Who Invented Venetoclax? The 25-Year Story Behind a Cancer Drug

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From Melbourne Lab to Global Cancer Drug: The Story Behind Venetoclax

On the morning of July 28, 2017, I walked into work expecting an ordinary Friday. Instead, television cameras filled the entrance to the main auditorium, and the seventh-floor tea room buzzed with anticipation. By the end of the morning, we learned that decades of research at the Walter and Eliza Hall Institute had culminated in an announcement worth up to US$325 million—one that demonstrated how fundamental science could ultimately change the lives of cancer patients around the world. This is the story behind that moment.

A Bench-to-Bedside Story: A 25-Year Journey

Venetoclax's origin story traces back to Melbourne, Australia, and the Walter and Eliza Hall Institute of Medical Research (WEHI). In the late 1980s, WEHI researchers, led by Professor Jerry Adams, Professor Suzanne Cory, and Professor Andreas Strasser made the foundational discovery that the BCL-2 protein helps cancer cells survive indefinitely by blocking their own apoptosis — the insight that, decades later, became the entire rationale for venetoclax. That basic-science finding was developed over roughly three decades into a clinical drug through a research collaboration between WEHI and the pharmaceutical companies Genentech (part of the Roche Group) and AbbVie, with early clinical trials led by Australian haematologists at the Royal Melbourne Hospital and Peter MacCallum Cancer Centre.


Here's how that journey unfolded

1988 - A discovery of a gene called BCL-2 that holds the key to cancer cell death

2000 - Structural biology opens a new window into protein structure, sparking breakthroughs in cell death research

2006 - BH3-mimetics as anti-cancer drugs are shown to kill cancer cells

2011 - Clinical trials of an anti-cancer drug for leukemia begin in Australia

2013 - Clinical trial results bring high hopes for advanced leukemia

The US$325 Million Royalty Payment

Because WEHI's foundational research underpins the drug's patents, the institute is entitled to royalties on venetoclax sales. In 2017, WEHI struck a deal with the Canada Pension Plan Investment Board (CPPIB) to sell a portion of its future royalty rights for up to US$325 million — a US$250 million upfront cash payment plus up to US$75 million in potential milestones — while retaining a partial royalty stream for itself. WEHI has said it plans to reinvest that income into its research endowment and future drug-discovery work, calling venetoclax proof that an Australian medical research institute can shepherd a discovery all the way from the lab bench to a globally used cancer therapy. Individual WEHI scientists who contributed to the discovery also share in a portion of the ongoing royalty payments.


From Lab Bench to Global Impact

It's a striking example of how a single basic-science observation — made with no drug in mind — can end up reshaping treatment for tens of thousands of leukemia patients decades later, and generate a funding pipeline that supports the next generation of discoveries.