Rare Leader: Terry Thompson, President, Kennedy Disease Association

The Basics
Name: Terry Thompson

Title: President

Organization: Kennedy’s Disease Association

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Disease focus: Kennedy’s disease, or spinal and bulbar muscular atrophy (SMBA), is an X-linked genetic progressive neuromuscular disease that causes severe muscle wasting and weakness and leads to severe disability. Both the spinal and bulbar neurons are affected causing muscle weakness and wasting (atrophy) throughout the body which is most noticeable in the extremities (legs/arms) and also in the face and throat where it causes speech and swallowing difficulties. Kennedy’s disease is estimated to affect about 1:50,000 people worldwide, primarily men, although some studies have indicated that this number underestimates the extent of the disease. Women are carriers and can experience some effects. Most documented cases have been in North America, Europe (including Scandinavia and Russia), and Japan. The gene mutation that causes the disease was discovered in the 1990s, and while more than 30 potential treatments have been successful in animal models, no treatment has yet been effective in people. There is no cure.

Headquarters: Coarsegold, California

How did you become involved in rare disease: Kennedy’s disease is in our family. My wife Kathy’s older brother was the first person in her family to be diagnosed with Kennedy’s disease. That was about 20 years ago. He subsequently died from a catastrophic accident that resulted from his inability to walk, forcing him to use a scooter. That’s when Kathy learned that she is a carrier of the KD gene. She has been on the KDA board for half a dozen years or more and organizes KDA’s annual conference. I’ve been helping her with the conference and offered to do a little more starting earlier this year, and I was surprised when the KDA board asked me to become the president.

Previous career: I served 30 years in the U.S. government, including three years in the Marine Corps and 27 years at the National Security Agency. I then worked as a cybersecurity consultant for 15 years in the U.S. and six other countries. Besides the KDA, I currently teach graduate cybersecurity courses at Johns Hopkins University.

Education: B.A. English and Master’s in Russian from Wayne State University; Ph.D. in Russian Studies from Georgetown University

The Organization
Organization’s mission: The primary focus of KDA is to raise money for SBMA research. In the past 20 years, we have provided over $1.6 million to SBMA research efforts in six countries. In 2019 and 2020, we awarded research grants to researchers in the United States, Canada, United Kingdom, Italy, and Japan. Education and awareness about Kennedy’s disease is also central to our mission, and we work to share information and build awareness of Kennedy’s disease in the medical community and general public. We also support KD patients and their families to the extent possible with advice and assistance.

Organization’s strategy: Our strategy is to identify and help as many patients with Kennedy’s disease as possible, and to fund research to develop treatments and a cure for Kennedy’s disease. To do this, we have developed a strong working relationship with SBMA researchers at the National Institutes of Health (NIH), and we are building alliances with national SBMA organizations in other countries. Our funding of research in multiple countries acknowledges that KD is a global disease affecting people of all ethnicities.

Funding strategy: KDA is a small organization with no institutional funding. Our funding comes from the patient community and families, and sometimes from the researchers. We are an all-volunteer organization and over 90 percent of the funds we receive goes directly to the international research community.

What’s changing at your organization in the next year: KDA is a U.S.-based charity that is developing a global reach. Over 370 people from 32 countries signed up for our virtual conference in October 2021. As a result, we are ramping up our communications activities, especially in social media, to reach the largest number of patients possible in every country where KD has been identified. We are also focused on becoming more of a patient-driven organization.

Another change in 2022 will be the beginning of clinical trials for therapies to treat Kennedy’s disease. Clinical trials have been done before, but the exciting news for us is that four pharmaceutical companies and one university medical center are planning clinical trials beginning next year. This is important for two reasons. First, SBMA is a slow progressing, rare disease. That means that it is difficult for pharmaceutical companies to make a business case to spend money to develop a drug for a relatively small population, the results of which may not be evident for months or years. Some potential therapies fall into the “valley of death” between the lab and the pharmacy because they are not commercially sound. The fact that so many companies are devoting resources to clinical trials is a sign that this situation may be changing. Second, and more important, the transition from the laboratory to clinical trials for several different potential therapies is a positive sign to KD patients that there is hope, if not for a near-term cure, then at least for one or more drugs that could potentially slow the progression of Kennedy’s disease or mitigate some of its worst effects.

To accelerate research and prepare for clinical trials, KDA launched a KD/SBMA patient registry late June 2021. By mid-October, the registry had 228 people from 36 U.S. states and 20 countries. Registry forms have been translated into Japanese and Italian, and the goal is to include more KD sufferers from these countries into the database that is already becoming a useful tool for KD researchers, clinicians, and pharmaceutical companies. We also believe that the registry will expand the international research community as more neurologists become aware of the global reach of the disease and ongoing research.

We also work very closely with the NIH SBMA research and clinical teams on studies they are conducting on the natural history and quality of life for KD patients. These studies, which identify and document key outcome measures, will contribute to the development of effective clinical trials and eventual FDA approval of SBMA therapies.

Management Style
Management philosophy: My personal management philosophy is based on teamwork, collaboration, and communications. I’ve been a team player since I started little league baseball at age 10 and have served as a team member and team leader throughout my career. Teaming is critical in an all-volunteer organization since no one person, no matter how committed and motivated they may be, can do everything. Fortunately, I have a wonderful board of directors who are very collaborative and very helpful. They all have more background in the disease than I do and I rely on them to educate me. Above all, clear, effective, and continuing communications are vital in any organization. The leader has to communicate to the leadership team and the organization’s members, and he must encourage communications at all levels in all directions. I spend a lot of time communicating within and about KDA and believe that is a major part of my job. I’ve also reached out and collaborated with other rare disease leaders and organizations that I think can help us achieve our goals.

Guiding principles for running an effective organization: Comprehensive and transparent communications are at the top of the list. Keeping people informed about the latest medical developments as well as educational opportunities and providing information about tools to help KD patients in their daily lives is so important. KDA is run by a volunteer board of directors, some of whom have full-time professional jobs. We meet virtually once a month and we can only be effective as a leadership team with full and open communications and with a spirit of collaboration that enables effective sharing and teamwork. Thankfully, we have that.

 As the new guy on the team, I have to make sure I leverage the strengths of each member of our board. We need to develop an agreed-upon set of organizational goals that will help us maintain our focus and momentum. We also need to integrate with the scientific and medical research communities. They hold the keys to successful treatments for KD patients, so we need to ensure they fully understand the concerns and needs of patients who suffer from this devastating and life-altering disease.

Best way to keep your organization relevant: KDA has provided significant funds to the SBMA research community for the past 20 years. That gives us a voice in the global research community and helps us remain relevant. We know our researchers have other sources of funding but they are keen to stay close to the patient community, evident in their participation in our annual conferences. We intend to continue funding SBMA research, especially for junior researchers just getting into the field. One thing we’re considering is the establishment of a fellowship at a research hospital or a clinic to provide focused funding on SBMA.

Beyond keeping KDA relevant, we want to make it even more important in the lives of KD patients. Many KD men need mobility aids but can’t afford them or don’t know where to find them. Some visit ALS or muscular dystrophy patient centers for treatments because there are very few clinics that focus on SBMA patient care. In addition, caregivers for KD patients who need help with the necessities of daily life also need support in the form of advice, training, and assistance especially when their loved ones have to travel to a clinic or research center. These are crucial focus areas for our organization in the years ahead. Funding these needs is a challenge, especially since we want to continue to devote most of the funding we receive to medical research with the hope of developing effective therapies and an eventual cure.

Why people like working with you: I like to think it’s because I listen, and I try to consider everyone’s point of view before making any decisions. I’m quick to give credit to others and encourage everyone to share in our success. One thing I’ve learned as a manager of a volunteer organization is you have to be very careful about making unilateral decisions. You need to have a consensus and make sure that your board is, if not in full agreement, mostly in agreement with the direction you want to go. That has not been a problem with our board. Our directors are all very supportive, and we’re all moving forward in a common direction. What I’ve heard from them is they like what I’m doing in terms of being proactive and communicating to the researchers and to the patient community, what we’re doing as an organization, and some of the things that I’ve brought into KDA, such as the patient registry and my focus on giving KD patients more of a voice with the scientists and clinicians.

Mentor: Mark Stone, CEO of the FSHD Society, is my number one mentor outside KDA. He has been a tremendous help advising me on things to think about, approaches to take, how to deal with the interaction between the patient advocacy group and the research community and clinicians. He has given me a lot of advice about clinical trials and how to deal with pharmaceutical companies. Within KDA, I rely an awful lot on Maria Monte, who is our organization’s treasurer and was the president for a couple of years before I took over. She and the president who preceded her, Andrew Cassar, have been extremely helpful in getting me up to speed on the organizational, administrative, funding, and other areas of managing the KDA. KDA Vice President Jameson Parker, Secretary David Yelton, and Webmaster Ron Moffett have all been very supportive. My wife, Kathy, is my inspiration for KDA and everything else in life.

On the Job
What inspires you: What inspires me on the job is the idea that we are moving towards therapies in several pharmaceutical companies that are on the verge of clinical trials for treatments that can slow the progression of SBMA. We are at an inflection point in the search for a cure for SBMA, and these trials are very important first steps. I’m optimistic and hopeful. The researchers are also optimistic. I know the pharmaceutical companies we’re talking to are optimistic, and the patient community is excited about the prospects for effective treatments. All that inspires me to work harder and to think of new ways that we can improve our outreach and awareness about Kennedy’s disease, and hopefully raise a lot more money that we can contribute to the scientific community so they can accelerate progress towards developing a cure.

What makes you hopeful: The words that I’m hearing from five pharmaceutical companies who are taking the results of research that our research community has done over the last 10 years and coming up with therapies to slow the progression of the disease. That’s a very hopeful sign that we will have something to at least treat some of these patients in a way that improves their quality of life. That gives me a lot of hope. I’m optimistic that eventually we’ll find a cure for the disease, possibly through genetic engineering, such as the mRNA technology that was used to develop the COVID vaccines. For example, techniques have been developed that can be used to deliver corrective genetic code to the mutant gene that causes Kennedy’s disease. This, in turn, can help slow the progression and perhaps even lead to a cure. All this is very exciting to me and inspires me to work harder.

Best organization decision: The best organization decision since I’ve been president in the last nine or 10 months is the establishment of the KD/SBMA patient registry at Sanford CoRDS. That has really taken off. As I mentioned earlier, more than 228 people have signed up from 20 different countries. This is going to be a great boon for the research community. We have connected with patient advocacy groups like KDA in the UK, Italy, Australia, Japan, and Russia. We recently learned of a KD patient group in France. All these groups have been working with their individual country researchers on various tests and evaluations to track their progress over time, to help them with exercises and nutrition, and to help treat their symptoms. But for the first time ever, we have now, in one place, a database that researchers can go to for data that they would not be able to get from their own cohort of patients in their country. They can also recruit patients for studies and clinical trials. This is a major step forward for the research community and KDA. We did that through our own resources in collaboration with our research community at the National Institutes of Health. KDA was the driving force behind that, and I think it’s going to be a boon for our research community.

Hardest lesson learned: I knew just a little about the causes and consequences of Kennedy’s disease when I became KDA president. Learning the science has been a steep learning curve for me, and I’m still working on that. The science is complicated. Genetic science is not something, as you figured out from my background, that I ever studied before. But thanks to my wife and others on the KDA board and many KD patients who have advised and educated me, I’m making progress. The hardest thing for me is learning to be patient. I’d like to do things faster and on a bigger scale. I’m finding that working in an all-volunteer organization that has no money for any excess activities beyond what we’ve talked about in terms of funding research, that’s hard to do. There are things we can do with technology to help even more, but we just don’t have the funds to do that. Taking the KDA to the next level with insufficient resources has been difficult. That’s been the hardest lesson for me.

Toughest organization decision: The hardest decisions are those requiring funding for things other than research. With more resources, we could stand up patient support centers around the country, like they have for ALS. We know we have patients in 40 states. The problem is they’re spread out all over the country, but every time one of these men has to travel to a clinic for a study of some sort, or eventually for a clinical trial, they need transportation, and they need accommodations. Mobility issues are a huge problem for KD men because they lose strength in their legs and arms and are confined to wheelchairs and scooters at some point. A lot of these guys can’t get up. One patient, for example, fell and his wife was at work. She had to call the fire department to get him back up into a chair because he couldn’t move himself and she couldn’t lift him. Some men who suffer atrophy in the bulbar musculature have trouble speaking and swallowing and have to be fed through a tube. It’s just heartbreaking to see the effects this disease has on people. Caregivers for KD patients are faced with having to provide lifetime support for their loved ones. That includes everything from helping them with bodily functions, to helping them get in and out of bed, to helping them travel, to helping find accommodations where there’s a walk-in shower. Many of these men can’t risk stepping over even a two-inch barrier get into a hotel shower. I would like to be able to provide mobility devices and other tools for every KD patient in the United States because many of our patients don’t have the resources to buy those on their own and their insurance doesn’t always cover it. Those are hard decisions. Those are tough decisions that we can’t make because we don’t have resources for that purpose.

Biggest missed opportunity: We applied for a Chan Zuckerberg Initiative grant but didn’t get it. That would have enabled us to reach more KD patients with more services more quickly. We’ll use that experience to apply for and hopefully win other grants.

Like best about the job: I like being engaged with the patient community, with the researchers, and other national patient advocacy groups. One of my strengths as a manager is to bring people together. We’re doing that and that gives me a lot of satisfaction. Helping the organization get to a level of greater efficiency and hopefully better funding through grants and fundraising opportunities that we’re going to be trying in the next year or so, should give me even greater satisfaction. Being able to work collaboratively inside and outside of the organization to move forward and help develop better quality of life for patients is what I’m all about in this job. We’re making progress. My goal is to do any little thing to help people achieve a better quality of life, both for the caregivers and the patients. That is what excites me the most.

Like least about the job: Not having the resources to do the things we’ve talked about. If we had several million dollars, there’s so much we could do in terms of advancing research—advance the technology to connect researchers and patients worldwide beyond the registry. I feel frustrated sometimes at not being able to keep going on the trajectory we started on to improve our organization’s outreach and funding capabilities because of those lack of resources.

Pet peeve: My one pet peeve is we are a virtual organization. I understand how and why we operate that way, but it does create certain limitations. There are delays and inefficiencies that are caused by our directors living in many different states. Sometimes I would like to have an actual organization with somebody responsible for answering the phone, opening the mail, collecting and depositing the checks—that sort of thing—all focused in one place, but that costs money that we don’t have.

First choice for a new career: I’m 73 years old. This is my new career. I don’t need another one. I’ve flunked retirement three times already, so I’m not looking for another change.

Personal Taste
Most influential book: The Great Influenza: The Story of the Deadliest Pandemic in History by John Berry. I read that last year and it was really enlightening in terms of the relationship between the medical community and the community at large when faced with a rapidly advancing pandemic. Also, the way that the medical community responded to that, to the great influenza, and parallels to the way the research and medical community responded to COVID. I found that book fascinating.

Favorite movie: Star Wars

Favorite music: Jazz and classical

Favorite food: Anything my wife makes. We eat well at our house thanks to her lifelong focus on nutrition and healthy food. She’s such a great cook; actually, I call her “Chef” as a sign of my respect for her knowledge and skill in the kitchen. (And she also had a professional career and raised two sons.)

Guilty pleasure: Golf—I feel a little bit guilty taking time out to play golf, but that’s what I do.

Favorite way to spend free time: Traveling and hiking with my wife. We go hiking in the mountains in Maryland and also at a local state park. We just got back from a trip over to the New Jersey shore. We have a lot of fun doing things together.