A Call to Arms: Transparency and Inclusion in Gene and Cell Therapy Development

The second day of the ASGCT Policy Summit continued to offer crucial diverse perspectives and considerations around gene and cell therapy development. One theme that kept coming through is that we as a field have a great opportunity to dramatically impact the health for many patients suffering with rare diseases and cancer. However, with that opportunity comes a great responsibility.

At the core of that responsibility, we must be inclusive and transparent about the potential of the therapy to address unmet medical need, and we also must be honest about the risks and unknowns.

Two sessions really hammered this concept home:

• Prioritizing Patients in Gene Therapy Development – Erin Ward (MTM-CNM Family Connection) and Pat Furlong (Parent Project Muscular Dystrophy) discussed the criticality of integrating the perspective of the patients and caregivers in drug development from the earliest stages.
• AAV Vector Integration – Graham Foster, PhD, FRCP (Queen Mary University of London) provided the perspective of the hemophilia patients’ historical experience of the UK blood product crisis where some patients receiving the products were unintendedly infected with hepatitis C and HIV.

At the core of these talks was a message to us all as drug developers to be thorough at acknowledging the risks and unknowns to the investigators, caregivers, and patients who participate in our clinical trials. This is often complicated by the fact that we are still learning about the potential risks of these novel therapies every day, and so sometimes the science, clinical knowledge, and regulatory considerations are out of step with each other. However, that doesn’t lessen our responsibility, rather it heightens it.

It is more important than ever that the field continues transparent data sharing and collaborative solution finding to meet the challenges we will continue to discover in the clinic. The speakers at these sessions offered many suggestions about how to do this, including:

• Active engagement of patient/caregiver advocacy groups in drafting informed consent documents to ensure not only thoroughness but also understanding.
• Rapid updating of informed consent documents to ensure the best, most current information about potential benefits and risks to a therapy.
• Public, collaborative natural history studies to look at baseline risks in untreated patients for emerging concerns about the therapy (e.g. work together to establish the baseline understanding of hepatocarcinoma in hemophilia for instance, that way we can better understand the risk of AAV therapy with time).
• Open discussions around inclusion/exclusion criteria regarding age restrictions, con-medication considerations, and cross-over designs.
• Inclusive discussion with patients, caregivers and providers around endpoints and definition of clinical benefits.

 I hope you were able to join these important sessions yesterday. And that you, like me, heard these recommendations as a call to arms to challenge ourselves every day to be open and inclusive about the work we are doing; both with others in the field, as well as with the patients we are trying to serve.

Read Dr. Walsh Martin's blog post about the first day of the Policy Summit here. Registered attendees can watch on-demand content on the virtual platform through Oct. 24.

Dr. Walsh Martin is a principal at Tremont Therapeutics currently on sabbatical and a member of the ASGCT Communications Committee.

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