For a disease like ALS that has few treatment options and no cure, clinical trials are the best hope for the future. Participants in clinical trials play a critical role to help determine if a new treatment can slow, halt or reverse disease progression, or if a new intervention can improve the quality of life for themselves and others living with ALS. At the same time, they may gain access to new therapies before they are widely available and may be among the first to benefit if those therapies work.

“Participation in research is very meaningful to people with ALS and their families. There is a tremendous desire to give back, to add to knowledge about ALS and its treatment,” says Dr. Wendy Johnston, a neurologist who is director of the University of Alberta’s ALS Clinic and a professor at the University of Alberta in Edmonton. “It is also recognized that those who participate in clinical trials do better overall than those who do not, whether the trial drug worked or not. This may reflect the beneficial effect of hope or better monitoring of the disease progression.”

The challenge? Clinical trials are costly and time-consuming to set up and execute from start to finish. Large drug companies and investigators run clinical trials in locations where they can be assured the studies will be executed effectively, efficiently, and in large enough groups of people to establish whether the new treatments can make a measurable difference.

Stronger Together

In Canada, a group of ALS clinicians and researchers is helping to pave the way for Canada to become a preferred country for companies to conduct ALS clinical trials. The group began working together at a grassroots level in the 1990s and adopted the name CALS – the Canadian ALS Research Network – in 2008 under the leadership of Dr. Lorne Zinman, Director of the ALS Clinic at Sunnybrook Health Sciences Centre in Toronto. Many other leading neurologists played a pioneering role in establishing the network of clinicians that led to the development of CALS.

“We started out as a formal network of neurologists working together to improve patient education and help people with ALS gain access to the drug riluzole,” explains Dr. Colleen O’Connell, a physical medicine and rehabilitation specialist who leads the ALS Clinic at the Stan Cassidy Centre for Rehabilitation in Fredericton, New Brunswick and is an assistant professor at Dalhousie University in Halifax, Nova Scotia.

“We decided as a group that creating a research network would improve the availability of clinical trials across Canada,” says Dr. Johnston who, like Dr. O’Connell, she has been involved with CALS since its early days. “The larger centres were routinely being approached, but smaller, newer centres were not. Operating together as one network meant that we could coordinate responses to proposals from drug companies and investigators, and get trials up and running in a timely fashion with excellent recruitment.”

CALS Proves Its Mettle

In 2009, CALS was put to the test to facilitate Canada’s participation in a multicentre, international trial for lithium, an existing drug prescribed for mental disorders including bipolar disorder, depression and schizophrenia. The excitement about lithium as a possible treatment for ALS resulted from a small pilot study in Italy among 16 people that showed dramatic slowing of disease progression when taken together with riluzole. It was believed that lithium could help remove misfolded proteins and other cellular components that were not functioning properly. To confirm or refute the pilot study findings, CALS collaborated with investigators in the United States to design and execute a multicentre trial in a larger group of people. The trial took place at 15 sites across Canada, including large sites in Toronto, Montreal and Vancouver, as well as smaller sites in New Brunswick and Manitoba.

The researchers had planned to run the trial for up to a year but stopped it after six months when results showed that lithium was worsening participants’ ALS. This finding was particularly important given that some people with ALS were using lithium off-label outside of the study: where clinicians were aware of this usage they were able to advise their patients that lithium was doing more harm than good. “Another notable benefit of the lithium trial was that it put Canada on the map as an ideal location for ALS clinical trials,” said Dr. Johnson. “We demonstrated that as a group, CALS could respond to a research proposal, get a trial approved by Health Canada and recruit participants with ALS in a timely manner.”

Gaining Momentum in 2017

Dr. O’Connell and Dr. Johnston recently took the helm of CALS as co-chairs of the network in April 2017. Together with CALS members at 15 sites across the country, they are building on a strong foundation focused on helping people with ALS. “One of the visions that Dr. Johnston and I share is to keep our fingers on the pulse of new treatment opportunities. Our goal is that if people are interested, they can access information and participate in clinical research,” said Dr. O’Connell.

With an unprecedented uptick in the level of activity of ALS research worldwide, CALS is ready to meet the clinical research needs of large pharmaceutical companies and investigators alike. This fall, several new studies will be launched at sites across Canada. “We have attracted two big industry-sponsored drug trials, we have two investigator-led trials in preparation, and we are ready to start the pimozide trial pending final ethics review and regulatory approval,” said Dr. Johnston.

As the number of ALS clinical trials in Canada grows, more Canadians living with the disease will have earlier access to leading-edge experimental therapies.

In addition to bringing Canadian researchers together to share learning in person at the annual ALS Canada Research Forum, ALS Canada is providing administrative support to CALS to help the team establish a communications platform that will streamline information sharing between study sites.

CALS is supported administratively by the ALS Canada Research Program. Learn more about the ALS Canada Research Program and consider donating today.

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