Geron gets go-ahead for first hESC trial

US biotech Geron has received clearance from the FDA for a clinical trial of human embryonic stem cell-based therapy in patients with acute spinal cord injury.

The Californian company said it expected to start a Phase I safety trial of its product, GRNOPC1, which contains hESC-derived oligodendrocyte progenitor cells, in the northern summer.

The trial will be recruit a small number of patients with complete Grade A subacute thoracic spinal cord damage within seven to 14 days of the injury. While safety is the main endpoint, the company said the study would also assess efficacy, such as improved neuromuscular control or sensation in the trunk or lower extremities.

If successful, the trial will be extended to patients with cervical spinal cord injuries and incomplete injuries.

The company’s application included research showing improved axon survival and extensive remyelination surrounding the rat axons. Oligodendrocyte progenitor cells give rise to myelin-forming oligodendrocytes in the central nervous system.

The studies also showed no evidence of teratoma formation 12 months after injection in animal models. Transplanted ESCs often result in these tumours forming. The product is only minimally recognised by the immune system, the company said, and had shown little migration of the injected cells outside the spinal cord.

Geron’s cells are derived from the H1 human embryonic stem cell line, which was created before August 9, 2001, when former President George Bush barred federal research funding from being used in the development of embryonic stem cell technology. Despite this, the company used no federal funding in the product’s development, it said.