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.
Professor Andrew Roberts, head of clinical translation at the Walter and Eliza Hall Institute, said the phase 1a clinical trial is designed to demonstrate the safest dose of ABT-199. “It will be tested in a small group of patients with CLL who have not responded satisfactorily to other treatments,” Professor Roberts said. “If the safety profile of ABT-199 supports further study, we anticipate that, ABT-199 could be available for a larger phase II clinical trial to determine whether it is effective for treating CLL in the next one to two years.”
ABT-199 has been co-developed for clinical use by two companies, Abbott (NYSE: ABT) and Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and was discovered in a joint research collaboration that also involved scientists at the Walter and Eliza Hall Institute. Professor Huang, who co-led the collaboration, said ABT-199 is designed to selectively inhibit Bcl-2.
Institute director Professor Doug Hilton said this collaborative arrangement enhanced the institute’s ability to rapidly translate its laboratory discoveries into benefits for patients. “The institute has supported research teams that are dedicated to improving outcomes for patients,” he said. “In this case we are seeing more than two decades of research culminating in what we hope will be a promising new drug.“
The head of business development at the Walter and Eliza Hall Institute, Dr Julian Clark described the collaboration with Abbott and Genentech as unique. “The combination of large biotech and pharmaceutical companies with an academic research institute is regarded as the industry benchmark,” he said. “We anticipate that other compounds discovered at the institute will be able to enter clinical trials through similar collaborative efforts.”
Research into Bcl-2 and BH3-mimetics at the Walter and Eliza Hall Institute has also been funded by the National Health and Medical Research Council, the Leukemia and Lymphoma Society, the Australian Cancer Research Foundation, the Leukaemia Foundation of Australia, Cancer Council Victoria and the Victorian Government.
Victorian Minister for Innovation, Services and Small Business Louise Asher congratulated the Walter and Eliza Hall Institute on this development. “This latest announcement demonstrates the capabilities of our local medical research sector, and hopefully bodes well for positive future outcomes in the fight against leukaemia,” Ms Asher said.
For further information
Strategic Communications and Marketing Manager
Ph: +61 3 9345 2345
Mob: 0417 125 700