Author: Sarmishta Majumdar, Research Scholar, Institute of Chemical Technology, Mumbai, India
With the advances in science and medicine, the fundamental approach toward diagnosis and treatment of various diseases has improved significantly over the years. This has provided effective solutions to otherwise earlier difficult-to-treat diseases and disorders. The mainstream treatment of cancer has been chemotherapy, radiation therapy, or surgery. However, the existing therapies continue to remain ineffective in the case of advanced cancers or various disorders.
How can we find solutions to these incurable disorders? What if we could beat these destructive diseases by finding the root cause?
Cell therapy and Gene therapy are new fields of medicine that would create huge breakthroughs in healthcare.
Gene therapy: Gene therapy is a technique where defective genes are either modified or replaced with healthy ones to treat genetic disorders. This technique is used to treat genetic disorders like sickle cell anaemia and various cancers. Gene therapy is further categorized on the basis of cell types used i.e., Somatic cell gene therapy and Germline cell gene therapy. In somatic gene therapy, a mutant gene within somatic cells is replaced by a transgene and since the somatic cells are not involved in reproduction the genetic modification made won’t be inherited. On the other hand, the modifications made in germline cells would be passed on to the next generation. However, germline gene therapy is ethically prohibited.
Cell therapy: As the name suggests, cell therapy refers to the use of viable cells for the treatment of a disease or a disorder. The idea of transferring cells to a patient in case of blood transfusions and bone marrow transplants is well established and has been in use for a long time now. Non-stem cell-based therapy using fibroblasts that were reprogrammed to be converted into induced pluripotent stem cells (iPSC) has laid the foundation of regenerative medicine. With the emerging research, newer treatment options such as monoclonal antibodies and adoptive cell transfer are revolutionizing cancer immunotherapy.
CAR-T cell therapy:
A form of adoptive cell transfer called CAR-T cell therapy engineers a patient’s own T-lymphocytes to express chimeric antigen receptors (CAR) specific to a tumor antigen. Once injected back into the same patient’s body, the aim of these modified T cells is to recognize and attack the cancer cells. The T lymphocytes monitor the body for any foreign antigen or cells infected by pathogens using the receptors present on their surface and kill them. However, as it is challenging to differentiate cancer cells from normal cells, in many cases the cancer cells are able to get past these security defences. The engineering of T-lymphocytes takes advantage of the immune system’s intrinsic ability to seek out and destroy abnormal cells in the body (Miliotou et al, 2018).
The US FDA has approved a few mentioned CAR-T therapies to treat certain blood cancers that do not respond to chemotherapy (Newick et al, 2017).
- Breyanzi, for adults with some types of reoccurring B cell lymphoma.
- Tecartus, for adults with reoccurring mantle cell lymphoma.
- Kymriah, is for people under 25 years of age with leukaemia and adults with reoccurring large B cell lymphoma.
- Yescarta, for adults with certain forms of reoccurring B cell lymphoma.
Although CAR-T cell therapy has proved to be a promising immunotherapy for certain blood cancers, numerous studies are been currently conducted to investigate its use in other cancer types.
The treatment tends to be effective, but there are formidable challenges related to cost, and complex manufacturing procedure that needs mitigation (Mohanty et al, 2019). Eventually, the goal is to achieve high-quality cost-effective healthcare for patients.
Experts in the field of cell and gene therapy as well as the biopharmaceutical industry would be discussing various emerging research in the Leadership Conclave 2023 GOA.
Please visit www.biosimilarworkshop.com and join us for this exciting meeting of the scientific community!
Registration link:
https://bit.ly/Registration2023GOA
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References:
- Miliotou, A. N., & Papadopoulou, L. C. (2018). CAR T-cell therapy: a new era in cancer immunotherapy. Current pharmaceutical biotechnology, 19(1), 5–18.
- Mohanty, R., Chowdhury, C. R., Arega, S., Sen, P., Ganguly, P., & Ganguly, N. (2019). CAR T cell therapy: A new era for cancer treatment. Oncology reports, 42(6), 2183–2195.
- Newick, K., O’Brien, S., Moon, E., & Albelda, S. M. (2017). CAR T cell therapy for solid tumors. Annual review of medicine, 68, 139–152.
