Dr Marian Sturm is a health scientist, with many years of experience working in a busy public hospital. Her main focus is the manufacture of therapeutic goods for patients. Marian is the Chief Scientific Officer for our resident startup Isopogen Pty Ltd and alumni of our “Entrepreneurial Mindset Boot Camp”. In addition to her role at Isopogen Marian is the Facility Director at Royal Perth Hospital’s Cell and Tissue Therapies WA (CTTWA) and an Adjunct Associate Professor at the Centre for Cell Therapy and Regenerative Medicine based at the School of Medicine and Pharmacology at UWA.
Can you summarise your specialised area of research/ field of research as well as your current project?
One of my most important contributions to the welfare of patients has come in the last ten years with the development of a stem cell therapy, which has broad applications across all the disciplines of medicine. It’s an adult stem cell that we obtain from a bone marrow from donors; we culture the cells, they are packaged and stored frozen to be delivered by a simple infusion into a patient. We’ve treated over 120 patients now and have performed about 550 infusions and the cells have proven to be safe. The wonderful thing about these cells is they modulate the immune system so they can be used to treat an array of immune disorders. These cells were developed for those patients who have had a bone marrow transplant with a graft from another person, and then the new graft has rejected their body and they get a nasty complication called graft versus host disease. So that’s why we developed the cells and they’ve proved very effective in bringing the condition under control.
What are the potential applications or impacts of your research in the wider community?
We’ve been trialling the cells in the organ transplant rejection setting and they have been shown to improve the outcomes of patients with lung rejection. So the lungs were coming to the end of their functional status and now we are commencing a clinical trial to use the cells in patients as soon as their lungs start to be rejected rather than waiting until their lung function has severely deteriorated.
We have also been using them in patients who have rejected their kidney and that too has had a good outcome. We’ve just completed another study where as soon as the patients have a kidney transplant they receive the cells in the hope they won’t go on to develop immune rejection of their organ and also to improve the immediate kidney function.
The other immune conditions for which the cells can be used are autoimmune disorders, such as Crohn’s disease, which is an inflammatory bowel syndrome where your body mounts an attack against its own gut. The outcome of that study has been quite remarkable. We would really like to progress treatment for these conditions further and there are plenty of others too that the cells could be applicable to.
The great thing about these cells is they are a universal donor cell so that you can put any one’s cells into anyone else’s body. They are better than a blood product as you don’t have to match them in any way. So they can be held in special freezers all around Australia, like a pharmaceutical off the shelf, then can be thawed and infused into anybody.
Why have you decided to commercialise and create a startup from your research/ technology rather than just publishing the findings?
We’ve done early phase I and some phase II trials, but there isn’t the funding in the public sector or even applying for grants to actually take it further and make it into a product that is available to all Australians. So really it needs commercialisation to access the large amounts of money need to conducted later phase III trials. Once they’re done, then we can apply for full licencing by the TGA and a listing on the PBS, so that there will be a reimbursement for the cell therapy. We’re sort of a cottage industry and we can only go so far, but the demand for the cells is quite great.
What are some of the problems you’ve faced or noticed on your commercialisation journey?
I think for the big picture, for the translational science, in terms of translating research into clinical therapies, there isn’t funding. There is very limited funding for new developments like this in the health system because Health is very activity based now for funding. This is really a kind of R&D, it is so exciting the field of cell therapies and tissue regeneration, but it’s actually hard to find the money to progress it. We’ve got some wonderful facilities, but the majority of the staff are funded on what’s called “soft money”, they’re not permanent positions. These are highly skilled, highly trained, competent staff members and I am always having to look for salaries through grants or other funding. We’ve got some other products in development but there is very limited funding for them. So I’d really like to see some more easy money become available for some of this basic R&D work.
What would you most like to achieve with your research?
I’d like to see us get to a position whereby our cell therapy is listed on the PBS. I hope they list it for a broad classification like immune modulation and, initially, as a life-saving treatment for many patients. I think once that happens the therapy will survive me. I’m very concerned as I am getting older and may not able to continually raise money to assist in the translation of this therapy that it may actually disappear with me. I’d like to see it cemented and available.
What has been the most important advice you’ve ever received?
One of the Professors I used to work with, Prof Taylor, he was a cardiologist, a pretty rough and ready character, and he always said the most worthwhile things in life are the hardest. You know, you don’t get the greatest satisfaction out of something that is given to you. You have to work at it and you get most rewards from actually working hard and getting there.
Is that true for your commercialisation journey so far?
Not sure yet! It’s costing me a lot of money and a lot of angst. But I’m very grateful to be here in CERI. I can tell you, it’s just a shame that the whole place hadn’t come into being many years ago. It would have made my journey a lot easier.
If you had unlimited funding, what research project (or startup) would you undertake?
I’ve got a wonderfully bright scientist that is working with me and there is a great cell therapy within reach. Really I’d like to hope that we might treat the first patient by the end of next year. It’s called CAR-T cell therapy. Basically, it’s modifying one of the patient’s type of immune cells by putting in a gene that recognises the particular cancer they’ve got. There is quite a lot of work being done around the world and the techniques are being improved all the time. We are developing our own piece of genetic material and it basically changes the immune cell so that it finds the cancer cells and knocks them off. That’s going to make a massive difference particularly to children who have cancer, because what it will mean is they won’t be receiving the chemotherapies or the toxic compounds to fight their cancers, which can do a lot of organ damage and leaves them at significantly higher risk of developing other cancers as they get older. This cell is a more friendly treatment, it’s not a chemotherapy. It would be delivered to the children, hopefully, knock out their cancers and cure them, and ultimately they’ll be in a better state.
So that’s one of the things I’d like to do. The other thing I’d like to do if I get enough time is to have a go at some of the genetic disorders in children. I’d really like to be able to establish a clinical therapy for those children with severe immunodeficiency syndrome by inserting the genes that correct their condition into their own stem cells and then transplanting the corrected cells back into them so that they will have the gene defect fixed.
What would you say to a fellow researcher considering commercialising their work?
The advice I always give them is come to see what clinical manufacturing is about early, not come to see us late in development because they need to be thinking of how they are going to get it to a position to be able to have a clinical product that gets past the regulator. If they don’t do that, they’ll never get anywhere. You’ve got to start and think about it right from the word go because it actually affects how you make a product. Otherwise, you might have a concept, but by the time you might be ready to translate, you might have to go right back to the beginning and change all your sourcing of your agents and parts of your procedures and re-perform all the validations etc. My advice is always to come and see someone in the field who knows how to do it, rather than just think they know because it’s another whole area most people don’t have knowledge about.
Which Western Australian researchers do you look up to, or admire most?
I think Barry Marshall and Robin Warren. I think they’ve got to be greatly admired, something terribly simple that they were passionate about and believed in. They had great conflict with other clinicians of the time and they absolutely revolutionised the treatment of gastric ulcers. I definitely look up to them and admire their great courage and perseverance. They took plenty of criticism but made it in the end. I think some great things have come out of Perth. I don’t even know the inventors but, for example, cardiac stenting came out of Royal Perth Hospital. People don’t generally know that and it’s used all over the world, that’s a wonderful thing. I think Steve Wilton and Sue Fletcher are right up there, all the years they put in, it’s so pleasing to see they’ve made it. I think they’ve done fabulous work, they worked really hard all their lives, it’s a narrow clinical application but probably the technology will have wider applications. Definitely, they’re just fantastic scientists.