Development of a Non-Invasive Gene Therapy for Multiple Sclerosis.

Multiple sclerosis (MS) is an inflammatory demyelinating disease that affects 2.8 million individuals worldwide. Myelin protects nerve fibers and is crucial for efficient propagation of electrical signals in the brain. In MS, myelin is damaged due to an attack of the immune system. A new public private partnership between the Neuroimmunology and Neuroregeneration research groups at the Netherlands Institute for Neuroscience and the biotechology company SanaGen will combine forces to develop a gene therapy to repair damage to myelin and restore function in an animal model for MS.  

The development of a long-lasting regenerative treatment for MS would provide a therapeutic option for MS patients that is radically different from the current anti-immunological treatments which slow down but do not provide a long-lasting treatment for the disease. Therefore the socio-economic benefit will be significant. This project is supported by key patient organisations, including the MS Vereniging Nederland and the European Multiple Sclerosis Platform.   

Non-invasive CNS-wide gene therapy for genes with pro-myelinating functions using a vector that passes the blood brain barrier to promote remyelination and functional recovery in MS is a new concept. This project will provide the first ever evidence for the effectiveness of this gene therapy strategy in a major neuro-immunological disease and has the potential to significantly improve the quality of life of millions of MS-patients.