Breakthrough for childhood brain cancer treatment

NHMRC

The Australian Government has announced over $700,000 in funding to support Dr Fatima Valdes Mora and a team from the University of New South Wales to develop a new treatment that targets diffuse intrinsic pontine glioma (DIPG).

DIPG is a type of childhood brain cancer with no known cure or treatment options.

This innovative research will be funded under the National Health and Medical Research Council's (NHMRC) Ideas Grant scheme.

DIPG is a type of malignant brain tumour that forms in the brainstem, impacting function of nerves that control muscles and sensation of the face and is one of the most aggressive of all childhood cancers.

Importantly more than 80% of patients diagnosed with DIPG carry the same genetic mutation that makes the cancer cells more aggressive.

Despite previous research efforts to understand the genetics of DIPG, clinical outcomes have seen no improvements in over 50 years, and more than 250 clinical trials have failed.

Dr Valdes Mora intends to develop a new treatment that targets this disease-causing genetic mutation commonly found in patients with DIPG.

The treatment will aim to reverse the molecular changes that make DIPG difficult to treat, ultimately improving patient outcomes.

Quotes attributable to NHMRC CEO Professor Steve Wesselingh:

  • "Finding effective treatments that address the genetic and epigenetic factors of DIPG has been a difficult health research challenge.
  • "Dr Valdes Mora's proposed approach to this research is simultaneously both innovative and creative, targeting treatments that centre on the disease-causing mutation associated with DIPG.
  • "NHMRC's Ideas Grant scheme is the enabling vehicle for such exceptional research as this- congratulations to Dr Valdes Mora and the team from the University of New South Wales.

Quotes attributed to Dr Valdes Mora, University of New South Wales:

  • "DIPG is a type of paediatric brain cancer with no cure and no treatment options.
  • "Even though the main genetic cause of DIPG (a mutation called H3K27M) has been known since 2012, the survival rate for DIPG patients hasn't improved over the last 50 years, due to the inability to target the mutation directly.
  • "Our goal is to identify specific DNA regions related to H3K27M that cause DIPG to simultaneously and specifically target them using advanced systems and technologies.
  • "This project stands out by bringing together a diverse team of national and international researchers to create a new type of therapy to treat DIPG based on the Nobel Prize-winning CRISPR/Cas technology in a way that no-one has attempted before."

Quotes attributed to Angie Daher and Tamer Daher, consumer advocates for DIPG research:

  • "Our daughter Eve was diagnosed [with DIPG] in March 2021 and given 12 months to live. Had it not been for researchers in Australia, our daughter would not still be fighting 33 months later.
  • "Having government support and funding into DIPG can change that narrative so that breakthrough research can continue to happen to further extend and save children's lives.
  • "Our hope is that families and children who receive a DIPG diagnosis in the future won't be told 'go home and make memories' like we were."

Disclaimer: We acknowledge the enthusiasm and engagement of the health and medical research sector, as well as their interest in the 2023 Ideas Grant funding outcomes. The full list of funded projects under the scheme will be announced in due course, and therefore all other Ideas Grants remain under strict embargo.

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