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
Diving into the depths. Using clearing techniques we can now investigate the innards of a tumour spheroid. Here BT-20 breast cancer cells were cultured in 3D and stained for their nuclei using DAPI and for cell-cell junctions using a cadherin antibody. In 2D these cells hardly form cadherin junctions but in 3D these are highly enriched and form on all sides of each cell showing the importance of spatial cues for the proper formation of these junctions. The crescent appearing in the middle of the image is the start of a core devoid of cells. Cutting through these spheroids one is struck by the changes in cell morphology and size. An image that visualises the importance of improved cell culture systems to properly mimic the in vivo tumour organisation for the testing of new therapies.
Iman van den Bout, Heidi Truter, University of Pretoria, South Africa.
