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.

Wednesday, September 18, 2013

Surgeon Expected to be Added to California Stem Cell Agency Board

Julie Freischlag, UC Davis Photo
The next new member of the governing board of the California stem cell agency is likely to be a surgeon from Johns Hopkins, Julie Freischlag.

Freischlag was named this week as the new vice chancellor for human health services and dean of the UC Davis medical school, replacing Claire Pomeroy, who resigned to become president of the Lasker Foundation.

Pomeroy had served on the stem cell board since 2005. She was named to the board by the UC Davis chancellor because of her position as dean of the medical school.

Freischlag, who is scheduled to begin her new job Feb. 10, is no ordinary surgeon. Currently she is chair of the Department of Surgery at Johns Hopkins. Prior to that she was chief of vascular surgery at UCLA. A Baltimore Sun profile of her in 2003 said she stands out because of her stellar career in “perhaps the most macho specialty in medicine.” The article said women surgeons are outnumbered 5 to 1 by men.

Responding to a query from the California Stem Cell Report, a spokeswoman for the UC Davis chancellor was noncommital about whether Freischlag would be appointed to the stem cell agency board. Luanne Lawrence, associate chancellor for strategic communications at Davis, said,
“This is one of the important and transitional decisions that hasn’t been addressed quite yet.”
However, the deans of the other major California medical schools sit on the stem cell agency board, which gives away $300 million a year for research. Given that context, it would seem prudent for the dean of the UC Davis medical school to serve on the board as well.

For more on Freischlag's background, see here and here.

Sunday, September 15, 2013

Inside Stem Cells: A Guide from a UC Davis Researcher

The Sacramento Bee today published an article on UC Davis stem cell researcher Paul Knoepfler and his new book, which explores the basics of stem cells and how to make good judgments concerning possible treatments.

The book, “Stem Cells: An Insider's Guide,” is aimed at a general audience while at the same time exploring the nuances of stem cell science.

Below is a copy of the article, which appeared on the main page of The Bee Web site early today and which was written by yours truly. In the print version of The Bee, the Knoepfler piece appeared on the same page as a New York Times story dealing with dubious stem cell treatments(Here is a link to the original version of the Times' piece.).

The Knoepfler story was also the No. 1 article that turned up this afternoon when the Google news category was searched using the term “stem cell.” It is likely to be published elsewhere in the coming weeks as it is circulated by the McClatchy News Service. 

UC Davis stem cell researcher warns consumers to beware of unproven or dangerous stem cell treatments

By David Jensen

Bee correspondent

PUBLISHED SUNDAY, SEP. 15, 2013

In his day job, UC Davis scientist Paul Knoepfler probes the inner workings of stem cells and cancer cells and what makes them behave the way they do.
On the side, the father of three daughters blogs about costly, unproven stem cell treatments and provides guidance for those seeking experimental therapies.
Knoepfler is a rare stem cell researcher who regularly explores the most problematic aspects of stem cell therapies on the Internet in full public gaze. He considers himself an advocate for patients as well as a scientist, having survived an aggressive form of prostate cancer at the age of 42.
Now 46, Knoepfler began his blog in 2010, shortly after his cancer was diagnosed. His blogging has encountered resistance from some colleagues, who are uncomfortable with such public endeavors. But he has polished and expanded the blog to the point that it has received international recognition. He will receive an award in December at the World Stem Cell Summit in San Diego for his advocacy efforts.
This month he moved beyond cyberspace and published “Stem Cells: An Insider’s Guide” (World Scientific Publishing). The book covers stem cells from A to Z and is aimed at the general reader, especially people considering stem cell therapy for themselves, a family member or friend.
The book comes during what has been described by Christopher Scott, a senior research scholar at Stanford University, as “an epidemic of transplant clinics offering so-called cures and therapies” both here and abroad. The international stem cell medical tourism business, widely promoted on the Internet, is taking in roughly $1 billion annually, according to an estimate in Stanford Medicine, a publication of the Stanford medical school.
Knoepfler is a believer in the potential and power of stem cells. But he says that some of the dubious stem cell treatments now being offered have resulted in deaths and injuries. Other unpleasant issues arise as well in stem cell research, including the tendency of some stem cells to generate cancer cells, and Knoepfler wants to talk about them.
Too often in the academic section of the stem cell field, people pussyfoot around the most important issues or do not even dare talk about them at all,” says Knoepfler.
He is “not on some crusade to dissuade people from getting risky stem cell procedures,” but says safety and training need to be encouraged. Reckless behavior endangers the entire field, he says.
There is no better illustration of the risks of unlicensed stem cell treatments administered by untrained doctors than the recently reported case of a woman who received a stem cell facelift, only to have bone grow in her eye,” he said, referring to a Scientific American report involving a Beverly Hills clinic.
To help patients, Knoepfler’s book begins with the basics: What are stem cells? He moves on to topics ranging from whether stem cells can treat baldness to whether they can help with afflictions such as Alzheimer’s, autism and arthritis. Along the way, he discusses the potential for regenerating limbs, made-to-order organ transplants and curing spinal paralysis, none of which have reached the stage where patients can be assured of using the techniques safely and effectively.
Stem cell treatments are not exactly new. They were first used in the 1950s in bone marrow transplants. More recently, what has excited researchers and the public are pluripotent stem cells, which have the ability to transform themselves into any part of the body. The full range of stem cells, however, includes adult, fetal, embryonic and induced pluripotent cells (iPS), which are also known as reprogrammed adult stem cells.
Each type of stem cell has clinical promise for specific diseases, but also certain weaknesses. Some stem cells also stir controversy. For example, embryonic stem cells have been at the center of ethical debates for more than a dozen years,” Knoepfler writes.
Embryonic stem cells are derived from an embryo only a few days after fertilization and require destruction of the embryo. It was that type of process that triggered the federal restrictions – now rescinded by President Barack Obama – on federal funding of research using human embryonic stem cells. Some people believe that the process amounts to killing a human being, while others do not.
Knoepfler has come up with a battery of questions and “rights” that persons considering a stem cell treatment should address as they contemplate spending tens of thousands of dollars for treatments.
His “patient bill of rights” includes the right to treatment by a trained provider, the right to continuing follow-up and the right not to be charged to participate in a clinical trial.
Just this month, he added on his blog, ipscell.com, the top 10 questions that patients should ask. They include such areas as the details of the methods to be used and costs, the cell numbers and types, regulatory compliance by the provider, data supporting the cellular product’s effectiveness and data supporting its safety.
As for mainstream media reports and research papers that seem to promise miraculous cures, Knoepler says, “My advice to patients is to be cautious when reading papers on the clinical use of stem cells. Do not believe everything you read and avoid placing too much weight on any one paper. If something is real, it should be reproducible by multiple groups.”
One of the areas of Knoepfler’s research involves cancer stem cells, a topic of special interest to him because of his own encounter with the disease. “Even with something as wonderful as stem cells, there also can too much of a good thing. Stem cells can cause cancer and in certain conditions they can make cancer especially hard to cure,” he explains.
In the case of embryonic stem cells, he cites their ability to form an unusual tumor called a teratoma. Knoepfler, who was an English literature major as an undergraduate, says, “The name ‘teratoma‘ literally means ‘monster tumor,’ and there is good reason for that nomenclature. These tumors look monstrous when observed by eye, akin to an animal put into a developmental blender.”
Knoepfler did not use stem cell therapy to treat his cancer, but he says that as a cancer survivor he understands all too well why patients turn to it when they’re dealing with a life-threatening or life-changing medical condition.
If you feel that your disease has put you in a place for which patience is not an option,” he says, “talk with your physician. Get second and maybe even third and fourth opinions before deciding whether or not to proceed.”
Knoepfler’s advice may not be appreciated by some of the firms he writes about, but others think well of him.
Jonathan Thomas, chairman of the $3 billion California stem cell agency, says the field needs more scientists like Knoepfler.
It's hard to overstate the value of what Paul does,” Thomas said in an email.
Paul is a powerful advocate for helping the public understand what research is being done, and why it is important. He is gifted at taking complex science and turning it into plain English so that anyone can understand what he's talking about.”
Knoepfler has received $2.2 million from the stem cell agency for his research, which is also funded by the federal government and other sources. He has been at UC Davis since 2006, part of the campus’ stem cell program, which has received $131 million in grants from the state stem cell agency since 2005, generating 333 research-related jobs.
Knoepfler, whose father was a physician and mother a counselor, sees a bright future for stem cells, indeed a “medical revolution.”
Stem cells are today’s new frontier of medicine that will no doubt have an unimaginable impact on our lives, but even more so on the lives of many of our kids and grandchildren,” he said.

Jensen is a retired Sacramento Bee journalist and has produced the California Stem Cell Report (californiastemcellreport.blogspot.com) on the Internet since 2005, writing nearly 3,600 items.

Friday, September 13, 2013

Selling Stem Cell Sizzle: The Future of a $3 Billion Effort

All stem cell research is not created equal, a truism that found fresh validity this week.  Particularly research that could play a role in whether the California stem cell agency can find more cash to continue its operations. 

The difference was highlighted yesterday by articles on the stem cell agency’s blog. The pieces dealt with findings – some esoteric and some not-so-esoteric -- that received international attention. The articles written by CIRM staffers Amy Adams and Don Gibbons were fine as far as they went. But it is one thing to deal with the nuts and bolts of research and another to look at it from the perspective of whether it resonates with the public. 

The research in question is from Spain and Stanford. Researchers in Spain  reprogrammed adult cells in a living mouse to become like embryonic stem cells. Those results received much “gee whiz” attention in the mainstream media, most of which overlooked problematic aspects  involving its cancer-linked results(see researcher Paul Knoepfler's take here and Gibbons' item here).  

The other findings out of Stanford dealt with people and Down syndrome, along with cognitive function, aging and Alzheimer’s.

The press release by Krista Conger from Stanford said,
"'Conceptually, this study suggests that drug-based strategies to slow the rate of stem cell use could have profound effects on cognitive function, aging and risk for Alzheimer’s disease in people with Down syndrome,' said co-author Craig Garner, PhD, who is the co-director of Stanford’s Center for Research and Treatment of Down Syndrome and a professor of psychiatry and behavioral sciences.”
Both the press release and the CIRM blog item briefly noted that funding from CIRM helped to sustain the research. Stanford buried the information at the end of its release. CIRM mentioned it much higher in its item. 

In neither case were specific funding figures mentioned. Nor was there any attempt to say whether this research would have been slow in coming or not coming at all without CIRM help. 

Why does that matter? The $3 billion state stem cell agency will run out of funds for new grants in about three years, not very long given the length of time it takes to develop major funding sources and the rather deliberate pace at which CIRM works on some matters. 

Currently the agency spends about $300 million year on research and is not likely to be able to renew its funding at that level. But if it wants to play at even the $50 million level, it will have to generate some sizzle from the research that it has funded. 

Sizzle is what the Stanford research has. It resonates with people. We all know somebody or a family with issues such Alzheimer’s, Down syndrome or cognitive problems. Missing largely, however, from the press releases, media stories and even the CIRM blog is some sort of way of assessing whether CIRM funding played a KEY role. 

And that is the clincher for agency. That is the sizzle that will sell the agency as absolutely necessary if it truly wishes to turn stem cells into cures.

(Editor's note: Shortly after this item was posted, we searched the agency's Web site. One of the results disclosed that agency gave $1.4 million to Michael Clarke of Stanford for the research. He has filed two progress reports on his findings.) The research received additional support from CIRM as well, but the amounts were not readily apparent.)

Shuffling the Files: Changes on the CIRM Web Site

The California stem cell agency has re-organized its cyberspace book shelves to help make its Web site easier to navigate.

Nothing has been removed, said Amy Adams, the major domo for the CIRM’s Internet operation. 

Writing on the agency’s blog, she explained one of the major changes dealing with “our funding” category.
“It turns out that people looking for information about research we've funded weren't thinking to look under 'our funding.' And since no other section of the site seemed like a likely place to find that content, people sent me helpful emails suggesting that I add pages about where our funding has gone (I politely directed them to that content).”
Most of the information on funding has now been moved to “our progress,” she said, leaving only information on how to get and manage grants under the “our funding” category. Other changes have occurred as well. You can read all about them here.

Wednesday, September 11, 2013

California Stem Cell Agency Blogs on Questionable Stem Cell Clinics

The California stem cell agency has taken notice of a piece in the New York Times that reports on questionable practices of enterprises that say they are stem cell clinics. 

In a blog item written yesterday by Don Gibbons, senior science and education officer at the agency, the agency said that the practices described in the article generated a cautionary advisory to patients last month by the stem cell agency and 12 other organizations. 

Gibbons noted that much of the Sept. 9 Times article by Laura Beil focused on one clinic,  the Regenerative Medicine Institute in Tijuana, which is just on the California-Mexico border. Gibbons’ item reported that one U.S. expert says that some of the clinic’s key procedures would not pass muster in this country.

Gibbons continued,
“Most important, the clinic has not produced any publishable data. We all have great hope for the promise of stem cell science, but I think it is fair to say that anyone who legitimately cares about that promise wants to find out what is the right type of cell to put in each patient. When is the right time for the transplant, and what is the best method of delivering the cells. We will never learn those things without collecting data in a well-designed clinical trial and sharing that data.
The Times article quoted UC Davis researcher Paul Knoepfler as saying,
“There is absolutely no legitimate reason for such clinics to be not publishing their data.”
Gibbons also said that the International Society for Stem Cell Research, which backed away under threats of lawsuits a few years ago from some criticism of dubious stem cell clinics, plans to release this week a new statement on these sorts of treatments.

We should note that Times article, which is syndicated to many newspapers in this country, did not pick up the widely reported instances of deaths and injuries from these treatments. It did report that problems exist in this country as well as abroad. The article said, 
“In the United States, too, it is easy to conduct business outside government oversight, said Dr. George Q. Daley, who studies stem cells for blood diseases at Harvard Medical School. Close down one shady operation, he went on, and more seem to randomly pop up.
“Even questionable publicity does not necessarily hurt business. Regnocyte, a company in Florida, posted an unflattering CNN report about it on its Web site under the heading ‘special coverage.’”

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.”

Tuesday, September 03, 2013

WARF Stem Cell Challenge: Appeal Says Patent Involves Cells Not 'Markedly Different' Than Found in Human Body

The battle over whether excessive protection of stem cell IP stifles research that can lead to cures was engaged once more today with a broadside against the powerful Wisconsin Alumni Research Foundation(WARF).

The attack came from California’s Consumer Watchdog organization and New York’s Public Patent Foundation which have been tussling with WARF for seven years. The dispute over intellectual property (IP) centers on a patent on human embryonic stem cells held by WARF and which the other organizations are challenging in a federal appellate court in Washington, D.C.

More specifically, the patent involves research by Jamie Thomson of the University of Wisconsin, and now also of UC Santa Barbara, in which he isolated human embryonic stem cells.

Consumer Watchdog of Santa Monica, Ca., this morning issued a news release concerning the organizations’ appellate brief that was filed last week. It cited the U.S. Supreme Court ruling earlier this year that said genes cannot be patented because they exist in nature.  The lead attorney in that successful case, Dan Ravicher of the Public Patent Foundation, is also handling the challenge to WARF.

The news release said that Thomson deserved credit for being first to isolate and maintain human embryonic stem cells, but “his achievement was not the result of his having created a patentable invention.” The brief said that the work involved was “obvious.” One of the main reasons for Thomson’s achievement, the news release said, was that “he had access to human embryos and financial support that other researchers did not have.

The brief said,
The claims at issue here cover human embryonic stem (hES) cells that are not markedly different from those in our bodies. Thus, the claims are invalid under 35 U.S.C. § 101 for covering ineligible subject matter, an issue the Court may and, as a matter of judicial economy and public policy, should address.”
The challenge to the WARF patent has drawn impressive support in the scientific community, including  Jeanne Loring, now director of the Center for Regenerative Medicine at The Scripps Research Institute, who was involved from the start. In 2007, Loring wrote in Nature that she became involved in the case because “scientists have an obligation not only to perform research but to make sure that our research can benefit the society that supports it.

The news release said,
“Later in the case Dr. Alan Trounson, then of Australia’s Monash University and now president of the California Institute for Regenerative Medicine, Dr. Douglas Melton of Harvard and Dr. Chad Cowan of Harvard filed affidavits supporting the challenge.

Friday, August 30, 2013

Tracking the Fruits of California Stem Cell Agency Research

The California stem cell agency yesterday shed some interesting light on the awards in its $41 million round this week and their pathway to actually producing a product that can be used to treat persons who are suffering from diseases.

It is a difficult and long journey to generate usable therapies, a process poorly understood by the public, which was promised in 2004 that the stem cell agency would produce cures for ailments afflicting half the population of the state.

Writing on the agency’s blog, Amy Adams, CIRM communications manager, dealt with the issue indirectly. She said,
“Many scientists who receive our early translation awards first got their idea for a therapy while carrying out research with one of our other awards. In fact, eight of the scientists in this round of funding had previous CIRM funding for an earlier stage of research. If a scientist's early translation award provides good results, the scientists are then able to apply for one of our disease team awards, which fund the effort of compiling data to convince the Food and Drug Administration to allow them to test it in people.  Other organizations fund only early discovery research or only preclinical research. Under those conditions, researchers continually pause their projects to look for new sources of funding as the project moves through the phases toward clinical trial.
One of the virtues of the California stem cell agency is its promise of a continued stream of funding. Former Chairman Robert Klein used to tout that particular aspect of the agency, particularly in light of limited federal resources.

Adams’ comments implicitly raise important questions concerning CIRM’s entire portfolio. How many CIRM grants have led to additional funding from CIRM? How many are basically one-off shots that have not led to research that has advanced the development of stem cell therapies, either via CIRM or other funding. What is the therapeutic and scientific significance of the research that is linked by more than one CIRM award?  What previously funded CIRM research could be fruitfully funded again to advance the science and not necessarily through the traditional grant rounds, which sometimes have awkward timing?

Unmentioned in Adams’ item is an application from a UC Irvine researcher that came up at Wednesday’s meeting of the governing board of the stem cell agency. The woman, whose name was not clearly audible on the Internet audiocast, publicly appealed rejection of her application by reviewers. She noted that it was an extension of work that was previously funded by the agency. She also noted that the score on her review was all but identical to work that was funded. The board, however, turned her appeal aside, which had already been rejected behind closed doors by CIRM staff.

Hers is not the only such case in CIRM history. But they are virtually impossible to track systematically because of the structure of the CIRM grant-making progress. It is also not clear whether the agency itself is tracking its research awards to determine if they result in continuing, fruitful research in a specific area. Nonetheless, the matter deserves some public attention. 

Wednesday, August 28, 2013

UCLA Takes Four of 13 Awards Today; One Business Wins

The California stem cell agency has made it official, sending out its press release on the $41 million in grants approved today for institutions throughout the state. 

Most of the 13 awards, as usual, went to organizations represented on the governing board of the agency. Individual board members, however, are barred from voting on specific grants to their organizations. 

UCLA topped the list with four grants. No other institution received more than one, including only one business, Numerate, Inc., of San Bruno, via John Griffin, the firm's chief scientific officer. The lack of awards to businesses has long been a sore subject in the biotech community.  

The only news story so far was written by Bradley Fikes of the San Diego U-T, which circulates in an area that is a hotbed of biotech research. Institutions there snagged $12.6 million in four grants. Fikes also identified one of the five researchers who lost their appeals on negative grant review decisions. He is Evan Snyder, leader of stem cell research at Sanford-Burnham Medical Research Institute in La Jolla, who had a $5 million request before the agency.

CIRM Board Meeting Adjourns

Today's meeting of the directors of the California stem cell agency concluded at 4:11 p.m. PDT. We do not anticipate posting additional news items today.

Stem Cell Agency Seeks Stronger Ties with Possible Industry Funding Partners

The California stem cell agency today triggered a new program aimed at recruiting major biotech and venture capital firms to assist in providing tens of millions of dollars for research by California enterprises.

The effort, part of an $80 million business-friendly initiative, was approved by the agency's governing board on a voice vote.

Participating companies will have a special relationship with the state agency, according to a staff document. The "industry collaborators" will have early input into concept funding proposals prior to their presentation to the agency's governing board. The companies will also be able to attend agency workshops and meetings involving hundreds of grant recipients. 

Other aspects of the proposal call for special event-hosting arrangements aimed at creating more collaborations along with posting of information from the selected collaborators on the CIRM website.

California Stem Directors Still in Closed Session

Directors of the California stem cell agency today are still huddled behind closed doors, discussing an evaluation of CIRM President Alan Trounson. It is not clear when the public meeting will resume. Normally, little is publicly disclosed about such private personnel sessions, which are permitted under state law.

UCLA Snags $3.6 Million from California Stem Cell Agency

UCLA scored today with at least two grants, totaling $3.6 million, from the California stem cell agency. 

Seeking the cash were Donald Kohn,  application 6823, and Gerald Lipshutz, application 6831. Both of the  grants are for $1.8 million each. 


Their applications were initially in the agency's tier two category, which means that CIRM's reviewers did not approve them outright for funding.  CIRM staff, however, did under a new procedure, and the agency's governing ratified the recommendation. 

Lipshutz also appeared before the board along with several patient advocates who made emotional appeals for funding. Lipshutz's research deals with urea cycle disorders, which occur in one out of 8,200 births. Current treatment is arduous and can involve liver transplants.  

Kohn's research deals with sickle cell disease, which afflicts primarily African-Americans. His efforts are aimed at correcting the sickle gene defect in the blood stem cells before transplanting them back into the patient.

$41 Million in California Stem Cell Grants Virtually Approved

Directors of the California stem cell agency today all but approved about $41 million in early translational grants, rejecting all appeals by applicants and accepting staff recommendations on marginal grants.

The roll call vote was held open this morning to record a vote by one board member who was not present at the time. It is virtually certain that the member will vote in favor of affirmative action on the applications in question.

One member of the board, Joan Samuelson, abstained from voting on any of the applications. She said she did not think the board had adequate information on its total grant portfolio, particularly in view of the declining amount of money available.

The agency has about $600 million in uncommitted funds and is scheduled to run out of cash for new grants in 2017.

The research acted on today is aimed at “proof of concept for development of a therapy candidate and/or studies to select a development candidate. The approved grants can be found on this CIRM website page and are listed in tier one and tier two.  Identities of the applicants are withheld by CIRM to avoid embarrassing rejected candidates and to avoid disclosing the names of applicants to board members before they vote. However, applicants often appear before the board, as they did today, and identify themselves.

Five applicants appealed negative decisions on their applications by grant reviewers. The agency declined to disclose the appeal letters or identify the applicants, information that was a public record under the previous appeal procedures. New processes were put in place this spring that moved the appeals behind closed doors and made them subject to staff instead of board review. Nonetheless, rejected researchers have a legal right to address the board on appeals or any other matters.

At the request of the California Stem Cell Report, the agency provided the numbers of the grants on which appeals were filed. They are: 06787, 06888, 06761, 06793 and 06830. Review summaries on the applications can be found here. 

We have asked the agency to provide its legal and policy justification for now withholding information that was once a public record.


Stem Cell Board Moves into Closed Door Lunch Session

Today's meeting of the California stem cell agency governing board is now in recess for a closed-door lunch session involving the evaluation of CIRM President Alan Trounson. It is not clear when the open session will resume, but probably it will be at least 90 minutes.

UCLA Application for $1.8 Million Moves Ahead at Stem Cell Board Meeting

Directors of the California stem cell agency this morning moved forward on a $1.8 million application from UCLA that the agency's reviewers declined to approve outright.

The application (No. 6831) was moved into a category that will be approved routinely later today by the board. CIRM staff recommended that the application be approved.

Advocates speaking on behalf of the application identified UCLA as the institution involved. A researcher also spoke, but his name was not clearly audible on the Internet audiocast.

California Stem Cell Board Moves Positively on 'Wobbler' Grant Application

Directors of the California stem cell agency this morning ratified a staff recommendation for funding of a $1.8 million application that was a "wobbler" when it was examined behind closed doors by agency reviewers.

The CIRM staff had recommended funding of the application (No. 6823) with the condition that the agency staff be satisfied with the support to be provided by a collaborator. Reviewers had declined to approve the application outright.

The agency has not disclosed the identity of the applicant.

Discussion Begins in $70 Million Grant Round

Directors of the California stem cell agency have begun discussion of applications in its $70 million early translational round. Above is the Power Point presentation to the board of some of the applications including the range on the scientific scores in the round.

Stem Cell Agency Pays Tribute to the Late Duane Roth

The governing board of the California stem cell agency today paid tribute to the late Duane Roth, co- vice chairman of the agency, who died recently as the result of a bicycle accident.

With members of Roth's family present, CIRM Chairman J.T. Thomas characterized Roth, 63, as a "voice of reason" on the 29-member board. The video included testimonials from both staff and board members.

He was described as a "kind person" who could find "common ground" on difficult issues. Roth was deeply involved in San Diego affairs that went well beyond the stem cell agency. More than 1,000 persons attended memorial services for Roth earlier this month in San Diego.

CIRM President Alan Trounson said following the video that he will "miss (Roth) terribly" and expressed  "hope that his memory will lighten and brighten the day for all of us."

Jeanne Loring, a stem cell researcher at Scripps, said Roth "inspired us to do more than we thought we could do." She said he was an unusual kind of businessman who respected science.  "I wish I could thank him one more time," she said.

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