Navigation Links
Drug fights cystic fibrosis
Date:2/5/2008

BIRMINGHAM, Ala. An experimental drug that has proven effective in treating muscular dystrophy also works for cystic fibrosis, according to researchers at the University of Alabama at Birmingham (UAB).

The new study is the latest on a compound called PTC124, which helps to rescue faulty proteins that lead to illnesses. The drug holds promise in treating more than 2,400 genetic diseases caused by a certain class of DNA mutation.

In the UAB tests performed on mice, PTC124 restored to normal function up to 29 percent of the cases of abnormal cystic-fibrosis (CF) protein.

The findings are published in the online version of the journal Proceedings of the National Academy of Sciences and will soon appear in a print edition.

The study adds to research published last year in the journal Nature, which showed PTC124 restored up to 25 percent of the missing or abnormal protein function in mice with Duchenne muscular dystrophy.

Our study shows strong pre-clinical evidence that PTC124 is capable of suppressing nonsense mutations that cause cystic fibrosis, said David Bedwell, Ph.D., professor in the UAB Department of Microbiology and lead author on the study. We think this provides strong evidence for clinical trials with PTC124 in CF patients with this kind of mutation.

A gene that carries a nonsense mutation produces a shortened or faulty protein that degrades in the body. The absence of that protein is what leads to disease, Bedwell said. An estimated one-third of gene defects responsible for human disease are thought to come from nonsense mutations. In the case of CF, the absence of a certain protein leads to an imbalance of salt and water in the linings of the lungs and other membranes.

The UAB study showed that PTC124 allowed the protein to be made in mouse cells where it was previously absent, and it was delivered in a specific location that helped restore salt and water balance in membranes.

The preclinical and clinical data on PTC124 support our hope that this drug will be an important disease-modifying therapy for cystic fibrosis, said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation in Bethesda, Md. We look forward to the next stage of clinical development to demonstrate the benefits of this promising therapy.

Also in the UAB study, PTC124 was shown to be highly selective for fixing only disease-causing mutations, while it spared normal genes, Bedwell said.

The compound has been granted orphan-drug status by the U.S. Food and Drug Administration for the treatment of Duchenne muscular dystrophy and CF, according to the biopharmaceutical company PTC Therapeutics, Inc. of South Plainfield, N.J. It works in an oral form.


'/>"/>
Contact: Troy Goodman
tdgoodman@uab.edu
205-934-8938
University of Alabama at Birmingham
Source:Eurekalert

Related medicine news :

1. MIT radar technology fights breast cancer
2. New Drug Fights Medication-Linked Bone Loss
3. Experimental Drug Fights Prostate Cancer
4. Connecticut Girl Fights Rare Neurological Disease
5. Two-Drug Combo Fights Brain Tumors
6. Guacamole: Fights Hunger, Cholesterol and Cancer
7. Secondhand Smoke Worsens Lung Function in Cystic Fibrosis Patients
8. Cystic Fibrosis Foundation reports upward trend for key health outcomes
9. Montana Passes Newborn Screening Law for Cystic Fibrosis
10. Ibuprofen Preserves Lung Function in Kids With Cystic Fibrosis
11. Ibuprofen associated with slower lung function decline in children with cystic fibrosis
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/10/2016)... ... 10, 2016 , ... Holiday shopping, decorating, parties, family gatherings, ... stimuli that can put a great deal of stress on consumers this and ... gifts, food and festive gatherings can lead to excessive behaviors, which has an ...
(Date:12/10/2016)... ... ... When faced with having to use a toilet away from home an ... and contamination of children when they use public toilets. This inspired her to design ... face. , The patent-pending SANI-POCKET enables a person to use a public toilet in ...
(Date:12/9/2016)... ... December 09, 2016 , ... Flottman Company is ... As a means of expanding capabilities Flottman has added a G&K Vijuk ... code professional inserts (PIs) and patient package inserts (PPIs) that will marry with ...
(Date:12/9/2016)... ... ... An inventor from Cana, Va., wanted to fulfill the need for a device ... The patent-pending SAFETY STRAP FOR AMPUTEES improves accessibility. It eliminates discrimination. It is also ... a matter of minutes, or even seconds. The SAFETY STRAP FOR AMPUTEES is a ...
(Date:12/9/2016)... PA (PRWEB) , ... December 09, 2016 , ... "I ... said an inventor from Winchester, Va. "I thought that if the nebulizer had a ... treatments rather than fearing them." , He developed the patent-pending NEBY to avoid the ...
Breaking Medicine News(10 mins):
(Date:12/9/2016)...   Hudes Laser Aesthetica announced their ... Astanza Trinity laser and Astanza Liberty IPL technologies. ... removal, IPL hair removal, pigmented and vascular lesion ... Aesthetica is setting high standards for aesthetic clinics ... to deliver effective, long-lasting results. "Hudes ...
(Date:12/9/2016)... SARASOTA, Fla. , Dec. 9, 2016  Harmar Mobility, LLC ... officer and a member of the Board of Directors. ... ... Mr. ... and development of organizations across a variety of industries. He brings ...
(Date:12/9/2016)... DUBLIN , Dec 9, 2016 Research ... Vaccines Market 2016-2020" report to their offering. ... The global travel vaccines market ... The report covers the present scenario and the growth ... the market size, the report considers the revenue generated from the ...
Breaking Medicine Technology: