May 31, 2009

expert reaction to stem cell genetic reprogramming, as published in Nature

Scientists have shown that they are able to reprogramme skin cells taken from patients with a rare inherited blood disorder to make cells that are virtually identical to embryonic stem cells, making them potentially suitable for use as a treatment.

Prof Chris Mathew, Professor of Molecular Genetics at King’s College London, said:

“This is an important development for families with this rare, inherited blood disorder. The patients have low numbers of blood stem cells in their bone-marrow, so there are very few target cells to correct by gene therapy. The new research shows that it is possible to re-program skin cells from these patients into stem cells in which the genetic defect has been corrected. In future it may become possible to transfer the corrected stem cells back into the patient, but much work remains to be done before this can be transferred from the lab bench to the bedside.”

Prof Chris Mason, Professor of Regenerative Medicine at University College London, said:

“”This research demonstrates a very novel method of generating cells to potentially treat a rare inherited disorder. There is no doubt that this paper will be the first of many to offer hope for conditions where today there is no real therapy, let alone a cure. If science is not to over-promise and under-deliver, significant resources must be committed to the next step, namely the translation into safe, effective and affordable therapies.

“Whilst the basic science research takes months and costs hundreds of thousands of pounds, the translation will take many years and cost hundreds of millions. There is no guarantee of success; without the commitment to stem cell translation, however, we will never progress beyond great scientific discoveries and therefore patients will never have the chance to benefit. The question is this: who is prepared to make this substantial investment? Is it big pharma, the traditional pioneer of new therapies, or does it fall to governments to reap the rewards of their investment in the basic science?”