- venture [ven(t)shɘr]
- verb - to proceed despite the risk of danger
- philanthropy [fi-lan-thruh-pee]
- the desire to promote the welfare of others
Where will the future leaders in biotechnology come from? Some of them may be studying at Yale University right now! Last week I was privileged to be invited as a guest presenter at the 11th Yale Healthcare & Life Sciences Club “Business of Biotechnology” course, a week-long seminar series at held at the Yale School of Medicine. The program, led by Constance McKee, Yale MBA and CEO of Manzantia Pharmaceuticals, is designed to teach graduate students in science and the school of management how to build a business in biotechnology. I was there to describe our Therapy Acceleration Program™ (TAP) and to discuss venture philanthropy and the role of charities in supporting drug discovery and development together with the biotechnology industry. Sohini Chowdhury, VP, Michael J. Fox Foundation also addressed this topic.
Other presenters in the course include venture capitalists and biotechnology entrepreneurs.
In my presentation, I described LLS’s partnership with the biotechnology company Celator Pharmaceuticals as a case study demonstrating the process we use to identify, qualify and fund a drug development program together with a biotechnology company. About four years ago, the LLS Research Team reviewed a proposal from Celator that described laboratory and Phase 1 clinical trial data on a new therapy for the treatment of acute myeloid leukemia (AML). The data suggested that the two chemotherapy drugs used as the standard treatment for AML, cytarabine and daunorubicin, could be made more effective and safer for patients by packaging them inside a droplet of fat called a liposome; a new therapy called CPX-351. I asked the students to consider the data and we discussed the critical scientific, medical and business considerations required to make a decision about such an investment. Most were in favor of investing in the project.
Then I presented what LLS actually decided to do: After critical review of the Celator data, the LLS team and our TAP Committee decided to support a Phase 2 clinical trial to advance CPX-351. The decision was a good one. The Phase 2 trial showed that twice as many AML patients went into remission when they received CPX-351, compared with standard treatment. And 3 times as many patients given CPX-351 were still alive one year after treatment. (These results were presented at the annual American Society of Hematology meeting last December.)
Based on these very promising results, together with Celator, we are now conducting a Phase III clinical trial with CPX-351 for patients with acute myelogenous leukemia (AML). The goal of the trial is to generate the data required to seek FDA clearance of this new therapy which would represent the most significant advance in AML treatment in nearly 30 years.
Together, charities focused on biomedical research like LLS provide billions of dollars in support to research each year ($4.7 billion in 2007). An increasing amount of this funding is deployed to support clinical stage research by biotechnology companies with treatments that are close to impact for patients. Courses like the one at Yale are educating the next generation of scientists and business people who will bring cutting edge therapies, and possibly even cures, to cancer patients. Being at the University and in front of students took me back to my days as a professor – it was really a treat and their excitement and enthusiasm heartening.