Nearly $12M Grant Will Fund ALS Cell Therapy Trial at Cedars-Sinai

Cedars-Sinai Medical Center, in California, has been awarded an $11.99 million grant to support a clinical trial that will test specifically engineered neural progenitor cells as a potential therapy for amyotrophic lateral sclerosis (ALS).

The work, funded by the California Institute for Regenerative Medicine, or CIRM, will build on years of prior research at Cedars-Sinai — also supported by CIRM, a state agency created in 2004 to fund stem cell research — that showed a similar stem cell-based technique was effective in rodent models of ALS.

“We are extremely grateful for CIRM’s support of our ALS work over many years,” Clive Svendsen, PhD, executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, said in a press release.

“The new grant allows us to follow the science wherever it takes to best serve our patients,” added Svendsen, also a professor of biomedical sciences and medicine.

He noted that, despite years of research, ALS “remains a remarkably stubborn and heartbreaking disorder” with no known cure and limited treatments.

The Cedar-Sinai trial expects to enroll 16 ALS patients, who will have neural progenitor cells transplanted into specific parts of their brain. As the name implies, neural progenitor cells are able to grow and differentiate into other kinds of nervous system cells, including neurons (nerve cells) and astrocytes.

Neurons are the fiber-like cells that send electrical signals through the nervous system. ALS is characterized by the death of a specific set of neurons, called motor neurons, that control voluntary movement. Astrocytes, meanwhile, are star-shaped cells that help to support the health and activity of neurons. Their supportive abilities are impaired in ALS.

Researchers think that, once the neural progenitor cells are transplanted into patients, they will differentiate into astrocytes. The cells also will be engineered to secrete high levels of a protein called glial cell line-derived neurotrophic factor or GDNF, a signaling molecule that promotes the survival of astrocytes.

The overall aim of the project is to improve the health of neurons and astrocytes in patients’ brains, thereby slowing the progression of ALS. The clinical trial will mainly be testing the procedure’s safety, but preliminary measurements of efficacy also will be assessed.

Scientists at Cedar-Sinai previously conducted a similar trial, in which neural progenitor cells engineered to produce GDNF were transplanted into participants’ spinal cords. Results from that trial, which also was supported by CIRM, are still under analysis.

“This new major CIRM award recognizes and supports Cedars-Sinai’s decade-long drive to create ALS therapies using unique methodologies developed at our institution,” said Jeffrey A. Golden, MD, vice dean of research and graduate education and director of the Burns and Allen Research Institute at Cedars-Sinai, who praised his team’s “innovative effort.”