New discovery could slow Parkinson’s disease and reduce life-threatening lung injury

Scientists at Florida International University have developed a promising new compound that could slow the progression of Parkinson’s disease and protect against life-threatening lung injury by targeting a key process inside cells.

The research centers on dynamin-related protein 1 (DRP1), which helps control how mitochondria – often described as the powerhouses of cells – divide and function. When DRP1 becomes overactive, it can lead to impaired mitochondrial function, inflammation and cell damage linked to disease.

In preclinical tests, the compound, CTS2444-32, reduced DRP1 activity by up to 45%, helping limit damage in both brain and lung disease models.

Kim Tieu, a professor of environmental health sciences at FIU Robert Stempel College of Public Health & Social Work, has spent years studying DRP1’s role in Parkinson’s, a disease marked by tremors, stiffness and the loss of brain cells. He collaborated on the discovery with Stephen Black, director of the FIU Center for Translational Science  and associate dean for research at the Herbert Wertheim College of Medicine (FIU Medicine), who is an expert in acute lung injury, and Adel Nefzi, a medicinal chemist at FIU Medicine.

“DRP1 plays an important role in mitochondrial division,” Tieu said. “But when it gets too active, it causes the mitochondria to split more than they should, which can lead to mitochondrial dysfunction, inflammation and cell death – key factors in how Parkinson’s disease progresses.”

In Parkinson’s models, CTS2444-32 reduced inflammation in the brain and lowered levels of toxic proteins such as alpha-synuclein, which are closely linked to the disease.

“Unlike current treatments that simply replace the missing chemical to relieve symptoms, this compound has the potential to actually protect brain cells and slow down how Parkinson’s disease progresses,” Tieu said.

The compound also showed strong results in models of acute lung injury, a serious condition that can occur during illnesses like sepsis or when patients require ventilators. In these cases, the lungs can fill with fluid, making it difficult to breathe.

In the study, CTS2444-32 reduced inflammation and protected mice from ventilator-induced lung injury (VILI) within 24 hours.

“We saw reduced levels of inflammatory molecules in lung fluid, which tells us this compound does a great job stabilizing mitochondria and protecting cells from harm,” Black said.

The compound was identified by screening a library of 35 million compounds. Researchers said the compound could have even broader potential.
 “This compound targets multiple disease pathways with a single drug, offering hope for conditions beyond Parkinson’s and lung injury, such as Alzheimer’s, heart disease and even certain cancers,” Nefzi said.

The team has secured a patent and is now working to refine the compound so it can be safely used in humans. Additional studies are underway, including testing in larger animals to meet FDA requirements. Clinical trials could begin within a year.

If successful, the treatment could improve outcomes for patients. People with Parkinson’s may see slower disease progression, while those with severe lung injury – conditions that can carry mortality rates of 30% to 40%, such as acute respiratory distress syndrome – could have a better chance of survival.

“This is about saving lives,” Black said.