Early results from two embryonic stem cell-based trials show promise

Yesterday, the company Advanced Cell Technology announced that two people in their clinical trials testing an embryonic stem cell-based therapy for forms of blindness is not only safe so far, but shows tentative early signs of restoring some vision in two patients. The work was published online January 23 in the journal The Lancet.

Of the two women discussed in the paper one had macular degeneration, the leading cause of blindness, and the other had the most common form of blindness in children, called Stargardt’s macular dystrophy. Both were participating in phase 1 trials testing cells derived from embryonic stem cells as a possible treatment for their blindness. All phase 1 trials are primarily designed to make sure a therapy is safe, but the researchers do also look for signs that the therapy might be effective. (We have more information about the phases of clinical trials on our website.)

The preliminary news that two women in these trials reported some improvements in vision warrants some cautious optimism. For those of us who have been following the field since the discovery of embryonic stem cells in 1998, this paper is a milestone. It’s the first published paper showing that—at least in this small number of patients for the first few months—the cells are safe.

However, two patients isn’t enough to show whether the therapy actually works. In fact, in a New York Times story Steven D. Schwartz, a professor of ophthalmalogy at UCLA’s Jules Stein Eye Institute, who is leading the research, said there was evidence that at least one of the two patients might have experienced a placebo effect. Sorting out the real improvements from the placebos or short-term changes takes time, which is why clinical trials are set up to follow patients for several years. Only after many more people receive injections of the cells and are followed for several years will we know that the cells were effective.

A story by NPR quoted Schwartz:

Schwartz and his colleagues stressed that the findings are extremely preliminary and it’s far too early to know anything for sure. The patients could continue to improve, or their vision could deteriorate again, he said. Many more patients will be needed to be treated for far longer to know whether the therapy is really safe and responsible for any improvement.

“My job is to decrease suffering, and if we overstate this and raise hopes falsely and then it doesn’t work out, it will hurt people rather than help them,” Schwartz said.

The Stanford University medical blog has more commentary on this trial from law professor Hank Greely. He’s been following the field of embryonic stem cell research since the beginning and says the news is, “at least, a little exciting – and in a field that saw its first approved clinical trial stopped two months ago, even a little exciting news is very welcome.” You can read more on their blog.

These are the only two trials currently underway testing an embryonic stem cell-based therapy. Along with the rest of the stem cell community we’ll be watching the results, and following the additional trials that are expected to start in the next few years. It’s through clinical trials such as these that safe and effective stem cell-based therapies will eventually reach patients.

A.A.

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4 thoughts on “Early results from two embryonic stem cell-based trials show promise

  1. Does the CIRM have any comment as to why funding for this science was denied to Advanced Cell Technology? Was there a conflict of interest by a reviewer of the applications?

  2. Very exciting news! This is why it's so critical that California AND the rest of the country continue to support stem cell research. We are riding the first waves of regenerative medicine!

  3. To better grasp this issue; it needs to be placed back in context; saving lives. Though some of these opposing views are not without merit, it is evident that the benefits outweigh the ethical concerns. If there is a potential avenue for curing a disease, it must be pursued; with zero limitations. Though there will be situations where this process will be used in pure-vanity, e.g., facial make-up, as addressed “Scientists who favor stem cell research are upset by the promotion of these injections.”[v] However, one must not base one’s argument on such narcissistic- vanity. The dilemma arising from Stem Cell Research is social, legal, scientific, and for those that really need it; psychological. So what are the prospects of stem cell research? It is claimed that it might help cure Spinal cord injury, Diabetes, Heart disease, Parkinson’s disease, Alzheimer’s disease, Lou Gehrig’s disease, Arthritis, Sickle anemia, Organ failure and not to mention HIV. If that’s the case, why interfere and obstruct a research that will possibly eradicate these diseases? We live in an utilitarian system where what is better for the whole should always prevail; take all the diseases that could be cured on the advocate’s side and weigh it against what is presented on the con-side; it is without logic that one arrives at the notion that saving lives in non-ethical. What is non-ethical; is denying treatment to those that will benefit from the fruits of the research. Who benefits from the fruits of stem cell research? Mankind. Who benefits from ‘No stem cell research arguments and legislation?’ Nobody. I found this article to offer the most on this topic http://www.newsonhealthcare.com/stem-cell-research-it-must-be-funded-no-it-must-not/ I hope that helped some more.

  4. CIRM Funding for Vision Loss $33.5 Million
    CIRM Funding for Geron $25 Million
    CIRM Funding for Peter Coffey $4.8 Million
    CIRM Funding Total for above $63.3 Million

    Total Taxpayer Funds Wasted $63.3 Million

    Like Alan Trounson stated in 2008…. He would rather go to lunch then listen to what Advanced Cell Technology has to offer.

    Like the Story of David & Goliath.

    As usual the underdog wins!

    CIRM Clinical Trials using HESC “0″
    ACTC Clinical Trials using HESC “2″

    Here are the real questions….

    Does the CIRM continue to waste tax payer money on the same Clinical Trials that are already in progress by ACTC?

    Does Alan Trounson man up and admit there was pure lack of judgement on the CIRM's behave and help fund these trials?

    What is the CIRM defense if Steven Kessler formerly of ACTC is interviewed Today and blow the CIRM corruption out of the water?

    Jonathan Thomas, the new guy on the block….you were involved with the early funding of ACTC. You know what happened. You have any muscle against Trounson? Or are you just his figure head?

    Signed
    Son of a Blind Man

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