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Disease Profile: Pediatric Multiple Sclerosis (MS)
Pediatric multiple sclerosis (MS) affects individuals under 16 years of age and accounts for 5% of the total MS cases. With 20,000 cases in the US, it is designated as a rare disease. MS is an autoimmune central nervous system disorder where the immune system attacks the myelin sheath or the brain and spinal cord axons directly. Axonal loss is irreversible causing neurological deficits that can lead to progressive forms of the disease. Patients with childhood-onset MS convert to secondary progression an average of 10 years later than patients with adult onset, however they are also 10 years younger (31- 40 years old) when they reach the progressive phase. The exact cause of MS is not known. Currently, six disease modifying injectables are available for treating pediatric MS. These treatments delay relapses, but do not prevent the transition of reversible stages to permanent stages. Recently, BPT Pharmaceutical has attained the orphan drug designation with the help of KGI student, Silviya Meletath, M.D. for their drug Proenzy designed to treat pediatric MS on the basis of their discovery of a new biomarker in the spinal fluid of the patients. The preliminary experiments evaluating the therapeutic value of Proenzy is very promising and indicates that oral administration or delivery of Proenzy via skin patch technologies significantly reduced axon loss and limited the progression of the disease to latter stages.
KGI's Center for Rare Disease Therapies' Mission is to make more treatments available to people with rare disease through its advocacy and education of its students and the public. KGI's unique position in research and partnerships with corporations opens up doors of opportunities for students to learn and gain experience in all the caveats of getting an orphan drug to market.
By Jennifer Woo (PPM '11) and Silviya Meletath (MBS '11)