UCL team sees promise in mouse study of pancreatic tumor-targeting compound

UCL's Stephen Neidle

Investigators at University College London (UCL) have identified a compound that targets unusual knots frequently found inside faulty genes and determined that it proved effective in shrinking pancreatic tumors in mice. And while they say this particular compound isn't suitable for clinical testing, the team believes that another member of this family of medications could prove fruitful in fighting one of the most lethal cancers in the book.

The compound is called MM41. Two groups of 8 mice were treated with different doses of the drug over 40 days. And at the highest dose the investigators registered an 80% reduction in the size of pancreatic tumors. Tumor regrowth was stopped among all the mice and in two of the mice there was a complete remission over the course of their natural life span.

The drug appeared particularly effective in blocking the K-RAS and BCL-2 genes, a pair of prime suspects in the development of pancreatic cancer.

"This research provides a potentially very powerful alternative approach to the way that conventional drugs tackle pancreatic cancer, by targeting a very specific area of the DNA of faulty genes," says UCL professor Stephen Neidle. "One of the genes that MM41 blocks--the BCL-2 gene--is involved in regulating apoptosis, the body's natural process which forces cells to die if they become too damaged or unhealthy to be repaired. BCL-2 is present in high amounts in many tumours and helps cancer cells to survive, but when the BCL-2 gene is blocked by MM41 in mice, the cancer cells succumb to apoptosis and die."

As the researchers note, pancreatic cancer is very tough to treat. Only three patients in 100 live for 5 years beyond their diagnosis.

The study was funded by the U.K. charity Pancreatic Cancer Research Fund and published in Nature Scientific Reports.

- here's the release
- read the research article

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