Gene Therapy for Sickle Cell Not Linked to Cancer, Bluebird Finds

Clinical trials were halted after the treatment’s vector that ferries in the healthy genetic sequence was identified in the genome of a patient’s cancer cells.

Written byLisa Winter

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The biotech company Bluebird Bio intends to resume clinical trials of a sickle cell disease gene therapy that were halted in February after a participant was diagnosed with acute myeloid leukemia five years after treatment. The company released a statement yesterday (March 10) claiming an investigation has found “it is very unlikely” the AML is related to the therapy and the firm is seeking approval from the US Food and Drug Administration (FDA) to resume the trials.

The therapy works by taking a patient’s own hematopoietic stem cells (HCS) and using a lentiviral vector to insert a healthy copy of a β-globin gene. After the patient who had received the gene therapy was diagnosed with AML, Bluebird examined the cancer cells and found the vector DNA incorporated into their genome. Upon additional testing from independent sources, researchers found that the vector landed within the VAMP4 gene ...

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Meet the Author

Lisa Winter
Lisa joined The Scientist in 2017. As social media editor, some of her duties include creating content, managing interactions, and developing strategies for the brand’s social media presence. She also contributes to the News & Opinion section of the website. Lisa holds a degree in Biological Sciences with a concentration in genetics, cell, and developmental biology from Arizona State University and has worked in science communication since 2012.
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