On Jul. 25, 2024, the California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, awarded nearly $35 million to fund clinical research to address a rare plasma cell disorder and six new Shared Resources Laboratories (SLR), an important component of CIRM’s infrastructure program.

The award was made through the Agency’s clinical program, which provided funding for eligible stem cell and gene therapy-based projects through all stages of clinical trial development. This award brought the number of CIRM-funded clinical trials to 109.

The $8 million clinical award was given to Ilya Rachman, MD, PhD, of Nexcella, to advance a therapy aimed at treating Amyloid light-chain amyloidosis (AL amyloidosis), a rare plasma cell disorder in which the body’s immune system produces abnormal forms of antibodies called “light chains.”

In the United States, approximately 4,000 new cases of AL amyloidosis are diagnosed annually. Patients with this condition face a significant reduction in their quality of life due to the debilitating symptoms and the need for frequent medical interventions. The burden of living with a rare disease also places emotional and financial strains on patients and their families.

There is no cure for amyloidosis and the current standard of care is ineffective in preventing the disease progression. The proposed therapy aims to eliminate the faulty plasma cells with a single treatment. It uses the patient’s own immune cells, which are genetically altered, to precisely target and destroy the problematic cells.