Data from Pharmawand - Curated by EPG Health - Date added 05 September 2018

The effectiveness of CAR-T therapy, which uses genetically modified immune cells to treat acute lymphoblastic leukemia (ALL) in children and young adults has been published to the New England Journal of Medicine. A new deal for NHS UK will utilise the CAR-T treatment called Kymriah for patients who have advanced B-cell acute lymphoblastic leukaemia that is not responding to standard therapies.

The treatment will be made available to those deemed eligible - predominantly children and young adults, of whom there are around 400 in the UK diagnosed with acute lymphoblastic leukaemia - a type of blood cancer

Personalised cancer therapies, such as immunotherapy and CAR-T treatments are seen by many as offering hope to patients suffering from cancer.

Further treatments and breakthrough data can be expected from the European Society for Medical Oncology (ESMO) who hold their annual Congress next month in Munich (ESMO Congress 2018).

What is CAR-T therapy?

Chimeric antigen receptor therapy, or CAR-T for short,  is an innovative immunotherapy that uses a re-engineered version of the patient's immune cells as a living drug. The adoptive t cell immunotherapy involves taking the patient's T-cells, a type of white blood cell in the body's immune system, and sending them to a commercial laboratory. CAR-T cell engineering takes place, priming the cells to specifically recognize cancer cells, training them in the same way in which T-cells in the body are primed to recognize a virus during an infection. The activated T-cells are then returned to the patient's blood, where they attack the cancer cells. CAR-T becomes a living drug, with each dose being unique and specific to the patient.


IMAGE demonstrates the immunotherapy technique known as chimeric antigen receptor t cell therapy. Courtesy of UT SOUTHWESTERN MEDICAL CENTER.

Groundbreaking trial

Researchers have known about the potential of CAR-T therapy, but it is only in recent historic steps forward that they have been able to realise this potential in the most common childhood cancer, Acute lymphoblastic leukemia (ALL). It is hoped that future developments will lead to CAR-T immunotherapy being available for the treatment of multiple myeloma (MM).

What is acute lymphoblastic leukemia?

ALL is a cancer of the blood in which the bone marrow makes too many white blood cells. Typically it responds well to traditional chemotherapy, but in cases where the cancer does not respond to initial treatment, or in which the cancer returns, subsequent rounds of chemotherapy are effective less than half the time. CAR-T therapy has been suggested as a new treatment strategy to treat ALL.

Leading author of the study, Dr. Laetsch from University of Texas Southwestern Medical Center explained the importance of this breakthrough, which examined a total of 75 young patients from around the world, all of whom who had acute lymphoblastic leukemia (ALL) that was resistant to treatment.

"While most children with ALL respond well to chemotherapy, the patients in this trial were patients whose cancer had returned, and they desperately needed an alternative. It was gratifying to be part of this pioneering effort using a genetically modified version of the patient's T-cells to attack their cancer cells, and to see such positive results for so many patients."

High remission rate

Of the 75 patients who were treated, 81 percent went into remission following treatment, a high number for any treatment. Based on these promising early results, CAR-T therapy for ALL patients 25 years old and younger was ratified by the FDA.

Dr. Laetsch and his team are now providing CAR-T treatment for young ALL patients whose leukemia did not respond to therapy or whose cancer relapsed more than once. It is hoped that using t cells to fight cancer will shortly be a treatment option available for other types of blood and bone marrow cancers, such as multiple myeloma.

Multiple myeloma (MM) is also a bone marrow cancer that affects plasma cells and causes them grow out of control. Normal plasma cells work as part of the immune system, but those damaged by cancer develop into multiple painful bone tumors causing secondary problems such as anemia, kidney failure and recurrent infections.


It is very gratifying to see children who previously did not have any good options, respond so well to this new therapy and continue to do well.

Dr. Ted Laetsch, Assistant Professor of Pediatrics with the Simmons Cancer Center at UT Southwestern.

Content developed from Simmons Cancer Center researchers part of historic CAR-T breakthrough UT SOUTHWESTERN MEDICAL CENTER


This article was originally published on 7 February 2018. It was Updated on 5 September 2018.


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