The Utility of CAR T-Cell Therapy in Myelofibrosis

T cells are a natural part of the immune system designed to fight infections and cancer, but in conditions like blood cancer, they may not be able to eliminate malignant cells, according to Dr. Matthew Ku, who went on to add that this is an unmet need.

This unmet need is then combatted by researchers who have developed a method to genetically engineer T cells to express receptors that specifically target tumor cells, allowing the modified T cells to recognize and attack cancer more effectively. Ongoing research such as the 'Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)' is studying how clinical and genetic patterns in low-risk myelofibrosis could inform disease progression and identify patients who may benefit from future targeted therapies.

Ku, a clinical and laboratory hematologist, is a professor and lymphoma stream lead at St Vincent’s Hospital, where he is also the principal investigator on multiple important clinical trials. He is a member of the Australasian Lymphoma Alliance, the Australasian Leukemia & Lymphoma Group and the American Society of Hematology.

Transcript:

Can you explain what CAR T-cell therapy is and how it works to target disease?

We all have T cells in our blood. These T cells are part of our natural, native immune system; they are meant to protect us against infections and cancers. However, when a patient has a cancer like lymphoma, it means the T cells have not properly done their job of eliminating that malignancy. This new method is a way of weaponizing T cells.

The process involves genetically modifying these T cells to express receptors that specifically target tumor cells. This can be done in two ways: autologous, where T cells are harvested from the patient's own blood or body, or allogeneic, where T cells come from another person, a healthy donor. A new DNA construct is introduced into these T cells using a viral method. This construct basically goes inside the T cells and makes them produce receptors that target the tumor cells.

The receptors will differ depending on the type of tumor cells being targeted. For example, if it's a B-cell tumor, the targets could be CD19 or CD20, but other types of receptors have also been created for other cancers. These modified cells are then reintroduced back into the patient, and ideally, they will persist, grow, and attack the tumor cells.

Transcript has been edited for clarity and conciseness

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