Biography
Biography: Petz Lawrence
Abstract
In 2007 a patient who was infected with HIV and who also had acute myelogenous leukemia received a hematopoietic cell transplant using stem cells from an adult donor who had a homozygous CCR5 mutation (CCR5-/-). Persons with this mutation are known to be resistant to infection by HIV and, indeed, the patient was cured of his HIV as well as leukemia. This patient remains the only person to have been cured of HIV. This cure has not been repeated because the CCR5-/- mutation is very unusual and transplants using stem cells from adults require a very close HLA match between donor and patient. Cord blood transplants require significantly less stringent HLA matching, and thus it is more feasible to transplant HIV-infected patients with CCR5-/- donor cells from cord blood. Combined haploidentical and cord blood (haplo/cord) transplantation eliminates two problems associated with cord blood transplants, i.e., prolonged time to engraftment and cell dose needs. Inventories of hundreds of cryopreserved CCR5-/- cord blood units have been developed and are now available for transplantation, and one such transplant was performed in 2017. Further, an estimated 800,000 cryopreserved cord blood units exist worldwide which indicates that transplantation with CCR5-/- cord blood units is feasible for large numbers of patients. Cure of HIV is very important because even patients who are adequately treated with antiretroviral drugs for HIV are not protected from serious adverse effects of long-standing HIV infection, including the life-long stigma associated with the infection. Cure by transplantation is also economically beneficial because the estimated lifetime cost for persons who become infected with HIV at age 35 is ~$326,500. A rigidly held opinion by many is that patients with HIV should not be transplanted with intent to cure the infection unless they also have an underlying indication for a transplant such as leukemia. However, even in this antiretroviral era, thousands of patients die of HIV annually, and it is our opinion that the serious adverse effects of long-standing HIV are greater than the adverse effects of haplo/cord transplants of young HIV-infected patients who have no co-morbidities. The time has come to accelerate research on this topic of tremendous public health potential including transplantation of CCR5-defective cells, especially those derived from cord blood.