Monday, March 20, 2017

The Gottlieb Effect: Dismantling of FDA Silos, Less Regulation of Stem Cells?

A California stem cell researcher is predicting a possible "roller-coaster ride" in the wake of the appointment of Scott Gottlieb as the new head of Food and Drug Administration(FDA)

President Trump named Gottlieb to the post earlier this month. Gottlieb has said in the past that "less is more" in terms of regulation of stem cell research, according to UC Davis research Paul Knoepfler.

He wrote on his blog last week about the Gottlieb's views and what they could mean for stem cell research. Knoepfler reviewed some of Gottlieb's publications and speeches for the analysis, including a 2016 speech in Berkeley which contained "some pretty radical language."  Mentioned was dismantling silos within the FDA.

The California scientist said,
"The use of powerful words like 'dismantled' by Gottlieb last year, now bode for potentially highly disruptive changes at the FDA in the Trump administration under his leadership. Since he stated that regenerative medicine products 'don’t easily fall into FDA’s current buckets,' I wonder what the could mean for CBER (a branch of the FDA).
"I’m hoping that Gottlieb will rightly see the central distinction between empowering more efficient stem cell and regenerative medicine product development by good actors in the field (he could do this, for example, by shaping how the 21st Century Cures Act regenerative medicine-related directives are implemented) versus enabling many dubious actors at a host of for-profit businesses. These companies don’t do preclinical studies, don’t respect the FDA, and put patients at risk."
Knoepfler covered additional ground in his March 14 piece, which may be the only serious, current analysis of Gottlieb and stem cells. Knoepfler concluded:
"Change at the FDA overall or on stem cells isn’t necessarily a bad thing, but in this case it could be a roller-coaster ride."
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Friday, March 17, 2017

California Stem Cell Agency Says Its Top Lawyer Is Departing After More Than 12 years

James Harrison, Remcho photo
The $3 billion California stem cell agency today said it is losing one of its top leaders, James Harrison, one of the authors of the measure that created the agency and who most recently is serving as its “unflappable” general counsel.

The agency announced this afternoon that Harrison would be departing at end of June “to focus full-time on his legal practice.”

Harrison has worked with the agency under contract since its inception in 2004. He was known for his exceptional legal skills and aplomb when matters became muddled or worse at meetings of the agency’s 29-member board.

Randy Mills, president of the agency, said in a press release,
“James is more than wise counsel at CIRM, he has also been a highly effective leader, responsible for designing and implementing many of CIRM 2.0’s more innovative features.
“He is unflappable and maintains a sense of humor and perspective, even in the most challenging of situations. We thank him for his many years of service and wish him the very best.”
Harrison was one of five persons who drafted Proposition 71, the ballot initiative that created the California Institute for Regenerative Medicine or CIRM as the agency is formally known. He is a partner in the Oakland law firm of Remcho, Johansen and Purcell, which also has an office in Sacramento. The firm specializes in political, election, ethics, constitutional and public policy law.

Harrison was known for his work on ballot measures well before he became involved in the Proposition 71 campaign, He also has worked in election law and campaign finance.

Harrison never was formally classified as an employee of the stem cell agency. His services came under a contract with his law firm. For the current fiscal year, that contract amounts to $575,000.

A 2009 memo to the stem cell agency board recounted Harrison’s value to CIRM, ranging from defending its constitutionality to its public records policy. The memo, prepared at the behest of Bob Klein, the agency’s first chairman, said that Harrison had been “has been involved in virtually every aspect of the agency’s operations, including defending the agency in litigation, drafting and reviewing agency policies, advising the agency on conflict of interest issues, and interfacing with constitutional officers and legislators on matters ranging from financing to proposed legislation.”

Mills said that Scott Tocher, deputy general counsel for the agency, will work closely with Harrison to ensure continuity during a transition period. Tocher is also a longtime veteran of CIRM affairs, joining the agency in 2005.
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A "Blind" Warning to Patients: Beware Unproven, Unregulated Stem Cell Treatments

“Disastrous” is how the New York Times this week described an ostensible stem cell treatment in Florida that left three older women blind or nearly blind. "Chilling reading" said the $3 billion California stem cell agency.

If you haven’t read about the matter yet, it is worth some attention. Denise Grady of the New York Times wrote,
“The cases expose gaps in the ability of government health agencies to protect consumers from unproven treatments offered by entrepreneurs who promote the supposed healing power of stem cells.”
Her article was based on a report in the New England Journal of Medicine that dug into the medicial and scientific details.

The matter involves loosely or non-regulated stem cell treatments in clinics that have shot up around the country, Paul Knoepfler, a stem cell research at UC Davis, and Leigh Turner, a bioethicist at the University of Minnesota, reported last year that at least 570 exist around in the country, with the most in California.

Kevin McCormack, director of communications at California's stem cell agency, wrote about the Florida matter on the agency's blog, He said,
"The report makes for chilling reading."
McCormack said it is a "warning to all patients about the dangers of getting unproven, unapproved stem cell therapies."

Knoepfler wrote on his blog,
“Is this just the tip of the iceberg for negative stem cell clinic outcomes given that there are around 600 such clinics in the US today largely operating generally without FDA approvals, lacking preclinical data to support what they are doing, and experimenting on thousands of patients for profit? 
“Where was the FDA in all of this and are they doing anything about it now?”
Knoepfler continued,
“The website remains a great, but also very problematic resource. Many patients seem to view anything listed on there as a legitimate, NIH-approved full-blown clinical trial and some clinics encourage that view, but that’s clearly not the case. I interviewed the leader of in 2014 and problems were already apparent to me back then from that discussion. needs to provide patients with much more information (e.g. IND status, fee as inclusion criteria, etc.) and consider excluding certain listings.”While it is quite possible that it lacks the budget, the flexibility, and/or the authority to make such changes quickly, a lot is at stake. They need to make changes ASAP.”
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Friday, March 10, 2017

California's Alpha Clinics: A Unique, Stem Cell 'Clincher' for Patients, Doctors and Researchers

California's $3 billion stem cell agency this week explored the status of its ambitious Alpha Clinic network, touting its impact and progress over the last two years involving hundreds of patients.

The network, which now has bases only in Southern California, is slated to add two more this year in what will be a $40 million investment by the state of California. It is likely to be a major part of the legacy of the California Institute of Regenerative Medicine or CIRM, as the Oakland-based stem cell agency is formally known. The agency runs out of cash for new awards in 2020.

Geoff Lomax, senior officer for CIRM's strategic initiatives, yesterday wrote about the Alpha network in an item on the agency's blog, The Stem Cellar.

He noted the state’s position as a world leader in stem cell research, helped mightily by the efforts of the 12-year-old agency. He wrote,

“But the real clincher is that California has something that no one else has: a network of medical centers dedicated to stem cell-based clinical trials for patients.”

Lomax, who was one of the first employees at the agency in 2005, said,

“So far, hundreds of patients have been treated at our Alpha Clinics. These top-notch medical centers use CIRM-funding to build teams specialized in overseeing stem cell trials. These teams include patient navigators who provided in-depth information about clinical trials to prospective patients and support them during their treatment. They also include pharmacists who work with patients’ cells or manufactured stem cell-products before the therapies are given to patients. And lastly, let’s not forget the doctors and nurses that are specially trained in the delivery of stem cell therapies to patients.

“The Alpha Clinics Network also offers resources and tools for clinical trial sponsors, the people responsible for conducting the trials. These include patient education and recruitment tools and access to over 20 million patients in California to support successful recruitment. And because the different clinical trial sites are in the same network, sponsors can benefit from sharing the same approval measures for a single trial at multiple sites….

“This collective expertise has resulted in a 3-fold (from 12 to 36 – two trials are being conducted at two sites) increase in the number of stem cell clinical trials at the Alpha Clinic sites since the Network’s inception.”

The agency is seeking applications to open two more Alpha Clinics this year, funding them with $8 million each. One of the criteria is geographic diversity. The application deadline is May 15.

In just 13 days, folks interested in Alpha Clinics can take part in a one-day symposium at the City of Hope in the Los Angeles area, which is one of the current Alpha sites. Advance registration is requested for the free event.

The other Alpha sites are at UC San Diego and UCLA/UC Irvine.
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Monday, March 06, 2017

California Award Recipient: 'Promising' Results from Chinese Stem Cell Trial for Knee Arthritis

Cellular Biomed has a four-year U.S. history
A Chinese biomedical firm that popped up last month with an award from the $3 billion California stem cell agency is working with the University of Southern California and Children's Hospital in Los Angeles to ready an arthritis therapy for a clinical trial in the United States.

The publicly traded enterprise is Cellular Biomedicine Group, Inc., which has an office in Cupertino, Ca., as well as China. It was awarded $2.3 million from the California Institute for Regenerative Medicine or CIRM, as the state stem cell agency is formally known.

The company said in a press release that the therapy, known as AlloJoin, is already in a phase one clinical trial in China where it is showing "promising interim 3-month safety data." The company described the therapy as an “off-the-shelf, allogeneic, human adipose-derived, mesenchymal stem cell" treatment.

Qing Liu-Michael, USC photo
Thomas Vangsness, USC photo
The company said it is working with C. Thomas Vangsness, Jr., at the Keck School of Medicine at USC, and Qing Liu-Michael of the Broad Center for Regenerative Medicine, also at USC. Vangsness will be principal investigator on the company's phase one U.S. trial.

The company said the CIRM grant is the first step in bringing its arthritis treatment to the United States. It is also contributing $572,993 to the work  being funded by the state agency.

In an interview last week with Javier Hasse of Benzinga, an online financial information site, Tony Liu, chief executive officer of Cellular, said his company is also working on  immune cell therapies involving cancer. He said his company's focus is on China because of the size of the market.

Liu said,
"In China, 57 million people have a knee issue; in the U.S., 27 million [people] have a knee issue. Stem cells can help knees regenerate by doing two things. First, by helping with the pain, providing symptom relief and functional improvements. Secondly, they regenerate the cartilage, which originally caused the knee problem. Nowadays, patients can only opt between pain pills or a knee replacement.
Today, if you do a knee replacement, you are looking at tens of thousands [of dollars]. So, any way you look at it, [it’s a] multi-billion [market] for knee treatments."
Liu continued,
“Our management team was educated in the U.S., and has experience managing large businesses.... “Our chief scientific officer is a former MedImmune/AstraZeneca plc (ADR) (NYSE: AZN) director. Some of our oncology scientists are from there as well. We also have scientists from the National Cancer Institute. We also have a person who is leading our manufacturing capabilities who worked for Harvard for 30 years and a top German company, leading research for seven years total.”
“So, we have this kind of people with skills come to China. Our company has 130 people with PhDs, and more than 30 with post-doctorate studies, so there is a lot of brain power, I believe, and we have a  common vision that is to create the best, first in class, biotech business in China.”

As for the company's finances, Liu said,
"CBMG’s stock is really thinly traded. Much of the stock is owned by those who have been with the company for a long time; so, they don’t sell. Having said this, there are many reasons that drive stocks: the U.S. election, the pricing discussion… Many investors don’t discriminate, and just punish biotech as a whole. However, CBMG is not really subject to most of these pricing pressures. In fact, because we have a different cost structure, I expect CBMG to do extremely well." 
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Thursday, March 02, 2017

'Pressure testing' a $150 Million California Plan to Lure Business into Development of Stem Cell Therapies

The California stem cell agency's far-reaching plan to crate a $150 million, public-private partnership to speed development of stem cell therapies remains on hold as the agency looks for ways to lure in private enterprises.

In an interview last week with the California Stem Cell Report, Randy Mills, president of the $3 billion agency, said he had nothing fresh to report on the project, which did not draw any eligible applicants last fall.

Mills said his team is "pressure testing (the project) to make sure people really understand the terms."

In December, Mills said that the firms that responded to the request for applications in October were seeking to make a "better deal" than the agency had offered. Under the proposal, a new company would be created to develop therapies. It would be backed by a $75 million state loan (discounted to 50 percent payback)  with a matching $75 million coming from a successful applicant. The company would have the pick of agency research that did not already have a commercial partner.

Mills said that the unusual nature of the proposal posed challenges for the company and the agency.

In the interview last week, Mills said the agency is seeking to determine if having more time to prepare proposals will trigger more applications. Another factor is whether the terms do not meet the needs of potential applicants. Sphere: Related Content

Sunday, February 26, 2017

California Popping for $40 Million for its Alpha Clinic Stem Cell Centers

CIRM video
California this year expects to add two new Alpha stem cell clinics to the three already in Southern California, boosting its investment in the effort to as much as $40 million.

The move came last Thursday when directors of the state's $3 billion stem cell agency approved seeking additional Alpha Clinic applications this spring. The purpose of the clinics is to accelerate development of stem cell therapies, a key goal of Proposition 71, the ballot measure that created the California Institute for Regenerative Medicine or CIRM as the Oakland-based program is known.

Expansion of the program could also mean that an Alpha site may be located somewhere in Northern California by the end of this year. One of the objectives of the latest proposal is to broaden the "geographic reach" of the Alpha network.

According to CIRM, the existing Alpha clinics already have 33 clinical trials underway for 12 different afflictions. Randy Mills, president of the agency, said in an interview Friday with the California Stem Cell Report that the clinics doubled the number of stem cell trials that they had prior to launching their CIRM Alpha programs. Many of those trials do not require agency funding, which allows the cash to be used elsewhere.

Maria Millan, CIRM's vice president for therapeutics, told CIRM directors last week,
"Once we built it, they did come."
The latest expansion is aimed at increasing capacity and access to stem cell trials, training physician/scientists in clinical trials and bringing additional assets to California's Alpha clinic network, which is also positioned to help establish the state as the global leader in stem cell treatment.

The request for applications, with a deadline of May 15, will also require that the clinics -- whether non-profit or for-profit -- create a "sustainability plan," which will keep them operating after the CIRM funding terminates in four years. The agency itself expects to run out of cash for any new awards even sooner -- June 2020.

CIRM is offering to provide two winners in the Alpha round $8 million each over a four-year period with milestones required to keep the cash flowing.

During Friday's interview, Mills said that applicants from Northern California will need to make a case for the "added value" that they will bring to their proposals and demonstrate that their populations are underserved. Additional applications are likely to come in from Southern California, which has the bulk of the state's population and patients.

The current Alpha Clinics are located at the City of Hope in Duarte, which is in the Los Angeles area, UC San Diego and UCLA/UC Irvine.

A symposium on Alpha Clinics is scheduled for March 23 at the City of Hope. Advance registration is requested.  

City of Hope video

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Friday, February 24, 2017


An item yesterday on the meeting of the governing board of the stem cell agency incorrectly stated that the board approved $37 million in awards. The correct figure is about $33 million. Sphere: Related Content