New funding to advance novel PAH treatment from Australian scientists
Monash University project aiming to create disease-modifying therapy
Researchers at Monash University in Australia will use newly granted funding to advance their work on a project that aims to create a novel disease-modifying treatment for pulmonary arterial hypertension (PAH).
That funding came from Biocurate, a venture led by the University of Melbourne and Monash that aims to help translate promising treatment approaches from the lab into medicines that can be used by people. Awarded from Biocurate’s Proof of Concept (POC) Fund, the funding is expected to help the researchers identify molecules that may serve as treatment candidates.
“We’ve found that there are quite limited resources in Australia for translational projects,” Helena Qin, PhD, a translational pharmacologist at Monash who is leading the project, said in a university press release. “The POC fund targets early-stage therapeutic projects, which aligned perfectly with the stage of our project.”
In addition to providing financial support, Biocurate has lent its expertise in discussions with researchers about how to translate the therapy approach. That includes ensuring that any medication developed through the project can be brought to market in a commercially viable manner.
“We were very impressed with their thinking about both the scientific and commercial aspects of the project,” Qin said. “Some of those discussions were challenging – they really encouraged us to push our boundaries and think about whether we were taking the right approach.”
Project aims to create treatment to slow PAH progression
PAH is a disorder marked by high pressure in the blood vessels of the lungs. This puts extra strain on the right side of the heart, which is responsible for pumping blood to the lungs to pick up oxygen. Several treatments are available for PAH, but they are primarily symptomatic, not disease-modifying — in other words, such treatment helps to ease disease symptoms, but has minimal ability to actually slow the progression of PAH.
The new project aims to address this critical gap in care by identifying small molecules that can modulate proteins involved in blood vessel inflammation and remodeling, which are key biological processes in the progression of PAH. Specifically, the scientists hope to identify molecules to target particular G-coupled receptors.
G-coupled receptors are a broad category of protein receptors that play many roles in biological signaling. These proteins stick through cellular membranes, with a receptor portion outside of the cell that can detect specific signaling molecules and signaling apparatuses inside the cell that can change cellular activity when the receptor is activated. G-coupled receptors are a useful target for medicines because their activity can usually be controlled by small molecules designed to interact with the receptor portion of the protein.
The Monash project includes a diverse group of researchers who hope to combine their expertise to create a new medicine that can benefit patients.
“We all have the same alignment of goals and mission – delivering a disease-modifying therapeutic for this devastating disease,” Qin said, adding that the “process has brought us closer to our consumers and end-users, which feels really special.”
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