Following successful laboratory and animal testing, Dr. Aziz Sancar's research into a novel brain cancer treatment using the EdU molecule has advanced to human trials. This development offers renewed hope for more effective treatments against this challenging form of cancer.
EdU's Mechanism and Potential
Dr. Sancar's research focuses on EdU's unique ability to cross the blood-brain barrier, a significant obstacle for many cancer drugs. Current drugs like Cisplatin are ineffective against brain tumors because they cannot penetrate this barrier. EdU, however, can not only cross this barrier but also selectively target and kill rapidly dividing cancer cells while sparing healthy cells. According to Dr. Sancar, "EdU can kill rapidly dividing cancerous brain cells while protecting non-dividing healthy brain cells," highlighting its potential as a breakthrough.
Preclinical Studies
The research process involved creating realistic brain cell models by implanting human brain tumor samples into mouse brains. The team observed EdU's effects on these tumors. While traditional cancer drugs initially showed better performance, EdU surpassed them in effectiveness after 30 to 40 days. Combining EdU with existing drugs further enhanced the results.
Human Trial Objectives
The human trials will focus on assessing EdU's side effects and confirming its effectiveness in humans, mirroring the results observed in animal models. Dr. Sancar emphasized the importance of this phase, stating, "We need to study its side effects in humans for two years before it can be used."
Implications for Brain Cancer Treatment
If the trials are successful, EdU could represent a significant advancement in brain cancer treatment, offering a new option for patients with limited alternatives. The ability of EdU to cross the blood-brain barrier and selectively target cancer cells addresses a critical unmet need in current therapies.