Patients are the key to the success of new projects at The Neuro that could greatly help in treating multiple sclerosis (MS) and understanding its cause. One project involves patients and novel therapies. The other project focusses on gathering and analyzing patients鈥 data in a comprehensive new database.
This database is called Data Extraction in Multiple Sclerosis To Inform Future Interventions, or DEMySTIFI. It will become part of The Neuro鈥檚 C-BIG Repository, a vast collection of biological samples, imaging and genetic data from both neurological patients and healthy control subjects. The Neuro鈥檚 Open Science policy makes this data freely available to world science.
鈥淲e have such a large MS footprint at The Neuro: about four thousand patients are seen in our MS Clinic, and about 50 staff members are doing clinical and laboratory studies,鈥 says the Dr. Paul Giacomini, the MS Clinic鈥檚 director, who is spearheading the DEMySTIFI project. 鈥淲e can move the field forward meaningfully by capturing patients鈥 clinical data and using the new bio-repository platform to better analyze and understand the nuances of MS.鈥
MS develops differently depending on whether a patient has the relapsing-remitting form of the disease or the progressive form.
鈥淪ome patients have a mild form and can go years without a relapse. Others have numerous relapses despite aggressive treatment. We think the best way to understand this is to get as much data as possible and take advantage of improvements in data science, including machine learning and artificial intelligence. By teasing out the nuances from this data set, and applying what we learn, we hope to better predict who will respond well or poorly to a specific therapy.鈥
Dr. Giacomini and his colleagues, including Dr. Douglas Arnold and Dr. Louis Collins, expect DEMySTIFI will enable them eventually to personalize treatment selection for individual patients.
鈥淥ncology has already moved into personalized medicine where a patient鈥檚 tumour is characterized by markers and receptors,鈥 he says. 鈥淲e hope that DEMySTIFI will give us the tools to treat MS in the same way so that each patient receives the best and safest treatment for them.鈥
DEMySTIFI will include data gathered from the latest MS clinical trials.
BTK inhibitors trial
鈥淥ne of the latest exciting developments is an experimental therapy using BTK inhibitors,鈥 says Dr. Alexander Saveriano, one of the neurologists who is overseeing MS clinical trials at The Neuro. 鈥淏TK inhibitors act on the effect of B cells in the central nervous system. B cells play an important role in generating inflammation in MS.鈥
Not all B cells contribute to this inflammation, however, and certain parts of B cells might have an anti-inflammatory effect.
鈥淭here are already approved therapies that target B cells to kill them,鈥 says Dr. Saveriano. 鈥淏TK inhibitors affect B-cell functioning, preventing their activation. They also have effects on macrophages, a type of cell in the immune system that has important roles in MS. Another advantage is that BTK inhibitors are better able to enter the central nervous system as compared to larger antibodies that are also used to treat MS.鈥
The most recently approved drug in Canada for patients with relapsing-remitting MS is ofatumumab, which targets B cells.
鈥淚t鈥檚 been highly effective in clinical trials, some of which The Neuro participated in. Ofatumumab can be administered subcutaneously at home, which is an advantage over a similar effective drug, ocrelizumab, which is administered intravenously in an infusion clinic.鈥
So far, there are no approved BTK inhibitor treatments for MS. Dr. Saveriano wants to recruit MS patients willing to participate in BTK inhibitor clinical trials.
鈥淲e hope to get approximately 10 to 15 patients. Some patients will not be eligible, however, because they are already being treated effectively with an approved therapy.鈥
MS patients who would like to participate in clinical trials can get information at or by writing to Sean Carlin: sean.carlin [at] mcgill.ca