For Immediate Release: July 21, 2020
KANSAS CITY, MO – BioNexus KC has awarded three $50,000 grants to area scientists to support research focused on pediatric genetic diseases. The partnership with the Paul Patton Trust launched in 2007 and has awarded 40 grants totaling $2.4 M for this important area of research. The funds to support these studies are provided by the Paul Patton Trust, Ted C. McCarter, William Evans, Jr., and Bank of America, N.A. Trustees.
“We are proud to work with the Paul Patton Charitable Trust and Bank of America to identify and fund important pediatric research projects,” said Dr. Keith Gary, Vice President of BioNexus KC. “Their funding supports new approaches to finding cures for genetic diseases that primarily impact children.”
A brief description of each grant recipient’s research is below.
Ewing sarcoma is the second most common type of pediatric bone cancer. This cancerous tumor often develops in the legs, pelvis, ribs, arms or spine but can also spread to the lungs, bones, and bone marrow. About half of all Ewing sarcoma tumors occur in children and young adults and the overall survival of metastatic Ewing sarcoma remains approximately 30%.
Dr. Mizuki Azuma from the University of Kansas has been studying this disease for 16 years and Dr. Chad Slawson from the University of Kansas Medical Center is a specialist in post-translational modifications. This collaborative effort has identified where a specific sugar genetically attaches to a protein resulting in accumulation of an abnormal number of chromosomes which drives cancer progression. The long-term goal of the team is to discover how the sugar modification on this protein causes Ewing sarcoma which may serve as a novel target to develop therapeutics for the disease. “I am humbled and grateful for this opportunity,” Azuma said. “The grant will help us to understand how Ewing sarcoma protein works, thus will support us to accomplish our goal to develop the drug for this tragic disease.”
Cleft lip/palate (CLP) is one of the most common birth defects, affecting 1 in 700 live births globally. The causes of CLP among most infants are unknown and in some cases occurrence and severity is impossible to predict. The severity of CLP determines how many surgeries will be necessary to improve appearance, breathing, hearing and speech and language development.
Dr. Timothy Cox from the University of Missouri – Kansas City will apply three complementary sequencing technologies to capture genetic differences in embryonic facial tissue to determine how this may impact the disease. His team strives to improve strategies for diagnosis, risk counseling for families and developments of interventional therapies. “This award solidifies our Kansas City partnerships with Children’s Mercy and KU Medical Center and enables us a timely opportunity to generate critical preliminary data and new bioinformatic workflows necessary to secure longer-term funding from the National Institutes of Health,” Cox said. “Although obviously exciting for our own work on cleft lip/palate, results from this project may ultimately help us understand how individual-specific genetic mutations (ie. those not inherited from a child’s parents) contribute to the risk of being born with any birth defect.”
Spinal Muscular Atrophy
Type 1 spinal muscular atrophy (SMA) is the number one genetic cause of death for infants in the US, affecting 1 in 11,000 births. SMA is due to a genetic defect in the SMN1 gene and affects the motor neurons that power the muscles in the body. Due to muscle weakness, young patients with type 1 SMA cannot sit or turn around, and even have difficulties in swallow. People with other types of SMA also suffer from lack of muscle strength, although the rate and severity can vary.
Dr. Jingxin Wang from the University of Kansas and Dr. Christian Lorson from the University of Missouri will work together to develop a new drug for the treatment of SMA. Wang will focus on the small-molecule drug design and Lorson is one of the pioneer scientists in the field of SMA research. Their objective is to chemically link a drug that is currently in clinical trials, with a previously FDA approved SMA drug and generate a new avenue to target SMA and other genetic cause of disease in infants. “Our research lab is focused on exploring new drug modalities for the treatment of SMA, and we are at a critical stage in a new drug design to reduce the side effects of an existing SMA experimental drug,” Wang said. “This grant will allow us to advance our technology, as well as establish and support key collaborations. If successful, our research will not only benefit SMA patients but also bring about a new avenue in drug development in general for other diseases.”
About BioNexus KC’s Research Development Grants Program:
The BioNexus KC grant program has awarded 108 grants totaling $5.6M since the program launched in 2002. The intent of the grant program is to better position researchers to compete for larger federal grants. For every grant dollar awarded by BioNexus KC, $11.10 returns to the region in from federal agencies. The total return on investment currently stands at $63 M and continues to grow.
Through its Research Development Grants program, BioNexus KC 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. BioNexus KC’s Research Development Grants program includes proposal review, evaluation by subject matter experts, written reviews for all applicants, and post-award management.
Information about the Patton Trust Research Development Grants, including eligibility, review criteria and application procedures, can be found on the BioNexus KC website.
About BioNexus KC:
BioNexus KC believes we can do more to care for people and animals. We inspire thinkers from different disciplines to combine their efforts for a common purpose — healthcare innovation. From bioinformatics to cancer research and beyond, our goal is to stimulate collaboration and advance emerging technologies from concept to reality.
The KC region is a global leader at the nexus of human and animal health benefiting all our citizens and the economy. Our mission is to highlight life sciences resources and their value to the community through collaboration and commercialization. BioNexus KC creates opportunities at the nexus of:
- Human and Animal Health
- Academia and Industry
- Kansas and Missouri
Dr. Keith Gary
BioNexus KC – Vice President
BioNexus KC – Marketing & Communications Manager