Improbable Developments

“Pharmaceutical companies sometimes have a bad rap of being, you know, big, bad pharmacy. But I learned that the patient or the people I am working with right now are just as passionate and just as dedicated to the patient community as I am.” Amy Grover, Director of Patient Advocacy at Catalyst PharmaceuticalsAmy Grover is a familiar face to the rare disease community. She spent 10 years at Global Genes before joining Catalyst Pharmaceuticals. At Global Genes she learned about rare diseases and the challenges they create for people. In her job now, she is working to bridge the gap between biopharma and the patients and families affected diseases like Lambert-Eaton Myasthenic Syndrome (LEMS). Sounds like a familiar mission.Amy moved from the patient side to industry much like I moved from industry to the patient world. We both were surprised by a few things that we saw as we passed through the looking glass between the two. We both recognize that one of the first steps to bridging this gap is mutual respect. If we work on this, the benefits of partnering will flow. I really enjoyed sharing insights looking at the problem from both directions. www.catlystpharma.com

“I don't think a bioethicist is normally who people have come to their elementary school career day. So, we are a novel breed to many people.” Alison Bateman-House, PhD That is how our latest guest on Improbable Developments reflected on her chosen profession, bioethics. It is a field where scientists do not ask the questions about whether they can run an experiment or clinical study, but whether they should. Until recently, most people would not even know the field existed let alone give a good example of where it made a difference. But COVID has changed that. Who should be vaccinated first? How do we ensure equity in distributing the vaccine? If we run low on oxygen or ICU beds, who do we treat? Should we expand access to experimental or repurposed drugs before they have been tested? These are ethical questions that require considering multiple dimensions and perspectives. They are difficult questions. They are very human questions. Tune in to learn how Alison Bateman-House was led into this challenging field and the work she is doing today on policy, clinical trial design, and ethics consulting. Her career path is fascinating to say the least.

In the latest episode of our Emerging Researchers Series, we talk to Emily McIntosh (@MCINTOSE) a recent Ph.D. from the University of Guelph in Guelph, Ontario. Living less than an hour from Toronto, she is a big Leafs fan (that is the Maple Leafs for all you non-hockey listeners.) Emily earned her doctorate in biomechanics after studying age-related muscle loss and how that influences balance and mobility. As she says, it seems so intuitive, but she wanted to know why this happens. Emily began her work with elderly people as part of a summer research project just before her senior year in undergrad. She went on to get her master’s and eventually started her Ph.D. studies. And that’s when the story got quite a bit more interesting. Emily had gotten a piercing headache that went away in a day or so. It was bad enough that she was worried but not so bad to call the doctor. She attributed it to dehydration and stress. When it happened again, she did call a doctor and was referred to a neurologist who found what he thought was a benign tumor in her brain. After getting a somewhat different and scarier second opinion, Emily used her well-earned scientific skills to start learning as much about this type of tumor as possible. It was this initiative and her ability to think clinically about her own condition that may have saved her life. You’ve just got to listen to hear what happened. You can reach Emily on LinkedIn (https://www.linkedin.com/in/mcinsite) and via e-mail (EMCINT03@UOGuelph.ca) Please help support Improbable Developments at www.patreon.com/salemoaks

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

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/

“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.

"For me it was a question of dying and going to heaven for 32 years." Dr. Kieran Geoghegan was inspired by the scientific and technological breakthroughs of the NASA programs in the 1960’s. This became a lifelong quest to get to the bottom of things and resulted in a wonderful career as a scientist himself. This innate curiosity and determination led him to pursue his PhD in biochemistry at Cambridge College in the UK. He began studying DNA, the very molecules of genetics. However, he found the variety and complexity of proteins much more fascinating. His primary motivation was to live the life of a scientist. He found that working within a company with the shared goal of bringing new medicines to patients offered him this opportunity. He enjoyed the challenges of the applied science and also found time to solve some more fundamental biochemical challenges. His 32-year career at Pfizer spanned the very era when biotechnology emerged as a new industrial force. At each fork in the road, he followed his passion, his skills, and curiosity. This led him to a very high position on the scientific ladder at Pfizer, Dr. Geoghegan has written two books. Creating Cures: A Young Scientist’s First Job in American Biopharma. He discusses life in an industrial research organization, the duality of business and science, and the opportunities working in such an organization brings. Enzymes, Wizards and Secret Passages: Intuitive Lessons in Biochemistry This is a more technical book about some of the hard-won lessons and shortcuts in biochemistry. It is probably most suited for those in the field.

This month listen to Dr. Carol Marzetta talk about how the urge to help other people has shaped her career and her life. From her earliest days as a Discovery Biologist, she has found he passion in work that tries to help people in need. She shares with us some stories about the emotional pendulum around some promising cholesterol lowering programs that did not make the grade. She also opens up about the challenges managing a groundbreaking clinical program for Viagra®, including learning how devastating male erectile dysfunction could be to individuals and their families. While setbacks are expected in Discovery and management pressure is expected for a high-value clinical project, Carol always comes back to the need for helping people to keep her going. In fact, after leaving industry, she spent years working with non-profits like the Bill and Melinda Gates Foundation to bring medicines and vaccines to children in the poorest of countries. Although I have known her for years, the discussion reminded me of why I liked working with her so much. She was always there to help.

Today we hear from David Pearce, PhD, President of Innovation and Research, Sanford Health and Vice Chair, Consortium Assembly, International Rare Disease Research Consortium (IRDiRC)Dr. Pearce’s rare disease background emanates from publishing over 100 research papers on Batten Disease.  David talks to us about how he got involved in Batten Disease research and where that work has taken him since.  In his role as President of Innovation and Research at Sanford he is responsible for overseeing the development of research programs across Sanford’s nine-state footprint, including more than 450 researchers, eight research centers and more than 300 ongoing clinical trials. This includes a national registry for rare diseases, Coordination of Rare Diseases at Sanford (CoRDS). The International Rare Disease Research Consortium comprises more than 50 international entities.  

We are honored to have Dr. David Fajgenbaum as our first guest on Improbable Develoipments. His story is intense. Before he even earned is medical degree, he was faced by a rare life-threatening illness for which there was no treatment, let alone cure. Tune in to hear how he took a novel approach to consolidating the scarce knowledge about his condition, Idiopathic Multi-Centric Castleman's Disease that helped him find a treatment that quite literally allowed him to be on our podcast. His new goal is to replicate that approach for patients with other rare disorders. We also get to hear about his book, Chasing My Cure: A Doctor's Race to Turn Hope Into Action which comes out on September 10, 2019.