We have been seeking the ‘Holy Grail’ to treat Parkinson’s Disease with stem cells for some time. Given the restrictions on embryonic stem cells, research has been focused on integumentary and pluripotent stem cells. A big step forward has been made by a group of researchers working together, all from different institution. Drs. Lorenz Studer, Sonja Kriks & Jae-Won Shim of the Sloan Kettering Cancer Center in NY, along with other researchers there, plus researchers at the Cornell Medical School, Northwestern Medical, and Rush Memorial (the last two in Chicago, IL) published the results of their study in Nature on 10 November 2011.
The research group reported they were able to produce mid-brain dopamine neurons (nerve cells) from pluripotent stem cells (PSC) and embryonic stem cells (ESC). These cells were derived and maintained in vitro (not implanted) for several months. These PSC derived neurons were also implanted in mice, rats, and monkeys, where functionality was maintained. However, the long term survival was only determined for cells that were derived from ESC (not the PSC), which afforded mice and rats (and some monkeys) that manifested Parkinson’s disease restoration of limb use and akinesia. All the implanted cells produced dopamine in their hosts.
The test mice lacked dopamine cells on one side of their brain (limited Parkinson’s disease). When injected with an amphetamine, these mouse rotated and spun- because one side already had dopamine cells. When injected with 100,000 human derived dopamine neurons on the side lacking the neurons, the mice regains normal movement and stopped rotating after a three to five month period. Rats exhibited similar behavior when injected with the dopamine producing cells..
Humans would need millions of such cells to, hopefully, regain standard movement. When 7 million such cells were injected into rhesus monkeys (two), the cells survived and functioned along with the existing cells in the monkeys.
Until this finding, most of the efforts have been based upon mouse embryo derived cells. When human derived cells were transplanted into mice, the cells either failed to perform as expected or led to the growth of tumor-like clusters in the animals. Those studies employed growth factors (specialized proteins) to convert the ESC to dopamine-producing neurons; this work used different small molecule activators (sonic hedgehog and purmorphamine) to induce the neuron formation.
Given these facts, one could consider that the next phase is more of an engineering issue than a scientific issue (i.e., getting the PSC to survive long term). The next step is to produce the stem cells under clinically acceptable conditions (like FDA regulations) and effect long-term animal testing. Hopefully, human trials could begin in 3 years or so.
Many of us are hopeful. The demands of living with PD and knowing it will continue to progress and increasingly require more meds and self effort, but not knowing at what pace of progression is something that can’t accurately be put into words. And I’m in early stage of it. Even I can’t fully imagine what life is like for mid and late stage sufferers.
Chevy:
I have tremendous hopes that this will be “engineered” in time for your needs. My grandfather succumbed to PD, and was among the very first (clinical trial) to have part of his brain frozen and to be treated with L-Dopa. It’s one of the reasons why I am involved in these efforts.
Roy
Stem cells have been held out as a way to treat many things. Now if only we could work around the whole situation more quickly but that could lead to another problem with population increases. I will watch to see what happens with this research
Roberta recently posted..Stuck trying to fill too many Business Roles
You are correct, Roberta. This issue has been tangled up in politics (for those who claim life is precious to them, this is always amazing.)
Roy