Navigation Links
Promising new drug being evaluated as possible treatment option for fragile X syndrome
Date:1/7/2009

(Chicago) A pilot trial of an oral drug therapy called fenobam has shown promising initial results and could be a potential new treatment option for adult patients with Fragile X syndrome (FXS). Findings of the open label, single-dose study by researchers at Rush University Medical Center and the University of California, Davis, Medical Center are to be published in the upcoming January issue of the Journal of Medical Genetics.

Results of an initial evaluation of the safety of fenobam, which is an mGluR5 antagonist, in adult males and females with Fragile X syndrome showed there were no adverse side effects from the medication.

"This is the first study assessing the safety and pharmokinetic metabolism of an mGluR5 antagonist in humans with Fragile X syndrome," said Dr. Elizabeth Berry-Kravis, pediatric neurologist at Rush and principal investigator of the study. "Also, some patients showed calmed behavior and rapid reduction in hyperactivity and anxiety, similar to effects of the drug in mouse models."

Fragile X syndrome is the most common inherited cause of mental impairment and the most common known cause of autism. Fragile X affects 1 in 4000 males and 1 in 6000 females of all races and ethnic groups (source Centers for Disease Control). About 1 in 259 women carry fragile X and could pass it to their children. About 1 in 800 men carry fragile X; their daughters will also be carriers. Symptoms of Fragile X syndrome include mental impairment such as learning disabilities, attention deficit, hyperactivity, autistic-like behaviors, and anxiety and unstable mood.

Fragile X syndrome is caused by lack of activity of the FMR1 gene, which is responsible for a protein called FMRP. Without FMRP, activation of cell pathways by a brain receptor protein called mGluR5 goes unchecked, and it has been theorized that this plays an important part in Fragile X syndrome.

To test this theory, past researchers have used laboratory mice without an active FMR1 gene, like in Fragile X syndrome, but with a reduced amount of mGluR5 protein. The mice showed an improvement in their brain structure and function, in their brains' ability to make key proteins, and in memory and body growth. This shows that the over-activation of mGluR5 is very important in Fragile X syndrome, and suggests a path for drug development to treat the syndrome.

In the current study, twelve participants recruited by Rush and the University of California, Davis received a single oral dose of 50-to-150 mg of fenobam. Prepulse inhibition (PPI) and continuous performance test (CPT) were obtained before and after dosing to explore the effects of fenobam on measures of sensory gating, attention and inhibition. In six of the 12 individuals there was a 20 percent improvement.

"Currently, there are no therapies on the market to treat cognitive deficits associated with Fragile X syndrome," said Berry-Kravis. "This pilot study has identified the potential beneficial clinical effects of fenobam, but further research is needed."


'/>"/>

Contact: Deborah Song
deb_song@rush.edu
312-942-0588
Rush University Medical Center
Source:Eurekalert

Related biology news :

1. UIC researchers find promising new targets for antibiotics
2. New book defines promising young field of adult neurogenesis
3. Sirtris unveils promising, novel SIRT1 activators for treating diseases of aging
4. Cancer and arthritis therapy may be promising treatment for diabetes
5. Unique whey protein is promising supplement for strict PKU diet
6. Promising new drug targets identified for Huntingtons disease
7. Promising new nanotechnology for spinal cord injury
8. Biodesigns Rittmann offers promising perspectives on societys energy challenge
9. UIC researchers make promising finding in severe lung disease
10. New treatment approach promising for lymphoma patients in the developing world
11. Immunotherapy in high-risk pediatric sarcomas shows promising response
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/18/2016)... , March 18, 2016 --> ... of Biometrics, ICT, Manned & Unmanned Vehicles, Physical infrastructure and ... security companies in the border security market and the continuing ... and Europe has led visiongain to ... improved success. --> defence & security companies ...
(Date:3/15/2016)... New York , March 15, 2016 ... new market report published by Transparency Market Research "Digital Door ... Trends and Forecast 2015 - 2023," the global digital door ... US$ 731.9 Mn in 2014 and is forecast to grow ... 2023. Growth of micro, small and medium enterprises (MSMEs) across ...
(Date:3/14/2016)... NXTD ) ("NXT-ID" or the "Company"), ... the airing of a new series of commercials on Time ... 21 st .  The commercials will air on Bloomberg TV, ... the Street show. --> NXTD ) ("NXT-ID" or ... market, announces the airing of a new series of commercials ...
Breaking Biology News(10 mins):
(Date:4/28/2016)... NEW YORK , April 28, 2016 /PRNewswire/ ... biotechnology acceleration company reports the Company,s CEO  was ... capital titled Accelerators Enter When VCs Fear To ... Life Science Leader magazine is an ... work for everything from emerging biotechs to Big ...
(Date:4/27/2016)... ... 2016 , ... Cambridge Semantics, the leading provider of Smart Data ... has been named to The Silicon Review’s “20 Fastest Growing Big Data Companies of ... serves the needs of end users facing some of the most complex data challenges ...
(Date:4/27/2016)... RESEARCH TRIANGLE PARK, N.C. , April 27, 2016 ... announced today that Martine Rothblatt , Ph.D., Chairman ... an overview and update on the company,s business at ... Conference. The presentation will take place on ... and can be accessed via a live webcast on ...
(Date:4/27/2016)... ... ... Global Stem Cells Group CEO Benito Novas announced that Duncan Ross, Ph.D. ... Labs in Miami. , In 2004, Ross received his Ph.D. in Immunology from the ... the suppression of graft vs. host disease (GVHD) under UM Professor Robert Levy Ph.D. ...
Breaking Biology Technology: