How does research provide hope for sick children?

By developing innovative ways to provide treatment.

Perhaps the saddest thing anyone can tell parents of children afflicted by disease is that there is no hope. This is what physicians currently tell loved ones of children with Canavan disease – a fatal childhood neurological condition that usually causes death by age 10. That’s what they tell them today, but tomorrow?

Tomorrow, if research conducted by Dr. Paola Leone produces a cure, that will change.

Leone is a pioneer of gene therapy targeted to the central nervous system and has worked with her team to develop a genetically modified virus that expresses a healthy copy of the gene that is mutated in Canavan disease. The development of this gene therapy technology may save lives.

Leone and her team at the Cell and Gene Therapy Center at the Rowan University School of Osteopathic Medicine were the first to demonstrate the long-term safety and tolerability of this virus-based gene therapy technology in the human brain. This clinical neurological application was the first such study approved by the National Institutes of Health as an Investigational New Drug application.

Based on this pioneering work, Rowan University entered into a collaborative agreement in July 2015 with Bamboo Therapeutics, Inc., to further develop this technology for the treatment of Canavan disease and provide a foundation for application to other degenerative diseases of the brain. In 2016, biopharmaceutical giant Pfizer Inc. acquired the clinical-stage company based in Chapel Hill, North Carolina, for $150 million in a clear signal of intent with respect to the potential of this technology to treat currently intractable diseases of the brain.

Thanks to Leone’s pioneering work, the effort by Rowan University researchers may now be coupled with the resources of Pfizer to produce effective therapeutics. Now there is even greater hope for children with Canavan disease.

Contact Us: For more information about any of these initiatives, please contact:
Dr. Joe Cardona, cardona@rowan.edu, 856-256-4236.