Novel Leukema Therapy

The goal of novel leukemia therapy is to treat sick children before time runs out

CAR T cell therapy revolutionized cancer treatment a decade ago. It offers individualized care for patients suffering from blood cancers such as leukemia.

However, it is expensive and time-consuming to provide this specialized treatment. For many young patients with aggressive forms of cancer, it can take up to two months to collect their T cells and reprogram them as cancer fighters.

These patients “don’t have months or even years to live,” Dr. Mohamed Kharfan -Dabaja, Director of Blood and Marrow Transplantation at the Mayo Clinic in Florida, said. They have weeks. Khalfan-Dabaja wasn’t part of the new research.

This novel method of CAR T (chimeric Antigen Receptor T Cell) therapy is designed to significantly reduce turnaround times. Researchers are testing whether universal CAR T cells can be used to treat cancer. CRISPR is used to modify these cells further so that they don’t get rejected by the patient.

Researchers at University College London, the United Kingdom, tested the safety of the experimental method on six children with advanced leukemia. These were mostly toddlers. Science Translational Medicine published the research Wednesday.

The phase 1 clinical trial had shown that chemotherapy and other conventional treatments were failing in six of the children. It was intended to test whether the new approach was safe. It will be determined by scientists whether the new approach is safe in larger future studies.

“These children are very difficult to treat,” stated Wassim Qasim (a study author) and professor of cell therapy and gene therapy at Great Ormond Street Hospital for Children, London.

The new treatment is not intended to cure the disease. Instead, it’s designed to bring patients into remission and make them well enough to receive a bone marrow transplant.

Dr. Stephen Gottschalk is the head of the Department of Bone Marrow Transplantation at St. Jude Children’s Research Hospital Memphis. The new approach “bridges to transplant.” “If they are not in remission before transplant, their chances of relapse are probably 80%.”

Gottschalk, who wasn’t involved in the trial, described the new research as “exciting.”

Qasim stated that it can take around 12 days to modify the T cells harvested with

CRISPR is a genetic tool that can cut or paste bits of DNA. Then prepare them for use by preparing them for use. The wait time for most patients is shorter, as the patient only needs to freeze the T cells.

Qasim stated, “If we want these treatments to be available worldwide, then off the shelf will be the way to do it.”

This “off-the-shelf” method also guarantees sufficient T cells to treat the condition. Gottschalk stated that these young children are so tiny it is very difficult to collect cells.

None of the children who participated in the trial suffered from dangerous side effects. Cytokine storm is a side effect of CAR T therapy that can prove fatal. This is when the body’s immune response to the therapy goes haywire.

Four children died from cancer, not due to treatment or a Cytokine Storm. Qasim stated that two of the children entered remission after treatment and are still alive and well.

Dr. Kris Mahadeo is an associate professor at the University of Texas MD Anderson Cancer Center in Houston. She said that the initial results were positive.

Mahadeo, who wasn’t involved in the study, stated that “the idea is to get all these children to live a full life.” “That’s what we’re working towards.”

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