CU Kids at Heart supports research aimed at the prevention, treatment, and/or cure of pediatric neurological disorders, including cerebral palsy, pediatric brain cancer, epilepsy, Sturge-Weber syndrome, and Moyamoya disease. We work closely with the renowned research investigators of Boston Children's Hospital and their research affiliates on specific initiatives to make possible medical discoveries, diagnostic tools, and treatment breakthroughs that not only serve patients in Boston, but serve children and adults worldwide. And, because research and funding allocations for rare diseases are traditionally limited, our fundraising efforts are immediately impactful.

We are pleased to announce the following research directives for the 2018-2019 season:

Radiation and chemotherapy are the standard treatment for most cancers. Both seek to kill as many tumor cells as possible before the cancer develops a resistance to the treatment. Recent research shows that these treatments leave behind cellular debris, dead tumor cells that can actually stimulate surviving tumor cells. Dr. Panigrahy and his team have made significant progress in the study of resolvins and protectins in cancer. These naturally occurring molecules aid in the removal of cellular debris and help to stop their signals. These research investigators are investigating new resolvin and protectin drug therapies that may help reduce the stimulatory signals that tumors receive, and believe that specialized proresolving mediators may be able to clear cellular debris, counter tumor growth, and even mitigate the negative side effects of many pediatric cancer treatments.

Surgeon-scientist Dr. Stone, with his team at Boston Children's Hospital, has spearheaded a surgical technique for epilepsy called “Stereoelectroencephalography,” or “SEEG”, which allows for extremely precise, targeted analysis of brain activity. In a less-invasive surgical procedure than many typical brain surgeries, these electrodes and sensors are placed in a child's brain for a period of time to document seizure activity in the brain and pinpoint its source. Only then is additional surgery performed to remove the seizure-causing tissue. Dr. Stone seeks to improve and advance these minimally invasive techniques to minimize recurrences of seizures and encourage functional regeneration and repair of the brain. His 2018-2019 research plans include the further development of custom micro-wires that will record seizure activity from even an individual neuron, or brain cell, as well as further clinical testing of these devices and this innovative technique.

Parents of children with cerebral palsy, specifically those of children diagnosed with perinatal stroke, are at increased risk for clinically significant symptoms of PTSD, anxiety and depression. Parental depression following diagnosis of perinatal stroke may impact the child's recovery, similar to how maternal depression has been previously associated with poorer cognitive and behavioral outcomes in children. Dr. Smith and his team will first evaluate the prevalence of these symptoms in the parents and then will collect MRI imaging on all children who are recruited to determine if the type of stroke the child sustained is a biomarker for parental emotional outcome. They seek to discover whether the neurologic outcome of a child is indeed associated with emotional outcome in the parents; whether these effects are greater for parents with children with neonatal middle cerebral artery territory strokes compared to children with other arterial territory strokes; or, whether parental education helps alleviate these feelings of guilt and sorrow.

A transient ischemic attack (TIA), sometimes called a “mini-stroke,” is a temporary period of neurologic disfunction caused by a short-term interruption to blood flow in the brain. It is known that 13% of children who present with a TIA will have a subsequent stroke, and some will also experience strokes or neurologic symptoms that mimic TIAs. Dr. Smith and his team are working to identify biomarkers, recognizable indicators, or medical characteristics, of a biological phenomenon, specific to TIA in children. Identifying these biomarkers would allow doctors to better facilitate diagnosis and, eventually, stroke prevention treatment. Because children with Moyamoya disease often present with a TIA caused by known arterial stenosis, these children may help doctors find these TIA biomarkers and, perhaps better understand the opportunities for revascularization surgery in children with Moyamoya. Dr. Smith and his team will conduct a longitudinal study collecting clinical and neurologic histories and evaluations of children with Moyamoya in the hopes of learning more about TIA.

The period following a diagnosis of a life-threatening pediatric illness or injury can be devastating and a complex emotional time for children and their families. In fact, post-traumatic stress disorder (PTSD) is commonly diagnosed in both parents and children following a serious medical diagnosis, and PTSD may be recognized in patients and their families months to years after the initial diagnosis. Although the prevalence of PTSD has been studied for some medical diagnoses, the prevalence and significance of PTSD in parent or child after a diagnosis and surgical treatment for Moyamoya are unknown. PTSD can have debilitating effects on a child's functional outcome including failure to follow medical recommendations and decreased psychosocial recovery. PTSD in parents of children, or children, with Moyamoya could similarly impede compliance with medical and developmental interventions and, consequently, may lead to poorer outcomes in the child and the family system as a whole. Dr. Smith and his team hope to evaluate the prevalence of PTSD, anxiety, and depression in children with and the parents of children with Moyamoya in the hopes that these studies one day may inform mental health interventions early in the disease course.

Sturge-Weber syndrome (SWS) is a rare, congenital disorder that can cause a range of neurological complications, including seizures, developmental delays, muscle weakness, and more. Dr. Sahin and his team, in partnership with the research teams from those medical centers that specialize in neurology or vascular biology, have made great advancements in our understanding of Sturge-Weber syndrome. Recent advancements include identifying the mutated gene responsible for SWS and its location in the blood-vessel cells. Dr. Sahin and his team continue to isolate and measure these cells in the hopes of identifying predictive biomarkers for SWS and gaining a better understanding of how these cells interact with the neurological system. The hope is, of course, to one day discover new drug screenings and innovative therapies to prevent neurological impairment.

This year, as part of the First Downs to Fight Pediatric Brain Cancer initiative, Credit Unions Kids at Heart will donate $30,000 toward the completion of two clinical trials currently sponsored by the Team Jack Foundation. These clinical trials will test the efficacy of two promising targeted agents, MEK162 and TAK580, to stop progression of pediatric brain cancers, and will allow for critical analysis of tumor material generated by these trials. The goal of these clinical trials is, ultimately, to support the development of chemically engineered smart drugs that target common mutations which drive brain tumor growth. Hopefully, these targeted agents will become more successful than those treatment protocols currently available, and cause less adverse effects. These jointly funded clinical trials are lead by research investigators at Dana-Farber Cancer Institute in Boston, Massachusetts.