Greek professor in Canada’s Alberta medical university, Dr. Evangelos Michelakis is co-leading a study introducing a novel drug which shows promising results targeting mitochondria to fight Pulmonary Arterial Hypertension (PAH), a debilitating disease of the lung blood vessels that causes heart failure and early death.
Michelakis is part of a team of investigators at the University of Alberta (Edmonton, Canada), and the Imperial College of Medicine (London, UK), that reported promising results of an early-phase clinical trial with a novel drug in PAH patients, already under treatment with approved drugs.
In this week’s issue of Science Translational Medicine, the investigators showed that the generic drug, Dichloroacetate (DCA), can decrease the blood pressure in the lungs of PAH patients and improve their ability to walk, without significant side effects at the doses tested.
DCA works by activating mitochondria, the energy-producing units of the cell, which also regulate the cell’s fate (i.e., whether the cell can grow or die by self-destruction), a process called apoptosis. Mitochondrial function is suppressed in PAH. This allows the cells lining the lumen of lung vessels to grow and avoid apoptosis.
The resulting overgrowth of cells narrows the lumen of vessel, making the heart work harder to push blood through for oxygenation. This overgrowth of cells resembles the growth of cancer cells, where mitochondrial function is also suppressed and DCA has shown promise as a potential cancer treatment.
“This is the first time that a drug targeting mitochondria is shown to be effective in patients with PAH” said Dr. Michelakis.
“Another important aspect of the study is that DCA is a generic drug, and thus the study was not sponsored by a pharmaceutical company, but from public funds and donations. This means that if future studies confirm our results and show a clear and definitive benefit of DCA in PAH, we may have an affordable, cheap treatment available to all,” said Michelakis.
Source: Medical Xpress