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

Highlights

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. 

Japanese and California Stem Cell Affairs: An Opportunity to Make a Connection

This baby is a spin-off in Japan from CIRM-financed research.  Kazuhiro Kawamura 

of the St. Mariana School of Medicine delivered the child, which he is holding.

 (Kawamura photo)

Scientists and other stem cell fanciers in Japan will have their first chance this Thursday to take part in a meeting of the governing board of the $3 billion California stem cell agency.

Ken Burtis
UC Davis photo

That's because one of the board members, Ken Burtis of UC Davis, is in Nara, Japan, for a visit on the day of the meeting in Burlingame, Ca. He will be linked to the session via a telephone connection. It will be a two-way hookup that the public can use to participate, a requirement of California state law.

Stem cells are a hot scientific and commercial topic in Japan. According to an article last November in the Japan Times, the country's regenerative medicine market is expected to climb to $15.85 billion in 2030, up from $260 million in 2012. Japan is also the home of the induced pluripotent stem cell, which was first produced there.

Burtis is a professor of genetics and provost at UC Davis. It was not immediately known whether his visit to Japan involved UC Davis, the stem cell agency or was personal.

Burtis' access to the stem cell meeting, which includes a lengthy briefing on the agency's development portfolio, will be from the Hotel Nikko in Nara. Interested parties will be able to participate from the room in which Burtis is monitoring the meeting. However, the meeting agenda does not specify a room number. That will have to be obtained by emailing the stem cell agency at info@cirm.ca.gov. It is best to do that well in advance of the meeting.

This week's meeting has nothing specific on the agenda related to Japanese stem cell affairs, but stem cell research is a global matter. Researchers and others in Japan may well learn something new, particularly from the briefing on the agency's portfolio, and will have an opportunity to pose questions. Additionally, the board will be considering $72 million in "concept" proposals to speed commercialization of stem cell research, which could well be of interest to Japanese stem cell researchers and biotech firms even if they are not eligible for awards.

The California stem cell agency, which is known as CIRM, has also had a collaborative arrangement with Japan Science and Technology Agency since 2008.

Masaya Nakamura
Keio photo

Aileen Anderson
UCI photo

The agreement has resulted in one collaborative funding project involving Aileen Anderson of UC Irvine and Masaya Nakamura of Keio University. Anderson has received $1.3 million from CIRM, which did not announce the amount of funding that Japan provided to Nakamura.

Aaron Hsueh
AFP photo

Aaron Hsueh of Stanford received $2 million from CIRM for work that later led to a novel way of treating some forms of infertility and further work with Japanese researchers. One child has been born in Japan using the techninque. Kazuhiro Kawamura (pictured at the top of this item) and others at St. Mariana University School of Medicine were involved in that effort, which was not funded by CIRM. Another woman was pregnant as of October 2013. No information about the result of that pregnancy was immediately available. (See here and here.)

(Editor's note: This item has been altered slightly from the original version to make it clearer what is on the agenda this Thursday and its relationship to Japan. The headline has been reworded. No information has been dropped.) 

Cost of a Stem Cell Therapy? An Estimated $512,000

(Editor's note: Updated figures on costs can be found in this 2017 item.)

The likely costs of potential stem cell therapies and cures receive almost no attention in the media as well as publicly from scientists and the biotech firms.

Usually any public discussion is obliquely framed in the context of “reimbursement,” as if industry is owed something instead of making a business decision about what will make a profit. Euphemisms and jargon cloak unpleasant realities such as astronomical patient costs. But what reimbursement really involves are, in fact, pricing decisions and profit margins along with lobbying campaigns for inclusion of therapies in normal coverage of health insurance and Medicare. 

And today a singular figure – $512,000 for one stem cell treatment – appeared in the Wall Street Journal . The story by Kosaku Narioka and Phred Dvorak dealt with what would be the first-ever human study of a treatment that uses reprogrammed adult stem cells.

They reported that the study received preliminary approval on Wednesday from a key panel of the Japan Health Ministry. The treatment involves a form of age-related macular degeneration, which has also been targeted by the California stem cell agency with different approaches.

Buried deep in the Wall Street Journal article, with little other discussion, was this sentence:

“One eventual obstacle, even if tests go well, could be cost: (Masayuki) Yamato (of Tokyo Women's Medical University) says initial estimates for the treatment run around ¥50 million ($512,000) per person."

The subject of costs for potential stem cell treatments has rattled around in the background for years without much deep public discussion. One reason is that high costs of treatments are controversial and can trigger emotional debate. Another reason is that it is very early in the therapy development process and estimates are not likely to be entirely reliable.

A few years ago, however, the California stem cell agency commissioned a study involving costs of stem cell therapies. The UC Berkeley report said,

“The cost impact of the therapy is likely to be high, because of a therapy’s high cost per patient, and the potentially large number of individuals who might benefit from the therapy. This expense would put additional stress on the Medicare and Medicaid budgets, cause private insurance health premiums to increase, and create an incentive for private plans to avoid covering individuals eligible for a therapy.”

The findings did not seem to be exactly welcomed. The agency sat on the 2009 study for seven months until it was uncovered by the California Stem Cell Report in April 2010. Then the agency was careful to say that the study did not reflect the view of CIRM management or board leadership.

Their wariness of being out in front on the issue could be well-advised. The pharmaceutical industry received some unpleasant attention this spring when more than 100 influential cancer specialists from more than 15 countries publicly denounced the cost of cancer drugs that exceed more than $100,000 a year.

Nonetheless pricing is critical to both patient accessibility and therapy development. If companies cannot make a profit on a possible therapy, it is virtually certain not to appear in the marketplace.

While the subject remains in the background, it does not mean there is a lack of interest. The copy of the Berkeley stem cell cost study that was posted online by the California Stem Cell Report has been read 11,701 times since it was made available in April 2010 on scribd.com.

(For a 2015 look at costs for non-government approved procedures, see this item.)

A copy of the Berkeley study can be found below.

Stem Cell Therapy Costs by DavidJensen