Sunday, May 13, 2007

Dear Fragile X Friends and Families,

Doris Buffett, president and founder of the Sunshine Lady Foundation, has donated $500,000 to FRAXA and challenged FRAXA to raise an additional $500,000 in new money by November 1, 2007. If we succeed in raising $500,000, Ms. Buffett’s foundation will match that, netting FRAXA a total of $1.5 million in new money for research!

It will be a race to the finish line, but with your help, FRAXA will have an additional $1.5 million that will go directly to research aimed at finding effective treatments and a cure for Fragile X, the leading inherited cause of mental impairment and autism.

The outlook for those affected by Fragile X is brighter than it has ever been. FRAXA’s research has identified the pathways in the brain affected by the loss of FMRP, the Fragile X protein. Our research teams have tested specific compounds in animal models of Fragile X, and the results have been remarkable. We now have several therapeutic strategies that have been proven effective in preclinical testing.

We have finally moved from identifying the problem to demonstrating that we can fix it.

In 2000, Dr. Kim Huber, a FRAXA Postdoctoral Fellow in the Brown University laboratory of Dr. Mark Bear, discovered that in Fragile X mice brains there is too much of a good thing – an excessive form of learning called Long Term Depression (LTD). Remarkably, this LTD is regulated by one class of receptor: mGluRs. Drugs (called mGluR5 antagonists) are being developed which can specifically block these receptors.

FRAXA has devoted much of its resources in the past six years to arrive at this breakthrough. We are now on the verge of the first specific treatment for Fragile X – a treatment likely to be effective for a wide range of autism spectrum disorders as well.

As we move into human trials, everything gets much more expensive. Our goal is close; the financial requirements are huge. We are working together with outstanding small pharmaceutical companies, but their resources are also limited.

Money is required every step of the way, and now we can double the dollars we raise!

We are counting on you to help deliver these treatments and a cure. Remember, every new dollar raised will be doubled by Doris Buffett’s Sunshine Lady Foundation, and our vital research will be accelerated!

Please help us raise $500,000 by November 1, 2007. Send your check in the enclosed envelope, donate online at, or call us to get a supply of envelopes and letters for your friends. Help us meet Doris Buffett’s Sunshine Lady Foundation Challenge.

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