Stem cell-based spinal cord therapy expanded to more patients – The San Diego Union-Tribune

An experimental therapy to repair spinal cord injury with stem cell-derived tissue is progressing smoothly, according to a leader of that trial who spoke at a conference on stem cell therapy.

The Phase 1 safety trial is proceeding with no complications, said Dr. Joseph Ciacci, a University of California San Diego neurosurgeon. The conference was held last week at the Sanford Consortium for Regenerative Medicine in La Jolla.

With safety looking good, the green light has been given to treat more patients, Ciacci said. However, to produce effectiveness, more cells will need to be transplanted.

Four patients have been treated with neural stem cells, injected into the spinal cord. They had experienced complete loss of motor and sensor function below the injury. They had been injured between 1 and 2 years previously.

Moreover, the cells show signs of integrating with the surrounding tissue in animal studies, Ciacci said. If the preliminary evidence holds up, Ciacci and colleagues plan to submit a paper detailing the results.

Curing paralysis from spinal cord injury was a big selling point for those who successfully advocated Proposition 71, which authorized selling $6 billion in state bonds to establish and fund the California Institute for Regenerative Medicine, or CIRM. The institute got $3 billion, the remaining half is going for interest over the life of the bonds.

While CIRM has been under pressure to show results, doctors are taking great care to establish safety first in the spinal cord treatment, because of potential risks in the procedure.

We are now enrolling and recruiting for the second cohort, which is for chronic cervical spinal cord injuries, Ciacci said. They are medically classified as C5-C7 ASIA A Complete.

Chronic injuries need to have taken place more than 1 year before treatment. For this study, the injury must also be under two years old. The trial is being conducted at UCSD with Ciacci serving as the principal investigator.

For more information on the Phase I Chronic SCI study, contact Ciaccis research group at (619) 471-3698, nksidhu@ucsd.edu.

In addition, the researchers have been approved to start another spinal cord injury trial with a different set of cells. These oligodendrocyte progenitor cells, derived from embryonic stem cells, can turn into several different types of neural cells.

The trial, sponsored by Asterias, treats newly injured patients, between 14 and 30 days after injury.

For more information on the Asterias trial, contact the UCSD Alpha Stem Cell Clinic at 858-534-5932 alphastemcellclinic@ucsd.edu or visit http://www.scistar-study.com and j.mp/ucsdast.

Asterias acquired the technology from Geron, which had undertaken the work with a CIRM grant. Geron later canceled the work and refunded the money to CIRM. Asterias got funding from CIRM to continue the work.

The Asterias trial will use the same technique as used with the Chronic SCI trial, a technique which can improve safety, Ciacci said. The cells will be injected in a series of progressively larger amounts that may give evidence of the dose relates to effectiveness, although safety remains the main concern.

This cell line is cryopreserved, its sent to us as a single dose the day of surgery, Ciacci said. Were going to study different doses 2 million, 10 million, 20 million cells per injection. Its going to be a direct injection, just like what weve done before.

As in previous treatments, patients will also receive immune suppression to prevent rejection of the cells. Likewise, they will be monitored for many years after treatment.

Another trial coming to UCSD will test for efficacy in ALS, Ciacci said.

Ciacci said hes looking for qualified patients for these trials, and urged those in the audience to help find them.