Gene Therapy and Cancer

In Siddhartha Mukherjee's seminal biography of cancer "The Emperor of Maladies" he debates the historical approach to addressing how to cure cancer1. Early in the book Mr Vaneveer Bush (in 1941 the director of the Office of Scientific and Research and Development) is quoted as pivoting from the wartime approach of targeting scientific endeavours towards a cancer cure and returns to a basic science strategy. "General knowledge and an understanding of nature and its laws...provides the means of answering a large number of important practical problems, though it may not give a complete specific answer to any one of them. Basic knowledge leads to new knowledge. It provides scientific capital. It creates the fund from which the pratical applications of knowledge must be drawn, [it] is the peacemaker of technological progress."


Nearly 60 years later, I couldn't agree with him more. One of the most exciting aspects emerging from the field of gene therapy is that it demonstrates how it can be applied to targeting cancer cells. As cancer is rarely a monogenic disease, this demonstrates the extraordinary reach that gene therapy applications have beyond their original use in addressing those orphan diseases many of us have worked on during our careers. The papers in this collection use standard AAV based delivery approaches, apply AI algorithms, and argue the pros and cons of genome engineering towards CAR-T treatments. This collection is a validation that basic science can be used to build on a greater foundation of scientific capital which can be applied to many aspects of human health.


Janine Scholefield, Editor-in-Chief


1Mukherjee, Siddhartha. The Emperor Of All Maladies: A Biography Of Cancer. New York: Schribner, 2010