A Canadian surgeon's painstaking research into a rare tumour has led him to a surprising discovery that could benefit millions and earn billions.
For two decades, Benjamin Alman, the head of orthopedic surgery at the Hospital for Sick Children in Toronto, sought a treatment for desmoid tumours, which afflict about 30 Canadians each year. Though non-cancerous, they require surgery – sometimes even amputation.
What he found is an old analgesic used in Europe that when reformulated has a new use. In doing so, he appears to have hit upon a discovery sought since ancient times: how to dramatically reduce scar formation. By turning an old molecule into a cream and applying it daily for three weeks, scars from surgery grow to less than half of the expected size.
The molecule – which was not identified pending publication of a scientific paper – constitutes one of the biggest projects developed by MaRS Innovation, a government-funded organization that helps commercialize the ideas of scientists working at Ontario's universities and teaching hospitals. The discovery has since spawned an agreement with Novotek Therapeutics Co. Ltd., in China.
"It's very exciting to think any one doing fundamental research can understand a gene defect causing a problem, be able to find a drug to target it, to actually having it potentially used for patients," said Dr. Alman, also chair of orthopedic surgery at University of Toronto, who has so far conducted animal studies. "That's what biomedical research is all about. That's about as exciting as it can get."
Every surgical patient is a potential customer for the cream, expected to retail for $350 per treatment course. Patients most likely to seek it are those undergoing reconstructive surgery due to tumour removal, burns, or from repairing congenital malformations, as well as women undergoing cesarean sections and seniors with joint replacements.
"It's a massive market that no one has been successful in achieving," said Ivan Waissbluth, project manager of Life Sciences for MaRS Innovation. The U.S. market is estimated at $4-billion; North American clinical trials on the cream are to be led in Toronto, in early 2013.
Commercializing science is a growing, necessary trend, say experts, as too many discoveries have been bought for next to nothing – insulin was sold for $1 by the University of Toronto – or fizzled out, due to lack of financing or interest.
"The reason why they decided to do this," said Rafi Hofstein, president and chief executive officer of MaRS Innovation, "is because they felt that there's a gap between the quality of academic research and commercialization of the outcome."
Commercializing science not only provides a product to help patients, but the proceeds will get re-invested into research and innovation, according to Mary Jo Haddad, president and chief executive officer of Hospital for Sick Children and board chair of MaRS Innovation, adding that it "has the potential to be very, very significant."
The hunt for a scar treatment has been sought since early times. Remedies of frankincense and fermented fruit juices have been scratched out on papyrus. People have applied a mixture of red ochre and kohl. Even gum was stuck to burns. More recently, incisions have been slathered with vitamin E, despite a lack of proof it works.
At least 19 different attempts to create an anti-scarring treatment have been documented by pharmaceutical companies in several countries including Canada, New Zealand, France and the United States – all to no avail.
In this case, Novotek will spend about $6-million (U.S.) to bring the drug called ScarX onto the Chinese market, according to its chief executive officer, Jubo Liu. In exchange for developing and commercializing it, Novotek keeps the Chinese market; MaRS Innovation receives data, milestone payments and royalties from China but is able to sell it in other worldwide markets.
"Scar forming has been a big concern for most of surgery patients after they have gone through a critical stage of their life, some time a life and death situation," wrote Dr. Liu in an e-mail. "… I believe this will help the patient to recover faster and enjoy a happier life."
Dr. Alman's research began about 20 years ago, when he was an orthopedic surgery resident and he saw a young boy with a desmoid tumour wrapped around the blood vessels and nerves in his arm, leaving him with a functionless hand.
"I just couldn't believe that somebody would actually get an amputation for what is supposed to be a benign tumour," Dr. Alman said.
He conducted research into the mutations that caused the tumour, then looked at how those mutations changed the way the cells behave. He then did drug screening to see what might inhibit the cells.
When MaRS Innovation got a hold of his research, they recognized the agent he found – a molecule used as a pain pill stronger than aspirin in some parts of Europe – targets myofibroblast cells in a tumour.
Dr. Alman hopes that by getting it approved for use as a topical agent in scar patients, he can get it developed in an oral form for that 1 out of a million patients each year, who are afflicted with the rare tumours.
"I can't imagine a drug company spending money to develop a drug that's going to be used for 30 patients a year," said Dr. Alman, noting the huge cost in bringing a drug to market. "But I can imagine a drug company spending money for a drug that's going to be used by millions of people a year."
THE SERIES The future of health care Over the next year, The Globe will explore the innovations that are reshaping Canada's health care.