Rare Leader: Neena Nizar, Founder and Executive Director of The Jansen’s Foundation

The Basics
Name: Neena Nizar

Title: Founder and Executive Director

Organization:  The Jansen’s Foundation

Disease focus:  Jansen’s metaphyseal chondrodysplasia is a ultra-rare autosomal dominant skeletal dysplasia characterized by short-limbed short stature (due to severe metaphyseal changes that are often discovered in childhood by imaging), waddling gait, bowed legs, contracture deformities of the joints, short hands with clubbed fingers, clinodactyly, prominent upper face and small mandible, as well as chronic parathyroid hormone-independent hypercalcemia, hypercalciuria, and mild hypophosphatemia.

Headquarters:  Elkhorn, Nebraska

How did you become involved in rare disease: I have the rare disease myself. I was 32 when I was diagnosed. By then, I had already passed it down to my two boys. There are only eight known cases of Jansen’s disease worldwide. That’s when I realized how rare this condition was. I also quickly realized that nothing had changed much since I was a little child; invasive surgical procedures throughout childhood was still the only option. I was pretty upset that despite all the advances in bone and mineralization, there wasn’t much being done in rare skeletal diseases. I wanted to change that. I wanted a different treatment option for the children and a chance at a pain-free childhood.

Previous career:  I was a teacher at an international baccalaureate school in Dubai.

Education: Bachelor’s degree in English and a double Master’s degree in English and Philosophy from All Saint’s College, Kerala, India; Doctorate in Interdisciplinary Leadership from Creighton University in Omaha, Nebraska.

The Organization
Organization’s mandate: Our mandate is to create awareness and fund research. Because of the rarity of Jansen’s disease, we focused our first year on bringing awareness to the condition. A second part of our mandate has been to get funding for research. We succeeded in getting an NIH grant last year. Our research team at Harvard Medical School & Boston Mass. General Hospital have a therapeutic treatment they have been working on and we are hoping to get that to market as soon as possible. The treatment aims to stop the over-active PTH receptor from turning over bone so quickly.

Organization’s strategy: We’ve been very careful in our strategy. When we started in 2017, we had these two big goals. We knew creating awareness of the disease would help us find others with Jansen’s and true enough we ended 2017, having found 3 new cases to take our community to eight in total. Our strategy has been to make the disease visible and make our patients’ voices heard. We did a Discovery channel documentary and got purposeful media coverage documenting the disease. We also aimed at creating an informative website that helped with disease identification and educational programs. I’m handicapped in the sense I can’t move around much because Jansen’s affects mobility; it’s not always easy attending every event in the rare disease space, so we pick and choose events wisely and strategize from living room board-meetings. Another important strategy is finding the right collaborations. As a NORD member and a Global Genes Foundation Alliance member, I have been successful in making successful inroads into the arena of drug development. We have an amazing core team of dedicated professionals and our goals are very clear. We knew right off the bat that crowd-funding was time-consuming with such a small disease-population and no drug company was going to be interested in such a rare disease, so we had to work wisely in getting our story out there and finding angel investors through our zeal for hope and faith.

Funding strategy: The funding strategy has been grants and we’ve been approached by a couple of angel investors interested in investing in the drug because of its crossover applications. If we develop this drug, then we may be able to help more common diseases like osteoporosis and osteoarthritis. Even though we are a small population, it’s been incredible the number of people who have jumped on board and said, “Yes, we want to help you” just by listening to our story.

What’s changing at your organization in the next year: We are getting prepared for our first clinical trial. We’ve been working on a pre-IND meeting with the FDA for this year. We’ve been contacted by a production company that wants to document our journey towards the l trial. We also have our very first skeletal dysplasia conference in the Midwest region and we are excited to get some very big names from all over the country coming to Nebraska on September 7, 2018.

Management Style
Management philosophy: Strengthening credibility across the board is important for all of us at the Jansen’s Foundation. We focus on developing values-based goals; we are constantly reflecting on our mission, not only thoughtfully and critically, but also with a sense of passion and ethics. We are looking not just for cures but also how we as an organization can serve the common good. We’ve been very transparent about what we want and how we intend to achieve our goals of advocacy, awareness, and education.  The board members understood the importance of making this a participatory and collaborative venture. We knew that serious changes were likely, and that change always carries risk. I think we all recognized that the best way to manage that risk was to make sure we were all on the same page.

Guiding principles for running an effective organization: The Jansen’s Foundation members have the ability to create and sustain the vision and mission; inspire and influence others to join us; prioritize; be decisive but inclusive; be open to innovation and manage risk, and hold ourselves accountable to the mission; all in an effort to achieve the organizational mission. The Jesuit principle of magis pervades the core of our foundation. The magis drives a person toward “something more, something greater”. Most people wonder about their capacity to act heroically should a momentous opportunity suddenly present itself. Our guiding principles force us to consider instead our capacity for heroism on a daily basis. Heroism is not just a response to a crisis, but a consciously chosen approach to life. It is judged not by the scale of the opportunity, but by the quality of the response to the opportunity at hand. Every day we are presented a choice, and we choose to devote each day to a work so vital it feels like the well-being of the whole world depends on it.

Best way to keep your organization relevant: We’re always looking to be compliant with federal policy and state policy. We are very much up to date with technology and advances in science. We also look at different ways of using science, research, and data to connect with patients. As a nonprofit organization, it is important to be engaged and relevant to the community we serve.  We believe in passionate discussion. By putting ourselves out there and having discussions with a wide range of stakeholders, we are able to find opportunities that align with our mission. Ultimately, we aim to create deeper and more meaningful relationships with all stakeholders. The Jansen’s Foundation’s responsibility is to not just advocate for the mission of but to be a voice for the broader vulnerable populations we serve. Leadership is transactional and transformative in creating cohesion throughout the community. We keep relevant by being consistent and involved, promoting opportunities for meaningful change.

Why people like working for you: I’m passionate about what I do. I believe change is possible. People see we’ve been successful in a short amount of time. They believe in the mission. I think that’s the biggest buy-in factor.

Mentor: My mentor has always been my father. He’s always been a fixer. He fixes everything that is broken. He doesn’t accept “this is not going to work.” I learned very early on life the lesson of perseverance. In the rare disease space, I have several mentors. I am new in the field and I love hearing everyone’s stories. I love connecting with advocates because they have already done things I’m just setting out to do. It’s nice to see people who have gone before me and learn from their success and mistakes.

On the Job
What inspires you: Hope. The idea that we can actually change something. When we start seeing these small successes, it inspires me to work harder. Every pursuit offers its own version of the same heroic choice. How one chooses profoundly affects personal satisfaction and performance quality; after all, how can you not be motivated when the “wellbeing of the whole world” depends on what you do, right?

What makes you hopeful: Science and faith; a juxtaposition of sorts, but it’s truly a remarkable period in time for science and medicine. When I was growing up there was nothing. Doctors told my parents to take me home and pray. I remember being tied to an ironing board because my bones were bending, and my parents didn’t know what else to do to keep them straight. Today, we have so many other options and a therapeutic treatment in the works.  How incredible!

Best organization decision: Backing up our research team and working with them. That’s been monumental. Such collaborations, if managed effectively, can contribute to more comprehensive research and more effective policy advocacy.

Hardest lesson learned: I come from a different country and I have had many hard lessons along the way. The toughest one has to be the cost of cures and life-saving treatments. That was a hard lesson I had to quickly learn. Other smaller, but still significant lessons, include learning to navigate the healthcare and insurance system. I had to learn how drugs are developed and to understand all the nitty-gritty of the role of pharmaceutical giants and changing place of patient advocates.

Toughest organization decision: I don’t think I’ve had any tough decisions so far. I am very clear in my mind as to what our mission is and so decisions are not hard to make with the support of our very committed board and scientific advisory team.

Biggest missed opportunity: We haven’t had a missed opportunity yet. There are some events I don’t get to attend that might have been great opportunities, but I don’t see those as missed opportunities, but rather as moments that weren’t mean to be at that specific point in time.

Like best about the job: That it’s not a job. We’re fighting for lives here. We’re fighting for cures. I wish I didn’t have this job. I wish cures were something everybody had by right. I don’t see this as my job. This is what needs to be done. It’s part of my every day.

Like least about the job: Travelling with the wheelchair and the physical toll it takes on me.

Pet peeve: Airline people breaking my wheelchair and being callous about it!

First choice for a new career: I see the need for a pharmaceutical company in my future…

Personal Taste
Most influential book: To Kill a Mockingbird by Harper Lee. I learned that book as a child and I got to teach that book to my first class. It has so many powerful lessons and so many sweet memories tied to it. I still find myself quoting from it regularly.

Favorite movie: Black Panther. The shining splendor of the Wakandan warriors poised and ready to fight is a collective soul as timeless and indestructible as vibranium itself.

Favorite music: ‘80s music.

Favorite food: Indian.

Guilty pleasure: Dark chocolate.

Favorite way to spend free time: Researching.

May 24, 2018