Making an Impact on Research
Scientists at the Sanford Children’s Health Research Center are seeking new treatments for birth defects and childhood diseases, but how quickly those findings progress depends on available funding and resources.
Generous donors to Sanford Research ensure important work, like that of Dr. Michael Kruer and his team, continues in the quest for improved treatments and cures.
Dr. Kruer, pediatric neurologist and associate scientist at Sanford Children’s Research Center, said good labs across the country are closing because federal funding is at a record low. Donors in this region can make a difference in the future of medicine with their support.
“It’s critical to partner with donors to continue to push important work forward,” he said.
Dr. Kruer and his team are studying cerebral palsy (CP), a disorder of movement that for decades was believed to be solely the result of prematurity and oxygen deprivation to developing brains. However, Dr. Kruer’s research proposes a different culprit: genetics.
“We are finding that for many kids the disorder is caused by mutations, or misspellings if you will, in the DNA blueprint in a single gene,” Dr. Kruer said. “This was a single gene that was critical for brain development but didn’t function appropriately; it wasn’t turned ‘on’ at the right time, and it’s responsible for the child’s symptoms.”
Dr. Kruer and his team are using genetics to characterize the biological basis of CP in order to develop therapies to restore normal brain function. They are also gathering information, identifying additional genetic causes of CP and trying to better understand how brain cells communicate to control movement.
“Our brain has an incredibly complex web of connections,” he said. “Those connections are not static; instead, they are dynamic and ever-changing, and connections between different brain cells and different brain regions can in fact be altered. This suggests that it might be possible, even in a development disorder like CP, to rewire the brain so that those connections form appropriately.”
Dr. Kruer and his team first discovered a new gene that leads to cerebral palsy when it is defective, altering essential brain connections. They went on to show that a natural compound derived from the sea sponge can help more appropriate scaffolds build brain connections. In follow-up experiments, the researchers saw this compound restore the structure of a simple brain.
“We are working with several complementary model systems to really flesh out all the different mechanisms at work here,” he said. “But we are pretty excited we can reverse these things in the laboratory, arguing that treatments targeting the root of the problem, not just managing symptoms, are possible.”
Donors are an essential component of this research. Their gifts are crucial in seeing this— and many other projects—through to conclusion and ensuring a better future for ill children.
“What we have is very promising, but we still need to take a series of very deliberate steps before we can advance to clinical trials,” Dr. Kruer said. “How quickly we bridge that gap completely depends on the resources we can dedicate to treating this disease.”
To learn more about Sanford Research or to make a gift, visit foundation.sanfordhealth.org or call the Sanford Health Foundation at (605) 312-6700.
Posted Date: July 2014