ALS in Children | Hackensack Meridian Health   

Neurologists and Pediatric Geneticists at Hackensack University Medical Center Publish Study on New Form of ALS in Children

Results Inspire Additional Genetic Research to Investigate Whether Disease Progression Can Be Slowed

Four clinicians from the Department of Neurology at Hackensack University Medical Center, and from the Division of Genetics, Department of Pediatrics at the Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, participated in research that led to the characterization of a new form of juvenile amyotrophic lateral sclerosis (ALS).

This newly characterized form of ALS begins in childhood, worsens more slowly than usual, and is linked to the SPTLC1 that manufactures a type of lipid called sphingolipids.

News of this research comes a year after the establishment of a interprofessional Amyotrophic Lateral Sclerosis (ALS) Association Affiliated Clinic Program which brings together all the specialists a patient and family may need in one clinic during one visit at Hackensack University Medical Center. The new ALS Affiliated Clinic is the second such program established in the Hackensack Meridian Health network; the other is located at Hackensack Meridian Jersey Shore University Medical Center.

Although additional research is needed, the international study — which included 11 patients — suggests that suppressing activity of the SPTLC1 gene may be an effective treatment strategy for this unique type of ALS. The study also provides new information that may help researchers understand and develop treatments for other forms of ALS.

The research paper titled “Childhood amyotrophic lateral sclerosis caused by excess sphingolipid synthesis” was published in Nature Medicine, a monthly peer-reviewed medical journal. Hackensack University Medical Center physicians involved in the research include:

  • Eric Mittelmann, M.D., neurologist, assistant professor of Neurology, Hackensack Meridian School of Medicine
  • Helio Fernando Pedro, M.D., pediatric geneticist, assistant professor of Pediatrics, Hackensack Meridian School of Medicine
  • Florian P. Thomas, M.D., Ph.D., co-director of the ALS Center, founding chair and professor, Department of Neurology at Hackensack University Medical Center and Hackensack Meridian School of Medicine
  • Aliza Zidell, genetic counselor

Learn More about other advancements and clinical innovations.

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