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A Patient’s Rare Cancer Mission

March 4, 2016
Like most people, Paul Poth had never heard the word cholangiocarcinoma. As a healthy 38 year-old new father with no predisposing factors and no family history, it was a complete shock to be diagnosed with this rare cancer. Paul was confident, however, that by being in Boston at a top cancer center, working with leading doctors who were at the forefront of both treatment and research, something could be done to help him.

But the shock only deepened as Paul found out that because his cancer was rare - 5,000 cases per year in the US - no treatments existed, and little research was being carried out. He was offered a standard of care chemotherapy regimen that measured success in extending life by a few months. Second line treatments were no more promising, and attempts at clinical trials were not successful. Paul died just before his 40th birthday, less than two years after being diagnosed.

Unfortunately Paul’s story is not unusual - in fact it is alarmingly common among those fighting many rare cancers. Unlike more common cancers, rare cancers suffer from significant research limitations that prevent effective treatments from being identified. Paul recognized this clear research need and even as his cancer progressed, he started TargetCancer Foundation in an effort to catalyze innovative rare cancer research.TargetCancer

Quickly, a major question emerged: In a world of multi-year, million dollar grants, could a small, individual-donor driven organization make a difference? As TargetCancer Foundation has grown in the years following Paul’s death, the answer has been a clear “yes.”

We learned early on of the “catch-22” that exists in rare cancer research. Scientists need to build basic research tools and publish discoveries in order to attract the attention of major funding sources. But those sources often will only fund research that already has these tools in place, so there is a funding gap that small grants can address.

When we started funding cholangiocarcinoma research at the Bardeesy Lab at the Massachusetts General Hospital Cancer Center, it became clear that there was a need for cholangiocarcinoma cell lines. While a more common cancer is likely to have hundreds of cell lines publically available, rare cancers like cholangiocarcinoma have hardly any - a major limiting factor.

With relatively small grants, we created a program that united key stakeholders -oncologists, pathologists, surgeons, and researchers - so that tissue at the time of resection would be rushed to the lab to begin the process of being generated into a cell line, rather than being discarded. It is a relatively simple and inexpensive step that has had a major impact, and led to a comprehensive cell line and patient derived xenograft collection that has become a critical element of that research program.

In esophageal cancer, we took a slightly different approach by addressing unmet research needs through a specific collaborative project. We had previously funded Adam Bass’ genomic studies of esophageal cancer, and were fortunate enough to meet Michael Goldberg, who focused in the growing field of immunotherapy. Despite the fact that these two experts were both conducting research at the Dana-Farber Cancer Institute (DFCI), they had only met in passing.

By bringing them together informally, over a cup of coffee at the DFCI cafeteria, an unconventional collaboration was born almost immediately. These two scientists, who would not have otherwise worked together, realized the possibilities of correlating genomic features of esophageal cancer with immunotherapeutic targets. By leveraging their own data and the available resources at DFCI, Dr. Bass and Dr. Goldberg have been able to accelerate the identification of treatment targets in esophageal cancer.

By identifying key research gaps and bringing scientists together, we’re seeing rapid and impactful progress from our grants. Even as we grow, our strategy of funding key innovative seed grants remains the same. Until all of those fighting cancer - rare or common - can learn that there is an effective treatment available for them, we remain committed to the mission put in place by our founder close to seven years ago.

Jim Palma has served as Executive Director of TargetCancer Foundation since September 2010, overseeing its growth from a small start-up to a nationally recognized rare cancer research foundation. In 2014, Jim was named a Patient Advocate Fellow for the 2014 Drug Information Association Annual Meeting in San Diego. Prior to joining TargetCancer Foundation, Jim spent eleven years at Fidelity Investments in Boston, MA.

 Editor’s Note: Throughout the week of Rare Disease Day 2016, BIO will be profiling stories of those affected by rare diseases, shedding light on these diseases and the families impacted by them.  America’s Biopharmaceutical companies are working on treatments of these and many other diseases.  For more information visit