FDA authorizes emergency use of JYNNEOS to increase monkeypox vaccine supply
The U.S. Food and Drug Administration (FDA) issued an emergency use authorization (EUA) allowing health care providers to use the JYNNEOS vaccine by intradermal injection for individuals 18 years of age and older who are at high risk for monkeypox infection, increasing the total number of doses available for use by nearly five-fold. This comes in response to the recent rapid increase in the spread of the virus and insufficient vaccine supply.
The Modified vaccinia Ankara (MVA) vaccine JYNNEOS was originally approved in 2019 for prevention of smallpox and monkeypox disease in adults 18 years of age and older determined to be at high risk. For those adults, the EUA now allows for a fraction of the JYNNEOS dose to be administered intradermally. Two doses given four weeks apart will be needed. The EUA also allows JYNNEOS to be administered subcutaneously in individuals younger than 18 years of age who are at high risk of monkeypox infection as two doses, four weeks apart. The results of a clinical study of the MVA vaccine demonstrated that intradermal administration produced a similar immune response to subcutaneous administration, meaning individuals in both groups responded to vaccination similarly.
Researchers develop STORK Test to rapidly detect abnormal fetal chromosomes
A National Institutes of Health (NIH)-funded study found that the same-day Short-read Transpore Rapid Karyotyping (STORK) test can help identify causes of miscarriage and potentially improve the in vitro fertilization (IVF) process by detecting extra or missing fetal chromosomes (i.e., aneuploidy). STORK collects samples from prenatal tests, such as amniocentesis and chorionic villus sampling, as well as tissue obtained from miscarriage and biopsies from pre-implantation embryos produced using IVF. STORK had an accuracy of 98-100% when researchers compared it to standard methods by testing 218 samples that included tissue from miscarriage, chorionic villi, amniotic fluid, and trophectoderm biopsies, which are used to evaluate embryos before IVF implantation. STORK was 100% in accordance with standard clinical testing in another set of 60 samples.
STORK is faster and cheaper than standard clinical tests. It provides results within hours versus several days and costs less than $50 per sample, if 10 samples are run at the same time, or up to $200 if a sample is run on its own. STORK can also be done at the point of care for a patient, eliminating the need to ship a sample to a clinical laboratory. STORK may be more useful in identifying genetic causes after a first miscarriage, compared to genetic tests conducted after multiple miscarriages, and may also be used to improve the IVF process. STORK’s ability to provide results within hours could eliminate the need for embryos to be frozen while genetic tests are run and analyzed before implantation, saving time and money.
Researchers identify 3 proteins that could prevent heart failure after heart attack
Heart failure is the primary cause of death and disability globally, affecting approximately 64 million people, and there is currently no effective therapeutic treatment. In a preclinical study supported by the British Heart Foundation, scientists working to develop new therapies and treatments for heart failure patients have discovered three proteins that have been shown to restore heart function following a heart attack in mice. The proteins can be injected immediately after a heart attack, with the potential to preserve heart function following an attack, as shown in positive preclinical data (Science Translational Mag 2022;14:660).
Researchers developed an innovative technology called FunSel, a protein “search engine” that looks for proteins with the potential to protect heart cells against the rapid cell death that typically occurs following a heart attack and without the bias that often comes with drug development. In a library of over 1,000 proteins, FunSel identified three (Chrdl1, Fam3c, and Fam3b) that have been shown to prevent cardiac damage in mice after a heart attack and preserve cardiac function over time. With the success of the preclinical tests, clinical trials in humans will take place in the next two years.
First targeted therapy for HER2-low breast cancer approved by FDA
Enhertu (fam-trastuzumab-deruxtecan-nxki), an I.V. infusion, is the first FDA-approved therapy for the treatment of patients with unresectable or metastatic HER2-low breast cancer, a newly defined subset of HER2-negative breast cancer. HER2-low describes a subtype that has some HER2 proteins on the cell surface, but not enough to be classified as HER2-positive. In the 280,000 new cases of female breast cancer projected by the National Cancer Institute in the U.S. in 2022, approximately 80%-85% would be previously considered as the HER2-negative subtype. About 60% of those patients are now considered as HER2-low and previously would have received endocrine therapy or chemotherapy. Enhertu has received priority review and breakthrough therapy designations by the FDA. Patients with HER2-low breast cancer are eligible to receive Enhertu if they have previously undergone chemotherapy in the metastatic setting, or if their cancer returned during or within six months of completing adjuvant chemotherapy.
The approval is based on data from DESTINY-Breast04, a clinical trial that studied 557 adult patients with unresectable or metastatic HER2-low breast cancer. Among the 557 patients, 24% were age 65 or older, with female patients making up 99.6% of the trial population. Trial participants’ race was reported as 48% White, 40% Asian, 2% Black or African American, and 3.8% Hispanic/Latino. The patients were divided into two groups: 494 hormone receptor positive patients and 63 hormone receptor negative patients. Of these patients, 373 randomly received Enhertu by intravenous infusion every three weeks, and 184 randomly received the physician’s choice of chemotherapy. The results showed improvement in both progression-free survival and overall survival in people with unresectable or metastatic HER2-low breast cancer.
Novel blood test helps evaluate severity in a rare lung disease
Pulmonary arterial hypertension (PAH) is a rare, life-threatening condition that causes unexplained high blood pressure in the lungs, affecting fewer than 50,000 people in the U.S. The condition is characterized by progressive narrowing and blockage of the small pulmonary arteries of the lungs, strain on the right side of the heart, and eventual death from heart failure. The damage to the lung in severe cases can require lung transplantation. Patients with PAH, a majority of whom are women, have a high death rate, and there is currently no cure. In a study funded by the NIH, researchers have found that a novel blood test that measures DNA fragments shed by damaged cells, called cell-free DNA, can be used to evaluate the severity in patients with PAH and predict survivability. Patients with PAH have more of these fragments, increasing the severity of their disease. The use of this blood test for PAH patients could allow doctors to intervene faster than with current testing methods, such as echocardiography, to prevent or delay progression of the disease and possibly save lives. Researchers analyzed cell-free DNA in blood samples from 209 adult patients diagnosed with PAH and compared the results to cell-free DNA measured from a control group of 48 healthy volunteers without PAH, finding that cell-free DNA was elevated in patients with PAH and that patients with the highest level of cell-free DNA had a 3.8-times greater risk of either death or a need for lung transplantation compared to those with the lowest levels. With the novel blood test, researchers can identify specific tissues affected by PAH, potentially leading to the development of new treatment methods.
Hydrogel system offers pathways to finding treatments for kidney disease
Washington University’s McKelvey School of Engineering and the School of Medicine in St. Louis discovered the use of a new hydrogel system for preservation of the biochemistry and mechanical environments of cultured podocyte cells, demonstrating the potential for new treatments for kidney disease (Science Advances 2022;8:35). Chronic kidney disease and failure affect 13% of the U.S. population and are currently incurable. Podocytes are unique cells with a hierarchical structure that include octopus-like tentacles forming the structure for the kidney’s filtering units. Diseases that affect podocytes, such as diabetic nephropathy, degrade the podocyte cells and disrupt these structures, leading to kidney failure that necessitates dialysis.
Development of the hydrogel system allows podocyte cells to be cultured outside of the body by taking podocytes from isolated glomeruli, the filtering units of the kidney, to be cultured on patterns of the proteins that would be in their vicinity in healthy and diseased kidneys. The cells rapidly adopt new shapes and express new protein structures that are associated with injury, enabling researchers to identify new ways to control the mechanisms that these cells use to heal themselves. Washington University has applied for a patent on the hydrogel technology, as they believe it has potential to transform development of therapeutics.
AI-powered Voluson Expert 22 is improving ultrasounds and advancing women’s health care
GE Healthcare recently unveiled the next-generation Voluson™ Expert 22, a tool powered by artificial intelligence (AI) that uses graphic-based beamformer technology to produce higher-quality images and offer greater flexibility in imaging functions. The advanced technology offers customizable touch panels and color and lighting options and is designed for consistency in exams and decreased number of tasks. The Lyric Architecture of the Voluson Expert 22 ensures higher resolution, detailed images, and increased independence from body habitus and other difficult scanning conditions. Its AI-powered features include SonoLyst, which identifies fetal anatomy and adds annotations and measurements for efficiency; SonoPelvicFloor, which automates plane alignment and measurements to simplify the exam process; and SonoCNS, which helps to align and display recommended views and measurements of the fetal brain from a 3D volume. The Voluson Expert 22 represents a major step forward in ultrasound innovation.