Wednesday, October 28, 2015

Need Work? The California Stem Cell Agency May Want You

Looking for a good job with an 11-year-old, but still fast-moving, cutting-edge organization?

The $3 billion California stem cell agency is looking for five folks who are willing to work in its new offices in Oakland and help turn stem cells into cures.


The director/associate director position carries a salary of up to $250,065. Title and compensation will vary depending on the qualifications of the individual.
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Friday, October 23, 2015

The Niche Rises -- Sort of Phoenix-like -- in California

California and the world has a “new” stem cell blog – at least new in name.

Welcome to The Niche (with an upper case T).

Paul Knoepfler, UC Davis photo
It is a rebranding of ipscell.com, the five-year-old effort by UC Davis stem cell researcher Paul Knoepfler.

He announced the change yesterday. Knoepfler wrote,
 “The Niche name better represents the sense that this site is a home and community including contributions by other writers and also commenters. I’m still working on a logo for The Niche.
“As The Niche continues to evolve look for it to include a wider range of contributors. As you may have noticed we have an increasing number of posts on genetics and genetic modification as well in addition to stem cells and regenerative medicine.”
The Niche, as some may recall, was the name of an excellent blog mounted by the journal Nature a few years back. It was shuttered by Nature for what must be presumed to be financial reasons.

Monya Baker and Natalie DeWitt were the overseers of that effort. Baker remains with Nature. DeWitt moved on to the California stem cell agency where she was a top aide to its then president Alan Trounson. She left the agency in June 2014 for Stanford and has been with NDA Partners LLC of San Francisco for the past five months.
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Thursday, October 22, 2015

Speedy Session Today for Directors of $3 Billion California Stem Cell Effort

The $3 billion California stem cell agency today whipped through a 28-minute meeting that featured no public comment on its activities and almost no questions from its governing board.

Approved unanimously were three items that its governing board treated in routine fashion. They included changes in its loan program and conference grants along with appointment of new grant reviewers. 

The changes in the loan procedures and other related grant administration rules must go through additional state processes before final action.

Today’s meeting had 22 teleconference locations throughout the state but no members of the public or the scientific or business community spoke up from those sites. The board has another teleconference meeting scheduled for Nov. 19. The next in-person governing board meeting will be in Los Angeles on Dec. 17. 
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Today's California Stem Cell Meeting Open to All at 22 Locations

If you would like to speak directly to the overseers of California’s $3 billion stem cell research effort, today would be a good day.

The agency will have the most ever sites available for public access in its nearly 11-year history to a meeting of its governing board. Twenty-two teleconference locations around California are scheduled to be accessible, ranging from Davis to Fresno to La Jolla as well as major population centers such as Los Angeles and the San Francisco Bay Area.

The meeting begins at noon. The public is entitled to speak to the board on any subject, including its proposals for spending its last $800 million to $900 million. (See here, here, here and here.)

Items on the brief agenda include three non-controversial matters. As usual, the California Stem Cell Report will provide live coverage of the session.

The teleconference sites are in San Francisco (3), Los Angeles (4), San Diego (2), La Jolla (2) and one each in Davis, Sacramento, Redwood City, Irvine, Oakland, Fresno, Berkeley, Elk Grove, South San Francisco, Stanford and San Jose. Specific addresses can be found on the agenda.
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Tuesday, October 13, 2015

California Stem Cell Loan Rules Coming Up Later This Month

The governing board of the $3 billion California stem cell agency has scheduled a brief, teleconference meeting for Oct. 22 during which the public or researchers can address any concerns they may have, including its priorities and spending.

The items on the agenda are routine, but as always there is an opportunity for the public to comment on any topic, whether it is on the agenda or not.  That includes how the agency plans to spend its last $800-$900 million.

There are plenty of teleconference locations spread around the state including two each in San Francisco, Los Angeles, San Diego, La Jolla and Irvine. Other locations are in Davis, Sacramento, Redwood City, Oakland and Fresno. Specific addresses can be found on the agenda.

On tap at the meeting is formal approval of loan and other rules in the clinical stage program, changes in conference grants and appointments of new grant reviewers.  

The agency is also in the midst of finalizing changes in its strategic plan for the next few years. Its money for new awards is scheduled to run out in about four years. Although the changes are not expected to be adopted until December, the public can speak directly to the board on the matter at Oct. 22 meeting. 

For more on the choices facing the agency over the next few years, see here, here, here and here.
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Saturday, October 10, 2015

LA Times: California's Stem Cell Agency and Rising Concerns About Drug Costs

Bloomberg News describes this $1 million gene therapy
as the world's most expensive medicine
Highlights
CIRM's affordability role
Risk-reward calculations
Burden on industry and CIRM

The Los Angeles Times has a Sunday readership of 2.4 million, and tomorrow they will be seeing this headline.
“Sky-high price of new stem cell therapies is a growing concern”
The article under the headline has much to do with California’s stem cell agency, which was founded on the promise that it would reduce health care costs, among other things, by as much as $18 billion.

The piece, available online last night, was written by Pulitzer Prize-winning columnist Michael Hiltzik. He noted that the agency is engaged in two, phase three clinical trials, the last step before a therapy is approved for widespread use.  

Hiltzik, author of the recently published book, “Big Science,”  wrote,
“If successful, they'll be firsts for the program, which is formally known as the California Institute for Regenerative Medicine. But they may also put CIRM smack in the middle of a burgeoning debate over how to ensure access for all patients to life-enhancing or life-saving cures.” 
Hiltzik pointed to stem cell and other advanced biologic treatments now reaching the global market that cost as much $1 million. One of those therapies was developed by Osiris Therapeutics when the current CEO of CIRM, Randy Mills, was the CEO of Osiris. The product, Prochymal, is being offered in Canada for $200,000 for a full treatment.

Hiltzik wrote that Mills “argues that CIRM's duty is to nurture therapies that can't be developed under conventional pharmaceutical business models and therefore may not yield profits like conventional drugs.”

Hiltzik continued,
“'If there was a risk-reward profile that was so attractive’ for such products, (Mills) says, ‘CIRM wouldn't have to exist.’ The public should have ‘an expectation of some return, but not of some great return,’ he says. 
“Mills argues that the public's concerns about drug pricing often may be misplaced, especially for treatments that reduce or eliminate lifelong treatment of chronic diseases. ‘In general, cell therapy may be more expensive to deliver,’ he says. ‘But if it's curative, that almost doesn't matter.’"
“He also cautions against allowing the political debate over drug pricing to distract from CIRM's ‘primary mission, which has been to bring cures to patients suffering from unmet medical needs.’ That explains the CIRM board's past resistance to the imposition of more stringent price regulation or higher revenue-sharing requirements. ‘We should be funding those things that, without our funding, wouldn't exist.’" 
Hiltzik concluded, 
“Still, public outrage over high drug prices isn't likely to ebb any time soon, and may only intensify as cutting-edge therapies reach the market at stratospheric prices. The burden will be on the biotech industry and its backers, including CIRM, to show taxpayers and patients that the price of treatment corresponds to the cost of research and development, rather than reflecting merely what the market will bear.”
(For more on the stem cell agency and affordability, see here and here.)
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Friday, October 09, 2015

Will Stem Cell Treatments Mean "Your Money or Your Life?"

Highlights
Outrage about prices
Industry euphemisms
Handy demons
California's stem cell direction

Halloween is just around the corner and some stem cell folks here in California are doing their best to wish away a particularly frightening specter.

They barely can bring themselves to name the force they fear, at least based on what this writer heard in a brief visit to the live Web cast yesterday of the “Stem Cells on the Mesa 2015” conference.

Some of the rhetoric amounted to no more than whistling in the dark. Investors, researchers and business executives danced around what almost certainly appear to be extremely high treatment costs for stem cell treatments.

Those costs are the type that have stirred recent outrage among consumers and among some physicians. The controversy has emerged anew in the presidential race and last week knocked the stock market around a bit. 

This week, NBC News is airing a series of reports called “Your Money or Your Life.” The episode
yesterday featured a woman with cystic fibrosis who said about a drug maker,
“They put a price on my life.”
Inevitably meetings like the Mesa conference rarely deal directly with the tough and emotional issues that are typified by the ire expressed by that woman, Klyn Elsbury, who lives a few miles north of the Mesa meeting.   Instead biotech executives retreat behind such euphemisms as “reimbursement,” which is a catch-all term for “how do we make a profit.”

Yesterday the matter of pricing did come before one panel. While this writer came in late and did not hear all the names, the general response could be called “if we build it, they will come.”

Many of the potential products being tested now involve “unmet medical needs,” and thus the demand could be extraordinarily high. In other words, if you want to live, you will have to pay our price.

It would be “super transformative” in the market place, one speaker said, if a company has produced the only drug that will save a person’s life. Another said “the system will eventually find a (pricing) model.”  Which is where whistling in the dark comes in. But if the industry doesn’t directly face the emotional and medical concerns about predatory business actions, the industry, in all likelihood, will be hoist on its own pricing petard.

Lawmakers and regulators – fueled by public outrage – may well react to overly aggressive prices and begin to impose what could amount to some sort of profit rationing. After all good public health is a virtuous thing. And if prices stand in the way, something needs to be done about it. Or so the reasoning will go. Every politician needs a demon to rail against. Big Pharma and related stem cell firms could be that handy demon.

The argument in some circles maintains that prices will start out sky high and then decrease over time. But that does not mean the public and other payers will wait for decades and patiently pay $1 million per treatment.

That figure popped up this week in an item by UC Davis stem cell scientist Paul Knoepfler.  He wrote on his blog, ipscell.com, about a pricing model that did, in fact, run as high as $1 million.

Knoepfler said the stem cell community needs to answer following question and soon.
“Where’s the stem cell price sweet spot where we can help the most patients, but also generate a needed profit for the biotechs?” 
California’s $3 billion stem cell agency, in particular,  has an economic dog in the pricing hooha. The agency is in the midst of determining how to spend its last $800 million or so. It can decide to put that money into research that offers the likelihood of relatively affordable treatments or instead into $1 million cash cow therapies for Big Pharma.

What the agency does now will affect whether it vanishes in a few years for lack of funding or can find additional support from the state and/or private sources. If its only product after running through $6 billion (including interest) is a $1 million therapy, some might look askance at providing additional cash.

The Science Subcommittee of the agency’s governing will take up the revisions in its strategic spending plan some time in November. The proposal is scheduled to be approved by the full board at its Dec. 17 meeting in Los Angeles. (See here and here for more on the plan.)

Comments and suggestion for the plan can be sent to kmccormack@cirm.ca.gov.
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Thursday, October 08, 2015

From Wounds to Regulatory Speed-Up: California Conclave Examines Stem Cell Business and Research

Highlights
The Grafix story
Japanese ambitions
California's Alpha Clinics

Hundreds of representatives of the world’s stem cell community are meeting today and tomorrow in California mulling over everything from pricing to the possibilities of commercial cures.

The occasion is the Stem Cell Meeting on the Mesa 2015 in La Jolla, Ca., and if you are not there, it is still possible to see some of the presentations live and later on video.

Some can be dramatic, including one from Lode Debrabandere, CEO of Osiris Therapeutics of Maryland. This afternoon he pulled up a slide involving an Osiris product called Grafix, which is “a cryopreserved placental membrane that is designed for direct application to acute and chronic wounds.”

The photographs on the slide showed an open wound with an exposed tendon before and after
Osiris/Meeting on Mesa graphic
treatment. Debrabandere said the Grafix treatment led to closure of the wound in five months. He said the patient "is walking around and still has his foot.”

Osiris is the firm once headed by Randy Mills, president of the $3 billion California stem cell agency, known officially as the California Institute for Regenerative Medicine(CIRM). Mills speaks tomorrow morning to the conclave. The agency is one of the major organizers of the three-day session and contributed $50,000 to the program.  Other organizers are the Alliance for Regenerative Medicine and the Sanford Consortium for Regenerative Medicine, which has received $43 million from CIRM.

The conference has received little attention in the mainstream media with the exception of the San Diego Union Tribune. The newspaper's biotech reporter, Bradley Fikes, has filed two major stories tied to the conference.

One dealt with the burgeoning number of stem cell clinical trials. The other explored the ambitious stem cell research effort in Japan. Fikes wrote,   
“In the second half of the 20th century, Japan emerged as a world leader in automobiles and consumer electronics. In the first half of this century, the country plans to do the same with stem cells and regenerative medicine.”
Fikes said the Japanese stem cell market “was estimated at $90 million in 2012, projected to reach $950 million in 2020, $10 billion by 2030 and $25 billion by 2050.”

Fikes also pointed out how the Japanese have streamlined the regulatory process, something that CIRM President Mills thinks the United States should emulate. Last week, Mills was in Washngton, D.C., talking to regulators and others, presumably advancing his case for faster action on stem cell therapies.

On the agenda tomorrow morning is a panel dealing with clinical trials at the Sanford Consortium. The effort is tied to the Alpha stem cell clinic effort at UC San Diego  (see here ), which is funded by $8 million from CIRM. The agency initiated the Alpha program, which totals $24 million, in an effort to develop  a world-leading, one-stop program for stem cell treatment.

The Mesa meeting program said, “The CIRM Alpha Stem Cell Clinic at UC San Diego provides infrastructural strength to enable the complex interaction required for success” in stem cell treatments. 
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Monday, October 05, 2015

Nominations for Stem Cell Person of the Year Being Taken This Week

Now is the time nominate a few good men or women for the 2015 Stem Cell Person of the Year.

UC Davis researcher Paul Knoepfler is collecting names for the annual award that he funds with a $2,000 prize. He is looking for the person who had “the biggest positive impact in the stem cell and regenerative medicine world in 2015.”

Knoepfler wrote last week,
 “This award is unique in a number of ways. For example, anyone in the world is eligible to be nominated: both scientists and non-scientists alike. The nominee should also be someone who thought outside the box and took risks, which are novel areas of emphasis for this stem cell and regenerative medicine award.” 
Paul Knoepfler, CIRM photo
The deadline for making nominations is Oct. 13. The finalists will be selected from the nominees by an Internet vote. However, Knoepler himself will choose the ultimate winner. Nominations should be emailed directly to Knoepfler at knoepfler@ucdavis.edu.

Knoepler listed the previous winners last week.
Dr. Masayo Takahashi won in 2014 and this year also received the inaugural Ogawa-Yamanaka Stem Cell Prize.
Dr. Elena Cattaneo received the award in 2013 and went on to get the ISSCR Public Service Award in 2014 along with colleagues.
“In 2012 the winner was top stem cell patient advocate Roman Reed, who went on in 2013 to receive the GPI Stem Cell Inspiration Award.”
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Thursday, October 01, 2015

Fifteen Clinical Trials: Better Marks for California Stem Cell Agency This Year

California's stem cell agency today received higher marks than nearly two years ago when it was last examined by the only state body charged with oversight of the $3 billion research enterprise.

The occasion was a meeting of the Citizens Financial Accountability and Oversight Committee, chaired by the state controller. One of its members, Jim Lott, in January 2014 had some harsh words for the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

But after a presentation that showed the agency will be involved in 15 clinical trials by the end of this year, Lott said CIRM had improved its pitch, although most of what he was said was lost in a poor quality audiocast of the meeting.

Jim Lott
The California Stem Cell Report asked him to summarize his views. Lott replied in an email,
"I've always felt that CIRM performs well. They needed to better communicate their achievements to justify the $2 billion the agency has spent thus far.  They needed to make their business case.  They did that today, and it would seem that their new president is the change that occurred to make this and other needed transformational efforts happen."
State Controller Betty Yee, chair of the oversight committee, also seemed satisfied with the agency's progress. Her comments were also largely lost in the audiocast. Her office declined to provide a summary of her views for this item today.

Jonathan Thomas, chairman of CIRM, and other agency executives provided an overview of the agency's progress and changes in its strategic plan, most of which is familiar to readers of the California Stem Cell Report.

Some fresh tidbits from the presentations.

-- The move to the agency's new headquarters in Oakland at the end of November will cost roughly $380,000. The agency is losing its rent-free office space in San Francisco and cannot afford the expensive leases in the city of San Francisco.

-- Randy Mills, president of CIRM since May 2014, has brought a sharp-eyed business approach to the agency, accord to Chairman Thomas. He said the agency "needed a business person" as opposed to an academic as the result of its emphasis on financing clinical trials. Thomas said Mills put "discipline and perspective" of business in place at the agency.

Michael Quick, USC photo
-- Thomas was "delighted" to hear Yee ask what the state could do to help the agency in the future. Thomas said the agency is developing a strategy in which the state could play a "very prominent role." Presumably that would involve more state funding since the agency is expected to run out of funds for new awards in about four years.

The controller's office also announced the addition of a new member to the oversight committee, Michael Quick, provost and senior vice president of academic affairs at USC.

As the school's No. 2 executive, he reports to Carmen Puliafito, president of USC and who also sits on the CIRM governing board. USC, which has held a seat on the board since 2004, has received $107 million from CIRM.

The oversight panel is required by state law to examine the agency on an annual basis. The last such session occurred 21 months ago. Asked to explain the delay, Taryn Kinney, a spokeswoman for Yee, said in an email,
"Since her inauguration earlier this year, (the controller) focused on building her staff and strengthening the many core internal functions of the state controller’s office."
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Oversight Panel Concludes Hearing on California Stem Cell Agency

Today's oversight meeting involving the operations of the $3 billion California stem cell meeting concluded at 11:10 a.m.  The agency presented a general update on its operations. No members of the review panel voiced major criticism of the agency. In fact, one who was sharply critical of the agency nearly two years ago appeared to be quite satisfied with its performance. However, much of what he had to say was muddled in the audiocast. We expect to carry an item with more details later today. Sphere: Related Content

Oversight Panel Begins Session Involving California Stem Cell Agency

Today's meeting of the Citizens Financial Accountability and Oversight Committee began about five to 10 minutes ago. Members are hearing presentation of routine audit results already aired publicly by the California stem cell agency.

At 9:18 a.m., the meeting achieved a quorum. That enables it to take official action. A new member was sworn in this morning. However, his name was not audible on the audiocast of the meeting. The California Stem Cell Report has asked for information on the appointee. Sphere: Related Content

California's Stem Cell Oversight Meeting to Begin Shortly; Directions for Listening Online

Today's 9 a.m. meeting of the oversight committee for the California stem cell agency is available online -- listen only -- using the following information.  The slides for the meeting are available here.
Public call-in number (listen only): (800) 260-0718 
Access Code: 369689
 
Listen to the meeting online: https://im.csgsystems.com/cgi-bin/confCast

Conference ID: 369689  


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