Monday, November 02, 2020

Prop. 14: USA Today Looks at UCLA Gene Therapy and California's Stem Cell Program

The Lagenhop family in Los Angeles for a clinical
trial to treat their children for a fatal affliction
Harrison Hill/USA Today photo


USA Today has published a lengthy piece involving initial, favorable results from a more than $12 million clinical trial backed by the California stem cell agency and involving a rare disease that usually ends the lives of children before they reach kindergarten age.

The article comes on the eve of the final day for voting on a ballot initiative, Proposition 14, to save the agency from financial extinction by giving it $5.5 billion more. Officially known as the California Institute for Regenerative Medicince (CIRM), the 16-year-old agency is running out of its original $3 billion and is scheduled to begin closing its doors this winter without a boost from the initiative.

The research involves three children from Ohio who are being treated at UCLA in a trial being conducted by Donald Kohn, who has performed other genetic therapy procedures for rare diseases. For the work, CIRM awarded Rocket Pharmaceuticals, Inc., a publicly traded, New York-based firm, $6.6 million in May 2019. The firm provided co-funding of $5.6 million. (Here is a link to the summary of the review of the application, CLIN2-11480.)

Over the years, CIRM has supported Kohn's work with $52 million, not including the Rocket funding. 

The USA Today article by Karen Weintraub began with the case of the family of Alicia and Jon Langenhop of Canton, Ohio. The piece delved into the history of the California stem cell program, but did not mention the agency or its official name.  Proposition 14 was mentioned twice, once in the headline. 

USA Today is a national newspaper. Circulation figures for California are not available, although it reports national, weekly circulation of 726,906. Today's story, which would resonate with many voters, was tucked away in its health section.  

The affliction involved is Leukocyte Adhesion Deficiency-I (LAD-I

"Patients with severe LAD-I can develop life-threatening infections because their white blood cells are unable to leave the bloodstream to fight them. Without a successful bone marrow transplant, severe LAD-I is most frequently fatal during the first 2 years of life," the Rocket web site said.

The company's stock price today closed at $28.74, up 70 cents. Its 52-week high is $30.43, and its 52-week low is $9.01

USA Today quoted some researchers as saying taxpayer spending has put the Golden State in "the forefront of global stem cell research." The article said, 

"George Daley, a stem cell biologist who is dean of Harvard Medical School, said he's envious of the California researchers who have access to this pot of money.

"'California has always been a very exciting place to pursue science, but prior to (the taxpayer funding), it wasn't exactly the place that was the first on the tip of your tongue as a powerhouse community for stem cell science,' he said. 'But there's no way that today it wouldn't be listed in the top three.'"

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Read all about California's stem cell agency, including Proposition 14,  in David Jensen's new book. Download it from Amazon:  California's Great Stem Cell Experiment: Inside a $3 Billion Search for Stem Cell Cures. Click here for more information on the author.

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