California Stem Cell Agency to Award $24 Million for Three Clinical Trials

If all goes according to plan, in just three days the California stem cell agency will be down to its last $69 million for clinical trials.

Directors are set to give away $24 million on Wednesday for three clinical trial programs as the agency runs out of cash this year for new awards. The agency started with $3 billion 14 years ago. It has allotted $144 million for research awards this year.

The agency, known formally as the California Institute for Regenerative Medicine (CIRM), is attempting to raise privately more than $200 million this year. It is also hoping that voters will approve $5 billion more in funding in the November 2020 election. 

Applications coming before directors this week involve $6 million for Huntington's disease, $6 million for immunodeficiency studies and $12 million for renal failure research. 

The agency withholds the names of researchers and institutions involved until directors act publicly.  The agency's reviewers earlier approved the applications during a closed door session. The agency board almost never overturns the decision of its reviewers. 

The public can participate in the teleconference meeting via the Internet. Instructions can be found on the meeting agenda along with summaries of the reviewers comments on the applications.

CIRM was created by California voters in 2004 and provided with $3 billion in bond funding. No other significant source of income was provided under the ballot initiative.

Correction: An earlier version of this item that was up briefly incorrectly described the $69 million as all that remained for all awards this year.    

California Kicks Off Major, New Foray into Gene Therapy: First by Any State in the Country

California today became the first state in the nation to launch itself into the sizzling field of gene therapy, backed by tens of millions of dollars and with the hope of creating treatments that could permanently cure afflictions ranging from hemophilia to cancer.

The move came today as the board of the $3 billion California stem cell agency opened its doors to funding gene therapy research that has reached the most advanced stage, clinical trials. The agency said that gene therapy -- minus stem cells -- is "valuable and worthy of pursuit."

"This is where the science is going," said Jeff Sheehy, chairman of the CIRM board's Science Subcommittee, at hearing earlier this month,

The state stem cell program has allocated $143 million for research programs next year that could include gene therapy.

A document prepared by the leadership of the agency, formally known as the California Insitute for Regenerative Medicine (CIRM), said, 

"For CIRM and the patients it aims to serve, it is vital to support technologies which prove to be highly complementary and augmenting to stem cells, such as gene therapy."

Gene therapy treatments are expected to be quite expensive, with some forecasts running in the $1 million to $2 million range. Supporters of gene therapy argue that the cost is justified because gene therapy can be a total cure that would eliminate the need for also very expensive lifelong treatments of chronic diseases. 

In its action today, the stem cell agency did not announce any specific research awards. Rather it created a procedure for declaring that a gene therapy project with a regenerative element is a "vital research opportunity."  That would allow CIRM to fund such a project under the terms of the ballot initiative that created the agency in 2004. Today's action is the first time that the agency has acted to use the "vital opportunity" provision. 

CIRM has budgeted $143 million for next year in two areas where the new gene therapy initiative could come into play, clinical trials along with translational research that is intended to advance basic research into clinical stages. 

The agency's new foray comes as its cash is running out. By the end of next year, it expects to have no more funding for new research. The agency is pinning its hopes of survival on a $200 million plus private fundraising effort this year and voter approval of a yet-to-be-written ballot initiative on the November 2020 ballot. 

The gene therapy field is moving swiftly and could generate a result that would resonate with voters and help to win approval of an additional $5 billion for CIRM.

CIRM board members have acknowledged that its new gene therapy effort will mean more competition for the state's research dollars, including possibly less for stem cell research, which is the agency's fundamental reason for being.

The agency, however, has already awarded millions of dollars for gene therapy research that has a stem cell link, including a program at UCLA that has led to a successful treatment for what is known as the "bubble boy syndrome." However, that research has not yet moved into the marketplace.

The target of the UCLA treatment is an immune deficiency that is fatal and for which there is no successful treatment outside of the experimental trials that are still underway. 

Bloomberg News has reported that Scott Gottlieb, head of the federal Food and Drug Administration(FDA), has described the field as "somewhat breathtaking." More than 500 experimental treatments are in the pipeline. 

Gottlieb has said that he expects the FDA to approve 40 gene therapies by 2022 and possibly a cure for sickle cell anemia within 10 years. CIRM is deeply involved in a major national push on sickle cell supported by the National Institutes of Health.

California's Stem Cell Agency Opening Door to Pumping More Millions into the Sizzling Gene Therapy Market

Gene therapy graphic from FDA

The California stem cell agency today crossed a key threshold into the "somewhat breathtaking" and potentially multibillion-dollar world of gene therapy, a field that it has skirted previously. 

The action came when a key panel of the agency's governing board this morning approved its first-ever procedure for awarding millions of dollars for research not connected to stem cells. 

"This is where the (regenerative) science is going," said Jeff Sheehy, chair of the Science Subcommittee of the board of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known. 

Under Prop. 71, the ballot initiative that created the $3 billion agency in 2004, CIRM is limited to financing research involving stem cells in some fashion. However, a provision of the measure also allows the support of a "vital research oppportunity" under certain conditions. Today's action formalizes the process for making that finding. 

CIRM said in in a document that it is "vital" that the agency expand its horizons.

"CIRM has supported projects that combine stem cell and gene therapy technologies, such as the gene-corrected stem cell transplants at UCLA that essentially cured 5-year old Evangelina Padilla Vaccaro and several CAR-T cell approaches using stem memory T cells that aim to tackle various cancers. 

"The support of stem cell research that contributes to these treatments is and will continue to be the core of CIRM funding. However, treatment opportunities in regenerative medicine that utilize gene therapy technologies but not necessarily stem cells are also valuable and worthy of pursuit."

The agency's statement continued,

"The field of regenerative medicine brings together technologies that include stem cells, gene therapy, and tissue engineering that in many cases combine to produce a therapeutic product. In some cases, one technology leads the way. For CIRM and the patients it aims to serve, it is vital to support technologies which prove to be highly complementary and augmenting to stem cells, such as gene therapy."

Several directors noted during today's meeting that the change could lead to less cash for purely stem cell projects. 

The field of gene therapy has attracted widespread interest within the regenerative medicine industry and among federal medical regulators. Scott Gottlieb, commissioner of the Food and Drug Administration, earlier this year announced steps to speed development of the therapies. 

Bloomberg News reported:

"'The pace of progress in gene therapy has been somewhat breathtaking,' he (Gottlieb) said, with more than 500 experimental drugs now in development. 'The promise is becoming very much a reality.'"

In April, Novartis AG ponied up $8.7 billion to buy AveXis Inc., of Illinois to strength its efforts to produce a marketable gene therapy.

Gottlieb has said that he expects the FDA to approve 40 gene therapies by 2022 and possibly a cure for sickle cell anemia within 10 years. 

CIRM's new gene therapy policy will go before the full CIRM board one week from today where it is expected to be approved. Additional information on the policy can be found on the Science Subcommittee agenda. 

Solomon and Stem Cells: California's Stem Cell Agency Is Slicing and Dicing Cash and Research Decisions

California's stem cell agency gave away $14 million this month, which could be described as less than a drop in its $3 billion bucket.

But the talk at the agency's awards meeting July 19 was not about largess. Instead it was about the lack of cash, lack of time and the need to split "babies" and "buckets."

"We are going to have to make some hard choices today," said one member of the agency's governing  board, Jeff Sheehy, a patient advocate member of the board for HIV.

"We are coming to the end," said another, Oswald Steward, director of the Reeve, Irvine Research Center at UC Irvine.

Sheehy and Oswald and others referred to the shrinking finances of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known. It expects to run out of cash for new awards by the end of next year. Its leaders are trying to raise more than $200 million privately to tide it over until November 2020. That's when they hope another ballot initiative/bond measure will be approved by California voters and pump an additional $5 billion into the 13-year-old enterprise.

That was the backdrop at the session earlier this month. It was a meeting that illuminated the changing criteria that the agency is beginning to use  -- as the cash squeeze tightens -- to make decisions on who and what to finance. No longer will patient advocates or researchers be able to assume that an excellent scientific score assures that an application will be ultimately approved. Instead, "programmatic" considerations will play an important and perhaps definitive role.

Those considerations range from whether a researcher has received past funding from the agency, whether the application fits well within the agency's mission, whether an application fills a void in the agency's portfolio and just how innovative the research is, plus much more.

The discussion about diminishing resources arose when directors were given the usual list of applications that were approved for funding by its reviewers, who met earlier behind closed doors.  Normally, the reviewer decisions are rubber-stamped. This time, however, the 14 reviewer-approved applications totaled $19 million, but only $10 million was budgeted. That's when some directors brought up the need for Solomon-like decisions that would "split" the babies and the "buckets" of cash.

One upshot was that two applicants, James Hagood of UC San Diego and  Vittorio Sebastiano of Stanford, received only partial funding with the expectation that the board would approve a revision in the budget in late October. Nonetheless, those researchers were better off than six applicants who were also approved for funding by reviewers, but not by the CIRM directors. Final action on their applications was put off, probably also until late October.

The $10 million round involved basic types of research, but with a two-year time table. Directors also took action in another, separate round involving preclinical work. It dealt with only one application for $3.99 million, which was approved with no discussion. It went to Poseida Therapeutics, Inc., of San Diego for research aimed at supercharging  "a patient’s own immune system cells to attack and kill a treatment-resistant form of prostate cancer," according to the CIRM news release on all the awards. Poseida matched the award with $998,023.

It was the second CIRM award for Poseida, bringing its total from the agency to $23.8 million.  Here is a link to the summary of comments from reviewers on the Poseida application.

Below is a list of the other approved awards, all of which went to institutions that have links to a CIRM governing board member. Those members were not allowed to vote on those applications. Here is a link to a document which contains summaries of the reviewer comments on each application as well as ones that were not funded on July 

Application number	Title	Institution	Amount	Principal Investigator
DISC2-11131	Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease	UC San Diego	$1,393,200	Eric Adler
DISC2-11157	Preclinical Development of An HSC-Engineered Off- The-Shelf iNKT Cell Therapy for Cancer	UCLA	$1,404,000	Lili Yang
DISC2-11036	Non-viral reprogramming of the endogenous TCRα locus to direct stem memory T cells against shared neoantigens in malignant gliomas	UC San Francisco	$900,000	Hideho Okada
DISC2-11175	Therapeutic immune tolerant human islet-like organoids (HILOs) for Type 1 Diabetes	Salk Institute	$1,637,209	Ronald Evans
DISC2-11107	Chimeric Antigen Receptor-Engineered Stem/Memory T Cells for the Treatment of Recurrent Ovarian Cancer	City of Hope	$1,381,104	Saul Priceman
DISC2-11165	Develop iPSC-derived microglia to treat progranulin- deficient Frontotemporal Dementia	Gladstone Institutes	$1,553,923	Li Gan
DISC2-11192	Mesenchymal stem cell extracellular vesicles as therapy for pulmonary fibrosis	U.C. San Diego	$865,282	James Hagood
DISC2-11109	Regenerative Thymic Tissues as Curative Cell Therapy for Patients with 22q11 Deletion Syndrome	Stanford University	$865,282	Vittorio Sebastiano

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California Stem Cell Agency Eyes Changes in Funding Decisions; Possible Impact on Bond Election, Development of Different Therapies

A CIRM slide outlining current programmatic criteria. GWG
refers to the group that reviews applications.  The
 subcommittee reference is to the panel of directors  who
ratify reviewers' decisions. ICOC is the abbreviation for
the name of the governing board.

The $3 billion California stem cell agency is re-examining its criteria for awarding hundreds of millions of dollars with an eye to placing more emphasis on what could be called non-scientific criteria.

The move could have an impact on hundreds of researchers in the state and the development of stem cell therapies that could benefit untold numbers of patients afflicted with a host of deadly and debilitating diseases. It could also have an impact on a possible ballot measure to provide an additional $5 billion for the 13-year-old stem cell program.

The changes could be acted on as early as tomorrow at a meeting of the governing board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. The meeting is in Oakland, but Internet access is available for those who wish to comment and hear the proceedings.

The move comes under the rubric of "programmatic review" of applications for funding. It has been an ill-defined term for years at the agency. But more specificity was disclosed yesterday in a series of 20 slides scheduled to be shown at tomorrow's meeting of the agency's 29 directors. The posting of the slides came less than two days prior to the meeting.

The agency's staff has laid out seven possible areas where changes might be made:

• "Annual Program Budget and Goals
• "Value Proposition of Proposed Project
• "Patient population, competitive landscape
• "Relevance of Project to Stem Cells
• "Contribution to CIRM Portfolio
• "Disease area, current award overlap
• "Previous CIRM Support of Project"

The full impact of increased use of any or all of those criteria was not clear from the slides provided by the agency. But it could mean that an application that received a high scientific score could be sidelined in favor of one that fills a void or bolsters a weak spot in the CIRM award portfolio.

CIRM slide on possible new award criteria 

Over the years, many CIRM board members have expressed frustration with how the scoring on some applications works. In the case of some applications, only one point separates those receive millions and those who receive none. The concern has been that a one point difference is less than meaningful.

The slides do not flesh out all the likely reasons for putting more emphasis on non-scientific issues, but the agency is approaching the end of its life. It expects to run out of cash for new awards at the end of next year.

A private fundraising effort is underway to tide the agency over until, it is hoped, voters approve $5 billion more for the agency in November 2020.

Changes in award criteria could lead to approval of research whose results are more likely to resonate with voters in time for a ballot measure campaign in two years.

CIRM was created in 2004 by voters who were swayed by a campaign that raised expectations that stem cell cures were just around the corner. The agency has yet to produce a therapy that is available for widespread use. However, it has helped to fund 49 clinical trials, which are the last stages before a therapy is approved by the federal government for general use.

California Stem Cell Agency Steps Up Public Access to Key Meeting This Week; Brain Cancer and Parkinson's On Tap

Stem cell researchers and the public -- from wherever they are around the world -- will have a chance Thursday to take part remotely for the first time during a meeting of the governing board of California's $3 billion stem cell agency.

The session will include approval of two awards totaling $9.5 million, approval of a $16.8 million operating budget, approval of a revised conflict of interest code along with a quasi-mysterious matter dealing with "programmatic tools."

At the time of this posting, the agency, formally known as the California Institute of Regenerative Medicine (CIRM), had not added online any background material supporting the cryptic, agenda item. However, the general subject deals with decisions that the board makes on applications for funding. In addition to scientific considerations, the board can base its decisions on non-scientific matters, such as whether the application really fits with the CIRM program.

(After this item was posted, the agency added to the agenda a number of slides dealing with the grant-making process and use of programmatic criteria. The California Stem Cell Report will carry an item on those slides tomorrow. Update: That item has now been posted.)

Interested persons will be able -- for the first time -- to make comments not only that matter but others from wherever they are. But they must be logged on to the audiocast/webcast of the session in order to be recognized. The agenda contains directions on how to do that. During the meeting, online participants will be asked if they have a comment. We recommend setting up the connection in advance of the meeting. It may require some tweaking, depending on your computer figuration.

The agency has successfully tested the new public comment feature on a few committee hearings. This will be the first experience with a full board meeting.

The awards expected to be made include a  $5.8 million application for Parkinson's disease and a $3.7 million application for glioblastoma, a form of brain cancer. Reviewers, meeting behind closed doors, have already approved the awards. The board must ratify their decisions in public. (Here are links to review summaries on the Parkinson's application and the glioblastoma application, plus comments by a Scripps Research Institute scientist on the Parkinson's application.

John Zaia of the City of Hope wrote a letter to the board commenting on the public summary of his glioblastoma application, critiquing reviewer comments. He said,

"Based on our experience with two previous FDA-approved pilot trials, we foresee no major hurdles to completing an IND, recruiting patients, manufacturing the therapeutic cell product, and eventually conducting the clinical trial."

The agency does not release the names of applicants until action is taken by the board, but when a letter is written by an applicant, his/her name becomes public along with the letter.

The proposed budget for the 2018-19 year stands at $16.8 million, up 7 percent from an estimated spending of $15.7 million for this fiscal year. The budget for research awards is separate and has been normally set in December. However, some board members have indicated that are are interested in trimming the amount of awards to be given annually in order to extend the life of the agency.

Under current spending rates, the agency expects to run out of money for new awards by the end of next year. It is pinning its hope for survival on a private fundraising effort now underway and voter approval of a proposed $5 billion bond measure in November 2020.  An update on the fundraising effort may be presented Thursday by CIRM board chairman J.T. Thomas.  

Diabetes to Arthritis: California Awards $33 Million for Clinical Stage Stem Cell Work

The California stem cell agency today approved nearly $33 million for clinical stage research projects testing treatments for type 1 diabetes, arthritis of the knee, ALS and an immunodeficiency affliction.

The awards were quickly approved with little discussion during a meeting at the Oakland headquarters of the California Institute for Regenerative Medicine or CIRM, as the agency is formally known.

The award likely to have an impact on the most people -- if it is successful -- is a relatively small, $2.3 million award to the Cellular Biomedicine Group,  a Chinese firm with operations in Cupertino, Calif. The stem cell agency by law only finances work in California. The research would also be supported by $572,993 in co-funding.

The project is aimed at treating osteoarthritis of the knee. More than 51 million people in the United States suffer from arthritis, which is particularly common in the knee.

The goal of the research is to regenerate knee cartilage through the use of a mesenchymal progenitor cell treatment, according to the agency's application review summary. The funding would go to manufacture the product and complete work to secure Food and Drug Administration approval for a phase one safety trial. A treatment for the public would likely be years in the future.

Here are the other winners today of California stem cell cash with links to the summaries of the reviews.

Caladrius Biosciences of New Jersey won $12.2 million for a clinical trial for young people ages 12-17 for newly diagnosed type 1 diabetes. The firm plans to use regulatory T cells from the patients themselves to treat the disease. Caladrius has a California location in Mountain View. (Caladrius' press release can be found here.)

St. Jude's Research Hospital in Memphis, Tenn., was awarded $11.9 million for a phase one/two trial to treat infants with X-linked severe combined immunodeficiency. The trial would aim at enrolling at least six patients suffering from the catastrophic affliction. The treatment would use the patients own bone marrow stem cells after the cells were specially handled. The agency said in a press release that St. Jude's is working with UC San Francisco. (St. Jude's press release can be found here.)

Cedars-Sinai Medical Center in Los Angeles was awarded $6.2 million for a phase 1/2A trial to test a treatment for ALS, which has no treatment or cure. The CIRM review summary said a "huge unmet need" existed. About 20,000 persons in the United States suffer from the affliction.

CIRM's press release did not identify the researchers involved in any of the awards.

The agency is on a push to support more clinical trials, which are the last and most expensive research prior to the possibility of winning federal approval for widespread use of a therapy.

Currently the agency is participating in 27 trials and is planning on adding 37 more in the next 40 months. The agency is expected to run out of funds for new awards in June 2020 and has no source of future financing.

The awards were previously approved behind closed doors by the agency's out-of-state reviewers, who do not disclose publicly their economic or professional interests. The agency's directors rarely overturn a positive decision by the reviewers.

All of the winners have links to two or more members of the 29-member CIRM governing board. Those members are not allowed to vote on applications where they have conflicts of interest.
About 90 percent of the funds awarded by the board since 2005 have gone to institutions that have ties to members of the board, past or present, according to calculations by the California Stem Cell Report.

(Editor's note: An earlier version of this item incorrectly said the total amount of awards was $37 million.)

Search for a Diabetes Cure: California Stem Cell Agency Hits $60 Million Mark with ViaCyte

The California stem cell agency next week is slated to award  an additional $4 million to ViaCyte, Inc., a San Diego firm that is working with the agency to develop a "virtual" cure for diabetes.

ViaCyte has already received $56 million over the years from the California Institute for Regenerative Medicine(CIRM), as the agency is formally known. The latest award will be used to help prepare for a clinical trial for a new treatment for the highest risk diabetes patients.

In response to a query this morning from the California Stem Cell Report, Paul Laikind, president and CEO of ViaCyte, said that the new product, PEC-Direct, will deliver the same biologic component as used in the firm's current product, which is now in a clinical trial.

Laikind said PEC-Direct will allow direct vascularization, which "is expected to allow for a very robust engraftment and cellular performance."

Because the new device will require chronic immune suppression, Laikind said,

"It is being developed to treat patients with type 1 diabetes that are at high risk for acute complications such as severe hypoglycemic events associated with hypoglycemia unawareness syndrome."

(The full text of Laikind's remarks and research update can be found here.)

The agency's summary of the closed-door review of the Viacyte proposal said,

"There are over 100,000 people in the U.S. with type 1 diabetes so severe that they are at constant risk of hospitalization and/or death. Within months after administration, this product could naturally restore those patients’ blood sugar to normal healthy levels and save their lives." 

In addition to the $4 million, ViaCyte is putting up $994,343 of its own cash.

CIRM's blue-ribbon panel of scientific reviewers has already approved the award, which is part of a $30 million package of applications coming up next Thursday.  The governing board of the agency will ratify the approvals at its meeting next Thursday during a one-hour telephonic meeting.

The largest award, $10 million, will go to Humacyte, Inc., of North Carolina,  to produce an artificial vein for use in hemodialysis as reported yesterday on the California Stem Cell Report. More details of all the applications and review summaries can be found on the meeting agenda.

The session will be audiocast and carried on the Internet on a listen-only basis. Interested parties can participate in the meeting from telephonic locations throughout the state.

The main site for the meeting will be at the Sanford consortium in San Diego. Other locations include the agency's headquarters in Oakland and sites in Davis, South San Francisco(2), Napa, Los Gatos, Sacramento, Irvine, San Francisco and Los Angeles. For specific addresses and instructions for online access, see the agenda.

Multimillion Dollar Clinical Trial Proposal Hits Roadblock at California Stem Cell Agency

In a refreshing bit of candor, a California regenerative medicine firm yesterday said it was having little luck with its funding pitch to the state’s $3 billion stem cell agency and was withdrawing its proposal.

The firm is Cesca Therapeutics, which is based in the Sacramento suburb of Rancho Cordova and also has offices in India. The publicly traded enterprise researches and develops cell-based therapeutics.

In a press release today, the firm reported the bad news about its application for funding

“aimed at supporting implementation of its FDA approved Phase III pivotal clinical trial for critical limb ischemia.”

The announcement said,

“The company anticipated CIRM's evaluation of its application to be concluded during the second half of the month of November. However, preliminary feedback provided in advance of a formal funding decision has cast significant doubt over the prospects for a positive outcome and, as a result, the company has withdrawn its current application.”

Robin Stracey, Cesca photo

Robin Stracey, Cesca’s CEO, said,

"We feel compelled to revisit those elements of our plan that appear to have given CIRM reviewers cause for concern.

"Given that we are in the process of gearing up a significant number of sites to begin enrolling patients in a trial that would cost over $20 million to conduct, we need to quickly digest the feedback and address the questions raised. We expect to spend the next several weeks re-validating and/or amending select elements of our plan, with a particular focus on the anticipated rate of patient enrollment, the overall timetable and the design of the statistical plan. Having said that, we remain very excited by the prospects for our program. We believe the science to be sound and the clinical results so far very compelling. Nevertheless, there may be opportunities to refine our approach. It is imperative that we get it right."

The decision by Cesca fits with the CIRM 2.0 effort launched by Randy Mills, president of the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known.  Mills initiated the program in an effort to speed money to researchers and to boost the quality of applications. That means that applications are examined rigorously early on and often sent back for more work, a significant departure from past practices.

It is unusual for publicly traded companies to announce financial difficulties involving their projects, even though federal law says public firms must disclose major financial events that investors should know about.

The review process at CIRM is conducted behind closed doors. The agency does not release information about issues raised at this stage and also withholds the names of applicants for taxpayer cash.

The firm’s stock price closed at 59 cents today, up six cents. The 52-week range runs from 48 cents to $1.24.

Parkinson's Advocates Dismayed by California Stem Cell Agency Action

This is a Summit4stemcell video prepared following an emotional protest at the July meeting of the board of  the California stem cell agency.

Highlights

Lack of speed, openness protested

Agency cites cost issues

Patient-CIRM relationships unique

A group of Parkinson’s patients from San Diego is less than pleased today with a move by the $3 billion California stem cell agency that the group says will stall efforts to secure much-needed funding for a possible cure for the disease. 

The events this week follow an emotional protest at an agency board meeting in Oakland in July and highlight the often intense relationship between patient advocates and the stem cell agency – not to mention the pressure on the agency to move fast to find cures.

Anger and the Parkinson's symptoms of trembling and speech difficulties marked the protests. One patient advocate, Jenifer Raub of San Diego, said it was "outrageous" that an award round being discussed at the meeting would not provide funding until possibly 2017.

The patient advocate group, Summit4stemcell, had planned to address the stem cell agency board again at a Sept. 24 meeting scheduled to be held in San Diego. However, yesterday the group was told that the meeting would not be held in their city. Instead the session was being turned into a teleconference meeting and would be only about one hour long. Most in-person board meetings last much of the day.

Responding to questions from the California Stem Cell Report, Sherrie Gould, executive director of the Summit group and a nurse practitioner, said in an email,

Sherrie Gould, Scripps Health photo

“We are both confused and disappointed that this PUBLIC (Gould’s capitalization) meeting has been essentially cancelled.  We were prepared to talk with the ICOC (the agency board) and answer all questions about our patient advocacy movement (Summit4stemcells) and our research….

“Unless we can be present in front of the CIRM board, Summit's strong voice is diminished.  Being physically present in human form is always more effective than a teleconference. Delays, which we realize are a part of any government associated agency, are devastating to people suffering from the progressive symptoms of Parkinson's disease.” 

Jeanne Loring, head of the Scripps stem cell program in La Jolla, also voiced her displeasure in an email to Kevin McCormack, senior director of communications for the California Institute for Regenerative Medicine(CIRM), as the agency is formally known.

She wrote,

“CIRM has rather pulled the rug out from under the patient advocates. These meetings are supposed to be public, so how is the public going to participate if there is no access?…. I'm disappointed and the Parkinson's patients are exceedingly disappointed.  The Parkinson's group is trying to find out the resolution, if any, of the timing of the grant applications.”

The Summit group has raised $2 million to support Loring’s research and to match what CIRM might award for her effort.

Asked for comment today, McCormack said in an email,

“The reason we switched from an in-person to a teleconference board meeting is simple, the agenda for this meeting consisted of just a few items that could probably be handled in one hour. We can’t justify the cost of an in-person meeting in San Diego – which includes transportation for all those going there, plus hotel rooms, meals and the cost of the ballroom for the day - for one hour’s worth of business. As a state agency we are always mindful of how we spend our money so we made the change.” 

The next in-person meeting of the agency board is not scheduled until Dec. 17. But, in addition to the Sept. 24 meeting, two more teleconference meetings are scheduled between now and Dec. 17. Both are expected to be brief, routine approval of awards, if they are held at all.

Under the agency’s procedures and state law, members of the public can listen in and speak during teleconference meetings but only from the specific location from which an individual board member is participating.  

On the other hand, the in-person meetings offer an opportunity to speak face-to-face with all board members, chat with them informally during breaks and to converse directly with CIRM staff. The meetings have been an important tool for many patient advocate groups to press their cases. At times, however, their lengthy presentations have tried the patience of the agency.

The relationship between patient advocates and CIRM is notable. One of the unique features of the agency is the influence of patient advocates, particularly on its board.  Twelve sit on the 29-member panel and often play important roles. Sometimes they are the only way the board can approve awards because most of the other members have legal conflicts of interest and cannot vote.

David Higgins of San Diego is one of the patient advocate members of the board, which has a

David Higgins

specific slot for a Parkinson’s disease advocate. He was interviewed in July following the protest of the many-months delay in possible funding for Parkinson’s research. His remarks were recorded on a Summit-produced video(see at top of this item). Higgins, who is a scientist and has Parkinson's, said,

“It’s tough, it’s really tough, to be involved in the Parkinson’s community, especially to have Parkinson’s disease, and to watch things go in what seems like slow motion.”

Contacted today for additional comments, Higgins said,

“In my year at CIRM I have only encountered enthusiasm and support for patient advocacy - both from the staff as well as the board. Patient and patient advocate participation in the process is welcomed. CIRM is first and foremost about patients.”

Following the protests at the July meeting, CIRM President Randy Mills told the board he would see if there is anything he could do to speed funding. He said he would report back to the board later.

McCormack did not directly respond to a question today about whether recent, proposed changes in the award scheduling would have an impact on the Parkinson’s research.  He said only that the changes would provide more flexibility.

(The text of remarks by the various parties follow this item.)

Text of McCormack and Loring Emails on Stem Cell Board Meeting

Here is the text of the Sept. 9, 2015, email message to Sherrie Gould, executive director of Summit4stemcell, from Kevin McCormack, senior director of communications for the California stem cell agency, along with one from Jeanne Loring, head of the stem cell program at Scripps, to McCormack.  Loring sent her email to McCormack after she received a copy of his email to Gould.

The Gould-McCormack email comes first with the Loring-McCormack email following.

“Dear Sherrie
“I just wanted to let you know that there has been a change in plans for our September board meeting. As you know, it was originally scheduled to be held in San Diego, however, that has changed and it is now simply a telephonic meeting. The reason is that the agenda is going to be lighter than originally anticipated and it didn’t make sense to incur the cost of an in-person meeting for one hour’s worth of business. 

“I know you were planning to have many of the Parkinson’s group attend the meeting so I wanted to let you know so you can alert them to the change.
 “Our next in-person meeting will now be in Los Angeles on December 17th.
“Cheers,
“Kevin McCormack”

Loring’s email to McCormack:

“Hi Kevin:
 “Two in a row??  CIRM has rather pulled the rug out from under the patient advocates -these meetings are supposed to be public, so how is the public going to participate if there is no access?  Does CIRM have a plan to give access?  More importantly, is CIRM doing this to avoid having public access?
 “I'm disappointed and the Parkinson's patients are exceedingly disappointed.  The Parkinson's group is trying to find out the resolution, if any, of the timing of the grant applications.  Has anything been done in that regard yet?
 “In addition to the advocates, my students will be disappointed.  I always bring my interns and my CIRM fellows to the meetings so they can meet their benefactors.
 “Any plans for access to the public would be welcome.
 “Jeanne”

Text of Comments by Sherrie Gould, Executive Director, Summit4stemcells

Here is the text of comments by Sherrie Gould, executive director of Summit4stemcell of San Diego, made in connection with changes in the stem cell agency board meeting Sept. 24. Gould made the remarks in response to a query from the California Stem Cell Report.

“We are both confused and disappointed that this PUBLIC meeting has
been essentially cancelled.  We were prepared to talk with the ICOC (the stem cell agency) board and answer all questions about our patient advocacy
(Summit4stemcells) movement and our research. The Summit4stemcell
project has been faithfully attending public meetings every other
month for the last two years.  We have flown in numbers to make sure
our voice is heard.  We have spoken to board members, and to the media
about our plight.  This San Diego meeting, now cancelled, was an
opportunity attend to discuss the funding possibilities for
Parkinson's disease and whether funding might be appropriate for our
project.  Our science is sound and we have a clinical arm to this
project. We believe CIRM could take ownership of this very exciting
translational research if we were indeed funded.

“Unless we can be present in front of the CIRM board, Summit's strong
voice is diminished.  Being physically present in human form is always
more effective than a teleconference. Delays, which we realize are a
part of any government associated agency, are devastating to people
suffering from the progressive symptoms of Parkinson's disease. And
although hope springs eternal, we are saddened by the repetitive
delays.”