Friday, December 13, 2019

'Natural Killer' Cancer Cell Treatment Advances; State Stem Cell Agency Pleased

The California stem cell agency this week touted a possible "off-the-shelf cancer killer" that the state enterprise is helping to develop with $4 million.

The company involved in the treatment is Fate Therapeutics, Inc., of San Diego, a publicly traded firm working on induced pluripotent stem cells (iPSC) that have been genetically engineered to enhance their anti-tumor activity. The product is called FT516.

Writing this week on the agency's blogKevin McCormack, senior director of communications at the agency, said,

"Fate Therapeutics has developed an off-the-shelf therapy (thanks to (agency funding) that could, theoretically, be stored at hospitals and clinics around the country and used whenever it’s needed for anyone who needs it."
McCormack said that a CAR-T approach uses a patient's own "re-engineered" cells to kill cancer tumors. He wrote, 
"But the thing that makes it so appealing – using the patient’s own cells – also makes it really complicated and expensive. Creating a custom-made therapy from each patient’s own cells takes time and costs a lot of money. But now (the Fate Therapeutics) approach could change that."
Fate uses NK (natural killer cells). Maxx Chatsko of the Motley Fool financial web site reported,
"It's the first cell product of any kind derived from a clonal master iPSC line to be evaluated in a clinical trial in the United States. The 'clonal master iPSC line' part essentially means it can be manufactured more easily than immunotherapies requiring cells to be extracted from patients. That's why it's called 'off the shelf.'"
McCormack continued, 
"At this year’s meeting of the American Society of Hematology (ASH) Fate announced that the first patient treated with this new approach seems to be doing very well. The patient had acute myeloid leukemia and wasn’t responding to conventional treatments. However, following treatment with Fate’s FT516 the patient responded quickly and...was able to leave the hospital and spend Thanksgiving with his family. 
"Equally impressive is that 42 days after being treated with FT516, the man showed no signs of leukemia in either his bone marrow or blood."
The product is being licensed exclusively to Fate by the Memorial Sloan Kettering Cancer Center

Fate Therapeutics' stock closed today at $18.79, down 13 cents. Its 52-week high was $22.82. The 52-week low was $11.00. Last September it raised $173 million in a stock offering.
Sphere: Related Content

Sunday, December 08, 2019

LA Times: $5.5 Billion Measure for California Stem Cell Agency Could Actually Be a 'Downfall'

The Los Angeles Times, California's largest circulation newspaper, carried a piece today that called for withdrawal of the proposed, $5.5 billion ballot initiative that would save the financial life of the state's stem cell agency.

The article by Pulitzer Prize-winning business columnist Michael Hiltzik said the measure would perpetuate many of the flaws in the ballot proposition in 2004 that created the agency, officially known as the California Institute for Regenerative Medicine (CIRM). 

In fact, Hiltzik said, the new measure "makes some of them worse."

"That’s dangerous," Hiltzik continued, "because although the measure could fuel the stem cell program for years to come, it might also prompt a repudiation by voters sensitive to its many imperfections. Such an outcome would be tragic for California and the advanced science already supported by CIRM."

Hiltzik's opinion piece was the first extended look at the proposed stem cell initiative by a major media outlet in California. It appeared on the front page of today's business section of the Times. On the paper's web site, the article was featured as a "weekend read" at the time of this writing. 

The Times says it has about 2 million readers on Sunday. It is a go-to source for other journalists when they are researching stories and trying to understand major issues facing the state. 

Hiltzik has followed the $3 billion agency since its inception and has been highly critical. Last spring, however, he wrote that the agency had "proved its value." In today's piece, he had a lot to say about the good work of the agency. 

In his first two paragraphs, Hiltzik declared that CIRM "has made great strides in advancing what’s known as regenerative medicine and placing California at the center of the developing science." Hiltzik also said that CIRM has financed state-of-the-art laboratories, attracted preeminent scientists and "brought scores of promising treatments for severe chronic diseases to the point of clinical trials."

But he also noted that the agency has "has so far failed to yield a single marketable clinical product." He continued, 
"That’s despite the sales pitch for Proposition 71 in 2004 -- that all that stood in the way of 'cures' for Parkinson’s, Alzheimer’s, spinal cord injuries and other maladies was money."
The greatest flaw of the agency, however, has more to do with public policy than science, Hiltzik wrote. And that is the exemption of the agency from the usual oversight by the legislature and the governor, an exemption contained in the measure that created the agency.

Voters provided CIRM with $3 billion in bond funding 15 years ago. 
Today that money has all but run out. Robert Klein, a real estate developer, first chairman of the agency and who sponsored Proposition 71, is now once again sponsoring the latest initiative to give it billions more. 

Hiltzik concluded,
"(Klein) should withdraw his measure, and CIRM’s leadership should write a new one or work with Gov. Newsom and the Legislature to map out the program’s next act. 
"CIRM’s leadership needs to show the public that it’s capable of taking charge of the program’s destiny. If it’s not willing to make its own case for CIRM’s continued existence, how can it persuade voters to give it one cent more?"
Sphere: Related Content

Wednesday, November 27, 2019

San Diego Biotech Reporter Bradley J. Fikes Dies at 61

Bradley J. Fikes, journalist
San Diego U-T photo

When biotech reporter Bradley J. Fikes died last Wednesday at the age of 61, he was described in his newspaper as "part Dr. Dolittle, part Inspector Gadget."

The piece in the San Diego Union-Tribune was a tribute to Fikes, whose family said he died of natural causes at home. The paper said he was "an ever-on-the-move ball of energy" and beloved by his colleagues.

Fikes occupied a special position in California journalism. He was one of the few daily newspaper reporters who regularly covered the biotech industry and its research community. Fikes was also one of the few who delved into the affairs of California's 15-year-old, stem cell research agency. 

Last month Fikes penned a lengthy piece for the San Diego Union-Tribune that involved the agency, known officially as the California Institute for Regenerative Medicine (CIRM). The story dealt with a blood cancer drug called fedratinib and the relentless, years-long journey before the research, which was partly funded by CIRM, came to fruition. 

Fikes began the story like this:
"Cancer patients hold onto hope the way some people hold onto the lap bars of roller coasters: As if their lives depend on it.
"And then they get ready for the ups and downs."
Kevin McCormack, the stem cell agency's senior director of communications, wrote on the agency's blog that Fikes' story captured the setbacks, twists and turns and the years that it took before the treatment ultimately emerged. 

McCormack said yesterday, 
"Everyone at CIRM was stunned and saddened by the news. Brad was a real character, and a real gentleman. His unique sense of fashion made him hard to miss, even at a crowded conference, but it was his curiosity and his clear love of science that really stood out. He didn’t just report on stem cell research as a job, it was something he loved and that came across in his writing. He had a tremendous gift for taking complex science and turning it into everyday English. He was also great fun to hang out with. He had a wonderful sense of humor and a wry way of seeing the world. I shall miss him. We all will."
One of Fikes' colleagues, Michelle Guerrero, a Union-Tribune illustrator and graphics reporter, said in Fikes' obituary
“He had the wonderment of a child, the complexity of a scientist and an artful way of coming up with the words to explain it all.”
Fikes cut a colorful figure as he divined the doings at Scripps and Salk and at the other hallowed halls of science.  The Union-Tribune's piece on Fikes said, 
"Fikes was impossible to miss. By his own admission, he was a walking fashion disaster. He wore odd-colored business shirts that clashed with his suspenders, and slacks that never made contact with an iron. At times, cellphone cables hung out of his pockets like limp licorice."
Fikes was a graduate of San Diego State University where he first began writing the news. The campus newspaper wrote its own account of the life and times of Bradley J. Fikes. The piece carried comments from his colleagues including Gary Robbins who sometimes teamed with Fikes on stories. Robbins praised not only Fikes' journalism but his humanity as well. Robbins said, 
"He had poise, he had professionalism, he was a gentleman.”
Sphere: Related Content

Tuesday, November 26, 2019

Media Coverage: California Stem Cell Agency Receives Slight Whack in Los Angeles Times

California's $3 billion stem cell research program, now down to only $27 million, popped up in the Los Angeles Times this week in a piece that dealt with presidential politics, term limits and dubious ballot measures.

The mention consisted of only two paragraphs. But it demonstrated, at least for the state stem cell agency, the flaws of excessive generalization in P.T. Barnum's famous saying that “Any kind of publicity is good publicity as long as they spell your name right.”

Los Angeles Times columnist Michael Hiltzik wrote yesterday about "crowd pleasing" pitches to voters that actually contain "few virtues."  The starting point was term limits for congressmen and women. Hiltzik noted that such measures, in fact, do not serve the public well because they enhance the power of lobbyists. And then he went on to discuss ballot initiatives in California. 

Hiltzik, a Pulitzer Prize winner and author of "Big Science," wrote, 
"It's not unusual for ballot measures to win the approval of voters despite provisions that work directly against voters’ interests. That includes Proposition 71 of 2004, which created California’s $6-billion stem cell program. (See below for a look at the $6 billion figure and the nominal cost of the stem cell effort.)
"Proposition 71 was sold to voters as a gateway to cures for a host of intractable diseases, a promise hopelessly incompatible with the way science is done. Its promoters also tried to make a virtue out of its nearly total independence from legislative oversight, another artifact of anti-government sentiment. The stem cell program has indisputably supported excellent science, but its lack of legislative oversight remains a flaw."
The lack of legislative oversight will continue under the proposed 2020, $5.5 billion ballot initiative that is now before California election officials. If approved by voters in 11 months, the initiative will save the California stem cell agency from financial extinction. 

The new initiative is sponsored by Robert Klein, the first chairman of the state stem cell agency. Klein's measure does not address a number of sharp criticisms of the program, including those found in a $700,000 study of the agency by the prestigious Institute of Medicine

Klein's new initiative is sort of a son of Proposition 71. Klein also directed the writing of the 2004 measure, which did not provide any source of stem cell funding beyond the $3 billion in state bonds. 

As for the $6 billion figure cited by Hiltzik, it originated in a 2004, state ballot analysis of Proposition 71. The figure was an estimate of the total cost, including interest, to taxpayers of the stem cell agency, which is financed by $3 billion borrowed by the state. 

The interest estimate did not anticipate the 2008 Great Recession and the related drop in interest rates. The latest estimate is that the total will be about $4 billion. Read more about that estimate here. 

Sphere: Related Content

Monday, November 18, 2019

Rare Media Coverage of $5.5 Billion California Stem Cell Ballot Measure

The journal Science is carrying an article about the "delicate negotiation" involving the California stem cell agency and the man who is sponsoring a ballot measure that would keep the research effort alive with $5.5 billion in cash. 

The piece by Kelly Servick followed a two-hour meeting last Friday of the governing board of the agency, formally known as the California Institute for Regenerative Medicine (CIRM). Klein was in attendance and agreed to additional changes in the measure beyond those he announced to the California Stem Cell Report. 

There was no disagreement between Klein and the agency concerning the amount of money involved, which is much needed by CIRM. It has only $27 million left, compared to the up to $300 million annually that it normally spends on awards. The $27 million is earmarked for sickle cell anemia. 

The piece in Science represented rare national or even California media attention to refinancing the agency, which has pumped $2.7 billion into stem cell research in the Golden State in the last 15 years. Beyond the refinancing, the Klein initiative would make sweeping changes in the agency and governance. It also does not deal in a significant way with some areas that have been the focus of sharp criticism over the years, such as conflicts of interest.

Servick's article captured some of the highlights of last Friday's CIRM board meeting. She also reported, 
"In July, Americans for Cures conducted a poll of about 1150 likely voters and found a 65% approval rating for re-funding CIRM, Klein says."
Servick wrote, 
"Many researchers credit the agency with creating a strong infrastructure for stem cell research in the state and shepherding dozens of experimental treatments into human trials. But no CIRM-funded stem cell therapy has yet won FDA approval, leading critics of the agency to ask whether the initial 2004 initiative, born from opposition to a ban on federal funding for embryonic stem cell research, promised 'cures' it couldn’t deliver." 
Klein has until 5 p.m. today to file his amended initiative with state election officials. If it qualifies for the ballot with more than 600,000 signatures it will be brought to voters next November.

Sphere: Related Content

Friday, November 15, 2019

$5.5 Billion Stem Cell Ballot Measure: Questions in California but Apparently No Foot-Dragging

Some directors of the California stem cell agency this morning expressed a number of reservations about a $5.5 billion ballot initiative to stave off its financial demise, but none appeared likely to oppose the proposal 

In a special, two-hour meeting of the agency's governing board, questions were raised about provisions that would restrict the board or mandate programs, ranging from training to lab equipment. A provision to increase the size of the board from 29 to 35 was criticized and supported. A new effort to deal with the affordability of stem cell therapies was praised. But some aspects were questioned.   

The topic of the day was a complex, 30-page, proposed ballot initiative sponsored by Robert Klein, the first chairman of the agency, who has already announced changes in his proposal. More alterations appeared to be in the works as the result of today's hearing, including elimination of some language dealing with pricing. The meeting did not go into details about that subject.

In addition to requiring the state to borrow $5.5 billion for stem cell and other "vital" research, Klein's proposal would expand the scope of the agency and alter its governance. It would be required to develop and finance efforts at making stem cell research more affordable. Brain disease research would receive special attention with a dedicated $1.5 billion out of the $5.5 billion. Training programs would be mandated and expanded at community and state colleges and much more.

(The CIRM summary of the initiative can be found here. A summary by the California Stem Cell Report can be found here.)

(Here is the text of the initiative as filed in October. Here is the text of the measure with Klein's proposed changes as of Nov. 14.)

The agency began its life in 2004 with $3 billion in state bond funding, authorized by California voters through a ballot initiative.  Known formally as the California Institute for Regenerative Medicine(CIRM), it is down to its last $27 million, which is devoted to a sickle cell program with the federal government. At its peak, the agency was giving away up to $300 million a year.

Today's meeting began with several of the board members declaring that the public should not perceive their questions and comments as indicating they did not support the effort to refinance the agency. Some had expressed concerns that opponents might use board comments to defeat the initiative next November. 

Steve Juelsgaard
CIRM photo
The board moved rapidly through the initiative during the morning meeting. CIRM director Steve Juelsgaard and others raised questions about the size of the board, declaring that large boards become disengaged from an enterprise. Os Stewart, who has served on the board since 2004, said he initially thought 29 members were excessive but feels differently after 15 years. 

Several members expressed concern about mandates that would tie the hands of the board. CIRM Director Francisco Prieto said, 
"The science can surprise us."
Klein told the board that "we have fundamental obligation to give the voters an idea where their money is going." He also said that the mandates do not have legal timetables.

Today's meeting was very lightly attended, according to an agency spokesman, mostly by CIRM staffers. Only one member of the public addressed the board, Don Reed, a longtime patient advocate and supporter of the board.

Questions about the measure arose publicly this month after longtime director
Jeff Sheehy
CIRM photo
Jeff Sheehy authored a 3,300-word critique of the Klein measure, declaring it amounted to "too little and too much."  At the end of today's meeting, Sheehy did not express reservations that would prevent him supporting the measure. 

Klein unofficially modified his measure this week. He has until Monday at 5 p.m. to actually file the changes with state elections officials. The official comment period for the initiative is closed. But Klein said yesterday that interested parties can send comments and suggestions to him through Saturday at this email address: 

Comments filed directly with Klein are not a public record. If a reader would like to share his/her comments with the public, please send a copy of them to the California Stem Cell Report at djensen@californiastemcellreport. We will carry them verbatim.

Here is a link to a guide to the stem cell initiative information maze, which is updated as other key information surfaces. Sphere: Related Content

California Stem Cell Agency's Summary of $5.5 Billion Measure to Save its Financial Life

The California stem cell agency late yesterday posted its own summary of a proposed $5.5 billion ballot initiative to keep it from basically going out of business next year.

The summary is about 1 1/2 pages long compared to the 30 pages of the actual initiative, which is the subject of a hearing this morning by the governing board of the agency. 

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known, is down to its last $27 million. It came into being in 2004 with $3 billion in state bond financing. 

Also posted on the meeting's agenda is a five-page memo from 2017 discussing what the agency can and cannot do in connection with election activities such as a ballot initiative campaign.  Last month the board was given a seven-page memo about such activities. Both memos confirm that the agency may use public funds to draft its own initiative.

Neither of the memos discuss the pros and cons of the ballot measure sponsored by Robert Klein, who ran the ballot campaign that created the stem cell agency 15 years ago this month.

Here is an updated guide to the key documents and information concerning the initiative.

Sphere: Related Content

Thursday, November 14, 2019

Robust Defense of $5.5 Billion California Stem Cell Measure; State Hearing Scheduled for Tomorrow Morning

The man behind the $5.5 billion California stem cell initiative this afternoon vigorously defended the measure and released details of fresh changes in the proposal, which will be scrutinized tomorrow morning by the directors of the financially strapped state stem cell agency.

Robert Klein file photo by California Stem Cell Report
Despite the fact that agency faces its fiscal demise next year, the initiative is the only proposal around to continue the agency's funding. It ran afoul last week of questions from some of the agency's directors. One of those directors wrote a 3,300-word critique of the proposal by Robert Klein, the first chairman of the agency and who directed the writing of the 2004 ballot initiative that created the agency. A special meeting was called for tomorrow morning.

In addition to providing $5.5 billion in state bonds, Klein's proposal would significantly expand the reach and change the governance of what is formally known as the California Institute for Regenerative Medicine(CIRM). The measure would require the agency to launch a major effort to make "affordable" what are extremely expensive stem cell and genetic treatments. The proposal would also enlarge its governing board from 29 to 35. The board has been criticized sharply for the size of its board.

Klein said in an emailed statement that the changes he made in the wake of CIRM directors' concerns basically follow the outlines of what he released yesterday. He stoutly defended the agency's current work and his plans to expand its scope.

He said in an eight-page statement to the California Stem Cell Report.
"Patients and their families should not have to suffer more delays because of ill-conceived agendas that obviously do not fit into the new initiative, with a scope that has already been court validated."
Klein added,
"The federal government remains a threat to scientists and physicians investigating the best cell sources for developing therapies for patients. Just within the last nine months there have been new restrictions imposed by the federal government based upon ideological grounds."
Klein, a Palo Alto, Ca., real estate developer, is under a state deadline of 5 p.m. on Monday to make any changes. As sponsor of the 2020 initiative, he is the only person legally entitled to make changes. The measure then will go through a lengthy state elections process. More than 600,000 registered voters will have to sign petitions for the measure if it is to make it onto the November 2020 ballot.

Klein noted that the state deadline has passed for officially making public comments. But he said suggestions and comments can be sent to him through Saturday Nov. 16 at this address:  Any such comments are not a public record. If a reader would like to share his/her comments with the public, please send a copy of them to the California Stem Cell Report at djensen@californiastemcellreport. We will carry them verbatim.

The public can listen in and participate in tomorrow's meeting via the Internet or at telephonic locations throughout California. An updated list of those locations is here. Information on Internet access is on the meeting agenda.

Below is the text of the statement Klein sent this afternoon to the California Stem Cell Report. In it, he says the full text of the current version of the actual initiative is available on the Americans for Cures web site, a stem cell patient advocacy group that he founded and chairs.

Sphere: Related Content

A Guide to Information on the $5.5 Billion California Stem Initiative

Trying to wade through the information maze surrounding the proposed, $5.5 billion California stem cell initiative? Here are links to key documents, online postings and information dealing with the proposal and the ballot initiative process.

This list of information sources was updated Nov. 14, 2019, at 8:54 PM PST.

How ballot initiatives work in California

Text of the 30-page, proposed $5.5 billion measure

Text of the revised version of initiative as of Nov. 14, 2019

A summary of the measure from the California Stem Cell Report

A summary of the measure from the stem cell agency

A critique of the proposal by CIRM board member Jeff Sheehy

A critique of the proposal by CIRM grant recipient Jeanne Loring

An eight-page statement from Robert Klein, author of the initiative, to the California Stem Cell Report.

Proposed changes in the initiative offered by its sponsor, Robert Klein

How CIRM can write its own initiative

An article on CIRM board reaction to the initiative, complaints about "handcuffs" Sphere: Related Content

Delays on Details of Changes in Proposed, $5 Billion California Stem Cell Initiative

The details of the changes in the proposed 2020 California stem cell initiative that were promised by its sponsor yesterday have not been received as of this writing. The California Stem Cell Report will carry them when they are received.  The governing board of the state stem cell agency is scheduled to meet at 9 a.m. tomorrow to consider the initiative. Sphere: Related Content