Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

St. Justine's Hospital30 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Pediatric Cancer

Eligibility

Sex: ALLMin age: 1 MonthMax age: 21 Years

Medical Language ↔ Plain English

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.

Locations (1)

Outcomes

Primary Outcomes

Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.

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.

Time frame: 24 months

Secondary Outcomes

Number of children with cancer who are suitable candidates for targeted therapy at our institution each year.

Time frame: 24 months

Number and type of driver mutation(s) found in our population of recurrent or refractory cancers.

Time frame: 24 months

Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.

Time frame: 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

Time frame: 24 months

Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

Overview

Conditions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts