Shameless Family Promotion: World's Best Leather iPhone Case

Looking for a terrific iPhone case with room for cash and cards as well? Look no further. 

The case comes in in different shades. Saddle black here.

You can find it at coveredplanet.com. The case is called The Traveler and is "the world's best leather iPhone wallet case." It provides "easy convenience and rugged protection" for people looking for more than the usual plastic. In other words, a perfect holiday gift.

But first a disclosure: Coveredplanet is an enterprise run by this writer's son out of Santa Barbara. He devised the case initially as a personal solution, inspired by the old days of cattle ranches, vaqueros and saddle-making on the California coast.

Coveredplanet says the case is "handcrafted here in the USA by some of world's best leather craftsmen, and we use only the highest quality materials - saddle grade leather, sturdy parachute thread, black matte finished polycarbonate cases and brass plated rivets - in our products. If you're not 100% satisfied, we also provide a 30-day return policy for your leather iPhone wallet case, but, we think, you'll never want to give it back."

Please check it out.  A 20 percent discount is available until Dec. 15. Type in FAF20 when ordering.

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.

California's New $105 Million Stem Cell Surge: The Creation of an Industrial Powerhouse

Highlights

National biotech interest likely

$75 million alone for public-private effort

High risk, high reward

More support for Alpha clinics

California is hoping to fire up the state’s stem cell industry this month with $105 million aimed at creation of an unprecedented, “industrial stem cell therapeutic powerhouse” on the nation’s West Coast. 

The money is coming from the state’s stem cell agency, which has already spent $2 billion on stem cell research in the last decade. The agency is now on its last $900 million and pushing hard to translate stem cells into therapies and cures. It is expected to run out of money for new awards in 2020.

Earlier this week, a key panel of the agency’s governing board approved the $105 million surge, a decision that is certain to be ratified by the full board at a meeting Dec. 17 in Los Angeles. The action will initiate a 2016 competition for the cash, which is likely to draw national interest, although the money can be spent only within the state.

The largest component of the $105 million is $75 million for a program that the agency has dubbed the “Accelerating Therapies through Public-Private Partnership” or ATP3. The award would be one of the largest ever made by the agency, which is formally known as the California Institute for Regenerative Medicine (CIRM).

Also approved was a $15 million round for an "accelerating center" and another $15 million round for a "translating center." All of the awards are scheduled to be made by late 2016. 

Under the ATP3 public-private round, the recipient would be required to match the $75 million from California taxpayers. The program would bundle together for continued CIRM funding the most promising current CIRM projects. Those projects -- a “portfolio of  ‘high risk’ but high reward projects” -- would be selected by the recipient for development for commercialization, the agency said in a memo.

CIRM’s plan for the project said,

“The aggregation of a basket of otherwise unpartnered CIRM projects offers the successful applicant ‘multiple shots on goal.’ This increases the probability of successfully developing and commercializing a stem cell treatment and makes significant industry investment in stem cell technology more attractive.”

The agency said in a presentation that industry is “beginning to show interest” in stem cell therapies but only 8 percent of CIRM’s 71 active therapeutic programs have industry partners.

CIRM’s presentation said the awardee must have a “top-tier” track record and “could be an established company, a spin-off or a new company with a team formed by Pharma, biotechnology or by an investor.”

CIRM said Californians would benefit from "creation of an industrial stem cell therapeutic powerhouse that increases the likelihood of the commercialization of stem cell treatments for patients with unmet medical needs."

The other CIRM award rounds that are up for ratification later this month aim also at bringing stem cell therapies into widespread use.

The $15 million accelerating center round, which is open to non-California-based organizations, would fund the following activities:

• “Regulatory management services (including IND preparation and submission)
• “Clinical trial planning and management, including site selection, contracting, and clinical site management (e.g., patient recruitment and operational and logistical support)
• “ Data management, biostatical and analytical services
• “Provision of services to (CIRM’s) Alpha Clinics Network, CIRM-funded investigators and sponsors with translational and clinical stage projects, and clinical sites seeking to join the Alpha Clinics Network”

The translating center would fund the following activities:

• “Project planning in coordination with CIRM and the Accelerating Center.
• “Project management for pre-clinical IND-enabling development programs, including pivotal pharmacology and toxicology studies.
• “Process development and product manufacturing activities suitable for production of cell products under good manufacturing practices (GMP).
• “Assembly and authorship of documents to support IND submissions and FDA interactions.
• “Development and execution of a business plan to sustain operations beyond CIRM funding.”

Less Than $1 Billion Left: California Stem Cell Agency Lays Out its Five-Year Bucket List

CIRM's spending plans by area -- CIRM graphic

Highlights

Three pages on risk of research

Funding ends in 2020

Two new centers proposed, up to $15 million each

Clear pathways to commercialization

The California stem cell agency today unveiled its plan for spending its last $900 million, calling for 50 new clinical trials and an increasingly hard-eyed focus on turning earlier stage research into therapies.

Between now and 2020, the agency’s strategic plan budgeted $620 million for clinical and translational research compared to $170 million for more basic efforts.   

The proposal builds on “radical” changes already underway at the agency and is not without risk. Indeed, the 45-page page plan devotes three pages to the subject of “risk.” Sherry Lansing, a longtime board member and former chair of the University of California regents, said in a press release,

“It’s an ambitious plan, but you never achieve anything worthwhile by playing it safe.”

Jonathan Thomas, chair of the agency’s governing board, said the strategic plan “began with us throwing out all our preconceived notions of what we do, and instead focused on what was possible with the time and money we have left.”

The California Institute for Regenerative Medicine (CIRM), as the $3 billion agency is formally known, is projected to run out of funds for new awards in 2020. It is unclear what if any cash can be raised from either public or private sources. However, Thomas is scheduled to discuss future funding prospects at the board’s Dec. 17 meeting in Los Angeles.

Much of the plan has been revealed in the past six months as Randy Mills, CEO of CIRM, briefed the governing board on its developing direction. Mills joined the agency in May 2014 after a career in the biotech business. The thrust of the strategic plan reflects his business background and a strong emphasis on measurable benchmarks and organizational clarity.  He launched an effort called CIRM 2.0 earlier this year in an effort to speed funds to clinical efforts and improve the quality of applications. (See here and here for earlier stories on the strategic plan.)

New to the spending plan -- at least in any kind of detail -- were two proposals slated for $12 million to $15 million each. One was dubbed a “translating center.” The other was called an “accelerating center.”

The translating center will be located at a single enterprise and be charged with development of cGMP compliant cell manufacturing processes, providing “core services” leading to FDA investigational new drug applications and coordinating with the FDA and the accelerating center.

The accelerating center will also be located a single enterprise that has stem cell-specific regulatory expertise. It will provide support for clinical trials in an effort to accelerate the regulatory process and conduct of clinical trials.

Over the past six months or so, Mill has repeatedly made the case for capturing promising early research and ensuring that it has a clear pathway to clinical trials and ultimately into the market.  

Under his strategic plan, “every program will be integrated into and coordinated with the overall effort” to fulfill the CIRM mission: “To accelerate stem cell therapies to patients with unmet needs.”

The plan additionally sets concrete standards for measuring the agency’s performance as well as each team’s performance within the agency, including such specifics as the percent of application reviews held as scheduled and percent of projects that advance to the next stage.

The proposal is set for discussion and action at the directors’ Science Subcommittee meeting next Monday and goes to the full board on Dec. 17 for final approval. The meeting will be based at CIRM's new headquarters in Oakland with remote, teleconference locations for the public in San Diego and Los Angeles. Specific addresses are on the agenda.

While the plan is called a draft, it is not likely to undergo significant changes. However, interested parties can weigh in with comments by emailing them to CIRM at this email address kmccormack@cirm.ca.gov by 5 p.m. Dec. 3.

CIRM's goals for the next five years -- CIRM graphic

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

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

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

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

Cesca Stock Plunges 50 Percent in Wake of California Stem Cell Agency News

The stock price of Cesca Therapeutics yesterday plummeted about 50 percent after it reported bad news about its application for $10 million from the California stem cell agency.

The stock closed at 30 cents a share in relatively heavy trading, compared to its close of 59 cents the previous day. Over the last 52 weeks, the stock has ranged from 29 cents to $1.24.

On Monday, the company, which is based in the Sacramento suburb of Rancho Cordova, announced that it had withdrawn its application from the $3 billion state-funded agency until it assessed critical reviewer comments. Cesca’s proposal involves a roughly $20 million, phase three clinical trial to test a product aimed at critical limb ischemia.

The firm had raised financing for the trial that was contingent on CIRM approval of a grant.  

In its Monday press release, company president Robin Stracey said,

“We expect to spend the next several weeks re-validating and/or amending select elements of our plan, with a particular focus on the anticipated rate of patient enrollment, the overall timetable and the design of the statistical plan. Having said that, we remain very excited by the prospects for our program.”

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

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

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

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

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

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

The summary also said,

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

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

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

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

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

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

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

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

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

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

The announcement said,

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

Robin Stracey, Cesca photo

Robin Stracey, Cesca’s CEO, said,

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

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

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

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

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

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

Scripps' Loring Picked as Stem Cell Person of the Year

Jeanne Loring (center with sunglasses) and her lab team at Scripps in La Jolla

Jeanne Loring, head of the stem cell program at the Scripps Research Institute, last week was named Stem Cell Person of the Year.

The award was made by UC Davis researcher Paul Knoepfler, who funds the $2,000 prize personally. Loring, however, has declined the cash, and Knoepfler is looking for a cause to donate the money to.

Writing on his blog, The Niche, Knoepfler said that overall Loring “has had a transformative,

positive impact at least in part via taking risks and thinking outside the box.”

Knoepfler said,

“Jeanne came out as the winner for her exceptional contributions in 2015 and throughout her many years in the field. She not only has made numerous advances scientifically, but also gone the extra mile in many respects as an advocate and educator.

“Her scientific contributions include outstanding research on human stem cells and in particular in stem cell epigenetics. See her publications on GoogleScholar.  She has been a great mentor to her trainees. You can visit her lab page here.

“She has also been a creative leader in producing IPS cells from endangered species, an area with huge potential ecologically and at a societal level in terms of preventing extinctions.

“Jeanne has mobilized patient advocates and catalyzed exciting work in the clinical pipeline in a number of areas including most prominently in the last few years for Parkinson’s Disease.”

As for the destination of the $2,000 award, Knoepfler wrote,

“I’m currently considering whether to donate the funds to a charity or put them towards a novel educational outreach project in the stem cell field.”

George Bush, the California Stem Cell Agency and the Daily Beast: A Story for 20 Million Readers

The Daily Beast this morning carried a story with the headline “George W., Father of the Stem Cell Revolution.”

If that gives you pause, consider the Daily Beast’s next two paragraphs.

“It wasn’t what President George W. Bush had in mind. In 2001, Bush restricted the use of federal funding for embryonic stem cell research, giving conservatives what looked like a major victory in the nation’s culture wars.

“Three years later California thumbed its nose at the ban by starting its own multi-billion dollar stem cell program, and several states followed suit. Even though the restrictions were lifted in 2009, the insurgent movement survived and grew.”

The article was authored by Guy Gugliotta, who writes on science and public policy. The piece appeared both on the Daily Beast, which claims more than 20 million readers a month, and Kaiser Healthline, which is also carried on the Daily Beast. The article offers a lesson in unintended consequences for those who thought the federal restrictions would crush research using human embryonic stem cells. Gugliotta said,

“Today at least seven states offer stem cell research funding or other incentives to local scientists and industry.” 

The article covered the scene in states across the country, but dealt in more detail with the $3 billion California stem cell agency. Quoted was Randy Mills, president of the agency, as well as yours truly. Gugliotta wrote,

“’Without George Bush, this agency would not exist,’ said David Jensen, publisher of California Stem Cell Report, a blog focused on the California institute.” 

Bush’s restrictions created the justification for California to march -- on its own -- into the wilderness of stem cell research 11 years ago this month. Absent Bush's actions, there would have been virtually no perceived need for the state to embark independently.  

Gugliotta recounted the history of the agency and summarized the issues that have come up since 2004. He wrote,

“C. Randal Mills, chosen in 2014 as the institute’s new president and chief executive officer, said the organization is adjusting to ‘a world that has changed significantly’ since 2004 by moving away from simply funding good ideas in isolation to what he describes as a ‘system-based agency.’

“Last year the institute had 10 programs in clinical trials, but expects to have 20 by the end of this year.

“'We’re setting up continuous paths to move basic research to clinical trials,’ he added. ‘It’s like a train moving down a track, where each grant is the link to the next step down the line.’” 

Noting that President Obama has lifted the Bush restrictions, Gugliotta concluded,

“Despite the improved national (stem cell research) climate, states, both for economic and scientific reasons, have continued to fund their own programs. NIH lists initiatives in six states, not counting Minnesota, and other reports have suggested that as many as 15 states either have dedicated programs or fund stem cell research or did so in the past.

“Yet in a discipline that is just beginning to enter a translational phase, it is hard to evaluate the effectiveness of individual programs: ‘It’s a huge field, and it’s still early,’ said Heather Rooke, scientific director for the International Society for Stem Cell Research. “States will continue to do basic research, and California has certainly already had important influence driving the research to the clinic.’

“Results will take time, agreed Minnesota’s (Jakub) Tolar, but it is worth the trouble: ‘We started on drugs a hundred years ago. Then we went to monoclonal antibodies—biologicals,’ he said. ‘We are now getting ready to use cells as a third way of doing medicine. We are at a historical sweet spot.’”