California Backs Research for Brain Cancer and Parkinson's with $9.5 Million

OAKLAND, Ca. -- The California stem cell agency today awarded $9.5 million for two late stage preclinical projects for development of therapies aimed at brain cancer and Parkinson's Disease.

A $5.8 million award for Parkinson's went to Krystof Bankiewicz of UC San Francisco and Clive Svendsen of Cedars-Sinai. A $3.7 million award for glioblastoma, a form of brain cancer, went to John Zaia of the City Of  Hope. Glioblastoma is the form of cancer that has afflicted U.S. Sen. John McCain.

In a news release from the agency, known formally as the California Institute for Regenerative Medicine (CIRM), its CEO, Maria Millan, said,

“Glioblastoma is the most common, and the most aggressive, form of brain cancer that led to the death of U.S. Senator Ted Kennedy and former Vice President Joe Biden’s son Beau Biden. 

"CIRM has supported a variety of stem cell-based approaches to target this devastating and currently untreatable condition.  The project approved by our board today is unique in that it seeks to use gene modified stem cells to allow patients to tolerate the high doses of chemotherapy while also making these tumors more susceptible to the chemotherapy.”

Regarding the Parkinson's award, the agency has already pumped $22.3 million into a phase one clinical trial being conducted by Svendsen for treatment of ALS with the same neural progenitor cell product that will be used in the Parkinson's research.

Here are links to the review summaries on the Parkinson's application and the glioblastoma application, plus additional information.

Application Number	Principal Investigator	Institution	Target	Amount Awarded	Public Summary of Review
CLIN1-10967	John Zaia	City of Hope	Glioblastoma	3,684,259	Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients
CLIN1-11059	Krystof Bankiewicz/ Clive Svendsen	UC San Francisco/ Cedars- Sinai	Parkinson’s Disease	5,811,340	MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson's disease

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.  

California's "Huge Ecosystem" for Stem Cells: The View from Europe

Some folks in Europe are worried about stem cell research, particularly about organizations like California's $3 billion stem cell agency.

The alarm was sounded just yesterday in Horizon, which calls itself  "The EU Research and Innovation Magazine."

The article in question (the second most popular on its web site at midday today) was headlined:

"Europe is in danger of being out-innovated in regenerative medicine"

Ton Rabelink, Arno Massee Fotografie

The piece consisted of an interview with Ton Rabelink, professor of internal medicine and head of Leiden University in the Netherlands. He cited the California agency as a "huge ecosystem" for developing much needed stem cell therapies.
nephrology at

Rabelink said that the European Medicines Agency is wrestling with finding the "the right mechanisms to support the field."

‘It is very important that they do this because the regulatory landscape in the US and Japan has changed over the past two or three years to accommodate recent advances. For example, Japan has an early access programme for treatments that seem promising but are not yet proven to work. If they appear safe in say, 40 patients, then doctors can start applying them. The US has created the 21st Century Cures Act that allows for clinical trials for stem cell therapies and fast-track access to market for those that appear effective."

Rabelink said,

"The risk is that if we don’t organise locally here in Europe, we’ll end up having to buy these treatments from those countries. We’ve already seen this with genetically modified cells, so-called CAR cells, to attack tumours in leukaemia. The treatment works quite well but costs about €500,000 ($582,670) per patient.

"It's very interesting to look at what happens in the rest of the world. You really need ecosystems — academia, but also legislatures, manufacturing and, of course, finance. The US has huge ecosystems like CIRM, the California Institute for Regenerative Medicine, which was founded through taxpayers’ money following a referendum, and invests about $250 million per year in this space. (The agency actually was created by a ballot initiative, which is much different than a referendum.)

The Dutch researcher continued,

"The situation in Japan is even more remarkable. The early breakthroughs were made by a Japanese scientist so they consider regenerative medicine almost as their national invention. And, of course, Japan has an ageing population so the concept is very appealing. The government set up a planned economy around regenerative medicine and adapted its regulatory framework, putting national systems in place to oversee quality and safety and organising private-public collaborations, bringing together academic institutions and big pharma. Fujifilm, which was originally a photographic company, is devoting resources to stem cell research and using its film technology to make biomembranes.’

New California Stem Cell Agency Board Member: Suzanne Sandmeyer of UC Irvine

A woman who is a medical doctor as well as a professor of both chemical engineering and microbiology and molecular genetics is joining the governing board of the $3 billion California stem cell agency.

She is Suzanne Sandmeyer, vice dean of research in the School of Medicine at UC Irvine. Her appointment by the UC Irvine chancellor was announced today by the California Institute for Regenerative Medicine (CIRM). 

UC Irvine has long held positions on the 29-member governing board of the agency, which was created by California voters in 2004. UC Irvine and its researchers have received $114.3 million from CIRM, ranking sixth among all recipients. Roughly 90 percent of CIRM awards has gone to institutions with ties to members of its board.

In its news release today, the agency quoted Sandmeyer as saying,

“Our country has one of the most expensive systems of medical care, and yet we don’t have the longest-lived population. I want to work toward reducing the burden of medical expenses for people. I am very excited about the potential of stem cells to treat many disorders and the potential for new technologies like CRISPR to further empower that approach.”

The agency's news release also noted Sandmeyer's broad range of interests outside academia.  She was quoted in a CIRM blog item as saying:

"I go through phases like everyone. There is never enough time. My favorites are astronomy, bird photography, guitar, biking, kayaking, reading and of course German shepherd dogs."

She replaces Howard Federoff also of UC Irvine, who is stepping down to devote more time to his research, CIRM said.

Stem Cell 'Ethical Tensions,' Recouping the Public Investment, Affordability and Much More

"Staggering" amounts of public money have been spent on stem cell research, and "care must be taken" to assure that commercialization does not exact an excessive "human or monetary price," according to an article this week in the journal Science.

The cautionary note was sounded in the prestigious publication while the world's largest stem cell gathering is underway in Australia with more than 2,500 researchers and others in attendance. Plus next week, the $3 billion California stem cell agency convenes its directors to mull over its own programs and give away more millions. 

Written by Douglas Sipp, a researcher at RIKEN in Japan, Megan Munsie of University of Melbourne and Jeremy Sugarman of John Hopkins University, the Science article said

"Ethical tensions related to stem cell clinical translation and regulatory policy are now center stage...."

They said that expedited government procedures for use of stem cell treatments have been set up in the United States, Japan and Italy. At the same time, they wrote, 

"A staggering amount of public money has been spent on stem cell research globally."

The article declared, 

"The state and the taxpaying public's interests should arguably be reflected in the pricing of stem cell products that were developed through publicly funded research and the regulatory subsidies. Detailed programs for recouping taxpayers' investments in stem cell research and development must be established."

They warned, 

"Care must be taken to ensure that entry of stem cell–based products into the medical marketplace does not come at too high a human or monetary price."

Another journal article this week also sounded cautionary notes. Written by Paul Knoepfler of UC Davis, it was titled: 

"Too Much Carrot and Not Enough Stick in New Stem Cell Oversight Trends"

Knoepfler addressed the Food and Drug Administration's efforts to speed use of stem cell therapies. He wrote in Cell Stem Cell and also on his blog, saying,

"It’s been remarkable to see the FDA approve up to 20 regenerative medicine advanced therapy(RMAT) designations in just over a year. However, I think there’s a strong possibility the agency has swung too far from the too slow review of stem cell and regenerative medicine investigational therapies in the past to now going at warp speed. Since none of the current 20 designated RMAT products had any kind of prior expedited review designation given, is it reasonable to think all 20 now meet rigorous enough standards and all because of new data? It’s hard to say, but there’s likely a spectrum of existing data behind these RMAT designated studies.

"We won’t have an overall RMAT verdict for years as the RMAT trials play out. However, I predict that the agency has lowered the bar too far. There are also concerns that the conditional approval system in Japan is too liberal, as evidenced by discussion over the approval of a recent IPS cell cardiovascular study. Taken together this is what I mean by 'too much carrot.'  Another issue with RMAT is that the criteria by which the designations are given (or not) are not clear." 

Next Thursday, the governing board of California's $3 billion stem cell agency is scheduled to meet to give away more millions for stem cell research in the Golden State. The agency will also be examining the non-scientific considerations that it uses in deciding which applications to fund. It may be that some of the issues raised by these four researchers will also come into play.

Safety and Effectiveness Concerns Raised about $5.8 Million California Stem Cell Research Proposal for Parkinson's

A California stem cell scientist yesterday commented on an application before the state's stem cell agency for $5.8 million for research into a possible therapy for Parkinson's disease, which afflicts as many as 10 million persons worldwide.

Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute, made the remarks concerning a proposal that is likely to be approved June 28 by the governing board of the $3 billion agency.

Her comments came in an email to the California Stem Cell Report after it asked for her thoughts on the review summary of the application (CLIN1-11059), which has been already approved behind closed doors by the agency's scientific reviewers. The vote was 9-6-0 with nine reviewers voting to approve it and six reviewers saying it needed improvement and should be resubmitted. No reviewers voted outright against it. The summary of the review and application can be found here. 

The name of applicant is not known. The stem cell agency withholds the identity of applicants until an application is ratified by the full board in a public session.

As Loring notes in her email, she is also conducting research on Parkinson's and also has funding from the stem cell agency, formally known as the California Institute for Regenerative Medicine. Here is the text of her email along with links to the two research papers that she cites.

“Researchers have explored many approaches to treatment of Parkinson’s disease over the last several decades. The tremors and freezing in PD are caused by the progressive loss a specific type of dopamine-producing neuron in a part of the brain called the substantia nigra. Half of these neurons have died by the time the disease is diagnosed.

“The only approach that has resulted in long-term reversal of the symptoms of PD is transplantation of human fetal tissue containing the precursors of that specific type of dopamine neuron. 

“The therapy that we are developing is based on the success of those fetal studies; our particular approach is to use patient-specific dopamine neurons produced from their own induced pluripotent stem cells (iPSCs). The arguments in support of this therapy have been published (see below).

“We share this idea for neural replacement therapy with several other groups worldwide: Roger Barker in the UK, Malin Parmar in Sweden, Jun Takahashi in Japan, and Lorenz Studer in New York are using human embryonic stem cell-derived neurons or unmatched iPSCs. We are the only group among these that is using matched neurons so there will be no need for immunosuppression.

“These are my concerns about any therapy using undifferentiated neural stem cells and gene therapy:

“Safety concerns:

“Neural stem cells are dividing cells that have been known to make tumors; one patient in California had a tumor grow in his spine after receiving neural stem cells from a clinic that did not have FDA approval, to treat a stroke. A few years ago there was a proposed treatment for Alzheimer disease that used neural stem cells, and in that case there was clear evidence that those cells made tumors in animals; it was funded by CIRM. Our cells are not dividing, and we have done a one-year study in rats to make sure no tumors form.

“Addition of a transgene for a neural growth factor, GDNF, to the cells, will require thorough testing because transgenes can disrupt the genome, and the expansion of the cells increases the risk of other mutations arising, such as the p53 mutations that we discovered in hESCs a few years ago. p53 is a very bad actor; mutations in this gene are found in half of all cancers. The applicants should be planning to sequence the genome of the cells to be improve the chances that the cells are safe; we sequence our cells.

“Effectiveness concerns:

“Survival vs. replacement.

“There are two general approaches to cell-based treatment of Parkinson’s disease. The neural stem cell approach is not intended to REPLACE the dying neurons. It is intended to help the remaining dopamine neurons survive longer. The other approach, neuron replacement therapy, can reverse the symptoms long-term.

“Immunosuppression. It is necessary to suppress the immune system whenever a transplantation of unmatched cells or tissues is performed. Immunosuppression is a concern for neurologists who are treating PD patients, because it weakens the immune system and makes patients more susceptible to serious debilitating infections.

“The neural stem cells making GDNF may be a good solution for the debilitating neuronal loss in ALS, and CIRM is already funding their use for that disease. But please ask a few neurologists what they would advise their patients with Parkinson’s disease. Those I’ve asked would prefer replacement of dopamine neurons, rather than a treatment using transplantation of dividing cells pumping a nerve growth factor into the brain, that requires immunosuppression. Historically, this has not been a good idea.”

Here are two links to articles that Loring cited. See here and here. 

California Stem Cell 'Renewal:' The Search for More Billions -- And Credit for Results

A KTVU video connected to CIRM's research support

California's 13-year-old stem cell agency, which is facing its demise as it lingers in a dim media shadow, last week snagged some credit for the work it has helped to finance in saving the life of a five-year-old girl. 

The news story appeared on San Francisco Bay Area television station KTVU. The piece involved Evangelina “Evie” Padilla Vacarro, who was born with what is known as the "bubble baby" disease. That is a genetic affliction that compromises the immune system so severely that it is nearly impossible for a person to survive.

Evie's story is powerful and well-known among those who closely follow the $3 billion California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known. But it has not seeped in the consciousness of California voters, who are likely to be asked to cough up $5 billion more for CIRM.

The agency expects to run out of cash by the end of next year. It is pinning its survival hopes on a private fundraising drive underway this year and passage of a proposed bond measure in November 2020. However, many competing priorities exist for that sort of funding. Plus the agency has yet to fulfill voter expectations that creation of the agency would lead to a stem cell treatment that would have widespread use.

In Evie's case, the treatment is available under exceedingly limited circumstances, like other treatments that the agency is supporting. They are still being tested before the federal government approves them for wider use.

The positive results that are, in fact, surfacing often do not mention CIRM's substantial backing, or they bury that fact so deeply it is all but invisible. (See here, here and here.) That is occurring despite the fact that many of those stories emerge from institutions that have received tens and tens of millions of dollars from the agency.

The fresh news peg for the KTVU story last week was the launch of a book, “California Cures: How the California Stem Cell Program is Fighting Your Incurable Disease," by longtime stem cell patient advocate Don Reed. The station reported,

"'Lives have been saved, and suffering eased, because California stood up for stem cells in 2004,' Reed said. 'Now as we approach the end of that (2004) voter-approved program, it is vital that everybody knows the story of (the institute) and why we must renew its funding.'"

Whether that "renewal" actually takes place may depend on whether the researchers and institutions that have benefited from CIRM's largess do a much better job of selling what they believe are the benefits and importance of the agency's programs and cash.

CIRM Board Member Sheehy Exiting as San Francisco Supervisor; Says SF Voters Favorable on Stem Cell Work

Jeff Sheehy, a member of the governing board of the $3 billion California stem cell agency, will be leaving his fulltime post as a member of the San Francisco board of supervisors later this year, but he says his unsuccessful election campaign disclosed favorable perceptions of stem cell research.

Sheehy disclosed his plans not to seek re-election in the media in the Bay Area. In a statement, he said it was "immensely rewarding" to have served the city. He also had this to say yesterday to the California Stem Cell Report,

"I have been asked about the implications for CIRM (the stem cell agency) and, based on polling around my bio that my campaign conducted, stem cell research remains very popular.

"When we asked voters about what prior work of mine resonated with them, the tagline, 'Fights for stem cell cures for deadly diseases and conditions as a founding board member of the California Institute for Regenerative Medicine,' was one of, if not the most popular items listed in my background.

"I had multiple conversations with voters about our work at CIRM and they were very supportive and very impressed with the what we have accomplished."

Sheehy is a patient advocate member of the stem cell board and has served on it since its inception in 2004. He is also chair of the Science Subcommittee of the board, participates in closed door reviews of applications for funds by scientists and oversees board discussion of the reviewers' decisions.

Sheehy's term on the 29-member board expires in 2020. The agency expects to run out of cash for new awards by the end of next year, 2019.

CRISPR, the Cancer Question and the California Stem Cell Agency: $13.5 Million Investment in Eight Projects

CRISPR was in the news this week, and it wasn't about how you like your French fries cooked.

CRISPR, for those of you who don't know already, is a relatively easy way to edit genes. The technique is used in at least eight research projects backed by the California stem cell agency, totalling about $13.5 million.

What made CRISPR generate the headlines was research -- as Business Insider reported -- that the "blockbuster gene-editing tool has been linked to cancer." The assessment was described as hype by some scientists.

Paul Knoepfler, the peripatetic stem cell blogger and researcher at UC Davis, explored the implications of the findings in an item earlier this week. He wrote,

"To say that CRISPR-Cas9 gene editing might 'cause cancer' seems premature to me so there probably were and are better ways for people to phrase the big-picture meaning of these new papers and others like them that may pop up in the future. However, to be clear safety is crucial, so this is an important development and risks of any potential therapy including gene editing-based approaches should be carefully weighed against potential benefits as trials are designed and then progress."

The California Stem Cell Report asked the agency, which monitors the research it funds, for a list of its projects involving CRISPR. Here is what the agency provided, including the disease focus, principal investigator and grant number.

• Sickle cell disease, $4.5 million, Childrens Hospital Oakland Research Institute, Mark Walters, TRAN1-09292
• Muscular dystrophy, $2.2 million, UCLA, April Pyle, DISC2-08824
• Vision loss, $232,200, UC San Diego, Karl Wahlin, DISC1-08683
• Neurological disorders, $1.9 million, Parkinson's Institute, Birgitt Schuele, DISC2-09610
• Blood disorders, immune disease, $984,228, Stanford, Rosa Bacchetta, DISC2-09526
• Blood disorders, $1.5 million, UCLA, Caroline Kuo, DISC2-10124
• Cystic Fibrosis, respiratory disorders, $2 million, Stanford, Matthew Porteus, DISC2-09637
• Blood Cancer, cancer, $235,800, UC Berkeley, Jacob Corn, DISC1-08776

Not a Dr. Ruth Explainer: California Stem Cell Agency's FAQ

Looking for a reasonably straight forward primer on the $3 billion California stem cell agency, a state program that is unique in California history and now in its 13th year of life? 

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), has compiled an FAQ (frequently asked questions) list which is available on its web site. Obviously, some of the material is colored by agency's own perspective, but the basic facts are there in one handy document.

The questions deal with such things as "when will there be cures," "shouldn't private industry fund stem cell research" and "what happens when the money runs out."  It's not exactly a Dr. Ruth's guide to all things CIRM, but many may find the document helpful.

Fire, Fury and $5 Billion: A Mini Preview of a Ballot Campaign for the California Stem Cell Agency

California's stem cell research effort, which is pinning its survival hopes on a proposed $5 billion bond measure in a couple of years, was slammed in a national publication last week as a "multi-billion dollar money suck."

The column in the conservative magazine National Review, which has about 90,000 circulation and a significant online presence, was a tiny preview of the fire and fury that is likely to erupt around the likely pitch to California voters in 2020 to give more cash to the agency.

Formally known as the California Institute for Regenerative Medicine (CIRM), the agency was created by a ballot initiative in 2004 and backed with $3 billion in state funds. It expects to run out of money for new awards by the end of next year.

Wesley J. Smith, a longtime critic of the agency, wrote in the National Review piece,

"The mendacious (2004) campaign promised Cures! Cures! Cures! with embryonic stem cells and therapeutic human cloning — even promising that disabled children would get out of their wheel chairs and walk. Good grief, campaigners also claimed that the money earned from all the coming cures would reduce California’s health-care budget.

"Some $2 billion later, none of it came to pass. Tens of millions were spent on a fancy-dancy building. Conflicts of interested have abounded. But the supposed point of the CIRM was not achieved. There have been extremely few human trials with embryonic stem cells — mostly dealing with eye conditions — and not all were CIRM-funded."

It is fair to say that Smith's characterizations omit much information about what CIRM calls its value proposition. Nonetheless, his points are likely to resonate with a substantial portion of California voters, who have seen little mainstream media coverage of CIRM. 

Even the institutions and recipients of multi-million dollar research awards regularly fail to note CIRM's contributions in their news releases about state-backed scientific discoveries. (See here and here.)

As of today, the agency has invested in 49 clinical trials, the last stage before a therapy is approved for widespread use. A discovery or treatment that would captivate the public could emerge from those trials between now and the election in November 2020. Meanwhile, given the nature of today's financially struggling media and limited science coverage, the agency and its backers are likely to find it tough to break through the news clutter and convince voters to cough up more cash.   

'Spontaneity' Live: A Powerful, Cyberspace PR Tool for California's Cash-Strapped Stem Cell Program?

Jeanne Loring, right, on a Scripps Research Institute
Facebook Live event last week.   Click here to go to the video. 

Stem cell researchers rarely have a chance to talk directly about their work to thousands of people at a time, including those in the farthest reaches of the globe.

But Jeanne Loring at the Scripps Research Institute did it last week. The California stem cell agency did it last month with Stanford researcher Gary Steinberg.  And it could well be that the technique that they used will emerge as a critical tool in the effort to stave off the death of the $3 billion, stem cell program. The end could come as early as 2020 if agency supporters fail that year to win voter approval of $5 billion more for the program.

The medium in use by Loring and the agency is something called "Facebook Live."  It is a live streaming service that allows viewers to ask questions and comment during a webcast conducted by researchers or stem cell research advocates.  It is considerably more compelling than pre-recorded videos, says Forbes magazine, which reports that presentations on Facebook Live are viewed for three times as long as a static video. The thinking is that the interactive connection and the initial live presentation -- with its informality, spontaneity and lack of predictability --  are more interesting than your usual, pre-recorded sessions.

Facebook Live has been around since 2016.  but has rolled out slowly. According to a Google search this past weekend, only a handful of stem cell-connected enterprises have taken advantage of it. (See a few below.)

Viral Boost for the California Stem Cell Message

In its presentation last month, the stem cell agency started off with about 80 viewers. By the time of this writing the number of views of the video had skyrocketed to more than 6,800. Loring's presentation now stands at 1,600. Her number grew by 100 as this article was being written. That viral strength likely surpasses many static offerings in print coverage or conventional electronic news, which are largely onetime shots.

(The agency, on the morning following this posting, filed an item on its blog, hailing the success of the program. The item said, "We had an amazing response from people during the event and in the days since then with some 6,750 people watching the video and almost 1,000 people reacting by posting a comment or sharing it with friends. It was one of the most successful things we have ever done on Facebook so it’s not surprising that we plan on doing many more Facebook Live ‘Ask the Expert’ events in the future.")

One might ask: Why is the impact of this single Facebook event important in terms of a stem cell ballot campaign?

In recent years, the stem cell agency has slipped out of the public eye and is hard-pressed to attract widespread media attention, a situation that is commonplace for most state government departments. The agency's "value proposition" is not well understood, say officials at the agency, formally known as the California Institute for Regenerative Medicine (CIRM). 

To win approval of a proposed $5 billion bond measure in November 2020, however, CIRM will need strong support for its efforts, which have so far failed to deliver on the public's expectations that miraculous stem cell therapies are right around the corner. Those hopes were raised more than 13 years ago during the campaign that created the stem cell agency. Today its funds are running out. Cash for new awards is  expected to vanish by the end of next year.

Selling Hope to Half of California Families

Nonetheless, one of CIRM's notable products is hope -- not to mention 49 clinical trials that could lead to therapies for deadly diseases and widespread afflictions ranging from cancer to urinary incontinence.

In 2004, backers of the stem cell ballot measure declared that "medical problems that could benefit from stem cell research affect 128 million Americans including a child or adult in nearly half of all California families."

Reaching those California families is the key to unlocking their votes on behalf of the stem cell program. Facebook -- notably and coincidentally -- reaches nearly half of the Internet users  in California.

Using Facebook Live, or rival services, may well be the most effective way to connect with those important voters, especially with the help of the patient advocates who have been heavily involved with the agency. They bring the personal stories that resonate with voters and create an emotional appeal that is likely to trigger a favorable vote for more billions.

More services like Facebook Live will probably arise in the next 12 months. But Facebook has an advantage: the huge number of its users and an established Internet base that includes the patient advocate community and its organizations.

Writing on Forbes about business applications of Facebook Live, Steve Olenski said:

"Live video comes with a generous helping of transparency. Your influencer's voice will ring truer in a live conversation with fans than it does in a blog post. Viewers can engage with your product or service and the influencer in open conversation handled in real time.

"If you're looking for a compelling way to build trust with your audience, a live video is a great place to start."

Here are links to a few Facebook Live events involving stem cell-related matters: Difference between gene therapy and stem cell therapy, CAR T-Cell Therapy for Non-Hodgkin Lymphoma, Gene Therapy for Sickle Cell Disease.

Viral Power: California Stem Cell Agency Hits 6K with Cyberspace Event on Stroke Therapy

Here is the Facebook Live event produced by CIRM. The number in the left hand 

corner is not static. It changes as the video is viewed by more persons. 

The $3 billion California stem cell agency last week mounted its first "Ask the Experts" session on Facebook, a look at a stroke therapy that left one patient crying with joy and  researchers "shocked" at the initial, beneficial results.

The Facebook Live event dealt with a stem cell treatment for the most common form of strokes, which claim 750,000 victims annually. It is the leading cause of disability in this country and third leading cause of death.

The webcast at noon last Thursday drew only about 80 viewers, the agency reported. But by late Monday, Facebook statistics showed that it had been viewed more than 6,400 times (more than 6,500 by this morning). The figures demonstrated the viral power of Internet communications in spreading the stem cell agency's message. Not to mention that it could help to stave off the agency's financial demise.

Featured on the webcast were researcher Gary Steinberg of Stanford University, whose work has been supported by $24 million from the agency, and Sonia Coontz of Long Beach, Ca., who said she wept after treatment in the clinical trial as she experienced immediate improvements. Steinberg said his team was "shocked" by the rapidity with which some patients improved from their ischemic strokes.

More than 172 comments were made during the session. Some simply sought more information. Others waxed euphoric. Lisa Bayha Deck, wrote on Facebook,

"I’m a patient of Dr. Steinberg’s and so thankful for his professionalism. He gave me my life back after four strokes and Moyamoya Disease."

The Facebook event was the latest effort by the agency, formally known as the California Institute for Regenerative Medicine (CIRM), to educate the public about the work it is financing. However, the agency expects to run out of cash for new awards by the end of next year. It hopes that voters will provide it with $5 billion more in state bond funding via a ballot measure in November 2020. 

During the event, Lila Collins, a senior scientist at CIRM, provided an overview of key stem cell trials around the country. Kevin McCormack, senior director of communication at CIRM, moderated and produced the session.   

Following the event, McCormack said he thought it went well for an initial effort, although he said he was caught up in the mechanics of handling the session. On Monday, McCormack said, 

"We are going through the stats now and plan on putting together a roundup of views, comments, etc. We are also planning on posting a blog about the event – with answers to questions we didn’t get around to during the hour – so that should be coming in the next few days."

Prior to the webcast, the California Stem Cell Report asked McCormack to elaborate on the agency's Facebook effort. Here is a lightly edited version of that email interview. 

Q: Is this the first such event for CIRM? 

A: It’s the first Facebook Live event we are doing. A few years ago we did a similar series using Google Hangout. We featured a number of our experts in ALS and cancer, heart disease etc. but that wasn’t as easy to do technically. Facebook Live is simpler and because so many more people are on Facebook on a regular basis it will hopefully allow us to reach a wider audience. 

Q: Why is CIRM doing this event? 

A: It’s just part of our outreach efforts to let people know how stem cell research is progressing and the role that CIRM is playing in advancing that. We have been doing similar things for years, reporting back to the people of California on the work we are doing. We do it regularly with in-person patient advocate meetings, and with talks and presentations all around the state so we just wanted to take advantage of this technology to see if we could reach a wider audience.

Q: Will it be saved on the CIRM web site for future viewing?

A: One of the great features of Facebook Live is that it is automatically saved at the end and you can watch them on Facebook again and again. And yes, we’ll be putting links to them on our website but the easiest way to find them is to just go to our Facebook page.

Q: How did this originate?

A: We are always looking for new ways to reach out to a wider audience and let people know about CIRM’s help in advancing stem cell research. We looked at Facebook Live and thought it could be a great tool for us. We are already on Facebook anyway so this was a natural extension.

Q: And, finally, is there anything else you want to say about this event and its significance? 

A: Earlier this year we held a patient advocate event at UC Riverside. It was our first excursion to the Inland Empire and we had a great crowd, more than 100 people showed up on a weekday. But our goal is always to reach as many people as possible, and so we are always exploring new ways, new tools to do that. 

Much as we would like to, we don’t have the staff or budget to travel constantly around the state doing in-person meetings so this is a great way of achieving the same goal in a much more efficient manner. The people of California put a lot of faith in us when they approved $3 billion in funding for stem cell research. As we see that funding really help accelerate the field – we have 49 projects that we funded in clinical trials, we are seeing patients cured of life-threatening diseases – we want to share that news with as many people as possible.

$115 Million Stock Offering by California State-backed Biotech Firm, Forty Seven, Inc.

A California company backed by the state's stem cell agency has announced that it is planning a $115 million initial public offering.

The firm -- Forty Seven, Inc., of Menlo Park -- filed a notice of its plans with the Security and Exchange Commission last Friday. The company has been awarded $15.2 million from the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is formally known.

The company was founded by renown stem cell researcher Irving Weissman of Stanford University, who serves on the Forty Seven board of directors and who has a 9.5 percent equity interest in the firm.

John Carroll of Endpoint News this morning wrote:

"Forty Seven has an interesting past. The 78-year-old Weissman was able to wrangle substantial support for his early research work on CD47 from the California Institute for Regenerative Medicine, even launching early human studies — a rare feat in academic circles. Weissman and former CIRM chief Alan Trounson enjoyed a tight relationship, which extended to Trounson’s appointment to the board of another startup that Weissman had helped found — StemCells, Inc. —shortly after his departure from the agency. And Forty Seven is still getting money from CIRM under its latest $19 million grant."

The company said in its SEC filing,

"We are eligible to receive up to $19.2 million in grants from CIRM and the Leukemia and Lymphoma Society, or LLS, of which $11.6 million has been received through March 31, 2018."

Weissman has received  more than $34 million from the agency, part of the $360 million going to Stanford projects from CIRM, whose 29-member governing board has included a member from Stanford since 2004. Stanford ranks as the No. 1 recipient of funds from the $3 billion California stem cell agency, which is slated to run out of cash for new awards by the end of next year. 

For more on Forty Seven, see here and here.

California Stem Cell Agency and First-of-Kind Fetal Transplant for Fatal Affliction

Nichelle Obar and her daughter, Elianna, at three weeks. NYT photo by Bryan Meltz

A uniquely California medical treatment -- the first of its kind in the world -- won national attention today with reports that it could open the door to similar operations before birth for such diseases as sickle cell and hemophilia.

The therapy was developed by a UC San Francisco researcher who treated "the first patient enrolled in the world’s first clinical trial using stem cells transplanted prior to birth," according to a UCSF news release.

Fetal surgery was also invented in California. And the transplant was backed by $11 million from the state's stem cell agency, a $3 billion enterprise that is unique among states in its size and scope.

Missing from the news, however, was any mention of the agency's multimillion-dollar contribution -- a not insignificant matter since the agency is facing its own financial demise in less than two years. The agency hopes voters will keep it alive and approve $5 billion more for stem cell research in 2020. The agency, however, is laboring under a lack of public awareness for its "value proposition." (See here and here.)

The research is being conducted by Tippi MacKenzie, a pediatric and fetal surgeon who has received a total of nearly $15 million from the agency. Her employer, UC San Francisco, has received $169 million in 81 awards.  Various deans of its medical school have also served on the agency's governing board since the inception of the agency, known formally as the California Institute for Regenerative Medicine (CIRM).  

A spokeswoman for UCSF said omission of the CIRM funding in its news release was not intentional. 

The agency was not mentioned also in a lengthy article on MacKenzie's research in the New York Times this morning. The piece was displayed on the front page of the weekly Science section of the Times, a prestigious position available only 52 times a year.

MacKenzie's work involved a family from Hawaii and their child, who was treated in utero for a nearly always fatal affliction. 

In the article by Denise Grady, the Times reported, 

"Elianna, born Feb. 1 with a robust cry and a cap of gleaming black hair, has a genetic disease that usually kills a fetus before birth. The condition, alpha thalassemia major, leaves red blood cells unable to carry oxygen around the body, causing severe anemia, heart failure and brain damage.

"The transfusions in the womb kept her alive, but only treated her illness. The bone-marrow transplant has the potential to cure it. Whether it will succeed is still too soon to tell.

"Elianna and her mother, Nichelle Obar, were the first patients in an experiment that pushes the limits of fetal therapy, a field already known for its daring."

The Times continued, 

"If the treatment works, it could open the door to using bone-marrow transplants before birth to cure not just Elianna’s blood disease but also sickle cell anemia, hemophilia and other hereditary disorders, some so severe that the prenatal diagnosis may lead parents to end the pregnancy.

"Bone marrow is considered a potential cure because it teems with stem cells, which can create replacements for cells that are missing or defective as a result of genetic flaws."

Asked for comment by the California Stem Cell Report, Maria Millan, president of CIRM and a pediatric surgeon, said in an email, 

“The New York Times picture of Nichelle Obar with her 3 week old daughter Elianna shows us something that’s hard to capture in a billion words.(See photo above.)  Elianna is afflicted with alpha thalassemia major, a fatal condition that usually ends up in death before birth.

"It is extremely gratifying to know that CIRM has been instrumental in supporting Dr. McKenzie’s research to develop a treatment for this severe unmet medical need. Dr. McKenzie’s approach combines surgical advances that allow her to treat the baby while still in the mother’s womb; transplantation with the mother’s blood stem cells provides healthy blood cells that the baby needs to survive. 

"We have supported Dr. McKenzie’s research from the early years when she was a new faculty working out the basic science and immunology in the laboratory and we helped her to develop her clinical plan. Most recently, we have partnered with Dr. McKenzie to bring this research to clinical trials. To see Ms. Obar take their child home from the hospital is encouraging. We know that this is just the beginning. This is what the people of California voted for when they approved Proposition 71. They depend on us to boldly partner with Dr. McKenzie to translate the promise of stem cell treatments to patients with unmet medical needs."

The phase one clinical trial, which is aimed primarily at safety, is scheduled to release its preliminary results by February 2022 and hopes to treat another nine patients. The treatment would then face another two phases of clinical trials for efficacy. 

As for the baby, her mother told the Times, 

“Elianna’s doing great. I’m not disappointed at all. If it works, great. If it didn’t, we’re O.K. with it. We’ll celebrate all the little accomplishments. I’m glad we did it."

Here is a UCSF video on the trial.

Incontinence to Cancer: California Kicks in $26 Million for Stem Cell Research

The California stem cell agency this week awarded more than $26 million in its hunt for treatments for afflictions ranging from cancer and incontinence to osteoporosis and eye disease.

The $3 billion agency's board ratified the five research grants in a 30-minute, telephonic meeting on Thursday with little debate. The applications were previously approved behind closed doors by the agency's out-of-state scientific reviewers.

The largest award -- $12 million -- went to a team at UCLA led by Steven Dubinett for a phase one clinical trial. The agency, formally known as the California Institute for Regenerative Medicine (CIRM), said in a news release:

Steven Dubinett, UCLA photo

"The team is using the patient’s own immune system where their dendritic cells – key cells in our immune system – are genetically modified to boost their ability to stimulate their native T cells - a type of white blood cell - to destroy cancer cells. 

"The investigators will combine this cell therapy with the FDA-approved therapy pembrolizumab (better known as Keytruda) a therapeutic that renders cancer cells more susceptible to clearance by the immune system."

The research application approved this week that would likely have the most widespread application involved urinary incontinence. The $6 million award went to a Stanford team led by Bertha Chen. CIRM said,

Bertha Chen, Stanford photo

"Despite being one of the most common indications for surgery in women, one third of elderly women continue to suffer from debilitating urinary incontinence because they are not candidates for surgery or because surgery fails to address their condition. 

"The Stanford team is developing an approach using the patient’s own cells to create smooth muscle cells that can replace those lost in UI. If this approach is successful, it provides a proof of concept for replacement of smooth muscle cells that could potentially address other conditions in the urinary tract and in the digestive tract."

Here is the full list of winners and links to the summaries of the reviews of their applications and their scores. The review summaries other than the one for Dubinett can be found on a single document, which also contains the review summaries of the 10 rejected applications:

• Steven Dubinett, UCLA, $12 million, application number CLIN2-10784, review summary, title advanced non–small cell lung cancer, co-funding not required but $400,000 provided
• Bertha Chen, Stanford, $6 million, application number TRAN1-10958, review summary, title Autologous iPSC-derived smooth muscle cell therapy for treatment of urinary incontinence, co-funding not required
• Cassandra Calloway, Children's Hospital Oakland Research Institute, application number TRAN2-10990, review summary, target development of a noninvasive prenatal test for beta-hemoglobinopathies for earlier stem cell therapeutic interventions. co-funding not required
• Farhad Parhami, Max BioPharma of Santa Monica, Ca., $1.7 million, application number TRAN1-10937, review summary, title therapeutic development of an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis, co-funding not required
• Magdalene Seiler, UC Irvine, $4.8 million, application number TRAN1-10995, review summary, title morphological and functional integration of stem cell derived retina organoid sheets into degenerating retina models, co-funding not required

Here is a link to an LA Business Journal story on the award to BioPharma.

A Stem Cell "Nose Job" and Updates on Regulation of Questionable Stem Cell Clinics

Tracking the latest on dubious stem cell clinics, the feds and the implications across the nation can be a bit of consuming task, if you are interested in that sort of thing.

But one Internet site does a pretty good job of keeping up with it. Here is what popped up last week on The Niche, which is based out of UC Davis.

•  A Q&A with a Georgia law professor concerning the federal crackdown on unregulated stem cell clinics. 
• A quick look at activities regarding the state of Washington's new law on stem cell clinics, the failed state regulatory attempt in the stem cell "hot bed" that is Florida plus questions about enforcement of California's new stem cell clinic law. 
• A video of a presentation by an unregulated stem cell clinic which discussed a product described as a  "magic concoction" that is sprayed up the patient's nose.  

Paul Knoepfler is the UC Davis stem cell researcher who produces The Niche and has long written about the dubious clinics that have proliferated across the nation, with the most in California. 

Feds Crack Down on Unregulated 'Stem Cell' Clinics, Including One in California

The Food and Drug Administration has moved to shut down two businesses that offer "stem cell" treatments, including one operation based in California,  following reports that some patients were blinded by treatments.

The lawsuits yesterday were characterized as "historic" by one California stem cell researcher, who has been reporting on unregulated and unproven "stem cell" treatments for several years. 

The firms involved are U.S. Stem Cell in Florida and California Stem Cell Treatment Center and its proprietors, physicians Mark Berman, a cosmetic surgeon, and Elliott Lander, a surgeon and urologist. The California enterprise has locations in Beverly Hills and Rancho Mirage with affiliates in 100 locations around the country.

Michael Hiltzik wrote in the Los Angeles Times,

"The lawsuits are the most aggressive steps yet taken by government regulators against rapidly proliferating stem-cell treatment clinics pitching scientifically unproven therapies to desperate patients, some with terminal illnesses. The lawsuits were filed at the request of the Food and Drug Administration, which last year announced that it would take a stern approach to what it described as the manufacture and use of unlicensed drugs."

Researcher Paul Knoepfler of UC Davis has been raising questions about unregulated and unproven stem cell treatments for a number of years. Writing yesterday on his blog, he called the federal action "historic." He said the suits could mark "a turning point for dealing with the for-profit stem cell clinic problem in the U.S."

Knoepfler also carried on his blog the full statements of the companies denying any wrong doing.

The California stem cell agency has also raised cautionary notes about unregulated treatments. 

Here are links to other stories on the lawsuits, which received national attention: Washington Post, The Associated Press and STAT. The Associated Press was carried by the New York Times and many other outlets online. The Times also carried this story by its own reporters.  Here is a recent column by Hiltzik on the California Stem Cell Treatment Center. The headline on the colum says "Patient lawsuits, federal investigations and a 'virtual' PhD: Inside a would-be stem cell empire." 

Removing the Barriers to Widespread Use of Stem Cell Treatments

If you are really interested in delivering stem cell therapies to the marketplace, the place to be early next month is in Los Angeles at a major, international conference that will examine the bottlenecks that stand in the way of bringing the treatments to the public at large.

The California stem cell agency, which is spending $3 billion to create therapies, says that "key unresolved issues" must be addressed before safe and effective therapies reach the mainstream.

The stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM), is co-sponsoring the two-day event June 5 and June 6 with the The International Alliance for Biological Standardization.  The session is drawing speakers from around the world. 

Abla Creasey, CIRM photo

Earlier this year, Abla Creasey,  vice president of therapeutics and strategic infrastructure at the agency, said, 

“The field of stem cell research and regenerative medicine has matured to the point where there are over 900 clinical trials worldwide. It is critical to develop a system of effective regulation of how these stem cell treatments are developed and manufactured so patients can benefit from future treatments.”

Her comments appeared on the agency's blog, The Stem Cellar, in an item written by Geoff Lomax, senior officer for CIRM strategic initiatives. Lomax also quoted Martin Pera, a professor at the Jackson Laboratory in Maine and who also directs the International Stem Cell Initiative Genetics and Epigenetics Study Group, and Robert Deans, chief technology officer of Blue Rock Therapeutics, Inc.,  of Cambridge, Mass.

Pera said,

“Participants at this meeting will survey and discuss the state of the art in the development of definitive assays for assessing the safety of pluripotent stem cell based therapies, a critical issue for the future of the field. Anyone active in cell therapy should attend this meeting to contribute to a dialogue that will impact on research directions and ultimately help to define best practice in this sector.”

Deans said,

“I believe standardization will be an increasingly crucial element in securing commercial success for regenerative cell therapies. This applies to all facets of development, from cell characterization and patent protection through safety testing of final product. Most important is the adherence of players in this sector to harmonized standards and creation of a scientifically credible market to the capital community.”

Here is a link for registration.

Golden State's Stem Cell 'Value:' Bubble Boy Disease and Bringing a Therapy to Market

California's $3 billion stem cell agency today performed a double, PR whammy in an effort to create a greater public understanding of the "key role" it can play in the most expensive and critical steps in creating a therapy that is widely available to the public.

The agency's pitch comes as it faces its potential demise in two years unless it is successful in raising more funds for its operations.

Donald Kohn, UCLA photo

The agency's starting point today involved research for cures for an affliction that has received highly visible, albeit intermittent, coverage over the past several decades. The fatal affliction is often referred to as the "bubble boy" syndrome or, more accurately, SCID,  Severe Combined Immune Deficiency.

In this case, it involves UCLA scientist Donald Kohn, a British firm, Orchard Therapeutics, Inc., opening new facilities in California (Menlo Park and Foster City) and a $20 million award. The cash, however, is only a relatively small piece of the state's immune deficiency investment. 

$141 million for immuno deficiencies

Over the years, Kohn's research has been backed by at least $52 million from the taxpayer-financed California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Overall, it has pumped more than $141 million into immune deficiency disorders, not all of which is directly related to SCID. 

Responding to a query, Kevin McCormack, senior director for CIRM communications, said,

"We’re a lot more than just a pretty face you know. We feel we’re helping change the face of medicine."

Or as Maria Millan, president of CIRM, said both in a news release and in an article on the agency's blog, The Stem Cellar: 

"We invest when others are not ready to take a chance on a promising but early stage project. That early support not only helps the scientists get the data they need to show their work has potential, but it also takes some of the risk out of investments by venture capitalists or larger pharmaceutical companies."

She continued, 

"Our funding and partnership has enabled the smooth transfer of Dr. Kohn’s technology from the academic to the industry setting while conducting this important pivotal clinical trial. With our help, Orchard was able to attract more outside investment and now it is able to grow its pipeline utilizing this platform gene therapy approach.”

De-risking stem cell therapies

In biotech, all this is referred often as "de-risking" and helping research advance beyond the "valley of death," the stage at which conventional financing for research becomes exceedingly difficult to secure. Both have been goals of the agency for some time and are part of what Millan refers to as the agency's "value proposition." 

Boosting public recognition of the value created by CIRM is likely to be a key element in winning approval of a proposed $5 billion bond initiative in 2020, seeking more cash for the agency. The agency estimates that it will run out of money for new awards at the end of next year. 

The agency noted the significance of the deal that GlaxoSmithKline(GSK) made last month with Orchard.  Although the agency tends to focus strongly on medical and scientific achievements, today's PR hit had a stronger emphasis on business matters. Without a successful business model, it is unlikely that any CIRM research will reach the general population.

California's key role

CIRM said,

"Under the deal, GSK not only transfers its rare disease gene therapy portfolio to Orchard, it also becomes a shareholder in the company with a 19.9 percent equity stake. GSK is also eligible to receive royalties and commercial milestone payments. This agreement is both a recognition of Orchard’s expertise in this area, and the financial potential of developing treatments for rare conditions."

Mark Rothera, Orchard photo 

The agency quoted the CEO of Orchard, Mark Rothera, as saying,

"The funding and advice from CIRM allowed Orchard to accelerate the development of OTL-101 and to build a manufacturing platform to support our development pipeline which includes 5 clinical and additional preclinical programs for potentially transformative gene therapies."

The GSK-Orchard deal, CIRM said, was "good news for both companies and for the patients who are hoping this research could lead to new treatments, even cures, for some rare diseases. It was also good news for CIRM, which played a key role in helping Orchard grow to the point where this deal was possible."