Pediatric Cancer
Conditions
Brief summary
A new research paradigm that involves sequencing tumor DNA/RNA to identify driver mutations, select among the Health Canada approved drugs (for adult cancers) known to block certain oncogenic pathways, and recommend these drugs to the treating physician, without taking into account the tumor histology. In this paradigm, the treatment is targeted to the actionable mutation(s) i.e. those driving oncogenesis. It is also personalized to the molecular signature of the patient's tumor, irrespective of its histopathological subtype. The experience of the investigators team in genomics, including next generation sequencing and bioinformatic analysis combined with the clinical expertise, bring at last this approach within our technical capacities. In parallel, the number of Health Canada-approved drugs (which have been tested in a pediatric setting) designed to interfere with oncogenesis pathways is increasing exponentially.
Interventions
None listed
Sponsors
St. Justine's Hospital
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Months to 21 Years
Healthy volunteers
No
Inclusion criteria
At the time of enrollment: * 21 year-old or less * Poor prognosis biopsy-proven cancer of any type : * Cancer (at initial diagnosis) known to be refractory to treatment * Or cancer refractory to treatment * Or relapsed cancer * Written informed consent by patient, parents, or the legal guardians
Exclusion criteria
* Estimated life expectancy of less than 3 months.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers. | 24 months | The study team will evaluate the timeline between decision of biopsy, the actual biopsy, availability of results of the whole-genome analysis, interpretation of results and divulgation of results to patient and family. |
Secondary
| Measure | Time frame |
|---|---|
| Number of children with cancer who are suitable candidates for targeted therapy at our institution each year. | 24 months |
| Number and type of driver mutation(s) found in our population of recurrent or refractory cancers. | 24 months |
| Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug. | 24 months |
| Feasibility of performing whole genome sequencing and data analysis, identifying a drug based on the genomic data and offering this information to the medical team, the patient and the family within 10-week time frame from diagnosis | 24 months |
Countries
Canada
Contacts
Primary ContactMonia Marzouki, MD
Backup ContactHemrique Bittencourt, MD
Outcome results
None listed