Translational Research Improves Medication Safety and Efficacy

At Children’s Mercy, research in Personalized Medicine and Clinical Pharmacology is driven by our goal to give children the best care possible.

“Traditional drug development approaches have relied on identification of the best dose of a medication to treat patients ‘on average’—on a population basis. This strategy breaks down when applied to individual patients, especially those who fail to respond to the recommended dose, or who experience side effects. So we’re actively exploring the extent of variability and the contributions of genetics and development in response to medications across all fields of pediatric medicine—then using this knowledge to achieve a higher level of individualized care.”

Children’s Mercy is integrating research with treatment by creating translational teams. The program pairs clinicians who identify clinically significant medication-related problems with basic science faculty members who have the expertise necessary to develop solutions to those problems. The ultimate goal is to improve clinical care by increasing both the safety and efficacy of medications used in children. A secondary goal is to provide a learning environment conducive to developing clinician scientists who will subsequently provide leadership for integrating personalized pediatrics into standard clinical care.

A prime example of this type of collaboration is the work of pediatric rheumatologist Mara Becker, MD, MSCE. Dr. Becker is completing fellowship training in Clinical Pharmacology at Children’s Mercy, which has the only pediatric training program in the U.S. accredited by the American Board of Clinical Pharmacology. Her research has focused on methotrexate (MTX), an old drug originally developed for specific forms of cancer, but now used to treat juvenile idiopathic arthritis (JIA). While MTX is generally 
considered to be safe and well tolerated, it only works in about 60 percent of children and can cause side effects such as nausea and liver inflammation in certain patients. Dr. Becker is seeking to understand what makes someone more or less responsive to MTX. In order to complete this work, we’ve established a productive collaborative team that includes members of the Rheumatology and Clinical Pharmacology divisions at Children’s Mercy as well as the Pharmaceutical Chemistry department at the University of Kansas, Lawrence.

Another example is our initiative to develop an experimental therapeutics program in oncology at Children’s Mercy, which is spearheaded by Kathleen A. Neville, MD, MS. Board certified in Hematology/Oncology and Clinical Pharmacology, Dr. Neville has had extensive training in experimental therapeutics for children with cancer. She is leading our Phase I research efforts to develop new agents for the treatment of pediatric cancer and supportive care for patients.

Bridgette L. Jones, MD, board certified in Asthma/Allergy and trained in Clinical Pharmacology, has focused on genotype/phenotype associations in asthma to gain a better understanding of how genetics affect histamine breakdown. She is also investigating a new technique called histamine iontophoresis to assess histamine response in the skin through the use of Laser Doppler Flowmetry.

These are just a few of the many translational studies that illustrate the collaborative environment and multi-disciplinary synergy at Children’s Mercy.

“Today, the technology is so advanced that one person can’t do everything. I’ve found some of the best collaborators outside of my own discipline. That’s the kind of environment that Children’s Mercy has fostered here over the past several years.”