Congratulations to C17 Grant Recipients: William Foulkes, Faisal Khan and Tal Schechter

The C17 Research Network is pleased to announce the recipients of the 2014/15 grant competition.

PI: Dr. William Foulkes (McGill)
Title: Towards a pan-Canadian DICER1 clinical and research network

Inherited mutations in the gene DICER1 have been discovered to cause rare types of tumours that occur primarily very early in an infant’s life, but little is known about how these mutations cause tumours. The purpose of the proposed study is to establish a network of physicians, pathologists and researchers with knowledge of DICER1-related cancers from across Canada to enable them to share valuable resources such as patient samples and treatment information. The benefits of this network will be twofold: first, it will allow the identification of most Canadian families with family members that may suffer from a DICER1-related cancer. This will provide these families with access to genetic testing and counselling, and increased surveillance for as yet unaffected infants. Second, collecting samples and information from DICER1 patients will provide researchers access to very valuable materials and information that can be used to better understand how these mutations lead to cancer development and potentially better ways to treat affected individuals. The network will create a link between the physicians who treat patients and the researchers who work at understanding the disease, to the benefit of all.

PI: Dr. Faisal M. Khan (Alberta Children’s Hospital Research Institute)
Title: Genetic Profiling of Killer Immunoglobulin-Like Receptors (KIRs) of Natural Killer Cells as Predictors of ATG-Conditioned HLA-matched Pediatric Allogeneic Hematopoietic Cell Transplantation (HCT) Outcomes

Bone marrow transplantation (BMT) is a life-saving treatment for childhood and adult blood cancers and other blood disorders. However, BMT is toxic, especially due to ensuing hostile attack of graft cells on patient’s tissues (known as graft-vs-host disease, GVHD) or infections, and relapse of cancer. Despite of using donors matched for important immunity affecting genes, BMT is truly beneficial for only ~35% patients. Remaining 65% of patients either die due to relapse, GVHD or infections or suffer long-term due to GVHD. It is therefore important to find other gene systems that can improve BMT outcomes by controlling bad immunity (one that leads to GVHD) and/or by enhancing good immunity (one that fight cancer and infections).

Present study is focused on one such gene system known as ‘KIR’ that dictate the functioning of one of the first type of blood cells that recovers back after BMT. Here, we will assess whether presence/matching of certain KIR genes in donor and recipients lead to improved outcome of pediatric BMT. If yes, we will further refine the KIR gene assessment so that for each pediatric BMT recipient a donor can be chosen that gives the lowest likelihood of complications. This should lead to improved survival and quality of life of pediatric BMT patients.

PI: Dr. Tal Schechter (Hospital for Sick Children)

Title: A population-based study of long-term outcomes in survivors of allogeneic hematopoietic stem cell transplantation during childhood, adolescence or young adulthood

With modern therapies, more patients who are treated with bone-marrow transplantation (BMT) will continue to survive. Unfortunately, a majority of the survivors will develop long-term side effects of their transplant and related therapies. These late-effects are often severe and can lead to premature death. There are very few publications addressing late-effects after receiving a BMT in childhood. There is even less knowledge on these long-term outcomes after BMT in adolescents and young adults or in patients who underwent BMT for indications other than cancer.

Our research group has extensive experience in the investigation of late complications. In the proposed study, we plan to estimate the risk of late death and the development of subsequent cancer in children, adolescents and young adults who underwent BMT. We also plan to evaluate other health consequences in long-term survivors of childhood, adolescent and young adulthood BMT by assessing parameters such as outpatient clinic visits, and the frequency of hospitalizations. The proposed work is the foundation required to estimate the specific health burden and inform patients and health-care providers regarding the actual risk for late effects. It will also allow to plan for life long risk-based survivor care in young patients who underwent BMT.

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