Wednesday, August 05, 2020

UC Irvine Named for Covid-19 Trial Backed by California Stem Cell Agency and Giuliani

A New Jersey firm backed by $750,000 from the California stem cell agency today announced that the California location of its Covid-19 trial will be at UC Irvine.

The firm is Celularity, Inc., and its proposed therapy is supported by Rudi Giuliani, President Trump's personal lawyer.  Celularity expects to complete its work by Nov. 30.

CIRM said the research involves "a phase I/II study of human placental hematopoietic stem cell derived natural killer cells for the treatment of adults with COVID-19."

The award was made June 26 by the agency's governing board, which expected its Covid grantees to be ready to start work within 30 days under its fast-track Covid round. Celularity had not responded to questions about the trial location prior to its news release today. The stem cell agency cannot fund research outside of California. 

The company's news release indicated there would be more locations in California, but did not disclose any. 

Tuesday, August 04, 2020

California's $5.5 Billion Stem Cell Campaign and Covid-19: How Science and Politics Marry

Editor's note: The following item written by the producer of the California Stem Cell Report appeared yesterday on the Capitol Weekly online news service. 
The California stem cell agency has just finished pumping $5.3 million into the fight to save the lives of COVID-19 victims. And — in a ballot-box bonus — its efforts are already surfacing in the ballot campaign to rescue the agency from its own demise.
The agency is running out of money. It will begin closing its doors this fall without major financial support that it hopes will come from Proposition 14, a $5.5 billion bond measure on the November ballot.
The $5.3 million in the fast-track, Covid program is tiny by comparison. But the awards are likely to play a heftier role than might be anticipated based on their size. Stories about the Covid awards have appeared on the campaign website in a move to tap into the deep public concern about the coronavirus.
The Covid research piggybacks on the federal “warp speed” drive to develop Covid treatments and is aimed at demonstrating that the agency is part of what CIRM officials describe as a worldwide, “all-hands-on-deck” effort.
Three clinical trials have been funded, which involve actual patients as opposed to laboratory research. If the trials are successful, patients who have benefited are likely to surface in campaign video advertising aimed at refinancing the agency, formally known as the California Institute for Regenerative Medicine (CIRM).
CIRM sorely needs research results that will resonate with voters. The 2004 ballot campaign that created the $3 billion program raised expectations that stem cell therapies were right around the corner. But the agency has yet to produce a stem cell treatment that is widely available to the public.
The agency launched its Covid round on March 27 when CIRM board chairman Jonathan Thomas declared, “The coronavirus is creating an unprecedented threat to all of us, and, as one of the leading players in regenerative medicine, we are committed to doing all we can to develop the tools and promote the research that will help us respond to that threat.”
Indeed, failure by CIRM to respond to the COVID-19 threat — it could be argued — would be a breach of CIRM’s public responsibilities.CIRM’s Covid research financing is a case where an urgent medical need marries nicely with ballot-box politics. The agency is forbidden by law to campaign for the ballot measure. But its charter also allows it to pursue a wide range of research possibilities.
Late last month, CIRM directors capped off the round by approving two awards and adding nearly $300,000 to the original authorization of $5 million. All of the awards have been made under an emergency process that requires awardees to be ready to begin research within 30 days and achieve “a clear deliverable within six months.”
The final awards went for basic level research. Earlier, CIRM approved funds to assist in the three clinical trials, which are the final stage before a treatment can be approved for widespread public use. The trial awards range from $1 million to $701,000 each, a bargain price for joining a trial. Most of CIRM’s previous clinical trial awards run into many millions.
CIRM now counts a total of 64 clinical trials that it is helping to finance. Two of the Covid trials are taking place at the University of California, San Francisco-UC Davis and the City of Hope in the Los Angeles area. The third involves a New Jersey firm called Celularity, Inc., which has failed to respond to multiple questions about the California location of its trial. The agency is limited to funding activities within the state. CIRM directors approved the Celularity award on June 26, more than 30 days ago.
You can find more information about all the awards in a series of items on CIRM’s blog, The Stem Cellar, using the search term “covid.”   Five CIRM/Covid stories, including CIRM blog items about the Covid round, have been placed on the campaign website by the campaign staff. 

Saturday, August 01, 2020

Quote of the Day

Jonathan Shestack, in 2013 and then a patient advocate member of the governing board of the California stem cell agency, speaking about patient advocates appearing before the board:
"We have a responsibility to look those people in the face.... So I would like to be very careful as we retool that process to make it neater and less chaotic and not upsetting to the press or to people in Sacramento, but to understand that is part of our responsibility, to look at those people and listen to them and take some of what they have to say into consideration."
From a meeting of the CIRM board Jan. 23, 2013

Monday, July 27, 2020

California's Stem Cell Covid Round Ends: Three Clinical Trials, 17 Awards

The California stem cell agency has finished its special round of Covid-19 awards, adding three clinical trials to bring its total to 64 plus 14 other awards totaling in all $5.3 million. 

The final awards were made on Friday in the emergency, fast-track round. However, the agency noted that additional Covid awards are possible through its existing awards process and today issued a "special call" for applications in its Quest awards program.  The deadline is Aug. 31.

The agency said in an email, 
"For this special edition of DISC2, the overall timeline and award amount have been capped at 12 months and $250,000, respectively. The expected outcome, at the end of the award, is a candidate therapeutic, diagnostic, medical device, or tool that can immediately progress to translational stage activities."
Last week's awards of $250,000 each went to Karen Christman at UC San Diego and Lili Yang at UCLA.

Christman hopes to develop a treatment for acute respiratory distress syndrome, the agency said, a life-threatening lung injury that occurs when fluid leaks into the lungs and is prevalent in COVID-19 patients.  Her research involves extracellular matrix hydrogels, a structure that provides support to surrounding cells.

Yang is seeking to use blood stem cells to create invariant natural killer T (iNKT) cells, a powerful type of immune cell with the potential to clear virus infection and mitigate harmful inflammation.

For more details, the agency's review summary for Christman's application (DISC2COVID19-12007 #2 ) can be found here. The summary of Yang's review (DISC2COVID19-12020) can be found here. Yang also sent a letter to the agency board as did Christman.

Quote of the Day

"In science, true hope is clear-eyed and brings a tight focus on the barriers and potential setbacks that exist along the path to desired results." 

-- , in his New Yorker article titled "The Long Game of Coronavirus Research."

Friday, July 17, 2020

Covid Clinical Trial Location Still Missing on $750,000 California Stem Cell Grant

The California stem cell agency stipulates that winners of research awards in its fast-track, Covid-19 round must be ready to start work within a month of approval, but a New Jersey firm that snagged $750,000 three weeks ago has yet to report the location of its California work.

The  firm is Celularity, Inc. Its proposed therapy is backed by Rudi Giuliani, the president's personal lawyer, who has also touted other possible Covid therapies, including one that has been rejected by the Food and Drug Administration.

The $750,000 is intended to finance a clinical trial someplace in California. The stem cell agency funds only California work.

In creating the Covid round, CIRM said award winners must be "ready to start work within 30 days of approval and propose achieving a clear deliverable within six months." It has been 21 days since the Celularity award was approved. 

The California Stem Cell Report has asked the firm repeatedly for the location of the California site but it has not responded with this routine information. The agency itself also has not disclosed the location and defers to the company.  

Celularity updated the information today that it files with the National Institutes of Health on the trial. However, the locations of the trial still did not show any in California. 

Thursday, July 16, 2020

California's $40 Million Stem Cell Genomics Center: Its Fate and Outcomes

California's stem cell agency hit something of a milestone six years ago when it approved $40 million to create a genomics center that was aimed at positioning the state as a global leader in stem cell genomics. 

The round triggered complaints about irregularities, unfairness, score manipulation and the role of its then president, Alan Trounson. The $40 million, at the time the largest single research award by the agency, was ultimately divided three ways and now has been spent. 

The California Stem Cell Report queried the agency this week about the award, its status and results. Here is the response in Q&A format.

Q: How was the $40 million shared and with which institutions?
A: The genomics grant was split into three awards: $22.7M to Stanford (GC1R-06673-A), $13.2M to the Salk Institute (GC1R-06673-B), $4M to UC Santa Cruz (GC1R-06673-C). (The application numbers carry links to the progress reports on the CIRM website.) 

Q: Is there any requirement for the grantees to come up with a plan for continued funding? 
A: There was no requirement for the grantees to come up with a plan for continual funding in the concept RFA.  Stanford and Salk provided co-funding to support the centers during the award period.

Q: If not, what prevents the doors from closing at the genomics center(s) in the next couple of years?
A: The Stanford sequencing center established by the genomics awards continues to operate with other sources of funding.  The Stem Cell Data Hub established by UCSC continues to be supported.  UCSC has no plans to shut down the site even though its CIRM award has ended. 

Q: How much time remains on the grant(s)? I See there was at least one extension given, but it is unclear why there was delay.
A: All three genomics grants have ended as of May 31st.  The sequencing centers at Stanford and the Salk were given six month award extensions to continue supporting projects with technical delays in sequencing activities.  The data hub at UCSC was given a 12 month extension to process data generated by the sequencing centers.

Q: What else do readers need to know about this award? 
A: A number of outputs were generated from genomic center projects, including:
  • 60 publications connected to CESCG funding
  • 10 data analysis/visualization tools created
  • 1 new sequencing technique developed
  • 300 new iPSC lines created
  • Central online data hub created with standard metadata, analysis pipelines, restricted access
  • Data repository of human single cell global transcription in heart, pancreas, blood, brain, brain
  • 84TB data generated from sequencing activities across projects

Tuesday, July 14, 2020

Former Chief of California Stem Cell Agency Returns to California as Head of Sanford Burnham

C. Randal Mills, who led California's $3 billion stem cell agency for three years, this afternoon was named as chief executive officer of the Sanford Burnham Prebys Medical Discovery Institute in La Jolla, Ca.

Randy Mills
Sanford Burnham photo
The institute said in a news release that Mills brought to Sanford "decades of experience as an entrepreneur and transformational leader in the biomedical industry."

Most recently, Mills was president of the National Marrow Donor Program/Be The Match nonprofit, an international leader in facilitating and supporting bone marrow transplantation.

Mills left Be The Match in February of this year, citing personal reasons.

Mills, who goes by Randy, was  president and CEO of the California stem cell agency from May 2014 until July 2017. 

During his time at the agency, which is formally known as the California Institute for Regenerative Medicine (CIRM), Mills instituted something he called CIRM 2.0. It streamlined and sped up the work of the agency, sharpening its focus with an emphasis on performance metrics. The structure of the effort remains in place today. 

When Mills left the agency, CIRM Chair Jonathan Thomas, who recruited Mills, said his efforts had created a "bold strategic plan, the results of which include an 82 percent reduction in approval time, a 3-fold increase in the number of clinical trials, and a 65 percent reduction in the time it takes to enroll those trials."

Sanford Burnham has received $39.4 million from CIRM over the years. Its president, Kristiina Vouri, sits on the governing board of the stem cell agency, which has included a Sanford representative since 2004. 

Vouris said in the news release that Mills "comes to us with extensive organizational and executive leadership experience. His diverse background and knowledge of translational research and drug discovery will help us propel the Institute into a bright and successful future.”

Mills will begin his work immediately at Sanford Burnham, which has 700 employees in the San Diego area.