The National Multiple Sclerosis Society—a group including patients, donors, and Society leaders—recently came out to meet Sanford-Burnham’s Barbara Ranscht, Ph.D., tour her lab, and learn how she and her team are laying the groundwork for future Multiple Sclerosis (MS) therapies.
In MS, a person’s immune system mistakes myelin—the protective coating around nerve fibers in the brain—for a foreign substance and attacks it. As a result, these fibers, called axons, become vulnerable and prone to inducing neurodegeneration and cell death. Ultimately, the brain has trouble telling the rest of the body what to do.
“Myelin is necessary for rapid nerve impulse conduction—those times when you need a quick reaction, like pulling your hand off a hot burner,” Ranscht explained in her talk. “It’s also needed to protect the axons and ensure the health of our neurons.”
Symptoms of MS can be mild, such as numbness in the arms and legs, or severe, causing paralysis, vision loss, or other impairments. Severity and progression vary widely from one individual to another. Approximately 400,000 people in the U.S. are living with MS.
“Many of these MS patients often visit medical clinics to see their doctors and receive treatment, but it’s not often that they get to hear about the science behind it all—what researchers are learning about the disease and what new therapeutic approaches might be coming down the pipeline,” said Richard Israel, president of the Pacific South Coast Chapter of the National MS Society.
Carrie Radant, the Society’s director of Development and Strategic Relationships, explained that they recently conducted a survey to learn what people touched by MS thought was most important and what the Society should be focusing on. Respondents overwhelmingly said they want the Society to fund more research on MS and draw more scientists and doctors to the field. As a result, the Society started a new campaign to raise money for MS research and award grants through a competitive review system. These grants, Radant explained, fund research aimed at either “stopping the progression of MS, restoring function, or ending the disease forever.”
Ranscht, a professor in Sanford-Burnham’s National Cancer Institute-designated Cancer Center, is a recipient of one of those Society grants. Her work falls into the “restore function” category of MS research. Most current therapies aim to temper the immune response. But, according to Ranscht, “We also need strategies to repair the myelin, to make sure axons are always covered.” Restoring myelin, and therefore preserving axon function, could retain vital functions in patients with MS.
Ranscht spoke to the group about her lab’s studies of Contactin-1, a protein anchored in the membranes of myelinating cells, where it supports axon contact. Contactin-1 seems to be especially important during the myelination process. In mice, Contactin-1 function is required for proper myelin formation, normal balance, and voluntary movement.
Ranscht believes that changes in Contactin-1 function contribute to myelin defects in MS. She also thinks the protein might make a good candidate for therapies aimed at repairing myelin. With the help of scientists in the Conrad Prebys Center for Chemical Genomics, Sanford-Burnham’s drug discovery center, Ranscht and her team are planning to develop new therapeutic drugs that promote myelination after injury.
After Ranscht’s talk, the visitors toured her lab, where MS patients met the mice that share their condition—a valuable research model that Dr. Ranscht and other scientists use to study the disease and look for new treatments.
“We’re honored to be funding the work being done here by Dr. Ranscht,” Israel said.