gene therapy cures scid patients

We have all heard what happened to Jesse Gelsinger and the bubble boy (SCID) patients when treated with gene therapy. Well, a modified version of that therapy has now cured young patients of SCID with no unwanted side affects. This is just more mounting evidence supporting the commercialisation of gene therapies, including ddRNAi.


"Anyone who has followed the long twisted road of developing gene therapy knows the story of the big oops that happened with the first large trial in Severe Combined Immune Deficiency (SCID). This fatal disease is sometimes called “bubble boy” disease referring to a patient that was kept alive by living behind a clear barrier that protected him from the infectious agents that inevitably kill children with this genetic disease.

In that infamous trial, researchers harvested the patients’ own bone marrow stem cells, used a retrovirus to insert the gene needed to correct the defect, and gave the cells back to the patients. This cured the disease in 18 out of 20 kids. But five of them developed leukemia a few years later.

This sent the field scrambling. The result was the story told this morning in the opening talk of a plenary session of the International Society for Stem Cell Research annual meeting. They did two things. First they formed a seven-center international consortium hoping the large diverse group could collectively come up with solutions more quickly, and when the next trial began, recruit patients more quickly. They also went from the bedside back to the bench to figure out exactly what caused the leukemias.

One of the three leaders of the consortium, Indiana University’s David Williams, described the extensive effort to figure out how the virus that carried in the therapeutic gene caused the unwanted gene alteration that led to leukemia. They found that the virus caused a gene to be inserted next to a cancer causing gene that turned it on. They then set out to alter the virus to avoid this gene misplacement. That task was relatively rapid, however, compared to proving to the regulatory bodies that the change really did the trick. That required many different experiments spanning more than a year.

The resulting clinical trial is underway at five centers, and five of the first eight patients seem to be cured. None have shown any unwanted effects and the first child is two years out from his treatment. The consortium now has clinical trials underway for four diseases. Worldwide, gene therapy has shown promise in early tests in patients for at least 10 diseases."

http://cirmresearch.blogspot.com.au/2013/06/isscr-gene-therapy-shows-power-of-going.html