12:00 AM
Sep 28, 2015
 |  BioCentury  |  Regulation

Serving nerves

How patient and industry priorities match up in HD, PD

Testimony at the first of FDA's patient-focused drug development meetings last week revealed that several programs in industry's pipeline for Huntington's disease are aligned with patient and caregiver desires for drugs that could halt progression and improve psychiatric and cognitive symptoms, although it will be difficult to demonstrate those effects in the clinic.

In Parkinson's disease, there appears to be a clearer route to meeting patient needs with clinical programs focused on solving PK problems with existing drugs to enable better motor control.

Huntington's disease (HD) is a fatal progressive neurodegenerative disorder characterized by involuntary movements known as chorea; psychiatric symptoms including irritability, depression and anxiety; and cognitive decline. The autosomal dominant disease results from an expansion of CAG repeats in the gene encoding huntingtin (HTT), which can be detected before symptoms develop.

The only drug approved in the U.S. to treat HD - Xenazine tetrabenazine from Valeant Pharmaceuticals International Inc. - only treats chorea.

Participants at last Tuesday's patient-focused drug development workshop said not all HD patients have chorea, nor do they consider it their most bothersome symptom. In a poll asking for the three symptoms that most impact patients' and caregivers' daily lives, cognitive impairment came in first at 65%, followed by depression or anxiety (47%), difficulty walking (37%) and chorea (30%).

Huntington's Disease Society of America President and CEO Louise Vetter told BioCentury after the workshop that according to an HDSA survey, patients find chorea less problematic than their caregivers do - but both agreed executive function is important.

"It's the cognitive impact that steals one's ability to work, to maintain the quality of independent life that individuals hold so dear," she said.

At the workshop, patients and caregivers also described how psychiatric symptoms reduced independence and took a toll on caregivers and families.

"My husband now refuses to leave the house because it sends him into a panic at the thought of it. The anger and irritability is so hard on our children and family as a whole," said Katie Jackson, who spoke on behalf of her husband Mike.

"I never feared for my or my children's safety, but I know families that live in fear daily," she added.

Participants also...

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