07/18/2013 03:38 pm ET Updated Sep 17, 2013

The Science of Pancreatic Cancer Advances

Pancreatic cancer is the most lethal of all cancers, yet there is reason for hope. The disease is no longer the mystery that it once was to those of us researching it at renowned institutions like the Cold Spring Harbor Laboratory, where I direct The Lustgarten Foundation Pancreatic Cancer Research Laboratory. We now understand why pancreatic cancer occurs, why it's hard to detect, and why it's hard to treat. With that understanding, we are at a turning point in fighting the disease and are well-positioned to change its outcome.

Pancreatic cancer is a silent disease because its signs and symptoms can go unnoticed until the cancer is in the advanced stage. The overall five-year survival rate for the disease is six percent, according to the American Cancer Society. More than 45,000 Americans will be diagnosed this year; more than 38,000 will die. There are no early detection tests, no effective long-term treatments and, unless the cancer is surgically removed in its earliest stages, no cure.

Despite that reality, however, there is hope because of recent research. Fifteen years ago, the pancreatic cancer research field was largely ignored - with only a handful of investigators working part-time in research peripherally relevant to the disease. Today, the field has nearly 1,000 researchers working on a focused approach to find a cure.

That research has revealed much about the disease. Researchers have clarified and confirmed why pancreatic cancer happens: critical mutations in genes, such as K-RAS, cause pancreatic cancer. We know this because, through research that The Lustgarten Foundation funded, scientists sequenced the genes in many pancreatic cancers, which resulted in the discovery of more than 60 DNA mutations.

That discovery provided a starting point for many new research studies, including the development of tests for early detection, creating new drugs that specifically target pancreatic cancer, identifying familial pancreatic cancer genes, selecting new combinations of drugs for clinical trials, and identifying which patients are most likely to respond.

Early detection is a major area of focus. We now know that pancreatic cancer develops and progresses to lethal stages slower than originally thought, potentially taking up to 10 years to fully develop. We also know that a specific combination of gene mutations can distinguish cancerous cysts from some non-cancerous cysts using small volumes of liquid obtained from patients by needle biopsy. This finding was the result of encouraging research funded by The Lustgarten Foundation and conducted by Dr. Bert Vogelstein at Johns Hopkins University.

Recent research also has revealed that pancreatic cancer may be hard to treat because getting drugs to penetrate the thick stromal blanket surrounding cancer cells is difficult. This discovery, which my team and I uncovered, proposed a new reason that patients do not respond well to chemotherapy and led to promising studies that helped drugs reach the tumor. Scientific methods to improve drug delivery are now being tested with patients in clinical trials.

Understanding these fundamental dynamics -- why pancreatic cancer occurs, why it's hard to detect, and why it's hard to treat -- has set us on a course to change the outcome for patients. The mandate now is increased research collaboration. We need to nurture a culture in which research into shared theories and hypotheses advances the field faster than individual research could.

Since its founding 15 years ago, The Lustgarten Foundation -- America's largest private foundation dedicated to funding pancreatic cancer research - has devoted more than $65 million to more than 175 research projects at over 50 medical and research centers worldwide. That funding has been pivotal in getting individual researchers to advance the field, and today, this funding continues to propel collaborative breakthroughs forward.

Much of this encouraging collaborative work is fostered through our Pancreatic Cancer Research Consortium, which is composed of six world-renowned medical institutions working together on research initiatives aimed at finding a cure for pancreatic cancer. Those institutions are Cold Spring Harbor Laboratory, Dana-Farber Cancer Institute in affiliation with Harvard Medical School, Johns Hopkins University School of Medicine, The David H. Koch Institute for Integrative Cancer Research at MIT, Memorial Sloan-Kettering Cancer Center, and The University of Texas M.D. Anderson Cancer Center. This collaboration is enhanced by the work of our new lab.

With the knowledge we now have in hand, the collaborations underway through this consortium and other initiatives, and the outstanding scientific capabilities of these world-class institutions, there is reason for great optimism in the face of pancreatic cancer's grim reality. More research is needed and more support for that research will be required. Research contributions from private foundations like The Lustgarten Foundation have been crucial, yet, with less than two percent of federal research funding directed at this lethal disease, more funding is needed.

The science is advancing, and hope rises with it. We're on the path to conquering this terrible disease, and the path is becoming clearer. With more research funding, a cure should be possible.

The author is Director of Research at The Lustgarten Foundation and Director of The Lustgarten Foundation Pancreatic Cancer Research Laboratory at Cold Spring Harbor Laboratory. For more information, visit