Yesterday Geron announced that they would be discontinuing their embryonic stem cell program, including the groundbreaking spinal cord injury trial that CIRM had been supporting. You can read more about the decision in Geron's announcement and in CIRM's press release. Geron has returned the $6.4 million they had received from CIRM with accrued interest.
It's not uncommon for companies to discontinue research programs for financial reasons. What makes this announcement different is the high-profile nature of the company and the trial. Geron was the first to pave a path through the Food and Drug Administration (FDA) to test a human embryonic stem cell-derived product in clinical trials. The trial was seen as a sign of hope for people waiting for therapies derived from embryonic stem cells, so its termination came as a shock to many patients and their families.
Today we wanted to take a step back and look at the announcement and what it means.
Q. What was Geron testing in their spinal cord injury trial?
A. Geron started with human embryonic stem cells and matured them into the precursor of type of cell that forms the coating on neurons. That coating is lost soon after a spinal cord injury, and without the coating electrical signals can't travel up and down the spine. The idea was that after injecting some of these precursor cells they would mature and form a new coating on the spinal cord neurons, restoring the ability of signals to cross the injury site.
This first trial was in its early stages and was testing a low dose of the cells to make sure they are safe. Remember that when this trial began the cells had only been tested in animals. Only after the company showed that the cells were safe would they be able to inject a dose that was expected to be high enough to show some signs of success.
Q. Why did Geron decide to discontinue the stem cell program?
A. The company decided to focus its resources on two oncology drugs that are in clinical trials. The decision was made based on financial considerations, not based on concerns about the science. Such a move is relatively common for biotechnology companies especially during the complex early-phase clinical trials such as this one.
Q. What does Geron’s decision mean for the field of stem cell research?
A. The field of stem cell research continues to be strong, with many promising therapies in the pipeline. Geron leaving the field does not alter the existing excellent programs that are working toward and carrying out clinical trials.
Q. What will happen to the patients who already received the cells?
A. Geron has been closely following the patients who have received the cells. So far, the patients have tolerated their injections and haven't shown any serious adverse effects from the cells. The company says it will continue monitoring those patients and keeping the FDA appraised of the results.
Q. Will the trial continue?
A. For now, the trial is discontinued. Geron says they are looking for partners to further develop their stem cell programs. Whether or not the trial continues depends on whether partners come forward with the resources and the interest in continuing it.
Q. What happened to CIRM's money?
A. CIRM had committed $24.8 million to support the trial, of which $6.4 had been dispersed. Geron has already returned the money in its entirety with accrued interest.
Q. Are there other trials testing products made from embryonic stem cells?
A. Yes. The company Advanced Cell Technology is testing an embryonic stem cell-derived product in two forms of blindness: macular degeneration and Stargardt's disease. They have already begun enrolling patients in those trials.
Q. What other trials are in the pipeline to test embryonic stem cells?
A. World-wide, many groups are moving closer to clinical trials testing cells derived from embryonic stem cells. CIRM has funded four disease teams that have the goal of submitting a request to the FDA to move forward with clinical trials (that request is also known as an Investigational New Drug or IND filing). Those disease teams are working on diabetes, stroke, macular degeneration and ALS (also known as Lou Gehrig's disease).
A.A.
No comments:
Post a Comment