Carly Kudzia, 7, with her mother, Heather Unsinger, in Swanton, Ohio. Carly participated in a study suggesting that a drug lonafarnib may extend life for children with progeria,
The children in the study were also involved in earlier research testing the same drug combined with two others. Within a period of about five years, survival was extended by about 19 months. It wasn’t clear if all three drugs or just
one or two of them contributed to the improvement.
Progeria being an incurable disease, the only hope of a successful treatment was wherein the lifespan of the patient is increased although not by much.
The first successful treatment was carried out in 2012, as a result of the first clinical drug trial of lonafarnib, a farnesyltransferase inhibitor (FTI) that was devloped keeping cancer patients in mind.
The oral medication, the FTI lonafarnib, was given twice daily over the course of the study, over two years. The research team assessed the children’s rate of weight gain, compared to their pre-study rate.
The clinical trial results showed significant improvements in bone structure, weight gain, and most importantly, the cardiovascular system,