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Research Road Map
The Caring for Carcinoid Foundation (CFCF) follows a 3-step Research Road Map to unlock the genetic causes of carcinoid and develop novel, targeted therapies. This Research Road Map was developed with the leadership and guidance of our esteemed Board of Scientific Advisors.
CFCF simultaneously supports all three steps of its Research Road Map in order to achieve its mission of a cure as quickly as possible. Scientific breakthroughs in any step will inform scientific research in the others. All CFCF-funded scientists are required to share their breakthrough research progress with all scientists - both via publication and at CFCF's annual symposium. In the best interest of patients, CFCF requires collaboration.
Our Research Road Map is based on the new genetic research paradigm.
The goals of our Research Road Map are:
- To map the fastest route to discovering a cure for carcinoid
- To prioritize critical gaps along the route
- To direct 100% of all donations to closing the gaps
The Caring for Carcinoid Foundation designed this Research Road Map based on guidance from our Board of Scientific Advisors.
Members of our Board of Scientific Advisors are pioneers in genetically-based research of carcinoid and other cancers, including pancreatic endocrine cancer, lung cancer, and gastrointestinal stromal tumors. Their work has contributed to the launch of several new cancer treatments, including Gleevec.
Therefore, our Research Road Map is based on proven models of successful cancer drug discovery.
The timing is right to pursue our Research Road Map.
Until recently, large-scale, genetically based carcinoid research was not possible due to the lack of a carcinoid tumor database. Such a database provides the tissue samples that scientists examine to unlock the genetic causes of carcinoid.
Two members of our Board of Scientific Advisors— Dr. Matthew Kulke and Dr. Daniel Chung—removed this barrier to genetically based carcinoid research. They spent several years collecting the country’s largest database of carcinoid/NET tumors, including tissue samples, blood samples, urine samples, and clinical data from carcinoid/NET patients. Due to their efforts, it is now possible to pursue genetically based research of carcinoid.
The Caring for Carcinoid Foundation simultaneously supports all three steps of our Research Road Map.
We aim to discover a cure for carcinoid as quickly as possible. It would be too slow to complete steps 1, 2 and 3 in sequence. Instead, we simultaneously support leading scientists who undertake cutting-edge, genetically-based carcinoid research in all three steps.
Our simultaneous approach is possible because we require research collaboration. All scientists funded by the Caring for Carcinoid Foundation must share their research findings and insights. Such collaboration leads to rapid dissemination of critical information and constant reshaping of carcinoid research to reflect the latest discoveries.
The work to complete our Research Road Map is highly challenging.
Dr. Brian Druker is one of the leading scientists who helped discover Gleevec. In The Oncologist, Dr. Druker described the extraordinary challenge of developing new targeted therapies for cancer:
"Developing a successful agent that targets a causal, molecular event in cancer is not easy. It requires knowledge of the molecular genetic events in a tumor, drug design, and testing.
All of this requires years of work from dedicated and talented scientists. The success of Gleevec will not have made it easier, but it has proven that the concept of targeting specific molecular genetic events in cancer can result in remarkably effective therapies."
Therefore, success in curing carcinoid will require generous financial support for dedicated and talented scientists. The Caring for Carcinoid Foundation is committed to raising these necessary funds.
Research Road Map
Step 1: Genetic
- Goal: To determine the genetic causes of carcinoid
- Research: Genomic and proteomic
All cancer is caused by genetic mutation, as described in the genetic research paradigm. This means that genes that normally control the orderly replication of cells become mutated and allow damaged cells to reproduce without restraint. Therefore, Step 1 in our Research Road Map is to unlock the genetic causes of carcinoid.
Recent scientific breakthroughs changed the landscape of cancer research and made possible this genetically-based approach to carcinoid research. First, the Human Genome Project (HGP) was completed in 2003, two years ahead of schedule. Begun in 1990, the HGP was a thirteen-year effort coordinated by the National Institutes of Health and Department of Energy. The HGP’s goals were to identify all the approximately 20,000-25,000 genes in human DNA, to determine the sequences of the 3 billion chemical base pairs that make up human DNA, to store this information in databases, to improve the tools for data analysis, and to transfer related technologies to the private sector. The success of the HGP marked a watershed in research because it enabled scientists to study cancer and many other diseases at the genetic level.
The second scientific breakthrough was the discovery of Gleevec. Gleevec represents a landmark event because it is a novel, targeted therapy that is highly effective in treating cancer patients with chronic myeloid leukemia and gastrointestinal stromal tumors. Moreover, Gleevec has no significant side effects.
"Gleevec proves a principle. It justifies an approach. It demonstrates that highly specific, non-toxic therapy is possible."
The Caring for Carcinoid Foundation aims to repeat the success of Gleevec for carcinoid.
Step 2: Molecular Biology
- Goal: To define how carcinoid spreads in the body
- Research: Cellular pathways
It is insufficient to understand the genetic mutations that cause carcinoid. Scientists must also understand how these genetic mutations affect cell development over the long term. Therefore, Step 2 in our Research Road Map is to map the pathways of carcinoid cell growth and proliferation.
This work focuses on understanding the interactions between various systems of a cell, including the interrelationships between DNA, RNA, and protein synthesis, and determining how these interrelationships are regulated. This molecular biology research requires contrasting the development of normal and mutated carcinoid cells to identify critical differences in replication, transcription, and translation of genetic material.
Step 3: Clinical
- Goal: To develop genetically targeted therapies to cure carcinoid
- Research: Drug screening, development, and approval
Today, therapies available to carcinoid patients only provide symptomatic relief. They are ineffective at shrinking and eliminating carcinoid tumors, particularly following metastasis. Therefore, Step 3 in our Research Road Map is to develop novel, targeted therapies for carcinoid similar to Gleevec.
This work involves screening, developing, and approving new therapies that target the genetic mutations that cause carcinoid. All new therapies will undergo clinical trials and be reviewed by the Food and Drug Administration, and the new therapies that prove safe and effective will be made available to carcinoid patients.
We believe this vision of launching new, genetically targeted therapies is bold and offers a profoundly exciting hope for carcinoid patients, families, and friends.