Rare Leader: Danae’ Bartke, executive director and co-founder, HCU Network America

The Basics
Name: Danae’ Bartke

Title: Executive director and co-founder

Organization: HCU Network America

Social Media Links:

Disease focus: Homocystinuria is a group of inherited metabolic disorders. Homocystinuria causes an elevation of the amino acid, homocysteine (protein building block coming from our diet) in our urine. Homocysteine can also be elevated in blood. People can have these problems from birth through their genetics (that which we inherited from our mother and father) or it can be acquired. Untreated high homocysteine levels can affect the Skeletal, Vascular and Central Nervous Systems as well as the eyes.

Headquarters: Batavia, Illinois

How did you become involved in rare disease: I am actually a rare disease patient myself. I have a younger brother also with homocystinuria. When he was five years old, he hit his head on a filing cabinet at school. He came home from school complaining he couldn’t see; this persisted for a couple of weeks. My mom took him in to see an ophthalmologist and they discovered that his lenses had dislocated in his eyes. He had immediate eye surgery to have his lenses removed. In the following work-up they determined it could be one of two things. They sent us to a geneticist and they confirmed the diagnosis of classical homocystinuria. Within a few months the rest of us were tested, and I was also confirmed to have classical homocystinuria.

Previous career: ESL teacher

Education: A.S. in early childhood education and teaching from Waubonsee Community College, B.S. in Elementary Education and Teaching from Northern Illinois University, ESL Endorsement teaching English as a Second or Foreign Language/ESL language Instructor from Aurora University

The Organization
Organization’s mandate:  HCU Network America strives to inform and provide resources for patients and families, create connections, influence state and federal policy, and support advancement of diagnosis and treatment for HCU and related disorders.

Organization’s strategy: Our four-pronged approach centers on various types of engagement and collaboration. Everyone has to work together for the betterment of not just homocystinuria patients, but for all rare diseases. Therefore, we believe in engaging patients and their families, clinics, industry and those at the federal and state level. We can’t do things in isolation if we expect to make strides forward.

Funding strategy: We try to be transparent as we develop as an organization. A lot of what we’re working towards in our transparency is making sure that a lot of our funds are either going towards our programs or our research. We don’t have a clear fundraising strategy. The big thing is being honest and upfront with the community about where their funds are going.

What’s changing at your organization in the next year: There are things that we have been working on and they’re going to carry on through. Homocystinuria is an umbrella. It covers classical homocystinuria, which my brother and I have, but it also encompasses several other homocystinurias. They fall under the cobalamin disorders and severe MTHFR. What we’re working on is bringing these various communities together and helping them understand how they all fit together under the term homocystinuria. We are also working on getting our committee to collaborate more on projects that line up together with those communities. That will also help with that larger goal of bringing the communities together.

We are still working steadily on revised newborn screening recommendations for homocystinuria and related disorders. We’ve been working on that for the past three years, working with consultants, the ACHDNC, CDC, and our Newborn Screening Advisory Board. This year we will be looking to collaborate more with state health departments and newborn screening labs to bring our vision to life.

Management Style
Management philosophy: We are all a team, but each individual member plays a vital role. They’re just as valuable as I am. We’re all working together to reach these goals that we have set out for our organization. We all have to pull our own weight, but we can lean on each other for support too.

Guiding principles for running an effective organization: We are all on the same team working together. It’s important to keep in mind when we are planning because we do cover not just classical homocystinuria, but the cobalamin disorders and other remethylation disorders. While each of us have our own unique needs and differences, we are all here together under that same mission. When we can keep that in mind we can do a lot more with the tools that we develop, the research that we fund, and even moving along research in general. If you look at things in too much isolation, you’re only going to get a narrow scope of things.

Best way to keep your organization relevant: It’s important to listen to the community members, the patients, the parents, the clinics, and I include our industry partners in this too because we all are helping and working together in this. The picture is not complete without every piece to the puzzle working in unison. Listening to your community, taking that information and feedback back to your different committees and boards, and seeing how we can incorporate their viewpoints and what else they have to offer into what we are working on. Remember to reevaluate your priorities so it’s meeting the needs of that community. It’s critical.

Why do people like working with you: I like rolling up my sleeves and being in the trenches with everyone else. I try to see things from different perspectives—the patients, the parents, the clinics, industry. Being able to take on those various roles and see things from these different lenses allows you to work well with others. First and foremost, while I am an executive director, I am a patient too. That goes a long way in working in a patient organization and patient advocacy.

Mentor: I don’t have a mentor per se, but there are people within the rare disease space who inspire me and who I look up to. Our president, Margie McGlynn. She’s been a phenomenal leader to our community. I don’t know if HCU Network America would exist without her. Because of her background in pharma and her familial ties with homocystinuria—she had two sisters who passed away from homocystinuria when it was a first recognized disorder—she brings so much expertise to the table and experience that is hard to find. There’s also Sarah Chamberlin, who’s very much involved with PKU News. She has a brilliant mind and always finds ways to make things better not just for the PKU community, but the low protein community as a whole.  She is always willing to brainstorm and collaborate, which is extremely helpful. I appreciate both of them tremendously.

On the Job
What inspires you: Patients and their families and their experiences and stories. It’s wonderful seeing the things that we do but knowing how much more we actually have to get done keeps me going. You sometimes lose sight of things, and you need to hear those stories every so often.

What makes you hopeful: Where we’re at with science and research, it’s absolutely incredible. Within homocystinuria, we have research going on with enzyme replacement therapy, gene therapy and several other areas. If you would have asked me even 10 years ago, “Do you see things changing?” I would’ve had a very different outlook. The research is moving forward fast now. It’s an exciting time to be a patient, and to be in the rare disease space. Patients are getting cures and those are words that we didn’t hear before. We had therapies. We had treatments. To know that there is a cure ahead that will change those newborn diagnoses. It’s extremely exciting and it makes me incredibly hopeful.

Best organization decision: The idea of organizing was a big decision. At the time I co-founded HCU Network America with Margie, I was actually the president of another nonprofit organization for another rare disease. I was very hesitant, but I have learned so much, connected with so many people, and just grown in ways that I would have never been able to foresee had it not been for helping establish HCU Network America. That was probably the best decision that we’ve made because we’ve been able to make such tremendous progress in five short years. It feels like the fastest five years of my life.

Hardest lesson learned: The hardest lesson learned has been to slow down. That’s a funny lesson to learn, but sometimes we’re so excited to roll things out and a little bit of extra time could make things just that much better. It’s like watching a little kid when they’re learning to walk. They get so excited and they get things moving too fast that they fall down. The idea of slowing down and making sure that you’re doing everything to the best of your ability is important. That’s something that I’ve had to learn.

Toughest organization decision: The toughest decision was one of our best decisions we’ve had to make. The toughest decision has been to open ourselves up to the larger homocystinuria community. It created a lot of debate, which in retrospect, I feel bad even having to admit. It’s an important thing to have to admit because our board was only affected by classical homocystinuria. We lived in isolation and we weren’t sure how to accommodate them and their needs. We knew that the disease affected them differently and their symptoms were different, but here they were. They have the same problem; they all have high homocysteine. While it was our toughest organizational decision, I have to say it was one of our best organization decisions because we’ve been able to work together with that side of the community. Things have moved along incredibly fast for them because they now have a home. We are seeing things in terms of therapies also moving along, which it’s exciting to see for them because they’re very underserved otherwise.

Biggest missed opportunity: I don’t think there’s really been any missed opportunities. We are careful in our approach. They’re not missed, they’re just postponed opportunities, I would say. Sometimes an opportunity presents itself, but it’s not the right time. We can look back and be sad about that, but also, other opportunities will come along that are better fit for the time and place that we’re at.

Like best about the job: I love the patient engagement. I love being able to help families find the resources they need. It’s important to be able to help families, regardless of their rare disease state, find what they’re looking for. Sometimes it’s about networking and being able to connect them with the right person. That’s my favorite part about the job—being able to help families be more successful in their diagnosis and treatment.

Like least about the job: Not being able to have all the answers. It’s hard when families have hard hitting questions and there’s not always enough research to answer those questions. It’s difficult not being able to give families the answers they need and deserve all the time. I do my best, but that’s about all I can do. If I can’t answer their questions, I try to find answers, and still sometimes they don’t exist. Hitting a dead end is difficult.

Pet peeve: One of my biggest pet peeves is when you have clinicians say classical homocystinuria is easy to treat. I’ve heard this multiple times. The fact is that even with that treatment, it’s still difficult. Classical homocystinuria requires a low-protein diet and formula, and it’s extremely restrictive. It’s similar to how PKU is treated. By the time patients are 20, 50 percent of them are off diet. If it’s so easy, why do patients struggle? if it’s so easy, why do patients have trouble accessing the care? It’s not easy for the patients, or their parents. It completely undermines their struggle when you get brushed aside and say, “Oh, that’s easy.”

First choice for a new career: I love my job and it would be really hard to envision a different one, but if I were told I could not continue with my job, I would like to go back to school and be a geneticist. I never thought about that as a job before entering the rare disease space, but after working so much in it, I think it would be great to be able to actually engage and work with patients on that level. That would be exciting to be on the forefront and help patients through their diagnosis and treatments.

Personal Taste
Most influential book: The most influential book that I’ve read was a compilation of biographies in a book called They Poured Fire on Us From the Sky: The True Story of Three Lost Boys from Sudan, by Benjamin Ajak, Benson Deng, and Alephonsion Deng. It’s about the journey of navigating for freedom in a space that is war torn. It’s an amazing book and puts a little perspective into how lucky our lives are.

Favorite movie: I love Disney movies. My favorite movie is probably Beauty and the Beast. When I was younger it was always about Belle’s castle and her library there.

Favorite music: Pop Punk

Favorite food: Pizza. I can’t eat cheese, so I eat cheeseless pizza. It’s still delicious though.

Guilty pleasure: Dunkin’ Donuts coffee. You can never have enough coffee.

Favorite way to spend free time: I like spending time outdoors, going on nature hikes. It’s a little hard to do right now in Illinois with three feet of snow on the ground, but If I can transport myself to somewhere warm with clear trails, that would be where I’d want to be.