Fast-tracking new aerosol treatments into clinical practice

Over the past decade, chronic respiratory conditions have been increasing in prevalence. Almost 7.4 million Australians (31% of the population) are now affected by respiratory disease, with respiratory infection and chronic obstructive pulmonary disease being the third and fifth leading causes of death, respectively. Despite a growing burden of disease, only a handful of new inhaled therapeutic products have successfully progressed to market. Only 27 such products have been approved by the USA’s Food and Drug Administration in the past five years, which is low in comparison to other health areas.

More recently, the COVID-19 global pandemic has placed a spotlight on respiratory research. The pandemic presents a challenge to rapidly identify effective drugs for prevention and treatment, and in this context, especially the development of targeted inhaled formulations. The pandemic has also highlighted the importance of sovereign capabilities for research and manufacture within Australia to meet similar challenges in the future.

While the pandemic is wreaking havoc around the world, it is an exciting time to be in respiratory research. My team works on several projects and we are exploring new technologies such as nanoparticles, mRNA vaccines and thermoresponsive gels. I also continue to work on establishing physiologically relevant in vitro methodological platforms using state-of-the-art technologies, including 3D printing, microfluidic chips and microsensors, to help streamline and fast-track development of novel inhalation therapies for improved patient care.

Engagement with industry is key to achieving a successful translational outcome.

Another exciting part of my work is working with national and international pharmaceutical and medical technology companies. Working with industry is different to working in academia. Companies are timeline-driven and milestone-oriented. Engagement with industry is key to achieving a successful translational outcome. It allows me to better understand what is required and how to navigate the complicated commercial pathways for taking therapies from the lab into the clinic. It is through these clinical impacts that my novel in vitro models have been recognised as the “gold standard” for studies on the interaction between pharmaceutical aerosols and human airways.

I knew early on in my career that the pharmacy profession was not for me, as I wanted to do more and be part of the process of creating the next cure or therapy that will make a difference to people’s lives. In this search for a new direction, I spent time interning at a pharmaceutical company, working in different departments, speaking to different professionals, and dabbling in research. It was here that I found my love for research – having the freedom to think outside the box and test new ideas.

I used the knowledge I gained in pharmacy to pursue research on developing these new inhalation pharmaceutical therapies. However, I always had a keen interest in biology, and particularly the mechanisms of the human body. Now my research integrates both by developing representative respiratory cellular models as new tools to study how “real” aerosols interact with the airways. My ultimate goal is to make safer and more effective therapies.

My PhD studies showed that the efficacy of inhaled therapies depends not only on the successful generation of small aerosol particles, but also on how those particles interact with biological barriers in the airway. I identified fundamental factors influencing particle transport, toxicity and efficacy – knowledge that can be used to inform better design and optimisation of inhaled therapies.

After my PhD, I broadened my research in the UK, applying my model to study nasal formulations and investigate how aerosols interact within the nose. I have built on this work at the Woolcock Institute of Medical Research, where I have researched how to use the nose as a portal for the treatment of various local and systemic diseases.

Academia and research are going to face a very tough road ahead if they are to retain talent and to continue to make breakthrough discoveries.

The models I have developed provide a more realistic measure, in a laboratory setting, of drug and formulation performance than the conventional in vitro “glass” model.

With my experiences in the pharmaceutical field and interactions with various industry partners, it was obvious that there was a severe shortage of skilled workers in this area in Australia. Recognising this gap, I developed and directed an industry-driven Pharmaceutical and Medical Device Development program at the University of Sydney. This program is designed to meet the current industry needs for highly trained and work-ready graduates.

Research is not always a straightforward and easy road. I think resilience in this field is key! Unfortunately, this may, at times, also not be sufficient, as the ability to perform research and manufacture therapies is fast disappearing in Australia due to limited research funding and the outsourcing of manufacturing to other countries. As a young emerging leader, female and a mother, juggling different responsibilities and the pitfalls of balancing work and family life, securing grant funding is even more difficult. Academia and research are going to face a very tough road ahead if they are to retain talent and to continue to make breakthrough discoveries.

I have always believed it is important to look for an alternative research income stream. With two of my colleagues, I helped set-up Ab Initio Pharma, a young and dynamic SME (small to medium enterprise) funded by MTP Connect. Based at the Royal Prince Alfred Hospital in Sydney, Ab Initio has state-of-the-art GMP (good manufacturing practice) manufacturing and R&D capabilities to help entrepreneurs, university academics and start-ups translate their concept from bench to clinic. Ab Initio will help build Australia’s sovereign capabilities and create a vibrant and sustainable pharmaceutical ecosystem.

I look forward to continuing to pursue my passion in research and supporting my team in research and commercialisation.