Grants Awarded to Area Researchers Tackling Childhood Diseases
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For Release Sept. 17, 2014 KANSAS CITY, Mo. – Three area research programs targeting treatments for devastating genetic childhood diseases will each receive $50,000 as recipients of the 2014 Patton Trust Research Development Grants.

The grant recipients were announced today by the Kansas City Area Life Sciences Institute (KCALSI).  The Institute has managed the scientific review of the Patton Trust grant proposals since 2007, identifying those research programs with the greatest scientific potential.

“The $50,000 grants fund the research necessary to identify valuable data that will allow the recipients to successfully compete for much larger, federally funded grants that can provide hundreds of thousands of dollars,” said Dr. Wayne Carter, KCALSI’s president and chief executive officer.  “This is a critical step in translating laboratory findings into life-changing treatments for infants and children.  We value our relationship with the Paul Patton Charitable Trust and Bank of America, the corporate co-trustee, for making these grants possible.”

The areas of research being explored by this year’s recipients include congenital heart disease, brittle bone disease, and Duchenne muscular dystrophy. Finding Heart Disease Answers in Quail Embryos A potentially groundbreaking hypothesis on the cause of congenital heart disease (CHD) led to a unique collaboration between Dr. Doug Bittel, director of the genetics research laboratory at Children’s Mercy Hospital, and Brenda Rongish, associate professor, department of anatomy and cell biology at the University of Kansas Medical Center.

Together with their colleagues, they are hoping to identify the cause of congenital heart defects, which result in 25 percent of all birth defects and are one of the leading causes of death in infants under one year in age. Their research uses sophisticated time-lapse imaging technology to watch, in real time, as hearts develop in quail embryos, which are very similar to human hearts. 

“We can actually see the cells move as they begin to form the heart,” Bittel said. Bittel and Rongish are particularly interested in watching spliceosomes contained within the cells as they process and translate vital genetic information that directs heart development.  Proper splicing is critical for normal heart development. “Our research will tell us if there’s something affecting the cell’s behavior,” Rongish said.  “We hope this will help clarify a big gaping hole in our understanding of how human hearts develop, and that understanding could help us develop therapies or possibly prevent this condition.”

Making Brittle Bones into Stronger Bones Imagine not being able to change your baby’s diaper or pick your baby up without breaking one of his bones.  Babies with osteogenesis imperfecta, often called “brittle bone” disease, are born with a rare genetic disorder of their type I collagen, the primary protein that makes up the bone. Two University of Missouri professors are hoping their research will show that the body can be “teased into making stronger bones” by responding to increased muscle tension or stress.  Their tests on mice with a new drug developed by Acceleron Pharma, Inc., of Cambridge, Mass., could mean the difference between walking or using a wheelchair for thousands of young patients.

“The premise of our research is how do we make a better bone?” said Charlotte Phillips, associate professor in the departments of biochemistry and child health, division of medical genetics at MU in Columbia.  Phillips, together with Dr. Sarah Dallas, professor, department of oral biology at UMKC’s school of dentistry, and PhD graduate student Youngjae Jeong in the department of biochemistry, believe the answer may lie with the muscle.

“One of the biggest factors telling a bone to stay strong is muscle.  When the muscle contracts, the bone feels this tension and asks ‘am I strong enough for this?’ And if the answer is no, it lays down more bone, making a thicker, stronger bone,” Phillips said.  “We want to maximize a child’s potential and tease their bodies into responding to make a thicker bone during development.”

The drug they are testing on mice traps some of the protein myostatin, which regulates muscle size, causing the muscle to get larger.  Larger muscles could lead to stronger bones, and that could greatly improve these little patients’ quality of life. Delivering Gene Replacement Therapy Safely The human immune system is wonderful at defending us against foreign bodies, such as viral infections. 

But sometimes the system works too well, rejecting medical treatments such as a therapeutic gene, cell or tissue meant to help patients suffering from disease. A gene therapy for Duchenne muscular dystrophy developed at the University of Missouri School of Medicine has been successfully tested on mice.  The challenge remaining, however, is to develop a method for safely delivering this therapy to human patients without their immune system rejecting it.

Dongsheng Duan, professor in the department of molecular microbiology and immunology at MU, and his team are testing on dogs a third generation viral vector – called an adeno-associated virus (AAV) – to determine if it can evade the body’s immune surveillance so that gene therapy will last.

“If we can minimize the immune response to our gene therapy vector, it will greatly increase the odds of success in treating patients,” Duan said.  “There is no cure for Duchenne muscular dystrophy, but the gene therapy we developed could be a game changer.” Duchenne muscular dystrophy is the most common lethal childhood muscle disease, resulting from a mutation in the X chromosome. 

It primarily affects boys because they have only one X chromosome. 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.  

From 2007 to 2012, KCALSI’s program managed 44 grants totaling $2,200,144 million, which enabled area researchers to successfully compete for $14,674,319 million in additional funding.  Importantly, grants totaling an additional $16,805,709 million are waiting for final funding decisions. KCALSI’s Research Development Grants program includes proposal review, evaluation by subject matter experts, written reviews for all applicants, and post-award management.

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For More Information, Contact: 
Dr. Keith Gary, KCALSI Director of Program Development, (816) 753-7700    

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