Study explores why some drug therapies are ineffective for brain tumour patients

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Study explores why some drug therapies are ineffective for brain tumour patients

New research will try and find the first effective non-surgical treatments for patients with neurofibromatosis type 2

Brain Tumour Research Centre of Excellence at the �� awarded funding so it can continue work until 2030

Study explores why some drug therapies are ineffective for brain tumour patients

New research will try and find the first effective non-surgical treatments for patients with neurofibromatosis type 2

Mr Alan Williams

Media and Communications Manager

Corporate Communications (Marketing and Communications)

12 December 2025

Scientists at the �� have been awarded significant funding to explore why certain drug treatments are proving ineffective in brain tumour patients.

The two-year project, supported by the Children’s Tumor Foundation (CTF), will specifically be aimed at NF2-related schwannomatosis (NF2), a rare genetic condition which frequently causes patients to develop multiple tumours. These can include schwannomas on the nerves responsible for hearing and balance, as well as under the skin and along the spine, and meningiomas that develop in the protective membranes covering the brain and spinal cord.

At present, the only available treatments for NF2-related tumours are surgery and radiotherapy. However, these approaches are not suitable for all patients, particularly those with multiple tumours throughout the body, or with tumours located in neurologically sensitive regions. Even in patients who are eligible for surgery or radiotherapy, treatment is often associated with significant side effects.

Numerous drugs used clinically to tackle other tumour types have shown only limited or no effectiveness in treating NF2-related schwannoma and meningioma tumours. In recent studies, carried out at the Brain Tumour Research Centre of Excellence at the University the scientists discovered that Multi-Drug Resistance (MDR) – caused by specific proteins located on the surface of tumour cells – may be responsible. These proteins can actively transport drugs out of the cells, thereby preventing the drugs from exerting their therapeutic effects.

To address this challenge, the new project will explore whether clinically tested and FDA-approved cancer drugs, specifically those that inhibit MDR mechanisms, can be repurposed for use in combination with the drugs that failed in NF2 clinical trials.

The goal is to develop the first truly effective therapies for patients – and because NF2-related tumours closely resemble their sporadic counterparts, treatments shown to be effective in NF2-related schwannoma and meningioma could subsequently be extended to the much larger population of patients with sporadic tumours, which are highly prevalent, particularly meningiomas, the most common type of primary brain tumour.

The project is being funded through a grant of over $175,000 from the CTF’s Drug Discovery Initiative (DDI) Awards programme and will be led by Associate Professor Dr Sylwia Ammoun and PhD researcher Summer Henderson, in collaboration with Research Fellow Dr Juri Na .

Associate Professor Dr Sylwia Ammoun

For many years, researchers worldwide, including our laboratory, have searched for new therapeutic targets and tested numerous drugs in NF2-related and sporadic schwannoma and meningioma tumours.

Although many of these agents showed promising effects in laboratory models, most ultimately proved to be ineffective or only partially effective in patients during clinical trials. This led us to suspect that there is a fundamental feature of these tumours that prevents the drugs from working effectively. Our research so far has shown that schwannoma and meningioma tumours are inherently drug resistant and therefore do not fully respond to drug therapies. If we are to find effective treatments for these tumours, that is something we urgently need to address. Our ongoing research is possible thanks to generous support from organisations such as the Children’s Tumor Foundation, as well as patients who donated samples, for which we are deeply grateful.

Dr Sylwia Ammoun
Associate Professor

Dr Ammoun and Summer Henderson have been working for the past three years to examine drug resistance in schwannoma and meningioma tumours, with their research focused on tumour cells taken from patients during surgery, and made available to researchers through the �� Biobank and Brain UK.

Their work has demonstrated that combining proposed NF2 treatments with direct MDR inhibitors clinically tested in other cancers can enhance therapeutic efficacy in meningioma and schwannoma tumours. Their studies further showed that MDR can also be targeted indirectly by inhibiting signalling pathways that regulate the expression and activation of MDR proteins, including the PI3K/AKT pathway.

Using clinically tested and FDA-approved drugs which target this pathway resulted in markedly improved treatment outcomes in laboratory cell models. These combinations have proved effective at low doses, offering the potential to minimise side effects experienced by patients, while improving therapeutic outcomes.

With the new funding, researchers will first test these drug combinations in the laboratory on tumour cells taken from schwannoma and meningioma patients. The most promising treatments will then be tested in living models in a follow-up study to assess how well they work within the body.

Since the drugs under investigation are either already approved by the Food and Drug Administration (FDA) agency, or currently in clinical trials for other diseases, the hope is that if the project is successful it can be swiftly translated into clinical trials and then be used in patient treatments.

The grant awarded by the Children’s Tumor Foundation is an exciting milestone for the project I have been working on over the past three years.

Securing the support of such a dedicated charity is truly inspiring; without funding of this kind, our research may not have been able to progress. Addressing multi-drug resistance in meningioma and schwannoma tumours has the potential to make a significant difference for patients, and we remain hopeful that our work will ultimately lead to better, more effective options for those living with these conditions.

Summer Henderson
PhD Researcher

Funding support from the Children's Tumor Foundation

In 2025, the CTF has awarded nearly $2 million to a range of projects that will accelerate the development of new therapies for neurofibromatosis type 1 (NF1), schwannomatosis (SWN), and NF2-related schwannomatosis (NF2-SWN).

This year’s awards mark the largest round of DDI funding to date, advancing bold ideas that tackle NF’s toughest challenges: shrinking tumours, overcoming drug resistance, and developing innovative targeted therapies. These projects reflect CTF’s unwavering commitment to transforming scientific discoveries into tangible, life-changing treatments for patients and families.

We funded this project because drug resistance has long been a barrier to developing effective therapies for NF2-related schwannomatosis. By exploring ways to restore drug sensitivity and test safer, low-dose combinations, this research could redefine treatment for NF2 patients – offering more effective tumour control, safer therapies, and more real options for those who urgently need them.

Irene Morganstern, PhD
Director of Preclinical Initiatives, Children’s Tumor Foundation

From Biological Sciences graduate to brain tumour researcher

Growing up in ��, I was always drawn to the beach, nature and animals, which led me to study Biological Sciences as a way of combining those interests with the science subjects I enjoyed. My degree provided broad training across ecology, plant sciences and biomedical sciences, and I discovered a particular passion for molecular biology and understanding how the smallest components of cells function.

For my final-year project, I conducted research into honeybee viruses at the Marine Biological Association, gaining valuable experience in core laboratory techniques such as DNA and RNA extraction. This confirmed my desire to pursue a research career. After graduating, I joined The Vaccine Group, a �� spinout, where I worked on the development of a bovine mastitis vaccine. Being trusted to work independently in the lab gave me a strong sense of responsibility and reinforced my enthusiasm for research.

After taking a year to travel, I was eager to continue my scientific development and applied for a PhD with Dr Sylwia Ammoun. My research focuses on understanding the mechanisms of multi-drug resistance in meningioma and schwannoma tumours and finding ways to combat them. Clinical trials for drug therapies in these tumours have had limited success, and our work aims to uncover whether multi-drug resistance proteins play a key role. We have begun identifying ways to inhibit these proteins to enhance drug effectiveness, and early findings have been promising – progress that we will now build on through this new project.

Summer Henderson, BSc (Hons) Biological Sciences graduate and now a PhD researcher in the Brain Tumour Research Centre of Excellence

Each day, I pass the Wall of Hope at our Brain Tumour Research Centre of Excellence. It’s a powerful reminder of the importance of this work and the people it aims to help. Being part of this research environment has also inspired me to carry out fundraising for Brain Tumour Research, giving me the opportunity to contribute not only through my work in the lab but also by supporting the charitable efforts that make it possible.

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Mr Alan Williams

Funding support from the Children's Tumor Foundation

From Biological Sciences graduate to brain tumour researcher

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