This week is Multiple Sclerosis Awareness Week, a time to learn about this often disabling neurological condition—and work to improve the lives of people with multiple sclerosis (MS). This condition affects about 400,000 people in the United States. People with MS can develop a wide variety of symptoms, including vision problems, fatigue, pain, and loss of coordination. The disease is diagnosed by a thorough examination that may use magnetic resonance imaging (MRI) to detect damage, or lesions, in the brain and spinal cord.
The majority of people newly diagnosed with MS go through periods of relative health followed by episodes of new or worsening symptoms, referred to as relapses. Since 1993, disease-modifying therapies have been available that can lessen the frequency and severity of relapses and prevent new MRI lesions. The Food and Drug Administration has approved 14 drugs to treat the symptoms and slow the course of this relapsing-remitting form of MS.
A PCORI-funded study is now testing whether some patients, after years of successful treatment, could safely stop taking those drugs.
Why is this an important issue? The average annual wholesale cost of the drugs is $90,000. Some of the treatments are taken orally, but others require injections. Most have side effects, some of which can be serious. “I give myself a shot three times a week. I don’t want to do that for the rest of my life,” says Robin Beland, MBA, a vice president of sales at a technology company, who was diagnosed with MS around 20 years ago.
Answering a Common Question from Patients
Her neurologist, John Corboy, MD, of the University of Colorado, Denver, says patients constantly ask, “‘Hey doc, how long do I need to take this?’ We really have very little data to answer a very common question,” Corboy says.
So, he and a team of both researchers and other members of the healthcare community have launched a project to begin to answer that question. The study will recruit 300 patients with MS at 15 centers across the country. The patients in the study have been continuously taking MS medications for at least five years with no relapses or new lesions. Half of the participants will be randomly assigned to stop taking their medicines, and the other half will continue.
The research team will closely monitor the patients for two years, performing MRI scans and tracking symptoms, to see how the patients who continue taking the disease-modifying therapies compare to those who halt their medicines.
Although it is important for patients to continue in their assigned group at least until they have a new relapse or MRI lesion, all participants will retain the right to stop, restart, or change their medicine should they feel that is appropriate for them.
I give myself a shot three times a week. I don’t want to do that for the rest of my life.
The study is enrolling people age 55 and older. “As patients age, relapses generally happen less often, and there’s a reduction in new lesions,” Corboy says. “And most importantly, studies have shown that as people age, the medicines tend to be less effective.”
It’s possible, Corboy says, that Beland, who is 55, and others are getting little or no benefit from their medications, while still experiencing side effects.
Weighing the Risks of Stopping Medication
Beland plans to sign up for the study. “I do MRIs yearly, and I don’t have any new lesions. I don’t know if that’s because of the drug or not,” she says. She’s willing to risk having a relapse if the study randomly assigns her to the group of the patients who stop taking medicines. “If I go off the medicine and something happens, then I’ll go back on,” she says.
PCORI has funded seven patient-centered comparative clinical effectiveness research studies, totaling $26 million, about multiple sclerosis (MS).
Two of those studies compare the pros and cons of drugs to reduce MS attacks or slow the disease’s progression. Another assesses whether patients with MS in rural and low-income areas benefit as much from rehabilitation therapies provided via telehealth as they do from clinic-delivered treatments.
For more information, see our fact sheet and a list of projects related to MS. You can also learn more about other PCORI-funded projects on MS in previous blog posts, When a Clinician Becomes a Patient and A Multifaceted Disease Requires a Patient-Centered Response. Learn more about the former post’s guest author, Stephanie Buxhoeveden, here.
Corboy’s team is getting ready to enroll patients at 15 study sites, three of which are supported by funding from the National Multiple Sclerosis Society. To gauge the likelihood of success in recruiting participants, the team did a preliminary survey asking patients whether they’d consider joining such a study.
“Some people are scared about going off medication,” Corboy says. “They say, ‘If it ain’t broke, don’t fix it.’ They won’t want to join our study, and we understand their concerns. But everybody is interested in the study’s results.”
If the research finds no downside to stopping medication, Corboy thinks a larger study should take place, perhaps homing in on the best age to stop taking the medications. If, however, going off disease-modifying drugs carries significant risks, Corboy says, “that would reassure physicians and patients that it’s worthwhile to continue to use these medications in spite of the costs and hassle and side effects.”
There could, of course, be a gray area in the results, where patients face a slight risk of relapse or lesion if they stop treatments. Each patient and doctor would then need to weigh the options together.
Patients and other stakeholders have been involved from the beginning of the study through two stakeholder advisory committees based at the University of Colorado and the Cleveland Clinic, one of the study sites. The advisory committees worked intensively on the language in questionnaires to ask patients about how satisfied they are with their treatment or lack thereof.
“Ultimately, patient satisfaction is the bottom line,” Corboy says.