For a child, the brown-eyed boy has had more than his share of misfortune. He was born with HIV, the virus that causes AIDS. Then several months ago he developed a rare form of leukemia.
On Tuesday, doctors at the University of Minnesota are planning to try an extraordinary procedure that they believe may cure him of both illnesses.
So far, only one person on the planet has had this type of treatment. And six years later, he’s considered free of HIV.
The treatment injects the patient with cells that are resistant to AIDS. If it works — and there are a lot of ifs — it could do more than save a boy’s life, says Dr. John Wagner, a specialist in children’s cancer who is leading the medical team. It would show the world that the first patient wasn’t just a fluke, and set the stage for future research on a possible cure.
“Of course, our goal is to cure him of both diseases,” said Wagner. “We’ve been waiting for the opportunity to do it.”
The standard treatment for the boy’s type of leukemia — a bone marrow or cord blood transplant — was ordinarily considered too risky for HIV patients, Wagner said.
But that changed in 2007, when a man named Timothy Ray Brown, who had both HIV and leukemia, had an experimental treatment in Germany.
Brown, who became known as “the Berlin Patient,” got a bone-marrow transplant from a donor with a rare genetic mutation that, scientists knew, guards against HIV infection. When people are born with this genetic quirk, the AIDS virus may knock at the door of their cells, but can’t get in.
After his transplant, Brown stopped taking his AIDS medications for good, and tests show he’s still free of the virus. “Now I’m basically immune to HIV,” said Brown, 47, who now lives in Las Vegas. “I’m told by doctors that I can’t get HIV anymore.”
Dr. Timothy Schacker, an AIDS expert at the University of Minnesota, was one of the skeptics — until he studied Brown’s test results with a panel of experts. Schacker said the doctors “ultimately said, in our best opinion, this man is cured of HIV.”
So far, the feat has yet to be repeated successfully — in part because it’s hard to find a perfect bone-marrow match.
But Wagner and his team thought they had a better idea: to use cord blood cells, which come from umbilical cords and are far easier to match. The university has pioneered such transplants, performing 1,300 in the last 20 years. “If anyone is going to try this, it should be here,” Wagner said.
Wagner and his team were just waiting for the right patient when they got the call from the boy’s hometown doctor in another state.
‘I like Rubio’
To the naked eye, the young patient looks perfectly healthy. Curled up in a lounge chair, he was chewing gum and playing a video game when Dr. Michael Verneris, a transplant specialist, stopped in to check on him last week. An impish grin crossed the boy’s face, as two thin tubes poked out beneath his “How to be a Ninja” shirt, linking him to a cluster of IV bags. At the moment, all he wanted to talk about was basketball.
“So you’re like becoming a Minnesota fan?” asked Verneris.
“Yeah, I like Rubio,” replied the boy, whose family asked that he not be identified.
After chatting with the boy’s mother, Verneris asked if they had any questions. “I’ve got a question,” the boy piped up. “Who’s going to win the playoffs?”
Despite the boy’s demeanor, his doctors say they don’t sugarcoat the dangers.
“It’s a rough road,” says Verneris. The transplant is riskier than most types of heart or brain surgery, he said; one in five die from the procedure. The recovery can be brutal, too, leaving patients sick and fragile for months.
“When I tell a parent that,” says Verneris, “if they’re not crying at the end of the discussion, they didn’t hear me.”
Without a transplant, though, the boy’s odds are even worse, he said. Because this particular procedure is experimental, the university got approval from the Food and Drug Administration to try it.
Last week, the boy had chemotherapy and radiation to destroy his immune system and, hopefully, the cancer and HIV infection at the same time. On Tuesday, he’s scheduled to receive an infusion of cord blood cells, which should replace his own immune system. Doctors made sure that the new cells, from a newborn’s umbilical cord, had the rare genetic mutation that resists HIV.
After the transplant, he’ll be in isolation at the hospital, and won’t be able to go home for at least 100 days, Verneris said.
While checking for signs that the leukemia is gone, his doctors plan to keep him on anti-AIDS drugs — for awhile. At some point, says Schacker, “we’ll stop the [HIV] drugs and, on pins and needles, wait to see if the virus comes back.”
No one suggests this kind of transplant is the cure for HIV, Wagner said. But if it works, it will be a “proof of concept” that could lead to simpler, less toxic ways of curing the infection.
Bill Tiedemann, executive director of the Minnesota AIDS Project, cautions that the research is still in its early stages. But he calls the university’s work “an incredible opportunity.” The hope, he said, is that it will serve as “a steppingstone” to more research, “and that we will eventually reach what we all want, which is a cure for HIV.”
For Timothy Brown — the Berlin patient — it’s noteworthy that scientists are even talking about a cure. Just last month, doctors reported that a Mississippi baby girl was cured of HIV through intensive treatment after birth. And Brown has launched his own foundation to promote the cause. “Since my case came out in the news, people are now talking about it, and medical researchers are looking for a cure,” he said. If Tuesday’s procedure is a success, he added, “it would give people a lot of hope.”
In the meantime, Brown said he plans to reach out to the boy’s family. “I’m very hopeful that he will get through this very well.”
The boy’s physicians are cautiously optimistic. “We really are kind of going into the unknown,” says Verneris, “with eyes wide open.”