Reprogrammed Skin Cells for Parkinson's
There are other methods for developing effective treatments and possibly cures for diseases through the adaptation of non-embryonic stem cells which are induced to have the pluripotent capabilities. There appears to be potential if these cells can live up to their promise.
Induced pluripotent cells
Non-embryonic stem cells induced/stimulated to
become pluripotent (iPS)
Embryonic stem cells
Already pluripotent (ESC) capable of being any cell
hESC
Human embryonic stem cells
hiPSC
Human induced pluripotent cells
Pluripotent
having the capability to be any cell
Gene expression
a measure of which genes the cell uses and how much of the
cell uses those genes
Genetic recombination
a strand of genetic material (DNA or sometimes RNA)
is broken and joined to a different DNA molecule
Botanists have been grafting for centuries but this is nano
micro-scale and complicated
Cre recombinase
Enzyme which catalyzes genetic recombination
Transgene
Genetic material transferredfrom one organism to another;
any DNA sequence introduced into an organism.
Factor-free
without being introduced via a virus factor
As you already know, researchers in Cambridge MA have been able to use skin cells which were induced into pluripotency to become dopamine-producing neuron cells. Unlike previous research, these iPS did not insert cancer producing genes into the DNA because that reprogramming was removed allowing the cells to maintain an ESC-like status.
The problem in the past has been that the reprogrammed cells had the potential to cause cancer, creating an out of the frying pan and into the fire situation. In the new process: the skin cells of a Parkinson's patient are reprogrammed to be iPS cells. And then reprogrammed again so that the potentially cancer-causing program was removed. At this point the human induced pluripotent cells (hiPS) are very similar to human embryonic stem cells (hESC)
In the past researchers were not sure that removing the reprogramming from these human cells would allow the cell to maintain a stable iPS state so that it could multiply. Now they are. This step opens the door...
Links:
http://www.cell.com/abstract/S0092-8674(09)00151-2
http://esciencenews.com/articles/2009/03/05/breakthrough.produces.parkinsons.patient.specific.stem.cells.free.harmful.reprogramming.genes
The Scientists:
Whitehead Institute for Biomedical Research, Cambridge, Mass: Frank Soldner, Dirk Hockemeyer, Caroline Beard, George W Bell, Elizabeth G Cook, Qing Gao, Miasam Mitalippova, Rudolph Jaenisch
Dept of Biology MIT, Cambridge, Mass: Rudolph Jaenisch
Udall Parkinson Disease Research Center of Excellence, Center for Neuroredegeneration Research, McLean Hospital/Harvard Medical School, Cambridge, Mass: Gunnar Hargus, Alexandra Blak, Oliver Cooper, Ole Isacson
Showing posts with label PD research. Show all posts
Showing posts with label PD research. Show all posts
Thursday, March 19, 2009
Wednesday, February 18, 2009
You Have to Kiss A Lot of Lab Rats
Not all Parkinson's research will be the big one.
Every so often we come across the blogger who criticizes, for example, the funding by the Michael J Fox Foundation because no results have been seen: no cure for Parkinson's disease. And that attitude miffs us. It doesn't get us angry because to do that would be to acknowledge that this was someone who knew what the heck was going on. But it is dangerous because the monies which must go into research are enormous and the backlash effect of these attitudes can jeopardize donations and research funding if believed.
Research often leads down a dead end road...for a particular condition.
These paths aren't marked that way from the outset...it just happens that this direction or that one didn't turn out to be viable. Sometimes they are a bridge and sometimes they are a dead end. But you've got to take the road to discover and that's not always a bad thing because you know how to improve, where not to look, whatever...
Some of these clinical trials can take a long time to open for participation. After that a trial can last for a significantly long time - years sometimes.
One of the recent directions of research found from an earlier study that in people who had naturally high levels of urate, an antioxidant, had a slower Parkinson's disease progression rate. This is especially important for the understudied young onset Parkinson's patients (YOPD). See: our 1/20/10 post
For those who were following the April 2008 announcement that the MJFF was one of the sponsors of the Phase II trial of Drs. Michael Schwarzschild and Alberto Ascherio studying patients with early onset Parkinson's to determine the safety and efficacy of Inosine which raises urate levels - a potentially risky business and contraindicated under certain conditions but by no means all - there is no news except to say that the study, Safety of Urate Elevation (SURE-PD) has not begun to enroll yet. (9-2009 update: study now recruiting. See: below)
You can find the contact information and the nine states where the trials will be conducted here:
Safety of Urate Elevation in Parkinson's Disease (SURE-PD)
Every so often we come across the blogger who criticizes, for example, the funding by the Michael J Fox Foundation because no results have been seen: no cure for Parkinson's disease. And that attitude miffs us. It doesn't get us angry because to do that would be to acknowledge that this was someone who knew what the heck was going on. But it is dangerous because the monies which must go into research are enormous and the backlash effect of these attitudes can jeopardize donations and research funding if believed.
Research often leads down a dead end road...for a particular condition.
These paths aren't marked that way from the outset...it just happens that this direction or that one didn't turn out to be viable. Sometimes they are a bridge and sometimes they are a dead end. But you've got to take the road to discover and that's not always a bad thing because you know how to improve, where not to look, whatever...
Some of these clinical trials can take a long time to open for participation. After that a trial can last for a significantly long time - years sometimes.
One of the recent directions of research found from an earlier study that in people who had naturally high levels of urate, an antioxidant, had a slower Parkinson's disease progression rate. This is especially important for the understudied young onset Parkinson's patients (YOPD). See: our 1/20/10 post
For those who were following the April 2008 announcement that the MJFF was one of the sponsors of the Phase II trial of Drs. Michael Schwarzschild and Alberto Ascherio studying patients with early onset Parkinson's to determine the safety and efficacy of Inosine which raises urate levels - a potentially risky business and contraindicated under certain conditions but by no means all - there is no news except to say that the study, Safety of Urate Elevation (SURE-PD) has not begun to enroll yet. (9-2009 update: study now recruiting. See: below)
You can find the contact information and the nine states where the trials will be conducted here:
Safety of Urate Elevation in Parkinson's Disease (SURE-PD)
NCT00833690
Another grant from MJFF went to Drs. Rowen Chang and Chuantao Jiang at the Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases, part of the University of Texas Health Science Center in Houston, TX. They are developing a vaccine targeting the protein alpha-synuclein relavent to the regulation and transportation of dopamine.
Every year there are tens of thousands more PD patients. The cost of the disease is enormous on so many levels. We just wanted to express our gratitude to not only the funders and the researchers but also the trial subjects for their courage and dedication. To the patients, families and caregivers we want to demonstrate that there are many people out there working to find solutions.
2015 Addenda
In 2013 the Michael J fox Foundation published this information about the SURE-PD (Safety of Urate Elevation in Parkinson's disease.
Phase 3 of the SURE-PD study will begin enrolling in 2016. Here's a link to one news report from Science Daily, the summary of which is below:
A study from members of the research team investigating whether increasing blood levels of the antioxidant urate can slow the progression of Parkinson's disease has found that the neuroprotective effects of urate extend beyond its own antioxidant properties. An NIH-funded phase 3 trial of a urate-elevating drug, led by the senior author of the current study, will begin enrolling patients next year.
As of September 2015, I'm sure you've already see reports from many PD organizations about the urate/inosine Phase 2 study results.
Another grant from MJFF went to Drs. Rowen Chang and Chuantao Jiang at the Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases, part of the University of Texas Health Science Center in Houston, TX. They are developing a vaccine targeting the protein alpha-synuclein relavent to the regulation and transportation of dopamine.
Every year there are tens of thousands more PD patients. The cost of the disease is enormous on so many levels. We just wanted to express our gratitude to not only the funders and the researchers but also the trial subjects for their courage and dedication. To the patients, families and caregivers we want to demonstrate that there are many people out there working to find solutions.
2015 Addenda
In 2013 the Michael J fox Foundation published this information about the SURE-PD (Safety of Urate Elevation in Parkinson's disease.
Phase 3 of the SURE-PD study will begin enrolling in 2016. Here's a link to one news report from Science Daily, the summary of which is below:
A study from members of the research team investigating whether increasing blood levels of the antioxidant urate can slow the progression of Parkinson's disease has found that the neuroprotective effects of urate extend beyond its own antioxidant properties. An NIH-funded phase 3 trial of a urate-elevating drug, led by the senior author of the current study, will begin enrolling patients next year.
As of September 2015, I'm sure you've already see reports from many PD organizations about the urate/inosine Phase 2 study results.
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