The Foundation Dedicated to Discovering a Cure for Neuroendocrine Cancer

Caring for Carcinoid Foundation

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Lauren and Nick.

Dave, Suzi, and Lauren at 2011 Focus on Neuroendocrine Tumors Conference in Philadelphia, PA

Dave, Suzi, Lauren - Philadelphia, PA

CFCF Awards Over $1,000,000 in 2012

Today, I am very pleased to share exciting news on the research front. The Caring for Carcinoid Foundation has awarded $1,050,000 in research grants to researchers at Cold Spring Harbor Research Laboratory, Dana-Farber Cancer Institute and Rockefeller University.

Last year, CFCF-funded researchers revolutionized neuroendocrine tumor research with breakthrough results. Through this latest round of funding, researchers will advance last year’s promising findings and generate models that the entire neuroendocrine tumor research community can use to develop new treatments for patients.

These projects fit into CFCF’s diversified research strategy that seeks, simultaneously, to:

  • discover novel targets for therapeutics and biomarkers,
  • advance the scientific understanding of promising targets, and
  • translate scientific findings into improved patient care.

In addition, CFCF supports foundational projects that are necessary for all phases of neuroendocrine cancer research, including the creation of model systems such as mouse models and cell lines.  These models are necessary and particularly useful for discovering new therapies and predicting their effectiveness on patients. From these models, we also learn valuable insights about how neuroendocrine tumors develop, and identify new targets for patient therapies or biomarkers.

CFCF’s newly funded projects bring tremendous momentum to CFCF’s research portfolio.  They could yield targets for new molecularly based therapies and biomarkers for patients as well as models to use to study neuroendocrine tumor development and develop new treatments.

We are so grateful to CFCF’s donors whose generous contributions will enable the Foundation to pursue all of these projects simultaneously without sacrificing support for the other components of our research portfolio, enabling us to make progress for patients as quickly as possible.

I have detailed the specific projects below for readers to learn more about these exciting new research projects.

The first project is a foundational project to create new model systems.

The next two projects are focused on advancing findings from CFCF-funded researchers who discovered mutations in the genes DAXX and ATRX among tumors from patients with non-functioning pancreatic neuroendocrine tumors. The precise role of ATRX and DAXX in neuroendocrine tumor development is yet to be understood and treatments exploiting these findings have yet to be developed.

DAXX and ATRX are epigenetic regulators, meaning they determine which genes are turned on or off under specific conditions in a cell. While genes contain the instructions for assembling proteins, it is through epigenetic regulation that cells are able to control whether those proteins are actually produced. Mutations within these epigenetic regulating genes can cause them to malfunction, leading to the inactivation of a cancer-suppressing gene, or activation of a cancer-driving gene.

Project One

Title: Mouse Model Project Using Forward Genetics

Investigator: David Tuveson, MD, PhD

Institution: Cold Spring Harbor Laboratory

Award: $300,000

The lack of model systems that accurately recapitulate the behavior of neuroendocrine cancers has long been a significant hurdle to developing targeted treatments for patients. With this funding Dr. Tuveson will complete his project to generate the first accurate mouse models of neuroendocrine cancers.

Dr. Tuveson is using a new method called “jumping genes” to generate mouse models of carcinoid cancer.   If successful Dr. Tuveson will not only create an important resource for the research community but also identify genes and pathways involved in neuroendocrine tumor development.


Project Two

 Title: Understanding the Tumor Suppressor Activities of ATRX-DAXX through Epigenomic Profiling and Animal Models

Investigator: C. David Allis, PhD

Institution: The Rockefeller University

Award: $450,000

In this project Dr. Allis and his team will conduct experiments and create models to understand the role of ATRX and DAXX in neuroendocrine tumor development with the ultimate goal of developing new therapies for patients by targeting these processes.  Furthermore they will establish the precise changes in chromosome structure resulting from mutations in ATRX and DAXX.  Knowledge of these changes could shed light on not only neuroendocrine cancers but many other cancer types as well.  Finally they will attempt to identify molecular subtypes of pancreatic neuroendocrine tumors to facilitate the development of molecular biomarkers and targeted therapies.

Project Three

Title: The Impact of MEN1, DAXX, ATRX and PTEN in Pancreatic Neuroendocrine Cancer Pathogenesis

Investigator: Kwok-Kin Wong, MD, PhD

Institution: Dana Farber Cancer Institute

Award: $300,000

In this project Dr. Wong will create mouse models to determine how certain recently identified genes impact neuroendocrine tumor development.  Next Dr. Wong will conduct experiments using his mouse models to identify the cellular pathways that are deregulated during the development of neuroendocrine tumors.  These pathways represent potentially new and novel therapeutic targets for treatment of patients with neuroendocrine tumors.

This project has tremendous potential to help rapidly advance treatment options for patients, Dr. Wong states, “In particular, we are enthusiastic regarding the possibility that we will uncover vulnerable pathways that might be druggable.  If these generated mutant mice do develop pancreatic neuroendocrine cancer, these will serve as ideal platforms for novel therapeutic testing.”


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