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Published online 20 July 2009 | Nature | doi:10.1038/news.2009.706
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Setback for Huntington's disease therapy
Brain-tissue transplants don't last very long in patients.
A once-promising clinical therapy for Huntington's disease needs to head back to the lab, research suggests.
Huntington's disease is an inherited, untreatable and fatal disease in which patients develop severe movement and cognitive problems.
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Brain tissue transplant needs to be coupled with a functional immuno-protectant scheme to prevent rejection. Obviously, giving immuno-suppresant drugs is not the best answer. In the context of Parkinson's disease it's been shown that co-transplantation of testicular Sertoli cells with neural grafts will not only produce an area of localized immuno-suppression (due to local secretion of GDNF by the Sertoli cells) but they will also coax stem cells specifically into dopaminergic neurons (the kind of neuron wiped out in PD). I am not aware of any analogous cell-based therapies for HD, however.
It is unfortunate that this news report did not have space to compare the results seen in our Huntington's disease transplant program to what we described in our transplanted patients with Parkinson's disease. In the latter program, only 5-8% of the transplanted neurons developed Lewy bodies. Also, grafts still survived and functioned clinically for over a decade. The article and the responses seem to cast a haze over the entire field of stem cells in general, which is certainly not accurate. The magnitude and mechanisms of neural degeneration that we reported was specific to grafts in patients with Huntington's disease. We are still enthusiastic about the potential of brain repair with stem cells for many different diseases. Our report also suggests new windows into opportunities for future research about the mechanism of and therapeutic options for Huntington's disease. Thomas Freeman, MD and Francesca Cicchetti, PhD