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Cell transplantation technologies are promising treatments for many conditions. Our expert panel discussed how to overcome the barriers preventing widespread adoption.
Cell transplantation is an emerging technology with great potential to treat cancer, cardiovascular diseases and neurological conditions.
While drugs focus only on treating symptoms or slowing disease progression, a cell therapy could additionally reverse the course of disease. As one-off treatments, they minimise side effects compared to ongoing drug treatments.
However, cell transplantation technologies still face significant barriers that prevent widespread adoption. Hosted by Professor David Nisbet for the Faculty of Engineering and Information Technology MedTech Platform, in ‘Overcoming clinical barriers to cell transplantation technologies’ a panel of experts discussed how to overcome barriers in the availability, quality and cost of treatments and methods to combat the immune response.
Stem cells aren’t a miracle cure
Are patients open to cell transplantation? Professor Megan Munsie, who leads research programs into ethical, legal and social implications of stem cell research, said: “The Australians that we’ve spoken to are extremely enthusiastic. In fact, so enthusiastic that they’re frustrated in the lack of opportunities to participate in clinical research.”
Some international clinics prey on patients’ hopes by advertising cell-based treatments for their conditions that aren’t substantiated by science.
Cell products should be backed by a solid biological mechanism – science that describes how they work.
“It really is not a ‘stem cell cures all’ sort of approach,” said stem cell researcher Professor Clare Parish from the Florey Institute of Neuroscience and Mental Health.
Barriers to cell therapy development and manufacture
Developing cell therapies takes time and money. Panellist Dr Richard Boyd’s cell therapy for ovarian cancer has taken $40 million and around seven years from lab bench to beginning clinical trials.
Australia lags behind the United States in suitable facilities for manufacturing cell products, Dr Boyd, who is Chief Scientific Officer at Cartherics, said. Working with GMP Pharmaceuticals, his team scouted more than 100 sites before settling on renovating old 7-Eleven corporate offices to create brand new laboratories and clean rooms.
“We need more support for translation,” said Dr Boyd.
The need for specialised manufacturing facilities and sophisticated transportation also create barriers for access to low and middle-income countries, Professor Munsie said.
Platform technologies would increase the availability of cell therapies.
“Things like stealth or cloaked cell lines … will enable us to do bulk manufacturing and cryopreservation of cell products that can be disseminated around the world,” said Professor Parish.
A matrix of regulators for cell products
Who regulates cell products in Australia? As a manufactured therapeutic product, cell therapies fall under the Therapeutic Goods Administration. However, the Office of the Gene Technology Regulator has oversight over gene technologies, including genetically modified cell therapies. Finally, the Australian Health Practitioner Regulation Agency regulate all clinical practice in Australia.
Australian regulatory bodies may need educating about cutting-edge technologies so that they’re able to make informed, ethical and appropriate decisions on cell therapies, Professor Parish said. Dr Boyd added that the US Food & Drug Administration are considered the gold standard for cell therapies – as well as representing one of the largest markets in the world.
Barriers to cell therapies in the immune system
A patient’s own body also presents a barrier to cell therapies.
“Transplantation rejection has been a long, long, long, long history of failures in many ways,” said Dr Boyd.
“The traditional way [to overcome it] is to immune suppress the patient, which has obvious benefits in retaining the graft, but it introduces a whole swathe of other problems – susceptibility to infections and so on. It’s kind of a Stone Age technology, though currently still necessary.”
New technologies add flexibility. Getting the closest genetic donor match to a host minimises problems. Thanks to genome-editing tools like CRISPR, researchers can also genetically modify cells so that they’re not seen by a patient’s immune system.
The future of cell transplantation
The panel agreed that treatments for conditions with a single type of affected cell – like Parkinson’s disease or eye conditions – are a likely candidate for success.
“It’s that defined cell population and focal delivery. As soon as it’s many cell populations or across large or diffuse areas, it’s a much, much greater challenge for us to manage,” said Professor Parish.
CAR-T cell therapies for blood cancer are particularly promising.
“When you hear the word ‘cure’ being openly used by clinicians, then you have to start sitting up and taking notice,” said Dr Boyd.
Learn more about MedTech research
First published on 24 April 2023.
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