By Kambiz Shekdar, Ph.D.
Fifty percent of people living with HIV/AIDS have HIV-associated neurocognitive disorders (HAND). Today’s powerful anti-HIV drugs do not eliminate HIV-associated mental decline, even in individuals who achieve undetectable viral levels in their blood. On October 20, 2020, the NIH issued a call soliciting proposals to develop methods to cure HIV infection, including in infected human brain cell types. My organization, Research Foundation to Cure AIDS (RFTCA), is among those that submitted applications.
“The purpose of this funding opportunity announcement (FOA) is to support research on the development and validation of innovative strategies to deliver anti-HIV gene therapies efficiently to specific target cells [in the body],” states the solicitation issued jointly by the National Institute of Allergy and Infectious Disease (NIAID), the division commandeered by Dr. Fauci, and the National Institute of Mental Health (NIMH).
“NIMH will support research to develop and evaluate novel techniques to deliver anti-HIV gene therapies across the blood-brain barrier and effectively target CNS cells that harbor latent virus such as macrophages, microglia, and astrocytes with limited brain toxicity.” CNS refers to the central nervous system, which includes the brain, and “blood-brain barrier” refers to the impediment that blocks standard HIV medications from reaching this exclusive site of continuing mental disease.
The first major announcement by the NIH in support of efforts to find a cure for HIV was issued on October 23, 2019 when the NIH and Bill & Melinda Gates Foundation announced that $200 million in funding would be provided to focus on curing HIV/AIDS and sickle cell disease. This announcement came just six weeks before the coronavirus would alter priorities. Ironically, the first cases of the interfering coronavirus epidemic have, by now, been traced to December 1, 2019, which is World AIDS Day.
I contacted Tony Fauci and his team to share our preliminary data and results in response to the recent NIH solicitation. Within an hour, the program officer in charge got back to schedule a call. During that discussion she encouraged me to submit at least three separate grant applications for funding, including one in connection with its most recent solicitation, which I successfully submitted on behalf of RFTCA on March 17, 2021, the deadline for submissions.
Organizations like mine require research funding from the NIH to advance our work in the laboratory. The leading edge of medical philanthropy is often driven by patients and their families. However, severe persisting feelings of shame and stigma associated with HIV/AIDS continue to cause many people living with the disease to keep their HIV status a closely guarded secret, hampering the ability to raise awareness and support for new and urgent work to develop a cure. Additionally, almost all HIV/AIDS organizations, including beloved groups such as the Elton John AIDS Foundation and Broadway Cares, expressly exclude any funding for research from their mandates and missions. Broadway Cares provides care for people living with HIV/AIDS; “Broadway Cures” does not exist.
RFTCA’s particular approaches to developing a cure for HIV/AIDS—and we have several inter-related technologies— derive from work done in connection with a biotechnology, originating from a Nobel prize-winning laboratory at The Rockefeller University, termed Chromovert® Technology. Chromovert is a method of cellular engineering.
The application of the Chromovert process to cure HIV/AIDS would look like this: First, blood is drawn from a patient. Stem cells of the body’s immune system, called hematopoietic stem cells, are then isolated from the blood. Next, genetic surgery is performed on the stem cells to render them resistant to infection by HIV. The genetic surgery can be performed using any one of a variety of methods, including, for instance, those known as CRISPR, and Zinc Finger Nucleases (ZFNs) or oligonucleotide-mediated triplex recombination (oligos). The various methods have already generated promising results in the laboratory, however, they have side effects and/or too few optimally engineered cells for use in subsequent cell therapy to cure HIV/AIDS. Chromovert is a validated biotechnology that has the potential to detect and enrich even rare, optimally engineered cells, enabling the creation of sufficient quantities of therapeutic cells for use in cell therapy cures. During early work on this method, my colleagues and I uncovered several findings that allow multiple pathways to develop a cure.
Anyone interested in the details of the Chromovert cellular engineering method may find them in the first scientific publication on the technology, March 8, 2021, in the Springer Nature journal Biotechnology Letters, available at this link: https://link.springer.com/article/10.1007/s10529-021-03101-5. I invented Chromovert during my time as a graduate student, at The Rockefeller University, in the laboratory of the late Nobel laureate Professor Gunter Blobel, M.D., Ph.D., where the lightbulb that became Chromovert lit up. Gunter and I co-founded Chromocell Corporation with our business partner Christian Kopfli as a biotech spin-out of Rockefeller in order to pioneer the use of Chromovert in unrelated commercial applications which include a novel, non-addictive clinical-stage pain blocker fast-tracked by the FDA.
The first known cure of a patient with AIDS made me realize that the technology I invented might one day yield a broadly-applicable global cure. With pro bono support from the law firm Morrison & Foerster, LLP I created RFTCA as a 501(c)3 not-for-profit organization to develop a truly global and socially responsible cure, establish the best charitable use of the technology, and leverage it to shake complacency, give hope, and raise the funds that are needed to end AIDS, worldwide. In 2019, Chromocell granted a worldwide, royalty-free license to its biotechnology in the field of curing HIV/AIDS to RFTCA, including the right to research, develop, and implement a global cure for HIV/AIDS on a pro bono basis.
Unlike organizations which fund research and development, such as amfAR, RFTCA has its own cutting-edge biotechnologies. As a result, to my knowledge RFTCA is the only group that is developing a cure for HIV/AIDS on a pro bono basis such that it can be made available to all those in need, regardless of ability to pay. The recent support of organizations like the NIH and Gates Foundation for efforts to cure is a welcome and vitally needed ingredient to develop a worldwide, global cure for HIV/AIDS.
Rockefeller University alumnus and biotech inventor Kambiz Shekdar, Ph.D., is the founder and president of Research Foundation to Cure AIDS. Contact Kambiz at kambiz.shekdar@rftca.org.
