in-PharmaTechnologist.comGene therapy risks must be evaluated in each clinical trial

A news published last October 1 in the Wall Street Journal (see HERE) affirms that the prospects for the human use of gene therapy enter a dark phase when the viability of its authorized clinical trials is questioned after a young man has died as a result of the gene therapy treatment that was applied to cure a neuromuscular disease in September in Japan.

According to this newspaper, it is not the only case: several young people subjected to this treatment have died. Based on the study of the Japanese laboratory Astellas Pharma on the case, mentioning that it also includes the previous deaths of three young people in treatment with this technique, the FDA called a meeting to analyze the feasibility of continuing with the authorized clinical trials.

See articles published on gene therapy risks HERE.

Some specialists claim that these side effects could be an effect of a large number of treatments started for which the risk/benefit balance should be evaluated before taking any measure.

Astellas Pharma proposes as a conclusion of the referred study to continue with the therapies and to include new volunteers, excluding those who have or have had liver disease, which seems to be the cause of these deaths. Most gene therapies pass through the liver before reaching the cells being treated, and the report states that large doses can seriously affect this organ.

The advanced technology of gene therapy has offered great hope of curing, mainly, hereditary diseases that are increasing due to the rising age of the parents (see HERE) and the significant increase in “in vitro fertilization” (see HERE).

The stock market price of the pharmaceutical companies that lead the treatments has dropped significantly, which is seen as a loss of investor confidence and a possible decrease in investments in research and development in gene therapy.

Beyond the economic aspects that this new phase presents, after the aforementioned deaths, the bioethical problem of continuing with gene therapies at this point in their development must be resolved, also assessing possible short and long-term side effects.

Photo: American Gene Technologies

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