Category PRECISION MEDICINE

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

Read More


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

Read More


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

Read More


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

Read More


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

Read More


Immunotherapy for Pediatric Solid Tumors: What’s the Latest?

Immunotherapy can be effective in treating certain types of pediatric blood cancers, but researchers are still exploring how this type of treatment could work for pediatric solid tumors. In clinical trials, scientists are combining immunotherapy agents in an effort to jump-start the immune system against pediatric solid tumors. Source DANA-FARBER Cancer Institute New treatments that

Read More


The Era Of Personalized Medicine Has Arrived – PMC’s Annual Progress And Outlook Report

The Personalized Medicine Coalition (PMC) released its annual “Personalized Medicine At FDA: A Progress & Outlook Report” (Report) that monitors current successes and challenges in bringing personalized therapies to market. Notable Developments for 2018 Several milestones were noted for 2018 – ranging from new drug approvals to direct-to-consumer genetic testing. 1.            A record number of

Read More