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星期四, 4月 28, 2016

世界醫藥創新論壇第三日諾華執行長談醫藥未來有四個重點

Highlights of the Day
Day Three of the World Medical Innovation Forum was short, but was filled with some of the most exciting aspects of the meeting. The first session touched on the needs of cancer patients who require alternative strategies to achieve optimal therapeutic benefit, and the advantages and challenges associated with combination therapy.

The opening session was followed by a fireside chat with Joseph Jimenez, CEO of Novartis, who provided insight into Novartis' successes and discussed current topics including CRISPR, CAR-T, a second generation of immuno-oncology, and the biggest threats to the future of innovation.

The morning continued with a session in which panelists discussed new cancer diagnostics driven by advances in genetics, genomics and proteomics, and the integration of these technologies into the delivery of care. This panel was followed by a discussion around the evolving role of disease-focused philanthropic foundations in patient care. For more in-depth highlights from Day Three, see below.

The capstone event of the Forum was the announcement of the Disruptive Dozen, the 12 technologies predicted to have the greatest impact on cancer care in the next decade. The main stage was filled with 12 influential leaders in cancer research and clinical care from Massachusetts General Hospital, Brigham and Women's Hospital and Dana-Farber Cancer Institute, who came together for an exciting and insightful discussion around these state-of-the-art medical technologies and what they mean for the future of the field.

The Forum closed with notes from Anne Klibanski, MD, Chief Academic Officer of Partners HealthCare, who noted that one of the greatest accomplishments of the Forum was the facilitation of a new community; a community that can shift paradigms and accelerate cures by having open dialogue, understanding priorities, and incorporating patient needs, expectations, and quality of life issues, and posed a challenge to the audience to maintain this dialogue even after conclusion of the meeting.

Next year, the Forum will focus on cardiovascular disease and will be held from May 1-3, 2017 at the Westin Copley in Boston, Massachusetts.




New Philanthropy: Patients Driving Innovation
As part of continued discussion around patient-driven innovation, the New Philanthropy session provided insight into the efforts of philanthropic leaders and how their community is redefining its role to develop new ways to help patients and drive breakthroughs. As disease foundations become more involved in all aspects of patient care, including the lab, clinic, engagement, and regulatory approval process, their role has evolved.

As gaps in funding for basic and translational research grow, these organizations have provided extensive financial support to the cancer research community, which has led to the development of breakthrough therapies and promising technologies. As Louis DeGennaro, PhD, President and CEO of the Leukemia and Lymphoma Society, pointed out their foundation funded the basic research that eventually led to the development of Gleevec and supported early efforts in the development of CAR-T technology at the University of Pennsylvania. Additionally, Judy Salerno, MD, President and CEO of Susan G. Komen for the Cure, emphasized the importance of funding young investigators and remarked that, as funding has dried up, we are starving a pipeline of next-generation investigators who are needed to advance progress.

To provide this support, fundraising is essential, and the panel discussed the role of athletic events and social media in driving their efforts. As Dr. DeGennaro observed, cancer patients "want to win," and athletic events such as walks, challenges, and races are a practical manifestation of that desire and are successful because "people give to people." Supporting that notion, Billy Starr, Founder and Executive Director of the Pan-Mass Challenge, added that today's technology allows people to reach out to their world and create bonds and opportunities that are distinct from the impact of direct mail or celebrity spokespeople. When asked about the influence of the ALS Ice Bucket Challenge and whether they would consider that type of engagement for their audiences, Judy Salerno, MD, President and CEO of Susan G. Komen for the Cure, noted that their focus tends to be more directed toward the recruitment and development of people who are passionate about their mission and can become advocates.

To push for faster cures, panelists noted that their organizations are facilitating ongoing dialogue between patients and the FDA to address issues, concerns, and preferences, and training advocates in science to better disseminate their message. Additionally, as money raised from the Pan-Mass Challenge is donated exclusively to treatment and research at the Dana-Farber Cancer Institute, Mr. Starr also noted the patient role in the design of the Yawkey Center for Cancer Care, which was intended to advance clinical care and enhance the patient experience.


Disruptive Dozen
After rigorous consideration by cancer faculty from the Brigham and Women's Hospital, Massachusetts General Hospital, and Dana-Farber Cancer Institute, the 12 technologies that are predicted to have the greatest impact on cancer care in the next decade were announced. After a short audio clip describing their disruptive technology, panelists representing each of the 12 areas enlightened the audience as to why their respective fields would have an impact on the future of cancer care in a series of sound bites.

Following a major theme of the Forum, and perhaps unsurprisingly, cellular immunotherapy was announced as the most disruptive technology for the next decade. Marcela Maus, MD, PhD, Director of Cellular Immunotherapy at MGH, highlighted the promise of this technology by stating that it has the potential to become a frontline cancer therapy and noting the unprecedented results seen in some patient populations thus far. However, she also noted that there are obstacles to overcome in the development of this technology for the treatment of solid tumors, which can be addressed by identifying new targets, using different engineering strategies to distinguish tumor from self, and targeting the tumor microenvironment.

The second runner-up for most disruptive technology was liquid biopsy for oncology, and Shyamala Maheswaran, PhD, Associate in Molecular Biology, Surgery at MGH, praised the benefits of more frequent, less invasive sampling methods as compared to tumor biopsy and described the applications of this technology in early detection of cancer, monitoring of therapy, evaluation of mutational status to direct therapeutics, and assessment of heterogeneity between tumor cells for a deeper understanding of tumor evolution.

Coming in at number seven, genome editing was represented by Keith Joung, MD, PhD, Associate Chief of Pathology for Research at MGH. When posed the question, "how crisp are CRISPRs?" by Forum Co-Chair Daniel Haber, MD, PhD, Director of the MGH Cancer Center, Dr. Joung explained that, while limiting off-target effects at this time is a challenge, we have a distinct opportunity to develop treatments for diseases in which there are no therapies currently available and should proceed with the understanding that the technology will evolve and improve over time.



Fireside Chat: Joseph Jimenez, CEO, Novartis
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Rounding out the fireside chat series, Joseph Jimenez, CEO of Novartis, sat down with Gregg Meyer, MD, Chief Clinical Officer at Partners Healthcare System to discuss:

• Changes that need to take place in the "healthcare system as a whole to a platform where all patients have access to therapies"

• The company's "IO strategy is to be strong with second generation approaches that target the tumor microenvironment" and the "shift away from a transactional approach to outcomes-based approach"

• The four pillars: clinical value, patient value, health system value and societal value

• The importance of intellectual property for driving innovation - "there are no generics without innovation"

• The relatively low cost and continuing benefits, often extending over decades, of a drug once it comes off patent should be included in discussions of drug pricing


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