The Niche

Crain’s Cleveland Business has an article reporting that investors and established companies are growing more interested in several adult stem-cell technologies. (Of course, given the current economic situation, investors are generally acting more shy than interested in anything.) The article discusses several companies focusing on those around Cleveland, Osiris is discussed, of course, but also Cytori, Athersys, Arteriocyte, AcelleRX Therapeutics. and Cell Targeting. (Our article In search of a business model also looks at this topic, primarily interviewing representatives of investors and big drug companies.)

Also relevant is a biotech start-up, Fate Therapeutics, which is using conventional drugs to target stem cells within patients’ own bodies. (See our article Stem cell therapies the old-fashioned way)

Posted by Monya Baker on January 07, 2009
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Salaries and appointments for CIRM officials
After lots of people (including me) indulged in headlines touting half-million suppositions, the CIRM board decided to pay Bob Klein $150,000 a year for what it deemed a half-time position. The San Francisco Business Times provides a nice overview, including the potential hiring of California Democratic Party Chairman Art Torres for the vice-chair position at just over $300,000 per year, and Governor Schwarzenegger’s concerns about paying both these positions. The Sacramento Bee has an article on Torres.

Some companies get tools grants
In the last round of grants for creating new pluripotent stem cell lines, biotech companies cried foul that only applications from academics got the dough. (See What got funded.) They have less to complain about in this round. And while San Diego was bitterly disappointed that San Francisco (my fair city) won the seat of the California Institute of Regenerative Medicine, they should be cheered that four San Diego biotechs won grants. In addition, Duane Roth, one of the biggest “go-to” people in the San Diego biotech community, is reported to be Schwarzenegger’s pick for the vice-chair position eyed by Torres. XConomy reports that funds will soon be flowing to NovoCell for a pouch that can be used to transplant insulin-secreting cells without triggering an immune response, to Invitrogen (now known as the hard-to-Google “Life Technologies”) to use stem cells to model neurodegenerative disease, to Vala Sciences to make heart cells from stem cells, and to a joint effort by Fluidigm (which is in South San Francisco) and StemGent for techniques to find better ways to induce differentiated cells to pluripotency. The San Diego Tribune describes some funded technology more fully, along with the disappointments of one of the industry applicants that did not get funded. The two other companies to win grants were Gamma-Medica Ideas (with offices in Northridge, California, plus Norway and Canada) for ways to visualize single stem cells in the body and Vistagen (based in South San Francisco) for ways to use stem cells to screen drugs for potential liver toxicity.

Here is a list of the 23 grants awarded to 18 institutions, along with links to each application. Awarded funds totaled $19 million. This round of grants was targeted to develop technologies that could speed the development of therapies rather than become therapies themselves.

Posted by Monya Baker on December 12, 2008
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Two papers show how ES cells change in culture, and the head of NovoCell heads to a nonprofit.

First, the gossip, actually a news article from the San Diego Union Tribune. The head of Novocell, Alan Lewis, has headed over to the deep-pocketed non-profit Juvenile Diabetes Research Foundation, which has become a leading force in stem cell research. ( Bob Klein, head of CIRM, is on its board of directors, and the agency, along with NIH and CIRM, are American members of the International Stem Cell Initiative.) Alan Lewis is featured in our article on stem-cell start up companies, In search of a viable business model.

Next, one more thing for those culturing stem cells to worry about.

A paper in PNAS shows that mouse ES cells pick up copy number variants after just a few passages in routine culture. Here is a link to the research highlight. (We reported on CNVs in human ES cells several months ago and also ran a commentary about how to assess a stem cell genome).

More recently, two Nature Biotech papers show amplifications of sections of chromosomes containing several genes. Here’s the draft of that, as reported by excellent freelancer Simone Alves.

Continue reading "ES cells change chromosomes; stem-cell CEO changes jobs" »

Posted by Monya Baker on November 26, 2008
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Three University of Wisconsin companies founded by ES-cell rock star James Thomson are joining up, according to an article from the Associated Press. The new company will be called CDI and combines Cellular Dynamics, Stem Cell Products and iPS Cells.

The first quote from the CEO is both vague and ambitious. Bob Peley’s company "intends to be the world leader in the industrialization of basic stem cell technology." Later, the article reveals the more-tangible goal of creating cardiomyocytes for drug screening. In fact, Cellular Dynamics had announced a deal with Roche to do this in the first quarter of this year. (See our article on this and other stem cell start-ups in http://www.nature.com/stemcells/2008/0810/081030/full/stemcells.2008.138.html In search of a viable business model )

Posted by Monya Baker on November 25, 2008
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The pharmaceutical giant made it official today. It has launched a regenerative medicine unit co-located in Cambridge, UK and Cambridge, MA. It will have about 70 full-time employees, but the cheery news for stem-cell start-ups is the focus on deal-making. The company’s press release hinted that several scientific collaborations would be announced this week, and Pfizer’s head of bio-innovation reportedly said that this initiative might very well help some young companies stay alive through the financial crisis. (See the Wall Street Journal blog. For a broader view, see In search of a viable business model.)

The first time I heard a Pfizer official talk about this was at a stem cell conference in September (See Companies have company at stem-cell conference). The company line was caution: stem cells would be tools to finding, vetting, and assessing small molecule drugs. Cell therapies were a stretch. Sure, at that point, Pfizer had already invested in a little cell therapy company for eye disease, but those funds comprised the tiniest fraction of a giant R&D budget (See Pfizer dips a toe in stem-cell research). Today, ten days after the election of a stem-cell-friendly US president Pfizer sounds more ambitious. “Scientists at Pfizer Regenerative Medicine will explore the use of stem cells to develop future treatments that may prevent disability, repair failing organs and treat degenerative diseases. The ultimate goal will be to deliver new medicinal products that can pave the way for the use of cells as therapeutics.”

Posted by Monya Baker on November 14, 2008
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Cheaper genome scanning
Next year, the cost of sequencing a human genome will fall to $5000, according to start-up Complete Genomics in a story in The New York Times. The start-up expects that individual people will be the chief customers, but I’d imaging those characterizing stem cell lines wouldn’t mind getting some additional data, both about the existing variety of stem cell lines and about how individual stem cell lines change genetically as they adapt to culture. (See our commentary on how to assess a stem cell genome.)

Paperwork for embryo donors
Besides providing more genetic diversity, newly derived embryonic stem cell lines could be derived and maintained under better conditions for culture and informed consent. While recent surveys show individuals are willing to donate unwanted frozen embryos for research, an article in The Los Angeles Times describes some of the paperwork burdens involved.

Private money for Stanford stem-cell building
Meanwhile, BusinessWire founder Lorry Lokey is giving $75 million to Stanford for a stem-cell facility, according to the San Jose Business Journal. In the article, the Stanford Graduate and entrepreneur compares stem cells to the silicon chip. The total cost of the building will be $200 million, of which $44 million is coming from tax-payer funded California Institute of Regenerative Medicine; the university and other contributors are supposed to foot the rest of the bill.
Also, here’s a story from the San Francisco Chronicle.

Posted by Monya Baker on October 06, 2008
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Posted by Monya Baker on behalf of Natalie DeWitt

What do the top venture capitalists interested in investing in iPS cells consider the major hurdles on the road to viable clinical products? Today stem-cell scientists Deepak Srivastava and Shinya Yamanaka of the Gladstone Institute in San Francisco were joined by Beth Seidenberg of venture-capital firm Kleiner Perkins Caufield and Byers to answer that question for an audience consisting mainly of investors.

Yamanaka was reluctant to give a timetable for clinical application. Alternatives to viral integration as a means to make iPS cells are clearly on the way, he said, but the similarity of reprogrammed cells to cancer cells presents another serious hurdle. Srivastava added that expansion of cells is another major focus—how to generate the large quantities of cells needed for human therapy.

As someone who is mainly preoccupied by the science, I found it fascinating to hear Siedenberg, a drug development expert for a venture capital firm, break the road to clinical development into the following seven steps, two scientific feats to be established, plus a series of questions that clinicians and regulators must figure out how to answer. (My own observations are in parentheses.)

• A way around viral transduction to reprogramming must be developed. (This is a hot area of research and already small molecules are being screened and developed to do this).
• Differentiate iPS cells into any cell type. (Many papers report differentiation of ES cells to useful cell types such as neurons or cardiomyocytes—but only in rare cases do these procedures achieve robust efficiency. It is proving to similarly be a challenge for iPS cells).
• What is the best way to administer the cells? Will novel biomaterials to form scaffolds be needed?
• What safety and regulatory process must be in place before using cell transplantation in humans?
• What dose will be required and how often?
• How do you gauge the efficacy of the cells destined for transplantation? How do you know if they are working?
• And a major hurdle, how to handle the manufacturing of cells to control costs and reproducibility?

The first two goals are well underway in scientific labs. But the next five are key for developing therapies, and are the questions investors and drug developers ask about any product in their pipeline. However, using cells for transplantation instead of small molecules raises serious challenges in terms of bringing a cost effective product to market. Do companies developing hES cell therapies like Geron have the answers? By now, the FDA has likely told them what experiments are necessary to move their cell product for spinal cord injury into clinical trials, but so far they’ve been silent.

See also FDA places Geron’s clinical trial on hold. This can get you to Monya Baker’s overview of the FDA’s meeting on how to assess whether embryonic-stem cell derived products were ready for human trials as well as transcripts of that meeting.

Seidenberg pointed out that the nearest term application of iPS cells is testing for drug toxicity. At present, 63% of drugs are withdrawn because of toxicity, usually to the heart and liver. Individuals are susceptible to varying degrees depending to some extent on their genotypes so iPS cells provide a great opportunity for testing toxicities in cells with a wide array of genotypes. And these investigations may, in turn, help answer some of the questions necessary to bring these cells to the clinic.

Posted by Monya Baker on October 02, 2008
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Maryland has just issued requests for proposals in stem cell research for a series of five-year grants for projects with supporting data plus two-year grants for more exploratory research. Graduate students and post-docs across the U.S. can apply to work in Maryland stem-cell labs for fellowships worth up to $55,000.

Elsewhere, initiatives to push stem-cell products toward commercialization are hitting snags. A few weeks after its head was ousted and its board resigned en masse, the Australian Stem Cell Centre has a new interim head and board of directors. (See The Age) The agency has been having a tumultuous time, sparked by debates over whether to follow basic or commercial research. Though a 2006 review of the centre gave it good marks, the ASCC board fired it head Stephen Livesey, after a negative review of the centre. He told an Australian newspaper, that he was frustrated by stakeholders’ skeptical attitudes toward commercialization. See Infighting clouds stem cell centre’s future .

The Australian quotes Alan Trounson, head of the California Institute of Regenerative Medicine, co-founded ASCC in 2002, said the organization “needed to restructure”. Ironically, the structure of the California Institute of Regenerative Medicine is currently undergoing a review of its structure and efficacy. (See The Great Beyond ) CIRM itself is obligated to help stem cells become commercial therapies. It is offering a loan program for biotechs. Biotechs are also eligible to apply for grants which carry an obligation to pay some royalties to the state for commercialized products. At the same time, CIRM must make sure that these therapies will be accessible to Californians. On Monday, California governor Arnold Schwarzenegger vetoed bipartisan legislationguaranteeing access to Californians and making it easier for the agency to fund other sorts of research.

As possibilities for commercialization increase, so will the tumult.

Posted by Monya Baker on October 01, 2008
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I didn’t expect to find so much industry here, at this year’s World Stem Cell Summitt in Madison, Wisconsin. The companies are diverse, from giant New York–based Pfizer to teeny, tiny start-ups, and so are their goals. Pfizer is creating a new division of regenerative medicine, and the new head, John McNeish, spoke of plans to use stem cells to identify new drug targets and to screen drugs for toxicity and efficacy. They’ve already run over a million compounds through assays that used stem cells (I think mouse embryonic stem cells).

But he’s also excited about harvesting the therapeutic potential of stem cells themselves. It’s not cell transplants he’s thinking of, though, so much as finding small molecules (the type that can be made into pills) that activate stem cells within the body. Perhaps the tissue-homing skills of certain stem cells could even be used as drug-delivery devices.

Of course, Pfizer is not alone. McNeish had a lengthy list of other public announcements to establish pharma’s interest in stem cells. At the International Society for Stem Cell Research meeting in June, I met plenty of industry scientists hoping to pick up bench tricks for using stem cells, mainly as a way to test small molecules.

For the most part, the smaller companies here are selling services tocharacterize or grow stem cells. Companies might scale an established line of cells up under rigorously controlled conditions or provide reagents to make growing stem cells more convenient. At a panel, Jonathan Gertler, a boutique investment banker from Leerink Swann, in Boston, Massachusetts, predicted an expanding market for these kinds of services: as less experienced people enter the field, both the market and need for these tools will grow. He estimated that companies that are potentially commercially viable already number almost 100; consolidation is coming, he said.

As the possibilities for making money edge nearer, the real intellectual property fights will begin. And right now the patent situation is very uncertain, said Owen Hughes, who thinks about intellectual property and regulatory issues for Pfizer. Taxpayer-funded reach-through policies (I believe this was code for the California Institute of Regenerative Medicine), he says, only add to the uncertainty.

Greg Simon of the think tank FasterCures, based in Washington, DC, said fiefdoms would hurt science. “We can’t have every state build an infrastructure for intellectual property and then build a castle around it.” He also called for academics’ work with industry to be disclosed, not so much for concerns over conflict of interest, but because that information could be useful for everyone in the space. (There are loads of people who would like to know what reasons the FDA gave for halting Geron’s clinical trial; that way they might be able to better plan out the tests they need to do).

If anyone has ideas for sharing data in their own self-interest for stem cells, please let me know.

Related stories
See Embryonic stem cell trial put on hold

See Thickets and gaps blocking stem cell research

Pfizer dips a toe into cell therapy

Here are all three blogs from the conference

Companies have company

Stem cell trials balancing hope and harm

Stem cell therapies, ready for success?

Posted by Monya Baker on September 25, 2008
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Shortly after the announcement that stem-cell pioneer (and promoter) Advanced Cell Technology was going broke, a venture capital firm has announced that it is looking for investors to pour $225 million dollars into the technology. Here’s the story in the San Francisco Business Times. The firm, Proteus Ventures, is no stranger to stem cell endeavors, including making investments in Saudi Arabia. (See Gulf states embrace stem cell technologies at home and abroad .)

Big pharma has also backed stem cell research, with the latest announcement of a five-year $25 million deal between GSK and Harvard. For the most part, drug companies have been interested in using embryonic stem cells for drug screening (See New tools for drug screening), but Pfizer has made a tiny investment in a cell-therapy startup as well.

And finally, adult-stem cell company Osiris has raised some money for future stem-cell products by selling its current one. The product, Osteocel, is currently sold not as a drug but as a surgical product, which does not have to meet the FDA requirements for an approved drug or cell therapy. According to the Baltimore Sun, Osiris has sold its stem-cell product Osteocel for $137 million and plans to use the money to finance development of biologic products as well as mesenchymal stem cells. These are in clinical trials for heart failure and graft versus heart disease (See Questioning the self cell and Stem cells for the heart, a new wave of clinical trials ).

Posted by Monya Baker on July 28, 2008
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One way of knowing that a technology might have commercial applications is when the patent wars start. This week BioCentury reports that a North Carolina company called Artecel just won an exclusive right to a composition-of-matter patent covering stem cells derived from adipose tissue. According to some reports, stem cells in fat appear to be capable of differentiating into other tissue types. Cytori, a California-based stem cell company that no longer has rights to the patent, was developing the cells for heart disease and reconstructive surgeries. Artecel is less specific about its goals, saying the cells will be used for “soft tissue and cosmetics applications.”

At issue was whether University of California, Los Angeles scientists who had licensed their technology to Cytori had a right to the patent. In 2004, University of PIttsburgh, who had licensed the technology to Artecel, filed a suit to remove the UCLA scientists from the patent. Earlier this month, a United States district court in California ruled in their favor.

Here is the press release from Artecel. In Cytori’s press release, the company said that the decision did not affect its ongoing business practices because the device that enriches collected samples for stem cells for reinjection is still patented. The decision might affect their product pipeline, though.

Posted by Monya Baker on June 25, 2008
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An article in TIME describes a San Diego company that is already offering a procedure using stem cells collected from fat to treat pets with bad hips. Fat is scooped from a dog's abdomen, and then the stem cells are isolated with centrifugation (spinning test tubes of cells so that the heaviest ones go to the bottom) and injected into the problematic area.

The techniques that I know of to isolate stem cells rely on identifying markers on cell surfaces; in fact, the company introduces a mixture of cells to the site of injury. Because these cells are minimally manipulated, they don’t require the FDA’s approval to be transplanted into the same patient they were collected from

But, it’s not so much the cells as it is their effects that matter, and the company has published an article on these effects in a journal indexed in PubMed. It examines results of treatment for 90 days and found improvement in the stem cell group. A follow-up would be interesting because these effects might be transient. Human transplants of mesenchymal stem cells for nonorthopedic indications sometimes show initial improvement that quickly fades away.

The TIME reporter writes that these cells then become cartilage and tendons, and it is true that mesenchymal stem cells, a sort that is found mainly in bone marrow but can also be derived from fat, can become cartilage-producing cells; however, there is quite a bit of debate about what the cells really differentiate into, and claims on this company’s website seem, to me, appropriately couched.

The company seems more keen to demonstrate efficacy than mechanism, which would make the most sense for the bottom line. It looks like there’s more to learn, though.

Posted by Monya Baker on June 25, 2008
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Here’s some news on the commercial front that shows stem cell therapy moving forward. The pharmaceutical giant Pfizer has made a teeny-tiny investment in stem cells, according to an article in Forbes.

Pfizer is financing a company called EyeCyte, which hopes to use cells from the blood and bone marrow to repair damaged blood vessels in the eyes of patients suffering from diabetes and macular degeneration. Pfizer spends over $8 billion in research and development and is investing $3 million in EyeCyte in exchange for having part ownership of the company and being its sole pharmaceutical partner. If the therapyworks, though, the payoff could be huge: biologics against macular degeneration have proved big business.

Posted by Monya Baker on June 25, 2008
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According to GenomeNews, Roche is going to use cardiomyocytes derived from human embryonic stem cells to test potential heart drugs for toxicity. The cells are coming from Cellular Dynamics International, founded by James Thomson, who is famous both for being the first to derive human embryonic stem cells and contributing to the broad patents that many researchers feel restrict their use. (See our commentary)
Lots of experimental drugs fail because they cause problems in heart rhythmns; it's one reason for that famous estimate that it takes over a billion dollars and ten years to bring a drug to market. Lots of companies are hoping to cash in on using human cells for this kind of drug testing. The hope is that not only will such tests be more accurate than animal studies, they can provide a cheaper and kinder alternative. A group of pharmaceutical companies (AstraZeneca, Roche, Pfizer and others) along with the UK government has formed a consortium to do just this (see our article), and the famous Singapore company has adopted cell screening as a business model, after deciding to put its therapeutic work on hold (See our article).
The situation is muddled in the US, because these cells are being used as research tools, and how stem-cell patents will be enforced on research tools used to show drugs' safety and efficacy is still up in the air (see our article).

Posted by Monya Baker on March 07, 2008
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