Friday, October 09, 2015

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

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,, 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
Sphere: Related Content

Thursday, October 08, 2015

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

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. 
Sphere: Related Content

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

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.”
Sphere: Related Content

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."
Sphere: Related Content

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:

Conference ID: 369689  

Sphere: Related Content

Wednesday, September 30, 2015

Connecting With the Public and Stem Cell Stakeholders: A New and Inexpensive Tool

For years the California stem cell agency has given the public access to the meetings of its governing board via a one-way audiocast over the Internet and by phone.

But patients, advocates, scientists and business folks have not had the opportunity to comment and make suggestions re the doings of the 29-member board of directors through the same method. The public could only listen unless they were on the scene.

However, that could change if the $3 billion stem cell enterprise follows the lead of the state Treasurer John Chiang. This summer he began opening up meetings of the many important boards in his office to live, two-way, teleconference connections with the public.

Chiang also uses the same sort of service and company as used by the stem cell agency. But he has added an inexpensive touch that genuinely opens up the meetings to the public.

We recently stumbled across Chiang's press release on the move, which may well be unique in California state government. We queried Drew Mendelson, a spokesman in the treasurer’s office, for more details. 

Mendelson said,
“Participants on the phone are in listen only mode, but when the meeting chair calls for public comments, callers can press a set of numbers to indicate they want to speak.  The conference call is monitored online by (treasurer’s) staff and the call monitor must click on the caller in the queue to allow the person to speak.”
 Mendelson continued,
“Under our state contract with AT&T, we determined that an hour long meeting with 10 callers would cost less than $15.  The cost increases significantly if you have AT&T provide conference monitoring services.  We chose to monitor the calls ourselves.  What we are using is a basic conference call service with an online component where the monitor sees the list of callers to know how many people are on the line and when someone indicates they want to speak.”
Mendelson said that the move was generated by Chiang's desire to enhance public access and his “desire to increase public participation in and increase awareness of the many boards, commissions and authorities" that he chairs.

On-the-scene public participation in CIRM meetings is slim. One of the reasons is that the sessions often require travel and overnight stays that pose barriers for many persons, particularly patients and their advocates. The agency would make it easier for its stakeholders and enhance its reputation for openness and transparency by instituting this inexpensive practice. It also fits with the goal of Randy Mills, the president of the agency, to provide more clarity in what the agency does. 
Sphere: Related Content

Tuesday, September 29, 2015

Oversight Panel of California Stem Cell Agency Schedules First Meeting in 21 Months

The only governmental body specifically charged with oversight of the $3 billion California stem cell agency, an enterprise that operates beyond normal state controls, announced yesterday that it would meet in just three days.

The session of the oversight panel comes 21 months after the last meeting of the Citizens Financial and Accountability Oversight Committee(CFOAC). The panel is required by state law to meet annually. Its last meeting in January 2014 saw the agency criticized harshly by one of the members of the oversight panel.

The committee is chaired by the state controller, who currently is Betty Yee. At the time of the last meeting, the controller was John Chiang, who is now state treasurer.

The agenda for this Thursday's meeting contains no items that would seem to be controversial, only a review of a routine audit and a presentation by the agency itself.

In January of 2014, Jim Lott, one of the members of CFAOC, made it clear at some length that he was not pleased with the agency's performance. He said in part:
“What can we say we've done to advance to a cure or to cures? It's fine that we've got all -- we've contributed to all. What can you say that we've actually done? We don't really have any -- I'm going to just say this because it's a bias and I know it's a bias. We don't have any tangible specific and measurable results that I can point to.”
The $3 billion state stem cell agency is exempt from the usual state budgetary controls. It receives its funds directly from bond proceeds without intervention by the governor or the legislature. The agency’s independence was authorized by voters when they created the agency in 2004 through a ballot initiative that altered the state constitution.

Two sites are available for the public to observe and participate. One is in Emeryville in the San Francisco area and the other is in Los Angles. Addresses can be found on the agenda. The public can also listen to an audiocast of the proceedings. Directions are also on the agenda. 

Sphere: Related Content

Friday, September 25, 2015

Building a 'Beautiful Machine:' California's Last $900 Million for Stem Cell Research

Intense, passionate, zealous – some of the words that could have applied yesterday to the CEO of a California enterprise devoted to the dispassionate world of scientific research.  

Randy Mills, president of $3 billion California stem cell agency, started with what could have been a mind-numbing overview of a revision of the agency’s strategic plan – a subject that draws yawns from many.
Randy Mills at an earlier strategic plan event in La Jolla.
San Diego UT photo

Plus he was not talking directly to his key audience – the directors of the governing board of the stem cell agency, who were listening to him from 18 different teleconference locations.

Mills eased into his presentation, backed by 30 pages of PowerPoint slides. He was speaking by phone from room 3803 in the Sanford Consortium in La Jolla, a facility partially financed with $43 million in agency funds.

His pace and fire escalated as talked about transforming the agency and fabricating a “great, beautiful machine.”

He told the directors,
“We have created very beautiful pieces – but they have existed as pieces”
Now is the time, he said, for California to create a “giant engine that will accelerate the research in a way that exists nowhere else in the world”  – a “giant coordinated stem cell machine.”

Mills said the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, is “by far the largest regenerative institute in the world.”  At the same time, it “needs to be better appreciated.” He promised to generate a “dramatic increase” in awareness of the agency.

Mills, who has been with the agency for about 16 months, skimmed over the numbers: $2 billion out the door, $900 million left for research awards, $400 million to go to clinical research, 50 new clinical trials.

Mills has already increased the speed of funding for clinical programs, dropping the time from years to months. But he wants to do more for what is called the translational stage – bringing developments out of  basic research into clinical phases in three years instead of eight.

Mills’ efforts yesterday were limited by time, however. The meeting was scheduled for only 90 minutes and the board had other business as well.

Individual board members had brief comments, including Jeff Sheehy, a communications manager from UC San Francisco. He raised questions about Mills’ plan to clear out obstacles at the FDA. Sheehy wanted to know more about how much it would cost and how it would be pursued.

Another asked whether there was a sufficient research base in California to achieve all that Mills proposed, a risk that Mills had identified in his PowerPoint slides. Yes, Mills said, it could be that CIRM will not be able to do 50 new clinical trials.

Mills’ report was a midpoint update on how the agency plans to spend its final $900 million in what could well be its last strategic plan.

The proposal will be examined once again by the Science Subcommittee of the agency’s governing board in November before it goes to the full board in December for approval.

The plan is likely to have a major impact on stem cell research in California and on the lives of hundreds of researchers. Only one scientist was present to comment yesterday at the meeting, Jeanne Loring of the Scripps Institute. She told Mills that his presentation was “terrific,” which sparked applause from about 20 patient advocates in the room at the Sanford Consortium.

As for the fate of the agency after 2020, when funds for new awards run out, CIRM Chairman Jonathan Thomas said that would be discussed at the December board meeting.

But as Mills noted, 
“We don’t have a lot of time left.” 
The agency is open for more comment from the public. Mills said suggestions, criticism, etc., can be sent to

More information about the strategic direction of the agency can be found here and here.

Those directly affected by CIRM spending – or who want to be affected by it -- would be well advised to listen to Mills’ presentation on a recording of the teleconference meeting. The recording can be found on the audiocast link on the meeting agenda, beginning at about 38 minutes into the meeting.
Sphere: Related Content