The man who brought Betadine throat gargle to Australian households against the will of big US pharmaceutical companies has set his sights on developing drugs to fight rare cancers.

Peter Molloy transformed a hospital antiseptic into Betadine throat gargle in 1984, and it has gone on to become Australia's largest selling over-the-counter sore throat treatment — but it never gained approval for sale in the US.

"The American company behind it saw it really as a hospital antiseptic but I saw it as something that could treat sore throats for example," Mr Molloy said.

"I think what it speaks to is the constrained thinking that you see in big companies."

Peter Molloy believes there is a role for Bisantrene. ( ABC News: Tom Gelai )

Mr Molloy has now formed a new company, Race Oncology, with the purpose of picking up what he calls the gems that "Big Pharma" discard or overlook because the sale potential is simply too small for them to pursue.

The first drug in his sights is Bisantrene, which could benefit sufferers of a rare blood cancer.

It never made its way to market after its discovery by big pharmaceutical companies in the US more than four decades ago, despite 40 clinical trials involving 2,000 patients during the 1990s.

Drug offers hope for AML patients

Bisantrene proved most beneficial to sufferers of the rare cancer acute myeloid leukaemia (AML), which is considered an "orphan disease" because it only affects around 40,000 people globally.

"In those studies … even in patients who had been treated with multiple rounds of other therapy and failed, we were still able to see a response from Bisantrene. That's a pretty good indicator that there is a role for this drug, Mr Molloy said.

"There are many of these drugs out there which have just been overlooked by Big Pharma.

"Particularly as a result of mergers and often because the drug doesn't meet a substantial market opportunity, and yet it could still solve some important medical conditions."

Race Oncology has secured the patent rights to develop Bisantrene and gained orphan drug designation in the US, meaning the company can apply for development incentives to undertake qualified clinical testing.

"The idea with Bisantrene is it could give new hope to those patients, it could represent an important salvage therapy when all other therapies have failed," he said.

Finding the answer to 'hell on earth'

AML took the life of four-year-old Perth boy Kai Nell in January and has haunted Australian professional golfer Jarrod Lyle for decades.

Kai Nell died from acute myeloid keukaemia. ( Supplied: Family )

Despite being given an up to 60 per cent chance of survival, Kai lost his battle with the disease.

"It's literally hell on earth for them," his mother Kara Nell said.

"We've held our own child as he's suffered … we've held him as he's died, which is why we feel so passionate no other family should go through this, that's why we can't turn away from it now."

The family searched the globe for a cure but failed to find any researchers looking for better treatment methods. They are now determined to highlight the lack of investment in treating rare cancers.

"We had all sorts of experts around the world looking at his medical records to try and get a second opinion, third opinion," Kai's father Richard Nell said.

Kai's parents Kara and Richard say they want more investment in treatments for rare cancers. ( Supplied: Family )

"[Princess Margaret Hospital] threw everything they had at it. It's only as you get into the process months and years down the line that you find out just how little there is going into research for new drugs — especially for the rare, less common cancers."

Clinical trials in the 90s showed Bisantrene could provide a vital alternative to anthracyclines, the first response drugs given to cancer patients.

Anthracyclines cause high levels of cardiac toxicity, which can cause consequent heart failure — meaning oncologists often have to cease treatment even if the drug is working — but studies have shown Bisantrene has much lower toxicity levels.

While the number of people living with rare cancers is small compared to more common ones, half of cancer-related deaths are attributed to the rarer strains.

Pro golfer's fight far from over

Victorian golfer Jarrod Lyle plays a shot on the 8th during day 2 of the Australian PGA Championships at Coolum on the Sunshine Coast on December 5, 2008. ( Dave Hunt: AAP )

Lyle was first diagnosed with the disease in 1998 at the age of 17 and the golfer has just started battling his second relapse, discovered by doctors in July.

The move to commercialise the drug brings the Lyle family hope there will soon to be a bigger medical arsenal to help fight the illness.

"It's a very promising sign, after reading up on it and seeing the advantages of it and what it does and how it doesn't affect the heart," Lyle said.

"Hopefully in years to come it will be available here in Australia and people can make use of it and access it.

Australian golfer Jarrod Lyle, with daughter Lusi, is receiving fresh treatment for AML. ( Supplied: Briony Harper )

"You kind of wish there was more people like Peter who were looking in the drugs that were going to help the less common cancers.

"You just wish the big pharmaceutical companies would see the benefits of it and help out the little fish a bit more."

Lyle is set to start another round of chemotherapy before a bone marrow transplant.

Race Oncology hopes to first gain approval to sell the drug on compassionate grounds in Europe to help fund the ongoing development of the drug, with a view to gaining Food and Drug Administration approval.