Showing posts with label knoepfler. Show all posts
Showing posts with label knoepfler. Show all posts

Thursday, May 10, 2018

Feds Crack Down on Unregulated 'Stem Cell' Clinics, Including One in California

The Food and Drug Administration has moved to shut down two businesses that offer "stem cell" treatments, including one operation based in California,  following reports that some patients were blinded by treatments.

The lawsuits yesterday were characterized as "historic" by one California stem cell researcher, who has been reporting on unregulated and unproven "stem cell" treatments for several years. 

The firms involved are U.S. Stem Cell in Florida and California Stem Cell Treatment Center and its proprietors, physicians Mark Berman, a cosmetic surgeon, and Elliott Lander, a surgeon and urologist. The California enterprise has locations in Beverly Hills and Rancho Mirage with affiliates in 100 locations around the country.

Michael Hiltzik wrote in the Los Angeles Times,
"The lawsuits are the most aggressive steps yet taken by government regulators against rapidly proliferating stem-cell treatment clinics pitching scientifically unproven therapies to desperate patients, some with terminal illnesses. The lawsuits were filed at the request of the Food and Drug Administration, which last year announced that it would take a stern approach to what it described as the manufacture and use of unlicensed drugs."
Researcher Paul Knoepfler of UC Davis has been raising questions about unregulated and unproven stem cell treatments for a number of years. Writing yesterday on his blog, he called the federal action "historic." He said the suits could mark "a turning point for dealing with the for-profit stem cell clinic problem in the U.S."

Here are links to other stories on the lawsuits, which received national attention: Washington Post, The Associated Press and STAT. The Associated Press was carried by the New York Times and many other outlets online. The Times also carried this story by its own reporters.  Here is a recent column by Hiltzik on the California Stem Cell Treatment Center. The headline on the colum says "Patient lawsuits, federal investigations and a 'virtual' PhD: Inside a would-be stem cell empire." 

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.

Sunday, December 03, 2017

Hope and the 'Helping Patients Part': The Uncertain Future of California's Stem Cell Program

One of the big selling points of the $3 billion California stem cell agency has always been hope. And it still is. 

It is a difficult matter to argue with. Hope underpins all our lives. But no more so than with persons suffering from terrible and incurable diseases. And those are precisely the targets of the 13-year-old stem cell research effort financed by the people of California. 

Currently the agency is on a course to run out of cash for new awards in 2019, ironically a fate dictated by Proposition 71, the ballot initiative that also created the agency in 2004. The measure provided $3 billion but no further stream of income. The agency is now wrestling with a variety of possibilities to extend its life for another decade or so, including mounting a $5 billion bond measure on the November 2020 ballot. 

A stem cell researcher at UC Davis, Paul Knoepfler, today addressed those critical matters -- and hope -- in a post on his blog, The Niche. He wrote, 
"The California Institute for Regenerative Medicine, more widely known as CIRM, has accomplished big things over the course of its history of about a decade and sparked a great deal of innovation, but what does the future hold for our stem cell agency?"
Knoepfler mentioned coverage of CIRM meeting last week by the California Stem Cell Report, noting that last week's item included the phrase "withering death." Knoepfler wrote,
"I don’t see things as so bleak and remain hopeful on the agency’s future. It’s unclear how California voters will be feeling about all this in 2020, but with our stem cell agency we are just now getting to the exciting part. The helping patients part. As a past grantee myself, I know how much the funding can make impact to advance science, whether basic, clinical, or somewhere in between. 
"At the recent brainstorming meeting on the agency’s future, there were apparently some upbeat reports of voter sentiment as described by (David) Jensen (producer of the California Stem Cell Report):

"'Robert Klein, a Palo Alto real estate investment banker who ran the 2004 campaign that created the agency, told the CIRM directors of a private poll that he said showed 70 percent of Californians supported stem cell research and continued funds for the stem cell agency.'
"What do you think about our stem cell agency’s future? 
"In terms of clinically relevant science, it is at the most promising point in its history with support for loads of clinical trials. Not all of them will work out, but I’m convinced that some will and other fresh trials that could be initiated with CIRM’s new funding in the future beyond 2020 would bring more hope."
The  California Stem Cell Report welcomes your thoughts on the agency's future and its strategy for getting there. You can submit them either by clicking on the word "comments" at the end of the item or by emailing them to djensen@californiastemcellreport.com.

Sunday, October 22, 2017

ViaCycte Update: Clinical Trials for a Diabetes Device Plus the Rain Gear Connection

ViaCyte, Inc., is a San Diego stem cell firm working on what could be a virtual cure for diabetes -- one that the $3 billion California stem cell agency has supported handsomely for years. 

UC Davis researcher Paul Knoepfler last week carried a piece on his blog on ViaCyte's progress. The article came in the form of a Q&A with the firm's president, Paul Laikind.
Paul Laikind, CEO Viacyte

The interview covered a lot of scientific ground as well as some financial matters dealing with the firm and its clinical trials. Perhaps a good part of the bottom line was contained in this paragraph from Laikind.
"Near term, as the PEC-Direct clinical trial advances into Cohort 2, full enrollment is expected in the second half of 2018 with evaluation of efficacy about 6 months later. The primary efficacy endpoint is clinically relevant insulin production, as measured by C-peptide 6 months after implantation. While the efficacy analysis is expected to occur in 2019, patients will remain in the study for two years, thus the expected completion date for the Phase 1/2 study is December 2020."
Also of interest is the use of a product from the company that produces Gore-Tex, a fabric that took the rain gear industry by storm, so to speak, decades ago. The fabric is now used in a wide variety of medical applications. Here is what Laikind said about the Gore-Tex connection.
"The goal of the research agreement with W.L. Gore & Associates is to cooperatively establish new methods of effectively delivering cell therapies, specifically with improvements in the Encaptra Cell Delivery System used in PEC-Encap. The semipermeable membrane that is a key component of the Encaptra device is made of the same material that makes up Gore-Tex. The Gore team are the world’s leading experts for manipulating and engineering this material.
"Gore has expertise in medical device development and drug delivery technologies, as well as previous research and development experience on cell encapsulation and implant programs for diabetes. Gore’s contribution to the material and design improvements of the Encaptra Cell Delivery System is expected to support the reliable and robust long-term engraftment that is required for the PEC-Encap product to be most effective.
"Gore’s participation in ViaCyte’s financing announced in May 2017 represents another external validation of the company and its technologies. It also reflects Gore’s great interest in, and commitment to, the development of a successful implantable cell therapy for all patients with diabetes who use insulin."

Friday, August 04, 2017

'A Reliable Voice of Reason:' A Look at a California Stem Cell Researcher/Blogger


Paul Knoepfler's TED talk has drawn more than one million views

The journal Science this week profiled UC Davis stem cell scientist and blogger Paul Knoepfler, describing him both as a consumer watchdog and a "dogged voice of caution."

In the article by Kelly Servick, George Daley,  dean of the Harvard Medical School, also called Knoepfler a "reliable voice of reason."  Daley noted that academics are "often more comfortable being provincial and insular, and not … mixing it up in the public debates."

Knoepfler began blogging seven years ago after -- at the age of 42 -- he was diagnosed with prostate cancer and given a roughly 50-50 chance of survival. He told Servick that he wanted to "expand how I had impact, beyond just the pure science." From the start, his blog went beyond science to patient advocacy.

Readers of the California Stem Cell Report are likely to be familiar with Knoepfler's blogging efforts, which are often reported here. We have followed his efforts since their inception. Over the years, his voice has grown to be heard in publications across the country. Just this week, he was widely quoted in coverage of the ground-breaking research into gene editing of human embryo.

(An aside from this writer, who is quoted in the Servick piece: One of the reasons that Knoepfler is quoted widely is that he returns media phone calls and can "speak English" -- meaning that he can explain science in terms that most persons can understand, at least those who read newspapers. That could have something to do with having an undergraduate degree in English.)

The Science article focused heavily on Knoepfler's writing about unregulated stem cell clinics, including research by him and Leigh Turner of the University of Minnesota that documented for the first time the size of that particular industry in this country. Their work counted 570 clinics with the most in California.

Why the focus on the unregulated treatments? Knoepfler told Science:
"'They were just saying, ‘Screw the rules, we're just going to set up shop and put up a website and start injecting people with stem cells....I saw that as a threat, first to patients, but to the field as well.'"  

Sunday, April 02, 2017

California's Unregulated Stem Cell Clinics: Will State Regulators Step In to Cure Abuses?

The state of California has laws regarding substandard and questionable medical care, and now questions are being raised about whether it should step in to deal with the unregulated stem cell clinics that appear to flourishing in the Golden State.

The concerns come as the Los Angeles Times reported during the weekend about an enterprise in La Jolla, StemGenex Medical Group, which describes itself as "the premiere leader in the United States for regenerative medicine." 

The Times' Pulitzer Prize-winning columnist Michael Hiltzik wrote about the case of Jim Durgeloh, 59, a construction contractor from Washington state, who was looking for a solution to his hip problems, as well as other cases. Durgeloh paid StemGenex $15,000 for a treatment.

Hiltzik interviewed Durgelogh who was in Southern California with his wife following treatment. Hiltzik wrote,
"They were preparing to fly home, infused with the hope communicated by the clinic staff, who 'seemed very optimistic,' Durgeloh told me.
"A lawsuit in San Diego federal court suggests that StemGenex may have given the Durgelohs nothing but hope. Three StemGenex patients — two with diabetes and one with lupus — say they were misled by the medical group’s marketing pitch to pay $14,900 each in 2015 and 2016 for therapies that have had no effect.
"The lawsuit, which seeks class-action status, claims that StemGenex has made its money by 'targeting the ill and the elderly' with 'false, fabricated and purposefully misleading' claims about patient satisfaction."
Hiltzik continued,
"StemGenex, in its reply to the ... lawsuit, asserts that the plaintiffs 'cannot prove' that its 'representations regarding the efficacy of its stem cell treatments are actually false.' The plaintiffs, it continues, 'do not cite to a single scientific study that disproves [StemGenex’s] advertised claims.'"
UC Davis stem cell scientist Paul Knoepfler wrote yesterday on his blog about Hiltzik's article. Knoepfler was the co-author of a 2016 study that attracted nationwide attention with the disclosure that 570 unregulated stem cell clinics exist in the United States. California leads the way with 113.

Knoepfler focused on Hiltzik's disclosure that one of the StemGenex physicians, Scott Sessions, was placed on three years probation last February involving treatments not related to StemGenex. Sessions was accused of negligence "related to cosmetic surgery and other procedures he performed on two patients at an unrelated facility in 2011 and 2013."

Knoepfler continued,
"Sessions photo was up on the Stemgenex website...and then suddenly it wasn’t. Hiltzik also mentions that Stemgenex has had other questionable information on its web site in the past.
"With California having the most stem cell clinics selling non-FDA approved interventions of any state I hope the state medical board here will wake up to the fact that it needs to give this arena more attention."
The state has wide-ranging authority to regulate physicians. A document on the state Department of Consumer Affairs web site says that physicians can be disciplined for such things as  "not using accepted, effective treatments or diagnostic procedures," "not referring a patient to a specialist when
appropriate" and "continuing to use a procedure that is unnecessary."

Monday, March 20, 2017

The Gottlieb Effect: Dismantling of FDA Silos, Less Regulation of Stem Cells?

A California stem cell researcher is predicting a possible "roller-coaster ride" in the wake of the appointment of Scott Gottlieb as the new head of Food and Drug Administration(FDA)

President Trump named Gottlieb to the post earlier this month. Gottlieb has said in the past that "less is more" in terms of regulation of stem cell research, according to UC Davis research Paul Knoepfler.

He wrote on his blog last week about the Gottlieb's views and what they could mean for stem cell research. Knoepfler reviewed some of Gottlieb's publications and speeches for the analysis, including a 2016 speech in Berkeley which contained "some pretty radical language."  Mentioned was dismantling silos within the FDA.

The California scientist said,
"The use of powerful words like 'dismantled' by Gottlieb last year, now bode for potentially highly disruptive changes at the FDA in the Trump administration under his leadership. Since he stated that regenerative medicine products 'don’t easily fall into FDA’s current buckets,' I wonder what the could mean for CBER (a branch of the FDA).
"I’m hoping that Gottlieb will rightly see the central distinction between empowering more efficient stem cell and regenerative medicine product development by good actors in the field (he could do this, for example, by shaping how the 21st Century Cures Act regenerative medicine-related directives are implemented) versus enabling many dubious actors at a host of for-profit businesses. These companies don’t do preclinical studies, don’t respect the FDA, and put patients at risk."
Knoepfler covered additional ground in his March 14 piece, which may be the only serious, current analysis of Gottlieb and stem cells. Knoepfler concluded:
"Change at the FDA overall or on stem cells isn’t necessarily a bad thing, but in this case it could be a roller-coaster ride."

Friday, December 09, 2016

Voting Underway for Stem Cell Person of 2016

The latest tally of voting for the stem cell person of the year shows that patient advocate Ted Harada, who died in October, is leading with 47 percent of the vote with the nearest contender at 11 percent.

However, only one vote counts since this is not a democratic election. The sole individual choosing the stem cell person of the year is Paul Knoepfler, a stem cell scientist at UC Davis.

Knoepfler originated the award a few years back and personally puts up $2,000 in prize money. He has compiled a list of 20 possible honorees, ranging from patient advocates to researchers. Voting is advisory only. Deadline for the ballot is 11:59 p.m. Dec. 15.

Knoepfler wrote on his blog last month about Harada, who was an ALS patient advocate. Knoepfler said,
"Ted approached all that life threw at him, whether it was ALS or a brain tumor, with a characteristic passion, sense of humor and classiness. I don’t recall anyone dealing with adversity as well as Ted did and I never heard him say anything like, 'why me.' Instead there was always kindness and grace."
Distant second to Harada this morning was Randy Mills, president of the $3 billion California stem cell agency.

Sunday, November 27, 2016

Dubious Stem Cell Therapies Attract Coverage in California's Capital

The leading newspaper in California's state capital this weekend published a lengthy piece exploring the world of unproven stem cell therapies, including one being offered in its own backyard. 

Reporter Claudia Buck of The Sacramento Bee wrote,
"For long-suffering patients...stem cells offer tantalizing hope. In the last few years, more than 570 stem cell clinics have popped up nationwide, advertising treatment for a range of maladies, from autism and Alzheimer’s to neuropathy and Parkinson’s disease, according to a recent UC Davis study. About 113 of those are operating in California. 
"But do they really work? According to most stem cell experts and the federal government, there’s no way to know yet."
Buck quoted Kevin McCormack, spokesman for the California stem cell agency, as saying,
“It’s quite clear that these people are offering treatments that haven’t been tested in clinical trials. It’s a little concerning,”
Buck wrote,
"'My view is that it’s a giant human experiment that doesn’t have FDA approval,' said Paul Knoepfler, a UC Davis stem cell expert, who co-authored the study identifying the 570 clinics. 'I don’t know how much patients are aware of how uncertain the benefits and risks are. As a scientist, it’s worrisome.'"
Knoepfler, who publishes a blog on stem cell matters, has written in the past about advertising in The Bee by a stem cell business called Nervana. The firm was mentioned in Buck's article, which, however, did not note that Nervana has taken out full page advertisements in the past in The Bee.

Knoepfler's most recent piece on Oct. 21 also noted that the firm had taken out a full page ad in the San Diego Union Tribune as well.  The UC Davis researcher said at the time,
"I don’t believe there is a solid, medical or scientific basis for what they are selling."

Thursday, July 28, 2016

NY Times: Stem Cell Theory vs. Flourishing, Dubious Stem Cell Clinics

The New York Times this morning took a crack at coverage of the first-ever study of the wave of dubious stem cell clinics in America. The headline on the Times' prominently displayed story said,
"Stem Cell Therapies Are Still Mostly Theory, Yet Clinics Are Flourishing"
The starting point for the article by Gina Kolata was the study by UC Davis stem cell researcher Paul Knoepfler and bioethicist Leigh Turner of the University of Minnesota.  They reported on June 30 that nearly 600 dubious stem cell clinics have sprung up around the country. It is the first study to document the reach of these businesses. The report, published in the scientific journal Cell Stem Cell,  received heavy mainstream media coverage.

Kolata highlighted the tentative nature of stem cell research. She wrote,
 "In theory, stem cells might be a useful treatment for certain diseases that involve the loss of cells, like Type 1 diabetes, Parkinson’s or osteoarthritis. They are primitive cells that can develop into a range of mature cells and perhaps serve as replacements. But progress is slow. After a flurry of stem cell excitement two decades ago, almost all the research today is still in mice or petri dishes. The very few clinical trials that have begun are still in the earliest phase.
"The problem is that stem cell therapies are still mostly theory. So what is going on? How can there be clinics, even chains of clinics run by companies, offering stem cell treatment for almost any disease you can think of — sports injuries, arthritis, autism, cerebral palsy, stroke, muscular dystrophy, A.L.S., cancer?"
She said that the Knoepfler-Turner study showed "what can happen when regulations fall behind." 
And she noted the limits of federal regulation as well as the failure of state regulatory bodies to deal with the issue. 

Kolata's story was displayed online this morning on the main page of the New York Times. The media attention to the work by Knoepfler and Turner has significantly raised the visibility of the issues involving dubious stem cell "therapies."  A narrowly defined search on Google news this morning turned up nearly 11,000 citations, up from about 7,000 early this month.

The coverage certainly has increased the likelihood of some sort of additional regulatory action. However, such moves take time, and it is not realistic to expect a crackdown anytime soon. Indeed, it may require legislative changes at the federal and state levels.

As for the ethical and medical concerns raised by Knoepfler and Turner and the Times piece, one Times reader -- identified online only as Susan -- said,  
"So what? A lot of what the scientific/medical establishment tries to sell us is just theory. Studies of this and studies of that. A study will come out and 6 months later another study will come out debunking the first study. I believe the problem is that some of these science/medical 'experts' are under the delusion that 'one size fits all.' I disagree. What may kill one person may help another."

Monday, June 20, 2016

Stem Cells and the FDA: California's Lobbying for Easier Approval Triggers Opposition

The $3 billion California stem cell agency came in for some sharp criticism last week in an opinion piece in the San Francisco Chronicle that was written by a UC Davis stem cell researcher who said the agency is engaged in politicking that "risks doing much more harm than good."

Paul Knoepfler, the scientist who also produces "The Niche" stem cell blog,  wrote,
"The California Institute for Regenerative Medicine (CIRM) has become politically active under its new president, C. Randal “Randy” Mills. Mills is lobbying for a radical departure in how the U.S. Food and Drug Administration (FDA) vets experimental stem-cell therapies, but such politicking risks doing much more harm than good."
Knoepfler wrapped in an attack on federal legislation known as Regrow Act. Knoepfler said the proposal would "force the FDA to conditionally approve still experimental stem cell therapies with relatively little data supporting them."

Randy Mills at left, USC photo
Knoepfler reported that Mills said he was not anti-FDA. Knoepfler also noted that the agency has not taken a position on the Regrow Act.  Knoepfler said the FDA effort and the Regrow Act are part of a larger effort to weaken the FDA, which has been under regular criticism for decades for moving too slowly.

The stem cell agency's attempt to modify the way the FDA approaches potential stem cell therapies has been underway for months. The California Stem Cell Report carried news on the subject as long ago as last winter(see here and here.)

Mills initiated the lobbying effort with the FDA after an informal survey of California "stakeholders" showed that they perceived the FDA as the No. 1 obstacle to making stem cell therapies available for widespread use.

In an item on the agency's blog, The Stem Cellar, last December, Mills said that “patients are dying” because the federal government is “being so careful about safety,"

Yesterday, Knoepfler provided some personal insight -- a "backstory" -- on what led him to write about the all of this. His piece was carried on his blog and emphasized that he is a "big backer" of CIRM and still is. Knoepfler has received $2.2 million from CIRM. The CIRM web site has 60 links to mentions of Knoepfler on its Web site, including this one below. 

Knoepfler wrote,
"Most of the stem cell scientists that I have talked to (admittedly with a few notable exceptions that include some long time stem cell advocates and scholars) are opposed to CIRM’s lobbying on this front, but are uncomfortable speaking out."
Uncomfortable is a rather mild word for it based on the fact that the agency holds the key to a $3 billion vault containing the hopes of funding for the entire California stem cell community.

We should note that lobbying is not new to the agency. Its former chairman, Robert Klein, had a passion for it. At one point in 2009, he engineered the hiring of an exceedingly well-connected lobbyist named Tony Podesta for $200,000.  Mills has not ventured into that sort of game, however.

Knoepfler has much more to say on the subject of regulation, the need for it and the impact on patients and the dubious stem cell clinics that seem to be multiplying throughout the nation, despite the fact that the FDA has never approved a stem cell therapy for widespread use.

The California Stem Cell Report will be visiting the matters surrounding these issues and the California stem cell agency. Look for more here during the next week or so.

Friday, October 23, 2015

The Niche Rises -- Sort of Phoenix-like -- in California

California and the world has a “new” stem cell blog – at least new in name.

Welcome to The Niche (with an upper case T).

Paul Knoepfler, UC Davis photo
It is a rebranding of ipscell.com, the five-year-old effort by UC Davis stem cell researcher Paul Knoepfler.

He announced the change yesterday. Knoepfler wrote,
 “The Niche name better represents the sense that this site is a home and community including contributions by other writers and also commenters. I’m still working on a logo for The Niche.
“As The Niche continues to evolve look for it to include a wider range of contributors. As you may have noticed we have an increasing number of posts on genetics and genetic modification as well in addition to stem cells and regenerative medicine.”
The Niche, as some may recall, was the name of an excellent blog mounted by the journal Nature a few years back. It was shuttered by Nature for what must be presumed to be financial reasons.

Monya Baker and Natalie DeWitt were the overseers of that effort. Baker remains with Nature. DeWitt moved on to the California stem cell agency where she was a top aide to its then president Alan Trounson. She left the agency in June 2014 for Stanford and has been with NDA Partners LLC of San Francisco for the past five months.

Thursday, July 02, 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.

Tuesday, November 04, 2014

Researcher Takahashi Honored as Stem Cell Person of the Year

Riken photo

Masayo Takahashi, the scientist in Japan who is leading the first clinical trial involving reprogrammed adult stem cells, today was named the stem cell person of the year.

The announcement was made by Paul Knoepfler, the UC Davis stem cell researcher who sponsors the award and personally funds its $2,000 prize.

Knoepfler said,
“In an astonishing feat, Takahashi's team transplanted its first macular degeneration patient recently on September 12, only 7 years after human IPSCs (induced pluripotent stem cells) were first ever published.”
The development of IPS cells, which also originated in Japan, has been hailed because they remove the moral objections that some persons have concerning the use of human embryonic stem cells. Many scientists, however, continue to consider the human cells as the gold standard.

Takahashi is a physician-scientist and a faculty member and project leader at the Laboratory of Retinal Regeneration at the Center for Developmental Biology at RIKEN.

Last December, Takahashi said she was wary of the high economic expectations surrounding potential stem cell therapies. In an article in the Financial Times by Jonathan Soble, she said, 
“Start-up companies are already involved and the road to commercialization is there, but to assume that the concept of iPSCs and regenerative medicine will yield a lot of money is naïve. Only parts of the field will become industries. It’s dangerous to think that all of regenerative medicine will.”
Takahashi was introduced to the field of stem science in 1995 when she followed her husband to the Salk Institute in the San Diego area in California.

Soble wrote,
“Paradoxically, because she had chosen clinical research, academia’s mix of glass-ceiling sexism and disdain for applied science worked in her favor. ‘I could do what I wanted because I was a woman,’ she says. ‘I didn’t think about career advancement.’”
She said barriers to women in science have largely disappeared in Japan although the comment was made before the STAP scandal that involved Riken. A key figure in the STAP research was a woman scientist, and persons prejudiced against women in science may well to use it to justify their biases.

Takahashi did say last year that traditional cultural values remain a problem in Japan. She said,
“'Japanese women don’t want to stand out, they don’t want to be leaders. They don’t think, ‘I want to have my own lab and reach the top in my field.’ I certainly didn’t until Salk.’ But Dr. Takahashi believes things are changing, in part because of the growing number of role models such as herself. ‘Sometimes,’ she says, ‘I meet young women who tell me, ‘You’re cool.’”

Wednesday, January 08, 2014

Knoepfler and Capricor Score Big This Week

Capricor's stock price has soared since Monday.
Chart from Google Finance
With the new year hardly begun, UC Davis stem cell researcher and blogger Paul Knoepfler has fulfilled one of his 10 predictions for the stem cell world in 2014.

On Dec. 29, Knoepfler predicted a “big announcement from Big Pharma” in 2014. He did not have to wait long to add some shine to his crystal ball.

On Monday, he wrote about the news that Johnson&Johnson was pumping $12.5 million into Capricor Therapeutics' stem cell therapy for heart disease. Capricor could see another $325 million from J&J if all goes well in its stage two clinical trial, which is being supported by $20 million from the California stem cell agency.

“I'd call that big money,” Knoepfler wrote. He also noted in his blog he has no financial interest in either company.

Investors liked the news as well. Capricor's stock closed at $7.05 today, more than double its $3.40 close on Friday, the business day just prior to J&J announcement. 

Wednesday, October 30, 2013

Italian Researcher Cattaneo Honored as 2013 Stem Cell Person of the Year

Elena Cattaneo, EuroStem Photo
Italian stem cell scientist Elena Cattaneo today was named the 2013 Stem Cell Person of the Year for her work as a researcher and her efforts to remove “partisan extremist politics” from financial support for science. 

UC Davis researcher Paul Knoepfler announced his choice this morning on his blog, ipscell.com. Knoepfler began the contest last year and personally provides the $1,000 award. The first winner was Roman Reed, a California patient advocate.

In Knoepfler's announcement, he cited Cattaneo's work on neural stem cells, particularly in connection with possible therapies for Huntington's Disease. Cattaneo is director and co-founder of the Unistem, the center for stem cell research at the University of Milano, Italy, and has been appointed a senator for life in Italy.

Knoepfler also said,
"She is that rare stem cell scientist who goes well above and beyond the call of academic duty to act as an advocate as well."
He said that she has "a track record of taking important public stands on key issues over the years."

Most recently, Knoepfler cited her efforts in connection with an organization called Stamina in Italy that was providing a stem cell therapy to large numbers of patients. The treatment this month was banned from testing in Italy after a panel of scientists determined it was excessively risky and not supported by evidence.

The decision followed a campaign by Cattaneo and others. Knoepfler wrote,
"She and a dozen fellow Italian scientists stood up to what was a flawed Italian governmental decision to allow Stamina to proceed with a gutsy letter. It takes courage to not only do your research, but also to take a stand against your own government's flawed decision and ultimately prevail. Her actions have helped to keep thousands of patients safer and protect the stem cell field."
Her efforts and those of others inspired a play called "Staminalia: A Dream and a Trial," which premiered in Portugal in 2010. According to the EuroStemCell Web site, the play "goes beyond the science of stem cells to tell the story of a stem cell researcher and a mother who sees ethical and political limitations impacting not only into her scientific freedom, but also into her private life."

Tuesday, September 24, 2013

Deadline Approaching on Stem Cell Person of the Year Awards

Only six more days remain to submit nominations for the Stem Cell Person of the Year.

This is the contest run by UC Davis stem cell researcher Paul Knoepfler, who will give the winner $1,000 of his own, personal cash.

The criteria are nearly wide open although Knoepfler is looking for the “most influential” person in the field. He wrote on his blog,
“Nominees can be almost anyone ranging from an advocate to an academic or industry scientist to a stem cell biotech leader to a policy maker to a physician…think outside the box! Surprise me!”
Knoepfler initiated the contest last year. The 2012 winner was Roman Reed, a San Francisco Bay Area patient advocate.

Knoepfler reported today on his blog that he has received two dozen nominations so far. He is planning on opening up the selection of the semi-finalists to online voting from the list that will be published in early October. Last year he selected the semi-finalists himself.

As for the names he has received so far, Knoepfler said,
“This group of nominees is very diverse, far more so than last year, and includes more scientists and physicians.”
Nominations should be sent to knoefpler@ucdavis.edu. More information about the rules can be found here.

Monday, September 09, 2013

Rise Up and Blog! Any Scientist Can!

The old saying has it that you can tell the pioneers by the arrows in their backs. 

Tell that to Paul Knoepfler, the UC Davis stem cell researcher who has been something of a pioneer in stem cell blogging and still is -- at least given that almost no other stem cell researcher blogs both as a researcher and patient advocate in addition to taking on wide-ranging business and public policy issues.

Knoepfler does have a few arrows in his back, some from enterprises that are less than happy with his vieww of their conduct and some from within the stem cell research community itself. But overall he has found the experience beneficial and rewarding. 

In an article in Nature Medicine Sept. 6, Knoepfler is recruiting more researchers for the stem cell blog brigade. To encourage them, he recounts his experiences and costs (such as $1,500 for his special domain name, although a normal domain name can be had for virtually no cost). His experiences include working late at night and on weekends. We can testify that the father of three puts in the hours, just based on the nature of the blog content and frequency of posting. 

Knoepfler continues his pitch in his piece in Nature, which is read primarily by researchers,
“To all this a reader might say, ‘Okay, this all sounds well and good, but you can't have your cake and eat it, too! Your science must have suffered from all this.’ In fact, I have lost some sleep, but I have not seen much in the way of negative scientific consequences. My lab has done very well during the past three years, and we have published many important papers, including one earlier this year that demonstrated the molecular similarities between induced pluripotency in stem cells and oncogenic transformation in cancer cells. I even secured tenure during this time.

“Reflecting on my personal transformation to the role of scientist−advocate, I have come to notice many tangible positive outcomes. In a general sense, my work has served to build bridges and stimulate new dialogue between industry and academia in the stem cell field. At the same time, I couple these efforts to accountability. Today, if someone does an Internet search for 'stem cell blog', that person will find my site, with all its educational outreach resources, at the top of the results list. A few years ago, the same search would mostly have yielded sites published by opponents of stem cell research or proponents of sketchy, for-profit endeavors to attract stem cell 'tourism'. I have also interacted with more than 100 patients and caregivers, helping them make more educated and, I believe, safer, stem cell−related medical decisions with their physicians.”
Knoepfler’s bottom line pitch? Any scientist can!

As he put it:
“Any scientist can, and should, do it. It is only logical that scientists would adapt to today's reality—a funding-poor environment that is nonetheless rich with opportunities for communication—by becoming advocates. I predict that any scientist who devotes a tiny bit of time to advocacy endeavors will find that the payoff is greatly multiplied. I will even help. Drop me an e-mail. Or better yet, leave a comment on my blog or message me on Twitter. Let's get the conversation going.”

Sunday, August 11, 2013

'Paradigm Shift' -- Researchers as Patient Advocates?

It is not easy for a stem cell scientist to break into “print” in that bastion of American capitalism, Forbes magazine. Much less one from that perceived antithesis of free enterprise, Reed College.

But researcher Paul Knoepfler of UC Davis has done just that.

John Farrell of Forbes wrote about Knoepfler on Friday in the wake of the announcement Knoepfler will be honored with a “national advocacy” award by the Genetics Policy Institute.
Paul Knoepfler -- Advocate for a "new ethos"
UC Davis photo

Knoepfler, who received a degree in English literature from Reed in 1989, is being recognized for his activities as a stem cell blogger since 2009.

Farrell quoted Knoepfler as saying in an email,
“With many stumbles, face plants, land mines, and even a few threats of litigation and career retaliation along the way for the last three and half a years, I turned my crazy idea into a reality.”
Farrell continued,
“But it was only possible, (Knoepfler) added, with guidance from many patient advocates and bloggers in other fields who generously helped him learn the ropes of blogging.
“'I see this award as a validation of the notion that advocacy by scientists has become a valued part of the stem cell field,' he said.
“'My hope is to catalyze a continuing paradigm shift whereby stem cell scientists and biomedical scientists more generally have a new ethos that not just accepts, but also deeply values advocacy.'”

Friday, January 18, 2013

UC Davis Stem Cell Researcher: 'Ivory Tower' IOM Recommendations Harmful to California Stem Cell Agency

The $3 billion California stem cell agency has funded in the neighborhood of 500 to 600 scientists and institutions, reviving and starting careers and stimulating construction of $1 billion in new research labs around the state.

None of those recipients, as far as we know, has come forward to comment publicly on the sweeping recommendations by Institute of Medicine for changes at the agency. Until today, that is.

UC Davis researcher Paul Knoepfler, who may be the only stem cell scientist in the United States with a stem cell blog, weighed in with his thoughts today, which do not align with those of the blue-ribbon IOM panel.

“Harmful” is one word that Knoepfler, who is a stem cell agency grant recipient,  used to describe the recommendations. He predicted “extremely negative repercussions” that “would actually make CIRM less effective and less responsive to patients and California citizens.”

He wrote that the IOM report, which will come before stem cell agency governing board next week “...seems more like an ivory tower intellectual exercise than an operative, realistic guide to a dynamic agency that must operate in the real world.”

He defended the CIRM governing board, which came under fire from the IOM for conflicts of interest. Institutions linked to board members have received about 90 percent of the $1.7 billion that the board has awarded, according to compilations by the California Stem Cell Report. The IOM said,
“Far too many board mem­bers represent organizations that receive CIRM funding or benefit from that funding. These com­peting personal and professional interests com­promise the perceived independence of the ICOC, introduce potential bias into the board’s decision making, and threaten to undermine confidence in the board."

Knoepfler said,
“(The) IOM itself admits there is no evidence that any conflicts of interest have ever guided (the agency's governing board) decisions. Not one example.”
Knoepfler also wrote,
“Interestingly, highlighting the extremely sensitive nature of this issue, while I’ve been talking with many bigwigs about this, at this point no one is wiling to go on the record with an opinion about it except one courageous soul, Don Reed (see his piece here).”
There is a reason for that. The IOM is the most prestigious organization of its sort. Its studies are described as the gold standard. And it has a rareified membership that many scientists seek to join. So few are ready to give the organization a smack on the nose. Likewise, California researchers are loath to publicly criticize the stem cell agency because it holds the strings to the purse that finances their careers.

California scientists, however, should be asking themselves a bottom-line question. Do they want to see the stem cell agency continue for another 10 to 20 years? Under the best of circumstances, that may be unlikely given the other pressing needs that the state faces. But if CIRM directors do not forthrightly address the recommendations of the IOM panel, the fate of the stem cell agency is exceedingly uncertain.

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