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LOS ANGELES (Oct. 24, 2024) -- Clinicians, researchers and the public are invited to learn about stem cell and gene therapies being developed throughout California at the , hosted by Cedars-Sinai on Oct. 25.

“This meeting isn’t just for scientists,” said , program director of The Alpha Clinic at Cedars-Sinai and professor of Medicine. “It is for clinicians, patients and members of the public who are interested in learning more about cell and gene therapies, emerging technologies, innovative clinical trial designs and issues surrounding accessibility to these therapies for patients, such as financial barriers.”  

The symposium, sponsored by the California Institute for Regenerative Medicine (CIRM), will take place from 9 a.m.-5 p.m. at the Cedars-Sinai Silver Screen Theater in the Pacific Design Center. Virtual registration is also available.

Scientists from all nine California Alpha Clinics are set to speak. Along with Cedars-Sinai, institutions with alpha clinics include University of California, San Diego; University of California, Los Angeles; University of Southern California and Children’s Hospital Los Angeles; Stanford University; University of California, Irvine; University of California, Davis; City of Hope; and University of California, San Francisco.

Directors of several Alpha Clinics will moderate panel discussions, and directors of the Alpha Clinics at UC Irvine, UC San Diego, University of Southern California and UC Davis will participate in a discussion of Alpha Clinic operations. Daniela A. Bota, MD, PhD, director of the UC Irvine Alpha Clinic, will talk about chimeric antigen receptor (CAR) therapies, and Mark Walters, MD, UC San Francisco Alpha Clinic director, will discuss current gene therapies, future opportunities and barriers to treatment. 

Lewis expects speakers to highlight the importance of diversity in clinical trials, promote collaboration among researchers and showcase advances in cell and gene therapies.

“The symposium will look to the current status of the field and, more importantly, to the future, as new approaches and therapies are likely to rapidly expand,” said Clive Svendsen, PhD, co-associate project director of Cedars-Sinai's Alpha Clinic and executive director of the  at Cedars-Sinai. Svendsen recently co-authored a review paper published in  about stem cell-based therapies for neurological conditions.

The review paper describes stem cell-based treatments for a number of neurological disorders, including amyotrophic lateral sclerosis, also known as ALS. The condition affects nerve cells in the brain and spinal cord that control movement. ALS eventually causes loss of the ability to move, speak, eat and breathe. At this time there is no cure.

The Cedars-Sinai Alpha Clinic is conducting a trial of a therapy, developed by Svendsen and his team, using specially engineered cells to protect damaged motor neurons. To date, six patients have been treated, with no major complications.

Cedars-Sinai’s Alpha Clinic and others throughout California are developing stem-cell based therapies for a variety of conditions: 

• Cedars-Sinai is studying a therapy for retinitis pigmentosa, which breaks down cells in the light-sensitive layer at the back of the eye, the retina, leading to vision loss. The therapeutic cells in the trial release proteins that help protect retinal cells from damage.

• Cedars-Sinai investigators also are testing a treatment for , a rare condition in which elevated blood pressure in lung blood vessels can damage the heart. The initial study was led by Lewis, director of Respiratory Care Services at Cedars-Sinai, and , executive director of the  and associate project director of The Alpha Clinic. Investigators concluded that infusions of specialized cell products derived from heart tissue seemed to improve patients’ heart and lung health.
• Alpha Clinics throughout California are studying stem cell-based therapies for several other conditions—including movement disorders such as Parkinson’s disease and Huntington’s disease; Alzheimer’s disease; stroke; epilepsy; spinal cord injury; and multiple sclerosis.

“The Alpha Clinic network is dedicated to accelerating the work of institutions in California, such as Cedars-Sinai, that are developing and testing cell and gene therapies,” Marbán said. “It is a highly collaborative group, and we share resources including our expertise, our infrastructure and our experience with cell delivery to help each other.”

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