Progress on Personalized Medicine? Updates from Harvard.

@HealthMed The 8th annual Personalized Medicine Conference took place at Harvard this week - a joint venture of Harvard Medical School, Harvard Business School and Partners Healthcare, lead by Professor Raju Kucherlapati, from the HMS Department of Genetics. Worth checking the excellent archive of past programmes, presentations and podcasts.
Meantime, some of the highlights?
- An excellent narrative on the partnership between Plexxikon (Peter Hirth) and Roche Diagnostics (Suzanne Cheng) to create a companion diagnostic/therapeutic pairing for vemurafenib (Zelboraf), the first FDA approved pairing for BRAF V600E positive metastatic melanoma
- Further case studies illustrating successful drug development using genetic approaches
- Personal case studies on the impact, clinical value and ethical and clinical challenges of genomic screening: from Joe Beery, Life Technologies, on detecting unrecognised treatable serious early childhood disorders, to John Lauerman, Bloomberg News, on consequences of sequencing for asymptomatic adults - questions on penetrance and future screening for onset e.g.  of metabolic disease and cancers

Harvard Medical School: New Research Building - Avenue Pasteur.

- Clinical potential, and regulatory and reimbursement challenges to introducing molecular diagnostics into clinical care pathways
- Leadership award to Randy Scott, In Vitae, whose discussion points included the relevance of Moore's Law (technology advancing) and Metcalfe's Law (people factors: exponential increase in interaction as network expands) to developments in personalized medicine
- Business models and their governance for use of genetic information
- A North Virginia (John Vockley, Inova) pioneering series of projects aiming to assess outcomes of neonatal genomic sequencing: from insight into preterm labour to prospective longitudinal follow-up to adulthood, supported by multi-generation family member sequencing combined with clinical histories
- The US Air Force Programme on Patient-Centered Precision Care (Dr Cecili Sessions), in partnership with the Coriell Institute and Johns Hopkins University,  aims of which include understanding the impact on health-related behaviour of providing personal genetic information on remediable medical disorders and on drug responses.
- A business school case study led by Professor Richard Hamermesh, Director of the HBS HealthCare Initiative, on reactive and proactive responses for development of companion diagnostics (1).
- Pros and cons of liberal vs. restrictive approaches to IP for genetic and other molecular diagnostics
- Engaging the policy community and the public in ethical, clinical, reimbursement and adoption issues for new diagnostics and treatments aimed at personalizing medicine, including case studies from the American Medical Association and the American Assocation for Cancer Research.

Personalized Medicine Conference website 
Companion and coupled diagnostics

More news on the Geneva EACPT 2013 Congress

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Around 10 months to go until the European Association for Clinical Pharmacology & Therapeutics 11th International Congress to be held in beautiful Geneva 28-31 August 2013. The image is one of a series on the congress website showing the best of the city.

Abstract submission is now open for the next EACPT Congress,

Over 900 participants are expected to attend including health professionals, scientists, policy makers, biotechnology and pharmaceutical professionals and others with an interest in basic and clinical pharmacology, pharmacotherapy, drug discovery and development, regulatory affairs and related areas.

Key themes at the congress will range from bedside pharmacology for special patient groups to pharmacology & toxicology, and pharmacology and society. 

Specific topics will include advances in personalised diagnostics to improve the safety and effectiveness of medicines, updates on new biological approaches to ocular disease, therapeutics of cardiovascular, cancer and inflammatory disease, clinical trial design and regulation, and drug safety and toxicology.

See here for more on key themes of the Congress. 

EACPT Geneva 2013 Congress website  

The European Association for Clinical Pharmacology and Therapeutics (EACPT) has its origins in a working party in the early 1980s under the auspices of the World Health Organisation (WHO-Europe). 

The EACPT's next biennial congresses after Geneva 2013 are in Madrid 2015 and in Prague 2017. The EACPT also arranges summer schools, and other scientific and professional activities.

Progress on stroke prevention

@HealthMed 
Stroke is an increasingly common cause of death and disability worldwide. See the summary below and comments related to our new research report just published online in the major American Heart Association journal Stroke, based on results from the Warwick Carotid Artery Disease Registry. 

See media interest in the research, both in the 'tests for spotting 'minuscule “breakaway” blood clots which usually go undetected' and in this potential new treatment approach. 

Finding and reducing high risk of stroke

Narrowing of carotid arteries in the neck is a very important cause of stroke. A major reason for the increase stroke risk is that the roughened carotid artery surface can trigger formation of small clots that can then dislodge into the brain circulation. These microemboli can block brain arteries to lead to weakness, disturbed speach, loss of vision and other serious stroke syndromes.

Doppler trace: red line shows micro-embolus signal.

A team of scientists at the University Hospital Coventry and University of Warwick led by Vascular Surgeon Professor Chris Imray and Professor of Therapeutics Donald Singer have developed a Carotid Research Group to find new ways to predict and prevent high stroke risk in patients with carotid artery disease. Using ultrasound scanning they are able to detect microemboli to find out which patients are at very high risk of stroke.

The major US journal Stroke has now published a report from Imray and Singer's team with two main findings. Despite single or dual anti-platelet treatment with aspirin and clopidogrel before carotid artery surgery, patients can develop high rates of microemboli. And the team looked at different rescue treatments aimed at stopping these microemboli. The anti-platelet drug tirofiban had previously been shown to be helpful in treating patients with acute coronary artery syndromes. In Stroke, Imray and Singer's team report that with rescue tirofiban there was a large decrease in the half-life of micro-emboli (23 vs. 60 minutes) and in time for these microemboli to be resolved (68 vs. 113 minutes), compared to an alternative treatment using infusion of the clot-preventing polysaccharide dextran-40.

Professor Imray said: 'These findings show the importance of ultrasound testing for micro-emboli in carotid disease patients. These biomarkers of high stroke risk cannot be predicted just from assessing the severity of risk factors such as smoking history, cholesterol, and blood pressure.'

Professor Singer  added 'These findings show that the choice of rescue medicine is very important when carotid patients develop microemboli despite previous treatment with powerful anti-platelet drugs such as aspirin and clopidogrel. We now need to go on to further studies of anti-microemboli rescue treatments, to aim for the right balance between protection and risk for our patients.' 

Press contacts

Advances in treating acute lung syndromes

@HealthMed Although only described as recently as 1967, a range of important contributory factors have been defined for acute respiratory distress syndrome (ARDS), which is now recognized to be at the severe end of a spectrum of acute lung injury, which imposes high risk for patients and which confers a major burden on health services. Outcome of treating the syndrome has been much improved by developments in devices to treat lung and other organ failure, supported by advances in expertise in intensive care. However, other than treatments for underlying causes, there is still important unmet need with regard to effective specific pharmacological treatments.

A timely review of ARDS by Dushianthan and colleagues is the Editor’s choice article in the September issue of the Postgraduate Medical Journal.  These experts from Southampton provide an update on knowledge of risk factors, including genetic biomarkers, for development of ARDS and other acute lung injury variants, and an up-to-date commentary on general and specific treatment options.

The authors note that ‘sepsis, pneumonia, and trauma with multiple transfusions’ account for most episodes. They highlight the importance for recovery of ‘general supportive measures such as appropriate antimicrobial therapy, early enteral nutrition, prophylaxis against venous thrombo-embolism and gastrointestinal ulceration’.

They discuss encouraging experimental evidence from trials of corticosteroids, nitric oxide, prostacyclins, exogenous surfactants, ketoconazole and antioxidants, however note that these findings have not as yet being translated into benefits for patients. They note as further treatment targets of interest, new approaches to modulating inflammation, and use of mesenchymal stem cells.

See the free full article for further details.