Cyndi Frank, co-founder of the Gaucher Community Alliance (GCA) spoke with us about driving their fledgling organisation through listening to the community.
What is the Gaucher Community Alliance and what services does it offer to individuals with Gaucher disease?
Founded in 2019, the GCA has established itself with a clear mission to provide peer support and community-building for individuals living with all types of Gaucher disease. The alliance is a diverse community of individuals living with Gaucher, family members and representatives of Gaucher disease. The team works closely with other representatives, including the executive director of the Canadian NGF, a professional advisory council made up of patients, doctors, industry representatives and other partnerships, who all meet twice a year and discuss how they can work in harmony to best support one another and patient needs.
Cyndi and fellow co-founder, Aviva Rosenberg, are enthusiastic about collaboration and “conquering that patient advocacy triangle” (those with the disease, the industry, and healthcare professionals). The GCA is passionate about keeping lines of communication open, with a strong emphasis on the importance of regular and, most importantly, transparent communication from all parties to tackle unmet needs and issues.
The GCA aims to ensure everyone who wants to be involved in the community is both enabled and encouraged through a “buddy system”. The buddy system offers volunteers partnership opportunities with other individuals who are interested in similar areas of volunteer work and who may have more experience of it. This often helps individuals to feel more involved in the community and feel supported.
What are the biggest needs for the Gaucher community and how well are these currently being addressed?
High prices of drugs and insurance issues are some of Cyndi’s biggest concerns when it comes to community needs. Cyndi explains that the GCA team is working hard to address this area and will not allow this topic to fall silent. The GCA has built a programme around this issue through the use of volunteers who have now formed their own committee.
Issues around treatment access for types 2 and 3 Gaucher are also a problem due to there being no treatment that can cross the blood–brain barrier. With more treatments now available for type 1 Gaucher disease, there is a clear demand for more transparent information to allow people to make informed choices. The GCA is working hard to ensure this content is more widely available to the community via reliable and trusted medical organisations.
Opportunities for research in Gaucher disease: issues around condition management
Gaucher disease affects individuals differently, even within the same type and family. Some individuals have mild symptoms while others will have more severe symptoms, and for some, bone pain, inflammation and fatigue are parts of their symptoms. Cyndi believes that more research is needed into controlled diets for managing symptoms associated with inflammation, such as bone pain. As some emerging studies are beginning to link diet to inflammation levels, Cyndi feels the timing could be right for research in this area for Gaucher and that conversations and research on the topic could lead to a substantial discovery in aiding condition management.
Leveraging technology for global education: GCA’s webinar events
Safe, warm environment where individuals can share information
For anyone considering joining one of GCA’s webinar events, you can expect to hear important discussions on topical news in the Gaucher community. The GCA’s live webinar events offer a secure space to interact with guest speakers by allowing viewers to ask questions in a chat room which can then be answered live. Cyndi describes the webinars as a “safe, warm environment where individuals can share information”.
GCA’s most attended webinar, Gene Therapy: A Personal Gaucher Story, can be accessed freely on the GCA website. The webinar hosts several guest speakers, including Dr Aneal Khan of Alberta Children’s Hospital in Calgary, who discuss both a new gene therapy trial to treat Gaucher disease type 1 and the science behind it. The webinar also features Shondra, the first individual to be dosed in the clinical trial for a new investigational gene therapy. Shondra shares a raw account of her journey from diagnosis to therapy and the reality of how it affected her.
Engaging their community: future plans for the GCA and how to deliver big ideas with a volunteer team
With great excitement and anticipation, Cyndi discusses her plans to run in-person events after Covid-19 restrictions lift, including a large-scale conference to feature speaker sessions and workshops.
Future plans also include a Youth Advisory Committee, a more interactive social media platform to connect with a younger community and welcome boxes for those who are newly diagnosed.
The GCA is committed to services that support long-lasting connections with their community, whether they are adults or young people.
By reaching out to universities and opening the door for internship opportunities, students can help support permanent volunteers and bring particular skill sets to help develop a larger social media presence and form crucial connections between individuals with Gaucher disease.
Get involved: why should individuals get involved in advocacy?
The GCA are always delighted to hear from new individuals who either want to share their personal stories about living with Gaucher or to get involved in volunteer work for the community. The GCA continuously responds to unmet need through listening to their community. The more people who reach out, start conversations and feedback, the better GCA can respond to the unmet need-providing benefit to the wider community and driving more developments in research, collaborations and individual connections.
Rare Revolution Editor