Tuesday, June 30, 2015

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 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.­­­­­

Friday, June 26, 2015

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.

Wednesday, June 24, 2015

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.

Monday, June 22, 2015

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. 

Thursday, June 18, 2015

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.

Monday, June 15, 2015

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. 

Thursday, June 11, 2015

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.)

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

Tuesday, June 09, 2015

NeoStem's New Name, Backed by $29 Million in New Cash

The caladrius at work
Say hello to the latest recipient of nearly $18 million from the California stem cell agency -- Caladrius Biosciences, Inc.

It is also a firm that is $29 million richer -- on top of the $18 million -- than it was when the $3 billion state research enterprise awarded the firm the money on May 21.

Caladrius is the new name for NeoStem, Inc., of New York, N.Y.  It is also the name of a video game once described by the Giantbomb web site as a “A 2013 bullet hell shooter from Moss.”

Caladrius has a longer history, however, than the video game. According to The Medieval Bestiary web site, the caladrius is an all-white bird – sort of a mythological diagnostic tool – that would refuse to look into the face of patients who were going to die. But the caladrius could also draw out the disease from the sick person and fly away with it.

David Mazzo, president of the firm, said yesterday,
“With the same objective of patient recovery, we, now as Caladrius Biosciences, are committed to bringing significant life-improving therapies to market, driving the evolution of the cell therapy industry and generating industry-leading growth through our innovation and executional excellence.” 
Caladrius sold more than 14 million shares of its stock earlier this month, raising nearly $29 million to help pay for the third stage clinical trial that has been backed by the stem cell agency. The firm is using the proceeds for, among other things, to meet the required match of the California state funding. The four-year trial is aimed at metastatic skin cancer. 

In his letter to shareholders yesterday, Mazzo laid out the future direction of the firm. He also cited the acquisition of an “asset” a year ago that provided the therapy now being funded by California. Mazzo said,
“Given that we acquired this asset approximately one year ago and, in the ensuing months, much of the external focus on our company was on our ischemic repair Phase 2 program, we believe that the market has not yet integrated the full potential of this mature immuno-therapy asset into the value of our company.”
Mazzo referred to the $124 million acquisition of California Stem Cell, Inc., of Irvine, Ca. Hans Keirstead, the president of that firm, is now senior vice president, research, and chief science officer of Caladrius.

Caladrius’ stock closed at $2.21 a share today, down from $2.30 on May 20, the day before it was awarded the California money. The stock has ranged from $2.03 to $7.23 over the last 52 weeks, but it jumped dramatically the day following the award and then fell back.

The Caladrius video game figure is markedly different than the bird depicted by medievalists(see below). For a review of the game, see here.  
Caladrius Blaze, the video game figure

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