Friday, September 22, 2017

Ballot Measures to Mergers: California Stem Cell Agency Examines Alternatives to Its Demise

Discussing the future of CIRM on Monday: Left to right, Chair Jonathan 
Thomas,  Vice chair Art Torres, Director Diane Winokur. Photos behind
 them are  of  persons helped by the agency's clinical trials. 
Photo by The California Stem Cell Report.
OAKLAND, Ca. -- Directors of California's $3 billion stem cell research effort are looking at ways to stave off the agency's death ranging from asking voters for billions more to being acquired by some sort of private entity.

The choices came before a new Transition Subcommittee of the agency's governing board last Monday. It is considering options as the money runs out for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Current projections estimate that cash for new awards will end in mid 2020. However, directors could alter the award rate and survive longer. That would shrink the flow of cash to possibly hundreds of researchers from Sacramento to San Diego.

CIRM's funds come from money that the state borrows. The ballot initiative that created it in 2004 provided for $3 billion in state bond funding but no other significant revenue. The agency has roughly $650 million remaining.

No directors at the meeting expressed support for simply letting financial nature take its course and permitting the agency to slowly expire.

One option that seemed to attract significant interest would call for the agency to ask the legislature and the governor to place another multi-billion dollar ballot measure before voters in 2020. That option would involve the governor who succeeds Jerry Brown in 2019.  Brown is wary of adding any height to what he calls California's "wall of debt."

Such an option requires a two-thirds vote of both houses of the legislature and the signature of the governor. (A CIRM memo on legislative options incorrectly said that only a majority vote was needed. It was corrected during the meeting.)

One possibility would involve another ballot initiative, a process that would not need approval of the legislature. However, under new state law provisions, the legislature is required to hold hearings on ballot initiatives. Such a process could result in changes in a proposed initiative.

CIRM directors seemed to acknowledge that either going to the legislature directly or using a ballot initiative would likely mean significant changes involving the agency. Director Steve Juelsgaard said the result could be a "very different CIRM."

The options considered this week also included private fund-raising. Some directors indicated that raising $200 million to $300 million a year was not entirely realistic. However, some combination of fund-raising and public support was also a possibility.

The last-ditch option involved acquisition of the agency by another enterprise including possibly a venture capital driven entity.  CIRM Chairman Jonathan Thomas said a possibility could involve an organization such as the Gates Foundation or the Wellcome Trust. He said California has a "tremendous asset" in CIRM and a "ton of IP(intellectual property)."

Director Jeff Sheehy said he had "never heard of a state agency that was merged or acquired."
"I wouldn't put my head in that noose," Sheehy said.

Thomas said the next step will be to hold a joint meeting of the directors' Science and Transition Committees in November and take the resulting recommendations to the full board in December. Sphere: Related Content

Wednesday, September 13, 2017

California Stem Cell Agency: $20 Million Cancer Award from 2012 Bears Clinical Trial Fruit

California's stem cell agency this week took the relatively unusual step of a firing off a double-barreled salute to the beginning of one of its clinical trials -- a $20 million effort based out of UCLA.

Antoni Ribas, UCLA photo
The press release and blog item heralded research by Antoni Ribas, who received the award in 2012 from the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

The potential treatment deals with the spread of cancer to other
parts of the body, which the agency said is responsible for 90 percent of cancer deaths. Ribas' trial will test a stem cell-based treatment that is better at tracking down and destroying hard-to-treat cancer stem cells. In a news release from UCLA, which is also funding the research, Ribas said,
"(F)ew options exist for the treatment of patients whose cancers have metastasized due to resistance to current therapies. This clinical trial will allow us to try a new approach that engineers the body’s immune system to fight metastasized tumors similar to how it fights germs and viruses.”
Siwen Hu-Lieskovan,
co-investigator, UCLA photo
Maria Millan, interim president of the stem cell agency, said,
“Backed by rigorous science, the stem cell derived T cell immunotherapy being developed by Dr. Ribas and his team has the potential to address advanced cancers that have spread beyond the primary tumor and which are associated with very low survival.
"This trial is the first step in developing a therapy that could alleviate the complications resulting from cancer metastases as well as potentially improving outcomes in cancer patients where there are currently no effective treatment options. We are confident that CIRM’s funding and partnership, in combination with the expertise provided by our Alpha Stem Cell Clinic network, will give provide critical support for the successful conduct of this important clinical trial.”
The $3 billion stem cell agency is actively supporting 33 clinical trials (five involving cancer), which are the last stage prior to approval of a therapy for widespread use. Why the CIRM salute in this case?  The blog item by Todd Dubnicoff said,
"The reality is this: the launch of a clinical trial isn’t a beginning. It represents many years of effort by many researchers and a lot of funding to take an idea and develop it into a tangible product that has been given clearance to be tested in people to potentially save their lives. That’s why this important milestone deserves to be recognized.
More information on the award (DR2A-05309) can be found here. Persons interested in participating in the trial can contact Clinical Research Coordinator Justin Tran by email at or by phone at 310-206-2090.
Sphere: Related Content

Monday, September 11, 2017

Curtailing Research Awards and Other End-of-Life Matters at California Stem Cell Agency

If you are interested in whether the $3 billion California stem cell agency is going to live or die, you may want to check in on a meeting one week from today.

A new committee of the agency's directors that was formed to deal with transition issues is scheduled to meet for three hours next Monday to consider various scenarios and how the agency might deal with them.

The impetus for the meeting is a projection that it will run out of cash for new awards in mid 2020 with no funding  in realistic sight.

One of the possibilities for extending the life of the agency is to curtail its award programs, which could possibly give the agency another one or two years of existence. The California Institute for Regenerative Medicine, as the agency is formally known, has roughly $650 million left but has been taking on clinical trials, some of which have been running $20 million a pop.

The session will be based out of the agency's headquarters, but teleconference locations are listed in Los Angeles, Duarte and La Jolla. The public can ask questions or make statements from those locations in addition to the main site in Oakland. Listen-only access is also available on the Internet. Full instructions and addresses can be found on the meeting agenda. Sphere: Related Content

Wednesday, September 06, 2017

San Francisco Business Times: Can Clinical Trials Save California's Stem Cell Agency?

Tippi McKenzie on front page of San Francisco Business Times

Long, difficult and expensive -- that's how the San Francisco Business Times describes stem cell therapy development in an article about the 33 clinical trials currently backed by the $3 billion California stem cell agency.

Will it all prove to be worthwhile? Reporter Ron Leuty addressed the matter in an Aug. 25, front-page piece that featured Tippi McKenzie of UC San Francisco. She recently received a $10.9 million award from the California Institute of Regenerative Medicine (CIRM), as the agency is formally known, to test her possible therapy on 10 pregnant women.

In what will be the first clinical trial of an in-utero blood stem cell transplant, McKenzie will transplant a mother's own stem cells into her fetus. Leuty wrote that it could be a precursor to pre-birth cures for other blood diseases, such as sickle cell disease.  He continued,
"It’s also the sort of high-profile medical breakthrough that could give the state stem cell funding research agency momentum as it prepares to (possibly) seek billions in fresh money to extend its life beyond 2020."
Leuty wrote,
"Still, any potential treatments are years into the future. Are trials such as MacKenzie’s too little, too late to help CIRM? "  
"It’s all an illustration of the long, difficult and expensive path for bringing any drug to market. Only four of every 10 late-stage trials succeed, according to a 2016 Biotechnology Innovation Organization study of clinical trials from 2006 to 2015. As a relatively new field, in which regulators and companies are seeking a new pathway to Food and Drug Administration approval, stem cell therapies take eight years to translate into clinical trials; non-stem cell therapies take 3.2 years, according to CIRM Chairman Jonathan Thomas. That’s a potentially awkward narrative as efforts ramp up to keep CIRM alive longer. "
Leuty said,
"CIRM can point to 33 projects that have reached clinical trials. For example, an $11.9 million grant to UCSF and St. Jude Children’s Research Hospital is paying for a mid-stage study in 'bubble boy disease.' The agency wants to fund another 40 clinical trials over the next four years, and it is pitching that scale and scope as a reason to keep CIRM going."
The article continued,
 "If anyone has a chance of showing CIRM’s value, it may be MacKenzie, an associate professor of surgery at UCSF’s pediatric surgery unit and head of its Fetal Treatment Center. Her lab since 2010 has been awarded $14.9 million in grants, including cash for basic research, translational research and now in the clinic. 
"CIRM is about more than funding for studies, MacKenzie said. Agency staff has provided advice about how to translate animal studies into work in humans, she said, as well as hiring an FDA consultant, writing an investigational new drug application and setting up a clinical protocol. 'I’m a clinician, but running a clinical trial is different,' MacKenzie said. 'CIRM’s been incredibly helpful in helping me navigate that.'"
Sphere: Related Content

Wednesday, August 30, 2017

The Dual Images of Stem Cell Medicine Highlighted in the Headlines

Google news stem cell headlines this week
A couple of notable headlines surfaced this week dealing with stem cell matters, capturing something of the dichotomy in a field that has been heralded for its possible production of miracle cures. 

One story told of a person who plays football for a living, Doug Baldwin, who is known as the $46 million man.  He decided to spend some time in England this summer receiving what he believes was a stem cell treatment to prevent his knees from deteriorating. It was the type of story that gives many others hope that some sort of stem cell therapy could cure a serious or fatal condition despite the fact that no such cure has been approved for widespread use in this country. 

The other story told of a modest crackdown by the Food and Drug Administration (FDA) on a couple of unregulated stem cell clinics in this country, a move that many believe was long overdue. More than 500 such clinics exist in the United States.  One of the California clinics identified by the FDA was using a restricted small pox vaccine as part of its treatment. 

Your average health care consumer could justifiably wonder, "What in the world is going on? Is this stuff safe or what?" Some scientists have been long frustrated by the dueling public faces of stem cell medicine. On one hand, stories of miracle cures involving professional athletes generate great attention and tend to create faith in the efficacy of stem cell treatments, although the therapies involved do not measure up to scientific standards. On the other hand, the FDA is now taking a more active role in policing dubious actors in this business.

Our average medical consumer might believe that the FDA action signals a new assurance of stem cell safety or is it the other way around? Is the stem cell glass half full or half empty?

Paul Knoepfler, a stem cell researcher at UC Davis, has been the bellwether for seven years regarding news about unregulated and dubious stem cell clinics. He once even went sort of undercover to attend a public marketing session produced by one clinic in the Sacramento area. 

Knoepfler wrote this week on his blog about the FDA action, its implications and the questions that remain. He said the FDA action was a "very big deal." 

But Knoepfler said six important questions remain:
  • "How much further will the FDA go with actual enforcement actions?
  • "Will the FDA define fat stem cells as a drug (or not) in upcoming guidances?
  • "How will the FDA handle non-homologous use of bone marrow cells in the commercial sector?
  • "Will the FDA work to deal with the growing problem of unapproved, amniotic stem cell offerings? 
  • "What about networks of stem cell businesses?
  • "Will other entities like state medical boards use this FDA action as a spring-board to get off the sidelines and take positive action too?"
Meantime, patients will continue to have to wade through conflicting claims, hype and the possibility of serious medical harm or death as they ponder whether they should undergo a stem cell treatment. 
Sphere: Related Content

Tuesday, August 29, 2017

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 CSLsaid 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. Sphere: Related Content

Monday, August 28, 2017

$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.

Sphere: Related Content