By Kambiz Shekdar, Ph.D.
One year before the coronavirus pandemic, Dr. Fauci attempted to direct the research prowess of the National Institutes of Health (NIH) toward curing what is still a largely incurable and festering global disease, HIV/AIDS, but with the explosion of COVID-19 that momentum was stopped abruptly. While right now the world is racing to inject vaccines to defeat the COVID killer, Dr. Fauci has renewed the $200 million call to find a cure for HIV/AIDS.
“This collaboration is an ambitious step forward, harnessing the most cutting-edge scientific tools and NIH’s sizable global HIV research infrastructure to one day deliver a cure and end the global HIV pandemic,” said Dr. Fauci when the $200 million joint initiative between NIH and the Gates Foundation was first announced in October 2019. “We aim to go big or go home,” added NIH Director Francis S. Collins, MD, PhD. Then COVID derailed the mother of all research plans. Until now.
Starting February 17, 2021, the National Institute of Allergy and Infectious Diseases (NIAID), the division of the NIH directed by Dr. Fauci, will accept proposals from the research community for what research to fund to cure AIDS. The renewed commitment was publicly announced on October 20, 2020. The announcement describes the most significant multi-year funding opportunity focused on curing HIV/AIDS to date, with applications due by March 17th of each of 2021, 2022, and 2023. The complete NIH grant solicitation may be accessed at this link: https://grants.nih.gov/grants/guide/rfa-files/RFA-AI-20-076.html.
The U.S. government paid Moderna—a company with an innovative approach but no prior medical products on the market—close to $1 billion to develop their COVID vaccine, and another $3 billion for 100 million doses. “Go for it,” encouraged Dr. Fauci. “Whatever it costs, don’t worry about it.” This kind of commitment is what is needed to eliminate disease. But is it sensible to fight HIV/AIDS in the midst of the coronavirus pandemic?
Make no mistake, HIV/AIDS and coronavirus/COVID-19 are different fronts in the same war. Coronavirus/COVID-19 is the burning hellfire, but HIV/AIDS continues to be our smoldering Vietnam—except that viruses accept no stalemates. Only by sharpening our biotechnological tools by actively and vigorously applying them to develop new vaccines and cures can we strengthen our scientific arsenal against viral outbreaks, including COVID-19.
Moreover, because the biotechnologies relevant to curing HIV/AIDS and sickle cell disease are often technologically related, the NIH/Gates cure collaboration includes funding to cure both. “This unprecedented collaboration focuses from the get-go on access, scalability, and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere, has the opportunity to be cured, not just those in high-income countries,” says NIH Director Francis S. Collins, MD, PhD. In this country, both HIV/AIDS and sickle cell disease disproportionately affect African Americans. COVID-19 demonstrated that the time to address our nation’s health inequity is now because #BLACKLIVESMATTER.
An estimated 37 million individuals are currently living with HIV/AIDS worldwide. There exist two individuals who have recently been cured of AIDS. Human Immunodeficiency Virus (HIV) binds to, infects, and destroys cells of the human immune system by attaching or “docking” to two proteins, primarily CD4 and CCR5, which are located on the cell surface. Left untreated, HIV deplete the immune system and causes Acquired Immune Deficiency Syndrome (AIDS).
A small number of individuals (less than 1% of the global population) are naturally resistant to HIV. This resistance arises because 32 units of DNA are deleted in these individuals’ CCR5 gene through a naturally-occurring mutation, resulting in a truncated variant of the gene, referred to as Delta-32 CCR5. The mutated version of CCR5 is not expressed on the cell surface, and is therefore unavailable for docking by the HIV virus. Individuals whose cells comprise Delta-32 CCR5 are thus naturally resistant to HIV.
To cure the first two patients of HIV/AIDS, stem cells from naturally HIV resistant donors were transplanted into the patients. This required a complex, multi-step process. The next steps relate to taking the science underlying these first two instances of a cure and developing a safe and effective method that may be used to cure all those in need, worldwide.
Two main pathways can be pursed to develop a broadly-applicable cure, ex vivo and in vivo pathways. Ex vivo cell therapy relates to taking a patient’s own stem cells out of their body and using molecular tools to perform ‘genetic surgery’ on the cells to render the cells resistant to HIV, followed by infusing the newly HIV resistant cells back into the same patient where they may grow and give rise to a new, HIV resistant immune system. In vivo cell therapy relates to performing the genetic surgery directly within patients, without taking their cells out as a first step, by delivering the molecular tools directly into the body.
There are multiple academic, biopharmaceutical and research foundations each developing their own promising biotechnologies to cure HIV/AIDS. At this time, funding and support from donors, foundations and the government are all welcome and necessary to accelerate the science and to develop the technology. For full disclosure, I am president and founder of Research Foundation to Cure AIDS, a 501(c)3 not-for-profit organization with its own biotechnology for in vivo and ex vivo cell therapy and I am applying for the NIH and Gates funding to develop a cure that is available to all those in need, regardless of ability to pay.
I am reminded of how the late AIDS activist Larry Kramer once described Dr. Fauci as “the only true and great hero” among government officials in the AIDS crisis. Dr. Fauci did not join the fight today. He first joined the NIAID as a clinical associate in 1968. In 1981, when he first heard of the new disease, he joined the earliest efforts to develop an HIV vaccine. Since 1984 he has led the nation’s research strategy to address HIV/AIDS in his role as NIAID director. We now have life-saving treatments and drugs. Next up, Cure.
Rockefeller University alumnus and biotech inventor Kambiz Shekdar, Ph.D., is the founder and president of Research Foundation to Cure AIDS. Contact Kambiz at firstname.lastname@example.org.