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

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Anthony-Quintano-CC

Gene Tarne - published on 09/09/14

You'd be amazed at the progress to date in treating patients using adult stem cells vs. nada from embryonic.

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However unintentionally, the ALSIce Bucket Challenge has again focused attention on the ethical problems inherent to human embryonic stem cell research (hESCR).  

At the same time, the challenge provides an opportunity to highlight the very real progress being made to provide therapeutic benefits for patients in research utilizing ethically non-contentious adult and other non-embryonic stem cells—for ALS and many other diseases.

The ethical hurdle that hESCR cannot overcome is that in order to obtain hESCs, a living human embryo must be destroyed; destruction of human life is inherent to the research. The Vatican is clear in its rejection of such research:

“The obtaining of stem cells from a living human embryo … invariably causes the death of the embryo and is consequently gravely illicit: ‘research, in such cases, irrespective of efficacious therapeutic results, is not truly at the service of humanity. In fact, this research advances through the suppression of human lives that are equal in dignity to the lives of other human individuals and to the lives of the researchers themselves. History itself has condemned such a science in the past and will condemn it in the future, not only because it lacks the light of God but also because it lacks humanity’ ” (at no. 32 and quoting Benedict XVI).

And in a 2003 address St. John Paul II told the Pontifical Academy of Sciences that “any treatment which claims to save human lives, yet is based upon the destruction of human life in its embryonic state, is logically and morally contradictory, as is any production of human embryos for the direct or indirect purpose of experimentation or eventual destruction.”

As is well known by now, the ALS Association (ALSA) is funding at least one hESCR project, causing many would be donors to seek other organizations for their contributions —a response demonstrating that concerns over the ethical problems inherent to hESCR have not diminished since hESCs were first isolated in 1998.  

But ALS support for hESCR goes beyond this one project. ALS describes itself as an “active member” of the Coalition for the Advancement of Medical Research (CAMR, now the Alliance for Regenerative Medicine), a major advocate for hESCR. Early in the Obama administration, ALSA posted a statement urging the new administration to lift restrictions on federal funding of hESCR and posted a link to a letter from CAMR that urged the same.

In addition, ALSA has also directed money to the Northeast ALS (NEALS) Consortium, which is funding a study that uses cells derived from an aborted fetus.

As news of ALSA support for hESCR spread, the organization issued a statement attempting to clarify its position. While the statement did not disavow support for hESCR, it was at pains to distance ALSA from it. The statement noted that ALSA is funding only one hESCR project; that the project is funded by a single individual; and that it uses a hESC line established years ago. “In fact,” the statement continues, “donors may stipulate that their funds not be invested in this study or any stem cell project” (emphasis in the original).

ALSA also scrubbed a page on its website entitled “A Primer on Stem Cells.” The original page, as of 8/19/14, included language on the necessity hESCR:

“Adult stem cell research is important and should be done alongside embryonic stem cell research as both will provide valuable insights. Only through exploration of all types of stem cell research will scientists find the most efficient and effective way to treat diseases.”

By 8/26/14, after news spread that ALSA funds hESCR, that
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
, in November, 2011. Geron, the company sponsoring the trial, cited financial difficulties in calling it off , but others were skeptical and called attention to other factors contributing to the trial’s demise, such as “the potential pitfalls associated with specific types of stem cell technologies – namely, embryonic stem cells…” (Note: California-based Asterias Biotherapeutics Inc. recently announced that it would resume this trial, picking up where Geron left off.)

Another telling development is the way state agencies that fund stem cell research have been devoting more and more resources to non-embryonic stem cell research.

Apart from the NIH, the California Institute for Regenerative Medicine (CIRM) is the nation’s largest funder of stem cell research. CIRM was approved by voter referendum in 2004, with a mission to give priority funding to hESCR. True to its mission, in its first two rounds of grants awarded in 2007, CIRM funded just over 100 research projects using hESCs and/or Somatic Cell Nuclear Transfer (SCNT, i.e., human cloning). No funds went to adult stem cell research projects.  

Over the years, however, CIRM has more and more shifted resources so that by 2009, up until today, CIRM has funded substantially more adult, induced pluripotent and other non-embryonic avenues of stem cell research. (here, here, here and here). Noticing this shift, in 2009, the Los Angeles Times reported:

“For 3 1/2 years, the agency focused on the basic groundwork needed to someday use human embryonic stem cells to replace body parts damaged by injury or disease. Such cures are still far in the future. Now the institute has a more immediate goal: boosting therapies that are much further along in development and more often rely on less glamorous adult stem cells.”

In 2012, the first CIRM-funded project to receive FDA approval for clinical trial was announced—a treatment for heart disease using adult stem cells.

A similar trend can also be seen in Maryland, which also provides state funds for hESCR.  The first round of grants, awarded in 2007, strongly favored hESCR. By 2013, 90 percent of the grants awarded went to non-embryonic stem cell research projects.  

President Clinton’s National Bioethics Advisory Committee (NBAC) was the first, in 1999, to recommend federal funding for hESCR — but it did so conditionally.

Recognizing the legitimacy of the ethical concerns raised by such research, NBAC said that destroying human embryos for research was “justifiable only if no less morally problematic alternatives are available for advancing the research” (at pdf pg. 78). While judging such alternatives were not available at that time, NBAC said that judgment could change as it “is a matter that must be revisited continually as science advances.”

Clearly those alternatives exist—so much so that the non-embryonic adult and induced pluripotent stem cells “alternatives” are clearly outpacing hESC in providing actual therapeutic benefits to human patients.

Gene Tarne is a senior analyst with the Charlotte Lozier Institute (lozierinstitute.org). He has been involved with the life issues for over 20 years.

Note: Should you participate in the Ice Bucket Challenge and want to contribute to ethically sound, non-embryonic stem cell research, here are some options:

The Midwest Stem Cell Therapy Center (MSCTC) at the University of Kansas Medical Center is only a year old, but is starting an increasing number of clinical trials and educational efforts. One potential future trial would be using adult stem cells for ALS.  Dr. Rick Barohn, an internationally recognized expert on ALS, recently joined the Advisory Board for the Center. The MSCTC does not do any embryonic or aborted fetal stem cell research, only adult and non-embryonic stem cell research and clinical trials. To donate click the “Make a Gift” link in the left column of their web page.

The John Paul II Medical Research Institute in Iowa City is doing research in several areas including ALS, and does not support embryonic stem cell research. To donate, click the button for “Donate Now” on their main web page

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