Open letter to the ALCCRF


Dr Sara Khan – ALCCRF Clinical Research Fellow

Dear Australian Lion’s Childhood Cancer Research Foundation and members,

In 2016 I was fortunate to be awarded the ALCCRF Clinical Research Fellowship to undertake work at the Monash Children’s Hospital and Hudson Institute of Medical Research. A Paediatric Oncology Fellow, this fellowship has enabled me to establish lab-based research programs into two of the most prevalent and aggressive childhood brain tumours, Medulloblastoma and Diffuse Intrinsic Pontine Glioma.

A principle aim of my project is to develop and validate a rapid and cost-effective clinical pipeline to determine Medulloblastoma patient tumour molecular subtypes by analyzing archived patient samples from the Monash Children’s Hospital and Royal Children’s Hospital. I am very pleased to report that I have already processed and analyzed all available fresh frozen tumour samples from both hospitals using a state-of-the-art methylation-based assay and demonstrated that my results completely match with the patient outcomes, international central reviews and comparative international data where available. I am currently acquiring and processing additional formalin-fixed patient samples (a more common specimen storage technique) from both sites for analysis. The establishment of this platform will rapidly translate into the clinical setting with change in clinical practices according to current international standards of treatment. This means that some patients will be identified as being suitable for de-escalation of therapy whilst others will have the opportunity to potentially access targeted inhibitors, both leading to improved outcomes and fewer long-term side effects.

Another major goal of my research program is to identify novel treatments for the universally fatal childhood brain tumour, Diffuse Intrinsic Pontine Glioma (DIPG). To date, I have comprehensively screened several patient tumour cell lines using a panel of drug compounds and identified top targets that will be further validated.  It is hoped that the outcomes of these experiments will lead to a potentially new therapeutic option for these patients. Given that there is currently no effective treatment for DIPG this would represent a significant step forward in the treatment of this disease.

In addition to my research, the ALCCRF Clinical Research Fellowship also provides me with the opportunity to continue to provide clinical services to patients and families at Monash Children’s Hospital and as an Honorary Fellow at The Royal Children’s Hospital, where I can participate and attend the state wide tumour board meetings. This is an important part in my continued clinical education and development and allows me to identify gaps in our current clinical understandings that I can begin to explore in the laboratory with my research colleagues.

I am extremely grateful for the opportunity this fellowship has provided me in being able to undertake important translation research whilst also continuing my clinical engagement and development. I look forward to sharing more of my research progress and outcomes in the near future.

Yours sincerely,

Sara Khan