$5.5 Billion California Stem Cell Countdown: Total Raw Signatures Now in With 924,213

One key step is now complete in the effort to qualify a $5.5 billion stem cell initiative for the November ballot in California and save its stem cell research program from running out of money.

The "raw" count of signature is finished with a total of 924,213, according to state election officials. That is almost exactly the same as the 925,000 figure that  campaign backers said they had submitted weeks ago.

The proposed ballot measure needs 623,212 valid signatures of registered voters to qualify. The count of valid or projected valid signatures stands at 75,484 as of late yesterday afternoon. The rate of rejected signatures is running at 22 percent. 

A number of populous counties have yet to report on their validation figures including Los Angeles, with 298,147 raw signatures, and San Diego, with 99,899. 

Counties have until June 24 to complete their count.

Look for an update later today or early tomorrow on the count right here on the California Stem Cell Report. 

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 djensen@californiastemcellreport.com. 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.")

"Emergency" Meeting Tomorrow Morning; California Stem Cell Agency Wants to Expand Scope of its Covid Awards

With only 24 hours notice, California's $3 billion stem cell agency today called a meeting for tomorrow to significantly expand the scope of its Covid-19 round to include vaccine development and to permit "supplemental funding."

If the changes are approved tomorrow, scientists could file their applications up until next Tuesday to be considered in the next application review session, probably within 10 days to two weeks.  They would have an opportunity to submit another application about two weeks later if all the Covid funds have not been awarded.

Agency directors allotted $5 million for the Covid round. They have awarded about $2 million.

Here is what the agency listed this afternoon to describe the proposed changes:

• "Increase scope of early stage opportunities (DISC and TRAN) to include vaccine development studies as a potential vital research opportunity.

 • "Include opportunity for studies on novel ideas and biological mechanisms by opening the DISC1 program announcement.

 • "Allow supplemental funding for active COVID-19 clinical trial awards to expand their efforts.

 • "Include requirement for outreach to underserved populations in all clinical trial proposals 

• "Increase award limit on DISC2 projects and adjust language on 6-month deliverables."

The DISC term generally refers to basic research. The TRAN term refers to translational research.

The full concept plan can be found here.

Normally the agency would not fund vaccine research because it does not involve stem cells. Under the proposal for tomorrow, it would be officially deemed a "vital research opportunity," a loophole in state stem research cell law that allows such funding.  The agency has already invoked the provision in a handful of other instances. 

The supplemental funding change would permit researchers who have already received funding under the Covid-19 round to ask the agency for more cash for their applications. The change would allocate up to $1 million for such awards. 

Under normal conditions the agency's governing board could not meet legally with only 24 hours public notice. The normal legal requirement is 10 days. However, the board is invoking emergency provisions to lift the 10-day notice. 

The 11 a.m. meeting will be available for public participation by the public via phone connections and the Internet. Details are on the agenda.

$5.5 Billion California Stem Cell Countdown: No Significant Changes Today

California election officials this afternoon reported no significant changes to the count to determine whether a $5.5 billion stem cell ballot initiative will qualify for the November ballot. 

Here are the results as of 4:55 p.m. PDT today:

Raw signature count -- Unchanged at 917,722, San Mateo County still not reporting

Valid signatures -- 5,202, up from 3,803 yesterday

Disqualification rate of signatures -- Nearly 23 percent, down from 24 percent yesterday

The proposal needs 623,212 valid signatures of registered voters to qualify for the ballot. The proposal is aimed at refinancing the California stem cell agency, which is running out of money. 

Sickle Cell Trial, Backed by $2 Million from State of California, Will be Based in Los Gatos

More details emerged today about a $2 million sickle cell award and clinical trial involving the California stem cell agency, including the location of the trial in Los Gatos and the involvement of a Stanford University researcher. 

The award was approved last Friday by the agency. It went to a Canadian firm, ExCellThera, Inc.,  which is partially matching the state award with $857,143. With the addition of ExCell, the stem agency is now involved in 63 clinical trials. 

                                                                                                                  

The company said yesterday that the Phase 1 trial, which involves safety, will use its ECT-001 product, and will be conducted at the Lucile Packard Children's Hospital. It will be led by Sandeep Soni of Stanford University. The safety phase trial is aimed at children and young adults.

Guy Sauvageau, CEO and founder of ExCellThera, said in a news release.

"The current standard of care for severe sickle cell disease is a blood stem cell transplant, which is only available to patients who have a matched donor. 

“However, the unique properties of ECT-001 cell therapy enable patients without a matched donor to receive treatment, with a greatly reduced risk of post-transplant complications. This study introduces a new indication into the ongoing evaluation of our ECT-001 technology, which we hope will make this life-saving therapy available to a far greater number of  severely ill patients."

The trial has not yet been posted on clinicaltrials.gov. It is not clear whether recruitment of patients is  underway.  Here is a link to a summary of  the stem cell agency's review of ExcellThera's application (CLIN2SCD-11674).