In the
1980s, getting HIV was the equivalent of a death sentence, inevitably leading
to AIDS. But today, thanks to advancements in medicine, HIV carriers can live
healthy lives for decades. And in France, researchers have now used HIV
to save the lives of two young boys who suffered from a rare and fatal brain
disease.
The
disease is called adrenoleukodystrophy, or ALD. It attacks the myelin, the
tissue that insulates nerves in the central and peripheral nervous systems. One
in 18,000 people suffer from the disease, most commonly boys and men, according
to the Houston-based Stop ALD Foundation. In the case of boys, they often first show
behavioral problems, such as withdrawal and concentration lapses. Eventually,
symptoms such as blindness, seizures, loss of muscle control and dementia are
known to set in. Within two to five years, patients often face disability or death.
The movie, "Lorenzo’s Oil," was based on an ALD case in which parents played by
Nick Nolte and Susan Sarandon struggled to find a cure for their son.
A team
based at Saint-Vincent de Paul Hospital at Paris-Descartes University—research
director Nathalie Cartier and pediatric neurology professor Patrick Aubourg—has
worked on their breakthrough treatment for 16 years. In essence, they used HIV
as a vector to transfer corrective genes into the patients, penetrating into
cell nuclei to introduce healthier genetic material. Before doing this, they
harvested cells from the children’s bone marrow, transferred in a functional
version of the defective gene via a non-toxic version of an HIV vector, and then
reinjected cells to the bone marrow so they could migrate to the brain and
correct the disease.
“The two
treated patients are doing well: The progression of the disease stopped a few
month after the [treatment],” Cartier said, as news of the breakthrough was
announced. “Even if we must remain cautious, this analysis shows that we have
no specific reason to fear a harmful effect linked to the integration of the
... vector.”
Added
Aubourg: “This is the first time a serious brain disease is successfully
treated by gene therapy, giving a new impetus for the treatment of human
diseases by this type of approach.”
Two
years after the treatment, the team has announced that the neural damage has
been either halted or reversed. The two patients now attend school and lead
normal lives, according to published reports.
The team
plans to extend the trial in France and the United States. They feel this kind of therapy
can be applied to other diseases as well, such as thalassemia, sickle cell
anemia, Wiskott-Aldrich syndrome and metachromatic leukodystrophy.
