The fight for gene therapy has been a long, uphill battle for the Batten community that has featured many losses and few victories.
It’s a battle that Suzette James is eager to help turn around. James, a BDSRA Board Member and Batten mother to Maya and Xavier, who are both affected by late-onset CLN2, will have another opportunity to help do just that.
She’ll be a featured panelist in the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) public virtual workshop on Thursday, April 13, from 11 a.m. to 3 p.m. ET. The workshop is titled, “Clinical Trials: The Patient Experience.”
This event will feature four panelists who’ll each give a 30-minute presentation followed by a Q&A session, according to James.
“I do have a different perspective and a different experience than probably the other three panelists in the interview, whereas we have not yet received gene therapy, but have been close, as have many others,” James said. “And so, I’ll talk about that process and that experience and the experience of having two different children at two very different points in the disease progression.”
The invitation was a pleasant surprise to James, who has previously participated in two FDA listening sessions, including one this past November where she helped prepare and present alongside three fellow CLN2 Batten mothers.
During her presentation in the first session covering gene therapy risks and benefits, battling through technical difficulties, James emphasized that the risk of not doing anything far greater exceeds the risk of doing something.
However, she wants others to know that doesn’t mean knowingly putting your children in danger.
“I think that I want to make sure that industry and the FDA know that parents are looking out for their children’s best interest and when we make a decision on any medical approach or treatment, we do that in a very methodical very thoughtful, and educated way,” James said. “We’re always relying upon our clinicians and our researchers, and the very work that the FDA is doing in their preclinical data collection.”
Suzette James (right) with her daughter, Maya (left), following a Brineura infusion.
Along with sharing Maya and Xavier’s stories, James aims to share experiences from other families.
In the meantime, James will consult with fellow Batten families, industry partners, and professionals in the Washington D.C. area who work closely with the FDA, as well as families who have experienced gene therapy trials in preparation for April 13th.
She currently faces a deadline at the end of the month to submit her presentation for approval.
“My question, first and foremost, for individuals that have been down that road and down that path (is) what information did you use to help inform you about making this decision?” James said. “When any of us tells a story, it’s not just our story. But I’m excited, I’m nervous, but I’m mostly excited.”
Overall, James believes the event can provide families with a guide on patient experiences and give them the tools to ask questions before making an educated decision on gene therapy.
And for part of that message and her presentation to be heard, James is going to need an assist from her electronics.
“I just really want all of my equipment to work on that day,” James said with a laugh. “I want the technology gods to be on my side on that day.”
