A human embryonic stem cell Phase I Trial for patients with spinal cord injury is to get a $25 million award from the California Institute for Regenerative Medicine (CIRM). This is the first clinical trial based on cells derived from human embryonic stem cells that has been approved by the FDA, and also the first time CIRM has funded stem-cell derived therapy research. The trial is run by the Geron Corporation.
Robert Klein, CIRM chairman said:
“Supporting the Geron trial is a landmark step for CIRM. However, we must remember that there will be successes and interim failures as human trials proceed through the refinements necessary to achieve a successful human therapy. We need to be prepared to stand by the heroic patients and the companies as they face these challenges and solve the problems that stand in the way of the recovery of patients from paralysis.
When the people of California voted for proposition 71 they did so with the hope of seeing new therapies for disabling diseases like Alzheimer’s disease, Parkinson’s disease, diabetes and other chronic diseases and injuries. By funding this trial, CIRM is taking a major step toward making that hope a reality.”
A limited number of patients are to be given injections – into the site of their injury – of oligodendrocyte progenitor cells that originate from embryonic stem cells.
Animal studies demonstrated that these cells turn into oligodendrocytes, which produce insulating tissue that surround neurons, protecting them – they form a myelin sheath around axons.
A oligodendrocyte is a type of central nervous system cell. They surround and insulate the axons (the long fibers) through which a nerve’s electrical messages are sent.
This three-year study initially aims to determine how safe the cells are when injected into human beings. Geron Corp wrote in a communiqué “..a dose of 2 million cells is being administered to assess safety”.
The trial will then include several degrees of spinal cord injury, including those in the cervical region as well as patients with incomplete injuries, where varying doses will be tested. One trial participant is already in another trial location (Stanford University Medical Center) after having received injections at the initial site in Georgia.
Jane S. Lebkowski, Geron’s Senior Vice President and Chief Scientific Officer, Cell Therapies, said:
“Bringing novel stem cell therapies to patients with an unmet medical need, such as spinal cord injury, is a goal that we share with CIRM. We are very pleased to announce the approval of this funding to support Geron’s clinical development of this first cell therapy derived from human embryonic stem cells to reach clinical trials.”
The CIRM award is, in fact, a product-backed loan. This means Geron Corp. has to pay it back when and if it starts making money from this project.
Spinal Cord Injury
This is caused by a serious injury (trauma) to the spinal cord that leaves the patient with loss of motor control, bowel and bladder control, or sensatory perception. A blow to the spine can dislocate or fracture vertebrae that can result in bone fragments or disc material damaging the nerve fibers and affecting the oligodendrocytes that insulate the axons. In the majority of cases, human spinal cord injuries are bruises (contusions) to the cord, and not severance of the nerve fibers.
12,000 Americans sustain spinal cord injuries annually.
Sources: CIRM, Geron Corp.
Written by Christian Nordqvist