Joe Slay arranged a news conference at the Richmond Children’s Museum in November 1991 — back when the museum was housed in the old Navy Hill School near the Richmond Coliseum — and announced, “This is the first day of a war.”
Such a pronouncement might strike some as hyperbole but not to Slay, a Richmond public relations executive, whose 5-year-old son, Andrew, had been diagnosed with spinal muscular atrophy, a rare and often fatal genetic disease affecting muscle strength and movement.
This was the battle of his life.
There was no cure for spinal muscular atrophy, and scientists still simply were trying to understand it. So Slay and his wife, Martha, formed an organization they called Andrew’s Buddies to raise money to help researchers.
Their first goal was to raise $100,000 to purchase a DNA sequencer, so scientists could try to identify the faulty gene behind the disorder.
Back then, the notion of a treatment was only a dream.
But sometimes dreams come true.
Just before Christmas, the U.S. Food and Drug Administration approved the first drug to treat patients with spinal muscular atrophy.
“So much has happened,” Slay said recently. “I don’t think any of us gave ourselves the freedom to think in our lifetime … there would be any kind of treatment. It was always, ‘Well, maybe … ’
“But here we are. It’s miraculous.”
This is not the top of the mountain, but it is a lovely scenic overlook. The drug, Nusinersen, which will be sold by Biogen under the brand name Spinraza, is incredibly expensive — hundreds of thousands of dollars to cover the cost of multiple treatments per year.
But based on clinical trials when injected into the fluid surrounding the spinal cord, the drug has the potential of increasing strength in some patients and halting the progression of the disease in others.
Finally, families who have been longing desperately for a breakthrough have this drug to give them hope.
“There are discussions going on now with insurance companies,” Slay said. “I think there are good intentions all around, but it’ll take smart, good people to figure it out so that this can be brought to as many families as possible.
“There are issues to sort through, but it’s very exciting. It’s quite a moment.”
The FDA approval wasn’t a total surprise to the Slays, who have been aware of the clinical trials and knew federal approval was likely — though perhaps not this quickly, as it turned out the FDA had fast-tracked the process.
The Slays also were familiar with the drug itself since, in a way, they were there at the beginning.
Andrew’s Buddies provided funding for Dr. Adrian Krainer in 2000, which helped his lab begin work on spinal muscular atrophy.
Krainer, an authority on a cellular process called “RNA splicing” who is a professor at Cold Spring Harbor Laboratory in Long Island, N.Y., had attended a workshop in 1999 where he became interested in focusing on spinal muscular atrophy.
“However, to begin work specifically on (spinal muscular atrophy), I needed some dedicated funding, and that’s exactly what Andrew’s Buddies’ seed funding enabled,” Krainer wrote in an email. “They strongly encouraged me, and when I submitted an application they were extremely quick in arranging funding for it.”
That early work, said Krainer, who has become a regular at Andrew’s Buddies/FightSMA meetings, led to the development — in collaboration with Ionis Pharmaceuticals — of Nusinersen.
Connecting the dots between Andrew’s Buddies/FightSMA and the creation of the new drug is precisely what Dr. Robert Leshner did on the other side of the continent in San Diego when he heard the news about the FDA approval.
Now retired, Leshner is the pediatric neurologist who during his tenure at Virginia Commonwealth University diagnosed Andrew Slay before he was 2 years old and has remained close with the Slays and other families associated with FightSMA.
I called Leshner and asked how soon after hearing the news did he think of the Slays.
“Within a half-second,” he said.
“It’s testimony to how a handful of very dedicated people can really make an incredible impact,” said Leshner, who marveled at the way Andrew’s Buddies/FightSMA has been able to raise money, promote research and continue to move forward through all these years.
“I think the significance of that can’t be overestimated. The Slays did things for the right reasons: no concept of self-importance, just an incredible passion for bringing together the brightest and the best to come up with answers. All the answers aren’t here yet, but there’s been a giant step taken.”
Leshner said he considers himself blessed that he “was here for this moment to see a dream realized.”
Andrew’s Buddies/FightSMA remains an all-volunteer, parent-led nonprofit. In a quarter-century, it has raised millions of dollars and has financed research at dozens of universities and research institutions around the world.
Slay is quick to point out that many hands across the country have been involved in the effort: “Lots of families and lots of organizations can feel good about this, and we’re one of them,” he said.
He also hastens to say the FDA news unleashed a torrent of emotions, not all of them tending toward pure joy.
“We’ve lost a lot of kids,” he said.
I asked Slay if deep down, behind his brave face and throughout his family’s relentless efforts, he truly thought he would see a treatment for spinal muscular atrophy in his lifetime.
“I’ve never known quite what to think,” he said. “You’re always hopeful. But years go by, and you sort of adjust your sights. Sometimes it felt like it was going slowly, but it’s really been incredibly fast, all things considered.
“It’s a nice accomplishment that we’ve all gotten to this place.”
And then there’s this:
The Slays’ son, Andrew, now is 30, living in Richmond and managing an investment fund.
“He’s doing great, and he’s very keen on this,” Joe Slay said of the new drug treatment. “He’s interested in exploring it.”