Targeted Therapy

Our laboratory focuses on the development of novel therapies for the most aggressive childhood cancers, with a goal of rapid translation to the clinic. A key area of our research focus is investigating new treatment strategies for malignant and incurable childhood brain tumours.

We have a strong research focus on Diffuse Intrinsic Pontine Gliomas (DIPG). DIPG are a highly malignant glioma that arise in early childhood in the brainstem, the most critical part of the brain. They are not amenable to surgery, or even biopsy. Patients don't respond to chemotherapy and radiation therapy only results in a short delay in tumour progression. Two hundred and fifty clinical trials have been conducted for DIPG and no successful treatments have been identified. Almost all children die of the disease within 12–24 months.

Funding for our research comes from sources including NHMRC, Cancer Australia, Kids Cancer Alliance, The Cure Starts Now, Cure Brain Cancer Foundation, The Kids Cancer Project, Robert Connor Dawes Foundation, Benny Wills Brain Tumour Fund, and Dainere’s Rainbow Brain Tumour Research Fund.

Group Leader

Associate Professor David Ziegler
    • DIPG cell cultures


      A critical problem in improving treatments has been the lack of surgical tissue for biological studies in the laboratory.

      To address this, we have initiated a national DIPG autopsy study. With tissue donated from autopsies, we have grown a panel of DIPG neurosphere cultures. These represent a unique platform on which to investigate the biology of DIPG and to test novel therapies. We have developed orthotopic mouse models of DIPG and performed robotic high throughput screens to identify novel therapeutic approaches. Further combination screens of the most effective compounds have identified potentially useful combination therapies.

    • Therapeutic strategies for DIPG and other high-risk brain tumours


      We are currently investigating a number of different therapeutic strategies to treat DIPG and other high-risk brain tumours. These strategies include:

      • modulation of cellular metabolism
      • targeting the mitochondria – the cancer cell’s powerhouse
      • targeting the epigenome
      • targeting cellular growth factors and tyrosine kinases
      • targeting the cell cycle apparatus
      • developing radiation therapy sensitising agents
      • investigating novel immunotherapeutic strategies.

      We recognise that none of these strategies alone is likely to be sufficient to eradicate tumours such as DIPG, therefore we have a strong emphasis on the development of rational combination therapies.

    • Personalised therapies for brain tumours


      A/Prof Ziegler is chair of a national precision medicine program that seeks to find personalised therapies for high risk cancer patients, including brain tumour patients.

      The Targeted Therapy research group leads the brain tumour program providing genomic analysis, in vitro drug screening and in vivo personalised xenograft models for all high risk paediatric brain tumour patients. The group aims to define novel therapeutic approaches for individual patients in real time, and leads a discovery program which utilises the rich database of genomic and drug screening data to investigate novel therapies for all high risk childhood brain tumour patients.

Staff List


Associate Professor David Ziegler


Dr Maria Tsoli


Dr Laura Franshaw

Dr Anahid Ehteda


Swapna Joshi
Jie Liu
Ornella Tolhurst


Aaminah Khan