The latest treatment for Parkinson’s disease was made possible by the actor’s audacious mission.
“It’s a lousy
1 disease to have,” said Michael J. Fox.
He was speaking, of course, about Parkinson’s disease, which the well-known actor, now 57, has had for nearly half his life. Like most Parkinson’s sufferers, Fox is afflicted with uncontrollable tremors and halting speech, among other symptoms. But he’s has never been one to complain about his illness, and he wasn’t doing so when I spoke to him the other day. He was simply trying to explain to me the importance of a new Parkinson’s medication that the U.S. Food and Drug Administration approved earlier this month.
The drug is called Inbrija, and while it doesn’t cure Parkinson’s, it’s a so-called rescue therapy that will greatly benefit Parkinson’s patients. I’ll explain how shortly. But first I want to note that Inbrija exists only because of the efforts of the Michael J. Fox Foundation, which Fox founded in 2000. What’s more, it’s the first drug to be approved for treatment in which the Fox Foundation played a significant role in both the approach and the funding.
I’ve been following the Michael J. Fox Foundation for about a decade. What initially drew me to the foundation’s work was its unusual model. Unlike most disease foundations, its emphasis was not patient advocacy. Rather, its goal was to find a cure for Parkinson’s, a disease that was woefully under-researched before Fox decided to start his foundation. The Michael J. Fox Foundation used its money to invigorate research around Parkinson’s, and to this day that’s where the bulk of its funding goes.
Fox’s first chief executive, Debi Brooks, came from Goldman Sachs, not the world of nonprofits. (The current CEO of the Fox Foundation is Todd Sherer, a scientist.) It does not have an endowment because Fox doesn’t want to hold onto money that might be better used searching for a cure.
Scientists who receive grants from the foundation have to agree to share their data with other Fox grantees — as well as with other Parkinson’s disease constituents, including the pharmaceutical industry. The foundation holds regular meetings where scientists talk about their work, with pharmaceutical representatives in the audience, asking questions.
Over time, the Foundation hired its own in-house scientists, rather than relying on a scientific board that met only occasionally, so it could make quicker funding decisions. Its scientists could also pull together promising ideas where others might not see any connection. And because the foundation had a deep knowledge of the Parkinson’s patient population, it could help scientists get the patients they needed for FDA trials. Brooks, who has never left the foundation — she is now its executive vice chairman — likes to say that one of its main roles is to “de-risk ideas,” giving industry and researchers incentive to pursue them.
Although there are a number of foundations that now take this approach, it was highly innovative when Fox started it. When I asked Fox and Brooks if they had this model in mind when they began in 2000, they laughed. “We started out at a more naïve and uninformed place,” said Brooks. “But as we started chipping away at the singular goal of speeding up better treatment to patients, this is how we came to see our role.”
In its 18 years, the foundation has raised — and spent — over $800 million. The scientists who have received Fox Foundation funding have made serious advances toward understanding what causes Parkinson’s. Once a research backwater, Parkinson’s research is now something scientists are eager to be involved in, thanks to the foundation. The Fox Foundation has become the fulcrum on which Parkinson’s research pivots.
Fox once thought, naïvely, that with the foundation’s help, scientists would find a cure within 10 years. Now he knows better. For every two steps forward, there has been a step back. This has been especially true in the area of drug development. There have been a number of drugs backed by the foundation that got as far as a Phase II FDA trial … only to be ultimately judged ineffective. Which is why Inbrija is such a milestone — and why, though it took nearly two decades, it offers a validation of the Michael J. Fox Foundation’s hands-on approach.
For starters, the biotech startup that developed Inbrija, Civitas Therapeutics, wasn’t even thinking about Parkinson’s when it began in 2009. According to the company’s former chief executive, Glenn Batchelder, it had come up with a technology that made it possible for certain drugs to be breathed in through an asthma-style inhaler. As the company considered possible uses for its innovation, Parkinson's came on its radar after Fox Foundation scientists explained that Parkinson's sufferers had times when their symptoms arose between their normal dosages. Being able to get quick relief via an inhaler -- the same way an asthma sufferer does -- could be could be a godsend. So that is where Civitas decided to focus.
Having helped plant the idea, the Fox Foundation backed it up with money. “We were a little company, with very little money,” said Batchelder. Fox seeded it, giving it $1.3 million between 2011 and 2013. As the company’s work progressed, it became part of the foundation’s “ecosystem,” as Batchelder calls it, getting ideas from the foundation, its scientists and others. The drug was so promising that in 2014, Civitas was acquired by Acorda, a larger biotech company, for $525 million. Batchelder wound up being so impressed with the Fox Foundation that he joined the board.
Still, helpful as it is, Inbrija doesn’t cure the disease — and that remains the goal. When I asked Fox whether he still thought Parkinson’s was a curable disease, he was adamant that it was. But his timeframe was, alas, more realistic. “It may not be there for me,” he acknowledged. “But I’m absolutely sure we are on a path to answer the questions” about what causes Parkinson’s and how to cure it.
Fox will always be best known for his “Back to the Future” films and his other acting roles — that’s only natural. But his most important legacy is the Fox Foundation, the model it created, and the hope it has given to people who have Parkinson’s disease.
This column does not necessarily reflect the opinion of the editorial board or Bloomberg LP and its owners.
To contact the author of this story:
Joe Nocera at jnocera3@bloomberg.net
To contact the editor responsible for this story:
Philip Gray at philipgray@bloomberg.net
https://www.bloomberg.com/opinion/articles/2019-01-23/parkinson-s-inhaler-the-michael-j-fox-foundation-gets-results
No comments:
Post a Comment