In a major leap forward for regenerative medicine, science in general, and potentially bioethics, researchers at the Salk Institute have combined stem cell therapy with gene therapy to cure a human disease. The details of the article are provided below:
Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells
The generation of induced pluripotent stem (iPS) cells has enabled the derivation of patient-specific pluripotent cells and provided valuable experimental platforms to model human disease. Patient-specific iPS cells are also thought to hold great therapeutic potential, although direct evidence for this is still lacking. Here we show that, on correction of the genetic defect, somatic cells from Fanconi anaemia patients can be reprogrammed to pluripotency to generate patient-specific iPS cells. These cell lines appear indistinguishable from human embryonic stem cells and iPS cells from healthy individuals. Most importantly, we show that corrected Fanconi-anaemia-specific iPS cells can give rise to haematopoietic progenitors of the myeloid and erythroid lineages that are phenotypically normal, that is, disease-free. These data offer proof-of-concept that iPS cell technology can be used for the generation of disease-corrected, patient-specific cells with potential value for cell therapy applications.
The first sentence above introduces the concept of induced pluripotent stem (iPS), which are pluripotent cells artificially created from non-pluripotent cells. iPS’s are generally created by forcing adult somatic cells to become pluripotent by inducing the expression certain genes. iPSC’s were first created in 2006 using mouse cells, and in 2007 using human cells. This was a hugely significant advancement in stem cell based therapies; the use of iPS’s will hopefully allow researchers to obtain pluripotent stem cells, which are important in research and are believed to have great therapeutic potential, without harvesting and destroying human embryos.
The remainder of the abstract can be translated loosely to: Gene therapy was used to correct a genetic defect present in a laboratory based human cell line – human cells growing in culture – which can then be coaxed to generate patient-specific iPS. Further, these iPS’s can also give rise to the progenitor cells that form the blood cells where the disease manifests itself.
Essentially – and this is simply a proof concept paper – this research suggests that one can isolate adult cells, genetically reprogram them, induce the genetically reprogrammed cells to become pluripotent stem cells, and induce those cells to become the appropriate progenitor cells, capable of giving rise to healthy somatic cells.
Researchers obtained samples of hair or skin cells from persons known to have Fanconi anemia. The defective gene was corrected within the patients’ cells, using gene therapy methods pioneered by the researchers themselves. The team then successfully reprogrammed the genetically altered and ‘repaired’ cells to form iPS cells by using a combination of transcription factors, SOX2, KLF4, OCT4, and cMYC. The resulting genetically corrected iPS cells were indistinguishable from either human embryonic stem cells or iPS cells generated from healthy donors. The article further reports that the genetically altered cells readily differentiated into the hematopoietic progenitor cells that will ultimately give rise to healthy blood cells.
There’s a larger issue buried within this good news as well – more good news buried within this good news. This breakthrough is a significant advance in stem cell based therapy that doesn’t involve the destruction of human embryos. Despite the media hype surrounding the use of embryonic stem cell therapies, there are currently more than 100 successful adult stem cell based therapies that effectively treat diseases such as brain injury, diabetes, bone cancer, aplastic anemia, stroke, retina regeneration, heart tissue regeneration, angina, nerve regeneration, cerebral palsy, cartilage regeneration, Parkinson’s, kidney damage, liver cancer, lupus, multiple sclerosis, leukemia and more. Thus far there are NONE for embryonic stem cell based therapies, and in fact embryonic stem cells have a history of inducing tumor formation.
Don Margolis maintains an excellent blog that details the successes of adult stem cell based therapies; you could spend all day looking at his site. A more condensed summary of successful adult stem cell based therapies can be viewed here.
As the evidence mounts, supporting the efficacy of adult stem cell based therapies, the mainstream media is ignoring it, reporting on the need for embryonic stem cell based therapies, despite the difficulties surrounding their therapeutic use.
Even worse than the media ignoring it our ultra pro-abortion President, Barack Obama, is actually undermining the effort to develop proven adult stem cell based therapies, and pouring more Federal monies into embryonic stem cell based therapies. Obama has co-sponsored a bill that would not only authorize the large-scale industrial production of human embryos, whose fate is specifically to be killed for use in medical research, but also effectively requires the killing of human embryos produced by cloning. The bill actually makes implantation and bringing a cloned embryo to term a federal crime.
Read that last sentence again.
Affectionately coined the ”clone and kill” bill, this bill will bring legally mandated abortion to the United States of America, something we tend to associate with oppressive government regimes, like that in China. In classic newspeak, Obama and the bill’s sponsors refer to this as an anti-cloning bill. The bill doesn’t ban the process of cloning a human, but rather bans allowing the cloned human embryos to survive.
This somewhat akin to calling opposition to the death penalty an anti-abortion position.
Even more disturbing than Obama’s position on the relative status of human embryos (think wisdom teeth), is his position on alternatives to embryonic stem cell based therapies. The article that prompted this blog post details one such alternative. Formation of iPS’s represent one feasible – and proven – alternative to the destruction of embryos as a source of pluripotent stem cells. However, when a bill was introduced in the US Senate that proposed investing a relatively small sum of federal money into research to develop alternatives to embryonic stem cells, Obama was one only a few senators who opposed it.
Why?
Why would anyone not desire a method to produce pluripotent cells scientists believe will move research forward that doesn’t involve the destruction of human embryos? Why mandate that scientists clone and kill human embryos when there are feasible, workable, and realistic alternatives that do not require destruction of human embryos?
Or as Robert George stated in his excellent article linked above: “It is as if Obama is opposed to stem-cell research unless it involves killing human embryos.”
Despite making grandiose claims about restoring “science to it’s rightful place…” Obama seems to be ignoring the science in favor of his own personal and/or political agenda. Were one to follow the data, and do what the science says we should, we would pour our research dollars into adult stem cell based therapies, not glorify a culture of death.
What is science’s rightful place? Based on what Obama has said, and how he’s forming his policy, it seems that Obama is confused. He seems to be letting science dictate his policy; he seems to be operating under an assumption that is loosely based around the notion that if we can do something we should do it. When science dictates policy it’s inevitable that atrocities will occur, the Nazi medical experiments are one such example. Indeed, American Eugenics, and ultimately Nazi genocide arose, logically from science, specifically the science of artificial selection and animal breeding. That is an example of science taken to its logical extreme and subsequently dictating public policy.
There is nothing scientifically incorrect about attempting to breed ‘better’ humans – whatever better means – but there is something morally repugnant about it.
Science has nothing to say about what should be done. Science has no foundation in ethics, and indeed produces no ethical conclusions. Science can tell us about the world around us, how things work, when things will occur, etc. Science can only tell us about what is possible, not what is moral. President Obama and his supporters would do well to learn the history surrounding this idea.
The Reactionary Researcher Blog 