IU School of Medicine researchers recently made news with significant scientific breakthroughs in two of the school’s research priority areas: triple negative breast cancer and Alzheimer’s disease. The discoveries clear the way for the potential development of targeted therapies to treat these devastating diseases.  

These breakthrough discoveries are the key first steps in developing targeted treatments for each of these diseases, where none have existed before. It is through scientific discoveries such as these that IU School of Medicine can lead the way towards developing precision approaches to treatment and prevention of such devastating diseases.

New promise for triple negative breast cancer treatment
Unlike the three most common forms of breast cancer, triple negative breast cancer has no currently approved targeted therapies for treatment. Xiongbin Lu, PhD, an IU School of Medicine researcher in the Vera Bradley Foundation Center for Breast Cancer Research and his collaborators recently reported in the prestigious journal Nature Nanotechnology several important findings related to triple negative breast cancer and its treatment future.

According to the paper now available online, TP53 is the most frequently mutated gene in triple negative breast cancer, meaning it is fueling the growth of this aggressive form of breast cancer. However, the problem with trying to target mutated TP53, specifically, is that it is not a druggable target, because of its potential toxicity–or ability to kill–nearby healthy cells. The team went hunting for an alternative target and discovered POLR2A. Using nano-technology, the team developed a modified version of POLR2A in the form of a “nano-bomb” that they delivered into cancerous cells, which significantly reduced tumor growth and left the healthy cells intact. Learn more about the study.

Breakthrough in potential to treat Alzheimer’s disease and CTE
Equally significant findings related to Alzheimer’s disease and chronic traumatic encephalopathy (CTE) were reported in the peer-reviewed scientific journal Nature by an international team of scientists, including IU School of Medicine researchers Bernardino Ghetti, MD, and Ruben Vidal, PhD. Together with their UK colleagues, Ghetti and Vidal discovered that the pathologies of CTE and Alzheimer’s disease are not the same. Their discoveries, which have major implications for treatment, dispel the long-held belief that the diseases had the same pathology.

Using cryogenic electron microscopy (cryo-EM), the researchers examined tau tangles extracted from the brains of an American football player and two professional boxers, all recognized neuropathologically as having suffered from CTE. What they determined is that the fold of the abnormal tau protein in CTE is different from the tau fold in Alzheimer’s disease. This same research team discovered the Alzheimer’s disease tau fold in 2017, a discovery which was featured on the cover of Nature.

The research team’s most recent findings could potentially lead to new and targeted treatments for both Alzheimer’s disease and CTE. Learn more about the study.

These breakthrough discoveries help us advance new findings into clinical testing of potential diagnostics and medicines for patients.  Fundamental scientific insights into life threatening diseases will deepen collaborations between IU School of Medicine and other industry leaders like Roche and Lilly, and help fuel Indiana’s life sciences economy.

Anantha Shekhar, MD, PhD, is Distinguished Professor and Associate Vice President of Research and Clinical Affairs for Indiana University; Director of the Indiana Clinical and Translational Sciences Institute; leader of IU Precision Health Grand Challenge; and Executive Vice President of Academic Affairs for Clinical Research at IU Health.

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