Explosion in Treatment Advances for Multiple Sclerosis

January 5, 2011— -- For three decades Tracy Ofri, 48, of Valley Stream, N.Y., suffered with multiple sclerosis.

At age 19, Ofri lost sight in one eye. Her unpredictable monthlong bouts of blurred vision, painful breathing and numbness throughout her body weighed her down.

And within her body, her own immune system attacked her myelin, the fatty substances that protected the nerves in her brain, spinal cord and optic nerves. Like many others who have the type of MS Ofri does -- relapsing and remitting MS -- the condition had her riding its ups and downs.

"When the doctor came into the room, he told me that there was no treatment, and that I had a disease with no medicine, and no cure," said Ofri, who was diagnosed in 1987. "I didn't even get a pamphlet. I had no information."

For decades she tried different drugs that brought on severe side effects. Then in 2007, Ofri enrolled in a clinical trial for a new drug that was soon hailed as the first oral MS medication to hit the market. Studies for the drug, Gilenya, showed that it reduced the number of relapses by keeping the white blood cells under control.

And for Ofri, it did just that. She hasn't relapsed since she started taking Gilenya, a drug approved just last year to reduce the frequency of clinical relapses and reduce inflammation in cells found in the central nervous system.

Experts say that the solo drug is not the only saving grace for many people with MS. The past decade has offered the greatest advances in MS, so much so that it may be difficult to believe that at one time the chances of successfully combating the condition were slim.

Twenty years ago, treatments for MS meant managing symptoms like urinary complications and joint pain. Now there are eight drugs approved for MS that control symptoms or modify the disease, half of which were approved within the past five years.

Three new disease-modifying treatments will be presented for FDA approval this year.

"We went from a time when one of my early mentors told me the attitudes for people with MS were 'diagnose and adios.' We had nothing to offer them," said Dr. Aaron Miller, director of the MS Center at Mt. Sinai Hospital in New York and chief medical officer for the National MS Society. "We've had an incredible explosion of new treatments."

The transformation is also due in part to the emergence of magnetic resonance imaging – MRI – that's not only used to diagnose the disease but to predict how the disease will progress.

"With MRI, we can also define the response to therapy," said Dr. Robert Fox, neurologist and medical director of the Mellen MS Center at the Cleveland Clinic.

Imaging also helps specialists segment patients to the right type of therapy based on their risk, Fox said.

Therapeutic progress in MS is almost exclusive to the relapsing and remitting form, or stage, of the disease. Before the development of disease-modifying therapies, about three-quarters of patients with this form of the disease developed a progressive form of MS.

Still, the challenge remains for the nearly 10 percent of patients who have primary and secondary progressive types of MS, where there are currently no therapies.

Unlike the relapsing and remitting form, the progressive form of the disease worsens more steadily without ever getting better.

"The number of trials in relapsing outnumbers the progressive form by 10 to 1," said Fox. "What we lack in progressive MS is understanding the basic biology of it; we lack imaging markers of it."

But that's not stopping research into emerging therapies for the disease, including stem cell transplantation.

While still in trial phase, researchers hope that transplanting mesenchymal stem cells, which are taken from a specific part of the bone marrow, may offer ways to keep white blood cells from attacking tissues in the central nervous system, and restore tissue already damaged in the brain and spinal cord.

Some experts said at best, the next renaissance -- this time one of understanding the condition's progressive forms and subsequent therapies – may be another generation out.

But for an area of research that once underwent a dry spell, the experts said, the coming decade offers a new and unpredictable spring.

"It's so gratifying now that we have so many options," said Miller. "And to know that as the years go on, we're seeing drugs that are not only easier to take – oral drugs rather than injectable drugs – but that have a higher degree of effectiveness."