Omar Khan, M.D., professor and chair of Neurology and director of the Multiple Sclerosis Center for the Wayne State University School of Medicine, published national guidelines on conducting research in a rare and potentially devastating neurologic disorder known as Neuromyelitis Optica or Devic's Disease.
Dr. Khan, a member of the International Clinical Consortium and the Neuromyelitis Optica Clinical Council organized by the Guthy Jackson Charitable Foundation, published "Challenges and Opportunities in Designing Clinical Trials for Neuromyelitis Optica" in Neurology, the official journal of the American Academy of Neurology.
Neuromyelitis Optica, or NMO, is rare disorder that can be fatal, Dr. Khan said. The condition has a predilection for optic nerves and the spinal cord. In 2004, a specific biomarker, anti-aquaporin-4 IgG, or AQP4-IgG, was developed and is now accepted worldwide. Detection of AQP4-IgG facilitates NMO diagnosis, however, an important proportion of cases is seronegative. "Acute NMO attacks, while variable, are generally more severe than those of multiple sclerosis and have higher mortality rates," Dr. Khan said. "Historically, reported NMO mortality rates range between 10 percent and 30 percent five years after diagnosis. Thus, minimizing the frequency and consequences of attacks are primary therapeutic goals."
The national guidelines published also noted that no drug has been proved to be safe and effective in NMO in randomized, controlled studies, and none has received regulatory approval. Opinions among investigators vary widely regarding ethics of placebo-controlled studies for maintenance treatment of NMO.
"As a result of these guidelines, which included input from internationally recognized experts in the field as well as from regulatory authorities, we have developed ethical and rational clinical trial design that will promote the development of efficacious and safe therapies in this rare but devastating disorder," Dr. Khan said.
Since the guidelines were developed, three large international randomized controlled trials are under way. "We are now able to investigate whether depleting B cells by targeting CD19 or interleukin-6 receptors or complement pathways with novel monoclonal antibodies will lead to dramatic reduction or even elimination of NMO disease activity. This is remarkable progress in NMO research and Wayne State University is leading cutting-edge translational research," Dr. Khan said.
The Wayne State University Multiple Sclerosis Center is one of the largest MS centers in North America, with nearly 5,000 patients. The center also conducts research in other neuroimmunologic disorders, including Neuromyeltis Optica.