Friday, September 23, 2016

A Look at Stem Cell Treatments in Mexico with Ties to California

First part of KBPS' stem cell treatment series

A San Diego television station this week aired a solid series exploring the world of unproven stem cell treatments, including therapies in Mexico and involving a California company.

The work was produced on KBPS by David Wagner and ran as a two-part series(see image above). UC Davis researcher Paul Knoepfler, who earlier this year documented the existent of 570 unregulated stem cell clinics nationally, described Wagner's work as "an important new piece...on for-profit investigational stem cell treatments."

Companies mentioned by Wagner included Stemedica Cell Technologies, Inc., Global Stem Cell Health, Inc., both in the San Diego area, and Hospital Angeles in Tijuana, just south of San Diego in Mexico.

Stemedica is the firm connected to nationally reported treatments of the late hockey great Gordie Howe and former professional quarterbacks John Brodie and Bart Starr. (See here and here.) Sphere: Related Content

Wednesday, September 21, 2016

California Stem Cell Research: $639 Million Left in the Golden State's "Big Bucket"

Randy Mills, head of the California stem cell agency, calls the cash
available for research awards "the big bucket." CIRM graphic
California's ambitious stem cell research agency has $639 million remaining in uncommitted cash as it continues to seek therapies and perhaps cures for afflictions ranging from spinal injury to diabetes.

Randy Mills, president of the 12-year-old program, announced the figure today at a San Diego meeting of the agency's governing board. He also said the program, formally known as the California Institute for Regenerative Medicine (CIRM), is expected to run out of funds in June 2020.

CIRM is financed by money that the state borrows -- $3 billion in bonds approved by California voters in 2004. Using borrowed money also leads to interest costs, increasing the ultimate taxpayer expense to roughly $6 billion. However, that estimate dates back to 2004 and no fresher figure has been produced by the state.

Mills reminded his directors that the agency is allowed only $180 million for operational expenses over the life of the program. About $61 million remains for those expenses.The current annual operational budget of $16 million pays for the roughly 55-person staff  to oversee $900 million in active awards and to make new awards.

The agency has handed out $2.1 billion over its life but many of those research efforts are finished. No therapies have been developed for widespread use.

The agency also has a "return rate" of 3 percent to 5 percent on awards.  The "return" is cash that is left over from an award or that is returned to the agency when a researcher does not meet research milestones, and the award is cancelled.  The returned cash is running at about $40 million annually.

Just what will happen to the agency come 2020 is unclear. Mills today said he and his team are monitoring the funds carefully so that awards and the operational funds run out at the same time.

The agency also may begin lose key staff as time passes and no plan for 2020 emerges, a possibility that Mills has publicly discussed, albeit briefly.

As for future funding, thoughts about another bond issue, public-private partnerships and more legislative funding have been bandied about. But nothing concrete has yet emerged.
Operational funds are described as "the little bucket" by Mills. CIRM graphic



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Wednesday, September 14, 2016

Time to Crank Up the Stem Cell Comment Machine and Weigh in on Proposed Federal Regulation

Folks interested in seeing more or less or different federal regulation of stem cell treatments and research have until Sept. 27 to file their arguments with the Food and Drug Administration (FDA) following two days of public hearings earlier this week. 

Patients, researchers and representatives of regulated and unregulated companies turned out personally Monday and Tuesday for FDA hearings on its new proposals. Witnesses included Randy Mills, president of the California stem cell agency. Questions were asked by an FDA panel but no conclusions reached. 

For those who missed the marathon sessions, they are recorded and are available online. One big advantage of watching them in a recorded format is the ability to skip through the content rapidly instead of having to sit and wait for the whole process to unwind. The first day can be found here and the second here

The live, online Internet broadcast chalked up about 770 views on the first day. On the second day, only about 550 were reported.  The numbers seem low to this writer based on the intense, emotional interest on the part of many patients. Industry also does not seem overly interested despite forecasts of the billions of dollars to be made from regenerative medicine, which may say something about the likelihood of handsome profits.

Only 15 written comments have been filed so far, mostly from patients. That number is likely to increase as industry and academia weigh in with more details. 

The FDA has not released a timetable for action which could affect many of the 570 unregulated stem cell clinics operating across the country. 

The hearings generated almost no media coverage. However, the Washington Post and Stat published good pieces (see here and here) that provided an overview of the issues ahead of this week's hearing. You can find coverage by the California Stem Cell Report here, here, here and here.

California Healthline carried a piece Monday with comments from Mills that basically summarized his presentation to the FDA. Today, the agency's blog carried an item with the text of the Healthline comments. The item by Kevin McCormack, senior director of communications, also said that Mills believes that "the rules the FDA is proposing will not fix the problem, and may even make it worse."

UC Davis stem cell researcher Paul Knoepfler summed up the second day of the hearings in a blog item yesterday. He quoted Leigh Turner, a University of Minnesota bioethicist, as saying, 
“The out-of-control marketplace for stem cell interventions needs effective regulatory oversight. I therefore hope the draft guidances are more than stage props and this hearing is more than public theater. When patient safety and public health are at stake, the FDA must do more than function as a paper tiger. It is time for action.”
Here is a link to the key Federal Register document laying out the process. Here is a link to the site for filing a comment electronically.
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Tuesday, September 13, 2016

The Wright Brothers, the Blind and Stem Cells: A Different Look at the FDA Hearing on More Regulation

A California stem cell researcher has filed a report that chronicles some of the details that help to provide a sharper picture of the events yesterday and today at 9000 Rockville Pike in Bethesda, Maryland.

Writing on The Niche blog, Jeanne Loring, head of the stem cell program at the Scripps Insitute, covered matters -- ranging from aircraft flight to the blind -- that came up at the Food and Drug Administration hearing into new regulation of stem cell therapies. .

Loring is at the hearing, which is underway again this morning, and will testify later today. Her item noted that the marathon yesterday included 42 presentations of five minutes each.

She wrote,
"The question asked by the FDA is 'what should we regulate?' and the answer from the majority of speakers was 'don’t regulate the things that we’re doing!'"
Loring continued,
"The clinics, in general, wanted the FDA to define the fat as having non-structural as well as structural functions. This would allow them to isolate cells from fat and inject them into the bloodstream, a popular treatment at many clinics, without FDA oversight.
"There were amusing incidents, such as when Randy Mills (president of the California stem cell agency)  used a metaphor to describe the FDA regulating rapidly developing stem cell therapies; it was, he said, as if the Wright brothers had just gotten their plane off the ground, and returned to find an FAA official who explained that he was going to regulate their planes. I also liked Arnold Caplan’s 'apology' for naming the stromal cells he extracted from bone marrow 40 years ago 'mesenchymal stem cells.' They aren’t stem cells, he said, but rather cells that secrete factors that may be useful for healing in some cases.
"At the periphery there were the victims of reckless stem cell clinics. A man wore a sign that said he was blinded by a stem cell procedure. A woman I met while standing in line for the bathroom told me that her husband and 6 others had been blinded at a Florida clinic. In his case, she said, he did not sign the paperwork that would prevent him from suing the clinic, so they’ve found a lawyer."
Loring predicted that today's session will include much from unregulated clinics. She said,
"From the applause (yesterday) whenever a speaker said they should not be regulated, I expect that most will be glowing. But I hope, for balance, that a few will report negative experiences. I haven’t decided what to say when my time comes (this) afternoon."
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Monday, September 12, 2016

California's Randy Mills: Flood of Demand for Stem Cell Therapies Requires New Development Pathways

The president of the $3 billion California stem cell agency today told federal regulators that patient demand is "very real" for stem cell therapies and called for "alternative" pathways for development.

Randy Mills at FDA hearing
Randy Mills said patients are "really suffering and their needs are not being met." He said the demand is like a flood of water and will find its way around any blockages that rise up.

Mills spoke during the first day of a two-day hearing by the Food and Drug Administration(FDA) into possible new regulation of the stem cell field.

The session came as the Washington Post published online a lengthy piece about unregulated stem cell therapies and the increasing scrutiny they are facing from regulators. Reporter Laurie McGinley wrote about a clinic in Beverly Hills with a self-described "Time Machine" box.

Mark Berman, who was described as the co-founder of the "biggest network of commercial stem cell clinics" in the country, processes stems cell through the box for a procedure that he says doesn't require FDA approval.

McGinley wrote,
"In mini-liposuction procedures, Berman extracts fat tissue from patients and puts it into the Time Machine box, where a centrifuge separates stem cells from fat. The cells are then injected back into the patients to supposedly treat a variety of health problems, such as arthritic knees, heart disease, amyotrophic lateral sclerosis and even autism."
The treatments run about $8,900 each. Berman said his network has treated about 5,000 persons, McGinley reported.

The Post piece said that such treatments and their regulation or lack of regulation has "ignited a fierce debate among physicians, patients, scientists and politicians about whether the (FDA) should crack down on therapies that critics deride as snake oil but that some patients swear by. And it is fueling a broader, longer-term debate over how cellular therapies should be regulated."

Stem cell researcher Paul Knoepfler of UC Davis , who is quoted in the Washington Post article, took in today's FDA hearing from his lab in California. Writing on his blog, he delivered some "take-homes" after the session concluded. Here are a few excerpts from what Knoepfler wrote.
  • "Some speakers advocated for a 'middle ground' between 'nothing' in the way of regulation for instance of the dubious clinics and the perceived 'mountainous' extreme of the current FDA oversight system. The middle ground was, for example, argued for by Janet Marchibroda of the BPC and then also in the next talk by CIRM President Randy Mills. Mills, who is a very powerful speaker, argued that there is a harmful binary system now and we need a middle ground between the extremes of little-to-no regulation and the current in his view constricted pathway."
  • "Several speakers invoked patient autonomy and patient rights, and argued that they are doing what they are doing as practitioners of stem cell therapies simply for the betterment of patients. There’s the pesky problem there with this argument of probably millions of dollars in profit being made from the patients by some clinics though. If it’s all about patients, I challenge the clinics to do the transplants strictly 'at cost' with no profit margin."
  • "Many parties asked the FDA for more clarity and consistency on oversight. Also, a number of speakers asked for concrete, real world examples of how definitions would apply.  I’ve been pushing the FDA for increased clarity for years including lay abstract summaries of their main guidelines and statements of current thinking. I really like the idea of flowcharts."
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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. Sphere: Related Content

Live on the Internet: Today's FDA Hearing on Stem Cell Regulation, Preview of California's Comments

The FDA's two-day hearing into stem cell treatments is underway this morning and can be seen live on the Internet, including a presentation this afternoon by the California stem cell agency, which is seeking to ease FDA regulation of stem cell clinical trials.

A likely preview of the comments from the agency popped up this morning on California Healthline in a piece by Emily Bazar. She carried the brief text of an interview with Randy Mills, president of the $3 billion California agency.

He said,
"The problem with (the FDA's current) strategy is twofold. It doesn’t address the patients, or the need side of the equation. And I don’t think it has a chance of actually working because the FDA will acknowledge that they do not have the resources to enforce these types of regulations at the clinic level.
"They would have to be essentially regulating the practice of physicians, which is well beyond their capabilities. Even if they were able to enforce it, it would just drive these patients somewhere else.
"We’re advocating for the creation of some middle pathway that would bring essentially unregulated therapies into the regulatory fold, but in a manner which could be complied with.
"I would rather know these clinics are being regulated and collecting data than have them operating under the radar screen of the FDA. I would like there to be a formal pre-market review of these therapies before they’re put on the market. I would like there to be safety and efficacy data.
"I’m going to try hard to get the FDA to see that just plugging this hole won’t make the problem go away."
Bazar's piece also contained the text of comments from Jeanne Loring, head of the stem cell research program at the Scripps Institute in La Jolla. She said,
"There’s no scientific evidence that the fat cells (unregulated) clinics are using are going to do the patient any good. And there’s no evidence that shows they are safe.
"I’m a stem cell scientist. I need scientific evidence before I will believe anything. Regulation will help determine the efficacy and safety of those fat cells.
"There are several lawsuits or potential lawsuits brewing over these stem cell treatments. People were promised they would get improvements and didn’t. And there are cases where people were actually harmed by stem cells.
"Some people truly believe they have been helped by the stem cell injections. I’m not going to argue with them. It’s a very personal and emotional response. It’s not something that can be scientifically validated. People are really desperate, especially really sick people and their families.
"That means patients are at risk. That’s what bothers me a lot. There’s nothing we can do to talk somebody out of going to a clinic if they feel that’s the only option they have. We just want to make sure nobody gets hurt. We also don’t want people to go broke. These treatments are not covered by insurance and they cost tens of thousands of dollars."

Here is a link to the actual broadcast, which is being watched by about 300 persons at this moment. An overview of the audience seems to indicate that it is being lightly attended. Mills is scheduled to speak at 4:23 p.m. EDT.

Here is a link to the agenda including a list of speakers and schedule. The broadcast also has closed caption capability. Sphere: Related Content

Friday, September 09, 2016

Center for Genetics and Society Calls Trounson Affair Scandalous

The Center for Genetics and Society, a longtime critic of the California stem cell agency, yesterday described the Trounson-StemCells, Inc., affair as "scandalous" and part of an "object lesson" in how not to set up a state agency.

Writing on center's blog, Pete Shanks referred to the disclosure that Alan Trounson, former president of the $3 billion research effort, had received $443,500 in total compensation from StemCells, Inc., of Newark, Ca., for his work on the company's board over a two-year period. Trounson was appointed seven days after leaving the agency, which came as an unpleasant surprise to the stem cell agency's governing board.

StemCells, Inc., was awarded $40 million while Trounson was president of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Shanks, an author and consultant to the Berkeley-based center, said,
"Did Trounson or StemCells Inc. do anything illegal? Quite likely not. Was this transaction appropriate? Absolutely not! It’s scandalous, but it’s the kind of scandal that was built into CIRM from its very inception."
Shanks recounted the conflict-of-interest issues that have troubled the agency even prior to voter approval of the program. He concluded,
"CIRM is now slowly running out of the $3 billion of public funds allocated to it in 2004, and is expected to wind up in 2020. It has provided an object lesson in how not to set up and run an independent public-funded agency. These latest revelations should end any speculation about extending its charter."
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Wednesday, September 07, 2016

Text of Irv Weissman's Comments Re Trounson Compensation

Stanford University stem cell researcher Irv Weissman has sent along the following comment in the wake of the publication of the Sept. 2, 2016, story concerning Alan Trounson's compensation from StemCells, Inc., a company co-founded by Weissman, who also served on its board of directors.
"I would like to correct some information that can be verified by CIRM officials and employees. I have known Alan Trounson for years before he came to CIRM both as a scientist and once on a fishing trip. After he got settled into his position at CIRM we met socially, and agreed to go on another fishing trip together. From that time forward he recused himself on all grants and CIRM related activities that involved either my lab or Stem Cells Inc. This was unfortunate for me, as there were times when he was the field expert and could have suggested appropriate reviewers, or mistakes in review, for grants coming from me at Stanford or Stem Cells, Inc. Although you mention the many successes we have had with CIRM grants, there were many more times when I failed to reach a funding score that was sufficient, sometimes with what I thought were inaccurate reviews,and sometimes with reviewers that I would have asked to be removed for conflicts outside of scientific expertise. I am happy to answer any questions on these issues for all grants on which I am a principal investigator, or as on the stem cell genomics grant, a participating but minor investigator who was the only person with purified stem and progenitor cells.
"I hope you dig into the actual current value of his or my stock, so that the public will know the real numbers. At least up to the wind-down of the company I believe I have never sold stock I hold, as I felt I always could have had information not generally available to the public, even when it was legal by SEC standards. In the early days of the company I contributed personal money to the company for stock so that the company could meet its payroll, and also I never sold those stocks. All of my SCI stocks are worth no more than and probably much less than 10% of that single contribution."
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