MS Clinical Trials–By Vernon Rowe, Doug Schell, Arlene O’Shea, Liegh Kreishel, LeAnn Cannon, Shannon Cone, John Hunter
MS Clinical Trials are an extremely important part of our MS center at the RNI, one of the major MS centers in the Kansas City Region. MS clinical trials, coupled with bench research and clinical care, help give our patients potential access to cutting edge therapies in MS.
But bench research and MS clinical trials for cutting edge therapies in MS cannot occur in isolation. They must in some way be tied to the stories of people who suffer with MS, and who need MS clinical trials so more effective and targeted therapies can be approved for MS treatment. In this post I’ll share two stories of real people and the benefits each received from clinical trial participation in studies with the RNI.
The first involves the clinical trial of alemtuzumab, now FDA approved as the drug Lemtrada. A young woman seven years ago wanted to be involved in this study. Initially, the FDA would not approve alemtuzumab because many other studies, including most of those in the rest of the world, were not well controlled. Yet most MS doctors in this country don’t believe it’s ethical to enroll patients in studies with a new drug, when that drug is not compared with known therapy that works. There were many other approved therapies for Relapsing forms of MS when alemtuzimab was introduced to the FDA. So the study we participated in was controlled with one of those therapies.
We enrolled this young woman with active disease, and since receiving this alemtuzimab, she not had a relapse for three years, and has had no serious side effects with therapy. So enrolling in the clinical trial allowed her to receive a powerful and effective therapy, used in the rest of the world, years before it was formally approved by the FDA in this country.
The second story involves a patient with primary progressive MS (PPMS) enrolled in the ocrelizumab study for PPMS. Unlike relapsing forms of MS, there is no approved therapy for PPMS anywhere in the world. Many drugs approved for relapsing forms of MS have been tried in PPMS, but so far nothing has proved effective.
Many of us who deal with disease every day, however, believe it’s possible that PPMS is a different disease from RRMS, and may involve B-cell-mediated nerve cell degeneration much earlier in the MS disease process. (B-cells are a part of the immune system that help our immune system generate antibodies to viruses and other threats to our bodies.) We at the RNI we have tried anti-B-cell drugs, even given into the spinal fluid, to try to help these desperate patients when nothing else worked.
For this reason, a human anti-B-cell drug, ocrelizumab, was studied for patients with PPMS, and we were selected as a participating site. We enrolled a middle-aged male patient with PPMS, who understood the risk and potential value of participation in this trial. His willingness to participate in the study gave him early access to what turned out to be the first successful trial of a drug for PPMS.
So participating in MS clinical trials not only gives patients early access to potential new successful treatments, but it also helps many people who suffer with phases of the disease, sometimes for which there is no treatment. And this is true not only for MS, but for Alzheimer disease, migraine, seizures, stroke, and other neurologic diseases, as well as sleep disorders.
So I salute the patients who have in the hundred or so pharmaceutical trials we at the RNI have been involved in over the years. Without them and people like them, the effective therapies we have today would not exist.