Friday, January 12, 2018

California Stem Cell Spending: Priming a $1.5 Billion Research Influx

California's $3 billion stem cell agency this week reported on "what you may not know" about the unusual, 13-year-old research organization. 

Its "disclosure" did not deal with such matters as the fact that it is the first such enterprise in state history or that it operates outside of the purview of the governor and the legislature. Rather it dealt with, among other things, leverage -- a term used often in the real estate business to describe, for example, using  $10 to create $1,000.

In the case of the California Institute for Regenerative Medicine (CIRM), as the Oakland-based agency is formally known, it said it has created $1.5 billion in leveraged funds. It also noted that it has served as a "validator" for a significant amount of research, meaning that its awards re-assure the nervous nellies of finance about making an investment in a particular project. 

CIRM's seal of approval has led to $528 million in additional "partnership" funding for specific research, the agency said, along with more than $395 million in awards from other agencies that were built on CIRM-backed research. . 

News about the agency often focuses on stories about people and their afflictions. Those emotional tales are powerful ways to tell the CIRM story, but its less well known ventures into leverage and validation also speak to its impact, both short term and long term. 

Kevin McCormack, CIRM's senior director of communications, wrote about all this earlier this week with specific numbers and details in the agency's blog, The Stem Cellar, in a preview of the agency's annual report.
"Our goal is to do all we can to support the best science and move it out of the lab and into clinical trials in people. Obviously, providing funding is a key step, but it’s far from the only step. For us, it’s really just the first step."
CIRM does report its impact in the best possible light. However, there is little doubt that it has played an important role in "de-risking" much stem cell research and creating a friendly environment that is more likely to attract additional financing for a still young field.  Sphere: Related Content

Wednesday, January 10, 2018

California Pumping $19 Million More into Stem Cell Effort to Improve Kidney Transplant Success

The California stem cell agency is set to make a nearly $19 million bet next week on a treatment that is aimed at significantly improving the success of  kidney transplants and helping to reduce healthcare costs.

The agency's board is expected to ratify a decision to award $18.8 million to Medeor Therapeutics, Inc.,  of San Mateo, Ca., for a phase 3 clinical trial, the last stage before its proposed product can be widely used.

Next week's award will bring to $25.4 million that the state has invested in the work. The agency awarded $6.7 million for the research in 2016.

The company's chief medical officer, D. Scott Batty, Jr.,  said last spring that its product, dubbed MDR-101, "has the potential to address the two most critical transplant patient needs: preventing organ rejection and mitigating anti-rejection treatment-associated toxicities. " He added that the technology could potentially be used in all solid organ transplant patients.

Fierce Biotech reporter Phil Taylor wrote last April,
"Patients who undergo organ transplants may no longer have to rely on lifelong immune-suppressing drugs, if Medeor Therapeutics has its way."
Taylor continued,
"More than 30,000 Americans get an organ transplant every year, and while success rates for these procedures are improving, it is estimated that up to a third of the most common transplants—such as heart, kidney, and liver—fail within 5 years."
The Medeor treatment uses adult stem cells to create a condition in which the transplanted kidney "is no longer viewed as foreign by the recipient," according a summary of the closed-door review of the company's application (CLIN2-10411). 

The agency's reviewers, who do not have to publicly disclose if they have  potential conflicts of interest, voted 11-1 to fund the research. The agency's board almost never overturns the decisions of its reviewers, who come from out-of-state. The names of persons reviewing specific applications are not disclosed by the agency, which also does not disclose the name of applicants until after board action. The California Stem Cell Report identified the firm from public records.

Samuel Strober, Stanford photo
Last November, Medeor announced it had raised $57 million in Series B financing, "led by RA Capital Management. Additional new investors included Sofinnova Ventures and 6 Dimensions Capital, who were joined by existing investors Vivo Capital and WuXi Healthcare Ventures."

The scientific founder of the firm is Samuel Strober, a professor of medicine at Stanford and who is a member of its scientific advisory board. 

The estimated date of completion of the trial is January 2022, according to clinicaltrials.gov. The governing board of the $3 billion stem cell agency is scheduled to meet Jan. 18 to approve the award. No other action is scheduled. Sphere: Related Content

Monday, January 08, 2018

California Stem Cells, 'Rapture' and a $5 Billion Bond Measure

This past weekend brought news about a "rapturous agreement among Republicans and Democrats" to support development of new therapies by financing biomedical research with billions of dollars.

The New York Times article indicated that the rapture could have something to do with the fact that many key political leaders on Capitol Hill are "aging in place."

The piece by Robert Pear also carried implications for the $3 billion California stem cell agency, which has virtually endorsed a yet-to-be-written, $5 billion bond measure to stave off its own death.


Pear wrote on Sunday:
"For the third straight year, lawmakers (in Congress) are planning to increase the budget of the National Institutes of Health by $2 billion. In the process, they have summarily rejected cuts proposed by President Trump.
"The push for additional funding reflects a fascination among legislators with advances in fields like molecular biology, genetics and regenerative medicine, even as they wage bitter battles over just how large a role the government should play in financing health care and providing coverage."
Pear continued, 
"Why is medical research so much less contentious than fundamental issues like health insurance coverage?
"Anthony J. Mazzaschi, a lobbyist at the national organization representing schools of public health, said that 'the charisma of the cure, the hope and promise of curing disease, seems to excite members of Congress,' including some in their 70s and 80s who are 'facing the prospect of disease and disability head-on.'"
The Times piece said, 
"The challenges facing patients and policymakers were illustrated this past week when a Philadelphia company said it would charge $850,000 for a new gene therapy to treat a rare inherited form of blindness. (The company, Spark Therapeutics, said it would pay rebates to certain insurers if the medicine, given in a one-time injection, did not work as promised.)"
California's stem cell agency is running out of money. It is looking at a November 2020 ballot measure to fill its shrinking coffers with $5 billion more. But such public policy decisions involve trade-offs concerning the health of Californians.

Several questions (among others) arise theoretically and otherwise:
  • Would it be better to spend $5 billion to provide much needed, immediate health care to poor Californians, including mothers and children, which could provide a quicker payoff in improved health. 
  • Is it better for the state to spend $20 million to help reduce infant mortality rates in Fresno and Mendocino that are nearly twice the statewide average or give the cash instead to a stem cell researcher (and his employing institution) who can also win the cash from the NIH. 
  • The impetus for creation of the California stem cell agency was based on a restriction in federal funding for human embryonic stem cell research. That restriction no longer exists, but could be restored by the Trump administration. But should California spend its billions when researchers can seek funding at the federal level? 
These are sort of bottom-line policy questions that ignore the fact that bond issues generally are used for long-lived assets, such as prisons, educational buildings, highways, etc., -- not services such as health care. The bond measure that provided the stem cell agency with its only significant cash was an historic exception.

Nonetheless, in the public's eyes the stem cell agency is all about health. As the campaign that created the agency in 2004 noted, medical problems that affect nearly half of California families "could benefit from stem cell research."

The likely cost of the therapies that are being developed in the agency's clinical trials are almost never discussed in public. Yet many are likely to be as expensive as the Spark Therapeutics blindness treatment. One can only imagine the kind of opposition advertising that is likely to surface in a campaign for a $5 billion bond measure: "Vote No on Million Dollar Cures for Billionaires!" On the other hand, that pitch might seem too harsh and blow back on agency opponents.

Nonetheless, Americans believe drug costs are too high, something that will come into play during a 2020 campaign. According a recent Gallup poll, 78 percent of the people also say total health care costs are too expensive. Drug costs have become an easy litmus test for the expense of staying healthy.

The New York Times story noted the importance of age as helping to create support for spending on medical research. California is headed for a sharp increase in the numbers of seniors in the next decade. One study is even projecting a "silver tsunami" of cancer in older patients nationally. Generally, however, older voters are considered more likely to be conservative. and may not buy into more government spending. How that plays out in California is not clear when it comes to stem cell research or the hope for stem cell cures.

Hope is one of the more powerful products of the California stem agency. Ultimately that could be the deciding selling point. But the November 2020 election is 34 months away, and much is likely to change.
Sphere: Related Content

Friday, January 05, 2018

Tracking the $3 Billion California Stem Cell Agency Month by Month

Directors of the California stem cell agency plan to meet 12 times during the coming year, including four valuable face-to-face sessions that are likely to be based at its Oakland headquarters.

Valuable because those sessions offer an opportunity to chat directly with directors and the agency's staff. The face-to-face sessions are scheduled for March 13, June 28, Sept. 20 and Dec. 13. Topics for the meetings will come later when agendas are posted 10 days before the sessions. But the June and December meetings have often dealt with important financial and strategic issues.

Researchers who have attended the board sessions in the past have benefited from gaining a greater understanding of the application review process and procedures of the board as it ratifies reviewer decisions and hears appeals.

The other eight meetings are telephonic and are scheduled to occur on Jan. 18, Feb. 22, April 26, May 24, July 26, Aug. 23, Oct. 18 and Nov. 29.

To be announced during the year on an ad hoc basis are meetings of the committees of the governing board, which regularly consider important issues and approve policies and actions that are often summarily ratified by the board.

All of the meetings can be tracked by subscribing to notifications from the agency and by reading the California Stem Cell Report, which tries to provide a deeper look at some of the matters facing the stem cell agency.

The public can attend all the meetings at either remote locations and the CIRM headquarters and can address the board directly. If you just want to listen, an audiocast is available. After the meetings, which are also accessible as a recording, transcripts become available a couple of weeks later.  Sphere: Related Content

Places to Go, Stem Cell Science to See in 2018

The $3 billion California stem cell agency did all you stem "cellists" out there a big favor this week with a rundown of the hottest and most interesting conferences coming up in 2018. A UC Davis researcher also has put his own list on his own blog.

First, the agency's list of what it describes as "awesome" conferences. They range from industry-oriented events to ones devoted to science. Coming up next week is a "state of the industry" briefing by the Alliance for Regenerative Medicine in San Francisco. The World Stem Cell Summit runs at the end of this month in Miami.

A discussion of the agency's Alpha Clinics program is scheduled for April 19 at UCLA. Last year's Alpha program at the City of Hope was excellent. December will bring a Cedars-Sinai conference on Translation of Stem Cells to the Clinic, Challenges and Opportunities, which could be illuminating as the stem cell agency pushes to fulfill the promises of the ballot initiative campaign that created the research effort.

Of course, there are many more -- much too many to list here.The Oakland-based agency has also provided links to the conferences it highlights

Over at UC Davis, scientist Paul Knoepfler's list included some of the same sessions. He also is soliciting information on conferences that he has not listed. You can send him your information by clicking here. 

Sphere: Related Content

Wednesday, January 03, 2018

Stem Cell Person of the Year: Michele DeLuca of Italy

An Italian scientist has been named Stem Cell Person of the Year for 2017 by The Niche, a stem cell blog produced by a California researcher.

Michele DeLuca, CMR photo
Paul Knoepfler of UC Davis announced the selection of Michele DeLuca on his blog last week.
Knoepfler wrote,
"I chose him because of his exceptional track record in stem cell and regenerative medicine science combined with a long record of advocating for responsible translational science."
The award carries of a cash prize of $2,000, which Knoepfler funds himself. The California stem cell scientist commented,
"Dr. De Luca also led an international team that recently published a groundbreaking paper on epidermal regeneration in a pediatric patient with epidermolysis bullosa. The new paper reported an innovative combination stem cell-gene therapy approach to this terrible disease. I reviewed the paper here in journal club style. The manuscript is an important starting point in new therapy development. It’s the kind of work that is very risky and needs to be done in a meticulous way. Dr. De Luca is one of the few people in the world who could have led this work."
Knoepfler is the sole arbiter on the award but does ask his readers to indicate their preference. In the case of DeLuca, he received 2.98 percent of votes cast. 

Knoepfler started the awards in 2012 with an initial prize of $1,000. Sphere: Related Content