Multiple endocrine neoplasia type 1 future or investigational therapies

Multiple endocrine neoplasia type 1 Microchapters
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Ammu Susheela, M.D. [2]

Future or Investigational Therapies

• Since MEN type 1 is a rare disorder clinical trials are difficult to pursue due to paucity of study population.
• National and international collaborations are encouraged to recruit more patients with multiple endocrine neoplasia type 1 for clinical trials.
• Use of TRK inhibitors for medullary thyroid cancer and pancreatic neuroendocrine tumors have been investigated under multicenter clinical trials.
• mTOR inhibitors for pancreatic neuroendocrine tumors are other trials undergoing investigation.
• Role of surgery for non functioning pancreatic neuroendocrine tumors are under investigation.
• The interaction between menin and mixed lineage leukemia protein 1(MLL1) also known as a histone H3 lysine 4 methyltransferase has been targeted to find cure for this disorder.
• Thienopyrimidine analogs that binds to wild type menin have been investigated as a possible treatment for Multiple endocrine neoplasia type 1.
• Molecular phenotyping of tumors have been proposed to use to detect MEN type 1 tumors at an early stage.