In addition to their far-reaching social impact, neurodegenerative diseases are rapidly becoming a significant global health concern. Currently, they trail only cancer as a leading cause of death, and are poised to become the leading cause of death within the next decade.
The impact of neurodegenerative disease is increasing because of the normal ageing process of human physiology, which makes the brain more vulnerable to them later in life. Globally, life expectancy is rising, meaning more people are living long enough to experience this ageing-associated deterioration.
However, that’s only one part of the problem. While a long lifespan is undoubtedly something to be admired, it unfortunately makes studying age-related diseases much more challenging.
Although animals, such as laboratory rats, are often useful models for human diseases, their application is limited. For one, they do not live as long as humans. A rat is lucky to reach three years of age, whereas humans routinely live past 70. Scientists believe that the processes that take place in the brain and its neurons are likely different if the lifespan is a few years compared to three score years and ten.
Although it is possible to model neurodegenerative diseases in rats, replicating the aging effects of a long human lifespan is a challenging task. Not to mention that humans and rats have evolved in different ways for the past 90 million years or so. In both species, the brain has developed unique adaptations for specific functions. Many of these adaptations are at the cellular level and are not shared with rats. While neurons in the rodent brain have evolved to function at high speed, human neurons are specialized to operate electrically with extremely high temporal precision, without fatigue, for extended periods.
This is one of the human-specific features of neurons, and it is believed that such long-term stability is crucial for our unique capacity to remain focused on intellectual tasks for lengthy periods at a stretch. Indeed, we know that we have the ability to concentrate on reading a book or answering exam questions for hours without interruption, if necessary or desired. This makes our brain and our neurons different from those of laboratory rodents, who usually stay focused on a task for no longer than tens of seconds.
These functional differences between species are generated by the slightly different molecular machinery of neurons in humans and rats. Importantly, these may also influence how diseases or drugs take effect. HCEMM’s Human Neuron Physiology and Therapy Core Group, led by Dr. Karri Lamsa, is working to solve this problem by removing rats from the equation.
Lamsa’s group works with live human brain tissue obtained from patients undergoing surgery, such as tumor removal. In such cases, minuscule amounts of healthy tissue are removed to reach target areas. Researchers use these samples to identify cellular characteristics unique to the human nervous system. Since neurons do not change much throughout our lives, these samples also have the advantage of being as old as their donor.
Lamsa’s group in Hungary and a second run by Dr. Vladimir Benes at HCEMM’s partner institute, the European Molecular Biology Laboratory (EMBL) in Heidelberg, use the latest technology to study gene activity in neurons removed from the brain during surgery.
At the EMBL GeneCore facility in Heidelberg, led by Benes, scientists can examine a cell’s interior with advanced techniques to study how its molecular machinery operates in real time. They observe how the machinery regulates gene expression as needed and, significantly, how this process differs from that of laboratory animals. This is important because the relationship between the molecular uniqueness of human neurons and their “human-specific” functions is not well understood.
Even at first glance, it is evident that human nerve cells have nearly twice as many active genes as rat nerve cells. Each gene contributes to the functional differences, such as electrical activity speed and stamina, in how rodent and human brains operate and could form the basis for a new drug. According to Lamsa and Benes, screening drug candidates in the early stages using genuine human brain tissue samples can substantially benefit companies developing therapies for age-related brain diseases.
Currently, drug development requires a significant investment of time and capital: 10-15 years and nearly USD 1 billion per drug that is finally approved. This is in no small part because drugs that seem effective in animals often do not work well in humans. Consequently, many seemingly good drug candidates developed in animal testing models fail the clinical test in humans.
Often, it remains unknown why a drug compound that works well in animals fails in human trials. Insights gathered using human brain tissue are thus twofold. First, they may enable scientists to better gauge the efficacy of drug candidates. Equally importantly, these insights may highlight drug targets and avenues of research that would have been overlooked using only animal models.
The Hungarian Center of Excellence for Molecular Medicine is a distributed institute whose scientists develop advanced diagnostics and treatment options supporting healthy aging. It works at the interface of academic and industrial research on topics related to translational medicine. The goal is to enhance the quality of life for an aging Hungarian population while simultaneously reducing the cost of healthcare provision through innovative applications in the field of molecular medicine. The HCEMM program is currently funded by an H2020 Teaming Grant (where Semmelweis University, the University of Szeged and the Hun-Ren Biological Research Center in Szeged cooperate with their advanced partner, the European Molecular Biology Laboratory, headquartered in Heidelberg, Germany) and a Thematic Excellence award, as well as a National Laboratory award from the Hungarian government. The various activities are coordinated by HCEMM Nonprofit Kft., headquartered in Szeged.
This article was first published in the Budapest Business Journal print issue of July 11, 2025.
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