- An off-label drug may be an effective alternative to approved treatments for multiple sclerosis (MS).
- Federal approval is unlikely, but off-label use will probably remain a viable option.
- The cost of MS treatment is generally high and this drug could be more affordable.
It’s understandable to have a lot on your mind when you’re diagnosed with multiple sclerosis (MS).
The cost of treatment shouldn’t be one of those things. However, drugs used to treat MS are notoriously expensive.
According to new research from Sweden, pricing relief might be found by treating MS with an off-label drug called rituximab.
Researchers add that rituximab might even be more effective than some MS treatments already approved by the Food and Drug Administration (FDA).
There are four types of MS with the most common being relapsing-remitting MS (RRMS). It’s characterized by alternating periods of flare-ups and recovery.
Dr. Barbara Giesser, a neurologist and MS specialist at Pacific Neuroscience Institute at Providence Saint John’s Health Center in California, told Healthline, “On average, most persons with RRMS who are not being treated with disease-modifying therapies may have one to three relapses per year.”
“A relapse may last up to several weeks or occasionally months if it’s not treated,” she added.
Those extended reactions are considered to be clinical relapses with symptoms you can detect, according to Dr. Nancy L. Sicotte, chair of the Department of Neurology and director of the Multiple Sclerosis and Neuroimmunology Program, and professor of Neurology at Cedars-Sinai in Los Angeles, told Healthline.
“We think of relapses as the ‘tip of the iceberg’ because we know from imaging that the disease is active even if we don’t detect clinical symptoms,” Sicotte told Healthline.
While RRMS tends to manifest in peaks and valleys, it can become progressive. Experts believe that the number of relapses is associated with this change.
So, in addition to treating what can be debilitating symptoms of RRMS, preventing relapses might also slow the disease’s progression.
There’s no shortage of options for treating RRMS.
“There are currently almost two dozen disease-modifying therapies that are approved for use in the U.S. that are indicated for treatment of RRMS,” said Giesser.
One of these drugs is dimethyl fumarate.
What’s not on the list of FDA-approved RRMS treatments? Rituximab.
Researchers wanted to know how well rituximab prevented relapses compared to dimethyl fumarate. Their study included 195 people who had been newly diagnosed with RRMS. These participants were randomly assigned to receive either rituximab or dimethyl fumarate for 24 months.
The researchers said they found rituximab to be the superior treatment. In fact, those receiving rituximab had a 5-fold lower risk of relapse than those receiving dimethyl fumarate. Further, MRI scans showed fewer MS plaques in people who took rituximab. There also wasn’t an increased risk of side effects when rituximab was used.
The researchers also noted that rituximab is more affordable.
The research was funded by the Swedish Research Council, but some of the study authors reported potential conflicts of interest with pharmaceutical companies involved with rituximab.
Nonetheless, the outside experts interviewed by Healthline agreed with the study’s conclusions.
“There are a lot of different versions of dimethyl fumarate now. They’re very expensive and it’s a very simple compound, so it defies logic that it’s so expensive,” said Sicotte.
“In some circumstances, rituximab is the preferred treatment over the FDA-approved versions because it is so much cheaper to use,” she added.
Rituximab was originally developed for lymphoma and has since been used for other autoimmune diseases.
“It was tested in RRMS 10 years ago and it was a paradigm breaker. It worked to decrease relapses effectively, and that really opened the door to a new class of medications for MS, including ocrelizumab and ofatumumab,” Sicotte said.
If rituximab is indeed more effective and less costly than some other RRMS treatments, why doesn’t it have FDA approval for RRMS?
“The cost for doing the large study required to formally prove that rituximab is effective wouldn’t be cost-effective for the pharmaceutical companies. Biosimilar generic versions of rituximab are available now, so it’s very unlikely that anyone will pursue FDA approval,” Sicotte explained.
This doesn’t mean that you can’t use rituximab to treat RRMS. It just means that coverage will vary from one insurance policy to another.
There are also other available options.
“Ocrelizumab and ofatumumab are slightly different and newer versions of rituximab. They belong to a class of medications called B cell depletors, and all have a similar mechanism of action,” said Giesser.
“Some drugs are taken orally, some are injected, some are administered intravenously (IV), and they can have a range of side effects and potential risks. So, it really is a shared decision between the person with MS and their doctor,” said Sicotte.
“These drugs are meant to be taken for the long term. You accrue the benefit by being on them for long periods of time. You want to pick a therapy that you’ll realistically be able to stick with,” Sicotte added.
One deciding factor might be which drug is the most effective at preventing relapses.
As of today, the answer to that question is still unclear. But two ongoing trials — TREAT-MS and DELIVER-MS — are seeking to determine the best approach to treating newly diagnosed RRMS.
There are still a lot of questions about how to best treat RRMS, but experts do have some advice.
“All of the evidence we’ve accumulated over the last 20 years indicates that getting on a therapy as soon as the diagnosis is made leads to the best outcomes in the future. We highly encourage anyone who thinks they have MS to seek out an evaluation,” said Sicotte.