While cancer treatments are becoming more effective every day they still leave a lot of room for improvement. Most treatments for common types of cancer come with significant side effects that can cause the patient deep discomfort or even lead indirectly to death due to a compromised immune system. Researchers from the Albert Einstein College of Medicine may have found a compound that kills cancer cells without damaging healthy cells.
The researchers tested a BAX Trigger Site Activator 1 (BTSA1) molecule on acute myeloid leukemia (AML) cells. The researchers made a startling discovery. The BTSA1 molecule “promptly induces” a form of cell suicide called apoptosis in AML cancer cells. In other words the molecule kills cancer cells without damaging healthy cells.
The new compound works by targeting a so-called “executioner protein” in cells called BAX. The BAX proteins overwhelm the cells mitochondria and punch holes in the cell membranes, effectively killing them. Cancer cells are able to evade this effect by producing large numbers of Anti-apoptotic proteins that inhibit BAX molecules.
Once the researchers discovered the BAX site they attempted to find a way to overcome the cancer cell’s defenses. They did this by using computers to screen over a million different compounds that might bind to a BAX site. This allowed them to narrow their selection down to the 500 most promising candidates. Of these BTSA1 was the most promising. Using blood samples from patients suffering from high risk AML the researchers found that it killed AML cancer cells while leaving the stem cells alive.
The researchers then tested the effectiveness of BTSA1 in mice. They infected mice with AML using grafted human cells. They found that mice treated with the BTSA1 molecule has a significantly better survival rate than the control group. Untreated mice survived around 40 days whereas treated mice survived an average of 53 days. Even more startlingly 43% of mice survived longer than 60 days and showed no signs of AML. The treated mice also showed no signs of toxicity, meaning that the treatment could well have less negative side effects than traditional chemotherapy treatments.
The researchers believe that the treatment is effective because cancer cells are more vulnerable to apoptosis. This means that the increases in BAX are harmless to healthy cells but will overwhelm the cancer cells.
The treatment is still in early testing and a lot can change between trials on animals and trials on humans but it is another promising step forward in the fight against cancer. The researchers hope the molecule that kills cancer cells without damaging healthy cells can be combined with other treatments in order to create safer cancer cures and hopefully increase survival rates for AML and other kinds of cancers.