Michael Makris on Rising Malignancy Concerns After Hemophilia Gene Therapy: Key Challenges Highlighted in NEJM
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, Editor-in-Chief of RPTH Journal, posted on LinkedIn:
”There is an interesting discussion about the issue of malignancy in persons with haemophilia who have had gene therapy in the New England Journal this week.
The number of malignancies is increasing as more patients undergo gene therapy.
The key issues are:
a) Need for a standardised set of investigations
b) Need for advice on what tissue sample to collect and how to store them, to allow for the more specialised investigations
c) The interpretation of the results
d) Payment for these very expensive investigations. Manufacturers have funded these so far because most of the cases were seen in the clinical trials, but will they continue to fund it long term? ”
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