In this blog, we dive into the exciting new world of CAR-T therapy, an innovative form of cancer treatment that has the potential to transform lives. We’ll explore the basics of using the immune system to fight cancer, delve into what CAR-T therapy is all about, learn about its FDA-approved applications, discuss potential risks and benefits, and provide you with reliable sources for further information.

Before diving into the overview of CAR-T therapy, we start with an inspirational story about the first pediatric patient to receive CAR-T therapy that is living proof of its game-changing potential 10 years later!

From Relapse with No Treatment Options to Cancer Free 10 Years Later: A Remarkable Story of the First Pediatric Patient to Receive CAR-T Therapy

In 2012, Emily Whitehead was a 6-year-old girl with acute lymphoblastic leukemia (ALL). She had already undergone two rounds of chemotherapy, but her cancer was still aggressive. Her doctors told her parents that there were no other treatment options available.

However, Emily’s parents were determined to find a cure for their daughter. They learned about a new experimental treatment called CAR-T cell therapy. CAR-T therapy is a type of immunotherapy that uses a patient’s own immune cells to fight cancer.

Emily was the first pediatric patient to receive CAR-T cell therapy for ALL. The treatment was a success, and Emily went into remission. She has now been cancer free for 10 years, demonstrating a real-world example of the exciting potential of CAR-T therapy.

To read more about Emily’s amazing story, explore the links below.

• https://www.chop.edu/news/emily-whitehead-first-pediatric-patient-receive-car-t-cell-therapy-celebrates-cure-10-years
• https://emilywhiteheadfoundation.org/our-journey/
• https://www.statnews.com/2022/02/02/cart-cancer-therapy-leukemia-treatment/

Immuno-Oncology: A Foundation for Understanding CAR-T Therapy

Our immune system is a remarkable network of cells, tissues, and organs working together to protect us from infections and diseases. Similar to how the immune system recognizes and fights infections, it’s constantly surveilling the body to find and eliminate cancer. The immune system is one of our greatest defenses against cancer – and leveraging its power has driven an explosion of research and development resulting in an entire field: Immuno-Oncology.

Immuno-oncology, or IO for short, is a branch of medicine that focuses on leveraging our immune system’s natural defense mechanisms to combat cancer. However, cancer cells can sometimes evade detection by the immune system, allowing tumors to grow unchecked.

CAR-T therapy is a pioneering approach within immuno-oncology that empowers the immune system to recognize and eliminate cancer cells.

What is CAR-T Therapy?

CAR-T therapy, short for Chimeric Antigen Receptor T-cell therapy, is a groundbreaking immunotherapy designed to bolster our immune system’s ability to fight cancer. The therapy involves a complex process that starts with harvesting a patient’s own T cells, a type of white blood cell.

These T cells are then genetically modified in the laboratory to express chimeric antigen receptors (CARs) on their surface. These CARs act as “guided missiles” that specifically target and bind to proteins found on cancer cells, triggering an immune response to destroy the cancerous growths.

How does CAR-T therapy work?

CAR-T therapy starts with collecting a sample of the patient’s T cells. These cells are then sent to a laboratory, where they are genetically engineered to produce a chimeric antigen receptor (CAR). The CAR is a protein that binds to a specific antigen on the surface of cancer cells.

Once the T cells have been engineered, they are multiplied in the laboratory and then infused back into the patient. The CAR-T cells then go to work, finding and attacking cancer cells that express the targeted antigen.

Types of Cancer FDA Approved for CAR-T Therapy

As of the writing of this blog post, July 2023, the Food and Drug Administration (FDA) has approved six CAR T-cell therapies:

• Abecma® (idecabtagene vicleucel)
• Breyanzi® (lisocabtagene maraleucel)
• Kymriah® (tisagenlecleucel)
• Tecartus® (brexucabtagene autoleucel)
• Yescarta^TM (axicabtagene ciloleucel)
• Carvykti^TM (ciltacabtagene autoleucel)

By cancer type, they are approved to treat:

• Acute lymphoblastic leukemia (ALL): Kymriah is approved for patients up to age 25 with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. ALL ranks among the most prevalent and deadliest types of cancer affecting children and young adults.
• B-cell lymphoma: Yescarta, Kymriah and Breyanzi are approved for adults with relapsed or refractory large B-cell lymphoma.
• Follicular lymphoma (FL): Yescarta is approved for adults with relapsed or refractory follicular lymphoma.
• Mantle cell lymphoma: Tecartus is approved for adults with treatment-resistant or relapsed mantle cell lymphoma.
• Multiple myeloma: Abecma and Carvykti are approved for adults with relapsed or refractory multiple myeloma.

Risks and Benefits of CAR-T Therapy

While CAR-T therapy has shown impressive results, it is essential to understand both the potential risks and benefits before considering this treatment option. First and foremost, since CAR-T is a relatively new approach to treating cancer, patients haven’t been followed for an extensive period to fully understand all effects, especially long-term.

Potential Benefits

CAR-T therapy has demonstrated remarkable success, offering durable remissions and, in some cases, potential cures for patients who have exhausted other treatments.

Potential Risks

• Cytokine Release Syndrome (CRS):
  • This immune reaction can cause fever, flu-like symptoms, and, in severe cases, organ dysfunction. However, medical teams are skilled in managing CRS, and a therapy has recently been FDA-approved for the treatment of CRS.
  • Tocilizumab (Actemra®) is FDA-approved for the treatment of adults and pediatric patients 2 years of age and older with chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS).
• Neurologic Toxicity:
  • Some patients may experience confusion, seizures, or difficulty speaking. Early detection and treatment are crucial to address these effects.
• Other Known Side Effects
  • The listed risks and side effects in this blog are not exhaustive, but other known potential risks include Tumor Lysis Syndrome, Macrophage Activated Syndrome, On-Target/Off-Tumor Toxicity (killing of healthy cells expressing the target), Anaphylaxis/Allergic Reactions, and others.

Sources and Where to Find More Information

If you wish to learn more about CAR-T therapy, its applications, and future research, reliable sources include:

• gov: CAR T-cell therapy: https://www.cancer.gov/about-cancer/treatment/research/car-t-cells
• National Cancer Institute: Chimeric Antigen Receptor (CAR) T-Cell Therapy: https://www.cancer.gov/publications/dictionaries/cancer-terms/def/car-t-cell-therapy
• American Cancer Society: CAR T-cell Therapy: https://www.cancer.org/cancer/managing-cancer/treatment-types/immunotherapy/car-t-cell1.html
• OHSU Knight Cancer Center: CAR-T Cell Therapy for Cancer: https://www.ohsu.edu/knight-cancer-institute/car-t-cell-therapy-cancer
• Leukemia and Lymphoma Society: Chimeric Antigen Receptor (Car) T-Cell Therapy: https://www.lls.org/treatment/types-treatment/immunotherapy/chimeric-antigen-receptor-car-t-cell-therapy#FDA%20Approved%20Treatments

Conclusion

As we conclude this introductory journey into the world of CAR-T therapy, we hope you now have a better understanding of this cutting-edge immunotherapy. It represents a beacon of hope for patients facing challenging types of cancer like Emily Whitehead and has the potential to revolutionize cancer treatment in the years to come. Remember to consult with medical professionals to determine the best treatment options for individual cases, and never hesitate to seek reliable information from trusted sources.

Here to Serve has been assisting families on their cancer journeys for over twelve years. In addition to organizing care communities, go-fund-me pages, and other daily needs, they have an extensive knowledge of resources that may be beneficial to finding new and effective cancer treatments. If you or someone you know is in need of assistance, please contact Here to Serve.

About the Author

Dr. Ned Swanson has a unique background combining scientific, clinical, and industry knowledge. He is passionately dedicated to improving outcomes for patients across the entire patient journey, from therapeutic innovation to comprehensive social support.

This blog is for informational and educational purposes only. For specific medical needs, please contact the appropriate medical professional.

All photos in this article are from the Here to Serve photo archive.