Bone marrow transplants in autism models

In an Oct 9 post, I cited a fascinating talk by Jonathan Kipnis that I heard at a meeting. His paper came out in March, showing that the autism-like behavioral abnormalities in a mouse model of Rett syndrome could be prevented by giving these mice a bone marrow transplant from a normal mouse. By using several manipulations, these experiments indicated that the transplant worked by replacing the Rett microglia with normal microglia in the brain. I noted the potential importance of these cells in a post on June 7.

Now, a very talented graduate student in my laboratory, Elaine Hsiao, in collaboration with the group of Sarkis Mazmanian here at Caltech, has just published results of manipulating the immune system in our mouse model of the autism risk factor, maternal infection. Elaine finds that transplanting bone marrow into this mouse model, even after these mice have developed the autism-like behaviors, can correct several of them. These results provide a further suggestion that the immune system can interact with the brain to regulate behavior – the primary topic of my book!

We emphasize that because the work was conducted in mice, the results cannot be readily extrapolated to humans, and we are not suggesting that bone-marrow transplants should be considered as a treatment for autism. We have yet to establish whether it was the infusion of stem cells or the bone-marrow transplant procedure itself—complete with irradiation—that corrected the behaviors. However, the results do suggest that immune irregularities in children could be an important target for innovative immune manipulations in addressing the behaviors associated with autism spectrum disorder. By correcting these immune problems, it might be possible to ameliorate some of the classic developmental delays seen in autism.

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8 Responses to Bone marrow transplants in autism models

  1. Congratulation Paul to you and all your staff this is a fantastic paper that has very wide implications for many in the ASD community. Including one little 11 year old boy. That’s the very personal endpoint…

    Looking forward to the continuing research. A question or two though do you have any numbers on 1. How many ASD children present with these type immune system irregularities ?
    2. How many link back to first trimester infection or the prenatal environment as a whole ?

    • phpatterson says:

      Thx ASDRI; your constant, amazingly complete scan of the literature is really useful. RE your questions: Tony Persico et al. find that 17% of their ASD population fits an immune-related phenotype. This includes “prenatal obstetric complications”, so maternal infection may be part of this classification. I’ll do a post on that study. Great questions.

  2. Beth Maloney says:

    I think that what we are going to eventually find out is that PANDAS is a similarly related disorder. Perhaps but for a certain variation, a PANDAS child might have developed autism. So I’m wondering how many PANDAS moms might have had the flu during pregnancy. I think I will ask that question on my Facebook fan pages and see what might turn up…totally scientific way to get the information…

  3. JBBW says:

    What kind of innovative immune manipulations are being thought about? It’s interesting that stem cell infusion by itself doesn’t seem to work. Did you you see the study out of UNC that stopped the development of Type 1 diabetes in a mouse model with antibodies that block the t cells that attack the pancreas? Cool!

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  5. Wow that was unusual. I just wrote an really long comment but after I clicked submit my comment didn’t show up.
    Grrrr… well I’m not writing all that over again.
    Regardless, just wanted to say excellent blog!

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