A Breakthrough Drug for Retardation Syndrome

The New York Times published an article on April 29, 2010 about an experimental drug which is improving the behaviors found in retardation and autism especially Fragile X syndrome, the most common inherited cause of mental disabilities. Fragile X has mental effects ranging from mild learning disabilities to retardation such as not speaking. In addition, the physical effects include elongated faces, large jaws, big ears, and enlarged testes. It affects mostly affects boys due to one arm of the X chromosome seems nearly broken, with part hanging by a thread. Statistically, 10% to 15% of autism cases result from Fragile X syndrome which was discovered in 1991.
The drug was made by a Swiss pharmaceutical giant Novartis who spent three decades conducting research. A trial was conducted with only a few dozen patients. Dr. Mark C. Fishman, president of the Novartis Institute of Mental Health was notified of the Novartis trial results and stated the drug will not be commercially available and could fail in further trials. For ethical reasons, Novartis tested the drug only in adults but the company believes this treatment may be effective in young children who brains are more likely to respond rapidly when barriers to learning are removed. The Novaris trial which began in 2008 in Europe had the researcher measuring behaviors such as hyperactivity, repetitive motions, social withdrawal and inappropriate speech. Dr. Fishman was reluctant to make this public because he felt more experiments are needed.
The article illustrated that only five researchers in the world were working toward a cure. These numbers are low considering how many children and adults are diagnosed with some version of retardation syndrome. Due to budget limitations, many agencies are faced with eliminated programs to assist with support services and healthcare assistance. It’s an urgent cause which needs immediate attention by the medical and healthcare professionals.

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