![]() The approach aims to replace diseased astrocytes, star-shaped cells that support motor neurons. Every approach takes square aim at the ultimate questions: Why do patients develop ALS, and how can we stop the suffering?įor nearly 20 years, Clive Svendsen, PhD, executive director of the Board of Governors Regenerative Medicine Institute and the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine, has cultivated a multipronged sneak attack against neurogenerative disease. They aim to differentiate between genetic and sporadic forms of ALS, and scour the models for the earliest signs of cellular decline. Having built the largest library of hyper-specific disease data, the team is reconsidering whether ALS is not one disease but a collection of conditions. Scientists are growing cells from ALS patients in petri dishes to model the disease. Because fresh progress in the disease is fueled by the body’s cells at their most naive state, the ALS Clinic team has embarked on a new clinical trial to test the safety of stem cell implantation directly into the cerebral cortices of ALS patients. ![]() The highly specialized, resourceful clinicians at Cedars-Sinai’s ALS Clinic, an ALS Association Certified Treatment Center of Excellence, can only leverage tools and technologies to support their chief goal: to preserve quality of life as patients become paralyzed and die.īuoyed by breakthroughs in the study of stem cells, Cedars-Sinai investigators are challenging assumptions and evolving their questions about ALS. In the absence of biomarkers, physicians can only diagnose ALS after it has already taken hold, and the three Food and Drug Administration approved treatments do little to slow its progression. Without the ability to biopsy brain and spinal tissue, little is understood about its mechanisms. ![]() Only 5% to 10% of patients carry genes known to cause the disease. Until now, ALS has frustrated researchers with a notoriously impenetrable monolith. We are encouraged enough with the results to proceed to more patients and attempt to slow disease progression.” “They knew we weren’t going to cure their disease, only pursue whether the cells and this surgical approach were safe. “These patients are the heroes of this research,” says Richard Lewis, MD, director of the Electromyography Lab and principal investigator of the study at the ALS Clinic. The findings, published in September 2022 in Nature Medicine, cleared investigators to study the therapy’s efficacy and continue refining the approach they hope that, ultimately, will slow or stop the disease. “She would do anything to be part of finding answers.”Īshley, along with the 17 others in the study, gave investigators their only opportunity to make a critical advance: The trial proved, for the first time, the safety of the implantation into the lumbar spinal cord of specialized stem cells-neural progenitors-engineered to express a powerful growth factor known to protect neurons. “She made this research a priority, and she was really proud of it,” Courtney says. Moments after Ashley’s death, in May 2021 at a hospital in Oregon, her daughter, Courtney Fisher Olsen, relayed to a nurse the urgent instructions impressed upon her in the previous months: Call Cedars-Sinai and ask them to collect Ashley’s spinal tissue. Their goal: to test the safety of a combination stem cell/gene therapy to treat the rare neurodegenerative disease, caused by the unexplained, unstoppable death of motor neurons in the brain and spinal cord. So, she sought out what she could do with the time she had left: She enrolled in a clinical trial.Įven after paralysis stole her speech, Ashley consented to a five-hour spine operation, took immunosuppressive drugs for a year and underwent extensive testing at Cedars-Sinai’s ALS Clinic, where a team monitored the impact of the procedure on her body-specifically, one of her legs. During the following three years, as the unstoppable disease took hold of her body, she lost the ability to work, hike by the beach or shop swap meets on Saturday mornings. Ashley Fisher was 48 when she was diagnosed with amyotrophic lateral sclerosis (ALS).
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