Could erectile dysfunction drugs help muscular dystrophy patients?

Washington, Mar 25 (ANI): Taking a step further from a recent rodent study, a Cedars-Sinai Heart Institute cardiologist is probing if drugs used to treat erectile dysfunction could also be used to improve muscle blood flow and reduce fatigue in muscular dystrophy patients.

A recent study showed beneficial effects of tadalafil (also known as Cialis) in mice with an animal version of Duchenne and Becker muscular dystrophy.

Only two doses of tadalafil improved muscle blood flow, allowing the dystrophic mice to perform more exercise with less muscle injury.

The new short-term clinical trial will move the testing from animals to human patients with Becker muscular dystrophy and examine the effects of acute tadalafil dosing on muscle blood flow during a bout of exercise.

Patients will take two doses of tadalafil prior to exercising. Then doctors will measure whether muscles receive increased blood flow and therefore are better protected during exercise.

“This is an exciting next step in the research I have been doing for 25 years, because we don’t need to create a new drug — the drug already exists. We now have the opportunity to find out if tadalafil can offer some hope for improving the lives of patients and allow them to do more exercise with less muscle injury,” said Victor.

The study is open to adult males 18 – 55 who have Becker muscular dystrophy as well as adult males who don’t have it.

And it includes includes hand grip exercise testing, measurements of muscle blood flow and oxygen delivery, and magnetic resonance imaging of the muscles. (ANI)

Antibiotic treatment may offer cystic fibrosis treatment

Washington, Apr 8 (ANI): Researchers from Technion-Israel Institute of Technology have developed a novel antibiotic treatment for human genetic diseases, including cystic fibrosis (CF).

By modifying the properties of the antibiotic gentamicin, commonly used to treat bacterial infections, the researchers claim to have developed a novel treatment for many human genetic diseases, including cystic fibrosis (CF), Duchenne muscular dystrophy, Usher Syndrome and numerous cancers.

Gentamicin belongs to a class of antibiotics called aminoglycosides.

The team led by Professor Timor Baasov of the Technion Faculty of Chemistry have modified existing aminoglycoside antibiotic drugs, and carefully monitored biological and toxicity tests of the resulting derivatives.

They came across “NB54,” a new chemical derivative of gentamicin.

“We’ve created a new purpose for aminoglycosides by removing their traditional, natural actions as antibiotics,” said Baasov.

“The loss of their antibacterial activity makes them highly selective, less toxic, and allows for their use in repairing ‘wrong’ genes in human beings,” he added.

The findings were published in the Journal of Medicinal Chemistry. (ANI)

New class of heart disease drugs may be effective against fatal muscular disorder

London, Feb 9 (ANI): Researchers at Columbia University Medical Centre say that a new class of experimental drugs for heart failure may be effective against fatal muscular disorder.

During a study, the research team identified a “leak” that weakens skeletal muscle in Duchenne, a disorder that affects boys usually before the age of 6, destroying their muscle cells and cardiac muscle in heart failure.

This leak allows the calcium to slowly seep into the skeletal muscle cells and excess calcium ultimately cause damage.

Similarly in heart failure patients, the calcium leak weakens the force produced by the heart, and turns on a protein-digesting enzyme that damages its muscle fibres.

Lead researcher Dr. Andrew Marks believes that a new class of experimental drugs developed at CUMC, designed to plug the leak in the heart might be effective against Duchenne.

While experimenting on mice with Duchenne, the researchers found these drugs to dramatically improve muscle strength and reduce the number of damaged muscle cells.

“This was extremely exciting to us,” Nature magazine quoted Dr. Marks, chair of the Department of Physiology and Cellular Biophysics and Clyde and Helen Wu Professor of Molecular Cardiology, as saying.

“If it works in people, our drug won’t be a cure, but it could slow the pace of muscle degeneration and extend the lives of people with Duchenne,” he added.

The study appears in the journal Nature Medicine. (ANI)