080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

New kids on the block

By By Penni Crabtree, SPECIAL TO THE UNION-TRIBUNE | Sunday, June 20, 2010 at 12:01 a.m.

. . .


Founded: 2007

Employees: 4

Product: Potential treatments for mental impairment disorders such as Fragile X syndrome and autism

When Jay Lichter read in the July 2007 journal of the National Academy of Science about a Nobel laureate’s theory for reversing an inherited form of mental impairment, the local venture capitalist had an “aha moment.”

That aha resulted in Afraxis, a tiny San Diego biotech that is developing a drug for treating Fragile X syndrome, the most common inherited cause of mental retardation and the most common known cause of autism.

Nobel winner Susumu Tonegawa, a professor of neuroscience at MIT, first proposed the idea of developing drugs for the syndrome after his lab reversed symptoms of the disorder in mice.

Tonegawa found that by inhibiting an enzyme called PAK, the symptoms of Fragile X reversed dramatically, both at the cellular and behavioral levels.

Fragile X is caused by a mutation in a single gene, which causes defects in dendrites, the signaling arms of neurons. Dendrites communicate through bumps, called spines, which become too numerous and misshapen with the gene defect.

In the mice experiments, inhibiting PAK reduced the number of spines and caused them to take on more normal shapes.

Lichter, managing director at San Diego’s Avalon Ventures, contacted Tonegawa and put together a licensing deal that involved MIT and another firm that could screen potential drug compounds against the gene target.

Now Afraxis has an experimental drug that appears to have a “profound effect” in reversing the damage caused by Fragile X and schizophrenia — at least in mice, said Lichter.

And while it’s a long way from mice to men — the company hopes to begin testing the drug in humans in 2012 — the early-stage work shows tremendous potential, said Lichter, who also serves as CEO for Afraxis.

“It is the first treatment that has actually been shown to reverse the disease, not just treat symptoms,” Lichter said. “We’re talking about growing new connections in the brain in places known to control thinking and behavior — that’s the goal, and the promise, of this.”


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