Sanfilippo ChildTeam Sanfilippo Collaborative:

Therapy for MPSIII through a research-family partnership

Rare disease, including lysosomal diseases, typically receive less attention in both basic and therapy development than common disorders. Team Sanfilippo is a research collaborative initiated by a group of family-based foundations to accelerate therapy development for Sanfilippo Syndrome. This effort is conceived as a research collaborative consisting of scientists at academic, industrial and government institutions conducting basic, translational and clinical research on MPSlll disease. The specific objective for which the families anticipate providing funding is to develop, within a three year period, a potential therapy that can be tested clinically to prevent or significantly delay the onset and progress of clinical neurological disease in MPSIII patients. The envisaged collaborative will conduct regular teleconferences to share and discuss research findings and set objectives. The parent-funders will conduct regular calls as well and the entire group will meet in person annually. Each of the scientists in the collaborative will bring unique expertise which fosters collaboration between the labrotories and enhances research efforts towards the common goal of developing treatments for MPSIII disease. The research will be focused on three areas necessary for identifying potential therapies suitable for clinical trials: High throughput screening for drug identification, animal models for drug testing and biomarker identification.

TS Logo Accomplishments to date include the identification and retention of a project manager with extensive pharamaceutical industry experience who has been successfully working on a similar collaboritive project for another Lysosomal storage disease. A scientific meeting has been schedueled for November, 2009. to review the status of research and development related to MPSIII disease. In attendance at this meeting will be more than a dozen leading scientists from around the world including the FDA and NIH, that are willing to share their knowledge and willingness to help formulate a strategic plan for achieving the objectives of Team Sanfilippo.

Understanding the drug development landscape:

Most pharmaceutical companies will not help you if you are facing a rare disease. Here are the statistics. Approximately 7,000 different rare diseases exist. According to the National Institute of Health, pharmaceutical companies have only developed treatments for 200 of the 7,000 rare diseases. Thousands of rare diseases are not being invested in by pharmaceutical companies because of small patient populations and lack of financial incentives to pharmaceutical and biotech companies.

This is why the NOH and sNCGC have started the Therapies for Rare and Neglected Diseases program (TRND). The government has to step in where pharma and bio companies will not. It takes 10 years to bring a new drug to market and costs $800+ million if a drug is not already in phase 2 or 3 for our specific disorder. It is unlikely that there will be options for us other than what already exists. It is unlikely that a drug company is going to invest over $800 million dollars for our rare disease.

Our researchers are tasked with finding near term treatments for Sanfilippo. We are not focused on creating new drugs, we do not have the time or funding to invest in this pathway. Rather, our focus is on the library of the over 3,000 approved drugs that could be used off-label and re-purposed for our needs. In addition, we will be testing over the counter supplements and create a master drug/supplement list and prioritize targets for testing.

In the case of Sanfilippo there are a lot of common symptoms and pathways shared with Alzheimer's as well as Ataxias. Research is being carried out diligently for these disorders and a treatment plan may carry over. For example, Curcumin and Vitamin D are non toxic supplements used in treating Alzheimer's and have been beneficial for other neurological disorders as well. Genestein is another example of a supplement that is being used to positively effect our children. We plan to explore these options further. With this knowledge in hand, we plan to leave no treatment option untested. We will move forward with this plan at a rapid pace.

Please join us in our reserach efforts now!!

Please visit Team Sanfilippo's website

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