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Back to Section One | Back to Arts & Entertainment
posted Friday, December 10, 2010 - Volume 38 Issue 50
Stem cell transplant cured HIV, doctors say
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Stem cell transplant cured HIV, doctors say

by Mike Andrew - SGN Staff Writer

Doctors who performed a 2007 stem cell transplant hoping to cure a patient's leukemia now say the procedure also cured his HIV infection.

Once known only as the 'Berlin patient,' the individual is Tony Ray Brown, a U.S. citizen now living in Germany. His identity was revealed in a December interview in the German news magazine Stern.

Stern also spoke with Brown's doctor, Dr. Gero Hutter, who told the magazine he was glad he was 'in the right place, at the right time.'

'[F]or me, it is important to have overthrown the dogma that HIV can never be cured. Something like this is the greatest thing one can achieve in medical research,' Hutter said.

Brown developed acute myeloid leukemia and received successful initial treatment, but subsequently suffered a relapse that required a transplant of stem cells in 2007.

Post-transplant follow-ups included regular testing for HIV infection.

To the surprise of his doctors, Brown's HIV remained undetectable by both viral load testing (RNA) and tests for viral DNA within cells.

Brown's HIV antibody levels declined to the point that he showed no antibody reactivity to HIV core antibodies, and only very low levels of antibodies to HIV envelope proteins.

The case was first reported at the 2008 Conference on Retroviruses and Opportunistic Infections in Boston. Berlin doctors subsequently published a detailed case history in the New England Journal of Medicine in February 2009.

They have now published a follow-up report in the journal Blood, saying that based on the results of extensive tests, 'It is reasonable to conclude that cure of HIV infection has been achieved in this patient.'

Brown received stem cells from a donor who had natural resistance to HIV infection due to a genetic mutation in which the CCR5 co-receptor was absent from his cells.

People with this mutation are almost completely protected against HIV infection because the most common variety of HIV uses CCR5 as its 'docking station,' attaching to it in order to enter and infect CD4 cells.

According to a USC study published in July, this particular mutation is present in perhaps 10% of Caucasians.

Researchers said this case may suggest ways to develop a cure for HIV infection through genetically engineered stem cells.

The German researchers and San Francisco-based immunologist Professor Jay Levy said that the findings in Brown's case point to the importance of suppressing production of CCR5-bearing cells, either through transplants or gene therapy.

Scientists were sufficiently intrigued by Brown's case that they met in Berlin in 2009 to discuss how to identify stem cell donors with the CCR5-deficient mutation and expand the supply of stem cells from these donors.

Others have suggested that gene therapy procedures which transform stem cells - and all their descendents - into cells resistant to HIV infection may be a more practical option than looking for matching donors.

Several US research groups announced in October 2009 that they had secured funding to explore techniques for engineering and introducing CCR5-deficient stem cells.

Even if these approaches are successful, they may not result in a universally applicable cure, however.

For one thing, they will be expensive, so they very likely will be reserved for people with no remaining treatment options or with a cancer - like leukemia - requiring bone marrow or stem cell transplants.

As Tony Brown's case shows, the course of treatment is also long and harrowing. It is too extreme and too dangerous to be used as a routine treatment, Levy said in 2009.

Brown suffered two relapses and underwent two stem cell transplants. He experienced temporary liver problems and kidney failure.

He also suffered a serious neurological disorder that led to temporary blindness and memory problems.

Brown is still undergoing physiotherapy to help restore his coordination and gait, as well as speech therapy.

When Stern asked if it would have been better to live with HIV than to have beaten it in this way, Brown replied, 'Perhaps. Perhaps it would have been better, but I don't ask those sorts of questions anymore.'

According to Levy, less invasive options to alter CCR5 could be on the horizon within the next five years.

'It's definitely the wave of the future,' he said. 'As we continue to follow this one patient, we will learn a lot.'

One drug currently on the market - maraviroc, or Selzentry - blocks CCR5 co-receptors. It was first approved in 2007 and is used in combination with other antiretroviral drugs.

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