Rare Leader: Allison Moore, CEO and Founder, Hereditary Neuropathy Foundation

The Basics
Name: Allison Moore

Title: CEO and Founder

Organization:  Hereditary Neuropathy Foundation

Disease focus:  Charcot-Marie-Tooth disease (CMT), named after the three doctors who first identified it, is one of the most common inherited nerve disorders. CMT is also commonly called hereditary sensory and motor neuropathy. This means that the disease runs in families and causes problems with the sensory and motor nerves, the nerves that run from the arms and legs to the spinal cord and brain. When the parts of the nerves—the axons and the myelin—become damaged, messages that run along the nerves move more slowly or have a weak signal. Over time, this causes muscles in the feet, legs, and hands to lose strength. Often, the muscle loss happens unevenly, which causes deformity as muscles waste away (atrophy) at different rates. CMT also can cause severe foot drop where patients have trouble walking and rely on mobility devices to stay mobile. CMT affects an estimated 1 in 2,500 people in the United States and 2.6 million people worldwide, although experts believe the number could be much higher.

Headquarters: New York City

How did you become involved in rare disease: When I was in my late twenties, I developed a rare cancer. I was given chemotherapy and the chemotherapy was contraindicated. I didn’t know I had a mutation for CMT.  I went from being an able-bodied person to a disabled person. I thought I would regain function, but it was clear after a couple of years that my function was permanently impaired. It took me two-and-a-half years to get a diagnosis. Once I was diagnosed, I was extremely upset because I wanted to find a way to reverse my situation. That’s how I became involved. I got the diagnosis in mid ’97 and I started the organization in January ’01.

Previous career: I worked in employee benefit sales for an insurance brokerage.

Education: B.A. with a minor in psychology and business from the State University of New York, Oneonta

The Organization
Organization’s mandate: Our mandate is to support the patient community, improve patients’ quality of life, and fund research that will hopefully lead to treatment and cures. We are patient-centric. About five years ago we started focusing a lot on patient-focused research with a goal to enhance research studies and support industry. Our studies have improved clinical trial design (better outcome measures and endpoints), accelerated patient recruitment, and now are being used to improve patient diagnosis and care.

Organization’s strategy:  Our strategy is to bring the voice of the patient to the stakeholders, so they can be more effective in their research and clinical trials. It’s starting to work. Right now, we have two drugs in clinical trials, two more that are approaching clinical trials, and many others that will hopefully get to that point within the next three years. It’s an exciting time for the CMT community.

Funding strategy: We work with private foundations and the public on grassroots fundraisers. We have participated in bike-a-thons, marathons, golf events, and cocktail parties to list a few. We have received several government grants and rely on funding from industry.

What’s changing at your organization in the next year: We’re going to be expanding our staff in order to accomplish our goals in the near future with a potential drug moving toward approval. Positive data was just announced for a drug called PXT3003, which will be the first drug to treat CMT. The sponsor is Pharnext, a French biopharmaceutical company dedicated to developing more treatments for CMT and other rare diseases.

Management Style
Management philosophy: I am super-collaborative. I’m absolutely head over heels in love with my team. They are the most incredible people. We all either suffer from CMT or are caregivers of family members with CMT. We’re all empathetic. If one is down and out, another steps in to pull the weight of that person’s jobs duties. It works well. It’s taken me almost ten years to find the perfect team.

Guiding principles for running an effective organization: You have to have a team that you are confident in and that you don’t micromanage. You have to allow your team to run with good ideas. You are dealing with a complicated disease and you don’t have all the answers. It’s important that you allow everyone on the team to have a voice. You have to be open to pushing the envelope and going outside of your comfort zone sometimes.

Best way to keep your organization relevant: It’s important to have a defined program and assess the program on a regular basis. I think we do that very well. It’s taken a number of years to get to that point. We make sure we are following the mission of the organization and serving stakeholders, who are not just patients, but physicians, researchers, industry, and even the FDA.

Why people like working with you: We’re a lot of fun and I really mean that. We’re dealing with a serious disease, but we all make the best of what we have to deal with and what is in front of us when living with a chronic and debilitating disease. These are super passionate people and we get stuff done. We don’t just talk about it, we do it.

Mentor: I have so many, my husband being number one on the list. He’s put up with me and my disease, which is challenging. Even more challenging is having me completely committed to this organization and to the many lives of patients. Family life does sometimes suffer because I’m traveling a lot and curing CMT is a priority for me.

On the Job
What inspires you: What inspires me the most is working with my close-knit team and working with industry. The empathetic researchers and physicians who have the same goal as I and the HNF do. It’s about improving the quality of life of patients and for better patient care with the ultimate goal of treatments for a future that is much better for our patient community.

What makes you hopeful: I’m hopeful that there are going to be multiple treatments in the next five years for many types of CMT. The days and years that I have wanted to give up, and almost did, whatever drove me to keep going, I’m glad I did. Now there’s a light at the end of the tunnel. Finally, we’re going to see what we set out to do. One day in my lifetime there will be treatments, and maybe even a cure.

Best organization decision: The best decision we ever made was reorganizing five years ago to add patient-focused research to our Therapeutic Research in Accelerated Discovery (TRIAD) program. Bringing the voice of the patient into the equation has really paid off. It’s been instrumental in much of our successes in the last few years.

Hardest lesson learned: You have to know how to fail fast. Sometimes we get so fixated because you want something to happen so much. You’ve got to know when to cut it and move on to the next project. Now, I have no problem cutting projects, but there was a time that I held on. I didn’t want to see something I thought potentially could work fail. That was the hardest lesson learned.

Toughest organization decision: The toughest organization decision was to discontinue our paid membership. We relied on those funds, but because the era of the Internet really gives people access to content and resources, we thought this was the right move. Also, we wanted patients to have free access with the idea that if they appreciated us, they would continue to donate.

Biggest missed opportunity: We’re a nimble organization. We’ve accomplished a lot without a lot of funding. The lack of funding has slowed our progress. My hopes for year-end are that we will raise more funds and accelerate more treatments to support our patients.

Like best about the job: I love working with industry and researchers who get us. You need people who get the patient community. It makes it bearable living with a progressive disease.

Like least about the job: I don’t like the admin grind, but I don’t do that anymore.

Pet peeve: When we work so hard on behalf of the community and sometimes the community doesn’t embrace what we’re doing by giving back. It’s very simple, by supporting our efforts we can better support the patients and stakeholder community. My pet peeve is that more people don’t pitch in by donating, joining our patient registry, or attending our annual summit. Even the smallest actions  make a difference, such as sharing a post, liking our Facebook page, and joining our online CMT Inspire Community.

First choice for a new career: I would love to work for industry and take everything I’ve learned and put it to work on the pharmaceutical side.

Personal Taste
Most influential book: I read a lot of self-help books. My favorite non-fiction book is A Prayer for Owen Meany by John Irving

Favorite movie: Some oldies – An Affair to Remember and the 1976 version of A Star is Born

Favorite music: I still love The Beatles

Favorite food: Avocados

Guilty pleasure: A nice cabernet with a delicious bowl of pasta

Favorite way to spend free time: With my family, but when alone, I love binge watching on Netflix

November 1, 2018