- CAR-T therapy can cure terminally ill cancer patients but it is prohibitively expensive, costing hundreds of thousands of dollars.
- Leveraging recent advances in chip technology scientists can create and mass produce small machines to re-engineer the immune system.
- But the scientific community needs public-private partnerships to ensure this medical breakthrough becomes accessible to everyone.
After suffering 16 months of chemotherapy for her leukaemia, treatment options for six-year-old Emily Whitehead had run out. Her parents began to fear the worst. As a last-ditch effort, the University of Pennsylvania enrolled Emily in a clinical trial that involved reprogramming her immune cells to destroy her cancer. The results were phenomenal. Emily not only survived, but nine years later she is a healthy teenager with no cancer.
Behind Emily’s recovery is CAR-T (Chimeric Antigen Receptor T cells ) a cell-based therapy that has become a revolutionary weapon in the treatment of previously incurable blood cancers.
CAR-T cell therapy genetically modifies a patient’s immune cells to hunt and kill cancer cells. It is a form of personalized immunotherapy that can provide lasting remissions, even to terminally ill patients who have just months to live and for whom classic treatment options have not worked.
More than 400 clinical trials of CAR-T therapies are currently in progress. Their impact could be enormous. According to the World Health Organization, cancer causes one in six deaths worldwide. Personalized cell therapy has the potential to save millions of lives. Preliminary data even suggests that engineering immune cells may one day be used to treat heart failure, autoimmune diseases, diabetes and HIV.
Why does CAR-T cost so much?
But unit economics are hobbling the rollout of CAR-T to the full number of patients whose lives it could save. The treatment alone can cost up to $475,000 and US hospitals can charge as much as $1.5 million to administer it, once ancillary costs are taken into account.
So why this high price? With conventional therapies, drug makers get economies of scale: the more they produce, the cheaper each dose becomes.
But CAR-T is tailor-made for each patient, and behind every treatment lies a highly sophisticated process, which is time-consuming and brutally expensive.
The patient’s immune cells are collected, purified in various steps, genetically modified, formulated at the right dose and reinfused. This complex manufacturing process requires shipments to different labs and frequent manual interventions, which introduce the risk of human error and potentially life-threatening side effects. Compounded by the fact that CAR-T consists of living cells that vary in potency, manufacturers need to continuously test results throughout the process.
The result is a production time that can take weeks, and an unaffordable price. Unless these economics change, this treatment will not reach patients whose lives it could save – it will only reach those privileged enough to afford it.
Technology holds the key to reducing costs
There is, however, hope. The most recent insights in nanotechnology, artificial intelligence (AI), biosensors, and the Internet of Things could help overcome the current roadblocks in making personalized cell therapies affordable.
The solution to democratize these therapies lies in automating their manufacturing process, which would reduce the cost, time, and risks significantly. This will require several engineering breakthroughs but is technically possible.
Recent advances in chip technology provide inspiration. The modern world’s insatiable demand for better computers, gaming consoles, and smartphones has resulted in the extreme miniaturization of transistors – the components which drive technology’s processing capacity – as more transistors on smaller circuits enables new and stronger technological abilities.
Enormous capital spending has gone into chip engineering, resulting in the development of materials and systems on scales so small a new window of opportunities opens up: the ability to screen, select, and even genetically modify cells.
If we leverage these techniques to advance medical research, modern technology holds the potential to create and mass produce small machines that are able to re-engineer the immune system. The result would be a true revolution in the treatment of diseases, unparalleled in effectiveness and safety.
What can governments do?
Speaking on behalf of the research community, we see four ways governments can accelerate progress:
- Invest in infrastructure that supports joint technology and clinical R&D.
- Allow us to work together more easily.
- Harmonize regulations.
- Provide longer-term research grants.
Various small scale infrastructure investments have already been made. By aligning strategies and pooling resources, governments can advance research dramatically.
The conventional approach to develop drug candidates that split discovery, clinical trials and manufacturing will not work as pharmaceutical companies are faced with the novel challenge of re-engineering living cells, which requires new and highly advanced manufacturing processes.
Programmes will therefore need to be designed in which both knowledge and infrastructure are shared simultaneously, allowing medical researchers to use technology they would otherwise not be able to access, while letting engineers further develop it.
Building the right ecosystems in which the necessary skill sets are combined will be crucial for these therapies’ overall success. Interdisciplinary collaboration between life scientists and experts in AI and nanoelectronics will be needed, as well as partnerships between pharmaceutical and technology companies.
Legislators should update and harmonize policies when possible to ensure restrictive regulations do not stall technological progress and consider reforming science funding. Even top academics can spend as much as half their time writing out applications for short-term grants. It is wasted time they are not spending in the lab.
Both US President Joe Biden and EU Commission President Ursula von der Leyen have pledged to rid the world of cancer. But any plan that does not include a detailed roadmap on how to make CAR-T therapy affordable is bound to fail.
Patient-derived cell therapies have the potential of saving lives when conventional approaches fail. However, with a price tag of hundreds of thousands of dollars for a single treatment, these therapies will remain far from accessible. The world is counting on its leaders to build the public-private partnerships that will ensure this significant medical breakthrough becomes accessible to everyone who needs it.
This piece is republished from WeForum.