The patient sits in the padded chair. A nurse swabs their arm before slipping a long needle beneath the skin to deliver genetically engineered antibodies into the patient’s blood.
Patients in this position hope the antibodies will melt the thick tumour in their lungs. They’ve likely been told this experimental drug is their last hope.
The drug coursing through their veins is built on the work of Professor Mark Smyth – a scientific fraudster.
The drug is called Nelistotug. It aims to use the immune system against cancer. Smyth’s lab made the key discovery behind the treatment’s development – or at least claimed to have.
Doubts now swirl around his work after a, partly, public unravelling. In 2021, an investigation by his own institute, QIMR Berghofer, found Smyth had committed “serious research misconduct” and referred him to corruption authorities.
Mark Smyth was once one of Australia’s top scientists.Credit:
Some of the world’s leading medical journals, including Nature Immunology, have retracted and corrected his research articles, labelling the data fabricated or falsified – or noting the experiments were likely never conducted.
Still, much about the scandal has remained shrouded from public view, even though taxpayers spent more than $42 million in scarce research funding supporting his work.
An investigation by this masthead can now reveal that serious concerns about Smyth’s conduct stretch back years to his time at the prestigious Peter MacCallum Cancer Centre. It also lays out for the first time how Smyth’s colleagues finally brought him unstuck, via secret recordings and claims Smyth fabricated data from mice he never treated.
The scandal has led to calls from the highest levels of the profession for an overhaul of how scientific misconduct is handled.
“It was like a priest in a parish: everyone knew this guy was fabricating data, he was a bully to his staff, and he went to Queensland. He was bringing in lots of money. People would just close their eyes to it,” said a senior executive in the sector, not authorised to discuss the matter publicly.
CSIRO chief executive Doug Hilton says the Smyth case highlights the need for reform.Credit: Martin Ollman
CSIRO chief executive Doug Hilton said the Smyth case clearly highlighted the need for an independent research misconduct watchdog instead of allowing universities to investigate allegations against their staff.
“I don’t think investigating your own is a good look – it does not pass the pub test.”
Smyth was once at the very top of Australia’s scientific firmament, winning global awards for his cancer research and receiving $42 million in taxpayer research grants, and more from charities.
In 2025, based on papers cited, Research.com still ranked him as Australia’s eighth-best scientist and our best immunologist.
“We almost put him on the pedestal of godlike,” says a former colleague, speaking anonymously to protect their career. “The god of immunology.”
A god who fell to Earth.
To piece together what went on in his lab, as well as previously unreported concerns about his earlier work, this masthead spoke to more than 20 people who worked with Smyth, co-authored papers with him, or investigated his alleged misdeeds.
Nearly all spoke on the condition of anonymity to protect their careers – some have been granted pseudonyms.
The investigation also obtained hundreds of pages of leaked investigations, emails, photos, videos and lab books from inside Smyth’s lab.
As for Nelistotug, the patent for the drug, now being trialled on patients, cites six studies co-authored by Smyth out of 42 underpinning its science. The tally includes one that was retracted because it was based on data Smyth faked.
GSK, the sponsor, insists its process is sound, but two of Smyth’s co-authors on the research behind Nelistotug say they fear the drug itself, which likely cost millions to develop, may not work as intended.
“I felt like the project must have been based on fabricated data,” said Casey*, one of Smyth’s former co-authors, who, like many, spoke anonymously due to fear of damaging their career.
“Even if it does not cause any harm, it’s still very wrong. The patients who take part expect to get a drug that actually works.”
Smyth’s research career has focused on the armies of immune cells that patrol our bodies. He was one of the early believers that these cells could be marshalled to kill cancer – and he was right.
Known as checkpoint inhibitor drugs, these medicines use immune cells to melt tumours and are one of the great triumphs of modern science. Smyth was on the forefront of the hottest field in medicine.
“His reputation was unprecedented, internationally. Huge impact factors all the time, high H index, high citations,” said one former colleague, referring to crucial industry measures of the importance of a researcher’s work.
Smyth was focused on a receptor called CD96, which he hoped to turn into a next-generation checkpoint inhibitor.
Mark Smyth, seen here in a still from a video taken while he was at Peter MacCallum Cancer Centre.Credit: Nine / Supplied
The drug being tested right now, Nelistotug, is exactly the one Smyth dreamed of inventing.
“We think that in future this method will be just as important and effective as other immunotherapy,” Smyth told The Australian in 2016.
We are “at the dawn of a new opportunity in terms of therapy”, he told Channel Nine, owner of this masthead, in 2014.
On camera, Smyth looks nervous and drawn – a scientist out of water.
But in recorded lectures to scientific colleagues, he looks far more assured. Dressed in black with his gelled grey hair, answering technical questions off the cuff, he is a man in command.
“He was a little bit aloof, he had a high opinion of himself. He saw himself as an upper-level person. But he was a nice-enough fellow,” said Brett*, a former colleague who spent time with Smyth outside work and, like others, requested anonymity to avoid professional repercussions.
“His arrogance comes across very quickly,” said a second.
“He had an ab-fab reputation,” said a third, who would later be charged with investigating him.
“That, more than anything else, is the biggest puzzle of all to me. He was not trying to achieve that level of reputation – he’d already achieved it. Why did he feel it was necessary to try for even higher acclaim than he’d already got?”
From the outside, Smyth was a rising star, winning awards, publishing important papers, and being showered in millions of dollars of taxpayer research funding.
But inside his lab, from the earliest days of his career, concerns were emerging.
Selena*, who worked closely alongside Smyth during his time at Peter MacCallum Cancer Centre, said he only ever wanted to know the good news – even though science is littered with negative results.
He “did not want to hear about things that weren’t working. He wanted to see finished results. He did not want to know how it was being done,” she said.
“You’d present raw data, and he’d say, ‘You can just leave those points out – they are outliers’.”
Other scientists sometimes could not reproduce his results. But rather than question Smyth, they often questioned themselves.
“Maybe he’s got better hands than I have. Or maybe the mice are different,” said Brett*. “There are all these variables.”
In 2004, Smyth was the senior author on a paper in top journal Nature Immunology, which was such a sensation that his co-author was nominated for a National Association of Research Fellows award, where Professor David Vaux was secretary.
David Vaux had concerns about Smyth’s research.Credit: Photographic
Vaux, 65, is one of Australia’s most important cancer researchers, past deputy director of the Walter and Eliza Hall Institute and winner of as many prizes as Smyth.
He is also one of the very few researchers willing to take on the scientific establishment when he believes someone is committing research misconduct.
A third thing to know about Vaux: when he goes to the doctor, he loves to read the Australian Women’s Weekly. The puzzles where you have to spot differences between two images are his favourite.
Vaux had first come across Smyth in 1995 when he was asked to comment on a paper the young researcher submitted. He came across paragraphs that seemed similar to Vaux’s own work. But when the paper was published, those paragraphs had disappeared. Still, Vaux kept half an eye on the rising star.
Years later, he found himself flicking through a 2004 paper Smyth had co-authored.
It contained rows of flow cytometry plots of immune cells. Each dot is meant to represent a cell.
“I just looked at them, fused the images, and it was immediately clear they had been duplicated and altered,” said Vaux.
The dot pattern kept repeating, as though someone had cut and pasted together the same images in a different order. Each plot contains 10,000 cells. “The chances of two plots having the same pattern of dots would be 1 in 10 to the power of 1000.”
Vaux emailed the paper’s authors.
“I can clearly see the problems – one dotplot has been duplicated and modified and used for at least 6 of the plots presented in that revised figure. I still haven’t been able to track Mark down,” one wrote back. “I feel sick.”
Nature Immunology launched an investigation and in 2006 retracted the paper because “it contains a number of errors, including duplications of some flow cytometry plots”.
To this day, it is not clear if it was Smyth who duplicated the plots. But a retraction is an enormous black mark on a scientist’s career.
Peter MacCallum Cancer Centre put in place compulsory research integrity training, including a seminar entitled “Scientists Behaving Badly: Fraud & Misconduct”.
This was only the start of Smyth’s troubles.
In 2014, a thin unmarked envelope was slipped under David Vaux’s office door.
Inside, under a cover note from “a concerned scientist”, was a copy of a secret Peter Mac investigation into Mark Smyth.
The investigation started in 2012, when one of Smyth’s PhD students was running a cancer experiment in mice. But the data wasn’t good. It looked like another negative experiment.
Then, according to the student’s evidence, Smyth provided him with a new spreadsheet. It contained records of 20 mice Smyth claimed he had kept as a “side project”.
Smyth said he’d been running the same experiment – with better results. He suggested combining the data, making the results much more positive.
At Peter Mac, mice were tracked closely on its Mighty Mouse database, which recorded their births, deaths and every experiment.
The student could find no record of Smyth’s additional mice on Mighty Mouse. He told Smyth, who suddenly advised tossing the new data.
Instead, the concerned student went to Peter Mac, which launched a preliminary investigation.
Smyth’s personal lab book contained “not entirely convincing” partial records for 14 of the mice, “crowded into unlikely spaces”, the preliminary investigative report, also obtained by this masthead, says.
A further six mice were recorded in a book belonging to a lab assistant.
But she told the inquiry she had no memory of monitoring the mice or writing the data in her lab book. She said the handwriting was not hers.
“The animal technicians are right on the ball. If they say a cage of mice, that they know Mark is talking about, never existed – it’s not really possible,” one investigator told this masthead, speaking under condition of anonymity to detail confidential information.
Peter Mac’s preliminary investigation found no independent evidence the mice ever existed and concluded Smyth had a case to answer.
“I thought it wasn’t marginal,” said the investigator. “I thought at the time: ‘This guy is in serious trouble here’.”
But under an unusual arrangement, the University of Melbourne is responsible for conducting research misconduct investigations at Peter Mac. The sandstone institution would conduct the full investigation.
The Peter MacCallum Cancer Centre in Parkville.Credit: Justin McManus
A finding he had made up about mice could end Smyth’s glittering career.
But Smyth’s luck turned.
On the day of his hearing in front of an expert panel, a Peter Mac employee produced a new datasheet.
It was apparently written by Smyth and then “mislaid”. The employee said they found it while clearing out his office.
It contained an error-riddled record for the 20 mice.
A handwriting expert brought in by the inquiry determined both this rediscovered loose sheet of paper and the records in the lab assistant’s lab book – the ones she said she did not write – were likely written by the same person.
That person may have been Smyth, the panel was told. But the expert couldn’t be sure.
Smyth claimed the central database that recorded mice was often faulty and not fit for purpose. Two of Smyth’s colleagues told the panel they had similar problems with the database.
One of those colleagues was the person who found Smyth’s “mislaid” data sheet.
The other, Robert*, now says the panel misconstrued his evidence.
“When they asked me directly if the mice in question could have existed, I was very clear and responded with a ‘no’,” he said.
“It does haunt me that my statements have been twisted to allow Mark to escape punishment.”
The panel concluded Smyth did not make up the data. He was in the clear.
“It’s very hard to understand how Melbourne University could say he wasn’t fabricating the data, making it up, and then six or seven years later he’s done exactly the same thing at QIMR,” Robert told this masthead.
“Melbourne University needs to take some accountability for allowing Mark to continue misleading scientists and patients.”
A senior Australian scientist with close knowledge of the case, speaking anonymously due to restrictions in their employment contract, is absolutely scathing.
“As they’d done with a number of integrity cases, [the University of Melbourne] … concluded there was nothing to see here,” they said.
“The institutional lens is: we have to avoid any suggestion the University of Melbourne has dodgy people, so let’s find him not guilty and move him on.”
University of Melbourne deputy vice chancellor Professor Mark Cassidy said in a statement that all complaints and allegations were taken seriously and addressed in line with the appropriate guidelines.
GSK said its oncology research and development program was “robust”.
“Our investigations of Nelistotug in combination with other therapies is always based on the full breadth of scientific evidence available,” it said.
Smyth was hired by QIMR in 2012, before the Peter Mac allegations were made, and left to join the Queensland-based institute in 2013 – before the investigation was concluded.
The allegations he faced soon became the subject of water-cooler gossip, both in Victoria and in Queensland.
“It was a pretty open secret at Peter Mac that Mark Smyth was fudging data,” said a former Peter Mac PhD student. “No one believed it. It all looked fake.”
When this masthead approached Smyth he was unloading golf clubs.Credit:
Smyth himself has never spoken publicly about the saga, he left QIMR and this masthead was not able to confirm where he was now working.
Approached recently at a house in a leafy Brisbane suburb a few minutes’ drive from his former QIMR lab, he said he was “not interested” in responding to the allegations, immediately turning down a printed list of questions as he unpacked golf clubs from his car.
“No thanks, I’ve been asked … a million times,” Smyth said.
Asked twice if he stood by his work and research, he said: “Can you please just get away. I’m not interested. See you later.”
Tomorrow: Inside Mark Smyth’s QIMR Lab
*Names in this story have been changed to protect sources.
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