Industry Science Updates

Promising New Cholangiocarcinoma Treatment Findings

An exciting paper was recently published in Cancer Discovery highlighting the promise of the recently FDA approved FGFR inhibitor drugs for an even broader population of cholangiocarcinoma patients with FGFR2 fusions. The Evan Schumacher Fund for Rare Cancer Research at TargetCancer Foundation directly supported Dr. James Cleary’s work at Dana-Farber Cancer Institute leading to these important developments. His work was also informed by TCF’s longstanding commitment to cell line creation for cholangiocarcinoma, beginning over a decade ago in Dr. Nabeel Bardeesy’s lab at Massachusetts General Hospital.

Continue reading

2018 NORD Summit: An Advocate’s Perspective

By Leslie Condon, TargetCancer Foundation Advocacy Council Chair

The National Organization for Rare Disorders was formed after a small coalition of rare disease patients came together in the late 1970’s and became a driving force that helped establish the 1983 Orphan Drug Act. Since then, the number of NORD patient organization members has grown to 280, including twenty-one rare cancer organizations in NORD’s Rare Cancer Coalition. Within NORD’s thirty-five year history, the organization established the first patient assistant program, and helped advocate and legislate for new laws, including the Rare Diseases Act in 2002 and the FDA Safety and Innovation Act in 2006. Continue reading

Considering Whether Criteria for Clinical Trials Exclude Too Many Patients

Clinical trials are a critical part of the development of new treatments. They rely on people who volunteer to participate and are then closely monitored by medical professionals in order to determine whether an investigational medicine is safe and effective for patients to use. When there is no treatment approved by the Food and Drug Administration (FDA) for a specific disease or when no approved treatments are successful for an individual patient, a clinical trial may offer the best chance of survival. This is especially true in rare cancers, which often have no FDA-approved treatment. Continue reading

Keytruda – what a 40% response rate has meant to me.

By Bill Drake, TargetCancer Foundation Advocacy Council Member

On September 11, 2013 I was diagnosed with an adenocarcinoma malignant tumor on my esophagus at my GI junction, inside my GI specialist’s office on Cape Cod. I was there for my routine “over 50” colonoscopy. Continue reading

Washington DC Recap: ACA Repeal and Its Impact on Cancer Patients

By TargetCancer Foundation Advocacy Council Member DJ Webster

Last Thursday, TargetCancer Foundation traveled to Washington D.C. for the annual in-person meeting of the Deadliest Cancers Coalition, a collaboration of national non-profit organizations that TargetCancer is a member of. The DCC is focused on addressing policy issues related to our nation’s deadliest (or recalcitrant) cancers, defined as those that have five-year survival rates below 50 percent, which includes both esophageal cancer and cholangiocarcinoma. Continue reading

United against rare diseases.

Did you know that in the US, a disease is considered rare if it affects under 200,000 people? “Rare” doesn’t mean much, however,  if you or someone you love is diagnosed a rare disease- you just want to know what to do, which doctor to see, and how to find the best treatment.

Continue reading