June 29, 2011
The FDA voted 6-0 to remove its endorsement of the use of Avastin, a monoclonal antibody designed to target proteins which increase the blood supply to cancer cells, for use in advanced breast cancer. Reports in 2008 had indicated that the addition of this drug to a traditional chemotherapy regime could increase survival by an average of 5.5 months. Later clinical studies did not support this optimistic view, showing a minimal effect on survival. In December, the FDA had signaled that it would begin the removal process, citing the lack of evidence to support the use of this therapeutic for this clinical indication. The manufacturer, Genentech, then took an aggressive response in the form of a petition to the FDA for a hearing on the proposed withdrawal. In addition, patients began petitioning the FDA ro keep Avastin's approval. After a contentious hearing, the FDA panel then voted to remove the approval of Avastin for metastatic breast cancer. Of course, this has tremendous implications for patients (the cost of the drug can be around $100,000 per year) who lose viable insurance claims, as well as for the company, which can not advertise the use of this drug for this purpose. Off-label prescribing can continue, but this whole episode is not only unnerving for the actual patients, some of whom have claimed significant benefit, but as a reminder that recently-developed biotech drugs are not legacy products, with an established risk and benefit profile. The field is obviously scientifically dynamic, but here we see the regulatory instability that attends to quickly shifting science.
June 27, 2011
On the heels of the report from 23andMe, the California-based personalized medicine company, that it successfully identified new genetic markers relevant to Parkinson's disease using its direct to consumer (DTC) genetic testing model, the company has teamed up with Genentech to launch a similar efforts for Alzheimer's patients. Here, the effort is aimed at identifying protective genetic mechanisms that could block or delay Alzheimer's onset. The call for participants asks for those 75 years and older who do not suffer any cognitive impairment. A 23andMe consent form details the collection of both self-reported and genetic data from a participant, the stripping of personal identifying information, the use of the anonymized data for comparison between affected and non-affected individuals to tease out relevant genetic differences. Genentech is one of the earliest biotechnology companies, founded in 1976 and has also become an investor in 23andMe. So this effort is notable for the collaboration between a "legacy" biotech company like Genentech (largely focused on biotech drugs) and the new wave of biotech companies that capture the use of voluminous genomic information to offer personalized testing services.
June 25, 2011
23andMe is one of the leading providers of personalized genetic testing, and has announced the results of their study in which Parkinson's disease patients were recruited to provide DNA samples for what is called a genome wide association study (GWAS), in which the genome is assayed to reveal its individual profile of genetic variations. The company enrolled 3426 self-identified Parkinson's patients online, who then sent in saliva samples for DNA testing. The company compared the GWAS results to non-Parkinson's controls, and have now published their study in PLoS Genetics, two new sites of genetic variation in the human genome that are linked to Parkinson's. The research was conducted in collaboration with several Parkinson's disease advocacy organizations, including the Michael J. Fox Foundation. What notable is the online management of the disease cohort by 23andMe; this is not the simple submission of DNA samples by an underinformed consumer who then can receive personalized genetic analysis that may lack firm scientific support (note the critiques of DTC testing from the Secretary's Committee on Genetics, Health and Society last year). Rather, this is an attempt to start with the phenotype of the disease diagnosis and work backward to find deeper genetic foundations of the disease. Nonetheless, as GWAS studies proliferate, there are legal issues of confidentiality, privacy, and data ownership that ensue from this work; here is a comprehensive (ELSI - ethics, law, social) analysis prepared for the Wellcome Trust by Dr. Jane Kaye and colleagues at the University of Oxford (UK). This effort from 23andMe now overlays the DTC component as well.
June 21, 2011
A surprise from the Supreme Court, which famously heard a patent case in 2006 (briefed and argued) regarding the patentability of diagnostic methods/scientific correlations and then decided against issuing a decision (the Metabolite case) - this case became known for the spirited/dejected dissent issued by Justice Breyer, Souter and Stevens. Moving on - the Court has accepted the Prometheus v. Mayo case (heard and reheard at the Federal Circuit, amidst much turbulence from the Bilski case involving the patent eligibility of business methods. The Court has granted cert in Prometheus, which means that a significant life science patent eligibility case returns to the Court, at a time when the controversy over the potential patenting of scientific correlations has grown, as witness the challenge to the method claims in AMP v. USPTO (Myriad gene patent) case, which awaits a decision from the Federal Circuit.
June 18, 2011
The use of genetic information to inform pharmaceutical treatments is known as pharmacogenetics/pharcogenomics. The FDA has argued for its authority to approve such tests, despite the fact that it does not approve most commercially offered genetic tests in the U.S. Now the FDA has approved another genetic test, the Dual ISH test manufactured by Ventana Systems, which will determine which women could benefit from the use of the monoclonal antibody treatment, Herceptin, which will be particularly helpful to patients whose cancer cells exhibit the HER-2 protein. The FDA notes: "HER-2 overexpression and gene amplification should be determined using FDA-approved tests with an indication for the specific tumor type being tested." Other HER-2 tests are available, including one offered by Genentech, which produces Herceptin.
June 15, 2011
In the middle of a chilly climate for most domestic federal expenditures, a ray of light for the National Institutes of Health (NIH) as they attempt to protect their upcoming 2011-12 budget appropriations against legislative hostility. NIH Director Francis Colllins testified to Congress regarding A letter being circulated by Senators Richard Burr (R-NC) and Bob Casey (D-PA) is asking their colleagues to protect the appropriations for the nation's leading biomedical research enterprise (federally funded). Contrast the approximately $30 billion or so that NIH receives with, for example, the resources of the privately funded Howard Hughes Medical Institute with a 2010 endowment of about $14.8 billion. The letter eventually was signed by 41 senators,enough to signal that NIH has a significant fan base in the Senate (or at least publicly-identified fans), which could help protect its allotment in upcoming budget negotiations.
June 7, 2011
A long-running dispute between Stanford University and Roche Diagnostics has centered on competing assignment documents that were signed by a Stanford research scientist, Mark Holodniy, who was employed at Stanford but who had gone over to Cetus Corp. to learn about then-developing techniques of polymerase chain reaction (PCR) DNA testing, a revolutionary advancement which allowed minute quantities of DNA to be tested and quantified. At stake in the patent litigation are the revenues derived from the development of a PCR-based blood test that measures HIV levels. Although Holodniy's employer-originted contract stipulated that inventions generated at the university would be assigned to Stanford ("agreed to assign), his subsequently executed contract with Cetus Corp. (predecessor to Roche) noted that his work at that site was immediately assigned ("will assign") to the corporation. The Supreme Court has decided in favor of Roche, disputing Stanford's assertion that the existing Bayh-Dole Act (allowing universities to claim patent rights over federally-funded inventions) automatically vested the patent rights in Stanford. This was clearly a case of somewhat conflicting assignments signed by an inventor who was legitimately dividing his work efforts between Stanford and Roche. The precision of the Roche assignment clearly favored the predominance of that document, as the Court spelled out. What's clear is that Bayh-Dole is certainly permissive of the scheme by which universities are allowed to exploit the federally funded-work through subsequent patenting; however, it is not a default mechanism in the absence of underlying documentation that explicitly transfers the rights of an inventor to the institution. What's also clear is that a the business relations governing the situation of a university-funded researcher who formally performs some research at a competing entity (whether academic or business) are no doubt less ambiguous and casually reviewed, as university tech transfer offices more carefully supervise the work efforts of employees and proactively attempt to avoid any casual or unsupervised contracting by employees which could defeat the expectations of the Bayh-Dole framework.