Receiving a diagnosis of a Neuroendocrine Tumor (NET) can be overwhelming, especially because these tumors can develop in many different organs, from the gut and lungs to the pancreas. NETs are unique in that they grow from cells that release hormones, meaning they can cause symptoms both from the physical presence of the tumor and from the excess hormones they produce. These hormonal symptoms might include severe diarrhea, flushing, or heart palpitations, drastically affecting daily routines and comfort. 

Treatment is essential to achieve two main goals: controlling the growth and spread of the tumor and managing the debilitating hormonal symptoms. Because NETs are highly diverse and often slow-growing, the choice of medication depends entirely on the tumor’s location, its grade (how fast it’s growing), and whether it is functional (releasing hormones) or non-functional. Management of this chronic condition requires ongoing monitoring and a highly specialized approach. 

Overview of treatment options for Neuroendocrine Tumor 

The overall treatment strategy for Neuroendocrine Tumors is often multimodal, meaning it combines several approaches. While surgery is often the preferred choice for removing localized tumors, medication plays a crucial role in controlling symptoms, shrinking tumors, and slowing disease progression, particularly if the cancer has spread (metastatic disease). 

The main goals of medication-based treatment are to inhibit tumor growth and block hormone release. Medications are typically used for chronic, slow-growing tumors that cannot be fully removed surgically. The selection of drug classes is highly individualized and depends on the specific receptors found on the tumor cells and the presence of hormonal symptoms, such as those associated with Carcinoid Syndrome (National Cancer Institute, 2024). 

Medications used for Neuroendocrine Tumor 

Medication management for NETs is sophisticated and generally involves three major classes: hormone-targeting drugs, targeted therapies, and chemotherapy. 

1. Somatostatin Analogs (SSAs): These are the first-line and most widely used drugs for many slow-to-intermediate grade NETs, such as those in the gut or pancreas. Common examples include octreotide and lanreotide. They are primarily used to control hormonal symptoms (like flushing and diarrhea) and have also been shown to slow tumor growth. SSAs are often given as long-acting injections administered every three to four weeks. 

2. Targeted Therapies: For fast-growing or SSA-resistant tumors, targeted oral drugs like mTOR inhibitors (e.g., everolimus) and tyrosine kinase inhibitors (e.g., sunitinib) are used, often for pancreatic NETs. These agents block molecular pathways, preventing tumor growth signals and new blood vessel formation (Endocrine Society, 2020). 

3. Chemotherapy: Traditional chemotherapy (e.g., streptozocin, capecitabine, or temozolomide) is generally reserved for high-grade or rapidly dividing NETs, which are sometimes called neuroendocrine carcinomas. Chemotherapy works by directly destroying rapidly dividing cells, and while it often has more pronounced side effects, it can achieve a faster response in aggressive tumors. 

How these medications work 

Each drug class addresses the disease uniquely, either by hormone regulation or direct cancer cell attack. 

Somatostatin Analogs (SSAs) mimic the natural hormone somatostatin. By attaching to receptors on NET cells, SSAs shut down hormone production, alleviating symptoms. They also inhibit cell growth and block the tumor’s blood supply. 

Targeted Therapies block specific cell signaling pathways. For example, mTOR inhibitors stop the mTOR protein, a key regulator of cell growth, effectively slowing tumor growth by “starving” it. 

Chemotherapy uses toxic agents to damage the DNA and cell division machinery of cancer cells, leading to their death (apoptosis). 

Side effects and safety considerations 

Side effects vary significantly depending on the class of medication used. SSAs are generally well-tolerated, with common side effects like injection site pain, stomach upset, and occasional gallstones. 

Targeted Therapies are oral drugs often causing fatigue, mouth sores, skin rashes, and high blood pressure. They require careful monitoring due to complex signaling pathway interactions. 

Chemotherapy can cause severe side effects, including nausea, vomiting, hair loss, and bone marrow suppression (anemia, increased infection risk). 

All patients on systemic NET treatment need frequent lab monitoring (hormone levels, kidney function, blood cell counts). Patients should seek immediate medical care for persistent high fever, uncontrolled diarrhea, or severe chest pain. Since everyone’s experience with the condition and its treatments can vary, working closely with a qualified healthcare provider helps ensure safe and effective care. 

References 

  1. American Cancer Society. https://www.cancer.org 
  1. National Cancer Institute. https://www.cancer.gov 
  1. Endocrine Society. https://www.endocrine.org 
  1. Mayo Clinic. https://www.mayoclinic.org 

Medications for Neuroendocrine Tumor

These are drugs that have been approved by the US Food and Drug Administration (FDA), meaning they have been determined to be safe and effective for use in Neuroendocrine Tumor.

Found 15 Approved Drugs for Neuroendocrine Tumor

Octreotide Acetate

Brand Names
Sandostatin, Octreotide, Bynfezia

Octreotide Acetate

Brand Names
Sandostatin, Octreotide, Bynfezia
BYNFEZIA PEN is a s o matostatin analogue indicated: Acromegaly : To reduce blood levels of growth hormone (GH) and insulin growth factor 1 (IGF-1; somatomedin C) in acromegaly patients who have had inadequate response to or cannot be treated with surgical resection, pituitary irradiation, and bromocriptine mesylate at maximally tolerated doses. (1.1) Carcinoid Tumors : For the symptomatic treatment of patients with m eta static c a rcinoid t u mo rs where it suppresses or inhibits the severe diarrhea and flushing episodes associated with the disease. (1.2) Vasoactive Intestinal Peptide Tumors (VIPomas) : For the treatment of profuse watery diarrhea associated with VIP-secreting tumors. (1.3) Limitations of Use Improvement in clinical signs and symptoms, or reduction in tumor size or rate of growth, were not shown in clinical trials performed with octreotide injection; these trials were not optimally designed to detect such effects.

Netspot

Generic Name
68GA-Dotatate

Netspot

Generic Name
68GA-Dotatate
NETSPOT, after radiolabeling with gallium-68, is indicated for use with positron emission tomography (PET) for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult and pediatric patients. NETSPOT, after radiolabeling with gallium-68, is a radioactive diagnostic agent indicated for use with positron emission tomography (PET) for localization of somatostatin receptor positive neuroendocrine tumors (NETs) in adult and pediatric patients. ( 1 )

Everolimus

Brand Names
Torpenz, Afinitor Disperz, Afinitor, Zortress

Everolimus

Brand Names
Torpenz, Afinitor Disperz, Afinitor, Zortress
TORPENZ (everolimus) tablets are a kinase inhibitor indicated for the treatment of: Postmenopausal women with advanced hormone receptor-positive, HER2-negative breast cancer in combination with exemestane after failure of treatment with letrozole or anastrozole.

Sutent

Generic Name
Sunitinib

Sutent

Generic Name
Sunitinib
Sunitinib malate capsules are a kinase inhibitor indicated for: treatment of adult patients with gastrointestinal stromal tumor (GIST) after disease progression on or intolerance to imatinib mesylate.

Pembrolizumab

Brand Names
Keytruda QLEX, Keytruda

Pembrolizumab

Brand Names
Keytruda QLEX, Keytruda
KEYTRUDA QLEX is a combination of pembrolizumab, a programmed death receptor-1 (PD-1)-blocking antibody, and berahyaluronidase alfa, an endoglycosidase, indicated: Melanoma for the treatment of adult patients with unresectable or metastatic melanoma.
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