A Light in the Darkness: CIRM Funds Gene and Cell Therapy Research

I fear that 2025 is ending with a tragic irony: the dismantling of progress in biomedical research, as infectious diseases return and resurge. Arrogance and ignorance are proving to be more dangerous pathogens than bacteria and viruses.

So I thought I’d close out the year with an uplifting look at a funding source not threatened by the current administration: the California Institute of Regenerative Medicine, aka CIRM.

Two Decades of Funding Research, Needed More Now Than Ever

CIRM was born 20 years ago, when 59 percent of California voters approved Proposition 71, and reaffirmed it in 2020. Since then, the taxpayer-supported organization has provided more than 1400 grants, totaling nearly $4 billion.

Grants support all stops on the journey from initial idea to delivering a new treatment. That means funding basic research at universities and supporting education, collaboration, scientific and medical meetings, as well as manufacturing facilities, clinics, hospitals, and community outreach programs.

“CIRM recognizes that making a medical breakthrough is only the beginning of the complex process of delivering new regenerative medicine therapies. That’s why CIRM funds regenerative medicine research and clinical trials, while simultaneously strengthening and expanding the educational opportunities for future members of the regenerative medicine workforce, and addressing barriers that might prevent patients in California from benefiting from cell and gene therapies,” said Chief Science Officer Rosa Canet-Avilés. Many of the funded programs include participation in states other than California.

From the Rare …

The gene and cell therapies that CIRM supports range from ultra-rare single-gene conditions to prevalent cancers, neurological disorders, and more.

A good example is a recent award to support development and testing of a gene therapy for CMT4J, a very rare form of Charcot-Marie-Tooth (CMT) disease, which is similar to ALS. DNA Science told the story of young Talia Duff and her family’s efforts to get a research collaboration and clinical trial up and running, through the organization CureCMT4J, back in 2017, here.

Amassing the funds and expertise to kickstart a clinical trial takes years. Elpida Therapeutics is developing the gene therapy.

Executive director of CureCMT4J Jocelyn Duff points out that the approach may be useful for the other hundred or so subtypes of CMJ, and perhaps even more prevalent neuromuscular conditions like ALS and Parkinson’s subtypes.

… To the Common

CIRM is also funding studies of common disorders – I found one mentioned on their website that I have, knee osteoarthritis. It affects some 30 million of us in the US.

I received my first steroid injection a few months ago, and recently returned from nursing my best friend through her total knee replacement surgery, following seven years of steroid and hyaluronic acid gel injections. You can’t get these shots forever – eventually, they rot the joint.     

I’d like to avoid knee replacement. Facebook groups, which of course attract the worst-case scenarios, offer horror stories of pain, infection, and very slow recoveries. So I’m hoping that the gene therapy that CIRM is supporting will be approved in time for me.

Developed by Genascence Corp, the gene therapy introduces copies of a gene that encodes an antagonist to the interleukin-1 receptor. And that blocks binding of the cytokine interleukin-1 (IL-1), which triggers the pain and swelling (inflammation). The gene is delivered aboard adeno-associated virus, a commonly deployed gene therapy vector.

The anti-inflammatory gene therapy approach for knee osteoarthritis was first tried in the finger joints of patients with rheumatoid arthritis, a more severe condition. If successful, the gene therapy for knee OI will only be required once to quell the inflammation, joint pain, and cartilage destruction.

I am thankful that organizations such as CIRM are complementing dwindling federal support for valid, promising, and scientifically logical new treatments.

A happy and healthy New Year!