Gene Therapy - Successess?

[darkwng]

What have been the successess of gene therapy? Besides the bubble boy "success", are there any other cases of curing monogenic diseases or other disease?

[kmunson779]

A google search for "successful gene therapy" (without the quotes) brings up a bunch of articles. Also see "Recent Developments" in the Wikipedia entry.

[Frank]

Quote from WIkipedia:

Scientist at the National Institutes of Health (Bethesda, MD) have successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells. This study constitutes the first demonstration that gene therapy can be effective in treating cancer. The study results have been published in Science (October 2006).

In addition there seem to be many positive results with gene therapies in mice and dogs, and also many ongoing clinical trials in humans. I expect the next ten years will see increasing numbers of succesful gene therapy treatments

[molecule2005]

Frank that is some great new information.

I think it is getting better as well!

[Cebe]

A success in this field should be carefully double-checked and confirmed before it can really be considered as a real therapeutic success. Up to now, only the gene therapy of some immunodeficiencies should be considered as a real cure!! there is no clear results when considering the cancer gene therapy strategy. We will of evidence learn a lot for the next ten years as mentionned, but the main problem will be to discriminate between "emphasized results" and " therapeutical success"!!!

[Frank]

Hi Cebe,

Originally Posted by Cebe

there is no clear results when considering the cancer gene therapy strategy.

You don't consider curing two patients a clear result?

Originally Posted by Cebe

We will of evidence learn a lot for the next ten years as mentionned, but the main problem will be to discriminate between "emphasized results" and " therapeutical success"!!!

I don't understand what you mean. With "emphasized results" do you mean the results emphasized by the proponents of the therapy?

[Frank]

Gene Therapy For Erectile Dysfunction Shows Promise In Clinical Trial

Science Daily — The first human trial of gene transfer therapy for erectile dysfunction (ED) indicates that gene therapy that lasts for months and eliminates the patient’s need for on-demand drugs (such as V iagra and Cialis), could become the future treatment of choice for this common problem, according to a paper in the most recent issue of Human Gene Therapy.

http://www.sciencedaily.com/releases...1201180435.htm

[Cebe]

Originally Posted by Frank

Hi Cebe,


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.
Cebe

[Cebe]

[quote=Cebe;1941]

Hi Frank

Just to explain myself a little more about "emphasized results"...

Many things have been said in this field since 1990. A lot of pseudo-positive results even reported sometimes by the investigators and/or the promoters of the trials have finally been shown exaggerated or wrong, ... I heard so many positive and enthousiastic talks with no therapeutical results nor improvments when looking at the results carefully that I don't feel very confident with results not confirmed by other labs. Some guys are very serious, .. Alain Fisher who conducted the SCID treatment in France is such a guy!! The main problem has been (ans is still) the control of the information by the biotech companies. The field suffered a lot from this very bad communication strategy

Cebe

[Frank]

Hi Cebe,

thanks for your input! You seem to know a lot about this subject, do you work in this field?