Huntington’s Disease Drug Development and Patient Groups


Scott Schobel, MD, MSc, clinical science leader for Roche’s Huntington Disease Program talks about the role that Huntington disease patient group, throughout the world, helped Roche develop their clinical program focused on Huntington disease.

Huntington disease is a neurodegenerative genetic ailment in which symptoms often appear suddenly when a person is about 40 years of age. As the person’s neurons die, the physical, cognitive and emotional symptoms associated with the disease can devastate the person and their family. Currently, there are no treatments that target the pathophysiology of the disease (i.e., the over production of the Huntingtin protein that results from mutations in the Huntingtin gene).

Roche is currently developing RG6042, a RNA antisense drug, that can reduce the synthesis of the Huntington protein and thereby, reduce its toxic effects in the brain.

As Dr. Schobel explains in this interview, working with patient groups like the Huntington Disease Society of America (HDSA), Huntington Disease Society of Canada, Huntington Disease UK, and many others, allowed them to formalize the best dose needed for their trials.

“We know many of the members of the patient community by name and we can have an interaction that keeps us close to what matters,” stated Dr. Schobel, adding that the relationships are “not only informative scientifically but is also hugely motivating.”

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