LOS ANGELES (Sept. 30, 2014) - The ALS Program at Cedars-Sinai has become the first in Southern California to be named an ALS Association Certified Treatment Center of Excellence - a distinction that recognizes the quality of its treatment and research programs for patients with Lou Gehrig's disease, also known as amyotrophic lateral sclerosis, or ALS.
The progressive disease attacks neurons in the brain and spinal cord that control muscles throughout the body. Patients generally live two to five years after diagnosis, and no treatment has been found to significantly extend length of survival.
To earn certification, Cedars-Sinai met rigorous standards established by The ALS Association for research initiatives and a comprehensive and collaborative approach to patient care and services.
"Cedars-Sinai's commitment to excellence in ALS care is evidenced by its thorough and thoughtful approach to the multidisciplinary clinic and the integration of community-based care provided by the Golden West Chapter. We applaud their efforts to discover and deliver effective treatments and a cure for ALS," said Nicole Yarab, The ALS Association's director of Certified Center Programs.
About 10 years ago, Cedars-Sinai partnered with The ALS Association Golden West Chapter to begin building a multidisciplinary clinic program. Cedars-Sinai invested heavily into a state-of-the-art patient care clinic and research effort in 2012 when Robert H. Baloh, MD, PhD, was named director of Neuromuscular Medicine in the Department of Neurology and director of the ALS Program. He joined Patrick Lyden, MD, chair of the Department of Neurology, and Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute, who had identified ALS as a top clinical and research priority.
Baloh's research group was one of the first to discover that mutations in a gene, TDP-43, cause inherited forms of ALS and frontotemporal lobar degeneration, a type of dementia that can accompany ALS. He also led studies that in 2009 produced a mouse model of ALS based on the TDP-43 mutation. This model is employed by researchers worldwide to study the disease.
Recently, Baloh's team turned skin cells of patients who had ALS into motor neurons that retained the genetic defects of the disease. In a lab dish, they identified the genetic defect that caused the disorder and inserted molecules made of small stretches of genetic material to block the damaging effects of the defective gene. The study is believed to be one of the first in which a specific form of ALS was replicated in a dish, analyzed and "treated," all in a single study.
In the clinic, Baloh sees the effects the disease has on patients; in his laboratory, he and his research team investigate underlying causes.
"Because there currently is no effective treatment or cure for ALS, much of our work in the clinic is focused on improving our patients' quality of life. At the same time, however, interactions with our patients provide motivation and direction for our scientists as we design and plan basic research studies and clinical trials. With technologies that weren't available even a few years ago, the ALS research community's understanding of this disease is growing rapidly, and new experimental drugs are in the pipeline," said Baloh, who in 2012 received The ALS Association's Golden West Chapter Commitment to a Cure Award.
The same year, the ALS Clinic became the first West Coast site providing an implanted breathing-assist device for ALS patients suffering chronic hypoventilation, the inability to take enough air into the lungs, under a protocol newly approved by the Food and Drug Administration. Cedars-Sinai doctors and researchers are currently participating in a clinical trial evaluating the device.
"The ALS Clinic is a core component of the overall program, where we strive to provide top-notch care using existing therapies, and to function as the conduit for patients to provide them opportunities to take part in research to identify new treatments for ALS. The ALS Association Golden West Chapter has partnered with us from the very beginning, by sending a patient care representative every week to the clinic, and by supporting part of the cost of our patient care coordinator, who is a key member of the team," Baloh said.
"We are thrilled that The ALS Association has recognized our efforts by certifying our clinic as a Certified Treatment Center of Excellence," he added. "We look forward to continuing to work with The ALS Association and the Golden West Chapter to maintain the highest level of care and research opportunities for our patients."
Baloh and his colleagues at Cedars-Sinai study a variety of approaches to treating ALS, including stem cell and gene therapies, all with a focus on developing patient-specific interventions.