Start2Cure Foundation Awards Grant to MS Gene Therapy Project
MS is an autoimmune disease characterized by the damage and loss of myelin — the protective layer around nerve fibers, or axons, that is crucial to the effective transmission of signals in the central nervous system, which includes the brain and spinal cord.
Most MS treatments are aimed at suppressing the immune system and reducing inflammation to protect the myelin coat. However, they do not prevent the development of the disease, nor can they reverse damage done by the disease.
“Unfortunately these therapies do not offer improvement of the symptoms for people whose nervous system is already damaged by MS. This damage is practically irreparable, and there are currently no therapies that repair damage to nerve cells,” Inge Huitinga, PhD, department head at the Netherlands Institute for Neuroscience (NIN), said in a press release.
To address this issue, a team of researchers at NIN proposed a gene therapy approach to repair the damage caused by MS. Gene therapy is based on the delivery of therapeutic genetic material into the body and has been widely studied as a potential treatment for MS.
NIN researchers will use brain tissue from MS patients who recovered from disease relapses, donated to the Netherlands Brain Bank, to identify the genes involved in the production of myelin and in repairing nerve fibers.
“Repairing this damage is precisely what we aim to do with this project, by means of gene therapy,” Huitinga said.
To test their ability to generate myelin and consequently reverse brain damage, the identified genes will be placed in a viral vector — a harmless modified virus designed to deliver genetic material into cells — and injected via the bloodstream. The vector will then reach the central nervous system, where it can deliver its cargo.
“This new form of gene therapy allows us, for the first time, to deliver a therapeutic gene to the widespread MS lesions in the brain,” said Joost Verhaagen, PhD, department head at NIN.
The team will first test their approach in an animal model of MS. The next step is to evaluate it through clinical trials involving patients with MS.
“When this project delivers positive results, the translation to the clinic can be made, in collaboration with (an) industrial partner(s),” Huitinga said. “A drug that promotes functional recovery of the nervous system is very important and potentially very meaningful for a large group of people with advanced MS.”
Netherlands-based Start2Cure Foundation is a newly launched nonprofit aimed at fighting neurodegenerative diseases, especially MS. It is dedicated to supporting research into innovative treatments on a global scale through grants and awards, with the ultimate goal of finding a cure for MS within the next 50 years.
“Awarding our first Therapeutic Innovation Research Grant is the starting point of the Start2Cure Foundation and its Global Research Grants and Awards program,” said Boaz Spermon, managing director of the foundation.
“The gene therapy research project of the Netherlands Institute for Neuroscience is a very challenging project given the complexity and the uncertain outcome. As a result, funding opportunities for this type of research are often scarce. Should this innovative approach, however, turn out to be truly successful, the impact will be even more significant,” Spermon added.