Speaker

Adrian Hernandez, MD, MHS
Vice Dean and Executive Director
Duke Clinical Research Institute
Duke University School of Medicine

Slides

Keywords

CRISPR; Gene Editing Therapies; Population Health

Key Points

• The epidemiologic forecast for 2050 is gloomy, with projected increases in the prevalence of diabetes, high cholesterol, hypertension, obesity, and resulting cardiovascular conditions. Innovative technologies and therapies – particularly gene editing – could potentially prevent these conditions and have a positive long-term impact.

• Gene editing technology could be used to develop targeted treatments for ultra-rare, rare, and common diseases. Several clinical trials are already underway, testing CRISPR-based therapies for cancers, chronic bacterial infections, lupus, and others.

• A little over a decade after the first discovery using CRISPR technology was published in Science, the FDA approved the first CRISPR-based medicine for the treatment of any disease. The recent, rapid, and revolutionary advances in gene editing therapies simultaneously hold promise and indicate the importance of ensuring that this technology is developed safely and effectively.

• When thinking through trial design, researchers need to consider durability. This means developing assessment methods for off-target effects and on-target safety validation, as well as planning for long-term monitoring.

• There are also ethical and regulatory points to consider, especially around obtaining informed consent, navigating the unknowns and potential permanence of gene editing interventions, engaging underserved communities, preventing misinformation, and defining long-term follow-up.

• The Precision Health Alliance was pulled together by Dr. Hernandez and his colleagues in an effort to bring together a variety of stakeholders – clinicians, researchers, healthcare systems, and life-science leaders – to think through what’s needed to develop research-to-care pathways with gene editing.

• Many questions still need to be addressed: How do we handle more and more personalized health data appropriately? If we know we can cure someone, should we? How do we ensure we don’t create greater health disparities? How do we overcome mistrust in revolutionary science? How do we address common questions, commonly?

 

Discussion Themes

– What’s your view on the current temperature of public perception in this space? And as you look to the future, how important do you think it’ll be to improve public understanding and acceptance of these types of therapies as a safe and effective strategy in clinical practice? There’s been some interesting early survey data in terms of, if you had an option for a permanent therapy that you took once, would you like to do it? And the answer was largely yes. Now if you were to say, well, we’re going to edit your genes, then the answer kind of changes. Some of the confusion comes from germline editing. So I think there’s a lot of education that has to be done in this area.

– As a person with a rare disease, I’ve been spending some time thinking about what I would do if I had this option, what the considerations are, and the ableism that all of these possibilities bring up. Contemplating and discussing with impacted persons is so critical. How would your initiative address that? It’s critically important to have lived experience involved, especially when you’re thinking about something that’s potentially durable over a lifetime. You want to make sure that you’re deriving meaningful benefit for patients that’s substantially beneficial rather than incremental. That’s also important for engaging the population.

 

 

Tags

#pctGR, @Collaboratory1