hemotherapy has been the cornerstone of blood cancer treatment for many decades! Although they still remain essential therapy options, new treatments have lately contributed to a change in the way blood cancer patients, both children and adults, are treated. Additionally, immunotherapy, treatments that mobilize and enhance a patient's immune system's ability to combat cancer, has quickly emerged as a very effective tool for treating patients who don’t respond well to other therapies. 

Some children with cancer have benefitted from immune system-boosting medications in multiple ways, and these therapy effects can last for years in a limited number of patients.  CAR T cell treatment, a type of immunotherapy, has been proven especially useful for treating children with blood cancer! Despite being less popular than immune checkpoint inhibitors, doctors are now quite excited about the effects of CAR T cell therapy for pediatric leukaemia, and more research is being done constantly on this medical process. This blog will discuss everything you need to know about CAR T Cell therapy.

What Is CAR T-cell Therapy?

Chimeric antigen receptor (CAR) T-cell therapy is a form of immunotherapy for some blood cancers. It transforms your T cells, or T lymphocytes, into more effective cancer-fighting tools. In CAR T-cell treatment, medical professionals insert a new gene into your T cells, altering them to better identify and eliminate malignant cells. That way, CAR T cell therapy for children can sometimes fully cure blood cancer. In other less successful cases, it prolongs the life of patients with certain blood cancers. 

Children and adolescents with reasonably advanced B-cell acute lymphoblastic leukemia (ALL) can get treatment with CAR T-cell therapy; however, remember that other pediatric cancers cannot. For patients with very resistant ALL, it is an option worth trying, and to apply this treatment to other forms of childhood cancer, research is still being conducted.

How Does CAR T-cell Therapy Work?

A lymphocyte is a kind of white blood cell crucial in the battle against cancer and other infections. Different kinds of lymphocytes exist in the body, and one of them is T cells. T cells search for and eliminate aberrant cells throughout the body, and that’s why the body roduces T cells to combat a particular virus or disease when the malignancy is detected.

For CAR T-cell therapy, the patient's blood is used to gather T lymphocytes through a technique called Apheresis. Scientists then transform the T cells into CAR T-cells in the lab, and that’s the reason this is sometimes referred to as genetically modifying the T cell. Scientists do this by introducing a gene into the T cell that triggers the production of chimeric antigen receptors (CARs), which are proteins on the cell's surface, and a particular protein on the surface of the cancer cells is recognized and targeted by the CARs. 

After collection, scientists make these altered T cells develop and proliferate in the lab, and once sufficient of them have been created, they put the CAR T-cells back into the body. After identifying the cancer cells, the CAR T-cells will then start attacking them! Because of the modifications they make in the lab, CAR T-cells can remain in the body for extended periods and will remain capable of identifying and eliminating particular cancer cells.  

CAR T-cell Therapies Currently Being Used for Treating Blood Cancer

Six CAR T-cell treatments are now used to treat pediatric cancer, so here is a list of the already approved CAR T-cell therapy for pediatric cancer treatment: 

Tisagenlecleucel	Tisagenlecleucel is used to treat children and young adults with acute lymphoblastic leukemia.
Brexucabtagene autoleucel	Mantle cell lymphoma is treated with brexucabtagene autoleucel
Axicabtagene ciloleucel	Several B-cell lymphoma types can be treated with axicabtagene ciloleucel
Lisocabtagene maraleucel	For the treatment of diffuse large B-cell lymphoma, lisocabtagene maraleucel is utilized.
Idecabtagene vicleucel	Multiple myeloma is treated with idecabtageneviolencel and ciltacabtagene autoleucel

How Does CAR T-cell Therapy Work for Kids

The patient initially undergoes a comprehensive checkup that includes organ assessments and tests to ensure they will be healthy enough to endure the entire procedure.

1. Cell collection

An apheresis catheter is a specialized catheter that a pediatric surgeon will insert into the kid while the patient is sedated throughout this procedure. On the same day, T cells are taken from the patient's blood using the new catheter as part of a cell collection procedure. Pediatric patients usually handle the surgery rather well, with the exception of slight pain when the new catheter is inserted,but the catheter is removed immediately once collection is finished.

2. Cell manufacturing

The cells are frozen in a cell therapy facility before being sent to a specialist lab for genetic modification, and it takes around three weeks until they are returned to the hospital. 

3. Bridging therapy

Patients will get a tiny dose of chemotherapy to prevent leukemia while the T cells are being altered, and this process is known as bridging treatment. 

4. Infusion

The patient receives chemotherapy that depletes lymph nodes to create space for the new CAR T cells and enable their proliferation. The patient's blood is then injected with CAR T cells, a procedure that takes about five to thirty minutes on average.   

What Are the Benefits of CAR T-cell Therapy? 

There are many CAR T cell therapy benefits for children, but the short treatment period required for it is one of its main benefits! 

Moreover, it is delivered with a single infusion and may only require two weeks of hospital care at most. 

Remember, CAR T-cell therapy is now licensed for the treatment of individuals for whom a transplant is unlikely to be curative or for those who relapse after receiving one. In a recent study, researchers found that following CAR T-cell therapy, over eighty per cent of the children, experienced complete remission, and about sixty per cent of them continued to be cancer-free for a full year.

Final Remarks 

Certain types of leukaemia in children can be treated using CAR T-cell therapy! This therapy is a more recent option for treating some types of blood cancer in children, but this therapy is used only when previous therapies fail or the cancer returns (recurs)! 

Not all patients with blood cancer are the right candidates for this treatment, and doctors go through a full medical history and perform a lot of tests before deciding whether this therapy will work on the patient! 

Another thing is that CAR T-cell therapy doesn’t work on all forms of cancer, so only an expert can give you the answer to whether this therapy can work on a particular child, depending on his or her blood cancer type.

Visit a super-specialty hospital to discuss your options with some of the best cancer specialists!  

FAQs

1. How effective is CAR T cell therapy in children?

Studies show high success rates, with many children achieving remission after CAR T cell therapy, especially for difficult-to-treat blood cancers.

2. What are the risks or side effects of CAR T cell therapy?

Common side effects include fever, low blood pressure, and neurological symptoms, which are usually temporary and manageable with proper care.

3. How long does the CAR T cell therapy process take?

The process, from T cell collection to infusion, can take a few weeks, including time for lab modification and the recovery period post-treatment.

4. Does my child need to stay in the hospital for CAR T cell therapy?

Yes, children usually need to stay in the hospital for monitoring after the infusion, especially during the first 1-2 weeks, to manage potential side effects.

5. Is CAR T cell therapy a cure for blood cancer?

CAR T cell therapy has led to long-term remission in many cases, but its effectiveness varies by individual. Follow-up care is essential to monitor progress.

Citations

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Boettcher, M., Joechner, A., Li, Z., Yang, S. F., & Schlegel, P. (2022). Development of CAR T Cell therapy in Children—A Comprehensive Overview. Journal of Clinical Medicine, 11(8), 2158. https://doi.org/10.3390/jcm11082158

CAR T cells: Engineering immune cells to treat cancer. (2022, March 10). Cancer.gov. https://www.cancer.gov/about-cancer/treatment/research/car-t-cells#:~:text=Since%202017%2C%20six%20CAR%20T,%2C%20most%20recently%2C%20multiple%20myeloma.

Edwards, S. C., Hoevenaar, W. H. M., & Coffelt, S. B. (2020). Emerging immunotherapies for metastasis. British Journal of Cancer, 124(1), 37–48. https://doi.org/10.1038/s41416-020-01160-5

Kulczycka, M., Derlatka, K., Tasior, J., Lejman, M., & Zawitkowska, J. (2023). CAR T-Cell Therapy in Children with Solid Tumors. Journal of Clinical Medicine, 12(6), 2326. https://doi.org/10.3390/jcm12062326

Shalabi, H., Qin, H., Su, A., Yates, B., Wolters, P. L., Steinberg, S. M., Ligon, J. A., Silbert, S., Dédé, K., Benzaoui, M., Goldberg, S., Achar, S., Schneider, D., Shahani, S. A., Little, L., Foley, T., Molina, J. C., Panch, S., Mackall, C. L., . . . Shah, N. N. (2022). CD19/22 CAR T cells in children and young adults with B-ALL: phase 1 results and development of a novel bicistronic CAR. Blood, 140(5), 451–463. https://doi.org/10.1182/blood.2022015795