Francis Collins: We need better drugs -- now | Summary and Q&A

TL;DR

Dr. Francis Collins discusses the challenges and potential solutions in translating basic scientific knowledge into effective treatments for diseases.

Key Insights

• 📊 The average life expectancy has increased significantly over the past century, but there is still a need for more treatments for diseases.
• 💊 Only about 250 out of the 4,000 known diseases with molecular bases have available treatments. There is a gap between basic science discoveries and their application in treatment development.
• 🌉 Developing new treatments is a complex and challenging process, akin to navigating through unpredictable obstacles and setbacks.
• 🔬 Advances in technology, such as the human genome sequencing, have made it easier to study the molecular basis of diseases and develop targeted treatments.
• ⚙️ Repurposing existing drugs for new applications, based on molecular understanding, can be a valuable and cost-effective strategy for developing treatments for rare diseases.
• 🤝 Collaborations between academia, government, private sector, and patient organizations are necessary to improve the speed and success rate of drug development and translation.
• 💡 Innovative methods like testing drug safety and efficacy on human cells-on-a-chip offer potential for personalized drug development and testing.
• 💰 Developing new treatments requires adequate resources, support, and attracting talented individuals from diverse backgrounds to contribute to the field of biomedical research.

Transcript

Translator: Joseph Geni Reviewer: Morton Bast So let me ask for a show of hands. How many people here are over the age of 48? Well, there do seem to be a few. Well, congratulations, because if you look at this particular slide of U.S. life expectancy, you are now in excess of the average life span of somebody who was born in 1900. But look what hap... Read More

Questions & Answers

Q: What is the average life expectancy for someone born in 2010?

The average life expectancy for someone born in 2010 is 79 years old.

Q: How many diseases have a known molecular basis?

Approximately 4,000 diseases have a known molecular basis.

Q: How many of the 4,000 diseases with a known molecular basis have treatments available?

Only about 250 of the 4,000 diseases with a known molecular basis have treatments available.

Q: What is the average timeline and cost for developing a new drug?

The average timeline for developing a new drug is 14 years, and it can cost upwards of a billion dollars.

Q: How can we make the process of drug development faster and more successful?

One way to make the process of drug development faster and more successful is by repositioning or repurposing existing drugs that have known safety profiles but were not successful in their original intended use.

Q: How has the human genome sequencing technology impacted the field of disease research?

The cost of human genome sequencing has significantly decreased over the past decade, making it more accessible for disease research. This technology has allowed researchers to better understand genetic causes of diseases and develop targeted treatments.

Q: What is an example of a successful drug development for a rare disease?

One example of a successful drug development for a rare disease is the approval of a drug for cystic fibrosis. However, it is important to note that not all cases of cystic fibrosis can be treated with this drug, highlighting the need for continued research and development.

Q: How can we test the safety and effectiveness of drugs without putting patients at risk?

Researchers are exploring the use of human cell-based models, such as the "organ-on-a-chip" technology, to test the safety and effectiveness of drugs. These models mimic specific organs in the human body, providing a more accurate and ethical alternative to animal testing.

Summary & Key Takeaways

• Average life expectancy has increased significantly over the past century, with the current average life expectancy being 79 years for children born today.

• Despite significant advancements in molecular understanding of diseases, only about 250 out of the 4,000 known diseases have treatments available.

• There is a need to bridge the gap between basic science and application in order to develop more successful and faster treatments for diseases.