Leukemia Drug Creates Optimism as Treatment for Multiple Sclerosis

The drug known as Alemtuzumab, the first monoclonal antibody made for use in humans, may offer new hope in treating early Multiple Sclerosis (MS), even though it was developed and approved for the treatment of chronic lymphocytic leukemia. Alemtuzumab works by seeking out and destroying certain immune cells that, when functioning normally, protect against infection. Since is believed that MS as well as other autoimmune diseases damage these cells, the result is the destruction of healthy tissue.

Initially, Cambridge University scientists tried treating patients suffering from advanced multiple sclerosis with Alemtuzumab, but they had little success. However, patients suffering from early relapsing-remitting MS who were treated with Alemtuzumab experienced significantly fewer relapses as well as a reduced the number of episodes of fatigue and physical impairments that indicate progression of the disease when compared with those patients treated with the currently approved treatment known as interferon beta-1a.

Surprisingly, three years after entry into the study, some patients who received the experimental drug suffered from less disabilities associated with MS than they were experiencing prior to the beginning of the study. This finding offers hope that the treatment may play a role in stopping the progression of the disease and preventing victims from ever reaching its crippling late stages. According to study co-author Alasdair Coles, Ph.D., “The ability of an MS drug to promote brain repair is unprecedented.” He went on to explain, “We are witnessing a drug which, if given early enough, might effectively stop the advancement of the disease and also restore lost function by promoting repair of the damaged brain tissue.”

The success of Alemtuzumab in fighting against MS does not come with out risks. Almost one in four of the patients treated with Alemtuzumab developed thyroid complications, and the disabilities of some patients worsened. In addition, 3 percent of the patients treated with the drug developed a potentially life-threatening autoimmune condition, resulting in the loss of one of the patient’s lives.

According to Genzyme Medical Director Susan Moran, M.D., the patient who passed away during the study died from an autoimmune-mediated blood condition known as idiopathic thrombocytopenic purpura (ITP). Moran said the death could have been avoided if the condition had been recognized as an adverse effect of the treatment. She stated, “Unfortunately, the patient had symptoms of ITP but did not seek medical attention prior to diagnosis because this was not recognized as an adverse event.” The five additional cases were identified and managed with treatment due to close monitoring of study patients once the risk was known.

Coles said that Phase III trials will soon begin and will determine if the benefits of Alemtuzumab outweigh the risks for MS patients. Eighty-five percent of people who are first diagnosed with MS suffer from relapsing-remitting MS according to the National MS Society. Coles noted, “The Phase II results are very exciting, but this is not ready for routine use.” He also acknowledged, “We need to know more about the long-term effectiveness and adverse effects. That is our challenge over the next few years.”

MS has no cure and affects about 400,000 people in the United States as well as almost 100,000 in Britain and millions around the world. The disease is caused by the body's immune system attacking nerve fibers in the central nervous system. Few effective treatments exist for MS. Symptoms of the disease may include depression, fatigue, cognitive problems and loss of sight and mobility.

The study was published in The New England Journal of Medicine.