Re: Drug Reverses mental Retardaton in Mice

[Guest guest]

RESEARCH

Drug Reverses Mental Retardation in Mice

_tinyurl.com/5klnat_ (http://tinyurl.com/5klnat)

Newswise - UCLA researchers discovered that an FDA-approved drug reverses the

brain dysfunction inflicted by a genetic disease called tuberous sclerosis

complex (TSC). Because half of TSC patients also suffer from autism, the

findings offer new hope for addressing learning disorders due to autism. Nature

Medicine publishes the findings in its online June 22 edition.

Using a mouse model for TSC, the scientists tested rapamycin, a drug

approved by the FDA to fight tissue rejection following organ transplants.

Rapamycin

is well-known for targeting an enzyme involved in making proteins needed for

memory. The UCLA team chose it because the same enzyme is also regulated by

TSC proteins.

" This is the first study to demonstrate that the drug rapamycin can repair

learning deficits related to a genetic mutation that causes autism in humans.

The same mutation in animals produces learning disorders, which we were able

to eliminate in adult mice, " explained principal investigator Dr. Alcino

Silva, professor of neurobiology and psychiatry at the Geffen School of

Medicine at UCLA. " Our work and other recent studies suggest that some forms of

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

" These findings challenge the theory that abnormal brain development is to

blame for mental impairment in tuberous sclerosis, " added first author Dan

Ehninger, postgraduate researcher in neurobiology. " Our research shows that the

disease's learning problems are caused by reversible changes in brain

function -- not by permanent damage to the developing brain. "

TSC is a devastating genetic disorder that disrupts how the brain works,

often causing severe mental retardation. Even in mild cases, learning

disabilities and short-term memory problems are common. Half of all TSC

patients also

suffer from autism and epilepsy. The disorder strikes one in 6,000 people,

making it twice as common as Huntington's or Lou Gehrig's disease.

Silva and Ehninger studied mice bred with TSC and verified that the animals

suffered from the same severe learning difficulties as human patients. Next,

the UCLA team traced the source of the learning problems to biochemical

changes sparking abnormal function of the hippocampus, a brain structure that

plays a key role in memory.

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

useful information, " said Silva, a member of the UCLA Department of Psychology

and UCLA Brain Research Institute. " Our findings suggest that mice with the

mutation cannot distinguish between important and unimportant data. We

suspect that their brains are filled with meaningless noise that interferes

with

learning. "

" After only three days of treatment, the TSC mice learned as quickly as the

healthy mice, " said Ehninger. " The rapamycin corrected the biochemistry,

reversed the learning deficits and restored normal hippocampal function,

allowing

the mice's brains to store memories properly. "

In January, Silva presented his study at the National Institute of

Neurological Disorders and Stroke meeting, where he was approached by Dr.

Petrus de

Vries, who studies TSC patients and leads rapamycin clinical trials at the

University of Cambridge. After discussing their respective findings, the two

researchers began collaborating on a clinical trial currently taking place at

Cambridge to examine whether rapamycin can restore short-term memory in TSC

patients.

" The United States spends roughly $90 billion a year on remedial programs to

address learning disorders, " noted Silva. " Our research offers hope to

patients affected by tuberous sclerosis and to their families. The new findings

suggest that rapamycin could provide therapeutic value in treating similar

symptoms in people affected by the disorder. " '

The research was funded by National Institute of Neurological Disorders and

Stroke, Autism Speaks and Deutsche Forschungsgemeinschaft (German Research

Foundation). Silva and Ehninger's coauthors included Yu Zhou, Shilyansky

and Weidong Li of UCLA; and Sangyeul Han, Vijaya Ramesh and

Kwiatkowski of Harvard Medical School.

Source: University of California, Los Angeles (UCLA), Health Sciences

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[Guest guest]

RESEARCH

Drug Reverses Mental Retardation in Mice

_tinyurl.com/5klnat_ (http://tinyurl.com/5klnat)

Newswise - UCLA researchers discovered that an FDA-approved drug reverses the

brain dysfunction inflicted by a genetic disease called tuberous sclerosis

complex (TSC). Because half of TSC patients also suffer from autism, the

findings offer new hope for addressing learning disorders due to autism. Nature

Medicine publishes the findings in its online June 22 edition.

Using a mouse model for TSC, the scientists tested rapamycin, a drug

approved by the FDA to fight tissue rejection following organ transplants.

Rapamycin

is well-known for targeting an enzyme involved in making proteins needed for

memory. The UCLA team chose it because the same enzyme is also regulated by

TSC proteins.

" This is the first study to demonstrate that the drug rapamycin can repair

learning deficits related to a genetic mutation that causes autism in humans.

The same mutation in animals produces learning disorders, which we were able

to eliminate in adult mice, " explained principal investigator Dr. Alcino

Silva, professor of neurobiology and psychiatry at the Geffen School of

Medicine at UCLA. " Our work and other recent studies suggest that some forms of

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

" These findings challenge the theory that abnormal brain development is to

blame for mental impairment in tuberous sclerosis, " added first author Dan

Ehninger, postgraduate researcher in neurobiology. " Our research shows that the

disease's learning problems are caused by reversible changes in brain

function -- not by permanent damage to the developing brain. "

TSC is a devastating genetic disorder that disrupts how the brain works,

often causing severe mental retardation. Even in mild cases, learning

disabilities and short-term memory problems are common. Half of all TSC

patients also

suffer from autism and epilepsy. The disorder strikes one in 6,000 people,

making it twice as common as Huntington's or Lou Gehrig's disease.

Silva and Ehninger studied mice bred with TSC and verified that the animals

suffered from the same severe learning difficulties as human patients. Next,

the UCLA team traced the source of the learning problems to biochemical

changes sparking abnormal function of the hippocampus, a brain structure that

plays a key role in memory.

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

useful information, " said Silva, a member of the UCLA Department of Psychology

and UCLA Brain Research Institute. " Our findings suggest that mice with the

mutation cannot distinguish between important and unimportant data. We

suspect that their brains are filled with meaningless noise that interferes

with

learning. "

" After only three days of treatment, the TSC mice learned as quickly as the

healthy mice, " said Ehninger. " The rapamycin corrected the biochemistry,

reversed the learning deficits and restored normal hippocampal function,

allowing

the mice's brains to store memories properly. "

In January, Silva presented his study at the National Institute of

Neurological Disorders and Stroke meeting, where he was approached by Dr.

Petrus de

Vries, who studies TSC patients and leads rapamycin clinical trials at the

University of Cambridge. After discussing their respective findings, the two

researchers began collaborating on a clinical trial currently taking place at

Cambridge to examine whether rapamycin can restore short-term memory in TSC

patients.

" The United States spends roughly $90 billion a year on remedial programs to

address learning disorders, " noted Silva. " Our research offers hope to

patients affected by tuberous sclerosis and to their families. The new findings

suggest that rapamycin could provide therapeutic value in treating similar

symptoms in people affected by the disorder. " '

The research was funded by National Institute of Neurological Disorders and

Stroke, Autism Speaks and Deutsche Forschungsgemeinschaft (German Research

Foundation). Silva and Ehninger's coauthors included Yu Zhou, Shilyansky

and Weidong Li of UCLA; and Sangyeul Han, Vijaya Ramesh and

Kwiatkowski of Harvard Medical School.

Source: University of California, Los Angeles (UCLA), Health Sciences

**************Gas prices getting you down? Search AOL Autos for

fuel-efficient used cars.

(http://autos.aol.com/used?ncid=aolaut00050000000007)