A Multifaceted Disease Requires a Patient-Centered Response

In 1986, I began experiencing bouts of numbness, fatigue, and lack of coordination on my left side. Those symptoms launched my 13-year journey toward a diagnosis of multiple sclerosis, or MS.

That nervous system disease can cause many symptoms including blurry vision or blindness, unsteady gait, tremors, weakness, bladder and bowel issues, and problems with concentration and memory. At least 2.3 million people worldwide have MS. The National Multiple Sclerosis Society has designated March 7–13 as MS Awareness Week.

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As both a patient and a former medical scientist, I know that we need patient-centered research to help speed accurate diagnosis and provide evidence that patients and clinicians can use to decide among treatment strategies. I’ve become involved with both PCORI and the MS Society, which work closely with patients and caregivers to fund projects in this area. PCORI will soon announce major new studies funded through an MS-specific initiative.

Puzzling Symptoms

Many people with MS go through a prolonged period before diagnosis. That’s because there’s a huge variety of experiences among people with MS, and the symptoms can seem unconnected or mimic other disorders. During the years before my diagnosis, I would be extremely tired for two or three weeks at a time, several times a year. Luckily, I didn’t experience other classic MS symptoms, which typically worsen at high temperatures—I was living in Florida and loved hot tubs and saunas.

I worked as a cardiac electrophysiologist and neuroscientist, so I had an idea of what was happening to me. In my head, I kept hoping it was something else, but in my heart, I knew it was MS. My doctors definitively diagnosed MS in 1999 when an MRI finally showed multiple lesions in my brain and on my spinal cord.

Even once it’s diagnosed, MS can be hard to manage. There’s little evidence to help patients and clinicians choose among treatments.

I have been fortunate in that my symptoms have been relatively mild, and a treatment has been effective. It’s one of a dozen disease-modifying therapies that may treat symptoms, slow disease progression, and hinder the growth of new lesions on the brain or spinal cord.

Three decades after my symptoms began, I’m doing well. I still have the occasional bout of fatigue if I overwork. I use crutches when moving in crowds or over longer distances.  I am fortunate to have the relapsing-remitting form of MS.

However, some people with MS see their health go downhill quickly—they have a form of the disease called progressive MS. Even among people with relapsing-remitting MS, individuals often have to try several disease-modifying strategies before they find one that treats their symptoms with manageable side effects. And for some people, a therapy helps only for a while because their disease becomes resistant or progressive.

For all of these reasons, we desperately need research on treating, modifying, and perhaps one day curing MS. And the studies need to be patient centered to develop options that fit a patient’s illness, circumstances, and wishes.

Moving Forward With MS Research

With my professional background and many years of personal experience with MS, I am in a unique position to appreciate the value of research. After retirement, I quickly threw myself into work with the National MS Society and since its formation, with PCORI. As a volunteer for the society, I have answered phones, attended health fairs, joined support groups, and teamed with other patients to speak on Capitol Hill.

People with MS and their families guide research investments and priorities of the MS Society in several ways. For example, frustration at the dearth of treatment options for people with progressive forms of MS led to the formation of the International Progressive MS Alliance, an initiative to speed development of treatments.

Another example of patient influence on research decisions is the society’s Wellness Initiative. It finds that people with MS consistently identify three areas of interest:  diet, exercise, and depression, which is common with this disease. For each of these areas, the initiative is surveying published research and making recommendations for future studies.

The MS Society recently funded a study in Boston that will gather opinions on pediatric MS research priorities from parents of children with MS, teenagers with MS, and adults who have MS that started in childhood. Its results will help drive research priorities. The society played a major role in training doctors and clinical centers to care for children with MS by helping establish the Network of Pediatric MS Centers. Several research studies by members of this network have also started to better understand the causes of MS in children and to identify safe and effective pediatric treatments.

I’m also involved with PCORI, where I serve on the Advisory Panel on Patient Engagement and help review research proposals. I have been deeply involved in PCORI’s recently announced initiative to fund large clinical trials or observational studies focused on MS treatments. These projects should help patients and clinicians address difficult questions that arise with an MS diagnosis, such as: What are the pros and cons of the various disease-modifying therapies? When should patients switch doses or medications? What other approaches ameliorate symptoms? Can technology-enhanced rehabilitation tools improve patient experiences?

These questions to be studied grew out of a series of PCORI workshops, which I participated in, attended by patients, caregivers, clinicians, pharmaceutical companies, insurers, researchers, and others with interest and expertise in MS. The workshops were fantastic, and their result—up to $50 million available for patient-centered MS research—was terrific for people with MS.

One ongoing project comes via PCORnet, PCORI’s national patient-centered clinical research network. In the Multiple Sclerosis Patient-Powered Research Network iConquerMS, people with MS can privately provide their health information to a growing data bank. Then the network collaborates with researchers to perform patient-centered studies. The network was initiated with another PCORI award.

In California, PCORI-funded researchers and patient partners adapted a tool they had previously built to help physicians track MS symptoms, clinical tests, disease course, and treatment. Patients can now use the tool to learn about the course of their own disease, informing treatment decisions they make in collaboration with their clinicians. (Read more about this BioScreen project in Tablet Tool Harnesses Data from Many Patients for Individual Choices.)

Another funded project is developing a patient-led MS research community in central Illinois. This project is part of PCORI’s Pipeline to Proposal Awards initiative, which helps people not usually involved in research form collaborations and infrastructure that can produce meaningful study proposals.

Research is incredibly important. It has already given us several effective therapies. We hope to have more in the future—and a better sense of which ones are right for which patients. Please let PCORI know using the comment area below if you have ideas for research.