We the Scientists by Amy Dockser Marcus

Author:Amy Dockser Marcus [Dockser Marcus, Amy]
Language: eng
Format: epub
Publisher: Penguin Publishing Group
Published: 2023-02-14T00:00:00+00:00

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When it was the parents’ turn to address the panel, there was a change in the room. Panel members leaned forward, intent on hearing the parents’ words. There was an energy that seemed absent when the scientists argued with one another over the data. Dillon Papier, a child with NPC disease who was taking Zavesca, sat quietly curled in his mother’s lap for much of the morning’s testimony. Now, as one parent after another came forward to talk, he jumped up and started walking up and down the aisle, slowly, shakily, but on his own. It was hard to know if Dillon was bored or if he wanted to tell the panel members something. As he walked back and forth, the boy raised the baseball he held in his hand to the light, like a beacon.

When it was Phil’s turn, Andrea and the four children gathered around him. Don’t base Zavesca’s fate only on the results of the patients’ eye movements, Phil urged. Consider the totality of his children’s lives when measuring the effectiveness of the drug. “I don’t think that when you are talking about people’s health that you take eye movement as the measurement and end point and when that doesn’t work you throw away the other evidence,” Phil said.

Wasn’t it important that Andrew got a high score on the school quiz listing the capitals of every state? Didn’t the committee members notice that Dana, seated in a wheelchair, reached out to rub her younger brother Andrew’s arm while their father talked? Dana’s disease was advanced, but she remained actively engaged with her family. Phil considered such milestones the benefits of Zavesca.

“Zavesa hasn’t stopped the progression of the disease in Dana. It hasn’t stopped the progression in Andrew. But we are giving these kids more time. As a parent, you don’t care if it is an hour, a day, or a week. The longer, the better,” Phil said.

Before Phil or Andrea went to bed at night, they rotated Dana in her bed. Then they got up two hours later to turn Dana again. They did this a few times a night. They changed her position to prevent bedsores and because they felt it improved her breathing. “When we’re in there taking care of Dana, she knows her mom and dad are taking care of her. She wakes up a bit, opens her eyes. She gives you a big smile. She reaches out a hand to hold your hand,” Phil said. “Every extra day I get of that is a blessing.”

At the end of a long day of testimony, the advisory panel debated whether to recommend that the FDA regulators approve Zavesca.

They struggled with the data, which they said often seemed like a set of subjective observations rather than concrete measurements. Perhaps the stability in the swallowing of some patients was not because they took Zavesca, but because the disease progressed more slowly in those particular patients.

The parents’ observations about the difference Zavesca made in

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