New Drug That Selectively Targets Blood Cancer Cells is Set for Human Trials


The drug, PCLX-001, is expected to have minimal adverse effects in treatment of patients with blood cancers.

A new precision cancer drug for blood cancer that selectively interferes with cancer cell signaling is set for human trials, according to study published in Nature Communications.

The compound, known as PCLX-001, targets enzymes that perform myristylation. This is a cellular process in which fatty acid myristate modifies proteins, allowing them to move membranes and become part of the cell signaling system, according to the study.

Investigators tested the drug compound against 300 different cancer cell types. The drug was most effective in combating blood cancer cells, including lymphomas and leukemia, which have fewer of the enzymes, according to the study. The drug also killed other cancer types at a higher concentration.

The drug stopped B-cell lymphoma tumor survival signals, as well as killed B-cell tumor cells, in both animal and test-tube experiments, while leaving non-cancerous cells unharmed, according to the study.

"We think PCLX-001 is a compound with a large therapeutic window that can kill the cancer cells at a much lower concentration than what is needed to kill normal cells...That is the holy grail of cancer therapies," research team leader Luc Berthiaume, PhD, said in a press release.

"Because of the highly selective nature of our drug, it's often referred to as a precision medicine, and we anticipate minimal side-effects.”

The drug is set for phase 1 human trials in patients with lymphoma, leukemia, breast, and colon cancers at the Cross Cancer Institute in Edmonton, the BC Cancer Centre in Vancouver, and the Princess Margret Cancer Centre in Toronto later this year.


New blood cancer treatment works by selectively interfering with cancer cell signalling [News release] October 22, 2020; Alberta, CA. Accessed October 22, 2020.

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