Searching for Profits Stymies Gene Therapies: A California Perspective on an International Problem
"Miracle medicine," untenable marketplace issues, total failure?
“Jakob is 2 years old. He has a deadly disease. But drug companies won't give him the cure — because they won't make a big enough profit.”
That was the headline yesterday on a matter involving a $43 million investment by California taxpayers, a London-based company, the University of California, the Golden State’s stem cell agency and a rare genetic immune deficiency often referred to as the “bubble baby” disease.
The article, written by Andy Dunn, appeared on Business Insider, a business and financial news website that reaches millions of readers internationally each month. It is rare that news involving the stem cell agency commands an audience of that size. The irony, if it can be called that, is that the California Institute for Regenerative Medicine (CIRM), as the agency is known, is not mentioned once.
The article dealt with the broader issues involving therapies for ADA-SCID and other rare diseases. Dunn wrote:
“A cure for Jakob's disease exists. It's an innovative form of gene therapy that restores the damaged immune system. It's miracle medicine, a proven wonder. But Jakob's family can't access it. Not because they can't afford it. Not because it's awaiting government approval. But because the drug companies that controlled the cure didn't think they could profit from it.
“Because Big Pharma is unconvinced of the profit potential, they're abandoning gene therapies — not just for SCID but for scores of other rare diseases - even when they know the cures work. As a result, kids like Jakob will needlessly suffer and die, denied access to a permanent fix for their illnesses. What's keeping Jakob isolated from the rest of the world, unable to play with a dog or make a friend, isn't his disease. It's the marketplace.
"‘SCID is the canary in the coal mine for rare diseases in general,’ says Matthew Porteus, a genomics researcher at Stanford University. ‘The thought that a curative therapy could be developed, get all the way through trials, and then not be delivered to patients just seems untenable to me. We've totally failed if we can't figure out how to solve this market problem.’"
Business Insider continued,
“This year's meltdown in biotech stocks has only exacerbated the fear that promising gene therapies for other rare diseases will also be stranded. Ethan Perlstein, the CEO of Perlara, a biotech working on treatments for ultrarare diseases, calls it ‘the Great Abandonment.’ In the past few months alone, gene therapies for Wiskott-Aldrich syndrome, MPS, Batten disease, phenylketonuria, chronic granulomatous disease, Rett syndrome, and Fabry disease have all been deprioritized or shelved. Biotechs and their investors just don't believe the drugs can make enough money.”
That said, California’s $12 billion stem cell agency (CIRM) has backed research into rare diseases with hundreds of millions of dollars. Over the years, it has supported work at UCLA leading to the development of the bubble baby treatment with $43 million. UCLA licensed the treatment to Orchard Therapeutics of London. Rules for CIRM’s intellectual property arrangements with the recipients date back to the earliest days of the state program -- well before gene therapies were on CIRM’s table.
The development of the therapies, which have price tags of upwards of $2 million, raises major and difficult ethical and financial issues. One is whether money provided by the people of California should be used to financially support companies that may not be ready or willing to take a successful therapy to the patients via the marketplace.
Orchard Therapeutics signaled the abandonment of its CIRM-supported bubble baby trial more than 2.5 years ago. The case did not receive any significant attention until May 2021 when the California Stem Cell Report wrote about the problem. However, the stem cell agency, which is now handing out $5.5 billion under its old intellectual property rules, has yet to address -- at least publicly -- the issues involving its grant recipients and their potential failure to commercialize successful, life-saving treatments.
Currently, the agency has invested in 80 clinical trials. On Thursday (July 28), CIRM’s 35-member governing board is expected to award nearly $8 million for two more clinical-stage projects, an occasion that presents an excellent opportunity for some of its directors to seek answers to how the agency is moving to avoid a repetition of the Orchard case.
(Editor’s note: If you are having difficulty in gaining access to the full Business Insider article, please email firstname.lastname@example.org. Additionally, this item has been modified from an earlier version to add more specifics concerning the $43 million.)
Labels: IP, gene therapy, bubble baby, patients, cirm future
From clinical trials to creation of a stem cell manufacturing network, the California Stem Cell Report has it all. Subscribe today to this indispensable and free newsletter.
And we all keep fighting for these children 💜