Friday, October 10, 2014

Non-Embryonic Stem Cell “Alternatives” Again Taking the Lead

The New York Times has long been – and continues to be – a vocal advocate for human embryonic stem cell research (e.g., here).  From the time hESCs were first isolated in 1998, the Times has published numerous editorials endorsing the research and calling for federal funding of it.

So a recent Times story providing a look at where stem cell research stands today was – no doubt unintentionally – revealing.

In marked contrast to the delirious enthusiasm with which advocates promoted hESCR,  promising it would lead to cures for virtually all diseases and conditions (one prominent politician at the time said human embryonic stem cells could become a “veritable fountain of youth”), the tone of the article is far more cautious and restrained in assessing what advances have actually been made to date in the field of  stem cell research.

The article’s title sets the newly sober tone: “The Trials of Stem Cell Therapy;” “trial” here can mean “to test,” as in a clinical trial, but also “difficult” as in the difficulties researchers face in bringing stem cells to therapy.  The article calls the process “halting” and notes that “progress has been slow.” 

But while progress may be “slow” and “halting” the article does note some promising developments.

And they are all being accomplished with adult and other non-embryonic sources of stem cells.

The article opens anecdotally, with the story of a heart disease patient who lost one-third of his heart’s functioning ability.  The patient volunteered for a medical trial in which researchers injected adult stem cells, derived from the patient’s own bone-marrow, directly into his heart.  While the article notes that “It’s impossible to know for sure whether the bone marrow cells’ descendants became heart muscle cells or if repairs were spurred some other way,” doctors nonetheless were able to tell the patient that his heart “is one-third of the way back to normal.”  The patient himself is quoted saying, “My quality of life is like night and day to before the treatment.”

The article further notes there are “as many as 4,500 clinical trials involving stem cells” currently underway in the U.S. for a whole host of diseases and conditions, but it does not clarify that virtually all of these trials are using non-embryonic stem cells.  That’s because only three trials using embryonic stem cells have been approved here: two are testing hESCs in two types of macular degeneration; they are ongoing and valid results have not yet been reported.  The third approved trial was for spinal cord injury; however, it was halted in 2011, a little over a year after it began. The California-based Asterias Biotherapeutics Inc. recently announced that it would resume the trial.

Another sign of progress mentioned in the article is that the California Institute of Regenerative Medicine (CIRM) has awarded over $2 billion since 2006 to stem cell researchers, and that it is enrolling patients for 10 clinical trials.
Unmentioned again, is the fact that the clear majority of these trials all involve non-embryonic stem cells.

A look at CIRM’s website shows a list of eight clinical trials “directly funded by grants from CIRM.”  Of these eight, just one employed embryonic stem cells, and it was the one mentioned above for spinal cord injury that was eventually shut down.

As for CIRM’s $2 billion in research grants awarded, this blog has noted several times that, over the years, more and more of the money awarded by CIRM has gone to fund adult and other non-embryonic stem cell research projects (here, here, and here).

The article also references the work being done using non-embryonic, induced pluripotent stem cells (iPSCs) for disease modeling and drug testing.  Dr.  Kevin Eggan of Harvard, the article notes, has used the iPSC process to create patient-specific stem cells from two patients with ALS (i.e., Lou Gehrig’s disease) and then coaxed those cells into becoming neurons.  Dr. Eggan noticed a signaling defect between those neurons that appeared to cause the neural degeneration typical of ALS. After extensive testing on the iPSC-derived neurons, Dr. Eggan has singled out a drug currently used to treat epilepsy that may correct the defect in the ALS neurons, which he expects to test on patients by the end of this year.

Commenting on the use of iPSCs for such disease modeling and drug testing, Dr. Eggan said that “the whole process is something that’s never been remotely possible before.”

Other researchers agree.  Earlier this year, in July, scientists used this same method to study Down syndrome, allowing them to discover a possible cause of the condition and a possible drug to treat it. “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,” said David Pleasure, a coauthor of the study.

The more sober approach to reporting on the therapeutic progress of stem cell research, as reflected in this New York Times article is, no doubt, a welcome development.

But however “halting” such progress may be, it is important to note, as the examples cited by the Times article – however unwittingly – show, that virtually all of it is being made on the non-embryonic stem cell front.

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