Children’s National Health System is initiating novel treatments that harness the body’s own cells to successfully attack leukemia, lymphoma, and non-malignant diseases such as immune deficiencies and sickle cell disease.
Much of this work involves T-cells.
What are T-cells?
T-cells are white blood cells that circulate around our bodies, scanning for abnormalities and infections. The T-cells themselves look like a blob of interwoven bits of curled up spaghetti.
At Children’s National labs, Catherine Bollard, MD, MBChB, a blood and marrow transplant specialist and head of the Program for Cell Enhancement and Technologies for Immunotherapy (CETI), and her research team are training these T-cells to fight tumors and target cells and viruses that occur in leukemia, sickle cell disease, and other ailments.
Patients Treated at Children’s National with T-cell Therapies
Since CETI’s opening in November 2013, Dr. Bollard’s team has treated 15 patients with T-cell therapies, often focusing on life-threatening infections that affect patients with a weakened immune system, specifically patients who have had a bone marrow transplant.
“There have been overwhelming success rates,” Dr. Bollard said.
Patients treated include those who have had received bone marrow stem cell transplants for cancers such as leukemia and lymphoma, immune deficiencies, and sickle cell disease.
Making T-cells in the Lab
The team takes blood from patients, and in a state-of-the-art cell therapy good manufacturing practice (GMP) laboratory.
“We train T-cells to kill virus infected cells and cancer cells,” said Dr. Bollard. “Once the T-cells are ready, they are injected back into the patient, where they can travel throughout the body to seek out, find, and destroy the cancer or the virus.”
These T-cells are malleable, and so specific to each patient, Dr. Bollard calls it the “ultimate personalized therapy.” Some functions of T cells include:
- Killer T-cells are able to see in our body and scan for trouble. These cells hunt down infection.
- Helper T-cells orchestrate the immune response and play an important role in all immunity.
Each T-cell Tailored to the Patient
Each T-cell therapy is carried out differently, depending on the mechanism of immunotherapy. At Children’s National, Dr. Bollard’s team has pioneered a new approach in T-cell therapy using cord blood, the blood that remains in the blood vessels of the placenta and the portion of the umbilical cord that remains attached to the baby after birth. Cord blood is a life-saving donor source, and has a greater flexibility to be matched to a patient with fewer rejection symptoms, so patients without a familial match can undergo treatments.
However, virus infection is a major problem affecting patients after cord blood transplantation. Therefore, the CETI team has developed a novel treatment to prevent or treat life-threatening virus infection. They borrow a small fraction of cord blood T-cells and teach them to effectively target and kill virus infected cells and then inject these cord blood T-cells into the patients after their transplant.
“We are the first group to show that naïve cord blood derived immune system cells can be ‘trained’ in the laboratory to kill three viruses simultaneously,” said Dr. Bollard.
T-cell therapy targeting viruses has fewer toxic side effects, compared to many of the drug treatments these patients need. The other advantage of the T-cells is that they live forever and protect patients from infection or relapse for the rest of their lives, Dr. Bollard said.
Treating patients with virus killing T-cells after a bone marrow or cord blood transplant has resulted in a 93 percent success rate. Based on these highly successful results, the Children’s National CETI team is building their own bank of trained up T-cells to match and use whenever a vulnerable child is in need.
Learn more about the Cellular Therapy Program at Children’s National.