Here is an interesting fact: you are made of roughly 37.2 trillion of cells (Ref) (yes, there is a paper about it on the web1). This means that you can be basically reduced to 37.2 trilllion functional autonomous parts that work very well in synchrony. This is beautiful! However, there is more we can apprehend from this like the fact that there are diseases that arise because some cells are either lacking or malfunctioning. This is an important information with potential interventional consequences. Think about diabetes type 1 for example: more than 20m people have type 1 diabetes in the world because somehow their body destroyed (auto immune disease) their insulin producing cells. All the rest 37,18 trillion cells are likely alright, but those 2-3 billion insulin-producing cells got cleared and this patient now has a lifelong problem. The same case could be made parkinsons, multiple sclerosis, heart failure, etc. There are many diseases that could potentially be curative if we could produce these cells.
And here comes science again: up until 2007 we didn’t have a technology to ethically produce unlimited amounts of human cells in the lab. Thanks to a Japanese group of scientists2 we now can cultivate an ethically source of cells that give rise to potentially any cell type in the body. This fundamentally changes our perspective on regenerative medicine.
After this breakthrough, there comes a time for more innovation. Researchers generally work with miniscule number of cells (think thousands to millions – this is almost invisible to the naked eye) in controlled environments to learn more about their biology. However, for regenerative applications to really take off, diseases like diabetes will need to dose hundreds of millions to billions of cells per patient. Now think with me for a second: imagine we can produce the right cell type for diabetes (Vertex is heading the pack and you can read more here) and you can prove beyond reasonable doubt the therapy works. How many of the 20 million patients will be able to get treatment? By following suit to what current successful cell therapies do today, you can estimate about 1000 patients a year. So, think about this: even if a curative therapy is developed and is successful, current manufacturing process could restrict patient access to less than 1% of patient population. That’s a huge problem, right? LizarBio Tx believes so and we are tackling it head on! We will dive deeper in future posts! Stick around!
Bianconi, E. et al. An estimation of the number of cells in the human body. Ann Hum Biol40, 463–471 (2013).
Takahashi, K. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell131, 861–72 (2007).