Multiple sclerosis (MS) is a disease of the central nervous system in which the immune system attacks the brain and spinal cord, typically resulting in muscle weakness, problems with vision and coordination, pain, and, in some patients, cognitive impairments. The disorder affects approximately 400,000 people in the U.S. and more than 2.5 million people worldwide. Patients with relapsing forms of MS are currently treated with one of three FDA-approved interferon-beta agents or with glatiramer acetate. Unfortunately, each of these treatments is not effective in a substantial number of patients. Therefore, there is an urgent need to develop new and more effective treatments, especially for those MS patients who fail to respond or respond only partially to standard immunotherapy. 

A team from the National Institute of Neurological Disorders and Stroke (NINDS) discovered that daclizumab, a humanized antibody to the interleukin-2 receptor alpha chain (IL-2Rα) approved in the U.S. for preventing organ transplant rejection, is effective in treating MS. They led small clinical trial of MS patients who did not respond to interferon-beta alone and found that adding daclizumab improved patient outcome. Patients who received the combined therapy had a 78-percent reduction in new brain lesions and a 70-percent reduction in total lesions, along with other significant clinical improvements. The daclizumab was also very well tolerated. Based on this trial, the NINDS team anticipated that daclizumab and other anti-IL-2Rα antibodies would be useful either as combination therapy or stand-alone treatment in MS, and patent applications disclosing these findings were filed by the National Institutes of Health (NIH). 

The technology is exclusively licensed to Abbott (formerly Facet Biotech/PDL) which, in collaboration with Biogen Idec, has initiated and is currently enrolling patients for a Phase III study. The licensee has recently concluded a study that enrolled 230 patients with MS, which confirmed that using daclizumab as an addon therapy helped patients whose symptoms had relapsed while they were taking interferon-beta. Several other small-scale clinical trials at NIH have led to the conclusion that daclizumab monotherapy is effective in most patients who experienced persistent MS disease activity with interferon-beta therapy. While the team helped the transfer of this technology from bench to bedside by conducting clinical trials and disseminating the results of their findings, the technology transfer professionals at NIH helped transfer this valuable technology to biopharmaceutical companies to ensure the development of Food and Drug Administration (FDA)-approved therapies that can help treat MS worldwide. 

Awardees

National Institute of Neurological Disorders and Stroke

• Dr. Bibiana Bielekova 
• Dr. Roland Martin
• Dr. Henry McFarland 
• Dr. Melissa Maderia 
• Laurie Arrants 
• Dr. Charlotte McGuinness

National Cancer Institute 

• Dr. Thomas Waldmann 
• Thomas Close 

NIH Office of Technology Transfer 

• Dr. Surekha Vathyam 
• Mojdeh Bahar 
• Richard Rodriguez