Breakthrough IBD treatment clinical trial passes milestones

A clinical trial delivering hope to people living with inflammatory bowel disease (IBD) has reached an important milestone, with initial results looking very positive.

IBD is the collective term that includes Crohn’s disease and Ulcerative Colitis – both inflammatory conditions of the digestive tract, often affecting the bowel.

The trial is testing a breakthrough treatment for one of the most difficult to treat conditions associated with IBD – complex perianal fistulising Crohn’s disease – a debilitating condition in which abnormal connections form between the lumen of the bowel and other sections of bowel, vagina, uterus and skin, causing faecal incontinence, recurrent severe infections, chronic inflammation and tissue damage.

Until now this condition has been largely incurable. Patients suffer from pain, infection, incontinence and poor mental health while the health system incurs significant economic costs with maintenance treatment.

The therapeutic candidate being tested in this trial, pioneered by Associate Professor Rebecca Lim and her team at Hudson Institute of Medical Research, uses extracellular vesicles (EVs) derived from human amniotic epithelial cells (hAECs), termed hAEC-EVs. hAEC-EVs  are nano-sized particles with anti-inflammatory, anti-fibrotic and pro-regenerative properties.

IBD treatment clinical trial reaches the half-way point

Now, in collaboration with Monash Health and Monash University, the clinical trial has passed the half-way point in patients treated.

Dr Charlotte Keung is leading the trial, using her expertise as an IBD specialist gastroenterologist at Monash Health and early career researcher at Monash University, and member of the Amnion Cell Biology Research group at Hudson Institute of Medical Research.

This is the first use of hAEC-EV therapy for Crohn’s disease at a significant scale worldwide and the second human EV trial in Australia. hAEC-EVs are administered via local injection to participants with complex refractory Crohn’s perianal fistulas.

Dr Keung says so far, the results have been positive and there have been no serious adverse events.

“We have achieved two milestones – we have treated more than 50% of targeted patient numbers on the trial and further we have successfully recruited more than 75% of our target number patients to the trial,” she said.

“It’s a debilitating condition with very few treatment options, so our goal is to increase access to effective treatments by developing a therapy with significant manufacturing cost reductions

“hAEC-EV therapy reduces the significant manufacturing, cold chain logistics, clinical administration and cost limitations associated with live cell treatment.”

1/3 of Crohn’s patients could benefit from this treatment

According to Global Data Crohn’s Disease has a current total market size of $9.2 billion expected to grow to $13.4 billion by 2026. One third of Crohn’s patients will develop fistulas.

Hudson Institute’s CEO and Director, Professor Elizabeth Hartland AM, said the primary aim of the trial is assessing the safety of the treatment candidate. The secondary aims include investigations to assess whether it is effective in healing perianal fistulae and improving patient quality of life. These aims will help researchers to further optimise the therapy.

This exciting regenerative therapy trial is overseen by internationally recognised clinician-scientist Associate Professor Greg Moore, who heads a large multidisciplinary IBD Unit at Monash Health. He is active in basic and translational research, clinical trials, education and clinical medicine, with a proven record in research infrastructure development. He is a director on the board of Crohn’s and Colitis Australia.

Exosome BioSciences P/L

Exosome BioSciences P/L is driving the trial, confident that its novel hAEC exosome platform is highly effective in pre-clinical models over a range of conditions including bronchopulmonary dysplasia, and stroke, as well as pulmonary, liver and kidney fibrosis.

Exosome BioSciences Pty Ltd is a spin-off company established in 2023 to commercialise a portfolio of Intellectual Property (IP) developed by Hudson Institute of Medical Research, Monash University and Monash Health. The company has an exclusive license for the relevant IP including Patent applications, trade secrets and proprietary materials.

In 2023 Exosome BioSciences was awarded a $1.5 million grant from CUREator to develop novel exosome therapies to fund this clinical trial. CUREator is an Australian biotech incubator delivered by Brandon BioCatalyst with funding from the Australian Federal Government’s Medical Research Future Fund.

What are extracellular vesicles?

EVs are small, membrane-bound particles released by almost all cell types. They play a crucial role in intercellular communication by transporting proteins, lipids, and nucleic acids between cells.

Depending on the cell of origin, EVs can play a role in:

• Cell communication: EVs facilitate communication between cells by transferring bioactive molecules, which can influence the behaviour of recipient cells.

• Immune response: Bioactive molecules may include anti-inflammatory cargo from parent cells. These can modulate the immune response to potentially reduce a pro-inflammatory environment.

• Therapeutic potential: EVs are being explored for their potential as a regenerative medicine delivering pro-regenerative cargo to damaged cells or tissues.