Genetically Altered Cells Proven Effective in Addressing Heart and Lung Disorders
In a groundbreaking study published in the Proceedings of the National Academy of Sciences, scientists at the University of California, San Francisco (UCSF) have developed a new approach to treating major diseases using specially engineered induced pluripotent stem cells, known as "HIP" cells. The study, with the DOI 10.1073/pnas.2022091118, is an example of "living therapeutics," a new pillar of medicine.
The research, led by Dr. Irina M. Conboy, Dr. Sonja Schrepfer (now at Sana Biotechnology, Inc.), and Dr. Lewis L. Lanier (both UCSF professors), subverts the immune response that is a major cause of transplant failure. HIP cells, the study's central component, were able to successfully treat major diseases while evading the immune system, without the need for immunosuppressive drugs.
The scientists tested the ability of these cells to treat three major diseases: Peripheral artery disease, chronic obstructive pulmonary disease from alpha1-antitrypsin deficiency, and heart failure. The study was conducted on mice with different tissue types, including diseased tissue and tissue with poor blood supply.
The researchers were able to alleviate peripheral artery disease in hindlimbs, prevent the development of lung disease, and alleviate heart failure in mice after myocardial infarction. They assessed the treatment's efficacy using standard parameters for human clinical trials focusing on outcome and organ function.
Dr. Schrepfer, a pioneer of immune engineering and co-inventor of the HIP cells, emphasizes the potential cost-effectiveness of the manufacturing of universal, high-quality cell therapeutics with HIP cells. However, she stresses that a careful and measured introduction into clinical trials will be crucial for the approach.
Dr. Lanier, co-senior author of the paper and co-leader of the Cancer Immunology & Immunotherapy Program at the UCSF Helen Diller Family Comprehensive Cancer Center, plans to explore the potential of HIP cells for treating other endocrine and cardiovascular conditions.
The strategy of immune engineering, as demonstrated in this study, comes with a reasonable price tag, according to Dr. Deuse, another key researcher involved in the study. He also plans to investigate the potential of HIP cells for treating other diseases.
This research marks a significant step forward in the field of gene-edited cellular therapeutics and offers hope for the treatment of various diseases in the future.
