After 21 years (and a few months), the California Institute of Regenerative Medicine (CIRM) has hit a major milestone: the first therapy supported by CIRM has been approved by the Food and Drug Administration for use in patients.

In 2004, when California voters created CIRM to fund research with stem cells — cells that can divide to form many different tissues in the body — stem cells held great potential, but scientists were still learning how to work with them in the lab. CIRM helped rapidly expand our understanding of stem cells and how they can be used in therapies. In 2020 when voters extended CIRM’s mandate to include gene therapy, which involves modifying genes to treat disease, that field was only recently emerging from a decades-long stand-still resulting from early safety concerns.

The fact that it took only 21 years to go from basic questions and concerns to an approved therapy is thanks to long hours and persistence on the part of dedicated scientists, lab workers, and students, as well as collaboration with regulators, funders (like CIRM), and patient advocates.

The milestone also required patients willing to participate in clinical trials. These participants and their families are heroes whose bravery makes therapies possible. 

This landmark therapy was developed in part through a $5,867,085 award to Rocket Pharmaceuticals, Inc. in support of a clinical trial at the University of California, Los Angeles (UCLA) to test a therapy for severe leukocyte adhesion deficiency-I (severe LAD-I). (Trial sites in London and Madrid were funded separately.) 

LAD-I is a rare genetic disease where a child’s immune system can’t properly fight infections, putting them at risk for serious illness. Children with the disease have recurrent, life-threatening bacterial and fungal infections that respond poorly to antimicrobials and result in frequent hospitalizations. 

We’ve written about some of the children who participated in the trial: Marly Gaskins and the Langenhop kids were all born with LAD-I, and all regained healthy immune function after participating in the UCLA trial.

Although individually rare diseases like LAD-I don’t affect many people, cumulatively rare diseases affect over 30 million people in the U.S., about half of whom are young children with limited expected lifespan. Roughly 1 in 10 Americans have a rare disease, of which 95% have no approved therapy.

While research funding may be uncertain elsewhere, California is expecting to spend hundreds of millions of dollars on research next year, from basic discovery through clinical trials and cures, not to mention training students to develop those therapies today and in the future. Much of this funding is directed toward rare diseases. This kind of sustained funding means LAD-I may be the first disease to have a CIRM-funded approved therapy, but there will be many more to come.

That progress toward therapies is also enabled by CIRM’s network of supported clinical centers that deliver cell and gene therapy clinical trials and approved therapies across the state. In fact, the successful UCLA LAD-1 trial took place within one of these CIRM-funded clinics.

Congratulations and thank you to everyone involved in making this therapy a reality for people with LAD-I and their families.