(Health): Novel Leukemia therapy aims to treat sick Children before is late

There is a great news for the health community as highly personalized treatments for aggressive pediatric leukemia are available and have proved effective for those who can wait for them. The question thereof is, what about those who can't wait?

About a decade ago, CAR T cell therapy changed the world of cancer treatment, offering a personalized approach to patients with blood cancers like leukemia. But getting that specialized treatment to patients is costly and time consuming. It can take up to two months to harvest a patient's T cells and reprogram them into cancer-fighters — a nonstarter for many young patients with aggressive cancer.

These patients "don't have months or years to live," said Dr. Mohamed Kharfan-Dabaja, director of Blood and Marrow Transplantation and Cellular Therapies at the Mayo Clinic in Florida. "They have weeks." Kharfan-Dabaja was not part of the new research.

A novel approach to CAR T (chimeric antigen receptor T cell) therapy aims to cut down that turnaround time significantly. Instead of reprogramming each patient's cells, researchers are testing the safety of using universal, or "off the shelf," CAR T cells from other patients, preprogrammed to fight cancer. These cells are then tweaked further using another gene-modifying technology — CRISPR — to ensure they are not rejected by the patient's own immune system.

Scientists at University College London in the United Kingdom tested the safety of the experimental approach in six children — mostly toddlers — with advanced leukemia. The research was published Wednesday in the journal Science Translational Medicine.

Conventional treatments, like chemotherapy, had already failed in the six children in the study, a phase 1 clinical trial. The goal was to determine whether the new approach was safe. The scientists will determine whether it is effective in future, larger studies.  

"These are very difficult children to treat," said Wassim Qasim, a study author and professor of cell and gene therapy at Great Ormond Street Hospital for Children in London.

The goal of the new treatment approach isn't necessarily curative; instead, it's to get patients into remission so that they are well enough for a bone marrow stem cell transplant.

The new approach is a "bridge to transplant," said Dr. Stephen Gottschalk, head of the Department of Bone Marrow Transplantation and Cellular Therapy at St. Jude Children's Research Hospital in Memphis. "If they're not in remission before transplant, their likelihood of relapse is probably 80%."

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