California Stem Cell Report Taking Break

The California Stem Cell Report is on holiday until Jan. 6. Look for a fresh posting then. Happy New Year to all.

Possible Parkinson's Stem Cell Treatment in San Diego, $6.5 Million and Loads of Media Coverage

KPBS 2018 video

A San Diego firm that has $22 million worth of financial roots in the California stem cell agency this month snagged a bonus -- a ton of favorable attention for its efforts to treat Parkinson's disease. 

The fledgling business is Aspen Neuroscience, Inc., which was co-founded by Jeanne Loring, professor emeritus at the Scripps Research Institute, and Andres Bratt-Leal, also formerly of Scripps. 

Earlier this month, Aspen received $6.5 million in seed funding from Domain Associates and Axon Ventures. The announcement of the cash infusion triggered a spate of news articles.

In a piece on the Biocentury web site, writer Elizabeth Eaton quoted Kim Kamdar, a partner at Domain, as saying, 

"Aspen is the only company taking an autologous approach to cell therapy for Parkinson's disease." 

The approach can avoid the immunosuppression necessary for allogeneic therapies. Eaton continued,  

"Such (immunosuppression) treatment, which can last for a year or more to ensure a patient's immune system doesn't react to the transplant, can make patients more susceptible to viral diseases and cancer.

"While companies like BlueRock Therapeutics L.P. are exploring allogeneic treatments, Aspen was able to break into the autologous space thanks to its genomic tools, co-founder and CSO Jeanne Loring told BioCentury.

"Aspen is built on Loring's research at the Scripps Research Institute and the PluriTest, an assay she developed after discovering that pluripotent cells have a distinct genetic signature. The patented test shows whether a cell has achieved pluripotency, which allows researchers to avoid conducting more time-consuming confirmatory tests."

Loring has received nearly $22 milllion from the state stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM), including $4.5 million as a co-PI. She told the California Stem Cell Report, 

"All of my CIRM awards contributed to this project. We used those funds to develop our genomics tools and databases and to develop improved methods for handling the cells.  The only specific CIRM grants supporting this project were DISC2-09073 and the progress award DISC2P-11595."

Loring also credited Summit for Stem Cell Foundation of San Diego for supporting and financing her work as far back as 2011. Joining Domain in the $6.5 million in funding were Alexandria Venture Investments, Arch Venture Partners, OrbiMed Advisors and Section 32.

Here are links to additional stories on Aspen and the $6.5 million in funding: San Diego Union-Tribune, San Diego Business Journal, Endpoints News, FierceBiotech, Xconomy, BioWorld, Parkinson's News Today and BioSpace. 

Aspen Neurosciences video

Latina Cancer Advocate Ysabel Duron Named to California Stem Cell Board

Ysabel Duron

The governing board of the $3 billion state stem cell program has a new member, Ysabel Duron, an award-winning Latina journalist and president of the Latino Cancer Institute.

Her appointment comes as Sherry Lansing, who was the first woman to head a major Hollywood studio, has left the board. Lansing is president of the foundation bearing her name, which is involved deeply in cancer research, and is a member of the University of California board of regents. 

On its blog, the stem cell agency said,

"Ms. Duron was a journalist and TV news anchor for more than 43 years winning numerous awards, including two EMMYS. She has been inducted into the Hall of Fame of the National Association of Hispanic Journalists and given the Living Legacy Award by the Chicana/Latina Foundation.

"As a journalist she covered her own battle against Hodgkin’s Lymphoma, using her reporting to help raise awareness about the disease and the health disparities involved in treating it in communities of color."

The agency quoted Duron as saying,

"Usually I am looking for the best return for the public health! This appointment gives me a new learning opportunity to understand a very complex issue, and, make it bite size so the public, patients and advocates will understand how these scientific revelations will impact lives in the short term and the long run. As a steward of taxpayer dollars, I also want to make sure there is equity for communities across California, and that the research serves all of us."

Lansing served on the agency's 29-member board since its inception in 2004. In her letter of resignation, she said,

"This is just the beginning for stem cell research, and yet we are at a critical inflection point in the life of this organization - with funds coming to an end and promising research that must wait for the next CIRM(the stem cell agency). It is for this reason that I am stepping down now, so that I can dedicate more time to getting the next initiative on the ballot."

State Controller Betty Yee appointed Duron. The agency's board currently has two vacancies.

'Natural Killer' Cancer Cell Treatment Advances; State Stem Cell Agency Pleased

The California stem cell agency this week touted a possible "off-the-shelf cancer killer" that the state enterprise is helping to develop with $4 million.

The company involved in the treatment is Fate Therapeutics, Inc., of San Diego, a publicly traded firm working on induced pluripotent stem cells (iPSC) that have been genetically engineered to enhance their anti-tumor activity. The product is called FT516.

Writing this week on the agency's blog, Kevin McCormack, senior director of communications at the agency, said,

"Fate Therapeutics has developed an off-the-shelf therapy (thanks to (agency funding) that could, theoretically, be stored at hospitals and clinics around the country and used whenever it’s needed for anyone who needs it."

McCormack said that a CAR-T approach uses a patient's own "re-engineered" cells to kill cancer tumors. He wrote, 

"But the thing that makes it so appealing – using the patient’s own cells – also makes it really complicated and expensive. Creating a custom-made therapy from each patient’s own cells takes time and costs a lot of money. But now (the Fate Therapeutics) approach could change that."

Fate uses NK (natural killer cells). Maxx Chatsko of the Motley Fool financial web site reported,

"It's the first cell product of any kind derived from a clonal master iPSC line to be evaluated in a clinical trial in the United States. The 'clonal master iPSC line' part essentially means it can be manufactured more easily than immunotherapies requiring cells to be extracted from patients. That's why it's called 'off the shelf.'"

McCormack continued, 

"At this year’s meeting of the American Society of Hematology (ASH) Fate announced that the first patient treated with this new approach seems to be doing very well. The patient had acute myeloid leukemia and wasn’t responding to conventional treatments. However, following treatment with Fate’s FT516 the patient responded quickly and...was able to leave the hospital and spend Thanksgiving with his family. 

"Equally impressive is that 42 days after being treated with FT516, the man showed no signs of leukemia in either his bone marrow or blood."

The product is being licensed exclusively to Fate by the Memorial Sloan Kettering Cancer Center

Fate Therapeutics' stock closed today at $18.79, down 13 cents. Its 52-week high was $22.82. The 52-week low was $11.00. Last September it raised $173 million in a stock offering.

LA Times: $5.5 Billion Measure for California Stem Cell Agency Could Actually Be a 'Downfall'

The Los Angeles Times, California's largest circulation newspaper, carried a piece today that called for withdrawal of the proposed, $5.5 billion ballot initiative that would save the financial life of the state's stem cell agency.

The article by Pulitzer Prize-winning business columnist Michael Hiltzik said the measure would perpetuate many of the flaws in the ballot proposition in 2004 that created the agency, officially known as the California Institute for Regenerative Medicine (CIRM). 

In fact, Hiltzik said, the new measure "makes some of them worse."

"That’s dangerous," Hiltzik continued, "because although the measure could fuel the stem cell program for years to come, it might also prompt a repudiation by voters sensitive to its many imperfections. Such an outcome would be tragic for California and the advanced science already supported by CIRM."

Hiltzik's opinion piece was the first extended look at the proposed stem cell initiative by a major media outlet in California. It appeared on the front page of today's business section of the Times. On the paper's web site, the article was featured as a "weekend read" at the time of this writing. 

The Times says it has about 2 million readers on Sunday. It is a go-to source for other journalists when they are researching stories and trying to understand major issues facing the state. 

Hiltzik has followed the $3 billion agency since its inception and has been highly critical. Last spring, however, he wrote that the agency had "proved its value." In today's piece, he had a lot to say about the good work of the agency. 

In his first two paragraphs, Hiltzik declared that CIRM "has made great strides in advancing what’s known as regenerative medicine and placing California at the center of the developing science." Hiltzik also said that CIRM has financed state-of-the-art laboratories, attracted preeminent scientists and "brought scores of promising treatments for severe chronic diseases to the point of clinical trials."

But he also noted that the agency has "has so far failed to yield a single marketable clinical product." He continued, 

"That’s despite the sales pitch for Proposition 71 in 2004 -- that all that stood in the way of 'cures' for Parkinson’s, Alzheimer’s, spinal cord injuries and other maladies was money."

The greatest flaw of the agency, however, has more to do with public policy than science, Hiltzik wrote. And that is the exemption of the agency from the usual oversight by the legislature and the governor, an exemption contained in the measure that created the agency.

Voters provided CIRM with $3 billion in bond funding 15 years ago. 
Today that money has all but run out. Robert Klein, a real estate developer, first chairman of the agency and who sponsored Proposition 71, is now once again sponsoring the latest initiative to give it billions more. 

Hiltzik concluded,

"(Klein) should withdraw his measure, and CIRM’s leadership should write a new one or work with Gov. Newsom and the Legislature to map out the program’s next act. 

"CIRM’s leadership needs to show the public that it’s capable of taking charge of the program’s destiny. If it’s not willing to make its own case for CIRM’s continued existence, how can it persuade voters to give it one cent more?"