Novel drug candidate may help treat inherited muscular wasting diseaseNovember 7th, 2009 - 2:29 pm ICT by ANI
Washington, Nov 7 (ANI): Scientists from University of Oregon and the University of Rochester School of Medicine and Dentistry in New York have discovered a potential drug candidate that may help treat inherited muscular wasting disease.
The compound called pentamidine carries approval of the U.S. Food and Drug Administration for treating a severe type of pneumonia in people with weakened immune systems, as well as leishmaniasis, sleeping sickness and some yeast infections.
The new study showed that pentamidine, when tested in genetically altered mice, counters genetic splicing defects in RNA that lead to type 1 myotonic dystrophy- one of nine types of muscular dystrophy - also known as DM1 and Steinart’s disease.
“The fact that a very small library of compounds yielded a molecule capable of reversing the splicing defects associated with DM1 in both cell and mouse DM1 models suggests that a small molecule strategy could lead to a drug for this disease,” said UO chemist J. Andrew Berglund.
In a separate commentary in PNAS, Thomas A. Cooper of the Baylor College of Medicine in Houston hailed the findings, noting that the compound is the first to show such promise of reversing splicing defects.
He noted that such a therapeutic approach is attractive because of the potential benefits to multiple organs affected by the disease.
Berglund added that pentamidine is an exciting lead compound because it is relatively easy to chemically modify, and hopefully one of these modified compounds could lead to a safe, long-term treatment in the future. (ANI)
- Effects of genetic defect in myotonic muscular dystrophy revealed - Jan 25, 2010
- Indian scientists find safe drug against kala azar - Sep 10, 2012
- Novel discovery may pave way for new muscular dystrophy therapies - Jul 17, 2009
- Golden retriever could hold key to treating muscular dystrophy - Dec 31, 2010
- Heart drug 'helps treat muscular dystrophy symptom' - May 05, 2010
- Drug candidate for treating spinal muscular atrophy identified - Nov 05, 2009
- Manipulating muscle stem cells could treat muscular dystrophy - Oct 10, 2010
- Malaria-fighting tobacco! - Feb 13, 2012
- Cancer drugs may help battle parasite Leishmania - Jul 17, 2010
- First clinical trial of gene therapy offers muscular dystrophy clues - Oct 07, 2010
- Protein modification involved in muscular dystrophy, cancer discovered - Jan 01, 2010
- New signaling pathway linked to inflammatory disease discovered - Dec 15, 2010
- Potential treatment target for congenital heart disease identified - Feb 22, 2011
- Muscular dystrophy gene mystery cracked - Aug 20, 2010
- Scientists identify risk factors for sudden death for adult muscular dystrophy - Jun 19, 2008
Tags: baylor college of medicine, baylor college of medicine in houston, dm1, drug candidate, food and drug administration, lead compound, myotonic, novel drug, pentamidine, pnas, rochester school, school of medicine and dentistry, sleeping sickness, therapeutic approach, types of muscular dystrophy, university of rochester school of medicine, university of rochester school of medicine and dentistr, wasting disease, weakened immune systems, yeast infections