Clinical trials: a roadmap to the future of innovation

Diagnosed with cystic fibrosis at 16 months, Kim Wood never truly knew what it felt like to take a full breath.

Thanks to her dedicated health care team and her parents’ careful commitment to a supportive health regime, Kim fared well during childhood and as a teen. In fact, she was so inspired by the caring treatment she’d received all her life she became a nurse.

While in her early 20s, Kim’s health took a downward turn. As her illness progressed, Kim experienced frequent lung infections that required hospitalization.

“I was almost spending more time in the hospital than out of it,” she says. “It was really challenging and overwhelming being stuck in a hospital room, having all of your control taken away from you.”

Then came Dr. Bradley Quon and his clinical trial evaluating Trikafta at St. Paul’s Hospital. Dr. Quon is the medical director of the CF Adult Care Program at St. Paul’s Hospital, and a researcher at the Centre for Heart and Lung Innovation. Fortunately, Kim was a prime candidate to try the drug. After decades of living with cystic fibrosis, Kim realized she could finally breathe – and live her life to the fullest.

“I started to feel that complete, unrestricted breath. It was astonishing because I had never felt like this, even when I was really young on a good, healthy day,” Kim recalls. “I was like, ‘Oh my gosh, this is what breathing is supposed to feel like’. It was absolutely mind-blowing.”

Medical breakthroughs from clinical trials benefit us all, but here in Western Canada we don’t have the proper infrastructure to support enough trials at their beginning stages. With early access to game-changing treatments, we could be saving lives a whole lot sooner.

And Providence Health Care is about to make this happen.

“Today’s interventions are the results of yesterday’s clinical trials,” says Dr. Darryl Knight, president of Providence Research. 

Clinical trials are life-changing and life-saving. A trial could test a drug, surgery or medical device, or a behavioural intervention.

They give clinicians the chance to find better treatments, make drugs safer, and discover new therapies. Researchers spend many years developing therapies and assessing their safety.   

There are several important stages of clinical drug trials in Canada:

Phase 1: The therapeutic or therapy is tested on a small group of people. This phase is also known as ‘first in human’. Researchers determine if the treatment is safe overall, if there are any side effects, and the safest dose to give.

Phase 2: The treatment is tested on a larger group of people (about 100-300) with a specific disease or health condition. In addition to testing whether the treatment is safe and has side effects, researchers determine whether the therapy effectively treats the condition and the optimal dose.

Phase 3: The treatment is tested on an even larger group of people (1,000 or more) with a specific disease or health condition. Researchers study the considerations in previous phases, and compare the new treatment to the existing standard treatment. Trial participants could receive the new treatment, the existing treatment, or a placebo.

Phase 4: This phase happens after Health Canada approves a drug. Researchers continue to study the treatment to gather more information, find new ways to use it, and determine its long-term benefits and risks.

For over a century, St. Paul’s Hospital has distinguished itself with incredible innovations to solve some of the most pressing health problems of our time.

We’ve already developed decades of expertise in clinical trials, conducting over 200 Phase 2 and 3 trials annually – which have saved lives, and improved quality of life for our patients. For example:

Dr. Bradley Quon’s Trikafta trial helped 25 patients with cystic fibrosis breathe again.

Dr. Liam Brunham’s pivotal trial is studying whether an injectable medication called Inclisiran, when taken alongside statin therapy, will reduce the risk of heart attacks, strokes, and cardiovascular deaths.

Dr. Christopher Ryerson is testing if inhaled Treprostinil, a medication for pulmonary fibrosis, works to improve lung function.

In Canada, there isn’t a straightforward path to guide new therapeutics from idea to research to approval. Our scientists have an excellent reputation in developing and discovering ideas we could bring to fruition, and fine-tuning them in the pre-clinical phase. We’re also skilled at participating in existing trials once they reach Phases 2 and 3.

But there are very few hospitals in Western Canada that have the capacity to conduct non-cancer, Phase 1 clinical trials.

And this gap is delaying life-saving therapies for British Columbians.

Phase 1 trials are a critical step in the research process. They can save lives, enhance quality of lives, and offer hope to patients who are out of options.

Without the meticulous scrutiny that takes place in the first phase to demonstrate outcomes and patient safety of potentially life-saving therapies, a trial cannot move ahead to the second and third phases. Once a drug reaches Phase 3, it is more likely to be considered for approval from Health Canada.

Yet by the time a drug reaches the second or third stage, and available for patients at our facilities in trials, it could take four years.

And for some patients, that might simply be too late.

When we don’t have the capacity to run Phase 1 trials in BC, we also risk losing vital clinical research and the brilliant minds behind them to other countries.  

For example, despite a wealth of basic scientific discoveries made here, Canada was not self-reliant and couldn’t significantly contribute to the global COVID-19 effort.

We couldn’t conduct early phase clinical trials rapidly, further hindering pandemic response efforts during the global health emergency. Of the nearly 130 clinical trials evaluating COVID therapies in Canada, fewer than 10% were first in human – and even fewer involved products were developed in Canada.

And imagine how many lives could have been improved if Dr. Quon had access to Trikafta during its first phase of testing.

A large hurdle to Phase 1 trials is quite basic: we need hospital beds to complete them.

During first in human drug trials, patients require 24/7 monitoring and care to ensure they are safe. Here in BC, researchers are competing with acute hospital care for beds – and as there often aren’t enough beds to go around, patients in acute need are a priority.

There are very few hospitals in Western Canada that can conduct non-cancer, Phase 1 clinical trials. We’ve taken a big step forward to address this.

Thanks to a $4.2 million investment from the BC government, we’re launching a Phase 1, non-cancer focused clinical trial unit at Mount Saint Joseph Hospital. The eight-bed unit will open in 2024.

And this is the just the beginning of British Columbia’s position as a destination for clinical trials that will yield new discoveries, create more high-quality jobs, and keep intellectual property in our own backyard.

Innovation has always been a metaphorical bridge connecting patients with advancements in therapies and treatments. With the Clinical Support and Research Centre (CSRC), we will have a real bridge connecting to the new St. Paul’s Hospital bringing clinicians and researchers even closer to work side-by-side to change and improve the lives of British Columbians now and for generations to come.

The CSRC will be one of the only facilities in Western Canada with the potential to run all phases of non-cancer clinical trials where our scientists could take critical, life-saving treatments from concept to completion, and keep our patented therapies within BC.

With the world’s leading clinicians, scientists, and researchers right next door to one another at the new St. Paul’s Hospital and CSRC, we’ll be able to easily connect and partner with patients interested in participating in clinical trials – and take therapies from the lab bench to the patient’s bedside quickly.

“We are at the forefront of both research and clinical care,” says Dr. Bradley Quon. “Working adjacent to the hospital, we can easily take discoveries in the lab and translate them into better treatments for our patients.”

The CSRC is a true collaborative ecosystem, where researchers and clinicians will come together with the patient to find treatments, and possible cures, for some of the most pressing illnesses we face today.

“There’s nothing else in Canada comparable to what we’re planning. If you’re involved in health care research in BC – as a scientist, a partner, or a patient – this is the place to be,” says Dr. Knight. “As a researcher myself, I can tell you that the CSRC will be a game changer.”

This kind of dedicated, push-the-envelope research means the world to patients like Kim Wood.

“Every day, I feel grateful for having the privilege to take that medication and have it affect my body in the positive way that it has,” she says. “I don’t know if I would be here without Trikafta, and I don’t know if I would be able to be the kind of mom I’ve always wanted to be for my little boy.”

Clinical trials are essential to developing newer, better medical treatments. Learn more about the life-changing research happening across Providence at the link below.