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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Thursday, December 13, 2018

California Stem Cell Agency Scores 50 Clinical Trials, Says It "Just Getting Started"

OAKLAND, Ca. -- California's ambitious stem cell agency this morning chalked up involvement in 50 clinical trials, an achievement that was regarded by some as unattainable 10 years ago.

The watershed event came when directors of the agency approved a $6.2 million proposal to help fight lymphoma.

Following the action, the first person ever to participate in a CIRM human embryonic stem cell trial, Richard Lajara, praised the efforts of the agency, which is running out of cash. 

He said the people of California have a responsibility to move forward with stem cell research and to continue to fund the agency. Lajara's appeal followed a statement from the agency that 35 persons are alive today because of clinical trials that it has helped to finance.

Lajara, who is paralyzed from the waist down, was enrolled in 2011 in the spinal injury trial initiated by Geron, Inc.,  and backed by the agency. 

Jonathan Thomas, chairman of the agency, which is formally called the California Institute for Regenerative Medicine(CIRM), said in a statement,
"We have come a long way in the past seven and a half years, helping advance the field from its early days to a much more mature space today, one capable of producing new treatments and even cures. 
 "But we feel that in many ways we are just getting started, and we intend funding as many additional clinical trials as we can for as long as we can."
In the early days of the agency, Robert Klein, its first chairman, and others widely regarded major participation in clinical trials, especially phase three, as largely beyond the financial capability of the agency. The cost was prohibitive, they said.

The cost of trials varies widely but can run into hundreds of millions of dollars, which could rapidly devour the $3 billion the agency had to spend, the reasoning went. Clinical trials are the last stage before a treatment is approved for widespread use by the federal government.


CIRM's first strategic plan in December 2006, which covered a 10 year period, anticipated some involvement in phase one and two trials. But it said,

"Because of their expense and because of the time required to reach this stage of clinical development, CIRM is unlikely to fund Phase III trials over the time span of the strategic plan."
The first patient to enroll in a CIRM clinical trial did not come until late 2011. 

Today, the agency has committed $541 million to clinical trials, including four that are in phase three, the ultimate trial hurdle to clear before a treatment wins approval. The phase three trials involve kidney disease and ALS.

The 14-year-old agency is now down to its last $144 million. Money for new awards will run out at the end of next year. CIRM is pinning its hopes for continued life on a proposed $5 billion ballot measure on the November 2020 ballot.

Key to winning approval would be a favorable result from its clinical trials -- one that would resonate with the voters. Their expectations were raised in 2004 by the ballot campaign that led to creation of the agency. However, the agency has yet to produce a stem cell treatment that is widely available.
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Wednesday, December 12, 2018

Trump, Fetal Tissue and the California Stem Cell Agency

The California stem cell agency says the Trump Administration moves against research involving fetal tissue have had no impact on the projects that it is financing, at least so far.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), was responding to a question originally raised by a reader of the California Stem Cell Report.

The inquiry was triggered by a number of reports over the last few months concerning the federal direction away from fetal tissue, a move that one researcher, Warner Greene of the Gladstone Institutes in California, called "scientific censorship of the worst kind."

The California Stem Cell Report queried CIRM yesterday about the federal actions, asking whether they have had "any impact, direct or indirect, on CIRM awards, existing or likely in the future."


Kevin McCormack, senior director for CIRM communications, replied, noting that the federal move is relatively recent.
"It could mean an increase in applications that use fetal tissue but it’s too soon to tell. Regardless, this is why the people of California created CIRM, so we don’t have to worry about federal funding for potentially life-saving research. Because we are independent, we can fund what we think is the best science."
McCormack alluded to the ballot initiative in 2004 that established the agency. The campaign was largely based on the need to bypass the Bush administration's restrictions on stem cell research. The anti-fetal tissue effort is likely to be the first step towards resurrecting similar restrictions on stem cell research. 

Politically and ironically speaking, new federal restrictions on human embryonic stem cell research could build support for continued funding of CIRM, which is hoping for passage of a proposed $5 billion bond measure on the November 2020 ballot.

While fresh restrictions are not good for the field overall, their imposition could help to preserve the stem cell agency. Any ballot campaign needs a nasty villain to campaign against.

And without Bush to campaign against in 2004, the stem cell agency probably would never have come into existence.

Here are links to a few of the recent stories on the Trump fetal tissue move: Washington Post, Science, STAT.
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Tuesday, December 11, 2018

Walking a Thin Line at the California Stem Cell Agency

California's stem cell agency has a delicate dance to perform as it edges closer to seeing a $5 billion measure on the ballot to provide more funding for its efforts.
The dance involves a prohibition on state agencies spending public money on behalf of bond measures. The $3 billion agency has a legitimate responsibility to keep the public informed about its activities, but when does that task step over a legal line? 
CALmatters, an online news site devoted to state government matters, touched on the issue this morning. Dan Morain, the site's senior editor, wrote about a couple of cases in the last election.
In his morning newsletter "What Matters," he cited a tiny fine imposed on the Bay Area Rapid Transit District and another case involving an $800,000 expenditure by Los Angeles County board of supervisors, which is unresolved.
As for the stem cell agency, it is certain to be accused of using public money to support any ballot measure that may emerge in 2020. 
That would be part of the political tactics of opponents to the measure -- a portrayal of the stem cell agency as unworthy of voter trust.
The agency is treading lightly in this area right now, a position that will serve it well as more clarity emerges on whether a ballot initiative will actually surface on the November 2020 ballot. 
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Sunday, December 09, 2018

California Setting Stage for Crackdown on Dubious Stem Cell Clinics

The California stem cell agency, state regulators and lawmakers are taking aim at the more than 100 dubious, unregulated "stem cell" clinics now operating in the Golden State.

The goal is to curb clinics that are using what they describe as stem cells in treatments costing thousands of dollars but that have not been tested  scientifically. Lawsuits have been filed around the country alleging damage to patients that includes blindness.

Art Torres, vice chairman of the state stem cell agency, is now  working with lawmakers to formulate legislation that is expected to be introduced by the end of January.

At the same time, the State Medical Board, which licenses and regulates physicians, has chartered a task force to look into the the growing business.

Earlier this fall, Torres told the governing board of
Kevin Mullin, LA Times photo
the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, that he was engaged with Assemblyman Kevin Mullin, D-San Mateo, on a bill.

Torres, a former state lawmaker, said the legislation is expected to involve certification of clinics by a state department. He said, 

"It involves a number of issues which we (CIRM) really can't be involved with in terms of licensing, but we certainly can be involved with the parameters and the distinctions that we ought to raise as to what constitutes an appropriate stem cell clinic in California."
The Medical Board is scrutinizing the promotional practices and harm caused by the clinics with the intent of crafting regulations to curb abuses.  

"There is reasonable concern about a growing number of providers and clinics in the United States that are undermining the field. Such providers and clinics have been known to apply, prescribe or recommend therapies inappropriately, over-promise without sufficient data to support claims, and exploit patients who are often in desperate circumstances and willing to try any proposed therapy as a last resort, even if there is excessive cost or scant evidence of efficacy."
Paul Knoepfler, a UC Davis stem cell scientist who has long been involved in examination of dubious clinics, has reported that at least 100 such clinics exist in California. 

Writing on his blog Nov. 30, Knoepfler said,
"Broadly, it may be going rapidly from the best of times to the worst of times for unproven stem cell clinics in the U.S., which would be a very good thing for patients and the stem cell field, if it actually happens. We’ll see."
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Friday, December 07, 2018

Former Biotech Maven Steve Burrill Sentenced to Prison

Famed and legendary he was called. Now he is facing 2.5 years in prison.

Steve Burrill, wn.com photo
He is Steve Burrill, who once was a featured life science maven/financier/visionary at international conferences such as BIO, which attracts upwards of 16,000 persons annually. Burrill also conducted a well-attended stem cell conference in San Francisco that showcased California's stem cell agency shortly after it came into existence.

Burrill, who was based out of San Francisco, was sentenced this week for defrauding investors and falsifying his tax returns. He pled guilty to siphoning off $18 million from his companies.

Here are links to the news stories on the case: GEN News, STAT, Xconomy. Sphere: Related Content

Thursday, December 06, 2018

California Stem Cell Agency Slated to Award $6.2 Million to Fight Lymphoma

California's $3 billion stem cell agency, which turned 14 last month, is expected next Thursday to give away another $6.2 million as it continues its efforts to fulfill the expectations of the voters who created it in 2004.

Also possibly on tap is an update on the status of efforts to raise privately some $200 million to tide over the agency as it looks forward to 2020 and a possible ballot measure to provide it with another $5 billion.

Known formally as the California Institute for Regenerative Medicine (CIRM), the agency expects to run out of cash for new awards in about 12 months. Its award budget for 2019 now stands at $144 million. Presumably, another $28.4 million can be added, which is the uncommitted cash for research left over from this year. 

The agency subsists on state bond funds approved by voters. Its bond issuance authority, however, is expiring. No other source of funding was provided by voters, and the agency has no expectations of being financed on annual basis by the legislature.

The 2004 ballot campaign that created the first-ever such agency in California history raised high hopes a stem cell therapy was right around the corner. CIRM has not yet backed a treatment that is available for widespread use. However, it is helping to fund 49 clinical trials, the last steps before a treatment is approved for the marketplace.

The application (CLIN2-11371) before the board next week has already been approved by the agency's reviewers. Normal practice is for the board to ratify in public earlier decisions made in private by reviewers.

The name of the recipient has been withheld by the agency until after ratification, as is the agency's standard practice. The proposal seeks to continue a phase one clinical trial  to help treat lymphoma.

The CIRM summary of the review of the application said the goal of the research is to "ameliorate or accelerate recovery from toxicities related to high-dose chemotherapy followed by HDT-ASCT for the treatment of lymphoma and other cancers."

CIRM said the method would involve "genetically engineered CD31+ cells derived from human umbilical vein tissue (engineered HUVEC)."
"There are currently only a few moderately effective treatments available to reduce the toxic side effects associated with aggressive cancer treatments – hence a high unmet medical need. New approaches are urgently needed to both improve quality of life and reduce the risks of high dose therapy."
The summary said that the $6.2 million award would be backed by $2.7 million from the recipient.

CIRM Chaiman Jonathan Thomas has been working to raise the private funds to help support the agency beyond next year. He often reports on his progress at board meetings.

The CIRM board meeting will be based in Oakland. Offsite locations where the public can participate are located in Stanford and San Francisco. The public can also log in online and ask questions or make comments. Instructions on how to participate are contained on the meeting agenda. If you are not familiar with the procedure, it is useful to log in about 10 minutes prior to the meeting's start (10 a.m. PST)  to avoid technical difficulties. Sphere: Related Content