At band rehearsals, it’s not all play and no work. Music occasionally mixes with shop talk, and the jargon-filled conversations can seem indecipherable to outsiders. “We scientists do have a hard time speaking English sometimes,” O’Shea says. They use acronyms and terms that are obvious to them, like BCL11A erythroid enhancer, polygenic risk score, electroceuticals and TIL cells.
“How’s your research?” Lenardo asks fiddler Dr. Will Sears, 35, the band’s newest member, during a late-August rehearsal. Lenardo is doing the dinner dishes at his Potomac home before the band begins to play.
“It’s basically not happening,” Sears says.
“Eventually it happens,” Lenardo tells him.
Sears, a fellow in the infectious disease program, has been trying to kill whipworms, parasites that infect the large intestine, with drugs that might also be used in humans. “The worms I got [for the experiment] were already dead,” he says, “so I couldn’t kill them again.”
Lenardo, who’s been at NIH since 1989, encourages Sears not to give up. A doctor’s son, Lenardo chose research over traditional medicine. “We study the basic mechanism of how cells of the immune system respond to pathogens,” he says. His clinic studies children with genetic abnormalities of the immune system. The children he treats “get referred to NIH because nobody else knows what to do.”
The program’s patients are being treated at four centers in Turkey, where the disease he studies is most evident, and Lenardo has made many trips there to oversee their care. “We apply a contemporary genomic approach, down to what gene is involved, why is it abnormal and what that tells us about how the immune system functions,” he explains. “We have had children very, very sick who had a problem with their gastrointestinal tract, chronic diarrhea, losing proteins, a number of complications. It’s almost as if they were starving when they were trying to grow. Most die between 10 and 20 years old.”
The disease—CHAPLE, which stands for complement hyperactivation, angiopathic thrombosis and protein-losing enteropathy—was identified only two years ago, and more than a dozen children have since been treated for it. “We are just getting referrals for patients in the U.S. with the same genetic problem,” Lenardo says. “We will treat them, too. Any kid in the world can be treated.”
Through the FDA’s expanded access program, known as “compassionate use,” Lenardo’s branch has been able to employ the costly drug eculizumab, which is not yet approved by the FDA for this specific disease. “It’s absolutely miraculous,” Lenardo says. “These kids immediately get better. Their protein levels improved. They start to grow, mature, run around playing, where they were in bed, effectively dying.”
Without genomic analysis “there is no way we would’ve thought of using this,” Lenardo says. “It’s really very heartening… . Everyone’s life is centered around this very sick child. When they get better, the whole family dynamic changes.” Lenardo and his staff of 25 toil on the 11th floor of the NIH Clinical Center. When he’s not working, there’s the band, in which he gets to play (and sing) his favorite song, the Beatles’ “Eight Days a Week.”
On a deeper level, he says, “I find that music—both listening and performing—unleashes creativity and optimism, which helps my scientific work on these devastating diseases.”
It’s getting late at Lenardo’s, where the Affordable Rock ’n’ Roll Act is practicing for a gig at an Immunology Interest Group workshop at The National Conference Center in Leesburg, Virginia. Grayson thinks it’s time to play “Seagull,” one of two songs he’s written that the band performs.
“Love is the great fire escape, until it all goes up in flames,” Grayson sings. “Until the world is washed away. I’ll see you on that judgment day. …I am the early morning cat that was outside the hospital…”
“I wrote that song—like the music and lyrics entirely—in like [the] two-minute walk from my house to the hospital in Boston where I worked,” he tells the band. “There was a cat outside the hospital. That’s why that’s in there. Kind of random, right? But I got into the elevator to go up to my office, and they have that fire escape sign, with the person running down the stairs, and somebody wrote, love is the great fire escape.”
“So it’s not your song,” O’Shea declares. “You stole it!” There’s a chorus of laughter, and the band plays on.
Eugene L. Meyer, a former Washington Post reporter and editor, is a contributing editor for Bethesda Magazine and the author of Five for Freedom: The African American Soldiers in John Brown’s Army.