The 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 goal.
And, 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 are high.
NIH 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 10 years.
Most 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.
The 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.
Even worse, he said, millions of people have no access to the treatment.
While 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.
The 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.
Sickle 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.
World 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.
There is much work needed to ensure safe, effective therapies.
Collins 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.
Written by Mark Riegel, MD
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