At the Advanced Therapy Treatment Centre (ATTC) network’s 2025 annual event, ‘Shaping a stronger UK ecosystem for advanced therapy clinical trials’, delegates were reminded that one of the key measures of success for advanced therapy medicinal products (ATMPs) lies in patient experience.

In a fireside chat, Jacqueline Barry, Chief Clinical Officer at the Cell and Gene Therapy Catapult, spoke with Chris Kessler about his family’s journey when his son Charlie was diagnosed with spinal muscular atrophy (SMA) type 1 and went on to receive gene therapy.

Diagnosis and treatment

Chris described how his family’s world shifted when, at just seven weeks old, Charlie started to become weak and struggle with breathing. Receiving a devastating diagnosis of SMA type 1, a condition once considered fatal in infancy, Chris recalled:

“It was a huge shock… our world collapsed at that moment,”.

Yet, unlike in the past, there are various treatments available, including a gene therapy. In December 2024, Charlie received this gene therapy and the results were striking. Now 13 months old, Charlie is able to sit on his own and has begun to talk.

For Chris and his wife, the possibility of a single-dose treatment outweighed the alternative options of daily medication or repeated procedures. The therapy carried physical demands and the need for long-term steroids, but these challenges feel manageable when set against the potential benefits.

Navigating complex choices

Chris’ family experience demonstrates that the challenges of these medicines lie not only in their delivery, but also in the complex decisions patients and families need to make on treatment choices during what is a very difficult time in their lives.

Chris explained that what mattered most was not just the clinical information available, but also how it was shared: “The individual relationship we had with doctors and clinics, and how they presented [that information] to us,” was what gave them confidence to proceed with the opportunity in front of them.

Support from other families also played a crucial role. Speaking with parents whose children had already undergone treatment provided reassurance and practical advice. For Chris, these peer-led communities were “the most important thing.” He reflected on how simple digital tools such as apps, beyond informal group chats, could make these exchanges even more effective and accessible to patients and their families and/or carers.

Lessons for ATMP delivery

Charlie’s story highlights several important aspects of patient experience.

Timing is critical. In the US, most newborns are screened for SMA, enabling treatment before symptoms develop. “The data has shown children treated pre-symptomatically are developing normally,” Chris observed, reflecting on how different Charlie’s experience might have been with earlier intervention.

Cost and value also matter. While gene therapies carry a high price tag, Chris pointed out that supporting a symptomatic child brings significant ongoing costs to families and the health system. From his perspective, treating pre-symptomatically “could potentially be cheaper,” once long-term care needs are considered.

Putting patients and families at the heart of delivery

Charlie’s progress is a testament to what ATMPs can achieve. Yet his story is also a reminder that delivery must focus not only on clinical outcomes, but on the day-to-day experience of patients and their families.

From the clarity of clinical communication and the reassurance of peer networks, to the wider systems that determine access and support, the success of ATMPs will be measured by how they improve the lives of patients and their families.

The fireside chat with Chris was a great way to end another successful ATTC event – By hearing patient stories like Charlie’s, we’re reminded of the true purpose behind our work.

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