So here’s something awesome.
Seemingly right on the tails of a few notorious articles debating the worth of funding for orphan drug research comes something completely unexpected. Bursting into the headlines yesterday came a story concerning an unexpected side effect of bexarotene, a substance sometimes used for cancer treatment and classified as an orphan drug.
In a study of mice in the early stages of Alzheimer’s disease, bexarotene has significantly reversed some of the effects of the illness – namely reversing brain abnormalities and restoring "declining mental function". The mice also showed improvement in tests of memory and social behavior.
While this sometimes seems debatable, there is in fact a large difference between human beings and rodents, so it is difficult to tell at this time what this study will mean for people affected by Alzheimer’s. Because of this, the pharmaceutical industry is hesitant to jump to any conclusions, but if nothing else this study is huge. It does make things easier, however, that the drug has already been approved for human use – not for Alzheimer’s, but for a rare form of cancer called cutaneous T-cell lymphoma. This doesn’t mean that clinical trials will start overnight, but matters will definitely move much faster than they would if this were a brand new substance.
While cutaneous T-cell lymphoma affects "only" 3,000 Americans a year, Alzheimer’s devastates millions. And here we are – a little orphan drug suddenly has an enormous and completely unforeseen potential. Good thing there was funding to develop it in the first place…just saying…
Interested in keeping up to date on developments in the orphan drug industry? Meet the best at the World Orphan Drug Congress USA