But right here in Newport Beach, Debra Miller was most likely beaming with pride and teaming with hope.
You see it was last May in this space, that I told you about Miller and the organization she founded, CureDuchenne — an organization born, quite literally, to save the life of her son, who has the disease.
She told me then about some of the researchers she is funding and how she didn’t believe it was going to take that many millions to put a serious dent in this disease.
And one of those researchers she funded, with a grant to the tune of $1.3 million five years ago, was Prosensa.
While $1.3 million may not seem like enough to cure a disease, the money came when Prosensa needed it most, filling a gap for the company at a crucial time and when it was in need of a human trial, Miller said.
“The most important thing about this is it is a whole new technology,” Miller told me. “By this being successful it really opens the door. It’s a huge breakthrough and we are really proud that we had the guts to go forward with it.”
I called Gerard Platenburg, the CEO of Prosensa, and asked him just how big a role this little Newport Beach-based non profit played in his company’s amazing breakthrough.
“They were kind of catalyst so to speak,” he said. “Obviously we have been working with several groups. But they have been instrumental for us to continue to grow. They were very motivating and very helpful in raising money. Most importantly we are now at the stage where we can test this therapy in trials.”
Miller said she is just happy she took the chance on Prosensa, which now has some $18 million worth of financing at its fingertips.
“So many of the academic researchers are just so focused on tinkering and perfecting things,” she said. “Prosensa kind of shook things up because someone went to trial with this drug.”
