Four leading children’s hospitals announce IPCHiP, a collaborative to develop new treatments for paediatric diseases

TORONTO - Ahead of Rare Disease Day (February 28), four leading children’s research institutions on three continents are joining forces to decipher paediatric illnesses, including rare diseases, and find better treatments. The four paediatric hospitals — Boston Children’s Hospital; UCL Great Ormond Street Institute for Child Health and Great Ormond Street Hospital (London); the Murdoch Children’s Research Institute with The Royal Children’s Hospital (Melbourne); and The Hospital for Sick Children (SickKids) (Toronto) — will pool their medical and scientific expertise to analyze medical and genomic data to accelerate discovery and therapeutic development.

The partnership, known as the International Precision Child Health Partnership (IPCHiP), is to the founders’ knowledge the first major global collaboration around genomics and child health. The founding partners anticipate that additional institutions will join the collaboration in the future.

Of the more than 7,000 rare diseases that affect millions of individuals globally, only a few hundred have approved treatments. Many of these diseases mean children suffer their whole lives, or may die early from complications, sometimes just a day after birth.

IPCHiP’s goal is to enable the world’s top experts in pediatrics and genomics to work together to improve diagnosis, implement personalized treatment decisions, and develop new therapeutic targets and treatments that will benefit children around the world.

“SickKids has been championing a new approach to paediatric medicine – Precision Child Health – which includes using genomics to better classify disease so it can be treated earlier and more effectively. Through IPCHiP, we can now share our expertises and resources to fuel new discoveries,” says Dr. Stephen Scherer, IPCHiP lead and Director of The Centre for Applied Genomics (TCAG) at SickKids. “By doing so, we are taking a huge leap forward in moving medicine toward data-driven treatments that take into account individual differences in each child’s genes.”

IPCHiP’s first project will involve epilepsy in infants, bringing together efforts already underway at the four centers. Through this project, IPCHiP will establish protected systems to evaluate data across different institutions without any information having to cross international borders.

Investigators at each site will prospectively enroll babies under age 1 with epilepsy in real time, sequence their genomes, change treatment based on the findings as appropriate, and follow their development long term. The study will compare infants who receive a genetic diagnosis with those for whom no genetic answer can be found.

“Rapid whole genome sequencing can help us find the cause of early onset epilepsy quicker and among a greater number of patients, giving clinicians the critical data they need to choose the most appropriate treatment sooner,” says Dr. Vann Chau, Neurologist and Project Investigator in the Neurosciences & Mental Health Program at SickKids, who is leading the SickKids arm of the epilepsy project. “Our hope is that this collaboration will help create new and tangible changes in medical practice to cut diagnosis from months to days, and lessen the burden on our patients and families living with epilepsy.”

“At SickKids, we have a successful track-record of conducting genomics research studies that translate from the bench to the bedside,” says Dr. Greg Costain, iPCHiP clinical lead, Staff Physician in the Division of Clinical and Metabolic Genetics, and Scientist-Track Investigator in the Genetics & Genome Biology Program at SickKids. “With this new international partnership, we hope to responsibly leverage research technologies and specialized expertise from world-renowned institutions to help ourselves and our colleagues provide the best care possible for all our patients and families.”

After initial seed funding by each institution, the collaborative anticipates additional funding through national health and medical research grants within each country, industry contracts, and philanthropic efforts. The collaborative as a whole has received support from the International Venture Philanthropy Network.

IPCHiP is contributing to Precision Child Health, a movement at SickKids to shift medicine from a one-size-fits-all approach to health care that is individualized to each unique patient.