Community Stories

Contributions that can be life changing

At CIRM, community voices are essential to our mission. Patients play a critical role in advancing stem cell and gene therapies by participating in clinical trials and providing valuable insights.

Their perspectives help ensure that research reflects the needs of diverse communities, guiding the development of therapies that are accessible and effective for everyone. Read more patient stories in CIRM’s Stem Cellar blog.

Real Stories, Real Impact

The patients in CIRM-funded clinical trials are making a real difference in advancing regenerative medicine.

Their experiences help shape the development of treatments that can improve lives and address unmet medical needs.

Listen to their voices

“I’m taking time out of life to advance science forward.”

“I want to contribute to science and help others – a lot of us are not doing this for us.”

“Small changes are necessary to progress science.”

“A few levels can be life changing.”

“CIRM opens up new frontiers and possibilities that once only existed in people’s dreams.”

“CIRM listens to what we want. This is about what the people want, and they hear us.”

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Veronica McDougall

Veronica was diagnosed with retinitis pigmentosa (RP), a rare degenerative condition of the retina that would eventually leave her legally blind. She enrolled in a clinical trial at UC Irvine funded by CIRM and led by the biotech company jCyte. In the trial, a dedicated research team injected retinal progenitor cells into her left eye, leading to steady vision improvement.

Photo Courtesy of Andrea Fernandez de Soto

Jakob Guziak

For Andrea Fernandez de Soto and her family in Alberta, Canada, the journey with ADA-SCID began when her son, Jakob, was diagnosed 10 days after birth. For the first few years of his life, Jakob relied on weekly enzyme replacement therapy to survive while his family waited for a more permanent solution. That solution came in the form of a gene therapy funded by CIRM.

Hataalii Tiisyatonii (“HT”) Begay

Shortly after his birth in the Navajo nation, Hataalii Tiisyatonii (“HT”) Begay was diagnosed with Artemis Severe Combined Immunodeficiency Disease (SCID). He was the first child to participate in a CIRM-funded UCSF trial where lead investigators collected his own blood stem cells, modified them with a healthy version of the defective gene, and then re-infuse the corrected cells back into his body to rebuild his immune system.

Photo Credit: Barbara Ries for UCSF

Photo Credit: Jaquell Chandler

Evie Junior

Evie Junior was born with sickle cell disease, a life-threatening condition that affects 100,000 Americans, most of them Black and Latino. People with sickle cell disease have blood cells that are shaped like a hook (or sickle) rather than smooth and round, which can create clogs causing intense pain, organ damage, and stroke.