Thursday, August 28, 2014

Asterias Stock Price Jumps Nearly 13 Percent Today Following hESC Therapy News

The California stem cell agency and Asterias Biotherapeutics today picked up a modicum of news coverage in connection with an advance on a spinal injury therapy that was once hailed as historic.

The news about the Menlo Park, Ca., firm’s clinical trial received major attention in the San Francisco Chronicle and more modest coverage in the San Francisco Business Times and on the blog.  

The news also helped to push the Asterias stock price up nearly 13 percent since yesterday to close at $3.08 today. The stock closed at $2.43 Tuesday, the day before the company released the clinical trial news, according to Google Finance.

Stephanie Lee’s piece in the Chronicle contained some history about the potential therapy, dating back to when it was developed by Geron and then abandoned. Geron was the first firm to win approval of a clinical trial for a human embryonic stem cell (hESC) treatment.

Lee also had a couple of interesting tidbits, including the fact that the stem cell agency’s $14.3 million award to support the trial will cover half its costs. Lee also reported,
“Geron treated severe injuries in the thoracic region of the spinal cord, which runs along the back. Asterias is targeting injuries that originate in the neck, citing an outside study that suggests injuries in this area are easier to treat.”
Enal Razvi
Select Biosciences photo
Lee additionally quoted Enal Razvi, managing director of Select Biosciences U.S., an international life sciences consulting firm with its U.S. headquarters in Fremont, Ca.,  as saying,
"This is just the start of a trial, not the approval of a drug, which are two very, very different things in this space…(but) this helps things go to the next level." 
The Chronicle story was the No. 1 story late afternoon today in Google news search results using the term "stem cell," ranking ahead of the STAP news out of Japan.

Paul Knoepfler, a UC Davis scientists who writes the ipscell blog, carried a Q&A with Jane Lebkowski, president of research and development at Asterias, who discussed another hESC product. She said,
“A second Asterias product is AST-VAC2, which are human embryonic stem cell derived dendritic cells. These cells are modified to express telomerase, a protein typically expressed in cancer cells. The aim is to use these telomerase expressing dendritic cells to stimulate immune responses against cancer cells. We are now preparing for clinical trials with this product.” 
That effort could well find its way to additional funding from the California stem cell agency if it meets the four-point criteria of the new president, Randy Mills, of the $3 billion research program.

In the San Francisco Business Times piece, Ron Leuty noted that the initial five-patient safety trial showed that “spinal cord injuries in four the patients had shrunk.” Leuty wrote,
“Whether that means Geron’s treatment is working in those patients is an open question. Geron’s study looked only at acute, or new, spinal cord injuries, so some of the results could be connected to normal healing over time.”

Wednesday, August 27, 2014

California's Stem Cell Trial for Spinal Cord Injury Moves Forward

A California firm, backed by $14.3 million in state cash, today announced that it has received a federal go-ahead to advance its clinical trial involving a human embryonic (hESC) therapy for cervical spinal cord injury.

Asterias Biotherapeutics, Inc., of Menlo Park, said in a press release that it is currently selecting clinical trial sites and expects to enroll patients beginning early next year. The trial is the continuation of an effort that Geron abandoned in 2011 for financial reasons.

Pedro Lichtinger, president and CEO of Asterias, said,
  “We are especially enthusiastic about working with our new partner, (the California Institute for Regenerative Medicine or CIRM), in executing this clinical trial. The FDA clearance provides Asterias with imminent access to the previously announced $14.3 million CIRM grant, which provides non-dilutive funding to support both the clinical trial and other product development activities for AST-OPC1.”
The phase 1/2a trial will involve doses of up to 10 times higher than what was used earlier. Up to 13 patients will be treated within 14 to 30 days after their injury occurred. The hope is that it will be easier to detect the efficacy of the treatment with such dosages.

As for the early stage trial involving five patients, the company said,
“These five patients were administered a low dose of two million AST-OPC1 cells and have been followed to date for 2 to 3 years. No serious adverse events were observed associated with the delivery of the cells, the cells themselves, or the short-course immunosuppression regimen used.  There was no evidence of expanding masses, expanding cysts, infections, cerebrospinal fluid leaks, increased inflammation, neural tissue deterioration or immune responses targeting AST-OPC1 in these patients.  In four of the five subjects, serial MRI scans performed throughout the 2 to 3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration.”
Asterias was awarded the cash by the California stem cell agency in May. (See here and here.)

Randy Mills, president of the stem cell agency, said today,
“This is exactly the type of treatment, focusing on an unmet medical need, that CIRM was created to address.”
Katie Sharify, one of the patients involved in the trial when it was started by Geron, said in the CIRM press release,
“A lot remains unknown about human embryonic stem cells and that's exactly why this research is so important. The scientific community is going to have a much greater understanding of these stem cells from the data that will be collected throughout the study and I'm glad to have been a part of this advancement."

Sunday, August 24, 2014

Earthquakes and Scientific Research: California's Exposure to Damage

The 6.1 earthquake today in Northern California once again demonstrated the vulnerability of scientific research in the Golden State to disruption by tremor.

As of Sunday morning, no research institutions – stem cell or otherwise -- reported damage from the quake. However, it was still early in the process of assessing the full impact of the temblor.

The closest stem cell research facility to the quake epicenter is the Buck Institute, located in Novato, which is roughly 15 miles from the epicenter.  A spokeswoman for Buck, which holds $34 million in awards from the California stem cell agency, said she had received no immediate reports of damage..

In response to a question from the California Stem Cell Report, Kris Rebillot said,
“I have not heard anything from our facilities staff about damage. The Buck is on pretty stable bedrock. I live in Petaluma which is closer to quake. No damage there.”
Later she reported that a walkthrough showed "no discernible damage."

The likelihood of severe shaking from an earthquake
is shown on this state map. The greatest potential is
marked by lavender with red and orange as less
 intense. The epicenter of the Napa quake was near
the north end of San Francisco Bay. 
 California is riddled with earthquake faults, many of which lie beneath or close by major research institutions, including StanfordUC Berkeley and UC San Francisco. Institutions in the south are in the same situation as well.

Problems that arose in the aftermath of today’s quake included lack of power, lack of water and natural gas leaks from pipelines. 

The situation recalled a lesser event that left one Southern California stem cell research institution without its normal power.  The institution, which will remain unnamed, had a backup generator that also failed. Fortunately the situation was caught before irreparable harm occurred.

The focus during events like today’s quake in Napa is on major damage and injury. But research institutions can suffer significant harm from what appears to be relatively minor damage – laptops smashing to floors, servers toppling, delicate instruments flying off tables, animal cages falling over and breaking and so forth.

Today’s event served notice that researchers should double-check their safety measures and physical security of important equipment and ensure that all data is backed up well offsite where it would not be destroyed by a quake.  That would include data at the California stem cell agency, which is headquartered in San Francisco.

Wednesday, August 20, 2014

Stem Cell Blowback on the Ice Bucket Challenge

Virulent opposition to research involving human embryonic stem cells is surfacing anew in the wake of the vast attention attracted by the Ice Bucket Challenge.

Sarah Pulliam Bailey wrote on Religion News Service that the objections have “gone viral” on the Internet because the beneficiary of the watery fundraising, the ALS Association, finances research involving human embryonic stem cell (hESC) lines.

For those of you who are not tuned into the icy effort, it involves celebrities and others pouring frigid water over themselves and challenging others to do likewise and donate to the ALS Association.  About $16 million has been raised so far and is still growing. Photos, news stories and blogs all chronicle the challenge on the Web.  A search this morning on the term “ice bucket challenge” turned up 57.2 million hits.

Our readers might ask how this involves the $3 billion California stem cell agency. The answer is that hESC research is the primary reason for the existence of the agency. It was created in 2004 by California voters after former President Bush restricted federal funding for research involving hESC.  Ironically Bush has participated in the challenge(see video above).

Bush’s restrictions, since lifted by President Obama, generated major controversy in the scientific community and raised fears that efforts to develop what seemed to be nearly magical therapies would wither and die. Without that concern, voters would have been unlikely to approve the measure, Proposition 71, and campaign contributors would have been unwilling to open their checkbooks.

The stem cell agency, however, has turned away from hESC research. Less than 250 of its 622 awards have gone to that field, according to the agency. Instead the agency is backing adult stem cell research, once an anathema to the organization, along with experiments involving reprogrammed adult stem cells. Leaders in the stem cell field, however, say human embryonic stem cells remain the scientific gold standard. (No figures were immediately available on the dollar value of the hESC awards.)

Continued funding of hESC research is linked to the agency’s search for alternative sources of cash as its current financing winds down. Efforts to develop a fresh stream from public sources will run up against the controversy involving hESC experiments, one of the reasons for the lack of interest by Big Pharma. The hESC opposition could also have an impact on development of private sources of funding who may not want to become embroiled in the flap, which can lead to picketing and public protests involving entities that support hESC research.

Kevin McCormack, senior director for the stem cell agency’s public communications, wrote about the ice bucket fad last week on the agency’s blog. He said it “shows how a little bit of creativity can create so much more interest in a disease, and the people suffering from it, than any amount of well-meaning, more traditional attempts at education.” And he poured a bucket of water over his head.
Nonetheless, the opponents hold their views with great passion and zeal and have little respect for hESC science. Here is what one person said today in a comment posted on the Religion News Service article. The individual was identified only as “jamadan.”
“My father died of ALS 6 years ago. My father was and my family is ‘pro-life’, meaning we believe abortion to be murder and embryonic research akin to Nazi medical testing on dead camp victims. Nothing can make us ‘accept’ abortion or anything that benefits from it. Not even our own lives. I think you’ll find that true for all Christians, who by definition, must be pro-life.” 
For other commentary involving objections to the Ice Bucket Challenge, see here, here and here.

(A footnote: This item marks the first video appearance of former President Bush and Kevin McCormack on this blog.)

Tuesday, August 19, 2014

California Edges Forward with $39 Million Stem Cell Diabetes Effort

California’s $39 million bet on a new stem cell therapy for diabetes today moved a notch ahead today with word that the treatment's developer has received federal approval to begin a clinical trial.

“Very encouraging news” was how the new president of the state stem cell agency, Randy Mills, described the announcement by Viacyte, Inc. “Exciting” was the word used by Jonathan Thomas, chairman in a pressrelease from the California Institute of Regenerative Medicine (CIRM), as the $3 billion agency is formally known. CIRM began financing Viacyte six years ago.

The go-ahead by the FDA was for an early stage trial aimed at testing safety and preliminary efficacy for treatment for type 1 (juvenile) diabetes. While the move is a good sign, only one out of any 10 potential therapies that begin a trial emerges as a full-blown treatment.  That statistic is for ordinary treatments – not therapies based on human embryonic stem cells (hESC). No hESC therapy has ever been commercialized, and they face unique regulatory and financial obstacles.

CIRM’s press release described the treatment like this:
“ViaCyte’s approach uses a thin plastic pouch, containing an immature form of pancreatic cells, to mimic the blood glucose regulating function of the pancreas. When the device is implanted under the skin these cells are designed to become insulin-producing and other cells needed to regulate blood glucose levels. It is believed that these cells will be able to sense when blood glucose is high, and then secrete insulin to restore it to a healthy level.” 
In addition to the news release, the stem cell agency ran a blog item on the announcement, which is useful, since there is more than one audience for the news. The agency did not link to the press release from Viacyte, which noted that the Juvenile Diabetes Research Foundation also supported the research.

Paul Laikind, president of Viacyte, said in his press release that he was “pleased” by the FDA action.  That was the most ebullient word in the company’s announcement, which focused on the more technical aspects of the potential therapy.

In news coverage, Bradley Fikes of the San Diego U-T wrote that the company has said the treatment could serve as a “virtual cure” for type 1 diabetes, which afflicts more than two million persons in the United States.

Monday, August 18, 2014

'De-risking' Stem Cell Therapy with Government Cash and Assistance

Nature Biotechnology this month took a crack at what it called “therapies of the state,” three government- financed efforts at turning stem cells into cures.

The piece by Beth Schachter discussed “thorny technical challenges,” safety issues and regulatory and IP risk – not to mention the dreaded “reimbursement” risk. (Reimbursement is a PR euphemism that revolves around industry fears of losing money.)

Greg Bonfiglio
Proteus photo
In addition to California’s $3 billion effort, Schachter wrote about Cell Therapy Catapult in the UK and Canada’s Center for Commercialization of Regenerative Medicine, whose board is chaired by Greg Bonfiglio, who is also founder and managing partner of Proteus Venture Partners of Palo Alto, Ca.

Schachter said that all three are to attempting to “de-risk the perilous process of advancing cell therapies that show potential in animal studies through human testing to commercialization.”

The Nature Biotech piece said that until recently Big Pharma and venture capitalists have steered clear of the cell therapies coming out of academia. Schachter wrote,
“For one thing, big pharma’s business model is very different from what is needed to translate cell therapies into practice. The pharma model involves mass manufacturing of products that can be stored in warehouses and distributed through pharmacies to large markets of patients. Cell therapies, on the other hand, may be highly individualized, are incompletely characterized, are expensive to produce, have a short shelf life and onerous supply chain, must be transplanted into patients by skilled healthcare workers and have complex regulatory requirements. These challenges, along with a dearth of cell-therapy successes, have kept away investors, too.”
As for the stem cell agency in California, Schachter wrote,
“In developing (its) extensive program, the CIRM has, in a sense, been standing in for the National Institutes of Health (NIH). It has also been taking the role that venture capital might have assumed, had the US government given the go-ahead on human embryonic stem cells, thereby building a robust preclinical/translational research program. As Bonfiglio it, ‘CIRM galvanized the industry, putting regenerative medicine on the map, both in terms of what the politicians and the lay people saw and in terms of maintaining scientific training in that arena.'” 
Schachter said that it is not clear how long taxpayer cash will be needed or wanted. She wrote,
“Also not clear is how each of the governments will assess the success or failure of these programs. How will governments know when the time is right to wean the programs from public monies? Will they consider job creation, the number of companies spun out, or launching successful products in the marketplace? Given the complexity of cell therapies and regulatory uncertainty, none of these things may happen for decades. Perhaps a better measure is whether any of the companies created gets traction with big pharma. That might indicate that the commercial promise of these therapies is finally evident.”

Wednesday, August 13, 2014

Scientist/Parkinson's Advocate With Biotech Background Appointed to California Stem Cell Agency Board

A retired San Diego scientist with a background in the biotech industry was named this week to the 29-member governing board of the $3 billion California stem cell agency.

David Higgins
Parkinson's Association photo
David Higgins, who is also president of the San Diego Parkinson’s Association, was appointed by State Controller John Chiang, replacing Joan Samuelson, who resigned from the board earlier this year.  Samuelson had been a member of the board since 2004.

Higgins fills a slot designated for a patient advocate for Parkinson’s Disease. He was diagnosed with Parkinson’s Disease in 2011.

The stem cell agency press release yesterday on the appointment emphasized Higgins’ personal and family perspective on the affliction. Chiang was also quoted as saying,
“As a trained molecular biologist, his involvement in drug development and business operations places him in a unique position, understanding both science and process.”
Higgins has worked at Invitrogen, Chiron and Idun Pharmaceuticals. Most recently he was executive vice president for business development and head of U.S. operations for BioMedica, Inc., a UK-based gene therapy company.

Higgins is currently a business/scientific advisor to iDiverse, Inc., a Del Mar, Ca., a gene technology firm that markets to biopharmaceutical firms as well as companies involved in fuel ethanol and industrial enzymes.

Higgins brings a perspective not found elsewhere on the board because of a variety of community service activities.

He serves on the executive committee of the Center for Ethics in Science and Technology in San Diego. He was once president of the board of the ACLU for San Diego and Imperial counties. He also served as foreman of the San Diego County Grand Jury, a 19-member citizens watchdog group, in 2006-7..

Higgins said in the CIRM press release,
“One thing I feel strongly about is that, yes I’m the Parkinson’s patient advocate on the board, but first and foremost I’m an advocate for everyone and I want to make sure that we spend our money wisely, and that we use our resources to identify and nurture the most promising stem cell projects across all target diseases.”
Higgins earned his Ph.D. in molecular biology and genetics from the University of Rochester followed by a postdoctoral fellowship at the National Cancer Institute.

The agency's board now has three vacancies on it left by the departure of Marcy Feit, Michael Goldberg and Robert Birgeneau. 

Monday, August 11, 2014

$40 Million California Stem Cell Genomics Agreement Signed; A Checkered Past

The California stem cell agency and a Stanford-led consortium have reached agreement on a $40 million stem cell genomics project that triggered complaints about irregularities, unfairness, score manipulation and the role of its then president, Alan Trounson.

The agreement was concluded last month with Stanford, UC Santa Cruz and the Salk Institute in La Jolla, five months after the award was approved by the governing board of the $3 billion agency, which is known as CIRM. The final signature came July 2 when Santa Cruz signed. Salk signed on June 26 and Stanford June 18, according to Kevin McCormack, a spokesman for the agency.

The effort is aimed at paving the way for therapies tailored to a patient’s genetic make-up and positioning California as a world leader in stem cell genomics.

Trounson’s role came under fire when he recommended approval of the Stanford application. The agency’s blue-ribbon grant reviewers, whose advice is rarely rejected by the CIRM board, also recommended funding three competitors. 

The round had a checkered history as a result of a conflict of interest involving scientist Irv Weissman of Stanford and scientist Lee Hood of Seattle, who own a ranch together in Montana. Trounson, who has visited the ranch as Weissman’s guest, recruited Hood to review the applications, including Stanford’s proposal which then specifically included Weissman.

The Stanford application that was ultimately approved did not include Weissman.  Michael Clarke, the No. 2 person in Weissman’s stem cell program at Stanford, was included, however, and was praised by name by Trounson during board consideration of the Stanford application. (See here for discussion of conflicts preceding the board action.)

Seven days after leaving CIRM at the end of June, Trounson was named to the board of directors of StemCells, Inc., of Newark, Ca., which holds $19.4 million in awards from the agency. The firm was co-founded by Weissman, who now sits on its board.  The Trounson appointment surprised the agency and triggered a rash of bad publicity for CIRM. (See here and here.)

The agency’s new president, Randy Mills, banned CIRM employees from communicating with Trounson about StemCells, Inc., matters and announced that he would not accept employment from CIRM grantees until one year after he leaves the agency.

Tuesday, August 05, 2014

Another Perspective on the Trounson Flap: Columnist Lists It as Example of 'Possible Corruption'

The $3 billion California stem cell agency was mixed up with some bad company this week – part of the fallout from last month’s Trounson Affair. 

The agency was lumped together with some ne’er-do-wells in a column in California newspapers that was headlined:
“California state government awash in corruption, conflict of interest”
The piece was written by Tom Elias, a middle-of-the-road, longtime columnist whose work appears twice weekly in 93 newspapers with a circulation of 2.2 million. Elias wrote,
“To some, it seems almost as if California has lately become New Jersey West. Incidents of possible corruption and conflict of interest are seemingly exposed at least once a month these days, with almost no consequences for anyone involved.”
Top on his list was the business involving Trounson, who left the California stem cell agency June 30. Seven days later, he was appointed to the board of StemCells, Inc., which holds $19.4 million in awards from the agency, formally known as the California Institute for Regenerative Medicine (CIRM).

Second on Elias’ list was a case involving a consultant at another state department that was reminiscent of another situation at the stem cell agency. Elias said,
“A month earlier, this column caught the state Energy Commission earmarking more than $28 million in ‘hydrogen highway’ grants for a new company co-founded by a consultant who only months earlier drew the map determining where hydrogen refueling stations will go and then trained commission staff on how to evaluate grants.
“No conflict of interest there, the commission insisted. Right.”
The case at CIRM involved a “special advisor” by the name of  Saira Ramasastry, managing partner of Life Sciences Advisory, LLC, of Emerald Lake Hills, Ca. In 2012, she was named to the board of Sangamo BioSciences, Inc., a publicly traded firm based in Richmond, Ca. At the time of her appointment to the board, the firm shared in a $14.5 million grant from the agency. She also worked as a consultant to Sangamo.

Since then, Sangamo has received another $6.4 million from CIRM. The president of Sangamo, Edward Lanphier, told the agency last January,
“We wouldn't be where we are today without you.”
In 2013, Ramasastry received $99,662 in cash and stock options from the company, according to its filings with the Security and Exchange Commission.

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