Scientists at the University of Copenhagen’s Center for Healthy Ageing have identified a compound that blocks the expression of a protein without which certain tumours cannot grow. This compound has the potential as an anticancer agent according to the research published in the journal CHBIOL: Chemistry and Biology this week.
The BLM protein is also known to be important in maintaining stability in cells when they multiply, thus preventing cancer. However, certain types of tumour need BLM to grow. This is typical of osteosarcomas – aggressive malignant tumours often seen in bone cancer – and also soft tissue sarcomas.
Now for the first time scientists have been able to turn off the BLM function in cells using an inhibitor called ML216, which stops cells that express BLM from multiplying, leaving cells without BLM alone.
Tumour treatment one step closer
Professor Ian D. Hickson, who led the research says: “Sarcomas and especially osteosarcomas are notoriously difficult to treat. This compound has the potential to lead to a treatment that could stop such tumours growing.”
Professor Hickson’s team is now working on finding derivatives of the compound that will be more potent and suitable to use as a basis for a drug.
“Once we have the compound in the right form, the next step is to test it using mice as a model and then, all being well, to move on to a clinical trial. However, we are several years off having an actual treatment.” He says.
The Bloom Syndrome
The BLM gene and BLM protein get their names from the rare genetic disorder Bloom’s syndrome, identified in 1952 by the New York Dermatologist Dr David Bloom. Bloom’s syndrome sufferers carry a mutation of the BLM gene that prevents the BLM protein from being expressed.
The BLM gene is a DNA helicase that unwinds the two strands of DNA. This unwinding is a feature of most DNA processes such as replication, or making a copy of itself. When it goes wrong mutations can appear that can lead to cancer. The average age for developing cancer for a Bloom’s syndrome sufferer is 25 years old.
Although the suppression of the BLM protein can have catastrophic results for Bloom’s syndrome patients, this research shows the potential benefit for treating other cancers. It is thanks to a deepening understanding of Bloom’s syndrome that this insight has been achieved.
University of Copenhagen