Matthew H. Kulke and Jennifer Chan discuss neuroendocrine tumors (NETs), which can occur in any organ that has endocrine cells.

1. Program in Neuroendocrine and Carcinoid Tumors

2. What is NET Cancer?
 Neuroendocrine tumors (NETs) can occur in any organ that has endocrine cells.
Sometimes Neuroendocrine tumors are called carcinoid tumors.
 They are found in both adults and children.
 They appear most often in the small intestine, appendix, pancreas, and lungs but can
also be found in the thyroid, breast, ovaries and prostate.
 NETs are difficult to diagnose and many patients have vague symptoms such as
abdominal pain, flushing, or diarrhea for eight or nine years before the correct
diagnosis is made.
 Tumors have often metastasized by the time they are diagnosed.
 NETs don’t respond to most chemotherapy drugs, and very few of the new targeted
drugs are effective. Surgery is the only cure and once the NET has metastasized, it is
difficult to remove the complete tumor.
Program in Neuroendocrine and Carcinoid Tumors

3. Types of neuroendocrine tumors
 Neuroendocrine tumors are usually
classified as either pancreatic endocrine
tumors which start in the pancreas or
carcinoid tumors which start in other
organs. Carcinoid tumors most
commonly start in the lungs, small
intestine, appendix, or rectum.
Program in Neuroendocrine and Carcinoid Tumors

4. Treatment approaches
 When neuroendocrine tumors are detected at an early stage, before there
has been spread to other organs, they can usually be removed surgically.
 Liver-directed therapies: Often involves embolization, an interventional
radiology procedure to cut off the blood supply to the liver metastases.
 Somatostatin analogs: Most neuroendocrine tumors have receptors for the
naturally occurring hormone somatostatin. Synthetic somatostatin analogs
are available that mimic the action of somatostatin and can be used to treat
neuroendocrine tumors.
 Chemotherapy
 Interferon: Interferon is considered a biological agent.
It is administered using a subcutaneous injection.
 Targeted therapies
Program in Neuroendocrine and Carcinoid Tumors

5. Incidence
 In excess of 100,000 people living with
NET cancer in the US
 16,000 new diagnoses each year in US
 Estimated more than 200,000 undiagnosed cases
in the US
 Time from onset of symptoms to proper diagnosis often exceeds
five years
Program in Neuroendocrine and Carcinoid Tumors

6. Government Funding
 Government funding for NET research is less than $3 million annually.
 In 2017 it is estimated that government funding for breast and
colorectal cancers is $699 million and $331 million respectively.
 Government funding for NET represents less than 1% of what is spent
on more common malignancies.
Program in Neuroendocrine and Carcinoid Tumors

7. About Dana-Farber
Unique Approach
• Dana-Farber’s 50/50 balance of research and patient care is the cornerstone of our lifesaving
mission.
• This powerful integration results in new discoveries and innovations that benefit patients faster.
Breakthrough Clinical Trials
• With one of the largest clinical trials programs in the country, Dana-Farber offers eligible patients
the opportunity to access the latest advances in experimental therapies.
Expansive Collaboration
• Dana-Farber’s team science model encourages lab scientists to work in tandem with clinicians, as
well as across the Harvard Medical School system, with MIT and the Broad Institute, as well as
preeminent research institutions around the world.
Next Generation Drug Discovery
• Dana-Farber investigators are creating new laboratory models of cancers and testing new drugs.
• Dana-Farber chemical biologists are creating new drug compounds to expand therapeutic
options for patients.
Program in Neuroendocrine and Carcinoid Tumors

8. Leadership
Matthew H. Kulke –
Director, Program in Neuroendocrine and
Carcinoid-Tumors DF/BWCC
Center for Gastrointestinal Oncology
Department Medical Oncology
Professor of Medicine, Harvard Medical School
Jennifer Chan –
Clinical Director, Program in Neuroendocrine and
Carcinoid Tumors DF/BWCC
Center for Gastrointestinal Oncology
Department Medical Oncology
Instructor of Medicine, Harvard Medical School
Program in Neuroendocrine and Carcinoid Tumors

9. Program in Neuroendocrine and Carcinoid Tumors

10. CHALLENGES: NET Research
 Neuroendocrine tumors are notoriously challenging to treat, and only ten years
ago few treatment options were available for patients.
 Sparse dedicated private funding sources and very few private foundations
dedicated to neuroendocrine tumor research
 Lack of laboratory models of NETS to test promising new treatments
 Very little public awareness about the disease
Program in Neuroendocrine and Carcinoid Tumors

11. Collect tumor
and blood
specimens
Analyze
specimens to
discover key
pathways critical
to driving tumor
growth
I. Identify Treatment Targets
II. Validate Treatment
Targets in Patient-
Derived Tumor
Models
III. Evaluate in Clinical
Trials
Take promising
treatments to
clinical trials
Use patient-derived
tumor models to
confirm that targeting
pathways impairs NET
growth
Identifying New Treatments for NET at DFCI:
A Patient-Based Approach
Bring new
treatments
to patients
Molecular and
genetic analysis
Program in Neuroendocrine and Carcinoid Tumors

12. Philanthropy is Catalytic…
For patients and families who are awaiting the next breakthrough treatment for
neuroendocrine cancer, we need a partnership between science and visionary
philanthropy to more rapidly pave the way for new therapies and shape the future of
cancer care.
I. People
 Scientists to advance the study of laboratory model systems and new targets
for therapy
II. Program
 Discover and validate new therapeutic targets and test new drugs
Program in Neuroendocrine and Carcinoid Tumors

13. Summary
 Neuroendocrine cancer is a deadly disease that has had insufficient research
attention and funding
 We now have potential to dramatically advance our understanding of these cancers
and to develop more effective rational therapies to precisely target the drivers of
tumor growth
 We have a unique combination of research and clinical efforts to attack these
cancers and are developing an integrated program to make real progress
Program in Neuroendocrine and Carcinoid Tumors