Wednesday, August 31, 2016

Good Safety News in Asterias California Stem Cell Trial

Jake Javier and family -- Javier family photo
For 18-year-old Jake Javier, it's a small matter of 10 million stem cells. And hope.

Javier is one of the patients in a clinical trial backed by $14.3 million from California's stem cell agency, which announced today some good news about the safety of that research.

For Javier, however, the details about "efficacy cohorts" and "AST-OPC1were a little more personal. The former high school football player is paralyzed from the chest down as the result of a swimming pool accident in June on the eve of his high school graduation.

Today he is one of a handful of patients enrolled in the spinal cord clinical trial by Asterias Biotherapeutics of Menlo Park, Ca., which picked up the human embryonic stem cell trial after it was abandoned by Geron in 2011.

The $3 billion stem cell agency announced today that Asterias "has cleared two key safety hurdles and been given approval to expand the therapy to a larger group of patients with a much higher dose of cells."

The next step is to insert 20 million stem cells into patients, up from the 10 million that Javier received.

Javier, who played football at San Ramon Valley High School, has been the subject of widespread media attention and fund-raising, including considerable reporting by San Francisco Bay Area television station KTVU.

Writing today on the stem cell agency's blog, The Stem Cellar, Kevin McCormack, senior director for communications, said,
"For people like Jake Javier this news is not about numbers or data, it’s personal. Earlier this summer Jake broke his neck at a pool party, celebrating graduating from high school. It left him paralyzed from the chest down with extremely limited use of his arms and hands. On July 7th Jake was enrolled in the Asterias trial, and had ten million cells transplanted into his neck.
"It could be months, even as much as one year, before we know if those cells are having any beneficial effect on Jake. But at least for now we know they don’t seem to be having any negative effects."
McCormack wrote,
“Our focus is always on the patient, so making sure a potential therapy is safe is an important first step,” says C. Randal Mills, Ph.D., the President and CEO of CIRM. “I recently met with Jake Javier, a young man who was treated in this trial, and heard first-hand what he and his family are going through in the aftermath of his injury. But I also saw a young man with remarkable courage and determination. It is because of Jake, and the others who volunteer to take part in clinical trials, that progress is possible. They are true heroes.”
For more on Javier and the research see: Facebook and the stem cell agency progress report.
Left to right, Dr. Marco Lee, a member of the transplant team, Jake Javier,
 his mother, Isabelle, and Randy Mills, president of the California Institute for
Regenerative Medicine, as the stem cell agency is formally known. 

Monday, August 22, 2016

'Unnerved' Researchers and Proliferation of Stem Cell Clinics: The Perspective from Los Angeles Times

The Los Angeles Times this past weekend picked up on the growing concern about the spread of dubious stem cell clinics triggered in many ways by a first-ever study by a UC Davis stem cell researcher. 

Pulitzer Prize-winning columnist Michael Hiltzik wrote,
"The proliferation of clinics nationwide offering stem cell therapies unnerves many researchers, who fear that unrealistic public expectations could undermine their own credibility. Patients with hard-to-treat conditions such as multiple sclerosis and Parkinson’s are easy marks who can be led to forgo established therapies in favor of unproven ones."
The headline on Hiltzik's piece said,
"These new stem cell treatments are expensive — and unproven"
Hiltzik interviewed a California lawyer who is working in the field. Hiltzik wrote,
"'Stem cell scientists include the best and the brightest doing great work, and I admire them,' says Janice Mulligan, a San Diego attorney currently assembling evidence for a potential class-action lawsuit against unnamed stem cell clinics. "At the other end of the scale, there’s snake oil.'"
Hiltzik also spoke to Paul Knoepfler, the UC Davis researcher who co-authored the study about stem cell clinics with Leigh Turner of the University of Minnesota. Hiltzik wrote,
"In an article just published in Stem Cells Translational Medicine, Knoepfler described his visit to one informational seminar sponsored by a clinic pitching a $5,999 stem cell treatment to about 30 attendees. Each was given a pack of forms to fill out, including a credit application. The spiel, he reported, featured personal anecdotes and 'questionable medical claims' — including the assertion that '90% of patients had a 50% or better improvement.'"

Monday, August 15, 2016

Odds for a Stem Cell Cure vs. Odds on Trump Winning the Election

The article explored one particular search for a cure for diabetes, but the headline was bleak.
"Will Embryonic Stem Cells Ever Cure Anything?"
The question came at the top of a piece by Aleszu Bajak in the Aug. 12, 2016, edition of the MIT Technology Review. 

Bajak quoted Doug Melton of Harvard as saying,
“The public definitely doesn’t appreciate that much of science is failure.”
Bajak, a veteran science writer and journalism instructor, continued with his own conclusion, 
"In fact, no field of biotechnology has promised more and delivered less in the way of treatments than embryonic stem cells." 
Hard, hard language that probably disturbs many in the field, including some at the California stem cell agency, which was founded on the promise of therapies from human embryonic stem cells(hESC). The agency has committed more than $2 billion to stem cell research since 2005 and is still looking for its first big score.

One can argue the fairness of the conclusion by Bajak, but it certainly seems tied to the excessive rhetoric that has surrounded the hESC field, particularly the $34 million ballot campaign that created the $3 billion state stem cell agency in 2004.

The rhetoric of the campaign came with a price. The agency is scheduled to run out of cash for new grants in 2020. Whether it can deliver on the campaign promises of 2004 will in large part determine whether it can conjure up additional funding.

The likeliest prospects for success today can be found in the list of clinical trials the agency is helping to fund and the scores of additional trials that it plans to assist in the next several years. They include efforts to deal with afflictions ranging from heart disease to diabetes.

By one widely cited measure, only one out of 10 conventional therapies entering clinical trials reaches the market place. Those are slim odds, ones that do not involve novel stem cell efforts, which are presumably more difficult. To put it another way, Donald Trump has better chances -- as of this writing -- of being elected president than of a clinical trial producing a stem cell cure.

Trump, however, is only going to have one chance. The California stem cell agency is expecting to take more than 50 shots at a therapy via its involvement in clinical trials over the next several years.

Nonetheless, it is a risky business, as venture capitalist Gregory Bonfiglio, pointed out to the California Stem Cell Report last December.  He said,
“There are risks inherent in the development of new, disruptive technology. The bigger risk is failing to deliver on their underlying promise to bring new regenerative therapies to patients…. The bigger risk is not doing anything.”

Wednesday, August 10, 2016

Digging into the 'Stem Cell Hard Sell:' Researcher Ventures Into 'Lion's Den'

A California stem cell researcher went "semi-undercover" recently and attended a commercial "educational seminar" that promoted stem cell treatments and handed out requests for credit information from those attending.

Paul Knoepfler -- UC Davis photo
Paul Knoepfler of UC Davis recorded his experience at the session in an article this week in the Stem Cells Translational Medicine, which was founded in 2011 with $600,000 from the California stem cell agency.

Knoepfler did not identify the stem cell clinic that conducted the session. He said he wanted instead to be able to use a "higher degree of frankness" in writing about the session. Knoepfler noted, however, that he was quickly spotted by one of the organizers, some of whom lurked around him during the session. Knoepfler wrote on his blog Monday, 
"It was an intense experience and one where I felt at risk. To some extent it is akin to  going into the lion’s den. I didn’t know what reaction I would get if I were recognized by those running the seminar, which in fact did happen. They did not seem happy at all that I was there even though I wasn’t disruptive in the least. I had hoped to ask a few questions from my place in the audience, but the format did not allow it. Still I wondered if I could be kicked out. That didn’t happen."
The clinic involved is one of at least 570 across the country. Earlier this summer, Knoepfler and Leigh Turner, a University of Minnesota bioethicist, were the first to report on the scope of what passes for the commercial stem cell clinic industry in the United States when they documented the existence of those clinics.

About 30 people attended the session Knoepfler was at, including some who obviously had "serious medical conditions." He wrote,
"The seminar team (mostly dressed in medical scrubs) handed out a clipboard to each attendee with three pieces of paper very much like those that one might be given on arriving at a new doctor's office. We were told to fill in our information and then to return the sheets back to them before we left. Notably, the top sheet was a credit application. On another page, the clinic asked for extensive personal information, including name, age, birthdate, address, e-mail and phone number. Additional questions asked about medical conditions, as well as medical tests that had already been conducted." 
Knoepfler said several medical claims were made during the meeting including that the treatments had no side effects, were safer than the normal standard of care, did not pose immune rejection problems, were federally approved and were specifically effective for arthritis and pain.

The price of the treatment was $5,999 but attendees could get $1,000 off "if you sign up today at the seminar." Attendees were told that the clinic was heavily booked already with a one to two month waiting period.

In the piece about his foray into the "lion's den," Knoepfler wrote,
“Given the growing realization that there are hundreds of stem cell clinics in the U.S. today in 2016 and the observation that many hold recruitment seminars, it is possible that thousands of members of the public each year are attending infomercial seminars that provide misleading or even outright factually incorrect information about stem cells and questionable medical claims. This could not only lead many patients to receive unnecessary, unsafe or ineffectual treatments, but it may also contribute to public confusion about stem cells and the field of stem cell clinical research. These seminars represent only one type of an assortment of recruitment methods, including Internet, radio, newspaper, and television ads for various clinics, which may also contain dubious statements. I believe that such stem cell clinic marketing poses a significant threat to public perception and understanding of the legitimate stem cell translational medicine field.”

Sunday, August 07, 2016

Pigs and People: Growing Human Organs in Animals, Changes at the Federal Level

Last week's news that the federal government is moving to lift its ban on funding research involving human-animal hybrids is a bit of old hat in California.

The state's $3 billion stem agency has long been okay with such research and has well-established protocols to ensure that it is conducted ethically and safely.

Last year, the agency, formally known as California Institute for Regenerative Medicine (CIRM), reported (see below) on its activities to the National Institutes of Health, declaring,
"CIRM has reviewed twelve protocols at seven different institutions where stem cells or neural progenitor cells were introduced into vertebrate animals. CIRM found that all protocols were reviewed and approved by a SCRO(standards review) committee consistent with CIRM requirements."

The Chimera of Arezzo -- photo by Joe deSousa
The announcement that the NIH is once again likely to fund the creation of chimeras -- organisms composed of genetically different cells -- received a significant amount of attention in the media. But none of the stories noted the origin of the word in Greek mythology, where it signified a fire-breathing female monster with a lion's head, a goat's body and a serpent's tail.

Sara Reardon of Nature wrote,
"Chimeras are a growing area of research. Currently, researchers use them to study early embryonic development and to create animal models of human diseases. But one major goal is to engineer animals to grow human organs. The organs could later be harvested from the adult animal and used for transplantation into a patient."
Kevin McCormack, senior director of communications for CIRM, told the California Stem Cell Report, last April,
"On a purely theoretical level CIRM has no objection to growing replacement organs or tissues in pigs, provided it met all CIRM’s rules and regulations. We fund research that does that all the time with mice and rats. Right now none of the research we fund is being used to do that."
Shen Ding -- Gladstone photo
Lenny Bernstein wrote in the Washington Post that researcher Shen Ding at the J. David Gladstone Institutes in San Francisco and who has received $6.7 million in research funding from CIRM, sounded a note of caution.
"While he does not favor the current (NIH) moratorium, Ding said, he believes scientists in this field must move slowly because 'we don't know how to precisely control where and how [cells] might contribute' to different organs."
Bernstein noted,
"The NIH imposed its temporary ban on funding last September, citing ethical concerns. These include worries over animals whose brains might contain human brain cells and what might happen if chimeras were able — and allowed — to reproduce."
The NIH is accepting comments over the next several weeks and then is expected to move forward with its proposal, perhaps with changes.

For Hank Greely, a Stanford law professor, it's about time. Last spring, he wrote an op-ed piece in the Los Angeles Times and said, 
"Today we face the possibility of babies getting organs grown in human/nonhuman chimeras — beasts that are pigs except for a single human organ. To the uninitiated, this may sound more like the dark arts than modern medicine, but pursuing careful research and potential clinical use of these chimeras is both proper and important."

Friday, August 05, 2016

The Human Egg Business: More Media Coverage of California Cash-for-Eggs Legislation

A renewed legislative effort in California to pay women for handing over their eggs for research is attracting more attention this year, including opposition from a former state senator sometimes called the mother of the state's stem cell agency.

Deborah Ortiz, former chair of the California Senate Health Committee, wrote an op-ed piece published this week in The Sacramento Bee. She noted that the stem cell agency prohibits paying women for their eggs. Ortiz wrote,
"I oppose AB 2531 (the number of the bill) on a number of grounds: First, we have very limited information on the long-term health effects of the egg retrieval process. Second, women who undergo egg retrieval are not research subjects in the traditional sense, as proponents assert. Finally, paying for women’s eggs for research purposes contradicts well-established national recommendations and state policy."
She continued,
"This is not an issue of equity with other research subjects. Women providing eggs for research are not comparable to research subjects in clinical trials. Their role is providing raw materials for pre-clinical research, rather than participating as subjects in medical research. And they are not afforded the safeguards or follow-up of subjects in clinical trials.
"Finally, AB 2531 conflicts with national recommendations and with policies in our state constitution. The 2010 guidelines of the National Academy of Science recommend that no payments beyond reimbursement for expenses be made for donating eggs for research."
Ortiz has been called the mother of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, because of her early role in support of stem cell research and authorship of an initial measure to use bond funding to finance it.

The cash-for-eggs bill also drew attention from Michael Hiltzik, a Pulitzer prize-winning columnist at the Los Angeles Times. He said that the measure should not be approved. Hiltzik wrote,
"There is scant research on the long-term health risks of egg retrieval, and nothing in the bill that would encourage more. To paper over that fact, the measure’s advocates have engaged in a neat bit of obfuscation. Assemblywoman Autumn Burke (D-Marina del Rey), who is carrying the bill for the ASRM, labels it a 'pay equity' bill....
"But it’s not about equity or discrimination. What worries the bill’s critics is that the measure may allow women to be misled into taking uninformed health risks by the prospect of easy cash."
The legislation surfaced at Buzzfeed in a piece by Cora Lewis and Azeen Ghorayshi. They quoted Sean Tipton, a spokesman for the fertility industry group, American Society for Reproductive Medicine (ASRM), that is sponsor of the legislation.
“'It’s (the bill is) needed to correct a strange and outdated feature in California law.'...Women are paid when they donate eggs to make babies, and people are paid when they are research subjects, he noted. 'But if you combine the two, you can’t be compensated. I don’t think that makes any sense,' Tipton said."
Another piece appeared on the Undark web site. It was written by Diane Tober, an assistant professor at UC San Francisco. She wrote,
"As a researcher myself, I can sympathize with the seduction of discovery, and the lure of developing new technologies and treatments that stem cell research may offer society at large. The millions of dollars of funding available to support such research would also be enticing.
"But the history demonstrates that the burden of human experimentation has rested on the shoulders of the socially and economically disadvantaged. The focus of AB-2531 is to obtain eggs for research, but it ignores the need for research into the impacts of the myriad drugs and procedures used to coax and extract those eggs in the first place." 
Leading the drive against the measure, vetoed by Gov. Jerry Brown in 2013, is the Center for Genetics and Society in Berkeley. It is circulating a flyer aimed at California lawmakers that says,
"This bill incentivizes invasive procedures that expose women to substantial short-term and unknown long-term health risks."
Brown has given no indication that he has changed his position on the measure.

Tuesday, August 02, 2016

Why Does It Matter? Clues for Scientists When Dealing with Reporters

Scientists -- like Boy Scouts -- should be prepared when that call comes in from one of the ink-stained wretches of the mainstream, print media or one of the harried online reporters who hardly have time to think.

Based on decades of experience on behalf of the folks who own the presses, I can tell you that is a good admonition. It was reinforced mightily by another few years on the opposite side of the reporters' notebook, where it became readily apparently that interviewees need to think carefully about the message they want to impart.

Today, I ran across some advice to journalists who aspire to query researchers. I am passing it along to the readers of the California Stem Cell Report, 99.9 percent of whom would never see it otherwise.

It helps give our readers a frame of reference and a clue to what to expect when a mass media opportunity comes knocking. So here it is, questions for journalists to ask scientists, as prepared by Elissa Yancey of the Poynter Institute, a well-respected Florida enterprise involved in journalism training.
  • "Why does this study matter?
  • "Why did you want to do this study?
  • "How does this study relate to your other work?
  • "What was your role in the study?
  • "What surprised you the most?
  • "Did you change any of your or your family’s habits as a result of anything you learned from this study?
  • "What are the economic stakes of the study/topic from your perspective? Your employer's or funder's perspective? (i.e., whose profits / earnings might be harmed or helped by this study? Who is invested in it?)
  • "What comes next? (More studies? Different funders? Legislation?)"

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