700;” box_shadow_style_image=”preset3″ locked=”off” global_colors_info=”{}”]<\/p>\n \u201cI know of no other rare genetic disease that has gone from gene discovery to clinical trial in under four years \u2013 a phenomenal testament to the hard work of The Progeria Research Foundation.\u201d<\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”1_2,1_2″ custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”false” padding_left_right_link_2=”false” _builder_version=”4.16″ custom_margin=”||25px” custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|20px||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/11\/redoof2007photocollage.png” title_text=”redoof2007photocollage” align_tablet=”center” align_phone=”” align_last_edited=”on|desktop” admin_label=”collage” _builder_version=”4.27.0″ custom_margin=”|-50px|||false|false” global_colors_info=”{}”] <\/p>\n Twenty-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.<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Who, Where, When, How and How Much\u2026” _builder_version=”4.27.0″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”The first three clinical trials were led by Mark Kieran MD” _builder_version=”4.27.0″ header_4_line_height=”1.2em” custom_margin=”||25px” custom_margin_tablet=”35px||” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n The first three clinical trials were led by Mark Kieran MD, PhD,\u00a0<\/strong>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.\u00a0 In 2017, he left his position at Dana Farber to work in the private sector. 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\u00a0Leslie Gordon, MD, PhD, Medical Director of PRF, Lecturer at BCH and Harvard Medical School, Professor of Pediatrics at Hasbro Children\u2019s Hospital and Brown University in Providence, RI.\u00a0 Dr. Kleinman has assumed the lead role of Principal Investigator.<\/p>\n The clinical trials are a collaborative effort, involving physicians at Boston Children\u2019s Hospital, Dana-Farber Cancer Institute, and Brigham and Women\u2019s Hospital,<\/strong>\u00a0all 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.<\/p>\n [\/et_pb_text][et_pb_text admin_label=”How did we get to this point?” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n In 2003, The Progeria Research Foundation\u2019s collaborative research team\u00a0\u00a0discovered the Progeria gene<\/a>.<\/a> This 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. Since the gene discovery, the support of researchers, clinicians, families of children with Progeria and people like YOU brought us to another crossroads in the search for a treatment. Researchers began an intense study of this enemy protein called\u00a0progerin<\/em>, and in 2006 they identified a potential drug treatment for children with Progeria, called farnesyltransferase inhibitors (FTIs), and conducted studies in the lab that supported a human trial with the drug. The FTI chosen was initially supplied by Merck and called lonafarnib<\/em>.\u00a0Click here<\/a>\u00a0for more details on the research.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Why did researchers think this drug would work in Progeria?” _builder_version=”4.16″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Normal cell, Progeria cell, Progeria cell after being treated with FTI.<\/p><\/div>\n The 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.\u00a0\u00a0Click here<\/a>\u00a0for more information on FTIs.<\/p>\n [\/et_pb_text][et_pb_text admin_label=”How did PRF fund the trial?” _builder_version=”4.16″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Thanks to the support of thousands, we were able to raise all the funds necessary to cover the trial costs.\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.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|35px|35.2344px|35px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”The FTI lonafarnib is now a proven treatment for Progeria.” _builder_version=”4.22.1″ text_font=”||||||||” header_font=”||||||||” header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”2012 2014″ _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”39px||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n In 2012, the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system. In May, 2014, a study showed one or more of 3 drugs \u2013 including lonafarnib \u2013 being tested in PRF-funded clinical trials extended lifespan; it was unclear which drug had this positive life-changing impact.\u00a0 However, in April 2018, a study published in The Journal of the American Medical Association (JAMA)<\/em> reported that lonafarnib alone extended survival in children with Progeria by at least 1.6 years. Click here<\/a>\u00a0<\/a>for details on the 2012 historic treatment discovery study,\u00a0here\u00a0<\/a>for details on the 2014 findings, and\u00a0here<\/a>\u00a0for details on the 2018 study.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_testimonial portrait_url=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cam-and-DadMarch09.jpg” quote_icon_color=”#00b2e2″ portrait_width=”140px” portrait_height=”140px” _builder_version=”4.16″ background_color=”#f7f7f7″ custom_margin_tablet=”” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” z_index_tablet=”500″ custom_css_testimonial_author=”color: #00b2e2;||font-size: 19px !important;||font-weight: 700;” custom_css_testimonial_meta=”font-style: italic;||font-weight: 700;” box_shadow_style_image=”preset3″ locked=”off” global_colors_info=”{}”]<\/p>\n \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.\u201d<\/em><\/p>\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\u2026the doctors, the researchers and the staff. You are our heroes!\u201d<\/em><\/p>\n \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>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_margin=”39px||” custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/trophies-3-kids.jpg” align=”center” align_tablet=”center” align_phone=”” align_last_edited=”on|desktop” admin_label=”aloha kids image” _builder_version=”4.22.1″ animation_style=”fade” animation_direction=”bottom” global_colors_info=”{}”] <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|30px|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Summary, etc.” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”39px||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Researchers 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. This much larger trial included 45 children from 24 different countries!<\/p>\n 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.<\/p>\n 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 would work as partners, to complement each other so that the progerin protein is affected more by combining the three drugs.<\/p>\n 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\u00a0<\/strong><\/p>\n 45 children enrolled in this trial, from 24 countries, speaking 17 languages. This includes children that participated in the FTI-only trial, the 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.<\/p>\n [\/et_pb_text][et_pb_text admin_label=”Trial Medications at a Glance” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”39px||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Pravastatin<\/strong>\u00a0(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.<\/p>\n Zoledronic acid\u00a0<\/strong>is a\u00a0bisphosphonate<\/strong>, usually used as a bone drug for improving osteoporosis, and to prevent skeletal fractures in people suffering from some forms of cancer.<\/p>\n Lonafarnib <\/strong>is an\u00a0FTI\u00a0<\/strong>(Farnesyltransferase inhibitor), a drug that can reverse an abnormality in Progeria cells in the laboratory, and has improved disease in Progeria mice. In July a\u00a0study\u00a0<\/a>** was published that showed\u00a0no significant improvements were found over and above the lonafarnib single therapy. **Gordon, et. al., Clinical Trial of Protein Farnesylation Inhibitors lonafarnib, Pravastatin and Zoledronic Acid in Children with Hutchinson-Gilford Progeria Syndrome, Circulation<\/em>,\u00a0\u00a0\u00a0 10.1161\/CIRCULATIONAHA.116.022188<\/strong><\/p>\n However, the \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 lonafarnib alone because we know that it is helping the children. Usually, clinical trials run their course and the patients are taken off all the drugs until FDA approval; this could take years.\u00a0 PRF has ensured that the children continue to take the one known treatment, while they and their research partners continue exploring additional treatment options (such as everolimus that is currently being tested).<\/p>\n [\/et_pb_text][et_pb_text admin_label=”Everolimus” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Everolimus is a form of the drug rapamycin; everolimus could be more easily given to children with Progeria because it requires fewer blood draws to measure drug levels. While lonafarnib may block the toxic progerin from developing, rapamycin appears to allow cells to more rapidly clear out progerin. Thus with rapamycin targeting a different pathway than lonafarnib, the combination may prove to be a \u201cone-two punch\u201d to Progeria \u2013 hopefully a better treatment than lonafarnib on its own.<\/p>\n Rapamycin<\/em> is an FDA-approved drug that has previously been shown to extend the lives of non-Progeria mouse models. 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.<\/p>\n Rapamycin 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.<\/p>\n \u00a0* 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 Cellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Syndrome Cells.\u201d Sci.\u00a0 Transl.\u00a0 Med.\u00a0 3, 89ra58 (2011).<\/p>\n The Progeria Research Foundation provided cells for this project from the\u00a0PRF Cell & Tissue Bank<\/a>\u00a0and helped fund the research through our\u00a0grants program<\/a>\u00a0\u2013 more proof that PRF\u2019s research-related programs are essential to advancements toward the cure.<\/p>\n This 2-drug trial has been a collaborative effort that built upon the knowledge gained from PRF\u2019s previous clinical trials. The children were seen by virtually the same team of physicians from Boston Children\u2019s Hospital and Brigham and Women\u2019s Hospital, all of whom now have world-renowned expertise in Progeria as well as in the drugs involved.<\/p>\n Sixty children from 27 countries were enrolled in this two-drug phase. Data from the 2-drug portion of the trial is being analyzed and results are being formulated and written up for publication in a peer reviewed scientific journal.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|30px|0px|false|false” custom_padding_tablet=”0px|35px|2px|35px|false|false” custom_padding_phone=”0px||0px” animation_direction=”right” border_width_top=”10px” border_color_top=”#8fd2ed” border_width_bottom=”10px” border_color_bottom=”#00b2e2″ global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Quest” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”25px||25px” custom_padding=”||” animation_direction=”right” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Our work with genetic therapies is advancing full-speed ahead! RNA therapy and DNA Gene Editing studies <\/strong>have shown a vast improvement in Progeria mice lifespan. <\/strong>PRF continues to invest substantial funds into their development, with the hope that these research efforts will lead to clinical trials, and, ultimately, the cure.<\/strong><\/p>\n These cutting-edge therapies have huge potential<\/em>! With your help, PRF can continue to move forward as quickly as possible toward the most effective treatments and the cure.<\/p>\n [\/et_pb_text][et_pb_button button_url=”https:\/\/www.progeriaresearch.org\/lonafarnib-map-program\/” button_text=”Patients, caregivers and doctors: Click here for details.” admin_label=”Lonafarnib pdf’s button” _builder_version=”4.22.1″ background_layout=”dark” custom_margin=”10px||10px||false|false” z_index_tablet=”500″ button_text_shadow_horizontal_length_tablet=”0px” button_text_shadow_vertical_length_tablet=”0px” button_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”] [\/et_pb_cta][\/et_pb_column][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_cta button_url=”https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR” button_text=”Donate Now” admin_label=”Together, we will find the cure!” module_class=”sign-btn” _builder_version=”4.16″ header_font_size=”25px” background_color=”#29327a” animation_style=”slide” animation_direction=”left” animation_intensity_slide=”25%” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” body_font_size_tablet=”” body_font_size_phone=”” body_font_size_last_edited=”on|desktop” z_index_tablet=”500″ border_radii=”on|25px|25px|25px|25px” global_colors_info=”{}” button_bg_color__hover_enabled=”on” button_bg_color__hover=”#8fd2ed” button_border_color__hover_enabled=”on”]<\/p>\n [\/et_pb_cta][\/et_pb_column][et_pb_column type=”1_2″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2024\/08\/2024-strip-footer-strip-copy.png” title_text=”2024 strip footer strip copy” _builder_version=”4.27.0″ _module_preset=”default” custom_margin=”35px||||false|false” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>\n","protected":false},"excerpt":{"rendered":" [et_pb_section fb_built=”1″ fullwidth=”on” disabled_on=”off|off|off” _builder_version=”4.16″ border_width_bottom=”55px” border_color_bottom=”#29327a” locked=”off” global_colors_info=”{}”][et_pb_fullwidth_header _builder_version=”4.16″ title_font_size=”55px” background_color=”#29327a” background_image=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/09\/Kids-on-Bridge.png” custom_padding=”11.5vw||11.5vw||true” custom_padding_tablet=”” custom_padding_phone=”|56px||” custom_padding_last_edited=”on|desktop” title_font_size_tablet=”45px” title_font_size_phone=”40px” title_font_size_last_edited=”on|phone” z_index_tablet=”500″ custom_css_main_element=”background-position: center 18% !important;” title_text_shadow_horizontal_length_tablet=”0px” title_text_shadow_vertical_length_tablet=”0px” title_text_shadow_blur_strength_tablet=”1px” content_text_shadow_horizontal_length_tablet=”0px” content_text_shadow_vertical_length_tablet=”0px” content_text_shadow_blur_strength_tablet=”1px” subhead_text_shadow_horizontal_length_tablet=”0px” subhead_text_shadow_vertical_length_tablet=”0px” subhead_text_shadow_blur_strength_tablet=”1px” content_link_text_shadow_horizontal_length_tablet=”0px” content_link_text_shadow_vertical_length_tablet=”0px” content_link_text_shadow_blur_strength_tablet=”1px” content_ul_text_shadow_horizontal_length_tablet=”0px” content_ul_text_shadow_vertical_length_tablet=”0px” content_ul_text_shadow_blur_strength_tablet=”1px” content_ol_text_shadow_horizontal_length_tablet=”0px” content_ol_text_shadow_vertical_length_tablet=”0px” content_ol_text_shadow_blur_strength_tablet=”1px” content_quote_text_shadow_horizontal_length_tablet=”0px” content_quote_text_shadow_vertical_length_tablet=”0px” content_quote_text_shadow_blur_strength_tablet=”1px” button_one_text_shadow_horizontal_length_tablet=”0px” button_one_text_shadow_vertical_length_tablet=”0px” button_one_text_shadow_blur_strength_tablet=”1px” button_two_text_shadow_horizontal_length_tablet=”0px” button_two_text_shadow_vertical_length_tablet=”0px” button_two_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” […]<\/p>\n","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\"]Kickstarting a New Clinical Drug Trial: Enrollment for Progerinin Trial Have Begun!<\/em><\/strong><\/h4>\n
Email: sphillips@progeriaresearch.org<\/a>
WhatsApp, Telegram, WeChat: 1-978-876-2407
Office Phone: 978-548-5308<\/p>\nRNA Therapy: Drug Administration<\/strong> Feasibility Study is Complete!<\/strong><\/h4>\n
What\u2019s Happening TODAY With the Progeria Clinical Trials?<\/strong><\/h2>\n
Trial History at a Glance<\/b><\/h2>\n
\n[\/et_pb_button][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”1_2,1_2″ custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Details on the Prior Trials” _builder_version=”4.27.0″ global_colors_info=”{}”]<\/p>\nDetails on the Prior Trials<\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_margin=”||25px” custom_padding=”|35px|0|0px|false|false” custom_padding_tablet=”35px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”May 7, 2007: The Start of the First-Ever Progeria Clinical Drug Trial Marks Historic Moment in Progeria Research History!” _builder_version=”4.27.2″ z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\nMay 7, 2007:\u00a0The Start of the First-Ever Progeria Clinical Drug Trial Marks Historic Moment in Progeria Research History!<\/strong><\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|desktop” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Twenty-eight (28) children from sixteen countries participated” _builder_version=”4.27.0″ custom_margin=”|||-30px|false|false” custom_margin_tablet=”35px||” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” global_colors_info=”{}”]<\/p>\nWho, Where, When, How and How Much\u2026<\/strong><\/h2>\n
How did we get to this point?<\/b><\/h4>\n
Why did researchers think this drug would work in Progeria?<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/FTI-3-cell-image-crpd-300x71.jpg\")
How did PRF fund the trial?
<\/strong><\/h4>\nThe FTI lonafarnib is now a proven treatment for Progeria<\/strong>.<\/h2>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ module_id=”threeCT” _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”The Progeria Triple Drug Trial Begins August 2009″ _builder_version=”4.22.1″ text_font=”||||||||” header_font=”||||||||” header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” global_colors_info=”{}”]<\/p>\nAlways Moving Forward: The Progeria Triple Drug Trial Begins August 2009<\/strong><\/h2>\n
Summary:<\/strong><\/h4>\n
Strategy:<\/strong><\/h4>\n
Goal:<\/strong><\/h4>\n
The Feasibility Trial:<\/strong><\/h4>\n
The Efficacy Trial:<\/strong><\/h4>\n
Trial Medications at a Glance<\/strong><\/h4>\n
All 3 drugs block the production of the farnesyl molecule that is needed for progerin to create disease in Progeria.
<\/em>
*<\/em>\u00a0\u201c<\/strong>Combined treatment with statins and aminobisphosphonates extends longevity in a mouse model of human premature aging\u201d<\/strong>, 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.<\/p>\nThe Addition of a Newer<\/strong> Drug: Everolimus<\/strong><\/h4>\n
The Science Behind the Addition of this Second Drug<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cao-with-caption.jpg\")
<\/em><\/p>\nOur quest for the cure continues\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=”1″ module_class=”footer” _builder_version=”4.21.0″ background_color=”#29327a” custom_margin=”-2px|||||” custom_padding=”0|0px|0|0px|false|false” z_index_tablet=”500″ border_width_top=”12px” border_color_top=”#00b2e2″ global_module=”133″ locked=”off” global_colors_info=”{}”][et_pb_row_inner column_structure=”1_3,1_3,1_3″ disabled_on=”on|on|on” admin_label=”Row” _builder_version=”4.21.0″ _module_preset=”default” min_height=”277px” custom_margin=”|20px|30px||false|false” custom_padding=”0px|30px|0px||false|false” disabled=”on” global_colors_info=”{}”][et_pb_column_inner type=”1_3″ saved_specialty_column_type=”3_4″ _builder_version=”4.21.0″ _module_preset=”default” global_colors_info=”{}”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/08\/Startbanner-1.png” title_text=”Startbanner” url=”https:\/\/www.gravoc.com\/” align=”right” _builder_version=”4.21.0″ _module_preset=”default” custom_margin=”|-50px||20px|false|false” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=”1_3″ saved_specialty_column_type=”3_4″ _builder_version=”4.21.0″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column_inner][et_pb_column_inner type=”1_3″ saved_specialty_column_type=”3_4″ _builder_version=”4.21.0″ _module_preset=”default” global_colors_info=”{}”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/07\/RFRFinish.png” title_text=”RFRFinish” _builder_version=”4.21.0″ _module_preset=”default” custom_margin=”|20px||-50px|false|false” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row column_structure=”1_4,1_4,1_2″ make_equal=”on” module_class=” et_pb_row_fullwidth” _builder_version=”4.16″ width=”89%” width_tablet=”80%” width_phone=”” width_last_edited=”on|desktop” max_width=”89%” max_width_tablet=”80%” max_width_phone=”” max_width_last_edited=”on|desktop” z_index_tablet=”500″ make_fullwidth=”on” width_unit=”off” custom_width_percent=”100%” global_colors_info=”{}”][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_cta button_url=”https:\/\/www.progeriaresearch.org\/newsletter-signup\/” button_text=”Sign Up Now” admin_label=”Sign Up for Newsletter” module_class=”sign-btn” _builder_version=”4.16″ header_font_size=”25px” background_color=”#29327a” animation_style=”slide” animation_direction=”left” animation_intensity_slide=”25%” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ border_radii=”on|25px|25px|25px|25px” global_colors_info=”{}” button_bg_color__hover_enabled=”on” button_bg_color__hover=”#8fd2ed” button_border_color__hover_enabled=”on”]<\/p>\nSign Up<\/h2>\n
for Our<\/h2>\n
Newsletter!<\/h2>\n
Together, we<\/h2>\n
WILL<\/em><\/h2>\n
find the cure!<\/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