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Realising high hopes for the new wave of medicine

21 September 2015

Regenerative medicine represents a new paradigm in healthcare, but with mini kidneys being grown in labs and titanium body parts being created with 3D printers, how close are we really to accessing ‘made to order’ body parts or combatting cancers in a significant way in Australia? 

Regenerative medicine treatments that modify cells to produce an immune response to cancer are showing exceptional promise in treating cancers like melanoma and myeloma. Other recent examples of regenerative medicine in Australia include, mini kidneys being grown in a lab, and a titanium heel was created with a 3D printer and successfully implanted to replace damaged bone. Reality is starting to resemble what we once thought of as science fiction in this new paradigm in human healthcare. 

A range of experts – including researchers, ethicists, and biotechnology companies - will gather at the upcoming AusBiotech 2015 national conference to discuss progress, developments and perspectives on regenerative medicine and stem cell therapies. The event includes a free public forum on stem cells, a one-day Regenerative Medicine Symposium, and a three-day conference program. 

Emeritus Professor Alan Trounson, from Melbourne’s Hudson Institute for Medical Research said: “The field of regenerative medicine is moving very quickly across a broad range of indications using a wide variety of cell types and drugs. The areas attracting most interest are cell therapies, tissue engineering and endogenous stem cell mobilisation.

“Perhaps the most interest presently is in the discovery of drugs and monoclonal antibodies for destroying cancer stem cells, gene editing of adult stem cells for genetic disease and the rapid escalation of companies involved in chimeric antigen receptor technology (CAR-T) being used to genetically arm Killer T cells (CD8+) to rapidly destroy cancer (blood and solid) tumours.” 

Dr Dominic Wall (CSO, Cell Therapies Ltd) is working on the practical considerations for delivering regenerative therapies on a larger scale, including maximising the effectiveness of supply chain, manufacturing and delivery to reach patient in need.

Dr Wall said: “Developments in T-cell immunotherapy, and extraordinary clinical outcomes, particularly in the area of chimeric antigen receptors (CAR-T), are changing the paradigm of how diseases are being treated. The clinical process for cell harvesting is painstaking and individualised, and the frontier is to be able to apply these therapies to large populations. Moving from treating a small number of patients, and the manufacturing and logistics considerations for doing so, will be challenging.”

Once a regenerative therapy is shown to be effective, getting the therapy to the patient (or market) is the final step of a long process. Dr Dawn Driscoll (Principal, DCi Biotech (USA)) will discuss market access considerations for translating these types of therapies from the research lab to healthcare market.

Dr Driscoll commented: “Regenerative medicine is an exciting new mode of therapy with huge potential. Results of trials in advanced stages of disease have been unprecedented and we are cautiously optimistic that some new treatments may be curative. These advances are fantastic, but somebody has to pay for these expensive therapies, and the payers will need to see justification for the costs. So the groups developing the therapies would do well to track not only the clinical aspects of the new medicines, but the economic factors too. These include the impacts of regenerative medicines on things like the length of time the patient is in hospital, the avoidance of more medical procedures, and getting patients back to their jobs and families.

“Some of the most promising results have been in children with devastating blood cancers. When we are able to get these young patients back on track, they can go on to live full lives and be socially and economically productive. So the big hope of regenerative medicine is that if we can save kids’ lives and free adults from years of traditional, debilitating treatments, then we’ve done something that is both clinically amazing and economically viable.”  

Rhenu Bhuller, Partner and Senior Vice President, Healthcare for Frost & Sullivan who heads the APAC healthcare practice for the firm, said: “Regenerative medicine represents a new paradigm in human health with the potential to resolve unmet medical needs by addressing the underlying cause of diseases. Although stem cell therapy will continue to be the largest segment of regenerative medicine, cross segment therapies that combine the use of immunology, genetic and stem cell therapy are rapidly advancing. 

“Regenerative medicine has been an area of interest for major pharma companies, many of which have set up their own R&D units or have invested in regenerative medicine companies. Globally there are over 700 companies with a regenerative medicine focus.

“Australia has started to develop critical infrastructure needed for research in this area and is also starting to gain ground internally, with partnerships that will enable knowledge sharing and hopefully accelerate discoveries in areas such as heart diseases, autoimmune diseases and musculoskeletal discoveries.” 

Ms Bhuller says this trend is expected to change as more stem cell therapy products for cancer and heart disease complete their clinical trials and are approved for patients to access.

Program Leader at Stem Cells Australia, Professor Martin Pera, who will open the conference’s Regenerative Medicine Symposium, said: “Novel innovative cell therapies for a wide range of medical conditions are now entering clinical trials. The data thus far are highly encouraging, but there are still many hurdles to cross. This conference will explore the progress and challenges facing the field as the stem cell revolution moves forward, and cell therapy emerges as the fourth pillar of health care along with drugs, medical devices and biologics.”

Professor Pera, who has made major contributions to the understanding of human embryonic stem cell biology, and Associate Professor Melissa Little (NHMRC Senior Principal Research Fellow, Murdoch Children’s Research Institute), who has created tiny kidney organoids in the lab using human stem cells, will explore how research is unlocking the potential of regenerative medicine. 

The AusBiotech national conference has been held regularly for almost 30 years and is the largest annual event for the biotechnology industry in Australia and the Asia Pacific Region, hosted by AusBiotech.

To access the full AusBiotech 2015 program, including the free public forum on stem cells, the one-day Regenerative Medicine Symposium, and the three-day conference program, please visit www.ausbiotechnc.org