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Biotech / Medical : Biotech Valuation
CRSP 56.86-2.3%3:59 PM EST

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To: scaram(o)uche who wrote (7761)1/17/2003 3:25:52 PM
From: Biomaven   of 52153
 
From a letter in this weeks NEJM from the French doctors involved:

We interpret these findings as the consequence of the insertional mutagenesis event, a risk that is potentially associated with retrovirally mediated gene transfer and that has previously been considered to be very low in humans.4 For this reason, a thorough reassessment of the potential risk of retrovirally mediated gene therapy is warranted. It is likely that additional factors may have contributed to the adverse event in our patient, including a varicella?zoster virus infection five months before clinically detectable lymphoproliferation, which may have stimulated immune reactivity of the / T-cell clone, or a selective growth advantage conferred by c expression in the transduced cells. Genetic predisposing factors for childhood cancer are also possible, since medulloblastomas have developed in the proband's sister and a first-degree relative.

So this might just have been bad luck.

Given that these particular patients die pretty quickly without this procedure, I suspect they will resume treatments using retroviruses for this disorder. But clearly this is a warning flag for retrovirus use for other, less fatal, diseases.

Peter
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