Revolutionising Healthcare: The Rise of Cell and Gene Therapies
Safety and clinical activity — Cell and Gene Therapies (CGTs) are transformative, offering potential cures with a single dose. Despite challenges in logistics, safety, and cost, their benefits, especially in CAR-T immunotherapy, outweigh the potential downsides. As innovation continues, CGTs are driving significant growth in the medical field.
A revolutionary approach within a complex landscape
— Cell and Gene Therapies (CGTs) are a class of drugs with immense potential. They are used to prevent and treat diseases and can go as far as curing them with just a single dose.
As promising as CGTs present themself, their successful administration faces challenges in multiple areas, such as logistics, communication, long-term safety, cost of manufacturing, and accessibility.
- Medical teams must coordinate smoothly across multiple contributors such as apheresis clinics, specialised shipping vendors, therapy manufacturers, chemotherapy teams, and post-treatment care providers.
- Decision-making involving the patient’s care requires greater reactivity, adaptability, and a higher level of input from external parties. From cell extraction to reinfusion, there are many steps, stakeholders, and external factors to be considered.
- CGTs are still considered to be a recent scientific advancement. Much remains unknown about their long-term effects on patients, such as the possible passing down of mutations to next generations, or the adverse effect on immune system response. This is why CGTs may take longer to research, approve, and adopt.
- These therapeutic agents are also expensive to manufacture and to administer, further limiting their accessibility.
Nonetheless, the medical community and clinical development industry acknowledges that the revolutionary benefits of CGT outweigh the risks. The market size and growth are clear evidence of this, worth $18.12 billion in 2023 and expected to reach $117.46 billion by 2034.
A closer look into CAR-T therapy
— One of the most clinically and commercially advanced embodiments of CGTs is Chimeric Antigen Receptor T (CAR-T) cellular immunotherapy. CAR-T therapy is a personalised cancer treatment, also known as precision medicine. This treatment utilises specific information about the patient and their tumour to provide the most suitable approach. By using the patient’s own immune system to fight the disease, fewer treatment cycles can be expected, potentially reducing harmful side effects and damage to healthy cells.
CAR-T therapy involves collecting the patient’s white blood cells (T cells) and then genetically engineering them in the laboratory to produce receptors (called Chimeric Antigen Receptors, or CAR) on their surface. The T cells used to create CAR-T cells can either come from the patient (autologous) or other sources (allogeneic). The generated CAR-T cells are then infused back into the patient where they will recognise and bind to cancer cells more efficiently.
— In the future, we can expect next-generation CAR-T therapies to encompass additional tumour types, particularly in solid tumours. Recent research has focused on expanding the approach to utilising donor T-cells or other immune cells, such as Natural Killer (NK) cells, which would in turn reduce the side effects of CAR-T products. With each advancement, we can expect to increase CAR-T therapy’s accessibility, reaching larger groups of patients and widening the range of diseases it can treat.
