Christoph Soeder/dpa/image society via Getty I
Cheryl Meany, a high school teacher from Camillo, NY, was excited when she learned she was carrying twins in 2014. But his joy quickly turned to worry as doctors flagged various health concerns, including possible brain damage.
So it took a moment for the process when a man, a respiratory therapist, proposed that we end up enrolling newborn babies in an experimental study of diseases. The trial was about a protective treatment for RSV or respiratory syncytial virus, a common respiratory virus that can be very serious in children.
“He took me by surprise, like, ‘What are you even talking about?’ “I don’t even know what you’re asking me now,” he said.
That was in 2014, several years before the recent AV crisis overwhelmed hospitals across the country. But Meany was worried about his health back then after seeing some of his friends’ kids end up in the hospital. Up to 80,000 children under the age of 5 are admitted each year for AVG.
So he enrolled his daughters in a trial of a monoclonal antibody that works to prevent RSV-infection of the lower respiratory tract in infants. His decision helped advance one of the most promising treatments to protect infants from severe RSV in decades.
In January, drugmakers AstraZeneca and Sanofi announced that the US Food and Drug Administration would officially review their application to get the treatment – called nirsevimab – approved in the US, amid results from a mid-twin trial.
AstraZeneca said the results of its third phase of the trial showed that the single-dose treatment was almost 75% effective in preventing severe RSV infection in children during the period. The data was published in March 2012 New England Journal of Medicine.
Dr. William Schaffner, medical director of the National Foundation for Infectious Diseases, who was not involved in this research, said the results of nirsevimab significantly reduce the number of children who are hospitalized each year for AVG.
“The potential impact on supporting a healthy childhood for a very large proportion of children here in the United States — and even beyond — is potentially very large,” Schaffner said.
Form, passive immunization
The drug — a long-acting antibody injection — is intended for newborns or other infants against their first RSV infection, and for infants up to 24 months of age with their second RSV infection, according to a release from AstraZeneca.
Dr. Joseph Domachowske, a pediatric disease specialist at Upstate Medical Hospital in Syracuse, helped conduct the first phase of the nirsevimab study.
“RSV is the number one reason why babies and children are hospitalized not only in the US, but around the world,” he said.
He explained that the antibody treatment is not a vaccine, but science is “passive immunization.” Antibodies against CPV in children’s bodies work to protect against the virus if the child is exposed.
“It doesn’t trigger an immune response,” he said, nor does it cause the body to develop immune memory. “But the time until it wears off provides protection,” he said. A similar treatment plan has been used to help immunocompromised patients against COVID.
Domachowske, who also led the hospital’s covid-19 vaccine trial for kids, is waiting for the green light from regulators in time to make nirsevimab available next time in the case of RSV. It has already been tested in Europe.
A long way to effective treatment
When Meany’s daughters received the injection in January 2015, they were the first babies in the world to receive it, according to AstraZeneca.
Domachowske, a lowly family member, said giving the twins protection against the virus was a significant milestone after researchers spent years trying to find a treatment to prevent RSV. Back in the 1960s, another treatment, a vaccine candidate, was under study. But he made the children sick from CPUL – and two babies died from it.
“In reality, the injury has taken over half of the immune system,” Domachowske said.
Progress did not come until two decades later. In 1998, the FDA OK’d the monoclonal antibody for premature and high-risk babies. But Domachowske said that a variety of medical reasons at the time severely limited eligibility for this treatment, and, he said, the effectiveness was not great.
“We don’t have to give monthly,” said Domachowske. “And it is effective for preventing hospitalization, not for preventing infection.”
That’s where the research got stuck for years until 2014, when Domachowske attended a medical conference in Argentina. A detailed discussion of the great discovery that much of the research has focused on AVG sometimes leads to injury.
“Everybody’s sitting there with their mouths agape like, ‘This is why all our work hasn’t been done for a decade,'” Domachowske said. “That was serious.” And you can see the pharma people who were listening, taking notes, calling their colleagues, saying, “Stop, stop the work.”
Not long after, Meany’s daughters were injected with a better, longer-lasting monoclonal antibody that protects infants against RSV with a single shot.
The twin girls, Cassidy and Stella, are now 8 years old and like to compete in ninja battles – they run through obstacles that topple ladders, monkey bars and Bosu balls.
On average, he said the girls never had any complications from school and never showed signs of AVG. She is proud of the role medicine played in history.
“What a thing, and this is for kids everywhere, not kids here,” Meane said.
Domachowske said the girls got AVG at a later time after the effects of the treatment wore off. But because older children’s immune systems are stronger, the symptoms were not noticeable.
A welcome RSV prevention tool
Physicians and infectious disease experts undertake potential treatment approval.
Schaffner of the National Foundation for Infectious Diseases said that if it had already been approved in the US, nirsevimab would have helped control the high infections seen this season, one of the worst recent seasons for the disease.
“This recent attack would have been surprisingly blunt,” he said.
Dr. Vandana Madhavan, clinical director of pediatric diseases at Mass General for Children, said the monoclonal antibody is a significant breakthrough in the fight against CP.
“This is a huge step,” he said.
#oneshot #treatment #protect #babies #HIV #coming