First Patient Treated with Federally Approved Lyfgenia™ for the Curative Gene Therapy Treatment of Sickle Cell Disease Leaves Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center | Newswise

Newswise — An 18-year-old patient has been discharged following the long process of undergoing the gene-modifying treatment at Joseph M. Sanzari Children’s Hospital, the pediatric facility at Hackensack Meridian Hackensack University Medical Center. The Joseph M. Sanzari patient is the first on the Hackensack University Medical Center campus to complete treatment outside of a clinical trial. 

The Children’s Hospital and John Theurer Cancer Center, also located on the Hackensack University Medical Center campus, were the first bluebird-designated Qualified Treatment Centers for the gene therapy treatment in New Jersey, following FDA approval of the treatment. 

The completion of the patient’s treatment for sickle cell is the second pediatric curative gene therapy treatment completed this year on the Hackensack University Medical Center campus. The Lyfgenia treatment comes on the heels of Joseph M. Sanzari’s treatment of a young girl who underwent treatment for another inherited blood disorder. The patient was treated with beta thalassemia using Zynteglo, which was approved by the FDA for the treatment of the inherited blood disorder at the same time as Lyfgenia was approved for sickle cell. 

The process of treating the first Sanzari patient with Lyfgenia began last year when the patient’s stem cells were harvested.  The blood cells were then sent to a lab where they were modified.  During the patient’s most recent monthlong hospital stay which began in April, the cells were returned to his body following the chemotherapy treatment that prepared the bone marrow for the re-engineered cells that will make it possible for his body to produce healthy red blood cells. 

Lyfgenia treatment is available to patients cared for at Sanzari and at K. Hovnanian Children’s Hospital at Hackensack Meridian Jersey Shore University Medical Center, the network’s other children’s hospital.

“The clinical team at Hackensack University Medical Center is committed to delivering clinical excellence and providing patient-centric care with transformative clinical treatments that is supported by evidence based research,” said Lisa Tank, M.D., president and chief hospital executive, Hackensack University Medical Center. 

“For decades, the campus of Hackensack University Medical Center has been a leader in the care of patients living with sickle cell, and in cutting edge research, providing curative stem cell transplantation first as bone marrow donor treatment, and now we’re able to offer gene therapy as the newest innovation in care,” said Mark Sparta, FACHE, president Northern Market, Hackensack Meridian Health.  “We are proud to continue to provide comprehensive medical management for patients living with this disease.” 

Lyfgenia is a one-time gene therapy approved last year by the U.S. Food and Drug Administration for patients ages 12 and older who experience vaso-occlusive events, also called VOEs. Vaso-occlusive events (VOEs) are painful complications associated with sickle cell disease. They occur when blood flow blockages deprive 

tissues and organs of oxygen and nutrients, which can lead to severe pain, hospitalization and organ damage. VOEs can also be called vaso-occlusive crises (VOCs) or sickle cell crises. 

Sickle cell is an inherited blood disorder affecting the red blood cells. When a patient has two copies of the sickle cell gene, their red blood cells are not shaped normally and are instead shaped like a crescent or sickle. Due to their sickle 

shape, the red blood cells may stick together and block blood flow to the blood vessels of different organs, tissues, and bones. Patients with sickle cell disease may experience serious medical complications, including repeated painful crises, strokes, infections, anemia, and blood clots. 

“Sickle cell is a disease that affects every organ in a patient’s body,” said Stacey Rifkin-Zenenberg, D.O., the pediatric hematologist/oncologist and section chief, Pain and Palliative Care, Joseph M. Sanzari Children’s Hospital, who has been a lead investigator of the largest clinical trials for curative treatments for sickle cell. “It affects tremendously not only the patient, but also the family.”

Lyfgenia treatment involves collecting patients’ stem cells and treating them with Lyfgenia before infusing them back into the patient via a stem cell transplant. Before the transplant, patients must undergo a round of chemotherapy to eliminate faulty blood stem cells. After the transplant, red blood cells generated from the engineered stem cells should produce the anti-sickling hemoglobin, limiting red blood cell sickling and future VOEs.

In addition to gene therapy treatment, which prior to the FDA approval of Lyfgenia was only available when enrolled in a clinical trial, Joseph M. Sanzari has provided curative treatment bone marrow stem cell transplants. Hackensack Meridian Children’s Health has the oldest and largest curative treatment program. It is a joint program in partnership with the Adult Stem Cell Transplantation and Cellular Therapy available at the John Theurer Cancer Center which is also located on the Hackensack University Medical Center campus.  The joint program – which serves adults and children as does the Lyfgenia gene therapy program – is accredited by Foundation for the Accreditation for Cellular Therapy (FACT). Earlier this month, Hackensack University Medical Center hosted Celebrate the Cure, a reunion attended by some of the more than 80 patients who have been cured of sickle cell disease, the majority as pediatric patients, during the past 20 years that cell transplantation and cellular therapy has been offered on the campus.  

Lyfgenia, previously known as LentiGlobin, received U.S. Food and Drug Administration (FDA) approval as a gene therapy treatment for sickle cell disease for adults and children aged 12 and older in December 2023. In 2019, Joseph M. Sanzari Children’s Hospital and the John Theurer Cancer Center, which is also on the Hackensack University Medical Center campus, became one of only a few U.S. hospitals, and the only in New Jersey to offer a national LentiGlobin gene therapy clinical trial. At an estimated $3.1 million, Lyfgenia is one of the highest prices ever for the treatment of a disease.