NEW YORK – Children's Mercy Hospital said on Wednesday that it has raised $16 million from donors to fund a new initiative called Genomic Answers for Kids that aims to accelerate the search for diagnoses and novel treatments for pediatric genetic conditions.
The effort is the flagship research project for the Children's Mercy Research Institute, whose new facility is slated to open in early 2021. Investigators hope to collect genomic data and health information for 30,000 children and their families, creating a database of nearly 100,000 genomes.
According to the project website, recruitment is dependent on the identification of potential participants by clinicians at the hospital. Researchers are seeking children who are suspected to have a genetic condition or a genetic component to their symptoms.
Tomi Pastinen, director of CMH's Genomic Medicine Center, said in a statement that the effort was conceived to address a systemic lack of funding for pediatric research, accelerating the discovery of genetic contributors to rare and life-threatening childhood disorders.
"On average, families [currently] wait five to seven years for a diagnosis and that’s simply too long – this database will be used to identify other genetic diseases, and research treatments and cures in the future. It will move kids to the front of the line," Pastinen said.
The program already has more than 1,750 families enrolled, he added, resulting in more than 6,000 new genomic analyses, more than 170 genetic diagnoses and the discovery of eight new genes.
Among 76 donors that have contributed, CMH highlighted Brad Bradley and Robbie Harding, a couple whose daughter was born with Down syndrome and later developed Acute Lymphoblastic Leukemia, from which she has now been in remission for over three years.
"Like most parents with children that have been diagnosed with a rare disease, I’ve asked myself many times ― why does this happen? … And in our case, why do kids with Down syndrome have ten times the risk of getting cancer?" Bradley said in a statement. "Learning the answers to some of these baffling questions will hopefully bring some comfort and even normalcy into what is a very dark and difficult time for many families."