What if a relatively low-cost, repurposed drug could help treat multiple sclerosis (MS), the least treatable of all autoimmune diseases? That could be a distinct possibility now that Dr. Marcus Koch, MD, Ph.D., and Dr. Wee Yong, Ph.D., with Cummings School of Medicine at University of Calgary, discovered that the anti-malarial drug hydroxychloroquine helped to slow the severity of a disability during an 18-month study conducted at the MS clinic in Calgary.

With results recently published in the Annals of Neurology, the promise of a low-cost generic drug used to slow down or even stop the worsening of the condition raises the hopes of many patients. In fact, in Canada, about 90,000 people have MS with about 15% of that total or 13,500 with primary progressive MS. This happens to be one of the highest rates in the world, reports the university’s newsroom.

The Drug

Hydroxychloroquine is an anti-malaria medication more commonly used to manage the symptoms of rheumatoid arthritis and autoimmune conditions such as lupus. It was chosen because it is widely used in rheumatological diseases and generally well-tolerated.

The cause of MS remains unknown. It’s a disease in which the body’s immune system attacks its own tissues. It is generally long-lasting, often affecting the brain, spinal cord, and optic nerves in your eyes. It can cause problems with vision, balance, and muscle control, although the effects are different for everyone who has the disease.Subscribe to the Trialsitenews “Autoimmune Disease” ChannelNo spam – we promise

Translational Research

Prior to the study, the study co-lead, Dr. Marcus Koch, MD, Ph.D., reported that the group conducted preclinical research in the laboratory studying models of MS. During this early translational research, the group “predicted that hydroxychloroquine would reduce disability in people living with MS.”

Emphasizing the translational research capability up in Calgary, Dr. Wee Yong, Ph.D., professor in the Department of Clinical Neurosciences and a Hotchkiss Brian Institute member, elaborated that “Calgary has a vibrant bench-to-bedside MS program and the work from Dr. Koch’s trial offers further evidence which we were pleased to see.”

The Study

A single-arm Phase 2 futility trial, the investigators tracked 35 participants between November 2016 and June 2021. Each study participant had a follow-up of six months and 18 months. The results were a pleasant surprise: while the investigators expected a 40% (14 participants) worsening among the study participant walking function, that group ended up at only eight participants.

Principal Investigator POV

Dr. Marcus Koch, MD, Ph.D., study co-lead, clinician-investigator in the Department of Clinical Neurosciences, and member of the Hotchkiss Brain Institute reported:

With primary progressive MS, there is no good treatment to stop or reverse the progression of disease. The disability progressively worsens over time. Dr. Yong’s research team, with whom we closely collaborate, has been screening many generic drugs over several years and the results with hydroxychloroquine show some promise. Our trial is a preliminary success that needs further research. We hope sharing these results will help inspire that work, specifically larger scale clinical trials into the future.”

Study Funding

  • The Westman Charitable Foundation
  • Swartout family
  • MS Translational Clinical Trials Program of the Hotchkiss Brain Institute

About the University of Calgary

The University of Calgary is a global intellectual hub located in Canada’s most enterprising city. In their spirited, high-quality learning environment, students thrive in programs made rich by research, hands-on experiences, and entrepreneurial thinking. Their strategy drives them to be recognized as one of Canada’s top five research universities, engaging the communities they both serve and lead.

About the Hotchkiss Brain Institute

The Hotchkiss Brain Institute was launched in October 2004, under the leadership of Dr. Samuel Weiss with the University of Calgary and former Calgary Health Region, as key partners. The Institute was enabled by a foundational gift from Calgary’s own Hotchkiss family.

Launched in May 2015, the Brain and Mental Health research strategy provides a unifying direction for brain and mental health research at the University of Calgary. This exciting strategy, led by the Hotchkiss Brain Institute, positions University of Calgary researchers to unlock new discoveries and treatments for brain health in our community by working in interdisciplinary teams.

Lead Research/investigator

Marcus Koch, MD, Ph.D., Cummings School of Medicine at University of Calgary

Wee Yong, Ph.D. Cummings School of Medicine at University of Calgary

Call to Action: Read the entire study here.

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