This week, we bring you news of a gene therapy with the potential to change lives for children with Duchenne muscular dystrophy, having experienced positive results in an initial small group of pediatric patients. There’s also news of novel blood tests for the early detection of Alzheimer’s and certain cancers; COVID-19 tracking via a new digital health tool – smart clothing; and updates in research on multiple sclerosis; autoimmune conditions; and psoriasis.
Gene Therapy Sees Encouraging Success in Child with Duchenne Muscular Dystrophy
A revolutionary gene therapy treatment for Duchenne Muscular Dystrophy has brought hope to many families, the treatment consists of a single infusion that fixes the genetic mutation that gradually causes muscle cells to die. Six children have now experienced positive results using the therapy, and although it does not restore lost muscle cells - it does allow those the patient is left with to function better. The success suggests that gene therapy could be poised to change the lives of thousands of children - usually boys - who have Duchenne. But scientists still want to see the results of a much larger trial of the therapy, which is likely to begin later this year.
Experimental Blood Test Detects Cancer up to Four Years Before Symptoms Appear
For years scientists have sought to create the ultimate cancer-screening test - one that can reliably detect a malignancy early, before tumor cells spread and when treatments are more effective. A new method has recently been reported which brings researchers a step closer to that goal. An international team of scientists was able to diagnose cancer long before symptoms appeared in nearly all of the people who went on to develop cancer by using an experimental blood test.
Genetic Risk for Autoimmune Diseases and Allergies may Exist from Birth
A study carried out by scientists in the UK and Australia from the Cambridge Baker Systems Genomics Initiative shows that variants in some genes may predispose people to developing autoimmune diseases or allergies from an early age. Autoimmune diseases such as type 1 diabetes, rheumatoid arthritis and inflammatory bowel diseases affect around 7% of the US population and even more, around 20% or higher, have some form of allergy.
Hopes Raised for Early Blood Test to Help Fight Alzheimer’s
Scientists say they have made progress towards a test for Alzheimer’s that could help researchers in the hunt for treatments for the disease. Several studies, presented at a conference in Chicago, showed it was possible to measure the levels of a protein called tau in the blood. Tau and another protein called amyloid have been targets for developing tests and treatments for Alzheimer’s for many years. They form clusters known as tangles and plaques in the brain that are well-known features of the disease. Any test will help research by identifying people who have the early stages of the disease and may not yet have any symptoms.
Protein Linked to Inflammation in MS also Helps to Control it
The pro-inflammatory signaling protein interleukin (IL)-17A, which is associated with nerve damage in multiple sclerosis (MS), also has an opposing, and crucial, anti-inflammatory role in cells, a recent study reports. These findings may explain why therapies that lower IL-17A levels have failed in clinical trials to treat autoimmune diseases like MS.
Digital Health Researchers Flock to Smart Clothing in Studying Sweat & COVID-19
Nanowear, a company that develops smart clothing, has announced a research collaboration with New York City-Metro Health Systems that is focused on remote detection of COVID-19. In the study, researchers plan to monitor patients with confirmed or suspected COVID-19 by using Nanowear’s cloth-based nanosensors, which were designed to detect clinical biomarker changes. Patients in the study will get a one-size-fits-all garment that's adjustable. This tool is supposed to measure ECG, systolic and diastolic blood pressure, respiration, lung volume and fluid, and temperature trends.
New Potential Advances in Treating Psoriasis: A Skin Condition Affecting 125+ Million People
Although psoriasis has been a well-recognized skin condition for many years, there has been significant progress over the last decade on effective therapeutic models and treatment modalities for this autoimmune condition - ranging from topical treatments, to injections and oral medications. There are many different mechanisms of action and physiological targets for psoriasis treatments; a few examples include reducing inflammation, slowing skin growth, or altering the immune system.