The California Institute for Regenerative Medicine
(CIRM) has awarded City of Hope’s Xiuli Wang
, Ph.D., research professor in the Department of Hematology & Hematopoietic Cell Transplantation, $3.8 million to develop chimeric antigen receptor (CAR) T cells that can target and kill HIV-infected cells. The treatment will also use the cytomegalovirus vaccine to stimulate the immune system to proliferate and maintain the CAR T cells.
Our approach has the potential to change how HIV patients are treated,” Wang said. “Our goal is for a person to receive this therapy once, which would make it possible for that patient to stop the anti-retroviral drug therapy regime for the rest of their lives.”
Xiuli Wang, Ph.D.
Based on anti-retroviral drug therapy cost calculations, HIV CAR T therapy could provide cost savings to the health care system. Even though the cost of CAR T manufacturing is higher, the cost of the therapy per year could be significantly lower due to the single infusion mode of treatment.
CIRM’s Grants Working Group, an independent panel of experts who review all of CIRM’s applications for scientific merit, unanimously recommended Wang’s initiative for the full funding requested, saying that it showed “exceptional merit, necessary significance and potential for impact.”
In its review, the group said they “were very enthusiastic [about] this novel, albeit high risk, bispecific CAR-T cell therapy approach to curing or effectively controlling HIV infection. Reviewers thought that the rationale focusing on cytomegalovirus (CMV) specific T-cells and engineering them to target HIV-infected cells was based on sound scientific rationale and was supported by the preliminary data.”
Jeff Sheehy, a CIRM board member and patient advocate for HIV/AIDS, said there is a real need for a new approach to treating HIV patients. There is no cure for HIV, and only half of HIV patients adhere to anti-viral drug therapy in North America. Every year, 16,000 HIV individuals die in the U.S.
“With 37 million people worldwide living with HIV, including 1 million Americans, a single treatment that cures is desperately needed. An exciting feature of this approach is the way it is combined with the cytomegalovirus vaccine,” Sheehy said. “Making CAR T therapies safer and more efficient would not only help produce a new HIV treatment but would help with CAR T cancer therapies and could facilitate CAR T therapies for other diseases.”
Wang will use the CIRM funds to optimize the clinical manufacturing of the therapeutic product at City of Hope, complete the characterization of the efficacy and safety profiles of the therapeutic product, and submit the regulatory documentation to the Food and Drug Administration to initiate a clinical trial at City of Hope, hopefully within 18 months.
CIRM, California's stem cell institute, was created by the voters of California in 2004 when they passed Proposition 71, which authorized $3 billion in funding for stem cell research in the state. The institute funds stem cell research at institutions and companies throughout California with the goal of accelerating treatments to patients with unmet medical needs.
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