Grants for Rare Disease Research

Representative Barbara Lee form California introduced the bill “Neuromyelitis Optica (NMO) Consortium Act” (H.R. 660) to provide grants to do research on the causes, risk factors, and biomarkers associated with NMO. So far, there has not been a comprehensive study that analyzes all the relevant clinical, biological, and epidemiological aspects of NMO that can be used to identify potential risk factors and biomarkers for NMO

NMO an uncommon disease syndrome can attack the central nervous system This devastating neurologic disease can lead to blindness and paralysis and it is estimated there are 11,000 patients with NMO in the U.S. Women are affected 7 to 9 times more than men and a large proportion of NMO patients are African-American.

NMO is a central nervous system disorder causing primarily swelling and inflammation of the eye nerves and the spinal cord. NMO occurs when the body’s immune system reacts against its own cells in the central nervous system that occurs mainly in the optic nerves and spinal cord, but sometimes in the brain

NMO was once thought of as a variant of MS and is still sometimes misdiagnosed as MS. Yet it differs from MS, since NMO does not often involve the brain, severe attacks are more robust as compared to MS, and the pathophysiology differs from MS.

According to NIH’s National Institute of Neurological Disorders and Stroke (NINDS), there is no cure for NMO, but there are therapies to treat an attack while it is happening to reduce symptoms and to prevent relapses. Doctors usually treat an initial attack of NMO with a combination of a corticosteroid drug to stop the attack and an immunosuppressive drug to prevent subsequent attacks.

Representative Lee’s bill would enable the Director of NIH in collaboration with the Director of the National Institute on Minority Health and Health Disparities to coordinate the efforts to establish a National Neuromyelitis Optica Consortium.

The legislation would enable the Consortium to provide grants for not fewer than five years so that the Consortium would be able to conduct research on a minimum of 25 individuals diagnosed with NMO.

The Consortium would be responsible to design a common study, develop standard protocols, methods, procedures, and assays to use with individuals enrolled as study participants, develop specific analytical methods for examining data, and provide provisions to review enrolled cases. Another objective would be to designate a central laboratory to collect, analyze, and aggregate data with respect to the research and then make this data and analysis available to researchers.

 Go to www.ninds.nih.gov/disorders/neuromyelitis_optica/neuromyelitis_optica.htm for more information on NMO.