Scientists at UC San Francisco have shown that gene-edited cellular therapeutics can be used to successfully treat cardiovascular and pulmonary diseases, potentially paving the way for developing less expensive cellular therapies to treat diseases for which there are currently few viable options.
The study, in mice, is the first in the emerging field of regenerative cell therapy to show that products from specially engineered induced pluripotent stem cells called “HIP” cells can successfully be employed to treat major diseases while evading the immune system. The findings subvert the immune response that is a major cause of transplant failure and poses a barrier to using engineered cells as therapy.
“We showed that immune-engineered HIP cells reliably evade immune rejection in mice with different tissue types, a situation similar to the transplantation between unrelated human individuals. This immune evasion was maintained in diseased tissue and tissue with poor blood supply without the use of any immunosuppressive drugs.” said Tobias Deuse, MD, the Julien I.E. Hoffman, M.D. Endowed Chair in Cardiac Surgery and a first author of the study.
Deuse’s research is an example of “living therapeutics,” an emerging pillar of medicine in which treatments are broadly defined as living human and microbial cells that are selected, modified, or engineered to treat or cure disease.
The study appears in PNAS (Proceedings of the National Academy of Science of the United States of America).
‘Universal’ Stem Cells Avoid Immune Detection
The prospects of generating specialized cells in a dish that can be transplanted into patients to treat various diseases are encouraging, the scientists report. However, the immune system would immediately recognize cells that were recovered from another individual and would reject the cells. Hence, some scientists believe that custom cell therapeutics need to be generated from scratch using a blood sample from every individual patient as starting material.
The research group at UCSF followed a different approach, using gene editing to create ‘universal stem cells’ (named HIP cells) that are not recognized by the immune system and can be used to make “universal cell therapeutics.”