
From the start, CIRM has aimed to advance stem cell research in California. We do this by funding promising work—reflected in our 51 clinical trials—and by bringing top scientists together to solve problems.
We have organized conferences and workshops on Parkinson’s disease, cerebral palsy, and tissue engineering. Each event gathered key leaders to brainstorm solutions and explore new therapeutic approaches. We encourage scientists who usually compete to partner and find the best path forward.
This collaboration is difficult, and results take time. Still, it is essential to our mission of accelerating stem cell therapies for patients with unmet needs.
Last week, we helped organize two major events and participated in a third.
The first, co‑hosted with Cedars‑Sinai, was the “Brainstorm Neurodegeneration” workshop. It brought together experts in stem cell science, genomics, big data, patient advocacy, and the FDA. They tackled challenges slowing progress on diseases such as Parkinson’s, Alzheimer’s, ALS, and Huntington’s.
We called the workshop “a cutting‑edge meeting to disrupt the field.” While two days of discussion didn’t solve every issue, they produced strong ideas and promising paths forward.

Two days later, we partnered with UC San Francisco to host the Fourth Annual CIRM Alpha Stem Cell Clinics Network Symposium. This brought together the scientists who develop therapies, the doctors and nurses who deliver them, and the patients who are in need of them. The theme was “The Past, Present & Future of Regenerative Medicine” and included both a look at the initial discoveries in gene therapy that led us to where we are now as well as a look to the future when cellular therapies, we believe, will become a routine option for patients.
Bringing these different groups together is important for us. We feel each has a key role to play in moving these projects and out of the lab and into clinical trials and that it is only by working together that they can succeed in producing the treatments and cures patients so desperately need.

As always, the patients surprised us. Cierra Danielle Jackson described what it felt like to be cured of sickle cell disease. Many expected her to focus only on the joy of losing a painful, life‑threatening condition. She did—but she also described the challenge of adjusting to a new identity.
Sickle cell had shaped every part of her life and relationships. Losing it created an identity crisis. Who was she now without the disease?
She noted that most patients return to a previous “normal” after treatment. But people with sickle cell have never known life without illness. Their normal disappears, and they must build a new one.
Her story reminded everyone that new treatments must address the whole person—their emotional and psychological needs, not just the physical.

The third event was the Stanford Drug Discovery Symposium. This invitation‑only meeting included leading scientists such as Nobel laureates Paul Berg and Randy Schekman and former FDA Commissioner Robert Califf. Over two days, speakers discussed philanthropy’s role in research, the rise of immunotherapy, and how artificial intelligence and big data shape medicine.
CIRM President and CEO Dr. Maria Millan spoke about California’s stem cell investment. She described how this work delivers “Something Better than Hope,” the title of our 2018 Annual Report. She highlighted several of the 51 clinical trials we funded and the lives changed by those efforts.
The presentations mattered, but so did the conversations outside the auditorium. Many collaborations begin over lunch or coffee, or when researchers hear directly from patients about trial needs.
Bringing together people who might never meet often sparks ideas that can change the world.
Was the need for stem cell-specific dose determination discussed, noted, mentioned? How can the results of stem cell clinical trails be interpreted soundly without knowing the stem cell-specific dose of treatments?
Hi James, that’s the importance of taking part in a clinical trial given the go-ahead by the FDA. They, and the researchers, agree on dosing level or levels, timing of administration, follow-up etc. Without those parameters the results, as you rightly point out, mean very little.