VentureBeat

(NOTE: Apologies — especially to RSS readers — if you’ve seen this post before, but an apparent server error ate it late yesterday and I was only able to recover it this morning. Enjoy, or ignore, as seems most fit.)

Featured stories:

• Stem-cell science, money and death
• Gene tests: Out of control?
• Clinical-trial data wants to be free
• Drug, biotech industries face uncertainty
• Short takes

Clones, regrown hearts, money and death — Last week, the San Diego biotech http://www.stemagen.com/ announced that it had cloned human embryos by transplanting the nuclei of adult skin cells into oocytes, or human egg cells. The cloned embryos reportedly into blastocysts, the five-day-old clumps of roughly 100 cells from which researchers can, if all goes well, extract stem cells. (The research isn’t aimed at creating cloned babies.) Although not of immediate practical use, the ability to use embryo cloning to make genetically matched stem cells would be a big step forward for the field, since the technique could be used to produce stem-cell lines specifically for the study of particular genetic disease. The Stemagen embryos, however, didn’t actually yield any stem cells, leaving some researchers skeptical that the company had actually achieved what it claimed. The NYT has more.

Another team of researchers at the University of Minnesota recently created a beating rat heart in the laboratory, although the work didn’t grow the heart from scratch and didn’t specifically use stem cells. The team used detergents to clear living cells from a dead rat heart, leaving the outer structure and valves as a scaffold that cold be “repopulated” by injected cells from newborn rats, which eventually developed into working heart muscle that pumped blood and conducted electrical signals. Eventually it may be possible to do the same with hearts taken from human cadavers and stem cells extracted from a patient’s bone marrow, potentially yielding a newly transplantable heart.

Meanwhile, stem-cell pioneer James Thomson, a University of Wisconsin biologist, argued for a major boost in state stem-cell funding, saying that Wisconsin needs to hand out $50 million a year in order to compete with California’s $3 billion program. And in sad news, a 9-year-old girl with a fatal genetic condition called Batten disease died after being treated in a clinical trial with neural stem cells intended to correct a brain-enzyme deficiency in Batten patients. Preliminary results suggested the death was a result of her disease and not the stem-cell therapy, developed by the Palo Alto, Calif., biotech Stemcells.

Test, genes and politics — A federal advisory panel raised concerns over the proliferation of genetic tests, complaining of misleading or false marketing as well as the possibility that patients could be harmed by basing medical decisions on inaccurate tests. Unsurprisingly, many tests fall through loopholes in federal regulation, partly as the result of divided responsibilities between Medicare and the FDA. No agency even knows how many such tests are currently on the market. New tests — or the prospect of them — are popping up all the time, such as a recent finding that five specific DNA variations may help predict a man’s risk of prostate cancer, a test that, predictably enough, will be commercialized within the next few months by a startup called Proactive Genomics. The panel doesn’t appear to have addressed personal-genomics services like 23andMe or deCODEme, but chances seem good that Washington will want to weigh in sooner or later.

Making the most of clinical data — Reported results of drug trials and other medical interventions tend to be heavily skewed toward “positive” trials that appear to demonstrate the effectiveness of whatever therapy is being studied. Efforts to encourage reporting of “negative” results — that is, those that show no effect, which can also yield useful information — have faced an uphill battle. Over at Fierce Biotech, the founder of Raven Biotechnologies — a company best known around here for its attempt to swallow the corpse of VaxGen — cites her personal experience to argue that neither drug companies nor scientific journals are inclined to publish negative results. “The publishing industry and pharma reinforce each other’s biases” toward positive news, Jennie Mather writes. Meanwhile, in the NYT, Memorial Sloan-Kettering Cancer Center biostatistician Andrew Vickers bemoans the culture of secrecy that discourages even academic cancer researchers from sharing their data from clinical trial more openly. Allowing others access to the data could not only improve clinical trial designs, but potentially point the way to new diagnostic tests and treatments.

Pharma, biotech under fire — Politicians are turning up the heat on the drug industry, the WSJ notes, listing efforts to allow reimportation of cheap Canadian drugs, force Medicare to negotiate drug-price discounts, permit generic forms of biotech drugs and enact healthcare reform as among the major threats to Big Pharma and Big Biotech. In California, a recent report from the California Healthcare Institute raises similar concerns about the biotech industry, arguing that increased government oversight could crimp drug development. New drug approvals are already at a 24 year low, the In Vivo blog noted recently, although research productivity at drug companies seems to be the major culprit.

Short takes:

• Pfizer last year won approval for the first drug to treat fibromyalgia, a chronic pain condition whose existence still isn’t well accepted by doctors — including the one who first defined it in 1990, but has since changed his mind. (NYT)
• California’s plan to become the first state to require electronic tracking of prescription drugs — a measure designed to thwart drug counterfeiting — may be delayed another two years, to 2011, as the drug industry upgrades its computer systems. (SF Chronicle)
• Northstar Neuroscience brain-stimulation device fails in stroke-rehabilitation trial, highlighting the hit-or-miss efforts to treat brain and nerve disorders with neuromodulation. (NYT)

Is the bell tolling for EPO? – The news keeps going from bad to worse for the wonder drugs of biotech — the anemia treatments known as ESAs or EPO, shorthand for “erythropoiesis stimulating agents” and “erythropoietin,” respectively. Earlier today, an FDA advisory panel recommended new warnings for the drugs, which stimulate the production of oxygen-carrying red blood cells, as well as fresh clinical studies on their safety. Recent studies in kidney-dialysis patients linked higher doses of ESAs to heart problems and strokes, while studies in cancer patients treated for chemotherapy-related anemia have suggested that the treatments don’t improve patient survival, and may even cut lives short — possibly by encouraging tumor growth.

New restrictions, which the panel didn’t spell out, could put a serious crimp in ESA sales, which currently amount to billions of dollars for Amgen and Johnson & Johnson. The two companies have also been taking a public-relations battering in terms of how they promote the drugs. Yesterday, the NYT ran a front-page piece that detailed how rebates offered by Amgen and J&J encourage doctors to overuse the drugs, and today the WSJ followed with a look at whistleblower allegations that J&J boosted EPO sales by pushing higher-than-approved doses.

It’s worth remembering that while the storm is currently walloping industry giants like Amgen and J&J, plenty of smaller biotechs that have staked their hopes on getting into the anemia-treatment game could eventually be affected as well. These companies include Affymax, FibroGen and Neose. Only Affymax is public; another potential ESA competitor, GlycoFi, was acquired by Merck last year.

First embryonic stem-cell trial edges forward – By early next year, Geron plans to be injecting recent spinal-injury patients with nerve cells grown from embryonic stem cells, in hopes of regenerating damaged nerve pathways. This trial was supposed to be underway already, but last year the FDA requested more animal data for safety purposes. Geron CEO Thomas Okarma says the treatment will have been tested in 2,000 animals before it ever reaches humans. The FT’s Clive Cookson has the story.

Aggressive treatment leads to worse “quality of death” in cancer patients – File this one under things you already knew but didn’t want to think about. A study of 243 advanced cancer patients revealed that a greater number of aggressive treatments — including the use of ventilators and non-palliative chemotherapy — in the last week of life was associated with greater physical and psychological distress and a lower chance of dying in a preferred location (often home). Money quotes:

[Said study lead author Gabriel Silverman:] “These results suggest that when patients are actively dying, the use of aggressive treatments should be considered with caution and only pursued with the full understanding of patients or their surrogate decision makers.

[...]

“As a doctor, if I had a patient or family who wanted aggressive, life-sustaining care toward the end of their life, I would view it as a red flag warning of patient or caregiver distress,” Dr. [Robert] Arnold [of the University of Pittsburgh] concluded. “Often patients and their families are suffering, sad, or distressed at the end of life, and when dying occurs in medical settings they may hope that aggressive treatment will help the suffering, but often it doesn’t.”

Tau gets a little respect – For the past decade or so, Alzheimer’s researchers have concentrated their attention on beta amyloid, the protein that clumps around neurons in “tangles” visible in the autopsied brains of many — though not all — Alzheimer’s patients. Now comes evidence that a dark-horse protein called tau may also bear some responsibility for the disease. Researchers reported last week in Science that they reversed memory loss in mice by tinkering with their genes to produce lower levels of the tau protein. It’s heartening to see competing theories getting some attention in the Alzheimer’s community, which has had an unfortunate tendency to shun researchers who strayed from the majority opinion, but don’t expect beta-amyloid supporters to give much ground until they have to. That might be soon, as a new batch of drugs designed to block formation of beta-amyloid tangles should begin reporting data from human trials later this year.

New genetic heart-disease link – Another whole-genome association study has identified a new genetic variation that appears to increase heart-attack risk by 60 percent in European populations. The catch is that the variation doesn’t appear to be associated with any known gene, and instead exists in the long stretches of non-coding, or “junk,” DNA, meaning that no one has any idea why it should have any effect on heart-attack rates. The NYT has more.

Stem-cell researchers make like Willie Sutton – Near the end of this otherwise unremarkable account of a talk by James Thomson, the Wisconsin researcher who first isolated and grew human embryonic stem cells, comes this interesting nugget: Thomson will open a “satellite laboratory” on the UC Santa Barbara campus for stem-cell collaborations with UCSB researchers. Coincidentally enough, having a presence in the state might also qualify Thomson for funding by California’s $3 billion stem-cell program. Willie Sutton, you’ll recall, is the outlaw who once proclaimed that he robbed banks “because that’s where the money is.” Some sentiments, it seems, are universal.

Hypocrisy in the generic-biologics fight? – The prospect of legislation that clears a path for “generic” versions of expensive biotech drugs appears to have dimmed significantly. But biotech consultant and blogger David Williams — no fan himself of the push for “biogenerics” — notes that biotech companies and their lobbyists may be shooting themselves in the foot when they argue that biogenerics could never be “identical” to branded products now on the market. It’s worth reading his entire post — it’s not long — but the gist is that changing the manufacturing process for name-brand biotech drugs, which happens all the time, opens up the same “equivalence” issues that BIO and its allies find insurmountable where biogenerics are concerned. The main difference is that name-brand manufacturers can handle the issue with short, inexpensive “bioequivalence” trials — but they insist that biogenerics must undergo expensive, full-blown clinical testing to assure their efficacy and safety. If the biogenerics issue heats up again, don’t be surprised to see this argument make a comeback.

Surgical robots in space, stem cells in rodent eyes – These are just two interesting stories from the San Jose Mercury News I haven’t yet had a chance to mention. Last Sunday, the Merc ran this piece on efforts to automate surgery, with the ultimate goal of building robots that could operate on astronauts in space or soldiers on the battlefield. Far off and far out stuff. Similarly, this piece outlined the possibility of growing new blood vessels using an early and highly regenerative stem cell called a hemangioblast. Ultimately, these fast-growing cells could one day regrow blood vessels in the heart, eyes or limbs that were damaged by injury or disease.