CAR T-Cell Therapy in Childhood Cancer

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Questions and Answers

What significant challenge did the first child infused with anti-CD19 CAR T cells face?

Infection risk
Resistance to treatment
Hypersensitivity reaction
Cytokine-release syndrome (correct)

What was a major outcome of the early phase trials of anti-CD19 CAR T cell therapy?

Long-term remission for many patients (correct)
Short-term benefits only
High rates of toxicity
Increased patient mortality

What was the impact of the success of CAR T cell therapy on regulatory approval?

Encouraged more restrictions on new treatments
Led to the withdrawal of existing therapies
Resulted in FDA approval of multiple CAR T cell products (correct)
Has not yet influenced regulatory decisions

How has the development of CAR T cell therapy impacted the broader field of anticancer treatments?

<p>Revolutionized the field of anticancer immunotherapy (D)</p> Signup and view all the answers

What distinguishes CAR T cell therapies from previous cancer treatments?

<p>Targeting specific antigens such as CD19 (A)</p> Signup and view all the answers

Flashcards

What is CAR T cell therapy?

CAR T cell therapy is a revolutionary treatment that uses a patient's own immune cells, specifically T cells, to fight cancer. These T cells are genetically engineered to express a chimeric antigen receptor (CAR) that targets a specific protein on the surface of cancer cells. This gives the T cells the ability to recognize and destroy cancer cells.

What is cytokine release syndrome (CRS)?

Cytokine release syndrome (CRS) is a potentially life-threatening complication associated with CAR T cell therapy. It occurs when the infused CAR T cells activate the immune system, leading to a massive release of cytokines, which are signaling molecules that can cause inflammation and other adverse effects.

What do anti-CD19 CAR T cells target?

Anti-CD19 CAR T cells target a specific protein called CD19, which is found on the surface of certain types of B cell cancers, including acute lymphoblastic leukemia (ALL) and diffuse large B cell lymphoma.

What are some examples of cancers where CAR T cell therapy has shown success?

The initial success of CAR T cell therapy was demonstrated in cases of follicular lymphoma and chronic lymphocytic leukemia (CLL), followed by remarkable results in a child with acute lymphoblastic leukemia (ALL).

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What are some clinical outcomes of CAR T cell therapy?

CAR T cell therapy has shown promise in achieving long-term remission and potentially cure in many patients with relapsed or refractory B cell malignancies, prompting the FDA approval of two distinct anti-CD19 CAR T cell products for the treatment of both B cell ALL and diffuse large B cell lymphoma.

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Study Notes

CAR T-Cell Therapy for Childhood Cancer

First child with acute lymphoblastic leukemia (ALL) treated with anti-CD19 CAR T-cells in 2012.
Experienced cytokine-release syndrome (CRS), a life-threatening toxicity.
Remarkable recovery and ongoing remission for over 6 years.
Demonstrated potential of CAR T-cell therapy for durable remissions.
Early-phase trials showed unprecedented success in treating relapsed/refractory CD19-expressing B-cell malignancies, leading to long-term remission/cure.
FDA approved two anti-CD19 CAR T-cell products for B-cell ALL and diffuse large B-cell lymphoma.
Over 100 global trials are underway for CAR T-cells targeting antigens beyond CD19.