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Genetic therapy cures thalassaemia

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Andy Battaglia:
I'll quote from the above article, which explains why chemo is needed for gene therapy.


--- Quote ---With the gene successfully transferred, the patient was subjected to a high dose of chemotherapy before transplanting the genetically modified haematopoietic stem cells. High dose chemotherapy treatment ensured that all diseased stem cells were destroyed. This ensured that the effects of the genetically modified stem cells introduced were not diluted and hence the outcome not compromised.
--- End quote ---

Cari:
I found this article in outre local paper. http://epaper.orlandosentinel.com/OS/OS/2010/09/23/ArticleHtmls/23_09_2010_002_003.shtml

Syaida Lee:
Andy,
In saying "There is no possibility of rejection as there is with BMT, so there is no host versus graft disease", are you saying even if there is a perfect bone marrow match for my child (he has 3 other siblings), gene therapy would be a preferred method of cure?

"Both procedures require chemo to work and in both cases, there are doctors who have found that using a less harsh mix of chemo produced better results in terms of side effects from the process. Dr Krishnamurti in Pittsburgh has been a pioneer in using less harsh chemo regimens for BMT and Dr Sadelain's group have been pioneers in finding less harsh chemo to use in gene therapy"-
Logically, for Thal Major, there's only 1 defective gene, as oppose to other reasons why one would require a stem cell transplant for cancer. That's my logical deduction (?)

Andy Battaglia:
Syaida,

It isn't yet possible to compare BMT and gene therapy because BMT is a reality and gene therapy is in development and won't be perfected for some years. There are two issues with gene therapy that have to be resolved. One is controlling it so that it doesn't grow out of control and cause tumors. Second, it has to sustain. If the effect doesn't last permanently, its value will be lessened.
But, the hopes are it will work and if this does work as hoped, it would have less risk than BMT. However, decisions have to be made in the real world, so until we know more about the successes of gene therapy, it's difficult to consider it as an option to BMT.

What has been happening in BMT with chemotherapy, is that some doctors have discovered that the regimens being used were not totally necessary. Refining the chemo reduces the side effects and makes it easier on the patient. The comparison between stem cells for cancer and for thalassemia is that in both cases, the patients bone marrow is destroyed and replaced. In both cases, the bone marrow is not functioning properly, so it is replaced with stems cells that do work properly.

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