The California Institute for Regenerative Medicine (CIRM) board approved $16 million for more than a dozen Discovery‑stage research programs. These projects span Parkinson’s disease, new drug targets for cancer stem cells, and methods for creating human airway stem cells.
Dr. Maria Millan, CIRM’s President and CEO, said the agency backs this work because breakthrough ideas often start in early research:
“This research is essential for advancing stem cell knowledge and forming the foundation for future therapies. Exploring and testing new ideas increases the chances of finding treatments for patients with unmet medical needs. Without CIRM’s support, many of these projects might never begin. That’s why funding early‑stage research is so important for the field.”
Discovery Inception Awards
At our Board meeting last week David Higgins, our Board member and Patient Advocate for Parkinson’s disease, made that point as he championed one project that is taking a new approach to finding treatments for the condition. As he said in a news release:
“I’m a fourth‑generation Parkinson’s patient, and I’m taking the same medicines my grandmother took. They help, but not everyone benefits and they don’t work long enough. People with Parkinson’s need new treatment options now. That’s why this project matters. It could identify promising candidates that may lead to new therapies.”
Zenobia Therapeutics received $150,000 through our Discovery Inception program, which supports bold early‑stage ideas that need initial funding to show their potential impact on stem cell research.
Zenobia plans to generate induced pluripotent stem cells (iPSCs) and turn them into dopaminergic neurons—the brain cells affected in Parkinson’s. They will use these cells to screen hundreds of compounds and look for therapy candidates. Testing compounds on human neurons, rather than animal models, could improve the chances of finding an effective treatment.
Zenobia’s project is one of 14 Discovery Inception awards approved. The full list in our news release highlights a range of innovative ideas, from creating human airway stem cells for new respiratory disease treatments to developing a novel drug that targets cancer stem cells.
Other Projects in Translational Stage
The CIRM Board approved $13.4 million for three Translation-stage projects. These programs showed promise in early research and need funding to move to the next stage.
• $5.56 million to Anthony Oro at Stanford to test a stem cell therapy for Epidermolysis bullosa, a painful blistering skin disease with wounds that don’t heal.
• $5.15 million to Dan Kaufman at UC San Diego to generate natural killer (NK) cells from embryonic stem cells and test them in patients with treatment‑resistant acute myelogenous leukemia (AML).
• $2.7 million to Catriona Jamieson at UC San Diego to test a new therapeutic approach that targets cancer stem cells in AML, which likely drive the disease’s high relapse rate.
CIRM aims to build a pipeline of projects that can ultimately help patients. We fund research from early Discovery through Translation and, ultimately, into clinical trials.
Victor Hugo wrote, “There is one thing stronger than all the armies in the world, and that is an idea whose time has come.”
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There has actually been remarkable progress on potentially curative stem cell-based therapy for Parkinson’s disease. Clinical trials for dopamine neuron replacement therapies will begin next year by groups belonging to GForce PD, a partnership to coordinate methods and results in similar studies. NYSTEM, New York state’s stem cell agency, has invested more than $20 million in a project to use dopamine neurons derived from human embryonic stem cells that will start clinical trials next year. The European Union has invested in a similar hESC approach in Sweden and the UK, also providing more than $20 million. The Japanese government has invested at least as much in a similar trial that uses non-matched iPSCs.
There is hope for Californians to also have access to this kind of therapy. The fourth member of GForce is my lab in California, who have used mostly private donations to develop a patient-matching autologous neuron replacement therapy for Californians suffering from the disease. But CIRM has invested only $2.4 million in our project, not nearly enough to do the studies required by the FDA, so we’ve had to do a lot of outside fundraising. But we still plan to start our clinical trial in 2019.
“Victory over Parkinson’s disease” is the mission of our largest private donor, the Summit for Stem Cell Foundation. http://www.summitforstemcell.org