Looking for Some Research Cash? $182 Million on Table in California; Briefing Thursday on How to Get It

California's $12 billion stem cell program is entering a new phase that will affect patients and researchers alike, and it is planning a show-and-tell this Thursday for the curious.

"With the voter approval of Proposition 14 CIRM is about to start a new chapter in its life. Because we are always trying to improve the way we operate, always trying to be better, there are going to be changes in the way we do things. Some of those changes are in the way we fund research, and the kinds of projects we are going to fund," the agency said in an announcement last week. 

Gil Sambrano, CIRM photo

Addressing many of those matters will be Gil Sambrano, the stem cell agency's
vice president of portfolio and review. Sambrano has been at CIRM (the California Institute for Regenerative Medicine) for years. He is in charge of the application review process, among other things. (Update: Here is a link to a video of the presentation.)

The CIRM session is aimed primarily at researchers and is expected to deal with some changes dealing with making CIRM-funded data more open along with the new diversity requirements in applications dealing with both potential patient populations and lab/research staffing. 

CIRM plans to award $182 million in the next six months. Based on discussions and action last month by the CIRM board, it would behoove applicants to be well-informed about the details of the new requirements and how they will affect the scoring of grant applications. 

Here are links to the specifics on the changes: discovery/basic research, translational and clinical.

Here are CIRM instructions for joining the webinar that begins at noon PST on Thursday. The Zoom address is https://cirm-ca.zoom.us/s/92287515387. 

Other methods: Phone one-tap, US: +16699006833,,92287515387# or +13462487799,,92287515387#, or join by telephone (for higher quality, dial a number based on your current location) US : +1 669 900 6833 or +1 346 248 7799 or +1 253 215 8782 or +1 301 715 8592 or +1 312 626 6799 or +1 929 205 6099; Webinar ID 922 8751 5387, passcode 210107.

Rudy Giuliani and Covid-19: A California Stem Cell Angle

Capitol Weekly, an online service that covers California government and political news, this morning carried an article dealing with an award last week involving a Covid-19 clinical trial. Here is an excerpt from the freelance piece by yours truly. 

"California’s stem cell agency last week awarded $750,000 to a New Jersey firm to help finance a clinical trial for the firm’s proposed Covid-19 treatment — a therapy that has been hailed by President Trump’s personal attorney as having “real potential.” 

"The firm is Celularity, Inc. Its president and co-founder is Robert Hariri, who describes himself as a longtime friend of Rudy Giuliani, Trump’s personal lawyer. The speedy passage of Celularity’s proposed product through the federal drug approval gauntlet has attracted attention from the New York Times as well as the respected STAT biomedical online news service and other media. 

"Last Friday, directors of the taxpayer-funded stem cell agency, formally known as the California Institute for Regenerative Medicine(CIRM), approved Celularity’s $750,000 application with no discussion. The headline on a STAT article on March 31 said, “Rudy Giuliani wants FDA to fast-track a stem cell therapy for Covid-19; critics see political meddling.”" 

For more details, see Capitol Weekly.

California's Big Research Tent: Beyond Stem Cells and Into VROs

California's $3 billion stem cell research program is unprecedented in state history, and it is now ready to mark another first: Backing a couple of clinical "VROs"  with $13.5 million.

VRO is a term that only a policy wonk could love or perhaps a researcher seeking funding under the rubric. It is not exactly posted on the home page of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. It is nestled comfortably among the 10,000 words of the 2004 ballot initiative that created CIRM.

VRO stands for vital research opportunity. It is fair to say that few persons are aware of the language or know that CIRM's funding authority is something of a big tent that can go well beyond stem cells. Or that CIRM's program could be more expansive under a new, proposed $5.5 billion ballot initiative.

CIRM's governing board set the stage for the awards last November when it approved a VRO process for gene therapy research that did not involve stem cells but involved an aspect of regenerative medicine.

According to the meeting transcript, an application for an award would be considered a VRO if, among other things, "the approach is intended to replace, regenerate, or repair the function of aged, diseased, damaged, or defective cells, tissues, and/or organ. This basically constitutes the definition of regenerative medicine and brings that as a requirement."
 
A VRO designation also requires a two-thirds vote of the grant reviewers, who conduct their meetings behind closed doors.

On Thursday, the board is expected to approve its first two VRO proposals. One for $8 million targets Parkinson's disease. The summary of the review said the research has "the potential to slow disease progression and provide amelioration of motor symptoms."

The other is a $5.5 million award for treatment of a rare autoimmune disease called IPEX. The summary of the review said the research offered "a valuable alternative to the current standard of care options, which have significant toxic side effects."

VRO awards are permitted under the 2004 ballot measure, which says:

"The institute shall have the following purposes: (a) To make grants and loans for stem cell research, for research facilities, and for other vital research opportunities to realize therapies, protocols, and/or medical procedures that will result in, as speedily as possible, the cure for, and/or substantial mitigation of, major diseases, injuries, and orphan diseases."

Currently on file with state election officials is a proposed ballot initiative that would provide an additional $5.5 billion for the agency, which is soon expected to run out of cash for new awards.

The measure would make a number of changes in CIRM, including permitting the agency to venture even farther afield than permitted under the 2004 language.

The measure would impose a new requirement that the agency support training programs for "careers in stem cell research and other vital research opportunities." It would establish experience in "other vital research opportunities" as acceptable criteria for the selection of governing board members and the chair of CIRM. The criteria would apply also to the grant review group and appointments to a new scientific advisory board.

The proposed ballot measure also defines a VRO as including personalized medicine, genetics and aging. Here is the proposed, new language:

"Vital research opportunity means scientific and medical research and technologies, includinq but not limited to qenetics, personalized medicine, and aqinq as a patholoqy, and/or any stem cell research not actually funded by the institute under paragraph (3) of subdivision (c) of Section 125290.60 which provides a substantially superior research opportunity, vital to advance medical science as determined by at least a two-thirds vote of a quorum of the members of the Scientific and Medical Research Funding Working Group (reviewers) and recommended as such by that working group to the ICOC (the governing board),or as determined by the vote of a majority of a quorum of members of the ICOC. Human reproductive cloning shall not be a vital research opportunity."

The language still can be modified by the initiative backers prior to being certified for the November 2020 ballot. Here is how that mechanism works. 

California Stem Cell Agency Posts $54 Million in Good News for Eight Researchers

Eight California researchers may be able to breathe a sigh of relief this morning with the news that the California stem cell agency has enough cash on hand to fund their applications to develop therapies for afflictions ranging from Parkinson's to eye diseases.

All eight of the applications have been approved already by the agency's reviewers. The agency's  governing board very rarely reverses the reviewers' actions. 

Until a day ago, it was uncertain whether the agency had the money to fund all the proposals. The good news for the scientists came in the form of a posting late yesterday on the web site of the agency, formally known as the California Institute for Regenerative Medicine (CIRM). The posting said that the agency has $54.2 million available for new awards as of Wednesday of this week (Oct. 23). The total for the eight application is about $52 million. 

Of course, it is possible that the board could move in a different direction based on considerations not readily apparent from the agenda for its meeting next Thursday. 

If the agency has $2 million or so left after next week's meeting, it is also unclear what it will do with the cash. One possibility is that it could retain the cash for awhile and combine it with funds that might be recovered from awards that have not met milestones.  So far this year, the agency has recovered $30.3 million.

CIRM has expected to run out of cash this year for new awards after dispensing $2.7 billion over the last 15 years to 1,017 recipients. It is hoping voters will approve $5.5 billion more via a ballot initiative in November 2020. 

Thursday's meeting does have another financial fillip. Listed on the agenda is a line that says a report will be made on private donations. That could be good news or not-so-good news depending on how successful its private funding efforts have been over the last couple of years. Results of that drive are yet to be publicly announced. 

(Editor's note for careful readers: The CIRM document that contains the $54.2 million figure also lists $57.2 million in applications. One of those applications, a $5 million proposal, was not approved by reviewers, however.) 

California Stem Cell Researchers Compete Next Week for $52 Million in Awards

Directors of the California stem cell agency will make yes-or-no decisions next week  on eight applications for $52 million to seek cures for afflictions ranging from Parkinson's to an incurable eye disease called retinitis pigmentosa.

All eight have been approved behind closed doors by the agency's grant reviewers, but it is yet to be determined whether the agency has enough cash to ratify that action.

Prior to voting on the applications, the 29-member board is scheduled to discuss how the agency's final funding will be parceled out. The agency expects to run out of cash for new awards at the end of the Oct. 31 meeting. However, also on its agenda are a few words promising a report on the status of its search for private donations. 

Below is a table on the applications. It includes links to the review summaries prepared by the agency, which is formally known as the California Institute for Regenerative Medicine. Links to the review summary can be found on the application numbers.

Some of the applicants have filed additional letters with the board seeking to bolster their pitches. The letters can be found by clicking on the researcher's name. Some of the applicants are expected to address the board personally Oct. 31 in Oakland.

The agency has a non-embarrassment policy of not disclosing the names of applicants and their institutions until a vote by its board.  However, when they write the board, their names become a public record. The names of applicants who fail to win approval are never disclosed by the agency.

Look for more stories in the upcoming days from the California Stem Cell Report on the agency's Halloween meeting.

Application Number	Amount In millions	Institution	Principal  Investigator	Target
CLIN1-11591	$5.5	Stanford	Rosa Bacchetta	IPEX
CLIN1-11472	$6.6	Not disclosed	Not disclosed	Retinitis pigmentosa
CLIN2-11620	$10.5	Not disclosed	Not disclosed	Retinitis pigmentosa
CLIN2-11661	$8.0	Brain Neurotherapy Bio	Krystof S. Bankiewicz	Parkinson’s Disease
CLIN2-11650	$10.3	UCLA	Sophia Deng	Limbal stem cell deficiency
TRAN1-11536	$4.9	UCLA	Caroline Y. Kuo	Immune deficiency
TRAN1-11555	$3.2	UCLA	Yvonne Y. Chen	Myeloma
TRAN1-11544	$2.9	Not disclosed	Not disclosed	Ovarian cancer

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."

California Stem Cell Agency Moving to Expand its Reach into Big Market for Gene Therapy

The definition of gene therapy under proposed changes for
 research funding by the California stem cell agency. CIRM chart

California's $3 billion stem cell research program is set to add gene therapy -- minus stem cells -- as research that it will be able to legally finance. 

The change comes as the stem cell agency is looking to generate results that are likely to resonate with voters in November 2020 who may be asked to provide an additional $5 billion in funding for the program. The agency expects to run out of cash by the end of next year. 

Gene therapy has received considerable attention in the last few years. Yesterday, Orchard 
Therapeutics, a British gene therapy firm that has links to CIRM (also see here) and research by Donald Kohn of UCLA, raised $200 million in an initial stock offering. The company said in its prospectus that the total market potential "in the diseases areas underlying our five lead programs could be greater than $2 billion annually."

Donald Kohn, UCLA photo

Kohn has received nearly $31 million in backing from the stem cell agency, which is formally known as the California Institute for Regenerative Medicine (CIRM).

The Science Subcommittee of the agency is expected to approve extension of its gene therapy efforts next Wednesday in a teleconference meeting that will be available globally through the Internet. 

Under the provisions of the ballot measure that created the agency in 2004, the agency is limited in scope. But exceptions are possible if a finding is made that a "vital research opportunity" exists. 

Next week's meeting is expected to formalize the process of making that determination in regards to gene therapy that does not involve stem cells. It will require a 2/3 vote of CIRM's grant review group, among other things. 

Members of the public can participate in the hearing remotely via the Internet or at locations in Oakland, San Francisco, La Jolla, Riverside and Napa. Directions can be found on the agenda.

Stem Cell Suspense to End Dec. 5 for Four California Researchers

Four California stem cell researchers could well receive a total of $16.6 million in awards next week from the Golden State's 12-year-old stem cell program.

The researchers were caught in a bit of a snafu earlier in November when the $3 billion agency appeared to come up $1.6 million short. The story about the snag also involved quorums, priorities and fiscal discipline, more than is necessary to discuss right here in this item. But you can read all about it here. 

The two researchers identified so far are Yadong Huang of the Gladstone Institutes, Jill Helms of Stanford and Ankasa Regenerative Therapeutics of La Jolla.  The identities of the others are being withheld by the stem cell agency.

Whether all four will receive funding will be determined on Monday Dec. 5 when the stem cell agency's board will hold a telephonic meeting out of its base in Oakland. The agenda gave no indication of how the agency plans to overcome the difficulties that stymied it on Nov. 17.

The public can attend the meeting at the agency's headquarters in Oakland or from telephonic locations in Napa, Beverly Hills, Elk Grove, Los Gatos, Irvine, Sacramento and two each in South San Francisco and San Diego.

The session is also expected to be audiocast on the Internet. More information and addresses can be found on the agenda.

California Stem Cell Research and a Super Doc: Inside a $4.4 Million Windpipe Grant at UC Davis

Paolo Macchiarini, Guardian photo 

Headlines around the world once hailed Paolo Macchiarini as a super-surgeon, a stem cell trailblazer who was responsible for the ground-breaking, first-ever stem cell-based trachea transplant.

It was good enough work, indeed, to be cited in 2013 as a starting point in a pitch by a team from the University of California, Davis, for $13 million from the $3 billion California stem cell agency. 

In their application, scientists Peter Balafsky and Alice Tarantal said they would build on the "first-in-human surgical successes with (the) stem cell-based tissue engineered airway implants" pioneered by Macchiarini

Last month, Macchiarini was fired from the prestigious Swedish Karolinska Institute. Six of eight of his patients have died. The institute said,

 "He has acted in a way that has had very tragic consequences for the people affected and their families."

The controversy has stirred up the international stem cell community with blog postings and sharp accusations. And in January, Vanity Fair carried an article that reported Macchiarini falsely claimed he was part of a “highly classified group of doctors from around the world who cater to the world’s VIPs,”including Pope Francis, Bill and Hillary Clinton, Emperor Akihito of Japan and President Obama.

The Italian doctor has denied the charges that led to his dismissal in Sweden and has been working in Russia.

The Macchiarini saga and its California connections offer a peek into the global nature of stem cell research and how scientists must rely on the integrity of others thousands of miles away --  as well as  the sometimes agonizingly slow search for cures. It also provides a deeper look at how the California stem cell agency goes about handing out money.

The California Stem Cell Report queried both Balafsky and Tarantal about their grant along with the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known.  

Kevin McCormack, senior director of communications, replied for CIRM. Asked whether the agency looked into the research by Belafsky as a result of the questions raised about Macchiarini, McCormack said,

"As with all the research we fund we have been carefully following the progress of Professor Belafsky’s project ensuring it continues to meet our rules and regulations."

Peter Belafsky, UCD photo

Belafsky, professor and director of the Voice and Swallowing Center at UC Davis, and Tarantal, professor in the Department of Pediatrics and Department of Cell Biology and Human Anatomy, said Macchiarini is not involved in the CIRM project. The point was stressed in boldface letters in their email response:

"Dr. Macchiarini has had no involvement in this project on any level, in any way, at any time."

(For the full text of the response, see here.)

Macchiarini was not always isolated from UC Davis and Belafsky. Macchiarini served on Belafsky's international team in 2010 when the Belafsky group performed the second-ever larynx transplant. The operation restored the voice of a Modesto, Ca., woman who had been unable to speak for a decade. 

UC Davis issued a press release saying Macchiarini served as an advisor and assisted in the surgery. The statement also said that Martin Birchall of the United Kingdom, a co-leader with Macchiarini on his ground-breaking 2008 transplant surgery, served as a scientific advisor and assisted with the California larynx surgery.

Birchall was identified as a collaborator on the CIRM grant that was approved for Belafsky and Tarantal.  A press release from University College London said that the work in Davis would serve as a "fundamental under-pinning" for two clinical trials in the United Kingdom.

Birchall received a $19,800 planning grant in 2011 to prepare an application for research that appears to be aligned with the current work at UC Davis. Birchall was listed by CIRM at the time as a researcher at the California campus.

Belafsky and Tarantal told the California Stem Cell Report that Birchall is no longer involved with their work. They said,

"Dr. Birchall was involved in our project at the onset, but due to the logistical difficulties of intercontinental collaboration, is not currently an active participant."

The Belafsky/Tarantal application originally sought $13.3 million. It was trimmed to $4.4 million on the recommendation of the agency's then president, Alan Trounson, who apparently agreed with reviewers that the initial scope and budget were high, but still recommended funding.

The application was given a score of 70 on a scale of 100 by agency's blue-ribbon scientific reviewers, all of whom came from out-of-state. As is their usual practice, the reviewers met behind closed doors and voted on the application. The action then went to the CIRM governing board, which has almost never rejected a favorable decision by its reviewers, whose economic and professional interests are not disclosed publicly.

The score of 70 placed the proposal just below the cutoff line of 75 for routine approval by the board, but the reviewers did not nix application.

The agency publishes a summary of reviewer comments, which does not identify applicants or their collaborators. The summary on the UC Davis application said the research "presents a unique opportunity to bring a world-leading regenerative medicine technology to California." The summary additionally said, without elaboration, "It was also noted that this project is unlikely to be funded by other agencies."

The summary said,

"Reviewers agreed that having already treated human patients (such as those involved with Macchiarini) using this approach is strong proof of concept(for Belafsky's work)."

Reviewers noted that "manufacturing and testing methods were not well described" but "the UK-based collaborators (Barchall and his group) will clearly play an important role in helping to establish the manufacturing process."

The summary added,

"Reviewers were unclear on the relationship between the California- and UK-based team members and whether the relationship and efforts were collaborative or duplicative."

Belafsky appeared before CIRM directors in Los Angeles in December 2013 to ask them to approve his research, saying good treatments for "complex breathing and swallowing problems" do not exist. With little debate, the board voted 8-0 to approve the award.

CIRM's McCormack said that the agency has paid out $3.3 million of the $4.4 million grant. He said, 

"Dr. Macchiarini has never been a part of the UC Davis project or any work that CIRM has funded.... The primary aim of the CIRM project is to determine the scientific reproducibility of research results from previously published studies regarding tissue-engineering for severe airway stenosis. The results are pending."

Belafsky said in his email,

"The translation of high-risk, complex innovation from the laboratory to suffering patient is not an easy road. The controversy surrounding Macchiarini has resulted in the redoubling of our efforts to explore the science behind what saves lives and what does not. This step is essential in order to lay a firm, evidence-based foundation upon which to build.

"Since your primary focus relates to the work of Macchiarini, I would like to re-emphasize that our project is fundamentally different. Our research is focusing on decellularized trachea only, whereas he has utilized synthetic grafts."

Belafsky continued,

"This is a work in progress, and we do not yet have the data to answer all the questions raised by the scientific community. We may apply to CIRM for funds to continue our research, but will not consider human implantation until we are satisfied that the science is sound and the technology is safe."

Text of Belafsky-Tarantal Reponse

Here is the text of the response from scientists Peter Belafsky and Alice Tarantal of UC Davis concerning questions asked by the California Stem Cell Report. The questions first and then the response.

The questions

"Has Macchiarini been involved in any way, including informal consultation or contact, with your CIRM award? If so, please describe the nature of that contact.

"What is the nature of the collaboration with Birchall?

"Have the disclosures concerning Macchiarini altered your direction or delayed your CIRM research?

"Has UCD inquired concerning your CIRM research in the wake of the Macchiarini disclosures? If so, what is the status of that inquiry?

"What is the current status of your research? The CIRM progress report on its web site appears to be old, and you are nearing the final stages of the term of the award.

"Do you plan to apply to CIRM to advance your research into a clinical trial?

"Is there anything else that would be useful for the public to know?"

The response

"Hi, David.

"I appreciate your inquiry and dedication to advancing regenerative medicine. I am a head and neck surgeon specializing in the care of patients with complex airway collapse. In June of 2008 an international team performed what was then considered to be the world’s first tissue-engineered organ transplant to help save a suffocating mother who was failed by contemporary medicine. The team took a donor trachea stripped of innate cells, implanted it with autologous stem cells, and transplanted it into her failing windpipe. The operation was a success, and the patient is alive and well. Based on this achievement, similar technology was used in 2010 to save the life of a 10 year-old boy; he is now a thriving 15 year-old.

"Since the initial success with decellularized/re-cellularized human donor trachea, work on synthetic trachea implanted with autologous stem cells has not shown equal promise, and people have died. We have had no involvement in these procedures or investigations.

"An article published in 2013, by Gretchen Vogel (Science. 2013 Apr 19;340(6130):266-8), questioned the success of this work and urged caution before moving the science forward. She and others, our team included, have suggested that more research is required to determine the fate of the cells and the long-term viability of the grafts before making the implants widely available to patients. Our team at UC Davis is uniquely qualified to address these questions and we coordinated the CIRM proposal to fund the research to critically evaluate the donor trachea method. Our university’s support in this endeavor has not wavered.

"The ultimate goal of our work is to provide better treatment options for people with life-threatening airway stenosis. The primary aim of our CIRM grant is to take a step back and understand what happens with the grafts in the lab before initiating operations in people. This is a work in progress, and we do not yet have the data to answer all the questions raised by the scientific community. We may apply to CIRM for funds to continue our research, but will not consider human implantation until we are satisfied that the science is sound and the technology is safe.

"To answer your questions, Dr. Birchall was involved in our project at the onset, but due to the logistical difficulties of intercontinental collaboration, is not currently an active participant. Dr. Macchiarini has had no involvement in this project on any level, in any way, at any time. (Belafsky's boldface italics)

"The translation of high-risk, complex innovation from the laboratory to suffering patient is not an easy road. The controversy surrounding Macchiarini has resulted in the redoubling of our efforts to explore the science behind what saves lives and what does not. This step is essential in order to lay a firm, evidence-based foundation upon which to build.

"Since your primary focus relates to the work of Macchiarini, I would like to re-emphasize that our project is fundamentally different.  Our research is focusing on decellularized trachea only, whereas he has utilized synthetic grafts.

"Warm regards,

"Peter Belafsky, MD, PhD

"University of California, Davis"

Attacking Barriers to Stem Cell Cures: California to Put Official Stamp on New Strategy

Call it California’s $900-million road map to a stem cell cure. 

That’s what up for final action next week by the governing board of the state’s $3 billion stem cell agency, officially known as the California Institute for Regenerative Medicine (CIRM).

The plan includes a $105 million lure to entice Big Pharma and other possible investors to join the Golden State’s regenerative medicine bandwagon and create an “industrial stem cell therapeutic powerhouse.” 

Randy Mills, president of the California stem cell agency, and his team are calling for “attacks” on barriers to clinical development of therapies. That includes the FDA which Mills and company say “appears to be literally lobbying against the very therapeutic modality they are responsible for promoting.” (See here and here.)

The key part of the $105 million stem surge would bring together next year the state of California and private investors in a joint enterprise. Under the plan, the private investors would have the pick of the best research from CIRM that has not already attracted partners.

The agency would pony up $75 million with another $75 million coming from investors.

(Here are links to the CIRM description of the three components of the $105 million effort: here, here and here.)

The agenda for the Dec. 17 meeting in Los Angeles includes other matters, such as action on a clinical research applications for millions of dollars. The review summaries of those applications are not yet available online, but most of the additional supporting material for the meeting has been posted. That is a healthy change from some recent past meetings where backup information has been missing until much too late.

(The morning of Wednesday Dec. 9, the agency posted a note on the agenda saying the applications were no longer under consideration. Often that means that the proposals have been withdrawn because of negative recommendations from reviewers.)

Also on tap is a major change in the scoring of applications in non-clinical programs. Here is how the change is described by a CIRM memo:

“For non-Clinical Program applications, therefore, we propose to revert to our former scoring system (1 to 100) with two tiers: (Tier 1) average score of 85 or above, recommended for funding, if funds are available, and (Tier 2) average score of 84 or below, not recommended for funding. In  addition, for those programs for which only one application is expected to be funded, we propose to specify that the application that receives the highest average scientific score shall be deemed to be the GWG’s recommendation for funding.”

These changes would be in effect for the three rounds involved in the $105 million surge.

The board is also being asked to raise the cap on payments to patient advocate members of the board from $15,000 to $30,000 annually. The move would be retroactive to the beginning of this year. 

A CIRM memo said that demands on patient advocate directors have increased dramatically both in terms of the numbers of meetings and their role. The memo said that prior to Mills’ arrival at CIRM patient advocates were involved in only three or four review sessions a year. In 2015, those sessions have already risen to close to a dozen.

While next week’s meeting will be based in Los Angeles, remote, telephonic locations where the public can participate will be located at Stanford and UC San Diego. Specific addresses can be found on the agenda.

Inside the Story of Cesca Therapeutics, the California Stem Cell Agency and its Kibosh of an $11 Million Proposal

The Sacramento Bee today carried a story taking a longer look at Cesca Therapeutics, Inc., its travails with the California stem agency and the firm’s application for $11 million to help finance a phase three clinical trial.

The freelance article was written by the publisher of this blog, David Jensen. Here is the full text of the story.

  Rancho Cordova firm comes up empty in bid to win $11 million from stem cell agency _ The Sacramento Bee

California to Nix $6.8 Million for Parkinson's Research, Flawed Proposal Cited

Directors of the California stem cell agency are scheduled to meet Thursday to reject a $6.8 million application to finance late stage research to develop a therapy for Parkinson’s Disease.

Twelve of the agency’s blue-ribbon, scientific reviewers last month voted unanimously against the proposal, declaring it was “flawed” and should not be resubmitted to the agency. Directors of the agency almost never overturn negative decisions of its reviewers.

This week’s meeting focuses unusual attention on the application. It is the only one scheduled to be considered at the meeting, which is part of the fast-track funding efforts this year by the stem cell agency. In the past, final approval or rejection of applications usually involved a number of applications. Reviewers’ decisions against funding have been rubber-stamped with no discussion. Scores on the rejected applications were not disclosed.

One of the goals of the agency’s “CIRM 2.0” effort, in addition to speeding funding, is to improve grant applications. It may well be that the agency is attempting to reinforce, via the application review summary, its message to researchers to step up the quality of their proposals for millions of dollars in taxpayer support.

The review summary found considerable fault with the rationale of the proposal. It said the data presented was “insufficient.” The dosage involved was “not clearly described.” Timelines may be “unrealistic,” the document declared.

The summary also said,

“It is not clear whether the focus of the applicant is an embryonic (ESC) or induced pluripotent stem cell (iPSC) derived product.”

“Reviewers....encouraged the (applicant) team to carefully consider feedback in any earlier stage application.”

The name of the applicant was not disclosed in keeping with the agency’s longstanding practice of withholding such information. Responding to a question, an agency spokesman said the application came from an academic institution. One Parkinson’s researcher who has been mentioned in the news in recent months, Jeanne Loring of Scripps, told the California Stem Cell Report that the application did not involve her. 

The rejection would be only the second in the CIRM 2.0 program, according to Kevin McCormack, senior director of communications for the agency. 

The public can weigh in on any subject during Thursday's meeting at teleconference sites in Beverly Hills, San Diego, South San Francisco, Napa, Irvine,, Los Gatos, Elk Grove, Redwood City, San Francisco and two in Sacramento. Specific addresses can be found on the agenda.