Wednesday, August 26, 2009

Lots and lots of info on stem cell therapy

Those are Max's stem cells, above. Aren't they just beautiful?

As you can imagine, I had a ton of questions for the doctor who's spearheading Duke University's stem cell program: Joanne Kurtzberg, MD, Professor of Pediatrics and Pathology and Director of Duke University's Pediatric Blood and Marrow Transplant Program. She is a brilliant, progressive-minded doctor and a person who manages to be both matter-of-fact yet warm at the same time. She first started researching stem cell therapy for kids with leukemia back in the 80s. Since then, she's treated more than 1600 kids with life-threatening cancer, metabolic and blood disorders, using donor stem cells, and 120 kids who had neurological conditions such as hydrocephalus, brain injuries from falls or drowning and cerebral palsy, using their cord blood. The stem cells are injected into the arm or leg, but it's possible that someday, they will be injected into the brain. It is still unknown where the best injection spot is.

Right now, Dr. Kurtzberg is only using stem cells from cord blood to treat cerebral palsy because the risks of using ones from a donor, even a sibling, are too high. In order to make donor stem cells "engraft" (or take) a child needs to receive chemo, a process that takes two months and can be dangerous. Since cerebral palsy is not a life-threatening condition, the doctor says, the risks outweigh any potential benefits (unlike, say, leukemia).

Dr. K told me that stem cell transplants have helped kids with cancer and other diseases. She and her team were able to do a biopsy on the brain of a child who died 10 months after getting a stem cell transplant. It was her progressive neurological condition that ultimately killed her. In fact, when they looked at her brain, they could see that 40 percent of the cells had reached there. With kids who have cp, the results are less clear-cut; reports come in from parents, but they may not be the most objective source. There is a lot of misinformation out there. Remember that video of Dallas Hextell I mentioned a couple of weeks ago, a video that showed he was able to walk after receiving a stem cell infusionl? That video and story were misrepresentative; Dr. K said Dallas was walking before he got the stem cell therapy. But she says that she has heard of kids being better able to use their limbs, and improvements in speech.

Results can start showing up around six months after the infusion. I'll be e-mailing Dr. K every few months to tell her how Max is doing, and we'll go back and see her in a year. Before we go home today we'll be visiting a pediatric neurologist at Duke U., who will assess Max and then see how much he may change by next year.

Dr. K believes that stem cells are best administered as soon as possible after a brain injury has occurred. Two other stem cell infusions happened yesterday, and one of them was for a six-week-old infant. I didn't feel comfortable asking what had happened, but I was thrilled for that child. I only wish, as I've said before, I could have done this sooner for Max.

For now, treatment is still considered experimental and unproven (more on that below), but with help from champions like Dr. Kurtzberg, perhaps in the near future we will get proof that it works. Once that exists, more hospitals around the country would start doing this. And that would be tremendous. As you know, there is no cure for cerebral palsy. Not yet, anyway.

A few more questions Dr. Kurtzberg answered for me:

Can you give a basic explanation of how stem cell therapy is supposed to be effective?
"We believe that stem cells can travel to the brain and then help repair damage caused by stroke, low oxygen or other vascular problems. The cells may work in two ways, first by releasing hormones that calm down inflammation and also attract cells from the brain to heal damaged areas, and second, by providing stem cells to contribute to the repair."

What are your hopes for the state of stem cell therapy five years from now? Ten years from now?
"I hope that we first learn if stem cell therapy works and also the best way to use it. We need to know what are the best cells to use, what is the best place to give them and what dose is most effective. We also need to better understand when children with cp sustain their brain injury and how to detect it as early as possible. I believe that stem cell therapy will help children with brain injuries but I also believe that the earlier it is given, the more effective it will be. I think that it may be possible, in the future, to scan babies in utero or at birth to determine whether they have an injury, and then to treat them within the
first few days to weeks of life."

What advice do you have for parents who did not bank their child's cord blood at birth?
"First, these parents need to know that we don't know if cells help yet. While it's very hard to wait, that's what they should do. I can promise that if we find that cells do help, we'll next find a way to safely use donor cells for children who didn't have their own cord blood cells stored."

Can you give any information on the upcoming trial?
"The upcoming trial will enroll 120 children with spastic cp between the ages of six months to six years. They will be tested with an MRI, neurodevelopmental testing and a neurological examination before treatment. Children will randomly receive their own cord blood cells or a placebo for their first treatment. One year later, they will be tested again and will cross over and receive the treatment they did not receive the first year. They will be studied again one year later. The treatment will be blinded, which means that no one will know which treatment each child received first and second until the end of the study."

If you are interested in finding out more information on getting your child into the trial, which they hope to kick off this fall, you can contact the Director of Clinical Operations, June Allison (the woman is a saint) at


  1. Wow. I am so optimistic about the future, now that "stem cell" isn't a dirty phrase like it was for eight of the last nine years!

    How interesting about having to use immune system supressants for donor cells. I agree with the doctor, too dangerous, at least for now. I think I'd be too afraid to risk it.

    I'm thinking it's probably better off if the scientists find a way to create stem cells from individuals themselves, so there's no rejection issues.

    It's all so fascinating, isn't it!!

    Thanks so much for your posts on this topic!

  2. Thank you for all this good information, I'm passing it all along to my neighbor whose daughter has CP.

  3. Thanks, Ellen, for all the great info on stem cells! I had hoped that sibling transplants might be in our future, but it sounds like that's more complicated than I thought - too risky indeed. Hoping and praying that Max's transplant will help him. Exciting stuff!

  4. Thanks for sharing that is great stuff very hopeful! I hope you see some great changes in max to gives us parents even more hope.

  5. Thanks so much for sharing such detailed and easy to understand info about stem cell therapy.

    I've recently been considering banking our baby number 3s cord blood, JUST IN CASE. A little disappointed to hear that it's still considered very risky and complicated to use it on a sibling (if indeed possible), but inspired enough that technology is rapidly developing in this area, to definitely make some further enquiries about the banking process.

    Thanks again for sharing!

  6. Hi Ellen,

    I am very interested to see what happens post-stem cell infusion. I worry about the danger too. Still, being somewhat in-the-field, I acknowledge the power of stem cells. As a mom, I am hesitant.
    I wish nothing but the best for Max and your family. I really appreciate your posting so much information on the subject.


  7. Thank you so much for taking the time to share this information with us and ask all those wonderful questions. I am so excited about the future of stem cell therapy. We did not save Sebastian's cord blood, but it is great to see that the research is moving in the direction of donor/sibling cord blood too. Good stuff. I am looking forward to reading about Max's developments over the next year!

  8. My daughter suffered a brain injury at 18 months which resulted in quad cp. We just got back from Costa Rica. While there she recieved donor stem cells. We have seen many GREAT improvements since then and are going back in 6 months for another round of treatment.

    She did not have chemo. We have only seen positive changes. Changes that even her therapists give credit to her donor cells.

    The answers to the questions on this blog are that of one doctor. One doctor that is governed by the FDA in a country that is 20 years or more behind in the medical field compared to other countries.

    For those that do not have cord blood.... research... there are other options out there then to just wait! My daughter is living proof of that!

    If you want to use your own stem cells go to Germany. They can extract your own cells out of your BM or fat. They are already DOING there what Duke has talked about TRIALING here.

    I am sure Duke is a great place with wonderful doctors. There is so much more out there then just Duke though!

    I can't wait to see how much this will help Max and so many other kiddos in the near future!

  9. (Remember that Duke is a private university so they do not have to follow all of the guidelines set forth by the FDA.)

  10. This is all so interesting! I certainly hope that you'll be able to see improvements with Max's mobility and speech - how exciting to think of the possibilities!

  11. Just wanted to reask on this post about stem cells for genetic things like Neurofibromatosis. Are you still working with this doctor? Can you ask her that?

  12. Hi

    my son is 4 month old and he is suffering from H.I.E. stage 2

    I am the father of a 4 month old who suffered from lack of oxygen at birth (hypoxic-ischemic encephalopathy (HIE)), along with seizures shortly after birth

    Delatils of MRI given below:
    The bilateral supratentirial compartment reveals multicystic encephalomalacic gliotic changes with underlying volume loss in both cerebral hemisphere predominantly involving the perpheral / subcortical white matter region of bilateral temporo-parieto-occipital lobes more than the frontal lobes.

    i have preserved the umbilical cord blood that is also a main source of stem cell
    please let me know if the above mentioned disease is treatable using Stem Cell Therapy

    I live in India

  13. No CP isnt a disease, its a non life threatening, non progressive condition. Did you read the post properly or not? If not, please read it carefully.


Thanks for sharing!

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