SUNY Upstate Clinical Trial Leads to RSV Treatment
Courtesy of Upstate Medical University
Joseph Domachowske, MD is receiving national attention this year for his participation in developing a new medication now shown to be safe and effective at preventing infant respiratory syncytial virus, or RSV.
Many people became aware of RSV for the first time in the past year due to the explosion of cases in the US that crippled hospitals and infected a record number of patients. A story about his role in a 2015 study that lead to the development of a new prevention for RSV on Syracuse member station WAER was picked up by NPR’s All Things Considered. It’s the culmination of years of work by Domachowske, in collaboration with other lead clinical investigators all over the world, that just so happens to coincide with a heightened focus on the virus that remains the number one cause of hospitalization of kids under five years of age.
“Not everyone's familiar with it, but awareness has been increased given what's happened over the last year or so,” explains Domachowske. RSV was discovered in the 1950s, and he says there’s never been effective vaccines or treatments for the virus.
“I always had an interest because it's a huge unmet need,” says Domachowske. “We don't have treatment. We don't have prevention.” It’s been a focus of his clinical and research efforts since he became a researcher in the 1990s. Everything changed though, after a major breakthrough in 2014.
“In 2014, we found out that we were working on the right protein, but on the wrong conformation of the protein.” Domachowske describes being in the room when the discovery was presented at the international RSV conference in South Africa. “There were gasps in the room, people's jaws dropped to the floor. I could see the pharmaceutical representatives at this meeting calling their colleagues. ‘Stop working on what you're working on. We have to revamp everything.’ Since then, there's been this explosion of progress in the prevention of RSV.”
Domachowske then began thinking about changing how we approach preventing RSV infections in babies. Instead of creating an active vaccine, is there a way we could extend infants’ natural immunity they receive from their mothers that wears off after a few weeks to cover the first six months of life, when hospitalization is most likely?
“There are certain things that we use antibodies for that we sure would like to be able to extend their half-life, keep them around much longer,” explains Domachowske. Pharmaceutical companies were already working on combining a new immunology discovery that prolongs antibody half-life with a current, RSV antibody for only the highest-risk newborns that must be given every month to remain effective. “When I put that all together in my brain, I said, ‘we have to do it. That's the clinical trial I want to bring to Syracuse.’”
“The first three babies enrolled in that trial were enrolled right here on this desk,” Domachowske says. He didn’t want to bring premature newborns to a clinic with sick patients and risk exposure, so he administered the first doses in his office.
Phase after phase of trials in different groups of newborns continued to prove safe for babies. “It gives me chills just thinking about seeing that data come in. In every group of babies that's been studied, it works. It's at least 80% effective at preventing RSV hospitalizations or medically attended visits for pneumonia in every newborn group studied.”
Domachowske explains that the data on safety has also been phenomenal. “we've been studying it for seven years; more than 6000 infants have gotten it. It's very safe. It's as safe as giving a saltwater placebo injection. There's no indication that there are any reactions of concern.”
Domachowske credits the families willing to enroll their babies in the clinical trial with a big part of this medical breakthrough. “It's the only way it happens,” he says. He has some advice for people and families that may be considering taking part in a clinical trial. First- he says no matter what decision you make, participation or not, your decision will not impact medical care for you or your baby.
“Convincing families is not my role. My role is to just present the facts as I know them. I try to present what is unknown and what we're trying to do to meet this unmet need.” he says. “It's very difficult to recruit in phase one or even some phase two trials because there tends to be a limited amount of information to share with the families. It must be the right families doing it for the right reasons.”
Second, Domachowske says you should make sure you fully understand what you’re signing up for and know that your consent can be withdrawn at any time if you decide you want to stop taking part in the trial. “If the decision is ‘yes, we will participate’, that need not be a permanent decision. The option to withdraw from the study is always available, no questions asked. All you have to do is say ‘no more, I'm withdrawing consent’ and that's it. I like to make sure they know that they have that level of decision-making because it certainly empowers people that may be on the fence.”
As for the RSV treatment, the phase three clinical trial data were submitted to the FDA in September and Domachowske says they’re expecting a decision this year. Once it’s approved and available for use, he thinks the fact that the public is so much more familiar with RSV after this past season will help parents choose to treat their children. “Newborns get a hepatitis B vaccine, so I'm hoping that, during RSV season, they also go home with a dose of this new RSV prevention product as soon as it's approved and available.”
If you are interested in participating in a clinical trial or want more information about the clinical trial process, you can find more at https://www.upstateresearch.org/clinical-trials/.
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