SOPHIE SCOTT, Australian Broadcasting Corporation reporter : In May this year, details of a ground-breaking new skin cancer treatment were revealed.
The discovery of the so-called super drug was led by renowned biologist Professor Levon Khachigian.
LEVON KHACHIGIAN, SCIENTIST, UNSW: It sends the tumour into a death spiral and as a consequence the body's own defence systems clean up the tumour.
SOPHIE SCOTT: The drug is a DNA enzyme called DZ13. It targets cancer cells by switching off a master gene, slowing down the growth of tumours.
The potential of the drug was hailed by experts in the field.
There were hopes the drug could go well beyond cancer treatment.
But even as the news was breaking, a very different story was unfolding behind closed doors as senior management at the University of NSW were confronted with serious allegations that the science which led to the breakthrough was deeply flawed and possibly even false.
DAVID VAUX, WALTER & ELIZA HALL INSTITUTE OF MEDICAL RESEARCH : Now I think that anybody who has concerns of scientific misconduct, you know, there's an ethical responsibility for them to raise those concerns with either the designated person to receive allegations of misconduct or with the journal editors or with the authors of a paper.
SOPHIE SCOTT: Professor David Vaux is an internationally acclaimed cell scientist at Melbourne's Walter and Eliza Hall Institute and lectures worldwide on research ethics.
He read a number of online papers about genetic research from Professor Khachigian's lab in 2009.
He was worried that some of the images in the papers were inappropriately duplicated.
He wrote twice to the Journal of Biological Chemistry to tell them he believed the images were not genuine.
DAVID VAUX (male voiceover): "Could you please try to obtain from these authors that readers of the JBC can trust, or retract the paper?"
SOPHIE SCOTT: In July 2010, the papers were retracted by the authors, who said they'd made a genuine error.
Then in February this year, David Vaux came across another paper from the same lab, this one focusing on Dz13.
When he studied the images, he became alarmed and wrote to the University of NSW.
DAVID VAUX (male voiceover): "I wish to alert you to concerns I have over a possible case of research misconduct at UNSW.
In the paper attached I have annotated the images that I'm concerned about.
They appear to contain duplications and/or alterations of images in such a way that the same data is used to represent two different conditions."
SOPHIE SCOTT: As an advocate for ethical research, he says he had to act.
DAVID VAUX: Well I think that it's important that when anyone has any concerns about science, that they report it as soon as possible. It's got to be done in a timely manner and shouldn't be delayed.
SOPHIE SCOTT: What Professor Vaux didn't know at the time was that one of the key University of NSW scientists working on Dz13 also had grave concerns about the research.
Dr Ying Morgan was one of the head researchers on Dz13.
She came to Australia in 1992, intent on building a scientific career specialising in cancer drug development.
Her big break came in 2007 when she won a sought-after postdoctoral position at the University of NSW.
Her task was to run the initial experiments on Dz13, which began in 2007.
Her supervisor was Professor Levon Khachigian.
YING MORGAN, SCIENTIST: I was so excited and I was also think, "Oh, you know, the supervisor's so famous and must be - will be good and I want to make some contribution on that project."
SOPHIE SCOTT: She played a central role in the research, testing the treatment on mice with skin cancer.
But within a year, she became worried about the results of some of the experiments done in the lab.
She thought the work had been done too quickly and the results were too perfect.
YING MORGAN: Data presented in the lab meeting appeared, "Oh, it's perfect." And then look like it's not real science, it's always not data perfect.
SOPHIE SCOTT: She then wrote to the university to make a formal complaint of research misconduct.
YING MORGAN (female voiceover): "I would not like to have my name associated with the research data and results on this project. I have no confidence with the research data and results."
SOPHIE SCOTT: In April 2010, she turned to fellow academic, historian and university union representative, Dr Sarah Gregson.
How did she strike you in terms of her demeanour and her personality?
SARAH GREGSON, NATIONAL TERTIARY EDUCATION UNION: Um, very stressed. I think you can't meet Ying without thinking she's very upset about this, being this is not her first language and she really struggles to get her meaning across. But I've spent a long time with her and I absolutely believe that she's genuine and very much concerned for patients.
SOPHIE SCOTT: Sarah Gregson was shocked by what Dr Morgan told her.
SARAH GREGSON: Ying came to us with an allegation that the professor who was supervising the lab where she worked was falsifying data and misrepresenting what that data said.
SOPHIE SCOTT: The university held a preliminary investigation.
Dr Morgan wasn't interviewed and the allegations were rejected.
It was wrapped up in two months and the university considered the matter closed.
Professor Khachigian told 7.30 that the investigation concluded, "Professor Khachigian took the concerns of Dr Morgan seriously ... took all necessary steps to ensure that Dr Morgan's concerns ... were investigated.
Professor Khachigian appropriately sought a review of experimental data and requested .. new DNAzymes studies.
The university was satisfied that Professor Khachigian did not breach his supervisory responsibilities."
Despite the university rejecting her allegations, Dr Morgan continued to press a complaint.
The university convened an external panel and Dr Morgan was interviewed twice.
Again the scientists were exonerated, there were no reasons given for the findings to Dr Morgan and the file was marked strictly confidential.
Professor Khachigian has told 7.30 he commissioned independent studies to verify the data.
PETER SMITH: The second case was referred to an external panel involving two very senior scientists and a very senior lawyer.
They investigated the complaints and they came up to two conclusions.
One is that there was no evidence for research misconduct.
And secondly, that the issue of the Dz13 trial, the matters that they considered had no bearing at all on that trial.
SARAH GREGSON: Oh, she was devastated, really devastated and committed to fighting on, but not sure where to go.
SOPHIE SCOTT: A further reason for Dr Morgan's distress was that her work revealed Dz13 worked for the first 20 days.
After that, it stopped working and the tumours started growing again.
She says her experiment showing a sharp increase after 20 days wasn't used.
Professor Levon Khachigian told 7.30 in a statement these were 20-day experiments and there was no manipulation of data.
LEVON KHACHIGIAN (male voiceover): "The research was not flawed. The independent inquiry panel found no case of research misconduct, nor any evidence of falsification, fabrication or misrepresentation of data."
SOPHIE SCOTT: But since the independent panel's findings, Dr Morgan has raised what she believes to be further serious irregularities in the research.
What were your concerns about these images?
YING MORGAN: (Inaudible) been manipulated.
SOPHIE SCOTT: Researchers in the same lab had done a study using Dz13 on blood vessels.
Dr Morgan alleges that crucial images in a paper published in the Journal of Biological Chemistry were misrepresented.
YING MORGAN: So, when I look at that paper, of course, absolutely shock. So, a lot of data hasn't been properly presented and then they are - misrepresentation, yes.
SOPHIE SCOTT: Dr Morgan believes the same specimen was used in the images of both the treatment and the placebo.
And what had been presented as two different images were actually one and the same.
And you could tell by looking at these images where you could see the same features pop up in both the treatment and in the control.
YING MORGAN: Yeah, that's right.
SOPHIE SCOTT: So Dr Morgan, are you 100 per cent convinced that you are right, that there is - in your opinion, that some of this data has been manipulated?
YING MORGAN: Yes, I'm - data be mispresented and have been manipulated and this is by the evidence.
SOPHIE SCOTT: These were the same images that caused David Vaux from the Walter and Eliza Hall Institute to write to the Journal of Biological Chemistry to say the images were not genuine and later to the university raising possible research misconduct.
DAVID VAUX: The thing about scientific misconduct is it must be deliberate, so it's somebody who's deliberately trying to distort or hide or make up results.
And - but whether mistakes are deliberate or whether they're accidental, they still need to be reported and they need to be corrected.
SOPHIE SCOTT: Professor Levon Khachigian wouldn't comment because it's currently under investigation, but said there was no deliberate misrepresentation.
Despite the unresolved concerns about the research, human trials of Dz13 began in 2010.
It was advertised with an assurance that animal studies had shown it was safe.
There was nothing to alert prospective patients about the concerns over Dz13.
Nine patients with basal cell carcinomas were recruited and injected with the drug.
The patient for Dz13 is held by Professor Khachigian and the University of NSW commercialisation arm.
The results of the initial trial showed the depth of tumours shrank in five of the nine patients and there were no serious side effects.
But Professor David Vaux was shocked to learn that a human trial had taken place and that a second trial was planned.
He wrote to the National Health and Medical Research Council, which was funding the study.
DAVID VAUX (male voiceover): "I believe it would be important to act quickly, as patients may currently be receiving the agent described in the publication, Dz13, as part of a clinical trial. If the results in this paper are not genuine, the HREC that approved the trial might have been misled, and the patients receiving the drug might not have been able to give properly informed consent."
SOPHIE SCOTT: Is it right that human trials on a compound should be conducted when there is some question marks over the science behind what led to that point, in your view?
DAVID VAUX: Well, clinical trials should only be conducted if they've been approved by a properly constituted human research ethics committee and patients have given informed consent.
SOPHIE SCOTT: As a trial of 38 patients with malignant melanoma was about to start, senior managers at the University of NSW began a third investigation into Dr Morgan's claims which is still underway.
Both Professor Vaux and Dr Morgan believe human trials shouldn't proceed until question marks hanging over the science are resolved.
YING MORGAN: Well you have to stop the trial because you have to reveal all the safety issues, because continue trial (inaudible) and then you can cause the patient harm.
SOPHIE SCOTT: After the ABC contacted the University of NSW, we were told that human trials of Dz13 had been suspended after concerns about the science leading up to the trial.
The university rejects any allegation of a cover-up into its handling of the investigation.
PETER SMITH: We take issues of patient safety in clinical trials extremely seriously and we will investigate whatever concerns or claims have been made.
We will through our processes determine if there has been a breach in the research - in the Carter research conduct, and if so, we will determine which individuals were responsible for it and then we will hold them to account.
SOPHIE SCOTT: Dr Morgan is no longer working as a scientist.
Her contract at the university wasn't renewed in 2009.
SARAH GREGSON : I don't think Ying thinks about employment at all, to be honest. I think she's so worried about this particular case and how unjust she sees it as and the implications for patients that I think she doesn't think about herself very much at all.
I really would like to see her go back into a lab.
I think she's dedicated and passionate about science and she's exactly the kind of person you would want doing research.
SOPHIE SCOTT: David Vaux would like to see an independent authority that scientists can go to if they suspect research misconduct.
DAVID VAUX: It would be great if Australia had an office for research integrity or an ombudsman for research integrity so any scientist who had any sort of concern or anybody reading a paper in any country in the world who saw something that they had concerns about, that they knew exactly where to go to and that they could have some place that could in an independent way find out what's really going on.
LEIGH SALES: Sophie Scott reporting there.
The story was produced by Jeanavive McGregor and Lesley Robinson.
Read an edited version of a statement provided by Professor Khachigian in response to questions from the ABC.