CIRM Approves Three Clinical Trials for Rare Disorders as CA Voters Extend Funding

Rare Daily Staff

As California continued to count election ballots, voters passed Proposition 14, which allows the state to raise up to $5.5 billion to support the work of the landmark California Institute for Regenerative Medicine (CIRM), which just approved four new clinical trials and ten new discovery research awards.

“Today is a momentous occasion as CIRM reaches 51 new clinical trials, surpassing one of the goals outlined in its five year strategic plan,” says Maria Millan, president and CEO of CIRM. “These four new trials, which implement innovative approaches in the field of regenerative medicine, reflect CIRM’s ever expanding and diverse clinical portfolio.”

Three of the four new clinical trials address rare disorders. CIRM awarded $3.7 million to Gayatri Rao from Rocket Pharmaceuticals to conduct a clinical trial using a gene therapy for infantile malignant osteopetrosis (IMO), a rare and life-threatening disorder that develops in infancy.  IMO is caused by defective bone cell function, which results in blindness, deafness, bone marrow failure, and death very early in life.

The trial will use a gene therapy that targets IMO caused by mutations in the TCIRG1 gene.  The team will take a young child’s own blood stem cells and inserting a functional version of the TCIRG1 gene.  The newly corrected blood stem cells are then introduced back into the child, with the hope of halting or preventing the progression of IMO in young children before much damage can occur.

Rocket Pharmaceuticals has used the same gene therapy approach for modifying blood stem cells in a separate CIRM funded trial for a rare pediatric disease, which has shown promising results.

Diana Farmer at UC Davis will receive almost $9 million to conduct a clinical trial of in utero repair of myelomeningocele (MMC), the most severe form of spina bifida. MMC is a birth defect that occurs due to incomplete closure of the developing spinal cord, resulting in neurological damage to the exposed cord.  This damage leads to lifelong lower body paralysis, and bladder and bowel dysfunction.

Farmer and her team will use placenta tissue to generate mesenchymal stem cells (MSCs). The newly generated MSCs will be seeded onto an FDA approved dural graft and the product will be applied to the spinal cord while the infant is still developing in the womb. The goal of this therapy is to help promote proper spinal cord formation and improve motor function, bladder function, and bowel function. The clinical trial builds upon the work of CIRM funded preclinical research.

Finally, David Williams at Boston Children’s Hospital will receive $8.3 million to conduct a gene therapy clinical trial for sickle cell disease (SCD). This is the second project that is part of an agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, to co-fund cell and gene therapy programs under the NHLBI’s  “Cure Sickle Cell” Initiative. The goal of this agreement is to markedly accelerate clinical development of cell and gene therapies to cure SCD.

SCD is an inherited disease caused by a single gene mutation resulting in abnormal hemoglobin, which causes red blood cells to ‘sickle’ in shape. Sickling of red blood cells clogs blood vessels and leads to progressive organ damage, pain crises, reduced quality of life, and early death.

The team will take a patient’s own blood stem cells and insert a novel engineered gene to silence abnormal hemoglobin and induce normal fetal hemoglobin expression. The modified blood stem cells will then be reintroduced back into the patient. The goal of this therapy is to aid in the production of normal shaped red blood cells, thereby reducing the severity of the disease.

Photo: Maria Millan, president and CEO of CIRM