The governing Board of the California Institute for Regenerative Medicine (CIRM) awarded two grants totaling $11.15 million to carry out two new clinical trials for children with Leukocyte Adhesion Deficiency-I (LAD-I) and soft tissue sarcoma.

These latest additions bring the total number of CIRM-funded clinical trials to 53. 

LAD-I

The awards include $6.56 million for Rocket Pharmaceuticals to run a clinical trial for infants with LAD‑I.

LAD‑I is a rare pediatric disease caused by a gene mutation that impairs the body’s ability to fight infections. Infants with severe LAD‑I often become ill immediately after birth. They suffer recurrent, life‑threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Mortality for severe LAD‑I is 60–75 percent before age two, and survival beyond age five is rare.

Rocket Pharmaceuticals will test a treatment that uses a patient’s own blood stem cells and inserts a functional version of the defective gene. After modification, the stem cells are returned to the patient to generate healthy immune cells capable of fighting infection.

This CLIN2 award will support a clinical trial to assess the treatment’s safety and effectiveness in patients with LAD‑I.

The project uses a gene‑therapy strategy similar to three other CIRM‑funded clinical trials at UCLA led by Dr. Don Kohn for X‑linked chronic granulomatous disease, ADA‑SCID, and sickle cell disease.

Soft tissue sarcoma

UCLA was awarded an additional $4.59 million to fund a clinical trial led by Dr. Theodore Nowicki for sarcomas and other advanced solid tumors. In 2018, an estimated 13,040 people in the U.S. were diagnosed with soft tissue sarcoma, and about 5,150 died. Standard treatment includes surgery, radiation, and chemotherapy, but patients with late‑stage or recurrent disease have few options.

Dr. Nowicki’s team will genetically modify peripheral blood stem cells and mononuclear cells to target solid tumors. The modified stem cells can self‑renew, offering the potential for durable benefit.

This CLIN2 award will support a clinical trial to assess the safety of this rare solid‑tumor therapy.

The project expands CIRM’s work in stem‑cell‑based cancer treatments. A previously funded UCLA trial uses the same approach to treat multiple myeloma.  CIRM has also funded two clinical trials using different approaches to target other solid tumors, one at Stanford and the other at UCLA. Lastly, two additional CIRM-funded trials conducted by City of Hope and Poseida Therapeutics, Inc. used modified T cells to treat brain cancer and multiple myeloma, respectively.

“CIRM has funded 23 clinical-stage programs utilizing cell and gene medicine approaches,” says Maria T. Millan, M.D., the President and CEO of CIRM. “The addition of these two programs, one in immunodeficiency and the other for the treatment of malignancy, broadens the scope of unmet medical need we can impact with cell and gene therapeutic approaches.”