Genetically Engineered Cell Therapies, The Next Frontier in Regenerative Medicine

Abstract

Genetically engineered cell therapies (GECTs) represent a significant milestone in regenerative medicine that entails the application of living cells in conjunction with genetic modification to treat or prevent an extensive range of disease at their etiological axis. Cell and gene therapies are included in this category, and these have been of particular interest for the management of complex and otherwise refractory diseases such as cancer and inherited genetic disorders. One of the greatest achievements in the field was the 2017 FDA's approval of Kymriah, a CAR-T cell therapy for pediatric leukemia, that ushered the doors to a new age of personalized medicine. Development of GECTs has progressed since then with the emergence of gene-editing technologies such as CRISPR, which allow more precise and effective interventions.

While therapeutic applicability is enormous, emergence of GECTs also presents important challenges. Areas of greatest contention involve ethical concerns about informed consent, long-term safety, and the possibility of unanticipated genetic consequences. Further, the cost of such therapy’s limits availability to many, raising essential questions about healthcare equity and best methods of public versus private funding to distribute these services widely.

Regulatory bodies such as the U.S. Food and Drug Administration are also changing their infrastructure to better accommodate these new treatments, attempting to balance safety with innovation. With all involved in the healthcare environment collaborating to address these issues, the science is moving very quickly.

Collectively, GECTs hold game-changing promise to redefine disease treatment and improve patient care, at the forefront of medicine's future. Additional innovation and equitable use will be essential to realizing their full promise.