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Nat Med: Gene therapy research should always focus on co-management, sharing and progress

 Last Update: 2021-05-08
 Source: Internet
 Author: User

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Gene therapy trials are increasing, but more work needs to be done to understand the long-term risks associated with this treatment.

On February 16, 2021, Bluebird Biological Company suspended its Phase 1/2 and Phase 3 clinical trials of chronic globin gene therapy for sickle cell disease.

Diagnostic Management FDA

Safety issues related to gene therapy are not new and have been highly publicized.

However, although progress has been made in dealing with the acute effects associated with gene therapy, the potential delayed effect remains a concern.

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In 2020, the FDA updated the long-term follow-up study design guidelines for delayed data collection of adverse events for gene therapy products.

Studies using integrative vectors and genome editing products will follow up patients at least.

The life expectancy should be considered based on the potential diseases and multiple comorbidities or the possibility of exposure to other agents that may themselves have long-term adverse effects.

Data needs to be shared openly and quickly with the scientific community Data needs to be shared openly and quickly with the scientific community

Delayed or incomplete data sharing is not only a safety risk for patients, it also prevents researchers from building on previous work, thereby slowing the progress of understanding of human health.

In 2018, Mila Makovec, a young girl who made global headlines for receiving gene therapy to treat rare and fatal brain diseases, passed away this year.

Now adopting a sound framework to understand the situation of patients receiving gene therapy will help balance the risks in the long run and will provide valuable insights on how to improve the long-term safety of gene therapy products so that most Patients benefit.

Gene therapy needs a long-term approach.

Gene therapy needs a long-term approach .
Nat Med 27, 563 (2021).
Https://doi.
org/10.
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