'This is an unproven theory." – Prof John Rasko'If you were the one sitting in a wheelchair, what would you do?" - Matt Battista
Airdate: 
Tuesday, July 15, 2014 - 20:30
Channel: 
SBS

If an experimental treatment existed for your illness – would you try it?

Thousands of desperate Australians are turning to controversial, unproven stem cell treatments in the hope it cure a whole range of illnesses.

They say they’re tired of waiting for the long process of clinical trials to conclude, while they get sicker and sicker.

An exemption in Therapeutic Goods Administration regulation allows Australian doctors to treat patients with their own stem cells. Stem Cells Australia believes over 40 local doctors now offer treatments that haven’t been rigorously tested.

Other patients are heading overseas for risky treatments that are strictly limited here in Australia. Some of them are returning back to Australia with startlingly successful results. Others have no improvement.

This week, Insight asks whether current stem cell treatments are cause for hope – or just misleading hype.

Presenter: Jenny Brockie 
Producer: Hannah Meagher 
Associate Producer: Amanda Xiberras 

Join the discussion by using the #insightsbs hashtag on Twitter, posting on our Facebook page

Transcript

JENNY BROCKIE: Welcome everybody, good to have you with us tonight. Phillip, four years ago how well could you see?

 

PHILLIP IRONFIELD: Jenny, I couldn't see very well at all. I had a growth, there was a failure of the stem cells and so like a veil, the covering on my iris was gradually coming down in an opaque sense.

 

JENNY BROCKIE: And this was your right eye?

 

PHILLIP IRONFIELD: It was my right eye and things were getting dimmer and dimmer.

 

JENNY BROCKIE: Now you had to retire early because of this that is right?

 

PHILLIP IRONFIELD: Yes, I did and then I subsequently went back to work.

 

JENNY BROCKIE: Tell us what you had done to your right eye in 2010?

 

PHILLIP IRONFIELD: I had my cornea scraped off and then I had stem cells that were taken from my left eye and grown up by a very talented medical researcher, sitting next to me, and the team of other capable people, put under my right eye whereupon I grew a new cornea in my right eye.

 

JENNY BROCKIE: And how well can you see now?

 

PHILLIP IRONFIELD: I can see very well, I can see you wearing two earrings, I can see you holding a pen, I can see everything.

 

JENNY BROCKIE: And you've gone back to work?

 

PHILLIP IRONFIELD: I did go back to work but last year I retired seriously.

 

JENNY BROCKIE: Nick, you're the researcher who was involved in this. Explain what you did to help Phillip?

 

ASSOCIATE PROFESSOR NICK DE GIROLAMO, UNIVERSITY OF NEW SOUTH WALES: So basically what we did was we took multiple biopsies and these are like 1 mm in diameter from his healthy eye and we put these biopsies on contact lenses and we put those lenses in a solution and put those lenses in an incubator so that we could expand the cells from those tiny biopsies. And once those contact lenses became confluent with cells I was, I phoned the ophthalmologist and Stephanie Watson, who performed the surgery, she was able to scrape away all the abnormal cells from Phillip's right eye with a scalpel blade and then we rested that contact lens with the stem cells over that eye.

 

JENNY BROCKIE: So you'd grown that and basically put it on the eye?

 

ASSOCIATE PROFESSOR NICK DE GIROLAMO: Exactly.

 

JENNY BROCKIE: Was this all part of a clinical trial?

 

ASSOCIATE PROFESSOR NICK DE GIROLAMO: It was a clinical trial which was registered - it had undergone ethics approval from two authorities, the University of New South Wales and the Area Health Service Prince of Wales Hospital.

 

JENNY BROCKIE: How quickly did you notice the difference?

 

PHILLIP IRONFIELD: I noticed it quickly enough so that by the time I went down to the South Coast for a bit of a break, I sat down and I read 'The Girl with the Dragon Tattoo’ without leaving the chair.

JENNY BROCKIE: How long was that after the operation?

 

PHILLIP IRONFIELD: That was probably two days later.

 

JENNY BROCKIE: Wow?

 

PHILLIP IRONFIELD: Yeah.

 

JENNY BROCKIE: Martin Pera, you're the head of Stem Cells Australia which is a government funded research initiative. How, explain to people how stem cells can help regrow or repair parts of the body?

 

DR MARTIN PERA, STEM CELLS AUSTRALIA: So stem cells are special cells, they exist in many tissues of the body and they are able to what we call self-renew, that's divide to form more stem cells, but they're also capable of what we call differentiation or specialisation to give rise to mature cell types. They're like a reserve or reservoir for regeneration and repair of the body.

 

JENNY BROCKIE: Okay, so you can harvest them and potentially use them to treat all sorts of things?

 

DR MARTIN PERA: That's correct, and we can make stem cells from embryos or we can convert adult cells now into cells that have properties very like embryonic stem cells and they can turn into all the tissues of the body.

 

JENNY BROCKIE: So most things are experimental, they're not proven yet?

 

DR MARTIN PERA: Yes, that's exactly right. So most of the stem cell treatments we're talking about are still experimental. Mainly we don't know whether they're safe and effective in humans.

 

JENNY BROCKIE: Kerri Pottharst, you won gold for Australia at the 2000 Olympics in beach volley ball. What state were your knees in after twenty years of playing volley ball?

KERRI POTTHARST: They were in pretty bad state, actually Jenny. I'd had six knee surgeries, that involved ligament ruptures, reconstructions, meniscus tears, cartilage pulled off bones, so I had almost everything you could do to a knee I did over those twenty years and I just kept on playing because I loved what I was doing.

 

JENNY BROCKIE: So how did you end up being treated with stem cells, how did that happen?

 

KERRI POTTHARST: Well I got approached by Dr Bright, his offices, Macquarie Stem Cells, and I kind of put my hand up and said yes, I'd be interested in having that done for sure. He was very clear that it doesn't work for everybody but I wanted to have a go because I was at the point where I was just going downhill.

 

JENNY BROCKIE: Did you know it was experimental?

 

KERRI POTTHARST: I knew it was trialled, being tried and I knew it was kind of the beginning of the procedures, that he'd only been doing it for a couple of years.

 

JENNY BROCKIE: And what was the treatment itself like?

 

KERRI POTTHARST: Well, the initial treatment to remove the fat which they took the stem cells, from which was basically liposuction wasn't pleasant and generally that's not a very pleasant experience.

 

JENNY BROCKIE: Ralph Bright, you're a cosmetic surgeon?

 

DR RALPH BRIGHT, MACQUARIE STEM CELLS: Yes.

 

JENNY BROCKIE: And you administered Kerri's fat stem cell treatment. You contacted her, why did you contact her?

 

DR RALPH BRIGHT: The imperative at that time was to help people to become more aware of the existence of stem cells and of the possibility of having the sort of treatment.

 

JENNY BROCKIE: But was it because she's a high profile Olympian and you were keen to have someone like that?

 

DR RALPH BRIGHT: Yes.

 

JENNY BROCKIE: Okay, so did she pay for the treatment or was it free?

 

DR RALPH BRIGHT: It was free.

 

JENNY BROCKIE: Did you pay?

 

KERRI POTTHARST: No.

 

JENNY BROCKIE: Ralph, you were the first person in Australia to treat osteoarthritis with fat stem cells, how did you get started doing this, working with fat stem cells? What motivated you as a cosmetic surgeon to start doing it?

 

DR RALPH BRIGHT: I'd been doing fat transfer for many, many years, since about 2000, and one of the patients at that time had a melanoma excised and it looked so darn ugly. And I said look, don't worry, you've just got a hole there, there's no fat so I'll just put some in fat in there for you and the first time I did it, because I needed to do it three times to build up the depth and the first time I did it she had no feeling whatsoever. The second time I did it she said ouch and the third time I had to use local anaesthetic. And I said what is in fat that this person should regrow all their nerves and that's when I started to look at fat to try and find out what was in it. And then I saw the work of Bill Walsh at Prince of Wales and he was growing bone from fat and I thought that was pretty amazing.

 

JENNY BROCKIE: Kerri, what happened after your treatment?

 

KERRI POTTHARST: I'm one year almost to the day now since I had the procedure and my knees are unbelievably fantastic and I couldn't, I couldn't say that highly enough because an example just this week, I was at Australia zoo with my seven year old. We were walking around for four hours, I had him on my back at times because he was tired. I then jumped on a plane and came back to Sydney and I had no pain, no stiffness and eighteen months ago I would have been in pain and having to take anti-inflammatories and probably wouldn't have been able to walk that much.

 

JENNY BROCKIE: Clint Bartram, you recently had to retire from AFL because of knee problems, we're going to hear a bit about knees tonight. How did you find out about stem cell therapy, about fat stem cell therapy, and I should define this because there are different types of stem cell therapy. How did you find out about this?

 

CLINT BARTRAM: Yeah, I've was at a very similar point to Kerri where my career was being halted by injuries. Very similar story, I had about six or seven arthroscopies which led to the fact that I actually couldn't perform the way I wanted to. The options were to retire, again very similar, or get a partial knee replacement. That wasn't really an option as a 24 year old so a club doctor Dan Bates came to me with another option which was stem cells, of which I was explained that this procedure still is experimental but it was something that I mean as a professional athlete you love what you do so that was something I was really passionate about, giving myself the best opportunity to potentially get back and play football.

 

JENNY BROCKIE: Now again, did you pay for this or because you're a high profile footballer were you approached to have it done? How did that work?

 

CLINT BARTRAM: The football club fully supports all theirs players that are in the system.

JENNY BROCKIE: Okay, so do you know if they paid for it?

 

CLINT BARTRAM: No, I'm unaware.

 

JENNY BROCKIE: Okay. What were the results like?

 

CLINT BARTRAM: So I've had partial success. The biggest difference I've actually noticed is day-to-day. For me I never used to be able to walk without pain. If I was on my feet for more than half an hour my knee would blow up and create quite a lot of swelling. Wasn't enough to get back to playing AFL football, certainly not but the success that I did have was enough to give me a really comfortable life now where I still can be relatively active. So I'm pretty impressed with what the stem cells have actually done.

 

JENNY BROCKIE: Okay. Ross Walker, your knees were a bit like Kerri and Clint's, although you weren't a professional athlete?

 

DR ROSS WALKER: Not in the slightest.

 

JENNY BROCKIE: No, not in the slightest. How did you hear about fat stem cell therapy?

 

DR ROSS WALKER: Okay, well I was one of those fools that played soccer and squash till I was 52 and I have a radio show every week and I was interviewing Professor Ben Herbert who is from Regeneus about regenerate medicine.

 

JENNY BROCKIE: Which is a company that deals with stem cells?

 

DR ROSS WALKER: Yes, so I have a segment on the show called regenerative medicine and I interview Ben every week and have been doing that for the last three years. As a doctor I thought yes, this is an experimental therapy. Yes, the evidence on the preliminary work has been reasonable and so I went ahead and had it done. Because I didn't want to be accused of cells for comment, I paid for it myself, it cost me $10,000 and it did nothing.

 

JENNY BROCKIE: Didn't work at all?

 

DR ROSS WALKER: Not at all but let me say in defence of the technique, I was told by a Dr Kuah who did the technique, he wasn't confident because my knee was so bad that I actually did need a replacement but I'm too young for that so it didn't work for me at all.

 

JENNY BROCKIE: And you're a cardiologist?

 

DR ROSS WALKER: I am.

JENNY BROCKIE: Did you have any misgivings about having a treatment that was experimental?

 

DR ROSS WALKER: No, it's my own cells. They took 200 grams of abdominal fat, I tried to negotiate for a little bit more, and they harvested about 70 million stem cells and injected them back in my knee and it was my cells so I thought at the worst it wasn't going to work and I'd lose $10,000. At the best I'd be out of pain.

 

JENNY BROCKIE: Donald Kuah in Thredbo, you work with the biggest stem cell company in Australia, you are also Ross's doctor. Why do you offer fat stem cell treatment when it's still experimental?

 

DR DONALD KUAH, SYDNEY SPORTS MEDICINE CENTRE: Well I suppose that, I suppose there's different degrees of experimental and that's important to note. There are at least twenty clinical trials available on the use of cell therapy in osteoarthritis. I'm not sure, as certain about other fields. In virtually all of them they have been safe and there's been positive results and on multiple of those studies there have been MRI changes findings, all arthroscopic findings to show that there have been positive improvements.

What some in the panel may want is really a level 1 study where there's double blinded randomised control trials. Now there are a lot of treatments in medicine that haven't had level 1 studies passed and are still being done. I think it really comes down to whether it is safe and I believe it is safe. There was a papercome out in 2011 which was a meta-analysis which means someone sits down and looks at all the studies with respect to stem cell use, and there were 24 studies from memory in that paper, and the conclusion was that it was safe.

 

JENNY BROCKIE: Are you involved in clinical trials in your work?

 

DR DONALD KUAH: Yeah, absolutely. So I've been involved in a publication of a preclinical trial that was published in August 2012 looking at the secretions of the cells because we believe that a lot of the work is done by the secretions. We're also involved in the largest and the only one in the world of an ethics approved registry. So a lot of these clinical published papers that I spoke about have got numbers of twelve and twenty and thirty patients in their cohort. We've got now over 400 patients in that cohort, over two years; many of those studies were six to twelve months.

 

JENNY BROCKIE: What are your results showing, in terms of the effectiveness of the treatment?

 

DR DONALD KUAH: Well we're showing, yeah, so certainly in about 75 percent of patients they get pain relief at six and twelve months and that pain relief on average is about 70 to 80 percent. So we rated patients as responders only if they improved by at least 30 percent with pain.

 

JENNY BROCKIE: Okay, Martin Pera, do you think there's solid evidence of the proven effectiveness of some of these stem cell treatments?

 

DR MARTIN PERA: I think in terms of osteoarthritis the evidence is still equivocal. Some studies find benefits, some do not. This is why we need to do these carefully controlled trials where we observe the patients very carefully over a long period of time to really get an idea whether the treatment is safe and effective.

 

JENNY BROCKIE: John Rasko, what do you think? You specialise in bone marrow transplants which is an established area of stem cell treatment, what do you think about this kind of treatment?

 

PROFESSOR JOHN RASKO, ROYAL PRINCE ALFRED HOSPITAL: Well we know that you do anything to a knee and 30 percent of people tend to get a little bit better. 30 percent of people tend to get a little bit worse and the other 30 percent remain pretty well neutral and God knows what happens to the other 10 percent. The bottom line is, the bottom line is that we don't know what happens when we inject these cells or any different types of cells until such time as we do a randomised trial.

 

JENNY BROCKIE: Ralph, what did you want to say?

 

DR RALPH BRIGHT: Just to make the point that if you are seeing cartilage growing on x-rays and MRIs then it's hardly a placebo.

 

DR JOHN MOORE, ST VINCENT’S HOSPITAL: But you don't know that, you don't know that unless you've got a placebo up. I mean I think, I'm also concerned about the use of payment for treatment in this sort of situation. A clinical trial should not have a cost involved in it and I think it's a very grey area of ethics personally.

 

DR RALPH BRIGHT: But if you have ever seen a placebo that's going to grow cartilage? Have you ever seen a placebo grow cartilage?

 

DR JOHN MOORE: I don't think so, I think the issue is that we need to know that it is of benefit for these patients and if you're charging people for it, yes, but no.

KERRI POTTHARST: If someone had said to me $20,000 and your knees will be like they feel today I would have had no hesitation in doing the procedure.

 

DR JOHN MOORE: Not everyone can afford 20,000 Kerri of course.

 

KERRI POTTHARST: I agree with you, I agree with you.

 

DR JOHN MOORE: I think we need to know whether this works.

 

JENNY BROCKIE: I think the point is, the point you're making is about science, right, that you want the science?

 

DR JOHN MOORE: We want the science without the overcrowding of a financial incentive involved in it, I think.

 

JENNY BROCKIE: And do you share that view Martin - that you don't think the science is in?

 

DR MARTIN PERA: I do share that view, yes.

 

JENNY BROCKIE: On fat stem cell therapy, or other types of stem cell therapy being offered in Australia as well?

 

DR MARTIN PERA: I think most types of stem cell therapy are still experimental, we don't know in fact, in many cases whether they're safe. We don't know in many cases whether they're effective.

 

JENNY BROCKIE: And so how can it be that those therapies are being applied to people?

DR MARTIN PERA: Because they are exempt from the normal regulations that govern cell therapies in this country.

 

JENNY BROCKIE: And that's because you're allowed to use?

 

DR MARTIN PERA: A patient's own cells.

 

JENNY BROCKIE: A patient's own cells?

 

DR MARTIN PERA: And may I point out, the application you've heard here, fat cells for osteoarthritis, is one a lot us would agree is a reasonable thing to try. But what's being done goes well beyond that now and"¦

 

JENNY BROCKIE: So do you see that as a loophole in the law?

 

DR MARTIN PERA: I do.

JENNY BROCKIE: You do and why are you concerned about it being a loophole in the law?

 

DR MARTIN PERA: Because it opens the door to a whole range of treatments and manipulations that in fact may carry real risk and really represent a genuine danger.

 

JENNY BROCKIE: Okay Donald, what did you want to say? I mean this idea that there's a loophole in the law that's enabling you to use this treatment?

 

DR DONALD KUAH: Well it's not a loophole, it's the same as that, that loophole that he refers to, I think John or Martin might have mentioned that, is a biological exemption. It is the same as someone saying that blood transfusion is a biological exemption.

 

JENNY BROCKIE: Ralph, how you do you respond to the suggestion you're operating in a loophole?

 

DR RALPH BRIGHT: Oh, yeah, good point. The loophole, the word loophole is a word that comes from Martin and it's not shared by the TGA. The TGA made a very deliberate decision to exempt these cells; it endorses the low risk nature of the cells that we use as opposed to the high risk nature of embryonic stem cells and the like.

 

JENNY BROCKIE: John, you're sitting next to Kerri, I mean she's saying this worked for me?

 

PROFESSOR JOHN RASKO: Absolutely and I'm certain it did. However, what I would say is maybe injecting plasma or normal saline, or washing out the joint might have had just exactly the same benefit in another group of people or herself. The point is not to have celebrity endorsements or individual reports. Ultimately we want to apply the best health we possibly can in our nation to everybody who has a crook joint and everybody should be allowed to take the benefit of the best that medicine and science has to offer. That's what Australia's health system offers and that's what we all wish to provide.

 

JENNY BROCKIE: Martin, are your own stem cells safer to use in these procedures? I mean is safety proven?

 

DR MARTIN PERA: That depends.

 

JENNY BROCKIE: As opposed to effectiveness?

 

DR MARTIN PERA: If we take the example of a red cell transfusion, that's an example in which the patient's own cells are taken, they're not really manipulated much and they're put right back in to do exactly the same job they would normally be doing. Now in instances where we take a patient's own cells and manipulate them in a laboratory or grow them, and then we're putting them back to do a completely different job, that entails a good deal more risk so there's a spectrum of risk here unquestionably. And yes, you do have to look at the risk benefit analysis, but the problem is under the current regulatory framework, this is allowing experimentation to go forward without the appropriate oversight.

 

JENNY BROCKIE: So far we've talked about stem cell treatment in Australia. Kristy, I wanted to talk to you because you travelled to Russia to have a bone marrow stem cell transplant to try and stop your multiple sclerosis and your story was recently featured on 60 Minutes. We have some footage of you before and after your treatment. Let's have a look at that and you might want to just tell us about that. So your mobility was limited here?

 

KRISTY CRUISE: I'm getting upset watching it, it was very limited. That was my day-to-day. That was me after, so obviously my hair's missing there. That was me after I got back from Russia, I could get back into exercise and no longer needed mobility devices.

 

JENNY BROCKIE: Tell us what you had done in Russia, what was involved?

 

KRISTY CRUISE: I had a hemataportic stem cell transplant which is essentially the same as a bone marrow transplant that they've been doing for cancers for many, many years. And it involved stimulating the bone marrow so that they could collect it through apheresis process and then chemotherapy and reinfusion of the stem cells to reboot my immune system. The whole idea behind it is that MS is widely accepted to be a haematologically rooted auto immune disease so the idea behind it is to stop that autoimmune process with the chemotherapy and rebuild a brand new immune system that no longer attacks your brain and your spinal cord.

 

JENNY BROCKIE: And why did you have it done in Russia?

 

KRISTY CRUISE: Because I couldn't get into Sydney so I applied to all of the reputable facilities I could find overseas, the ones that have contributed to clinical trials and who I knew were good.

 

JENNY BROCKIE: John, you're doing the only trial in Australia on this kind of treatment. How experimental do you think it is for MS patients?

 

DR JOHN MOORE: Well, I think take it back a few steps, the rationale is solid but there is no randomised trial in MS, I can say up front, as opposed to other conditions. We initiated a trial at St Vincent's in, this is our fourth trial but in 2010, including multiple sclerosis, given that by that stage we felt there was enough evidence to start looking at it. So we've treated nine patients and it has been difficult to include all the patients that we'd like to include, but for us we felt first of all we wanted to make sure it was safe.

 

JENNY BROCKIE: So when you hear Kristy's story about going and you see that footage, what's your reaction to that?

 

DR JOHN MOORE: Well, I suppose I'd like to be able to offer Kristy that in Australia and we did have a conversation before she went and, but"¦.

 

JENNY BROCKIE: So she wanted to be in your trial?

 

DR JOHN MOORE: Yes.

 

JENNY BROCKIE: And you wouldn't, you didn't include her?

 

DR JOHN MOORE: Well it way it's been approved by our ethics committee is that the patients have to have failed multiple lines of therapy and that's pretty standard really when you think about this as, this is a procedure that has a 5 percent chance of dying and so we only accept patients that neurologists feel have no other therapeutic options.

 

JENNY BROCKIE: So you thought she hadn't tried enough things?

 

DR JOHN MOORE: Well I have to be guided by the ethics committee and so by the way the trial was set up I couldn't accept her in the trial.

 

JENNY BROCKIE: And Kristy, why did you leapfrog that process of trying the other drugs and trying the other procedures?

 

KRISTY CRUISE: I found a number of studies which showed a median success rate of around 80 percent but a success rate of over 90 percent in the patients who had early intervention, HSCT.

 

JENNY BROCKIE: So you wanted to get in there?

 

KRISTY CRUISE: I wanted to get in and I wanted to have the high success rate and I didn't want to waste time. I was, I was out of work, I couldn't work as an ED nurse any more so I didn't want to waste years becoming more disabled and trialling multiple drugs with side effects and risks.

 

JENNY BROCKIE: I want you two to talk to one another here because I'm really interested, because you know"¦

 

DR JOHN MOORE: We did talk to each other.

 

JENNY BROCKIE: I'm sure you did.

 

KRISTY CRUISE: We did, we did. I was actually surprised because before I went to Russia, Dr Moore did give me the privilege of a phone call that was lengthy and he was very sympathetic and, you know, I was very frustrated and we discussed that there were some trials in which early intervention patients did better and he explained his position in having to treat the most disabled. You know, so it was good phone conversation but obviously I would have preferred to have stayed in Australia.

 

DR JOHN MOORE: Yeah.

 

JENNY BROCKIE: See this is interesting to me because in all these stories there's the meeting of science and the patient, you know, and you've got the patient saying I want to try something, I've got, I've got a disease, you know, that I am concerned about the development of, I want to move fast, I want to do something quickly which is really understandable.

 

DR JOHN MOORE: But Jenny if we'd gone in quickly fifteen years ago and done people early, and some of them had died, then everyone would say we haven't followed due process. And so, you know, there are two sides to the story.

 

KRISTY CRUISE: You've got to balance the need for science versus the human condition.

 

JENNY BROCKIE: And how do you all think that balance is going at the moment?

 

PHILLIP IRONFIELD: Well it's not going very well. When I undertook my stem cell transplant I made sure it was a clinical trial in the public system and under the review of an ethics committee. You know, heaven forbid we should find a doctor out there practising with some God complex and we have all of these things set up to protect us and self-regulation in the medical profession is not good Jenny.

 

JENNY BROCKIE: Ralph, your response to that, because you are able to just do this, aren't you? I mean you don't have to prove to anyone that you, that you, you know, have scientifically examined the whole process?

 

DR RALPH BRIGHT: Sometimes we practice medicine and we see things happen and we say oh, why did that happen? And if I do it again will it happen again? And quite often we really don't know why we have got some improvement, but just because we don't know the science behind it doesn't mean to say that it doesn't work.

 

JENNY BROCKIE: Response?

 

PROFESSOR JOHN RASKO: That's a definition of pseudo-science right there, we've just heard the definition of pseudo-science.

 

JENNY BROCKIE: But science isn't absolute either.

 

PROFESSOR NJOHN RASKO: That is snake oil sales.

 

JENNY BROCKIE: Science isn't always definitive either?

 

PROFESSOR JOHN RASKO: Absolutely not. Science is not always definitive and we can never speak in absolutes, but what we can be clear about is whether there's a benefit and the only way to do that is with a placebo controlled randomised, often crossover trial, a technically approved and well regulated trial that's then published, subject to review and replicated elsewhere. If cowboys have activities in their own rooms and do things that aren't being supervised, and certainly not intended or approved by the TGA, the activities that are occurring in a number of doctors' premises were not intended by the exemption order.

 

JENNY BROCKIE: So cowboys?

 

PROFESSOR JOHN RASKO: Absolutely.

 

JENNY BROCKIE: Are there cowboys in this room?

 

PROFESSOR JOHN RASKO: Absolutely I'm afraid so. This is an unproven therapy for which people are taking money and I take exception to that.

 

MATT BATTISTA: It's easy just to sit there and go oh yeah, if you were the one sitting in a wheelchair or couldn't be yourself or you couldn't do anything with your kids or couldn't stand up? What would you do? Would you be, would you be, okay, this is the law and this is how it all works, would you not just go I'm going to try something?

 

PROFESSOR JOHN RASKO: I take - I have no pleasure in being placed in a position that I find myself tonight. Overall and above everything, must be compassion and respect for individuals to decide on their own course and I find myself, after 25, 30 years devoting myself to making certain that stem cells and gene therapy one day will be safely and properly administered to people, I find myself in an impossible situation appearing to be saying don't do it.

 

MATT BATTISTA: But everything takes so long, they have these trials and they take like twenty years.

 

KRISTY CRUISE: And no money for clinical trials in Australia.

 

ASSOCIATE PROFESSOR MEGAN MUNSIE, STEM CELLS AUSTRALIA: But are stem cells the solution for everything? We just talk about stem cells as though they're a silver bullet and I think that we have to take a step back and that's why we need clinical trials. We need to understand whether HSCT will work for MS. We don't, we can't just lump everything together and think that stem cells are going to save everything.

 

JENNY BROCKIE: Megan, you've looked at patient expectations around stem cell therapy, haven't you?

 

ASSOCIATE PROFESSOR MEGAN MUNSIE: Yes.

 

JENNY BROCKIE: What have you found?

 

ASSOCIATE PROFESSOR MEGAN MUNSIE: In our research we interviewed Australians who have been overseas and a lot of people feel really good about having done that, having gone overseas. And I think this, the feeling of empowerment, to feel as though they have tried all options, even though there wasn't necessarily a physical improvement, they felt better for having gone, it gave them hope.

 

JENNY BROCKIE: Because Mike you have MS.

 

MIKE HEMINGWAY: Yes.

 

JENNY BROCKIE: Have you been tossing up what to do and thinking about, I mean when you see Kristy's story, for example when you hear about these treatments, what's been your response?

 

MIKE HEMINGWAY: I'm a little bit more sceptical. One thing I would ask Kristy is how long had you been diagnosed Kristy when you or had you had MS when you had your treatment?

 

KRISTY CRUISE: I've had MS for a long time but I went undiagnosed for a long time, it took a severe deterioration for me to actually into the GP and say I think I have MS, refer me to a neuro. But in hindsight, I had blindness in my eye in 2007, my history is extensive, and that's a common misconception after 60 Minutes is that I got MS one day and went and had a stem cell transplant. I was diagnosed and then had a stem cell transplant very quickly because I did nothing but research for six months and called good doctors and spoke to reputable people. But yeah, I had MS for a long time.

 

JENNY BROCKIE: Jodi, you have MS too and you're waiting to hear if you get into John's trial. Is that right?

 

JODI RUSSELL: I am, I am hoping to I get into John's trial because I'd love to come and have treatment in Sydney.

 

JENNY BROCKIE: Yes, I was going to say, it's a very good opportunity for lobbying here. What have you done in the meantime though while you're waiting?

 

JODI RUSSELL: Well I've actually had MS for, diagnosed for fourteen and a half years. It's been a yearly relapse for me and I've been through every single disease modifying drug that's available on the market and now, I mean that's including chemotherapy over two years which was amazing for me and put me into a remission of two to three years. Now I'm at the point where I've got nothing left to lean on and I need something, I need some hope and the light at the end of the tunnel. I saw the 60 Minutes program.

 

JENNY BROCKIE: This was about Kristy going to Russia?

 

JODI RUSSELL: It was about Kristy, I was alerted to it by a lot of friends and relatives. When I watched it I was sceptical at first because you know, you hear about these miracle cures.

 

JENNY BROCKIE: So have you booked in to go to Russia?

 

JODI RUSSELL: I've booked in to go to Russia in January because I have, well I'm trying to get into Sydney, but"¦

 

JENNY BROCKIE: No pressure John, no pressure.

 

MALE: And what money are you willing to pay?

 

JODI RUSSELL: I'll pay you.

 

KRISTY CRUISE: Yeah, that's the issue, I have 80 people, 80 Australians going to Russia in next eighteen months, 80 people that I've helped with applications and I've spoken, I've responded to 37,000 emails in four months and I have 12,000 in my account right now that are not replied to.

 

JENNY BROCKIE: How much does it cost?

 

KRISTY CRUISE: It costs 40,000 US plus expenses, air fares and cares and that sort of thing.

 

JENNY BROCKIE: How do you know it was the transplant that resulted in your improvement?

 

KRISTY CRUISE: Well here's the thing, from February till I arrived in Moscow from August, I got progressively worse. I tried steroids in hospital, they didn't work. I just kept getting worse and worse and more disabled. After I returned from Moscow I waited at least four months to have an MRI because I didn't want anyone to say it was the steroids interfering with my MRI report. All of my lesions are smaller and two are no longer visible on MRI, they cannot find them. And"¦

 

JENNY BROCKIE: These are the lesions in your brain?

 

KRISTY CRUISE: Lesions in my brain and a placebo effect cannot remove MRI lesions or shrink the ones that are there.

 

JENNY BROCKIE: Do you think there could be another explanation though for why you've improved since you went to Russia?

 

KRISTY CRUISE: No.

 

JENNY BROCKIE: I mean do you think there could be, because MS symptoms can wax and wane, can't they – they can come and go? In some cases, not in all cases.

 

KRISTY CRUISE: They can wax and wane. But they usually wax and wane for at the most maybe two months.

 

MATT BATTISTA: Is it better for you the earlier you go is it better for you the earlier you go before"¦

 

KRISTY CRUISE: The data that I found said yes.

 

MATT BATTISTA: But over here it's basically you've got to be at death's door, then we might have a look at you.

 

KRISTY CRUISE: That's right. So the facility that's doing the stage 3 clinical trial, they have trial criteria that's very similar. They want people who have failed these, these drugs. But they also have a non-trial acceptance policy and I was accepted off trial. I'm fine to be off trial, I just want the right to be treated.

 

DR JOHN MOORE: But it's $200,000 to do that with the Chicago group. So what I'm trying to do, you talk about the difference between compassion and science, what I'm trying to do is, I don't want to accept the $200,000, what I want to do is go through the appropriate channels and I have through the bureaucracy and it goes to a Health Minister's meeting on the 18th of July. I wrote a document, I'm trying to get it done so that we have an even playing field and not everyone has to go to $200,000.

 

JENNY BROCKIE: So this will be available in Australia?

 

DR JOHN MOORE: We can do it and people paying money for it I don't think is the right way to go. I can get the authorities to pay for it, I would love that.

 

JENNY BROCKIE: So does that mean you have evidence it works, scientific evidence it works?

 

DR JOHN MOORE: There is plenty of evidence that it works, correct, but we need the randomised trial and that's why we have committed at St Vincent's to do the randomised trial and it will cost us to do that, but we want to do it because we want to know that the science is correct.

 

DION RUSSELL: Do you understand how frustrating is to people who don't care who pays, I don't care if I pay or I don't care if the taxpayer pays. I just want my wife to be better again.

 

DR JOHN MOORE: Correct, I listen to it daily and I do feel for people.

 

JODI RUSSELL: Why in the trial, why do we have to be at a certain disability range? I mean what is the point of waiting say to be non-mobile before you get the treatment to halt your MS? I don't understand the logic behind that, nobody in their right mind wants to halt the MS when they're already in a wheelchair.

 

DR JOHN MOORE: Because there are therapies that may work and I mean obviously clearly"¦

 

JODI RUSSELL: -- but I've been on every one of your other therapies.

 

DR JOHN MOORE: So you may be eligible but there are people who may have, may go on to interferon for example and be fine for many years.

 

JENNY BROCKIE: But it sounds like there's reason, there's a reasonable basis for believing that this procedure should be available in Australia that's being offered, that Kristy had to go to Russia for.

 

DR JOHN MOORE: Yes Jenny, I hope the state Health Minister is watching.

 

JENNY BROCKIE: Ralph, back in your surgery in Sydney, what other conditions are you treating with fat stem cell therapy?

 

DR RALPH BRIGHT: I think that osteoarthritis responds very well and I think that migraine is another disease that also shows an extremely promising response rate and that's something that I would like to progress.

 

JENNY BROCKIE: You're also treating though spinal cord injury, MS, Parkinson's, motor neurone disease, all with fat stem cell therapy, is that right?

 

DR RALPH BRIGHT: I have had some patients with Parkinson's disease and I think that overall I'm not convinced that it's the best treatment to have.

 

JENNY BROCKIE: Well you've treated Geoffrey up the back here?

 

DR RALPH BRIGHT: Yes, and I'm very happy with his response but I think that he's a little bit unusual.

 

JENNY BROCKIE: Geoffrey, how did the treatment go?

 

GEOFFREY LAMBERT: I had the initial, the extraction of the stem cells in December 2011. Within two weeks I noticed an improvement in my connectivity with the world around me, my vitality, and I subsequently had top-up treatments several times and the tremor in my right arm was almost, pretty much disappeared and neither of them have come back. I've since written a novel which has been published, so my cognitive abilities didn't deteriorate any more than they do in any other 70 year old. I'm happy I had the treatment.

 

JENNY BROCKIE: Okay, Martin, your reaction?

 

DR MARTIN PERA: Well we don't really know what would happen to stem cells under those circumstances or they're not pure stem cells obviously, it's a crude preparation of fat. It seems unlikely to me that they would reach the brain and be able to benefit Parkinson's disease.

 

JENNY BROCKIE: John, your reaction?

 

PROFESSOR JOHN RASKO: I couldn't be more thrilled for Geoffrey, I couldn't be more thrilled for Kristy, so without wanting to dramatise it Jenny, a person with MS who might have a life expectancy of 10 or 15 years being with their children and seeing their kids grow up might die with a 5 percent or higher risk in the first three months following that autologous transplant.

 

KRISTY CRUISE: The worldwide risk rate is about 1 percent of treatment related mortality. The doctor who did mine has done 280 transplants with zero deaths. So we have to look at the different facilities and we can't just say it's 5 percent because that's absolutely not true. It may be 5 percent in Sydney, I don't know, but it's not 5 percent in every election in the world.

 

JENNY BROCKIE: Okay. Donald, you wanted to say something, did you want to join in this discussion?

 

DR DONALD KUAH: I suppose I wanted to address an earlier comment about cowboys and for fear of being labelled as a cowboy, I want to point out that I have to be referred patients by other doctors, I have to write back to those doctors explaining my rationale for treatment and what I've told the patients. I also work under the auspices of a medical advisory committee of the hospital that I perform this procedure in. So we have many treatments in the medical field which would be deemed experimental that are commonplace and I can name quite a few, certainly in musculoskeletal medicine.

 

JENNY BROCKIE: I want to talk a little bit about the placebo effect because I don't think we can, you know, have this discussion without talking about that. Donald, in the absence of those studies, those trials that actually do things like establish what happens when people are given a placebo, how can you know that some of the outcomes you get aren't the placebo effect, that people aren't just responding to any kind of treatment?

 

DR DONALD KUAH: There's no doubt that there's a strong placebo effect, there's been other trials that have shown that the more that you do, the more that you interfere, the more that you treat someone, the greater the placebo effect. I suppose when you look at things like cartilage regrowth, changes in biomarkers, so for example where we can look and measure products of cartilage degradation in placebo group and in treatment groups and there was a difference in that North Shore trial.

 

JENNY BROCKIE: Martin, what about where things are measurable, can you, like the growth of cartilage, can you eliminate the placebo effect with treatment?

 

DR MARTIN PERA: Well I think it's important to recognise that there may be a placebo effect, but also physically manipulating the tissue, putting a needle in, may do things we don't really appreciate that could be a consequence of those manipulations. So that's why it's important to know precisely what is causing the beneficial effect. So the treatment has to be matched as closely to a control as possible.

 

JENNY BROCKIE: Geoffrey, have you wondered about the placebo effect with treatment or not?

 

GEOFFREY LAMBERT: Yes, I have, and my view was if it works in me, I don't care whether it's placebo effect or"¦

 

JENNY BROCKIE: What it is?

 

GEOFFREY LAMBERT: What it is. If I feel better, it's now three and a half years and I do still feel better, I still enjoy red and white wine, I can do things that I wasn't able to do prior to 2011.

 

JENNY BROCKIE: Kerri, did you wonder about it?

 

KERRI POTTHARST: I don't think it even crossed my mind because I'd been in pain for so many years while I was playing and then since retiring, I retired ten years ago, that I've tried everything and I've tried everything, I've had injections in my joint before, I've had"¦

JENNY BROCKIE: Had a lot of treatment?

 

KERRI POTTHARST: I've had a lot of treatment and it took a while for me to really believe that it had worked. It took six months for me to really start to test it and now I'm running on the sand and I didn't even run on the sand when I was playing.

 

JENNY BROCKIE: Ross, what about you, you were a bit more sceptical about things?

 

DR ROSS WALKER: No, I don't do placebo very well actually. No, look, there's no doubt about the placebo response. A study came out last year about arthroscopy for osteoarthritis and they did sham arthroscopy and a proper arthroscopy where they tidy up the joints. There was absolutely no difference in benefit with a significant placebo response by just having the anaesthetic and having the injection.

 

JENNY BROCKIE: Perry, you're hopeful that stem cell therapy eventually might help your spinal cord injury. What sort of treatment have you had so far?

 

PERRY CROSS: I've taken part in a clinical trial with embryonic, human embryonic stem cells in India. It was a trial for people who were incurable and terminal. I've been doing that for a few years now and seen functional recovery that, you know, haven't been able to explain by my Australian doctors. So you know"¦

 

JENNY BROCKIE: So what sort of things?

 

PERRY CROSS: Mainly the function in my diaphragm. I was injured playing rugby twenty years ago, my life expectancy was ten years post injury because I was fully ventilated so about fourteen years after my injury I went to India to get treatment and started to regain function into my diaphragm. The last series of treatment I started get a bit of function in my right shoulder. So"¦

 

JENNY BROCKIE: Has it been as much as you'd hoped in terms of a"¦

 

PERRY CROSS: Well I went into the treatment thinking, you know, any improvement would be amazing because there is no cure for a spinal cord injury at the moment, you know, that's pretty obvious.

 

JENNY BROCKIE: So how many times you have been?

 

PERRY CROSS: Five, six times, probably, yeah.

 

JENNY BROCKIE: And how many more times do you think you'll go?

 

PERRY CROSS: Well at this stage it's all about, you know, money, because I have to pay to take part in their clinical trial and it's not cheap.

 

JENNY BROCKIE: So you have to pay in addition to the travel costs, you have to pay as well?

 

PERRY CROSS: Yes.

 

JENNY BROCKIE: How much has it cost you so far?

 

PERRY CROSS: Within the vicinity of maybe, it's going to get me out of my wheelchair, probably a quarter of a million dollars I'd say.

JENNY BROCKIE: Quarter of a million?

 

PERRY CROSS: Yes.

 

DR ROSS WALKER: How do they administer the treatment Perry?

 

PERRY CROSS: IV injection.

 

JENNY BROCKIE: This is embryonic stem cells?

 

PERRY CROSS: Yes, human embryonic inter muscular, epidural, all sorts of, yeah.

 

DR ROSS WALKER: Thank you.

 

JENNY BROCKIE: Martin, what's your view of this treatment?

 

DR MARTIN PERA: There are trials now on-going of embryonic stem cell derived products in spinal cord injury we don't know how those trials will turn out.

 

JENNY BROCKIE: Matt, you've also been overseas for stem cell treatment for your spinal cord injury. Where did you go?

MATT BATTISTA: I went to Germany and I went there because they use my own stem cells.

 

JENNY BROCKIE: So the nature of the treatment was very different, yeah?

 

MATT BATTISTA: Yeah, I didn't want to, put something in my body that wasn't mine.

 

JENNY BROCKIE: And what were the results for you?

 

MATT BATTISTA: Yeah, not much, nothing really happened but if I didn't go I'd still be sitting in here going what about if I did go?

 

JENNY BROCKIE: Now that clinic you went to has since been shut down, is that right?

 

MATT BATTISTA: Yeah, yeah, I think so, they put themselves into a young child's brain and it didn't work out.

 

JENNY BROCKIE: And the child died?

 

MATT BATTISTA: Yeah.

 

JENNY BROCKIE: So where has that left you now in terms of what you're prepared to try?

MATT BATTISTA: Now I wouldn't really try anything unless I was like a proven deal.

 

JENNY BROCKIE: So what's it like for us listening to this discussion tonight?

 

MATT BATTISTA: Yeah, I see everyone's point of view but you know, you're out in public and you are worrying about wetting your pants, and as a man, you know - it’s devastating. If there is only a touch of hope, you’ll go and that is never going to change. I can speak to someone who has had a spinal cord injury tomorrow and tell him my experience but he is going to have some hope cause where he is at the moment is probably not the best place he’d want to be so"¦ yeah.

 

JENNY BROCKIE: Perry, there was a lot of hype around stem cell treatment for spinal cord injuries some years ago, there was a lot of hope, a lot of, you know, promise in that. Particularly during the period that Christopher Reeve was championing it and calling for more research funds and so on. Has there been much progress do you think in the research in that time?

 

PERRY CROSS: Well there's, you know, there's a lot more, there's clinical trials happening, you know, all over the world now so we eventually got to a point where it started to come to fruition. You know, things were happening, there was momentum being gained. So I believe that, you know, we're on the right track now. There are things happening in the US that are looking encouraging so we are on the right track. The essence of this story, is that if I sit around and wait for the government to fund the doctor that's going to cure me, I'll probably be dead. So that's just the real truth.

 

JENNY BROCKIE: John?

 

PROFESSOR JOHN RASKO: I again feel very uncomfortable in this corner but the question is simply how would you know whether you got a benefit or not unless it was done in the context of a controlled clinical trial?

 

JENNY BROCKIE: Nick, what's happened to the trials that you're doing around stem cell therapy for eye problems?

 

ASSOCIATE PROFESSOR NICK DE GIROLAMO: Well, I'm saddened to tell you the truth because we never got funding from the federal government, despite putting many rounds of grants in. We did get funded by the Australian Stem Cell Centre at the time which provided a few hundred thousand dollars and the University of NSW chipped in $50,000. So you know there was a money constraints and a time constraint on us because we only had ethics committee approval for five years. The results show that 60 percent or 65 percent of our patients have improvement in eye health and importantly, improvement in vision. So it's not fool proof but certainly a large majority of our patients have benefited.

 

JENNY BROCKIE: So where are you up to? Can you finish your work?

 

ASSOCIATE PROFESSOR NICK DE GIROLAMO: We can't progress any further. What we want to do we want to try to improve the therapy and get it out there, but we can't without funding.

 

JENNY BROCKIE: Is the federal government's new medical research fund going to make a difference to any of this? Who'd like to comment on that, people running at a million miles an hour backwards on that question, I'm wondering why. John?

 

DR JOHN MOORE: A lot of doctor visits to make a billion, or whatever they said.

 

JENNY BROCKIE: It will take a lot of doctor visits - A lot of co-payments to make up the money?

 

DR JOHN MOORE: I think it's ludicrous personally, I think it's a smoke screen that medical research fund. I'd love if it happened but I don't think it will happen.

 

JENNY BROCKIE: What do you think John?

 

PROFESSOR JOHN RASKO: I embrace any medical research funding. If the government is actually committed to providing 20 billion dollars in the years forward and making it accessible in the next couple of years I certainly would encourage them to pursue that as the highest level.

 

JENNY BROCKIE: Nick, what do you think?

 

ASSOCIATE PROFESSOR NICK DE GIROLAMO: Show us the money.

 

PERRY CROSS: I agree. I think it's a great idea. I think this is one thing Australia can't do is build cars obviously so we have to do something with our kids, we've got to educate them, they've got to be educated. Australia's got the nouse to lead the world in medical research and if we sit by and just wait for every country to go and, you know, make amazing breakthroughs we'll get left behind in making cars and medical research. We've got be an opportunity here to invest in medical research and put ourselves on the map.

 

JENNY BROCKIE: What I want to do is wrap up and just get a final comment from people about the message they'd like to get out to people watching this as a result of this discussion. What's the message you'd like to get out to people Martin?

 

DR MARTIN PERA: I think from a scientific point of view we're very optimistic about this field. I think the progress we have made has remarkable. I'm delighted to see clinical trials proceeding. I understand everything we've heard here but we do have a responsibility not to raise false hope and responsibility to do no harm.

 

JENNY BROCKIE: Kristy?

 

KRISTY CRUISE: I would like Australians neurologists to see the success stories coming back from overseas and to perhaps open their minds a little bit and work with haematologists to make this more accessible in Australia.

 

JENNY BROCKIE: Matt?

 

MATT BATTISTA: I would like Australians to hurry up, get it happening now. I've already waited, you know, when I had my accident it was '08 and it was all new then, that was the thing. You know, I thought honestly six years later there would be something more happening than what's going on.

 

JENNY BROCKIE: So hurry up?

 

MATT BATTISTA: Yeah, pretty much.

 

JENNY BROCKIE: Okay, we'll leave it there, thank you all very much for joining us for this discussion tonight. That is all we have time for here but of course you can keep talking on Twitter and Facebook. I'm very interested to hear what you think about our discussion here tonight.