Dr. David Fajgenbaum Found his "Cure" for Castleman Disease

From My Medical News Readings This Week

Dr. David Fajgenbaum found his "cure" for Castleman disease by meticulously analyzing his own medical records and existing research to identify an overactive pathway in his immune system, called the mTOR pathway. He then identified the drug sirolimus, an mTOR inhibitor typically used to prevent organ transplant rejection, and successfully persuaded his doctors to try it on him. This experimental treatment has kept him in remission from the life-threatening disease for over a decade. 

From a Life-Threatening Illness to a Personal Mission

• Near Death: 

  In 2010, as a third-year medical student, Dr. Fajgenbaum was diagnosed with Castleman disease, a rare and aggressive lymphoproliferative disorder that attacks the immune system. He was hospitalized in critical condition and nearly died five times. 
• Hope into Action: 

  Realizing conventional treatments were not working, he shifted his perspective from hoping to survive to actively seeking a solution, beginning a rigorous research process. 

The "Chasing My Cure" Approach

• Deep Dive into Data: 

  Fajgenbaum scoured his own extensive health records and delved into scientific literature to understand the underlying mechanisms of the disease. 
• Crowdsourcing Knowledge: 

  He connected with other Castleman disease patients and physicians, creating the Castleman Disease Collaborative Network (CDCN) to crowdsource promising research questions and share knowledge. 
• Drug Repurposing: 

  Through this methodical research, he identified a hyperactivated pathway in his immune system (mTOR) and found that sirolimus, a drug developed for organ transplant patients, could inhibit it. 

The Resulting "Cure" and Mission

• Successful Treatment: 

  After persuading his doctors to try the drug, Fajgenbaum experienced a dramatic reversal of his condition and has remained in remission for over a decade. 
• Inspiring a New Era of Research: 

  His success paved the way for further research and the establishment of his non-profit organization, Every Cure,which uses artificial intelligence to identify existing drugs for new uses to treat various diseases. 

Meanwhile, My Food For Thought for Today:

This is what I learned when I was promoted to the Position of Chemistry Team Leader in the Division Of Anti-Infective Drug Products, Office of New Drugs Chemistry, Food and Drug Administration in 1998.

When you are a leader, you are no longer responsible for doing the job; you are now responsible for inspiring, coaching, mentoring and motivating your team to become the very best version of themselves, which will help them produce their very best work. It’s all about people.

Lastly, 

A growing body of research suggests that added sugar may be more detrimental to heart health than cholesterol. A large-scale US study highlighted in JAMA revealed that individuals consuming 17-21% of their daily calories from added sugar had a 38% greater risk of dying from heart disease.

Unlike cholesterol, excess sugar contributes to heart problems by driving inflammation, raising blood pressure, and disrupting fat metabolism. To minimize this risk, health experts advise limiting your intake of added sugar to less than 10% of your total daily calories.