FDA approval brings first gene therapy to the United States

March 29, 2018 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, M.D.  “New technologies such as … Read more

Heart’s twisting motion could predict mitral valve surgery outcome: Study

March 27, 2018 The researchers examined 50 patients with the condition for a median of two-and-one-half years following mitral surgery, according to an American College of Cardiology statement.  All of them exhibited advanced heart failure symptoms and had received the maximum guideline-based therapy for more than 6 months.  Of the 50 patients, 19 had died … Read more

How Patient Preferences Contribute to Regulatory Decisions for Medical Devices

March 22, 2018 In August 2017, for the first time we cleared an expanded indication for a home hemodialysis machine so it could be used without a care partner being present, a decision based in part on asking kidney patients about their tolerance for risk. Home dialysis may improve a patient’s quality of life, allowing … Read more

Gene therapy reverses symptoms, slows progression of MS in mice

March 21, 2018 In MS, the immune system attacks myelin, the fatty substance that coats and protects nerve fibers in the brain and spinal cord.  Its cause is still unknown, however, researchers think it stems from self-reactive effector T cells that target myelin.  Current treatments for MS focus on reducing the number of relapses — … Read more

How poliovirus unleashes an attack on glioblastoma

March 15, 2018 Now that same team has released a study that delves into the process by which the virus fights malignancy.  It appears to do so by activating an inflammatory process, the researchers believe.  That inflammation interferes with the ability of cancer cells to escape immune attack, according to a press release.  The research … Read more

Will Amazon really get into drug sales? It’s in talks with PBMs already, report says

March 13, 2018 The retail giant has stayed quiet about its pharma ambitions; however, a Goldman Sachs analyst in August 2017 outlined some potential pharmaceutical strategies the company might use.  Now, Leerink Partners has published a report saying the category-killer is in talks with mid-size Pharmacy Benefits Managers (PBMs) “in an effort to get into various … Read more

Can the culprit for cancer spreading also boost immuno-oncology treatments?

March 08, 2018 Lymphatic vessels have the ability to wrap around tumors and weave through them, thereby allowing cancer cells to leave the primary site and spread throughout the body.  However, in patients being treated with immune checkpoint inhibitors like Bristol-Myers Squibb’s Yervoy and Opdivo, those same vessels enhance the power of cancer-killing T cells, … Read more

Blood test could mean earlier diagnosis, better treatment for macular degeneration

March 06, 2018 The Massachusetts Eye and Ear team studied metabolites, or small molecules in the blood of patients with early, intermediate and late-stage AMD, as well as from individuals without AMD.  “The metabolome — the set of metabolites present in an individual — is thought to closely represent the true functional state of complex diseases,” … Read more

Platypus venom could give rise to a new class of diabetes treatments

March 01, 2018 Like humans, monotremes — platypus and echidnas — produce GLP-1 in their gut.  The hormone triggers insulin secretion, which lowers blood glucose levels; however, in people, it breaks down in less than two minutes, making it of little use for controlling blood sugar in patients with Type 2 diabetes. The researchers, from the … Read more