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Archive - Aug 31, 2017

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Variants in Fetal Genome Associated with Risk of Preeclampsia in Mother

For the first time, a relationship has been found between fetal genes and the risk of preeclampsia in the mother. Norwegian researchers, including a team from the Norwegian University of Science and Technology (NTNU), figure prominently in the international group that presented the discovery in Nature Genetics, first published online earlier this summer, on June 19, 2017. The article is titled “Variants in the Fetal Genome Near FLT1 Are Associated with Risk Of Preeclampsia.” Preeclampsia affects approximately 3 per cent of births in Norway; worldwide, that number is estimated to be about 5 per cent. In the vast majority of cases, the mother has mild symptoms, typically high blood pressure. Nevertheless, preeclampsia is one of the leading causes of death in both mothers and babies around the time of birth, and sometimes the only way to treat it is to deliver the baby, even prematurely. "For the first time ever, we have discovered a fetal gene that increases the risk of preeclampsia," says Dr. Ann-Charlotte Iversen at the Department of Clinical and Molecular Medicine at NTNU. She says there has not been much research on the role of fetal genes in triggering the illness. Preeclampsia usually begins with a problem in the placenta, which is mainly composed of fetal cells. For that reason, it makes sense that fetal cells might have a hand in causing the illness, Dr. Iversen said, even though it is the mother's symptoms during the last part of the pregnancy that lead to the diagnosis. Dr. Iversen is head of a research group at NTNU's Centre of Molecular Inflammation Research (CEMIR).

FDA Approval of Novartis’ Kymriah Brings First Gene Therapy to United States; CAR T-Cell Therapy Approved to Treat Certain Children and Young Adults with B-Cell Acute Lymphoblastic Leukemia (ALL)

On August 30, 2017, the U.S. Food and Drug Administration issued a historic action making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases. The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, MD. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.” Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse. Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.