Clinical Trials of the future - Episode 2 The day when there will be no more clinical trials

May 12, 2024 (Initial release: Aug. 2, 2019)

"Sakura, in the future, you should study data science!"

The effects of the drug I was participating in when I was a high school student were better than I expected, and as the trial was nearing its end, Rock suddenly said to me as I was studying for the university entrance exam.

"Where did you learn such words?"
"A magazine from about 20 years ago that I accessed on the Internet earlier said that the sexiest job of the 21st century is data scientist."

Some time after that conversation, when the clinical trial ended, Rock returned to the robot collection company and we were separated. After graduating from high school, I entered the Faculty of Pharmacy at a university in the Kansai region, where I fell in love with bioinformatics and sometimes remembered the words that Rock had said to me.

"Even though he was a clinical trial assistant robot, he was even able to predict the future. That's amazing, Rock."

After that, I relied on my part-time job at university to get a job at a biotech venture. My motivation was that I wanted to repay the kindness I had received through the clinical trials I had participated in by developing a new drug. The company's significant investment in computational science was also a motivating factor. Above all, I was interested in the new technology developed by the research team led by the president.

Using this technology, developed about three years ago, it is possible to recreate copies of each person's genome in a virtual space called virtual twins, which have the same genetic sequence as each person's genome. By using these virtual twins, it is possible to calculate the expression of specific genes, the structure and characteristics of the proteins synthesized by them, and the effects on tissues and organs in the body, and it is possible to calculate the effects on the metabolism of drugs when they enter the body. It is now possible to perform calculations with various conditions set, such as simulating changes in blood concentration and blood concentration for each gene type. In theory, it has become possible to administer drugs to subjects in clinical trials and examine their effects on toxicity, metabolism, and disease, all on a computer.

In fact, in the fields of organic chemical synthesis and exploratory research, this kind of revolution driven by information technology has already occurred more than 30 years ago. We design compounds on a computer, calculate more than 10 synthetic routes and conditions, and calculate what structures the compounds have, what properties and activities they exhibit, how much toxicity they have, etc. Calculations using computers have become the norm. Around this time, in addition to the words in vitro to refer to reactions in a test tube and in vivo to refer to reactions in living organisms, the term in silico to refer to results calculated on a computer appeared in papers.

Until now, it has been extremely difficult to accurately calculate chemical changes in the human body because of the combination of various conditions, but with the development of Virtual Twins technology, clinical development has finally caught up with this situation.

In order to launch a drug to the market, various tests were conducted on healthy people and actual patients, and it took more than ten years to get approval. By collecting and analyzing the necessary amount of data calculated using the Virtual Twins and verifying it using iPS cells, it became possible to receive approval without conducting clinical trials on humans. As a result, work that previously took more than 10 years can now be completed in about 6 months. If iPS cells can be produced more efficiently and in large quantities in the future, data analysis could theoretically be completed in about three months. Moreover, since human bodies are not used, safety issues such as side effects occurring during clinical trials are completely eliminated.

Of course, it has become more important than ever to carefully track and manage side effects after release, but we also conducted a simulation of administering the drug in advance to Virtual Twins of patients who will receive the drug. By conducting tests using iPS cells created from patient cells, if necessary, it has become possible to predict not only the effectiveness of the drug but also the possible side effects. This is truly the realization of personalized medicine.

"Sakura-san, as a next step, please use synthetic data from 3000 cases to analyze whether there are differences in the metabolism of this drug depending on race."

Right now, before my eyes, the conventional wisdom that clinical development was based on collecting and aggregating human data was about to be overturned. In the photo frame on the desk, Rock turned towards me and seemed to smile a little.

"Rock, have I become a little more like a sexy data scientist?"