Medical scientific research has yielded incredible advancements in the treatment of HIV, which was once a death sentence.
Now persons living with HIV (PLWH), who take their antiretroviral therapy (ART) as prescribed and maintain an undetectable viral load, can live long healthy lives. Moreover, these individuals effectively have no risk of sexually transmitting HIV to a partner.
In recent years ART has efficaciously reduced HIV-infection to a chronic disease. However, ART requires life-long therapy that is expensive and has the risk of significant side effects. In addition, drug resistance is growing, requiring new and often more costly medications. These facts are compounded by the reality that life-long therapy can be challenging, and unfortunately stigma and discrimination associated with the disease continues to exist throughout the world.
Even though ART has improved the standard of care in HIV, a significant subset of PHLW are drug resistant due to suboptimal therapy prior to combination treatment and/or poor adherence. This subset is notably present in major cities in the United States in California, New York, Illinois, Florida and certain countries, such as Brazil and Thailand, where there was widespread use of mono- and duo-therapy in the 1980s and 1990s. Plus, substantial numbers of HIV patients fail to adhere to their ART regimen due to a variety of reasons, including adverse side effects, drug abuse, mental disorders, socioeconomic status, literacy, and social stigma. Such patients have ongoing viral replication, often with significant levels of plasma viremia, and, therefore, have severely compromised immune systems, with limited options and an inexorable pathway to significant morbidity and, ultimately, death.
Despite significant efforts, little progress has been made to ameliorate patients with unsuppressed plasma viremia. Indeed, the challenge is so significant that it has escaped solving by researchers for decades. The Seraph Research Institute team are investigating new approaches designed to treat people with uncontrolled HIV viremia.
It has been proven that gene-editing in autologous hematopoietic stem cells (HSC) – which reduces the expression of CCR5, a door HIV needs to enter, replicate within, and kill CD4+ T cells — combined with transplantation, can lead to an HIV cure. However, this approach is very expensive and it requires the ablation (almost total destruction) of the patient’s bone marrow and immune system using very high dose combination chemotherapy in order to achieve sufficient engraftment (uptake) of gene-modified HIV-resistant cells to achieve durable control of HIV.
Seraph Research Institute, Executive Director & Director of Research, Dr. Serhat Gumrukcu has pioneered a novel approach that has a potential to allow sufficient engraftment of the gene-modified HSC without the necessity of high and very toxic doses of chemotherapy or radiation. This approach, when combined with gene-modifications to confer HIV resistance, could eliminate the need for ART, effectively curing HIV.
Under the leadership of Dr. Gumrukcu, and with the collaboration with other scientists in the field, Seraph Research Institute is on the path to become one of the world’s leading institutions in fighting against HIV/AIDS through the pursuit of novel and innovative prevention, treatment, and cure strategies.
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