Cell therapy has come a long way in just over a decade. What began as an experimental, last-resort approach for a handful of patients has matured into a field with approved products, blockbuster revenues, and entirely new therapeutic frontiers.
Yet despite its progress and the growing excitement around next-generation technologies like in vivo CAR-T, cell therapy remains a story of patience, complexity, and relentless execution.
In a recent conversation, longtime cell therapy pioneer Pascal Touchon reflected on where the field stands today, why progress took longer than expected, and why the next chapter may be just as transformative as the first.
A Field Born From Urgent Patient Need
For many leaders in cell therapy, the motivation was deeply personal.
The early days of CAR-T therapy were defined by patients with no remaining options — children with refractory leukaemia, adults with aggressive lymphomas, and families facing impossible choices. In 2012, as the first CAR-T treatments emerged from academic centers like UPenn, the results were nothing short of astonishing.
Children resistant to chemotherapy were achieving complete remissions. Patients who had exhausted every line of treatment were surviving.
“Cell therapy was truly transformational, able to do something no other treatment could.”
That fundamental truth remains unchanged today.
From Scientific Breakthrough to Commercial Reality
While the science moved quickly, commercialization did not.
The first approved CAR-T therapies, such as Kymriah and later Yescarta and others, delivered unprecedented clinical outcomes. Over time, some became blockbuster products, particularly in lymphoma and multiple myeloma, where CAR-T has helped turn a fatal disease into a chronic one for many patients.
But the road to scale was far longer and harder than expected.
Why?
Because CAR-T was not just a new drug, it was an entirely new healthcare delivery model.
Why Early CAR-T Commercialization Was So Complex
Launching the first CAR-T therapies required solving challenges that traditional pharma had never faced before:
- Manufacturing and logistics across individualized treatments
- Supply chain coordination between hospitals and manufacturers
- Pricing and reimbursement for one-time, high-cost therapies
- Restricted patient access due to safety monitoring requirements
In the U.S., hospitals had to be formally certified before they could administer CAR-T therapies. Even world-renowned cancer centers required external validation to ensure they could safely deliver the treatment.
At the beginning, only a limited number of certified sites existed, meaning many patients had to travel hundreds of miles and remain near treatment centers for weeks.
These constraints slowed adoption, despite extraordinary clinical data.
Autologous vs Allogeneic: Progress Took Longer Than Expected
The early success of autologous CAR-T created enormous excitement and naturally sparked hope that allogeneic (off-the-shelf) cell therapies would follow quickly.
The logic made sense:
- Faster access
- Lower costs
- Scalable manufacturing
But biology proved stubborn.
Allogeneic cell therapy has advanced more slowly than many anticipated, particularly in oncology, where challenges around persistence, rejection, and durability remain significant. While progress continues, widespread commercial adoption is still ahead.
This pattern, rapid excitement followed by slower execution, has become a recurring theme in cell therapy.
A Surprising Breakthrough: CAR-T in Autoimmune Disease
One of the most exciting recent developments in cell therapy didn’t come from oncology at all.
Researchers in Germany and Asia explored a novel idea: using CD19 CAR-T therapy in autoimmune diseases. By fully depleting B cells, including the pathological clones driving disease, patients experienced something remarkable.
- Long-lasting symptom-free periods
- Drug-free remission for years in some cases
- A potential reset of the immune system
It was one of those moments in science that feels obvious in hindsight, yet had never been tested before.
This discovery has reignited excitement across the field and opened an entirely new therapeutic frontier.
The Rise of In Vivo CAR-T: The Next Wave?
Today, attention is rapidly shifting toward in vivo CAR-T, where the body is prompted to generate CAR-T cells internally rather than through ex vivo manipulation.
The level of industry interest is unmistakable:
- Deals involving hundreds of millions in upfront cash
- Total deal values reaching into the billions
- Participation from nearly every major pharma player
The promise is compelling, but so is the challenge.
Transforming early, small-scale patient data into a regulatory-approved, commercially viable therapy will take time.
Execution, clinical development, and regulatory alignment will determine whether in vivo CAR-T fulfils its potential.
Evolution, Not Replacement
Importantly, new technologies are not making earlier ones obsolete.
Autologous CAR-T, allogeneic approaches, bispecifics, and in vivo CAR-T are complementary, not competing, solutions. Each may serve different diseases, patient populations, and clinical settings.
A single platform will not define the future of cell therapy, but rather progress, by indication, built on rigorous data and careful execution.
Why Cell Therapy Still Matters
Despite the challenges, the conclusion is clear: cell therapy remains one of the most exciting and meaningful areas in medicine today.
It has already saved lives that would otherwise have been lost. It continues to redefine what’s possible in oncology and autoimmune disease. And while timelines have stretched, the long-term impact may be even greater than originally imagined.
The promise is still there, and the next chapter is only beginning.
