LONDON – Four years ago, 28-year-old multiple sclerosis patient Karen Ayres was wheelchair-bound and paralyzed.
"I was trapped in a body that wouldn't do anything," she says. Now, following an experimental drug treatment, she has regained mobility and is studying for a doctoral degree.
Like Ayres, many of the other patients in the study experienced results so remarkable that some MS experts, while expressing caution, are now taking a second look at the preliminary experiment.
A three-year controlled study is being launched at 10 centers across the United Kingdom to further investigate the potential of the drug combination. The results of the initial trial, led by Dr Mike Boggild at the Walton Centre in Liverpool, will be published next month in the Journal of Neurology.
Mitoxantrone is an anti-cancer drug so powerful that it is potentially toxic and can only be used safely in the short term. So, Boggild and his colleagues combined its use with copaxone — a notoriously slow-acting drug.
"We decided to overlap the treatments because we wanted to give some time to copaxone to build up its effect," says Boggild.
What happened next was dramatic. "Patients who were just the worst of the worst did remarkably well," Boggild said.
"We think we've tapped into an unexpected synergy between the two drugs that gives you more than the sum of the parts," he says. With a few exceptions, Boggild says most of the patients treated with the drug combination are now essentially "trouble-free."
Though one patient developed acute leukemia — a known side effect to mitoxantrone treatment — Boggild says the majority of patients haven't had disease relapses.
Patients were first given a limited course of mitoxantrone, before being started on copaxone, usually a few months later. In Ayres' case, recovery was rapid.
"From barely being able to wave my hand to walking out of the rehabilitation center took a few weeks," she says.
While the study has clearly generated some impressive results, many experts say more time is needed.
"It's a small study with no control group," says Dr Robyn Wolintz, co-director of the MS Center at Maimonedes Medical Center in New York. "They also gave different people different doses of mitoxantrone, and that's not standard," says Wolintz, explaining that changing the dosages and the frequency of the treatment makes it difficult to reproduce these results to verify the drugs' efficacy.
"We're talking about an early, small study," says David Harrison, of the MS Society in the United Kingdom. Though Harrison characterized the results as "encouraging," he believes it is necessary to conduct a large-scale comparative investigation to establish how the mitoxantrone-copaxone combination ranks against other known and potential drug cocktails.
Still, some experts believe Boggild and his colleagues have stumbled upon a valid hypothesis. "Single drugs are not what gets the job done," says Dr John Richert, vice-president of research department in the United States' National MS Society. "It really is likely that more and more combination therapies will be used."
For patients with particularly aggressive MS, the proposed treatment may provide some hope. "The people who could benefit from this have nothing else at their disposal," says Wolintz.
And unfortunately, medicine is far from an exact science.
"Many physicians make their decisions based on incomplete data," says Richert. "Even though there's not sufficient data to make any kind of formal recommendation, there is enough data to say that it is reasonable to make this option available to physicians and their patients when they've weighed all the pros and cons of the situation."
For patients like Ayres, who still takes daily injections of copaxone, life without the treatment is unimaginable.
"When I was lying in my hospital bed, not even able to twitch my toes, I was jealous of anyone who could walk," she told The Associated Press.
Despite the potential side effects, which include leukemia and cardiac problems, Ayres says the treatment was worth the risk. "I didn't have a lot of options," she says, "and to be completely normal now was worth everything that I went through."