Parkinson Disease Gene Therapy PR001 Granted Fast Track Designation, Telerehabilitation Similar to In-Clinic Rehab for Stroke, New Therapeutic Technologies In Epilepsy

July 13, 2019

Neurology News Network for the week ending July 13, 2019.

This week, Neurology News Network covered Prevail Therapeutics' AAV9-based gene therapy PR001 for treatment of Parkinson disease with a mutation in the GBA1 gene, telerehabilitation, which has shown to have similar efficacy in rehabilitating and improving motor status for patients who had a stroke, and a conversation had with Michael Sperling, MD, at the 2019 International Epilepsy Congress about a number of new therapeutic technologies in the treatment of epilepsy (transcript below).

Jenna:

Welcome to Neurology News Network. I’m Jenna Payesko. Let’s get into the news from this week.

The FDA has granted fast track designation to PR001, an investigational, disease-modifying, single-dose AAV9-based gene therapy for treatment of Parkinson disease with a mutation in the GBA1 gene, according to Prevail Therapeutics.

In June, the FDA accepted the company’s IND, which allows for the initiation of a phase 1/2 clinical trial that will examine the safety and tolerability of PR001. The trial will also measure key biomarkers and exploratory efficacy endpoints in patients with Parkinson disease with GBA1 mutation. Dosing is expected to occur in the second half of 2019.

Telerehabilitation has been shown to have similar efficacy in rehabilitating and improving motor status for patients who’d had a stroke when compared with traditional in-clinic rehabilitation.

In an 11-site, 124-patient trial led by Dr. Steven C. Cramer of the University of California Irvine School of Medicine, the use of activity-based training produced substantial gains in arm motor function in both the home-based telerehab or traditional in-clinic rehab groups. The investigators concluded that these data suggest that telerehabilitation could potentially increase access to rehabilitation therapy, substantially and on a large scale.

At the 2019 International Epilepsy Congress, in Bangkok, Thailand, Dr. Michael Sperling, director of the Jefferson Comprehensive Epilepsy Center, gave an informative talk at the meeting about a number of new therapeutic technologies in the treatment of epilepsy.

In a conversation with NeurologyLive, he described the advancements in the surgical technology, saying that “practically, it means that people come into the hospital and go home the next day. For pain relief they use acetaminophen—they don’t need narcotics—and they can go back to work a couple of days later. It’s at that level, as opposed to 3 days in the hospital with a craniotomy, significant analgesics, people are out of work for 4 to 6 weeks. Some of these minimally invasive techniques, while still invasive, are far better than what we have had.”

For more direct access to expert insight, head to neurologylive.com. This has been Neurology News Network. Thanks for watching.