Blazing the Way to New Genetic-based Therapies for Neurological Diseases

Penn Neurology's many efforts to understand and diagnose the genetic causes of neurologic diseases has positioned the clinical team as one of the top neurogenetic programs in the country.

Now, a new initiative is taking things one step further by translating this work into the next generation of treatment. 

Launched in March 2020, the Translational Center for Excellence (TCE) for Gene and Molecular Therapy is already supporting clinical trials for Amyotrophic Lateral Sclerosis, Parkinson's disease, Spinocerebellar Ataxia, Huntington's disease and Spinal Muscular Atrophy.

Penn's Translational Center for Excellence for Gene and Molecular Therapy

"Our goal is two-fold," says program director and neurologist, Steven S. Scherer, MD, PhD.

"First, we're intending to achieve a definitive genetic diagnosis for as many patients as possible. Most treatment strategies for neurologic disorders use genetic-based therapies requiring an exact genetic diagnosis," he explains. "This process will also give us a database of eligible patients for new treatments and clinical trials. We'll be one step ahead of the game."

"Our second goal is to create a team of clinicians, nurses, genetic counselors and clinical research staff that is devoted to clinical trials of novel gene and molecular therapies," explains Sara-Claude Michon, PhD.

"Having a team of experts assembled would help streamline the process so we don't have to reinvent the wheel with each trial," she continues. "We'll have the expertise to run trials in our center and collaborate on trials with other teams. We already have 9 trials at various points of the study startup and recruitment phases."

Additionally, the Center has been approached by outside companies looking for help with the development of their new clinical programs, which will lead to industry-sponsored trials down the road.

"Companies come to us because we have the physician expertise and the patient populations for these trials," explains Dr. Michon. "It's one of our biggest selling points. Industry sponsors are also looking for help developing their clinical programs. We are building the team and it's growing very fast."

With a PhD in Neuroscience and 10 years' experience working in clinical trials, Dr. Michon is both the face of the Center and its main operations person.

"Penn is the epicenter of gene therapy for humans," says Dr. Scherer. "The value we offer to companies looking to do gene therapy and related trials is immeasurable. We are taking a proactive approach and front-loading the center with clinical trial specialists and genetically diagnosed patients, so it's only natural that companies are seeking us out."

"We are responding to a need," adds Dr. Michon. "Companies need to determine if their clinical trials and products are safe and effective. Through our center's initiative, we are streamlining that process with the goal of establishing new treatments for patients with neurological disorders."

One additional area of expansion for the TCE for Gene and Molecular Therapy in the future would be to develop a training program for neurologists and students who want to devote their expertise to trials and gene therapy. This would be the first program of its kind in the nation.