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Gene Therapy: The Future of Medicine?

14th June, 2024

Gene therapy is emerging as a revolutionary approach to treating a wide range of diseases, such as spinal muscular atrophy (SMA), cystic fibrosis, and haemophilia, offering the possibility of a one-time treatment with lifelong effects. The recent panel discussion at Cheltenham Science Festival, “Gene Therapy: The Future of Medicine?”, sponsored by the Innovation Hubs for Gene Therapies and Cell & Gene Therapy Catapult, brought together experts Professor Bobby Gaspar, Dr Rajvinder Karda, Dharmisha Stezaly, and Vivienne Parry to explore the current state and future potential of gene therapy. This article summarises the key messages from the event, highlighting the promise of gene therapy and the challenges that must be overcome to make it widely available.

Gene therapy as a one-dose treatment

Unlike traditional medicines that require regular, lifelong administration, gene therapy has the potential to provide a permanent cure for individuals, including children, by addressing the underlying genetic causes of diseases. This marks a significant shift in the advancement of medical treatments, offering hope for those affected by genetic disorders and providing the possibility of a cure rather than mere symptom management.

Gene therapy is a versatile and multifaceted approach to treating genetic disorders, offering a wide range of tools and techniques tailored to specific indications. Rather than being a one-size-fits-all solution, gene therapy can be thought of as a toolbox, where the appropriate tool must be selected based on the unique requirements and specifications of each disease. The choice of gene therapy depends on several key factors, such as the type of cells that need to be repaired, the desired method of cell repair, and the available tools to achieve the expected outcome.

One of the main challenges in gene therapy is delivering the therapeutic gene to the correct cells. Dr Rajvinder Karda introduced the idea of using an parcel delivery analogy to better explain this process, where the viral vector is the driver, the DNA sequence is the package, and the target cell is the delivery address. Designing a gene therapy that reaches the intended destination and performs its desired function is a complex and time-consuming process, requiring extensive collaboration among scientists worldwide.

What are the obstacles associated with availability of gene therapy for patients?

Gene therapy has significant cost implications due to its unique nature as a potentially curative treatment administered in a single dose. Unlike conventional medicines that often require life-long administration, gene therapies can provide benefits lasting for over decades after a single treatment. This development poses a challenge to the current healthcare system, which is used to paying for medicines on an ongoing basis. Pricing a gene therapy is complex, as its benefits can extend over the patient’s lifetime, potentially spanning 20 to 40 years. Governments, operating on shorter timeframes, must adopt a long-term perspective to ensure patient access to these life-changing treatments.

Professor Bobby Gaspar noted that independent bodies like The National Institute for Health and Care Excellence (NICE) in the UK assess the value of medicines based on factors such as available alternative treatments, life-years gained, and quality of life improvements. These assessments help determine the appropriate pricing for gene therapies, considering the significant benefits. However, as Vivienne Parry pointed out, the high cost of these treatments raises ethical questions about the allocation of limited healthcare resources and the potential diversion of funds from other essential services that benefit a larger population.

Gene therapies offer a paradigm shift in the treatment of rare genetic diseases, calling for a re-evaluation of how we value and fund medical treatments.

Importance of early screening

Early screening plays a crucial role in the effective implementation of gene therapies, allowing for the identification of genetic disorders at the earliest stage, often before the symptoms develop. This is especially important for conditions where early medical intervention can significantly improve a patient’s well-being and potentially provide a curative treatment.

Vivienne Parry mentioned the benefits of the upcoming Generation study, funded by Genomics England, which aims to screen approximately 100,000 children for 230 genetic diseases. It is a significant step towards understanding the applicability and the benefits of integrating early genetic screening into standard care for children. However, the speakers discussed the ethical implications of screening for genetic disorders, especially in cases where no effective treatment is currently available.

Professor Bobby Gaspar explained the importance of the timing of delivering gene therapy, particularly for neurological conditions. Early intervention is essential to prevent irreversible damage and achieve the best possible outcomes. Once symptoms develop and brain cells begin to die, it becomes particularly challenging to reverse the damage caused by the disease. Therefore, the primary goal is to halt the deterioration of brain function as early as possible.

Dharmisha Stezaly’s personal experience with her son Sebastian, who was diagnosed with Spinal Muscular Atrophy (SMA) at 13 weeks old, emphasised the critical role of early screening and the need for improved access to diagnostic services. Her story also highlights the long-term impact of gene therapy, as she mentioned that they still see the benefits of the treatment in their child’s health, even three years after receiving it.

What is next in the field of gene therapy?

The future of gene therapy is promising, with experts predicting a substantial increase in approved treatments in the coming years. While gene therapies are currently focused on rare genetic diseases, the knowledge gained from the research to-date may be applied to more common conditions, like Alzheimer’s, inflammatory bowel disease, and certain types of cancer.

Artificial intelligence is expected to play a crucial role in advancing gene therapy by diagnosing genetic diseases more efficiently and accurately. Professor Bobby Gaspar mentioned that AI and machine learning technologies can effectively analyse large datasets and identify patterns, enabling quicker and more accurate diagnoses. This is particularly important for gene therapies, as early diagnosis is crucial for achieving the best treatment results.

As the field of gene therapy continues to evolve, it has the potential to revolutionise the treatment of not only rare genetic disorders, but also a wide range of more common diseases.


Author: Mariya Spatar, LifeArc Communications Strategist

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