Wednesday, July 24, 2019

The "Hard Case" of Stem Cell Sound Bites and a Campaign to Raise Billions

The journal Nature today reported on the $5.5 billion plan to save what it called the "struggling" California stem cell agency, which is fast running out of cash for new research awards. 

In an article by Jonathan Lambert, Nature caught up with news that it is not so new to readers of this web site. 

The piece carried information from Robert Klein, who expects to lead a new ballot initiative effort late next year to provide the billions more for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

Nature also had this from a man who served on the Institute of Medicine team that evaluated the California program in 2012.
Aaron Levine, Georgia Tech photo
"Aaron Levine, a science-policy researcher at the Georgia Institute of Technology in Atlanta, says that CIRM has put California at the center of stem-cell research worldwide. 'CIRM has been really important in driving stem-cell research forward, especially in the preclinical and proof-of-concept space,' he says. 
"But he isn’t sure whether that will convince voters to keep supporting the agency." 
The Nature article continued,
"'It will be interesting to see if the campaign is one of promised cures, or something a bit more nuanced,' says Levine. 'It takes time for a whole new field of research to result in cures, but that’s a hard case to make to voters in short sound bites.'"
Nature said Klein told it that he will form a non-profit lobbying arm in October or November to help support the campaign next year. 

The journal also said changes in the stem cell research program were anticipated in the new ballot initiative.  
"These include creating a dedicated staff of 10–15 people who would work with insurance companies and patients to improve access to clinical trials and future therapies."

Tuesday, July 23, 2019

UC Researcher on Stem Cells and California's Stem Cell Agency

The California stem cell agency recently highlighted a brief look at the field with a Q&A that ranged from the impact of the agency itself to dealing with questions about "miraculous" stem cell "cures."

The item appeared on the agency's blog and originated at UC Davis, which is among the top five recipients of funds from the agency, known formally as the California Institute for Regenerative Medicine (CIRM).

The school has received $143 million, making it fifth on the list of recipients in terms of dollars.

The Q&A involved Jan Nolta, head of the stem cell program at UC Davis. Some excerpts from what Nolta had to say:

Jan Nolta, UC Davis photo
"Perhaps the most promising and exciting research right now comes from combining blood-forming stem cells with gene therapy. 
"Along with treating the famous bubble baby disease, where I had started my career, this approach looks very promising for sickle cell anemia. We’re hoping to use it to treat several different inherited metabolic diseases.... 
"The beauty of this therapy is that it can work for the lifetime of a patient. All of the blood cells circulating in a person’s system would be repaired. It’s the number one stem cell cure happening right now. Plus, it’s a therapy that won’t be rejected. These are a patient’s own stem cells. It is just one type of stem cell, and the first that’s being commercialized to change cells throughout the body."
About the five Alpha stem cell clinics initiated by CIRM, Nolta said,
"These are clinics where the patients can go for high-quality clinical stem cell trials approved by the FDA [U.S. Food and Drug Administration]. They don’t need to go to 'unapproved clinics'a and spend a lot of money. And they actually shouldn’t."

Thursday, July 18, 2019

California Medical Regulators Take Up Dubious Stem Cell Clinics Next Month

The California State Medical Board next month will discuss clinics that offer unregulated "stem cell" treatments that have allegedly led to blindness and tumors in some cases. 

The meeting comes a year after the board created a "task force" to address the issue.  It also comes as more news emerged this month concerning what was described as a "gruesome case" of an unregulated treatment that went wrong and a step-up in legal action against a La Jolla clinic. 

Canada this week also told dozens of clinics to stop selling unproven stem cells.

California is the location of the largest number of these dubious clinics in the United States, according to UC Davis stem cell researcher Paul Knoepfler and Leigh Turner of the University of Minnesota. The total nationally is currently believed to be more than 1,000. 

State legislation to regulate them is on the shelf in Sacramento. The state medical board formed a two-person task force last July to address the matter. The task force only recently held its first meeting on June 27. 

Queried by the California Stem Cell Report about the status of the board's work, Carlos Villatoro, a board spokesman, said this week, 
"There will be an update on the Stem Cell Task Force at the August meeting, as the Task Force has met. In addition, an interested parties meeting is being scheduled for early September."
The task force met with no public notice, which Villatoro said was not legally required. 

Villatoro has said that the board does not regulate clinics -- only physicians and some other medical professionals. The board describes itself as a consumer protection agency. 

(Here are links to what the board describes as a "complete listing" of laws dealing with its regulatory powers: California Law and Guide to the Laws Governing the Practice of Medicine by Physicians and Surgeons.)

The board meeting next month will be in Burlingame Aug. 8 and Aug. 9.  The agenda has not been posted, but the meeting is expected to streamed on the Internet. 

Friday, July 12, 2019

More News Reports on Halt in California Stem Cell Funding Applications

The journal Science this week joined the publications beginning to report on the financial travails of the $3 billion California stem cell agency. 

In a piece by Jocelyn Kaiser, the journal briefly summarized the agency's activities and its outlook for the future. Kaiser wrote, 
"Some researchers who explore the basic science of stem cells had already been looking for other funding sources as (the agency) began to emphasize clinical work and their support wound down. But others, especially those planning clinical trials, will be hit hard.
April Pyle, UCLA photo
"'It’s going to be a huge impact on my lab and many others if they end,” says April Pyle of UC Los Angeles (UCLA), whose 11-person group works on using muscle stem cells to treat muscular dystrophy. Her last CIRM grant ends in March 2020 and although she also has some NIH funding, it does not support the animal testing and other studies needed to move her work toward a clinical trial."
CIRM is the abbreviation of the official name of the stem cell agency, the California Institute for Regenerative Medicine

Pyle has received $4.6 million from CIRM and UCLA $289 million, according to the agency's figures.

Kaiser also wrote, 
"Ongoing payments for approved projects continue, but scientists are already tightening their belts for a funding gap. They are also contemplating the end of a boom in stem cell research in the state. California’s voters may be asked to renew CIRM with another bond initiative next year, 'but there’s no guarantee,' says Arnold Kriegstein, who heads a stem cell center at the University of California (UC), San Francisco, and has received CIRM funding in the past."
Kriegstein has received $4 million from CIRM and UC San Francisco $192 million.

The shutdown of CIRM applications was first reported by the California Stem Cell Report on June 20.

Others have recently followed, in one form or another, including The Scientist, Genome Web, Capitol Weekly, National Review, The Beacon, Spine Review and LifeNews.

Thursday, July 11, 2019

Live Online: The 'Inside Scoop' on $3 Billion, California Stem Cell Research Effort?

The headline was provocative, and the question was "now what?"

It is the latest posting on the blog of the $3 billion California stem cell agency, which expects to run out cash for new awards as early as this fall. 

"Getting the inside scoop on the stem cell agency" -- That was the headline for the article, which promoted an online event July 25 involving three of the directors of the nearly 15-year-old agency, formally known as the California Institute for Regenerative Medicine (CIRM). 

"Now what?" is one of the questions they will address during the Facebook Live session. Viewers will have a chance to submit questions during the event and hear the answers. The immediate response is one of the benefits of such an event. Another is that they are preserved online for later viewing, multiplying the potential exposure in a significant way. 

That staying power is a benefit of considerable utility for the agency, which is not exactly a topic at the breakfast table in the homes of California voters. But they are the folks who the agency hopes will approve a proposed ballot initiative in November 2020 for an additional $5.5 billion for the research program. 

CIRM is currently engaged in a bit of an extra effort to educate Californians about the positive aspects of its work. In recent years, it has functioned in the usual obscurity enjoyed by most state agencies. However, unlike most state agencies, it does not survive financially on the usual budgetary process. 

CIR was born in 2004 with $3 billion, but nothing more.  So today the task is demonstrate to the people of California its value proposition. 

Taking up that task online in a couple of weeks will be CIRM directors Anne Marie Duliegeexecutive vice president and chief medical office Rigel Pharmaceuticals; Joe Panetta, president of BIOCOM, and Dave Martinchairman and CEO of AvidBiotics. And CIRM is inviting Californians to join in the Facebook Live session "to understand how we got where we are, how the rest of the field is doing and what happens next."

Tuesday, July 09, 2019

The California Stem Cell Media Mix: 'Not Meant to Last Forever'

The Scientist magazine this week caught up with California stem cell matters, declaring that state stem cell agency "was not meant to last forever."

The piece by Chia-Yi Hou was a brief overview of California's stem cell agency, bringing the magazine's readers up-to-date about the current condition of the $3 billion research effort. 

"The California Institute for Regenerative Medicine (CIRM) was not meant to last forever, as it received a finite amount of government funds when it was formed," she wrote, referring to the stem cell agency by its official name.

Noting that the program was slow to begin its spending, The Scientist article continued, 
"Eventually, research ramped up and, with the help of CIRM, California has become a hotspot for the field. CIRM helped start stem cell labs in California and attract investments from pharmaceutical companies. 'I think it launched the whole field,' says stem cell researcher Jeanne Loring of Scripps Research in an email to The Scientist. 'At a time when the [National Institutes of Health] was not supporting much translational research using pluripotent stem cells, CIRM was investing heavily in that area.'" 
The piece said,
"Where CIRM funding has been crucial is funding preclinical studies that help get research 'from the bench to the bedside,' says stem cell and gene therapy researcher Stephanie Cherqui of the University of California, San Diego, who is the recipient of two awards totaling more than $17 million. Not many granting agencies have the means to provide millions of dollars to fund the toxicology, pharmacology, and manufacturing studies that are required by the US Food & Drug Administration before potential treatments can go into clinical trials, according to Cherqui."

Friday, July 05, 2019

USC vs. UC San Diego: Unprecedented $50 Million Settlement in Academic Recruiting War

The University of Southern California in Los Angeles is coughing up $50 million and publicly apologizing for its tactics in recruiting a star Alzheimer's resarcher from UC San Diego, it was reported Thursday.

The Los Angeles Times story about the unprecedented settlement described the case as an "ugly academic war." It had the potential of bringing $340 million in research grants to USC.  

The move settled a $185 million lawsuit that at one point involved two directors of the $3 billion California stem cell agency, along with researcher Paul Aisen.

The Times story said the "unprecedented litigation in which UC accused its private rival of repeatedly stealing away top scientists and their lucrative research grants with 'predatory' practices and a 'law-of-the-jungle mind-set.'"

Aisen was a neurology professor at UC San Diego. He and his lab staff left the La Jolla school in 2015. The Times reported that the departures were secretly orchestrated by top administrators at USC.

The Times story, written by Harriet Ryan and Teresa Watanabe with additional reporting by Bradley Fikes, said,
"The self-described 'quarterback' of Aisen’s recruitment was then dean of USC’s Keck School of Medicine Carmen Puliafito, subsequently revealed to have been using drugs and partying with criminals during the time he was courting the scientist."
At the time, Puliafito and David Brenner, dean of the UC San Diego medical school, were both members of the governing board of the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. Aisen, however, has not received funding from CIRM, which has financed $56.5 million in other Alzheimer's research. 

According to the Times, the apology said that the recruitment tactics "did not align with the standards of ethics and integrity which USC expects of all its faculty, administrators and staff."

The Times story continued,
"UCSD Chancellor Pradeep Khosla welcomed the settlement and said he was open to working with USC in the future.
"'For California and the country, it’s good that two great research universities can work on the Alzheimer’s problem,' he said in an interview. 'I look forward to a constructive collaboration in the future in solving other societal problems.'
"It is not unusual for professors to move to other institutions, but it is often a collegial process in which the universities work together to transfer grants and research."
The Aisen case was not the first instance of USC researcher poaching. The Times wrote,
"In 2013, Puliafito lured two well-funded brain researchers from UCLA, outraging the state university, which complained to government regulators. USC agreed to pay UCLA more than $2 million in a confidential settlement."

Wednesday, July 03, 2019

California Stem Cell Agency: 'Inarguable' Source of Hope, Says Article in Scientific American

The headline this morning on the Scientific American web site said:
"A Bulwark against Trump's Stem Cell Ban
"California's Institute for Regenerative Medicine, a kind of mini-NIH, does crucial basic research without federal funding"

Authored by Zachary Brown, a researcher at UC San Francisco, the article used as a peg the Trump administration restrictions on fetal research funding. Brown contrasted those restrictions to work being done here in California financed by the state stem cell agency. 

Brown recalled that a major impetus for voter creation of the agency in 2004 was presidential restrictions on federal backing for human embryonic stem cell research. Brown wrote,
"The future of embryonic stem cell research appears uncertain once again, as researchers are forced to scramble to adjust to arbitrarily changing norms uninformed by science."
Brown said,
"Almost 15 years have passed since Proposition 71 became law, and California voters made a three-billion-dollar bet on the promise of stem cell technology. At the time of its passage, the policy proposal was as groundbreaking as it was subversive."

Subversive because it challenged the "very relevance" of the predominant federal funding model. 

Brown continued,
"The measure was not perfect. Robert N. Klein II, one of the largest donors in support of Proposition 71, ended up as head of the governing body for seven years, and questions concerning bias in the disbursement of its ample endowment linger — curiously, more than 90 percent of awards" have gone to institutions with ties to governing board members.
"However, its role as a source of hope, both symbolic and realized, for the field of stem cell research is inarguable. The federal government weakens the image of the U.S. as a hub for discovery and medical ingenuity every time it prioritizes political gain over scientific progress."

Monday, July 01, 2019

Hard News for Patients, Scientists: California Shuts Down Applications for Its Stem Cell Research Funding

The California's stem agency's rundown on its clinical
 Some of the trials have saved lives.
The $3 billion California stem cell agency today served up the bad news with only a smattering of sugar coating.

No more applications for research funding are being accepted. The cash is running out, perhaps as early as the end of August.

In a posting on its blog, The Stem Cellar, the agency declared,

"It’s never easy to tell someone that they are too late, that they missed the deadline. It’s particularly hard when you know that the person you are telling that to has spent years working on a project and now needs money to take it to the next level. But in science, as in life, it’s always better to tell people what they need to know rather than what they would like to hear."
The news is no surprise to persons who follow the agency. But today brought a more clearly emerging sense of finality.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, was created in 2004 by voters who also provided $3 billion in bond funding. However, the CIRM ballot measure also contained the seeds of destruction for the agency. No other cash was provided. No other significant means of funding was laid out.

Today, the agency is pinning its hopes for survival on a yet-to-be-written ballot initiative for the November 2020 ballot. To bridge the gap between now and then, CIRM has been attempting for months to raise privately more than $200 million. So far, no results have emerged publicly. 

As of last month, the agency had in its pipeline applications for $88 million in research funding. But it had only $33 million left for new awards. 

CIRM reports it has enough cash on hand to administer its portfolio of awards, which stretch out a couple of years.

Fifteen days ago the agency quietly announced the application shutdown. Little public notice of the action was taken even in California's stem cell community, which has grown mightily over the nearly 15-year life of the agency. 

During that period, CIRM has helped finance 55 clinical trials targeting diseases ranging from cancer and heart disease to diabetes and arthritis. It has served up 1,015 research awards. The scientists it has supported have published more than 3,000 research papers. 

However, CIRM has yet to fulfill the campaign-generated expectations of the 7,018,059 voters in 2004 who voted to create it and who thought they would see new, widely available, miraculous cures. Impressive results, some of which have saved lives, have surfaced from some of the clinical trials. But the elusive stem cell cure that would be ready for the general public is yet to hit the streets.

The Oakland-based agency is not done yet nor is it out of business.  Its reviewers are expected to meet later this month to make the de facto decisions on some of the pending applications. And then again in August. 

More needs to be done in terms of the private fundraising effort. And more needs to be done in crafting a new ballot measure that would bring $5.5 billion to CIRM.  

In the CIRM blog item today, written by Kevin McCormack, senior director of communications, the agency declared, 
"Over the years we have built a pipeline of promising projects and without continued support many of those projects face a difficult future. Funding at the federal level is under threat and without CIRM there will be a limited number of funding alternatives for them to turn to.
"Telling researchers we don’t have any money to support their work is hard. Telling patients we don’t have any money to support work that could lead to new treatments for them, that’s hardest of all."

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