For the first time, scientists are beginning to conduct clinical trials of experimental treatment of severe forms of recurrent multiple sclerosis (MS) with stem cells. The obtained results will be compared with the most effective currently available biological treatments for this disease.
A study, sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institute of Health, will compare the safety, effectiveness, and economic benefits of different therapeutic approaches.
Multiple sclerosis is an autoimmune disease in which a body’s own immune cells attack the central nervous system.
The new experimental treatment uses a mixture of four chemicals that destroy the cells of the immune system. Before starting therapy, a certain amount of his/her own (autologous) hematopoietic stem cells is taken from the patient, which after treatment is transplanted back to the patient.
These cells restore the immune system, allowing it to “reboot”, after which the updated immune cells no longer attack the central nervous system. This form of treatment is called autologous hematopoietic stem cell transplantation, or AHSCT.
“For many people with MS — a chronic, debilitating, unpredictable and currently incurable disease — daily life can be a challenge”, – said NIAID Director Anthony S. Fauci, M.D.
“AHSCT has the potential to halt the progress of relapsing MS, eliminate the need for a person to take lifelong medication, and allow the body to partially regain function. However, we need to be certain that the benefits of this form of treatment outweigh its serious risks.”
Multiple sclerosis affects more than 2.3 million people worldwide, mostly women, including more than one million people in the United States. Symptoms of the disease vary widely and may include weakness, fatigue, chronic pain, problems with the motor apparatus and speech.
The most common form of the disease is relapsing-remitting multiple sclerosis, which is characterized by phases of remission or absence of symptoms, interspersed with exacerbation of symptoms or relapses. Incomplete recovery from relapses often leads to increased disability. With age, the disease can get worse and turn into a progressive form, which may also include relapses.
The Food and Drug Administration (FDA) has approved more than a dozen medications to treat recurrent forms of MS. These drugs vary in effectiveness, safety and cost. For many people with severe forms of relapsing-remitting MS first and second-line drugs are not able to adequately control the disease.
Previous studies have suggested that AHSCT can be an effective and long-term treatment for such people, but formally it has never been directly compared with available third-line biologic drugs that are very effective but can have dramatic side effects. In addition, AHSCT also carries the risk of serious side effects.
Given these risks and benefits, the researchers intend to determine whether autologous hematopoietic stem cell transplantation is a suitable treatment option for people with severe forms of recurrent multiple sclerosis who would otherwise receive one of the best available third-line biological therapies.
The new study is called BEAT-MS (BEst Available Therapy versus autologous hematopoietic stem cell transplant for Multiple Sclerosis). Head of the work is Jeffrey A. Cohen, M.D., a professor of neurology at the Cleveland Clinic Lerner College of Medicine and the director of the Experimental Therapeutics Program in the Mellen Center for Multiple Sclerosis Treatment and Research at the Cleveland Clinic.
BEAT-MS will be attended by 156 adult patients aged 18 to 55 from 19 hospitals in the United States and the United Kingdom. Participants will be randomly selected to receive AHSCT or one of the best high-efficacy biologic drugs. Then they will be observed by specialists for 6 years. Neurologists who will conduct periodic examinations of patients and assess their level of disabilities will not know what type of treatment they were prescribed.
The main indicator that researchers will study is the amount of time that has elapsed since the treatment was assigned to the participant and the patient’s relapse or death for any reason, if any, during the first three years of the observation period.
The researchers are also studying the mechanisms of action of both treatment strategies and will compare the restored immune system of participants receiving AHSCT with the immunological characteristics of participants who receive the best available biological medicines. In addition, researchers will compare the impact of two treatment strategies on other indicators: the activity and severity of the course of the disease, cost-effectiveness in terms of health care costs and individual performance, and the quality of life of participants.
“We hope that BEAT-MS will clarify the best way to treat people with relapsing MS”, – said Dr. Cohen.