Toronto-area high school student Marshall Zhang took first place this week at a national science contest for developing what could become a new drug cocktail to treat patients with CF, a genetic disorder that affects the lungs and digestive system.
Zhang, a Grade 11 student in Richmond Hill, Ont., used the Canadian SCINET supercomputing network at the Hospital for Sick Children in Toronto to identify how two compounds interacted with a protein on a mutant gene that's responsible for most cases of CF, called Delta F508.
Using the computer modeling, he looked at what these compounds might do at the molecular level to "correct" the genetic defect that marks CF. He found that two drugs each interacted with different parts of the mutant protein and then worked together in a whole new way as well.
"Originally, I thought it would be a 'one plus one equals two' kind of thing, but when we got our results back from the lab, it actually turned out that one plus one equals three. The two compounds were working together synergistically to correct that disease-causing defect," Zhang told CTV's Canada AM Thursday
"But beyond that, I've also laid a foundation for future structure-based drug design, in identifying certain chemical structures that have a key role in correcting that defect."
Zhang's discovery on the computer was the first step. Proving his 'virtual' findings using living cells in culture was the next step.
To his surprise, he discovered that his hypothesis was correct: the cells that were treated with both compounds were able to function as if they were healthy cells. Zhang was stunned to find that his experiment worked on the first try.
"A lot of the time, you make your hypothesis, you do your experiment and a lot of the times, it doesn't work out," he says.
Zhang really needed his hypothesis to bear out quickly because he only had enough time to try the experiment once. So he was delighted when it worked.
"It was just like ‘Wow'. You don't believe what you're seeing. It was amazing. It's really rare for an experiment to work on the first time," he said.
While the serendipitous finding is exciting, it remains to be seen whether it translates into living humans. Zhang notes that oftentimes, the way something works in cells cultured in a lab ends up being much different from the way it works in humans.
"We would have to test for toxicity and how the human body metabolizes all these compounds. Certainly, while this is a first step for these two compounds, it's a long way from developing these drugs," he said, noting that any resulting drug is probably 15 years down the pipeline.
"But I think what's more important about my project is laying the foundation for identifying these chemical structures that we might be able to use on different compounds in the future."
Zhang's project mentor Dr. Christine Bear, a researcher at the Hospital for Sick Children's Research Institute, says Zhang's findings show that computational methods can drive the discovery of compounds that lay the groundwork for drug development.
"I think that Marshall has tremendous potential to be a scientist in the future because of his intelligence, motivation and determination," Dr. Bear said in a statement.
Zhang's discovery so impressed eight scientists at the National Research Council of Canada laboratories in Ottawa, they awarded him first prize today in the 2011 Sanofi-Aventis BioTalent Challenge.
Zhang earned $5,000 for his first-place finish, a prize he will now share with his school, Bayview Secondary School in Richmond Hill.
He will go on to compete against American and Australian teams at an international challenge in Washington, called the International BioGENEius Challenge, in late June.
"That's going to be really exciting," he said.