Joseph M. Sanzari Children’s Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in Adolescents and Adults

Clinical trial shows potential for some sickle cell patients to be their own stem cell donor

The Children’s Cancer Institute at the Joseph M. Sanzari Children’s Hospital at Hackensack Meridian Health Hackensack University Medical Center and the John Theurer Cancer Center have announced they are participating in a multicenter Phase I/II clinical trial of an investigational gene therapy from bluebird bio, Inc. This trial is specifically for adolescents and adults with severe sickle cell disease (SCD) who cannot be effectively treated using standard therapies such as antibiotics, vitamins, blood transfusions or any pain relieving medications. The study is evaluating the safety and effectiveness of LentiGlobin® for sickle cell disease, a gene therapy produced using the patient’s own modified stem cells to treat their sickle cell disease.

By using the patient’s own cells to produce functional hemoglobin that can prevent sickling of their red blood cells, LentiGlobin for SCD offers patients the opportunity to treat their disease without the need to have a matched bone marrow donor.  The John Theurer Cancer Center is one of a limited number of centers internationally, and the Joseph M. Sanzari Children’s Hospital is the only pediatric site in New Jersey, where the study, which is enrolling patients age 12-50, is taking place.

“Sickle cell affects 100,000 Americans. It affects one in every 365 African American births and one in every 16,000 Hispanic American births,” said Alfred P. Gillio, M.D., director, Children’s Cancer Institute and section chief, Pediatric Stem Cell Transplantation and Cellular Therapy Program, Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center. “This trial is for patients who have severe sickle cell disease and seek advanced treatment options but do not have a well-matched stem cell donor. Only 15% of sickle cell patients have a matched sibling donor and only 25 percent of patients have a matched unrelated volunteer donor.”

“Sickle cell affects every organ in a patient’s body,” said Stacey Rifkin-Zenenberg, D.O., FAAP, pediatric hematologist/oncologist, Children’s Cancer Institute, and section chief, Pain and Palliative Care, Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center. “This disease really has a tremendous effect not only on the patient, but also the family.”

Sickle cell disease is an inherited disease caused by a mutation in the beta-globin gene, resulting in abnormal hemoglobin and sickle-shaped red blood cells. Symptoms and complications of the disease include anemia, infections, stroke, poor quality of life and early death. To date, the only cure for sickle cell disease is receiving a stem cell transplant from a matched donor, but this is not a therapeutic option for many patients. Supportive care including hydroxyurea and blood transfusions can ameliorate symptoms of the disease. To date, without a marrow donor, there has been no alternate curative therapy. Life expectancy of a person with sickle cell disease is 20 to 40 years of age. In some cases, patients using disease modifying medications can live to 50 or 60.

“This therapy may be a major advance for sickle cell patients and so far, the results look very promising,” said Scott D. Rowley, M.D., FACP, hematologist, medical director, Stem Cell Transplantation and Cellular Therapy and medical director, BMT Cell Lab, John Theurer Cancer Center, Hackensack Meridian Health Hackensack University Medical Center, who is enrolling adult patients.  “This investigational treatment, which is a one-time therapy, may be an option for our patients who have no other treatment options.”

“The results from early clinical studies are encouraging,” said Dr. Gillio.  “With this treatment, the patient is their own donor and we are modifying their own cells to add copies of a functional beta globin gene.”

In the current study:

A patient's stem cells will be collected from his or her blood and sent to a lab. LentiGlobin for SCD is used to add functional copies of a modified form of the ?-globin gene (?A-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the ?A-T87Q-globin gene, they have the potential to make functional red blood cells.

The patient will receive chemotherapy to prepare the bone marrow for the modified hematopoietic (blood) stem cells (HSCs) that now carry the ?A-T87Q-globin gene.

The modified stem cells are then returned to the patient, so that they will repopulate the bone marrow and produce red blood cells that are healthy.

Participants will be followed for two years after treatment with LentiGlobin for SCD to assess the treatment's safety and effectiveness, based on blood tests and frequency of sickle cell disease symptoms and complications.

Sickle Cell Disease Treatment at Hackensack University Medical Center

Since 1988, Hackensack University Medical Center has been on the forefront of sickle cell disease care, providing curative stem cell transplantation as well as comprehensive medical management for patients living with this disease. The goal of the program is to offer the most current treatments options and access to the most up to date research protocols to the patients we serve. To learn more about this study, please contact the study research staff pedsresearch@hmhn.org, or call 551-996-5600.