$400 Million Deal Leaves California-backed HIV Trial Hanging

A California-financed clinical trial testing a promising stem cell therapy for HIV is up in the air this morning following the purchase of its California backer by an Australian corporation in what could be a more than $400 million deal.

The firm being acquired is Calimmune, which has an $8.3 million award from the California Institute of Regenerative Medicine (CIRM), as the state stem cell agency is formally known. 

CSL Behring announced this morning that it is buying Calimmune for $91 million and as much as $325 million more depending on whether its research meets success milestones. 

However, CSL is not interested in the early phase Calimmune trial, which began in 2013 and was scheduled to be completed by this October. John Carroll, writing on the Endpoint News web site, reported this morning, 

"CSL, though, plans to let go of control of Calimmune’s lead, clinical program on HIV as soon as possible. In a follow-up to a query, a company spokesperson replied:
'We are currently evaluating our options for developing this pipeline candidate, which could include licensing or partnering. Given our areas of focus, it is unlikely that we will develop this candidate on our own.'"

The California stem cell agency had no immediate comment on the potential impact of the deal. In response to a query this morning, Ronald Mitsuyasyu of UCLA, listed by the NIH as the principal investigator, said he had no comment.

The latest CIRM progress report on the research indicated that the trial had been advancing. A notice on the NIH clinical trials web site said, however, that it is no longer recruiting patients. The CIRM progress report said,

"The objective of the Cal-1 therapy is to increase the number of protected cells in the body of an individual infected with HIV to the point where the virus is incapable of causing harm. This would potentially reduce or eliminate the need for a lifetime of antiretroviral therapy."

Calimmune was co-founded by David Baltimore, a Nobel Prize winner and former member of the governing board of the state stem cell agency.

California Stem Cell Agency's First Royalty Payment: Beginning of a $1 Billion Flood? No One is Saying

The check is in the mail for California's $3 billion stem cell research effort. But it is something of a secret.

So reports Ron Leuty of the San Francisco Business Times concerning the expectation that the agency, formally known as the California Institute for Regenerative Medicine (CIRM), would generate $1 billion in royalties. In an article online yesterday afternoon, Leuty said,

"We are expecting the first check to be delivered to the state very soon," CIRM spokesman Kevin McCormack said in an email.

"Yet even as a public state agency, CIRM officials are holding tight to key information about the first royalty check: How large (or small) is the check? When will the check actually be forwarded to the general fund? And from which CIRM-funded project did it spring?

"'The royalty check is something that is still being worked out so it’s premature to say anything at the moment,' McCormack said in a followup email. 'Sorry to sound so secretive but it is a big deal, the first of what we hope will be many such repayments for the state’s investment.'"

During the ballot initiative campaign that led voters in 2004 to create the stem cell agency, supporters said that the agency's research could generate up to $1.1 billion in royalties. None have yet surfaced. And at least one description of the royalty promise has called it a "cynical ruse."

California Stem Cell Agency Pushes $23 Million into Clinical Trials for Leukemia, Bone Marrow Transplants

California's 12-year-old stem cell research program this morning popped for $23 million to help finance clinical trials to "confuse and kill" leukemia cells and to develop a better alternative to bone marrow transplants.

The two awards are part of the stem cell agency's effort to produce a widely available stem cell therapy before its money for new awards runs out in less than three years. So far, the agency, known formally as the California Institute for Regenerative Medicine (CIRM), has not been successful in producing such a therapy.

CIRM has now funded 35 clinical trials, which are the final stage of testing before a therapy is approved by the federal government for widespread use.  Thirty-one of the trials are still active.

Today's largest award, $18.3 million, will go to Thomas Kipps of UC San Diego. An additional Oncternal Therapeutics, also of San Diego.  Kipps is a scientific advisor to the firm. The other award, $5 million,  goes to Angiocrine Bioscience, Inc., of San Diego. The company will add $2.7 million to the effort.

Thomas Kipps, Oncternal photo

$13.3 million will be provided by

The Kipps project targets the most common form of lymphoma, chronic lymphocytic leukemia (CLL) the agency said in a news release. The agency said Kipps is "using a combination strategy to kill the cancer stem cells that help CLL – and other cancers – survive traditional therapies, such as chemotherapy, and cause a relapse."

Every year, about 20,000 people nationwide are diagnosed with CLL.

The Angiocrine research is aimed at increasing the supply of cord blood for bone marrow transplants, when no matched donor is available.  Its product is called AB-110.

Maria Millan, interim CEO of the stem cell agency, said,

 "This (Phase 1) clinical trial will be the first step in determining if AB-110 is able to provide a way to improve the results of cord blood transplants."

Today's award appears to advance work previously funded by CIRM at a cost of $4 million.  Paul Finnegan was listed as the principal investigator on that work.

Angiocrine's key technology  is licensed from Weill Cornell Medical College and was invented and developed by Shahin Rafii.

The awards were approved in less than 10 minutes this morning with no discussion by the CIRM governing board, which simply ratifies the decisions of its out-of-state, grant reviewers, who considered the applications in July behind closed doors.

A summary of the reviewers' comments can be found here for Kipps' application, CLIN2 -101, and here for Angiocrine's, CLIN2-10386. Information on the Kipps trial can be found here on clinicaltrials.gov. Catriona Jamieson of UC San Diego is listed as the principal investigator on the Kipps' award.

The Angiocrine trial was not yet listed on clinicaltrials.gov at the time of this writing.

$185 Million California Rumpus over Researcher Raiding

To some outside the California scientific and academic community, it might seem like a cat fight replete with allegations of  a "law-of-the-jungle mindset," loyalty oaths, "petty academic politics" and -- literally -- paper clip theft.

All of which is mentioned in court filings in the squabble, if you want to call it that. But this is not minor stuff. It involves a $185 million, 2015 lawsuit by the University of California (UC) against the University of Southern California (USC), a private school in Los Angeles. Basically, UC wants the money from USC because UC thinks one of its high-powered scientists was hijacked by USC.

Some of the details emerged today in the Los Angeles Times, which wrote about the case in connection with the Carmen Puliafito affair. He is the former dean of the USC school of medicine as well as being a member of the governing board of the $3 billion California stem cell agency, a position he no longer holds.

The headline on the story said,

"USC dean drug scandal could take costly toll on school's legal battle with UC system"

Puliafito was dean of the medical school when it hired away from UC San Diego a star Alzheimer's disease researcher, Paul Aisen, who had the potential of bringing $340 million in research grants to USC. In addition to the prestige of having Aisen on campus, the school would receive a significant percentage of the grants as part of overhead costs.

The Times reported that during Puliafito's eight years at USC, he spearheaded an effort that lured more than 70 "transformative faculty" from UC schools, Stanford, Harvard and other prestigious rivals.

One was USC's longtime athletic rival, UCLA. According to the Times, the successful wooing of Arthur Toga and Paul Thompson, two well-funded neurology researchers, in 2013 "outraged" UCLA, which complained to federal regulators.

The Times wrote,

"It was not unusual for professors to move to other institutions, often with the first university cooperating in the transfer of grant funding to the new school. But in UC’s view, USC had acted beyond accepted norms by targeting academics based on grant funding and strategizing secretly with those researchers while they were still employed by UC about moving grants to USC. The schools reached a confidential settlement requiring USC to pay UCLA more than $2 million, according to a copy of the agreement obtained through a public records request."

The lawsuit is now in San Diego Superior Court. No trial date has been set.

Thinking About Its Demise: California Stem Cell Agency Launches Examination of Alternatives

California's $3 billion stem cell research agency, which is facing its financial demise in a few short years, has formed a team of its directors to tackle transition planning and examine possible alternatives, including ones that would extend its life.

The first meeting of the group of directors is tentatively scheduled for Sept. 18. Jonathan Thomas, chairman of the governing board of the California Institute for Regenerative Medicine(CIRM), as the agency is formally known, said earlier this summer:

"The legislature has asked that we put together and start thinking about a transition plan, which can contemplate a variety of factors."

In response to a question last week, a spokesman for the agency, Kevin McCormack, said that a notice with more details would be posted 10 days prior to the meeting.

At a meeting in June, Thomas laid out the need for the transition team. He said all options are on the table including asking the legislature for cash or to place a measure on the ballot for more bond funding.

The agency's only real source of money is state bonds, authorized by voters in 2004. It has roughly $600 million left. The agency has projected it will run out of cash for new awards in mid 2020, although that could vary, depending on whether it slows down the pace of awards.

Several directors at the board meeting in June expressed a "sense of urgency" about dealing with the fate of the agency. CIRM Director Jeff Sheehy, a member of the San Francisco board of supervisors and an HIV/AIDS patient advocate, voiced concern about the uncertain nature of the agency's future.

Sheehy said,

"It seems to me that we will be talking about a substantial scaling back of the organization in 2020....We've kind of created this expectation that we were going to go to 2018 and come back with new money."

Sheehy referred to talk that a new bond initiative might be launched in 2018, a move that the board's former chairman, Robert Klein, has publicly advanced. Sheehy said, however, that he spoke with Klein, who told him that he was now considering 2020 instead.  Klein's method would require the gathering of hundreds of thousands of valid voter signatures to place the proposal on the ballot and would bypass the legislature. 

The year 2020 includes a presidential election, which has higher voter turnout and generally is considered a better time to win approval of bond measures. Presumably, the agency might be able to secure extra funding to span any financial gap or, alternatively, lower the frequency of awards to stretch out the cash. 

The members of the transition group are Thomas, Sheehy, Art Torres, Steve Juelsgaard, Joe Panetta, Kristiina Vuori, Linda Malkas, Diane Winokur, Shlomo Melmed, Al Rowlett and Judy Gasson. Short bios on each of them can be found via this page. 

The California Stem Cell Report will carry an item with the date and location of the September meeting when it becomes available. 

Crossing the Two Million Mark: A Dozen Years of Readership on the California Stem Cell Report

Google's page view count for the California Stem Cell
Report as of 11:01 a.m. today 

The California Stem Cell Report this morning hit a readership milestone -- two million page views.

The count was recorded only about 18 months after chalking up one million page views, a number that took 10 years to reach. The blog began publication in January 2005, just as the Golden State's stem cell agency was getting underway. Since then 4,492 items have appeared. 

For those not familiar with Internet terms, a page view is recorded by Google each time a person opens his or her Internet browser on a particular page. It is an industry standard that it is used to help define readership, something akin to circulation numbers for newspaper.

The California Stem Cell Report (CSCR) is somewhat unique. It is the only independent web site devoted to the $3 billion ($6 billion if you count interest costs) California Institute for Regenerative Medicine (CIRM), as the agency is formally known. The report was created to dig deeply into the agency, focusing largely on public policy and economic issues and recording the agency's activities.

David Jensen, Mazatlan, Mexico

The blog is published and written by David Jensen, a retired editor and journalist who spent most of his career at The Sacramento Bee, but who also worked seven years for United Press International and two years as a press aide to Gov. Jerry Brown (1974-76). Over the last 12 years, Jensen has covered the agency while living on a sailboat as it navigated the west coasts of Mexico and Central America. He traveled back to California from time to time for meetings of the agency and interviews with its leaders.

As reported in 2015, the one-million figure then and the two-million figure now are both large and small. The figures are picayune compared to the readership for major news sites. But they are relatively large given the size of the potential audience.

The report's long-time estimate has been that only about 3,000 to 4,000 persons worldwide are deeply interested in stem cell matters. When Randy Mills, former president of the stem cell agency, heard the estimate last year, he said he thought the number was even less. 

The readership for the blog is mostly found in the United States, according to Google's statistics. But a significant component comes from Europe as well. Readers range from individuals seeking information about stem cell therapies to folks at such places as the National Institutes of Health, major research institutions in California and elsewhere including Harvard, Cedars-Sinai, Sanford-Burnham, Stanford, UC San Francisco, UC Irvine. 

The most highly read article during the past 12 years deals with the cost of a stem cell therapy. It has had 23,498 page views, far beyond the second best-read piece that counted 2,754 page views. Presumably the "cost" piece drew considerable attention from persons considering a stem cell treatment. The No. 2 article dealt with a call by the state controller for online posting of the economic interests of members of the CIRM governing board.

The blog has also served as a starting point or helpful reference for many journalists looking into the agency and has been described as "indispensable" by Los Angeles Times columnist Michael Hiltzik. 

The blog began shortly after the passage of the 2004 ballot initiative that created the agency and provided the bond funding for the program. CIRM is unique in California history and is one of the largest, if not the largest, single source in the world of funding for stem cell research, especially involving human embryonic stem cells. CIRM has had a major impact on stem cell science in California and has helped to sustain research interest in the field at a time when others were wary of involvement.

Its birth was unusual, probably the only scientific research effort ever triggered by the use of petitions signed by more than one million voters and then placed on the ballot for further voter approval. It additionally represents a rare confluence of big science, big business, big academia, big government, big politics, religion, morality and life and death. 

The blog itself is financed personally by Jensen, who has no financial ties to biotech, academia or industry or the agency. Google does place ads on the site, which generate about $150 every six months or so based on the number of people who click on an ad. A crowd funding pitch awhile back was unsuccessful. However, the blog does spin off occasional freelance pieces to The Sacramento Bee and Capitol Weekly. 

The Demise of California's Stem Cell Agency: Gone in Three Years or Perhaps Some Other Date?

The $3 billion California stem cell agency goes out of business in less than three years: True or false?

For those of you seeking certainty, the "definite" answer is maybe. The agency has been saying for a couple of years now that it will run out of funds for new awards by roughly the middle of 2020. However, prior to that estimate, the death knell for the research program was forecast for much earlier. In fact, it would have come this year, 2017.

The difference is the result of different projections at different times by different leadership of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known. When Alan Trounson, an internationally known scientist, was president of CIRM, it produced  the 2017 figure. When Randy Mills, a longtime biotech business executive, took over three years ago, he redid the estimate and came up with 2020.

The difference is based on assumptions. And they involve the "burn rate" of CIRM's cash. With the support of the board, Mills reduced the rate at which money would be awarded thus extending its effective life.

Steve Juelsgaard, Iowa State photo

CIRM board member Steve Juelsgaard, a former top executive with Genentech, in June briefly highlighted the vagaries of CIRM's possible demise. According to the transcript of the board meeting, he told CIRM directors:

"So there are ultimately three possibilities, right? The one that's most looming is the ending of current funding at some point in the future. Right now we're targeting 2020..... However, we're the ones who can determine when that funding will end. If we think it's better to run past 2020 to give ourselves more runway, then we should formulate a plan that would allow us to go to 2021 or even a little bit beyond that to see what happens. So nothing is cast in stone yet (concerning) exactly when we're going to end up with our funding.

"The second one would be the opportunity to have a new bond measure passed, let's say, in 2020.  So what's the plan? If that were to happen, then the third one is going back to the old private-public partner approach which, as I understand it, is still on the table.

"So, in any event, I think what we need are side-by-side plans that look at all of those, that as time goes on and it becomes clear which of those is likely to occur, we can just put it into play. The one that's most likely to be put into play is the one that ends the organization. And then if one of the others comes in after the fact, we can then plug it in and continue on."

Later this week, the California Stem Cell Report will look at a new governing board committee that is examining the various end-of-life, CIRM scenarios. As an aside, during the 2004 ballot campaign that created CIRM, it was widely and erroneously believed that CIRM would have only a 10-year life. The 10-year figure dealt with the agency's bonding authority from the date when bonds were first issued.

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

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

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

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

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

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

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

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

Why the focus on the unregulated treatments? Knoepfler told Science:

"'They were just saying, ‘Screw the rules, we're just going to set up shop and put up a website and start injecting people with stem cells....I saw that as a threat, first to patients, but to the field as well.'"  

California Stem Cell CEO Search: Lid Still on Until Late September

The  governing board of the $3 billion California stem cell agency, which is seeking a new president, does not plan to have another meeting of its presidential search committee until possibly this fall.

The committee has held only one meeting since May 2, when Randy Mills announced he would be leaving at the first of July.  Maria Millan, formerly vice president for therapeutics, has been serving since July 1 as interim president. She has the backing of Mills to fill the slot permanently. 

The meeting of the search committee occurred July 17. And the agency has remained virtually mum on the process of selecting a new president to oversee what some call the final stage of its life. It is scheduled to run out of cash for new awards in about three years. 

Last Monday (July 31) , the California Stem Cell Report carried an item on the dearth of information about the process of selecting a new president, including the lack of a public timetable and addressing such questions as whether a search firm would be involved. The position carries a salary of up to $575,000.

Following publication of the item, Kevin McCormack, senior director for communications at the agency, sent the following email. 

"Just to be absolutely clear, this is exactly the same process we followed when we were looking for a replacement for Alan Trounson. Then, as now, we did not disclose the results of the closed session meetings of the Presidential Search Subcommittee nor described the contents or outcomes.  From the very beginning of this process, and we mentioned this at the last board meeting, the intention was for the subcommittee to meet and evaluate the options for finding a replacement for Randy and then for the matter to be taken up at the very next meeting of the full ICOC (the governing board). There is no 'lack of information' nor is there any intention of holding another Presidential Search Subcommittee meeting before the full board meeting in September."

The agency, however, has a record of difficulty in recruiting new presidents, including rejected offers and board discord on the desired qualities in a CEO.  It also has in the past disclosed such things as timetables and search firms and more. (See here, here and here.)

The Smell of Money and Gene Editing of Human Embryos -- Not to Mention the Breakthrough Science

"Horror to be avoided," "superbabies" and "what it means to be human" -- Just some of the language that turned up today concerning the news about the first human embryo editing experiment in the United States. 

Publications ranging from Wired to the Financial Times all had pieces discussing the work led by Shoukhrat Mitalipov of the University of Oregon in collaboration with researchers from California's Salk Institute, China and Korea. A search on Google news this afternoon turned up more than 1,000 citations dealing with the work.

Details of the science can be found in the journal Nature with a critique by UC Davis stem cell scientist Paul Knoepfler on his blog. He praised the research and said it "raises the stakes on future CRISPR use in humans." Elsewhere, the implications of the work also generated both heat and caution.

 The headline on a commentary in the Chicago Tribune said,

"Don't fear the rise of super babies. Worry about who will own genetic engineering technology."

The piece was written by bioethicist Arthur Caplan. He said,

"Freaking out over impending super babies and mutant humans with the powers of comic book characters is not what is needed....How close are they to making freakish super people using their technology? About as close as we are to traveling intergalactically using current rocket technology."

Caplan continued,

"We need to determine who should own the techniques for genetic engineering. Important patent fights are underway among the technology's inventors. That means people smell lots of money. And that means it is time to talk about who gets to own what and charge what, lest we reinvent the world of the $250,000 drug in this area of medicine."

Pam Belluck of the New York Times reported that the findings are "sure to renew ethical concerns that some might try to design babies with certain traits, like greater intelligence or athleticism."

She interviewed Hank Greely, director of Stanford's Center for Law and Biosciences. 

"'If you’re in one camp, it’s a horror to be avoided, and if you’re in the other camp, it’s desirable,' Dr. Greely said. 'That’s going to continue to be the fight, whether it’s a feature or a bug.'

"For now, the fight is theoretical. Congress has barred the Food and Drug Administration from considering clinical trials involving germline engineering. And the National Institutes of Health is prohibited from funding gene-editing research in human embryos."

Marcy Darnovsky, executive director of the Center for Genetics and Science in Berkeley, said in a news release,

"We have not yet engaged in processes that would promote the`broad societal consensus’ about human germline modification that the National Academies of Sciences and other prominent advocates of gene editing have recommended. Until that is achieved, we call on scientists around the world to refrain from research aimed at refining gene editing for use in human reproduction."

Bradley Fikes of the Union-Tribune in San Diego, a hotbed of biotech activity and the home of Salk, wrote that the study

"...brings to a head fundamental questions about what it means to be human, and whether changing the human genome would also change the human identity. And scientists — including those involved in the study — say it’s time for the public to speak up."

Given that this type of research is not funded by the United States government, just where the money came from is of interest. The Salk Institute had the answer. It said in a news release that the funding was provided by the "Oregon Health and Science University, the Institute for Basic Science, the G. Harold and Leila Y. Mathers Charitable Foundation, the Moxie Foundation, the Leona M. and Harry B. Helmsley Charitable Trust, and Shenzhen Municipal Government of China."

Some of the money "compensated" the women who provided the eggs, according to an article by Kelly Servick in the journal Science. Compensation, as opposed to expense reimbursement, is problematic in some areas of research. For example, it is banned in research that is funded by the $3 billion California stem cell agency.

The stem cell agency wrote about the research last week after the findings leaked out early. In February 2016, the agency convened a day-long session to examine issues involving gene editing. Last summer it issued new regulations that say that consent forms involving CIRM research must be modified to include a mention of genetic research. Added was the following phrase: "donated embryos [blastocysts], derived cells or cell products may be used in research involving genetic manipulation."

Regarding the future of the research, Mitalipov told the Financial Times that he wanted to perform regulated clinical trials at some point in the U.S. Unless something changes, he said that “unfortunately this technology will be shifted to an unregulated place."

Zipped Up in Oakland: California Stem Cell Agency Mute on Search for New CEO

More than mum -- a good description for the $3 billion California stem cell agency this morning as it wrestles with what is likely its most important decision of 2017.

And that is finding a new president to oversee what some call the "last stage" of its life and its search for a stem cell therapy that will fulfill the expectations of the voters who created the research program in 2004.

The governing board of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known, has held one meeting of its presidential search committee since Randy Mills announced his resignation May 2.

No public utterances emerged, however, from the July 17 session. Did the panel set a timetable for selecting a new president? No response. Will the board hire a search firm to help recruit? No response. Is the search committee going to meet again? No response. Simple questions that have been dealt with openly in past presidential searches at CIRM.

The search for a replacement for Mills has special significance. The agency expects it will run out of cash for new awards in three years or less. In the world of stem cell research, that may not be a nanosecond but it is a very short period of time.

Maria Millan, CIRM photo

It raises the likelihood of an exodus of staff from the program, which has also lost another linchpin in the organization, general counsel James Harrison. Without some certainty and confidence about the next three years, some CIRM employees, of which there are only about 50, may look to other enterprises for their professional futures. The likelihood of more departures could increase unless the board moves to shore up stability and to help retain the energy that Mills brought to the program.

Maria Millan, formerly the vice president for therapeutics, is interim president. Mills publicly endorsed her to fill his slot, which carries an annual salary of up to $575,000. She has been in place for one month. She too may be wondering about the stability of her position and whether she should be looking elsewhere for a position.

The next meeting of the governing board of the agency is set for Aug. 24. CIRM Chairman J.T. Thomas usually gives a report to the board at many meetings. He skipped that at the board meeting in July. Will he will brief the board at the August meeting on the presidential search? That has not yet been publicly disclosed.

An FDA Signal: Good News for California's Stem Cell Agency's Search for Therapies

The California stem cell agency this week highlighted a couple of milestones that it suggests may be creating a trend towards wider application of its research.

The springboard was an announcement that the Food and Drug Administration (FDA) had granted a "rare pediatric disease designation" to a therapy that the agency has backed with  nearly $52 million.

The FDA action comes as the agency is in what some call its "last stage." The agency, formally known as the California Institute for Regenerative Medicine (CIRM), expects to run out of cash for new awards in about three years. No additional funding appears readily available for the agency, which was created by voters in 2004. It has yet to fulfill voter expectations that it would produce a stem cell therapy that is widely available.

The agency this week, however, touted the FDA designation, which involves a treatment for what is commonly known as "bubble baby syndrome." The treatment is called OTL-101 and is being developed by Orchard Therapeutics Ltd, which is based in the United Kingdom The firm is working with Donald Kohn of UCLA, who has been researching in the area for decades.

Writing on the $3 billion agency's blog, The Stem Cellar, Kevin McCormack, senior director for communications, said,

"The treatment) involves taking the patient’s own blood stem cells, genetically modifying them to correct the SCID mutation, and then returning the cells to the patient. Those modified cells create a new blood supply, and repair the child’s immune system."

He wrote,

"The importance of the rare pediatric disease designation is that it gives the company certain incentives for the therapy’s development, including priority review by the FDA. That means if it continues to show it is safe and effective it may have a faster route to being made more widely available to children in need."

McCormack continued,

"This is the second time in less than two weeks that a CIRM-funded therapy has been awarded rare pediatric disease designation. Earlier this month Capricor Therapeutics was given that status for its treatment for Duchenne muscular dystrophy. 

"Two other CIRM-funded clinical trials – Humacyte and jCyte – have been given regenerative medicine advanced therapy designation (RMAT) by the FDA. This makes them eligible for earlier and faster interactions with the FDA, and also means they may be able to apply for priority review and faster approval. 

"All these are encouraging signs for a couple of reasons. It suggests that the therapies are showing real promise in clinical trials. And it shows that the FDA is taking steps to encourage those therapies to advance as quickly – and safely of course – as possible."

McCormack added, however,

"Getting these designations is, of course, no guarantee the therapies will ultimately prove to be successful. But if they are, creating faster pathways means they can get to patients, the people who really need them, at a much faster pace."

USA Today Reports on Financial Complaints Involving Stemedica

The San Diego stem cell company that received national attention for treatments of sports heroes such as Gordie Howe and Bart Starr is now facing legal complaints involving its financial affairs, USA Today reported this morning. 

The story by sports writer Brent Schrotenboer said, 

"The company, Stemedica Cell Technologies, had drawn praise from the families of Bart Starr, the legendary former NFL quarterback, and Gordie Howe, the hockey legend. Both families credited the firm’s stem cells for helping the two bounce back after suffering debilitating strokes. John Brodie, another stroke victim and former NFL MVP quarterback, also praised the company’s stem cell products after being injected with them in foreign countries.

"But even after that boost from big names, a spate of recent legal and financial issues has challenged the company, whose business relies on selling hope in the form of new but unproven regenerative medicine."

The lengthy article went on to discuss lawsuits involving misuse of funds and unpaid bills, including the alleged failure to pay a $500,000 loan despite numerous extensions. 

The company told USA Today that the misuse of funds allegations are "completely false" and has contested the other allegations in court. 

Former USC Med School Dean Loses Board Seat at $3 Billion California Stem Cell Agency

Former USC medical school dean Carmen Puliafito, who reportedly led a secret life involving drugs and prostitution, is no longer a member of the governing board of the $3 billion California stem cell agency.

In response to a query last week from the California Stem Cell Report, Evan Westrup, press secretary to Gov. Jerry Brown, said,

"This individual is no longer on the board."

Carmen Puliafito
Photo by Tibrina Hobson, FilmMagic

However, as of this writing, the web site for the agency listed Puliafito as a member of the governing board, carrying a short biography and a photo.

In response to a question this morning, Kevin McCormack, senior director for communication for the agency, said,

"If the governor says he is not in the board then he is not on the board. We will change the web page."

The governor's office did not respond to requests last week for more details concerning Puliafito's departure. But his term expired last December. Agency board members may continue to serve until a replacement is named.

The position that Puliafito held on the board of the California Institute for Regenerative Medicine(CIRM), as the agency is formally known, is required to be filled by an executive from a California university. Since the inception of the agency in 2004, the seat has been filled by the dean of the USC School of Medicine.

Puliafito (far left) at ceremonies opening stem cell center at
USC. CIRM provided $27 million for the $80 million project.
 Then CIRM board chairman Bob Klein is second from right.
Then Gov. Schwarzenegger stands next to Puliafito, who he
appointed to the CIRM board. 

It is not clear whether the governor will replace Puliafito with another representative from USC. Brown may look askance at the school in the wake of reports about how USC handled the Puliafito affair over a period of more than a year. 

A headline on a column last week by Los Angeles Times columnist Steve Lopez said,

"USC bosses flunk the leadership test amid shocking allegations about former medical school dean"

USC ranks 6th among California institution in the amount of awards it has received from CIRM, collecting a total of $110 million.

NY Times: The Race to Create 'Transformative' Cellular Treatments for Cancer

Cellular treatments of cancer received a big media boost this morning with a front page story in the New York Times that said the door is opening on a radical new class of therapy.

The headline on the piece in the print version of the Times in California said,

"Racing to Alter Patients' Cells to Kill Cancer"

The article by Denise Grady was also high on the Internet home page this morning of the Times.

The peg for the lengthy story was the expected approval of gene therapy for leukemia in the next few months. Grady wrote,

"Companies and universities are racing to develop these new therapies, which re-engineer and turbocharge millions of a patient’s own immune cells, turning them into cancer killers that researchers call a 'living drug.' One of the big goals now is to get them to work for many other cancers, including those of the breast, prostate, ovary, lung and pancreas. 

"'This has been utterly transformative in blood cancers,' said Dr. Stephan Grupp, director of the cancer immunotherapy program at the Children’s Hospital of Philadelphia, a professor of pediatrics at the University of Pennsylvania and a leader of major studies. 'If it can start to work in solid tumors, it will be utterly transformative for the whole field.;'"

High in the story (the fifth paragraph), Grady mentioned that cellular treatments are also being studied in connection with glioblastoma, the type of brain cancer afflicting Sen. John McCain. 

The Times carried a host of caveats about the likely new therapy, its dangers and even deaths that have occurred during the research. But "studies are forging ahead," Grady reported, despite the fact that they are expected to cost hundreds of thousands of dollars. 

The article did not mention the research that has been financed by California stem cell agency, which has pumped $90 million into developing cellular-connected treatments for cancer. The California Stem Cell Report wrote about that effort last week and filed a freelance story on the subject for The Sacramento Bee. 

McCain's Cancer and the California Stem Cell Agency: The Promising Approaches of Genetic Engineering

City of Hope video on T-cell treatment of glioblastoma

The type of aggressive brain cancer that is now afflicting U.S. Sen. John McCain is a disease that has been long targeted by California's $3 billion stem cell agency.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), has spent more than $90 million for research dealing with brain cancer, which claims the lives of 13,000 people each year.

Particularly deadly is glioblastoma, the form of cancer involving McCain.

In January, Karen Ring, a stem cell scientist and overseer of CIRM's social media, wrote on the agency's blog about an early stage clinical trial involving glioblastoma, describing it as "a new cell-based therapy that melted away brain tumors in a patient with an advanced stage" of the disease.

The research was conducted at the City of Hope's Alpha Clinic, an effort created by the stem cell agency.

In March, Behnam Badie, who is leading the research, discussed the therapy at a symposium dealing with results from year two of the Alpha Clinic program. Badie, whose father died of brain cancer, described how the use of T-cells beneficially affected the patient, Richard Brady, who was also a surgeon. 

Richard Brady, City of Hope photo

In a video presented at the symposium, Badie's colleague, Christine Brown, described T-cells as the "soldiers of the immune system." Brady also appeared in the video, saying, 

"I find myself in disbelief that I am here." 

On its web site, the agency says,

"Stem cell approaches look promising for treating gliomas. Certain types of stem cell tend to migrate toward the tumor cells wherever they are in the brain. CIRM-funded researchers are trying to genetically engineer those stem cells to produce cancer-killing molecules. Transplanted into the brain, these cells would seek out the cancer cells and deliver their therapy directly where it is needed. This approach could significantly decrease toxic side-effects to normal tissues, preserving or improving the patient's quality of life."

Brady, however, was not cured by his treatment and ultimately succumbed to the spread of the cancer. Ring wrote in January,

"The effects of the immunotherapy lasted for seven-and-a-half months. Unfortunately, his glioblastoma did come back....Patients with advanced cases of glioblastoma like Richard often have only weeks left to live, and the prospect of another seven months of life with family and friends is a gift."

Correction

Two items today and yesterday on the California Stem Cell Report incorrectly said that Carmen Puliafito was reappointed as a member of the stem cell agency's governing board in 2010 by Gov. Jerry Brown. The re-appointment was made by then Gov. Arnold Schwarzenegger. Brown was elected in 2010 but did not take office until 2011.

'Secret Life' Flap: Former USC Med School Dean-Stem Cell Agency Board Member on Leave from School

The former dean of the USC medical school, who is also a member of the governing board of the $3 billion California stem cell agency, is on leave and no longer seeing patients, the Los Angeles Times is saying today.

The news came after the Times reported yesterday that Carmen Puliafito had a "secret life" involving illegal drug activity, some of which was captured on video.

Puliafito was appointed in 2008 to the 29-member board of the California Institute for Regenerative Medicine (CIRM), as the agency is officially known,  by then Gov. Arnold Schwarzenegger and reappointed by him in 2010. Puliafito's term expired last fall but he is permitted to serve until a replacement is named.

The governor's office has not responded to requests yesterday by the California Stem Cell Report for a comment about the matter.

Puliafito has made no comment about the Times' reports.

(Editor's note: An earlier version of this item incorrectly said that Puliafito was reappointed by Gov. Jerry Brown. Brown was elected in 2010 but did not take office until 2011.)

LATimes: 'Secret Life' of Former USC Med School Dean and California Stem Cell Agency Director

Carmen Puliafito (right), a member of the governing board of the California
stem agency, and Robert Klein, then chairman of the agency. USC photo 2009

The headline in the Los Angeles Times this morning said:

"An overdose, a young companion, drug-fueled parties: The secret life of USC med school dean"

Put together by a team of five reporters, the article called Carmen Puliafito a "towering figure," a "renowned eye surgeon" and a prodigious fund raiser, bringing in more than $1 billion for USC by his own estimate. And then it said, 

"During his tenure as dean, Puliafito kept company with a circle of criminals and drug users who said he used methamphetamine and other drugs with them, a Los Angeles Times investigation found.

"Puliafito, 66, and these much younger acquaintances captured their exploits in photos and videos. The Times reviewed dozens of the images."

Puliafito resigned his $1.1 million position as dean in March 2016, declaring he wanted to pursue outside opportunities. He still serves, as a gubernatorial appointee, on the board of the California Institute for Regenerate Medicine (CIRM), as the stem cell agency is formally known. The board position pays $100 a day for meeting attendance. The board meets 10 to 12 times a year. 

USC has received $110 million from CIRM since 2004, ranking No. 6 among all California institutions that have won stem cell awards from the state agency. USC has had a representative on the board since its inception in 2004.

Paul Aisen, San Diego UT photo, Howard Lipin

Puliafito remains on the USC faculty and continues to accept patients. He is a "central witness" in a $185 million, legal wrangle involving UC San Diego researcher Paul Aisen. Puliafito has described himself as the "quarterback" in the effort to hire Aisen away from UC San Diego. The Times wrote,

"Curing Alzheimer’s is a top priority for government agencies and pharmaceutical companies, and Aisen’s lab was overseeing groundbreaking research, including drug trials at 70 locations around the world. More than $340 million in funding was expected to flow to his lab, according to court records.

"UC contended in its suit that its private school rival went beyond the bounds of academic recruiting by targeting professors and labs based on grant funding. The suit accused USC of civil conspiracy, aiding and abetting breach of fiduciary duty and other misconduct."

Puliafito's current term on the stem cell agency board term expired Nov. 3, 2016, according to a governor's office document. However, members of the board may serve until a replacement is named. He was appointed in 2008 by then Gov. Arnold Schwarzenegger and reappointed in 2010, also by Schwarzenegger, according to the governor's office document.

In response to a query, Kevin McCormack, senior director of communications for the agency, said via email,

"We were surprised to read the allegations about Dean Puliafito in the Los Angeles Times. Since being appointed to the CIRM Board by Governor Arnold Schwarzenegger, in late 2008, Dean Puliafito has served with distinction, bringing knowledge, expertise and deep commitment to our mission."

The California Stem Cell Report also asked Brown's office for comment. When it responds, the full text of the comments will be carried.

The reporters who put together the story are Paul Pringle, Harriet Ryan, Adam Elmahrek, Matt Hamilton and Sarah Parvini.

(Editor's notes: An earlier version of this item said that Puliafito was reappointed by Brown in 2010. The re-appointment was by Schwarzenegger. Brown was elected in 2010 but did not take office until 2011.
(This article was picked up Capitol Weekly, a news and information service devoted to California public policy issues. The Capitol Weekly version can be found here.)

Two Rounds of Transplants, 70 Drugs: An Advocate's Journey into Stem Cell Safety

Lukas Wartman
Science magazine, Whitney Curtis photo

The California stem cell agency today related a compelling story concerning a physician named Lukas Wartman who received "a life-saving stem cell treatment that is now threatening his health."

The headline on the item on the agency's blog, The Stem Cellar, said,

"One man’s journey with leukemia has turned into a quest to make bone marrow stem cell transplants safer"

Karen Ring, a scientist herself and overseer of social media at the California Institute for Regenerative Medicine (CIRM), wrote about the case after she read an article by Jon Cohen in Science Magazine concerning Wartman. He is a physician at the Washington University School of Medicine. Ring wrote,

"He was first diagnosed with a type of blood cancer called acute lymphoblastic leukemia (ALL) in 2003. Since then he has taken over 70 drugs and undergone two rounds of bone marrow stem cell transplants to fight off his cancer."

The first transplant came from his brother but the second round was from an unrelated donor and was different. Ring said, 

"While the second transplant and cancer-fighting drugs have succeeded in keeping his cancer at bay, Wartman is now suffering from something equally life threatening – a condition called graft vs host disease (GVHD). In a nut shell, the stem cell transplant that cured him of cancer and saved his life is now attacking his body."

Ring discussed the currently available treatments and continued, 

"Another promising therapy is called Prochymal. It’s a stem cell therapy developed by former CIRM President and CEO, Dr. Randy Mills, at Osiris Therapeutics. Prochymal is already approved to treat the acute form of GVHD in Canada, and is currently being tested in phase 3 trials in the US in young children and adults.

"While CIRM isn’t currently funding clinical trials for GVHD, we are funding a ($20 million) trial out of Stanford University led by Dr. Judy Shizuru that aims to improve the outcome of bone marrow stem cell transplants in patients. Shizuru says that these transplants are “the most powerful form of cell therapy out there, for cancers or deficiencies in blood formation” but they come with their own set of potentially deadly side effects such as GVHD."

Ring said that Wartman's battle with the disease has made him "one of the strongest patient voices advocating for new treatments" for his affliction. Ring said Wartman is an inspiration to funding agencies and other scientists. She said,

"We don’t want these patients to suffer quietly. Wartman’s story is an important reminder that there’s a lot more work to do to make bone marrow transplants safer – so that they save lives without later putting those lives at risk."