Friday, May 22, 2020

Parkinson's and the Unseen Offspring of California's Stem Cell Agency

On the surface, Aspen Neurosciences, Inc., a tiny firm in La Jolla, Ca., does not seem to have much of a connection to California's $3 billion stem cell agency.

It is not alone. A good chunk of the collateral benefits of agency is all but invisible. While the agency's web site is chock-a-block with videos, statistics, testimonials and pie charts, not everything significant rises to the top. Most of the information can be classified as high profile. Aspen, however, is barely a footnote for the agency. 
Yet the firm, over the last six months, has received a $76 million validation from the private sector, more than any business has received from the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. Viacyte, Inc., of San Diego, is No. 1 among the business beneficiaries of CIRM, collecting $72.3 million.  

The lack of visibility of some benefits from CIRM may not seem all that important to some. But when plans are afoot to ask California voters for $5.5 billion to forestall the financial demise of the stem cell agency, voter perceptions of the agency become paramount. 

Aspen's story has elements that could resonate with many voters. It involves a well-regarded woman scientist, pursuit of millions of dollars, failures and success and patients with stories of their terrible times with a dreadful affliction.  

The tale begins within walking distance of what is now Aspen's headquarters in La Jolla. Aspen is located just down the street from the Scripps Research Institute, where Jeanne Loring was director of the Center for Regenerative Medicine and who also earlier co-founded a firm that morphed into Viacyte

While she was at Scripps, she embarked on research aimed at Parkinson's Disease, stimulated and aided by a patient advocacy group called Summit for Stem Cell. It is an energetic group and once collected $120,000 in a fundraising effort that took nine Parkinson's patients on a hike at Machu Pichu in Peru. 

Summit members also trekked to meetings of the governing board of the stem cell agency to build a case -- sometimes with tears and trembling voices -- for more money and more speed on Parkinson's research. (See here also.)

Loring filed for grants for her research as well. She ultimately collected nearly $22 million from CIRM, but the agency did not follow through on additional applications. Loring cast about for other possibilities, and she and Andres Bratt-Leal, also of Scripps and Summit for Stem Cell, co-founded Aspen Neurosciences. 

Loring strongly supports renewing the financially endangered stem cell agency with $5.5 billion. She has told the California Stem Cell Report
"All of my CIRM awards contributed to (Aspen). We used those funds to develop our genomics tools and databases and to develop improved methods for handling the cells." 
Loring is now Aspen's chief scientific officer. Bratt-LealHer is vice president of research and development. Their ongoing work now at Aspen is aimed at creating the "first autologous neuron replacement therapy to treat Parkinson's disease," Aspen said in a news release. 

Aspen expects to use its private financing to launch a phase one trial for the therapy, which begins with a patient's own skin cells. They are reprogrammed to become pluripotent and converted into neurons. The neurons are transplanted into a patient to replace the ones that Parkinson's has destroyed. 

Whether Aspen can produce a commercial treatment that is widely available remains to be seen. Whether CIRM's supporters can produce a $5.5 billion victory in November remains to be seen as well. It is likely to depend entirely on how voters see the benefits of CIRM's work over the last 15 years -- invisible and otherwise. 

(Note to readers: If you know of other unseen offspring of the stem cell agency -- good or bad -- please send a note to Your emails will be held in confidence. Thank you.)

(After this item was posted, the agency posted a blog item dealing with Parkinson's. In addition to information on the search for a cure or treatment, the item carried the following advice for persons seeking some sort of unregulated treatment. Here is what the agency had to say in a Q&A format,

"If you go online you can find lots of stem cells clinics, all over the US, that claim they can use stem cells to help people with Parkinson’s. Should I go to them?

("In a word, no! These clinics offer a wide variety of therapies using different kinds of cells or tissues (including the patient’s own blood or fat cells) but they have one thing in common; none of these therapies have been tested in a clinical trial to show they are even safe, let alone effective. These clinics also charge thousands, sometimes tens of thousands of dollars these therapies, and because it’s not covered by insurance this all comes out of the patient’s pocket.

("These predatory clinics are peddling hope, but are unable to back it up with any proof it will work. They frequently have slick, well-designed websites, and  'testimonials' from satisfied customers. But if they really had a treatment for Parkinson’s they wouldn’t be running clinics out of shopping malls, they’d be operating huge medical centers because the worldwide need for an effective therapy is so great.

("Here’s a link to the page on our website that can help you decide if a clinical trial or “therapy” is right for you.")

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