The process of getting a drug to patients is cumbersome and heroic. For Drs. Sonia Vallabh and Eric Minikel, who founded the Prion (pronounced “pree-on”) Alliance to develop therapeutics for human prion disease, their path has been, they say, like “driving at night in the fog,” quoting E.L. Doctorow: “You can only see as far as your headlights, but you can make the whole trip that way.”
We recently talked with Sonia about where she and Eric find themselves now, more than 10 years since Sonia discovered she had inherited a genetic variant that puts her at high risk for prion disease. We wanted to know not only where are they in identifying drug candidates to treat the disease, but where are they in the process of clinical trials to get drugs to patients?
