Repurposed Cancer Drug Shows Promise in Treating HER2-Positive Breast Cancer

• Researchers have identified a repurposed cancer drug that shows promising results in preclinical models for treating HER2-positive breast cancer, a subtype affecting approximately 20% of all breast cancer cases.
• The investigational drug has the ability to cross the blood-brain barrier, offering a potential therapeutic approach for brain metastasis, a common complication in HER2-positive breast cancer.
• The drug, already tested and approved for human use, is expected to rapidly advance to a Phase II clinical trial, potentially offering a new treatment option for patients with this aggressive form of breast cancer.
• The research team is validating their findings with oncologist collaborators, supported by grants from the Cancer Council and initial funding from HMRI donors.

A team of researchers at the Hunter Medical Research Institute (HMRI) has made a significant breakthrough in the treatment of HER2-positive breast cancer, a subtype known for its aggressive nature and tendency to metastasize. The research focuses on a repurposed cancer drug that has shown promising results in preclinical models, offering hope for patients with treatment-resistant disease.

The Challenge of HER2-Positive Breast Cancer

HER2-positive breast cancer, characterized by high levels of the human epidermal growth factor receptor 2 (HER2), accounts for approximately 20% of all breast cancer cases, affecting around 5,000 Australians annually. Dr. Luiza Steffens-Reinhardt notes that while about 90% of early-stage cases can be treated with chemotherapy and targeted therapy, around 20% of these patients experience a recurrence, which is often incurable and life-threatening. This subtype is particularly challenging due to its propensity to metastasize to the brain, lungs, and other organs.

A Repurposed Drug with Novel Potential

The research team, led by Dr. Chen Chen Jiang, Professor Hubert Hondermarck, and Dr. Luiza Steffens-Reinhardt, is focusing on a repurposed cancer drug that can cross the blood-brain barrier. This is a critical feature, as it allows the drug to target brain metastases, a major cause of morbidity and mortality in HER2-positive breast cancer patients. According to Dr. Steffens-Reinhardt, the drug is being directed to a highly specific target, enhancing its efficacy and potentially reducing side effects.

Path to Clinical Trials

Because the drug has already been tested and approved for human use, the researchers anticipate a rapid progression to a Phase II clinical trial. This expedited timeline could bring a new treatment option to patients sooner than with novel drug development. The team is currently validating their results with oncologist collaborators, supported by a Cancer Council grant and initial funding from HMRI donors. This validation process is crucial for ensuring the robustness and translatability of their findings.

HER2-Positive Breast Cancer: Key Facts

HER2-positive breast cancer is driven by the overproduction of the HER2 protein, which promotes the growth of cancer cells. While it is not hereditary, this subtype tends to grow and spread more quickly than other types of breast cancer. However, it often responds well to HER2-targeted treatments, making the development of new therapies crucial for overcoming resistance and improving patient outcomes.