Scientists have discovered the first compound that directly makes cancer cells “self destruct” while leaving healthy cells completely alone.
This novel treatment was used to attack acute myeloid leukemia (AML) cells but the researchers believe it could have profound implications for other cancers too.
The compound, discovered by scientists from Albert Einstein College of Medicine, activates apoptosis – an important process in the human body that rids the body of unwanted or malfunctioning cells.
While some chemotherapy treatments have been found to indirectly cause apoptosis, this new treatment would use it as the main weapon against cancerous cells.
Apoptosis occurs when BAX – the “executioner protein” in cells – is activated by pro-apoptotic proteins. It then targets the parts of the cell that create energy and destroys them.
Sadly, cancer cells are very good at fighting this and are able to suppress BAX by producing anti-apoptotic proteins.
However, this is where the new compound comes into play.
“Our novel compound revives suppressed BAX molecules in cancer cells by binding with high affinity to BAX’s activation site,” says senior author Dr. Gavathiotis. “BAX can then swing into action, killing cancer cells while leaving healthy cells unscathed.”
To hunt down the right compound that can boost the ability of BAX, Dr Gavathiotis and his team used computers to screen over a million different compounds that showed potential.
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They were then finally able to filter this even further until they had 500 compounds that met their requirements and eventually this was whittled down to just one.
“A compound dubbed BTSA1 (short for BAX Trigger Site Activator 1) proved to be the most potent BAX activator, causing rapid and extensive apoptosis when added to several different human AML cell lines,” says lead author Denis Reyna.
The researchers tested BTSA1 on human blood samples from patients which had high risk acute myeloid leukemia. Strikingly they found that the BTSA1 caused the AML cells to self-destruct while leaving the healthy cells completely untouched.
To take this a step further they then tested this on mice and found that just as before, the mice which were treated with BTSA1 not only had a longer survival rate but of those treated, 43% survived longer than 60 days and had no trace of AML in their system.
“BTSA1 activates BAX and causes apoptosis in AML cells while sparing healthy cells and tissues―probably because the cancer cells are primed for apoptosis,” says Dr. Gavathiotis.
The next step is to broaden their reach. The team are hoping to test BTSA1 on a wider ranger of cancers in the hopes that it will be just as effective.
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