National Cancer InstituteCancer treatment is an added challenge for those already facing the diagnosis, but new research could bring significant changes.
Luc Berthiaume, chief scientific officer at Pacylex Pharmaceuticals and a professor in the department of cell biology at the University of Alberta, has led his research team at Pacylex to develop a new class of drugs. These drugs are N-myristoyltransferase (NMT) inhibitors that specifically target and kill cancer cells, reducing unnecessary damage to healthy cells.
All cells, including cancer cells, require adenosine triphosphate (ATP) to function. When oxygen is present, ATP is mainly produced through a metabolic process called oxidative phosphorylation — which typically involves fat burning. When oxygen is unavailable, cells instead burn glucose via glycolysis to produce the necessary ATP. According to Berthiaume, Pacylex’s drug inhibits both of these pathways, essentially starving cancer cells.
Berthiaume expressed that a significant feature of this drug is that it specifically affects cancer cells more than normal ones. It inhibits the enzyme NMT, which is essential for cell viability, and exists as a pair in all cells.
However, only one NMT is needed for survival — very much like kidneys, Berthiaume said. He also mentioned that in cancer cells, one of the NMTs is usually already lost, therefore a drug that removes the remaining NMT will kill cancer cells, while normal cells will survive. For Berthiaume, this was the key discovery and point of attack in Pacylex’s anti-cancer strategy.
Clinical trials have shown “90 per cent growth inhibition of the solid tumour”
According to Berthiaume, clinical results have been promising. Pacylex’s new drug has proven especially effective against various types of blood cancer, such as lymphoma and leukemia. In mice with five different blood cancers, Berthiaume’s team “saw [the] eradication of the tumors.”
Other types of cancer, including testicular, lung, breast, and brain, have all shown sensitivity to the drug, suggesting potential for treating solid tumors as well. According to Berthiaume, “we see 90 per cent growth inhibition of the solid tumor.”
The treatment has also been effective in human trials. In their phase one clinical trial, Berthiaume’s team received a multitude of patients who only had about three months to live, many of whom had already tried all pre-existing treatments.
On NMT-inhibitor drugs, numerous patients lived past their prognosis, some even reaching 16 to 18 months. “Not only did we show that the drug is safe at the maximum tolerated dose in people, we showed that it is also efficacious,” Berthiaume stated.
Berthiaume has been conducting this research for 10 of the last 29 years that he has been at the U of A. While conducting this research, monetary challenges were not uncommon, Berthiaume said.
Funding is difficult “if you work on something that is outside the box, or something more unusual,” Berthiaume stated. “Never give up hope, but always have a plan to make your hope or desire come true.”
Berthiaume acknowledged that when a company is developing a new drug, financial risks are high. It is not an easy market, as many pharmaceutical giants hold cornerstone positions, he said. Despite this, Berthiaume is confident in his company — this confidence is based on positive empirical results.
“Always learn, push yourself to the limit, and become all you can be,” Berthiaume said.
