Improbable Developments Salem Oaks

We continue our Emerging Researchers series with an interview with Robin Powers, a student just starting her master’s degree work.

Robin is an enigma. She has a wealth of energy and a very fast-moving mind. She has dreams and aspirations that will change the world. Yet she has yet to find the right experiences and coaches to refine her approach. She most certainly has not come from a place of privilege.

Robin is a patient who has been managing and advocating for her own Ehlers-Danlos Syndrome for 17 years now, including identifying the issue and securing the genetic testing required to confirm the diagnosis. She has presented at conferences, been a PCORI Ambassador, and volunteered with other rare disease organizations. She has done all this independently without the luxury of programs or an organization paving the way.

Robin talks about how she came to Buffalo State College and designed her own degree program, Biochemical Pharmacology of Rare Diseases. She also discusses her work to earn a degree in patient advocacy and public policy.

The goal of our Emerging Researchers series is to highlight people like Robin who are full of potential but lacking the experience our other guests may have. These younger people have fresh ideas and the freedom to be bold. For Robin, her life depends on it.

We are excited to introduce our Emerging Researcher Series. Over the next 4 months, we will be talking to some people who are at various stages in their education. Each of them is excited about the research they are doing now and are looking forward to promising futures. We thought it would be interesting to hear what motivated them to pursue science and what they have learned so far. We also thought this would be a great way to showcase some diamonds in the rough.

Our first emerging researcher is Layne Rodden who is studying for her Ph.D. at the University of Oklahoma Health Science Center under the mentorship of Dr. Sanjay Bidichandani. Layne is currently looking at the contribution of DNA-methylation to the silencing of the frataxin gene in Friedreich’s Ataxia. Layne has a unique ability to communicate about the science she is doing to non-scientists.

Layne hopes to earn her Ph.D. in early 2021 and begin looking for her first “real job.” She has a curious mind and is open to many different possibilities. Hearing her talk about this crossroads will throw you right back to those times when you were about to take the leap into a new chapter.

If you would like to connect with Layne, you can reach her at layne-rodden@ouhsc.edu. You should follow her on LinkedIn linkedin.com/in/layne-rodden-57877b74


Sound Design: Jake Tompkins

Graphics: Heather McCullen

We first met Dr. Ethan Perlstein on our other podcast Raising Rare. Dr. Perlstein is a biotechnology entrepreneur.

Dr. Perlstein has nearly two decades of experience in biomedical research and over five years in biotech company formation and executive leadership. He holds a doctorate in molecular and cell biology from Harvard University and was a Lewis-Sigler Fellow at Princeton University from 2007-2012.

In 2014, Dr. Perlstein founded Perlara PBC, the first biotech public benefit corporation with a legal mandate to co-develop drugs in collaboration with families and communities affected by genetic diseases. During that time, Dr. Perlstein served as a member of the Medical and Scientific Advisory Board of Global Genes, the leading rare disease community-led advocacy organization. Prior to becoming a biotech entrepreneur, Dr. Perlstein conducted and led cross-disciplinary academic research in genetics, cell biology, neuroscience, pharmacology, and drug discovery.

In 2019, Ethan joined the Christopher & Dana Reeve Foundation as their first Chief Scientific Officer, but due to unexpected COVID-related financial constraints, his team was let go in Spring 2020.

Dr. Perlstein currently advises https://cureGPX4.org and a few other small foundations. His story is one of a relentless pursuit of ways to help families affected by rare disease.

https://www.perlara.com/

Put me in the game coach.

These six simple words can be putting athletes, soldiers, and employees at great risk. Lynne Becker knows this from her experience building a database of traumatic brain injuries (TBI) for the department of defense. A bit closer to home, both of her daughters have experienced TBI. She has dedicated her life to helping these patients find their way back.

Despite her work as a clinical researcher and biostatistician, Lynne found herself struggling to make sense of her daughter’s signs and symptoms the first time this happened. Because of her work experiences, she was ready to do something about it. She has used all these experiences to create an AI-powered TBI dashboard that helps patients get a correct diagnosis, track their progress, and regain some control of a very scary situation.

In this Episode of Improbable Developments, you will hear Lynne’s story and how a series of events has shaped her career. Ultimately, this journey has led to the establishment of Power of Patients, an organization dedicated to supporting the victims of TBI.

https://powerofpatients.com/

Mike Wenger is Vice President of Patient Engagement at TrialScope. A brain tumor patient and software developer, Mike is passionate about patient access to clinical trials. He is spearheading TrialScope Connect, the industry’s first patient referral collective. This initiative is creating a paradigm shift for patient recruitment, focusing on collaboration vs. competition.

Previously, Mike worked at the Michael J. Fox Foundation where he helped develop the Fox Trial Finder, a clinical trial matching platform that educates patients on trials and helps them locate trials best suited to their needs. He also was a lead software developer at Epion Health, transforming the doctor’s appointment into ¬a personalized digital experience where patients could share information with their doctor and take ownership of their own health management.

Mike was named one of the 2020 PM360 ELITE 100 in the Patient Advocates category and a 2020 Force for Change Illuminator. He also was a finalist in the Patient Advocate of the Year category in the 2020 Medigy HITMC Awards.

Mike became interested in working in healthcare when he had a health scare himself. Because of this scare, he started keeping a headache diary, which helped him learn to manage those headaches. He felt very fortunate to have been able to use these data to improve his condition. This, in turn, motivated him to find ways to help others.

“I think it is really important for the public and the patient Community to learn a little more about FDA advisory committees because these are public meetings and they're accessible for anyone to attend and learn from.” Mary Rofael, MD

An FDA Advisory Committee is one of the biggest moments in the development of a new treatment. As a drug developer, I consider the FDA Ad Comm as the pinnacle of my career. During these hearings, a company is given a short time to make the case for the approval of their New Drug Application (NDA) and answer questions from a panel of independent experts. The FDA has a similar opportunity to present their concerns and question to that panel of experts. And all this is done in public.

Needless to say, consolidating 12-15 years of collected data into a couple of hours is daunting. Over the past 20+ years, Mary Rofael, MD has built a business, ProEd Communications, that helps guide companies through this major event. In this episode were learn how those companies prepare, what ProEd Communications brings to the table, and why Mary finds this work so fulfilling.