Modern medicine relies on the statistical evaluation of new therapies versus placebo or the current standard of care (clinical trials) to identify the best treatments to alleviate or cure diseases. The approaches to understanding and treating disease have evolved significantly over time. Nowadays, the great basic-research advancements in our understanding of human genetics, physiology, biochemistry and cell biology in health and disease have opened the doors to many therapeutic strategies ranging from drugs and vaccines to the more cutting edge gene therapies and cell-based therapies.

What Are Cell-Based Therapies?

Cell-based therapies aim to deliver live whole cells for the treatment of a disease. This means using modified or unmodified cellular material obtained from the patient or from an immunologically similar donor. This approach is generally believed to be rather new, but it isn’t; blood transfusions date back to 1628, bone marrow transplants to 1968, and pancreatic islet transplantations to 1990.

What Is the Biggest Challenge In Cell-Based Therapies?

Similar to the case of organ transplants, the biggest challenge for cell-based therapies is avoiding tissue rejection. This is characterized by a strong reaction of the host’s immune system against the transfused cells/tissue/organ, and it is a serious and even deadly condition that can be mitigated by identifying donors with close histocompatibility to the recipient, or better yet by using the patient’s own cells/tissues. These approaches are termed allogeneic and autogeneic transplants, respectively.

The Advantages of Cell-Based Therapies

Our increased knowledge of immunology, cancer biology, developmental biology, stem cell biology, and our recent ability to efficiently manipulate human DNA to modulate gene expression has placed cell-based therapies at the forefront of modern medicine. Several cell-based therapies have come out of the intersection of these disciplines including the successful chimeric antigen receptor (CAR) T-cell therapy for cancer treatment, and the promising use of stem cells for treatment of many conditions including diabetes, cardiovascular disease, spinal cord injuries, liver disease, and also promising for accelerating wound healing.

The scientific, medical and investing excitement around these new approaches revolves around the fact that they all aim to provide autogeneic solutions for personalized medicine i.e. modifying patient-derived material to then treat the same patient. Such approaches also provide standard off-the-shelf solutions for use by any patient and solutions to regenerate and/or replace lost or injured tissue.

CAR T-Cell Therapy to Target Cancer Cells

In CAR T-cell therapy, T cells are isolated from the patient, manipulated to express a chimeric receptor engineered to detect a specific cancer marker (antigen), and then transplanted back into the patient. Cancer-antigen recognition by the CAR T-cells activates them and results in cancer cell destruction. This approach has proven to be very successful for treatment of hematologic cancers, but not so much for the treatment of solid tumors.

Stem Cells to Replace Damaged or Lost Tissue

A different approach involves the use of stem cells to regenerate damaged or lost tissue. In this case, patient’s stem cells are differentiated into mature cells (e.g. cardiac cells), and these cells are then transplanted into the patient. These approaches are very promising for the treatment of several conditions.

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Future Prospects

Cell-based therapies have come a long way from blood transfusions and bone marrow transplants. It is likely they will now begin to have an even more significant impact on human health as it is expected that many new successful therapies and cures will come directly or indirectly from studies using cell-based approaches. There remains, however, like any new groundbreaking technique of such precision, a number of hurdles to overcome before employing such therapies in modern medicine.

To stay up-to-date on all the new cell therapy publications please see: https://www.celltherapynews.com