In this gift-giving season, it seems appropriate to list the most incredible medical breakthroughs made this year, because each of them is a gift that will save millions of lives. Another step closer to an insomnia cure In America, about one in three adults struggles to get enough sleep, which is linked to overeating, obesity, diabetes, heart disease, and problems on the job. Our sleep cycles are tied to an internal body clock (circadian rhythm)—and now scientists have discovered not only a gene that controls our normal daily biological rhythm, but also the mechanism by which that gene operates. Because of the importance of sleep to health and well-being, these discoveries are expected to have vast implications for human health and well-being. Hep C treatment This year, a groundbreaking study revealed a single receptor cell in the human body that can recognize Hepatitis C, a viral disease that can lead to diminished liver function, liver failure, and/or death, and from which an estimated 2.7 to 3.9 billion Americans suffer, according to the Centers for Disease Control and Prevention. That single cell may be a key step in developing a vaccine and/or cure for Hepatitis C. But that’s not the biggest news on the Hep C front. The bigger news is that in late August, the FDA approved a new drug for the treatment of Hep C. in the U.S. The new drug, called “Mavyret” (generic name: glecaprevir and pibrentasvir), is an eight-week treatment for patients with or without cirrhosis, and with or without moderate to severe kidney disease (both of which are Hep C complications). “This approval provides a shorter treatment duration for many patients, and also a treatment option for certain patients,” says Edward Cox, MD, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research in the press release. Nerlynx to treat breast cancer Last year, researchers from the UK discovered that a novel combination of two existing cancer-treating drugs—Herceptin (generic name: trastuzumab) and Tyverb (generic name: lapatinib)—dramatically reduce the size of tumors caused by a particularly aggressive form of breast cancer. This year, the FDA approved a new drug for that same form of breast cancer. Called Nerlynx (generic name: neratinib), it’s been approved for patients with early-stage disease after an initial regimen that includes the drug Herceptin. Its purpose is to further lower the risk of the cancer recurring. “HER2-positive breast cancers are aggressive tumors and can spread to other parts of the body, making this additional therapy an important part of the treatment plan,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research in a press release. “Now, these patients have an option after initial treatment that may help keep the cancer from coming back.” Parkinson’s innovation An estimated 50,000 Americans are diagnosed with Parkinson’s disease each year, according to the National Institutes of Health. Parkinson’s is a movement disorder that occurs when cells in the brain that produce the chemical dopamine become impaired or die (dopamine helps transmit signals between the areas of the brain that produce smooth, purposeful movement). Look out for these early warning signs of Parkinson’s disease. “Parkinson’s is a relentless disease without a cure,” said Eric Bastings, MD, deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research in a press release. Currently, the treatment protocols are not consistently effective and can have unfortunate side effects, but there is promise in the newly FDA-approved Parkinson’s drug, Xadago (safinamide), as an add-on treatment for patients whose medications have ceased to work well. New understanding of cholesterol If your doctor says you have high cholesterol, it means the LDL reading (bad cholesterol) is building up and may cause blockages in your arteries, a risk factor for heart disease and stroke. A healthy LDL reading from a blood test is less than 200 mg. With certain new drug combinations, LDL levels can be reduced by 75 percent. While this stat is remarkable, many patients and doctors have been wondering, how low is too low? This year, doctors at the Cleveland Clinic revealed that from what they can tell, there is no such thing as “too low.” A number of trials have recently shown a 20 percent reduction in the risk of cardiovascular death, heart attack, and stroke in patients who took statins combined with a new class of cholesterol-lowering drugs (PCSK9 inhibitors) to reach ultra-low LDL levels. What this means, according to the Cleveland Clinic, is that doctors now “have the tools and the research to take the fight to LDL cholesterol. With over 400,000 coronary disease deaths and 102 million Americans living with high cholesterol levels, these new strategies hold the promise to turn the tide in 2018.” A new definition of “high blood pressure” High blood pressure may show no symptoms, but it’s a major risk factor for cardiovascular disease. In fact, high blood pressure doubles your risk of death from stroke, heart disease, or other vascular diseases, according to the American Heart Association Task Force on Clinical Practice Guidelines (the Task Force). Your blood pressure reading is made up of two numbers: systolic pressure, or how much force the blood places on the walls of the arteries when your heart beats, and diastolic pressure, the same force but when your heart is resting between beats. Previously, what was considered high blood pressure was a score of anything above 140 (systolic) over 90 (diastolic), which affected about 1 in 3 adults in this country, according to Science News. However, under new guidelines issued by the Task Force, 130 is essentially the new 140, and 80 is the new 90. Under these new guidelines, nearly half of Americans fall under the definition of high blood pressure, and the American Heart Association recommends that Americans look to their lifestyle choices first when trying to reduce hypertension. Childhood cancer breakthough The most common childhood cancer in the United States, leukemia affects about 3,100 kids every year. Now there’s a cure within reach for children who were previously out of options, thanks to a groundbreaking new treatment pioneered by Kevin J. Curran, MD, at Memorial Sloan Kettering Cancer Center in New York City. The therapy, known as CAR T-Cell Therapy (also known as Kymriah), was recently approved by the FDA for treating acute lymphoblastic leukemia in kids and young adults. It works by directing the body’s immune system to attack cancer cells. CAR T-Cell Therapy put 15-year-old Zach Swart, a patient of Dr. Curran’s who’d been battling ALL for ten years, into remission within a month after initiating treatment. Soon, Zach was strong enough to receive a bone marrow transplant that just may have saved his life. “I was initially afraid about CAR-T’s side effects,” Zach tells Reader’s Digest, “But then I didn’t have any at all. I was so lucky, and it feels so good not to be sick anymore and to be home and see my friends. I’m determined to leave the cancer far behind me.” “Every day I see Zach smile, laugh, and just be a kid, it is truly a miracle,” his mom says. Promising pancreatic cancer developments Pancreatic cancer is tough to detect and so difficult to treat that just 20 percent of patients survive a year past diagnosis, and fewer than 10 percent make it to the 5-year mark. But recent discoveries are offering new and improved detection and treatment of pancreatic cancer. Specifically, STAT News reports that 2017 marked four breakthrough discoveries in the field of pancreatic cancer: Researchers reported in Science that they’d found gut bacteria in pancreatic cancers that actually break down chemotherapy drugs, rendering them ineffective. With this understanding, scientists can devise new treatments withstand the body’s microbiome. Researchers found a link between new-onset diabetes and pancreatic cancer, which could help with early detection. Apparently in some people, diabetes is caused by cancer growth in the pancreas. Soon, a more sensitive test to help determine the root cause of diabetes may reveal pancreatic cancer much earlier. A team of scientists at the Gastrointestinal Cancer Center at Dana-Farber Cancer Institute discovered not only a genetic mutation that is present in more than 90 percent of pancreatic cancer patients, but also different variations that are associated with different outcomes. This has the potential to lead to novel, gene-based treatments for pancreatic cancer. Immunotherapy has shown promise as a treatment. A small Phase 1 studyfound that combining two immunotherapy drugs may help some patients. “This is a glimmer of hope into using immunotherapy,” said Dr. Zev Wainberg, co-director of the GI Oncology Program at University of California, Los Angeles. “Until this point, there have been no responses with any immunotherapy drugs for pancreatic cancer.” An autism gene Autism affects about 3.5 million Americans, and about one in 68 U.S. children, according to the Centers for Disease Control and Prevention, and no single cause has been identified. (These are the signs of autism to watch for.) But scientists at University of Southern California (USC) recently identified a hotspot for autism-related mutations in a single gene known as TRIO. The findings were published in the journal Nature Communications. “I have never seen this number of autism-related mutations in such a small area,” said the study’s corresponding author, Bruce Herring, PhD, a neurobiologist at the USC’s Dana and David Dornsife College of Letters, Arts and Sciences. “The likelihood that this number of mutations occurs by chance is 1 in 1.8 trillion. We’re pretty confident these mutations contribute to the development of autism-related disorders.” It is hoped that this major discovery will be the key to developing more effective strategies for treating autism spectrum disorders. HIV: kick and kill Current HIV treatments are allowing people with the virus to live longer, healthier lives, but they couldn’t rid the body of the virus—until now. A recent study led by the David Geffen School of Medicine at UCLA has developed a new approach aimed at eliminating dormant “reservoirs” of the HIV virus. The treatment involves “waking up” any dormant virus in the body, causing it to begin replicating—so that it can be destroyed. It’s kind of like smoking out the bad guys, but scientists refer to this approach as “kick and kill.” Scientists are now exploring ways to make the still-experimental treatment less toxic, and to evaluate its effectiveness in larger animals before it can be tested in humans. Earlier this year, a different group of researchers made this astonishing discovery that may help lead to an HIV vaccine. Solutions to the organ shortage A groundbreaking new method of safely reviving frozen organs promises to save millions of lives by significantly addressing the critical shortage of organs available for donation. A research team out of the University of Minnesota has discovered a process to successfully rewarm large-scale animal heart valves and blood vessels preserved at very low temperatures. The discovery is a major step forward in saving millions of human lives by increasing the availability of organs and tissues for transplantation through the establishment of tissue and organ banks, according to a press release by the University of Minnesota. Bonus discovery: New research in the field of organ transplantation has opened up the possibility of pigs becoming organ donors in the not-so-distant future. Head transplantation It may sound wacky, but Italian neurosurgeon Sergio Canavero, MD, claims to be on track to complete the first-ever transplantation of a human head to different body. A 31-year-old Russian man suffering from spinal muscular atrophy has volunteered to be the first person to undergo the surgery, which Dr. Canavero pitched to the American Academy of Neurological and Orthopaedic Surgeons in the hopes of convincing surgeons to assist him in this risky procedure, ideally before the end of this year. According to a study published in the journal CNS Neuroscience and Therapeutics, Dr. Canavero and a team of Chinese doctors severed the spinal cords of 15 rats and attempted to reattach nine of them. All but one of the rats survived for an “astonishing 30 days” after the experiment. It’s still unknown whether Dr. Canavero has been able to recruit enough support to perform the surgery on the Russian man, so stay tuned for updates. source
