California Stem Cell Agency Cites "Real Progress" on Calimmune's HIV Clinical Trial

The California stem cell agency this morning announced that one of its early stage clinical trials has produced “encouraging safety data” in its search for a therapy that could eradicate AIDS.

The agency said in a press release the trial will now move forward to its next stage which will focus on efficacy. The gene-based therapy, dubbed Cal-1, was developed by Calimmune of Tucson, Az.

Randy Mills, president of the stem cell agency, said,

“While still early in clinical development this announcement demonstrates real progress towards this mission. The accomplishments of Calimmune’s team is a great example of how CIRM partnerships are working to impact patient’s lives today.”

Louis Breton, CEO of Calimmune, said that the safety benchmark achievement “is an important step in bringing this potential one-time therapy to the patients, and takes us closer to our ultimate goal of eradicating AIDS." (Here is the company press release.)

Kevin McCormack, senior director for public communications for the agency, said in an agency blog item that the therapy “takes blood stem cells from people who are HIV-positive, genetically modifies them so they carry a gene that blocks the AIDS virus from infecting cells, and then re-introduces the modified cells to the patient. The hope is that those stem cells will then create a new blood system that is resistant to HIV.”

Calimmune has a powerful scientific pedigree. Nobel Laureate David Baltimore, one of the firm’s founders, is chairman of the Calimmune board. He also once served on the governing board of the stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM).

The Calimmune Web site says that the company was formed in 2007 around stem cell technology discovered in the labs of Baltimore, while he was at Caltech, and Irvin Chen at the UCLA AIDS Institute in early 2000.  Delaware state records show that the firm was incorporated in 2006.

Baltimore served on the CIRM board from 2004 until June 6, 2007. He resigned from the agency board about 18 months before the application process began for the grant round that ultimately funded Calimmune. 

In addition to Baltimore, another member of the the Calimmune board is Harry George, managing general partner of Solstice Capital, whose Tuscon’s office shares the same address as Calimmune.

The trial, which is taking place in San Francisco and Los Angeles, is partly funded by CIRM, which has awarded Calimmune $8.3 million. Originally, the Calimmune research was part of a $20 million disease team grant involving UCLA. That was split, however. CIRM now lists the original award as a closed, $9.9 million grant shared by Calimmune  and UCLA.

Calimmune and the stem cell agency discussed the therapy at today's session of BIO2014 in San Diego, which is attended by about 15,000 persons in the biotech industry.

Here is a copy of the Calimmune contract with the stem cell agency. 

   California Stem Cell Agency Contract with Calimmune

Calimmune, the Berlin Patient and $20 Million from California Stem Cell Agency

UCLA Photo

An obscure Arizona company that is sharing in a $20 million HIV/AIDS research grant from the California stem cell agency has posted more details on the Internet about its origins and plans, which include a tie to the famous Berlin Patient.

The firm, Calimmune, Inc., disclosed that its chairman is David Baltimore, former president of Caltech, Nobel Laureate and former member of the stem cell agency's governing board. One of Calimmune's advisors is the German physician, Gero Hutter, who successfully treated the Berlin Patient, Timothy Ray Brown, the only person in the world who has been cured of HIV and AIDs and who now lives in San Francisco.

 In 2009, CIRM's governing board awarded a $20 million disease team grant to UCLA researcher Irvin Chen and Geoff Symonds, an Australian who is chief scientific officer of Calimmune of Tucson, Ariz. Earlier information on the Internet reported that Chen and Baltimore were co-founders of Calimmune.

Baltimore was not on the CIRM governing board at the time the grant was awarded. He resigned June 6, 2007.

Calimmune said on its website that its "singular purpose" is to bring "a one-time, cost effective therapy to the HIV community." According to the website, the firm was established "to develop innovative cell-based therapies for HIV" based on work from Chen's and Baltimore's labs.

Delaware state records show that Calimmune was incorporated in that state in 2006 although the company's website reports that it was founded in 2007. We have queried the company concerning the discrepancy.

Calimmune said it is seeking "to provide HIV-positive patients with a similar type of genetic resistance to HIV that occurs naturally in 1% of the European population.  By treating a patient's own stem cells and T cells (cells specifically targeted by HIV), we aim to protect patients from the ravages of AIDS and eliminate the need for daily medication." Essentially, that would replicate the case of the Berlin Patient.

The company appears to have strong ties to Johnson&Johnson, or at least its executives have had in the past. Symonds worked in Australia for the firm from 1992 to 2009. Calimmune's chief financial officer, Bhavin Raval, was finance director for J&J Research in Australia from 2002 to 2009. Louis Breton, CEO of Calimmune, told the California Stem Cell Report in 2009 that none of the firm's funding originated with Johnson&Johnson.

Breton has not responded to queries from the California Stem Cell Report since 2009 including queries during the past week. The unanswered questions involve the number of its employees, whether the firm has any business beyond the CIRM grant activities and the address of the location where the CIRM work is actually performed. CIRM cannot fund work that is performed out-of-state.

Melissa King, executive director of the CIRM governing board, told the California Stem Cell Report that the firm "conducts its laboratory research at leased lab space on UCLA's campus." It also has laboratories in Australia, according to documents on the Internet. King said Calimmune's "corporate address" is on Wilshire Boulevard near UCLA. The Wilshire address appears to be only an address for service of process, according to California state records. Calimmune's Tucson address is the location of an investment firm, based on a visit by the California Stem Cell Report to the location last fall. We were told at the time that Calimmune rents space in the office.

In 2009, CIRM's grant reviewers scored the Chen-Symonds application at 79. They had high praise for the research team, but one reviewer expressed reservations. The summary of reviewer comments said,

"….the resources and investigators are outstanding and the team is superb, both scientifically and in therapy development. The Disease Team comprises a collaboration between two complementary groups, one academic and one corporate. Each brings unique expertise to the project, with the academic group providing scientific know-how and proof of concept and the corporate group providing expertise in biologics development and commercialization. The team leaders are accomplished, highly productive investigators with a demonstrated track record in the field of HIV research, gene therapy, and/or clinical drug development. Key members of this team made the initial scientific observations leading to their hypothesis and demonstrated proof of concept in tissue culture and relevant models....The principal investigator has built up a rich and well-considered network of collaborations and resources and there is no doubt that the environment will be sufficient for this task."

CIRM also said,

"Another reviewer expressed the view that although the treatment will likely find a significant niche, the complexity, cost and potential toxicity of the approach will likely limit its use, even if successful."

A Look at Tucson's Calimmune and $20 Milllion

The global headquarters of Calimmune, Inc., which shares in a $20 million grant from the California stem cell agency, stands on one of the more unlovely thoroughfares in Arizona.

The three-year-old firm is only a short walk on East Broadway in Tucson from O'Reilly Chevrolet and a Burger King. While Calimmune's address may not electrify folks on Wall Street, it has a pedigree that it is hard to quibble with.

By one account, Calimmune was founded by David Baltimore (see photo), a former member of the board of directors of the $3 billion California stem cell agency. Baltimore also is a Nobel Prize winner and former president of Caltech. Other accounts state that Irvin Chen, director of the UCLA AIDS Institute, was a co-founder.

Late last month, California's stem cell agency approved a $20 million grant to Chen and Geoff Symonds, chief scientific officer of Calimmune, a little-known company with no Web site but a laboratory in Australia, lab space in Pasadena, Ca., and more facilities soon to come at UCLA.

CIRM said the grant was aimed at generating an FDA application in four years for a clinical trial “to treat HIV/AIDS using an RNA interference approach to modify the patient’s blood-forming stem cells. When transplanted back, those cells will produce T cells that are resistant to HIV infection.”

The proposal echoes Calimmune goals articulated earlier on the Web site of Grayhawk Capital, a private equity investment company in Phoenix, Az., that has cash in Calimunne. Some initial research involving Symonds and UCLA was published last February with partial funding from Johnson & Johnson in Australia.

In response to email queries, Calimmune's CEO, Louis Breton, told the California Stem Cell Report that Symonds will perform his CIRM-funded work in California. He said the closely held firm is aware that CIRM is barred from funding out-of-state grants. Breton did not respond to questions about the number of Calimmune employees and their locations, although he said he anticipated more hiring for the CIRM grant. He said the Tucson location houses a small administrative staff. The company is incorporated in Delaware.

As for Calimmune's funding, we asked Breton whether any originated with Johnson & Johnson. Until the last year or so, Symonds was senior research director at Johnson & Johnson Research Pty Ltd. in Sydney, Australia, where the Calimmune lab is located. According to one report earlier this year, he holds more than $10,000 in Johnson & Johnson stock. At least one of the other Calimmune scientists in Australia worked as well for Johnson & Johnson until February. Breton, however, said none of the company's funding originated with Johnson & Johnson. Grayhawk did not respond our email query.

A Johnson & Johnson company document said in 2008 that the purpose of the Johnson & Johnson research operation involving Symonds was “to identify new medical discoveries in Australia and facilitate their commercial development into new products for Johnson & Johnson.”

CIRM reviewers were enthusiastic about Chen and Symonds disease team project. A CIRM summary of the application said,

“Reviewers stated that the resources and investigators are outstanding and the team is superb, both scientifically and in therapy development. The Disease Team comprises a collaboration between two complementary groups, one academic and one corporate. Each brings unique expertise to the project, with the academic group providing scientific know-how and proof of concept and the corporate group providing expertise in biologics development and commercialization. The team leaders are accomplished, highly productive investigators with a demonstrated track record in the field of HIV research, gene therapy, and/or clinical drug development. Key members of this team made the initial scientific observations leading to their hypothesis and demonstrated proof of concept in tissue culture and relevant models. A subset of the team has direct experience with a gene therapy trial in humans.”

The Latest on Calimmune and California Cash

The story of Calimmune and a $20 million CIRM grant has a brief, new chapter this week – an installment that includes the word “mysterious.”

For those of you who may have forgotten, Calimmune, which is based in Tucson, is involved with UCLA in some CIRM-funded research. The young firm has connections to Johnson & Johnson, Australia and former CIRM director David Baltimore, a Nobel laureate. We carried an item with more details last November.

Yesterday, Paul Knoepfler, a stem cell scientist at UC Davis, carried an item dealing with Calimmune on his blog. The piece was part of an ongoing discussion about CIRM funding to businesses. CIRM is barred from funding research out-of-state, be it conducted by business or academia.

He wrote,

“David Jensen reported that Calimmune has lab space in Pasadena. The only reference to that that I can find is here, where it is mentioned as a company in the "Pasadena Biosciences Group", a group of tech companies sharing a building in Pasadena.

“Being somewhat puzzled, I inquired with CIRM about Calimmune and they responded that they have verified that Calimmune has a California facility.  So as far as I can tell, all companies with CIRM funding have ongoing science in California.

“Calimmune still seems mysterious to me. Perhaps the most unusual things about Calimmune is it has no website and there is nearly zero information on it on the Internet. No apparent publications. No press releases. No industry analysis. It has been referred to as 'tightly held'...I guess so!”

When we originally reported about Calimmune, Louis Breton, the president of the firm promised more details around the beginning of this year. Nothing was forthcoming at that time, and he failed to respond to an email we sent to him on March 9.

HIV Clinical Trial Hailed by California Stem Cell Agency

The California stem cell agency today scored what it called an “important milestone” with the announcement of the start of a clinical trial involving a therapy to help protect persons infected with HIV from the effects of the virus.

The trial is partially funded from a $20 million award from the stem cell agency, which is known as CIRM, to researchers at UCLA and Calimmune, a Tucson, Az., company. Calimmune's share of the award was $8.2 million.

Alan Trounson, president of the $3 billion state agency, said in a statement,

“CIRM funding of this Phase l/ll trial is an important milestone for us. One of our goals is to support research that moves the most promising science out of the lab and into clinical trials in people. To be able to do that with a disease as devastating as HIV/AIDS highlights the importance of our funding and the potential impact it could have on the health of people around the world.”

The trial was announced by Calimmune this morning. The company said,

“The first patient has begun treatment in a Phase I/II clinical trial designed to determine whether a pioneering genetic medicine approach can help to protect individuals infected with HIV from the effects of the virus. The study, “Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection,” utilizes a gene medicine called Cal-1, developed in the lab of Nobel Laureate Dr. David Baltimore and by Calimmune.”

Baltimore served on the CIRM board from 2004 until June 6, 2007. He resigned from the board about 18 months before the application process began for the grant round that ultimately funded Calimmune, a company he helped to found. He is currently chairman of the Calimmune board. 

Asked for comment, Jeff Sheehy, a member of the CIRM governing board and communications director for AIDS research at UC San Francisco, said,

"This trial will hopefully offer several important insights into the safety and feasibility of genetically modifying blood forming stem cells in an HIV patient as a potential therapy.  We are very early in this research, and with this Phase I trial's goal of establishing safety and the risks involved, I applaud the courage and altruism demonstrated by the patients who are willing to participate in this study."

The Calimmune press release said the principal investigators on the clinical trial are Ron Mitsayasu of UCLA and Jacob P. Lalezari of Quest Clinical Research of San Francisco. Quest is currently soliciting patients for the clinical trial as well as UCLA. (Persons interested in participating in the trial can find email contacts at this website. Twelve are needed.)

The principal investigators on the CIRM award are Irvin Chen of UCLA and Geoff Symonds of Calimmune.  

Here are links to the CIRM press release on the subject and the agency's blog item.

(An earlier version of this story did not include the fact that Calimmune's share of the CIRM award was $8.2 million or the links to the agency press release and blog.)

$15 Million for Calimmune and California Stem Cell HIV Trial

Calimmune, Inc., which has received $8.3 million from the California stem cell agency, today announced it has rustled up another $15 million to help out with the work that the agency is backing.

Calimmune of Tucson, Ariz., was co-founded by a former agency board member and Nobel Prize winner David Baltimore. It is currently engaged in a clinical trial in Los Angeles and San Francisco involving a treatment for HIV.

John Carroll, writing on Fierce Biotech, said,

“Calimmune hasn't had much of a profile outside of the HIV world, but (CEO Louis) Breton is looking to change that. The company has a staff of 40 now, he says, and is looking to expand and possibly strike a partnership deal with a Big Pharma in the space. And unlike some of the leading gene therapy companies in the industry which are targeting tiny populations, Calimmune is tackling a treatment for a disease with a huge, global population of patients. Discussions about million-dollar therapies, he says, won't work for something like HIV. 

"‘Our mission,’ says Breton, ‘is to provide broad capacity; democratize gene therapy for the masses.’"

Calimmune said the latest investors include a “large pharmaceutical company” and Alexandria Venture Investments of Pasadena, Ca. They are in addition to original investors, RA Capital Healthcare Fund LP and Translational Accelerator LLC.

Carroll is right about Calimmune’s low profile. From its earliest days, it has declined to disclose such things as the number of employees. The figure of 40 that was cited by Carroll was the first time it has been disclosed.

(See here, here and here for more information on Calimmune.)

Calimmune's HIV Clinical Trial and the California Stem Cell Agency: Decisions Looming on the State-backed Research

The HIV clinical trial involving Calimmune and the California stem cell agency appears to be headed today for several major decision points following the purchase of the company in a deal that could total more than $400 million. 

CSL Behring announced yesterday that it was acquiring the company. One published report said that CSL was not interested in the Calimmune HIV research, which has been funded with $8.3 million by the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. 

Responding to a query from the California Stem Cell Report, Natalie deVane, a spokeswoman for CSL, said today, 

"Our only comment regarding (the trial) was the following – 'We are currently evaluating our options for developing this pipeline candidate, which could include licensing or partnering. Given our areas of focus, it is unlikely that we will develop this candidate on our own.'

"We made no comments regarding timing, and at this point is would be premature to comment further."

Asked for a comment, Kevin McCormack, a CIRM spokesman said yesterday,

"Calimmune plans to complete the clinical trial that CIRM is funding, and CIRM has every intention of continuing the partnership with Calimmune in funding this essential research. Based on the findings of the trial, Calimmune hopes to make a decision as to whether or not it is feasible to move ahead with the next phase of this work."

The trial is scheduled to be completed in October.

$400 Million Deal Leaves California-backed HIV Trial Hanging

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

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

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

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

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

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

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

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

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

The California Stem Cell Campaign for $5.5 Billion More Mounts a Web Site, Hires PR firm

The ballot campaign to pump an additional $5.5 billion into California's stem cell stem cell agency is now moving briskly and has a web site plus a well-known public relations firm that has handled more than 20 other ballot measures in the Golden State. 

The campaign has also spent $1 million, which is a tiny amount given that the campaign could cost upwards of $50 million, give or take some millions. The latest campaign disclosure statement shows that it had a zero balance as of Dec. 31 last year. 

The state stem cell agency is running out of the $3 billion originally approved by voters via a 2004 ballot measure. If the new ballot initiative is not approved in November, the agency is expected to whither and die. Its only significant source of cash has been the $3 billion in state bonds. 

The campaign web site is called "Californians for Stem Cell Research, Treatments and Cures." It carries a list of 43 organizations that it says support the ballot initiative, which has not yet qualified for the ballot. 

The groups range from the Alliance for Regenerative Medicine, an industry lobbying group in Washington, D.C., and the Loving Mind Institute, which deals with mental and addiction issues,  to the Arthritis Foundation and the International Society for Stem Cell Research, the largest organization of stem cell researchers in the world. 

Also listed as supporters are patient advocates, scientists and private parties. They include luminaries such as Nobel Prize winner David Baltimore. He served on the board of the stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM) from 2004 until June 6, 2007. 

Baltimore was also a co-founder and chairman of the board of a firm backed with millions by the stem cell agency. The firm, Calimmune, was incorporated March 23, 2006, in Delaware.  The firm has received $8.3 million from CIRM. Calimmune's initial award came as part of a $20 million award on Oct. 28, 2009.   Calimmune was sold to CSL Behring in 2017 for $91 million. 

(See here, here and here for more on Calimmune.)

A number of current board members of the stem cell agency are also listed by the campaign as supporters. The board has not yet taken a formal position on the initiative, but there is little doubt that it will support the proposal. Other supporters include a number of researchers who have received CIRM funding. 

The campaign web site features "success stories" in CIRM program, all of which have been carried earlier on the official CIRM web site. The campaign site asks for donations as low as $5 via credit cards. It contains a list of five stories and columns that are favorable to the agency, covering the period from 2016 to last month.

The campaign has also hired a well-known California public relations firm, Fiona Hutton and Associates, which has offices in Los Angeles and Sacramento. Hutton was involved in the 2004 stem cell campaign as well. 

Hutton's main web page promises "communications that shake up things and move mountains."

The American Association of Political Consultants says that Hutton is "one of only two women-owned businesses ranked in the Top 10 of national public affairs agencies and the Top 10 Los Angeles-based firms by leading PR trade publication O’Dwyer’s."

CIRM's Trounson Has Month-Long Gig in Australia in July 2012

The Australian Centre for NanoMedicine has announced that the president of the $3 billion California stem cell agency, Alan Trounson, will be spending July 2012 as its "visiting professor."

The announcement apparently came as a surprise to some directors of the state's research program, which paid Trounson $490,118 in 2010. There was no date on the announcement but it appears to have been placed on the Internet sometime this spring.

The center is part of the University of New South Wales and was formed in April 2010. Trounson, who emigrated from Australia to take the CIRM post, is the second visiting professor in the program.

The center's announcement did not specify whether Trounson would be paid for his stint in Australia next year. Nor did it explain his exact duties. The center has at least one very slight connection to CIRM. Geoff Symonds, a longtime Australian researcher and chief scientific officer of Calimmune of Tucson, Az., made a presentation at the center's international conference last June 30-July 2. Calimmune, whose founders include former CIRM director, Nobel Laureate David Baltimore, shares in a $20 million grant with Irvin Chen of UCLA.

For more on Calimmune, see here and here.

We are querying CIRM for more information on Trounson's commitments in July 2012.

$28 Million California Search for HIV/AIDS Stem Cell Cure Advancing to Clinical Trial

The California stem cell agency today announced that one of its multimillion dollar efforts to come up with a cure for HIV/AIDS is moving into the first stage of clinical trials involving human beings.

The effort includes the City of Hope in Duarte, Ca., the University of Southern California and Sangamo BioSciences, Inc., of Richmond, Ca.

John Zaia
City of Hope photo 

The $3 billion agency is pumping $5.6 million into the clinical trial. Overall, the agency has awarded the lead scientist in the project, John Zaia of the City of Hope, $28.2 million for work involving HIV/AIDS. 

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), said the FDA had approved the initial trial to determine the safety of the treatment.

CIRM’s press release said that the plan is to “take blood stem cells from HIV infected individuals, then treat them with zinc finger nucleases (ZFNs), a kind of molecular scissors, to disrupt the CCR5 gene in those cells. The hope is that this will make those stem cells, and their progeny, resistant to HIV. The modified cells will then be reintroduced into the patient with the hope that they will create a new, AIDS-resistant immune system.”

The effort is intended to replicate what occurred with the “Berlin Patient,” a man who has apparently been cured of AIDS as the result of a mutation.

Jeff Sheehy, a longtime CIRM board member and HIV/AIDS advocate, said in the press release,

“This trial is enrolling HIV patients whose immune cells have not returned to normal levels even after success in suppressing the virus with antiretroviral therapy, and even if it doesn’t lead to a cure it could still result in a therapy that offers clinical benefit to patients at risk for opportunistic infections.”

(Here is a link to how Sangamo describes its ZFN pipeline.)

Zaia said,

‘While we have a number of drugs that are effective in holding HIV at bay, we have nothing that cures it. In addition, for many patients, these medications come with significant long-term problems so there is a real need for a therapy that can help eradicate the virus from a patient completely. That is where our work is focused.”  

The agency is also funding a clinical trial involving an alternative approach to HIV/AIDS. That effort includes UCLA and an Arizona company, Calimmune, Inc., co-founded by a former member of the CIRM governing board, David Baltimore. Calimmune also has an address near UCLA in Los Angeles.

CIRM said,

“Calimmune, an HIV gene medicines company focused on developing cell-based therapies for HIV, began its human clinical trial in July 2013 and has already shown that the first group of patients treated did well enough for the company to start treating a second group more intensively.” 

Jonathan Thomas, chairman of the CIRM board, said,

“This kind of work is too important to just try one method at a time and sit back and wait to see if it is effective.”

A $230 Million California Stem Cell Web

The publicity last week from recipients of $230 million from the California stem cell agency portrays an impressive web of scientists in the Golden State and beyond.

The list below began as a simple update of an earlier item with links to news releases from the beneficiaries of CIRM largess, in this case the agency's largest ever research round. But, as we gathered the information, what became abundantly clear are the significant financial and scientific ties between institutions that might seem to be rivals under other circumstances.

No doubt they continue to compete in other areas and will be competitors in the future for grants, talent and more. But in this case researchers put together powerful teams that won tens of millions of dollars for themselves and their institutions.

The releases from the institutions contain more information about the researchers and their projects than is contained in CIRM's press release on the disease team grants. But CIRM also provides separately summaries of the scientific reviews of the applications, which carry analysis, criticism and praise of the proposals.

Here is the latest list of news releases issued by the institutions and businesses.

Beckman Institute, shares $15 million with City of Hope and USC

Burnham Institute, no news release available, shares $19 million with UC San Francisco and Ludwig Institute

Calimmune, Inc., of Tucson, Az, no news release available, shares $20 million with UCLA

Cedars of Sinai Medical Center, $6 million

Children's Hospital, Los Angeles, no news release available, shares $9 million with UCLA

City of Hope, $18 million and shares $15 million with USC and Beckman Institute

Ludwig Institute for Cancer Research, no news release available, shares $19 million with UC San Francisco and Burnham, $16 million with Salk and UC San Diego

Novocell, shares $20 million with UC San Francisco

Salk Institute. shares $16 million with UC San Diego and Ludwig Institute

Sangamo Biosciences, Inc., of Richmond, Ca., shares $15 million with City of Hope

Stanford, $32 million, and shares $20 million with UCLA and USC, $20 million with UCLA

UCLA, shares $20 million with Calimmune, Inc., of Tucson, Az.; $9 million with Children's Hospital, Los Angeles; $20 million with Stanford and USC; $20 million with Stanford

UC San Diego, $20 million , shares $16 million with Salk and Ludwig

UC San Francisco, shares $39 million with Ludwig Institute, Novocell and Burnham

UC Santa Barbara, shares $16 million with USC

The international partners listed in the CIRM news release are the Medical Research Council of the United Kingdom($8 million via the Weatherall Institute of Molecular Medicine, Oxford University, no news release available from Weatherall); the University Health Network ($35 million)of Toronto, Canada.

Here is a link to CIRM's disesease team press package, including a video of the lengthy news conference. Only one reporter from a major publication was present at the news conference, Andy Pollack of the New York Times. No television stations sent crews.

(Editor's note: The earlier item that we posted concerning the institutional press releases has vanished from this Web site for reasons probably only fully understood by Google, which provides the blogging service that we use. Also, an earlier version of this item incorrectly said Pollack was the only reporter present at the news conference. CIRM reports that two other reporters from local outlets were on the scene.)

"Comfort News" for California's Stem Cell Research Effort

The California stem cell agency has enjoyed a spate of good financial and scientific news this week from the biotech industry as the research effort pushes on with its mission of turning stem cells into cures.

The $3 billion agency is scheduled to make its last grants in less than three years and, given the glacial pace of medical research, needs all the help it can get by then to bring a stem cell therapy close to the marketplace – the promise it made to voters when the agency was created nine years ago.

CIRM, as the agency is known, requires not only steady scientific progress but also a rosy outlook for the industry, which has languished in past years as major investors shunned the field. This week, CIRM garnered good news on both fronts.

There was enough so that the agency even touted it on the agency's research blog in an item by Neil Littman, CIRM's business development officer. He said it all helps to leverage CIRM investments and create a favorable investment climate. The good news included yesterday's announcement that Viacyte, Inc., of San Diego, Ca., has come up with $10.6 million needed to match a $10.1 million, much-ballyhooed award from CIRM last fall. The Viacyte financing includes important support from Big Pharma, in the form of Johnson & Johnson. CIRM has pumped a total of $39.4 million into Viacyte.

Another CIRM award winner, Cellular Dynamics International, Inc., of Madison, Wisc., yesterday announced its price on its upcoming stock offering to raise up to $53 million. Cellular Dynamics scored $16 million from the agency last March.

The “comfort news” for CIRM also included Monday's announcement that Capricor, Inc., a private Beverly Hills company benefiting from $27 million from the California stem cell agency, is merging with publicly traded Niles Therapeutic, Inc., of San Mateo. The merger is aimed at providing better access to capital.

And then there was Tuesday's news that a $20 million CIRM disease team award is paying off with the beginning of a clinical trial by Calimmune of Tucson, Az. for an HIV treatment.

All on top of the news in June when bluebird bio of Masschusetts brought in $101 million on its stock offering. Bluebird is the recipient of a $9.4 million CIRM award.

The rosy news comes amid a generally better outlook for biotech in general. John Carroll, editor of Fierce Biotech, this week noted that there were only 11 biotech stock offerings last year. He wrote,

“In the last 6 months, though, the industry has seen a tremendous rebound, with almost twice that number of IPOs in half the time. And there's no sign that the great leap into the public market is waning, with 10 more IPOs in the queue.”

Carroll's comments were echoed in a piece by Peter Winter on Bioworld headlined “Bubbleology and Biotech's Bull Run.”

All of this plays into what some might call the “everybody's-doing-it dance" or the “lemming syndrome,” depending on your point of view. The reality is that big investors and venture capitalists are timid souls and need the comfort of companionship-in-risk as they fork over tens or hundreds of millions of dollars on something that may not pay off for a decade or more. No one wants to be the out-front pioneer who winds up with financial arrows in his or her back. Being in a crowd provides an illusion of safety.

Of course, there is always the caveat about how markets and investors are fickle. A piece of bad news can translate quickly into major reversals as Apple has learned over the last year. Nonetheless, the folks at the stem cell agency have to  be feeling good today.

Trounson Talks Stem Cells in Qatar

Qatar Conference Center

If our readers in the Middle East are looking for a first-hand assessment of the state of stem cell research, they might want to take in the four-day conference this week in Qatar, which features the president of the $3 billion California stem cell agency.

Alan Trounson is one of a number of international stem cell notables at the session at the new Qatar Conference Center in the tiny nation in the Persian Gulf. The country is putting on the conference as a means of developing its own stem cell research capabilities.

Qatar had a gross national product of $129 billion in 2010, with a per capita income of $138,000, according to the U.S. State Department. The population is about 1.7 million, more than 75 percent of whom are foreigners with temporary residence status.

In addition to Trounson, other California and CIRM-connected researchers are speaking at the conference in the Qatar center, which just opened in December.  They include David Baltimore, Nobel Laureate and a former director of the stem cell agency. A company Baltimore co-founded, Calimmune, of Tucson, Az., is sharing in a $20 million CIRM grant. Other CIRM grant recipients or representatives of recipient companies appearing at the conference are Irv Weissman of Stanford; Deepak Srivastava of the Gladstone Institute, and Ann Tsukamoto Weissman of Stem Cells Inc. of Newark, Ca.

Social activities at the conference include sand dune "bashing" in off-road vehicles, camel tracking along with a look at their "robot jockeys" and a visit to the original Arabic Oryx farm.

The California Stem Cell Agency Numbers

​For today’s 10-year anniversary piece in The Sacramento Bee on the California stem cell agency, the agency was asked for statistics and numbers that would be useful in illustrating what it believes is the progress of the program.

The freelance article was written by David Jensen, publisher of this blog. Given the space limitations of the print media, the California Stem Cell Report is carrying all of the information provided by the agency.

Kevin McCormack, senior director for communications, supplied the following, which has been edited slightly for clarity.  

“One (graphic) is a pie chart that breaks down all the different disease areas where we are funding research at the most advanced levels, the pre-clinical (or translational as the graphic calls it) and the

clinical trial level (shown at right).  As you can see this covers 80 different programs and involves $627 million in funding. Obviously a lot of other money goes to fund other projects such as basic research, developing tools and technologies, etc., but I think this is a graphic that people will be interested in because it highlights the areas where we are closest to developing treatments for patients.

“For other numbers here are some that might be useful: 

“More than 1,750 published discoveries

“Trained more than 1,500 students for future careers in stem cell research

“Lured more than 130 senior scientists, and their teams, to California

“Research facilities helped leverage $543 million for the state 

“Generated more than $280 million in new tax revenues by the end of this year

“Generated more than 38,000 job years by end of 2014

“The second item is our Ten @ Ten list (10 clinical trial investments after 10 years) which shows the ten clinical trials we're funding right now. It's actually a little (off) because we have only been funding research for seven years – it took a couple of years after Prop. 71 was passed for us to get through the legal challenges and set up the infrastructure for the agency, etc., before we began funding – but Ten @ Ten has a much nicer ring to it. 

“HIV, Calimmune 

“Heart disease, Capricor 

“Solid Tumors, UCLA 

“Leukemia, UCSD 

“Sickle Cell Anemia, UCLA

“Solid Tumors, Stanford

“Diabetes, Viacyte

“Spinal Cord Injury, Asterias

“HIV, City of Hope

“Blindness AMD, USC

“We also funded research that led to six other clinical trials and you can find out information about them here: 

http://www.cirm.ca.gov/our-progress/cirm-funded-clinical-trials

“In terms of money there are probably a few things to mention, and as always it gets a little complicated. So far the Board has awarded $1.9 billion in funding but only $1.4 billion has actually been spent. When you talk about our accomplishments to date, as I am sure you will, the $1.4B is the most accurate number to use because it reflects what has gone out the door and been used for research. 

“We also estimate that we'll be able to recover around $100 million of the money that has been awarded through cost savings on projects and on projects that have been cancelled because they failed to meet their milestones. So, if you add that to the $843 million that we still have left to award that means the Board has close to $1 billion left on hand for funding. And that means that, at our current rate of spending, we'll be able to continue funding new projects until 2020, and because those are multi-year awards we'll be continuing to supervise that funding for several more years after that date.”

Hope and Home Runs: Nature and the Chron on the California Stem Effort

It wasn’t exactly a stem cell tag team, but both the journal Nature and the San Francisco Chronicle this past week carried lengthy pieces examining the state of affairs at California’s $3 billion stem cell agency.

 Neither of the articles was a valentine, but overall the agency should be satisfied with them -- if only for the reason that they will make more people aware of what the agency is doing. The agency received something of a cyber bonus with a video on the Nature Web site that accompanied its online article(see video above). Coverage of the agency, especially in the mainstream media, has been minimal over the last several years.

 Meanwhile the agency has been trying to spread the word as it tries to fend off its financial demise in less than three years, when funds for new research awards run out. 

Both pieces covered familiar ground for readers of this Web site. And both emphasized the looming financial crunch for the agency, which is examining the possibility of some sort of private-public financing arrangement. The agency has not ruled out another bond measure to secure voter approval for as much $5 billion in additional financing. Neither article discussed the likelihood of voter approval, which is problematic. 

Both pieces took a run at providing summaries of the work that the agency has financed and its contributions. Both noted that the agency has yet to produce a stem cell therapy despite the apparent promises of the ballot campaign 10 years ago that created the agency. 

Erika Check Hayden, who wrote a Nature overview of the agency in 2008, was the reporter again on the latest article on July 2.  It was headlined, 

"Stem cells: Hope on the line"

The subhead said,

“A decade ago, voters in California changed the biomedical research landscape by directly funding embryonic stem-cell research. Now the organization they created needs a hit to survive.”

Hayden wrote,

“The institute has navigated a difficult path, however. CIRM had to revamp its structure and practices in response to complaints about inefficiency and potential conflicts of interest. It has also had to adapt its mission to seismic shifts in stem-cell science.

A "home run" is now in order in the words of agency board member Sherry Lansing, the Nature article said.

Hayden interviewed former agency chairman Robert Klein about his plan for a $5 billion bond measure, perhaps in 2016. She quoted Klein as warning that the nation and California could see a “theocratic government” in the near future that would endanger research. Hayden wrote that Klein said,

“We have to protect science's access to the full range of cellular types now. And in doing that, we will protect the freedom of science to ethically pursue knowledge in this country outside of religious ideology.”

Klein also said,

“If we don't take a position now the next ten years may see a theocratic government at the state and federal level that restricts scientific research in this country for the next 50–100 years.”

As for the agency’s accomplishments, Hayden wrote,

“(I)n California, researchers are making nerve, heart, eye and skin cells from iPS cells and embryonic stem cells — a range of work rare for a single state — and they aim to test many of these in humans. They are developing drugs against cancer stem cells, which are thought to perpetuate the disease. And they are leading the world's only two trials of treatments that combine gene editing and cell therapy to treat HIV. They are doing all this with an unmatched infrastructure, including a network of 12 new or newly renovated facilities, and a funding pipeline that acts as a beacon to young scientists.

“Almost every country would be jealous of what they've got in California,” says Christine Mummery of the Leiden University Medical Center in the Netherlands.”

Mummery is a member of the agency's scientific advisory board.

Stephanie Lee was the writer on the San Francisco Chronicle story that was on the first page of the business section on July 5.

It was headlined,

“Stem cell researchers under pressure to produce”

The headline was drawn from this paragraph by Lee,

“Fund recipients are under pressure to show results - commercially viable therapies, ideally. Universities and other nonprofit groups have received most of the money, but the pressure is especially heavy on biotechnology companies that have staked their livelihoods on such therapies."

Lee mentioned StemCells, Inc., (along with photos at the company’s lab), Asterias, Capricor, Calimmune and ViaCyte as companies that are making progress.

 She continued,

“As (clinical) trials add more patients, they become more expensive. The challenge in the future will be to find money to keep these trials going when the stem cell agency runs out of money.

"’It's certainly not going to make life easier,’ said (Martin) McGlynn (CEO) of StemCells, which reported a $28 million operating loss last year."’To their credit, the California voters stepped up to the plate, and $3 billion is a lot of money,’ he said. ‘But an awful lot more is going to be needed to finish the job.’"

Lee continued,

“It is not realistic to expect a stem cell therapy to reach consumers in five years, especially when the field is so young, said Enal Razvi, managing director at Select Biosciences, a life sciences market research company.

"Even so, by 2017, ‘if they don't have products already on the marketplace, they should not be expecting public money to fill their portfolio,’ he said. ‘Apple doesn't go to California and keep asking for money to build their own iPhone.’"

California Stem Cell Agency Pulls $19 Million Grant

The California stem cell agency has terminated a $19 million grant to a UC San Francisco researcher involved in the agency's ambitious attempts to push stem cell therapies into clinics.

The agency said the research effort led by Mitchel Berger, chairman of the department of neurological surgery at UCSF, "did not meet a go/no-go milestone" stipulated in the grant. His research was funded in 2010 to treat brain tumors with genetically modified neural brain cells. No further explanation for the termination was provided by CIRM in a report prepared for tomorrow's meeting of the CIRM governing board. The agency estimated the cancellation would save $13 million.

The California Stem Cell Report has asked Berger and his co-PIs for comment on the CIRM action. The other researchers are Evan Snyder of Sanford-Burnham and Webster Cavanee of the Ludwig Cancer Institute. Their remarks will be carried verbatim when they are received.

The CIRM action was disclosed in the progress report on the $230 million disease team effort launched by the agency in 2009. The amount climbed to more than $250 million with contributions from partnering countries. Three of the 14 funded applicants – Irv Weissman and Gary Steinberg, both of Stanford, and Karen Aboody of the City of Hope – were approved only after they appealed to the CIRM board to overturn rejections by grant reviewers. (See  here , here and here for their written appeals. See here and here for coverage of the 2009 board action.)

One other disease team grant was modified to limit its scope and revise its funding. No savings were announced by CIRM. The PI on the $20 million project is Dennis Carson of UC San Diego. Co-PIs are Catriona Jamieson, also of UC San Diego, and John Dick of the University Health Network of Canada. The research is aimed at leukemia.

The actions on the disease team grants were not entirely unexpected. From their inception, CIRM directors have been told not to expect all the grants to finish successfully.

Ellen Feigal, senior vice president for research and development at CIRM, prepared the 19-page update on the disease team efforts. The grants are aimed at generating an investigational new drug application with the FDA within the four-year term of the grant.

She said that the funding decisions were made following evaluation of the projects by panels of clinical development advisors. Their recommendations were then considered by CIRM staff.

Feigal's report laid out accomplishments of the research so far and discussed changes in direction.

She said two companies have been formed since the grants were awarded to commercialize the hoped-for products. She said that in June 2011 Aboody founded TheraBiologics Inc., Newport Beach, Ca., of which she is chief scientific officer and director. Another company, Regenerative Patch Technologies, Glendale, Ca., was created by the team working on an hESC treatment for age-related macular degeneration. That $16 million grant involves Mark Humayan and David Hinton of USC, Dennis Clegg of UC Santa Barbara and Peter Coffey, formerly with University College, London, but now at UC Santa Barbara. The effort has generated seven patent filings.

The Feigal update also discussed the efforts of companies involved in other disease team grants. The lack of CIRM funding for biotech firms has been a bone of contention with industry and troublesome for some CIRM directors.

CIRM indicated the projects involving the firms were moving on schedule with no major difficulties reported. The companies involved are ViaCyte of San Diego, Calimmune of Tucson, Az., and Sangamo Inc. of Richmond, Ca.