The first clinical drug trial for children with progeria has been an astounding success! At the center of it all is a drug called Lonafarnib. First, what is progeria?
The miracle drug - Lonafarnib Lonafarnib is a type of farnesyltransferase inhibitor (FTI) that was originally meant to treat cancer, however it has proven very effective for Progeria. Throughout the 2 ½ year clinical trial, with 28 children from 16 countries, every child has shown improvement in one of four areas: gaining additional weight, improved bone structure, improved hearing, and increased flexibility of blood vessels. FTIs work by blocking the attachment of the farnesyl group onto the lamin A protein, thus reducing the buildup of farnesyl-lamin A complexes that disfigure the shape of the nuclear lamina. A good way to think about this is to imagine a line of perfectly parallel parked cars as being the normal structure. Once a person in a bigger car, like one of those mammoth-sized Ford F150 Raptors, tries his or her luck at fitting into a parking space meant for a Toyota Corolla, each car is going to ram into another like dominoes. The street would be a disaster and nobody would be able to travel across the street for some time. Likewise, a disfigured nuclear lamina would not be efficient at genetic regulation. FTIs, in our car example, would cut off a portion of the Ford F150 so that it fits in the spot. FTIs in our body allow for farnesyl to be separated from lamin A so it can join into the nuclear lamina. Scientists have discovered that progerin is actually produced in the general population, leading many to believe that progerin is involved with the normal aging process. Things to keep in mind FTIs are still very new and the long term effects of these drugs has not been observed. After only 2-3 years of clinical research, trials would need to continue for much longer to make sure the drug does not carry any adverse side-effects, which may include heart, liver, or bone complications. Hopefully, Lonafarnib and other FTIs sustain their success and save the lives of progeric children around the world. I recommend everybody to watch the TED talk below by Sam Berns, a truly inspirational child with progeria. Please watch. Thank You!
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