Other US News of Gene Therapy


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Currently three groups of researchers are planning to carry out trials in the USA. All three spoke on the Gene Transplant panel at the National Gaucher Foundation's Conference held in Philadelphia November 1994.

Dr John Barranger University of Pittsburgh spoke in more technical detail on the same subject at our own conference.

The second speaker Dr Donald Kohn Children's Hospital Los Angeles has already been involved in actual gene therapy of patients with ADA deficiency. Although these trials have to date shown no adverse side effects from the therapy, they have not been successful in treating the disorder.

His method is to transplant the genetically altered material into the bone marrow. His work indicates that gene transplants in Gauchers patients may have to involve chemotherapy and radiation in order to kill the remaining unaltered bone marrow cells. However this involves risk as it would take 2 to 4 weeks for the genetically altered cells to grow to replace those killed and there is the risk of death for up to 5% of patients. There is also hair loss and nausea associated with the treatment.

It was noted that none of the three trials of gene therapy will currently involve chemotherapy or radiation. Dr Kohn's study is in conjunction with Drs Brady and Barton at the National Institute of Health, Washington DC. Dr Kohn's group are using a different genetically altered retrovirus to the one used by Dr Barranger.

Dr Friedrich Schuening Fred Hutchinson Cancer Research Centre, Seattle reported on trials using dogs who were subjected to the procedure proposed by Dr Barranger for human clinical trials. He reported how their experiments in dogs had shown that those which had received chemotherapy or radiation had some uptake of genetically altered material while those without had little or none. He concluded that current capabilities for gene therapy were modest. On the positive side, he reported that gene therapy appeared to be relatively safe as he had healthy dogs five years after they had received a gene transplant.


Source: Gauchers News February 1995
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