Drug Development: Filling the Gap Between Experiments and Costly Trials


Finnish researcher Karri Lämsä, a group leader at HCEMM in Szeged.

At the Szeged-based Hungarian Center of Excellence for Molecular Medicine (HCEMM), a group headed by Finnish researcher Karri Lämsä is leading the way when it comes to providing human brain tissue samples for drug development.

HCEMM is an EU-supported Molecular Medicine Research Institute focusing on healthy aging, which collaborates closely with academic and industrial partners on a national and international level.

A Finn who led a research group at a University Pharmacological Department in Oxford for eight years before moving to Szeged (175 km southeast of Budapest), Lämsä has now lived and worked in the city for more than seven years.

Lämsä and his team offer a biomedical experimental service with fresh human brain tissue removed during brain surgery to startups and smaller-scale biomedical companies engaged in the costly business of testing drugs. They can now trial on human tissue samples in parallel with testing on what is called common experimental animal tissue, usually sourced from rodents.

Being able to test on human brain tissue enables researchers to find out earlier how the drugs they are developing work on humans.

The tissue is cultured from so-called resected brain tissue obtained from units carrying out neurosurgery. Resected brain tissue is removed during surgery because it is sick, malformed, damaged, or cut away by the surgeon to access the area of the brain on which they need to work.

The removed matter is typically sent to a tissue bank or discarded as biological waste. As Lämsä explains, “We’ve seen the value of cultured human brain tissue to benefit drug research and ultimately patient health.”

Saving Time and Money

Up until fairly recently, researchers were content to only use common experimental animal tissue, particularly from rodents, to test their drugs. This is, of course, generally far easier than accessing human brain tissue samples.

Testing using rodent tissue has given science invaluable insights into how the human brain works. But, as Lämsä says, “Human brain cells are not identical to rodent brain cells. There’s a 70-million-year evolutionary distance between rodent and human brains. Although these differences are typically quite small at the molecular level, together they become meaningful because there are thousands of different active molecules in a single brain cell, and the small differences add up.”

He continues, “When we get into extremely fine issues, such as how a drug works on a brain, there are clear differences that affect testing.”

So much so that, in the past, drugs that worked when tested on rodents have performed sub-optimally or even failed in clinical trials on humans.

Lämsä and his research team explore molecular, functional, and pharmaceutical features of human nerve cells, searching for properties unique to humans. Or at least not occurring in the animals commonly used for experimentation.

Many of these properties are related to brain development and plasticity, and the team believes some could be relevant to human brain degeneration processes and the effects of medication.

The service offered by Lämsä’s group at HCEMM enables companies to qualify the critical effects and results of their tests on rodents using actual human tissue before they start the costly clinical trials phase with human patients.

Clinical trials tend to be seriously expensive and time-consuming. If a drug doesn’t perform as expected or even fails in a clinical trial that happens at a late stage in its development, it could be a painful pill for a startup or small company to swallow.

As Lämsä says, “Our platform can allow them to do a final check with their product before they start the expensive clinical tests.”

Hungary in Top Research Category

Lämsä has worked in preclinical disease models and biomedical science for more than 20 years. During this time, he has seen “that drugs and therapeutic interventions developed using animals often just do not work as expected in the human body.”

He is not the only one to come to this realization. But, while skin tissue and blood are relatively easy to obtain, researchers all over the world wanting to use cultured human brain tissue have gotten tangled up in red tape and have often been defeated by bureaucracy.

But the differences between animal and human brains and the ways in which they work are becoming too large to ignore. Pressure to test on human brain tissue has been growing, Lämsä estimates, for at least the past 10 years.

Hungary had a head start, Lämsä suggests, because the country is particularly strong in the area of neuroanatomy, the anatomy of the nervous system.

“Neuroanatomy hinted at the substantial differences between rodent and human brain cells years ago, but now it’s becoming a real issue when it comes to drug testing.”

As a result, Hungarian research in this specific field is in the top category globally. While it can’t compete with prominent institutes in, for example, the United States or China, Hungary is able to focus on specific challenges offered by small teams associated with academic institutions and carry out high-quality research.

A particularly promising area for Lämsä and his team is connected to biological drugs and interventions in addition to classic pharmaceutical drugs.

Biological drugs are treatment strategies that aim to repair or heal tissue using biological mechanisms. For example, taking targeted gene expression regulation in human tissue for specific cells to correct how they function so they become normal and healthy.

Ultimately, by uncovering the hidden secrets of human neuron function and drug sensitivity, the work done by Lämsä and his team has the exciting potential to enable us to understand our brains better.

This knowledge could enable us to develop more powerfully effective cures against devastating brain diseases such as Alzheimer’s or Parkinson’s that affect millions of people.

Contact to learn more about the work of Karri Lämsä and his team.

This article was first published in the Budapest Business Journal print issue of November 7, 2022.

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