NIH Conference Review 2002 The conference, "The Mucopolysaccharidoses: Therapeutic Strategies for the Central Nervous System was held September 24-25, 2002 in Bethesda, MD. This conference was the result of almost 2 years of planning between the NIH (National Institutes of Health) and The National MPS Society. We are very grateful to the commitment and financial support of the NIH, without whom this meeting would not have been a reality. Members of our Scientific Advisory Board, Dr. Elizabeth Neufeld, Dr. Mark Haskins, and Dr. Edward Schuchman pledged their time and expertise to identifying scientists whose work focuses on the central nervous system. This is the first time a conference has been devoted to the central nervous system and attracted many new researchers. The goal was to promote MPS research in this area and expand the number scientists involved. Dr. Danilo Tagle, Program Director, Neurogenetics National Institute of Neurological Disorders and Stroke introduced the workshop goals: identify problems delivering therapeutics across the blood-brain barrier, determine strategies and delivery methods with the greatest potential for overcoming these problems, determine how animal models can facilitate therapy development, determine the efficacy and outcome measures of treatment and facilitate the exchange of ideas and promote collaboration. Dr. Joseph Muenzer presented the clinical manifestations of MPS and the current status of therapies, followed by Dr. Mark Haskins' overview of the animal models of MPS. Presentations on the pathology of the brain were presented by Drs. Arthur Lander, Steve Walkley and Elizabeth Neufeld. Dr. Lester Drewes reported on the blood-brain barrier from a molecular perspective. Talks on methods to cross the blood-brain barrier were given by Drs. William Pardridge and Todd Zankel. Gene transfer approaches to getting enzyme into the brain were reported by Drs. Mark Sands, John Wolfe, Gianvito Martino and Beverly Davidson. Presentations on the use of stem cells as therapeutic agents were given by Drs. Eva Mezey, Edward Schuchman, Greg Stewart, and Joanne Kurtzberg. The final session was on imaging to follow the course of disease and progress of therapy, given by Drs. Afonso Silva, William Ball and Harley Kornblum, The final afternoon session was devoted to recommendations, future research priorities, potential collaborations and funding strategies. There was agreement that much more needs to be learned about stem cells before they can become a therapeutic agent. It is likely that multiple approaches or treatments will be needed, to treat both CNS and physical problems. The idea of multi-center collaboration was discussed along with the need to increase awareness to the medical profession about MPS conditions. The attendees were enthusiastic in their praise of the conference, the focus on CNS, the informal discussions and the potential for future collaboration. The National MPS Society hosted an evening reception for the conference attendees. Barbara Wedehase presented a brief history of The National MPS Society, its accomplishments and goals. Les Sheaffer followed with a talk "Science, Government and Parents - Building the Future". The National MPS Society is very grateful to the support of BioMarin Pharmaceutical, Inc, Genyzme General Therapeutics, and Transkaryotic Therapies, Inc. for their financial support of the two conference lunches and of the refreshments served during the conference poster session.