Summary

In this episode, Scott Schliebner discusses the unique challenges and complexities of rare disease trials. He highlights the difficulties in recruiting patients, the multidisciplinary nature of care, and the importance of collaboration among various stakeholders. The role of AI in rare disease trials is also explored, with a focus on its potential for patient recruitment and data analysis. 

Scott shares success stories in the field and discusses the expanding use of gene therapy to target multiple genes. The conversation concludes with a discussion on the recent FDA approval of Iovance's Amtogvy as the first T-cell therapy for a solid tumor, Melanoma. In this conversation, Scott discusses the potential of cell therapy in targeting solid tumors, particularly melanoma. He highlights the importance of expanding the use of cell therapy to other tumor types and the potential for curative treatments. The conversation also delves into the challenges and questions surrounding pricing and ethics in the gene and cell therapy space. The impact of curative therapies on quality of life and healthcare costs is explored, along with the success of curative therapies in hepatitis C. The high bar of clinical development and regulatory standards is discussed, as well as the potential for cost-effective treatments. The conversation concludes with advice for young professionals and established clinical professionals in the field of drug development.

Takeaways

• Cell therapy shows promise in targeting solid tumors, such as the breakthrough in melanoma treatment, and has the potential to provide curative treatments for various cancer types.

  
  



• The pricing and ethical considerations surrounding gene and cell therapy raise important questions about accessibility and affordability.

  
  



• Curative therapies have the potential to significantly improve quality of life and reduce healthcare costs by eliminating the need for long-term treatments.

  
  



• The success of curative therapies in hepatitis C demonstrates the potential for similar breakthroughs in other disease areas.

  
  



• Young professionals interested in a career in clinical drug development should seek opportunities to gain experience and consider starting in roles such as clinical research coordinators or clinical research associates.

  
  



• Rare Givers is a nonprofit organization that supports caregivers in the rare disease space.

  
  

Chapters

00:21 - Collaborative Approach in Rare Disease Trials

3:32 - The Role of AI in Rare Disease Trials

6:11 - Success Stories in Rare Disease Trials

11:34 - Expanding Gene Therapy to Target Multiple Genes

16:56 - New News: FDA Approval of Iovance's Amtagvi, The Breakthrough of IOVANCE for Melanoma

19:27 - Targeting Solid Tumors with Cell Therapy, Expanding the Potential of Cell Therapy

20:23 - The Impact of Curative Therapies on Quality of Life and Healthcare Costs

21:11 - Challenges and Questions around Pricing and Ethics

22:37 - Cures for Hepatitis C

23:25 - The High Bar of Clinical Development and Regulatory Standards

24:05 - Pricing Considerations for Different Cancer Types, The Potential for Cost-Effective Treatments

27:45 - Advice for Young Professionals in Clinical Drug Development

31:49 - Advice for Established Clinical Professionals

33:21 - Scott's Hobbies and Interests

Learn about and DONATE to Rare Givers:

https://www.raregivers.global/ 

How to reach us:

Scott Schliebner

https://www.linkedin.com/in/scott-schliebner-1087789/ 

Dr. Chad Briscoe

https://www.linkedin.com/in/chadbriscoe/ 

Gregory Austin

https://www.linkedin.com/in/gregoryaustin1/

New News Link:

https://www.fiercepharma.com/pharma/fda-approves-iovances-amtagvi-first-cell-therapy-solid-tumor 

Image Credits

Iovance, https://www.iovance.com/ 

FDA approved, https://www.tipranks.com/news/iovance-nasdaqiova-fda-approves-cell-therapy-stock-gains

Amtagvi, https://www.amtagvi.com/