Fifty New Clinical Trials, a $150 Million Partnership and Much More: California's Coming Stem Cell 'Powerhouse'

Highlights

50 new clinical trials

$150 million public-private partnership

"Long overdue," says venture capitalist
$30 million end plan

LOS ANGELES -- Directors of the California stem cell agency this morning approved an $890 million plan for the next five years as it surges forward with a risky and ambitious effort to build an “industrial stem cell therapeutic powerhouse” in the Golden State.

The agency proposes 50 new clinical trials on top of 15 already underway. Next year it expects to set up a $150 million partnership with private investors to turn research into cures. Investors would have first pick of the best research that CIRM has to offer that currently lacks a partner. 

Other ventures and goals for what could be the agency's last five years of life include:

• Introduction of 50 new therapeutic or device candidates into development
• Working to create a more favorable federal regulatory environment
• Reduction by 50 percent the time it takes basic research to move into a clinical trial
• Creation of two centers at $15 million each to assist in much of the "backroom" work needed for clinical trials
• Creation of an online, hook-up center for researchers looking for collaborators to advance their work

Directors of the agency, formally known as the California Institute 71 for Regenerative Medicine (CIRM), approved the plan unanimously at a meeting here. (Here is a link to its press release.)

Arlene Chiu, former director of CIRM's scientific programs and now with the City of Hope, told the agency board that the plan was audacious and bold. CIRM board member Sherry Lansing, former chair of the UC board of regents, praised the plan's "sense of urgency."  Another board member, Steve Juelsgaard, former executive vice president of Genentech, said the plan contained projects that he had never seen before in the biotech industry.

Asked for comment, CIRM board member Al Rowlett, who is chief executive officer of the Turning Point mental health program in Sacramento, said,

“It’s ambitious, but then isn’t that what the people of California were when they approved Proposition 71. They wanted to create something that was going to change the face of medicine. That’s what we hope to do….”

Proposition 71 was the ballot initiative that created the $3 billion agency in 2004. The campaign led voters to believe stem cell therapies were close on the horizon. None has been produced, and the agency's state bond funding is expected to run out in 2020.

CIRM directors later today will be briefed on a $30 million “wind-down” plan that has attracted $7 million in private donations. More funding is being sought. One source may be the state legislature.

The agency’s plan for the next five years says it would benefit the people of California by creating  “an industrial stem cell therapeutic powerhouse that expands the tax base, adds high quality jobs and increases the likelihood of the commercialization of stem cell treatments for patients with unmet needs.”

Randy Mills, CEO of the agency since May 2014, said the plan was devised to have "the greatest possible impact for our patients. We didn’t want something ‘good enough.’ We wanted something transformational."

He acknowledged the risk and size of the task, which he said is aimed at transforming regenerative medicine. But he remains optimistic that the agency’s tiny team of about 55 persons can pull it off. He gave his team full credit for developing the spending proposal. 

The CIRM plan calls for spending $620 million on clinical work and “translational” research, which is aimed at taking basic discoveries beyond the most preliminary stage. Basic research would receive $170 million. Educational programs total $50 million. Another $50 million would go for “infrastructure.” 

One of the riskier elements of the proposal may be the agency’s plan to offer $75 million to private investors to begin a partnership in which they would have access to the best of the CIRM-funded research that doesn’t already have a private partner. 

The investors, who could be a Big Pharma firm, an existing smaller company or venture capitalists, would have to add $75 million of their own money. The agency would also continue to support the selected research, thus minimizing the risk to the private investors.

For months, Mills has been commenting on the reluctance of private investors to engage in stem cell therapy development, which is expensive and novel. “De-risking” is important in attracting business interest, Mills says.

Competition for the $75 million is scheduled to begin behind closed doors early next year. But questions have been raised about the risk that no private investors would find any CIRM research attractive.

Gregory Bonfiglio, managing partner of Proteus Regenerative Medicine, a Portola Valley, Ca., a venture capital firm, said in an interview, however, such efforts by the agency are long overdue.

He said, 

“There are risks inherent in the development of new, disruptive technology. The bigger risk is failing to deliver on their underlying promise to bring new regenerative therapies to patients…. The bigger risk is not doing anything.”

Another important element of the plan involves creation of accelerating and “translating” centers, funded at $15 million each, beginning next year. The agency is also likely to expand its Alpha Clinic effort, which is aimed at providing one-stop treatment centers.

The translating and accelerating centers would work with the Alpha clinics and other researcher to provide much of the “backroom” work needed to negotiate federal rules and regulations and win ultimate approval of a therapy. 

California's $30 Million Stem Cell 'Pitching Machine'

LOS ANGELES -- Above is a look at the California's stem agency's plan to support efforts to advance research into clinical trials. Two, $15 million centers are planned. They would perform the "back room" work that does not have appeal to research scientists.

Transforming Regenerative Medicine: Discussion Begins on California's $890 Million Approach

LOS ANGELES -- Directors of the $3 billion California stem agency this morning began discussion of its spending plans for the next five years, an $890 million effort that is designed to "transform regenerative medicine."

Jeff Sheehy, chairman of the Science Subcommitte of the board, praised the effort, especially the "metrics" that measure the agency's teams performance at all levels. He said the staff developed metrics within each area.

David Higgins of San Diego, another board member, said that CIRM's plan is much different than other strategic plans that are "boring" and "go into a desk drawer." He said the CIRM plan is "much different" and creates a pathway to success.

The $2.8 Billion Award Bucket of the California Stem Cell Agency

LOS ANGELES -- Here is the financial state of awards by the $3 billion California stem agency. The effective burn rate is $170 million a year -- the amount of money that can  be awarded -- because of return of funds on grants that have been terminated.

Randy Mills, CEO of the stem cell agency, said that the rate of returns has jumped from 10 percent to 22 percent, which could mean an additional $150 million for future awards, if the rate stays at the level.

A Look at the Portfolio of the California Stem Cell Agency

Look at the status of CIRM funding by disease

LOS ANGELES -- The California stem cell agency this morning presented a fresh breakdown of its spending by disease areas. The largest two components are in the neuro and cancer areas, which account for nearly half of the agency's portfolio.

CIRM Board Meeting Opens

LOS ANGELES -- The meeting of the governing board of the California stem cell agency opened at 9:12 a.m. today.  The public audience is quite small, less than 10 to witness approval of an $890 million expenditure of state funds.

Follow All The News Today on California's New $890 Million Stem Cell Plan

LOS ANGELES -- The California Stem Cell Report will bring you gavel-to-gavel coverage beginnin this morning of today's meeting of the governing board of the California stem cell agency, which is expected to approved an $890 million spending plan for the next five years.

The proposal includes an ambitious, $150 million effort to join next year with a private partner to develop the best of the agency's offerings that do not yet have a private partner. To entice business, the agency is putting up $75 million. It expects the private partner to put up another $75 million.

You can read about all that and more on the California Stem Cell Report as the day progresses. Stories will be filed as warranted.

Today's meeting begins at 9 a.m. PST.

More Info on the $30 Million California Stem Cell Agency Plan for Life After 'Death'

More information emerged this afternoon concerning the California stem cell agency’s $30 million plan for its life beyond 2020, when its funding runs out for new awards.. The agency posted documents late yesterday saying that it had pledges of $7 million from private donors contingent on raising an additional $23 million. One of the presentation slides posted online used the term “wind-down.” Earlier today, the California Stem Cell Report asked the agency for clarification of the sketchy information posted on the agenda for tomorrow’s stem cell board meeting. Kevin McCormack, senior director for communications for agency, responded,

“The $30 million is to help ensure that we have funds to cover the administration of all the awards we make in the next five years. As you know those are multi-year awards so if we can continue making new awards till, say, 2020 we also want to make sure we have enough money in our admin bucket (which is separate fro the research bucket) to cover the supervision and support for those awards.

“We are also but separately working on finding other sources of funding so the agency as a whole can continue funding and administering research after our current money runs out , but that is a separate task.”

Presentation documents posted online made reference to public funding, but had no specifics. Currently the agency survives on money borrowed by the state under the terms of the ballot initiative that created the research program in 2004. However, that $3 billion source is shrinking, and the agency is down to its last $890 million. The online documents made no mention of plans to continue with new awards or loans beyond 2020. Jonathan Thomas, agency chairman, is expected to provide more details tomorrow to the board on the financing plan.

Surviving after 2020: California Stem Cell Agency Reports $7 Million in Private Donations

The California stem cell agency today unveiled the first concrete indication that it will have significant cash after 2020, when its funds are scheduled to run out for new awards.

The news came in a memo quietly posted last night on the agenda for tomorrow’s meeting in Los Angeles. The memo said the agency had received pledges of $7 million in private support that appear to be the first installment in a $30 million, four-year funding plan beyond 2020.

Details were not immediately available. Jonathan Thomas, chairman of the agency, is scheduled to brief board members on the funding plan tomorrow. But based on the memo and a presentation document, Thomas appears to be looking forward to private funding along with some public support, presumably from the state of California. However, the presentation labelled the funding as a “wind-down” operation.

Bill Bowes, UCSB photo

The William K. Bowes, Jr. Foundation is providing $5 million to the agency, and the Franklin and Catherine Johnson Foundation is adding $2 million, the memo by James Harrison, the agency's general counsel said.

The Bowes Foundation in 2012 gave $5 million to the UC Santa Barbara stem cell program. Bowes is the founder of Amgen.

The memo said,

“These gifts are contingent upon CIRM raising an additional $23 million before June 30, 2019 and not having access to additional funds for its administrative costs.”

Currently the agency is financed with money that state borrows (bonds). The cash flows directly to the agency without intervention or control by the governor or the legislature, an arrangement established by voters in 2004 when they approved creation of the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

California Stem Cell Agency: FDA No. 1 Impediment to Stem Cell Therapies, 'Patients Are Dying'

California’s $3 billion stem cell agency this morning made it clear that it is not backing away from taking on the $4.9 billion Federal Drug Administration(FDA).

Randy Mills, MCC photo

“Patients are dying” because the federal government is “being so careful about safety,” Randy Mills, president of the agency, said in an item on its blog, The Stem Cellar.

 “Doing nothing is not okay,” said the headline on the piece, written by Kevin McCormack, senior director for communications for the agency. 

The FDA is too slow, too concerned about safety and needs to remove rules that are “bad for patients and regulators,” said the item.

Mills has been beating a drum concerning the FDA for a number of months, citing a non-scientific survey of agency stakeholders that showed 70 percent of them identified the FDA as the No. 1 impediment standing in the way of CIRM’s goals. 

Significantly, McCormack’s piece comes one day before the board of the California Institute of Regenerative Medicine (CIRM), as the agency is formally known, meets in Los Angeles to approve a plan that includes 50 new clinical trials over the next five years. All of those trials require FDA approval. 

In the proposal, Mills identifies FDA foot-dragging as one of the risks to successful completion of the $890 million effort.

 McCormack’s item said CIRM is “caught between a rock and a hard place. And CIRM is going to try and help them get out from under that.”

He quoted Mills’ speech to the World Stem Cell Summit last week. Mills said,

“We have had the current FDA regulatory structure for cell therapy in place for 15 years, and in that 15 years not one stem cell therapy has been approved. The scoreboard is not lying, there’s a zero on it. Not one therapy has been approved. There is an issue here, we can’t ignore that fact and so we made it part of our proposed new Strategic Plan to try and remove this burden. 

“There is an excessively long translational pathway to get an Investigational New Drug (IND) approval from the FDA (a necessary step to proceed with testing a therapy in a clinical trial). For non-cell therapies it takes 3-4 years to get an IND. For cell therapies it takes 6-8 years, twice as long.” 

Mills continued,

“We are not anti-regulation, we are not anti-FDA, and we are not calling for the removal of rules and regulations around stem cell therapies, that would be bad for patients and research. These therapies have risks and we are not proposing any strategy that puts things on the market without any testing or safety data. But right now we are being so careful about safety to ensure patients are not put at risk while those same patients are dying from their disease.”

In addition to Mills, one of the speakers at the World Stem Cell Summit was from the FDA. He said the primary mission of the FDA is safety. On the other hand, the mission of the California stem cell agency has long been “to accelerate stem cell therapies.” 

Full Coverage Thursday of California's New Stem Cell Direction

For all the latest doings by the $3 billion California stem cell agency, follow the California Stem Cell Report on Thursday for on-the-scene reporting as the agency officially approves its new course for the next five years.

Directors of the agency are scheduled to meet in Los Angeles to ratify a spending plan for its last $900 million. Also on tap is a risky, $75 million venture aimed at pushing the best of CIRM's research into the marketplace. 

The California Stem Cell Report will provide gavel-to-gavel coverage, but avid followers of the agency can listen in on the Internet or from two telephonic locations in San Diego and at Stanford. Details are on the meeting agenda.  

The Klein Legacy: Vestiges of California's Stem Cell Past to be Scrubbed This Week

No doubt exists that Bob Klein left his mark on the $3 billion California stem cell agency. Sometimes he is described as the father of the agency. He was its first chairman and led the drive to win voter approval of the research effort in 2004.

Bob Klein, Elie Dolgin photo

The agency has been under new leadership since 2011. And this Thursday some of the marks left by Klein are going to be erased.

Minor stuff now, but they recall some of the issues that rumbled through the agency in earlier days.

For example, the agency’s rules currently restrict to 12 the number of employees in the chairman’s office out of an agency total now of 55-56. The restriction is almost certainly to be removed on Thursday by the agency’s governing board. A memo by agency general counsel James Harrison said mildly that “disagreements” existed during the Klein regime about staff resources, leading to the limitation.

Those disagreements were actually sufficiently harsh that the stem cell board at one point in 2007 felt compelled to strip six employees from Klein’s office of chair, limiting him to four.

During Klein’s tenure, he also scheduled board meetings with jam-packed agendas that often took two days. The lengthy sessions tested the patience of board members, some of whom fled for the doors in an effort to catch their flights home as the hours wore on. The result was that the supermajority, legally required quorums required to do business were lost.. (See here and here for
more on quorum problems at the agency.)

To help avoid those unseemly situations, rules allowing telephonic attendance by key members of the board were enacted. Nowadays, the meetings proceed with dispatch, often ending early, much to the satisfaction of board.

So the board on Thursday plans to expand the use of telephonic meetings, which could well be a plus and a minus. The move will increase the number of offsite locations where members of the public and researchers can weigh in remotely with comments during meetings, a feature that has been lightly used. On the other hand, there will be less face-to-face contact between members of the board, something that is an important aid in finding solutions to touchy problems.

Head of NIH Troubled by Flagrant Violations of Law Requiring Public Disclosure of Research Results

The headline read “Failure to Report: Law Ignored, Patients at Risk.” The story this week began,

"Stanford University, Memorial Sloan Kettering Cancer Center, and other prestigious medical research institutions have flagrantly violated a federal law requiring public reporting of study results, depriving patients and doctors of complete data to gauge the safety and benefits of treatments, a STAT investigation has found.

"The violations have left gaping holes in a federal database used by millions of patients, their relatives, and medical professionals, often to compare the effectiveness and side effects of treatments for deadly diseases such as advanced breast cancer

"The worst offenders included four of the top 10 recipients of federal medical research funding from the National Institutes of Health: Stanford, the University of Pennsylvania, the University of Pittsburgh, and the University of California, San Diego. All disclosed research results late or not at all at least 95 percent of the time since reporting became mandatory in 2008."

Francis Collins, head of the NIH, said he finds the failure to report "very troubling."

The story was written by Charles Piller, West Coast editor of STAT, which is a new online news operation dealing with the life sciences. It was started by the owner of the Boston Globe. The STAT web site says it reports "from the frontiers of health and medicine."

Aussie Clinical Trial for California Firm's Stem Cell Therapy for Parkinson's Disease

A Southern California firm that repeatedly and unsuccessfully sought funding from the $3 billion California stem cell has received the go-ahead in Australia for human testing of a treatment for Parkinson’s Disease, it was reported today. 

In an article today in the San Diego Union-Tribune, reporter Bradley Fikes described the move involving International Stem Cell Corp. (ISCO) of Carlsbad as appearing to be a “medical first.” The company's stock price jumped nearly 16 percent today on the news.

Fikes wrote,

“If all goes according to plan, doctors will implant replacement brain cells into 12 Parkinson’s patients, probably in the first quarter of 2016, said Russell Kern, the company’s chief scientific officer. These are called neural precursor cells, a slightly immature kind of neuron. The cells will finish maturing in the brain into the kind of neurons destroyed by the movement disorder.

“The neural precursor cells are derived from the company’s parthenogenetic stem cells, which are produced from unfertilized human egg cells.”

International Stem Cell is a publicly traded firm whose researchers pitched proposals with some regularity to the stem cell agency a few years back. It was not known whether any of the applications involved the Parkinson's therapy. The firm’s personnel also attended CIRM board meetings with some frequency. (For earlier items on the company, see here, here, here, here and here.)

The day after Fikes' story appeared, the stem cell posted an item on its Stem Cellar blog about the effort.

Fikes wrote that the firm’s trial will be the first Parkinson’s trial  using replacement brain cells grown from stem cells, according to clinicaltrials.gov. The trial will be conducted by the firm’s Australian subsidiary, Cyto Therapeutics. 

Fikes reported that company’s effort is similar to other research in the San Diego area.

“That’s also the approach Summit for Stem Cell will take, said stem cell scientist Jeanne Loring, a leader of the Summit for Stem Cell project. The cells make proper connections with the brain better when they are still maturing, said Loring, who’s also head of the regenerative medicine program at The Scripps Research Institute in La Jolla.”

Loring is applying for an award from the stem cell agency but her research is at an earlier stage than that of the Carlsbad firm. Fikes wrote,

“Loring said she views ISCO as a partner in fighting Parkinson’s. One of her former students is working for the company, she said….

“ISCO’s choice of Australia for its streamlined regulatory process makes sense, Loring said. Her team, with U.S.-based academics and medical professionals, doesn’t have the same flexibility as ISCO in looking for clinical trial locations, she said.”

The firm’s stock closed at $5.00 today, up nearly 16 percent. Its 52-week high was $12.30 and low was $1.25. Here is a link to the company’s press release on the news today.

(The information concerning the agency posting an item on the San Diego news was added to this item 24 hours after it was first published.)

San Francisco Business Times: Risky Downside to California's $75 Million Stem Cell Venture Plan

The stem cell agency plan is dubbed ATP3. Here is some of the criteria.

California’s $75 million, stem-cell dip into venture capital waters poses an “embarrassing risk,” according to a story in the San Francisco Business Times.

The article by by Ron Leuty said,

“The strategy offers a potential high return for CIRM and the stem cell field in general, and it's not without the classic scientific risk of biotech investing. But it holds a larger, potentially more damaging and embarrassing risk as well if applicants peruse CIRM's shelves but find nothing to buy after the agency has invested hundreds of millions of taxpayer cash.”

Leuty wrote yesterday about the agency’s proposed plan, first reported last week by the California Stem Cell Report. The agency plans to entice -- with $75 million -- Big Pharma or other investors into a partnership. The private partner would also have to pony up $75 million. The combined enterprise would have first pick of the best of CIRM’s unpartnered research.

Leuty wrote,

"'The plan is not without risk, no doubt about that,' said CIRM President and CEO Randy Mills. 'Nothing about this plan is attempting to do something easy or easily achievable. This is hard and requires a lot of effort, but if we're successful the outcome of it should be transformative to CIRM and regenerative medicine.'”

Leuty continued,

“But there's a potential downside as well if nobody shows up to window shop at CIRM, much less to buy, because stem cell therapies are largely unproven, said Andy Schwab, a managing partner at Menlo Park venture capital firm 5AM Ventures.

"’The mechanism of action still is unknown. It's not like gene editing and gene therapy, where it's very specific,’ said Schwab, mentioning two of the hottest areas of medical science and investment action. ‘It's tough when you don't know the mechanism of action.’

“CIRM also hasn't invested in areas around white-hot CAR-T therapies, where chimeric antigen receptors on the surface of immune system T cells are genetically engineered to amp up their recognition and ability to kill cancer cells.

"’CIRM has really missed it,’ Schwab said. ‘They haven't been leaders in the right space.'

“Still, Schwab said, CIRM does have plenty of ‘really interesting research’ in its portfolio that could interest an investor. The issue, he added, is whether science projects can be commercially viable in a short timeframe.”

Luety continued,

"’It's an innovative solution to what they've been trying to do — to rush basic research concepts into actual treatments that meet people's medical needs,’ said John Simpson, an advocate and former stem cell project director with Consumer Watchdog, a Santa Monica nonprofit that for years tracked CIRM policies and spending.

"’There are potential pitfalls if (the spinout) goes to somebody's buddies,’ Simpson said. ‘But as long as the board does its job and closely vets the awards, this could have some real payback.’"

Responding to a query today from the California Stem Cell Report, Kevin McCormack, senior director for CIRM communications, said that "under CIRM’s IP (intellectual property) regulations, a company that licenses CIRM projects and commercializes them would owe a royalty to the general fund of the State of California(not CIRM). However, we are contemplating awarding funds to the successful ATP3 applicant as a loan, and under Proposition 71, the proceeds of a loan are paid to CIRM for the purposes of making additional research awards. So it is possible that, in addition to the royalties it would owe the general fund as a result of licensing and commercializing CIRM projects, the successful awardee may also owe money to CIRM. "

The $75 million proposal comes up for ratification by the agency board next Thursday at a meeting in Los Angeles.

(Editor's note: The paragraph in this item dealing with IP was inserted shortly after the original version of this item was posted.)

Stem Cell Cures: Who is Going to Pay?

Money and the FDA were on the agenda today over at The Stem Cellar.

The Cellar is the blog of the $3 billion California stem cell agency, whose minions were on the scene in Atlanta for the World Stem Cell Summit.

Kevin McCormack, the agency’s senior director for communications, wrote about the FDA, only a couple of weeks after the agency took on the FDA for slowness and excessive caution. Don Gibbons, who also works in communications for the agency, wrote about the minor matter of money -- just who is going to pay for stem cell therapies.

First, the money bit, since as Gibbons points out,

“The bottom line: stem cell therapies will never be widely available if insurers won’t pay for them”

Gibbons wrote about a panel dealing with that dubious euphemism -- “reimbursement:”

“Elizabeth Powers of the IMS Consulting group suggested the audience pay close attention to the cancer market.  She said insurers and other payers of health care services are tired of paying for ‘statistically significant’ improvements in survival that only translate to a few weeks on average. She said payers are moving away from just whether a new therapy is different from prior therapies and want to be shown true value.”

Use of the word “reimbursement” is Big Pharma’s way of NOT saying that profits must be made if treatments are going to be produced. Unfortunately, the use of the term ill serves the industry by creating the impression among the public that they are being hornswoggled with jargon. The word smacks of entitlement, as if someone should guarantee the drug companies billion-dollar earnings.

McCormack focused on what he called a “clarion call” by Robert Califf, the deputy commissioner for the Food and Drug Administration (FDA), who has been with the FDA only eight months. Califf called for deeper patient involvement in drug development decisions.

McCormack also wrote,

“(Califf) says the first goal of the FDA has to be to protect the public, and that it’s hard to balance safety and innovation. ‘That’s an issue we struggle with every day.’”

McCormack did not link to the strong language concerning the FDA found in his agency’s new plan for spending $900 million over the next few years in search of a stem cell therapy.

Underlying both pieces on The Stem Cellar are two issues for the CIRM board to consider next week as it eyes the future. Does the agency, which will cost the California public roughly $6 billion including interest, want to fund research that is likely to lead to enormously expensive treatments that insurers and the federal government (via Medicare and Medicaid) will be reluctant and even refuse to pay for.

The usual argument is that prices will ultimately come down. However, we have already seen strong evidence to the contrary. The concerns have risen to the level of presidential politics and are not likely to vanish, regardless of the hopeful expectations of industry.

As for the FDA, when safety is the all-pervasive driving force of the FDA, it naturally will lead to slower action and caution on new treatments.  California’s stem cell agency, however, is looking for significant results in the next five years. Its money is running out. Cash for new awards will vanish in 2020. And the possibility of raising significant new funds is bleak if the agency cannot show major results after what will be 16 years of effort.

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California Stem Cell Agency: FDA Stalling Development of Stem Cell Cures

The FDA is one of the favorite whipping boys in Washington, D.C., but it is not often that a state governmental agency also decides to thrash the body that is charged with assuring the safety of the nation’s drugs and medical treatments.

California’s $3 billion stem cell agency, however, is doing just that. As part of its new plan for spending its last $900 million, the agency is lambasting the FDA for standing in the way of speedy development of stem cell therapies.

The general chorus about the failings of the FDA is large. This morning a Google search on the term “FDA whipping boy” turned up 155,000 results. A search on the term “criticism FDA approval process” produced 569,000 results including an entire Wikipedia entry. Of course, not everyone agrees that the FDA is too stringent in its drug regulation. (See here and here.)

It should also be noted that stem cell therapies provide novel challenges, much more so than conventional, new drug offerings.

Nonetheless, here is the text of what the California Institute for Regenerative Medicine had to say about FDA in its new strategic plan, which is expected to be approved Dec. 17 in Los Angeles.

“FDA Challenges and Obstacles

“We heard a resounding chorus frommost stakeholders of the enormouschallenges with the regulatory burdensplaced on cell therapy in general, andstem cell therapy even more so, by theFDA. Instead of the ever growing bodyof work in cell therapy, with its overallexcellent safety record, making thepathway to approval smoother, it seemsto many that the requirements imposedby the FDA are increasing.

“A recent therapy touted by the FDAas a success had such a high clinicaldevelopment burden placed on it thatby the time it was finally approved,standard of care had evolved andits market was significantly reduced,leading to liquidation of the company.Companies, and sadly patients, mustgo outside the United States, as faraway as Japan, to find regulatoryagencies willing to work successfullytowards approval.

“Ultra-orphan diseases still must reachthe same statistical burden, whichrequires larger effect sizes than seenin the majority of approved, successfuldrugs and biologics. Everyone hastheir own list of how the FDA makesit seemingly impossible to take stemcell therapy through the regulatoryprocess. In 2014, Japan recognizedthe differences in regulatory approachesneeded for regenerative medicine andtook action. However, the FDA does notappear to have the same motivation. Infact, in a disturbing turn of events, FDAhas recently began providing certainmembers of the U.S. Congress withcompletely one-sided information on thedangers of cell therapies encounteredin clinical trials. However, FDA failedto provided any context or balancedinformation regarding the safety recordof cell therapies that comprises the vastmajority published clinical literature. TheFDA appears to be literally lobbyingagainst the very therapeutic modalitythey are responsible for promoting.

“CIRM needs to join with Congress,academia, and patients, to bring aboutreal change to meaningfully balancerisk-benefit in FDA regulations andmore importantly, the FDA’s behaviorand willingness to grant licensure toeffective therapies.”

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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