For Immediate Release: August 29, 2016
Kansas City, MO – The Kansas City Area Life Sciences Institute (KCALSI) has awarded grants supporting research focused on genetic diseases affecting children. The areas of research being explored by this year’s awardees include Zika virus, neurodegenerative disorders such as ALS, and spinal birth defects. Three area scientists will each receive $50,000 as recipients of the 2016 Paul Patton Trust, Ted C. McCarter, William Evans Jr., Bank of America N.A., Trustees Grants.
“These grants fund research focused on genetic diseases that primarily affect children and may lead to life-changing discoveries and treatments,” said Dr. Wayne Carter, KCALSI’s president and chief executive officer. “We value our relationship with the Paul Patton Charitable Trust and Bank of America, the corporate co-trustee, for funding these research grants. Historically for every dollar invested in these grants, over $8.50 has been returned to the community and researchers in future grant dollars because these grants allow the recipients to successfully compete for much larger, federally funded grants that can provide hundreds of thousands of dollars for follow-on research.” KCALSI has managed the scientific review of the Paul Patton Trust, Ted C. McCarter, William Evans Jr., Bank of America N.A., Trustees grant proposals since 2007, identifying research programs with the greatest scientific relevance and potential impact.
This year’s grant recipients and a brief description of their research programs follow.
Neurodegenerative Disorders Caused Damaged Protein Accumulation
University of Kansas Medical Center (KUMC) will focus on a gene producing a specific protein that targets damaged proteins for removal from nerve cells. Neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease) and dementia, share common abnormalities including buildup of damaged proteins that may give clues to the cause of the disease. Studying unique transgenic animal models may ultimately lead to earlier disease treatment.
A team of scientists at KUMC is developing a mouse model to study the changes in the brain and spinal cord throughout the course of the disease. The team will specifically study the abnormal function of the ubiquilin-2 gene that allows accumulation of the damaged protein TDP-43 (TAR DNA binding protein, 43 kD). Boys with this gene mutation appear normal at birth, but are wheelchair-bound before age 10 and die as teenagers. Girls affected by the disorder develop the disease later in life.
The KUMC team is also developing a cell culture model to test drugs that may prevent the formation of the disease. “This type of study may ultimately lead to recognition of earlier stages of disease development which may be more sensitive to treatment,” says Kathy Newell, Associate Professor of Pathology and Lab, Medicine Department at KUMC. “We hope to gain insights that will help affected children while realizing the potential of also helping others with similar disorders, including older individuals, with other neurodegenerative disorders.”
Zika Virus Infection Resulting In Microcephaly
MRIGlobal will collaborate with the University of Missouri to develop unique animal models to investigate Zika virus infection. The mosquito-borne virus can be passed from mother to fetus via an unknown mechanism, resulting in reduced head circumference due to incomplete brain development called microcephaly. Individuals with this condition have many disabilities. Although there are no treatments for the physical abnormalities associated with this condition, therapies can control seizures, hyperactivity, and other symptoms.
“Zika virus is not well researched and is now known to cause serious birth defects. There is a desperate need to understand how the virus spreads through mosquitos and from mother to baby,” says Herbert Gelhaus, Principal Scientist at MRIGlobal. Our research through animal models will examine how Zika virus spreads to provide tools to the research community and better understand how Zika virus causes microcephaly in a developing fetus. Our hope is that we will identify drug and vaccine targets that can be further developed to stop the devastating effects of Zika virus infection.”
Causes of Spinal Birth Defects within the Uterus
A team of investigators, which is headed by Dr. Jeff Gorski at the University of Missouri–Kansas City (UMKC) School of Dentistry and includes Drs. Jeffrey Price and Paul A. Trainor of the Stowers Institute for Medical Research, will begin new research on spinal birth defects. The most common causes for spinal birth defect are conditions experienced during pregnancy such as diabetes, lack of oxygen, ethanol consumption, or vitamin A excess. The mechanism underlying these conditions leading to spinal defects is unknown and no treatment exists to limit these defects. Mouse models are commonly used to study underlying causes of birth defects.
The deletion of the gene (Mbtps1) results in hindlimb paralysis in mice, a similar feature also occurring in babies born with certain spinal defects. Many of these conditions are thought to share Mbtps1-dependent mechanisms as a cause for spinal defects. “The goals of our research are to develop methods to monitor the effect of exposure to hypoxic or hyperglycemic conditions in utero and to ultimately prevent these types of spinal birth defects,” said Jeffrey Gorski, professor UMKC Department of Oral and Craniofacial Sciences.
Information about the Patton Trust Research Development Grants, including eligibility, review criteria and application procedures, can be found on the Kansas City Area Life Sciences Institute’s website at www.kclifesciences.org.
For More Information, Contact:
Dr. Keith Gary, KCALSI Vice President
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.