UVA has enlisted two top doctors, Dr. Lawrence G. Lum and Dr. Daniel “Trey” Lee, to bring new innovative therapy for challenging childhood cancers.

Dr. Lawrence G Lum

Dr. Lum is a pediatric immunologist and has developed a novel immunotherapy using immune cells in the blood (T Cells) to target and kill cancerous cells such as neuroblastomas and osteosarcomas.

Dr. Daniel “Trey” Lee

Dr. Lee is a pediatric oncologist and has developed immunotherapy treatments with “CAR T” cells to treat previously untreatable childhood leukemia.

The therapy consists of a very specific targeted treatment that focuses just on the actual tumor site versus attacking both healthy and unhealthy cells in the area.

The treatments of the past 40 years kill everything in the area, including the healthy cells. This treatment creates an immunity against the tumor, which could potentially and hopefully prevent future relapses. Dr. Lum’s work in immunotherapy has potential to destroy cancer in many areas of the body. It involves removing T-cells from patients, the cells that are already designed to destroy abnormal cells, and arming them with two antibodies that tell the T-cells to go to and kill the harmful cancer cells. The trial consists of weekly infusions, 2 times a week, for 8 weeks.

T cells are immune cells that our body uses to fight off viruses. Adoptive T cell cancer immunotherapy involves taking the patient’s T cells and retargeting them to kill specific cancer cells by linking together two antibodies on the surface of the cell to create a bispecific antibody. One end of antibody binds to T cells and the other end binds to the tumor cells. The T cells then become specific anti-tumor killing machines that can attack and kill multiple times. Outpatient infusions of the bispecific antibody targeted T cells (BATs) have been shows to vaccinate and boost the patient’s immune system against their own tumors.

Six patients with neuroblastoma and osteosarcoma have already been treated safely without severe side effects. One child cleared his bone marrow of neuroblastoma calls and another reduced his osteosarcoma so he could receive successful facial surgery.

This cancer therapy also hopes to reduce relapses.

Relapsed leukemia is responsible for the most deaths in children due to cancer, followed closely by brain tumors. For the first time, there is now an ability to harness a child’s own immune system to attack their cancer. Dr. Lee was one of a handful doctors who developed a way to teach T cells how to effectively kill tumor cells by genetically engineering them to make a new protein called a Chimeric Antigen Receptor (CAR). In one week, Dr. Lee is able to manufacture hundreds of millions of these CAR T cells that will attack a protein on the tumor cells, called CD19. After an infusion to the patient, the CD19 CAR T cells start to kill the tumor. Each time they kill, they secrete immune factors that recruit other parts of the immune system into the fight. Also, the CD19 CAR T cells will make many copies of the cell, creating daughter cells that also contain the CAR and are able to kill more tumor cells. This is the real strength in this therapy – infusing a million cancer-fighting CAR T cells will turn into hundreds of millions of CAR T cells in the body in just a few days.

In Dr. Lee’s first clinical trial at the NCI/NIH, 70 % of the patients went into remission. These were children who had no other hope for even short-term survival. In fact, with standard chemotherapy only about 10% will survive 2 years. However, over 60% of the children treated with CD19 CAR are alive at 2 years.

These impressive results have generated extensive enthusiasm in the cancer community. This innovative therapy will be available at UVA Children’s Hospital this year to help treat children who have previously failed treatment for leukemia, neuroblastoma, and other pediatric cancers.