September 7, 2016
By Mark Terry, BioSpace.com Breaking News Staff
A new cancer drug developed in part by Australia’s Eliza Hall Institute of Medical Research, has received fast-track approval by the U.S. Food and Drug Administration (FDA).
The drug, Venetoclax, is a BH3-mimetic, and inhibits Bcl-2, a programmed cell death protein. Venetoclax was based on a discovery at the Eliza Hall Institute made in the late 1980s. In April, the FDA approved the drug to treat chronic lymphocytic leukemia (CLL). Although not yet approved for use in Australia, the U.S. FDA approval is a “strong indicator that the drug is on track to becoming registered and available in Australia.”
Andrew Roberts, a clinical hematologist and head of clinical translation at the institute in Melbourne, Australia, reported significant reduction of leukemia cells. “Some patients have remained in remission four years after their treatment began,” he said in a statement. “In many cases we have seen the cancerous cells simply melt away.”
The drug was co-developed by AbbVie and Genentech . In January 2016, AbbVie announced that the FDA granted the drug Breakthrough Therapy Designation in combination with rituximab to treat patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL).
“This second Breakthrough Therapy Designation for venetoclax granted by the FDA underscores the significant potential of this therapy in treating relapsed/refractory CLL patients, and reflects AbbVie’s commitment to providing breakthrough therapies for cancer patients,” said Michael Severino, AbbVie’s executive vice president of research and development and chief scientific officer, in a statement. “AbbVie will continue harnessing our collective expertise to accelerate efforts to bring new treatment options to patients battling this difficult to treat blood cancer.”
Venetoclax has shown positive results positive results in about 80 percent of clinical trials. Approximately 20 percent—one in five—patients that finished the program were completely cancer free.
John Seymour, who assisted in overseeing the trials at the Peter MacCallum Cancer Centre in Australia, said to 7.30, “Cells, when they are born, are destined to die and cancer cells and particularly leukemia cells delay that death by using a protein called BCL2 that stops the normal time of death. Venetoclax works by specifically blocking the action of that BCL2 and allows the cells to die in the way that they were destined to.”
The Phase II trial involved 107 patients at least 18 years of age who had already received at least one type of treatment. They also had to have a deletion on chromosome 17p. That particular deletion includes a gene that suppresses cancer growth.
The medication is oral. Patients took one pill daily for five weeks, with dosing starting at 20 mg and slowly escalating to 400 mg. By the end of the trial, patients and researchers at 31 sites in the U.S., Canada, the UK, Germany, Poland and Australia were involved.
Richard Pazdur, with the U.S. FDA’s Center for Drug Evaluation and Research, said in April, “These patients now have a new, targeted therapy that inhibits a protein involved in keeping tumor cells alive. For certain patients with CLL who have not had favorable outcomes with other therapies, Venclexta may provide a new option for their specific condition.”
