FDA Awards Orphan Status to Brain Cancer Vaccine Developed at Roswell Park Cancer Institute

Excerpt:

“The U.S. Food and Drug Administration has awarded orphan drug status to a promising immunotherapy vaccine developed at Roswell Park Cancer Institute. The FDA notified MimiVax LLC, a Roswell Park spinoff company, on Aug. 3 that its application for orphan status for SurVaxM as treatment for glioblastoma, a type of brain cancer, had been approved.

“Orphan status is a special designation awarded to encourage innovation and exploration of approaches to treat rare diseases that affect relatively few people. SurVaxM, also known as DRU-2017-5947, is an immunotherapy drug that targets survivin, a cell-survival protein present in most cancers.”

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Molecular Pathways: MERTK Signaling in Cancer

“MERTK is a receptor tyrosine kinase of the TAM (Tyro3, Axl, MERTK) family, with a defined spectrum of normal expression. However, MERTK is over-expressed or ectopically expressed in a wide variety of cancers, including leukemia, non-small cell lung cancer, glioblastoma, melanoma, prostate cancer, breast cancer, colon cancer, gastric cancer, pituitary adenomas, and rhabdomyosarcomas, potentially resulting in the activation of several canonical oncogenic signaling pathways. These include the mitogen-activated protein kinase and phosphoinositide 3-kinase pathways, as well as regulation of signal transducer and activator of transcription family members, migration associated proteins including the focal adhesion kinase and myosin light chain 2, and pro-survival proteins such as survivin and Bcl-2. Each has been implicated in MERTK physiologic and oncogenic functions.”


Molecular Pathways: MERTK Signaling in Cancer

“MERTK is a receptor tyrosine kinase of the TAM (Tyro3, Axl, MERTK) family, with a defined spectrum of normal expression. However, MERTK is over-expressed or ectopically expressed in a wide variety of cancers, including leukemia, non-small cell lung cancer, glioblastoma, melanoma, prostate cancer, breast cancer, colon cancer, gastric cancer, pituitary adenomas, and rhabdomyosarcomas, potentially resulting in the activation of several canonical oncogenic signaling pathways. These include the mitogen-activated protein kinase and phosphoinositide 3-kinase pathways, as well as regulation of signal transducer and activator of transcription family members, migration associated proteins including the focal adhesion kinase and myosin light chain 2, and pro-survival proteins such as survivin and Bcl-2. Each has been implicated in MERTK physiologic and oncogenic functions.”


Molecular Pathways: MERTK Signaling in Cancer

“MERTK is a receptor tyrosine kinase of the TAM (Tyro3, Axl, MERTK) family, with a defined spectrum of normal expression. However, MERTK is over-expressed or ectopically expressed in a wide variety of cancers, including leukemia, non-small cell lung cancer, glioblastoma, melanoma, prostate cancer, breast cancer, colon cancer, gastric cancer, pituitary adenomas, and rhabdomyosarcomas, potentially resulting in the activation of several canonical oncogenic signaling pathways. These include the mitogen-activated protein kinase and phosphoinositide 3-kinase pathways, as well as regulation of signal transducer and activator of transcription family members, migration associated proteins including the focal adhesion kinase and myosin light chain 2, and pro-survival proteins such as survivin and Bcl-2. Each has been implicated in MERTK physiologic and oncogenic functions.”


Utility of survivin mRNA as a diagnostic biomarker in lung cancer with malignant pleural effusion

Since survivin is frequently overexpressed in lung cancer, it might play a role in oncogenesis and progression of the tumor. The detection of survivin by RT-PCR seems to be a promising assay to diagnose malignant pleural effusions, using the appropriate cut-off point. Survivin mRNA has a significant role in differentiating benign from malignant pleural effusion.