Improbable Developments – Détails, épisodes et analyse

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

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/  

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: mrofael@hcg-int.com mnrofael@gmail.com https://proedcom.com/

Sound Design Jacob Tompkins (losstudiosnc@gmail.com)

Music “Origins” Per Kilstofte https://machinimasound.com/

“AS – Tribal O1” Aaron Spencer https://machinimasound.com/

Graphic Design Heather McCullen (heathermccullen@salemoaks.com)

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.) 

"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.

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.

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.

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 (losstudiosnc@gmail.com)  

Music: “Origins” Per Kilstofte https://machinimasound.com/ 

“AS – Tribal O1” Aaron Spencer https://machinimasound.com/ 

Graphic Design: Heather McCullen (heathermccullen@salemoaks.com)  

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 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.