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.

California Hits Its Mark on Faster Delivery of Cash for Stem Cell Research

• Caladrius to get first installment on nearly $18 million 
• Agency President Randy Mills Pleased with CIRM 2.0 Overhaul

 SAN FRANCISCO -- The California stem cell agency may not be faster than a speeding bullet, but it is running way more quickly than it was a year ago.

For researchers and patients, that means the agency’s millions are moving more rapidly into development of therapies and cures with the goal of beginning to save lives sooner rather than later.

The signal event came last week when the agency, formally known as the California Institute for Regenerative Medicine(CIRM), signed off on a contract with Caladrius Biosciences, Inc., of New York.

As soon as Caladrius signs the agreement, it will receive a check for $3 million, the first installment on a nearly $18 million award that was approved by directors less than a month ago. 

Randy Mills

CIRM President Randy Mills was delighted last Friday when he told the California Stem Cell Report about the action on the Caladrius award. (Caladrius was formerly known as NeoStem.)

Mills, who has been head of the agency for only a little over a year, mentioned the news during a 45-minute conversation in his office at CIRM headquarters.

It was the first award paid out under Mills’ new, CIRM 2.0 program, an effort designed to speed cash to researchers and improve the quality of applications. It is also the first CIRM award in a stage three trial, the last step in the process of winning government approval for widespread public use of a therapy.

The agency approved the actual Caladrius contract last Thursday, 21 days after the directors’ approval. Mills’ goal was to act on the contract within 45 days.

CIRM’s 2.0 clock shows Caladrius’ application for the melanoma therapy coming in at the end of February, 113 days ago. Mills’ goal is to have action completed on an application within 120 days from the time it officially enters the 2.0 system -- instead of up to two years as in the past. 

The firm offered its initial application at the end of January, but it was sent back for improvement. 

Next up in the first round of CIRM 2.0 awards is final action on a $5 million award to Shaomei Wang of Cedars-Sinai that was also approved on May 21 by CIRM directors.

Mills said last Friday that CIRM 2.0 has not been perfect and that in some cases implementation required "brute force." But he is pleased overall. Later this year, Mills and his team plan to apply CIRM 2.0 to basic and translational awards, all of which will mean significant changes for hundreds of California scientists. 

California Stem Cell Agency Symposium: 'Vague Fears' vs. Potential Genetic Alteration of Human Race

California’s $3 billion stem cell agency has called a high-level meeting for next fall to examine a
"red-hot" issue that many prominent researchers say could lead ultimately to alteration of the human race.

The topic is a technique that makes it much easier to alter the genetic make-up of individuals in a way that can be inherited by their offspring.

In April, scientists in China reported genetically modifying human embryos in what was regarded as a “world first.” (See here and here.)

That came after a group of leading scientists in March called for a global moratorium on the genome-editing practice, stating that the therapeutic benefits were tenuous.

More recently Marcy Darnovsky, executive director of the Center for Genetics and Society in Berkeley, wrote on May 28 about how the subject was viewed at a meeting called, “Biotech and the Ethical Imagination: A Global Summit.”

Steven Pinker

“The far end of techno-enthusiastic perspectives, both on human germline modification specifically and on biotech in general, was represented by Harvard experimental psychologist and popular science writer Steven Pinker. In his opening remarks, Pinker counseled bioethicists to ‘stay out of the way of progress.’ In his closing comments, he cast ‘vague fears’ as standing in the way of saving millions of lives. In between, he epitomized the ‘bad boy scientism’ that too frequently characterizes the biotech field.” 

Jonathan Thomas, chairman of the stem cell agency, and Geoff Lomax, the agency’s main person on bioethics, wrote on June 1 about the matter and the upcoming meeting. In an item on the agency’s Stem Cellar blog, they said,

“Given CIRM’s support for research activities where genome editing may be employed, we believe it is important to have effective policies to guide our future funding decisions. Therefore, we have charged the CIRM Medical and Ethical Standards Working Group with convening a public workshop to elucidate the scientific and policy considerations surrounding genome editing in the context of CIRM supported research. This workshop is a first step in evaluating whether CIRM should make changes to its existing policies governing stem cell research.”

 CIRM’s standards group consists of some of the top bioethicists in the country. Early on, they developed the agency’s rules for research on stem cells, which were the most advanced in the country at the time. The chairman of the group is Bernie Lo of UC San Francisco.

The genome-editing issue to be discussed in November was originally scheduled to be brought up at an April meeting of the standards group. However, it was put off for a variety of reasons. Thomas subsequently called the meeting for next fall.

At the April meeting, Ted Peters of the Pacific Lutheran Theological Seminary and a member of the stem cell standards group, said the issue was “red hot.”  Jeff Sheehy, a member of the agency board and a communications manager at UC San Francisco, said the agency needed to clarify where it stood.  

Robert Taylor of the Emory School of Medicine and also a member of the standards group, said,

“There are companies that are offering to do this for your cell line, so it's really out there.”

Lo said,

“I think Jeff has raised a really important, complex and breaking topic. I think the idea of having a symposium to deal with both the science, and the ethics policy could be a really important thing to do.” 

 Suggestions regarding the meeting can be sent to Lomax at glomax@cirm.ca.gov.