Progressive Multiple Sclerosis (MS)Drug Discovery Project led by Dr. Gianvito Martino
Dr. Martino’s Lab - San Rafaelle Institute
MS is a chronic, often disabling disease that attacks the central nervous system, made up of the brain, spinal cord and optic nerve. The disease attacks and destroys myelin – the protective fatty insulation around the nerve fibers of the brain and spinal cord – interrupting signals between the brain and the body. The damage caused by these attacks is where the disease gets its name ‘multiple sclerosis’ literally meaning ‘many scars’. The damage is lasting and stays with you forever.
Symptoms may be mild, such as numbness in the limbs, or severe, such as poor balance, painful spells, paralysis or loss of vision. The progress, severity and specific symptoms of MS are unpredictable and vary from person to person and from time to time in the same individual, which makes it hard to diagnose and treat.
Each individual’s experience of MS is unique, with a different combination of symptoms and severity. But everyone with the disease lives with the uncertainty of whether it will progress and whether they will lose the ability to do the things that matter most to them. There are 2 types of MS, Relapsing-Remitting, and a more acute version, known as progressive MS. A diagnosis of progressive MS takes the challenge of living with MS to a new level because, unlike Relapsing-Remitting MS, there are limited effective treatments for Progressive MS.
Every day, people with Progressive MS lose some of their ability to move, think, and connect with those they love and the greater world. While there are many approved disease-modifying treatments for Relapsing forms of MS, there are limited approved treatments for Progressive MS. These treatments are still insufficient to fully address Progressive MS. We need to better understand this debilitating form of MS, which is a complex and challenging problem and discover disease modifying therapies.
The International Progressive MS Alliance was established to foster global collaboration among MS organizations, researchers, clinicians, pharmaceutical companies, and people living with progressive MS to fuel necessary research to speed up treatment development for progressive MS.
Initially the Alliance received 52 applications from around the world, involving almost 500 investigators. Following extensive peer and technical review, involving key academia and industry experts, three projects and collaborative networks were funded for a duration of 4 years. In 2018, The Foundation For A Better World committed to fully fund one of these efforts.
The project is being led by Gianvito Martino, M.D. at the Division of Neuroscience at the San Raffaele Institute in Milan, Italy and involves a collaborative network of 13 different investigators from Europe, Canada and the U.S.
The team is working to identify molecules that may have a protective role in nerve cells or neurons and/or the capacity to promote myelin repair. They are focusing their efforts in three phases:
Identifying potential drugs or compounds using sophisticated bioinformatics tools specifically developed to virtually reproduce pathogenic mechanisms of MS.
Screening these compounds for their ability to protect nerve cells or promote myelin repair in laboratory tests using both rodent and human neurons and myelin forming cells.
Evaluating in animal models of progressive MS the therapeutic potential of the ‘candidate’ compounds identified through the in vitro screening.
The research team expects to identify a limited number of previously unidentified molecules with a high chance of therapeutic power in progressive MS patients. They expect that within four years from the start of the project they will identity one or two human grade compounds that can be used in Phase I/II clinical trials in patients with Progressive MS.
The Foundation For a Better World is intimately involved in reviewing the progress of the project. We have been impressed with the leadership of Dr. Martino in integrating the research being done in many centers of excellence around the world. Despite the obvious setbacks caused by the COVID-19 pandemic, Dr. Martino’s project is still on schedule and already showing remarkable results.
