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:9/19/2017)... ... September 19, 2017 , ... ... its executive team as the President of its HealthBI division. McFarland will ... and contact centers. , “Scott is a utility player who balances his ...
(Date:9/19/2017)... ... September 19, 2017 , ... MelaKids, a Laredo-based company, has introduced a ... When we’re born, Nature gives us a full supply of melanin – in the ... lose this natural glare-reducing pigment; however, around the age of thirty, we develop a ...
(Date:9/19/2017)... ... September 19, 2017 , ... ... the present, Mirixa Corporation recently unveiled the MirixaPro 6.0 platform. As the ... how medication management services are delivered. Based on industry research and market ...
(Date:9/19/2017)... ... September 19, 2017 , ... Back Pain Centers of ... back pain with a reputable physician in their area, announces the addition of ... as Nevada Spine Clinic was founded by board-certified spine surgeon, Jaswinder Grover, M.D. ...
(Date:9/19/2017)... ... 2017 , ... The ISO 9001 standard sets out the requirements for a ... and improve customer satisfaction. ISO 9001:2015 was released in 2015 and certified organizations have ... September 2018. , As described in this white paper , ISO ...
Breaking Medicine News(10 mins):
(Date:9/12/2017)... , Sept. 12, 2017  ValGenesis Inc., the ... (VLMS), is pleased to announce the appointment of ... of its Board of Directors and Chairman of ... enables life science companies to manage their entire ... of paper in this process. Furthermore, ValGenesis VLMS ...
(Date:9/9/2017)... -- Eli Lilly and Company (NYSE: LLY ) ... lasmiditan, an investigational, oral, first-in-class molecule for the acute ... to placebo in the Phase 3 SPARTAN study. Detailed ... of the International Headache Society (IHC) in ... demonstrate lasmiditan,s potential to reduce pain and provide freedom ...
(Date:9/7/2017)... Caris Life Sciences, a leading innovator ... of precision medicine, today announced results from two ... molecular profiling approach in guiding therapeutic strategies in ... (CGP+) with Caris Molecular Intelligence ® to ... level, leading to more therapeutic options and improved ...
Breaking Medicine Technology: