Programming Medicine | Summary and Q&A

TL;DR

Scientists are exploring the future of medicine by programming living things to act as medicines, revolutionizing the treatment of diseases.

Key Insights

• ⌛ Natural remedies discovered in nature have been the foundation of medicine since ancient times.
• ⚾ Chemistry-based medicines have greatly advanced the treatment and management of diseases.
• 💊 Biotechnology enabled the production of complex molecules like insulin, revolutionizing medicine.
• 💊 Programming living things to act as medicines through synthetic biology and gene and cell engineering is the future of medicine.
• 🧑‍⚕️ Current advancements include engineered cells fighting cancer, precise gene editing for genetic diseases, and reprogramming microbes for improved gut health.
• 🫒 Containment and termination methodologies need to be developed to ensure the safe usage of living medicines.
• 🥺 The ability to program living medicines will lead to increasingly effective and broad-spectrum treatments for various diseases.

Transcript

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Questions & Answers

Q: How did humans discover their first medicines?

Humans discovered their first medicines in nature, using natural remedies like willow bark for pain relief, which led to the discovery of aspirin. Additionally, accidents like leaving a petri dish on a lab bench led to the discovery of penicillin.

Q: What were some limitations of using chemistry to develop medicines?

Chemistry has its limitations, especially with complex molecules like insulin. Insulin sourced from pigs or cadavers was not ideal as it didn't match human insulin perfectly. However, the discovery of recombinant DNA and biotechnology allowed for the production of complex molecules like insulin.

Q: How are living things being programmed to act as medicines?

Living things like cells, genes, and microbes are being engineered to recognize and attack diseases. For example, cells can be engineered to activate the immune system to fight cancer. Gene therapy involves editing specific genes to treat diseases caused by single gene mutations. Microbes can be programmed to break down amino acids or release anti-inflammatory agents, benefiting conditions like PKU or Crohn's disease.

Q: What are the potential risks of using programmed living medicines?

One challenge is ensuring containment and termination. Scientists are working on designing sophisticated sensors, logic circuits, and kill switches to ensure living medicines only act when needed and terminate their function once the disease is alleviated. Controlling the behavior of these living medicines is crucial to prevent unintended consequences.

Summary

This video discusses the history and future of medicine, focusing on how humans have been able to make and develop medicines over time. It explores the discovery of medicines in nature, the use of chemistry to create novel molecules, and the advancements in biotechnology that allow for the programming of living things as medicines. The speaker highlights examples of using engineered cells, genes, and microbes to sense, execute programs, contain themselves, and terminate their functions for various diseases such as cancer, genetic disorders, and microbiome-related conditions. The video concludes by emphasizing the promising future of increasingly sophisticated and effective living medicines in finding cures.

Questions & Answers

Q: How were the first medicines discovered?

The first medicines were discovered in nature, with ancient Egyptians as early as 2500 years ago using the bark of the willow tree to treat pain. Nature is filled with examples of tools and tricks that it uses to ward off invasive species, providing a rich source of medicinal compounds. For example, the bark of the willow tree is the active ingredient in aspirin, and penicillin was discovered when a scientist accidentally left a petri dish on a lab bench.

Q: How did chemistry contribute to the development of medicines?

Chemistry played a crucial role in the development of medicines. Centuries ago, early apothecaries started developing novel molecules that do not exist in nature but have therapeutic effects. This led to the modern pharmaceutical industry that relies on chemistry-based medicines. Chemistry has allowed us to create countless medicines to treat and manage diseases, expanding our ability to heal and care for loved ones.

Q: What are the limitations of chemistry in medicine production?

While chemistry has been invaluable in medicine development, there are limitations to what can be done solely through chemistry. Some medicines, like insulin, are large and complex molecules that are difficult or impossible to synthesize using chemistry alone. Insulin was historically sourced from pigs or cadavers, which had drawbacks and potential negative effects on patients. Chemistry alone cannot produce certain medicines, and alternative approaches are needed.

Q: How did the discovery of recombinant DNA impact medicine production?

Almost forty years ago, scientists discovered recombinant DNA, a tool that allowed them to produce complex molecules like insulin. They introduced the human insulin gene into bacteria, programming them to produce insulin on our behalf. This breakthrough gave rise to the modern biotechnology industry. Recombinant DNA technology has enabled the production of medicines that were previously unattainable through chemistry alone, expanding our therapeutic options significantly.

Q: What is the next frontier in medicine regarding living things?

The next frontier in medicine involves programming living things to serve as medicines themselves. Instead of medicines acting on cells, genes, or microbes, advancements in synthetic biology and gene and cell engineering allow for the programming of these living entities to become the medicines. For example, cells can be engineered to recognize and kill cancer, genes can be modified to repair mutations, and microbes can be re-engineered to produce therapeutic molecules. This marks the beginning of the era of programming medicines.

Q: How are cells, genes, and microbes programmed to become medicines?

Programming cells, genes, and microbes as medicines requires two fundamental frameworks: understanding their mechanism of action and considering the specific biochemical processes inside the body. For cells and microbes, they need to sense the presence of disease, execute predetermined programs to combat it, be contained to the site of disease, and terminate their function when the disease state is alleviated. For genes, they need to be delivered using vehicles with low immune response and machinery to precisely edit the targeted genes. Scientists are already working on engineering sensors, logic circuits, and termination mechanisms to achieve these programming imperatives.

Q: What is CAR-T therapy, and how does it work?

CAR-T therapy is a cancer therapy that takes a patient's immune cells and engineers them to react to the patient's own tumor. These engineered cells, known as CAR-T cells, are reintroduced into the patient's body, where they circulate and fight cancer, recruiting the patient's immune system to aid in the process. CAR-T therapy has shown remarkable results in terms of remission rates and even cures. However, currently, this therapy is mostly applicable to leukemias, and more sophisticated methods are needed to extend its applicability to other types of cancer.

Q: How can cells be engineered to sense disease and execute programs?

Cells can be engineered with sophisticated sensors, such as receptors, allowing them to search for signals of disease throughout the body. By detecting these signals, cells can execute specific courses of action to combat the disease, such as recruiting the immune system or releasing anti-cancer drugs. Gene circuits can function as logic circuits, computing inputs and determining the best response, leading to the activation of the desired program. These advancements in cell engineering enable cells to become more effective therapeutic agents.

Q: What has been the hurdle in advancing gene therapy?

Gene therapy has faced a significant hurdle related to containment. In the past, gene therapy trials led to catastrophic immune responses and even deaths when the introduced genes spread throughout the body uncontrollably. This containment issue raised concerns and halted progress for approximately twenty years. Addressing this problem has been crucial to advance gene therapy and extend its use to a broader range of genetic diseases.

Q: How can the challenges of containment in gene therapy be addressed?

Containment in gene therapy can be addressed through the engineering of viruses used as vehicles for delivering the therapy. Scientists are working on redesigning and re-engineering viruses to have low immune response and target specific cell types. By improving the precision and specificity of viral delivery, the risk of gene therapy going to unintended areas in the body can be minimized. Another important aspect is the development of machinery, such as CRISPR, to accurately edit and repair genes with high precision, ensuring that only the targeted mutations are addressed.

Q: How is the microbiome being explored as a therapeutic strategy?

The microbiome, which plays a critical role in regulating health and is involved in various diseases, is being explored as a therapeutic strategy called "bugs as drugs." Microbes can be engineered to perform beneficial functions within the body. For example, an engineered microbe has been developed to break down an amino acid in a disease called phenylketonuria (PKU). By introducing this microbe into patients who cannot break down the amino acid themselves, toxic build-up is prevented. There is ongoing research to enhance the capabilities and potential of microbiome therapies.

Takeaways

The history and future of medicine have been shaped by the discovery of medicines in nature, the development of molecules through chemistry, and advancements in biotechnology. Programming living things to serve as medicines is an emerging frontier that offers immense potential. The ability to engineer cells, genes, and microbes to sense disease, execute programs, contain themselves, and terminate their function holds promise for treating a wide range of diseases, including cancer, genetic disorders, and conditions related to the microbiome. These living medicines can lead to increasingly sophisticated and effective therapies, ultimately leading us closer to finding cures.

Summary & Key Takeaways

• Throughout history, humans have relied on natural remedies discovered in nature, such as the use of willow bark for pain relief.

• Chemistry has played a crucial role in developing man-made medicines that are effective in treating and managing diseases.

• Biotechnology has allowed scientists to produce complex molecules like insulin, and now, advancements in synthetic biology and gene and cell engineering are paving the way for the programming of cells, genes, and microbes to serve as medicines.