Team Zoey, the fundraising group that was formed to support research into a rare disease affecting a Verona toddler, is announcing some hopeful results in a key drug trial.
According to the group, the results of the first-ever clinical drug trial for children with Progeria, the fatal rapid-aging disease that affects Zoey Penny, show the efficacy of a farnesyltransferase inhibitor (FTI), a drug originally developed to treat cancer. The clinical trial results show significant improvements in weight gain, bone structure and the cardiovascular system. The study results were published today in Proceedings of the National Academy of Sciences.
Progeria, also known as Hutchinson-Gilford Progeria Syndrome (HGPS), is a rare, fatal genetic disease characterized by an appearance of accelerated aging in children. All children with Progeria die of the same heart disease that affects millions of normal aging adults (atherosclerosis), but instead of dying at 60 or 70, children with progeria may suffer heart attacks and strokes as early as age five years.
“To discover that some aspects of damage to the blood vessels in Progeria can not only be slowed by the FTI called lonafarnib, but even partially reversed within just 2.5 years of treatment is a tremendous breakthrough, because cardiovascular disease is the ultimate cause of death in children with Progeria,” said Leslie Gordon, M.D., Ph.D., lead author of the study, medical director for PRF, and mother of a child with Progeria. In addition, Dr. Gordon is a staff scientist at Boston Children’s Hospital and Harvard Medical School, and associate professor at Hasbro Children’s Hospital and Alpert Medical School of Brown University.
Twenty-eight children from 16 countries–including Zoey Penny–participated in the two-and-a-half year drug trial. The children traveled to Boston every four months to receive comprehensive medical testing through Boston Children’s Hospital’s Clinical and Translational Study Unit.
so pleased to hear there is a breakthrough for these special children – God Bless and keep going!
Zoey has not participated in any drug trial yet…..but would love to!
Oops. Sorry Ian. Thanks for the correction.
No worries! Thank you for writing about Progeria Research Foundation’s progress.