Lessons from the Frontlines of CGT Trials

“There's a big distinction between hope and expectation,” Jennifer Seidman said. “And if we ignore the emotional experience of families, we’re leaving out half the story."

Considering a gene therapy clinical trial means navigating big decisions that can shape a loved one’s future. During the closing session of ASGCT’s Empowering Patients Summit, advocates and parents Seidman and Megan Mack offered raw, insightful reflections on navigating personal values, goals of care, and the human side of cell and gene therapy (CGT) trials.

Both women entered the CGT space not as professionals, but as mothers. Seidman's son, Benjamin, and Mack’s children, Honor and Jude, were all diagnosed with Sanfilippo syndrome. What started as a search for answers became a relentless push to drive research, fundraise, and partner with scientists and companies to give their children, and future kids, an opportunity to get treated.

Watch the session 

From Advocates to Architects of Hope 

Seidman described families as both the drivers and passengers in this journey. Families often lead the charge in jumpstarting research for their loved one’s condition and gearing it toward clinical trial readiness. Yet once a therapy enters the formal pipeline, those same families may find themselves abruptly sidelined. Progress takes time and their children do not have decades to wait.

One of the session’s most powerful themes was the evolving nature of hope. When a child is diagnosed, hope may start with dreams of a cure. But over time, especially in the face of clinical trial limitations, eligibility barriers, or loss, it transforms. Mack described how, after her son Jude was disqualified from a clinical trial due to pre-existing antibodies, her expectations had to shift. “It was one of the most devastating days of our lives,” she shared. “But we had to find a new kind of hope.”

Hope can be an evolving thought. It can mean the hope that a child is free of pain, or can sit comfortably on the floor, or that their experience contributes to future breakthroughs.

Their session also tackled risk tolerance which is a key concern in CGT trials. Families living with rare diseases often face impossible choices and are forced to develop a unique capacity for risk assessment.

“The assumption is that we’ll do anything to save our child,” Seidman said. “But we also know how to say no when the risk doesn’t match the reward.”

The conversation underscored the need for ongoing education, emotional support, and clearer communication from industry, regulators, and care teams. Patient families need more than protocols and informed consent; they need to be fully equipped for the road ahead. Resources like the Courageous Parents Network’s clinical trial materials, developed with insights from advocates, help fill that gap.

In closing, both women reflected on what they’ve built not just for their own children, but for generations to come. Their stories show extraordinary resilience, “I couldn't face the thought of their lives being lived in vain,” Mack said. “But now, I can say things have progressed because of the work we’ve done.”

Their stories are a testament to the unique power of patient families, not just as participants in trials, but as partners, leaders, and teachers. This courage helps shape a system that can greatly benefit from the perspectives of those who live it.

Lexi Starosta is Manager of Advocacy Programs

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