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I recently attended a biotechnology forum in which several Boston-area industry leaders and oncology experts gathered to discuss the current state of oncology research and cancer drug development. Xconomy, a Fool content partner and source of online news/commentary surrounding innovative businesses or the exponential economy, organized the event, and it was hosted by Millennium: The Takeda Oncology Company (OTC BB: TKPHF.PK).
The forum was a mixture of corporate presentations, roundtable discussion, and ended with a keynote chat with Tyler Jacks, the director of the David H. Koch Institute for Integrative Cancer Research and moderated by former CNBC reporter Mike Huckman.
The forum was termed "Boston's War on Cancer" and examined the current state of what has been a long and ongoing struggle. The first shot in the war on cancer was fired with the passage of the National Cancer Act of 1971, which directed funding to the understanding of underlying biology of cancer and the search for effective therapies.
The opening presentation was provided by Millennium CEO Dr. Deborah Dunsire, who was recently named one of the top 10 women in biotechnology by Fierce Biotech. She noted that the oncology market is growing alongside U.S. and world demographics, with the $70 billion market of 2009 expected to become a $94 billion market as early as 2015.
And yet …
Despite massive investment and effort to date, and the presence of as many as 2,470 agents in current development (predominantly preclinical phase), there have been -- disappointingly -- few truly transforming treatments in the past few years. Only modest improvements in mortality have been achieved for many forms of cancer.
But while there have been a few bright lights, the industry is capable of impressive achievement. There's Gleevec (imatinib), a drug marketed by Novartis (NYSE: NVS ) that has revolutionized the treatment of chronic myelogenous leukemia. There's crizotinib, in development at Pfizer (NYSE: PFE ) , which may do the same for non-small cell lung carcinoma.
Cancer is a very diverse disease, with many different factors and transformations contributing to unchecked cell proliferation. The most promising therapies to date are, not surprisingly, based on where the disease genesis and corresponding therapeutic targets are best understood.
Change is needed
Dr. Dunsire noted that the current model for the development of cancer therapeutics needs to change. Advancement of therapeutics is unfortunately too frequently driven by serendipity; Takeda/Millennium's own successful drug Velcade (bortezomib), a first-in-class proteosome inhibitor, is one example. The rising cost of oncology drug development, competitive clinical environment, and the increasing unwillingness of regulatory and reimbursement-setting agencies to accept only modest incremental improvements combine to call into question the sustainability of the current development models.
Dr. Dunsire is encouraged, though, that advancement in early detection, companion diagnostics that match patients to therapies, and new targets for and approaches to cancer therapeutics seem to indicate we are on the cusp of revolutionary advancements in the treatment of cancer. As Dr. Dunsire told me an interview, "It may take years rather than months to accomplish our goal, but we will see patients receive care based on their unique genetic profile in the coming decade."
One new approach to oncology therapeutics was presented by Doug Fambrough, CEO of privately held Dicerna Pharmaceuticals. The strategy is to adapt the revolutionary technology of RNA interference to cancer therapeutics. By directing its effects to the messenger RNA rather than the protein products derived from it, RNAi offers the opportunity to attack targets that have been previously difficult to drug or are not protein-based.
It is a strategy Dr. Fambrough is quite familiar with, as he was previously instrumental with the establishment of RNAi based Sirna Therapeutics and its eventual sale to Merck (NYSE: MRK ) in early 2007. A challenge in the development of RNAi-based therapeutics and its predecessor, antisense-based technologies, has been systematic delivery of these drug types. Recent advances in this area, including those made with liposomal delivery vesicles and used by RNAi company Alnylam Pharmaceuticals (Nasdaq: ALNY ) , as well as newer conjugate strategies developed by Dicerna, provide hope that this issue has been effectively solved.
Mark Goldsmith, president and CEO of privately held Constellation Pharmaceuticals presented on an arena of promising untapped therapeutic targets involved in epigenetic changes and transformation. Epigenetics is the field of heritable factors that do not involve changes in DNA sequence, but rather incorporate the changes in how DNA is stored and expressed in different cell types.
Epigenetic factors are behind whether a cell becomes one or another tissue type, for example, and are also often involved in cells' cancerous transformations. Some recent drug approvals have come from this approach, including Merck's Zolinza (vorinostat) and Celgene's (Nasdaq: CELG ) Vidaza (azacytidine). Constellation hopes to turn the large set of epigenetic transforming agents into the rich mine of therapeutic targets that protein kinases has become, a very ambitious goal that may well attract pharmaceutical partnerships.
In vitro diagnostics
The roundtable segment of the forum (which I'll be covering in greater detail tomorrow on Fool.com) included a discussion of diagnostics partnered with therapies. It is this field that David Okrongly, President and CEO of Quanterix (private) is looking to innovate. He noted that in-vitro diagnostics is a $30 billion current market, and while commanding only about 2% of health-care dollars, factors in as much as 70% of health-care decisions. Quanterix's mission is to provide better sensitivity to the detection of protein targets in body fluids (such as blood), approaching single molecule detection levels.
Dr. Okrongly pointed out that improvements in sensitivity in other diagnostic areas such as imaging and genetic analysis have revolutionized their use and greatly broadened applications. Enabling a similar improvement in protein measurement should similarly transform the field and enable the discovery of new detection tools and earlier assignable therapeutic decisions.
Come back to Fool.com tomorrow for more coverage of Xconomy's "War on Cancer" forum.
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