UAB ALS Clinic co-directors Mohamed Kazamel, M.D., and Nan Jiang, M.D. (at left and right), Department of Neurology Chair David Standaert, M.D., Ph.D. (center back), and clinic patient and ALS advocate Kelly Butler (at front).Eight medical professionals. One visit. A new clinic for amyotrophic lateral sclerosis, or ALS, at UAB lets patients see eight health care providers during a single visit, with a single co-pay.
“They’ve got my back,” says Venus Richardson of Mobile, Alabama, who was first diagnosed with ALS in 2019 and began seeing UAB physicians a few months later. “I’ll be making a road trip to UAB every three months now to see the full team. The more you interact with your care team, the better you are able to express how the disease is affecting you. It means better communication and interaction.”
The new clinic is supported by a grant from the Alabama Department of Commerce Innovation Fund. It allowed UAB to expand the existing clinic to see more patients more often. Most patients will come every three months, and during their visit they will interact with their neurologist, physical and occupational therapists, a dietitian, speech therapist, psychologist, social workers, and respiratory therapists. The visit is facilitated by a nurse coordinator with experience working with ALS patients.
“If I have questions or need any information on medications, symptoms, or just questions about diet or lifestyle, they are there to help,” Richardson says. “I think God handpicked the perfect team for me. They take their time to talk to me and listen to me. They don’t just hear me; they listen.”
“The opportunity for patients to see all of their ALS care providers at one visit is a significant benefit,” says clinic co-director Mohamed Kazamel, M.D., associate professor in the Department of Neurology. “Besides convenience and peace of mind for the patient, it allows the care team to work together to build a comprehensive care program for each patient paired with frequent follow-up.”
ALS was identified in 1869 by French neurologist Jean-Martin Charcot, but it became more widely known internationally in 1939 when it ended the career of one of baseball’s most beloved players, Lou Gehrig. For many years, ALS was commonly known as Lou Gehrig’s disease.
The disease affects motor neurons, which reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. They govern voluntary movements and muscle control.
ALS causes these motor neurons to degenerate over time until they eventually die. The disease is progressive, meaning the symptoms get worse over time. When voluntary muscle action is progressively affected, people may lose the ability to speak, eat, move, and breathe. ALS has no cure and there is no effective treatment to reverse its progression.
The new clinic opened in January. It sees about 150 patients currently and expects to add about 30 new patients each year. “Beyond expanding our clinical services, the Alabama Innovation Fund grant will allow us to hire an ALS clinical trials coordinator so we can initiate and participate in more national or international clinical trials that shed new light on the disease and may lead to new knowledge about ALS that can point us toward treatments or even a cure,” says Nan Jiang, M.D., Ph.D., associate professor in the Department of Neurology and clinic co-director.
Last year, UAB investigators began enrolling patients in an international trial of a promising new ALS drug. Preliminary studies reported in 2021 indicate the drug, masitinib, may help prolong overall survival. “The 2021 study, which enrolled 394 patients primarily in Europe and South America, showed that masitinib could prolong survival by up to two years as compared with placebo, provided that treatment starts prior to severe impairment of functionality,” says Peter King, M.D., professor in the Department of Neurology and principal investigator for the study at UAB.
The new international study is enrolling more than 400 patients from countries around the world. UAB will enroll 12-15 individuals with mild, early symptoms of ALS. This is the first drug study for ALS at UAB since 2014.
Masitinib inhibits a class of cells called myeloid cells, which includes mast cells, macrophages, neutrophils, and microglia, all part of the human immune system. King was involved in preclinical studies, in collaboration with scientists in Uruguay, showing that Masitinib significantly attenuated inflammatory responses and tissue injury in a rodent model of ALS.
King, along with co-investigators Kazamel and Jiang, says the immune system may contribute to the progression of ALS by doing its job too well. “In ALS, it appears the immune system can be protective in the beginning, but ultimately becomes too active,” King says.
Over the years, King has established a bank of tissues obtained from ALS patients that has allowed him and collaborators to correlate discoveries in animal models with human disease.
“Our work established that there is a robust inflammatory response in peripheral neuromuscular tissues of ALS patients including mast cells, neutrophils, and macrophages,” he says. “Masitinib inhibits the action of these inflammatory cells as well as microglia in the central nervous system by targeting the signaling pathway that activates them. The hope is that dampening the inflammatory response may slow down the progression of ALS.”
-By Alicia Rohan
For more information on the UAB ALS clinic, call 205-934-2120.