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ALS Ice Bucket Challenge Prompts A Deeper Look at Stem Cell Research

Anthony-Quintano-CC

Gene Tarne - published on 09/09/14


language had been removed  from the primer.

In marked contrast to the distancing from hESCR, the statement all but embraced non-embryonic stem cell research, especially research using (iPSCs):

“The ALS Association primarily funds adult stem cell research … Many labs have replaced ESCs with induced pluripotent stem cells (iPS cells). These iPS cells begin as adult human skin cells but are then reprogrammed to become stem cells, which are then ready to become other cells types (emphasis in the original)”.

There’s good reason why ALSA would want to emphasize support for adult and iPSC research: this research is proving the most promising in leading to clinical trials and in providing real therapeutic benefits for patients, despite all the hype that for more than a decade has exaggerated the medical potential of hESCR to cure any number of diseases and conditions.  

Another organization focusing on ALS, the ALS Therapy Development Institute, describes itself as the “#1 nonprofit biotechnology organization dedicated to developing effective treatments for ALS.” A reporter for the Massachusetts Citizens for Life blogspot called ALSTDI to ask if it funds hESCR and was told “they (ALSTDI) do not do any research with embryonic stem cells because they think induced pluripotent adult stem cells are the best avenue to a cure.”

Already, researchers are enrolling ALS patients for using adult stem cells. One of those trials is in collaboration with BrainStorm Therapeutics, an Israeli company. BrainStorm has already seen positive results in a separate trial using adult stem cells.

In animal models, researchers have used iPSCs to treat ALS, with very positive results.

As far back as 2008, researchers using the iPSC process took skin cells from an ALS patient to produce motor neurons and glia, giving researchers an abundant supply of cells carrying the genetic defects associated with ALS to study the progression of the disease. One report called this “a veritable godsend for ALS research.”

More recently, another team of researchers did the same with Down Syndrome patients, giving them a supply of cells to model the disease and enabling the researchers to gain new insights into the origin and development of Down. According to one of the researchers, “the advent of induced pluripotent stem cell technology has created exciting new approaches to model neurodevelopmental and neurodegenerative diseases for the study of pathogenesis and for drug screening.”

And not just for neurological diseases; the iPSC process is being used to create patient specific stem cells to create models to study other types of disease as well, greatly enhancing progress in the field of regenerative medicine.     

Adult stem cells have also provided therapeutic benefits for a whole host of diseases and conditions, including heart disease, Type 1 diabetesParkinson’s, MS, spinal cord injury and many others.

Adult stem cells have also been used to generate whole organs, including tracheas (one of which was used to replace a patient’s cancerous trachea, saving his life);  nose, ears, tear ducts and blood vessels; bladders, and a rudimentary liver, among other developments.

In marked contrast, hESC have been tested in human patients in only three experimental clinical trials.  Two of those trials tested hESCs on two different forms of macular degeneration; the trials are ongoing, and no valid results have been reported.

A third trial using hESCs was to treat patients with spinal cord injury.” The trial began in October, 2010, but was halted just over a year later

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