Thursday, January 26, 2012

Media giving vulnerable hope

The latest headline reads:

"Once they were blind, now they see. Patient's cured by stem cell 'miracle'"

This article was dated 24th January 2012 and posted on The Independent website (link below).

Are we not getting a little ahead of ourselves?

The article refers to the Advanced Cell Technology stem cell trials and their latest findings. As previously mentioned, they have treated two patients, one with Stargardt's, with RPE embryonic stem cells. This trial is purely for safety, and is not measuring the effectiveness of embryonic stem cells in the treatment of the diseases.

It is very exciting that of the two patients, so far they have not experienced any adverse effects - tumours or reactions. From what I have read it has been 4 months since the patients received the cells and they are doing well, that is to say are not doing badly.

Because the ACT trial is proving to be safe, another 2 Stargardt's patients will receive the stem cells, one in the US and one in the UK. This is great news for stem cell research.

The patients that have been treated have also stated improvement in their vision. ACT are not focusing on the visual improvement in this stage of trials and there is a link below to the published report.

So I am putting my skepticism hat on again and I am angry that the media can claim that this is a 'miracle' and 'the blind can see again.' These trials are for SAFETY and are not measuring whether stem cell treatment actually works! To say that the blind are cured is getting just a little too ahead of what is actually going on. ACT have stressed that these trials are safety trials and the media should pass the information to the public, instead of giving false hope to people like me with Stargardt's.

There is still a long way to go with stem cells. I will continue to wait in anticipation.

Below are links to recent articles about the ACT trial.

The Independent

Advanced Cell Technology

Australian Ageing Agenda

The Lancet


  1. Hi,
    I understand your caution since you are directly affected by this disease and do not want to have false hope but your statement:

    "These trials are for SAFETY and are not measuring whether stem cell treatment actually works!"

    is actually not perfectly correct. These trials are PhaseI/II and although primary measure is safety, secondary measure is efficacy (

    Needles to say that even if it's only for safety you can't tell the already treated patients which have visual improvement that they should not be excited because these trials are only for SAFETY and that injected RPE cells should be only safe. These cells are living cells which belongs into the eye anyway so you can't tell them to not do their proper work. Forget about molecular compound drugs, these are living cells!!

    And keep an eye on first treated UK as in opposite to US patients, there is apparently no embargo on the information as he was giving interview right after the treatment..

    I wish you the best and also wish the best to already treated patients.

  2. Hi Mrakoslap

    I am not saying that the treated patient's or others with Stargardt's should not be excited, but I do caution the use of "miracle" and "the blind being cured" which is not what has happened!

    Embryonic stem cells are in the very early stages of trials, and even though we know they can turn into any cell, they have never been directly put into human tissue before and this is a new experience. Of course they should not only be safe but safety is paramount in the medical world and we need to explore all possibilities and over greater lengths of time to ensure that there are no complications down the track.

    I closely follow ACT and they have said that the study is focusing on safety and tolerability:

    "It is a prospective, open-label study, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt&rquo;s macular dystrophy at 12 months.."

    This statement is form their most recent news release on their website. (

    My main point was the sensationalisation of the media which is not fair to patient's who so desperately are hoping for a cure.

  3. Hi C.R,
    thank you for your reply. Regarding safety/not efficacy, please check out this link

    There is secondary outcome measures: Engraftment

    I guess that if you follow the ACTC you understand what engraftment of RPE cells precludes. You should also be familiar with mice/rat preclinical studies.

    Regarding some media headlines, i believe the "blind being cured" is from The Independent. This is sure questionable as patients didn't regain the full vision but just a fraction of it (but considering that SMD is untreatable isn't this miracle?)

    Again I wish you the best. Do you know of iCell site when you can learn tons of things regarding ACTC and more?

  4. No I do not think what has happened is a "miracle" . A patient has regained sight which was their subjective view - and even Dr. Schwartz who is conducting the study said that this is most likely a placebo effect as she had improvement in both eyes, one of which wasn't treated.

    Engraftment in this case is the cells Implanted will grow and reproduce - not whether vision will improve.

    We must remember that this trial has treated 3 patients so far and there is no control group. It has been 4 months since stem cells were implanted, which is a fraction of time when talking about being treated. Stargardt's patients are usually diagnosed in their teens and early twenties - which means a treatment needs to work for most their life, and 4 months isn't long enough to base this on just yet.

    Here is an article from the New York Times that supports what I've said:

  5. No, Dr.Schwartz never said that it's "most likely placebo effect". Get your facts straight please. So you are basing your opininon on one article in Nytimes because it supports your view and you purposedly omitting peer-review Lancet journal + dozens of other articles as it does not support your view..

    Now it's 6 moths since initial injections and the patients are still experiencing placebo effect visual improvement by your words.

    yes 4 months is not enough but don't you think that it's possible to have multiple injections within lifetime? Wet AMD is treated by 2 different drugs, one is every month and second is every 2 months so what's your point?

  6. Btw. "A patient has regained sight which was their subjective view..."

    Who can tell first hand whether he see better then before then a patient? Plus there are opthalmologic methods to measure vision improvement like EDTRS chart etc.

    Please read the peer-review artcile in Lancer journal before you jump to a statements that Dr.Schwartz said "most likely placebo effect"...

  7. You mentioned you weren't directly affected by Stargardt's, could you explain why you are so passionate about it, as it is very rare to find someone who actually knows what the disease is!

  8. Hi C.R. i'll be honest with you I'm ACTC shareholder. But I'm also fan of sci-fi and all modern science and I simply want this to work as it will be an amazing progress in medicine. I've first came across ACTC 3 years ago and choosed to believe that the science will work (3 years ago I've also bought shares for the first time and then some over time).
    So to me these first preliminary results are just fantastic as so far only "terminal" stage patients were injected where Bruch membrane was severely damaged and almost no photoreceptors left to rescue. Sure we need to wait till more patients are injected but it's very exciting to see this science show some progress.
    There has been 1 patient injected in UK with SMD and he has given interview just days after the injections. Apparently EU EMA is not so strict as US FDA so patient identit is public since the beggining. You may want to contact him and ask him about his feels and progress as he has same disease as you. Btw. I'm from EU (Czech Republic)

  9. Hi C.R.

    The SMD patient showed engraftment per the Lancet article. The members at iCell fully expected this, and what was surprising to everyone I believe was that improvement was seen in Sue's eyes (the AMD patient) without attachment. That's what prompted the whole placebo effect thing and folks seems to be extending it to the overall results. Doc Lanza and ACT have been at this for a long time, and I think you'll see even better results as the dosage is escalated per the trial design. In terms of a control group, only one eye is being dosed. Good luck and I wish you the best.

  10. Let me start by saying i have Stargardt's as well. I was diagnosed about 10 years ago, when I was 16, and i'm just now starting to cope with it.

    With that said, the news media routinely promotes half-truths and conjecture. Don't let them get to you.

  11. I completely agree with what CR has to say about the media in their ferver
    to report stem cells and other advances in the field of retinal disorders. I was unfortunate enough to be diagnosed with Viteliform Macular Dystrohpy though doctors are still not sure (read cofused) on the exact nature of my disorder except that my sight has become limited and incurable. Some sayit is Best's disease some say it is Stargardt's. I think it is Stargardt's simply becuase at the age I was affected, Stargardt's is most common.
    Coming back to my point, media can do a much better job in helping people like me to be aware of the 'real' advances going on around the world so that we can have some meaningful hope of someday seeing better.
    However as much as we sing prases for stem cells, I have my doubts about its efficacy to cure genetic diseases. I have a leevl of understanding in genetics and biological sciences since I have a background in Bioanalytical Chemistry. My knowledge tells me that even if stem cells are embedded in place of the original 'bad' cells, once in place they will get the chraracteristics of the genes that I have inherited and thus revert back to their unusual characteristics. Of course in saying this I may be completely missing some important point about how stem cells work and I pray to god that I am actually wrong in my current understanding.Because so far I have lived with a hope and believe me hope is what drives the world (and people like you and me) that one day I will see better. And in my willingness to hope positively media can actually help by reporting only that is truth and not get carried away with little triumphs that may not actually mean any thing in the long run.

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