Where We Are in the War on Cancer

Xconomy, a Fool content partner and source of online news and commentary on innovative businesses, recently organized a forum with industry and research leaders on the subject of oncology. Termed Boston's War on Cancer, the event was hosted by Millennium: The Takeda Oncology Company.

My first article covering this forum focused on the corporate presentations, but today I will detail the roundtable discussion and keynote chat segments.

The players
The panel discussion segment was run by Xconomy columnist Syvia Pagan Westphal. Participants included Millennium chief medical officer Dr. Nancy Simonian; Aveo Pharmaceuticals (Nasdaq: AVEO  ) CEO Tuan Ha-Ngoc; Infinity Pharmaceuticals (Nasdaq: INFI  ) CEO Adelene Perkins; and Alexis Borisy, venture capital partner with Third Rock Ventures and CEO of recently formed Foundation Medicine.

The representatives and their companies provided a diverse set of experience and approaches to the development of cancer therapeutics. Aveo Pharmaceutical's most mature therapeutic is tivozanib (AV-951), a VEGF inhibitor in Phase 3 clinical development. Drugs of this type prevent the recruitment of new blood vessels to tumors and include the successful Avastin (bevacizumab) from Genentech/Roche.

The therapeutic candidates in the pipeline at Infinity Pharmaceuticals are at an earlier stage of development. These include IPI-493, an inhibitor of heat shock protein 90, a chaperone that assists in protein folding; and IPI-926, an inhibitor of the hedgehog pathway, an embryonic development pathway that gets switched back on in some cancers. Foundation Medicine is a diagnostic company that seeks to match genomic and other biological information to the clinical development of oncology therapeutics and ultimately to the decisions regarding their most effective use.

Serendipity
The discussion began with a question on the role serendipity still plays in the development of an effective cancer therapeutic. Dr. Simonian related that looking for a fortuitous event, such as the complete response occurrence in early clinical evaluation that was part of the Velcade development story, is not a sustainable model. Dr. Perkins noted that she has observed responses in groups established as partial control arms that drove further drug development in a direction contrary to the original expectations based on the biology used in the drug design.

As Louis Pasteur once said, chance favors the prepared mind. Yet the panel has been encouraged by recent examples of success that predicatively followed drug design, and that the design biology and models are ever improving.

Ms. Westphal questioned the panel on matching the right patient to the right therapy. More cancer therapeutics have companion diagnostic tests associated with them today that identify which patients are best likely to benefit (or not) from a given therapy. A well-established example is the use of Herceptin (trastuzumab) from Genentech/Roche for metastatic breast cancer in those patients who exhibit an overactive HER2 receptor. Mr. Borisy compared the older manner of treating cancer to throwing a grenade into a room, and that we are now being equipped with newer, better weapons.

Dr. Ha-Ngoc noted that the terminology around cancer is even changing from one based on organ of origin to biological characterization, such as HER-positive. He also noted, though, that the different organ origins can still vary substantially in behavior and response to treatments. Mr. Borisy additionally pointed out that the cost of broad genomic analysis is reaching a point where it can and should be incorporated into many oncology trials.

There followed a discussion on the challenging ethics of oncology trials. This issue has gained public awareness -- a recent New York Times story detailed the experiences of two cousins with skin cancer in the same clinical trial who responded very differently. One cousin had been randomly assigned to the control arm of the trial. These types of issues have always been present in clinical cancer research.

Dr. Simonian noted that determining whether response rates result in real benefits requires real tests and controls. Dr. Ha-Ngoc noted that having effective controls does make a benefit visible sooner. The FDA does have an accelerated approval process in place for cancer and other life threatening illnesses. A recent example is abiraterone acetate, in development by Johnson & Johnson (NYSE: JNJ  ) for castration-resistant prostate cancer. It showed that trials can be and are designed to determine early whether a therapeutic has a positive enough effect to justifies switching the control arm to the drug in development. Dr. Perkins additionally noted that trial design based on the efficacy of individual drugs, while necessary, makes it difficult to fully explore drug synergies. Most cancers do eventually escape treatment, and cocktails of therapies need to be effectively evaluated.

Wrap up
The closing segment of the forum was a keynote chat with Tyler Jacks, director of the David H. Koch Institute for Integrative Cancer Research, and moderated by former CNBC reporter Mike Huckman, currently director of media strategy at MSLGROUP.

The Koch Institute is one of several designated basic research centers identified by the National Cancer Institute. Dr. Jacks noted that the institute is designed to force interaction / collaboration by researchers, and emphasized the bringing together of the often different approaches of scientists, who like to ask and answer questions, with engineers, who like to craft effective solutions. 

Dr. Jacks noted that the institute will have a basic research focus, but it will be especially interested in apparent paradoxes, the exploration of which often drives new insights. He noted that we are getting better at characterizing disease types, but we still need to work toward linking the changes that occur to create disease states. Better understanding in this area should lead to earlier and more effective treatments. Both the public and the industry want more than merely marginal improvements, and he is optimistic that we are on the cusp of seeing just such advances.

A question related to immunotherapy in the treatment of oncology offered a rare moment in which Dr. Jacks noted that he was initially quite skeptical of such approaches. But he has come around to be very optimistic due to recent successes and a better appreciation and understanding of the mechanisms cancers use to evade immune responses.

His closing remarks were related to a concern on the increasingly restricted interaction of researchers, doctors, and pharmaceutical companies. While acknowledging that doctors and researcher do need to operate fully ethically, Dr. Jacks stated that research needs big pharma, and big pharma needs research, and he worried that heavy-handed restrictions on their interaction would not serve to further advancements in the field.

Overall, the forum was an intellectually stimulating event, and I give kudos to Xconomy and Millennium for arranging such an affair. As is the purpose of these occasions, the event has me considering and reconsidering some aspects of oncology research, pharmaceutical operations, and of course, attractive investments in and surrounding these enterprises.

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Motley Fool Options has recommended a diagonal call position on Johnson & Johnson, which is a Motley Fool Income Investor recommendation. The Fool owns shares of Johnson & Johnson. Try any of our Foolish newsletter services free for 30 days.

Ralph Casale owns shares of Johnson & Johnson. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.


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