Carolina Nor, William Donald Nash Brain Tumour Research Fellow

 Dr. Carolina Nor, Research Fellow

Project: Rationale targeting of very high risk medulloblastoma of childhood

Location: Hospital for Sick Children, Toronto, ON 


Read about the project

Brain tumours are the most common solid malignancies of childhood, and account for the majority of the cancer related deaths. Medulloblastoma is the most common brain cancer in children for which treatment consists of surgery, radiation and high dose chemotherapy. Only 65% of patients survive and the survivors are usually left with severe side effects. More specific therapies with less injury to the brain are urgently needed. The most aggressive medulloblastomas present with a defect in a gene called p53, which is responsible for preventing cancer formation. When this particular gene is defective either in the tumor at diagnosis or at relapse, the tumors are resistant to all current therapies.

In this project I will evaluate two novel drugs that were designed to target p53-defective cancer cells. The drugs are called PLK4 inhibitor and ATM inhibitor. I will use a pre-clinical model developed in our laboratory that very precisely mimics the treatment given to children with medulloblastoma. The drugs will be given before and during irradiation to prevent the development of drug resistance. This novel approach has the potential to rapidly move forward to clinical trials, and may significantly improve the survival and quality of life of children with medulloblastoma.

Read about what being awarded this Fellowship means to Carolina

"Being awarded the William Donald Nash Brain Tumour Research Fellowship means that I will have the opportunity to conduct a project I believe can have a significant impact in the treatment of brain tumors in childhood. The traditional search for new therapies has been flawed where models do not exactly reproduce the human treatment conditions. My passion is to develop new and novel therapies in realistic pre-clinical models of brain tumours closely mimicking human condition.  As a pharmacist, I am excited to develop new therapies for high risk medulloblastoma that will be more effective and less toxic leading to better survival and quality of life for children with brain tumours.  I would like to thank the Nash Family and the Brain Tumour Foundation of Canada for your ongoing generosity and contributions to funding this fellowship."

From the first year report

Medulloblastoma is the most common malignant brain tumor of childhood and treatments for this devastating disease are very limited. A major roadblock in developing new treatments has been a lack of an efficient system to evaluate new approaches. My project aims to generate an accurate pre-clinical pipeline of drug development for medulloblastoma, which reproduces, with precision, the treatment currently given to children with brain cancer. Read the first year report (PDF).

Read more about William Donald Nash, the man who inspired and gave to launch this important research program.
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Susan Ruypers
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