PEABODY — The Progeria Research Foundation’s (PRF) annual ONEPossible campaign is nearing the finish line of raising $110,000 by the end of July.
So far the campaign has raised nearly $77,000, but thanks to an anonymous donor, the campaign is on solid footing to reach its goal sooner rather than later.
“We had an anonymous donor who said he will match $10,000 in donations through July 15, so that should help us reach our goal,” said PRF President and Executive Director Audrey Gordon. “This is our annual mid-year campaign that focuses on our belief that even one person making even a small donation can make a difference and help make a cure possible.
“We are all about research seeds blooming into a cure. We want people to know how important they are to us as it all adds up; whether it’s $25 or $2,500, we can’t attain our mission without them.”
The goal of the campaign, which kicked off in June, is to help PRF continue its work toward developing new treatments, and ultimately finding a cure for progeria, an extremely rare and fatal rapid-aging disease that strikes down children and young adults in their teens, usually by heart disease or strokes more commonly found in the elderly population.
Gordon said that PRF had identified 195 children with progeria, which represents about half the children suffering from the disease worldwide. About 60 of those children, representing 30 countries, are participants in a PRF trial in which families incur no expenses. Donations, food, transportation and lodging are provided by PRF, while drug costs are covered by health insurance.
Since awarding its first research grant in 1999, PRF has worked with world-class scientists to develop novel breakthroughs and treatments which help children with progeria live longer, healthier lives. PRF has sponsored five clinical drug trials and funded 77 grants totaling $8.4 million. PRF has also created a cell and tissue bank along with a medical and research database containing information on 191 children, and has sponsored 13 international scientific meetings.
“We pretty much do it all, one-stop shopping style, with everything being conducted in-house,” Gordon said. “We thought the best route was to control our own resources.”
In November 2020, 13 years of research that included four PRF-coordinated clinical trials culminated with FDA approval of Zokinvy (lonafarnib), the first-ever treatment for progeria, opening the door for children and young adults to get the drug by prescription instead of by clinical trials — the drug adds two 1/2 years to life expectancy. Without treatment, average life expectancy is only 14.5 years.
“We’ve learned that now instead of talking about children with progeria, we are now talking about young adults as they are living longer,” Gordon said.
The FDA approval was only the beginning of what turned out to be a huge windfall for PRF. Three days after approval, PRF announced that it had entered into an agreement to sell a priority review voucher to Eiger BioPharmaceuticals (Eiger)for $95 million. Under the terms of the voucher program, PRF received 50 percent of the net proceeds.
“It’s basically a government incentive program designed to fast track drug development. Once you have FDA approval, pharmaceuticals can then approach us to buy into the process on a 50-50 basis,” Gordon said. “We basically sold the voucher and split the gain, which was a wonderful boost for us. It came at the perfect time, knowing we needed to foot the bills for a cutting-edge process that could cost up to $50 million or more because we are trying to pursue all avenues to get to clinical trials so we can develop new drugs and find a cure.”
The sale proceeds boosted PRF total fundraising to more than $85,000,000.
The Peabody-based foundation was started in 1999 by Gordon, her sister, Dr. Leslie Gordon, and her husband, Dr. Scott Berns, a year after their son, Sam, was diagnosed with progeria at the age of two. He died in 2014 at the age of 17.
Following the diagnosis, his parents questioned why there was virtually no research on or treatments for the disease.
“Sam was an amazing young man who was incredibly inspiring and full of life, a real blessing,” said Audrey. “But we learned that there was no research, most likely because progeria is the rarest of all rare diseases that affects only a small number of people. We knew that we needed to start a research foundation, otherwise these children would continue to suffer with no hope of a cure.
“We had never really heard about progeria. I used to think that I could never go through what some families have to go through with things like this, but we all learned we can — we just had to do it and that’s what we did.”
To donate to ONEpossible, use the mobile app https://app.mobilecause.com/vf/ONEpossible or go to https://www.progeriaresearch.org/2021/06/01/prfs-onepossible-campaign-kicks-off/. You can also make a donation by texting ONEpossible to 71777.
Anne Marie Tobin can be reached at [email protected].
