It’s an exciting time to be working in clinical trials at the UNM Health Sciences Center Multiple Sclerosis Specialty Clinic. We’re moving ever closer to more impactful treatments that show real promise for our patients, who make meaningful contributions to science by participating in research studies.
It all adds up to something very important: Hope for patients with MS.
A chronic and often debilitating condition, multiple sclerosis is a neuroimmune condition that develops when the immune system damages the brain, spinal cord, and optic nerve. Inflammation causes lesions that damage the myelin sheath that surrounds important nerve cells. Symptoms can include fatigue, balance and coordination problems, difficulty with vision and speaking, and tremors.
In the U.S., nearly one million people are living with multiple sclerosis, and almost 200 people are newly diagnosed each week – about four times as many women as men. From 1994 to 2017, the number of people with MS in the U.S. has quadrupled.
There are three primary types of MS:
- Relapsing-remitting multiple sclerosis (RRMS): About 85% of patients with multiple sclerosis are initially diagnosed with RRMS, in which symptoms can come and go. There are several effective disease-modifying treatments for RRMS on the market.
- Secondary progressive multiple sclerosis (SPMS): Patients with RRMS can progress to this stage, in which symptoms slowly worsen despite not developing new brain lesions that can be detected with MRI imaging. There are no approved therapies for inactive SPMS.
- Primary progressive multiple sclerosis (PPMS): This type of MS begins with a steady onset of symptoms without remission. There is only one approved treatment for PPMS.
UNM Health Sciences Cetner researchers and patients are working together to develop new treatments for all stages of MS through several ongoing clinical studies.
VISTA Trial: Exploring Remyelination
We have recently completed enrollment in a particularly exciting clinical trial called VISTA, or “Study to Evaluate the Safety and Efficacy of PIPE-307 in Subjects with Relapsing-Remitting Multiple Sclerosis.” This phase 2, randomized, double-blind study evaluates whether adding a medication called PIPE-307 to current treatments improves MS symptoms better than a placebo.
In phase 1 studies, the investigational compound was found to block M1 receptors, which can lead to remyelination, the process of regrowing myelin. To measure patients’ progress, we carefully studied improvements in their low contrast visual acuity and the thickness of their optic nerve.
These visual indicators of health are uniquely interesting. The optic nerve is particularly sensitive to treatment effects, so we look for signs that the treatment is improving the structural integrity of the optic nerve.
Enrolled patients continue on their existing disease-modifying therapy (DMT) while enrolled, preventing new clinical relapses. PIPE-307 could add the ability to repair myelin damage to these treatments that are already approved to prevent relapse.
Related reading: Patient-Centric Multiple Sclerosis Care: How Our Trainees Blend Innovation & Personal Attention
BTKi Trials: An Opportunity to Slow Progression
To help advance treatment for patients with SPMS and RRMS, we’re involved in several studies of a class of drugs called bruton tyrosine kinase inhibitors (BTKi). This new class of DMT targets immune cells that are involved in MS.
BTKi’s unique mechanism of action makes it particularly interesting. B cells (in the peripheral lymphoid tissues like the spleen and lymph nodes) and microglia (in the central nervous system) are immune cells with BTK enzymes that drive inflammation and cause myelin damage. BTKi medications stop this process.
We suspect that because BTKi work on the microglia in the central nervous system, these medications could help slow or halt the progression of MS.
Studies we have or are participating in, including GEMINI, FENhance and FUSION, among others, are providing our patients with hope—and giving participants access to leading treatments before they’re available to the public.
Related reading: Neuroimmunology Fellowship Charts the Leading Edge of Multiple Sclerosis Care
[h3] FUSION Trial
In this trial, we’re studying a combination of DMTs to learn whether taking them both will create a synergistic effect in the immune system, providing more relief for patients than either drug could achieve on its own.
In Part 1, one set of participants will take either investigational BTKi or diroximel fumarate (DRF). In Part 2, a different set of participants will take either a combination of investigational BTKi and DRF or DRF alone.
Researchers will assess relapse rates, physical examination and MRI scans to learn how these treatments affect patients’ health, especially new areas of inflammation in the brain. To enroll, patients must have a diagnosis of RRMS. If you’re interested in taking part in this exciting trial, call 505-925-4124 for more information.
Related reading: Patients First: Our Team Approach to Multiple Sclerosis Treatment
Practice in an MS Clinical Trials Hub
UNM HSC is excited to participate in clinical trials. We are at the precipice of advancements that can make a real difference in patients’ quality of life. At any given time, we are participating in several distinct clinical trials for MS. By the time of this publication, for example, we are enrolling in newer clinical trials for MS not mentioned here. It is truly a rapidly evolving landscape.
New drugs such as remyelinating agents, the BTKi class of drugs, and emerging monoclonal antibodies offer hope for a distinct mechanism of action that could give patients with all types of MS, from RRMS, inactive SPMS and PPMS hope. For patients with this chronic and progressive disease, hope is a very important part of health.
But we couldn’t make any advances without patients who agree to participate in research. I call them heroes, because it takes bravery to put yourself at the forefront of science for the benefit of others. Patients experience many benefits to participating in research, including:
- Access to leading treatments before they’re available to the public.
- Very close monitoring of their health. Trial participants receive more careful monitoring, including frequent blood tests and MRI, and more physical exams than in a standard clinical setting.
- All services provided during a clinical trial are at no cost to the patient.
- In trials for RRMS, patients are either on the investigational drug or an existing FDA- approved disease-modifying therapy. There is typically no placebo arm.
Our patient population is very diverse. Diversity in clinical trials is paramount because it helps ensure that DMTs are safe and effective for people of all backgrounds. We have the unique opportunity to improve our understanding of MS through providing access to MS research.
Our understanding of the pathophysiology and treatment of MS is always evolving, so participating in research that keeps us at the cutting edge is important. Through active participation in clinical trials, I am able to understand the mechanism of action of these emerging drugs and see how they directly impact patients. This helps me provide a more comprehensive approach to clinical care.
For trainees, there are many benefits of learning in a center where advancing science through research is paramount. Our trainees are exposed to the latest advances while developing research expertise, sharpening their critical thinking skills, learning to apply evidence-based practice, and improving their competitiveness for future fellowships and academic positions. Most importantly, they participate in our culture of continuous learning and innovation, which can serve trainees well throughout their practice.
Interested in Joining an MS Clinical Trial?
If you have been diagnosed with MS and want to join a clinical trial, call 505-925-4124 for more information and to see if you may be eligible.