What Does it Mean for a Global Cure?
By Kambiz Shekdar, Ph.D.
Within the research community, the Holy Grail to cure AIDS had been to find new drugs to “Shock and Kill” the festering virus from its hiding places (“latent infections” and “viral reservoirs”). No such drugs have been developed, yet three persons have been cured using highly imaginative stem cell strategies. The most recent stem cell cure reported in February 2022 is beginning to cause the field to re-think and expand strategies to develop a global cure, including new initiatives by NIH.
Many readers of this newspaper will remember the height of the AIDS epidemic. New York City’s West Village, where this newspaper is based, has always been one of the epicenters of the AIDS epidemic in the United States. The neighborhood’s St. Vincent’s Hospital, now closed, once served as the center for compassionate care of dying patients. Today, an AIDS Memorial sits adjacent to where St. Vincent’s once stood, as though AIDS is history. In fact, the best way we can memorialize those lost to AIDS and impact the lives and health of future generations to come all around the world is to build a research incubator and hub to accelerate a global cure, right here in New York City.
A brief timeline of 40 years of AIDS: the modern HIV epidemic started in the early 1980’s. The first life-saving AIDS drug cocktail was introduced by Dr. David Ho in 1995. Global access to the drugs was initiated by President George W. Bush in 2003 via PEPFAR (President’s Emergency Plan for AIDS Relief). In 2012, the FDA approved the use of existing AIDS medications to prevent HIV infection. AIDS may have been tamed, but it did not go away; it has been smoldering ever since.
HIV-associated neurocognitive disorders (HAND) occur in some 50% of people living with HIV. HAND is not in the least bit addressable by any of the many existing AIDS medications available today, not even in people who may achieve undetectable viral levels using existing AIDS medications. The disease continues to ravish the poorest and most disenfranchised communities, in the U.S. this started with the gays and now includes black women, our prison populations, trans individuals and our inner cities.
40 million people around the world are living with HIV, making Planet Earth a massive petri dish for the emergence of new strains. COVID’s Delta, Omicron, and Deltacron variants illustrate how infectious agents can change and evade existing defenses. So far, drug makers have been able to develop stronger and newer drugs to new strains of HIV, but this is getting more challenging all the time. Moreover, young gay men in the U.S. have largely stopped using condoms, creating ideal conditions for the spread of any drug-resistant HIV.
With treatment and prevention drugs alone, we can maintain a smoldering HIV/AIDS epidemic, but we cannot end it. With a cure, a vaccine or both, we can end AIDS for good and wipe HIV off the face of the planet.
ENTER CURE: Timothy Ray Brown is the first person cured of AIDS. Mr. Brown recounted how he was cured at a symposium at Columbia University organized by Research Foundation to Cure AIDS. He said that he suffered from both leukemia and AIDS. His physician, Dr. Gero Hutter, had an idea. Dr. Hutter was not an HIV or AIDS specialist, but he recalled from his days in medical school how certain rare individuals are naturally resistant to HIV/AIDS. Mr. Brown needed a bone marrow stem cell transplant to cure his leukemia. What if, Dr. Hutter reasoned, the stem cell transplant was sourced from a donor who is selected from the group of individuals who is naturally resistant to HIV. Could the transplanted stem cells give rise to a new HIV-resistant immune system that would cure Mr. Brown of both AIDS and leukemia? Mr. Brown was a willing guinea pig. The answer to this pair’s experiment was a resounding “YES!”
Who was this leukemia doctor with claims of using stem cells to cure AIDS in 2007, when stem cell science was new and when almost all the experts in the field believed new drugs, not stem cells, were required to achieve a cure? Not even long-time AIDS activists popped any champagne bottles. Was this yet another case of drinking goats’ milk to cure AIDS? It took years for news of the innovative cure to catch.
DRIP BY MONUMENTAL DRIP, the three cures to date and the new knowledge gleaned from each success have chipped away at the calcified and crusty assumptions with the real-world data and results of what works to cure AIDS. Each of the cures and their contribution to the field of curing AIDS using innovative stem cell strategies are summarized below.
STEM CELL CURE #1, reported in 2007, used HIV resistant stem cells to cure AIDS. But because only one person was cured, it was formally possible that something else or extra about the biology or genetics of either the donor or the patient, or both, was at play. In addition, because the patient suffered from leukemia as well as AIDS, he underwent radiation to eliminate his cancerous immune system first. Also, the donor-derived stem cells that were transplanted into the patient caused a phenomenon known as GVHD (graft versus host disease) where the implanted cells attacked the recipient’s own cells. Any of these factors could have contributed to the cure of the patient in addition to the curative role of the HIV-resistant stem cell transplant.
STEM CELL CURE #2, reported in 2019, was achieved in much the same way as the first. Now two people were cured, not just one, providing clinical proof that the cure could be repeated and that the first case was not a fluke. Also, by this time, similar cures were attempted in additional patients who suffered with both leukemia and AIDS but using regular stem cell transplants and not HIV-resistant stem cells. All these attempted cures failed, suggesting that radiation to kill off a patient’s cancerous immune system without adding back HIV resistant stem cells is not sufficient to cure AIDS.
STEM CELL CURE #3, reported in 2022, makes a compelling trifecta. This third was achieved in a multiracial woman. With the first cure being that of a White man, the second that of a Latino male, and the third a multiracial woman, together, the diverse backgrounds of the three individuals cured to date dispels the possibility that any particular biology or genetics of the individuals involved was likely a key factor in achieving any of these cures. Moreover, GVHD did not take hold in patient #3, increasingly pointing to a central curative role being played by the naturally HIV resistant stem cells that were in common across all three cures to date.
Any time when the first patients are cured from a disease that has been lingering for decades, the primary goal of the research community must be to investigate all possible factors, especially all of the factors that were involved in the index cases, that could have contributed to the outcome. 15 years ago, Dr. Hutter showed us all a cure is possible. It took the NIH until the end of 2019 to make its first major announcement prioritizing the development of a global cure for AIDS. Regrettably, the COVID pandemic hit just six weeks later. Since then, I have seen one narrowly-defined call from the NIH for research proposals expressly focused on curing AIDS.
Much more can and must be done to make up for lost time and mobilize a cure. During the last 40 years of living with AIDS, the focus has been on drugs to treat and prevent HIV/AIDS. Now, the cure of three patients using stem cells points to the need to invigorate and add to the field. Room must be made to add new teams that bring novel and differentiated cellular biotechnologies needed to harness natural and curative biological resistance to HIV to develop a global cure.
For full disclosure, I am President and Founder of Research Foundation to Cure AIDS (RFTCA). RFTCA is not about treatment or prevention; we’re about the cure. Together with our collaboration partners at Columbia University Medical Center, New York Stem Cell Foundation, New York Blood Center and others, RFTCA has applied for NIH grant funding to advance innovate stem cell science originating from The Rockefeller University.
The imaginative and breathtaking results that Dr. Hutter demonstrated when he combined pieces of knowledge known to all—but synthesized by none before him—into a coherent sequence of events that cured AIDS for the first time ever, inspired me ever since I heard about his work. The recent news of the third patient cured of AIDS and how this cure specifically informs the field makes me more hopeful than ever about the prospects of developing a global cure.
Rockefeller University alumnus and biotech inventor Kambiz Shekdar, Ph.D., is the president of Research Foundation to Cure AIDS and Science & LGBTQ editor at WestView News. To support RFTCA, go to https://rftca.org/.