The kernicterus Research Fund (KRF)

Dr. Steven Shapiro created The Kernicterus Research Fund (KRF) at the Medical College of Virginia Research Foundation, a 501(c)(3) organization.  The KRF transferred to Children’s Mercy when he relocated there in 2011. He transferred it to an independent subsidiary of PICK, another 501(c)(3) organization in 2021. Donors over the years include families and friends of children and adults with kernicterus. 

 

The KRF mission is to support research to better understand bilirubin neurotoxicity (BNTX) and kernicterus and to develop new treatments for kernicterus. Previously, funding has been used to determine where, when and how the brain is susceptible to BNTX, to identify which cells in the brain at the molecular level are responsible for the devastating dystonia of kernicterus, to understand how bilirubin affects the auditory nervous system during development, and to help fund preliminary studies of stem cells and neuroprogenitor cells that may eventually be used to treat and perhaps cure kernicteric dystonia.

 

Dr. Shapiro now hopes to use your tax-exempt donations to the Kernicterus Research Fund at PICK to support new and potentially impactful research to understand, treat and cure the most devastating effects of kernicterus, especially the dystonia and loss of voluntary movement characteristic of severe dystonic kernicterus in individuals who are typically cognitively intact.

 

Dr. Shapiro is planning to form a research committee composed of physicians, scientists and individuals with kernicterus or their families to send out requests for proposals. The committee will discuss and vote on grants for research. Our goal is to encourage young investigators with access to a top-notch research labs that will commit time and effort to researching kernicterus-related projects. For example, stem cell treatments, new medications to treat dystonia, and new ways to improve communication or voluntary movement or independent living. New testable ideas will be sought out. It is the hope that small start-up grants will lead to larger external grants from the NIH or other funding agencies.

 

Examples of some areas of research that a KRF committee might consider supporting include:

  • Stem cell therapy in animal models of kernicterus as a prelude to stem cell treatment in humans with disabling dystonia.

  • Discovery of rescue medicines or treatments that can “rescue” a newborn with extreme hyperbilirubinemia and ABR (acute kernicterus). Are treatments like hypothermia or EPO (erythropoietin) that are effective in improving outcome in babies with hypoxia-ischemia (low oxygen and blood flow) causing brain damage? Will these or other treatments work for bilirubin neurotoxicity?

  • The prevention of kernicterus in premature babies with new supplements e.g. choline.

  • The identification of new clinical syndromes of Kernicterus Spectrum Disorders (KSDs) including the validation of a KSD-Toolkit to bring some uniformity to the assessment of the likelihood, severity, and subtypes of KSDs.

  • The use of Brain Computer Interfaces (BCIs) to augment communication in individuals with dystonic motor and/or auditory prominent kernicterus.

  • Objective analysis of infant cry as a sign of acute bilirubin encephalopathy (kernicterus).

  • How to distinguish the dental enamel dysplasia of deciduous (baby) teeth from enamel dysplasia from other causes.

  • The Tooth Fairy project: determining whether a specific pattern of abnormal dental enamel exists in shed deciduous (baby) teeth that could be an objective marker for past exposure to excessive tissue unconjugated bilirubin.

  • Do auditory neuropathy and hearing loss from kernicterus improve with time? If so, how can we know who will improve and who will not? Is there any treatment that will increase the likelihood or amount of improvement?

  • Is there any way to predict in the newborn period who will develop dystonia, and how severe the dystonia will be? If so, are there treatments that will increase the likelihood or amount of improvement?

 

Other suggestions from individuals with kernicterus, their families, and donors will be welcome and encouraged.

 

A goal of the Kernicterus Research Fund is to seek applications for start-up grants in these or other areas that would benefit understanding and treatment of KSD, and award these to investigators who have the talent and opportunity to contribute with their research. Applications from young investigators who have the opportunity to work with established research laboratories or clinical research teams will be specifically sought in order to train the next generation of bilirubin neurotoxicity, kernicterus researchers.

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