Tiny brains, hearts and lungs can help cure disease
Mini-organs help researchers study many illnesses for which there are currently no treatments.
By Idun Haugan and Christina Benjaminsen – Published 17.10.2024
Many great discoveries and inventions stem from basic research, especially in medicine. Penicillin, for example, has saved millions of lives since Alexander Fleming discovered that Penicillium mould had a bactericidal effect.
Many other important drugs also originate from discoveries made through basic research.
Repairing cells
“Almost all medical treatment can be traced back to basic research,” says Magnar Bjørås, a professor of molecular biology at NTNU who has worked with basic research for three decades.
He also has a position at the University of Oslo/Oslo University Hospital.
Bjørås’s main line of research is investigating what happens inside our cells, especially cells that are diseased or damaged.
Once he and his research group have analysed what happens inside the diseased cells, they can begin their work to figure out how the cells can be repaired.
Among other things, the research group works to understand how to repair DNA damage caused by infections, heart failure, cancer and rare neurodegenerative disorders.
The latter are diseases that affect the brain and nerve cells, such as dementia, Parkinson’s and Alzheimer’s – and childhood dementia.
Bjørås has received funding from the Research Council of Norway to build expertise in molecular medicine. In recent years, his work has also been funded by donations from private sources who support the research.
Professor Magnar Bjørås and his research group are looking for new treatments for brain diseases. Many of the diseases that they are studying currently have no cure.
Photo: Geir Mogen
Major breakthrough in Japan
The brain is extremely complex. Finding treatments for brain diseases requires researchers to understand and analyse how the brain works.
Researchers made a major breakthrough nearly 20 years ago in basic stem cell research.
This allowed for entirely new approaches to understand the brain and to test treatments for brain diseases.
The Japanese researcher Shinya Yamanaka revolutionized stem cell research when he successfully reprogrammed a skin cell to become a stem cell. Yamanaka was awarded the Nobel Prize in Physiology or Medicine for his work.
A few years later, researchers managed to grow the first mini-organs from stem cells.
This is exactly what Magnar Bjørås’s research group is working on. They grow mini-organs in the laboratory: tiny brains, hearts, lungs and eyes that are smaller than a grain of rice.
The mini-organs are grown from skin cells of patients who are carriers of diseases for which the researchers are searching for therapies or medications.
Gene therapy
The mini-organs are fed with oxygen, carbohydrates, fats, minerals and amino acids – everything that cells and humans need to live.
Once the mini-organs have developed sufficiently, the researchers can test medicines and new therapies to investigate whether they can treat the diseases that the mini-organs carry. This research has produced promising results.
The medicines they are testing are combined in new ways, in an approach called combination therapy.
The mini-organs are also used for testing gene therapy. Gene therapy involves editing genes or introducing new genes into the patient’s body.
“Our most important job is to combine basic research with innovation. This involves making groundbreaking discoveries by understanding new aspects of diseases, and based on this, investigating which treatment therapies are possible,” Bjørås said.
Basic research
Basic research develops fundamental concepts, determines what can be considered reliable knowledge, and sets methodological standards. Basic research enables new insights into mechanisms that can be further developed and used by commercial players. Applied research focuses on practical goals and applications in industry and the business sector.
In Norway, basic research faces more challenges than in many other countries, and it is becoming increasingly difficult to obtain funding. Both basic research and applied research are important for society, and are closely intertwined.
Product development
Bjørås has just started a company called Zenit Science with researchers from NTNU, the University of Oslo and Oslo University Hospital in collaboration with Marigold Innovation, a Danish innovation company that focuses on medicine and life sciences.
The goal is to develop new tools and new treatments for complex diseases.
“The establishment of Zenit Science is an excellent example of the importance of basic research as a platform for product development towards the ultimate goal of better healthcare in the future,” says Toril Nagelhus Hernes, Pro-Rector for Innovation and Professor of Medical Technology at NTNU.
New medicines can be developed faster
Groundbreaking research is also being conducted on mini-organs at SINTEF. A technology called organ-on-a-chip can help develop new treatments for endometriosis. This disease causes severe pain in women and has previously been largely overlooked and insufficiently studied.
Developing new medicines is a time-consuming and expensive process. On average, it takes more than ten years to launch a new medicine on the market.
Much of the reason for this is that medicines are first tested on laboratory animals, but then are found not to be useable in humans.
What if there was a technology that could speed up drug development? Researchers around the world are currently working on this.
This approach is called organ-on-a-chip technology and consists of two technologies in one:
- Mini-organs: imitating the organs we have inside our bodies.
- Microchips: based on the same technology found in computers and mobile phones.
Reduces the need for animal research
“We can use this technology to study and imitate the foetal stages, imitate a menstrual cycle or study how cancer spreads, to name just a few applications” says researcher Frøydis Sved Skottvoll at SINTEF Digital.
In addition, this technology is expected to minimize the amount of animal testing, she says.
Frøydis Sved Skottvoll in the laboratory. Her work involves microsystems that make it possible to replace laboratory animals.
Photo: Yngve Vogt, University of Oslo
Channels as thin as a strand of hair
The research group that Skottvoll is part of is working on developing the technology around mini-organs. The mini-organs are placed in microchips called an organ-on-a-chip.
The microchips build a structure around the mini-organs which are nourished through hair-thin channels that transport fluid into the chips.
“We do this by using methods from the computer chip industry. We develop customized microchannels and sensors that make it possible to measure how the mini-organs are performing inside the microsystem,” Skottvoll says.
“The University of Oslo is helping us research this technology with the goal of creating a lab-on-a-chip. It is hoped it will make the process of testing new medications faster and more efficient than it is today,” she said.
Endometriosis
At KU Leuven University in Belgium, researchers have cultivated mini-organs that develop endometriosis. These mini-organs can now be used to test new medications and can produce safer and faster results than through animal testing, because the mini-organs originate from a female human body.
The technology will also enable treatment to be tailored to individual patients.
“They have discovered that endometriosis develops differently in different women, which suggests that the disease cannot be treated using a one-size-fits-all approach,” says Skottvoll.
The blood-brain barrier
The technology can also be used to simulate what is called the blood-brain barrier.
This is the body’s safety mechanism to protect the brain from harmful or unwanted substances in the blood. Generally speaking, it serves an important purpose, but it can also be an obstacle when we want to administer medications into the brain.
Historically, it has been very difficult to study this function in humans. However, organ-on-a-chip technology has now made this possible.
Photo – top four pictures: Idun Haugan/NTNU. Illustration of mini-brains: Marianne Gilbu/NTNU
