Groundbreaking Gene Therapy Now at Goryeb Children's Hospital

Continuing its longstanding tradition of offering novel treatments to children with rare genetic disorders, Atlantic Health System’s Goryeb Children’s Hospital is now able to offer a newly approved gene therapy to treat spinal muscular atrophy (SMA), a condition that affects the nerves and spinal cord, leading to loss of muscle function, suppression of respiration and, often, early death.

Zolgensma, approved just recently by the U.S. Food and Drug Administration, is a one-time infusion that delivers a functional copy of the SMN1 gene.

In clinical trials, babies who received Zolgensma showed greater mobility, reached a number of motor-skills milestones and largely were able to avoid being placed on ventilators. Researchers are hopeful that Zolgensma offers long-lasting treatment for SMA.

Jahannaz Dastgir, DO, medical director of the Pediatric Neuromuscular Medicine Program at Goryeb Children's Hospital, has served as a primary investigator and investigator for a number of clinical trials for pediatric patients with SMA and other neurological and neuromuscular disorders and diseases.

“It’s such an exciting time to be a doctor treating pediatric neurological and neuromuscular disorders because of the incredible advancements in the last few years,” Dr. Dastgir said. “With these new treatments, we are able to literally change the course of these childrens’ lives and give them not only a better quality of life, but reduce the impact of or in some cases, nearly eliminate their disease.”

Because of its experience working with insurance and pharmaceutical companies on these novel and often very expensive medications, and the team’s comfort and experience infusing patients with these novel therapies, Atlantic Health System is able to offer newly approved medications sooner than other hospitals. Under Dr. Dastgir’s leadership, Goryeb Children’s Hospital was the first hospital in the state to administer nusinersen, another medication to treat SMA.

Using gene therapy, DNA containing a properly functioning gene is inserted into the patient via IV infusion. That infused gene then independently produces the missing protein in the patient’s cells.

“Imagine trying to build a brick wall, but the bricks you have available keep cracking and falling apart after you stack them,” Dr. Dastgir said. “You then realize that you need better bricks. Gene therapies are able to deliver what is essentially a new factory to your cells that independently produces the good brick you need. Now the wall can stand, or in the case of spinal muscular atrophy, the motor neurons are stable and will not continue to die off, improving mobility.”

Atlantic Health System has been at the forefront of genetic disorders research and treatment, offering clinical trials for a multitude of genetic diseases for the past eight years.

“The future of medicine is genomic, and we have been working to streamline the process for patients and bring gene therapies, genetic research and clinical trials to Goryeb Children’s Hospital,” said Darius Adams, MD, Medical Director, Jacobs Levy Genomic Medicine and Research Program, and Division Chief, Pediatric Genetics and Metabolism at Goryeb Children’s Hospital. “We are incredibly pleased to be able to offer them to our patients.”

Atlantic Health System also offers clinical trials for conditions including: phenylketonuria, infantile neuroaxonal dystrophy, fragile X Syndrome and lysosomal storage disorders such as Fabry disease, Gaucher disease, mucopolysaccharidoses, Pompe disease and Niemann-Pick Disease.

Specialty services for children with neurological and neuromuscular disorders and diseases at Goryeb Children’s Hospital include services for:

• Autism and development – Goryeb Autism and Development Center (GADC)
• Brain and spinal tumors – Goryeb Neuro-Oncology Program (GNOP)
• Concussion – Goryeb Concussion Program (GCP)
• Epilepsy – Goryeb Epilepsy Program (GEP)
• Headache and Migraine – Goryeb Headache Program (GHP)
• Muscle and Nerve diseases – Goryeb Neuromuscular Program (GNP)
• All general neurology problems

For more information on Zolgensma or other neurological and neuromuscular disorders and diseases, visit: https://www.atlantichealth.org/conditions-treatments/childrens-health/pediatric-neurology/pediatric-neuromuscular-program.html or call 973-971-5700.