Rare Leader: Tim Ogden, President, Bardet-Biedl Syndrome Foundation

The Basics
Name: Tim Ogden

Title: President

Organization: Bardet-Biedl Syndrome Foundation

Social Media Links:

Disease focus: Bardet Biedl syndrome (BBS) is a rare genetic disorder with highly variable symptoms which may include retinal degeneration, obesity, reduced kidney function, polydactyly (extra digits of the hands or feet) among many other features. While there are more than 20 genes associated with BBS, the underlying cause regardless of gene is malfunction of primary cilia, a key component of cellular communication. BBS is thus categorized as a ciliopathy, or a disease of the cilia.

Headquarters: The Internet (we really don’t have anything like a physical location)

How did you become involved in rare disease: My older son, Nathanael was born in 2006. If not the first, he was one of the first children to be diagnosed with this syndrome in utero through a set of lucky circumstances. We ended up being referred to a nephrologist at Children’s Hospital of Philadelphia who had seen patients with Bardet Biedl Syndrome, which is pretty rare. He recognized it on the sonograms that had all of the other doctors stumped as to why my son was even alive based on what they were seeing. Right from the very beginning you have this diagnosis that is relatively uncertain, but it’s pretty clear there is something wrong. You plunge into that world of, “What is this? What happens to children with this syndrome? Who can we talk to?” It all began with that diagnosis in November 2005.

Previous career: Currently heads a research center at NYU-Wagner exploring how financial services can better meet the needs and improve the lives of people in poor households.

Education: B.A. International Political Economy from Davidson College

The Organization
Organization’s mission: Our mission is to improve the lives of all people affected by Bardet Biedl Syndrome.

Organization’s strategy: With a rare disease, the most important strategy is being visible so you can find and connect to people with the disease. Bardet Biedl Syndrome is exceedingly rare. The definition of “rare” according to the government is that it affects less than 1 in 2000 people. The best estimate of Bardet Biedl Syndrome in North America is about one in 250,000. We’re rare among rare, and rare enough that many people, presumably, are not even diagnosed with the disease. Even those who are diagnosed often don’t think to reach out that much, because it’s so rare. They don’t know that there are other people out there. First and foremost, our strategy is to make ourselves findable for anyone who decides to do a search for Bardet Biedl Syndrome so they can connect. Beyond that, our major effort is to involve not only the people of North America, who we directly serve, but everyone in the world around a clinical registry because within the means that we have as a very small organization that affects very few people the difficulty of raising enough funds to directly fund research is pretty insurmountable. But we can lower the costs of other people doing research, including pharmaceutical companies and basic researchers. And we do that by getting as many people as possible in a clinical registry, so that it’s easy to find patients and easy to document over time the issues those patients face.

Funding strategy: All of our funds come from direct fundraising by families affected by the syndrome. With most nonprofits, and certainly most rare disease organizations, there are a handful of families who have taken up the mantle to lead on fundraising and started fundraisers that have grown to meaningful size $10,000, $15,000, $20,000, $30,000 a year. And then a much wider group of families who donate either directly to the foundation or run smaller fundraisers with friends and family raising $500, $1,000, $2,000 at a time.

What’s changing at your organization in the next year: We are at an inflection point. The rate of diagnosis of Bardet Biedl has gone way up in the last 10 years because 25 different genes have been found. The most common of those genes are part of a pretty standard genetic disease screening tool that geneticists and neonatologists use when they’re not quite sure what is going on with a newborn. We are seeing many more people diagnosed early. That gives us some ability to work more closely with those families and over time to grow our ranks and expand our reach to make sure that we have found the 3,000 or so families in North America who are affected by this disease. The other big thing that’s changing is, as with many rare diseases that have been able to identify causal genes, there’s a lot of action going on in gene therapy. There are a couple of different organizations now who are working towards human trials of gene therapy for some of the major BBS genes.

Management Style
Management philosophy: You need to be able to cast a vision that there is a place to be going—that joining in, supporting an organization like the BBS Foundation, isn’t just about having a shoulder to cry on and finding people who can relate to you. That’s important, but that’s not enough. You have to be able to encourage people that there is a reason to come together and to work together, that progress is possible through collective action. A lot of what we spend our time doing is making sure that people do understand that no matter how rare this is, there are a group of people who care, who are working hard to try and find ways to better care for patients and ultimately develop treatments. And that we can accelerate that. The overall philosophy is to be relentlessly positive but directed. You’re not pie in the sky, everything’s going to be okay, but there are actual concrete actions that we can take now that are going to lead to better outcomes for the people of our community.

Guiding principles for running an effective organization: One of the particular challenges we have with Bardet Biedl Syndrome is that it is not only rare, but it’s highly variable. Not only are there a bunch of different genes, but even a specific gene is not all that predictive of the symptoms that a particular individual will have, or the severity of the symptoms. You can have two children in the same family with the exact same causal gene who have quite different symptoms from each other. It would be easy then for the community to splinter around a gene or a particular set of symptoms, but you can’t do anything when you take something that’s super rare and then split it up further than that. The big focus for us is that collective action is the only path to real effect. That involves making sure that we’re hearing the needs of the community that are quite varied from a symptomatic standpoint and severity standpoint and keeping that tent as big as possible and everyone focused on the ultimate goal.

Best way to keep your organization relevant: We have to be able to communicate useful information. We are in a transition over the last 20 years from “we don’t really know what this is” to “we found the first gene” to “now we know what that gene is supposed to do, or at least what protein it is supposed to   create” to “we know what that protein is supposed to do and understanding it sort of its role in symptoms.” There was a span of time where we were explaining to people what was going on with a syndrome, but you can only do that for so long. You have to keep doing it for all the people who are newly diagnosed, but to stay relevant, you’ve got to be able to offer something for all of those families that isn’t just explaining what this disease is and why their child, or their loved one, or they themselves have this strange group of symptoms. We have to evolve with the times. What are the needs of the community? We need to be able to hear those needs and find ways that we can respond to those needs at the same time. That is the core of the challenges with any disease that includes degenerative features and that primarily affects developmental issues. People are running out of time every day. Every day that a child loses some retinal cells, every day that there isn’t effective therapy around some of the other symptoms, it’s harder to get those things back. Keeping people motivated around progress that is slower than everyone would like is a big part of the challenge of relevance.

Why people like working with you: I don’t think I could effectively lead this organization if I wasn’t living through all of these challenges myself and seeing firsthand what it means to be a parent, knowing that your child is going to lose vision, and wonder if there is going to be kidney disease, or some other major health problem. Is my child going to need a kidney transplant? How am I going to arrange that? How can I make sure that my child gets the services that he needs in school in hierarchies and bureaucracies of education that are designed to deny services as much as possible and limit what the school’s responsible for. Because I’ve lived that I can relate to the parents who are first diagnosed. I was there. I experienced all of those emotions.

Mentor: I don’t know that I have someone I would call a mentor in relation to rare disease. That being said, I have a brother who’s 10 years younger than me who had significant development delays for reasons we still don’t know. I watched my parents spend a couple of months in the hospital and an ICU with my younger brother, watched them navigate through that process of helping him achieve everything that he could, and navigate through the delays and disabilities that he had. I did have a model for how to be a parent of a child with challenges.

On the Job
What inspires you: I am constantly amazed that there are a large number of people who are dedicating significant amounts of their lives to understanding what’s happening with people affected by BBS—that there are researchers who have devoted years and years and years to Understanding this syndrome. What are the genes? What do those genes do? How do these systems work? They are still working on these questions of why is it that the symptomology is so different, even when people have the same mutations? These scientists are dedicating themselves to figuring out whether and how gene therapy will be possible. As a parent, I don’t think you can help but be inspired by people who devote so much of their time to something that can make a difference for your child, but that doesn’t affect them. The scientists doing this research don’t have children with BBS. They just decided that they did not want to live in a world where no one understood this disease and there was nothing anyone could do about it. I find that tremendously inspirational. It if those researchers are willing to do that, then I have to do what we can do to make sure that they have the resources necessary to do the work.

What makes you hopeful: We have seen unbelievable progress. My son Nathanael has the BBS10 gene. I happen to know the researcher who was working on characterizing the BBS10 gene. She started working on it around the same time that Nathanael was born. To go from a place where we’re still trying to identify how many genes there are to a place where we are involved in gene therapy experiments with mice, and seeing those mice not only experience less retinal degeneration but also regain some vision they had lost within 15 years is astounding. The message I try to give people in our community is that our children are going to see again. I don’t know exactly when, and I don’t know which technology is going to do it, but I am thoroughly convinced that given the resources that so many different people are pouring into this, that in his lifetime, my son is going to get his vision back.

Best organization decision: When I joined the organization, it was not incorporated. It was a family association. It was not a nonprofit. I volunteered, I guess you could say, to take over the association at that point and convert it into an actual nonprofit that could engage in collective action and fundraising. The  very first thing that we did  was work with Robert Haws at the Marshfield Clinic to establish CRIBBS, the clinical registry investigating Bardet Biedl syndrome. I was convinced  that the thing that we could do, given how rare this disease was and how scattered everybody was, was to create a clinical registry that would capture all of this information and would make the work dramatically easier for researchers. We started that in 2012. It has made a tremendous difference. Rhythm Pharmaceuticals is applying for final approval of the first pharmaceutical treatment for one of the major BBS symptoms, insatiable hunger. Based on my conversations with them, they worked on Bardet Biedl syndrome because there was a registry. Some of the organizations working on gene therapy are doing that because they know that there is a way to start to reach patients. As they make progress, they can find patients for clinical trials. Unquestionably the biggest, most important thing that we’ve been able to do is create and fund the clinical registry.

Hardest lesson learned: The window for every person affected by BBS closes a little bit every day. Finding ways to connect with people, get them motivated, channel their energy and their desire to help their loved ones is a hard thing to do. We have to continue to try and reinvent and find ways of doing things that are going to be relevant to everyone in the community.

Toughest organization decision: This is a constant tough decision: how we allocate resources between our mission to connect to as many people as possible and advance treatments. Those are different things, and both of them need funds, need effort, and trying to find the right balance between those is a difficult thing every day.

Biggest missed opportunity: We are the largest organization focused on Bardet Biedl Syndrome in the world, in part because the United States is a big and wealthy country with relatively good, high-end medical care. We get a lot of inbound requests for help from families in countries where there isn’t  infrastructure like we have in the US. Last week I had an email from a Libyan family living in a refugee camp in Greece asking for help because their child has  Bardet Biedl Syndrome, but at this point there isn’t much we can offer. I would love to be able to help families in Russia and China and India and Pakistan and Libya and Guatemala. All of these are places that we get emails from, but we don’t have the resources—financial and linguistic—to help a lot of those families. My heart aches because if I had been in this place where there was no one to help me, there were no doctors in my country that knew very much about this disease, I didn’t know what to do for my child, I don’t know what I would do. It breaks my heart that we can’t do more for them.

Like best about the job: The moments that I enjoy the most are connecting with parents whose child has just been recently diagnosed and being able to tell them there’s somebody out there who cares about this. There are other families that have survived this. There is hope and there are people to walk alongside you. When I get to do that, those are my favorite moments.

Like least about the job: Trying to figure out how to get the million things I want us to be able to do done with the budget to do a hundred of them.

Pet peeve: I guess the thing I have the hardest time with is that the world is filled with pseudo-science now. A lot of that pseudo-science preys on parents of children with rare diseases who are desperate to hear that there’s something that they can do. I hate having to spend the time, and feeling like the Grinch, when you tell people that this dietary supplement or, that off-brand medication, isn’t actually going to do anything for your child. It’s terrible. It’s necessary, but it’s terrible.

First choice for a new career:  I mentioned earlier that we’re at an inflection point. Right now, my fantasy career would be to have the resources to do this all the time. Now is the time where we could make huge progress if we had the staff time to go out and talk to and connect families, help organize them, reach out across countries, create resources for people outside of North America. If I had the resources to do that, that’s what I would do.

Personal Taste
Most influential book: Heart of Darkness by Joseph Conrad is something that I’ve always carried with me from when I read it in college. I read it in high school and didn’t get it, and then read it in college with a great professor who really helped me understand what was happening there. One of the core ideas is that it’s not enough just to coast along. It’s not that there are good and evil people. It’s that we all have the possibility to end up doing terrible things to other people, so we have to, every day, choose to do good. Seeing Like a State: How Certain Schemes to Improve the Human Condition Have Failed by James C. Scott is the other book. It’s about the process by which organizations have to simplify the world in order to accomplish things, and all that can be lost when you do that.  The converse is true. How do people understand the organizations that are trying to deal with them? It influences a lot of how I think about how we organize ourselves, how we relate to people, but also how we communicate to people so that they understand how the organization actually works and why it’s doing the things that it’s doing.

Favorite movie: Memento

Favorite music: Crowded House

Favorite food: I hate mushrooms, that is about it. I really enjoy a very wide variety of food.

Guilty pleasure: I coach three soccer teams, not for my kids, but because I really enjoy coaching soccer. I’ve devoted a substantial amount of time to it because I like doing it. I like working with the boys and helping them improve. I feel guilty about the amount of time I spend coaching soccer.

Favorite way to spend free time: Coaching soccer.