KCALSI Awards KC Scientists $50,000 Each to Advance Childhood Diseases Research

For Immediate Release: September 1, 2017

KANSAS CITY, MO – The Kansas City Area Life Sciences Institute (KCALSI) has awarded The Paul Patton Trust, Ted C. McCarter, William Evans, Jr., and Bank of America, N.A. Trustees (Paul Patton Trust Grant) to three recipients to support their research focused on genetic diseases affecting children. The areas being explored by this year’s awardees include pediatric narcolepsy, muscle deterioration and respiratory distress, and chronic kidney disease. Each researcher will receive $50,000 for one year as recipients of the 2017 Paul Patton Trust Grant.

“We are proud to work with the Paul Patton Charitable Trust and Bank of America to find and fund crucial pediatric research for the past ten years,” said Dr. Wayne Carter, President and Chief Executive Officer of KCALSI. “The funding provides an important stepping stone to finding cures for genetic diseases that primarily impact children. With these research grants, the recipients have a much better opportunity at competing for larger federal grants to continue research programs.”

Below are this year’s grant recipients and a brief description of their research programs.

Genetic Cause and Treatment Suggestions for Pediatric Narcolepsy
A team of investigators at Children’s Mercy and the University of Kansas Medical Center (KUMC), led by Dr. Sarah Soden, will assess narcolepsy in children through the discovery of new genetic risk factors and identification of behaviors. The diagnosis of narcolepsy is often delayed due to incorrect judgment of symptoms and limited treatments for symptom management.

“Narcolepsy is a rare sleep disorder that can arise in childhood for reasons that aren’t fully understood. This study is designed to discover genetic risk, through genomic sequencing of families affected by narcolepsy,” says Dr. Soden. “We will collaborate with the Institute for Advancing Medical Innovation and the Transgenic and Gene Targeting Facility at KUMC to identify a strategy for developing research tools targeting candidate genes or biological pathways contributing to narcolepsy, that will guide future drug and biomarker discovery efforts.”

Understanding How Gene Mutations Can Result in Muscle Atrophy
Dr. Monique Lorson from the University of Missouri will focus her research on two childhood diseases, Spinal Muscular Atrophy with Respiratory Distress 1 (SMARD1) and Charcot Marie Tooth Type 2 (CMT2), both resulting from gene mutations.

SMARD1 is a fatal disease where children typically die before the age of two, due to paralysis of the diaphragm muscles. CMT2, while not fatal, is a progressive disease that significantly impacts the quality of life of patients through muscle deterioration and weakness in arms and legs. Currently there are no treatments or cures for either SMARD1 or CMT2.

“Our laboratory is interested in developing an animal model based on identified gene mutations,” said Dr. Monique Lorson. “By understanding the similarities and differences between these mutations, we hope to better understand the biology of SMARD1 and CMT2 disease development, motor neuron degeneration and muscle atrophy, and provide a tool for the development and testing of potential therapeutics.”

How Does Maternal Obesity Affect Kidney Development in Newborns?
Dr. Tarak Srivastava from Children’s Mercy will receive funding to determine the affect maternal obesity has on kidney development in newborns. Studies show that maternal obesity increases kidney abnormalities resulting in chronic kidney disease (CKD). “We propose that the effects of disease processes in the mother on fetal development are mediated by a maternal chemical causing inflammation known as IL-6,” said Dr. Srivastava. Recent research suggests that IL-6 can alter gene expression within the cell. “With our research, we plan to answer two questions: 1) Does elevated maternal IL-6 alter the anatomy and functional development of kidneys; and 2) which pathways are regulated during kidney development by elevated maternal IL-6?”

This project will lead to a model examining the effect of increased levels of IL-6 in mothers involved in abnormal kidney development that would increase the likelihood of chronic kidney disease in their children.

Information about the Patton Trust Research Development Grants, including eligibility, review criteria and application procedures, can be found on the Kansas City Area Life Science Institute’s website.

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About Kansas City Area Life Sciences Institute:
Through its Research Development Grants program, KCALSI manages a variety of individual grants for area corporations and trusts, helping them identify proposals with the best scientific, medical and technical merit. Research grants are awarded to generate initial results and stimulate the submission of major multidisciplinary research proposals to government or private agencies. KCALSI’s Research Development Grants program includes proposal review, evaluation by subject matter experts, written reviews for all applicants, and post-award management.

Media Inquiries:
Dr. Keith Gary
KCALSI – Vice President
kgary@kclifesciences.org
816-753-7700

Shaylee Yount
KCALSI – Communications and Events Manager
syount@kclifesciences.org
816-753-7700

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