The development of such technologies has the potential to completely transform the way that we view health care, offering patients the ability to recover from illnesses that have until now been considered incurable — such as neurodegenerative diseases, which can progressively damage organs and tissues over a long period of time. According to a report published in The Lancet in 20181, “neurological disorders are now the leading source of disability globally, and ageing is increasing the burden of neurodegenerative disorders.” One of these disorders is Parkinson’s disease, which is estimated to affect more than 10 million people worldwide — more than one million of whom are in Europe.2
When we talk about CGTs, we refer to therapies that involve transferring genetic or cellular material into the body of a patient to restore biological functionality to an organ or tissue that has been impaired by disease. Until now, available treatments tended to focus on addressing the symptoms of such diseases, rather than targeting the cause, meaning that they focused on disease management for individual patients rather than curing them of the disease altogether. In the case of Parkinson’s, this means treatments to restore decreased levels of dopamine caused by nerve cell damage in the brain, the depletion of which can diminish processes such as memory and movement. Popular symptomatic treatments such as dopamine substitutes (like levodopa) are commonly used to mitigate the symptoms of the disease, which include dyskinesias (involuntary movements), as well as rigidity and cramping. Other treatments include deep brain stimulation or focused ultrasound. However, a huge drawback of these types of medical treatments is that their effectiveness reduces as the disease progresses, and there is currently no disease-modifying treatment available.
Transforming lives through science
This is where biomedical research companies like Bayer come in. By developing technology that targets the disease at its root cause, the CGTs they are developing aim to go beyond symptomatic treatments. This means that for the first time in history, it may be possible to stop and reverse degenerative diseases like Parkinson’s. It’s exciting — using authentic dopaminergic neurons, Bayer is working alongside one of its subsidiaries, BlueRock, to reinnervate the affected regions of the human brain and reverse the degenerative process, potentially restoring motor function. For those who suffer with this disease or those close to them, even the potential to alleviate its damage will come as a relief.
An opportunity to prosper
The benefits of being able to potentially reverse the degenerative process on the lives of patients, as well as the broader population, would be far-reaching. Not only would it mean an end to the suffering that has blighted far too many lives, but on a societal level it would reduce the burden on the economy of these types of disorders — the annual European cost of Parkinson’s disease is estimated at €13.9 billion1 — and the financial pressure and indirect costs they so often place on the families of patients, who may need extra support to help them cope or who may feel the need to reduce working hours.
Moving toward advanced treatments for Parkinson’s would also have a positive impact on overall health systems, by reducing the need for intensive medical intervention in those who have received treatment, while CGT research is likely to create new jobs and boost economic growth across Europe. The potential of advanced CGTs is recognized by the recent Pharmaceutical Strategy for Europe and the European Commission’s study regarding the status of new genomic techniques under EU law.
However, there is a need to improve certain framework conditions that are impeding progress in CGTs, such as delays to clinical trials that are caused by the need to comply with genetically-modified organism (GMO) requirements in certain cases, which is complex as rules vary across member states. This makes the EU less attractive than other regions for conducting clinical trials with gene therapies and also delays access to transformative medicines. Recently developed COVID-19 treatments and vaccines could only be developed so quickly as they were exempted from some provisions of the GMO requirements, which is a clear recognition of the complexities at hand.
In other areas of the world, key regulatory differences mean that many more clinical trials are already under way for CGTs. When you consider in the 2014-18 period, growth in trials for advanced therapy medical treatments (ATMPs) was up by 36 percent in the U.S., by 28 percent in Asia and just 2 percent in Europe, it’s clear the latter has some catching up to do. In China, for instance, the investment climate has been primed to encourage further development in this field, and a national data bank on DNA has been created to support research. This is contributing to pressure in Europe to prioritize policies that support progress in CGTs, alongside the implementation of stronger cooperation in the field of advanced therapies included in the framework of the EU and the U.S.’s Transatlantic Agenda. In addition to simplifying and harmonizing rules for clinical trials, further priority areas for policy should include an improvement of venture capital access in the Continent, as well as the development of an innovative regulatory framework for the use of health-related data, while upholding the high data protection standards in Europe. The set-up of an Important Project of Common European Interest (IPCEI) in the area of biotechnology could further support investment and cross-border cooperation in the area of CGT.
The technology has great potential
The opportunities unlocked by developments in treatments for Parkinson’s disease should not be underestimated; as with other technological advances, progress in one area often means progress in many. For instance, new innovative mRNA technology expedited the development of effective COVID-19 vaccines. In the field of CGTs, there is potential for advanced therapies to help create new cancer treatments, or to move forward with efforts with applying these technologies in cardiovascular diseases.
When policy is aligned with the goals of biomedical innovation, the outlook is promising. A future in which certain degenerative diseases are no longer seen as incurable is now a realistic goal. What is clear is that there is much to gain if businesses and policymakers are able to work effectively together to create a framework that is able to support the pace of innovation.
1 The Lancet Neurology. (2018). Global, regional, and national burden of Parkinson’s disease,1990–2016: a systematic analysis for the Global Burden of Disease Study. Available at: https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(18)30295-3/fulltext
2 EPDA. (N/A). The European Parkinson’s disease Standards of Care Consensus Statement. Available at: https://www.epda.eu.com/media/1181/epda-consensus-statement-en.pdf
3 COI of Brain disorders. (2011). Cost of disorders of the brain in Europe 2010. Available at: https://www.researchgate.net/publication/51649021_Cost_of_disorders_of_the_brain_in_Europe_2010
4 Alliance for Regenerative Medicine. (2019). Q1 2019 Data Report. Available at: https://alliancerm.org/publication/q1-2019-data-report/
5 Alliance for Regenerative Medicine. (2020). 2020: Growth & Resilience in Regenerative Medicine. Available at: https://alliancerm.org/sector-report/2020-annual-report/
6 Nature reviews Neuroscience. (2020). The future of stem cell therapies for Parkinson disease. Available at: https://www.researchgate.net/publication/338413735_The_future_of_stem_cell_therapies_for_Parkinson_disease