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FDA, Pharma Turn to PGx to Tackle Adverse Events


By studying the genetic underpinnings of adverse drug reactions, the US Food and Drug Administration is hoping to avoid recalling drugs based on their negative impact on a few and depriving the greater population of beneficial treatment.

In late September, the FDA and a collection of industry and academic partners kicked off the Serious Adverse Events Consortium, a collaborative effort that hopes to identify genetic markers that might be used to identify patients at high risk for adverse reactions to particular drugs.

If tests based on these genetic markers were available, “[We] can keep the benefit of a drug and keep it available for people who are going to benefit from it, if you could identify those people who are at risk for having a very serious side effect, and make sure they don’t ever get the drug,” according to FDA Deputy Commissioner Janet Woodcock.

The SAEC is a nonprofit entity comprised of large pharmaceutical companies, research organizations, and regulatory bodies. The FDA will guide the consortium with scientific and strategic input.

The formation of the consortium comes as several large pharma companies have had to pull drugs already on the market or kill investigational treatments very late in clinical development. Two SAEC partners, Pfizer and GlaxoSmithKline, have recently had such setbacks.

Pfizer last year had to suspend a large, phase III trial for the investigational cardiovascular therapy torcetrapib, which was being studied in combination with the popular statin atorvastatin (Lipitor), due to an increased rate of mortality in patients receiving the torcetrapib/atorvastatin combination.

In another instance, sales for GSK’s type 2 diabetes drug Avandia took a nosedive since May, when it was associated with a potentially significant increase in the risk of heart attack and heart-related deaths. By the end of June, GSK’s quarterly US sales for Avandia had fallen 31 percent, to $461 million, from the previous quarter.

— Turna Ray

Pharma, Gov’t to Test Stem Cells in ADME-Tox

A group of US and UK drug makers and a government agency last week launched an initiative to explore whether stem cells can serve ADME/tox functions in compound libraries, and provide guidance and funding for stem cell research in the UK.

The initiative, dubbed Stem Cells for Safer Medicines (SC4SM), is organized as an independent, nonprofit company, and has issued a call for proposals from potential research partners interested in developing techniques to use stem cells for ADME/tox testing.
SC4SM is the first public-private partnership on human embryonic stem cells and marks the pharmaceutical industry’s first foray into the field of hESC research. AstraZeneca, GlaxoSmithKline, and Roche have each committed an initial £100,000 toward SC4SM, while the UK Department of Health, which initiated the project, will provide £750,000.

The consortium is led by the Association for the British Pharmaceutical Industry, and is also supported by the UK Department for Innovation, Universities, and Skills; the Scottish government; the Medical Research Council; and the Biotechnology and Biological Sciences Research Council.

Philip Wright, CEO of SC4SM and director of science and technology at the ABPI, says that although Glaxo, AstraZeneca, and Roche are the three founding pharmaceutical members of the consortium, the group is “in discussions with other pharmaceutical companies, and will be looking to have others join SC4SM during the first year.”

Wright says opportunities exist for companies to participate on a project-by-project basis, although they will only have access to the projects that they are directly involved in.

The launch of SC4SM is the first step in a five-year, £10 million initiative to develop stem cell technology for use in predictive toxicology assessment testing. The initiative is divided into a one-year pilot phase running through late 2008 or early 2009, followed by a four-year main phase.

— Charlotte LoBuono

Short reads

Multiplexed biomarker testing lab Rules-Based Medicine raised $25 million in a series A round of private equity financing led by Equity Group Investments. Other investors were Cross Creek Capital and Stephens Capital Partners. The money came just as RBM was ordered to pay Luminex $12.5 million to settle a lawsuit.

HiFi DNA Tech filed a lawsuit against the US Food and Drug Administration for allegedly failing to respond in time to the company’s petition to reclassify DNA tests for human papillomavirus from class III to class II devices.

Genomic Health announced that its Oncotype DX showed positive results in predicting the likelihood of recurrent breast cancer in postmenopausal women. The test is currently used to predict recurrence and the likelihood of benefit from chemotherapy in women with estrogen-receptor positive, node-negative breast cancer.

The Critical Path Institute will use a $2.1 million Arizona state grant to work with the US Food and Drug Administration and the National Cancer Institute to standardize how companion diagnostics and therapies for cancer are evaluated. Ventana Medical Systems will test the resulting processes.

A new report by the National Research Council calls for a national human toxicogenomics initiative to better understand toxicity.

Eli Lilly signed a three-year collaboration with GE Healthcare
and GE Global Research to develop in vitro diagnostics to predict the response of patients to targeted cancer therapies. The deal covers targeted cancer therapeutics in progress at Lilly and GE’s multiplexed tissue-based assays and image analysis tools.

Dutch diagnostics firm Agendia, which makes breast cancer test MammaPrint, joined the Personalized Medicine Coalition, a consortium focused on public policy and regulatory issues related to the adoption of personalized medicine.

TorreyPines Therapeutics and Eisai extended an agreement started in 2005 to identify and validate genes that may be used as targets for Alzheimer’s drug compounds. The partnership uses whole-genome, family-based association screening.

Abbott and Epigenomics formed a non-exclusive license and collaboration agreement through which they plan to develop an in vitro colorectal cancer diagnostic based on a methylation biomarker from Epigenomics.

Taiwan’s Department of Health said that it has updated the label for the anticonvulsant drug carbamazapine to warn patients of a genetic link to potentially serious side effects, and said that it plans to test patients for adverse reactions to the drug.


US Patent 7,282,576. Coactivators in the diagnosis and treatment of breast cancer. Inventors: Anna Riegel, Ronald Reiter, and Anton Wellstein. Assignee: Georgetown University. Issued: October 16, 2007.

The patent deals with “the AIB1 protein as a coactivator that potentiates the transcriptional activity of nuclear hormone receptors. ... Over-expression of .DELTA.3-AIB1 plays an important role in sensitizing breast tumor cells to hormone or growth factor stimulation.”

US Patent 7,282,361. Systems and methods for transgenic and targeted mutagenesis screening. Inventor: Timothy Hodge. Assignee: Transnetyx. Issued: October 16, 2007.

The patent covers a technique and tools “to conduct transgenic and targeted mutagenesis screening of genomic DNA. It also provides a system for screening DNA for a designated genetic sequence. The system includes a computer [which] receives instructions concerning the designated genetic sequence and other screening parameter … and an automatic screening device that analyzes samples of genomic DNA for the designated sequence.”

US Patent 7,266,457. High throughput functional genomics. Inventor: James Hickman. Assignee: Hesperos. Issued: September 4, 2007.
According to the abstract, “this invention focuses on the marriage of solid-state electronics and neuronal function to create a new high-throughput electrophysiological assay to determine a compound’s acute and chronic effect on cellular function. ... This innovative technology can be applied to neurotoxicity, and to screening compounds from combinatorial chemistry, gene function analysis, and basic neuroscience applications.”

US Patent 7,272,508. Small molecule mimetics of erythropoietin. Inventors: Ian Wilson, Oded Livnah, Enrico Stura, Dana Johnson, and Linda Jolliffe. Assignee: The Scripps Research Institute. Issued: September 18, 2007.
This patent covers “computer-assisted methods for identifying molecules which will bind to the EPO receptor and act as an erythropoietin (EPO) mimetic. Preferred EPO mimetics identified using the method of the invention act as agonists of the EPO receptor in one or more in vitro or in vivo biological assays of EPO activity.”

Data points

$300 million

Approximate purchase price PerkinElmer plans to pay for Viacell, a Cambridge, Mass.-based specialty stem cell company that will help PerkinElmer expand its neonatal and prenatal screening business.

$5 million

The Montreal Heart Institute has been awarded a CAN$5 million grant through Genome Quebec to fund a research project based on its academic and private partnerships in pharmacogenomics.

The Scan

Enzyme Involved in Lipid Metabolism Linked to Mutational Signatures

In Nature Genetics, a Wellcome Sanger Institute-led team found that APOBEC1 may contribute to the development of the SBS2 and SBS13 mutational signatures in the small intestine.

Family Genetic Risk Score Linked to Diagnostic Trajectory in Psychiatric Disorders

Researchers in JAMA Psychiatry find ties between high or low family genetic risk scores and diagnostic stability or change in four major psychiatric disorders over time.

Study Questions Existence of Fetal Microbiome

A study appearing in Nature this week suggests that the reported fetal microbiome might be the result of sample contamination.

Fruit Fly Study Explores Gut Microbiome Effects on Circadian Rhythm

With gut microbiome and gene expression experiments, researchers in PNAS see signs that the microbiome contributes to circadian rhythm synchronicity and stability in fruit flies.