New Drug Aids Muscle Function in Myasthenia Gravis

FRIDAY Aug. 17, 2007 -- A new drug -- oral EN101antisense -- reduced the severity of muscle weakness in patients with myasthenia gravis, researchers report.

Myasthenia gravis (MG), which affects about 20 in 100,000 people, causes fatigue and reduced strength in voluntary muscles. Other symptoms may include double vision, drooping eyelids, difficulty swallowing, or slurred speech.

The small open-label study included 16 MG patients who took daily doses of oral EN101antisense for four days and were monitored for a month. On average, the patients experienced a 46 percent reduction in symptom severity. Side effects included dryness of the eyes and mouth.

The study, published in the Aug. 14 issue of Neurology, was supported by the biotechnology company Ester Neuroscience Ltd.

"This is the first time we've been able to show that antisense is effective and safe when taken orally for a neurological disease," study author Dr. Zohar Argov, of the Hadassah Hebrew University Medical Center in Jerusalem, said in a prepared statement.

Further research is planned to study the effects of the drug over a longer period of time.

Antisense is a synthetic, short segment DNA that blocks production of specific proteins. Oral EN101antisense inhibits production of an enzyme called acetylcholine esterase, which plays an important role in the function of the neuromuscular junction, where nerves connect with muscles.

More information

The American Medical Association has more about myasthenia gravis.

Posted: August 2007


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