A recent study conducted by researchers at Cold Spring Harbor Laboratory (CSHL) has shed light on the role of a long non-coding RNA known as MALAT1 in the progression of triple-negative breast cancer (TNBC). This innovative research, published in Molecular Therapy: Oncology, tracks the levels of MALAT1 throughout the treatment journey of a 59-year-old woman diagnosed with stage 1 TNBC.
Traditionally, cancer therapy targets focus on protein-coding genes, aiming to disrupt their role in cancer progression. However, CSHL’s team has pivoted to explore the significance of MALAT1, which has been linked to over 20 different types of tumors. The study reveals that MALAT1 levels were elevated at the time of diagnosis and decreased during the patient’s standard treatments. Notably, the levels surged at a distant metastatic site, indicating a potential role in TNBC’s spread.
Disha Aggarwal, the graduate student who led the study, emphasized the novelty of tracking MALAT1 levels over the course of treatment. “Even though MALAT1 has been implicated in different cancers, including breast cancer, nobody has looked at how MALAT1 levels change over treatment and disease progression,” she stated.
The research involved meticulous examination of tissue samples collected during the patient’s two-and-a-half-year treatment journey, which included surgery, chemotherapy, radiation, and immunotherapy. Despite experiencing a temporary regression, the cancer eventually metastasized, leading to the patient’s unfortunate passing three and a half years after her diagnosis. The insights gained from her samples may pave the way for improved therapeutic strategies for future patients.
CSHL Professor David Spector noted the rarity of such comprehensive sample analysis. “Researchers typically get to see an initial sample and an end sample, but not progressive samples in the depth that we were able to look at here,” he explained. This unique perspective may ultimately contribute to the development of new treatment modalities.
Since 2015, Spector’s lab has partnered with Ionis Pharmaceuticals to develop a drug specifically targeting MALAT1. Currently, the team is in discussions with biotech companies, with hopes of initiating a clinical trial within the next few years. In addition to therapeutic applications, the research also explores the potential of MALAT1 to predict the risk of cancer recurrence or metastasis, which could significantly impact treatment decisions for women diagnosed with TNBC and possibly for those with other forms of breast cancer.
This groundbreaking study not only enhances understanding of TNBC but also highlights the importance of exploring novel targets like MALAT1 in cancer research. As the search for effective therapies continues, findings like these hold promise for improving the lives of patients facing this challenging diagnosis.
For further details, refer to the study by Disha Aggarwal et al, titled “Longitudinal tracking of MALAT1 level over a breast cancer patient’s course of treatment and disease progression,” published in Molecular Therapy: Oncology in 2025.
