The Basics
Name: Bina Shah

Title: Founder and president

Organization: Project 8p Foundation

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Disease focus:  Chromosome 8p is a rare genetic condition with approximately 350 patients around the world and counting. A chromosome disorder typically impacts every cell in the body. Associated symptoms and findings may vary greatly in range and severity from case to case. Intellectual disability, congenital heart defects, epilepsy, autism, agenesis of corpus collosum, apraxia, GI dysfunction, and sensory processing disorders are some of the diagnoses found with a Chromosome 8p karyotype. Common features include growth deficiency; cognitive impairments; mild malformations of the skull and facial (craniofacial) region, such as a small head (microcephaly) and vertical skin folds that may cover the eyes inner corners (epicanthal folds); heart (cardiac) abnormalities. Additional craniofacial features may also be present that tend to become less apparent with age, such as a short, broad nose; a low, wide nasal bridge; and/or a small jaw (micrognathia). The majority of 8p individuals have developmental delays including disordered acquisition of cognitive and social competence and delays in reaching developmental milestones.

Headquarters: New York City

How did you become involved in rare disease: My daughter is truly the inspiration for Project 8p. She has given me the freedom to lead the cause for treatment. She is 6-and-a-half years old and she has chromosome 8p inversion, duplication, and deletion. Throughout the journey of finding her diagnosis and trying to understand exactly what the disorder was, what the prognosis would be, we realized that we had a plethora of unknowns and questions. There was no central resource for physicians or families or relevant literature.  I realized that there was a huge need to establish an organization to find where these patients were around the world, unite them as a community, and to try to fund translational research with the key objective of trying to find treatments to empower who we call the 8p heroes to be the best they can be.

Previous career: Investment banking working on mergers and acquisitions, valuation consulting work,  and later real estate development and finance.

Education: B.S. in finance from the Stern Business School at New York University, MBA in finance and real estate from Columbia University

The Organization
Organization’s mission: to accelerate the discovery of treatments for chromosome 8p disorders with a translational research program. There are two components that are critical for us to achieve this mission, which are to unite the community and create a standard of care.

Organization’s strategy: What we’re trying to do is establish a collaborative network of clinicians and researchers. Identify all of the expertise not held in just one institution, but to communicate and work together amongst multiple settings and scientists. It takes a lot of effort to successfully facilitate meaningful collaboration keeping in mind what is best and pragmatic for patients and families at the heart of it all.

Funding strategy: Right now its family funded, although we are a public charity. In the last few days we started sharing our story. Fox news covered us and that drew people to our website. We were able to raise money organically that way. Our family has dedicated a sizable amount for now, but that’s not sustainable. We are trying to come up with ways that are efficient with a small team, right now just me.

What’s changing at your organization in the next year: We hope to establish a medical and scientific advisory board and give grants to more researchers to help understand the genes that are responsible for the neurodevelopmental challenges. We’ve been talking to several researchers and hope to have sponsored research agreements underway. We plan to hire more staff and produce one to two peer-reviewed publications with our PIs to ignite some interest in 8p.

Management Style
Management philosophy: It’s important to listen to the questions, opinions, and guidance of the community. We don’t have a full team right now. As we grow and recruit a team, we intend to hire on the development and scientific side. I’m very aware of what I can do well. It’s important to recruit a team that’s passionate, has a hard work ethic, and can wear multiple hats at a small ambitious organization. I have to build a long-term dedicated team. But I’m very careful and methodical about who we bring on board.

Guiding principles for running an effective organization: You need to have milestones and measurable deliverables. You need to have a clear roadmap and hold each other accountable to it. I believe in-person meetings are important. It’s also important to see and meet your “client,” in this case 8p families. I believe that anyone who is going to be on our team needs to interact with the patient families and get to understand them because we’re all kind of detectives trying to identify all the clues and the patterns. I have this mindset that if you’re on the team, this is a family and we all have to be able to depend on each other and rely on each other. I don’t think you can work for an ultra-rare disease organization and look at it as just a job.

Best way to keep your organization relevant: I think it’s important that we invite all of the stakeholders to the table and that all of us are involved in research strategy and refining goals to think out of the box. I think brainstorming together is important. I want our organization to stay as current about the latest and greatest in biopharma, what’s happening in leading academic institutions, and understanding the technologies,  drug repurposing and screening, assays, bioinformatics, and of course gene therapies. Regulatory support is also critical.  

Why people like working with you: I would like to think it’s because I’m very clear about roles, responsibilities, and expectations. At the same time, I view myself equal to everyone else and I really do believe that we are a team. I also delegate with trust. Everyone holds each other accountable. I’m open to that kind of 360-degree feedback.

Mentor: I had several and I think that they all give me guidance that’s unique, but still complements the others. One mentor is extremely task oriented in terms of ensuring that I’m organized in terms of how I get to my objective. And then I have another mentor who is very much big-picture oriented about what we need to do to support professional and personal growth and development.  And then I also have another mentor who is critical in terms of ensuring that I am the best person that I want to be, and just make sure that I maximize all of the potential in me as a human being, as a mother, as a spouse, as a sibling, as a daughter. They all balance each other and enrich my perspective on everything that I’m doing.

On the Job
What inspires you: That’s easy. We just had this conference in the last two-and-a-half days. I am so touched and motivated and driven. I get an endless source of energy from the families that we just met. We had 63 families from all over the world who came together with the chromosome 8p diagnosis. That keeps me going every day, morning and night—to know that everything that we’re doing as Project 8P Foundation is helping them. It’s therapy in itself to just be able to meet each other, and the strangers behind social media posts came together and became friends, and then the friends became family. That is truly the inspiration for all of this—that my daughter will have best friends who are just like her.

What makes you hopeful: The advancements in science and medicine, and the compassion of the rare disease world, and the camaraderie that I’m starting to understand and be a part of. We’re fortunate that this is the time for us to be pursuing a translational research strategy for an ultra-rare disorder.

Best organization decision: To stay focused. There are a lot of different objectives or mission that we could have gone after that are still disease-specific. I think that our focus on translational research, that we’re not just going to fund research that gives us a great paper on phenotypes, but that we’re going to fund research that gives us a great paper on genotype-phenotype correlation, that will help us understand which critical region or genes we’re going to go after. That in itself will translate immediately into the lab and give researchers a  specific focus that is tangible and a hypothesis that can be tested.

Hardest lesson learned: That it’s going to be a long arduous journey and we can’t just rely on researchers and clinicians to solve this. We have to be partners alongside the researchers because we are the drivers. That’s a lesson we have learned.

Toughest organization decision: Which researchers are going to be our partners and our cheerleaders and have the expertise to recruit the right team and bandwidth to focus on us.

Biggest missed opportunity: It’s taken a lot longer to get this thing moving. Sometimes I’ve trusted that things will get expedited. Unfortunately, researchers’ capacity is only so much. That’s been the toughest part and the lesson learned is that we really need to build a larger network of researchers and diversify.

Like best about the job: It is intrinsically rewarding. You wake up every morning and you’re touching people’s hearts and you’re helping them and you’re giving them hope. I wake up fresh and early and I’m ready to go. I often don’t know what time I should end my day because it’s the natural adrenaline rush for me knowing that these families feel like they have a home, they have somebody that is going to be their champion,  and that I want them there right alongside me. It’s truly gratifying.

Like
least about the job: It’s painstakingly slow. You really have to have a lot of patience when it comes to working with the researchers and scientists. I’m used to a fast-paced, New York City / Wall Street mentality and it’s just very different when we’re working with the medical / academic institutions. It requires a lot of patience.

Pet peeve: That is my pet peeve. I’ve offered to personally drive stem cell lines across town in New York. I can do it in 20 minutes. Why does it take months (MTAs to be signed, delays, logistics)?

First choice for a new career: I wish I went the science route. It is so fascinating to me. We had this conference where I carefully crafted 20 hours of agenda. There was 45 minutes with a technical presentation on brain organoids. That completely fascinated me.

Personal Taste
Most influential book: The Gene: An Intimate History by Siddhartha Mukherjee and How Children Succeed: Grit, Curiosity, and the Hidden Power of Character by Paul Tough

Favorite movie: The Godfather

Favorite music: R&B and Reggae

Favorite food: Indian food

Guilty pleasure: Warm chocolate chip cookies

Favorite way to spend free time: Sit and relax by any body of water.

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