Tuesday, November 15, 2011

Q&A about Geron's decision and its implications for the stem cell field

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.

4 comments:

  1. So the CIRM has spent 1.25 Billion of California tax payer money and currently have ZERO hesc clinical trials?

    The CIRM did not recommend for funding 3 applications for funding by Advanced Cell Technologies, whom is the only company to currently in clinical trials for Hesc?

    CIRM should gift all 50 Million of the Targeted Clinical round of funding to Advanced Cell Technology.

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  2. Once again the platform (suffering patients) for stuffing money into coffers to benefit others (university chairs,management,faculty, equipment companies, etc) is the modus operandi.

    Did you get this model from corporate america where the stock goes down but the pay package increases? I know, I know...everyone on the payroll is brilliant...brilliant but no results!
    SOS(doesn't represent the distress signal..you're all smart you'll figure it out)

    Stem-cell trials halted over funding concerns

    Daniel Heumann, a board member of the Christopher and Dana Reeves Foundation which aims to cure spinal cord injury by funding new paths of research, said: "I'm disgusted. It makes me sick. To get people's hopes up and then do this for financial reasons is despicable. They are treating us like lab rats."

    Read more: http://www.edmontonjournal.com/technology/Stem+cell+trials+halted+over+funding+concerns/5719316/story.html#ixzz1dtHb3ted


    Dan I think we all are!!!

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  3. Is Canada ready for human trials on stem cell therapy?

    As desperation leads patients to experimental treatments overseas, a leading neurosurgeon says it’s time


    Before his accident, Mike Kowalski loved fast motorcycles. At 26, he rode his bike from his home in Markham, Ont., three hours north to Haliburton—it has “nice roads and less cops,” he says—when he took a turn too fast. Hitting a gravel patch, Kowalski lost control. His first and only motorcycle crash left him paralyzed from the chest down. Devastated, he tried to be optimistic: rapid advances in stem cells seemed to suggest powerful new treatments on the horizon. “I was mentally prepared for five years,” Kowalski says. “Not to be back where I was, but that I’d be using a cane instead of a wheelchair.”

    Kowalski kept up with the latest research, attending conferences and chatting with scientists about their work in stem cells. As time went by, and treatments failed to materialize in North America, he got increasingly frustrated. Two years after his accident, he went to Taiwan, where an experimental “nerve cocktail” was injected into his spine. Five years later, he went to Beijing and received an embryonic stem cell transplant. Neither treatment, which cost about $20,000 each, made much of a difference, he says. He kept waiting. “Five years came and went, and then 10.” It’s now been 11 years, and Kowalski still uses a wheelchair. “It seems incomprehensible that we can fix rats in a lab and fly rovers to Mars,” he says, “but we can’t regenerate some nerves in my spine.”

    Dr. Michael Fehlings, medical director of the Krembil Neuroscience Centre at Toronto Western Hospital, makes his case in the October issue of Neurotherapeutics. Cell therapies shouldn’t be “stalled at the animal model stage,” he writes. No rat, pig or monkey can perfectly mimic the human condition; after years of working on these animals in the lab, we should pursue “focused, safe and ethical” trials in humans. All new therapies carry their risks. Stem cells “can make anything, but that’s also the danger,” he told Maclean’s in an interview. If they were to grow out of control, these cells could potentially turn into cancer. One argument for moving forward is to stop patients like Kowalski from making desperate trips overseas. “I’m not in favour of medical tourism,” Fehlings says. If patients receive stem cell treatments, “it should be in a carefully regulated clinical trial, with fully informed consent, and they shouldn’t be charged.” After all, we still don’t know if these treatments will work. “We’re hopeful,” Fehlings says, “but there’s a difference.”

    Beyond that, scientists debate whether lab rats tell us enough about spinal cord injury to safely attempt these treatments in humans. In his paper, Fehlings calls it an “unrealistic hurdle” to expect researchers to validate all their findings in bigger animals, like pigs and monkeys, before launching a clinical trial. “If every treatment had to be replicated in monkeys, we would never move forward,” he says. But Miller points out that a lab rat isn’t the ideal stand-in for a person with an injured spinal cord. “The size of a lesion in a rat is very different, and their spinal cord is somewhat different,” she says. “Even if you injure them badly, they can get some hind leg locomotion back, which is not the case for humans. You can’t predict what would happen in humans on the basis of a rat.”

    http://www2.macleans.ca/2011/11/16/taking-a-chance-on-a-miracle/

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  4. Thanks for the post--the question and answers were very helpful to clarify for folks what is going on.
    Keep hope!
    Paul
    http://www.ipscell.com

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