Wednesday, July 27, 2011

Advances in Diabetes Research -- without hESCs

Prominent foundations for diabetes research, led by the Juvenile Diabetes Research Foundation, have long been leading advocates for human embryonic stem cell research (hESCR), giving scant attention to the promise of adult stem cells and other alternatives to embryonic stem cells to treat this disease.

How ironic, then, that two of the most promising advances in treating diabetes have nothing at all to do with embryonic stem cells.

First, a little background. With regard to diabetes, it has long been medical orthodoxy that once the body’s insulin producing beta cells, found in the pancreas, are destroyed they are gone forever – they do not regenerate. Thus, the promise of stem cells to treat diabetes was that they could be coaxed into becoming insulin producing cells suitable for transplantation for those suffering with diabetes. Proponents of hESCR claimed that embryonic stem cells would prove ideal for this task.

It seems things are turning out differently from what they so confidently predicted.

Scientists have actually had very disappointing results in their efforts to transform hESCs into pure colonies of beta cells suitable for transplant.

However, in a recent issue of Cell Stem Cell, scientists report that induced pluripotent stem cells (iPSCs) derived from beta cells are very efficient at generating insulin producing cells that could be used for transplant.

The scientists involved in the study believe that because the iPSCs are derived from beta cells, they retain a “memory of what they once were,” thus making them actually more efficient than embryonic stem cells for generating insulin producing cells.

Lead researcher Professor Shimon Efrat of the Tel Aviv University stated, “This discovery promises to advance the development of cell replacement therapy for diabetics, possibly leading to an effective alternative to organ transplants.” He added, “When generated from human beta cells, pluripotent stem cells, these memory cells act as though they are receiving a prompt from their past life; the cells already have some understanding of their purpose, making them more efficient in generating beta cells.”

As promising as this development is, others involved in research on diabetes point out that as long as the body continues its auto-immune attack on the pancreas, where the insulin producing beta cells are found, transplanting new beta cells will not provide a permanent cure – the body will just attack these newly transplanted cells as well. So these researchers have turned their attention on finding ways to stop the auto-immune reaction against the beta cells.

In June, before a meeting of the American Diabetes Association in San Diego, Dr. Denise Faustman presented very positive findings in her research to stop the underlying auto-immune attack responsible for diabetes.

In her research with mice (which Do No Harm has closely followed and reported on over the years) Dr. Faustman found that BCG, a relatively inexpensive vaccine typically used to treat tuberculosis, also stops the auto-immune attack that destroys the insulin producing cells in the pancreas. At first, she thought this was just the first step in treatment, the second being to transplant donated islet cells now that the threat of attack had been ended.

But to her amazement, it seemed that the pancreas began to regenerate islet cells on its own, without the need for any newly-transplanted cells. This directly contradicted the prevailing orthodoxy that once an insulin producing cell is gone, it is gone (scientific consensus, anyone?). When she first published her results in 2001, they were so controversial that she was not allowed to use the term “regenerate” because the scientific community “knew” an organ could not regenerate itself.

In a 2003 paper published in Science, Faustman confirmed her finding and speculated the adult stem cells found in the spleen were responsible for aiding the pancreas to regenerate the islet cells.

This was met with even greater skepticism. Nonetheless, by 2007 her findings had been replicated in six other labs, both here and overseas.

At the June meeting in San Diego of the ADA, Faustman reported positive findings for the first use of her protocol in humans. Six patients who were diagnosed with diabetes for an average of 15 years were divided into one group that received low-level doses of the BCG vaccine and one group that received a placebo. All those receiving the vaccine showed reduced levels of the cell responsible for attacking the pancreas, as well as indications that new production of insulin had begun.

Faustman now hopes to expand her trial with a larger number of patients and to win FDA approval to increase the amount of BCG used to treat them.

So within a month of each other, we hear of two very promising findings in the treatment of diabetes. One showed that iPSCs should prove very useful in producing insulin producing cells for transplant should they be needed. And the other showed that transplants may not be needed at all if the auto-immune attack on the pancreas can be stopped, allowing the cells to regenerate on their own.

And the thundering silence you hear are the reports about all the advances using hESCs to treat diabetes.

The Spinning Never Stops

please note: this blog was originally posted on 6/21/11

The National Science Foundation (NSF) on June 10 issued a press release calling attention to a study the NSF funded on stem cell research and published in the June 9 issue of the scientific journal Cell.

“Social scientists study impact of human adult stem cell research” the headline reads, followed by: “Researchers say human adult and embryonic stem cell research is complementary.”

The release then sums up the study’s main finding: “New research says studying both adult and embryonic stem cells can benefit medical science, but banning the study of either type could harm studies of the other.” The remainder of the release elaborates on this point to the effect that any effort to cut back or eliminate federal funding for human embryonic stem cell research (hESCR) would have negative effects for adult stem cell research as well.

Now Cell is a very specialized scientific journal with a subscription rate of $212 annually, meaning most outside the scientific/research community will not have access to the full study described in the press release; for interested lay people, any information on the study will in all likelihood come from this press release.

But if you do read the original study in full, you will be struck by a rather amazing fact: what the press release reports actually has nothing to do with the study itself.

The study reports on the intersection of research using induced pluripotent stem cells iPSCs) and human embryonic stem cells (hESCs). It has nothing at all to do with adult stem cell research, adult stem cell research is never addressed or discussed and in fact the word “adult” only occurs twice in the whole study and on the last page at that (one is a reference to “two adult stem cell researchers” who brought a lawsuit challenging federal funding of hESCR and the other is at the very end of the study reflecting an opinion of the authors).

So outrageous was the deceptiveness of the press release vis-a-vis the actual study itself that David Prentice, PhD, a Do No Harm founding member and Senior Fellow for Life Sciences at the Family Research Council, said he was “appalled that the National Science Foundation would publish an ideological paper that promotes embryo-destructive research by attempting to link such research to advances in iPS cell research. While even this possible linkage is questionable based on the limits of the data presented, NSF in its headlong rush to promote ES cell research goes over the edge in confusing and prejudicing the public.”

So where’s the confusion and why does it matter?

By equating iPSCs with adult stem cells, the press release deliberately confuses what are two separate and very distinct types of stem cells. As Dr. Prentice noted in his statement on the study and release: “While iPS cells provide an ethical method to form pluripotent stem cells almost identical to ES cells, from any person, but without embryo destruction, iPS cells are not adult stem cells” (emphasis added).

Why does this matter?

Because the release (which, as already noted, is where most interested lay people would find out about this study) would have you believe that the proven and ongoing success of adult stem cells to actually provide real therapeutic benefits to patients depends on continued federal funding for embryo destructive stem cell research. And this is simply not true, nor has it ever been true.

Adult stem cells have provided therapeutic benefits for human patients for 73 diseases and conditions, including spinal cord injury; heart disease; multiple sclerosis; lupus; sickle cell anemia and Parkinson’s, among others. In contrast, only two patients have been treated with hESCs, both in a clinical trial for spinal cord injury (no results have been reported yet) and two more are slated to receive injections of hESCs for two different types of vision loss.

The success that adult stem cell research has had in treating patients did not come from the use of hESCs and does not in any way depend on continued federal funding for embryo-destructive research. In fact, the case could be made that the exact opposite is true: continued federal funding for hESCR may actually be hurting adult stem cell research by drawing limited funds away from it -- the very research that is actually helping patients now. As evidence of this, consider that a federal appeals court agreed with two adult stem cell researchers that by funding ESC research, the Administration is depriving adult stem cell researchers of the opportunity they would otherwise have to access these funds and advance stem cell research that is ethically non-controversial and which everyone accepts. This “competitive disadvantage” is what gave the two adult stem cell researchers standing to sue the Department of Health and Human Services.

The NSF press release is just another misdirection, the latest in a long string of deceptions and falsehoods proponents of embryo-destructive research have used to advance their agenda.

Wednesday, July 20, 2011

New Advances

An article in the current issue of the New Scientist focuses on some recent – and major – developments in the both real and potential therapeutic benefits from stem cells.

“Stem cell therapies ready for the real world” the article states.

Ready for the real world. For all the hype over embryonic stem cells for at least 10 years now, you might think the article would be about them, right?

Wrong. The article instead celebrates ongoing therapeutic advances utilizing adult stem cells.

The first involves the successful transplant of an artificial trachea (windpipe) into a patient whose own, cancerous trachea had been removed. Modeled on the diseased trachea, a new one was constructed of a “novel polymeric material.” This was then coated, inside and out, with the patient’s own cells that had been derived from stem cells in the patient’s bone marrow. The Karolinska University Hospital in Stockholm, Sweden, where the transplant took place, issued a statement noting that "because the cells used to regenerate the trachea were the patient's own, there has been no rejection of the transplant and the patient is not taking (anti-rejection) drugs." The patient has recovered and been sent home.

The next advance noted in the New Scientist involves a South Korean company which became the first in the world to receive official approval for the sale and use of a stem cell treatment for heart disease. Called “Hearticellgram-AMI” the treatment takes the patient’s own bone marrow derived adult stem cells, multiplies and then injects them directly into the coronary arteries to regenerate cells damaged or lost due to acute myocardial infarction.

The article also refers to a recent development that saw Japanese scientists successfully grow teeth using tissue stem cells derived from fetal mice (the New Scientist mistakenly refers to them as “embryonic” stem cells). The treated stem cells were grown in a box placed within a mouse’s kidney; they then developed into fully formed teeth with ligament and bone; when transplanted into the mouse, the teeth connected with the animal’s blood and nerve supply to function as naturally formed teeth. According to one news report, Professor David Leavesley, from the Institute of Health and Biomedical Innovation at Queensland (Australia) University of Technology, said this development suggested adult stem cell could be used to create other complex organs such a liver, pancreas and even eyes.

Finally, the article notes that scientists had isolated the “mother” stem cell for blood, from which both red and white blood cells are derived. “This raises the possibility of being able to completely reconstitute a patient's blood - perhaps after chemotherapy for leukaemia - from a single cell extracted from bone marrow before treatment began,” the New Scientist notes. The study was published in Science.

Embryonic stem cells are nowhere even close to duplicating these advances. Yet more reason to direct limited resources away from embryonic stem cell research and into research that is actually helping patients…adult stem cell research.

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