DAVID ROSENFELD

For Cathy Gregory, who suffers from secondary progressive multiple sclerosis (MS), the benefit of the drug trial she’s enrolled in may represent more hope for others than herself. And that’s all right with her.

“I can’t turn back time for myself,” said Gregory, whose MS has progressed to the point where she’s wheelchair-bound and has difficulty performing simple tasks. “But this drug might halt additional damage. And hopefully others can utilize the drug when they find out they have it.”

The trial is taking place among roughly 500 patients at more than 70 centers in Canada and the United States. Three of those patients, including Gregory, are under the care of Dr. Steven Cohen, PhD, at the Suncoast Medical Clinic in St. Petersburg.

“I’m very confident that one day MS is going to be in the history books like polio,” Cohen said.

Roughly 200 patients a week are diagnosed with MS, an autoimmune disease that affects an estimated 400,000 people in the United States, according to the National Multiple Sclerosis Society. There is no definitive cause of the disease, but it’s believed to be genetic or related to environmental factors.
Patients suffering from less severe forms of MS than Gregory have some effective FDA-approved treatments such as Novantrone — which Gregory tried without success — to slow down the disease. But so far nothing has proven able to consistently slow or completely stop the progression of secondary progressive MS, its most debilitating form.

Halting the deterioration of motor skills is exactly what the drug known as MBP8298 purports to do for people with secondary progressive MS such as Gregory, who comprise roughly 40 percent of all MS patients.

Developed by BioMS Medical, the drug is injected every six months. The double blind Phase III trial, which began around August, will last for three years. It’s one of the last steps before FDA approval.

What brings Cohen so much hope? The science behind the understanding of MS is getting better and clearer for researchers everywhere.

Scientists believe that patients develop MS when the fatty tissue that surrounds the central nervous system, known as myelin, disappears. This breaks down communication between the brain and the body, which leads to the physical symptoms.

MBP8298 works similar to an allergy shot. The injection introduces a myelin basic protein that helps the immune system build tolerance to the disease.
“The hope is that we’re going to make the patients immune system change so this protein is no longer thought to be foreign,” Cohen said.

Other drugs to combat MS work somewhat differently. A similar approach, known as peptide therapy, triggers an immune response to myelin-destroying white blood cells.

But don’t presume that MBP8298 is a miracle cure, said Dr. Jack Burks, clinical professor at the University of Nevada School of Medicine and chief medical officer of the Multiple Sclerosis Association of America. Burks also serves as an adviser to BioMS Medical.

“There are probably a dozen promising drugs,” Burks said. “This is one of them.”
But only MBP8298 specifically targets patients with secondary progressive MS. Burks says the results so far should be viewed with some perspective.

“There has only been one publicized study with small numbers of patients while other promising drugs have two or three studies,” Burks said. “The preliminary data, however, was pretty spectacular.”

In the study published in the European Journal of Neurology in August 2006, the drug worked to slow the disease on average by up to five years.

There’s a fair chance that Gregory might not be receiving the actual drug. A certain number of patients in the study are taking a placebo, of which not even their doctors are aware. There’s also a chance that even if she is receiving the drug, Gregory might not have the right genetic makeup to be affected. By even its most hopeful estimates, MBP8298 will not work on 25 percent of secondary progressive MS patients because they lack a specific gene sequence.

“If it’s not working or if I’m not getting the drug, that’s okay,” Gregory said. “I don’t worry about stuff like that. And anyway, I’ll be able to get it in three years (the estimated time it could take for FDA approval).”

This latest study caps more than six years of clinical trials. BioMS Medical has amassed significant losses in its quest to develop MBP8298. For the first nine months of 2007, the company lost $35.5 million.

That would explain why in December, Eli Lilly and Company (NYSE: LLY) bought the rights to MBP8298. According to the agreement, the two companies will team up on the drug’s development with Eli Lilly being responsible for much of its future costs. BioMS received $87 million and sales up to $410 million and other royalties in the agreement.



April 2008