Improbable Developments Salem Oaks

“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

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 https://www.nlorem.org/ (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.nlorem.org/) https://www.ionispharma.com/ (https://www.ionispharma.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 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 shandra.trantham@gmail.com


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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 (EMCINT03@UOGuelph.ca)

Please help support Improbable Developments at www.patreon.com/salemoaks

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.