Wednesday, January 31, 2018

The Unregulated Stem Cell Wildfire: Fresh Look at Practices Not Approved by the FDA

They have crept across the country like a malignant rash, popping up from coast to coast. They take tens of thousands of dollars from desperate people from seeking a cure for afflictions ranging from cancer to blindness. Instead some become more ill.

You can see it yourself on your computer screen in the comfort of your own home. Just click on the map above. It was brought to you by Paul Knoepfler of UC Davis and Leigh Turner of the University of Minnesota. Back in 2016, they published the first ever look that documented the size of the unregulated stem cell clinic market. And California led the way with the most clinics.

Recently, they probed more deeply. Knoepfler wrote last week on his blog,
"In our new paper, Leigh and I also found that clinics marketing certain conditions and types of stem cells exhibited specific trends over the years. For instance amniotic stem cell clinics, while lagging behind bone marrow and fat stem cell clinics in numbers, show a sharp upward trend. This spike fits with increasing mainstream media advertising efforts by amniotic clinics.
The Regenerative Medicine article is ensconced behind a pay wall, but here is a snippet from its summary.
"Between 2009 and 2014, the number of new US stem cell businesses with websites grew rapidly, at least doubling on average every year. From 2014 to 2016, approximately 90–100 new stem cell business websites appeared per year. In contrast, from 2012 to the present, regulatory activity in the form of FDA warning letters has been limited. These data point to a problematic disconnect between a rapidly expanding US direct-to-consumer stem cell industry and limited FDA oversight of this marketplace. More consistent, timely and effective FDA actions are urgently needed."
Knoepfler said on his blog,
"While Leigh and I documented the nearly 600 clinics as of 2016, my sense is that there are likely to be upwards of 700 clinics today. What will the map of stem cell clinics look like in 2019 or 2020? What do you think? Fewer? More? Or about the same number of clinics? What would similar temporal maps of clinics in other countries look like?"

Thursday, January 25, 2018

California's Stem Cell Story Gains National Attention on NPR

California's $3 billion stem cell research effort broke into national news this morning when National Public Radio (NPR) picked up a lengthy overview of the Golden State's 13-year-old program to develop therapies that could treat everything from cancer to incontinence.

While the story chronicled the pluses and minuses of the work of the California Institute for Regenerative Medicine (CIRM),  as the agency is formally known, overall the piece bolstered the agency's efforts to tell its story to millions of more persons. It could be said to fit into the category of "I don't care what you say about me, just as long as you spell my name right."

NPR says it reaches 99 million people monthly across its platforms.  Roughly 12 million of those persons are likely to be found in California, given that the state has about 12 percent of the nation's population.

The stem cell agency piece first appeared last week in California on the web site of a popular media outlet, KQED, which produces TV and radio news. KQED's syndicated California Report is expected to air a broadcast version of the story and has linked online to the longer version as well.

All of this meets the need of the agency to reach the millions of California voters who are likely to vote on a possible, $5 billion bond measure in November 2020. The agency is slated to run out of money by then and will whither away without additional cash.

While some in the media would be loath to admit it, they tend to run in packs, chasing the same stories for a variety of reasons. The widespread appearances of the article by David Gorn could well stimulate additional media coverage in the shorter term and also help to shape coverage over the next couple of years or so.

Wednesday, January 24, 2018

A Man Named Lucas and the Future of the California Stem Cell Agency

Lucas Lindner, photo by Cait Covers the Bases
The news last fall from a California stem cell firm was leaden and dense. Terms such as "Phase 1/2a SCiStar study" and "Full enrollment of Cohort 3 (AIS-A; 20 million AST-OPC1 cells)" littered the company's statement. 

Today the state's $3 billion stem cell agency turned the jargon into a heart-warming, human story -- the type that is critical to sustaining the life of the 13-year-old research effort, which is running out of cash.

The story involves Asteria Biotherapeutics, Inc., of Fremont, the California Institute of Regenerative Medicine(CIRM), as the stem cell agency is formally known, a clinical trial for a treatment for spinal injury and a young man named Lucas Lindner.

He is one of the patients in Asterias' clinical trial for a human embryonic stem cell therapy for spinal cord injury.  CIRM has funneled $21 million into the research. 

Writing on the agency's blog, Kevin McCormack, senior director for CIRM communications, said,
"On a Sunday morning in early 2016, Lucas Lindner was driving to get some donuts for his grandmother. A deer jumped in front of his truck. Lucas swerved to avoid it and crashed, suffering a severe spinal cord injury that left him paralyzed from the neck down."
But after the treatment, McCormack wrote,
"Lucas can now type 40 words a minute, use a soldering iron and touch his pinkie to his thumb, something he couldn’t do after the accident.
"In August of last year Lucas did something else he never imagined he would be able to do, he threw out the first pitch at a Milwaukee Brewers baseball game. At the time, he said 'I’m blown away by the fact that I can pitch a ball again.'"
Of course, there are scientific caveats and qualifications in the story. But what the tale really can do is resonate with nearly all California voters if the story ever reaches them.

Those voters are key to continuing the work of the agency, which expects to run out of state funds in 2019 and is hoping that voters will pump an additional $5 billion into the agency through a bond measure on the November 2020 ballot. (A private fund-raising effort is underway to bridge the cash gap.)

The agency has largely functioned in obscurity during the last 10 years or so. Its story is not well known by the public. And it has yet to help finance a therapy that is available to the general public. But CIRM's 44 clinical trials, including the one by Asterias, provide hope for patients and the agency.

The key lies, however, in how the story is told and how well CIRM supporters can turn mind-numbing scientific jargon and public policy issues into compelling yarns that will open both the hearts and purses of California voters.

Friday, January 19, 2018

California Makes a $25 Million Kidney Transplant Wager: Its 44th Stem Cell Clinical Trial

The California stem cell agency this week more than doubled down on its bet on a potentially breakthrough treatment for kidney transplants, raising to $25.4 million its support for a project that is entering its final stages.

The hope is that the treatment will not only improve the success rate of kidney transplants but also lead to use in liver, heart and other solid organ transplants. If successful, the therapy would eliminate the need for immunosuppressive drugs in genetically matched kidney transplant patients.

Maria Millan, CIRM photo 
Maria Millan, president of the agency, said in a news release:
“These immunosuppressive drugs not only can cause harmful side effects, but they are also expensive and some patients lose their transplant either because they can’t afford to pay for the drugs, or because their effectiveness is not adequate."
The award also could help save the life of the stem cell agency, which is facing its financial demise as its funding runs out.

The award brings to 44 the number of clinical trials being assisted by the $3 billion agency, formally known as the  California Institute for Regenerative Medicine(CIRM). Clinical trials are the last stages of research prior to certification by the federal government of a treatment for widespread use.

The stem cell agency is hoping that one of its trials will soon produce a therapy that will resonate with California voters who may be asked in 2020 to provide $5 billion more. The agency expects to run out of state funding next year and is attempting to raise more than $200 million privately to tide it over until a bond election in  November 2020.

Action on the award was swift on Thursday. It took less than eight minutes for the agency governing board to unanimously approve an $18.8 million award to Medeor Therapeutics, Inc., of San Mateo, Ca. The award comes on top of a $6.7 million investment in the firm's research in 2016.

Steven Deitcher, Medeor photo
The vote simply ratified a decision on the Phase 3 trial that was already made by the agency's reviewers, who gave the research strong support during a meeting behind closed doors weeks earlier.

Steven Deitcher, co-founder and president of Medeor, said in the agency's news release,
"CIRM funding accelerates our timelines, and these timelines are what stand between needy patients and potential transformative therapies. This CIRM award combined with investor support represent a public-private collaboration that we hope will make a difference in the lives of organ transplant recipients in California, the entire U.S., and beyond."

Tuesday, January 16, 2018

A Look at Whether California's Stem Cell Agency Can Carry the Day Two Years From Now

One of California's more respected news operations today posted an overview of the state's $3 billion stem cell research agency, asking the question:
"Should California Voters OK $5 Billion More for Stem Cell Research?"
The piece by David Gorn on KQED's web site recapped the history of the program, its current condition and included comments from Robert Klein, the man behind the $5 billion proposal and who is often regarded as the father of the agency.

Barbara Koenig
 UCSF photo
Also included were comments from folks not always heard from publicly in the discussions about the agency. Quoted was Barbara Koenig, head of the bioethics program at UC San Francisco, who said she supports stem cell research but voted against the ballot measure that created the agency in 2004. She said,
“I didn’t like the overhyping of the immediate idea that there were cures around the corner.  I think we need to be honest about how we’re investing in research.”
Gorn continued,
"Ask Koenig how she might use that proposed $5 billion differently, and she responds with a moment of stunned silence.
"'Oh my, so many things,' she said. 'I would try to figure out how to make sure every child in California has access to basic health services, nutrition, clean water . . . not just make high-priced products, but to improve public health.'
"She said stem cell research privileges these quick-fix biotech approaches, which may make a lot of money but may not benefit the general public.'". 
Gorn's piece had some reach into the general public in the Golden State. In addition to its online news site, the San Francisco-based KQED operates a public TV station including news and  a public radio station with syndicated programming throughout the state. A version of today's story is expected to run next week on KQED's California Report, which the station says reaches 775,500 persons each week.

Gorn's report was pretty much down the middle, exploring the expectations for cures that emerged from the 2004 campaign directed by Klein, who also oversaw the writing of the initiative. His view is that that agency has "out-achieved" the promises of the campaign. Gorn wrote that Klein said that the campaign "never promised cures during the lifetime of the stem cell agency, only progress toward attaining them."

The governing board of the California Institute for Regenerative Medicine, as the agency is formally known, has all but endorsed Klein's current plan to place another initiative on the 2020 ballot, a presidential election year that will generate the large turnout that tends to favor bond measures.

Bonds -- money that the state borrows -- are the only significant source of cash for the agency. Ironically, the measure that created the agency -- also provided the seeds of its death by terminating its bonding authority after a specified period. Today the agency is down to its last few hundred million.

Zev Yaroslavsky, UCLA photo
Bonds additionally are usually used for long-lived assets such as roads and buildings and roughly double the cost of the research to taxpayers because of the interest expense. 

Gorn concluded with a comment from Zev Yaroslavsky, an authority on state politics and government at UCLA, as saying  that even if voter attitudes are highly favorable toward stem cell research that may not be enough to carry the day.

Yaroslavsky said,
"People do see stem cell research as something they have a stake in, but you’re going to have to explain what we got with the first $3 billion. I suspect their case with the voters will be that we need to keep momentum going. But the question is, ‘Will they buy it?’"

Friday, January 12, 2018

California Stem Cell Spending: Priming a $1.5 Billion Research Influx

California's $3 billion stem cell agency this week reported on "what you may not know" about the unusual, 13-year-old research organization. 

Its "disclosure" did not deal with such matters as the fact that it is the first such enterprise in state history or that it operates outside of the purview of the governor and the legislature. Rather it dealt with, among other things, leverage -- a term used often in the real estate business to describe, for example, using  $10 to create $1,000.

In the case of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known, it said it has created $1.5 billion in leveraged funds. It also noted that it has served as a "validator" for a significant amount of research, meaning that its awards re-assure the nervous nellies of finance about making an investment in a particular project. 

CIRM's seal of approval has led to $528 million in additional "partnership" funding for specific research, the agency said, along with more than $395 million in awards from other agencies that were built on CIRM-backed research. . 

News about the agency often focuses on stories about people and their afflictions. Those emotional tales are powerful ways to tell the CIRM story, but its less well known ventures into leverage and validation also speak to its impact, both short term and long term. 

Kevin McCormack, CIRM's senior director of communications, wrote about all this earlier this week with specific numbers and details in the agency's blog, The Stem Cellar, in a preview of the agency's annual report.
"Our goal is to do all we can to support the best science and move it out of the lab and into clinical trials in people. Obviously, providing funding is a key step, but it’s far from the only step. For us, it’s really just the first step."
CIRM does report its impact in the best possible light. However, there is little doubt that it has played an important role in "de-risking" much stem cell research and creating a friendly environment that is more likely to attract additional financing for a still young field. 

Wednesday, January 10, 2018

California Pumping $19 Million More into Stem Cell Effort to Improve Kidney Transplant Success

The California stem cell agency is set to make a nearly $19 million bet next week on a treatment that is aimed at significantly improving the success of  kidney transplants and helping to reduce healthcare costs.

The agency's board is expected to ratify a decision to award $18.8 million to Medeor Therapeutics, Inc.,  of San Mateo, Ca., for a phase 3 clinical trial, the last stage before its proposed product can be widely used.

Next week's award will bring to $25.4 million that the state has invested in the work. The agency awarded $6.7 million for the research in 2016.

The company's chief medical officer, D. Scott Batty, Jr.,  said last spring that its product, dubbed MDR-101, "has the potential to address the two most critical transplant patient needs: preventing organ rejection and mitigating anti-rejection treatment-associated toxicities. " He added that the technology could potentially be used in all solid organ transplant patients.

Fierce Biotech reporter Phil Taylor wrote last April,
"Patients who undergo organ transplants may no longer have to rely on lifelong immune-suppressing drugs, if Medeor Therapeutics has its way."
Taylor continued,
"More than 30,000 Americans get an organ transplant every year, and while success rates for these procedures are improving, it is estimated that up to a third of the most common transplants—such as heart, kidney, and liver—fail within 5 years."
The Medeor treatment uses adult stem cells to create a condition in which the transplanted kidney "is no longer viewed as foreign by the recipient," according a summary of the closed-door review of the company's application (CLIN2-10411). 

The agency's reviewers, who do not have to publicly disclose if they have  potential conflicts of interest, voted 11-1 to fund the research. The agency's board almost never overturns the decisions of its reviewers, who come from out-of-state. The names of persons reviewing specific applications are not disclosed by the agency, which also does not disclose the name of applicants until after board action. The California Stem Cell Report identified the firm from public records.

Samuel Strober, Stanford photo
Last November, Medeor announced it had raised $57 million in Series B financing, "led by RA Capital Management. Additional new investors included Sofinnova Ventures and 6 Dimensions Capital, who were joined by existing investors Vivo Capital and WuXi Healthcare Ventures."

The scientific founder of the firm is Samuel Strober, a professor of medicine at Stanford and who is a member of its scientific advisory board. 

The estimated date of completion of the trial is January 2022, according to The governing board of the $3 billion stem cell agency is scheduled to meet Jan. 18 to approve the award. No other action is scheduled.

Monday, January 08, 2018

California Stem Cells, 'Rapture' and a $5 Billion Bond Measure

This past weekend brought news about a "rapturous agreement among Republicans and Democrats" to support development of new therapies by financing biomedical research with billions of dollars.

The New York Times article indicated that the rapture could have something to do with the fact that many key political leaders on Capitol Hill are "aging in place."

The piece by Robert Pear also carried implications for the $3 billion California stem cell agency, which has virtually endorsed a yet-to-be-written, $5 billion bond measure to stave off its own death.

Pear wrote on Sunday:
"For the third straight year, lawmakers (in Congress) are planning to increase the budget of the National Institutes of Health by $2 billion. In the process, they have summarily rejected cuts proposed by President Trump.
"The push for additional funding reflects a fascination among legislators with advances in fields like molecular biology, genetics and regenerative medicine, even as they wage bitter battles over just how large a role the government should play in financing health care and providing coverage."
Pear continued, 
"Why is medical research so much less contentious than fundamental issues like health insurance coverage?
"Anthony J. Mazzaschi, a lobbyist at the national organization representing schools of public health, said that 'the charisma of the cure, the hope and promise of curing disease, seems to excite members of Congress,' including some in their 70s and 80s who are 'facing the prospect of disease and disability head-on.'"
The Times piece said, 
"The challenges facing patients and policymakers were illustrated this past week when a Philadelphia company said it would charge $850,000 for a new gene therapy to treat a rare inherited form of blindness. (The company, Spark Therapeutics, said it would pay rebates to certain insurers if the medicine, given in a one-time injection, did not work as promised.)"
California's stem cell agency is running out of money. It is looking at a November 2020 ballot measure to fill its shrinking coffers with $5 billion more. But such public policy decisions involve trade-offs concerning the health of Californians.

Several questions (among others) arise theoretically and otherwise:
  • Would it be better to spend $5 billion to provide much needed, immediate health care to poor Californians, including mothers and children, which could provide a quicker payoff in improved health. 
  • Is it better for the state to spend $20 million to help reduce infant mortality rates in Fresno and Mendocino that are nearly twice the statewide average or give the cash instead to a stem cell researcher (and his employing institution) who can also win the cash from the NIH. 
  • The impetus for creation of the California stem cell agency was based on a restriction in federal funding for human embryonic stem cell research. That restriction no longer exists, but could be restored by the Trump administration. But should California spend its billions when researchers can seek funding at the federal level? 
These are sort of bottom-line policy questions that ignore the fact that bond issues generally are used for long-lived assets, such as prisons, educational buildings, highways, etc., -- not services such as health care. The bond measure that provided the stem cell agency with its only significant cash was an historic exception.

Nonetheless, in the public's eyes the stem cell agency is all about health. As the campaign that created the agency in 2004 noted, medical problems that affect nearly half of California families "could benefit from stem cell research."

The likely cost of the therapies that are being developed in the agency's clinical trials are almost never discussed in public. Yet many are likely to be as expensive as the Spark Therapeutics blindness treatment. One can only imagine the kind of opposition advertising that is likely to surface in a campaign for a $5 billion bond measure: "Vote No on Million Dollar Cures for Billionaires!" On the other hand, that pitch might seem too harsh and blow back on agency opponents.

Nonetheless, Americans believe drug costs are too high, something that will come into play during a 2020 campaign. According a recent Gallup poll, 78 percent of the people also say total health care costs are too expensive. Drug costs have become an easy litmus test for the expense of staying healthy.

The New York Times story noted the importance of age as helping to create support for spending on medical research. California is headed for a sharp increase in the numbers of seniors in the next decade. One study is even projecting a "silver tsunami" of cancer in older patients nationally. Generally, however, older voters are considered more likely to be conservative. and may not buy into more government spending. How that plays out in California is not clear when it comes to stem cell research or the hope for stem cell cures.

Hope is one of the more powerful products of the California stem agency. Ultimately that could be the deciding selling point. But the November 2020 election is 34 months away, and much is likely to change.

Friday, January 05, 2018

Tracking the $3 Billion California Stem Cell Agency Month by Month

Directors of the California stem cell agency plan to meet 12 times during the coming year, including four valuable face-to-face sessions that are likely to be based at its Oakland headquarters.

Valuable because those sessions offer an opportunity to chat directly with directors and the agency's staff. The face-to-face sessions are scheduled for March 13, June 28, Sept. 20 and Dec. 13. Topics for the meetings will come later when agendas are posted 10 days before the sessions. But the June and December meetings have often dealt with important financial and strategic issues.

Researchers who have attended the board sessions in the past have benefited from gaining a greater understanding of the application review process and procedures of the board as it ratifies reviewer decisions and hears appeals.

The other eight meetings are telephonic and are scheduled to occur on Jan. 18, Feb. 22, April 26, May 24, July 26, Aug. 23, Oct. 18 and Nov. 29.

To be announced during the year on an ad hoc basis are meetings of the committees of the governing board, which regularly consider important issues and approve policies and actions that are often summarily ratified by the board.

All of the meetings can be tracked by subscribing to notifications from the agency and by reading the California Stem Cell Report, which tries to provide a deeper look at some of the matters facing the stem cell agency.

The public can attend all the meetings at either remote locations and the CIRM headquarters and can address the board directly. If you just want to listen, an audiocast is available. After the meetings, which are also accessible as a recording, transcripts become available a couple of weeks later. 

Places to Go, Stem Cell Science to See in 2018

The $3 billion California stem cell agency did all you stem "cellists" out there a big favor this week with a rundown of the hottest and most interesting conferences coming up in 2018. A UC Davis researcher also has put his own list on his own blog.

First, the agency's list of what it describes as "awesome" conferences. They range from industry-oriented events to ones devoted to science. Coming up next week is a "state of the industry" briefing by the Alliance for Regenerative Medicine in San Francisco. The World Stem Cell Summit runs at the end of this month in Miami.

A discussion of the agency's Alpha Clinics program is scheduled for April 19 at UCLA. Last year's Alpha program at the City of Hope was excellent. December will bring a Cedars-Sinai conference on Translation of Stem Cells to the Clinic, Challenges and Opportunities, which could be illuminating as the stem cell agency pushes to fulfill the promises of the ballot initiative campaign that created the research effort.

Of course, there are many more -- much too many to list here.The Oakland-based agency has also provided links to the conferences it highlights

Over at UC Davis, scientist Paul Knoepfler's list included some of the same sessions. He also is soliciting information on conferences that he has not listed. You can send him your information by clicking here. 

Wednesday, January 03, 2018

Stem Cell Person of the Year: Michele DeLuca of Italy

An Italian scientist has been named Stem Cell Person of the Year for 2017 by The Niche, a stem cell blog produced by a California researcher.

Michele DeLuca, CMR photo
Paul Knoepfler of UC Davis announced the selection of Michele DeLuca on his blog last week.
Knoepfler wrote,
"I chose him because of his exceptional track record in stem cell and regenerative medicine science combined with a long record of advocating for responsible translational science."
The award carries of a cash prize of $2,000, which Knoepfler funds himself. The California stem cell scientist commented,
"Dr. De Luca also led an international team that recently published a groundbreaking paper on epidermal regeneration in a pediatric patient with epidermolysis bullosa. The new paper reported an innovative combination stem cell-gene therapy approach to this terrible disease. I reviewed the paper here in journal club style. The manuscript is an important starting point in new therapy development. It’s the kind of work that is very risky and needs to be done in a meticulous way. Dr. De Luca is one of the few people in the world who could have led this work."
Knoepfler is the sole arbiter on the award but does ask his readers to indicate their preference. In the case of DeLuca, he received 2.98 percent of votes cast. 

Knoepfler started the awards in 2012 with an initial prize of $1,000.

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