Current Research into MPS III

At present there is no cure for MPS III. Various experimental methods have been used to try to replace the missing enzyme, but none so far have been of any significant long-term benefit. Bone marrow transplant has been tried on Sanfilippo patients, but with disappointing results. Because of the devastating neurological symptoms of the disorder, the challenge is to get the missing enzyme into the brain.

Present research is focusing on the use of animal models to evaluate various experimental therapies. Fortunately a number of naturally occurring animal models and others which have been created. There is a mouse and dog model for MPS IIIA, a mouse and emu model for MPS IIIB, and a goat model for MPS IIID. It is interesting to note that the Sanfilippo mouse looks quite scruffy when compared to its "normal" counterpart.

The research is focused in three main areas:

• The development of "vectors" such as herpes viruses and adenoviruses to allow the spread of enzyme throughout the brain.

• Gene therapy techniques are used to correct cells.

• Experimentation in getting the enzyme into the body through either direct injection of corrected cells into the brain using vectors and the transplantation of neural stem cells (NSC).

NSC’s are self-renewing cells which give rise to both neurons and glial cells. They have been shown to migrate widely in the brain and to differentiate into apparently normal cells. This may be a future treatment.

Further information

More information on MPS III is available from our National Office, including an MPS III syndrome booklet which can be purchased with the enclosed order form. 

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