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/

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