Global Growth in 2020 for Regenerative Medicine While California's Stem Cell Program Faces Extinction

ARM graphic

SAN FRANCISCO -- Leaders of the stem cell and regenerative medicine industry gathered yesterday at a sumptuous hotel here, and the talk was of billions of dollars in business, not to mention medical treatments that can cost millions. 

Just 11 miles away across the famed San Francisco Bay, however, was another sort of stem cell scene. It could be found in the not-so-posh, Oakland headquarters of the state stem cell program, known officially as the California Institute for Regenerative Medicine (CIRM) . 

CIRM is going broke. Its original $3 billion has shrunk to $27 million over the last 15 years. 

On the other hand, global financing for therapies and treatments in the regenerative medicine field totaled nearly $10 billion in 2019, marking the second highest year ever, the leading U.S. industry group reported at the session at the Parc 55 hotel.

Janet Lambert, CEO of ARM -- ARM photo

"2019 (was) a year of significant growth in the regenerative medicine sector," said Janet Lambert, CEO of the Alliance for Regenerative Medicine (ARM), told a standing-room-only crowd of more than 500 gathered for its annual briefing. "We enter 2020 poised for continued expansion."

Lambert said,

"The pipeline is robust, with several next-gen technologies entering the clinic and an increase in clinical trials for indications with large patient populations."

Her comments came as CIRM faces a financing shortfall that could mean its virtual extinction. 

The agency, created in 2004 by state voters, is down to its last $27 million of uncommitted cash. CIRM has often spent close to $300 million a year. The agency is hoping voters next November will provide it with $5.5 billion more under the terms of a ballot initiative yet to qualify for the ballot. CIRM's only significant source of funding is bonds issued by the state. 

Lambert's organization has more than 350 members, including CIRM. The largest percentage of ARM's membership comes from cell and gene therapy companies. The global financing it reports is almost entirely private. 

ARM's 2019 survey tracked nearly 1,000 companies globally, including 534 in the United States. The second largest number, 237, includes Europe and Israel. Lambert said the fastest growing area is Asia, which has 180.

ARM counted 94 phase three clinical trials globally, the last stage before a therapy is approved for widespread use by the federal government. 

CIRM is helping to finance 60 clinical trials, including three that are still active in phase three. It has yet to back research that has led to a widely available product.

Here are links to CIRM's phase three trials: BrainStorm Cell Therapeutics,  $16 million, ALS, PI Ralph Kern; Humacyte, Inc., $10 million, kidney failure, PI Jeffrey Lawson; Medeor Therapeutics, $11 million, kidney failure, PI Karen Smith. 

Here is a link to Lambert's slides. In the near future, ARM expects to mount online a video of her presentation including two additional panel sessions that examine the current and future outlook for regenerative medicine.   

Gold-Plated Stem Cell Treatments, Multi-billion Dollar Benefits and Potential Industry Profits

One of the nation's leading regenerative medicine industry groups is touting multi-billion dollar savings that may be achieved with the type of stem cell and gene therapies that are being developed with cash from California's financially beleaguered stem cell program.

The industry group is the Washington, D.C.,-based Alliance for Regenerative Medicine (ARM). It is tackling one of the major issues facing development of commercial stem cell therapies -- sticker shock at their expected prices, running upwards of a $1 million or more.

Without a willingness from health care insurers to cover the costs and provide a pathway to profit, it is unlikely that the biotech industry will embrace production of the therapies.

In a study released last Friday, the group said,

"Advances in molecular biology and genetics are leading to new treatments for rare diseases that require new ways of assessing value. CGTs (cell and gene therapies) are directed at the underlying cause of a condition and offer durable, potentially curative, or near-curative benefits. These transformative therapies create challenges for current reimbursement frameworks as they (the therapies) require significant upfront costs but are expected to provide a lifetime of benefits. The recurring treatment costs of chronically-managed patients can be greatly reduced and even eliminated with a one-time administration or short course of these novel therapies. 

"As CGTs arrive on the market, payers need new models for assessing their value. These treatments could potentially end the patient’s burden of illness, resulting in cost offsets (eliminating or reducing the need for long-term treatment, hospitalizations, and other care) and productivity gains that span a lifetime. Manufacturers incur a high per-patient development cost for these therapies and payers who bear the cost of treatment may not realize the long-term financial benefits due to health plan switching."

The ARM study predicted cost savings of as much as $33.6 billion over about a decade in connection with three afflictions: sickle cell disease (SCD), multiple myeloma (MM) and hemophilia A (Hem A). 

California's stem cell agency was not mentioned in the study, but it has funded research in all three areas. The agency is a member of ARM.

The study, backed by ARM and performed by the Marwood Group, said,

"Access to CGTs for even a modest number of patients with MM, SCD, and Hem A each year can reduce overall disease costs by nearly 23% over a 10-year period. The savings from lowering healthcare costs and raising productivity are considerable, approaching $34 billion by 2029. Of the savings, $31 billion are from a reduction in healthcare costs and $3 billion are from productivity gains."

The model used by ARM assumed CGT prices as high as $2 million. The study said,

 "The model has tested more than 180 different prices across the three potential CGTs that ranged from a minimum test price of $150,000 and up to a maximum price test of $2,000,000. The prices entered into the model created 60 different cost savings curves for all three of drugs in this model. Prices were distributed with more than 50% of test prices in the $100,000-$600,000 price per administration range."

The discussion of the costs of stem cell therapies has special resonance in the Golden State where voters are likely to be asked next fall to give $5.5 billion more to its stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM).

The agency, funded with $3 billion in 2004, is down to its last $27 million. A new, proposed ballot initiative is focusing hard on affordability of stem cell treatments. The initiative has no specific solutions but stipulates that a new version of CIRM  -- if the ballot measure is approved -- should devise some ways to come up with answers for insurers who are not likely to warm easily to $1 million therapies.

California Stem Cell Agency's Business Partnering Program and BlueRock Therapeutics

A $240-million deal yesterday involved an East Coast biotech firm that has loose ties to California's nearly 15-year-old stem cell research program. 

The firm is Blue Rock Therapeutics, Inc., a privately held cell therapy company based in Cambridge, Mass.  Bayer owned about 60 percent of the company and picked up the remainder for $240 million in what it called a "major milestone." 

Last year, BlueRock was one of two companies that joined a program of the $3 billion stem cell agency to create a "built-in concierge service for the stem cell space." The agency, formally known as the California Institute for Regenerative Medicine (CIRM), called its new effort the Industry Alliance Program (IAP).

The agency said at the time, 

"The CIRM IAP is designed to give pharma, biotech and VC firms direct access to CIRM’s growing stem cell portfolio. These partners work in the stem cell and regenerative medicine field and will be connected to CIRM-funded scientists working on projects relevant to their interests."

Regarding the deal yesterday, Ben Fidler of Xconomy wrote,

"The deal gives Bayer rights to a company using cells harvested from healthy donors to develop cell therapies for a variety of different diseases—from neurodegenerative disorders like Parkinson’s to heart failure to graft versus host disease. But the buyout is a gamble on an ambitious technology whose first human study, in Parkinson’s, will start later this year."

So far, CIRM has not announced any fresh results from its industry alliance program. 

Stem Cell 'Teething Problems' -- Pricing to Manufacturing, the European View

From Europe this morning comes a perspective on the "three obstacles" that are blocking cell therapy from the mainstream.

It is a subject, of course, that resonates in the United States and particularly in California, where the $3 billion state stem cell agency is running out of cash and needs a boost.

The article appeared on Labiotech, which bills itself as "leading digital media covering the European biotech industry." Written by Helen Albert, the piece actually lists more than a trio of "teething problems" for the stem cell industry. They are:

• "A reliable, consistent and scalable manufacturing system" 
• "Excessively high pricing"
• Safety, regulation and a knowledgeable workforce

Albert interviewed a number of industry executives in Europe. She also mentioned Orchard Therapeutics, which has a tie to California.  It has received an $8.5 million award from the state stem cell agency, formally known as the California Institute for Regenerative Medicine.

The United States is ahead of the game on regulation, according to the folks in Europe. Albert wrote, 

"The US regulators are currently in the lead when it comes to keeping up with the research and being open to changing their processes, according to (Alan O’Connell, partner at Irish life sciences VC Seroba). Although he says Europe is not too far behind. 'Certainly, the EMA (European Medicines Agency) is responding, but from my perspective, the FDA is taking the lead, at least currently.'"

The Albert piece said, 

"With the first CAR T-cell therapies (technically a combination of gene and cell therapy) to treat blood cancers gaining US and European approval last year and global investment of over €6.7B in cell therapy in 2018, the question on everyone’s lips is ‘is cell therapy about to hit the big time?’"

Labiotech's answer? "2019 certainly appears to be the start, if not the end, of cell therapy’s move to the big time."

A Half Full Stem Cell Cocktail on Twitter

From a Twitter feed today

It was an interesting juxtaposition today on Twitter -- a vision of stem cell hope vs. a reality check.
The apparent contradiction is not uncommon in health science news.

It is a variation of the glass half-full or half-empty expression. But the stakes are not trivial. They involve deep emotions and the finances of the patients who face decades of impairment and death.

The perspectives also involve the mindsets of researchers and entities such as the $3 billion California stem cell agency. It has been wrestling for 12 years with the problem of producing a viable stem cell therapy for any sort of affliction. Its funds could well run out -- as expected in about three years -- with no cure to demonstrate for California voters who created the program in 2004.

Should the agency and researchers surrender because the task is enormous and is likely not to bear real fruit during their lifetimes? Should they move on -- if they could -- to something that would provide a more immediate, major public health benefit.

Can or should taxpayers support what some might call dreamy scientific aspirations while the state could use the billions to improve education and daily medical care for the poor?

Some may be able to come up with firm, black-and-white answers to these sort of policy and personal -- at least for researchers -- questions. This writer cannot.

As for the agency, its funds are legally locked for stem cell research or something akin to it. All the agency can do is spend the money well in that area -- a restriction approved by voters in the ballot initiative process.

Meantime, the public is hit with contradictory messages, becomes cynical and less ready to back research. All part of the challenge that faces patient advocates, researchers and drug companies today.

Industry Seeks More Clarity on Proposed Federal Stem Cell Research Rules

The nation's leading industry group for regenerative medicine today called for a clearer federal pathway to help bring stem cell products to the marketplace. 

The Alliance for Regenerative Medicine (ARM), which has 240 business, academic and patient advocacy members, testified today at a Food and Drug Administration hearing into proposals for more regulation of the stem cell industry. Randy Mills, president of the California stem cell agency, is scheduled to speak later today.

In remarks prepared for delivery, Michael Werner, executive director of the group, said,

"What all manufacturers have in common...is that they seek a clear and predictable regulatory pathway to market. In general, ARM believes that while the draft guidances are a positive step forward, they still leave some questions unanswered regarding interpretation of regulations . Consequently, ARM believes that when FDA finalizes these guidances, it should take actions to provide even more clarity."

Werner went on to make specific suggestions for more explanation on  requirements for product characterization and associated claims for each type of product.

The full text of his remarks can be found here.  You can find additional information in the texts of the group's comments that were submitted in writing  on "minimal manipulation" here and and on "homologous use" here.

The two-day hearing is being broadcast live on the Internet.

More Than a Minor Headache: The Stem Cell Snake Oil Problem vs. Legitimate Research

Just two weeks ago, the headlines from Bloomberg News offered a glowing view of the prospects for stem cell therapies and the industry. The story in the online publication, which counts nearly 9 million readers monthly, said,

"Stem cell crusader sparks new hope....Regenerative medicine could be a $120 billion industry by 2030"

But only three days later, that article was overwhelmed by a wave of stories with a much different flavor. They carried reports of unproven therapies, possibilities of fraud, lack of regulation and fearful, expensive consequences for desperate patients.

That theme continued even as recently as this past weekend as The Economist carried a piece headlined,

"A dish called hope: The flourishing, unregulated industry in expensive, experimental treatments"

So which is it? Hope for legitimate cures and oodles of cash for stem cell companies? Or "hope" for treatments that do not work and have sometimes damaged both the bodies and wallets of the patients?

The situation is more than a minor public relations headache for the supporters of stem cell research, be they patients, stem cell companies or the $3 billion California stem cell agency, which is on a campaign to ease federal regulation of stem cell clinical trials.

For all practical purposes, the public generally does not make much of distinction between the legitimate research conducted by institutions such as Stanford and the so-called "miracles" reported about the late hockey great Gordie Howe and others. It all goes into the same cognitive bag. The stories about mysterious and fearsome tumors found in little-known patients attract little widespread attention.

But UC Davis scientist Paul Knoepfler and bioethicist Leigh Turner of the University of Minnesota ripped open that bag with their study last month that reported for the first time that nearly 600 dubious stem cell clinics are peddling their unproven therapies across the country.  Their report supported the view that the stem cell field is rife with snake oil merchants. For the general public, which does not delve deeply into medical research, the perspective could well be described as, "You seen one stem cell therapy, you seen 'em all."

Does this mean that these clinics should be ignored and shoved quietly off into a corner in order to avoid besmirching legitimate efforts? Of course not. It certainly appears that the FDA and other  state regulatory bodies can do a better job. But stiffer regulations are not going to come anytime soon, despite an FDA hearing on the topic in September.

However, the situation DOES mean that folks like Knoepfler and Turner should continue to speak out along with many other scientists who do not want their efforts blackened by the snake oil men. Researchers can work with their institutions' PR departments to place op-ed pieces, find speaking engagements and gin up TV and radio interviews. Blogs, like the one produced by Knoepfler, can be started. The International Society for Stem Cell Research should revive its public education efforts to help patients and the general public understand the facts about stem cell research. It should also reinvigorate its warnings about dubious therapies, which were throttled back a few years ago, reportedly after legal threats were made.

California's stem cell agency has a special concern. It is trying to "de-risk" development of therapies  by a variety of means and lure biotech and Big Pharma into the stem cell game in a bigger way. This summer the agency is offering $75 million to entice a private partner into the state's first-ever, public-private partnership to create a stem cell cure.

The private sector has shied away from the stem cell business, as the stem cell agency's CEO, Randy Mills, has remarked on multiple occasions. One of the reasons involves the public climate and perception of the field. It is hard for companies to invest hundreds of millions of dollars or billions when the stem cell field is burdened with public perception and regulatory obstacles.

Selling the stem cell story in a realistic way is not necessarily an easy task. Nuances must be respected, but excitement is also needed. Balancing it all is a challenge for the men and women in the trenches. But without a good push, development of therapies will be slower, and, as Mills has noted, people will undoubtedly die who likely would have benefited from a more timely treatment.

Profitability in a $50 Billion Market: Fujifilm Projections and Cellular Dynamics

A Japanese firm that is the beneficiary of a $16 million award from the California stem cell agency was the subject yesterday of a piece in the Wisconsin State Journal.

The article by Judy Newman involved Fujifilm and Cellular Dynamics International (CDI) of Madison, Wis. The Japanese business bought Cellular Dynamics last April for $307 million. By that time, Cellular Dynamics had received the California cash to help create the world's largest public stem cell bank at the Buck Institute north of San Francisco. 

The stem cell agency has pumped more than $32 million into the project.

The occasion for the Wisconsin story was an interview with Kazuyoshi Hirao, CDI’s new chairman and CEO, and Chris Parker, executive vice president and chief business officer. The story also reported that Jamie Thomson, the famed University of Wisconsin researcher who founded the firm, is no longer employed by it. The newspaper did not elaborate.

Here are some highlights from the Q&A.

Parker said that Fujifim intends to bring to bear large-scale manufacturing practices on cellular production. He continued,

"Fujifilm believes regenerative medicine (using stem cells to treat diseases) will be a $50 billion market by 2025. Manufacturing will have to ramp up with it. We will have to look at leveraging other technologies to scale the business.

"The concept is that some day, we’ll each have our own iPS cells made and banked. So that when you need heart cells after a heart attack or you have eye damage from age-related macular degeneration, you pull your own cells out of the stem cell bank. Or you can use them to test drugs for toxicity before you take them.

"We’re envisioning replacing existing cells that will stay in the body and not be sloughed off later."

Hirao said, 

"We have a very aggressive target: 60 percent growth in revenue each year, for the next five years. We want our cells to be used for drug research and screening and for cell therapy — treating diseases."

Parker said, 

"CDI already has three cell therapy programs going — for age-related macular degeneration; Parkinson’s disease; and to replace scarred heart muscle after a heart attack — all using our iPS-derived cells. We anticipate being profitable by the end of 2017.

"CDI has 160 employees, about 120 of them in Madison. We expect to hire another 15 to 20 by the end of 2016, at least 10 of them in Madison.

Hirao said,

"We are trying to change the model. Current drug testing is performed, mostly, using animal cells. By 2019, we want the majority of drug testing to use iPS cells instead.

"Once the market is created, there will be a big jump in sales. That’s why Fujijilm has great expectations for this."

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.”

From Wounds to Regulatory Speed-Up: California Conclave Examines Stem Cell Business and Research

Highlights

The Grafix story

Japanese ambitions

California's Alpha Clinics

Hundreds of representatives of the world’s stem cell community are meeting today and tomorrow in California mulling over everything from pricing to the possibilities of commercial cures.

The occasion is the Stem Cell Meeting on the Mesa 2015 in La Jolla, Ca., and if you are not there, it is still possible to see some of the presentations live and later on video.

Some can be dramatic, including one from Lode Debrabandere, CEO of Osiris Therapeutics of Maryland. This afternoon he pulled up a slide involving an Osiris product called Grafix, which is “a cryopreserved placental membrane that is designed for direct application to acute and chronic wounds.”

The photographs on the slide showed an open wound with an exposed tendon before and after

Osiris/Meeting on Mesa graphic

treatment. Debrabandere said the Grafix treatment led to closure of the wound in five months. He said the patient "is walking around and still has his foot.”

Osiris is the firm once headed by Randy Mills, president of the $3 billion California stem cell agency, known officially as the California Institute for Regenerative Medicine(CIRM). Mills speaks tomorrow morning to the conclave. The agency is one of the major organizers of the three-day session and contributed $50,000 to the program.  Other organizers are the Alliance for Regenerative Medicine and the Sanford Consortium for Regenerative Medicine, which has received $43 million from CIRM.

The conference has received little attention in the mainstream media with the exception of the San Diego Union Tribune. The newspaper's biotech reporter, Bradley Fikes, has filed two major stories tied to the conference.

One dealt with the burgeoning number of stem cell clinical trials. The other explored the ambitious stem cell research effort in Japan. Fikes wrote,   

“In the second half of the 20th century, Japan emerged as a world leader in automobiles and consumer electronics. In the first half of this century, the country plans to do the same with stem cells and regenerative medicine.”

Fikes said the Japanese stem cell market “was estimated at $90 million in 2012, projected to reach $950 million in 2020, $10 billion by 2030 and $25 billion by 2050.”

Fikes also pointed out how the Japanese have streamlined the regulatory process, something that CIRM President Mills thinks the United States should emulate. Last week, Mills was in Washngton, D.C., talking to regulators and others, presumably advancing his case for faster action on stem cell therapies.

On the agenda tomorrow morning is a panel dealing with clinical trials at the Sanford Consortium. The effort is tied to the Alpha stem cell clinic effort at UC San Diego  (see here ), which is funded by $8 million from CIRM. The agency initiated the Alpha program, which totals $24 million, in an effort to develop  a world-leading, one-stop program for stem cell treatment.

The Mesa meeting program said, “The CIRM Alpha Stem Cell Clinic at UC San Diego provides infrastructural strength to enable the complex interaction required for success” in stem cell treatments. 

Stock of California Firm Jumps 17 Percent on News of Golden State Award

Google chart

ImmunoCellular Therapeutics’ stock price shot up 17 percent today in the wake of formal approval of a $20 million award from the California stem cell agency for a clinical trial involving a rare brain cancer.

The price has been climbing since last week when the California Stem Cell Report first disclosed that the company was set to secure the funding for the phase three clinical trial, the last step before widespread commercialization.

In an interview today, Andrew Gengos, president of the Calabasas, Ca., firm, confirmed that the trial is expected to cost $40 million to $50 million. The company has said it will add $34.4 million to the $20 million from the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Gengos said the five-year trial is expected to get underway in November with the enrollment of the first patients. He said it will probably start in the United States with expansion to Canada and Europe. About 400 patients are expected to participate.

ImmunoCellular has not had any revenues since 2010. Asked about the source of the $34 million, Gengos said the company had $31 million on hand at the end of June with no debt. He added that the company can access additional cash through the sale of more stock.

In reponse to a question, Gengos said he first became aware of the possibility of funding the clinical trial with the help of CIRM as the result of a conversation with a colleague at the J.P. Morgan health care conference in San Francisco last January.

Gengos followed up by asking an ImmunoCellular co-worker to look into the process. As the result, the company made its initial application which CIRM sent back with suggestions for improvement. In the second review, reviewers voted 9-0 to fund the project.

Gengos told the CIRM board today that the trial would take five years to complete.

“Frankly, this time period is outside the interest of most public market investors in terms of their investment horizon and therefore, in their eyes, handicaps our project compared to other projects that can execute in a shorter time frame.

“The result is that investment capital is hard to come by for these types of promising and highly innovative therapies when the investment horizon is long, and a small company without product revenues is at the helm.”

Gengos continued,

“I do not think it is an overstatement to say that without CIRM’s support, this program would not go forward. California’s innovative biotechnology community needs institutions like CIRM. Clearly – we need CIRM. Brain cancer patients need CIRM.”

Here is the text of the company’s press release today.

Here is the text of Gengos statement to the CIRM board.

   Statement by CEO of ImmunoCellular to CIRM board Sept. 24, 2015

Stem Cell Stocks to Grant Writing: One Researcher's Perspective

The president of the United States has his “state-of-the-nation” perspective but one California researcher has a somewhat more modest view of a smaller world.

Paul Knoepfler of UC Davis gave something of a “state-of-the-stem-cell-world” overview earlier this week on his blog, ipscell.com. He covered everything from grant writing to his investments in a stem cell company.

He wrote,

“Doing this blog for 5+ years has made it really sink in just how crucial the commercial side is to all of our shared goals. This means that things like stocks, patents, etc. are really important for us all to think about including academics, patients, students, and more.”

 He noted that he made a “very small” investment in Ocata Therapeutics, formerly known as Advanced Cell Technology.

But he said that the firm has been “getting its bell rung this year and in particular more recently it has dropped off a (small) cliff.”

On winning grants to continue his research, he said,

“In my 2+ decades in science in various positions I’ve never seen it quite this bad. These days it seems like I’m always working on at least one grant and sometimes several simultaneously. It eats up a lot of time, focus, and energy, but that seems to just be the new reality.”

 There is more. You can read it all here. Plus here is his perspective on other wide-ranging matters and his predictions about developments involving stem cells.

"Comfort News" for California's Stem Cell Research Effort

The California stem cell agency has enjoyed a spate of good financial and scientific news this week from the biotech industry as the research effort pushes on with its mission of turning stem cells into cures.

The $3 billion agency is scheduled to make its last grants in less than three years and, given the glacial pace of medical research, needs all the help it can get by then to bring a stem cell therapy close to the marketplace – the promise it made to voters when the agency was created nine years ago.

CIRM, as the agency is known, requires not only steady scientific progress but also a rosy outlook for the industry, which has languished in past years as major investors shunned the field. This week, CIRM garnered good news on both fronts.

There was enough so that the agency even touted it on the agency's research blog in an item by Neil Littman, CIRM's business development officer. He said it all helps to leverage CIRM investments and create a favorable investment climate. The good news included yesterday's announcement that Viacyte, Inc., of San Diego, Ca., has come up with $10.6 million needed to match a $10.1 million, much-ballyhooed award from CIRM last fall. The Viacyte financing includes important support from Big Pharma, in the form of Johnson & Johnson. CIRM has pumped a total of $39.4 million into Viacyte.

Another CIRM award winner, Cellular Dynamics International, Inc., of Madison, Wisc., yesterday announced its price on its upcoming stock offering to raise up to $53 million. Cellular Dynamics scored $16 million from the agency last March.

The “comfort news” for CIRM also included Monday's announcement that Capricor, Inc., a private Beverly Hills company benefiting from $27 million from the California stem cell agency, is merging with publicly traded Niles Therapeutic, Inc., of San Mateo. The merger is aimed at providing better access to capital.

And then there was Tuesday's news that a $20 million CIRM disease team award is paying off with the beginning of a clinical trial by Calimmune of Tucson, Az. for an HIV treatment.

All on top of the news in June when bluebird bio of Masschusetts brought in $101 million on its stock offering. Bluebird is the recipient of a $9.4 million CIRM award.

The rosy news comes amid a generally better outlook for biotech in general. John Carroll, editor of Fierce Biotech, this week noted that there were only 11 biotech stock offerings last year. He wrote,

“In the last 6 months, though, the industry has seen a tremendous rebound, with almost twice that number of IPOs in half the time. And there's no sign that the great leap into the public market is waning, with 10 more IPOs in the queue.”

Carroll's comments were echoed in a piece by Peter Winter on Bioworld headlined “Bubbleology and Biotech's Bull Run.”

All of this plays into what some might call the “everybody's-doing-it dance" or the “lemming syndrome,” depending on your point of view. The reality is that big investors and venture capitalists are timid souls and need the comfort of companionship-in-risk as they fork over tens or hundreds of millions of dollars on something that may not pay off for a decade or more. No one wants to be the out-front pioneer who winds up with financial arrows in his or her back. Being in a crowd provides an illusion of safety.

Of course, there is always the caveat about how markets and investors are fickle. A piece of bad news can translate quickly into major reversals as Apple has learned over the last year. Nonetheless, the folks at the stem cell agency have to  be feeling good today.

Light Coverage of Cellular Dynamics IPO But One Exec Says It's Good for Stem Cell Biz

A handful of media outlets today carried stories about the public stock offering announced yesterday by Cellular Dynamics International, Inc., a Wisconsin firm that will benefit to the tune of $16 million-plus from the California stem cell agency.

Kathleen Gallagher of the Milwaukee Journal Sentinel described the company, founded by stem cell pioneer Jamie Thomson, as in the business of making “fully functioning human cells in industrial quantities.”

Judy Newman of the Wisconsin State Journal in Madison, where the company is based, quoted Beth Donley, chief executive of Stemina Biomarker Discovery, as saying,

“It can’t help but increase the value of other stem cell companies.”

Thomson is a professor both at the University of Wisconsin in Madison and at UC Santa Barbara, and we queried Dennis Clegg, co-director of the Center for Stem Cell Biology and Engineering at UC Santa Barbara, about the school's ties to Cellular Dynamics, which hopes to take in $57 million in its public offering.

He replied in an email that Santa Barbara has a collaboration with Cellular Dynamics and the University of Wisconsin to develop a vision-restoring, stem-cell-based therapy for people with advanced retinal diseases. That $900,000 effort is financed by the Foundation Fighting Blindness.

The California stem cell agency grant to Cellular Dynamics is for work at the stem cell bank being created at the Buck Institute in Novato, north of San Francisco.

The Milwaukee Business Journal and Genomeweb also carried stories on the IPO.

ACT's Top Scientist Sells $1.5 Million in Company Stock

The mainstream media waxed enthusiastic last month when a California hESC clinical trial reported positive results dealing with blindness.

The report was first published account of a human trial of embryonic stem cell based therapy and involved Advanced Cell Technology, which is headquartered in Santa Monica, Ca. Despite a glowing reception of the trial's results, the firm is years away from being able to market the therapy at a profit – if it ever can do so.

The firm's chief scientific officer, Robert Lanza, moved quickly, however, to capture some monetary gain from the news, which was announced in a press release Jan. 23 by ACT.

On Jan. 23 and 24, Lanza sold 7.7 million shares in ACT for $1.5 million, according to SEC documents. He sold the stock at 18 and 19 cents a share. That compares to an ACT price of about 8 cents at the end of 2011. Lanza still holds 26 million shares in the firm. The acquisition price of the stocks is unknown.

There is nothing to suggest anything untoward about Lanza's sale. But it is a reminder that creating a successful stem cell therapy is about making money. Without a profit, there will be no therapy, as Geron reminded everyone last November when it dropped its longstanding hESC trial.

The California Stem Cell Report has asked Lanza if he has any comments about the sale of the stock. We will carry his remarks verbatim when we receive them.

The Seeking Alpha web site appears to have been the first to report the sale. Here is their complete item.

"Advanced Cell Technology, Inc. (ACTC.OB): ACTC is a development-stage biotech focused on the development and commercialization of human embryonic and adult stem cell technology in the field of regenerative medicine. On Wednesday, Chief Science Officer Robert Lanza filed SEC Form 4 indicating that he sold 7.7 million shares for $1.5 million, ending with 26.0 million shares after that sale. ACTC shares have rallied strongly since the beginning of the year, up from 8.2 cents at the end of last year to currently in 14-15c range after rising above 20c just earlier this week."

CIRM Leads National Effort to Forge 'Pathways' with FDA

California's $3 billion stem cell agency has created a broad-based, 43-member, interstate consortium of researchers and businesses to work with the FDA to develop “well-defined regulatory pathways for stem cell therapies.”

CIRM President Alan Trounson is high on the effort, which will be making another quasi-public appearance in just a few days. Last spring, Trounson told CIRM directors that he is “thrilled” with the initiative, which is spearheaded by CIRM General Counsel Elona Baum.

The next consortium program comes up on Sept. 28 – a “webinar” on preclinical considerations for stem cell therapies. The session, which requires advance registration, will feature Mercedes Serabian, chief of a key pharmacology/toxicology branch in the FDA; Robert Deans, senior vice president of Athersys, Inc., of Cleveland, Ohio, and Melissa Carpenter, a stem cell industry consultant and formerly with Geron of Menlo Park, Ca., StemCells, Inc.,of Palo Alto, Ca., and Novocell(now ViaCyte) of San Diego.

Baum has already conducted one webinar on issues on product characterization, which is now available for viewing on the CIRM Web site along with additional materials. Trounson said Baum “has been fantastic in getting the FDA along.”  Trounson told CIRM directors in April,

“If you were able to take the time to listen to these webinars, they're terrific. The information that's coming from the FDA and from the scientists is very, very helpful. And it's a learning process that's going both ways.”

The 43 members of the consortium include George Daley of Harvard, Irv Weissman of Stanford, Owen Witte of UCLA, Tom Okarma, CEO of Geron; Ed Field, president of Aldagen, Inc., of Durham, NC; John West, president of ViaCyte, Inc., of San Diego; William Caldwell of Advanced Cell Technology of Santa Monica, Ca.; Randy Mills, president of Osiris Therapeutics, Inc.,of Columbia, Md., and executives from Pfizer, Johnson & Johnson, iPierian of South San Francisco, Ca.; Genzyme of Cambridge, Mass., and the WiCell Research Institute of Madison, Wi., among others.

On its CIRM-based Web site, the consortium said that the stem cell field will move “faster when interested parties share best practices and resources.” The consortium said,

“This group provides a forum for members to discuss amongst themselves or with the FDA issues of importance to the industry. The RMC's mission also includes serving as a technical resource during development of guidelines and standards.”

The consortium Internet site contains a wide range of links to FDA, ISSCR and other materials. In addition to the Sept. 28 webinar, the consortium has a link to a Nov. 2 FDA workshop on cell and gene therapy trials.

Stem Cell Information Resource

A Silicon Valley firm has compiled a fine list of sources dealing with stem cell issues and regenerative medicine, ranging from investment info to links to reports on stem cell research in China and Singapore.

The list of resources was put up by Proteus Venture Partners. Many of the sources are familiar but the list is long and comprehensive. Sad to say, it does not include the California Stem Cell Report.

You can find the information roundup here.

ACT Seeks hESC Clinical Trial

Advanced Cell Technology has become the second U.S. company to seek permission to begin clinical trials for a therapy based on human embryonic stem cells.

The Santa Monica, Ca., firm said yesterday that it has applied to the FDA to start trials aimed at reversing vision loss with retinal cells created from the stem cells.

The firm, which has labs in Massachusetts, is only the second business to seek to perform clinical trials involving hESC. Geron, another California company, was the first, but its efforts are now on hold until late next year because of FDA safety concerns.

Neither of the firms has received funding from the California stem cell agency. ACT, however, moved its headquarters to California, partly to seek funding from CIRM.

The firm's announcement received modest news coverage. Steve Connor of the Independent in Great Britain quoted Robert Lanza, ACT's chief scientific officer as saying,

"'We've seen absolutely no adverse effects whatsoever in any of the preclinical experiments and our cells are more than 99.9 per cent pure. We certainly expect them [the FDA] to come back with comments and questions but our hope is that we will start sometime early next year. We're optimistic and certainly confident in our own data. We've been in dialogue [with the FDA] and we know what was on their mind and what they wanted us to do. We're hoping, assuming no hitches, to begin early next year, perhaps March."

Here are links to additional coverage: Los Angeles Times, Science, Nature, MassHighTech.

Scripps' Loring Teams with International Stem Cell Corp.

The Scripps Research Institute and scientist Jeanne Loring are collaborating with International Stem Cell Corp. of Oceanside, Ca., to study parthenogenic stem cells and their potential therapeutic use, it was announced today.

In a news release from the company, Loring, director of the Center for Regenerative Medicine at Scripps, said,

“"Parthenogenetic cell lines are uniquely valuable for our quest to understand the genetic and epigenetic processes that control stem cells. We are excited about the opportunity to collaborate with scientists at ISCO."

International Stem Cell said its scientists have derived a type of cell found in the human eye from parthenogenetic stem cells that may have application in treatment of macular degeneration. The company said it and Loring and will study these cells as part of the collaboration.

Kenneth Aldrich, chairman of International Stem Cell said,

“Our intent is to begin to provide these parthenogenetic stem cell lines to advance the field of regenerative medicine, as well as to commercialize our cells for cell transplant therapies.”

The firm has applied for grants from the California stem cell agency, but so far has not been successful. CIRM, however, is currently trying to link more closely with industry.

Oceanside Firm Disappointed by NIH hESC Proposal

A California stem cell company has filed a strong critique of the NIH's proposed rules for human embryonic stem cell research.

International Stem Cell Corp. of Oceanside Tuesday said it was “surprised and disappointed” by the draft guidelines.

The company said the government is proposing to fund research that involves the destruction of human embryos while barring funding for research that uses cells from unfertilized eggs (parthenogenetic stem cells). International Stem Cell uses unfertilized eggs.

The firm said in a news release that use of such cells has been approved by CIRM and three independent embryonic stem cell research oversight committees in the United States.

International Stem Cell also said the proposed NIH rules will limit opportunities for U.S. researchers, potentially lead to lost jobs and researcher flight to other countries and create a de-facto monopoly for two organizations that control most of the patents for current embryonic stem cells.