Four for Krabbe's

4th Annual Krabbe’s Translational Research Network Meeting sponsored by The Legacy of Angels Foundation

Ft. Lauderdale, FL-- In March, one of The Legacy of Angels Signature Grants, the Krabbe Translational Research Network (KTRN) gathered for its 4th annual meeting to advance knowledge and translate research from the bench to the bedside, striving to accelerate access to better treatments and improve outcomes for those affected by Krabbe’s disease.

From far and wide this highly renowned group of internationally recognized experts in Krabbe’s disease united again at the beautiful beachside B Ocean Hotel in sunny Ft. Lauderdale, Florida to share the highlights and progress of each of their lab’s work and amazing progress. With more than 25 clinicians, researchers, consultants, advocates, and family members participating, it was another year showing great advances towards improved clinical treatments.

The goal of this latest meeting was two-fold; to share knowledge about Krabbe’s disease by increasing communication among researchers and advancing specific projects that have the potential to develop into new therapies for patients.

As we’ve always wanted to focus our philanthropy on the education and awareness of this disease and help fund research for improved treatments and ultimately a cure for Krabbe's disease, my wife Sue and I could not be happier with the growth and success of these meetings.

-- Paul Rosenau, Co-Founder of The Legacy of Angels Foundation.

Once again, Maria L. Escolar, M.D. the world-wide recognized premier physician who directs The Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at the University of Pittsburgh Medical Center and has created a Virtual Medical Home and Clinic for Children with Krabbe’s Disease located on the web at:, hosted the meeting.

Research Topics addressed this year included:

  • Natural progression Krabbe disease, unresolved questions
  • New conditioning approaches to UCBT
  • Canine GLD: benefits of the model and what we have learned so far
  • Challenges of bone marrow transplant and gene therapy in the monkey
  • Development and characterization of O-cells (DUOC-01 cells), novel cord blood-derived bridging cell therapy for patients with lysosomal storage disease
  • Glial progenitor cell-based strategies for treating pediatric disorders of myelin
  • Combination therapies for Krabbe disease – update
  • Translational approaches for Krabbe disease with AAV gene therapy
  • Lenti-based gene therapy for Krabbe disease evolved to address the pathology of and the current therapeutic regimen for Krabbe disease
  • Updates on gene therapy with AAVrh10 in the mouse and dog models of Krabbe disease
  • Effects of mutations and polymorphisms on trafficking and processing of GALC
  • Development of novel pharmacological strategies as potential treatments for Krabbe disease
  • KTRN Pilot: novel gene therapy approaches to treatment of Krabbe’s disease in the twitcher mouse – description of synergism
  • Global studies of neuropathogenic mechanisms active in Krabbe’s disease
  • Gene therapy from bench to bedside, the FDA approval process for steps leading to an IND
  • NIH initiatives and funding mechanisms for networks and translational research


Upon completion of the individual presentations, workgroups were strategically put together to identify the main challenges in the following topics:

  • Strategies for assessing current treatments (what knowledge-tools are needed to design efficient trials, availability of specimens, tissues, clinical gaps in improving patient care, newborn screening.
  • Translational obstacles for FDA approval (design of preclinical studies and clinical trials for rare diseases)
  • Challenges in preclinical studies (laboratory assays, treatment strategies, delivery methods, toxicology studies, outcome measures in animal studies)

At the conclusion of the meeting, all meeting participants agreed upon what the “Next Steps” for KTRN will be. Very exciting! The upcoming 2015 will bring us a step closer to our desired outcome after another year’s worth of hard work by The Legacy of Angels Foundation grantees and the additional scientists and clinician’s who work daily to help the children. 

We are gratified to see the progress being made, it is unprecedented!

-- Sue Rosenau, Co-Founder of The Legacy of Angels Foundation.