How Patient-Derived Cells Are Changing ALS Research

Research Investigator Julia Kaye, PhD, stands for a portrait in a lab at Gladstone Institutes. Photo courtesy of Gladstone Institutes. Before there can be any therapies for disease, scientists need to understand what goes wrong in the first place. Are cells lost that could be replaced? Is there too much or too little of a … Continue reading How Patient-Derived Cells Are Changing ALS Research

Remembering CIRM board member and ALS patient advocate Fred Fisher

CIRM honors the contributions of Fred Fisher, MSW, LCSW, a dedicated member of our Independent Citizensโ€™ Oversight Committee (ICOC) and an enthusiastic patient advocate. Fred passed away in September. He joined the ICOC as the ALS patient advocate in June 2021 and served in many roles, including coโ€‘chair of the Standards Working Group and member … Continue reading Remembering CIRM board member and ALS patient advocate Fred Fisher

CIRM-funded stem cell-gene therapy shows promise in ALS safety trial

Senior author of the study Clive Svendsen, PhD (center) With funding support from the California Institute for Regenerative Medicine (CIRM), Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons … Continue reading CIRM-funded stem cell-gene therapy shows promise in ALS safety trial

The present and future of regenerative medicine

One of the great pleasures of my job is getting to meet the high school students who take part in our SPARK or Summer Internship to Accelerate Regenerative Medicine Knowledge program. It's a summer internship for high school students where they get to spend a couple of months working in a world class stem cell … Continue reading The present and future of regenerative medicine

How two women are fighting back against Lou Gehrigโ€™s disease

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Mary Ann Wittenberg (left) and Nadia Sethi Lou Gehrig's disease, or ALS, is a nasty degenerative condition that destroys the brain cells controlling movement. People with ALS suffer a progressive loss of ability to walk, talk, eat and breathe. The average life expectancy for someone diagnosed … Continue reading How two women are fighting back against Lou Gehrigโ€™s disease

Joining the movement to fight rare diseases

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Itโ€™s hard to think of something as being rare when it affects up to 30 million Americans and 300 million people worldwide. But the truth is there are more than 6,000 conditions โ€“ those affecting 200,000 people or fewer โ€“ that are considered rare.   Today, … Continue reading Joining the movement to fight rare diseases

A step forward in finding a treatment for a deadly neurological disorder

THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST MRI section of a brain affected by ALS with the front section of the brain highlighted Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigโ€™s disease, is a nasty disease that steadily attacks nerve cells in the brain and spinal cord. Itโ€™s pretty much always fatal … Continue reading A step forward in finding a treatment for a deadly neurological disorder

You can’t take it if you don’t make it

Biomedical specialist Mamadou Dialio at work in the Cedars-Sinai Biomanufacturing Center. Photo by Cedars-Sinai. Following the race to develop a vaccine for COVID-19 has been a crash course in learning how complicated creating a new therapy is. Itโ€™s not just the science involved, but the logistics. Coming up with a vaccine that is both safe … Continue reading You can’t take it if you don’t make it

A guide to healing

Dr. Evan Snyder Having grown up in an era where to find your way around you had to use paper maps, a compass and a knowledge of the stars (OK, Iโ€™m not actually that old!) Iโ€™m forever grateful to whoever invented the GPS. Itโ€™s a lifesaver, and I daresay has also saved more than a … Continue reading A guide to healing

A true Hall of Fame winner

Dr. Larry Goldstein: Photo courtesy UCSD You know you are working with some of the finest scientific minds in the world when they get elected to the prestigious National Academy of Sciences (NAS). Itโ€™s the science equivalent of the baseball, football or even Rock and Roll Hall of Fame. People only get in if their … Continue reading A true Hall of Fame winner