Research published this week describes a promising start to using stem cell therapy for multiple sclerosis. In a small Phase I trial, patients who had stem cells directly injected into their brain appeared to tolerate it well and experienced no serious adverse effects. The treatment may have also stopped or slowed the progression of their symptoms, the scientists say, but larger studies will be needed to confirm its effectiveness.
Multiple sclerosis is an autoimmune condition caused by the steady deterioration of our nervous system’s myelin sheath—the protective coating around nerve cells. People with MS experience various neurological symptoms, including numbness, muscle weakness, pain, and difficulty walking. There are different forms of MS, classified by the duration and progression of their symptoms. Most people with MS have symptoms that fade and reoccur at first, for instance. But eventually, many develop secondary progressive MS, which is when symptoms stop disappearing and only get worse over time.
There are several treatments available today that can reduce the frequency of MS relapses and extend the time it takes for MS to become progressive. But even with medication, up to two-thirds of people with relapsing symptoms will develop secondary MS within 25 to 30 years of their initial diagnosis.
Lab and animal studies have suggested that stem cells—so-called building blocks that have the ability to become many different types of cell—could hold the key to stopping or even reversing the progression of MS. And we’re now starting to see the first human trials testing out the potential of stem cell therapy.
This new research, published Monday in Cell Stem Cell, is a collaboration between scientists from the University of Colorado, the University of Cambridge in the UK, and the University of Milano-Bicocca in Italy. It involved 15 patients from Italy diagnosed with secondary MS. The patients were given neural stem cells originally derived from a single donor (a fetus that died of miscarriage) via an injection directly into the brain. Then they were observed for a year’s time.
Phase I trials primarily focus on a treatment’s safety and tolerability. And the therapy did appear to pass this test, with no serious adverse events or deaths reported during the trial. Other adverse events were mild, short-lasting, and possibly related to the other medications people were taking for their MS. Importantly, patients also experienced no progression of their symptoms. And in a subset of patients, the researchers found evidence that a greater dose of stem cells was tied to a slower reduction in brain volume, possibly a sign that the treatment can reduce MS-related inflammation.
These findings are still very early, and many treatments that seem to work well in Phase I research go on to fail when tested out in more extensive trials. But for now, the researchers are cautiously optimistic about the future of stem cell therapy for MS. The team is already planning to conduct larger studies, while other scientists are working on their own trials.
“We recognize that our study has limitations—it was only a small study and there may have been confounding effects from the immunosuppressant drugs, for example—but the fact that our treatment was safe and that its effects lasted over the 12 months of the trial means that we can proceed to the next stage of clinical trials,” said study author and Cambridge researcher Stefano Pluchino in a statement from the university.