Susan Solomon: The promise of research with stem cells

Summary

The speaker discusses the transformative potential of stem cell research, highlighting its role in modeling diseases, understanding illness, and developing personalized medicine. They emphasize the need for new technologies and research models to bridge the gap between academic research and pharmaceutical development, ultimately aiming to revolutionize drug testing and treatment.

Takeaways

• 🌟 Embryonic stem cells are pluripotent, meaning they can develop into any cell type in the body, and are crucial for disease modeling and drug development.
• 🔬 Stem cell research is transforming our understanding of diseases and could lead to the development of more effective treatments and preventive measures.
• 💡 The New York Stem Cell Foundation Laboratory was established in 2005 to provide a safe haven for stem cell research, free from political and financial interference.
• 🤖 A new technology combining software and hardware has been developed to generate a diverse array of stem cell lines, essentially creating 'avatars' of humans for disease modeling and drug testing.
• 🧬 The potential of human genome sequencing is being realized through the use of stem cells, allowing for more personalized and effective clinical trials.
• 🧪 Induced pluripotent stem cells (iPS cells), derived from reprogrammed skin cells, offer a powerful tool for disease modeling and drug discovery, though they are not as versatile as embryonic stem cells.
• 🧬 A breakthrough in 2008 involved turning skin biopsies from ALS patients into motor neurons, providing the first model of a disease in living human cells.
• 💊 Current drug testing methods are inefficient and costly, with only 1% of tested drugs successfully making it to market, highlighting the need for better disease models and testing platforms.
• 👩‍🔬 The development of genetically diverse stem cell lines is essential for personalized medicine, allowing for more targeted and effective drug treatments based on individual genetic profiles.
• 🔎 The pharmaceutical industry needs to move away from a one-size-fits-all approach to drug development, considering the genetic diversity of the population to ensure safety and efficacy.
• 🏥 Automated robotic technology for stem cell production is set to revolutionize drug discovery, making the process more scalable, consistent, and personalized.

Q & A

What are embryonic stem cells and why are they significant for medical research?

Embryonic stem cells are pluripotent cells that have the potential to develop into any cell type in the human body. They are significant for medical research because they can be used to replace damaged or diseased cells and have the potential to revolutionize the understanding and treatment of various diseases.

How can stem cells be used to model diseases and contribute to drug development?

Stem cells can be used to create models of diseases by reprogramming them into the specific cell types affected by the disease. This allows researchers to study the disease process in a controlled environment, understand why cells become sick, and test potential drugs for effectiveness and safety.

What is the significance of induced pluripotent stem cells (iPS cells) in medical research?

iPS cells are a type of pluripotent stem cell derived from adult cells that have been reprogrammed to an embryonic-like state. They are significant because they can be generated from a patient's own cells, avoiding ethical issues associated with embryonic stem cells, and can be used for disease modeling and drug discovery.

What is the New York Stem Cell Foundation Laboratory and what was its purpose?

The New York Stem Cell Foundation Laboratory was established in 2005 to provide a private, safe haven for stem cell research. Its purpose was to advance stem cell research without political or financial interference and to bridge the gap between academic research and the development of drugs and treatments by the pharmaceutical industry.

What challenges did the speaker identify in the current system of drug development?

The speaker identified several challenges, including the long time frame (13 years on average), the high cost (4 billion dollars per drug), and the low success rate (only 1% of drugs make it to market). Additionally, current drug testing often relies on animal models, which may not accurately represent human diseases.

What is the concept of 'avatars of ourselves' in the context of stem cell research?

The concept of 'avatars of ourselves' refers to the use of genetically diverse stem cell lines that represent different individuals. These avatars can be used to conduct clinical trials in a dish with human cells, allowing for more personalized and effective drug development.

How does the speaker describe the potential impact of stem cell research on the future of disease treatment?

The speaker believes that stem cell research will lead to a future where major diseases like Alzheimer's and diabetes are viewed as preventable, similar to how polio is viewed today. This is due to the ability of stem cells to model diseases and contribute to the development of more effective and safer treatments.

What is the role of genetics in the development of personalized medicine using stem cells?

Genetics plays a crucial role in personalized medicine as it helps in understanding the individual differences in how diseases affect people and how they respond to treatments. By using stem cells from a genetically diverse array of individuals, researchers can develop drugs and treatments tailored to specific genetic profiles.

What was the issue with the drug Vioxx mentioned in the script, and how could stem cell technology have prevented it?

Vioxx was a drug that helped alleviate severe arthritis pain but caused severe and sometimes fatal heart side effects in some patients. Stem cell technology could have prevented this by allowing for testing on a genetically diverse array of cardiac cells, identifying which genetic profiles would be affected by the side effects and thus preventing the drug from being prescribed to those individuals.

What is the significance of the automated robotic technology developed by the New York Stem Cell Foundation Laboratory?

The automated robotic technology is significant because it can produce thousands of genetically diverse stem cell lines, which can be used to test drugs and treatments on a wide range of genetic profiles. This technology has the potential to revolutionize drug development by making it more efficient, cost-effective, and personalized.

How does the speaker view the current state of personalized medicine in relation to stem cell research?

The speaker views the current state of personalized medicine as being on the threshold of a new era, thanks to stem cell research. The ability to create genetically diverse stem cell lines and test drugs on them allows for the development of treatments that are tailored to individual genetic profiles, moving away from a one-size-fits-all approach.