Using CAR-T cell techniques to treat myelofibrosis

Myelofibrosis (MF) is a type of blood cancer that falls within the myeloproliferative neoplasm (MPN) category, a group of rare conditions that affect the blood. In MF, scar tissue to form in the bone marrow (the spongy material inside some of our bones). Key cells in the bone marrow called ‘stem cells’ undergo unusual alterations and multiply, taking over the bone marrow and causing it to fail. Sometimes, this can turn into another more aggressive type of blood cancer called acute myeloid leukaemia. MF can be caused by an alteration in specific genes, one of these is the ‘CALR’ gene. At present, the only treatment that offers a cure for younger people with MF is a stem cell transplant that involves intensive chemotherapy which can cause severe side effects and complications.

Dr Pule and his team believe that CAR-T cell therapy which has been used to treat other blood cancers such as leukaemia and lymphoma will be an effective treatment for people with MF caused by the CALR gene alteration. CAR-T cell therapy is a treatment which harnesses the immune system to destroy cancer cells. It involves taking immune cells from a patient and altering them in the lab to seek out and destroy cancer cells, before transferring them back to the patient. Dr Pule wants to use this technique to develop CAR-T cells that recognise the CALR gene alteration, so they destroy these harmful cells. He wants to understand whether this could be a viable treatment option for young people with MF.

This is early work, but if successful, Dr Pule and his team plan to test this further in young people with MF. Ultimately the hope is to be able to use this adapted CAR-T cell therapy as an effective and potentially less harmful treatment for young people with MF, giving them a better quality of life and chance of survival.