Rare Leader: Akiva Zablocki, President, The Hyper IgM Foundation
September 5, 2019
Name: Akiva Zablocki
Organization: The Hyper IgM Foundation
Disease focus: Hyper IgM Syndrome is a very rare and life-threatening genetic mutation that severely compromises the immune system. The most common form of Hyper IgM is X-Linked Hyper IgM, which primarily affects boys, except in rare cases. Other forms of Hyper IgM are inherited as autosomal recessive traits and can affect both boys and girls. X-Linked Hyper IgM compromises the body’s ability to produce responses to pathogens, such as bacteria and viruses. In a normal functioning immune system, a person will produce immunoglobulins or antibodies in response to an encounter with a pathogen. These antibodies act like specialized forces, trained and capable to confront a more complicated pathogen. A person with Hyper IgM Syndrome, however, can only create very basic, primitive and largely ineffective IgM antibodies, and cannot “class switch” into the more effective and specialized antibodies to fight off the pathogen and create lasting immunity to the virus or bacteria. As a result of their inability to produce antibodies, patients with Hyper IgM are at significant risk for opportunistic and repeated infections. In addition, the defect in the T-cells results in a decreased ability to identify and fight cancer cells, as well as an inability to produce a response to pathogens. Combined with repeated infections over time, this defect in the T-cells poses a significantly increased risk for cancer.
Headquarters: New York City
How did you become involved in rare disease: My background in rare disease started 14 years ago when I was diagnosed with an “inoperable” brain tumor in my brainstem, a rare location for a pediatric tumor, at age 25. I had to advocate for myself, as most doctors did not think I would live more than 2 years. After getting opinions from over 30 specialists, I chose to fly to Phoenix, Arizona to have brainstem surgery that most of my New York doctors thought would kill me. I survived the surgery and, although the recovery was difficult and I still have several deficits, the entire tumor was resected. The following year, I got involved with the Children’s Brain Tumor Foundation, eventually joining their board of directors.
About eight years later, in 2013, my first son was diagnosed with a very rare, one-in-a-million immune deficiency called X-Linked Hyper IgM Syndrome, and my world was turned upside again. My wife and I learned all we could about this rare disease, and, with little information out there, proceeded to get opinions from more than 30 doctors on the best treatment. Our son had to undergo two stem cell transplants and multiple hospitalizations as we chased a normal life for him. Along the way, we connected to others with this rare disease, started an online support group for the families, and eventually founded the Hyper IgM Foundation in hopes to improve the treatment, quality of life and the long-term outlook for children and adults living with Hyper IgM Syndrome through research, support, education, and advocacy. Our son is now considered cured, and we’re determined to make that a reality for all patients with this disease.
Previous career: My career has always been dedicated to healthcare. After receiving my master’s in public health from the Mailman School of Public Health at Columbia, I worked in healthcare consulting for a multinational consulting firm. In that role, I advised Fortune 500 companies on their health and wellness plan offerings and helped devise new initiatives to improve the welfare workforce for my clients.
Education: B.A. in Economics and Philosophy from Columbia University;
M.P.H. in Healthcare Management and Policy from the Mailman School of Public Health at Columbia University
Organization’s mandate: Our mission is to improve the treatment, quality of life and the long-term outlook for children and adults living with Hyper IgM Syndrome through research, support, education, and advocacy.
Organization’s strategy: Our primary objectives are to find a cure for Hyper IgM Syndrome, to develop and enhance the current understanding of Hyper IgM, and to provide education and support to our families. We focus much of our resources on connecting with researchers and working with doctors to better understand our disease and drive research into finding a cure. Importantly, we have cultivated an international network of experts that can connect with our patients (even in the remotest of areas) and a rich online support group for our families.
Funding strategy: We are still relatively small but growing every year. Since we are entirely volunteer-based, we look to our community and families to run fundraising campaigns for most of our funding. We utilize our own networks and social media to reach a wide spectrum of donors as well as work to cultivate existing donors.
What’s changing at your organization in the next year: A primary goal over the next 12 months is to expand our board of directors. We hope to grow the board by 40 percent in 2020, something we believe will lead to increased sources of funding that can be used to make research grants and otherwise support the programs and objectives of our organization.
Management philosophy: I like to give our volunteers a lot of independence and control over their projects and make sure they feel appreciated. If it is social media awareness, patients and family support or a fundraiser, I like to allow volunteers with good ideas to take ownership and run with it, while supporting them with resources and knowledge as needed.
Guiding principles for running an effective organization: I like to make sure everyone involved feels as connected to the organization as its founders do. Give ownership and leadership to the volunteers that want it and continuously work on getting more involved.
Best way to keep your organization relevant: From a social medial and technology perspective I try to stay ahead of the game, whether it is fundraising or accepting new forms of donations. Also, make sure we are continuing to serve our community and our patients while seeking out collaborations with larger organizations.
Why people like working with you: I think I am responsive and open for partnerships on many levels, and I am always happy to give advice and share what we have learned along the way, helping new rare disease groups get started.
Mentor: My mentor is Eric Talbert, Western Regional Director at MedShare, a humanitarian aid organization. He was the development associate at Children’s Brain Tumor Foundation when I joined its Board and ran their young professional group. He taught me a lot about fundraising and volunteer managements and the skill set I developed working with him has guided me ever since.
On the Job
What inspires you: My son, Idan, who is 7 years old and has been through two stem cell transplants. He spent most of his early life in isolation and hospitals. To see him now, adapted to the real world, enjoying school and camp, and living a worry free and disease-free life, inspires me every day to make sure we don’t rest until all children and adults with Hyper IgM can be worry and disease free.
What makes you hopeful: Advances in gene editing is where we are hoping a real cure for Hyper IgM comes from, and the fact that there are amazing researches working on our rare disease and finding a cure makes me hopeful that sometime in the next five to 10 years we will have a real cure for our disease.
Best organization decision: Switching to and paying for a donor management system. I had spent the first three years using an excel file and individually writing thank you letters, which was taking up much of my time. The donor system is not perfect, but it at least allows us to target donors with relevant communications and track donors over time.
Hardest lesson learned: It’s been donor attrition, especially as time goes by from my own son’s diagnosis. When starting the Foundation, we relied mostly on our own network of donors that had followed our son’s story. Now that our son is healthy and has moved on to another stage in life, so have many of the donors that followed his story. It is the same for some of our other Board members as well. Figuring out how to keep telling our Foundation’s story, and communicating the need our community has for a real cure, is a continuing challenge.
Toughest organization decision: The toughest decision was deciding not to financially support individual families. Our donors expect the funds we raised to go towards our mission to fund research and education and advocacy, and this is what we do. Regrettably, that means that we do not fund or help families who need the money to save their child with a transplant or treatments not available in their country. We get contacted all the time by families who ask for financial help, and we refer them to a few organizations that can assist with their financial needs, but we can only offer expertise, knowledge and other forms of non-financial resources and support.
Biggest missed opportunity: I think if we had started the Foundation a couple of years earlier when our son was first diagnosed, we may have been able to reach a critical mass of followers with our son’s story to raise much more funds to accelerate a gene editing treatment. Of course, we were busy trying to save his life and it took us time to be able to be in a place that we felt we could focus on running the Foundation and its mission.
Like best about the job: Getting to know families with Hyper IgM around the world and seeing the value the Foundation, with its resources and support, brings to their lives and the lives of their children that are affected.
Like least about the job: I get to know each and every family we serve personally, and sadly, with Hyper IgM Syndrome, 50 percent of our patients do not make it past age 24, and 80 percent do not make it into their 30’s. When we lose a child or adult in our community it hits very hard and is very personal because of the tight knit community we have built. Before we existed, these families dealt with their loss alone, and they were just statistics in research papers for the other families who would have never known them. With the Foundation, we have made the Hyper IgM world smaller, but this is a double-edged sword, as each loss hits very hard on our whole community.
Pet peeve: Medical teams and doctors who don’t really understand our rare disease but feel like their general expertise is enough to treat or advise our families. It is not.
First choice for a new career: I am doing what I am most passionate about right now. If I were ever to change careers, it would be to empower all patients, not just ones with Hyper IgM, to make the best decisions regarding their treatments.
Most influential book: Making Miracles Happen by Gregory White Smith
Favorite movie: The Princess Bride
Favorite music: Currently it is Baby Shark by PinkFong, which my kids listen to 30 times a day on Alexa.
Favorite food: Italian, preferably in Rome.
Guilty pleasure: Currently I am off all desserts beside these amazing raw chocolate truffles I make with my 7-year-old, which is mainly just dates, raw cacao and almonds.
Favorite way to spend free time: A lazy Saturday morning with my wife and kids.
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