May 19, 2009

Translational Research – Under Construction! Louise M. Perkins, Ph.D., Chief Scientific Officer

How should the next generation of medicines be discovered and advanced to clinical testing?  Who should pay for and conduct this work?  These were among the key topics at the Translational Medicine Alliance Forum that I attended in Philadelphia on May 14 and 15.  This forum brought together thought leaders from foundations, academia, government and the private sector to identify barriers, possible solutions and the role that each of these key stakeholders should play in this important field.

So, what is translational research and why is it so important?  It is the process of advancing new medical approaches from the research laboratory to patients– that is, translating research “from bench to bedside”.   The MMRF has long supported this type of research, and the MMRF Scientific Agenda charts strategies aimed at translating an understanding of the genomics and proteomics of myeloma to targets and then to treatments and personalized medicine.  This may sound easy on paper - but actually making it happen in an efficient way is the real challenge. 

What was clear from this meeting is that disease-focused foundations like the MMRF are viewed as critical players in this complex, expensive and time-consuming process.  I was privileged to be invited as a panelist discussing the role of non-profit foundations in translational research along with other venture philanthropy leaders, like Bob Beall - MMRF Board Member and head of the Cystic Fibrosis Foundation (http://www.translationalmedicinealliance.org/forum/agenda.cfm).

While the specific approaches of the different groups vary, the common thread is that leading non-profit foundations like the MMRF fund commercial as well as academic research to overcome the barriers related to bringing new treatments forward.  The MMRF and its sister organization the MMRC have already made tremendous advances, such as opening clinical trials 30%-40% more quickly than the industry standard.  But, despite these improvements, there are still significant needs.  For example, there is a growing urgency to devise and fund new solutions that incentivize clinical development of drugs in MM since financial constraints facing the entire pharma and biotech industry threaten continued innovation and progress. 

Commercial investors who provide funding for the development of innovative products were also at the meeting.  These venture capitalists uniformly acknowledged that programs like the MMRF Biotech Investment Award that support promising biotech drugs are not just a source of cash.  Importantly, they also represent a “seal of approval” that can trigger the influx of venture capital to those companies.  Why is this important?  Simply put, it means that MMRF donor dollars are complemented with additional money that might not otherwise come to the field of myeloma thereby extending our reach and enhancing the chance of success for those drugs.

All in all, these were an exciting two days at the Translational Medicine Alliance Forum and it was rewarding to see so many scientists and business people working diligently to develop increasingly optimized models to translate research findings to real-world therapies for all patients, including those with multiple myeloma.