WASHINGTON (Reuters) - Treatment with an immune-suppressing drug may help people with the incurable disease multiple sclerosis, researchers said on Monday.
Their small study showed that treatment with high doses of cyclophosphamide, a generic cancer drug that has been around for half a century, cut the level of disability in MS patients, improved their physical functioning and reduced the number of brain lesions related to the condition.
Nine patients were tracked for two years after getting the drug. Five of them had no signs of disease activity, and the other four showed dramatic improvement, said Dr. Douglas Kerr of Johns Hopkins University in Baltimore.
The patients were able to recover physical functions that had been lost to the disease, Kerr said.
Existing drugs tend at best to slow the deterioration in MS patients and typically need to be given again and again.
“Every other therapy that’s out there and, as far as I’m aware, every other therapy that’s on any drug company’s drawing board, is designed to hold the disease at bay for as long as you take the drug,” said Kerr, whose study was published in the journal Archives of Neurology.
“This is one in which you give it once to hopefully reset the immune system back to a naive state so it’s no longer attacking the brain and spinal cord. And so that, I think, makes it quite different and quite exciting,” Kerr added.
The nine patients in the study were given large doses of cyclophosphamide intravenously for four consecutive days in a bid to “reboot” the immune system — and then no more of it.
The patients in the study had relapsing-remitting MS, the most typical form of it in which patients have periods of symptoms followed by periods of remission with no symptoms.
Kerr said the findings could herald a significant advance in the treatment of MS. He said the researchers aim to begin a large, multi-center clinical trial of the approach next year.
Multiple sclerosis is thought to be an autoimmune disease in which a person’s immune system mistakenly attacks parts of the body as if they were foreign.
In MS, the body attacks the myelin sheath, the fatty material that surrounds and protects nerve cells. This causes interference in the messages between the brain and body, triggering vision problems, muscle weakness, difficulty with coordination and balance and thinking and memory problems.
Kerr said the nine patients were the “worst of the worse,” with eight of them failing all other treatments and the ninth having had no previous treatment.
After two years, the patients experienced an average 40 percent reduction in disability and an 87 percent improvement in tests measuring physical and mental function, the researchers said. Brain imaging also showed a decrease in the average number of brain lesions from 6.5 to 1.2 lesions.
Kerr said cyclophosphamide has been used with mixed results in the past in MS treatment. Monthly infusions at a much lower dose worked modestly at best, he said. The drug also has been used alongside others in conjunction with bone marrow transplant treatment, he added.
There is one other immunosuppressant drug used for MS.
The U.S. Food and Drug Administration in 2000 approved the cancer drug Novantrone, also known as mitoxantrone, for MS treatment. It is marketed by OSI Pharmaceuticals Inc.
Editing by Maggie Fox and Cynthia Osterman