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Stem cells offer new hope for MS patients
CLEVELAND, Ohio—A group of researchers here are collaborating on a clinical trial that aims to treat or even reverse the debilitating effects of multiple sclerosis (MS) by harnessing the power of stem cell research.
The trial—the first of its kind in the United States—is the work of a consortium of researchers from the Cleveland Clinic, University Hospitals' Seidman Cancer Center and Case Western Reserve University (CWRU). Researchers are harvesting mesenchymal stem cells (MSCs)—or adult stem cells found in the bone marrow—of patients with MS, cultivated them in a CWRU lab and then injecting them intravenously back onto the patients.
The primary goal of these efforts is to test the safety and feasibility of using the body's own stem cells to treat MS. More long-term, though, the researchers hope to find evidence that these transplanted cells can help moderate the overactive immune systems of MS patients—or to develop regenerative strategies to repair or even stop the debilitation seen in the more progressive stages of the disease.
The researchers are interested in MSCs because they have several properties that could possibly make them helpful in treating MS, says Dr. Jeffrey Cohen, lead investigator of the trial.
"One reason is that they have the ability, in a number of animal models of other human diseases, to increase repair in tissue damages—not just by replacing the cells themselves, but also by creating an environment that supports normal repair mechanisms that are already present in tissue," says Cohen, who in addition to seeing patients in a clinical setting, directs the Experimental Therapeutics Program at the Cleveland Clinic's Mellen Center for Multiple Sclerosis Treatment. "In addition, the anti-inflammatory effects of these cells are quite potent in modulating a number of immune mechanisms, may of which have been implicated in MS. Finally, these cells appear to have the ability to migrate through the blood into tissues that are inflamed and seek out areas of damage. This is much more convenient than directly injecting them into the brain."
The study is the clinic's first foray into performing stem cell research on MS patients, Cohen says. The researchers have enrolled four patients in Phase I studies so far. After a formal safety review, they hope to enroll a total of 24 patients by the end of 2012.
Getting the trial off the ground hasn't been easy. Researchers first had conversations about it in early 2007, but had to work earnestly for a few years to obtain funding.
"This study is fully funded by grants, rather than industry," Cohen notes.
But the researchers' main stumbling block, he says, was obtaining the necessary regulatory approval to proceed.
"Even though there has been some experience with using these cells in other disease areas, the overall published experience has been very modest," Cohen explains. "We hope to get the reassurance we need to move at a more rapid place."
The researchers have not yet considered a commercial partner to bring the results of their findings to market, he notes. Instead, they are focused more on furthering the science involved in using MSCs.
"We're less encumbered by commercial constraints, and more concerned with how a treatment would be offered more broadly down the road," he says. "Even if the cells are not commercializable, the data we are generating will surely help other people who have commercial plans underway."
The outcome of this trial is expected to be a significant advance in the field of MS research. About 10,000 people in the United States are diagnosed with MS every year, and the disease primarily affects women. There is no known cure. Cohen notes that medications currently approved for the treatment of MS focus on preventing disease activity, but do not address damage. They can also have adverse effects or be poorly tolerated.
"There is a huge unmet need for therapies for MS patients," Cohen says.
MSCs have also been tested on and demonstrated potential in several other conditions such as heart disease, stroke, spinal cord injury and non-healing bone fractures, he adds.
"MSC transplantation is a very effective treatment for conditions where anti-inflammatory actions are discovered," Cohen says. "It's being evaluated in a wide variety of conditions. The areas that have received the most attention are cancer, graft-versus-host disease, bone marrow transplants, coronary disease and peripheral vascular disease. There are also a number of smaller studies in diseases like irritable bowel syndrome. But the overall experience there is relatively modest."