Originally Posted by Frank
You don't consider curing two patients a clear result?I don't understand what you mean. With "emphasized results" do you mean the results emphasized by the proponents of the therapy?
More than two patients actually!! In the Fisher's trial, we can consider that about 9 patients have been cured (I mean really cured) with one failure (an english kid who then received a standard bone marrow graft). I even consider that the three patients with leukemia-like side effects have been cured according to their immunological status. It seems good results have also been obtained by the Italian team leaded by Bordignon in a different SCID type. The main problem is that only the treatment of SCID by gene therapy can be considered now as a real success (I don't feel very confident with the information about melanoma treatment... so many things have been said from 15 years now , ... not only by the media but even in the gene therapy congresses, this is what I called "emphasized results" sorry my english is not clear-cut enough... I consider that being extremely careful is the only way to "rationalize this field",.... ). Of course, the SCID treatment is "the proof of concept" ... but, in the gene therapy treatment of SCID, the genetically modified T cells showed a "natural" proliferative advantage over non modified cells and colonized the organism up to the cure of the kids... a very specific effect to this approach which saved the protocol!! The vector technology was not better than in other protocols dealing with gene transfer into hematopoietic cells performed on the same time, the gene expression strategy was not innovative at all, the gene transfer procedure was very classical, the purification of stem cells was a routine procedure... the proliferative advantage has "erased" all the failures/weaknesses of the gene therapy technology, ... but It is unique and it helped only THIS clinical trial. All the other trials are still suffering from multiple technological and biological problems. We should not forget that gene therapy is still weak in term of therapeutical results: more than 1200 clinical trials (as announced, I think much less have been really done), more than 3000 patients, only 8 patients cured ... of course, there are a lot of explanations but it is a bad results!!! Even if I think there is a great potential and a future for gene therapy, this is what I call not being up.