You are 26 years old, and you watch your mother die of prion disease. You test positive for the gene. It's not really a matter of if you succumb to the same fate, but a matter of when. What's a girl with a law degree to do? Fuck law. Biomedical research it is. She makes it her life's mission - a race against time to find a cure. A new drug candidate designed to slow the progression of prion disease is entering a phase 1 clinical trial, which will evaluate the potential medicine for safety and tolerability. Read more here. It's too soon to say if we'll be privy to a great feat in human history, thanks to Dr Sonia Vallabh and her team. But you can't blame a guy for being optimistic. The results we've seen so far (and I know it's very early) are more promising than anything we've seen at all. It's prions, after all. Pending the results of these trials, we may have the most promising treatment against prion disease, and the only treatment in human history against an entire class of this disease at all. We may have our first weapon against the prion. Her team is dedicated to keeping their findings completely open source. There is already another drug trial based off of her work undergoing with Ionis Pharmaceuticals with their drug ION717. “Standard drug development is, for all sorts of reasons, not well set up to be a learning system,” Vallabh said. “But we can’t afford to treat each shot on goal as a self-contained unit – everything needs to feed the mission. I wish the absolute best for this exact molecule, and I also know that however it performs in the clinic, this experience must move us forward. It’s our job to figure out how.” What a champ. We'll have a few metrics to compare. Here is a link to her AMA she did over 10 years ago for those curious. I've been rooting for her since I was a teen. Keep her and this trial in your hearts. It's time humanity got its revenge on the prion.   submitted by   /u/HerpesFanatic01 [link]   [comments]