2-drug combination for MS shows dramatic results in British study

LONDON — Four years ago, Karen Ayres was paralyzed from the neck down by multiple sclerosis.

"When I was lying in my hospital bed, not even able to twitch my toes, I was jealous of anyone who could walk," she said.

But she started receiving an experimental combination of drugs, and within weeks, she was walking — right out of the hospital.

That was in 2002. Ayres, 28, who is studying for a doctoral degree in health and psychology, hasn't had relapses of her disease since.

She was among 27 patients with aggressive MS treated with the anti-cancer drug mitoxantrone plus Copaxone, which is used to treat relapsing MS.

Like Ayres, many of the other patients experienced results so remarkable that some MS experts, while expressing caution, are taking a second look at the preliminary experiment.

A three-year controlled study is starting at 10 centers across the United Kingdom to further investigate the drug combination. The results of the initial trial, led by Dr. Mike Boggild at the Walton Centre in Liverpool, will be published in August in the Journal of Neurology.

The cause of nerve-damaging multiple sclerosis is unknown and there is no cure. The most common form causes periodic flare-ups of symptoms, which include trouble seeing and walking.

Mitoxantrone is so powerful that it is potentially toxic and can be used safely only in the short term. Boggild and his colleagues combined it with Copaxone, a slow-acting drug.

"We decided to overlap the treatments because we wanted to give some time to Copaxone to build up its effect," Boggild said.

With a few exceptions, he said, most of the patients treated with the drug combination are essentially "trouble-free."

Though one patient developed acute leukemia — a side effect of mitoxantrone — Boggild said most of the patients haven't had relapses.

Patients were given a limited course of mitoxantrone before being started on Copaxone, usually a few months later.

While the study has generated some impressive results, many experts say more time is needed.

"It's a small study with no control group," said Dr. Robyn Wolintz, co-director of the MS Center at Maimonides Medical Center in New York.

"They also gave different people different doses of mitoxantrone, and that's not standard," said Wolintz, explaining that changing the dosages and the frequency of the treatment makes it difficult to reproduce the results to verify the treatment's efficacy.

"We're talking about an early, small study," said David Harrison of the MS Society in the United Kingdom. Though Harrison called the results "encouraging," he said a large-scale comparative investigation is needed to establish how the mitoxantrone-Copaxone combination ranks against other drug cocktails.

Still, some experts think Boggild and his colleagues have stumbled upon a valid hypothesis.

"Single drugs are not what gets the job done," said Dr. John Richert, vice president of the research department of the U.S. National Multiple Sclerosis Society. "It really is likely that more and more combination therapies will be used."

For people with particularly aggressive MS — 5 percent to 10 percent of MS patients — the treatment may provide hope. "The people who could benefit from this have nothing else at their disposal," Wolintz said.

Ayres said that despite the potential side effects, which include leukemia and cardiac problems, the treatment was worth the risk. She still injects herself with Copaxone.

"I didn't have a lot of options, and to be completely normal now was worth everything that I went through," she said.