Episodi
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“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 Pharmaceuticals
Amy 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
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Imagine you are one of only 10 people in the world with a disease. People with ultra-rare conditions have little hope of attracting research funding to find treatments to work for them. The investments just don’t make sense to for-profit businesses.
Our guest, Dr. Stanley Crooke, MD, PHD is leading an effort to change this. After an illustrious career in pharma and founding the RNA Therapy company Ionis Pharmaceuticals, he is blazing a new trail. He has founded n-Lorem, a non-profit organization that works with families affected by these ultrarare conditions. N-Lorem works to find, develop, and provide new RNA treatments for free, for life.
RNA therapy, which includes anti-sense oligonucleotides, has been a dream for researchers for decades. Dr. Crooke has been at the forefront of this work. Now that years of labor are starting to bear fruit, he is looking to a new chapter. A way to give back. The question has changed from, “Can We?” to, “How can we not?”
https://www.nlorem.org/ https://www.ionispharma.com/
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Episodi mancanti?
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“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.
https://med.nyu.edu/faculty/alison-bateman-house
https://www.youtube.com/watch?v=WM8bTdBs-cw To see how MTV inspired Alison to think about bioethics as a career.
https://med.nyu.edu/departments-institutes/population-health/divisions-sections-centers/medical-ethics/research/working-group-compassionate-use-preapproval-access
https://med.nyu.edu/departments-institutes/population-health/divisions-sections-centers/medical-ethics/research/working-group-pediatric-gene-therapy-medical-ethics
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In the last of our Emerging Researchers Series, I speak with Shandra Trantham, a Ph.D. candidate at the University of Florida. Shandra was destined for a life in science. As a young child, science and math were her favorite subjects. She and I share the childhood dream of becoming an astronaut. I never followed my dream, but Shandra didn’t even have the choice.
When she was 9 years old, she began to lose her balance and things got progressively worse. When she was 12, she was diagnosed with Friedreich’s Ataxia. Our regular listeners may be familiar with Friedreich’s Ataxia because we have talked to other’s about the rare disorder (Kyle Bryant in episode 2 way back in 2019, and Layne Rodden in episode 013). For Shandra, her diagnosis gave her a focus on science, a reason to study, and a purpose.
Her story is informative and inspiring.
You can contact Shandra at [email protected]
Plese support us a https://patreon.com/salemoaks
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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 ([email protected])
Please help support Improbable Developments at www.patreon.com/salemoaks
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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.
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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 [email protected]. You should follow her on LinkedIn linkedin.com/in/layne-rodden-57877b74
Sound Design: Jake Tompkins
Graphics: Heather McCullen
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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/
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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.
Sound Design: Jacob Tompkins
Cover Art: Heather McCullen
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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.
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“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.
Contact Mary Rofael: [email protected] [email protected] https://proedcom.com/
Sound Design Jacob Tompkins ([email protected])
Music “Origins” Per Kilstofte https://machinimasound.com/
“AS – Tribal O1” Aaron Spencer https://machinimasound.com/
Graphic Design Heather McCullen ([email protected])
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The impact of COVID-19 is literally being felt around the world. Humanity has been racing to find weapons to fight this invisible enemy. We know so very little about how the virus is operating. This can leave us feeling confused and isolated.
We speak to Dr. Rob Lambkin-Williams from his home where he is locked down for three months because he is vulnerable to complications of COVID. Rob is a UK-based virologist who has been studying influenza for most of his career. His expertise applies directly to the COVID pandemic.
We talk to Rob about the scientific and regulatory steps that must be taken to bring an effective vaccine to the millions who need it. He lays out a map of these steps and gives us insight into the challenges each one brings.
Join us on this look into what is likely to unfold in the next year or two.
You can follow Rob's work and his blog at VirolologyConsult.com
(Note: We recorded this conversation at the end of March 2020. The situation is constantly changing, and some details may already seem out of date.)
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"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.
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Rob Weker’s story is remarkable for many reasons.
First and foremost, he has beaten cancer.
Not once. Not twice. But three times.
Getting the third diagnosis, this time it was pancreatic cancer, really got his attention. Only 7% of pancreatic patients live beyond 5-years.
I met Rob at last year’s Patients as Partners conference and really enjoyed his perspective. His plea was for the conference attendees to walk in his boots. And yes, he had those boots on stage.
You see, Rob is also a veteran of pharmaceutical R&D. He had a deeper message for his professional colleagues. In his professional life, he turned his propensity for asking questions into a career looking at the process improvement and quality.
Rob is very methodical in how he analyzes problems – whether at work or as a patient. His experience has taught him that it is very important to keep asking questions until there are none. In this episode, he shares a few stories of how this has worked well for him as he battled for his life.
Obviously, on the health side, he has beaten the odds. He just passed the 5-year mark since his diagnosis.
And now he is turning his energy to bridge the gap between patients, industry, and the rest of healthcare.
You can read Rob’s blog on LinkedIn (click here) It’s about being proactive and not relying on luck.
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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.
You can listen to Improbable Developments directly or subscribe on iTunes, Spotify, Google Podcasts, and more. You can follow us on twitter @ImDevPodcast.
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We’ve all been there. You are at a party and someone asks, “So, what do you do?” Dr. Kirsch always found it hard to explain his career as a discovery scientist. He decided to write a book about it. The Drug Hunters: An Improbable Quest to DIscover New Medicines is a colorful, fact-filled narrative history of the search for new medicines from our Neolithic forebears to the professionals of today, and from quinine and aspirin to Viagra, Prozac, and Lipitor.
We talk to Dr. Kirsch about his own experiences as a scientist, the daunting odds of finding an actual medicine, and the additional human hurdles that can make these discoveries even harder. He also talks to us about the things he learned as he wrote The Drug Hunters with his co-author Ogi Ogas, Ph.D.
Credits:
Sound Design: Jacob Tompkins ([email protected])
Music: “Origins” Per Kilstofte https://machinimasound.com/
“AS – Tribal O1” Aaron Spencer https://machinimasound.com/
Graphic Design: Heather McCullen ([email protected])
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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.
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We welcome Kyle Bryant of the Friedreich’s Ataxia Research Alliance (FARA) to discuss his journey as a patient with Friedreich’s Ataxia (FA), as an active patient leader, and in his job as a liaison between the FA community and researchers.
After being diagnosed with FA when only 17 years old, Kyle has dedicated his life to helping other people with FA and exemplifying the adage “Life is how we react to it!” He has reacted by becoming a long-distance bike rider, podcast host, author, and full-time patient advocate.
Kyle opens up about his drive to help others deal with the challenges of life with FA and other disorders. It started when he wrote a daily blog during his first big bike ride from San Diego to Memphis. The comments and responses to this blog made him realize “Oh my gosh, we can make a real impact” by giving people hope and community. It was life-changing for him.
Kyle has a very healthy perspective on participation in clinical trials. He talks about his understanding that he may be just one small “mouse” in a study, but he can be the most powerful mouse that ever lived, an essential ingredient to make a drug a reality.
Kyle Bryant is the author of Shifting Into High Gear: One Man’s Diagnosis and the Epic Bike Ride that Taught Him What Matters. He is also the co-host of The Two Disabled Dudes Podcast (@2DDPodcast) with his friend Sean Baumstark. He and Sean are featured in the award-winning documentary The Ataxian.
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We are honored to have Dr. David Fajgenbaum as our first guest on Improbable Developments. 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.
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In this, our first episode, Kevin lays out the objectives and plan for the Improbable Developments podcast. He also shares a personal story about a medicine he worked on earlier in his career.
