Scientists Don’t Ever Stop Sciencing

There are interesting things afoot in the cell and gene therapy (CGT) space. And not only what is tackled in our feature article on the risks and rewards of CGT trials, which is a hot take on the David vs. Goliath theme running through trial sponsorship for CGTs. With two recent FDA approvals for Bluebird Bio in back-to-back months, attention on CGTs and personalized medicine continues to grow.

At the recent DPHARM conference in Boston, former FDA Commissioner Scott Gottlieb, MD, told attendees during his keynote that he was writing a book on the history of CGTs. Weaving the tale of Carl Jung, the University of Pennsylvania, and Novartis, Gottlieb noted the serendipitous events that almost failed—but ultimately succeeded—in bringing Kymriah, the first CAR-T therapy approved by FDA, to market.

But, as Gottlieb noted, the biggest obstacle to CGT innovation right now is in the manufacturing—“the process is the product.” And, currently, FDA is not equipped to approve processes over products. Gottlieb explained that constructs currently exist within the agency to enable this change, but it needs a different framework. Ever the realist, Gottlieb foresees that this framework will be realized, albeit in an iterative process and may require an act of Congress to allow FDA to define the parameters for the process.

In our sister publication, Pharmaceutical Executive, this month, I wrote about the current state of research innovation hubs around the US. Cambridge/Boston, San Francisco, San Diego, Philadelphia all take top spots for leading-edge therapeutic innovations. Juxtapose this against the current financial market in biotech, a market correction is underway. It’s definitely not a chilling effect, but it is suggested that uncertainty around the manufacturing process and the regulatory implications of such is impacting investment due to an opaque regulatory model.

However, as we also know, and as stated in the main feature, scientists don’t stop researching and seeking out the best for patients. One notes: “As a scientist, when you are doing something in biomed research, [the] goal is to translate bench work to the clinic for [the] wellness of people.”

Lisa Henderson is Editor-in-Chief of Applied Clinical Trials. She can be reached at lhenderson@mjhlifesciences.com.