Wolfgang Miesbach: How Safe Is AAV Gene Therapy?
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn about a recent article by Lindsey A George et al., published in Molecular Therapy, adding:
“6,000+ people treated. Nine tumors reported.
When a cancer shows up after AAV gene therapy, was the treatment to blame, or just chance?
That’s the new Opinion piece in Molecular Therapy by Lindsey George, Rebecca Ahrens-Nicklas and Frederic Bushman.
First, the reassuring part.
AAV gene therapy has a strong safety record. Across an estimated 6,000 plus people treated, only nine tumors have ever been reported afterward – and just one carries clear molecular proof that the vector itself caused the cancer.
That one case is sobering.
A child treated for MPS I at 13 months developed a brain tumor four years later.
The vector had landed inside a gene called PLAG1 and switched it on – using its own built-in regulatory elements to do it.
Encouragingly, the child remains neurologically intact.
The deeper lesson is about proof.
Vector DNA often inserts in a scrambled, rearranged mess, tumor tissue is scarce, and routine clinical labs simply don’t look for it.
In this case the team’s first four methods couldn’t reconstruct what had happened. It took long-read sequencing to finally map it.
Rare doesn’t mean impossible.
The authors make a strong case for rigor, transparency and long-term follow-up as these therapies reach more patients.”
Title: Evaluating cases of possible insertional mutagenesis after AAV gene therapy
Authors: Lindsey A George, Rebecca C Ahrens-Nicklas, Frederic D Bushman
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