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So now we find out: The DNA used to make Celera Genomics’ celebrated map of the human genome was not picked at random from anonymous donors, as previously stipulated. Its source was none other than the company’s flamboyant founder and erstwhile chief executive, Craig Venter.
While some Celera executives are chagrined that their former boss bypassed elaborate privacy safeguards, the company’s lawyers should be relieved. With Venter’s DNA sitting in their database, Celera can sidestep some of the sticky social and legal questions that have hobbled other efforts to commercialize genomic information. He’s not likely to sue for having mishandled his own genetic data.
Genetic samples are the mother’s milk of efforts to develop drugs that target our bodies’ precise molecular machinery. This research relies not only on DNA data such as Venter’s–taken from a blood sample–but also from pieces of tissue, such as cancerous tumors, the diseased pancreases found in diabetes or the placentas discarded from troubled births. Facilitating that are a number of bold new commercial enterprises that collect and manage such samples and make the information available to researchers. But these pioneering efforts are threatened by new rules about to be adopted by the Department of Health and Human Services. Should Washington’s busybody bureaucrats succeed, the result will be to hobble many of the most promising efforts at developing new drugs.
DNA information by itself only has limited value. It is a static picture of the body’s complex machinery. Its true worth emerges when these DNA markers are correlated against their donors’ medical histories, especially in samples of their diseased tissues and organs. This is where Venter’s blood sample becomes important. From his blood, scientists can discern the sequence of codes contained in his strand of DNA. By stretching Venter’s sequence out and comparing it against other samples, they can search for telltale genetic markers that might be correlated with certain traits and diseases.
To understand the value of this information, take a hypothetical scenario duplicated in hundreds of biomedical labs every day. A scientist searching for new ways to treat cancer collects DNA samples from people with the disease, then looks for telltale genetic markers that correlate with their propensity to develop tumors. This is genomic data, but it only answers half the question. By taking the next step–correlating genetic markers with tumor samples, what scientists call phenotypic data–scientists can identify proteins responsible for a cancer’s growth. Finding a way to block these proteins is often pharmaceutically easy, and it is through similar efforts that we have breakthrough drugs such as Herceptin for breast cancer and even ImClone’s Erbitux.
But the process of collecting genetic material has been slowed by a patchwork of ill-conceived regulations. Biotech companies say it can take up to nine months to finalize an agreement with an academic institution for a single tissue sample, due primarily to red tape. In Europe, by comparison, uniform standards for collecting genetic information have spawned a thriving industry in the banking and sharing of this vital data. In Iceland, a company called DeCode Genetics has one of the most extensive collections of population-based genetic information anywhere in the world. It recently used that database to discover a gene linked to Alzheimer’s disease.
In the United States, a half-dozen private tissue banks have sprung up in recent years, with the sole purpose of collecting patient DNA samples in what would normally have been discarded tissue. Working with the rest of the biotech community, these companies are developing powerful safeguards on donor confidentiality and uniform consents and computer programs to make accessing the tissues a breeze for researchers. One of these companies, Ardais, is collaborating with many of the leading academic medical centers, as well as working in parallel with the National Institutes of Health and the National Cancer Institute. Their systems safeguard privacy by de-linking donors from their samples at the same time that they foster medical research. It is the closest thing we have to a national standard for the collection and sharing of this vital data.
HHS is threatening to undermine all this, through rules included in the Health Insurance Portability and Accountability Act that would impose onerous burdens on how samples are procured and used. So called “safe harbor” specifications–originally intended to address the use and disclosure of patient information for things like quality assurance audits of HMOs–have been broadened to regulate standards for research. The new rules would require, for example, that genetic samples exclude certain health-related information, such as age or diagnoses–rendering the corresponding information useless to researchers. HHS’s intervention would upend the uniform standards that have grown up in the private market and which enable the sharing of information between scientists. And it would effectively shutter private banking efforts, by redefining who can have access to the data.
Fast and full access to tissue samples is increasingly important as the drug discovery process increasingly leaves the level of the genome and zeroes in on the proteins involved in the nuts and bolts of managing bodily processes. In a diseased cell, this elaborate protein network is somehow disrupted, deranged or hyperactive. Genetic defects are often the cause. Many cancers, for example, occur when mutations activate so called oncogenes that cause uncontrolled cell growth by signaling cell proliferation.
DNA data is crucial, but researchers also need actual tissue samples to isolate the proteins that triggered the disease and then find ways to neutralize them. And getting access to those samples “is very rapidly becoming the biggest bottleneck for solving the most important medical problems,” says Lloyd Segal, CEO of Caprion Pharmaceuticals, a Canadian-based biotechnology company that specializes in uncovering unique protein switches found on the surfaces of every cell.
Segal would know, since his company relies on fresh tissue samples to carry out its innovative research. Like many such companies, Caprion collaborates on tissue sampling with an academic medical center–in this case, McGill University in Toronto. But collaborations like this one can take a year or more to negotiate, which is time and money that energetic scientists would rather spend at their research tables. That’s where companies like Ardais come in.
One of the medical community’s dirty little secrets is that good researchers always had access to the tissue they needed. Sometimes they quietly took it from their own patients; sometimes they trolled the operating rooms and asked surgical colleagues: “Do you need that?” Sometimes they looked to places like China, where money talks. Companies like Ardais render this black-market system obsolete.
But those advances will be for naught if HHS doesn’t come to grips with the impact of what it’s proposing. The department has solicited opinions on how to modify its HIPAA (Health Insurance Portability & Accountability Act) regulations to accommodate the private tissue banks, but so far hasn’t changed the draft rules. Nor are any legislative fixes floating around Capitol Hill. The conventional wisdom in biotechnology circles is that if it’s left up to Congress or to the bureaucrats of HHS, then the regulations will stand.
Those who fear a “post-human” future, Big Brother and the boogeyman, should take heart knowing that the existing research framework has enabled elaborate systems for ensuring their privacy, adopted by companies that want to stay out of court. Common rules championed by private firms and shared across research institutions have successfully addressed privacy concerns. Craig Venter’s genome now sits on thousands of computers–does anyone seriously think we now know “more than we should” about his genetic history?
Indeed, one result of Mr. Venter’s charity is to increase his body’s value, since researchers around the world will undoubtedly want to try to correlate his DNA with actual pieces of his organs. In the future, if government rules still allow scientists to access tissue samples, some of those accusing Mr. Venter of hubris could have the chance to pick apart his brain and try to understand what was really going on.
Scott Gottlieb is a resident fellow at AEI.
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