With the death of Steve Jobs, the world mourns the passing of a technological visionary who changed the world and challenged us to “Think Different”. It seems ironic therefore, that he (along with 35,000 other Americans per year) should succumb to a disease that has continued to get short shrift when it comes to research dollars, and the attention of scientists. A disease whose treatment options have not changed in 40 years.
Over the past 10 years the genomics revolution fundamentally changed the way in which drugs are discovered, by allowing scientists to target a specific protein which contributes to a disease. We’ve seen acute forms of cancer be transformed into manageable chronic diseases. Moreover, pharmaceutical companies are shifting their portfolios to include biologics such as monocolonal antibodies, and therapeutic peptides. At some point in the future, those portfolios may also include siRNAs, and gene therapy, and engineered bacteria & viruses, but these are still a ways off.
Even more significant is the recent shift from the search for the next big cancer monotherapy, to a pathway-focused, “cocktail” approach. Pharmaceutical companies and institutions like TGen are actively engaged in looking for ways to combine existing drugs to attack multiple pathways simultaneously. An approach that originally was used to turn AIDS from a death sentence to a treatable chronic illness.
Even with this slow thaw in the mindset of pharmaceutical companies, the standard of care for pancreatic cancer patients has not changed in 40 years. Gemcitabine remains the drug of choice for prolonging the lives of pancreatic cancer patients, despite its lack of efficacy Pancreatic cancer is still a death sentence. Over the years I’ve followed a progression of comparative drug trials which have made little or no improvement in the situation. But only recently has there been up-tick in interest from the research community in pursuing multi-therapeutic “cocktail” approaches to treating the disease. If we are to make any progress with respect to Pancreatic Cancer, we will need to ‘Think Different’.
Changing The Funding Equation
Let’s start by changing how we think about funding research. Traditionally, pharmaceutical companies have been the drivers behind the development of new drugs and therapeutic approaches. They’ve also been the ones investing in basic research into disease areas. Paradoxically, over the past 5 years, pharmaceutical companies have been significantly reducing their R&D spend. They have cut therapeutic areas from their portfolios, and laid off thousands of researchers. They’ve grown their pipelines by acquisition and alliances instead of internal innovation, and cut down their investments in basic research required to understand the biomolecular basis for various diseases. The reasoning goes something like this: “Science by it’s very nature is unpredictable. Discoveries do not occur on a predictable schedule. And predictability is what makes shareholders happy.” The result of this is that most companies are now focused on managing the drug development end of the business — basically shepherding drugs through the clinical trials and approval processes. What is known in certain circles as the NRDO approach — No Research, Development Only.
But, as Sun Tzu observed, you can’t defeat an enemy that you don’t understand. And real progress in defeating pancreatic cancer won’t come without a deeper understanding of the biology of this class of diseases. This is where organizations dedicated to pancreatic cancer research can make the biggest difference by identifying those researchers who are making the greatest contributions, and creating new collaborations and funding opportunities.
One great example of this, is the work being done at Johns Hopkins University at the Sol Goldman Pancreatic Cancer Center by researchers like Ralph Hruban, Christine Iacobuzio-Donohue, Sian Jones and many others. Ralph Hruban is most widely known for his work in mapping out the early stages of the pancreatic cancer and created the Pan-IN model that describes the genetics changes that precede pancreatic cancer. Christine Iacobuzio-Donahue discovered the gene expression patterns in pancreatic cancer, and changed our understanding of the long-term course of the disease. Sian Jones discovered the core signalling pathways that drive the disease.
In addition to the Goldman Center, the Lustgarten Foundation, and the Pancreatic Cancer Action Network (PANCAN) have been instrumental in focusing research dollars and attention on this terrible disease.
How can you help? Take your iTunes budget for this month, and use it to honor the man who invented iTunes. Use it to honor the thousands of other pancreatic cancer victims. Use it to make a difference.
Lastly, Congress has put forth a bill (HR Bill 733) to change the funding priorities at the NIH, and thus route more funding dollars towards basic research in pancreatic cancer. You can help, by following the link above, and contacting your representative to voice your support for the bill.
Progress won’t be measured by the number of compounds screened, or the number of papers published — only in the number of lives saved.