As the era of personalized medicine emerges, important ethical and legal questions arise about how personal genetic testing information is managed and used. For example, how should genetic information be used, by whom, and who should have access to it? As a result, there is a lot of current research on the ethical, legal, and social implications (ELSI) of scientific and medical advances from genomic DNA research. Many nations have allocated resources toward addressing these areas, such as the National Institutes of Health (NIH) ELSI Program.

In the May 11 issue of Science, researchers working in ELSI related to genomics advances from several countries, initiated a program to facilitate research work on these issues more globally using a web-based platform designated as ELSI 2.0. The international ELSI "collaboratory" as it is described, is designed to enable research on the ethical, legal, and social implications of genomics to become more coordinated, responsive to societal needs, and better able to apply the research knowledge it generates at a global level" with a purpose to "accelerate the translation of ELIS research findings into practice and policy."

The hope is that the web-based infrastructure with enable researchers from around the world to build off of more diverse research work, connect with other scholars, expand study and participant groups, access a broader range of research tools, and conduct more diverse studies. It is estimated the project will require about $2 million per year for the first 2-3 years to establish. Currently, they are inviting interested individuals to participate and access the open source tools that are currently available as a pilot program.

For more information on this project, you can also read the press release, as well as link to pilot ELSI 2.0 site.

Researchers at the University of Pennsylvania completed a 10-year long follow up gene therapy safety study of patients that received an engineered gene which enabled immune system T-cells to recognize the HIV virus. In three trials between 1998 and 2002, immune system T-cells from the blood of 43 HIV-positive patients were infected with an modified retrovirus carrying an engineered gene called CD3-zeta. As a result, the CD3-zeta gene, which combined parts of the natural CD3 and CD4 genes to enable it to recognize the HIV virus and turn on the T-cell's immune response, became part of the genomic DNA in the T-cells. The T-cells were then injected back into the patient's blood stream. Of the 43 patients, 41 still had detectable levels of 0.01-0.1% of modified T-cells circulating, indicating the cells proliferated as a normal part of population. Perhaps more important than the length of time the cells remained, is that none of the patients appear to have any abnormalities nor do the cells appear to behave any differently from normal T-cells.

The findings indicate this gene therapy approach with T-cell populations seems reasonably safe and could be an effective technique to treat other blood borne disorders as well as HIV infection. This is in sharp contrast to a similar gene therapy approach also run in 1998-2000 to correct a genetic disorder in children. This other study also used a retrovirus vector to deliver the gene but they placed the gene in immature blood stem cells. Four out of 11 children developed leukemia and one subsequently died. The results from this recent follow up study indicate T-cells seem to be much better behaved targets for retroviral gene therapy. This is one of several recent studies indicating somatic-cell gene therapy may be a practical and effective approach to treatment for many types of diseases.

Read a summary of this study on the Doctor's Lounge website or at MedicalXpress.com.

The White House just released a National Bioeconomy Blueprint which outlines initiatives and policies for Federal government agencies to advance and promote development of a broad range of biology-related industries from biofuels through environmental sciences. Of course, a large portion of the report the report focuses on the largest segment of the bioeconomy, biomedical research.  Two of the six outlined objectives target the major challenges with developing and launching new drugs, diagnostic tests, and medical devices.

In an effort to speed up the discovery of new drugs and disease markers, the blueprint talks about increasing support for translational research. Activities supporting this objective includes the National Center for Advancing Translational Sciences (NCATS) started the end of last year. NCATS is a publicly funded enterprise within the NIH that provides research support to identify and validate new drugs and drug targets.  This initiative addresses difficulties at the start of the drug development pipeline by expanding the potential validated drug candidates and finding additional druggable targets beyond the several hundred GPCRs, proteases, and kinases that are the focus of most current drug screening.  However, the blueprint also focuses on the regulatory challenges that limit the approval and launch of new therapeutics.

The same translational research objective that includes the above initiative also mentions the NIH/FDA collaborative Regulatory Science program.  Implemented to speed up development and adoption of advanced technology, such as nanotechnology and microdevices, in regulatory evaluations, this program aim to "more effectively evaluate product safety, efficacy and quality" of new drugs and diagnostics.  Another objective in the plan specifically targets regulations reform "to reduce barriers, increase the speed and predictability of regulatory processes, and reduce costs...." and include several activities targeted toward streamlining and improving FDA regulatory procedures.  It seems these actions might be very timely given recent criticism from some sectors that the FDA approval process is having a negative effect on drug development.

Read the National Bioeconomy Blueprint.

Researchers in the UK appear to have found a set of epigenetically regulated genes that influence aging. The study published in the on-line journal PLoS Genetics associated chemical modifications in DNA (i.e., epigenetic marks) with age and aging characteristics, such as lung function, bone mineral density, blood pressure, of 172 female identical twins between 32 and 80 years old. They found 490 genes with that had changes in epigenetic marks that correlated with the age, but just 4 that correlated with differences in age-related traits, specifically LDL cholesterol levels, lung function, and maternal longevity (mother's lifespan).

The findings indicate that, with few exceptions, such as the examples related to cholesterol and lung function found in this study, most epigenetic changes related with aging do not appear regulate health conditions associated with old age.   However, some of the age-related epigenetic marks do appear to occur very early in life so it is still unclear what lifestyle and environmental factors might influence these, or even if they are inherited.

For more details, you can also read the announcement from King's College in London.