Rare Leader: Ron Garber, Co-Founder, Yaya Foundation for 4H Leukodystrophy

The Basics
Name: Ron Garber

Title: Co-founder and president

Organization: Yaya Foundation for 4H Leukodystrophy

Social Media Links:

Disease focus: 4H leukodystrophy is a rare and devastating disease of the central nervous system. The name 4H stands for hypomyelination, hypogonadotropic hypogonadism and hypodontia. The disease is also known as POLR3-Related Leukodystrophy or 4H Syndrome. Hypomyelination means that there is lack of myelin in the central nervous system, which results in problems in transmission of messages from the brain throughout the body.  Hypogonadotropic hypogonadism means delayed or abnormal puberty because the central nervous system is not able to initiate it properly. Hypodontia means that not all teeth are present or there is abnormal development of teeth. Symptoms tend to get progressively worse over time, and people with 4H Leukodystrophy suffer from motor problems, resulting in ataxia and loss of the ability to walk. They often develop difficulties swallowing, which leads to difficulties with eating and breathing. There is not yet a known cure.

Headquarters: Minneapolis

How did you become involved in rare disease: My daughter Yaya was diagnosed with 4H leukodystrophy when she was 8 months old and died a little more than three years ago when she was 13 months old. She was our first child and an amazing and wonderful little girl. Losing her was devastating to my wife and me. Before Yaya, I didn’t even know the rare disease world existed. Learning about the science of her disease and others, as well as others’ efforts to battle their own rare diseases, helped me slowly come to terms with our loss. Joining others to advance efforts in our disease area became a way for me to keep Yaya alive and help other kids like her.

Career: Currently serves as a healthcare mergers and acquisitions attorney at Medtronic, a medical technology company

Education: Bachelor’s degree in business administration and management from the University of Michigan Ross School of Business, J.D. from University of Michigan Law School; Forward/Center/Benchwarmer on the University of Michigan Men’s Basketball Team

The Organization
Organization’s mission: Our mission is to accelerate and support research that will lead to the development of a therapy and cure for patients, to provide educational and emotional support for families that are affected by 4H leukodystrophy, and to raise awareness of the disease.

Organization’s strategy: Our research strategy is to build a collaborative community/team-based approach to conducting research—to build a collaborative research network in our disease area. We have amazing researchers that are already working on our disease at a couple of different labs. Our strategy is to act as a hub to connect them and make sure that they are working together as much as they are able, not duplicating efforts, and taking a community approach to identifying and conducting the highest impact studies. We recognize that as amazing as they are, they’re stretched. They have clinical practices as well, which we’re extremely grateful for, and that stretches them even further. It takes a lot of different skills and perspectives and funding to get a therapy developed. We want to burnish their work by attracting new skill sets, new perspectives, and new funding to our community to support and accelerate that. We want to work with them and the community that we’re trying to build to identify the highest leverage research questions to answer and build the disease area infrastructure so we invest the scarce resources we do have and leverage it across our disease community.

Funding strategy: Our funding strategy is centered around families. The Yaya Foundation is the product of a collaboration by the few families in the United States who were doing organized fundraising to support 4H leukodystrophy research. We each had our own independent fundraising efforts, but we decided that if we pooled our efforts, the whole would be greater than the sum of its parts. We’re continuing that approach and trying to attract more families, which we’ve done in the short time we’ve been around, with some success. What we try to do is have events centered around each of these families. That’s because what we’ve seen is that you can’t see one of our families and what we’re going through and what our children are going through and not want to do something to help. It really moves people to see a family that is going through so much dealing with the health of their child to want to go to the effort to put something together to try to help other kids. This started with an amazing family in Ohio, really the pioneers of supporting research in our disease community, that put on an annual golf tournament. Then a banquet-style event hosted by another wonderful family in Oregon. Then my wife and I came along. In the past year, we’ve more than doubled the number of families that are doing fundraising on our behalf. We’ve had a grandpa ride his motorcycle across the United States to raise funds. And a number of families are doing social media fundraising. Each of us get to put our own personal stamp on our efforts, and our communities really rally around us.

What’s changing at your organization in the next year: A lot is changing for us in the next year. We’ve been around for only about a year and a half. Right now, we’re trying to scale up our efforts. We’re launching our collaborative research effort this year. We’ve recently received a significant grant from the Chan Zuckerberg Initiative’s Rare As One Project that is changing the trajectory of our efforts and that we are very grateful for – it will allow us to execute on our research strategy on a much bigger scale. In the next year we’ll be hiring our first employees, including a research director, and we’ll be hosting our disease area’s first ever scientific meeting. From there, we will be working with our research community on building a comprehensive research strategy for our disease area and prioritizing studies to fund and execute.

Management Style
Management philosophy: What we’re trying to do is recognize our strengths and weaknesses. Half of our board are patients and family and half of our board are friends of families, so there is a great deal of passion on our board. We’ve been successful at fundraising. We’ve been successful at community building. We don’t know drug development. We don’t know scientific research yet. We’ve gotten smarter at all of those things, but we’re not experts. What we try to do is partner with people. We try to understand what we do well and try to understand what we don’t do well, and partner with people who can help us get better at it. We’re collaborative. We’ve collaborated closely with the researchers in our community and with researchers and experts outside of our disease area. We’ve collaborated closely with other patient leaders to become more thoughtful and sophisticated about running our organization and learning how to impactfully support research. So maybe collaboration is our management philosophy.

Guiding principles for running an effective organization: It’s keeping our arrows pointed towards the kids in our community, the patients in our community, at all times. We just find that if we are keeping our children top of mind in what we are doing, we will be effective more often than not. People will want to help us to the collaboration point because they see that our interests are where they should be. And I think we arrive at the right outcomes if we’re putting our children first. That’s a big number one. We also recognize that we have scarce resources and so we look for leverage. We look hard for ways that we can expend a small amount of resources or effort that will then have a big impact across our community—invest in things that members of our research community can use and build upon.

Best way to keep your organization relevant: The best way to keep our organization relevant is to be responsive to our patients and families. That means responding to them the same day when we hear from them. It means making sure that our patients and families are driving our research priorities. We have families with all different sorts of needs, and if we can help support them emotionally, support them educationally, help them think through care challenges that they have or make a connection that would be helpful to them, we do, and we do it responsively. Over the long-term, that means supporting research that is going to be helpful to patients and families and that’s going to change the lives of people who are affected by this disease.

Why do people like working with you: We are really passionate about our kids and our families. We want so badly to help our kids and I think that is apparent to anyone we work with. We also have a steep learning curve and we depend on learning from the people we collaborate with, and I think they can see that we not only learn from them, but also go out and take productive action based on what we have learned.  We’re professional, respectful, and grateful. Also, we haven’t been around long, but when we say we are going to do something we do it, and partners appreciate that.

Mentor: First is the Triptow family in Ohio. They are amazing. When Yaya was diagnosed, we went online and tried to look for the big patient organization for 4H Leukodystrophy, and there wasn’t one. But we found a family that was hosting an annual golf tournament in Ohio. They were the first ones in our area to get serious about fundraising. It inspired us. They’re amazing parents. They have a daughter that’s affected by 4H leukodystrophy and is quite a bit older than Yaya, so we really look at them and see the effort they put into caring for their daughters, as well as what they pour into their work together. They’re amazing parents and pioneers in our disease area and they’re inspirations to my wife and me. And then three other people—Walt Kowtoniuk at Third Rock has really helped us become thoughtful and strategic about supporting research. Betsy Bogard [who served as the director of global research development for the International FOP Association] has been an amazing source of wisdom and inspiration. And Steve Laffoon, who leads a patient organization called Wylder Nation. He is also a parent who has lost a child and wanted to do something to help other people in the wake of that and set a really great example for us.

On the Job
What inspires you: My daughter Yaya and other kids like her. They are dealt such difficult circumstances and the zest with which they approach their lives and the efforts that Yaya and other kids put in to doing things the rest of us take for granted inspires me every day. Yaya dealt with her diagnosis with incredible grace and every day attacked life and play, and physical therapy and the relationships with her family with enthusiasm and love. I know so many other kids and families that are doing the same.  That inspires me to muster as much of that approach as I have in me and bring it to our work.

What makes you hopeful: Our kids make me hopeful. Our scientific community—the passion and brilliance they bring to their work—makes me hopeful. If we can support them effectively, we will make progress. What also makes me hopeful is that there is an increased focus on rare diseases generally. There are amazing organizations like the Chan Zuckerberg Initiative through which we’ve received funding. The NIH and their rare disease collaborative research network, which recently funded a consortium for leukodystrophies. There’s amazing support and focus, and sophisticated support and focus, on rare diseases. I think that will make a huge difference. I’m hopeful that it will change the trajectory for our specific disease community and for the leukodystrophies more broadly.

Best organization decision: When we started, we intended to just fund research proposals on a one-off basis. But listening to Walt [Kowtoniuk] when he told us to take a more strategic approach to research instead of just funding individual proposals, that’s the best decision we’ve made. It’s a lot of work and it takes more time. Our story is not yet written. We’re in the middle of trying to execute on that strategy. If we can execute on it, I really sincerely believe our organization will have a much greater impact than it would have if we had just funded a few early proposals with the limited funding we had raised to that time.

Hardest lesson learned: The hardest lesson learned is how hard this is and how much time it takes. When our daughter was first diagnosed, we wanted to do something right away and that was not possible. We want there to be a cure now and I’m just learning that it’s hard work and it takes time.

Toughest organization decision: It was hard to not fund research proposals early on. We had proposals from labs telling us that they wanted to do this work. We had good reason for holding off. It was the right decision to ask our researchers to hold off until we had a real strategy.

Biggest missed opportunity: We missed on the first big grant we applied for with our community’s principal researcher. We put a lot of effort into that and we were hopeful that our participation along with her amazing work and credentials would ultimately lead to success, but we did not get the grant and were disappointed.

Like best about the job: Before Yaya was diagnosed, before I was diagnosed, I didn’t even know that this world existed. And since discovering it, I’ve just been so fortunate to meet so many amazing people, so many kids and families in our disease community and across the rare disease community.  I’ve met researchers who are amazing and work so hard. One doesn’t come to rare disease for fame or fortune, but they bring such effort and passion to their work. Then the other patient and family leaders across the rare disease community who are always willing to take time to help, who have amazing insights into our work and into life generally, because of their experience. I love it and it gives me hope, not just for rare disease, but for humanity generally.

Like least about the job: What I like least about the job is seeing disease progression in our kids. We are close with a number of families and it’s hard to see.

Pet peeve: When someone says they are going to do something and then doesn’t do it.

First choice for a new career: It would be amazing if we could discover a cure for 4H and shift our efforts to support the great efforts that other advocates are doing.   

Personal Taste
Most influential book: Orphan: The Quest to Save Children with Rare Genetic Disorders by Phil Reilly

Favorite movie: Rushmore. It’s about love, loss, and revolution. I loved it before Yaya was born and even more after.

Favorite music: Hip hop and R&B

Favorite food: Middle eastern – hummus and kefta especially

Guilty pleasure: Reading National Basketball Association rumor websites about trades and free agency.

Favorite way to spend free time: Being outside with my family and on a bicycle preferably.