On March 1st, PRF Ambassador Meghan Waldron turned 19, and we celebrated with March Madness 2020: Where in the world is Meghan Waldron? We’ve shared photos from Meghan’s travels and hope they’ve inspired you to think about where your next adventure will be once we’re all able to travel again.
Here’s Meghan’s article on the cover of The Boston Globe:
Emerson student with progeria, disease that causes premature aging, heartened by medical advances
Meghan Waldron is an 18-year-old freshman at Emerson College who has already lived longer than most people with her extraordinarily rare disease. She’s taking an experimental drug for progeria that appears to prolong the life of people with the disease.
By Jonathan Saltzman Globe Staff, February 22, 2020, 2:32 p.m.
In many ways, Emerson College freshman Meghan Waldron seems like a lot of students in Boston. She adores pop star Ed Sheeran. She loved the latest film version of “Little Women” and wants to see it 10 more times. She had a great time backpacking alone in Europe over the summer but conceded her parents were “probably freaking out.”
She also has progeria, one of the world’s rarest diseases. The fatal genetic disorder causes premature aging and has been identified in only 169 children and young adults alive today worldwide, although researchers estimate that as many as 400 have it. Most children with progeria die of hardening of the arteries, a common killer of the elderly, at an average age of just 14.
Waldron has already lived considerably longer ― she turns 19 on March 1. She credits lonafarnib, an experimental medication she’s taken since 2007 in clinical trials at Boston Children’s Hospital. A California drug firm plans to complete its application for approval by March 31, with the hope of a favorable ruling from the Food and Drug Administration by year’s end. It would be the first approved drug for the ultra-rare disease.
“It’s been proven that it helps in extending life,” Waldron, a Deerfield native, said recently over hot chocolate at Caffe Nero near Emerson. “I’m almost 19. The life span is technically 14.” A winsome smile brightened her face. “Looks like it’s doing a good job.”
Since 2007, Children’s Hospital has run four clinical trial of lonafarnib. Waldron has participated in all four, and researchers say the results are encouraging.
In perhaps the most compelling finding, a study published by the Journal of the American Medical Association in 2018 reported that children with progeria who took lonafarnib capsules twice a day had a dramatically lower mortality rate than those who didn’t.
After slightly more than two years, one in 27 children who took lonafarnib, or 3.7 percent, had died compared with nine in 27 who didn’t get it, or 33 percent, according to the article by a team of researchers from the Progeria Research Foundation, Brown University, and Children’s Hospital. Lonafarnib appeared to slow the progression of cardiovascular disease, although it had little or no effect on other symptoms, including stiff joints, stunted growth, wrinkled skin, and loss of body fat and hair.
“The data looks fantastic,” said Dr. Leslie Gordon, lead author of the JAMA study and medical director and cofounder of the Progeria Research Foundation, the Peabody-based nonprofit that funded the trials. “You’ve got a fatal childhood disease with no treatment, and you’ve shown a survival benefit.”
For Gordon, a professor of pediatric medicine at Brown’s medical school who practices at Boston Children’s Hospital and Hasbro Children’s Hospital in Providence, the quest to treat progeria is profoundly personal.
Her son, Sam Berns, a Foxborough High School junior, died of progeria in 2014 at age 17. Like Waldron, he began taking lonafarnib in 2007 in the clinical trials. An avid sports fan who played the snare drum in the Foxborough High School marching band, he was the subject of the 2013 HBO documentary “Life According to Sam.”
Gordon had never heard of progeria when Sam, her only child, was diagnosed with it at 22 months. She has since become an authority. In 2003, she was on the research team led by Dr. Francis S. Collins, director of the National Institutes of Health, that discovered the defective gene that causes the disease. She cofounded the progeria foundation with her husband and sister.
The genetic mutation that causes progeria results in an overabundance of the protein progerin. A buildup of progerin occurs within a cell in normal aging, but the rate of accumulation is dramatically accelerated in children with the disease. Progeria has no effect on a child’s intellect, as anyone who meets Waldron — who took an Advanced Placement class in European history in high school and rhapsodizes about Michelangelo — can tell in an instant.
Lonafarnib was originally developed by the pharmaceutical giant Merck as a potential treatment for cancer. But researchers found that it can reverse an abnormality in cells of laboratory mice with progeria. Merck has licensed it to Eiger BioPharmaceuticals, a small drug maker in Palo Alto, Calif. David Cory, chief executive of Eiger, says the company has hired a chief commercial officer and a vice president of medical affair in anticipation of FDA approval.
Researchers are working on other potential treatments, including one that targets the genetic root of the disease. David Liu, a chemistry professor affiliated with the Broad Institute, Harvard University, and the Howard Hughes Medical Institute, recently announced that he and a team of scientists had used a new form of genome editing to correct the DNA mutation that caused the disorder in mice, extending their lives.
Waldron, who serves as an “ambassador” for the progeria foundation, said she was diagnosed with the disease when she was about 2. Her mother, a housekeeper at an assisted living facility, and her father, a solar energy contractor, were worried because she wasn’t growing or gaining weight, and her hair was falling out.
Waldron realized she had progeria as an adolescent when she went on the foundation’s website and saw pictures of kids who looked like her, she said.
“Obviously, I knew that I was different before that,” she said. “But it wasn’t an awareness I-have-progeria thing until at a certain point.”
The disease has hardly stopped her. She ran for the cross-country and track teams at the public Frontier Regional High School in Deerfield. She played violin in the middle school orchestra and cello in the high school orchestra.
She has met about a dozen other children with progeria from around the country at family weekends at the nonprofit Hole in the Wall Gang Camp in Connecticut for seriously ill children and their families.
When she started considering colleges, Waldron said, she had no interest in going to school in Boston. But she fell in love with the city on a visit to Emerson.
“You can walk down the street or hop on a train and go anywhere,” she said, citing the North End as one of her favorites places.
“I have great friends,” she added. “I always have.”
Emerson has made several accommodations for her. For example, the college provides a stool for her to rest her feet on when she sits at a desk in her four classes. The handle on her wardrobe in her dorm room was lowered so she could reach it more easily.
Waldron says she generally feels fine despite problems with her joints. She has dislocated her right shoulder four times doing ordinary tasks, such as reaching for a light switch.
None of this has dimmed her spirit for adventure.
“Meghan has a very strong personality. She’s driven,” her father, Bill Waldron, said in a video posted last year on the progeria foundation’s Facebook page. “I don’t think she pays attention to the fact that she has progeria.”
Indeed, after graduating from high school in June, she traveled in Europe alone for a month. The initial attraction was seeing Anne-Marie, a singer and occasional Ed Sheeran collaborator, perform in London. But Waldron decided she also wanted to experience Renaissance art. She visited Milan, Florence, Rome, Paris, and Dublin, staying in youth hostels along the way.
Waldron’s parents were nervous, she said. She was, too, but only briefly.
“There was a point of about five minutes when my parents said goodbye and I was getting on the plane where I started freaking out,” she said, laughing. “But then I was like, ‘Oh, well.’ And then I was fine.”
- What a remarkable young woman! I hope the medication gets approved. The world needs more people like Meghan.
- Meghan, I have a feeling you will do more amazing things! What a wonderful story.
- Meghan is awesome, what a great attitude and I am glad the medication is working so well. Many more bright years ahead to come for this Emerson College student!
- You go girl!
- Go girl!
- Meghan, you are a fantastic young woman.
I hope that you have a great time in Boston while you are studying at Emerson.
- What a lovely young woman! May she have many more years of success and happiness. It is amazing what the medication has done. Kudos to Leslie Berns and the Progeria Foundation!
- WOW Meghan. You are incredible and an inspiration! Best wishes to you and enjoy Emerson and Boston.
- Another thanks to the Globe writers who make the effort to cover humanity!
- Great story, great young woman. Wow!
- God bless you young lady!
- You are amazing and an inspiration.
- I was pleased to hear how much she enjoys classical music and Renaissance art. There’s a lot to celebrate in life if we just look around us. Here’s to her next trip to Italy!
- Thank you, Mr. Saltzman, for sharing Ms. Waldron’s inspiring story with us! What a lesson on living life to its fullest.
- I’m sure all of us wish her much love and much more life to live and to enjoy! And if I’m privileged to see her, I’ll tell her “you truly are my hero!”
- Here’s a story we should all read and keep with us for those times when we’re feeling like we have it tough.
- I had exactly the same reaction. This young woman is an exemplar of strength, courage, and dignity.
- And share with our children and grandchildren. Meaghan you rock!
- Rock on, Meghan!
- I remember Sam. A fine young man who left a hole in his community and school with his passing. His parents are tremendous people. Their profound loss, and the way they continue to choose to address it, is a big reason Meghan has lived to attend Emerson. I hope she gets to see the “Little Women” sequel and saves an empty seat for Sam.
- You are so inspiring, Meghan! Here’s to your drive and future achievements! Hoping the Globe will continue with updates on your story. So glad to know the drug has been successful, and hoping for many more medical breakthroughs ahead
- And may God bless the medical and research professionals who captain Team Meghan.
- God has a “special place” for Meghan when they meet each other in Heaven.
- God bless her and best wishes to her! I hope she is with us for a very long time. I’m sure she will do amazing things.
- Never waste an empty seat. Fill it with love.