Innovative New Leukemia Therapy Comes to Johns Hopkins All Children’s

Posted on Aug 28, 2018

Simulation-based Clinical System Testing (SbCST) for CAR-T therapy
Simulation-based Clinical System Testing (SbCST) for CAR-T therapy at Johns Hopkins All Children's Hospital

Johns Hopkins All Children’s Hospital is the first and so far only certified treatment center in Florida offering a cutting-edge cellular immunotherapy for advanced leukemia in children and young adults.

Scientists believe such immunotherapies—a new approach to medicine—hold promising possibilities for treating a host of diseases. Johns Hopkins All Children’s offers chimeric antigen receptor T-cell (CAR-T) therapy, which uses a patient’s own cells to fight acute lymphoblastic leukemia (ALL).

ALL affects about 3,100 young people in the United States each year, about 90 percent of whom respond well to standard treatments and become long-term survivors. The gene therapy is designed for those patients who do not respond to standard treatment or who relapse multiple times. In a clinical trial, 83 percent of these high-risk patients who received the CAR-T treatment were in complete remission in three months.

“CAR T-cell therapy has already shifted the paradigm of how we treat certain children with ALL and other cancers,” says Benjamin Oshrine, M.D., who works in the hospital’s Cancer & Blood Disorders Institute. “It is currently approved for use in children and young adults who’ve had multiple relapses of ALL or ALL that doesn’t respond to initial therapy. In the past, these patients would have very few, if any, treatment options that could realistically achieve a cure.”

CAR-T therapy involves removing the patient’s normal immune T-cells and then genetically reprogramming them to target a protein on the tumor cells’ surface. The altered cells are returned to the patient, where they multiply and attack a specific protein, CD19, which appears on the leukemia cell.

Johns Hopkins All Children’s, which offers one of the largest pediatric cancer and blood and marrow transplant programs in Florida, has specific expertise with blood disorders and is one of a limited number of pediatric hospitals across the country offering the treatment.

“We’re happy to have this promising new treatment available at Johns Hopkins All Children’s,” says Peter Shaw, M.D., deputy director of the hospital’s Cancer & Blood Disorders Institute. “This is an exciting development in the treatment of leukemia and all blood cancers. The use of the patient’s cells creates a living drug that specifically targets that patient’s cancer.”

The therapy, commercially marketed by Novartis and known as Kymriah, was developed at the University of Pennsylvania. The Johns Hopkins All Children’s clinical team received training in how to administer the treatment and how to manage potential side effects.

“We are excited about this advancement as it allows us to offer an effective treatment to children and young adults throughout Florida, the southeastern United States and beyond who had no other options and would be looking into phase I experimental trials or hospice care,” Shaw says. “To have this weapon in our arsenal against leukemia is amazing.”

Visit for more on the Johns Hopkins All Children’s Cancer & Blood Disorders Institute.

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