A $6 Billion Question: Progress of the California Stem Cell Agency

The headlines march like legions across the Internet and throughout the world.

“New type of stem cell helps your fingers regenerate”

“Stem cell technology can mass-produce cancer-killing cells to target tumours”

"Stem-cell treatment restores sight to blind man”

“Special stem cells could heal hearts”

But then there is this extraordinarily rare headline that sounds a harshly different note:

“Stem cells: what happened to the radical breakthroughs?”

All these headlines go to address, in one form or another, a request/question posed last month by an anonymous reader of the California Stem Cell Report. The comment came on an item about the California stem cell agency's $70 million plan to establish a network of “Alpha” stem cell clinics in California.

The reader said,

“It would be nice to have an overall update on how much as been spent on California's stem cell research project and what progress has been made.”

On the surface, the answer is easy. The agency has given away $1.8 billion. The agency says it has made tremendous progress and expects to make even more with the about $600 million it has left. The prestigious Institute of Medicine has said the agency has “achieved many notable results.”

However, no thorough, rigorous evaluation has been made of the details of the agency's scientific contributions, specific grant awards or its impact on the field of regenerative medicine. No one has attempted to genuinely assess whether the work of the agency is or will be worth the roughly $6 billion(including interest) that California taxpayers will have paid for the agency's ambitious efforts.

Then there is the question of “progress towards what?” Is the progress to be measured against the promises of the 2004 ballot campaign that resulted in creation of the stem cell agency or more modest goals that eschew the hype of the campaign?

The stem cell agency is burdened in a way that most science is not. The 2004 campaign created a sort of contract with voters. They were led to believe nine years ago that the cures for diseases that the campaign said afflict nearly one-half of all California families were, in fact, right around the corner. Few, if any California stem cell researchers were publicly warning that a hard and long, long slog remained before therapies reached patients.

Last week, however, Simon Roach of the British newspapers, The Guardian and Observer, shed some light on the early, rosy promises of stem cell science compared to the world as it exists today.

He wrote that in 1998,

“(B)iomedical engineer Professor Michael Sefton declared that within 10 years, scientists would have grown an entire heart, fit for transplant. 'We're shooting big,' he said. 'Our vision is that we'll be able to pop out a damaged heart and replace it as easily as you would replace a carburetor in a car.'

“Fifteen years on, however, we've had some liver cells, eye cells, even a lab-grown burger, but no whole human organs. We could be forgiven for asking: where's our heart? It does seem strange that a field stoking so much excitement could be so far off the mark. Speaking last week about the vision that he and his colleagues outlined in 1998, Sefton said they had been 'hopelessly naïve.' As time plodded on and an understanding of the biological complexity increased, the task seemed bigger and bigger. Even now, a cacophony of headlines later, we are not much further ahead.

“Chris Mason is a professor of regenerative medicine at University College London and believes that concentrating on organ regeneration is missing a trick. 'These organs are immensely complex,' he said. 'They've got nerves, blood vessels, in the case of the liver, a bile system – there are huge degrees of complexity. These things take a long time to grow in humans, let alone in the lab without all the natural cues that occur in the growing embryo.'"

The final paragraph in Roach's article said,

“There's a tension in medical research between the glory of the big discovery and the assiduous commitment to real application. 'We're hoping the scope and possibilities of this project will catch the public's imagination,' Sefton concluded in 1998. It did, but perhaps the public's imagination isn't always what science should be vying for.”

Little doubt exists that the California stem cell agency has made a significant contribution to stem cell science, although the size of that contribution – beyond dollars – remains to be measured. For now, the key for the agency and the public is to focus on activities that will generate the greatest value over the next few years and advance the science that has already been financed by the agency.

As the $700,000 Institute of Medicine report said,

“The challenge of moving its research programs closer to the clinic and California’s large biotechnology sector is certainly on CIRM’s agenda, but substantial achievements in this arena remain to be made.”

Stem Cell Agency's Duane Roth Eulogized at Memorial Services

An estimated 1,000 persons attended services last week for Duane Roth, co-vice chairman of the California stem cell agency, who died at the age of 63 following a bicycle accident.

The San Diego U-T reported,

“A Who’s Who of San Diego’s technology, business and civic community gathered Friday to bid farewell to Duane Roth. The biotech entrepreneur, community leader and director of Connect died last weekend of injuries sustained in a cycling accident.

“Among attendees were Gayle and former Gov. Pete Wilson, who had just celebrated his upcoming 80th birthday with former colleagues and friends in Sacramento, county Supervisor Ron Roberts, former Assemblyman Nathan Fletcher, Chamber head Jerry Sanders, Preuss School benefactors Peggy and Peter Preuss, SDG&E CEO Jessie Knight, and many biotech and high-tech leaders. These included Irwin Jacobs, Ted Waitt and Denny Sanford.”

'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.'”

California Stem Cell Agency on Lacks: Informed Consent Cannot Remove All Questions

(Photo and caption from the stem cell agency blog item this morning.)

The $3 billion California stem cell agency today weighed in on the Henrietta Lacks-NIH arrangement restricting the use of her cell lines in research.

Writing on the agency's blog, Geoff Lomax, the agency's senior officer for its standards group, noted that the DNA sequence of her cell line was published without the knowledge of her descendants. Lomax said,

“The family was understandably upset by the lack of consultation and in response the research team removed the genome data from public access.”

Lomax continued,

“CIRM has benefited from these efforts. We are currently supporting an initiative to collect tissue samples from thousands of people with a range of incurable diseases and create reprogrammed iPS cells from those tissues (here's more about that initiative). These cells will be a resource for scientists worldwide working to understand and treat diseases. Part of this initiative includes a consent process to make sure people who donate fully understand how their cells will be used. (This process is formally called informed consent.) 

“The informed consent process includes a form that identifies the purposes of the research and describes the way cells will be used. We are also developing education materials that will help potential donors quickly and easily understand the basic aspects of research that will be conducted with those cells. The end result of this collaboration with our grantees will be a process that is truly informative to donors.

“The informed consent process can’t entirely eliminate all future questions on the part of the donor, but it does ensure that donors have a chance to understand how their cells will be used and what information will be made public—something Henrietta Lacks and her family never had.”  

Skloots, Collins and More on Henrietta Lacks' Cell Line Deal

More details about the unprecedented arrangement involving Henrietta Lacks' cell line emerged today in a wide range of publications, including a Nature journal piece that said it was not a precedent.

The article was co-authored by Francis Collins, head of the NIH, and Kathy Hudson, deputy director for science, outreach and policy at the NIH.

“It is important to note, however, that we are responding to an extraordinary situation here, not setting a precedent for research with previously stored, de-identified specimens. The approach we have developed through working with the Lacks family is unique because HeLa cells were taken and used without consent, and gave rise to the most widely used human cell line in the world, and because the family members are known by name to millions of people.”

The restrictions on use of the cell lines came about after a flap erupted about their recent use without the knowledge of her descendants. (The California Stem Cell Report carried a commentary on it yesterday.) Rebecca Skloots, author of the best-seller, “The Immortal Life of Henrietta Lacks,” wrote about the controversy in a March 23 op-ed piece in the New York Times. She said,

In the article, Skloots said,

“Imagine if someone secretly sent samples of your DNA to one of many companies that promise to tell you what your genes say about you. That report would list the good news (you’ll probably live to be 100) and the not-so-good news (you’ll most likely develop Alzheimer’s, bipolar disorder and maybe alcoholism). Now imagine they posted your genetic information online, with your name on it. Some people may not mind. But I assure you, many do: genetic information can be stigmatizing, and while it’s illegal for employers or health insurance providers to discriminate based on that information, this is not true for life insurance, disability coverage or long-term care.

“'That is private family information,” said Jeri Lacks-Whye, Lacks’s granddaughter. “It shouldn’t have been published without our consent.'”

Nature also carried a Q&A with Collins in which he said,

“This has wrapped in it science, scientific history, ethical concerns, the bringing together of people of very different cultures, a family with all the complications that families have.”

In the Wall Street Journal this morning, Ron Winslow described the arrangement with the NIH like this.

“Under the pact, two descendants of Ms. Lacks will serve on a six-member panel with scientists to review proposals from researchers seeking to sequence the DNA of cell lines derived from her tumor or to use DNA profiles of such cells in their research. That gives family members a highly unusual voice in who gets access to personal health information.

Terms call for controlled access to the genomic data and credit to the Lacks family in papers and scientific presentations based on the research done with the DNA data.”

In an interview in The Scientist, Skloots, who was involved in the Lacks-NIH negotiations, said the Lacks family asked for her participation.

“The only reason I was involved in this is because scientists did this without the family’s consent and then it got all of this press coverage, and no one asked the question, 'Did the family give consent?' So I sort of waded back in.”

She continued, 

“That OpEd that I wrote was the first time I’d ever publicly expressed an opinion, which was, 'Really?!? Are we going to continue to not ask the Lacks family questions?' I was kind of shocked in a sense that nobody thought to raise that issue.”

The Henrietta Lacks Story and Eggs, Money and Motherhood

The legacy of Henrietta Lacks popped up again today in a piece in the New York Times that should resonate among stem cell researchers and within the stem cell industry.

It even has a current hook involving California legislation to permit women to sell their eggs for the purposes of scientific research – a bill that is now on the desk of Gov. Jerry Brown.

The issues in the Lacks saga involve ownership of human cells, trafficking in them and informed consent, all of which surface in one form or another in the state legislation.

But first a refresher on Henrietta Lacks. She was an African-American woman who died in 1951 of cervical cancer at the age of 31. Shortly before her death, physicians removed some of her tumor cells, and, as recounted in today's NYTimes article by Carl Zimmer,

“They later discovered that the cells could thrive in a lab, a feat no human cells had achieved before.

"Soon the cells — nicknamed HeLa cells — were being shipped from Baltimore around the world. In the 62 years since — twice as long as Ms. Lacks’s own brief life — her cells have been the subject of more than 74,000 studies, many of which have yielded profound insights into cell biology, vaccines, in vitro fertilization and cancer.”

But Lacks never consented to her cells' being studied, a situation not uncommon at the time, nor did her family know about the situation until 1973. The complete story was chronicled in 2010 in a best-selling book, “The Immortal Life of Henrietta Lacks," by Rebecca Skloot.

Zimmer noted in today's article,

“For 62 years, (Lacks') family has been left out of the decision-making about that research. Now, over the past four months, the National Institutes of Health has come to an agreement with the Lacks family to grant them control over how Henrietta Lacks’s genome is used.”

The particulars involving her genome are in Zimmer's story. But the article implicitly raises anew questions that make many scientists uncomfortable. Often they contend that the situation involving Lacks could not occur today because of higher ethical standards. Standards ARE higher today. But problems continue to arise in the scientific community, including the sale a few years ago of willed body parts at UCLA for $1.5 million to private medical companies.

Development of products based on human stem cells promises even greater rewards, with billion-dollar blockbuster therapies not out of the range of possibilities. Profit and the desire to record a stunning research triumph are powerful motivators. They can lead to short cuts and dubious practices, such as seen in the Korean stem cell scandals of 2006.

So we come to whether women who donate their eggs for stem cell research can give truly informed consent when they surrender all rights to whatever products may result from parts of their bodies, as is common on such consent agreements. Or for that matter, what about the men who give up adult cells for reprogramming to a pluripotent state? Can they really understand the likelihood of a billion dollar product being generated with the help of their contribution? On the other hand, can the donors also truly understand that they are probably more likely to be struck by lightning than have their body parts result in a medical blockbuster?

These considerations may seem insignificant to some in science. But to grasp their full implications, one only has to read a few of the nearly 200 reader comments today on Zimmer's article today. Here is a sample.

From Frank Spencer-Molloy in Connecticut:

“(T)the Lacks family was robbed. Scores of companies profited to the tune of tens of millions of dollars from products they made derived from Henrietta Lacks' cancerous cells. Maybe this will provide some impetus to a wider consideration of the rights patients are entitled to when their tissues are cloned and disseminated to other researchers and ultimately put to use in profit-making ventures.”

From Robbie in New York City:

“At the very least, this family needs to be financially compensated for the anguish of their discovery and for the time and energy they've put into pursuing their rights. In my opinion, they also deserve a portion of any commercial gain that's been made using the HeLa cells. It is only through having to give away money that the powerful learn manners.”

From Julia Himmel in New York City:

“It is absolutely true that scientists have had a blind spot when it came to the human element of the HeLa cells.”

The pay-for-eggs legislation (AB926) now before Gov. Brown requires informed consent from those who provide eggs. Opponents of the measure, however, argue that truly informed consent from some women could be actually impossible because of economic pressures felt by the women. Writing in The Sacramento Bee last month, Diane Tober and Nancy Scheper-Hughes said,

“Allowing a market in eggs for research would reach beyond the current pool to target women who may be motivated by dire need. How many low-income women might consider selling their eggs, multiple times, to feed their children or pay the rent?”

Even the fertility industry group sponsoring the legislation acknowledges that informed consent can be problematic. A 2012 news release from the American Society for Reproductive Medicine said, 

“Prospective egg donors must assimilate a great deal of information in the informed consent process, yet it remains difficult to determine the extent of their actual understanding of egg donation and its potential risks.”

The story of the treatment of Henrietta Lacks and her descendants is a poor commentary on science and medicine. Yet it resonates with the public, which is keenly sensitive to scientific and medical abuses, even in situations that did not appear to be abuses at the time.

Stem cell research already is burdened by its own particular moral and religious baggage. With commercialization of new, pluripotent stem cell therapies coming ever closer, the last thing the field needs is contemporary version of the Lacks affair. It would behoove researchers and the stem cell industry to walk with more than normal care as they manipulate products that are tied inextricably to visions of both motherhood and money.  

Californians Top List of Stem Cell Honorees

Californians dominated the list of those to be honored at the World Stem Cell Summit coming up in San Diego this December.

They include fellow blogger Paul Knoepfler, a stem cell scientist at UC Davis, and Roman Reed, the San Francisco Bay Area stem cell activist and son of another stem cell advocate, Don Reed. Both are among the 2013 Stem Cell Action Award honorees for this year.

Others include Denny Sanford, a philanthropist whose name now adorns the Sanford-Burnham Institute in La Jolla, and Malin Burnham, who is also linked to the institute. Also being honored is Mary Ann Liebert, whose firm publishes peer-reviewed journals in science and biomedical research.

Malin is a San Diego businessman who joined with an anonymous donor in 1996 to contribute $10 million to the La Jolla Cancer Foundation. It was renamed to reflect that contribution. In 2010, Sanford pledged $50 million to the organization, and it was renamed again.

Memorial Services Friday for Duane Roth, Co-vice chairman of the California Stem Cell Agency

A memorial service for Duane Roth, co-vice chairman of the California stem cell agency, will be held Friday at 11 a.m. at Immaculata Church at the University of San Diego.

Roth died Saturday from injuries suffered in an accident last month while bicycling in the mountains east of San Diego. He was 63.

San Diego has seen an outpouring of tributes in the wake of Roth's death for his contributions to the community in the life sciences, philanthropic and technology areas. He had served on the stem cell agency board since 2006 and had been scheduled to become of chairman of the Sanford-Burnham Institute this fall. He was CEO of Connect, a non-profit organization aimed at support entrepreneurship in the technology field.

Ted Roth, Duane's brother, remembered him in a piece in the San Diego U-T as the oldest of five sons growing up in Wayland, Iowa. Ted Roth wrote that their parents relied on Duane "to set an example for his brothers, and he was the one they called upon in their later years. He was a lifelong mentor and friend to his brothers, someone that was always there to share in life’s experiences." 

Ted also wrote about his brother's involvement in technology and business.

 "Duane was captivated by the possibilities that innovation provides in improving the world in which we live." 

The family has suggested that in lieu of flowers that donations be made to the Otterson Fund at Connect, Challenged Athletes Foundation or the Copley-Price Family YMCA.

Here are links to some of the other recent articles on Roth: San Diego U-T (see here and here), La Jolla Patch, La Jolla Light.

CIRM's Roth Dies Following Bike Accident

Duane Roth, co-vice chairman of the California stem cell agency, died yesterday from brain injuries suffered in a bicycle accident two weeks ago.

Duane Roth, Connect photo

Roth, CEO of Connect, a San Diego organization aimed at fostering technology entrepreneurship, succumbed yesterday afternoon at the UC San Diego Medical Center, the San Diego U-T reported. He was 63.

An avid bicyclist, Roth was injured while biking in the mountains east of San Diego July 21. Roth hit an outcropping and his helmet was broken in the accident.

Roth was a long-time member of the 29-person governing board of the $3 billion California stem cell agency and was a strong advocate for industry. He chaired the agency's loan task force, was vice chair of the Intellectual Property and Industry Engagement Subcommittee and a member of the executive committee.

J.T. Thomas, chairman of the stem cell agency, released the following statement this morning.

“On behalf of all the CIRM family, we mourn the loss of our colleague and dear friend Duane Roth.  Throughout his tenure with us, he was one of the true stewards of the mission, offering countless insights on the role of industry in the world of regenerative medicine and how best and efficiently to drive therapies through to patients.  He was unfailingly a voice of reason and optimism and always sought to find ways to make things happen, refusing to take 'no' for an answer.  Though one of 29 Board members, his extensive participation as co-Vice Chair of the Board, co-chair of Intellectual Property and Industry Engagement Subcommittee and a member of our Executive Committee gave Duane a singularly important and resonant voice in our organization.  His passing will be deeply felt by all of us as well as by the many patients and other CIRM stakeholders whom he touched over the years.  We send our deepest sympathies to Renee, Duane's brothers and the rest of the Roth family.”

Roth recently was involved in raising funds for cancer, and reporter Bradley Fikes wrote in the San Diego U-T,

“Contributions in Roth’s name can be made to Pedal the Cause, a fund-raiser for cancer research that Roth supported. More than $10,000 has been raised since Roth's accident."

Pay-for-Eggs Legislation: A Comment on Risk

The author of the Forbes piece cited in the eggs legislation item today has responded to a comment filed by two persons opposed to the measure that would remove the ban in California on paying women for their eggs for scientific research.

Here is the text filed by Jon Entine, executive director of the Genetic Literacy Project.

“Diane and Nancy, I'm shocked that you are either unaware or do not acknowledge that there are studies of oocyte retrieval surgeries that show very persuasively that the potential harm from this procedure is manageable. While you refer to 'stories' of women being harmed--that's called anecdotal evidence and is the antithesis of science--you ignore the established research in this area, which makes it clear that you are reacting hysterically rather than responding to empirical evidence. I would suggest that you read the National Academies Press workshop report: Assessing the Medical Risks of Human Oocyte Donation for Stem Cell Research (http://www.nap.edu/catalog.php?record_id=11832). It cites numerous studies, including a German study that examined the outcome of approximately 380,000 oocyte retrieval surgeries during 2000-2004. For the procedures for which there was information, the rate of complications was very low: only 0.002 percent—2 in every 100,000—had complications that required surgery to correct.

“Studies have also examined the potential risks of retrieval for a woman's future fertility.
“According to one large study, the rate of infection after oocyte retrieval was about 1 in every 200 IVF cycles, and surgery is needed to treat pelvic abscesses in less than 1 in 1,000 IVF cycles. 

“About five hundred egg donations take place in Canada each year, according to the Canadian Fertility and Andrology Society.The CFAS told me that, between 2001 and 2010, only two donors in Canada, out of a total of 4,177 donations, suffered from “severe” OHSS, which usually involves hospitalization. Fourteen others had “moderate” OHSS. These numbers are collected in a database called the Canadian Assisted Reproductive Technologies Registry.

“So sure, you can find your 'stories' but they do not represent a scientific review of the available data--you are trying to legislate based on fear. That's not science; that's the dark ages, and it's exactly the tactics used by anti-abortionists (and indeed by organizations like the Center for Genetics and Society which opposes such beneficial advances as mitochondrial replacement surgery).

“Furthermore, because women have a set of two ovaries and two fallopian tubes, they can remain fertile even if one set is damaged, and there is no evidence that both might be threatened simultaneously by the side effects of retrieval surgery. 

“Today doctors have had two decades of experience with the use of hormone treatments to maximize the number of eggs that can be harvested from a woman, and they have become quite proficient in the production of oocytes. During that time they have also worked to improve the safety of the procedure and decrease the potential risks. Despite these improvements some risk will remain, because hormones have a powerful effect on the body—they could not increase egg production so dramatically if this were not true—and anything with a powerful effect on the body has the potential for harmful side effects as well. 

“Egg donations are done for a reason. There are risks and benefits. For you to exaggerate the risks based on 'stories' and ignore the evidence is unconscionable. It's exactly what anti-abortion groups do and what opponents of genetically modified foods do--you promote fear around manageable (or in the case of GMOs, negligible) risk. 

“Your call for 'further studies' is the age old technique of reactionaries trying to control other people and impose their values on other people. You know darned well, because of your fundamental ideological opposition to this procedure, no study results could ever meet your standard of acceptability. 

“You are trying to control other women's bodies, claiming you have superior knowledge and wisdom--those are pro-life talking points. Your views, and that of the organizations that you represent, are illiberal.”

Comment re Pay-for-Eggs Item and Forbes Article

One of the authors of an op-ed piece in The Sacramento Bee has filed a comment in connection with an item today on the California Stem Cell Report. The item dealt with the California pay-for-eggs bill, which was also the subject of an op-ed piece in The Sacramento Bee as well as an article yesterday on the Forbes magazine website that discussed the op-ed piece critically.

A quotation from the article was contained in this item earlier today.

Here is the text of the comment from Nancy Scheper-Hughes, a professor of anthropology at UC Berkeley and director of Organs Watch.  Diane Tober, associate executive director of the Center for Genetics and Society of Berkeley, was the other author.

“Dr. Diane Tober and Prof. Nancy Scheper-Hughes  are 'pro choice'  social scientists who are concerned about the absence of any evidence-based medicine on the long term effects of hyper-stimulation for oocyte (egg) production in young women research subjects. We are not concerned about abortion, right to life, or obstructing  needed and valuable research on stem cells. We are concerned about the safety for potential research subjects who are being actively recruited to participate in  invasive medical procedures without any medical research studies on the possible risks and consequences of egg multiplication and extraction. We are on record that we  fully support stem cell research but not at the expense of unprotected egg donors.”  

Pay-for-Eggs Legislation Now Before California Gov. Jerry Brown

California's pay-for-eggs bill is now officially on Gov. Jerry Brown's desk, awaiting his signature or veto.

The measure, AB926 by Assemblywoman Susan Bonilla, D-Concord, was sent to the governor at 4:45 p.m. PDT yesterday. On July 1, it easily won legislative approval and has been held in legislative processing since then. The governor has 12 days to act on the measure or it becomes law without his signature.

The legislation would remove the state ban on payment to women for their eggs for scientific purposes. Currently women who provide their eggs for fertility purposes can be compensated. Fees run as high as $50,000 in some cases, depending on the characteristics of the woman providing the eggs, but generally are in the $10,000 range or less. The bill does not affect the ban on the use of funds from the California stem cell agency to compensate egg providers.

Bonilla's bill is sponsored by the $5 billion-a-year fertility industry, which is backing it on motherhood and sexual equity grounds. Supporters say women should receive payment for their eggs just as men are paid for their sperm. They also argue that more eggs are needed for research into fertility problems. In the stem cell field, scientists have also said it is nearly impossible to find women who will provide eggs unless they are paid.

Opponents contend that the process of stimulating production of eggs can be risky or dangerous. They say that the longterm effects of the process have not been studied well. They also argue that it will lead to exploitation of low income and minority women to produce eggs that then can become a profitable commodity for the largely unregulated fertility industry. (For more informationon on the bill, see here, here and here.)

In one op-ed piece in The Sacramento Bee, opponents cited the late philosopher Ivan Illich, who was much admired by Jerry Brown, who considered him a friend. Illich was quoted as warning "against the processes of medical industries which 'create new needs and control their satisfaction and turn human beings and their creativity into objects.'"

The industry group says, however, that Brown is committed to signing the bill.

The measure surfaced in the news yesterday in an article on the Forbes magazine website by Jon Entine. He wrote,

“Should activist groups, working through legislators, exercise their control over women’s reproduction? Do we really 'own' our own bodies? Or does that tenet only hold when nanny groups say it’s okay?”

(One of the authors of The Sacramento Bee op-ed piece criticized in the Forbes article later filed a comment concerning their position.)

The egg legislation may have implications for regulation of stem cell research by the state Department of Public Health(again not involving the California stem cell agency). Last month the California Stem Cell Report asked Hank Greely, a Stanford law professor and chair of the state department's Human Stem Cell Research Advisory Committee, about the measure. He replied,

“Well, if (when?) AB 926 is signed, I think our committee should meet to consider what recommendations we would make to the (the department) as a result of the bill.  Those recommendations could lead, if the committee and the department agree, to a revision of the state guidelines.  As a matter of law, a statute, particularly a subsequent statute, trumps a guideline where they are in conflict, but basically I expect we'll see what the committee thinks and what the department decides.  I don't wish to guess at the results of either process.”

Another question that was not discussed publicly during the debate on the legislation deals with whether human eggs provided with compensation would be subject to state sales tax at any stage in the process. A check of the tax code, however, makes it clear that eggs are tax free. The code states that “any human body parts held in a bank for medical purposes, shall be exempt from taxation for any purpose." The definition of “bank” includes research facilities, and "medical purposes" includes research.

CIRM's Roth Remains Hospitalized

Duane Roth, a co-vice chairman of the $3 billion California stem cell agency, is displaying no apparent brain damage following a bicycle accident 11 days ago in the mountains east of San Diego.

Bruce Bigelow of Xconomy reported this morning,

“MRI diagnostic imaging showed no apparent brain damage, but he remains sedated, according to his brother, Ted Roth. While doctors see nothing that would prevent Duane Roth from a good recovery, told me by phone yesterday his caregivers are moving slowly to give him time to heal.”

Roth is also CEO of Connect in San Diego, a technology industry assistance organization.

Skimpy Coverage of Alpha Clinic Concept Approval

News coverage of approval of the California stem cell agency's ambitious, $70 million Alpha clinic plan has been quite light but does include one article in the Los Angeles Times, the state's largest circulation newspaper.

The concept proposal was ratified last week by the agency's board with RFAs scheduled to be posted in October. The agency is seeking to build a basis for a robust stem cell clinic business in California that would have an international reach and give the state dominance in the industry.

Karen Kaplan's story in the Times last week quoted CIRM President Alan Trounson as saying in 2010 about agency's goals.

“If we went 10 years and had no clinical treatments, it would be a failure. We need to demonstrate that we are starting a whole new medical revolution.”

The stem cell agency was created by voters in 2004 and funded with $3 billion in borrowed money. It will run out of funds for new grants in 2017.

Outsourcing-Pharma.com caught up with the plan this week in a story that said,

“The opportunity to run trails under the well-funded CIRM could be a boon for CROs (contract research organizations)....But the difficulties of handling the stem cells and gathering enough patients to enroll in a trial may prove daunting for whatever company tries to conduct the trials.”

The article also quoted CIRM spokesman Kevin McCormack as saying,

 “No one has reached out to us yet because the specific details of what we are looking for in the clinics have not yet been decided.”

That said, considerable information is available here, here, here, here and here.)

Also reporting on board approval of the Alpha clinic plan was GenNews.

California Stem Cell Agency Looking for New Home in Two Years

The California stem cell agency is located south of Market Street in San
 Francisco, close to the San Francisco Giants ballpark(upper right).  Since
 the agency has been there, the area has grown from seedy to gentrified. 

Some not-so-good news surfaced today in San Francisco involving the $3 billion California stem cell agency.

The news has little to do with its science efforts but everything to do with where it is located and its overhead expenses. The agency will be forced out of its free office space – 20,000 square feet – in two years. The free space was provided under an $18 million recruitment package and is worth at least $1 million a year, according to the agency's auditors.

The bad news is that the San Francisco office-space market is sizzling hot. Google, Yahoo, Microsoft and other technology firms are scrambling for space in Baghdad-by-the-Bay, as the city is sometimes known. According to a story this morning by James Temple in the San Francisco Chronicle, the firms are looking for a total of about 800,000 square feet and are prepared to pay well for it.

One nearly completed deal involving Yahoo would cost about $48 per square foot for a 10-year lease. If CIRM paid at that rate, it would have nearly $1 million in additional costs annually. However, leasing rates are expected to rise substantially in the next year or so. Also involved in a move would be the cost of parking, which could run about $360,000 a year.

The stem cell agency is already examining its options for new offices, including some sort of special deal with the City of San Francisco.

Former State Sen. Art Torres, onetime chairman of the state Democratic Party and co-vice chairman of CIRM, briefed agency directors on the matter at its meeting in May. He said,

“I met with the mayor of San Francisco(Ed Lee), who's a dear friend, and he encouraged us to be aware that he's very committed to helping us find some space in San Francisco. Whether it means tax credits or incentives to a potential landlord, we still have to work that out. Obviously we still have to work out what the space will be. But the fact that the mayor has indicated very explicitly that he wants to keep us in San Francisco, I think it will bode well for us down the road.

“The current owner of the property (Stockbridge Capital Partners) has not been happy that for ten years they've had to supply free rent to us. And what they didn't anticipate was having to provide for over $755,000 in operating costs, which they thought some donors would take are of. Those donors -- some of whom passed away and others who chose to give money to other institutions, UC San Francisco, in particular, to the stem cell lab, which was very much appreciated, I know, by UCSF – but at the end of the day, there's no room for negotiations with this current owner.”

CIRM Director Joan Samuelson asked Torres whether future rent would also be free. Torres, who is also president of San Francisco's Public Utilities Commission, replied,

“I would not work on that assumption. I would work on the assumption somewhere between a dollar and more, again, dependent upon what kind of tax incentives the City of San Francisco would provide. We're very fortunate that my son(Joaquin Torres) is the deputy mayor for economic development, so we also have him working on this as well.”

Samuelson replied,

“I'll ask more questions offline.”

California Stem Cell Official Duane Roth in Improving Condition

Duane Roth, the co-vice chairman of the California stem cell agency, is improving after he was hospitalized for treatment of a serious brain injury sustained in a bicycle accident Sunday in the mountains east of San Diego.

According to a report on Xconomy.com, Roth's brother, Ted, said yesterday, 

“Were certainly moving in the right direction. We're now looking at the recovery phase.”

The article by Bruce Bigelow said Roth has passed through the most critical period following surgery at the UC San Diego hospital.

Roth, the 63-year-old CEO of the San Diego technology organization, Connect, is in serious condition in a medically induced coma.

The governing board of the California stem cell agency yesterday took special note of Roth at its meeting and sent its best wishes to him and his family.  

Stem Cell Agency Board Concludes Meeting

The governing board of the California stem cell agency concluded its meeting today at 2:33 p.m. PDT. The California Stem Cell Report has also concluded its coverage today's session.

CIRM Posts Alpha Clinic Press Release

The California stem cell agency this afternoon posted its press release on the $70 million Alpha stem cell clinic plan. Here is where it can be found.

Another $23 Million to Recruit Star Stem Cell Scientists to California

California's $46 million effort to lure stem cell research stars to the Golden State was expanded today by another $23 million.

Directors of the stem cell agency approved the funds on a 14-4-1 vote. CIRM directors Jeff Sheehy and Francisco Prieto were among those opposing the move. Prieto declared,

“We are coming up against finite resources. We have better ways to spend our money."

 Sheehy said that CIRM is contributing to inflation in stem cell science with its lucrative recruitment grants. 

Those supporting the expansion said that the grants have had a great impact on the field, not only bringing in individual scientists, but accompanying researchers in their labs along with grants from other sources.

The additional funds will go to institutions that have not already benefited from one of the earlier grants in the program. Up to four awards are expected to be made.

The CIRM staff proposal on the plan said,

“A number of California institutions have not yet been able to secure a confirmed Research Leadership award but would benefit greatly from the recruitment of emerging or established leaders in stem cell biology. Participation in the CIRM program could bring additional, exceptional researchers to California, strengthen and synergize with other efforts to build up local sustained research communities in stem cell biology and medicine and provide ongoing leadership at the cutting edge of California regenerative medicine.”

All of the California institutions involved with the winning researchers have representatives on the governing board of the stem cell agency. They are not allowed, however, to vote on grants to their institutions or researchers -- only on proposals such as today's $23 expansion.

Applications are due in January with final approval scheduled for next May. The program is not open to businesses.

$35 Million Research Grant Round to Remove Stem Cell Roadblocks

Directors of the California stem cell agency today approved a $35 million program aimed at removing bottlenecks to pushing stem cell therapies into the marketplace.

The plan would provide grants of up$1.2 million for about 20 awards with competition open to both business and non-profit institutions. Pre-applications are expected to be due in October with approval next summer.

California Stem Cell Agency Launches $70 Million Alpha Stem Cell Clinic Project

The California stem cell agency today approved a $70 million plan to create a network of “Alpha” stem cell clinics that is aimed at making the Golden State one of the leading purveyors and developers of stem cell therapies in the world.

The 29-member governing board of the California Institute for Regenerative Medicine (CIRM)adopted the plan on a 19-1 vote. The negative vote came from Joan Samuelson, who questioned whether the plan was premature and whether existing scientific research justified development of the clinics. 

Sherry Lansing, a patient advocate board member and former head of a Hollywood studio, said the proposal is “one of the most exciting proposals that we have ever had in front of us.” She said it was the “beginning of this dream coming true.”

Under the far-reaching proposal, which CIRM President Alan Trounson has been promoting for two years, the agency will finance five stem cell clinics at established institutions in California with grants of up to $11 million. Another $15 million will be allotted for a stem cell information and coordination center. Major matching contributions will be expected from award winners over the five-year terms of the grants.

The effort is aimed at drawing in clinical trials and patients from the around the world and creating a central bank of knowledge, know-how and regulatory expertise. It will also guide efforts to build profits into stem cell therapies and to develop strategies to attract investors and philanthropists. (For more information on the plan, see here, here, here, here and here.)

Trounson said in a statement,

“These clinics have the potential to revolutionize how we deliver stem cell therapies to patients. Stem cell therapies are a completely new way of treating diseases and disorders so we need a completely new way of delivering those in a safe and effective manner. These clinics will help us do just that and the clinical trials carried out in this network will fulfill the agency’s promise of bringing new therapies to patients who need them.” 

The journal Nature Medicine has reported that the Alpha clinics would be the first-ever “clinical trials network focused around a broad therapeutic platform.”

The CIRM board heard no negative comment on the plan other than the remarks by Samuelson. . However, not everyone sees a need for it. Mahendra Rao, director of the Center for Regenerative Medicine at the National Institutes of Health(NIH) , says its surveys of researchers have not shown a demand for such centers. In May, a researcher at institution that likely would be an applicant filed a blistering, anonymous comment on the California Stem Cell Report, describing it as a "boondoggle" and "irresponsible." The scientist said,

“Another boondoggle for some medical schools but made to order for private operators like for-profit cancer, dialysis, and laser eye specialty clinics that do one procedure.  I can see each of the medical schools gifted with one as they each were gifted with about 25 million dollars for stem cell institute buildings.”

The researcher continued,

“The NIH at various times has tried to organize clinical trials groups with infrastructure, like quick reaction forces, ready to gear up for a new trial at the drop of a hat. They mainly did nothing but suck money, kept staff employed, because there are generally few drugs ready for early human trials and each treatment that is brought along requires a unique contract, ethics reviews, and different facilities, equipment and staff than planned for.  The latest incarnation are CTSAs or CTSIs, clinical and translational science centers funded by the federal NIH that most if not all California medical schools already have.”

The RFA for the proposal is expected to go out in October and approval of funding coming one year from now. Here is the link to today's CIRM press release on the plan. 

Alpha Clinic Applicant Qualifications

The chart above outlines the criteria for applicants for grants in the $70 million Alpha clinic plan.

Stem Cell Directors Take Up $70 Million Alpha Clinic Proposal

Directors of the California stem cell agency have begun discussion of the $70 million Alpha stem cell clinic plan. The staff is laying out the proposal at this hour.

California Stem Cell Agency Has $577 Million Remaining for New Research Awards Out of $3 Billion

The California stem cell agency has only $577 million left for future research awards over the next four years, its directors were told this morning.

The figure was reported by CIRM staff this morning as part of its regular briefing on the financial status of the research effort, which began work in 2004 with $3 billion that the state is borrowing through issuance of state bonds. The method of funding will cost roughly $3 billion more in interest costs.

The agency expects to run out of funds for new grants late in 2017 and is considering ways to raise cash for future operations..

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Stem Cell Directors Begin Meeting with Report from Alan Trounson

Today's meeting of the governing board of the California stem cell agency began at 9:35 a.m. today with a a report from chairman J.T. Thomas, updating the board on his activities. CIRM President Alan Trounson has now begun his report dealing with stem cell research publications over the last few months.

California Stem Cell Board Meeting Delayed

The business portion of today's meeting of the governing board of the California stem cell agency was scheduled to begin at 9 a.m. PDT. However, that session has been delayed as the board hears a presentation on MS. The business session may begin about 15 minutes or so.

Coming Up: Live Coverage of Today's California Stem Cell Meeting

The California Stem Cell Report will provide live, wall-to-wall coverage of today's meeting of the governing board of the $3 billion California stem cell agency.

At the top of the agenda is a $70 million proposal aimed at creating a string of Alpha stem cell clinics in California that would serve as a foundation for the state's stem cell business. Also on tap are other proposed grant programs, including a $23 million expansion of a researcher recruitment effort and a $35 million round aimed at removing roadblocks to turning research into cures.

Stories will be filed as warranted throughout the day based on the Internet audiocast of the proceedings. 

Interested parties can also listen in on the meeting via the Internet. Instructions can be found on the agenda.  

Stem Cell Lines and Paid-for Eggs: Stem Cell Agency Delays Action on Easing Restrictions

A key panel of the California stem cell agency today balked at approving a plan to ease restrictions on using stem cell lines derived from women who were paid for their eggs.

The proposal had been scheduled to be taken up tomorrow by the governing board of the $3 billion agency, but the board's standards working group delayed action.

In response to a question, Kevin McCormack, a spokesman for the agency, said in an email,

“It was felt that more discussion was needed before moving to a vote so another meeting is going to be scheduled.”

In 2006, the CIRM governing board approved regulations that banned the use of CIRM funds for stem cells lines derived using compensation. That rule would be modified under today's plan, which would permit the CIRM governing board to approve the use of such lines following a staff study evaluating scientific and ethical issues. Their use would be allowed if the lines would “advance CIRM's mission.”

The delay came after four organizations, including the Center for Genetics and Society in Berkeley, argued that the plan is vague and did not adequately address safety issues.

The four-page statement by the groups said that the plan does not appear to have met “numerous concerns” raised in 2009 in a document co-authored by the CIRM staff. Those concerns include long-term risk and ethical issues.

Under the proposal, the groups said that the agency governing board

“...will decide whether to approve a grantee’s request to use a stem cell line created with paid-for eggs on the basis of whether doing so 'will advance CIRM’s mission.' This criterion is much too vague, and doesn’t include consideration of the health or welfare of the women who undergo egg retrieval. Protecting the well-being of women providing eggs is not even mentioned (though perhaps it could be considered as an element of the fifth of five 'factors to be considered by the ICOC(the agency board),' 'whether the donation…was consistent with `best practices’ at the time of donation').”

The standards group also heard from a UCLA researcher who argued on behalf of the change. Kathrin Plath said she and her colleagues wanted to use a paid-for stem cell line from the Oregon experiment that cloned human stem cells.

(An earlier version of this item said the change under consideration would ease restrictions on "purchasing" stem cell lines. The word "purchasing" was changed to "using.")

Here is the text of the statement by the four organizations.

CGS July 2013 Letter Opposing Changes in CIRM Cell Line-Egg Regulations

$70 Million Alpha Stem Cell Clinic Project Garners Mainstream Media Attention

California's $70 million plan for a chain of “Alpha” stem cell clinics today received its first major attention from the mainstream media.

The story came in the state's largest circulation newspaper, appearing this morning on the home page of the website of Los Angeles Times.

The Alpha project would create five clinics around the state and a coordination/information center under a concept that comes before the governing board of the state's $3 billion stem cell agency at its meeting tomorrow in Burlingame, Ca. Funds could be awarded as early as a year from now. (For more information, see here and here.)

Reporter Eryn Brown quoted Natalie DeWitt, special projects officer for CIRM, as the stem cell agency is known, and Maria Millan, a CIRM medical officer. Brown wrote,

“Clinics to conduct trials of stem cell therapies have different needs than clinics designed to deliver conventional therapies, DeWitt and Millan said. They need special facilities for handling the cells safely, as well as imaging equipment to track the cells once they're delivered into a patient’s body.  Some of this infrastructure already exists, but other parts of it still need to be perfected.  Establishing clinics to house multiple trials might create the critical mass needed to get the infrastructure in place, they said....

"Additionally, they said, CIRM hopes that such collaboration would encourage stem cell companies to share information -- speeding their own work and also helping out policymakers and insurers who are trying to figure out how they'll pay for stem cell therapies in the future.”

The Times quoted the California Stem Cell Report as saying last week,

 “The Alpha clinics are aimed at creation of a sturdy foundation for the stem cell industry in California, capitalizing on the burgeoning, international lure of stem cell treatments.”

The proposal envisions Alpha stem cell clinics at major, established institutions around the state. It is possible that two could be located in the Los Angeles area at institutions such as UCLA, USC, Cedars-Sinai or the City of Hope, all of which have representatives on the stem cell agency's governing board. Other likely locations are in the San Francisco Bay area and San Diego, again at facilities such as Stanford, UC San Francisco and UC San Diego that have representation on the agency board.

Institutions competing for the grants, including businesses, will be subject to closed-door. peer review prior to final action by the full governing board.  

UCLA Researcher Calls for Easing of Restrictions on Stem Cell Lines Derived from Eggs From Paid Providers

A UCLA researcher has spoken out in support of a proposal to allow use of California stem cell agency funds to purchase stem cell lines derived from eggs provided by women who have been paid for the service.

Kathrin Plath, an associate professor, said in a letter to the agency that she and her colleagues would like to use a line from the Oregon SCNT experiment by Shoukhrat Mitalipov in which human stem cells were cloned. Currently agency funds cannot be used for that purpose as a result of regulations that are the extension of a state law that bars use of agency funds for payment for eggs.

The agency's standards group meets later today to consider changing those regulations. The proposal will then go before the full board tomorrow.

Plath, who has received $5 million from CIRM, said,

“In my lab, we are ... interested in understanding what happens to the somatically silenced X chromosome when differentiated cells are reprogrammed by SCNT. The key question is: are these SCNT-ESCs more similar to iPSCs or fertilization-derived ESCs with respect to the epigenetic state of the X chromosome. Furthermore, it has been shown in mouse reprogramming that the active X chromosome becomes deregulated during SCNT-based reprogramming, and we would like to address this problem in the human system as well.

“We believe that the comparison of the epigenetic states between fertilization-derived ESCs, SCNT-ESCs and human iPSCs is important for a better characterization of these cells and understanding of their epigenetic nature.”

Co-vice Chairman of California Stem Cell Agency Remains in Critical Condition

Duane Roth, the co-vice chairman of the California stem cell agency, remains in critical condition with brain injuries following a weekend bicycle accident.

According to a report by Bradley Fikes in the San Diego U-T, Roth's family is preparing for a recovery that will take weeks. Roth is currently in a medically induced coma.

Roth's wife, Renee, released a statement through the UC San Diego Medical Center that said,

“His condition is currently listed as critical. Duane is in the great hands of the university’s doctors and nurses and we are very happy with the outstanding care he is receiving. We do ask that our privacy be respected as Duane heals. We will be in touch to share more.”

The accident occurred Sunday in the mountains east of San Diego when Roth hit a rocky embankment or outcropping and his helmet split open on the impact.

Roth is CEO of Connect, a technology business group. He has served on the stem cell agency board since 2006.

(An earlier version of this item incorrectly described Roth as CEO of Current instead of Connect.)

Correct Link to CIRM Report on Business Aspects of Alpha Clinics

The "Inside Alpha Clinics' Business" item yesterday had a bad link to the CIRM report on the proposal. Here is the correct link.  A thank you to Kevin McCormack of CIRM for pointing this out.

How to Stay On Top of California's Stem Cell Research Cash Cow -- At Least This Week

Persons interested in listening to Thursday's hearing on California's $70 million “Alpha” stem clinic proposal can do so by two different methods via the Internet.

In addition to Internet access, the public can participate in the meeting in Burlingame, Ca., and also at two teleconference locations in Los Angeles. The Internet access does not allow two-way participation.

If you have money in the game, our advice is to attend the meeting in Burlingame. Only a handful of business people and scientists attend the agency's governing board meetings, but they seem to benefit from the information and discussion as well as from the ability to meet face-to-face with CIRM officials and board members.

Of particular interest may be a scheduled discussion of CIRM's goals and direction this year and next along with other proposals that will funnel $128 million to researchers in California, including the clinic plan.

If stem cell agency fans do not have time to sit through the day-long proceedings, they can follow developments via the California Stem Cell Report, which will be covering the meeting live via the Internet and filing stories as warranted.

Instructions for Internet access can be found on the meeting agenda. One method is a relatively straightforward audiocast. The other involves WebEx, which may involve some configuration of your computer. However, the method also shows the Power Point presentations that are used during the meeting and are otherwise not available in advance or on the day of the meeting. They often contain valuable information that informs the discussions of the board of the $3 billion agency.

In either case, you will want to check out the procedures in advance if you have not used them before. The dial-in number for the audiocast is 866-320-4708 with the password of 297273. Here is the link for the WebEx connection: https://cirm.webex.com/cirm/onstage/g.php?d=286602980&t=a&EA=acheung%40c...

As for the teleconference locations, the specific addresses are on the agenda. But you should ask in advance for more information on the Cedars-Sinai location to be sure you can find it on the day of the meeting. The Cedars complex is very large.

Tomorrow the agency's standards group will take up a proposal to allow CIRM grant recipients to purchasestem cell lines derived from eggs from paid donors. Unfortunately, that session is not available either by Internet or teleconference, but only in San Francisco. The proposal is expected to move forward to the full board on Thursday but it could be modified.

Co-vice Chairman of California Stem Cell Agency Hospitalized with Brain Injury

Duane Roth
Connect Photo

Duane Roth, co-vice chairman of the $3 billion California stem cell agency, has been hospitalized in a medically induced coma following a bicycle accident yesterday in the mountains east of San Diego.

Roth hit a rock embankment and broke his helmet, according to news reports. He was flown to the UC San Diego Medical Center, where he underwent surgery to remove a piece of his skull to relieve pressure on his brain, according to an account by Bruce Bigelow on Xconomy.

Bradley Fikes of the San Diego U-T reported that Roth is expected to kept in a coma for at least several days until his condition improves. 

J.T. Thomas, chairman of the stem cell agency, said in a statement,

“Our thoughts, prayers and best wishes for a full recovery are with Duane and his family.”

Roth, CEO of Connect, an organization that supports the technology industry, has served on the governing board of the $3 billion California stem cell agency since 2006. He has been a strong voice for business at the agency and likely would have been active participant in board discussion this Thursday of one of the agency's major initiatives that will benefit business – a $70 million stem cell clinic plan.

Inside the Business in California's $70 Million Alpha Stem Cell Clinic Proposal

Want to know more about the business aspects of a $70 million proposal to create a chain of “Alpha” stem cell clinics in California?

More details can be found in a report from the California stem cell agency titled “Alpha Stem Cell Clinics: Delivering a New Kind of Medicine.”

Among other things, the 23-page report discusses how the plan would guide efforts to build profits into stem cell therapies and to develop strategies to attract investors and philanthropists. Not all of the business questions are answered. However, the report does paint a more complete picture of how the clinics would lead to actual production and marketing of a therapy. Overall, the proposal is aimed at creating a sturdy foundation for the stem cell industry in California, one that surpasses anything found elsewhere in the world.

The stem cell agency's governing board is set to move forward on the Alpha clinics at its meeting on Thursday. This week's action involves approval of the concept with an RFA coming in October. Awards to six recipients could be made as early as July of next year.

The business aspects of the proposal are critical because without a clear pathway to profits no stem cell therapies will be available on anything approaching a significant scale. 

CIRM President Alan Trounson first advanced the Alpha concept two years ago. Last fall the $3 billion agency brought together 70 folks from the stem cell world to ponder the idea and to make suggestions. CIRM's report on the discussions at the workshop served as the basis for the proposal to be considered by its governing board this week. Here are excerpts from the report.

“Workshop participants...emphasized the need for engaged experts who can consider and implement strategies for reimbursement (i.e.generating profits) for stem cell therapies, and to gather evidence for their value and effectiveness that will be important for coverage by payers. Having clear pathways in place will help in building financially sustainable clinics as stem cell therapies gain approval and are implemented as standard medical practice. The Alpha Coordinating Center (funded at $15 million) would work with Accountable Care Organizations (funding gouprs that tie performance to payment) and multi-stakeholder collaborations to generate evidence for informing coverage and payment policies.”

“Incentivizing company sponsors of clinical trials to participate in data sharing will be challenging, and it will be important to protect their interests as appropriate, by respecting confidentiality of proprietary information, while at the same time requiring an appropriate degree of data sharing to advance the mission of the Alpha Stem Cell Clinics Network(five clinics funded at $11 million each). It is interesting to note that at this time, the landscape of disclosing clinical trial results is in flux, and there is increasing international pressure for companies to disclose information on safety and efficacy to the research and medical communities.”

“Alpha Stem Cell Clinics will create unique models for assessing the financial viability of stem cell therapies in a clinical setting. Cost and revenue data may serve as the basis for developing reimbursement options among the public and private sector as therapies accumulate. Identification of reimbursement options can reduce investor uncertainties and encourage further investment by pharmaceutical companies, investors, funding agencies and philanthropists.
“The elements of the Alpha Stem Cell Clinics Network, namely the clinical sites and central coordinating center, will interact with external entities to generate revenue streams that would ultimately ensure financial sustainability independent of CIRM funding (Figure 3).”

“The Coordinating and Information Management Center could develop a fee for service model for its consulting services, and operate like a CRO. Sponsors could choose whether to use the consulting services and whether to use the Alpha Stem Cell Clinics Sites. The Alpha Stem Cell Clinics Sites would generate operating revenue by running the clinical trials and could perhaps support the Center with revenues, as the volume of trials grows and the Network builds steam.

“In a second model, clinical trial sponsors would all contract with the Coordinating and Information Management Center, which would then set prices according to services needed. From this revenue, the Center would pay the Alpha Stem Cell Clinics Sites for costs associated with the clinical trials.

“By the time the CIRM seed money is depleted, the clinics should have established a strong track record and brand for excellence in conducting clinical trials in stem cell therapies, which would attract more clinical trial sponsors, including companies. To have the largest possible impact on healthcare, it will be desirable to increase industry involvement for cell therapies. However engaging corporate involvement could be challenging, given that many cell therapies are not considered patentable, and many of the ongoing trials are conducted in academic settings. It was suggested that the Alpha Stem Cell Clinics Network align as much as possible with corporate “mentality” to maximize corporate participation.

“Overall, the Alpha Stem Cell Clinics Network will help companies increase their 'bandwidth' and run stem cell therapy clinical trials more effectively and successfully. Many companies will already have sufficient in-house expertise for administration and regulatory guidance, and may not need to engage the Center’s consulting services. They would be attracted to the Alpha Stem Cell Clinics Network because of advantages for clinical trials such as benefiting from the collective databases, know-how and experience of the 'brain trust,' positive interactions and lines of communication with the FDA and regulatory experts, positive branding, quality control, accreditation for their trials, access to patient registries for outreach and patient recruitment and, through its clinical trial management resources, help with enrollment.

“Workshop participants also emphasized that attaining reimbursement for stem cell therapies will be necessary for delivering approved therapies into medical practice. It is likely that the business models for at least some of the clinics will resemble bone marrow/HSC transplant clinics, where for stem cell therapies that are proven superior to the standard of care, the clinic will directly receive reimbursement from insurers for the procedure and follow-up care.

“Reaching this point will require advance advocacy and preparation, and the engagement with the CMS at an early stage of product development. It was recommended that the Alpha Stem Cell Clinics Coordinating and Information Management Center should employ experts in reimbursement methodologies to facilitate entry of approved products into healthcare delivery. One workshop participant, Jeff Sheehy(a stem cell agency board member), suggested that early engagement with Accountable Care Organizations (ACOs) could be helpful, given that stem cell therapies offer the promise of cures, which in the long run will offer high value and effectiveness, albeit with ' high front end costs and the necessity of developing the appropriate infrastructure for delivering complex cell based procedures.' Consortiums such as the Green Park Collaborative are being established to develop methodological standards to demonstrate the effectiveness and value of new technologies. These data could be used to inform healthcare payers of the overall benefits of cell therapies and their long-term value, as compared with existing therapies."

“In 2016 and beyond, CIRM will initiate the 'Delivery' phase, in which priorities will be 'facilitating commercialization (and non-commercial adoption, where appropriate) of therapies, advancing therapies to patients, and enabling business models for stem cell-based therapies.' Connecting this network to other networks and centers with a similar mission outside of California will further accelerate the development and delivery of stem cell therapies, through the global exchange of information and expertise. International testing of experimental therapies developed in California will be essential for ensuring their sustainability, as regulatory approval and widespread delivery of these products in major markets throughout the world will ensure commercial viability and sustainability of companies and universities that produce them, which will in turn help deliver returns to investors. Ultimately, establishing a solid foundation for investor engagement will ensure the viability of stem cell therapies as they move into clinical practice.'”