RARE Daily

Rare Leader: Jeff Levenson, Co-President, Adult Polyglucosan Body Disease Research Foundation

May 21, 2020

The Basics
Name: Jeff Levenson

Title: Co-president

Organization: Adult Polyglucosan Body Disease Research Foundation

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Disease Focus: APBD is a glycogen storage disease (type IV) caused by mutations on one gene. This neurodegenerative disease, often misdiagnosed as MS or peripheral neuropathy, results from the low activity of glycogen branching enzyme (GBE), which is used in the production of glycogen. When there is low activity of GBE, newly formed glycogen is manufactured incorrectly into long, insoluble stands, called polyglucosan bodies (PB). These PB cannot efficiently be used for fuel, and instead, build up inside nerve cells leading to poor capacity for the central nervous system (brain and spinal cord) to communicate with the peripheral nervous system. Eventually, the muscles controlled by these nerves cease to function. APBD appears in patients most often when they are in their 50’s and 60’s, but it may manifest in patients as young as in their 30s. It has been diagnosed most frequently among individuals of Ashkenazi Jewish descent, although it is also found among other populations. APBD’s initial indicators include numbness in the toes, feet, and hands; bladder and bowel dysfunction; and unusual fatigue. Most individuals will at some point require assistance walking, starting with a cane or walker, and later a wheelchair. APBD-afflicted individuals may develop mild cognitive impairment. In its natural progression, patients die when their autonomic nervous system fails.

Headquarters: New York City

How did you become involved in rare disease: My journey with the Adult Polyglucosan Body Disease Research Foundation started 15 years ago, when I met Edwin Kolodny (then chief of the Department of Neurology at New York University Medical Center) and the Weiss brothers—Gregory, Emil, and Michael—who were diagnosed with APBD. But, in fact, my family has been living with the ravages of APBD for more than 40 years. For the first 20 years, the disease did not have a name. It was mistakenly thought to be MS. We watched my father and his brother slowly deteriorate, while undergoing treatments that did not work. There was nothing that we could do other than make their lives as meaningful and comfortable as possible.

Previous career: Until the COVID-19 pandemic, I was practicing general dentistry full-time. Now, I do not know when I will resume my practice.

Education: NYU College of Dentistry AB (biochemistry concentration) and DDS

The Organization
Organization’s mission: The foundation’s mission is to improve the diagnosis and treatment of APBD, support affected individuals and their families, increase awareness of APBD among health professionals and the public, and facilitate the translation of research into treatments and cures.

Organization’s strategy: Our strategy is to partner with APBD and allied diseases stakeholder groups. We foster collaborative relationships across the industry, research, and clinical communities to accelerate APBD breakthroughs for new diagnostic tools, treatments, and cures. We develop awareness initiatives both for the medical community and the public. We also support patients with educational resources, opportunities to connect, and ways to get involved in supporting our mission.

Funding strategy: As an ultra-rare disease foundation, we are a small community working diligently on expanding our reach. We are working on a major gifts program and beginning development of a Corporate Partners program to be launched in 2021. We also participate in peer-to-peer awareness and fundraising events such as the New York City Marathon. Our priority is to leverage the funds we raise via matching opportunities, seed grants, and alliances with organizations whose interests link with ours.

What is changing at your organization in the next year: We are honored to have been selected as one of 30 members of the Rare As One Project sponsored by the Chan Zuckerberg Initiative. This project is introducing us to a dedicated and talented group of rare disease stakeholders who have already, in just a few months, impacted our foundation in profound ways. As a result of the Foundation’s urging, APBD was recently included in several pre-pregnancy screening programs. As a consequence, we are continuing to change our awareness campaigns to include connecting with and educating asymptomatic APBD patients. In addition, we also are planning an extensive outreach program for an at-risk population in coordination with the publication of an APBD prevalence paper. Finally, we are planning a virtual biomarker workshop for this summer. The topic and the virtual format will be a first for our foundation.  

Management Style
Management philosophy: For the first ten years that I volunteered with the APBDRF, I did anything that was needed. Then, in order to help clarify my role, I became the foundation’s chief strategy officer. Now, as I move into my new position as co-president, I will concentrate on leading the foundation’s CZI grant initiative and on strategizing with and supporting coordination across committees, task groups, and initiatives in order to ensure that all of our assets are maximized. I like to build constructive and collaborative working relationships. I want new ideas to emerge and be considered no matter how outside the box they may initially appear. I also look for initiatives where there are clear, actionable items with realistic deliverable goals. Wherever possible, we strive for consensus on our programs and on our approaches to raising awareness, fundraising, and finding treatments and cures.

Guiding principles for running an effective organization: Communicate, communicate, communicate. Because we are a small foundation, where a few people do so much, we deliberately keep roles fluid and flexible. My goal is to continue nurturing a respectful and collaborative environment where new ideas can flourish. I like to work with volunteers and suggest ways to contribute that are meaningful to them and helpful to the Foundation.  Our goal is to surround ourselves with positive, engaged, honest, hard-working, and goal-oriented people. 

Best way to keep your organization relevant: Ask for and listen to the voice of the patient. Recruit talented, dedicated, open-minded, and empathetic volunteers and staff. Constantly evaluate new technologies to build capacity. Continually assess the evolving needs of the community which comprises all our stakeholders—patients, caregivers, health professionals—and communicate with them. We are working on more creative ways to engage our community, beyond face-to-face interactions both during the COVID-19 pandemic and after (i.e., various virtual formats).

Why do people like working with you: To be honest, I am sure that some do not. I can be—in a word—intense.  I asked some of my colleagues and they told me they like my boundless energy, enthusiasm, and optimism. Furthermore, they like how I seek to turn any negative into a positive. I would like to think that I have built up their trust due to my commitment over many years.

Mentor: Gregory Weiss, founder and co-president of APBDRF is my mentor. I met Gregory fifteen years ago, shortly after he and his brothers launched the APBD Research Foundation. All of us met for the first time in Edwin Kolodny’s NYU Medical Center office. Gregory, out of the conviction that a cure must be found, inspired us all—patients, family members, friends, and colleagues. Gregory kept us optimistic and our spirits high despite his severe health challenges. Gregory became a close friend and mentor. He and I spent countless hours on the phone with dedicated researchers delving into how the Foundation could facilitate research into unexplored areas of APBD science. Gregory was an amazing listener who then would ask very probing questions.  Gregory also always took an interest in the lives of others, asking them about their family members and other areas of their lives outside of our work. He was effusive in his gratitude to all those who helped the Foundation and made everyone feel important and integral to the cause.

On the Job
What inspires you: In 15 years, we have had 13 scientific advisory board convenings. It has been my honor to help organize these convenings and listen to known and not so well-known scientists collegially deliberate and constructively work through problems. Each of these researchers and clinicians is my hero and deserves to be named individually. I stand on their shoulders. It is also heartening to see our patients find support and take care of each other, even though so many of them have never met in person. Our work at the Foundation makes this community possible.  There are many who stand out and inspire me. One of those patients, Robert Zuckerman, was the subject of a documentary, “Life Through a Lens,” that one of my children, Ronete, and I co-directed and produced. It has screened at more than 20 film festivals.

What makes you hopeful: I am very hopeful about the growing activism within our community.  Every voice, patient, carrier, caregiver, health professional, industry representative, can change the future for our community. Now that we have brought aboard talented individuals who are experienced in pharmaceutical development, we are better able to collaborate with pharmaceutical companies. Moreover, the foundation is leveraging quality of life surveys to get our patients’ voices heard and confirm our clinical trial readiness. We are also working toward the publication of a journal article describing the prevalence of APBD. The article estimates that there are 2000-3000 patients in the United States alone who have been misdiagnosed with multiple sclerosis. You can imagine the impact of finding the correct diagnosis on the individual lives of each of these patients, as well as on the work of the foundation.

Best organization decision: Early last summer, we learned about the Rare as One Project. As a small, volunteer-run foundation, the responsibility for writing the LOI and the grant fell on a small group of dedicated individuals. It took over our lives for many weeks, and, of course we had no idea if we would be awarded the grant. Fast forward nine months, as a CZI grant recipient we are being introduced to tools and individuals that will accelerate our research and community-building initiatives. We are working on projects— including addressing misdiagnoses, adopting new technologies to connect our community members with each other, adopting best practices, among others—that were just dreams. Going after this grant, against the odds, was the best decision we made as a foundation. 

Hardest lesson learned: Being involved in therapeutic development is extremely exciting, but it also comes with a risk. This is a lesson we learned the hard way. What happened was that the drug we were developing, guaiacol, had no appeal to investors. By way of brief background, guaiacol is a 19th century cough medicine. Through testing, it was determined that guaiacol would successfully down-regulate glycogen synthase, thereby, reducing glycogen and polyglucosan bodies. (Importantly, that down-regulation is also valuable for other glycogen storage disorders.) As a re-purposed drug, guaiacol was ready for clinical trials. In fact, Israel was poised to initiate its first patient-sponsored clinical trial for guaiacol. Unfortunately, however, guaiacol is no longer a patentable substance and we have been unsuccessful in getting a U.S. pharmaceutical company interested in investing in the clinical trial.

Toughest Organization decision: As incoming co-president in late April 2020, I have not had a lot of time to be faced with tough decisions. Ask me this question again next year.

Biggest missed opportunity: There is an opportunity that we missed recently, but not as a result of any inaction on our part of which we are aware. Nevertheless, we missed the chance two years ago for APBD to be included among the conditions that should be considered prior to making a multiple sclerosis diagnosis. The inclusion of APBD would have prevented APBD-afflicted individuals who were MS-misdiagnosed from taking unnecessary MS medications, participating in and tainting MS clinical trials, and allowed them to find the APBD community and the care and support they need. In addition, especially for ultra-rare diseases, patient numbers matter when validating potential biomarkers, presenting solid natural history studies, and harnessing well-populated patient registries to support clinical trials.

Like best about the job: I love meeting and working with like-minded people. In the rare disease community there is a realization that we cannot do this work alone. There is a camaraderie and an unprecedented sharing that takes place. It comes out of genuine empathy and understanding of what each of us is going through. I am especially appreciative of enlightened organizations like the Chan Zuckerberg Initiative and the Orphan Disease Center, which are empowering and nurturing the rare disease community. We are all in this together.

Like least about the job: In the absence of a treatment or cure, APBD ultimately takes its toll, bringing devastation into the lives of the people with whom I work, love, and admire. Watching them struggle is heartbreaking. 

Pet peeve: People who think they know the absolute truth and do not engage in a collaborative and constructive way. It is either their way or the highway.

First choice for a new career: Rescue Dog trainer. Although I already am a certified dog trainer, I would train and rehabilitate rescue dogs full-time. Or maybe I would be a vegan chef.

Personal Taste
Most influential book: There are a lot of them. If I must pick one—Man’s Search for Meaning by Viktor Frankl.

Favorite movie: More recently, the Mr. Rogers film starring Tom Hanks, A Beautiful Day in the Neighborhood. Mr. Rogers is my type of hero: soft-spoken, gentle, and genuine, with a keen understanding of human nature.

Favorite food: My homemade cashew cheese, which I make with a little turmeric, nutritional yeast acidophilus, topped with cayenne and Aleppo pepper. 

Guilty pleasure: Sour beer 

Favorite way to spend free time: With my family and dogs, cooking healthy vegan food, and gardening.

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