Gene therapy as treatment for MS
Neurological symptoms as a result of Multiple Sclerosis (MS), such as loss of cognitive or motor function, lead to a great many problems for over 2.5 million people worldwide. Nerve damage to the brain and the spinal cord cannot be repaired. Researchers of the Netherlands Institute for Neuroscience want to change this by means of the use of modern gene therapy technology. Funding from the Start2Cure Foundation will enable the Netherlands Institute for Neuroscience to embark on an ambitious project to repair the brain damage caused by MS.
The current treatments for MS are mainly geared towards inhibiting inflammatory cells from invading the brain. They reduce the number of MS attacks, but do not stop the disease from developing. In the later stages of the disease they virtually cease to have an effect. “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. But repairing this damage is precisely what we aim to do with this project, by means of gene therapy,” says professor Inge Huitinga.
Gene therapy involves introducing therapeutic genetic material into the body. For this a viral vector, a carrier of therapeutic genetic material, is used. It has recently become possible to reach the brain and the spinal cord by injecting the viral vector via the bloodstream. “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,” explains professor Joost Verhaagen.
Molecules that promote recovery
The aim of the new project is to start with identifying the molecules that stimulate the creation of myelin and the repair of axons in brain tissue donated to the Netherlands Brain Bank by MS patients who recovered very well from MS attacks.
Myelin is the protective fatty substance surrounding the nerve cell axons that transmit the signals in the brain and spinal cord. In people with MS this myelin layer degrades, which means that signals by nerve cells are no longer transmitted properly and loss of function ensues. The newly discovered molecules will then be introduced into viral vectors, together with a number of already known factors, and tested in an animal model for MS for their ability to produce myelin and restore neurological function.
“When this project delivers positive results, the translation to the clinic can be made, in collaboration with (an) industrial partner(s),” says Huitinga. “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”.
Start2Cure is a new foundation and aims to cure MS within 50 years by enabling scientists to develop breakthrough treatments. “Awarding our first Therapeutic Innovation Research Grant is the starting point of the Start2Cure Foundation and its Global Research Grants and Awards program,“ says Boaz Spermon, Managing Director Start2Cure. “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.”