U.S. National Institutes of Health has the goal to cure HIV and
sickle cell disease using gene therapies. The agency is going to
invest $100 million over the course of five years to get to that
the company, in its effort, is working with The Bill & Melinda
Gates Foundation, which is also investing $100 million into the
research. The goal is to create affordable therapies that anybody
can access, including people in developing countries where diseases
Director Dr. Francis Collins said it’s a bold goal but one they
chose to go big with. The idea is to create therapies that can be
tested in clinical U.S. and sub-Saharan Africa trials within seven to
of the 38 million people who have HIV are in developing countries,
with another two-thirds in Sub-Saharan Africa. Most cases of sickle
cell disease are seen in this region as well.
National Institute of Allergy and Infectious Diseases Director Dr.
Anthony Fauci said the agency has tried for decades to come up with
an HIV cure. While present antiretroviral therapy treatments are
effective in virus suppression, it’s not a cure, and people must
take the medicine every day.
worse, he said, millions of people have no access to the treatment.
scientists have been working to come up with gene-based HIV cures,
the methods tend to be expensive and unable to be replicated on a
large scale. Some therapies involve the removal of cells from a body
and re-infusing them using a costly, tedious intervention.
partnership will focus on coming up with cures using “in vivo”
methods, which can happen within the body. For instance, scientists
can remove the CCR5 receptor gene – something HIV uses to get
inside the cells. Another option is to take out the HIV proviral DNA
that’s replicated itself into the human genome and hides in the
body even after the treatment has been ongoing for years.
cell disease therapy could also rely on the in-vivo therapy to repair
the genetic mutation that leads to the disease. It would mean a
gene-based delivery system could potentially attack the mutation.
Health Organization Regional Director for Africa Matshidiso Rebecca
Moeti said a new way of thinking and a lifelong commitment is the
only surefire way to beat the diseases. She said she was happy to see
the type of collaboration necessary to address and beat the greatest
public health issues in Africa.
is much work needed to ensure safe, effective therapies.
said it would years before the dream to become a reality, which is
why the 10-year effort is so appealing.
In February 2019, the Trump Administration announced it was developing a 10-year plan to eliminate HIV and end the crisis altogether.