Precision Medicine in MS – What Will it Take?

I’ve just come back from an exciting meeting in Dallas hosted by ACTRIMS (Americas Committee for the Treatment and Research in MS).  The focus was precision medicine. It’s the idea of using a person’s genetic background, environment, lifestyle and other factors to develop a customized approach to treat MS.
More than 1200 clinicians, young investigators and seasoned researchers gathered to consider what precision medicine would look like for people with MS, and the challenges and opportunities in getting closer to precision medicine. If you’re interested, you can browse the summaries of the presentations here.
Informally, MS doctors carry out some form of customized medicine every day during office visits. They draw on their experience and what they know about a person, the person’s history of disease activity, lifestyle, and other factors when recommending any particular MS therapy.
I was interested in a talk given by Dr. Gavin Giovannoni of Queen Mary University of London, who described a more formal checklist approach to look at these personal factors to help his team make treatment recommendations. He pointed out that the most advanced precision medicine approach to treatment we have right now relates to the use of Tysabri, where before beginning therapy and while they are taking it, people are screened for antibodies to the JC virus, which causes the serious brain infection PML (read a summary of his presentation).
Some advances in precision medicine are emerging from the field of cancer, where, for example, people with a specific gene alteration are known to need aggressive treatment for breast cancer.
But we’re still a long way from being able to take a blood or saliva sample, analyze it with advanced technologies, and come back with definite information on a person’s likely MS disease trajectory and the best treatment options to slow, stop or reverse that trajectory.
One of the challenges in MS is how many different forms MS takes. It can be aggressive or mild, relapsing or progressive, or both. The person may be dealing with other disorders besides MS—such as high blood pressure or heart conditions (read a summary of a talk on “comorbidities” by Dr. Ruth Ann Marrie, University of Manitoba). A person’s ethnicity also influences disease course (read a summary of a talk about race and ethnicity by Dr. Lilyana Amezcua, University of Southern California). Each of these aspects is important, and right now there’s not a simple way of predicting what one individual’s disease course may look like down the road.
There’s a lot of research looking for biomarkers—such as molecules detected in the blood—that could facilitate diagnosis, predictions and treatment decisions. One biomarker being explored in MS is neurofilament light. It is a molecule that is part of the debris that enters the spinal fluid and blood when nerve wires (axons) are damaged—whether from head trauma, MS or some other disorders. Dr. David Leppert of the University of Basel showed how levels of neurofilament light rise with injury and fall with effective treatment. With more testing, neurofilament light may be validated as a marker of treatment response in clinical trials and even in the doctor’s office (read a summary of his presentation here).
One clear take-home message for me is that there is an explosion of informatics science, and eventually, one might use millions of data points to create a sophisticated profile of a person’s genes, the molecules their body is processing, the microbes in their guts, and other various biomarkers. These data are being generated in research settings right now, and the challenge will be how to integrate the most important datasets to determine the very best treatment approach for this person.
I am excited to see so much activity and intent by researchers and funders to tackle these challenges, and by the hope precision medicine can offer for better outcomes for people living with MS. 

Read more details about ACTRIMS 2019 on our website.
Tags Research, Treatment      3

Mark Allegretta, PhD

Dr. Mark Allegretta is the Vice President of Research at the National MS Society, leading commercial research including partnerships developed through Fast Forward. He brings expertise in immunology and 28 years of experience in biotechnology and pharmaceutical operations to help drive the development of new therapies to stop MS and restore function.