The U.S. Food and Drug Administration approved Omisirge (omidubicel-onlv), a substantially modified allogeneic cord blood-based cell therapy, to cut the risk of infection in patients with blood cancer following a myeloablative treatment, such as radiation or chemotherapy.
Omisirge is administered as a single, patient-specific intravenous dose for blood cancer patients ≥12 years to hasten recovery of neutrophils. The treatment is composed of human donor stem cells from umbilical cord blood that are processed and cultured with nicotinamide.
The approval was based on data from a randomized, multicenter study comparing transplantation of Omisirge to transplantation of umbilical cord blood, in 125 patients with blood cancer (ages, 12 and 65 years). Among patients randomly assigned to Omisirge, 87 percent achieved neutrophil recovery with a median of 12 days following treatment versus 83 percent of participants randomly assigned to receive umbilical cord blood transplantation (median 22 days for neutrophil recovery). Within 100 days of transplantation, bacterial or fungal infections were seen in 39 percent of those receiving Omisirge versus 60 percent who received umbilical cord blood.
An overactive defense response may lead to increased blood clotting, disease severity, and death from COVID-19. A phenomenon called NETosis—in which infection-fighting cells emit a web-like substance to trap invading viruses—is part of an immune response that becomes increasingly hyperactive in people on ventilators and people who die from the disease.
A team led by University of Utah Health and PEEL Therapeutics, in collaboration with Cold Spring Harbor Laboratory and Weill Cornell Medicine, report the findings in a new study published in the journal, Blood.
“This study tells us about a potential mechanism for lung injury in COVID-19 that had not previously been recognized as a possible target for treatment,” says Elizabeth Middleton, M.D., the study’s first author and a critical care specialist at U of U Health.
The investigation also reports that a naturally occurring protein—originally found in umbilical cord blood—quiets this NET immune response in laboratory experiments, potentially opening new avenues for treatment.
It is estimated that up to 10% of people with COVID-19 become critically ill with respiratory distress. Causes of lung damage are a subject of intense investigation, and increasing evidence demonstrates that increased blood clotting may lead to complications caused by the disease.
Middleton, U of U Health physician-scientists and co-senior authors Christian Con Yost, M.D. and Joshua Schiffman, M.D., and colleagues, took a closer look to see if a specific immune response, called NETosis, could be involved.
Researchers followed 334 mother-child pairs, a subset of CCCEH’s ongoing urban birth cohort study in New York City, from prenatal life through adolescence. Researchers collected umbilical cord blood at birth and blood from the children at ages 2, 3, 5, 7 and 9. Over time, levels of BDE-47, the most frequently detected component of the pentaBDE mixture in humans, decreased by about 5 percent per year from 1998 to 2013. When examining only blood samples collected postnatally, researchers observed a 13 percent decrease per year between 2000 and 2013.
Children that were toddlers (ages 2-3) before the phase-out took effect in 2004-2005, had significantly higher levels of BDE-47 in their blood than children who turned age 2-3 following the phase out. Phase-out aside, 2-3 year olds had the highest concentrations of BDE-47 in their blood than at any other age, possibly because they spend more time on the floor and have more contact with dust at this age.
Though levels of these flame retardants are decreasing over time, investigators found PBDEs in every child blood sample. “These findings suggest that while pentaBDE levels have been decreasing since the phase-out, they continue to be detected in the blood of young children nearly 10 years following their removal from U.S. commerce,” says first author Whitney Cowell, PhD, pediatric environmental health research fellow at Mt. Sinai and former doctoral student from the Columbia Center for Children’s Environmental Health.
“These findings reinforce the decision to phase-out PBDEs from consumer products,” says senior author Julie Herbstman, PhD, associate professor of Environmental Health Sciences. “However, it’s important to remain vigilant. Since the phase-out of PBDEs, we have begun to detect other flame-retardant chemicals in children, which are likely being used as replacements.”
Collaboration among hospitals across the U.S. as part of a new research consortium is giving hope for the future of babies born with a rare congenital heart defect called hypoplastic left heart syndrome. A baby from Alabama named Ryals is one of the first to benefit from the consortium.
“He’s amazing,” Andrea Sexton says, beaming about her infant son. “He is the strongest little boy … If you were just looking at him, you’d never know anything was wrong.”
Sexton and her husband, Heath Sexton, never imagined that would even be possible for their son Ryals after he was diagnosed with a congenital heart defect while still in the womb.
“All of a sudden, to see the ultrasound tech blinking and staring at the monitor, and then getting a little frantic and rushing out to get the doctor, I knew something was wrong,” Heath Sexton says.
The diagnosis: hypoplastic left heart syndrome, or HLHS. Essentially, there was no left side of baby Ryals’ heart.
It’s a relatively rare condition. Only about a thousand children are born with HLHS each year in the U.S.
Coming home from the hospital we were wondering how is this going to change our lives … Obviously, the care of a child that possibly would need a lot of help, maybe a lot of long-term help,” Heath Sexton says. “And then, of course, in the back of your mind, obviously, you’ve read that a lot of children don’t make it past three months. And, so, just thinking about, you know, am I going to be prepared … for a funeral?”
The Sextons packed up and temporarily left their home in Alabama to move to Philadelphia. The plan was to place Ryals’ hopes of survival in the hands of a team of doctors at Children’s Hospital of Philadelphia (CHOP), one of the leading medical centers for HLHS surgeries and treatment.