'Best of Times, Worst of Times' -- A Tale for Stem Cell Fans

Highlights:

Sinking stock prices vs. $40 billion market

Profits drive therapies

Test for the California stem cell agency

During the past week, followers of the stem cell world have been treated to sharply contrasting perspectives on the likely success of therapies involving regenerative medicine.

One perspective was bleak; the other robustly optimistic.

One borrowed slightly from the “Tale of Two Cities,” with a headline that said “Tale of seven stem cell stock woes….”

The other carried a headline that said “global stem cell market predicted to reach $40 billion in five years….”

One was a story about hard-eyed investors backing away from stem cell research. The other was about a bright, near-term future for the field.

Don Gibbons, chief communications officer at California’s stem cell agency, wrote about that bright global market on his agency’s blog. He said that the $40 billion figure was generated by the worldwide consulting firm Frost and Sullivan, which was promoting its study of the field. The 2014 report is going for as much as $7,500, a price tag that helps illustrate one of the features of the stem cell field: It needs a lot of cash.

Indeed, Gibbons noted that Frost and Sullivan cautioned that funding for early stage, clinical stem cell work is not abundant.

Which brings us to Paul Knoepfler’s tale of stem cell stock “woe.” He is a stem cell researcher and blogger at UC Davis, which has benefited mightily from cash from the $3 billion California stem cell agency.

He wrote,

“Lately, stem cell companies have not been doing so well financially to put it mildly and their stock prices have generally been going down, down, and down further. A lower stock price and market capitalization are not just a headache for investors and bad for the companies, but they also strongly interfere with the progress of the clinical science.”

And he asked,

“To be blunt, why are their stocks doing so miserably?”

Knoepfler did not really attempt to answer that question. However, it is clear that stem cell stock prices are down because nobody wants to buy them. The reason? Investors do not expect to make money any time soon by purchasing shares in those firms, which struggle perennially to raise cash.

That is a financial reality that the $3 billion California stem cell agency needs to fully integrate into its plans for spending its last $800 million, which is slated to run out in less than five years.

The agency’s goal is to produce a stem cell therapy as promised to California voters 11 years ago when they approved creation of the agency, which will cost the state about $6 billion, including interest, before the effort expires.

Without backing of industry, there will be no therapies. But the priorities of a business are not necessarily the same as the agency’s. Profits necessarily come first with a business. Otherwise it will vanish into a financial abyss. Witness the dumping of the nation’s first hESC clinical trial by Geron, in which the agency invested $24 million only three months before the trial was summarily terminated by the company for financial reasons. 

The California stem cell agency’s president, Randy Mills, should understand the vagaries of all this better than most. He made his career as a biotech business executive, not as a researcher. Over the last year, he has done much to sharpen the focus of the agency and begin to create a clear path for emergence of a therapy, a “radical” change he has dubbed CIRM 2.0.

Whether he can successfully put together the needs of business and the needs of science and medicine is perhaps the key question for the California stem cell agency between now and 2020. 

California's Salk Institute Looking for New CEO

San Diego is a hotbed of biomedical research, so when one of the leaders of its top institutions leaves, it is significant news.

William Brody, Salk photo

William Brody, head of the Salk Institute and a former director of the California stem cell agency, will be retiring at the end of the year, the San Diego Union-Tribune reported yesterday.

Brody, 71, came to Salk in 2009 and has wrapped up a $300 million fundraising effort that has stabilized the nonprofit after a period of financial uncertainty, Gary Robbins reported in the newspaper’s story.

Robbins wrote,

"'Salk has always had a much smaller endowment than other comparable institutions, but with decline in funding for biomedical research we needed a buffer and that is what Bill achieved,' said Terry Sejnowski, a Salk neuroscientist. 'None of his predecessors were able to do this. We don't have wealthy alumni or grateful patients, just the best basic science.'

"But if you compare where we are today with Scripps (Research) and medical schools around the country that are bleeding faculty and debt we are solvent if not plush, and continue to recruit the best young faculty. Looking to the future we need to find another Bill Brody."

Brody served on the governing board of the $3 billion state stem cell agency from August 2009 to the end of 2012. His predecessor at Salk, Richard Murphy, also served on the board and as interim president of the agency. Salk has not had a representative on the board since Brody left. 

Salk had received $52 million from the agency, which ranks the institute 12th on the list of recipients of awards. When Brody left the agency board in late 2011, Salk had chalked up $37 million in stem cell agency awards.

Salk officials said they will launch a nationwide search for a replacement. Scripps Research, also located nearby in the San Diego suburb of La Jolla, is also looking for a new CEO with top notch fundraising abilities. 

California Stem Cell Agency Ends Today's Session

OAKLAND, Ca. -- Today's meeting of the governing board of the California stem cell agency has concluded. We will have more on its proceedings during the next few days. Here is a link to the agency's press release on the meeting. It identifies the other recipient of an award ($8.5 million) made today as Mehrdad Abedi of UC Davis. Donald Kohn of UCLA received the other award ($7.4 million). 

Concerning the changes in the basic research and translation award programs, the press release said,,

"By scheduling both the Discovery and Translational awards more regularly CIRM is creating a system that helps move the most promising projects along a pipeline and also provides predictability to the grant system, making it easier for researchers to know when they can apply for funding.

"The stem cell agency estimates that each year there will be up to 50 Discovery awards worth a total of $53 million; 12 Translation awards worth a total of $40 million; and 12 clinical awards worth around $100 million."

Parkinson's Patients Dismayed by Slow Work at California Stem Cell Agency

OAKLAND, Ca. -- Directors of the California stem cell agency today unanimously approved $40 million for a program to advance or "translate" basic research during a session that was marked by emotional pleas from Parkinson's Disease patients for much faster action.

The contingent from the San Diego area told directors and agency officials they were not moving fast enough to fund a research effort that they supported.

One patient advocate, Jenifer Raub of San Diego, said it was "outrageous" that the effort could not even be considered by reviewers until next March with no funding until about 2017.  Another unidentified patient said,

"Don't let policy, bureacracy and procedures stop this for another year."

Agency officials said that the time is required to meet legal requirements for the competitive grant process and reviews of the proposals that would be submitted.

Individuals appearing at the meeting were largely associated with Summit4StemCell of San Diego and have raised to $2 million to back research by Jeanne Loring, head of the stem cell program at Scripps Research in La Jolla.  The comments appeared spontaneous in reaction to the CIRM staff presentation on the new program. Loring told the California Stem Cell Report she did not organize the comments.

The agency's new, four-round, translation research program promises cash to researchers within nine months of submission. The program says that applications will be accepted in September and March of each year. Asked about the timetable, Mills told the California Stem Cell Report that it will begin in 2016.

Mills later told the board that the agency needed to be responsive to patient needs. He said he would "see if there is anything we can do." He said he would report back to the board on the matter.

(The last paragraph in this item was added about one hour after its initial posting.)

California Stem Cell Agency Approves $53 Million for Basic Research

OAKLAND, Ca. -- Directors of the California stem cell agency this morning approved a $53 million "discovery" research program that is aimed at funding basic research and creating special incentives to help assure that it will lead to a widely used therapy.

Randy Mills, president of CIRM, said a new, $150,000 bonus award will be created as part of the program. He also said recipients will be newly required to explain how their proposal could lead to a therapy, among other things.

Mills told directors that the new requirements would help to prevent past situations in which only 5 percent of basic research awards advanced along a path towards development.

Directors voted unanimously to go forward with the effort following a discussion that focused mainly on technical details of the plan. (See here and here for additional information on the plan.)

UCLA's Kohn Wins $7.4 Million Stem Cell Research Award

OAKLAND, Ca. -- Donald Kohn of UCLA is the recipient of a $7.4 million award from the California stem cell agency to develop a therapy to treat a rare genetic condition called chronic granulomatous disease.

Kohn's proposal was approved unanimously by the directors of the $3 billion agency meeting here this morning. The agency did not identify Kohn as the recipient. However, it was learned shortly after the approval that the Los Angeles investigator had made the application.

See here for more information on his proposal.

$16 Million in Two Grants Approved by California Stem Cell Agency

OAKLAND, Ca. -- Directors of the California stem cell agency today approved $16 million for two clinical trials involving AIDS lymphoma and another involving a rare genetic affliction that creates immune deficiencies problems.

Names of the recipients were not immediately disclosed. See this item for more information on the awards.

$100 Million for Clinical Stage Stem Cell Research in California

OAKLAND, Ca. -- Directors of the California stem cell agency today approved $100 million for clinical stage research for the 2015-16 fiscal year. The funds were allocated on a unanimous vote with no discussion.

The effort extends the CIRM 2.0 program, which began in January of this year. The agency awarded $25 million during the first half of this year out of a $50 million budget.

Oakland Gains HQ of the California Stem Cell Agency

OAKLAND, Ca. -- The California stem cell agency will be moving to new headquarters this fall in Oakland, across San Francisco Bay from its current home near the San Francisco Giants ballpark.

The address of the new headquaters is 1990 Harrison Street in downtown Oakland. Details of the cost and the square footage were not immediately available.

California Stem Cell Agency: 'Lots of Money and Lots of Time"

OAKLAND, Ca. -- The California stem cell agency today said it has committed $1.97 billion during its nearly 11 years of life and anticipates spending about $170 million to $190 million over the next five years.

Agency President Randy Mills presented the figures at a meeting of the agency's board of directors. 

He said he anticipates a "net commitment" of $170 million annually over the next five years.

He said,

 "We have lots of money and lots of time."

California Stem Cell Meeting Begins in Oakland

Directors of the California stem cell agency today began their meeting here at 9:11 a.m. PDT with a report from Chairman Jonathan Thomas.

Looking for Clarity at the $3 Billion California Stem Cell Agency

OAKLAND, Ca. -- Scientists and others seeking the short version of the plans for funding of basic and translational research by the $3 billion California stem cell agency should look no further than an 18-page Power Point presentation.

The explanation is available on the agency’s Web site and in some ways is better than the more prolix description offered in three formal memos. While the presentation offers a fast read, seriously interested parties will need to plow through all three documents. (See here, here and here.)

Critical to understand the direction of the agency, however, is another Power Point presentation -- this one by Randy Mills and which is not supported by a memo or other formal document. But some of what Mills lays out in the slides has been brought up at earlier meetings, the voluminous transcripts of which are available on the agency’s Web site, if you can find them.

A search of the agency’s Web site late yesterday using the term “transcript” turned up 879 results. No transcripts of remarks by Mills or of meetings of the agency’s board of directors were found in the first 14 pages of the results.

In his slides today, Mills is covering his thinking on both basic and translational funding and how therapy development should have a continuous and predictable pathway. He likes to talk about it in terms of rail lines, a homely but apt analogy.   

While we are on the subject of Power Point presentations, the agency’s slides have improved greatly since Mills became president of the agency in May 2014.  Nonetheless, Power Point presentations do not necessarily enhance understanding, especially when the presenters do little more than read the notes on the slides. The result is known as “Death by PowerPoint!”

Slides are no replacement for a single, nuanced, written explanation of a proposal, including charts, something more than the agency usually offers in its memos to the board. That said, their memos have also improved and are more comprehensive and straight forward than under the regime of the agency’s former president, Alan Trounson.

Coming Up: Full Coverage of California Stem Cell Meeting Today

The California Stem Cell Report will be on the scene today in Oakland as the Golden State's $3 billion stem cell agency is set to allocate $243 million for a wide variety of research efforts plus a high school training program. News reports will be filed as warranted throughout the day.

The public can participate in the meeting at its physical site in Oakland and at teleconference locations in Beverly Hills, San Diego, South San Francisco, Stanford, Sunnyvale and two in La Jolla. An Internet audiocast is also available. Details are available on the agenda for the meeting, which begins at 9 a.m. PDT.

NDA Appoints Ellen Feigal as Partner

Ellen Feigal, formerly the No. 2 person at the $3 billion California stem cell agency, yesterday was named as a partner in NDA Partners, an international consulting firm to the medical products industry.

Feigal resigned last fall as senior vice president at the stem cell agency, about five months after Randy Mills was named as its new president.

NDA's web site says it is a "a global strategy consulting firm specializing in expert product development and regulatory advice to medical products industries" and that it deals with "emerging biopharma." The web site does not disclose the names of its clients, and it is not known whether any are involved with the California stem cell agency.

Full Coverage of Tomorrow's Stem Cell Meeting

The California Stem Cell Report will provide gavel-to-gavel coverage tomorrow of the directors' meeting of the $3 billion California stem cell agency. Up for consideration are plans for spending $243 million, among other things.

The public can participate in the meeting at its physical location in Oakland and at teleconference locations in San Diego, South San Francisco, Stanford, Sunnyvale and two in La Jolla. An Internet audiocast is also available. Details are available on the agenda for the meeting, which begins at 9 a.m. PDT.

California Ready to Approve $243 Million for New Stem Cell Research

CIRM President Randy Mills uses a railroad analogy for therapy development

The California stem cell agency Thursday is set to approve $243 million to finance everything from testing therapies on patients to exposing high school students to research.

The multi-faceted effort will come before the 29 directors of the agency at their meeting on Thursday at the Oakland Marriot City Center. The initiatives have already cleared the directors’ Scientific Subcommittee.

The effort is the second installment in the CIRM 2.0 program begun earlier this year by the agency’s president Randy Mills. He assumed his post in May 2014 at the California Institute of Regenerative Medicine or CIRM, as the $3 billion agency is formally known.

Mills has described his changes as radical. He says they are aimed at improving the quality of applications and speeding development of therapies. He also has called for deeper involvement by the agency in the direction and work of researchers.

Mills, who has made his entire career in the biotech business, says he wants to provide a relatively smooth track, a “continuous, predictable pathway” from basic research to clinical applications. He uses a railroad analogy to illustrate the desired progression of research(see graphic above).

The $243 million up for approval this week is about 30 percent of the agency's remaining $800 million. However, the money will be spent over the next several years -- not just this year. Under Mills' spending projections, the agency will not run out of cash until 2020. 

The largest program coming before CIRM directors this week is $100 million during 2015-16 for more advanced research related to clinical development. Directors earlier approved $50 million for the effort for the first six months of this year. However, they have awarded only $25 million as of the end of June.

Another $53 million is set for a variety of mostly basic research, which Mills calls “discovery.” Requests for applications are scheduled to begin late this summer or fall. Some of the rounds will have application openings twice a year.  Cash is scheduled to flow to researchers within about 10 months of application.

Mills is also asking the board for substantial delegation of authority to him during the reviews and is calling for an “optimized review process” because of a high anticipated volume of applications in discovery.

Under the $40 million translation research round, Mills would also be delegated authority in the grant review process to pluck out applications and advance them even if reviewers disagreed. Applications will be accepted in September and March. Cash will flow to researchers about nine months after applications are submitted.

Both the discovery and translation rounds are open to both businesses and nonprofit enterprises.

A revised training program called “Bridges 2.0” for college level students is budgeted for $46 million over a five-year period. A training program for high school students, changed and rebranded as “Spark,” will receive $4 million over five years. Applications for that program are due Oct. 1. It is open to colleges, universities and non-profit academic institutions. 

Applications for the Bridges program are also due Oct. 1. They will be limited to “California public universities or colleges or private, nonprofit academic institutions, which did not receive a CIRM-funded Major Facility or Shared Research Laboratory Award (and, hence, do not have a major stem cell research program or a critical mass of stem cell researchers).”

In addition to the Oakland location of the directors' meeting, the public can participate at teleconference locations in San Diego, South San Francisco, Stanford and two in La Jolla. Addresses can be found on the agenda, which includes directions for listening to the Internet audiocast of the meeting.

Email comments for the CIRM directors on items on the agenda may be sent to mbonneville@cirm.ca.gov.

California to Hand Out $16 Million for Stem Cell Research This Week

The California stem cell agency this Thursday is scheduled to give away nearly $16 million for clinical trials for possible therapies connected to AIDs and a rare genetic affliction involving immune deficiencies.

The larger of the two awards -- $8.5 million -- will go to an unidentified recipient for an early stage trial involving AIDS-lymphoma patients.

The agency’s blue-ribbon reviewers earlier voted 10-0 behind closed doors to approve the application. On Thursday, the full board of the agency will ratify the vote in public. The board has almost never overturned a decision by reviewers to fund an award.

The summary of the review of the application, which was not disclosed, said that the proposed therapy “would be a significant improvement over standard of care in the proposed AIDS lymphoma patient population. 

“With the proposed patient population (AIDS lymphoma patients), impact will be somewhat limited. However, demonstration of success in this limited context would serve as an impetus to solve additional problems such as non-ablative conditioning to achieve engraftment in the larger HIV patient population.” 

Despite another “paucity of patients,” the stem cell agency is also expected to approve $7.4 million for an early stage trial involving X-linked chronic granulomatous disease.

Reviewers voted 5-1-4 to approve the application. The voting translates to five for the application, one indicating it needs improvement and four indicating that it is “sufficiently flawed that it does not warrant funding, and the same project should not be resubmitted for review.” 

The review summary said,

“Reviewers were…divided as to the feasibility. Some reviewers did not think that there are sufficient patients afflicted with this disease who also meet the enrollment criteria to allow for completion of the trial with enough patients to yield meaningful outcomes. Other reviewers were optimistic that, given the track record of this team and the willingness of patients with intractable orphan diseases to participate clinical trials, the trial could be fully enrolled and yield results to potentially support moving the therapeutic toward approval by the FDA and/or advance the field.” 

The target of the potential therapy is an inherited condition that weakens the immune system. According to the Rare Disease Network, it occurs in only one out of every 250,000 persons. Only 20 new cases a year are reported in the United States, according to Wikipedia. The review summary used the expression “paucity of patients.”

Reviewers said, however, the approach has “potential value in a number of other diseases.”

The application carried with it $4.6 million in co-funding promised in a publicly unspecified form via the applicant.

Both the applications had been reworked and resubmitted to the agency under its new CIRM 2.0 award process. However, this week is the first time the board has considered them.

(Please note that the agency has cobbled together the review summaries for both applications. That means that some readers seeking the summary related to the $8.5 million award could be misled when the immune deficiency award turns up at the top of the document. The full application is also never disclosed by the agency, only the summary.) 

Rejected by reviewers was a $2.9 million application from another unidentified group. The vote was 2-6-3, meaning that the proposal can be reworked and resubmitted.

The board’s meeting will be in Oakland at the Marriott City Center. Public teleconference locations will be in San Diego, Stanford, South San Francisco and two in La Jolla. The public, including scientists, can participate from those locations as well as in Oakland.

An audiocast is also available. Full details can be found on the agenda.

National Controversy Snares California Stem Cell Firm; Congressional Investigation, Presidential Politics Involved

A California stem cell firm is enmeshed today in a national flap linked to abortion, buying and selling body parts and the 2016 presidential race.

The firm is StemExpress of Placerville, just east of Sacramento in the Sierra Nevada foothills. It was mentioned in a YouTube video that was recorded secretly by abortion foes who were posing undercover in a discussion with Planned Parenthood officials.

The video has been viewed nearly two million times as of this writing. The New York Times, the Washington Post and other major news outlets are covering the story.  It has triggered virulent headlines on anti-abortion and conservative web sites.

One headline on inquisitor.com said in red type,

“Buying And Selling Aborted Fetus Body Parts For $30 To $100? ‘Stem Express’ Website Down After Undercover Video Exposes Planned Parenthood” 

StemExpress provides researchers with “high-quality stem cells they need for their research,” an employee of the firm told the California Stem Cell Report in an email last May.  The firm also supplies “human blood, tissue products, primary cells and other clinical specimens to biomedical researchers around the world,”

Reporter Sammy Caiola wrote today in The Sacramento Bee,

“The eight-minute undercover clip, which has garnered over 1 million views (as of yesterday) and inspired the popular anti-abortion Twitter hashtag ‘#PPSellsBabyParts,’ is edited in a way to suggest Planned Parenthood and its affiliates profit from fetal tissue and organs procured during abortions, which is against federal law.”

Caiola said that the video contains “a supposed image of the StemExpress website (that) shows an order form, where users can designate type of order, number of specimens, tissue type and shipping options.”

Caiola continued,

“In a statement released by Planned Parenthood, spokesman Eric Ferrero said that while many women who undergo abortions choose to donate fetal tissue to scientific research, there is no financial benefit for either the patient or Planned Parenthood. Sometimes, Planned Parenthood is reimbursed for the cost of transporting the tissue to labs, he said.

“’We do this just like every other high-quality health care provider does – with full, appropriate consent from patients and under the highest ethical and legal standards,’ Ferrero said.” 

The California Stem Cell Report this morning asked StemExpress for comment and will carry it verbatim when it is received. However, all of the news stories so far say that the company has not responded to similar requests.

(The company responded -- see here for full text -- just as this item was posted and said,

"StemExpress prides itself on complying with all laws. Our compliance protocols and donor consents have been established by an FDA-compliant Independent Review Board. Written donor consent is required for any donation, including bone marrow, tissue of all types or blood."

(The company also said,

"Everything we provide is solely at the request of the nation’s and the world’s great research institutions. Producing the isolated cells researchers need from donated tissues requires the development of complicated, often unique, research techniques using millions of dollars of scientific equipment. We are hopeful the events of the last few days will not diminish our efforts to support the research community or hinder our partners from continuing their important work.")

The company was founded in 2010 by Cate Dyer.  Inc. magazine ranked the firm 363rd on Inc. magazine’s list of the fastest-growing private companies in America last year.

Ariana Eunjung Cha of the Washington Post wrote that GOP presidential hopefuls Ted Cruz, Jeb Bush, Ben Carson and Bobby Jindal all have spoken out on the controversy.

In Congress, Republican leaders have ordered an investigation. Jackie Calmes and Nicholas St. Fleuer of the New York Times said,

"House Republican leaders on Wednesday announced a congressional investigation of Planned Parenthood, a day after anti-abortion activists released a video of an unsuspecting official from the organization explaining how it provides fetal tissue to researchers.
 "Echoing the activists’ allegation, Speaker John A. Boehner and other top Republicans suggested that Planned Parenthood was selling fetal parts, which is illegal if done for profit. But Planned Parenthood said that while it charges for expenses such as processing and transporting, it makes no money from the fetal tissue donated by women who get abortions. The official shown in the video repeatedly says something similar to two activists posing as biotechnology representatives."

Text of StemExpress Comments

The following is the full text of StemExpress’ comments about the ongoing controversy involving it and Planned Parenthood. The statement was sent by Beau Phillips, a spokesman for the firm, in response to a request this morning by the California Stem Cell Report.

Statement of StemExpress Concerning Recent Media Stories: 

StemExpress is a small life sciences company that supports leading research institutions in the United States and internationally—including medical schools, pharmaceutical companies, and federal agencies—to provide stem cells and other human tissue critical to medical research.  Cells produced by the physicians, scientists, medical technicians and nurses at StemExpress are currently used in research globally aimed at finding cures and treatments for cancer, diabetes, HIV/AIDS, cardiac disease, and other significant medical conditions.  StemExpress prides itself on complying with all laws. Our compliance protocols and donor consents have been established by an FDA-compliant Independent Review Board. Written donor consent is required for any donation, including bone marrow, tissue of all types or blood. 

We are proud of the role we play in helping the global research community as they strive to achieve medical breakthroughs to stamp out global disease and improve quality of life. Everything we provide is solely at the request of the nation’s and the world’s great research institutions.

Producing the isolated cells researchers need from donated tissues requires the development of complicated, often unique, research techniques using millions of dollars of scientific equipment. We are hopeful the events of the last few days will not diminish our efforts to support the research community or hinder our partners from continuing their important work.

Lawsuit Alleges Biotech Guru Steve Burrill Embezzled $17 Million

Steve Burrill, wn.com photo

California biotech maven Steve Burrill, once a keynote speaker at such events as the annual BIO industry conference, has been charged with embezzling more than $17 million from an investment fund bearing his name.

The lawsuit surfaced in a spate of articles this month beginning with a piece July 10 by Dawn Lim of the Wall Street Journal. She wrote,

“The lawsuit, filed on July 6 with the Superior Court of California in San Francisco, alleges that Mr. Burrill, as well as corporate entities and individuals tied to him, embezzled more than $17 million from Burrill Life Sciences Capital Fund III L.P. between late 2007 and 2013.

 “The lawsuit also claims that the improper use of the fund’s money drained it of capital to support further investments in companies it backed, causing 'more than $30 million in investment losses.'

 “Earlier this year, investors in Burrill Life Sciences Capital Fund III L.P. installed executives from San Francisco-based Kearny Venture Partners to manage the fund. A corporate entity affiliated with Kearny authorized the lawsuit."

John Carroll of Fiercebiotech wrote,

“'Burrill was also a fraudster,’ the suit goes on. ‘Shortly after his company assumed the helm of the Fund as its General Partner, he started looting the Fund's cash and diverting it to entities he wholly owned or controlled.’” 

Reports of financial irregularities surfaced a little more than a year ago when Burrill was removed from control of his fund.

Over the years, Burrill raised more than $1 billion for life sciences financing via his investment banking firm. He produced an annual state-of-the-industry study that was something of a bible.

In 2006, his firm organized an international stem cell conference that was partly a celebration of the California stem cell agency. 

None of the stories published about the latest lawsuit reported comments from Burrill or his representatives.

Here are links to other stories on the matter: San Francisco Business Times, Biospace, Biocentury and Giacor Consulting.

'Brutal Facts,' Parkinson's and the California Stem Cell Agency

Randy Mills in San Diego, Union-Tribune photo by K.C. Alfred

The $3 billion California stem cell agency took its strategic-plan road show to San Diego yesterday and heard impassioned pitches for development of a Parkinson’s therapy.

Reporter Bradley Fikes of the San Diego Union-Tribune wrote that “sometimes emotional” exchanges arose at the meeting.  And he reported that the agency’s president, Randy Mills, said one of the “brutal facts” is that that “drug and medical companies aren’t really interested in stem cell treatments.”

The hearing was one of three this week to gather information to help reshape the agency’s plan for spending its last $800 million over the next five years. The other hearings will be in Los Angeles today at noon and in San Francisco tomorrow at noon. (See here for addresses.)

At yesterday’s session, Fikes quoted Mills as saying,

"We do not have enough industry pull for our technology….We don't have enough buyers for our intellectual property."

The article continued,

“Stem cell therapies are still considered too risky by most companies, Mills said.”

 About 70 persons attended the meeting, according to Fikes. That is about 10 times the size of the audience at most meetings of the governing board of the California Institute for Regenerative Medicine or CIRM, as the San Francisco agency is formally known.

Many in the audience were there to pitch for Parkinson’s. Fikes wrote,

“Patient advocate Sherrie Gould asked Mills to support funding a potential Parkinsons' disease therapy being developed by a coalition of scientists, doctors and Parkinson's patients called Summit4StemCell. Dozens of coalition supporters attended the meeting.

“The group seeks to grow replacement brain cells to relieve symptoms of the movement disorder, derived from the patient's own skin cells. It's affiliated with the Parkinson's Association of San Diego.”

Fikes continued,

David Higgins, Linked In photo

“We are ready, the patients are ready, the cells are ready,’ Gould said. ‘Time is running out for these patients.’" 

The 29-member CIRM board has a Parkinson’s patient advocate as a member, David Higgins of San Diego, who is a former president of the Parkinson’s Association, according to CIRM.

Face-to-face With the California Stem Cell Agency: San Diego to San Francisco

Californians will have a chance next week to go eyeball-to-eyeball with the top leaders of the state’s $3 billion stem cell agency and tell them what they think.

Meetings will be held in San Francisco, Los Angeles and the San Diego area. Plus there is a “light” lunch, but you do need to RSVP to info@CIRM.ca.gov.

Kevin McCormack, senior director of communications for the agency, wrote about the sessions earlier this week. He said they were part of the revision of the agency’s strategic plan – how it intends to spend its remaining $800 million or so.

The agency earlier posted on online survey for the public. But McCormack said on the agency’s blog:

“There is a big difference between ticking a box and having a conversation. These upcoming meetings are a chance to talk together, to explore ideas and really flesh out the details of what this strategic plan could be and should be.”

Originally the initial cut of the plan was scheduled to be presented to agency directors late this month. However, McCormack told the California Stem Cell Report that presentation was being postponed.

“The reason is pretty simple; the strategic plan is going to be the blueprint for the future of the agency and  will play a vital role in shaping the direction in the next few years so we take we want to make sure the board has a chance to discuss it in detail. Because so many board members are not going to be able to attend the July meeting – family vacations etc. - we felt it just made more sense to postpone the discussion of the plan to our next in-person board meeting, in September.” 

Here are the locations and times for the sessions.

La Jolla, Monday, July 13 at noon at Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive.

Los Angeles: Tuesday, July 14 at noon at Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at USC, 1425 San Pablo Street, 1st floor conf. room Los Angeles.

San Francisco: Wednesday, July 15 at noon at CIRM, 210 King Street (3rd floor), San Francisco. 

New 'Railroad' Coming to the $3 Billion California Stem Cell Agency; Basic Research Financing to Get New Ticket

"If you want a bike, don't ask for a toy"
Scientists welcome to comment

Directors of the $3 billion California stem cell agency this month will take a crack at building a new, basic research “railroad” with the hope of creating a “predictable path” to development of therapies and cures.

The move will involve funding for basic and translational research, the initial steps that lead to producing therapies that can be widely used by the public.

The effort is scheduled for approval at a meeting July 15 of the agency directors’ Science Subcommittee. The agenda for the meeting was posted last week minus details of the “concept plan” for the move. However, the changes are part of the CIRM 2.0 effort initiated by Randy Mills, president of the California Institute of Regenerative Medicine (CIRM), as the agency is formally known.

Mills kicked off CIRM 2.0 in January. His initial foray involved the more advanced research efforts that are engaged in testing treatments on human beings. Mills described the CIRM 2.0 program as “radical.”

In May, Mills sketched out the thinking behind extension of the CIRM 2.0 approach to basic research.

He told directors at their meeting that month,

“The first thing is we need is a continuous predictable path from discovery to clinical. So if we have a discovery program (basic and translational research), the purpose of that discovery program ought to be to deliver something downstream to where it ultimately impacts patients. That kind of makes sense.”

Mills quoted CIRM senior science officer Lila Collins as saying about the approach,

“If you want a bike, don’t ask for a toy.”

Under past procedures, Mills said basic research findings financed by CIRM only advanced 5 percent of the time. He acknowledged that it is “really difficult” to foresee where basic research is headed. But he said an analysis of the CIRM grants showed that when the agency specified that it wanted the research to progress, it advanced 30 percent of the time instead of 5 percent. 

Using primarily a railroad analogy, Mills said,

“We need to put intent into the RFA that says we want you to be creating something, and we want you to be thinking forward ….Maybe you don't take it there, but how is it going to move ultimately downstream? We want you to be thinking about that at the time you apply because if you do, we know it has a much better chance of going to thenext station.” 

Mills said,

“We need to have track that's continuous….We need to make sure thetrains run on time. We need to have predictable progression. We call this baton passing. We want the cargo on the train to proceed down the track. And we need to be really creative on how we're going to do that.
 “We need to be responsive to new developments and downstream bottlenecks. And then, lastly, we need to have multiple development pathways so we basically serve all of the destinations we want to serve.”

Even if scientists are not entirely pleased with the new approach, they are likely to climb aboard. Research funds nationally are scarce. CIRM itself has not offered a basic biology grant round since March 2013.

However, Mills’ latest proposal is still subject to modification. The Science Subcommittee may ask for changes before sending it to the full board for approval on July 23 at a session in Berkeley. Researchers and the public are welcome to comment and make suggestions.

Details of the concept plan are likely to be posted at least a few days before the Science Subcommittee meeting July 15. The meeting is open to the public with sites available in San Francisco, Cedars-Sinai in Los Angeles, UC Irvine, Pasadena and San Diego. See the agenda for addresses.

Written comments can be emailed to CIRM directors via mbonneville@cirm.ca.gov. 

Stem Cell Stocks to Grant Writing: One Researcher's Perspective

The president of the United States has his “state-of-the-nation” perspective but one California researcher has a somewhat more modest view of a smaller world.

Paul Knoepfler of UC Davis gave something of a “state-of-the-stem-cell-world” overview earlier this week on his blog, ipscell.com. He covered everything from grant writing to his investments in a stem cell company.

He wrote,

“Doing this blog for 5+ years has made it really sink in just how crucial the commercial side is to all of our shared goals. This means that things like stocks, patents, etc. are really important for us all to think about including academics, patients, students, and more.”

 He noted that he made a “very small” investment in Ocata Therapeutics, formerly known as Advanced Cell Technology.

But he said that the firm has been “getting its bell rung this year and in particular more recently it has dropped off a (small) cliff.”

On winning grants to continue his research, he said,

“In my 2+ decades in science in various positions I’ve never seen it quite this bad. These days it seems like I’m always working on at least one grant and sometimes several simultaneously. It eats up a lot of time, focus, and energy, but that seems to just be the new reality.”

 There is more. You can read it all here. Plus here is his perspective on other wide-ranging matters and his predictions about developments involving stem cells.

Correction

A June 30 item involving the Alpha clinic program incorrectly stated that the program involved $34 million. The correct figure is $24 million.

Three Top Execs Named Today at $3 Billion California Stem Cell Agency

The California stem cell agency today marked the completion of a year-long reorganization with the appointment of three top executives, including a veteran of Genentech.

Randy Mills, who became president of the $3 billion agency in May 2014, said the appointments represent a “significant inflection point.”

Mills said in a press release,

“People are everything when it comes to the potential and performance of an organization.  With the appointment of these three exceptionally talented individuals to the leadership team, CIRM is now better positioned to achieve its mission and forever change the practice of medicine.”

The trio consists of Ramona Doyle, vice president of therapeutics; Maria Millan, senior director of medical affairs and stem cell centers, and Maria Bonneville, director of administration. Millan and Bonneville both were promoted from within the organization.

Ramona Doyle, Rhodes Project photo

Doyle comes from Genentech. She was senior group medical director for respiratory product development at the South San Francisco business, where she has worked since 2009. She worked for Gilead Sciences for two years. Doyle has also taught at UC San Francisco and at the Stanford Medical School, where she worked with Millan.

Beginning Monday, Doyle will oversee projects involving neurological and ocular, cancer and blood related and cardiovascular, lung, liver and other organ system-related conditions. She is also the only person designated as vice president within the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

Doyle received her M.D. from Emory University and was a Rhodes Scholar after receiving an undergraduate degree in English and literature from Sewanee: The University of the South. When she was at Sewanee, she was editor of the Mountain Goat literary magazine and wrote prize-winning poetry.

Also named today as senior director of medical affairs was Maria Millan, who formerly was a

Maria Millan

medical officer at CIRM, which she joined in 2012. Prior to that, she was vice president and chief medical officer at StemCells, Inc., and director of the pediatric liver and kidney transplant program at Stanford, where was also an associate professor. Millan, a surgeon, is overseeing the agency's $34 million Alpha stem cell clinic program. 

Prior to Bonneville's appointment today as director of administration, she was executive director of the CIRM board. She will have oversight over the agency’s board relation, human resources, communications and information technology departments. Before joining CIRM, she was a consultant with Tramultola LLC and worked for former state Treasurer Bill Lockyer as finance director for Northern California.

Other members of the agency’s “leadership team” are Chila Silva-Martin, finance director, Gil Sambrano, director of review; James Harrison, general counsel; Gabe Thompson, director of grants management, and Patricia Olson, executive director of discovery(basic research).

All have been with the agency for some time. Harrison is an outside contractor.

Not including Mills, the 8-member leadership team at CIRM now consists of five women and three men. 

California's Stem Cell Program, Brain Cancer and the City of Hope

The City of Hope said yesterday that it has treated the first brain tumor patient at its state-financed Alpha stem cell clinic, part of an ambitious program aimed at creating four one-stop stem cell treatment centers in California.

The City of Hope announced that the unidentified patient was involved in a clinical trial using genetically modified neural stem cells to help deliver chemotherapy to brain cancer cells.

The press release said,

“The aim of this neural stem cell research is to develop a treatment that is more potent and less toxic than existing treatments for brain tumors.” 

 The Alpha Clinic program was created last October by the $3 billion California stem cell agency. It awarded $24 million for clinics involving UCLA, UC San Diego and UC Irvine in addition to the City of Hope, which is located in Duarte in the Los Angeles area.

Karen Aboody, co-leader of the Developmental Cancer Therapeutics Program at City of Hope, said,

Karen Aboody, City of Hope photo

  “Rather than putting chemotherapy through the whole body and possibly causing significant side effects that affect quality of life, the neural stem cells produce active chemotherapy only at the sites of the tumor, killing surrounding cancer cells.”

Maria Millan, who oversees the Alpha program for the stem cell agency, said,

 “This work does more than help just one person. Because they are part of the Alpha Clinics Network, City of Hope is demonstrating how by working together, providing collective expertise, efficiencies and critical resources, we can help accelerate the development of stem cell treatments for patients with unmet medical needs.”

 The City of Hope study now has several patients and is looking for more.­­­­­

Today is the Last Day to Weigh in on the Direction of the California Stem Cell Agency

Don't like what the California stem cell agency is doing? Want more clinical trials and less basic research? Do you think the agency is too cozy with business or not cozy enough?

Here is your chance to tell the agency directly what it should do. But today is the last day that you can participate in the survey of the public -- that includes researchers -- concerning the direction of the state's $3 billion enterprise.

The agency has mounted an online questionnaire that can be completed anonymously. It has 13 questions -- possibly the agency's lucky number -- with plenty of space to add comments.

The survey is part of the agency's revision of its plan for spending its remaining $800 million over the next few years.

"No one has a monopoly on good ideas," said Randy Mills, president of the agency, in soliciting comments from every quarter. Mills is expected to bring his latest thinking on changes in the agency's strategic plan to its governing board in late July.

The California Stem Cell Agency Celebrates a First

The California stem cell agency today marked a signal event in its most advanced clinical trial by declaring: 

“Some things never get old. Like watching the sunset over the Grand Canyon. Listening to a baby laugh. Watching the San Francisco Giants win the baseball World Series. Now you can add to that list learning that one of the clinical trials we are funding has just treated their first patient.”

­­­­­­­­

The piece by Kevin McCormack, senior communications director for the agency, referred to Caladrius Biosciences, Inc., which in May was awarded nearly $18 million by the agency. (Caladrius was formerly known as NeoStem.)

The money is going into a phase three trial for a treatment of metastatic cancer, the most lethal form of skin cancer. It is the first phase three trial in which the agency has participated.

McCormack wrote,

“Caladrius’ approach is a personalized one. They use the patient’s own tumor cells to create a therapeutic vaccine called (for now at least) CLBS20. It’s designed to engage the patient’s own immune system and destroy the cancer.

“This first patient was treated at Thomas Jefferson University Hospital in Philadelphia. Altogether Caladrius hopes to enroll some 250 patients at more than 40 sites worldwide, for the trial. Seven of those sites are here in California; that’s the portion of the project we are funding.”

Caladrius, which is headquartered in New York City, also issued a press release in which David Mazzo, CEO of the firm, described the enrollment of the first patient as a “milestone.”

Caladrius recently announced that it had “entered into a material transfer agreement with the University of Southern California and the California Institute of Technology, concerning next-generation strategies for its core cancer technology.”

The company’s stock today closed at $2.00, down 3.85 percent. Its 52 week low is $1.82. The 52-week high is $7.22.

The California Stem Cell Agency Wants Help in Charting New Directions

How to spend $800 million?

Friday deadline for comments

Want to tell the $3 billion California stem cell agency where to go? Here’s your chance.

The agency said today it wants to hear from you. It is open to suggestions, rude and otherwise. But it really wants to hear constructive comments and critiques.

“No one has a monopoly on good ideas,” said Randy Mills, president of the agency, in a statement that unveiled the enterprise’s most recent effort to elicit proposals and thoughts from the public.

What this is all about is revision of the agency’s strategic plan, which is an indirect way of asking the question of “how are we going to spend our last $800 million.”

The agency has mounted an online questionnaire with plenty of room for comments beyond the formal structure of the survey. Thirteen questions are posed. They include such matters as how much risk the agency should take and just how it should be measured. The agency wants to know whether the top three measurements should include such things as the number of scientific articles published or the number of patients treated. It wants to know whether you think the agency is sufficiently focused on its mission.

Time is short for persons who want to weigh in on the survey, which is designed to be answered anonymously. It must be completed by this Friday.

Jonathan Thomas, chairman of the agency board, noted that the effort is part of the agency’s responsibility to the public. He said,  

“We are a state agency. We were created by the people of California, and we answer to the people of California.”

 Mills said,

“That’s why we want to hear from a diverse group of people, scientists and non-scientists alike, to learn what they think about how we should best use our money, resources, and expertise to reach our goal. This new strategic plan will help create a clear vision for how we move forward, one that sets priorities and an actionable approach to accomplish our mission.” 

At the end of July the board is scheduled to hear what Mills has prepared up to that point on the strategic plan for the next five years or so, which is about how long the agency's current funding will last. 

The Hard Business of Stem Cell Economics and Patient Needs

Premature clinical trial shutdown?

Implications for California stem cell agency

The vagaries of Big Pharma and development of stem cell therapies -- plus the frustrations of a prominent Stanford researcher -- were the topic this week in a major California newspaper.

Irv Weissman, Stanford photo

The researcher is Irv Weissman, head of the stem cell program at the Palo Alto university. The companies are Sandoz Pharma, Ciba-Geigy and Novartis. The story also includes a $392 million deal involving Weissman.

The basic storyline, as reported June 14 by Lisa Krieger of the San Jose Mercury News, is that years ago Weissman developed a “unique way to grow and deliver blood stem cells to desperate patients with aggressive cancers, boosting survival rates.”

According to Krieger, Weissman in effect sold the potential stem cell therapy to Sandoz in 1991 for $392 million in an effort to place “his innovation into the hands of a company large and prosperous enough to accelerate research.”

Sandoz then “merged with Ciba-Geigy and became Novartis, which bought the remainder of Weissman's company (Systemix) for $76 million in 1997 -- and, with it, all patents,” according to Krieger.

Novartis shut down the trials in 2000 along with Weissman’s company. Krieger reported that the firm “ended the program because it couldn't produce blood stem cells in large enough numbers to develop a commercial market -- and it was not in the business of producing personalized ‘custom-made’ therapies.”

Krieger continued,

Karuna Jaggar, BCA photo

"'Well before the launch of the War on Cancer, the corporate profit motive has driven the cancer research agenda,' said Karuna Jaggar, executive director of the Breast Cancer Action, a San Francisco-based patient advocacy group. 'Time and again, history has shown that it is not only the possibility of saving lives but the potential of making money that has steered the research agenda toward some areas -- and away from others.

"'As long we have a health care system that puts profits before patients we will always be at the mercy of corporations looking to make profits. Any new innovation needs to be evidence-based and proven safe and effective no matter what money is to or is not to be made,' she said.”

Krieger wrote,

“Now, a quarter-century after it was conceived, the technique is finally back in Weissman's hands at Stanford -- although Novartis still holds the patent.” 

She reported that Weissman hopes to take the potential therapy forward in a non-profit setting.

Krieger has much more in her story, which offers considerable food for thought, particularly in connection with California’s $3 billion effort to bring a stem cell therapy into widespread use. It has not done so yet after nearly 11 years of trying and the expenditure of roughly $1.9 billion.  The agency also suffered a significant blow at the hands of one company, Geron, that dropped -- for financial reasons -- the first-ever clinical trial  for a hESC stem cell therapy, which the agency had backed with a $25 millon loan. 

The agency is currently revising its strategic plan. Randy Mills, president of the agency, says he is serious about developing a solid plan that will be followed assiduously with measurable benchmarks along the way.  

Our view: One important element in the new plan should focus on avoiding situations such as those involving Geron (see here and here) and the morass that Weissman fell into.

Whether one likes it or not, the facts of life in the biomedical world – pleasant or unpleasant – mean that business must be firmly engaged if a stem cell therapy is to be widely available to the public. Government agencies, however, often fall short in their dealings with private firms. For the stem cell agency, that means it needs a keen eye and clear goals when it deals with Big Pharma and stem cell firms. And if the California’s stem cell agency is to deliver on the promises made to the people of California, it must be willing to walk away from a deal if the terms aren’t right.