TITLE:
Prolonged Disease Stabilization and Tolerability in a Nuclear Protein in Testis Midline Carcinoma Patient Treated with Dual Histone Deacetylase and Phosphoinositide 3-Kinase Inhibitor CUDC-907
AUTHORS:
Pamela Munster, Nilson Wu, Meaghan McMahon, Robert Gharavi, David Tuck
KEYWORDS:
NMC, MYC, CUDC-907, HDAC, PI3K
JOURNAL NAME:
Case Reports in Clinical Medicine,
Vol.7 No.7,
July
24,
2018
ABSTRACT: Introduction: Nuclear protein in testis midline carcinoma (NMC) is a rare and extremely aggressive carcinoma (median survival Case Report: A 61-year old female NMC patient enrolled on study CUDC-907-102 (NCT02307240) after rapidly progressing through two prior treatments. The patient’s assessable sites of disease consisted of right pleural effusion, right hilar soft tissue, and segment IV liver. Treatment was well tolerated with toxicities primarily consisting of manageable diarrhea and thrombocytopenia. The patient remains on active treatment after more than 32 months of stable disease. Discussion: Dysregulation of MYC in NMC is believed to play a central role in pathogenesis. CUDC-907 has demonstrated potent suppression of MYC expression and anti-tumor activity in preclinical NMC models, providing a mechanistic rationale for the prolonged disease stabilization observed here. The treatment of additional NMC patients with CUDC-907 is needed to further evaluate this promising report. Conclusion: This case demonstrates a rare success in the treatment of a devastating disease using only a novel small molecule, warranting further investigation of CUDC-907 in NMC.