In a groundbreaking clinical trial, scientists are exploring whether drugs initially developed to treat HIV and AIDS could be repurposed to combat brain tumours. This innovative approach is being spearheaded by the Brain Tumour Research Centre of Excellence at the University of Plymouth, focusing on patients with multiple brain tumours caused by the rare genetic condition, Neurofibromatosis 2 (NF2).
The Condition: Neurofibromatosis 2
Neurofibromatosis 2 (NF2) is a rare inherited disorder that results in the formation of various types of brain tumours, including schwannomas (such as acoustic neuroma), ependymomas, and meningiomas. These tumours develop on the membranes surrounding the brain and can significantly impact a patient’s quality of life and overall health. NF2 is characterized by the development of benign tumours on the hearing and balance nerves, which can lead to hearing loss, balance problems, and other neurological complications. While the tumours are usually benign, their location and growth can cause severe health issues.
The Trial: RETREAT
The clinical trial, named RETREAT, is led by Professor Oliver Hanemann and aims to evaluate the effectiveness of anti-retroviral medications, Ritonavir and Lopinavir, in treating brain tumours associated with NF2. This trial builds on prior research conducted by Dr. Sylwia Ammoun and Professor Hanemann, which demonstrated that these repurposed drugs could reduce tumour growth and survival rates in preliminary studies.
These anti-retroviral drugs have been pivotal in managing HIV/AIDS by inhibiting the virus’s ability to replicate. The hypothesis driving the RETREAT trial is that these drugs may have similar effects on the cellular mechanisms that allow brain tumours to grow and survive. By targeting these mechanisms, the drugs could potentially slow down or stop tumour growth in patients with NF2.
Methodology
The RETREAT trial will involve patients undergoing a tumour biopsy and blood test before starting a 30-day treatment regimen with Ritonavir and Lopinavir. Following the treatment period, patients will undergo a second biopsy and blood test to assess whether the drug combination has successfully penetrated the tumour cells and achieved the desired effect. This trial, which will run for a year, represents a significant step forward in the quest to find effective treatments for brain tumours.
Patients participating in the trial will be closely monitored throughout the treatment period. The initial and subsequent biopsies will provide crucial data on how the drugs interact with tumour cells at a molecular level. The primary endpoints of the trial will be to determine the drugs’ safety, tolerability, and efficacy in reducing tumour size and growth. Secondary endpoints will include improvements in symptoms and quality of life for the patients.
The Role of Brain Tumor Research
Brain Tumour Research, a key partner in this trial, is dedicated to funding sustainable research at specialized centres across the UK. The charity also advocates for increased government and large cancer charity investments in brain tumour research to accelerate the development of new treatments and ultimately find a cure. They are actively campaigning for a national annual research funding of £35 million, aiming to improve survival rates and patient outcomes for brain tumour patients to levels comparable to those of breast cancer and leukaemia patients.
The organization’s efforts are not only focused on fundraising but also on raising awareness about the significant impact of brain tumours. Brain Tumour Research supports a network of Centres of Excellence, which conduct cutting-edge research into the causes, diagnosis, and treatment of brain tumours. By fostering collaboration between these centres and facilitating the sharing of knowledge and resources, the charity aims to expedite the discovery of new, effective treatments.
Plymouth’s Centre of Excellence
The University of Plymouth’s Brain Tumour Research Centre of Excellence is one of three universities in the UK collaborating with Brain Tumour Research to enhance brain tumour treatments and outcomes. The centre specializes in low-grade brain tumours, which are typically benign and slow-growing but have the potential to become malignant over time. The centre’s research focuses on identifying and understanding the mechanisms underlying brain tumour development and exploring strategies to halt or reverse these processes.
The Centre of Excellence at Plymouth is renowned for its multidisciplinary approach, combining expertise in neurology, oncology, genetics, and molecular biology to tackle the complex challenges posed by brain tumours. Researchers at the centre employ advanced techniques such as genomics, proteomics, and bioinformatics to uncover the genetic and molecular drivers of tumour growth. Their ultimate goal is to translate these scientific discoveries into new therapies that can improve patient outcomes.
The Broader Impact
Around 16,000 people in the UK are diagnosed with a brain tumour each year. The innovative research being conducted at Plymouth’s Centre of Excellence, supported by Brain Tumour Research, holds the promise of significant advancements in the treatment and understanding of brain tumours. By repurposing existing drugs like Ritonavir and Lopinavir, scientists hope to find more effective treatments with faster timelines and lower costs compared to developing new drugs from scratch.
The potential benefits of this research extend beyond the immediate impact on NF2 patients. Success in the RETREAT trial could pave the way for similar approaches to be applied to other types of brain tumours and cancers. The concept of drug repurposing—finding new uses for existing medications—offers a promising avenue for rapidly developing new treatments without the extensive time and financial investment required for new drug development.
The RETREAT trial marks a significant milestone in the fight against brain tumours. By investigating the potential of HIV and AIDS drugs to treat brain tumours, researchers are opening new avenues for treatment that could dramatically improve the lives of patients with NF2. This trial not only exemplifies the innovative spirit of contemporary medical research but also underscores the importance of sustained funding and collaboration in the quest to conquer one of the most challenging forms of cancer. The outcomes of this trial could have far-reaching implications, potentially revolutionizing the way brain tumours are treated and offering hope to thousands of patients and their families.
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