A man whose full-blown AIDS seems to have been eradicated after a bone marrow transplant has given new hope to Albany Medical Center researchers working on what might be an eventual cure for the virus.
Although it will be nearly impossible to duplicate the circumstances involving an unidentified American patient in Berlin, who had AIDS before receiving a bone marrow transplant to fight leukemia, local researchers said his recovery shows them they are on the right track.
They’re using an experimental medicine that the FDA just granted approval for this year. Watching the recovery of the Berlin patient lets them peer into a future where their medicine is successful, said Dr. Ralph Liporace, director of clinical research for the AIDS Treatment Program at Albany Medical Center.
Liporace’s team has spent the past four years studying a gluco-protein on normal cell walls that HIV needs in order to replicate itself.
When they began their research, they said they could stop HIV in its tracks if they could eliminate that gluco-protein from patients’ bodies.
Without the gluco-protein, HIV cannot expand, giving the patient’s immune system time to fight off the virus — although the war would continue for at least 17 years, Liporace said.
So far, they’ve developed an antibody that “turns off” the gluco-protein when injected into a patient’s body. The antibody only works for two to three weeks, but during that time, HIV can’t expand.
It worked in human trials, leading the FDA to approve it as a medicine late this year. Liporace has high hopes for the patients he is treating with it.
“We have a lot of patients doing well,” he said. “But you still wonder. You still want to know how long it will work.”
Now, thanks to that fluke bone marrow transplant in Berlin, he thinks he has his answer.
“It tells us this may be possible,” Liporace said. “We were able to see a model of it working. Eradicating CCR5 [the gluco-protein] appears to be an extremely effective treatment.”
Local patients aren’t celebrating yet.
“I think people are hopeful, but also wary,” said Daniel Butterworth, who runs Schenectady’s outreach center for HIV patients. “We as a group have seen this before, and then it doesn’t work for everyone or it doesn’t work generally. So there’s limited excitement — but the medical community is hopeful, and that makes us hopeful.”
He called the antibody treatment “really exciting” but dismissed the so-called cure in Berlin as useless to the rest of the HIV population.
“It sounds like it was a chain of really unusual circumstances,” Butterworth said.
Liporace also stressed that he can’t duplicate the Berlin treatment.
“Bone marrows have been tried in the past and failed miserably,” he said.
That’s because only the rarest form of bone marrow will work — and most patients’ bodies would reject it.
In the Berlin case, a doctor treating a cancer patient was also aware of studies into the fraction of humans whose cells do not have the gluco-protein.
The mutation, found in 1 percent to 2 percent of the world’s population, blocks all but one strain of HIV.
The patient was, luckily, genetically capable of accepting a wide range of bone marrow, so Dr. Gero Hutter went looking for a donor with the mutation, according to a report in The Wall Street Journal.
His hope, he said at a press conference, was to simply delay the ravages of HIV during the leukemia treatment. The patient had to stop taking AIDS drugs while being treated.
The donation slowed HIV down, all right — and then appeared to eradicate it. For 600 days, no sign of the virus has been found in the patient’s blood, brain tissue or rectal tissue.
Doctors warn that HIV could be hiding in nerve endings and other unreachable locations, unable to expand because of the mutated gluco-protein but still lurking in the shadows.
Still, it was a remarkable recovery for a man who had full-blown AIDS, and doctors said he is “functionally cured.”
Liporace said there’s little chance of repeating the cure.
First of all, there are few humans with the mutation — far fewer than the number of HIV patients — and most people reject bone marrow transplants unless the blood cells are very similar to theirs.
Doctors often find that the only acceptable donor is a close relative, and the donor must almost always be of the same ethnicity. However, the mutation is rarely found in people of African, Asian and South American descent.
Even if the perfect donor is found, Liporace isn’t confident of patients’ chances with a transplant. The procedure has a 30 percent fatality rate.
“People die during bone marrow transplants all the time. It’s a very tricky process,” he said. “It’s not the kind of treatment that would move out into the mainstream. But it adds another piece to the puzzle. It adds a lot of new information that’s useful.”
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