By:

• Ellen Eldridge

LISTEN: Patients at Children's Healthcare of Atlanta can now receive cellular and gene therapy treatments for a host of diseases including cancer. GPB’s Ellen Eldridge has more.

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Children's Healthcare of Atlanta currently is planning or conducting cellular and gene therapy clinical trials in efforts to address a variety of diseases, including dilated cardiomyopathy, hypoplastic left heart syndrome, neuroblastoma, osteogenesis imperfecta, high-risk leukemia and lymphoma, neuroblastoma and sickle cell disease.

Credit: CHOA.org

Clinicians use gene editing to correct genetic defects to help treat disease.

Now, Children’s Health Care of Atlanta is conducting cellular and gene therapy clinical trials to address a variety of diseases, including dilated cardiomyopathy, hypoplastic left heart syndrome, neuroblastoma, osteogenesis imperfecta, high-risk leukemia and lymphoma, neuroblastoma and sickle cell disease.

Children’s Marcus Center for Cellular Therapy at Arthur M. Blank Hospital is one of the only labs in the country with this technology, said Dr. Doug Graham, the chief of the hospital’s Cancer and Blood Disorders Center.

There are two ways that you can think about how cells are used as drugs, he said. In one sense, cell therapy is a regenerative medicine approach that helps grow back and sustain healthy tissue, such as for patients who have a heart cardiac defect and need surgery.

One type of cell therapy clinical trial is for hypoplastic left heart syndrome, a type of disease in which the heart can't function, the blood flow isn't correct in the heart and affected infants need multiple surgeries over years-long period.

"We use cardiac stem cells to be able to decrease recovery time in these procedures so that you get better patient outcomes in children," Graham said.

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Another way in which cell therapy is used is to fight disease directly, attacking the core of what the disease is, Graham said.

A new kind of cell therapy that's been progressing over the last decade is called CAR-T cell therapy.

"It's almost like science fiction that this even works," Graham said.

In the past, if a child diagnosed with leukemia failed to respond to standard of care treatment, doctors would have been more or less out of options. Not so with CAR-T cell therapy that uses the patient's own cells.

"We train the T-cells in the laboratory to be able to identify leukemia cells and kill leukemia cells, and then we infuse these T-cells back into the patient," Graham said. "So, the patient now gets their own cells that are equipped to seek and find the leukemia and destroy the leukemic cells."

Graham said the next phase is to extend care beyond Atlanta and deliver treatments across the country.

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About the author

Author

Ellen Eldridge

Senior Health Care Reporter

Ellen Eldridge (she/her) is the senior health care reporter for Georgia Public Broadcasting.