FDA Clears First Embryonic Stem Cell Trial Using Patients to Begin

Aug 02, 2010   

The U.S. Food and Drug Administration (FDA) has given the “go ahead” to the worlds first authorized human trial of a treatment derived from human embryonic stem cells.  This trial will test cells developed by the University of California, Irvine and the Geron Corporation.  The cells will be used on patients with recent spinal cord injuries.

This trial was originally cleared by the FDA to begin in January of last year.  However, before the study could begin, the FDA put a hold on the Investigational New Drug (IND) application because cysts were found in some of the mice injected with the cells during pre-clinical animal trials.  Another animal study had to be conducted and the testing of cell purity had to be improved before the trial could proceed.

On July 30, Geron announced that FDA had lifted the hold on the study and that it would move forward with the worlds first clinical trial of a human embryonic stem cell (hESC)-based therapy in humans.  This Phase I trial is designed to establish the safety of GRNOPC1 in patients with spinal cord injuries.  GRONPC1 is Gerons lead hESC-based therapeutic candidate.

Embryonic stem cells have the ability to turn into any type of cell in the human body.  In this clinical trial, embryonic stem cells are turned into precursors of neural support cells called oligodendrocytes, which are injected into the spinal cord at the site of the injury.  The goal is for the injected cells to repair the insulation around nerve cells which would restore the ability of some of the nerves to carry signals.  The initial trial will focus on a very small number of clinical subjects to establish the safety of the treatment. 

Geron plans to seek FDA approval, once safety is established, to extend the study to an increased dosage of GRONPC1, enroll subjects with complete cervical injuries, and expand the trial to include patients with severe injuries to enable access to the therapy for a broader population of patients.  The biopharmaceutical company believes that there could be other potential indications for GRONPC1 such as treatment for Alzheimers Disease, Multiple Sclerosis, and Canavan Disease.

The FDA clearance of this clinical trial is a major milestone for the field of stem cell research and medicine.

For more information on Fuerst Ittlemans experience handling the FDA regulatory framework of stem cells, drugs, and biologics, please contact us at contact@fidjlaw.com.