New leukaemia drug in clinical trials

A scientific discovery made 20 years ago at the Walter and Eliza Hall Institute has led to the development of a potential new anti-cancer agent that is now entering clinical trials to treat chronic lymphocytic leukaemia (CLL), the most common type of leukaemia.

Patients with CLL have begun to receive the agent, ABT-199 (GDC-0199/RG7601), as part of a worldwide phase 1a clinical trial coordinated locally by Cancer Trials Australia.

ABT-199 is a so-called BH3-mimetic drug, which is designed to block the function of the protein Bcl 2.

In 1988, it was discovered that Bcl-2 allowed leukaemia cells to become long-lived, a discovery made at the Walter and Eliza Hall Institute by Professors David Vaux, Suzanne Cory and Jerry Adams.

Subsequent research led by them and other institute scientists, including Professors Andreas Strasser, David Huang, Peter Colman and Keith Watson, has explained much about how Bcl-2 and related molecules function to determine if a cell lives or dies.

These discoveries have contributed to the development of a new class of drugs called BH3-mimetics that kill, and thereby rapidly remove, leukaemic cells by blocking Bcl-2.

Read more at Walter and Eliza Hall Institute