Sunday, January 13, 2019

Is the Governor of California a $5 Billion Stem Cell Friend?

Then San Francisco Mayor Gavin Newsom
 at 2005 announcement of stem cell HQ 

California's newly installed governor, Democrat Gavin Newsom, is no doubt a friend of the state's 14-year-old stem cell research program. 

But is he a $5 billion friend?

The question arises because the stem cell agency expects that its funds for new awards will dry up by the end of this year. It is pinning its hopes for survival on a proposed, $5 billion bond measure that may be placed on the November 2020 ballot. And the agency will need all the support it can muster to convince California voters to approve such a measure. 

Newsom's ties to the agency go back more than a decade. As then mayor of San Francisco, he was a key backer of the city's successful effort to lure the agency's headquarters, and he conjured up a $17 million package of incentives. 

He said at the time that the decision to locate the headquarters in San Francisco was a "proud moment." He said the incentive package demonstrated the "city’s unwavering commitment to innovation as scientists search for new methods to treat the world’s most challenging diseases and injuries."

(See here and here for more on Newsom and the selection of San Francisco as the agency headquarters.)

The agency is now based in Oakland. The free rent deal in Newsom's package expired, and San Francisco was too expensive for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

Newsom was busy last week with non-stem cell matters, such as a $209 billion budget he laid out for lawmakers. Given the nature of his and state priorities, he is unlikely to weigh in soon on a tentative ballot measure that is two years in the future. 

Newsom has sounded cautionary financial notes as he proposes ambitious spending plans. Some news stories have highlighted his willingness to pay down the state's debt, which figures into how the stem cell agency operates. 

In 2004, voters created the agency and also approved $3 billion for research awards. Those billions came from debt (bonds) taken out by the state. It is the first instance of a state funding scientific research with borrowed money. 

The interest on the bonds roughly doubles the cost of the agency, meaning a $10 million research grant really costs the people of California $20 million. 

Today, California is flush with cash because of a solid economy and a rainy day fund created by former Gov. Jerry Brown, also a Democrat. Some financial experts and economists are warning, however, that the good times will not be so good in 2020. That could color how voters perceive spending more on stem cell research.

Newsom may also have other, competing spending priorities come November 2020. He may want to husband his political capital to assure their approval. It is exceedingly unlikely, however, that he would oppose a new stem cell bond measure. But he could sit out a campaign for more billions for the agency. 

Given that possibility, maintaining a warm relationship with Newsom likely stands as an important priority for the agency and its backers -- one that they undoubtedly hope demonstrates both the value of stem cell research and the value it brings to the Newsom administration.
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Wednesday, January 09, 2019

The Golden State's Stem Cell Agency Sells its 'Powerful' Story: 2,700 Discoveries, 50 Clinical Trials, Billions Awarded

Ronnie's California stem cell story

"Something Better Than Hope" -- That's the new anthem of California's $3 billion stem cell research program, which is scheduled this year to run out money for new awards.

The 14-year-old stem cell agency trumpeted its new slogan this morning when it released its annual report for 2018. It's a "powerful story," the agency declared on its blog. 

The 28-page document chronicled the state of affairs at the California Institute of Regenerative Medicine (CIRM), as the agency is formally known. The report is titled "Something Better Than Hope. Right Now." CIRM said, 
"While once there was only hope, now we know that cures are imminent."
Kevin McCormack, senior director of communications, celebrated the agency's annual review on its blogThe Stem Cellar. He wrote,
  • "50 clinical trials funded to date, 7 this year alone
  • "$2.6 billion in CIRM grants has been leveraged to bring in an additional
  • "$3.2 billion in matching funds and investments from other sources.
  • "1,180 patients have been involved in CIRM clinical trials"
The report itself said,
"CIRM has funded 1,000 projects at more than 70 institutions in California and is the largest single funder in the world of clinical research for stem cell and regenerative medicine. More than 2,700 medical discoveries have been peer reviewed and published in scientific journals. But, most importantly, lives have been saved, second chances have become possible and cures have risen beyond hope."
Stories of the lives of patients in clinical trials were not neglected, including Ronnie, the toddler in the video at the top of this item.

Maria Millan, the CIRM CEO who was a pediatric surgeon earlier in her career, wrote,
"CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs.  
"Think about patients like baby Elianna on page 2. She was treated with stem cells for a life-threatening blood disorder while still in her mother’s womb. Ronnie (page 15), who was born with what was previously considered a fatal immune disorder, is alive and thriving today. These stories inspire and motivate us to continue to build upon the great strides CIRM has already made "
The annual report is expected to serve as an important tool as the agency attempts to raise privately more than $200 million to back its efforts until the fall of 2020. That is when CIRM is hoping that California voters will approve $5 billion more for the agency so it can continue its work. 

Annual reports from businesses and government agencies are predictably tilted towards the most positive view of their performance. CIRM's report is no exception. Nonetheless, the agency has run up a record of accomplishment that is significant and important. However, it has not yet fulfilled the expectations of voters in 2004 who were led to believe that nearly miraculous stem cell cures were right around the corner.

Don Reed is a patient advocate who has not lost faith in the promise of stem cells. The California stem cell agency is also indebted to him for his ability to turn the phrase that adorned the cover of the annual report. He was quoted in the report as saying:

"Today, thanks to the 7.2 million voters who authorized the California Institute for Regenerative Medicine, or CIRM, we have something better than hope; we have results, accomplishments, people made well— and a systematic way to fight chronic disease."
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Monday, January 07, 2019

California Stem Cell Board Meetings: A Chance to Meet its Directors and Speak to its Staff

The $3 billion California stem cell agency reports that its governing board will meet 12 times this year, mostly in teleconference sessions that will also have access on the Internet.

The business of the board is largely ratifying awards approved earlier by its grant reviewers. However, its financial survival will be a topic of regular discussion this year. The agency expects to run out of cash for new awards before the end of the year. It is hoping that voters will renew its funding with $5 billion in the fall of 2020.

Four "in-person" meetings are scheduled this year during which most of the board will gather at the agency's headquarters in Oakland. They are scheduled to occur in March, May, September and December. Those will be also accessible on the Internet, both for listening or participating.

The meetings of the board offer opportunities for members of the public, including researchers and business representatives, to speak directly to board members and staff of the agency. The in-person sessions are particularly valuable in that regard because of the informality before and after the meetings, as well as during breaks.  Seating is somewhat limited at the meetings at the headquarters of the agency.

Dates and times of the meetings may change, however, and should be double-checked as the sessions near. The first meeting of the year is scheduled to be conducted via teleconferencing and is set for Jan. 30.

The official name of the board is the Independent Citizens' Oversight Committee. The formal name of the stem cell agency is the California Institute for Regenerative Medicine Sphere: Related Content

Thursday, January 03, 2019

Hype and Fraud and Their Impact on the California Stem Cell Program

Image result for cartoons stem cell fraud
Cartoon by Johan Thyberg from "For Better Science"
The New York Times this week published a piece about how the lust for success could be corrupting science in a greater way. 

The questions it raises have particular relevance in the stem cell field, which is still very much in a formative stage. 

Aaron Carroll, a professor of pediatrics at the University of Indiana, produced the opinion piece for the Times. He wrote, 
"How might grant funding and career advancement — even the potential for fame — be biasing researchers? How might the desire to protect reputations affect the willingness to accept new information that reverses prior findings?"
He also said, 
"Moves toward open science, and for a change in the academic environment that currently incentivizes secrecy and the hoarding of data, are perhaps our best chance to improve research reproducibility Recent studies have found that an alarmingly high share of experiments that have been rerun have not produced results in line with the original research."
Questions arise with some regularity about hype and fraud in stem cell research. Back in 2014, bioethicist Art Caplan asked, “Why so Much Fake, Unduplicable Stem Cell Research?”

Just this past fall, a multimillion-dollar stem cell research scandal involving Harvard surfaced once again in the news.

Credibility, of course, is everything in science. For the California stem cell agency, it is a matter of survival. It will be out of cash for new awards in less than 12 months and is hoping voters will then give it an additional $5 billion.  More news about stem cell hanky panky, wherever it occurs, will not serve the agency well when it makes its pitch once again to the people of the Golden State.  
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Sunday, December 23, 2018

Serious Infections and Fecal Contamination Alleged; Feds Battle Rising Tide of Dubious Stem Cell Clinics

The federal government last week stepped up its efforts to curb dubious stem cell clinics, declaring that 12 persons have been hospitalized for infections as the result of treatments involving a San Diego firm.

Some of the products from the firm have been found to have been contaminated with fecal bacteria . 

The federal action (see here and here) comes after years of ignoring the problem, both by the Food and Drug Administration and state regulators. Meanwhile, the growth of unregulated clinics as multiplied. Current estimates are that as many as 700 or more clinics exist in the United States, compared to at least 570 in 2016. 

California has the biggest share of the clinics, which is not unusual since it is the most populous state in the nation. Legislation backed by the California stem cell agency is expected to be introduced in California next month to step up regulation of dubious clinics.

The federal enforcement action came in the form of a "warning" letter from the FDA and involved a firm called Genetech (no relation to the well-respected biotech firm Genentech, but obviously a name designed to lure unsuspecting patients.)

The FDA said 12 patients have contracted serious infections as the result of injections involving Genetch procedures and products. The Center for Disease Control has reported that some of its unopened products contain E. coli and E. faecalis.

Genetech has not responded to multiple efforts by media to obtain a comment on the federal action.

Some stem cell scientists, most prominently UC Davis researcher Paul Knoepfler, have warned repeatedly for years about the unregulated treatments. In addition to the harm to patients, they have noted that the activities of the dubious clinics damage the reputation of the field in general. (See here, and here and here. Sphere: Related Content

Monday, December 17, 2018

A Deep Probe by STAT into the Man Behind the CRISPR Babies

Want to know more about the researcher who spawned what are now known as the CRISPR babies? 

STAT published a cracker jack of a piece this morning about He Jiankui, including details about his life and his pathway into the gene editing game.

STAT promoted the article this morning in a newsletter with this squib:
"Every superhero, and antagonist, has his own origin story. He Jiankui, the scientist who stunned the world with a claim that he had already gene-edited two baby girls, has one too." 
The lengthy article was written by Sharon Begley and Andrew Joseph and covers He's path from physics to biology. They quoted UC Berkeley scientist Jennifer Doudna as saying, 
“His demeanor was an odd combination of hubris and naivete. He was very confident in his work, and not totally understanding what an explosion he had caused.
The STAT piece is based on extensive reporting. Its authors wrote,
"With details reported for the first time, it describes the many times He met with and spoke before some of the world’s leading genome-editing experts, the low opinion they had of his research, and the hints he dropped about his grandiose aspirations. It is based on interviews in Hong Kong and with experts on four continents, with scientists and others who have crossed paths with He, as well as on documents and published accounts. He did not reply to requests for an interview."
The article's final paragraph ends like this:
"Even those who condemn his experiment doubt it will be more than a speed bump on the road to editing of embryos to prevent severe inherited diseases. 'We have to acknowledge there is interest in using [CRISPR] clinically,' Doudna said. To those calling for a moratorium or an outright ban on such research, she has one response: 'It’s too late.'"
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