FDA Grants BrainStorm Meeting for NurOwn, Gene Therapy Study of AB-1005 Underway, VISION Trial to Test DBS Enhancement in PD

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

According to a new announcement, the FDA has granted BrainStorm Cell Therapeutics a meeting to discuss NurOwn, the company’s investigational stromal cell therapy, and its regulatory path forward as a treatment for patients with mild to moderate amyotrophic lateral sclerosis (ALS). The meeting is set to take place on December 6, 2023, and the company stated that it plans to discuss a Special Protocol Assessment (SPA) with the agency for the overall protocol design of a potential confirmatory phase 3 trial. In September 2023, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 17-1-1 (17 No; 1 Yes; 1 Abstain) that the current data on NurOwn was not sufficient in demonstrating efficacy as a treatment for patients with mild to moderate ALS.

According to an announcement from AskBio, the first patient has been dosed in its phase 1 REGENERATE MSA-101 study (NCT04680065) assessing AB-1005, an adeno-associated viral vector serotype 2 (AAV2) gene therapy in development for patients with multiple system atrophy (MSA)-parkinsonian type (MSA-P). The first dosing took place at the Ohio State Wexner Medical Center while other institutions such as the University of California Irvine and the Quest Research Institute are expected to have patients as well. AB-1005, also known as AAV2-GDNF, is designed to encode the human glial cell line-derived neurotrophic factor transgene that is delivered to the putamen.

Surgical Information Systems (SIS) recently announced a new post-marketed study, dubbed VISION, that will evaluate whether use of the company’s SIS visualization technology can enhance the accuracy of deep brain stimulation (DBS) in patients with Parkinson disease (PD). The SIS System, an FDA-cleared visualization technology, provides patient-specific imaging for planned procedures like DBS that target the subthalamic nucleus (STN) and global pallidus internus (GPi). It utilizes standard clinical imaging (T1- and T2-weighted) MR images with validated accuracy of less than 1mm. VISION, a prospective, open-label study, will include 90 patients across several sites, with the aim of determining if using the SIS System results in less distance between the planned DBS target location and the actual implanted lead location.

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