Collaborating with Patient Advocacy Groups to Advance Rare Disease Awareness and Support

Posted March 29th, 2022 by Ivana Magovčević-Liebisch, in From The Trenches, Patients

By Ivana Magovčević-Liebisch, CEO of Vigil Neuroscience, as part of the From The Trenches feature of LifeSciVC

Over my many years in the biopharma industry, working across disease areas, including orphan indications, I have learned that to impact patients’ lives credibly and meaningfully, we must raise awareness about the conditions we are attempting to treat. One of the most effective ways is to engage with patient advocacy groups who are more closely connected to patients and caregivers and have a deep knowledge of their needs. Access to patient groups is particularly important for work related to rare disease indications given the challenging combination of urgent patient needs, and limited resources and knowledge.

Founders of patient advocacy groups typically have close connections to the condition, either through a personal or familial diagnosis. These ties to patient communities can enhance patient advocacy groups’ knowledge about how best to support the community mentally and emotionally. Partnering with patient advocacy groups can also spark new ideas for ways to meaningfully engage with and support the patient and caregiver community.

Raising Awareness: First ALSP Awareness Month

March marks the very first awareness month for adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP). ALSP is a rare neurological disease caused by an autosomal dominant genetic mutation in the CSF1R gene with high penetrance. It affects an estimated 10,000 people in the US, with about 1,000-2,000 new cases annually. The disease epidemiology is similar in Europe and Asia.

The patient journey with ALSP varies from person to person – even patients from the same family can experience different symptoms. The disease can affect judgement, personality, behavior and movement. Although ALSP is fatal and has a devastating impact on the daily lives of patients and their families, there are currently no FDA-approved treatments and symptoms are treated off-label with existing medications.

Few people have heard of ALSP. This lack of awareness has a big impact for ALSP patients, who are often misdiagnosed or not diagnosed early. More common neurological conditions, such as multiple sclerosis, have public resources and expertise readily available at diagnosis. However, ALSP patients will not find a wealth of information available online or via a pamphlet in their doctor’s office. There is an urgent need to increase awareness in the medical and lay communities of this disease to help improve diagnosis, drive investments in treatments, and ensure patients and caregivers have information easily available as they navigate living with ALSP.

ALSP Awareness Month represents an important effort between Vigil and the Sisters’ Hope Foundation to raise awareness and support ALSP patients, caregivers and families.

Sisters’ Hope Foundation is a non-profit organization supporting families fighting ALSP. President and Founder of Sisters’ Hope, Heidi Edwards, has lost five family members to this condition, including her sister Heather and twin sister Holly, both who passed away in the last two years. Heidi started the Sisters’ Hope Foundation to honor her sisters, raise awareness and educate people about the disease.

The theme for the first ALSP Awareness Month is “Bridging the Gap in ALSP Awareness,” which signifies the urgent need to raise awareness and close the gaps in diagnosis. Recently, Heidi shared with me why ALSP Awareness Month matters, and how she hopes it will help bridge gaps, “My twin sister, Holly and I always started celebrating our birthday on March 1,” Heidi said. “We celebrated for 31 days. This year I will celebrate my first birthday month without Holly. Holly lost her battle with ALSP on July 20, 2021. Today, I know I am not celebrating alone thanks to all of you and the wonderful support from the ALSP community. As we build community together, we are closing gaps in knowledge and spanning the distance between the patients and families fighting this disease around the world.”

On March 1, together with Sisters’ Hope Foundation, we lit up four bridges across Massachusetts in pink and purple, the official ALSP awareness colors that represent love, compassion and hope. For Heidi, the lighting of bridges is designed to provoke conversation and spark people to learn the meaning behind the bridge beacons. We hope it also encourages people to learn more about ALSP and ultimately brings support and comfort to patients and their families.

Building a Support System

When a person is diagnosed with a rare disease, they may feel lost, discouraged or lonely. Patient advocacy groups provide a platform for patients and their families to connect with one another, educate themselves about the condition, and seek resources and assistance.

As a biotechnology company that is adequately resourced to support patient engagement, we feel honored to support the community and enable the connection and empowerment of patients and caregivers.

Through the Sisters’ Hope Foundation, we have connected with patients and caregivers to better understand how their daily lives are affected by ALSP. Throughout March, stories from patients and caregivers were shared across online channels, such as the Sisters’ Hope Foundation website and social media, to highlight patient, family and caregiver experiences.

When we began compiling these stories, I was surprised to discover many patients and caregivers thought they didn’t have a story to tell when each story was inspirational and unique. By sharing their experiences, we believe patients and caregivers can inspire, motivate and positively impact others who are walking a similar path.

For Heidi, collaboration with Vigil has allowed her organization to execute on meaningful ideas to connect and empower patients and their families. The partnership also enables each patient to be a part of Vigil’s endeavors to advance research and improve treatment options. Recently, we launched the global ALSP patient registry, to help enhance the understanding of the patient and caregiver journey, disease burden and health economic outcomes. The information we gather from the registry will ultimately support patient-centric therapeutic development for this devastating disease.

When done right, relationships between industry and patient advocacy groups can be mutually beneficial. Pharmaceutical and biotechnology companies have the expertise and resources to support the patient advocacy community and advocacy groups bring unique insights and deep knowledge of the patient journey that can help inform drug development, clinical trial design and patient engagement. This deep collaboration is crucial for serving the true needs of patients and caregivers, and ultimately, to improve lives.

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