A Drug Offers Hope For Autism And Retardation
3 CommentsBy Ed Silverman // April 30th, 2010 // 7:39 am
Here’s a rare bit of good news concerning retardation and autism. A Novartis clinical trial for an experimental drug is prompting researchers to wax optimistic about coping with the fragile X syndrome, which is the most common inherited cause of these mental disaibilities. The trial revealed ’substantial improvements in behaviors associated with the syndrome,’ The New York Times writes.
“We have been reluctant to make this public because we still need to do more experiments, do them correctly and in a bigger way. But our group feels pretty good about the data,” Marc Fishman, who heads Novartis Institutes for BioMedical Research, tells the paper. There are caveats - the trial involved only a few dozen patients, only some of whom benefited. And a drug is still years away.
One child in five thousand is born with fragile X syndrome, with mental effects ranging from mild learning disabilities to retardation. Patients seem to experience an overload of unchecked synaptic noise, the Times explains, noting that the Novartis drug is intended to lower the ‘volume’ so memory formation and high-level thinking can take place, which would allow children to develop normally. Between 10 percent and 15 percent of autism cases result from fragile X syndrome.
The Novartis trial is causing excitement because that it was a difficult test of the drug’s effects. For ethical reasons, Novartis tested the drug only in adults, but the drugmaker and outside researchers believe that such compounds may prove most effective in young children, whose brains are far more likely to respond rapidly when barriers to learning are removed, the Times writes.
This is what we have been working for and hoping for since our son was diagnosed with fragile X 17 years ago,” Katie Clapp, president and co-founder of the Fraxa Research Foundation, a nonprofit organization dedicated to financing fragile X research, tells the paper. “This may be the key to solving the mystery of autism and other developmental disorders.”
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David
I read this news with great interest but was disappointed to hear nothing about its pharmacological effects.
K. Fon R.Ph.
Need more details. What is the name of the drug and what is the side effect profile, risks and benefits.
Andrew
Cellceutix Corporation is also developing a compound for the treatment of autism at its core. They have posted data on initial studies conducted on rat pups and are continuing to move forward with the development of KM-391. You can view the data on the Cellceutix website at http://www.cellceutix.com.
There is hope!