First-in-human Phase 1 CRISPR Gene Editing Cancer Trials: Are We Ready?

Francoise Baylis, Marcus McLeod
  • Current Gene Therapy, January 2018, Bentham Science Publishers
  • DOI: 10.2174/1566523217666171121165935

Human Gene editing

What is it about?

Scientific validity is an important ethical requirement for first-in-human trials. In this article, we conclude that this standard has not been met by the proposed Phase 1 CRISPR gene editing trial in the United States for patients with melanoma, synovial sarcoma, and multiple myeloma.

Why is it important?

It is critically important when moving from preclinical studies to clinical studies to meet minimum standards of scientific validity and to hold relevant parameters constant. One preclinical study in mice (which is the basis of the application for the first-in-human Phase 1 CRISPR gene editing cancer trial in the United States) does not satisfy the ethical requirement of scientific validity. Moreover, the translational distance between the pre-clinical study and the proposed clinical trial is unnecessarily wide – the quality of preclinical evidence is seriously deficient.


Francoise Baylis
Dalhousie University

To avoid (some) of the problems with gene transfer research, we need to exercise more caution with gene editing research.

Read Publication

The following have contributed to this page: Francoise Baylis

In partnership with: