Significant scientific advances in neurological diseases and disorders were reported at the recent 74th Annual Meeting of the American Academy of Neurology (AAN). What may be important for healthcare professionals is certainly encouraging news for patients awaiting effective solutions.
UCB is a research-driven company with a strong patient focus in neurology. “In everything we do, our guiding question is: what difference will it make for patients? says Charl van Zyl, UCB Executive Vice President Neurology and Head of European and International Markets.
The next consideration is usually how we help ensure that patients who receive treatment can access it. “Third, we aim to create the best possible experience for patients to help them navigate through the complexity of their condition and all of its consequences.”
Unmet needs in epilepsy
In epilepsy research, for example, many advances have been made in recent years, which have helped to improve the lives of many people living with the disease. Thanks to its legacy of more than 20 years in creating an understanding of this complex disease and the heterogeneous population it affects, UCB has gone from an initial focus on stopping symptoms to stopping all seizures.
“While approximately 70% of patients with epilepsy today are well controlled and largely able to enjoy a ‘normal’ quality of life, either without seizures or with a much lower frequency of seizures, we are working to understand the underlying causes of the symptoms of the remaining 30% who are still unresponsive to available therapies,” says Charl. Industry and academic researchers aim to further scientific understanding of the causes of problems such as drug resistance , as well as on rare forms of epilepsy where the pathways are better understood, allowing for more precise targeting and better individual outcomes.
To support the wide range of patients affected by this complex disease, who suffer from a wide range of symptoms, it is essential that research in this area continues. “At UCB, we try to understand the full range of triggers causing seizures, from genetic factors, from brain damage to stroke, cancer or other trauma,” says Charl.
“It’s really important that people have access to a wide range of therapies, from treating emergencies, to relieving symptoms like reducing the frequency of seizures, to treating the root cause of the disease. We are committed to continuing our efforts to be a leader in this space.
New developments in neuroinflammation and neurodegeneration
Beyond epilepsy, there are a range of other neurological disorders that are becoming better understood. Charl believes we are witnessing the “decade of the brain” with significant advances in scientific understanding of disease pathways and genetic correlations.
“Genomics has opened up entirely new insights into what exactly can cause certain neurological conditions. This advanced understanding gives me confidence in our ability to provide additional help and relief to those in need.
As part of these new possibilities, UCB is currently focusing on movement impediments due to neuromuscular conditions, with the aim of improving functionality and quality of life. Beyond epilepsy, there are two next areas of interest and rapid development: neuroinflammation and neurodegeneration.
Neuroinflammation looks at the neuromuscular junction with inflammation where different pathways are expressed in different diseases and can cause dysfunction. “This research has led us to rare diseases like myasthenia gravis, where patients typically suffer from fatigue and a combination of symptoms impacting their quality of life, ability to be active and function,” says Charl .
“We are currently investigating two different mechanisms to treat this disease, a complement inhibitor and an anti-FcRn, which essentially aim to reduce inflammation and create normal functionality for patients with MG.”
Another area of UCB’s focus is neurodegeneration; specifically Parkinson’s and Alzheimer’s disease. Additionally, particular emphasis is placed on exploring the potential of gene therapy to develop targeted treatments for these and other neurodegenerative conditions.
“Particularly in neurodegeneration, we need to develop an ability not only to treat the symptoms, but also to modify the disease and slow, stop or even reverse its progression,” Charl is convinced. “We need to diagnose and intervene much earlier and monitor patients for much longer periods of time than we do today. Much more research is needed, including large-scale clinical trials, which involves some investment and some risk.
The value of external science and strategic collaboration
To stem these demands, complement internal pipelines with promising external science, and create differentiated value for patients, many pharmaceutical companies are currently seeking strategic collaborations and acquisitions.
For UCB, this opportunity has presented itself with the recent acquisition of Zogenix – an acquisition that promises to enhance both UCB’s epilepsy pipeline and its strategic priorities in rare diseases.
“With the acquisition of Zogenix, we have added a new treatment to our portfolio to reduce seizures in two rare and specific forms of epilepsy: Dravet and Lennox-Gastaut syndrome,” says Charl. “This acquisition fits perfectly with our ‘value for the patient’ strategy of bringing highly differentiated solutions to patients. We are truly excited to use our capabilities to advance and optimize these new treatments and make them available to people in need, wherever they live.
UCB’s strategic collaborations with Roche and Novartis are another example of incorporating know-how, science and external resources. “Because it takes decades of clinical research and investment to hopefully defeat widespread diseases like Alzheimer’s disease and Parkinson’s disease, we decided to collaborate in this area,” notes Charl.
“Each company adds tremendous scientific expertise and, of course, we share resources and risks for this monumental piece of research and development.”
Over the next decade, UCB plans to continue its strong growth in both neurology and immunology. A steady stream of new launches is envisioned to offset the value of therapies that lose their exclusivity. Patients will benefit from new therapies, line extensions, combination therapies as well as digital health offerings.
The future of epilepsy and neurology
“As we look to the next decade, the most exciting scientific approach I see right now is the advancement of gene therapy and its potential to address disease pathways,” Charl looks to the future with optimism.
The promise of gene therapy has the potential, and certainly the ambition, to cure certain neurological diseases. Much scientific research is currently underway to establish the value and safety of gene therapy. “Its scientific hypothesis coupled with an ever-stronger understanding of relevant disease pathways can essentially modify a faulty genetic condition and, therefore, pave the way for potential cures.”
Another major trend, spurred by the pandemic, is a rapid increase in digital health technologies. Most patients and their doctors are much more closely linked than before the emergence of the coronavirus. Many have developed a preference for being treated in an online environment with convenient remote ‘visits’, often receiving more information, faster.
“The ability for patients to easily monitor in real time, be better informed and efficiently access healthcare through a digital environment is a major improvement. This will lead to better prevention, better compliance with prescribed diets, more disciplined participation in clinical trials, faster recruitment, less attrition, faster and more reliable results. I think we will see a lot of benefits from digital health technologies coupled with sound scientific knowledge over the next few years.
A third development that Charl anticipates is a further increase in disease-modifying treatments. “We are already seeing many scientists stepping up their efforts in the area of neurodegeneration. With the quantity and quality of research in this space, we could see breakthrough solutions begin to alter major conditions like Parkinson’s disease and Alzheimer’s disease much earlier than at typical clinical diagnosis points today. today.
“Which would be really fantastic for the many people living with these diseases; and even more important for future generations of patients who could be diagnosed and treated earlier with a better chance of modifying, managing and – one day – potentially curing some of their conditions,” concludes Charl.
About the interviewee
Charl van Zyl is Executive Vice President Neurology and Head of European and International Markets at UCB. Charl van Zyl manages the development and worldwide commercialization of innovative solutions that create value for people with neurological disorders, such as epilepsy and neurodegenerative diseases. He is also in charge of UCB’s corporate activities in Europe and on international markets. He is a member of the Executive Committee of UCB. He is also a board member of BIO (Biotechnology Innovation Organization).
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people with serious diseases of the immune system or central nervous system. With more than 8,000 people in around 40 countries, the company generated a turnover of 5.8 billion euros in 2021. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.