Earlier last month, the second clinical trial for progeria was announced as completed. The trial was again funded by The Progeria Research Foundation, spearheaded by Drs. Scott Berns and Leslie Gordon, who are parents of the late Sam Berns.
Sam was, for many, the inspirational face of progeria, starring in a Ted Talk about happiness and an HBO documentary. Unfortunately, he died in early 2014 of the disease, which causes rapid premature aging and fatal heart problems.
To learn more about progeria, click here.
Sam was a subject in the previous progeria trial, which proved some efficacy with a farnesyltransferase inhibitor called lonafarnib in slowing the symptoms and complications of progeria. However, that study used lonafarnib as a monotherapy, and this study used lonafarnib in combination with pravastatin and zoledronic acid, with the hope that this would prove more effective. 37 patients in total were dosed with these therapies.
Overall, comparisons with the lonafarnib monotherapy trial revealed that there was an additional bone mineral density benefit with additional therapies. However, there was unfortunately, no added cardiovascular benefit noted.
Even so, the primary study outcome of increased rate of weight gain and decreased carotid artery echodensity was successfully achieved; these factors help lessen the inevitable complications that progeria brings on, so the study was a success overall.
Progeria proves to be a difficult condition to treat, but The Progeria Research Foundation is continually working tirelessly on new drug therapies and combinations to help the patients that suffer from this disease.
However, since progeria is an ultra rare condition, research, and capital for research, are largely being developed by the foundation itself and the inspirational and determined people running it, like Sam Berns’ parents and the One Possible fundraising teams.
The Foundation is currently trying to raise an additional $200,000 to kickstart another progeria clinical trial. Click here to read more about the already-published trial and to learn how you can help donate to the progeria cause!
