When someone has a disease, like ALS, their quality of life is affected in many different ways as the disease progresses. “Quality of life can mean many different things to different people. For some, it means functional abilities, like walking to the mailbox, but for others, it may mean leisure activities, or family and social relationships,” said Dr. Vanina Dal Bello-Haas, a clinical and research expert in rehabilitation for people with ALS and dementia at McMaster University.

Yet, when clinicians assess the quality of life of people living with ALS, they often use a standard assessment tool that was not developed with input from people with ALS. “Existing measures used in ALS do not appropriately reflect the preferences or values of people who are living with the disease,” said Dr. Ayse Kuspinar, a clinician and researcher at McMaster University who specializes in quality of life research and developing patient-reported outcome measures.

To address the shortcomings with existing ALS tools, Dr. Kuspinar and Dr. Dal Bello-Haas recently received a $125,000 project grant from the ALS Canada Research Program to create a brand-new measure called the Preference-Based ALS Index, in partnership with people living with the disease.

Dr. Dal Bello-Haas and Dr. Kuspinar anticipate that the new Preference-Based ALS Index will help healthcare providers identify gaps in care and tailor interventions that may improve the lives of people with ALS. They also expect it will be a valuable tool to assess the impact on quality of life of new therapies in future clinical trials.

Incorporating the voice of people with ALS

For their project, Dr. Kuspinar and Dr. Dal Bello-Haas will ask over 400 Canadians with ALS to help them develop a list of factors that affect their day-to-day quality of life. They expect that the factors will include the physical impacts of the disease, such as mobility issues and ability to speak, but also emotional and social impacts such as changing relationships with family and friends, ability to work and their social life.

After generating the list of factors, the researchers will draft a series of ALS-specific questions for the tool in collaboration with people with ALS and a team of ALS expert clinicians from the Canadian ALS (CALS) Research Network, a consortium of 15 academic ALS clinics across Canada. Co-investigators on the project include recognized neurologists Dr. Wendy Johnston, Dr. Colleen O’Connell, Dr. John Turnbull, and Dr. Marvin Chum. The research team also includes a senior knowledge translation expert, Dr. Joy MacDermid, who will assist with coming up with a plan to spread the study results to the wider ALS community.

Once the questionnaire is prepared, Dr. Dal Bello-Haas and Dr. Kuspinar will interview people with ALS, asking them to answer two to three questions to make sure that they are appropriate and take into consideration the realities of living with the disease.

A weight to each factor will be assigned depending on an individual’s values and preferences and calculate a total score that reflects their pluses and minuses. This approach is a significant improvement over existing tools that calculate a total score by adding up unadjusted ratings. The researchers will also translate the Preference-Based ALS Index into French and test it with volunteers with ALS to ensure the meanings are the same in both languages.

Advancing ALS research

Incorporating the Preference-Based ALS Index into clinical trials may also help speed drug approvals for new therapies. As Health Canada and provincial funding authorities consider a new drug approval and funding process that will require information about the value of a new treatment based on the lived experiences and preferences of people living with diseases, this database will allow the voice of people living with ALS to be brought forward. “When making decisions about which treatments should be approved, regulatory bodies are shifting their approach to look for treatments that are the most cost-effective and have the highest impact on quality of life,” said Dr. Kuspinar. “Faster drug approvals will mean quicker access to new therapies for people with ALS in the future.”

The project will begin recruiting volunteers with ALS to contribute their input to the development of the tool this spring. The first sites will open in Hamilton, Edmonton and Fredericton. If you are interested in participating, please contact Ayse Kuspinar, Project Lead, (kuspinaa@mcmaster.ca) for more information.

This research project is one of 8 research projects funded in 2018 by the ALS Canada Research Program, which is the only dedicated source of funding for ALS research in Canada. The funding of the project followed a rigorous scientific assessment by panels of global ALS experts. The panelists evaluated a larger pool of applications to identify the projects that demonstrate scientific excellence and have the potential to most quickly advance the field of ALS research in order to develop effective treatments.

ALS Canada is a registered charity that receives no government funding. Everything we do – from funding research to providing community-based support for people living with ALS – is possible only because of donor generosity and partnerships with provincial ALS Societies who contribute to the ALS Canada Research Program.

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Posted in: Research