A UMass Medical School professor and Nobel Prize winner helped pave the way for a drug after discovering a different kind of ribonucleic acid.
Forbes reported Tuesday that Cambridge pharmaceutical company Alnylam Pharmaceuticals was successful in developing a drug to combat ATTR amyloidosis with polyneuropathy, a rare hereditary disorder that causes patients to lose their ability to dress themselves, use utensils or walk.
The disorder is fatal, as patients usually die within 15 years of diagnosis.
In 1998, Craig Mello, a UMass professor, discovered RNAi, a different kind of ribonucleic acid or RNA that tricks cells into destroying messenger RNA before it can be used to make a protein, in effect, blocking genes.
The discovery was heralded as a breakthrough and brought a deeper understanding of how genes work. Now, Alnylam, in conjunction with another company, has developed the drug patisiran that studies show can be effective in fighting the disorder by using RNAi to block the hereditary disorder.Â
The death rate in patients who took the drug was slightly lower compared to those on a placebo (4.7 percent compared to 7.8 percent), but 37.7 percent of patients on a placebo stopped treatment, compared to 7.4 percent on patisiran.
Shares in Alnylam Pharmaceuticals recently rose 40% to $105 in morning trading.
