A new drug to treat chronic lung disease that is easier for people to take has entered human clinical trials.
The need
The burden of taking multiple oral medications can be a daily struggle for people living with chronic lung disease.
Sometimes this burden is worsened by adverse effects in the gut that cause patients to stop the treatment altogether. Researchers at the University of Melbourne are trialing a new treatment, TLB001, that delivers medication by aerosol to minimise adverse effects.
Initially targeting lung fibrosis, the new treatment could also benefit patients with chronic asthma, chronic obstructive pulmonary disease (COPD), and lung cancer.
As they commence Phase 1 clinical trials on humans, the research team hopes the new drug, TLB001, will reduce the adverse effects of anti-fibrotic medication for patients with Idiopathic Pulmonary Fibrosis (IPF) by delivering it directly into the lungs.
About chronic lung disease
IPF is a chronic lung disease that affects approximately 100,000 people in the US and 9,000 in Australia.
Early in the disease symptoms can be mild, which can make it difficult to detect, and this leads to a delayed diagnosis.
The disease is typically fatal within 3-5 years of diagnosis, and current forms of medication can cause severe nausea, vomiting, diarrhea. These side effects can limit quality of life to such a degree that patients decide to stop treatment altogether.
TLB001 avoids these gut effects by being delivered directly to the site of the disease in the lungs.
The Australian Lung Foundation recognises the impact of chronic lung disease with the expression ‘if you can’t breathe, nothing else matters’.
Making an impact
Professor Alastair Stewart from the University of Melbourne’s Department of Biochemistry and Pharmacology says the first 'in human' trial of TLB001 is a "significant step towards relieving the distress and slowing the progress of lung scarring in chronic respiratory disease."
“This milestone marks an advance in our team's efforts to translate many years of foundational work into new and more effective treatments for IPF."
Research collaborator Professor Spencer Williams from the School of Chemistry says the team overcame many technical challenges to manufacture the drug to the exacting standards necessary to support the clinical trial.
“In the trials, healthy volunteers will inhale TLB001 aerosol, allowing the drug to directly reach their target lung tissue. The trial is assessing the safety, tolerability, and pharmacokinetics of single and repeat doses of TLB001 administered via inhalation to healthy volunteers."
People
Professor Alastair Stewart, Department of Biochemistry and Pharmacology and Director of the ARC Centre for Personalised Therapeutics Technologies, is Principal Investigator of the research, along with medicinal chemistry support provided by co-Principal Investigator Professor Spencer Williams, School of Chemistry.
Funding
This research is being undertaken by TianLi Biotech, a joint venture between the University of Melbourne and Beijing No.1 Biotechnology Group Co., Ltd. The foundational work for the TianLi Biotech clinical program has been funded by the NHMRC, the ARC, University of Melbourne, the Kaye Merlin Brutton bequest to the Faculty of Science, Asthma Australia, the Victorian Government VMRAF scheme and Therapeutic Innovation Australia. Dr Bowen Zhang (Board Chair) and Dr Yan Han (CEO) have provided critically important and extensive financial, clinical, scientific and business support to the enterprise.
First published on 12 September 2024.
Share this article