Category PRECISION MEDICINE

Multiple myeloma treatment granted FDA Orphan Drug Designation

Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Glenmark Pharmaceuticals’ bispecific antibody candidate GBR 1342 for the treatment of patients with multiple myeloma who have received prior therapies. “Patients who are struggling with diseases like

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Research Continues to Drive Advances Against Cancer following AACR Cancer Progress Report 2019

AACR Cancer Progress Report 2019 details 27 new cancer treatments, a record high number of U.S. cancer survivors, and the benefit of increasing federal investments in medical research through the National Institutes of Health and the National Cancer Institute The American Association for Cancer Research (AACR) today released its annual Cancer Progress Report highlighting how research largely

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Novartis’ Phase III studies of ofatumumab achieve primary goals in relapsing multiple sclerosis

Novartis has reported positive findings from the Phase III ASCLEPIOS I and II clinical trials of ofatumumab (OMB157), which met their primary endpoints in relapsing forms of multiple sclerosis (RMS) patients. Ofatumumab is an anti-CD20 monoclonal antibody (mAb) designed to cause B-cell lysis and depletion. It has been formulated for once-monthly, subcutaneous self-administration. The identical

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Fedratinib Approval Further Validates JAK Inhibition for Myelofibrosis Treatment

Source Targeted Oncology JAK inhibition continues to drive progress in myelofibrosis treatment as fedratinib (Inrebic) now joins ruxolitinib (Jakafi) in the treatment landscape for myelofibrosis. Since its FDA approval in August 2019, fedratinib has become both an additional treatment and a second-line option for patients who respond poorly to ruxolitinib. The approval of fedratinib was

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Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer

Source https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(19)30339-X?elqTrackId=39785ec7f12f4ed79ff115249bc871f5&elq=81c0af21c850457daca48d5be457f158&elqaid=26028&elqat=1&elqCampaignId=10599 Invariant natural killer T (iNKT) cells are potent immune cells for targeting cancer; however, their clinical application has been hindered by their low numbers in cancer patients. Here, we developed a proof-of-concept for hematopoietic stem cell-engineered iNKT (HSC-iNKT) cell therapy with the potential to provide therapeutic levels of iNKT cells for a patient’s

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T-cell therapy receives FDA’s orphan drug status for soft tissue sarcoma

The FDA has granted orphan drug designation to ADP-A2M4, an investigational specific peptide-enhanced affinity receptor T-cell therapy, for the treatment of soft tissue sarcomas. ADP-A2M4 (Adaptimmune) is an autologous, genetically modified SPEAR T-cell therapy that targets the MAGE-A4 antigen on the surface of cancer cells. Certain soft tissue sarcomas express MAGE-A4 at high levels and

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Using MicroRNA to Starve a Tumor?

Source September 5th, 2019 by Dr. Francis Collins Tumor cells thrive by exploiting the willingness of normal cells in their neighborhood to act as accomplices. One of their sneakier stunts involves tricking the body into helping them form new blood vessels. This growth-enabling process of sprouting new blood vessels, called tumor angiogenesis, remains a vital area of

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NIH, Cincinnati Children’s scientists develop potential strategy against leukemia drug resistance

Two-pronged approach stymies AML escape plan, could have applications to many cancers. Scientists from the National Institutes of Health and Cincinnati Children’s Hospital Medical Center have devised a potential treatment against a common type of leukemia that could have implications for many other types of cancer. The new approach takes aim at a way that

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