State agency launches ‘Championing Change for Sickle Cell in California’ campaign

/ California Institute for Regenerative Medicine (CIRM)

CIRM has launched “Championing Change for Sickle Cell in California,” a campaign to raise awareness of sickle cell disease and support research aimed at improving treatment options.

More on sickle cell

More than 100,000 Americans and 20 million people worldwide live with this debilitating condition. CIRM is committed to funding advanced cell and gene therapy programs that may transform care for people with SCD.

Sickle cell disease is a genetic disorder that causes red blood cells to take on a sickle shape under stress. These cells block blood flow, trigger severe pain crises, and damage organs.

This disease disproportionately affects people of African American and Latinx heritage. It is estimated that about one in 365 Black or African American newborns has sickle cell disease. Latinx Californians make up 7.3 percent of cases, and Latinx newborns account for 11 percent of new diagnoses.

Collaborations

In collaboration with groups such as Axis Advocacy, the NHLBI Cure Sickle Cell Initiative, and the UCSF Sickle Cell Center of Excellence, CIRM is working to champion change and advance sickle cell treatments.

Flyers and social media graphics are available for download at CIRM’s website.

Community partners and patient advocates play a crucial role in advancing these treatments, and CIRM recognizes their importance. Key calls to action include becoming a sickle cell advocate, sharing information about clinical trial options, downloading a fact sheet, and learning about the NHLBI Cure Sickle Cell Initiative.

Sickle cell research

The campaign also highlights how CIRM funds stem cell, gene therapy, and other regenerative medicine approaches for sickle cell disease. To date, CIRM has invested more than $65 million in sickle cell research, from basic discovery through clinical development.l trials.

Evie Junior participated in a CIRM-funded clinical trial for sickle cell disease at UCLA. Photo: Jaquell Chandler

CIRM and the NHLBI are co-funding two clinical trials that are investigating different gene therapy approaches for patients with severe sickle cell disease. These trials include a Phase 1/2 trial led by Dr. Mark Walters of UCSF Benioff Children’s Hospital Oakland and a Phase 2 trial led by Dr. David A. Williams of Boston Children’s Hospital. These clinical trials are essential in the search for new therapies to treat sickle cell disease.

CIRM’s new campaign comes on the heels of two recently FDA-approved gene therapy treatments for sickle cell disease. Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have brought forth a new era of treatment options for people living with the condition.

CIRM introduced the “Championing Change for Sickle Cell in California” campaign at Oakland’s Black Joy Parade in February 2023. Pictured is CIRM’s Community Outreach Manager Aditi Desai.

“As the first regulatory authorization for a CRISPR-based gene-editing therapy in the world, this approval not only marks a milestone in the advancement of cell and gene therapies, but it also marks a transformative step in the treatment landscape for the millions of people impacted by sickle cell disease,” said CIRM Vice President of Therapeutics Development, Abla Creasey, PhD.

For more information on CIRM’s Sickle Cell Disease campaign, ongoing research projects, and ways to champion change, please visit cirm.ca.gov/sickle-cell-disease. To get involved in the campaign, please contact CIRM’s Community Outreach Manager Aditi Desai at adesai@cirm.ca.gov.

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