Promising Lou Gehrig's disease drug fails trial

LONDON (Reuters) - An acne drug that had shown early promise in treating the degenerative nerve condition known as Lou Gehrig's disease actually worsens it, a study in Lancet Neurology said on Thursday.

The findings from one of the first randomised trials of the drug minocycline in patients with amyotrophic lateral sclerosis (ALS) also casts a shadow on studies planned for people with Huntington's disease, stroke and multiple sclerosis, it said.

"Our finding that minocycline has a harmful effect on patients with ALS has implications for trials of minocycline in patients with other neurological disorders," Columbia University researcher Paul Gordon and colleagues wrote.

There is no cure for ALS, often called Lou Gehrig's disease after a hall-of-fame New York Yankees baseball player who was diagnosed with it in 1939, and its causes are not clear.

It kills quickly -- usually between two and five years after symptoms start -- and doctors diagnose about 120,000 new cases each year, according to the International Alliance of ALS.

The U.S. researchers looked at the antibiotic that had reduced motor neuron loss and boosted survival in earlier trials with mice. Researchers targeted the drug in hopes it would slow cell death in the brain that causes the disease.

But the test of 412 human patients with ALS showed people treated with minocycline deteriorated at a 25 percent faster pace than those given a placebo.

"Like other recent trials in which drugs had no benefit or seemed to cause deterioration in patients with ALS... our trial could not replicate in patients the positive findings in laboratory studies," the researchers wrote.

The failed trial joins a list of many others that have failed to repeat promising results in animal studies and highlights the need for new approaches, Michael Swash, a neurologist at Royal London Hospital wrote in a commentary.

The findings also reinforce the need for early diagnosis and suggests researchers should look to shorter, less expensive trials in humans before larger studies, he added.

"Recent studies of ALS have led to an expectation that therapies will be found to stop progression of the disease, if not restore lost motor function," Swash wrote. "Unfortunately, this expectation is far from becoming a reality."