A cancer drug previously used to fight leukemia significantly reduces relapses in people with multiple sclerosis and could be a “substantial advancement” in treatment of the disease, according to British researchers. But there is also concern that the company that manufactures the drug may increase its price.
In two phase III clinical trials published in The Lancet, researchers at the University of Cambridge found that MS patients who took the drug alemtuzumab had nearly half the relapses as patients who receive standard MS treatment. Nearly two out of three remained relapse free, compared to 47% of those getting standard treatments. Alemtuzumab was also found to reduce the risk of disability and brain shrinkage.
“Our research shows the transformative effect that alemtuzumab can have for people with MS,” said Alasdair Coles, principal investigator and professor at the University of Cambridge. “Patients who continue to show disease activity while on their initial therapy are especially difficult to treat. Now, we have shown that alemtuzumab works where first-line drugs have already failed. It not only reduces the chances of disability associated with MS, but may even result in long-term clinical improvements.”
In one study, researchers looked at 334 patients with active, early relapsing MS who were not previously treated. The patients received either alemtuzumab or interferon beta-1a treatments and were monitored for two years. They found that alemtuzumab reduced MS relapses 55% over the standard treatment.
The second study followed 840 MS patients who had previously been treated but relapsed during therapy. They had check-ups every three months for two years and yearly brain scans. The relapse rates were 35% for patients who were given alemtuzumab and 51% for those who received interferon beta 1a.
The brain scans showed that alemtuzumab not only reduced the number of new lesions, but also reduced the rate of brain shrinkage that occurs in MS when tissue is damaged.
In both trials, alemtuzumab appeared to increase the risk of certain autoimmune disorders, particularly those affecting the thyroid. The researchers noted those side effects are potentially serious but can be treated. The studies were sponsored by Genzyme, a Sanofi company (NYSE: SNY), and Bayer Schering Pharma.
In an editorial also appearing in The Lancet, the authors noted that the drug has been withdrawn from the U.S. and European markets by Genzyme, which has submitted applications to the U.S. Food and Drug Administration and the European Medicines Agency to have alemtuzumab approved for treatment of multiple sclerosis.
“There is concern that with a license for multiple sclerosis, the cost of alemtuzumab could rise and might become too expensive for many patients and health systems,” they wrote. “Finding promising treatments such as alemtuzumab is important. But so is keeping alemtuzumab accessible and affordable if its early success in these trials proves to be of enduring value.”
Alemtuzumab has been used off-label to treat MS for years. MS is a chronic autoimmune disease that attacks the body’s central nervous system and destroys the myelin sheath that protects the nerve cells. That breakdown stops the nerves from communicating with each other, eventually leading to progressive physical and cognitive disability.
An estimated 400,000 Americans have the disease and more than 2 million worldwide. There is no cure.