Drug may repair disease that causes autism

[cliaz]

June 23, 2008 - 12:48pm

Evan Haning, WTOP Radio

Stacy Wells, Special to WTOPnews.com

 

 

WASHINGTON - An FDA-approved drug may be the key to correcting the brain

dysfunction that causes autism.

 

 

Researchers at UCLA discovered that rapamycin reverses the effects on the

brain caused by a genetic disease called tuberous sclerosis complex, or

TSC. Half of all TSC patients suffer from autism and epilepsy.

 

 

TSC disrupts how the brain works and often causes mental retardation. The

disease strikes one in 6,000 people and causes learning disabilities and

short-term memory problems even in mild cases. Rapamycin, a drug approved

to fight tissue rejection following organ transplants, was given to mice

with TSC because it targets an enzyme involved in making proteins needed

for memory.

 

 

"Memory is as much about discarding trivial details as it is about storing

useful information," said Dr. Alcino Silva, principle investigator and

professor of neurobiology and psychiatry at the David Geffen School of

Medicine at UCLA.

 

 

Silva suspected that the brains of the mice were filled with meaningless

noise that interfered with learning, keeping them from distinguishing

between important and unimportant information. Silva and the others traced

the learning problems of the mice to biochemical changes sparking abnormal

function of the hippocampus, the part of the brain that regulates memory.

 

 

After only three days of treatment, the TSC mice were learning as quickly

as the healthy mice.

 

 

"The rapamycin corrected the biochemistry, reversed the learning deficits

and restored normal hippocampal function, allowing the mice's brains to

store memories properly," said Dan Ehninger, a postgraduate neurobiology

researcher.

 

 

Silva is currently collaborating with with doctors studying TSC at the

University of Cambridge to conduct human trials of rapamycin.

 

 

"Our work and other recent studies suggest that some forms of mental

retardation can be reversed, even in the adult brain," said Silva.

 

 

(Copyright 2008 by WTOP. All Rights Reserved.)

 

 

Evan Haning, WTOP Radio

Stacy Wells, Special to WTOPnews.com

 

 

WASHINGTON - An FDA-approved drug may be the key to correcting the brain

dysfunction that causes autism.

 

 

Researchers at UCLA discovered that rapamycin reverses the effects on the

brain caused by a genetic disease called tuberous sclerosis complex, or

TSC. Half of all TSC patients suffer from autism and epilepsy.

 

 

TSC disrupts how the brain works and often causes mental retardation. The

disease strikes one in 6,000 people and causes learning disabilities and

short-term memory problems even in mild cases. Rapamycin, a drug approved

to fight tissue rejection following organ transplants, was given to mice

with TSC because it targets an enzyme involved in making proteins needed

for memory.

 

 

"Memory is as much about discarding trivial details as it is about storing

useful information," said Dr. Alcino Silva, principle investigator and

professor of neurobiology and psychiatry at the David Geffen School of

Medicine at UCLA.

 

 

Silva suspected that the brains of the mice were filled with meaningless

noise that interfered with learning, keeping them from distinguishing

between important and unimportant information. Silva and the others traced

the learning problems of the mice to biochemical changes sparking abnormal

function of the hippocampus, the part of the brain that regulates memory.

 

 

After only three days of treatment, the TSC mice were learning as quickly

as the healthy mice.

 

 

"The rapamycin corrected the biochemistry, reversed the learning deficits

and restored normal hippocampal function, allowing the mice's brains to

store memories properly," said Dan Ehninger, a postgraduate neurobiology

researcher.

 

 

Silva is currently collaborating with with doctors studying TSC at the

University of Cambridge to conduct human trials of rapamycin.

 

 

"Our work and other recent studies suggest that some forms of mental

retardation can be reversed, even in the adult brain," said Silva.

 

 

(Copyright 2008 by WTOP. All Rights Reserved.)

related story tags

 

 

Alcino Silva Biochemistry Cognitive Science Dan Ehninger Life Sciences

Sciences Science and Technology University of Cambridge

[Thews40]

I was shocked to hear that one in 150 has some form of autism. Even if this wonder drug only helps a small percentage, it could pave the way for the next wonder drug.