Often, after decades of intensive scientific research, even the most promising potential new therapies come down to a critical final phase: courageous people stepping forward to participate in a clinical trial.

For Clinical Trials Day this year, we’re celebrating a few of the many patients who inspire us, while highlighting important things you should know about this crucial step in developing new treatments and cures.

Incredibly, there are more than half a million registered clinical trials worldwide. Researchers and regulators use them to learn whether a new medicine is safe and effective with careful monitoring at each step.

As part of its mission, the California Institute for Regenerative Medicine (CIRM) helps fund and support more than 100 clinical trials, as well as nine Alpha Clinics across the state that offer clinical trials. California voters created CIRM in 2004 to develop life-changing therapies for the people of California. CIRM’s unique model of state-funded support for the development of these treatments from basic research all the way through late-stage clinical trials has become a model for other states.

Through the Alpha Clinics — and a network of Community Care Centers of Excellence in smaller communities — that dot California, CIRM also makes treatment more accessible for people who may not live near a large urban center like San Francisco, Los Angeles, or San Diego.

Clinical trials and safety 

Even before a potential therapy begins a clinical trial, scientists have spent years researching and testing it before it’s ever used in people.

Once they’ve cleared those initial hurdles in the lab, scientists then carefully design a clinical trial with one core principle in mind: patient safety.

Regulators then review the study design before it begins. Whether it’s gene therapy, stem cell therapy, or cell-based cures, these trials move in phases. The researchers report the results from each phase of the trial.

But for the trial to proceed, there must be patients willing to volunteer. Although designed with safety in mind, clinical trials still involve an element of the unknown for both patients and researchers.

Deciding to participate 

Will the new therapy work? How might it impact a patient trying it for the first time?

Once informed of the risks and given a clear idea of what their participation will entail, the decision is up to the patient or their guardians. In 2019, Jordan Janz became the first person to participate in a clinical trial for a new gene therapy to treat cystinosis. Cystinosis is a rare disorder that can lead to organ failure, blindness, and, in some cases, premature death.

“It was a little scary, right, like you don’t know what to expect,” Janz said recently during a webinar. He’s doing well now and has a tough time remembering what his life was like before the treatment.

Janz decided to move forward not just because he hoped the treatment would help him, but also because his participation could benefit others with the condition. But knowing about the support from the lead researchers, as well as the nurses and doctors overseeing the program, made his decision to participate easier.

“The team was amazing,” he said.

Partners in research

Today’s trials aren’t passive or one-sided. Patients have the unique opportunity to help shape the research they might benefit from by sharing their experience and asking questions. For researchers, understanding patients’ lived experiences in a clinical trial helps refine treatment protocols, dosing, and safety monitoring.

Understanding the effectiveness of a specific treatment requires that researchers include a broad range of participants of different backgrounds, ages, and ethnicities. And understand how they each respond.

Justin Graves lived with epileptic seizures for years before enrolling in a CIRM-funded clinical trial in 2023. Within a month after his experimental treatment began, his most severe seizures ended.

“I love the idea of being able to help not just a few people with some issues, but the entire world,” Graves said about his participation in the clinical trial. 

Clinics aimed at expanding participation  

Alpha Clinics improve access for more people from across California.

These clinics also have coordinators, education support, and patient navigators who help participants throughout treatment. This approach reduces stress, removes barriers, and ensures that people feel respected and supported.

Built on science, clinical trials also depend on relationships between researchers and patients, and their families, often during the most stressful moments of their lives. When someone joins a clinical trial, they can help themselves and many, many others. 

When she was already seven months pregnant, Alicia Langenhop and her husband Jon learned that their third child, Landon, had a life-threatening immune disease called leukocyte adhesion deficiency-1 (LAD-1). They quickly learned that his sisters Ava, 3, and Olivia, 2, also had the condition.

In 2019, Alicia and Jon enrolled all three of their children in a CIRM-funded clinical trial. The trial included patients at several sites with three principal investors. The process was and is overseen by Dr. Donald Kohn,  a professor of microbiology, immunology, and molecular genetics at the University of California, Los Angeles.

“It was a big decision, but we’ve always trusted science and medicine,” Alicia said.  “I hope that this medical advancement becomes available for everyone.”

Indeed, in March of 2026, the therapy to treat LAD-1 — made possible in part by the participation of the Langenhops in a clinical trial — received FDA approval. Their bravery ensures that doctors have this new treatment to offer others facing a similar life-threatening diagnosis in the future.

You can explore CIRM-supported clinical trials here. 

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