discoveries magazine
Discoveries

Hope and ALS

There is no cure for amyotrophic lateral sclerosis (ALS), a usually fatal neurodegenerative disease that mainly strikes when a person is between 40 and 70 years old. Patients with ALS become slowly paralyzed when they lose motor neurons that control movement—but scientists don't yet understand what causes the neurons to die.

Existing treatments only extend lifespans by a few months and very few are on the market—the first ALS drug was approved by the Food and Drug Adminstration in 1995, and a second became available only 22 years later. So at Cedars-Sinai's weekly ALS Clinic, care focuses on diagnosis, symptom management and providing optimal support for patients and their families.

Every three months, a team of physicians, therapists, a social worker and other specialists evaluate patients' evolving needs and equip them with new machines, medicines or services to maintain and improve their quality of life.

But investigators are inching toward a greater understanding of the disease, made possible by ALS patients who take an active role in research. The ALS Clinic at Cedars-Sinai is running 13 ongoing trials involving about 170 patients, including studies into stem cell therapy.



One approach pioneered at Cedars-Sinai is the latest project in a line of study that has been ongoing for 12 years. In a new trial, a surgeon will transplant neural stem cells modified to release a powerful growth factor that can protect motor neurons directly into the brain of patients with ALS.

A previous study showed that these cells can be safely transplanted into the spinal cords of patients. As a secondary outcome, clinicians will be able to monitor patients' hand movements to determine whether the stem cells can slow down neuron death and help maintain that movement over time.

Investigators say the potential to find new treatments for ALS depends largely on the generosity and trust of patients in clinical trials—patients say the trials act as balm even when they know the study will not cure the disease. Here, people who live and work with ALS share what drives them to continue seeking solutions despite decades of only incremental progress.

ALS Clinic physician, principal investigator of the new stem cell trial


Youstina Makary

ALS clinical trial coordinator


Eric Kentor

ALS patient and clinic trial participant





Executive director of the Board of Governors Regenerative Medicine Institute, who initiated the stem cell trials