Tag Archives: Australian health

gene therapy

From cell to accessible therapy: the future of regenerative medicine

The cell and gene therapy industry is the fastest growing sector of regenerative medicine. Commercial cell therapies are being developed to treat several major diseases, including cardiovascular disease, cancer and autoimmune conditions. However, developing and manufacturing cell therapies is lengthy, labour intensive and expensive.

The CRC for Cell Therapy Manufacturing (CTM CRC) began in 2013, operating at the interface of cell biology and materials science. The CRC aims to help the cost-effective manufacture of cell therapies and assist their rapid translation into clinical practice.

CTM CRC’s research programs are driven by commercial imperatives and initially brought together 15 participant organisations across four states, including two international companies. That approach has led to the development of new immunotherapies and novel materials and surfaces to optimise cell and gene therapy manufacture.

From the outset, CTM CRC has focused on developing strategies to ensure its work continues beyond the funding period. “With two CTM CRC legacy vehicles to continue the excellent work carried out to date, the strategy to transition towards self-sufficiency has paid off,” says CTM CRC CEO

Dr Sherry Kothari. The CRC has incorporated its first spin-out company, Carina Biotech, and a second company, TekCyte, will also soon be incorporated. Both Carina and TekCyte will further develop and commercialise CTM CRC technologies, and are poised to continue the CRC’s work of making cell therapies more affordable and accessible.

Carina Biotech — A promising future for cancer treatment

In the last five years, researchers have achieved promising results in clinical trials of a revolutionary new treatment for blood cancers called Chimeric Antigen Receptor (CAR)-T cell therapy. CAR-T cell therapy is an immunotherapy that harnesses the patient’s own immune system to fight their cancer.

Since 2012, CAR-T cell therapy trials in adult and paediatric patients have recorded complete remission rates of up to 93%, offering huge potential for leukaemia and lymphoma treatment. The replication of this success in the treatment of solid cancers is a new focus of this approach, and it’s also the basis on which the CRC for Cell Therapy Manufacturing (CTM CRC) company, Carina Biotech, was founded.

“To effectively translate the unprecedented cancer-killing activity of CAR-T cells in blood cancers into solid cancers would represent the Holy Grail in the cellular immunotherapy industry,” says Dr Justin Coombs, CEO of Carina Biotech.

T-cells, the backbone of CAR-T cell therapy, are the ‘warriors’ of the immune system and they attack undesirable cells in the body. CAR-T cell therapy involves isolating a patient’s T-cells from a sample of blood and engineering them so they recognise and attack specific markers on cancer cells. These new CAR-T cells are then infused back into the patient to seek and destroy the cancer.

Carina Biotech’s first lead technology in cell and gene therapy research is a CAR-T cell that attacks a cancer-specific marker on solid cancers, but not on healthy cells. Early data indicates these CAR-T cells can kill a diverse range of solid cancer cells in vitro, including breast, ovarian and brain cancers and melanoma. Pending positive results from in vitro pre-clinical studies, slated to begin in 2018, the first-in-human clinical trials could follow within two years.

It is clear there is great potential for CAR-T cell therapy to play a leading role in the race to cure cancer, but as Dr Coombs cautions, “Solid cancers are shaped by evolution to defend themselves from attack. Carina is aiming to develop weapons for immune cells to destroy all solid cancers.”

TekCyte — Moving rapidly from lab to commercial scale

TekCyte, the translational facility of CTM CRC, was set up to respond to manufacturing challenges in the evolving cell and gene therapy industry. TekCyte’s focus is to translate technologies from the lab to pilot scale.

“Pilot-scale manufacturing is where many technologies stall because they cannot be replicated in commercial settings,” says Dr Tony Simula, who leads TekCyte with Dr Andrew Milligan. “There are unique challenges in scaling up processes involving living cells and TekCyte addresses these as an important step towards commercial manufacture of cell therapy products.”

TekCyte is currently validating two CTM CRC technologies for the commercial market: the delivery of stem cells for the treatment of chronic wounds, and an antithrombotic coating for vascular stents to reduce thrombosis and restenosis. With positive preclinical data to date, it is imperative that TekCyte is able to consistently produce both products in large volumes, as well as meeting stringent regulatory requirements and demonstrating reliable performance. TekCyte’s infrastructure and expertise enables it to fulfil this critical translational role so it can bridge the gap between the laboratory and commercial development.

“TekCyte is unique because it combines materials surface and cell biology expertise, with the know-how and infrastructure required to manufacture at pilot scale,” says Dr Milligan.

“This capability has given TekCyte a competitive advantage and enables it to expand its offering to include product development for companies.”

TekCyte aims to establish itself as an important player in the global supply chain for the regenerative medicine industry. It is evolving into a world-class translational facility, able to develop and supply specialised coatings and processes for cell and gene therapy manufacture and other biomedical applications.

 

science policy

Make your expertise available

Featured image: President of Science & Technology Australia, Professor Jim Piper (left), hosts a meeting between Science meets Parliament delegates and Prime Minister Malcolm Turnbull (centre) in 2016

Darren, what’s your particular area of research and how can it help to inform policy in Australia?

I am a medical researcher, working to understand the biology of cancer and neurodegeneration, and use that knowledge to design new therapies. Both diseases have a huge health and financial impact in Australia and internationally, and with an ageing population this impact will only increase, with obvious implications for health funding and policy.

When you first attended Science meets Parliament, how did you prepare for your research pitch?

I really didn’t know what to expect so I was actually pretty underprepared. I won’t make that mistake this time!

Did your pitch have the desired outcome? What would you do differently next time?

I had a great discussion with a Greens senator from Western Australia who had a strong interest in environmental issues. We talked about the importance of science in understanding the environment and gathering data as a foundation for drafting good evidence-based policy in areas such as fisheries management and forestry. In some ways I didn’t really have to do much convincing! 

This time I plan to research the electorate of the parliamentarians I’ll meet and the issues that might be important in that context. I’ll make sure I understand the issues they have flagged as important to them and think about how my background and research interests might align with those issues. I also plan to ask them questions to find common ground for discussion.

Describe your experience at Science meets Parliament (SmP). What did you think of the event?

I was really enthused by SmP, and impressed by the level engagement of the politicians and policymakers who attended. I found it an invaluable learning experience and a fantastic opportunity to meet scientists across a broad spectrum of specialities.

Seeing the workings of government up close (if only briefly) was a real eye opener and the various briefings and workshops were constructive and informative. I still draw on the things I learnt there.

In many ways it was a catalyst to me becoming much more interested and active in science policy and communication.

What advice do you have for other researchers who are trying to turn their knowledge into action?

Keep a constructive mindset and focus on how science might help, rather than just presenting a list of problems or complaints.

Listen to the concerns and issues that are important and make yourself available as a source of expertise and advice on the process and outcomes of science by fostering relationships.

Be aware that politics and policy development work to different timelines and use different language to science.

Try to take a bipartisan approach.

What have been the major challenges in getting your science heard by policymakers in Australia, and how have you overcome them?

The most difficult barriers to progress have been the relatively regular turnover of ministers, a challenging funding environment (which always seems to dominate discussions) and hostile attitudes to evidence and rejection of “expertise” in some quarters. 

Overcoming these is really challenging and incredibly time-consuming. My approach is to attempt to build dialogue wherever possible, and to be proactive in making science relevant and interesting to the general public.

I take every opportunity I can to tell people about the outcomes and process of science. Public support for science might eventually translate into it being heard at the policy level.

How do you think the relationship between science and politics in Australia compares with other countries, and what lessons could we take from overseas?

I believe we can learn a lot from other countries. For example, we could benefit from aspects of science and policy partnering schemes employed in the UK, science diplomacy schemes in the US, and the appointment of ministers with relevant experience and qualifications in places like Canada.

Most government departments in the UK have a Chief Scientific Adviser (CSA) to provide scientific advice and PhD students can undertake three-month internship placements in the Government Office for Science.

The American Association for the Advancement of Science (AAAS) have a Centre for Science Diplomacy which aims to use to promote scientific cooperation as an essential element of foreign policy.

What are you most looking forward to at Science meets Parliament this year, and what do you hope to see more of in the future?

I look forward to meeting interesting and driven people, gaining new insights and hopefully gaining some traction with politicians about the importance of science and its ability to help drive the health and prosperity of Australians.

Click here to find out more about Science meets Parliament.