A doctor, a mutation and a potential cure for AIDS

An American living in Berlin suffered from both AIDS and leukemia.  Hoping to extend his life, the 42-year-old underwent a bone marrow transplant for leukemia, but instead he might have just won his battle against AIDS.

The virus has been undetected in the American’s body for nearly 600 days now.  He has stopped taking his retroantivirals, which in your average AIDS patient would cause the virus to stampede the body within days or weeks.

But that isn’t the case here, thanks to 39 year-old hemotologist Dr. Gero Hutter (left) and his bone marrow gambit.

The breakthrough appears to be that Dr. Hütter, a soft-spoken hematologist who isn’t an AIDS specialist, deliberately replaced the patient’s bone marrow cells with those from a donor who has a naturally occurring genetic mutation that renders his cells immune to almost all strains of HIV, the virus that causes AIDS.

The development suggests a potential new therapeutic avenue and comes as the search for a cure has adopted new urgency. Many fear that current AIDS drugs aren’t sustainable. Known as antiretrovirals, the medications prevent the virus from replicating but must be taken every day for life and are expensive for poor countries where the disease runs rampant. Last year, AIDS killed two million people; 2.7 million more contracted the virus, so treatment costs will keep ballooning.

Researchers are cautious to point out that this isn’t necessarily a cure all for those suffering from the disease because the virus could mutate and something this difficult and costly is almost impossible to bring to Africa, where the disease is so rampant estimates think nearly 30% of Sub-Sahara Africa is infected.

The good news that has the science community excited is for the potential breakthroughs in gene-therapy, an often vilified practice, but one that is responsible for curing the American’s infliction.  Researchers are hoping to replicate the results of the bone marrow transplant in pill form as soon as possible.  Essentially, they are attempting to isolate the gene mutation and recreate that.

Back in 1996, when “cocktails” of antiretroviral drugs were proved effective, some researchers proposed that all cells harboring HIV might eventually die off, leading to eradication of HIV from the body — in short, a cure. Those hopes foundered on the discovery that HIV, which integrates itself into a patient’s own DNA, hides in so-called “sanctuary cells,” where it lies dormant yet remains capable of reigniting an infection.

But that same year, researchers discovered that some gay men astonishingly remained uninfected despite engaging in very risky sex with as many as hundreds of partners. These men had inherited a mutation from both their parents that made them virtually immune to HIV.

The mutation prevents a molecule called CCR5 from appearing on the surface of cells. CCR5 acts as a kind of door for the virus. Since most HIV strains must bind to CCR5 to enter cells, the mutation bars the virus from entering. A new AIDS drug, Selzentry, made by Pfizer Inc., doesn’t attack HIV itself but works by blocking CCR5.

Bone marrow is where the immune-system cells are generated.

Regardless, this breakthrough is excellent news.

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