Category Archives: Genetic Medicine

There is a tremendous opportunity in genetic medicine for innovation and for new players to make significant contributions, because it is still experimental, noted biologist and Nobel Laureate Dr David Baltimore said yesterday.
“Today, it is mainly the province of biotechnology companies and universities, not big pharmaceutical companies,” he observed in a keynote presentation at the Qatar International Conference on Stem Cell Science and Policy 2012.
There are new genetic tools available – though they are still experimental - to treat diseases which involve adding, subtracting or modifying genes in the cells of the body.
“However, they are powerful tools and I am confident they will be an important part of the medicine of the future,” he said.
Speaking on ‘The hematopoietic stem cell (HSC) as a target for therapy against cancer and Aids,’ Dr Baltimore explained that HSCs are one of the few cell types routinely used for bone marrow transplant.
The HSCs are easily accessible, retroviruses can be used to carry genes into these stem cells, the genes are then expressed in all of cells that derive from the HSC and can correct inherited defects and bring genes that perform therapy under a programme called engineering immunity.
“Though the human immune system is a wondrous creation of evolution yet it is not without certain limitations. One, in particular, is its poor ability to stop the growth of cancer cells– another is its hosting of HIV.
“In the case of cancer, the machinery of immunity can attack cancers but it rarely attacks with the necessary power. For HIV, the ability of the virus to use the CD4 and CCR5 proteins as receptors means that CD4 cells are the major cell type in which the virus grows.
“We have been trying to supply genes to the immune system by gene transfer methods that would improve its ability to block cancer and block infection of CD4 cells by HIV.
“For cancer, we have focused on T cell receptor genes. For HIV, we have used a small interfering ribonucleic acid (siRNA) targeted to CCR5. We have been quite successful in mice with both strategies and are now moving to humans.
“In both cases, our experiments with mice have focused on putting genes into HSCs as, once these cells are altered, they provide modified blood cells to the body for life.
“In our human cancer trials we first used peripheral T cells for modification with dramatic effect but it has been transient.
“We are now moving to stem cells. For the siRNA against CCR5, we plan to initiate trials within six months using autologous, gene-modified stem cells,” he added.
The ensuing panel discussion on ‘Opportunities and challenges for stem cell research,’ saw Prof Irving Weissman (Stanford Institute for Stem Cell Biology and Regenerative Medicine) cautioning against ‘phoney organisations engaged in stem cell therapy.’
Prof Juan Carlos Izpisua Belmonte (Salk Institute for Biological Studies, US) stated that stem cells derived from umbilical cord blood should be considered as one of the key cells for use in regenerative medicine.
The session also featured Dr Alan Trounson (California Institute of Regenerative Medicine), Prof Roger Pedersen (The Anne McLaren Laboratory for Regenerative Medicine, University of Cambridge), Dr Lawrence Corey (University of Washington) and with Dr Richard Klausner (managing partner of biotechnology venture capital firm The Column Group) as moderator.
Earlier, Ambassador Edward P Djerejian (founding director, James A Baker III Institute for Public Policy, Rice University, Houston, Texas, US) spoke about the collaboration with Qatar Foundation on stem cell research.

Read more:
‘Scope for innovation in genetic medicine’

Source: Data Sneak Peek: Groups Involved in Patient Adherence Teams

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Via Scoop.it – inPharmatics

Companies on the forward edge are putting a heavy emphasis on digital in their commercial models — but how can mobile technologies assist pharmaceutical and biotech firms in this transition?   Tablets to support the sales force – While sales forces are shrinking, they still play a vital role in educating prescribers on new medications. The pharma industry, taking the lead of companies like GSK, is starting to incent sales reps based on quality of service versus amount of sales (read more here in the WSJ). One of the tools that is helping deliver better service is the tablet. Reps with an iPad can deliver more interactive and engaging product information, capture signatures for compliance and make the most of a few quick minutes with a doctor in the time it would take a laptop to boot up.   Online and mobile drug sampling programs – Companies now have the ability to leverage PDMA-compliant mobile apps and websites that allow physicians to request free product samples that they can distribute to their patients to gauge efficacy and assist with adherence. Because the Internet never sleeps, physicians can do this no matter what shifts they are working, independent of time zone or location, 24 hours a day.   Direct-to-HCP mobile advertising – It used to be that most online and mobile advertisements for drugs were placed only in industry magazines, blogs and online communities geared toward healthcare professionals and general consumer websites. We see this changing, with emergence of mobile networks focused on healthcare such as Tomorrow Networks, which is comprised of more than 50 medical apps. Pharma companies can now buy ad placements in mobile apps made exclusively for physicians and other healthcare professionals. A physician can be looking up treatment information at the point of care and see an ad for a medication that is relevant to their patient’s ailment. That’s incredibly powerful for the physician and advantageous for the advertiser.   mDetails – Physicians want to learn about the best drugs and treatments for their patients. mDetails are multimedia mobile product presentations that provide information about drugs in a way that allows physicians to absorb detailed information at their own pace — and in their own time. Because mDetails are distributed on smartphones – it lets physicians fit pharma product education into ‘found time’ at any point during their day that’s convenient for them.   By employing a multi-channel approach and by helping healthcare professionals do their jobs better instead of just selling to them, pharmaceutical companies can reach their target audiences and develop deeper value-based relationships. The aforementioned examples are just a few of the ways that pharmaceutical companies can leverage the ever-growing mobile channel; there are many more evolving every day.
Via http://www.mhimss.org

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Via Scoop.it – inPharmatics

Companies on the forward edge are putting a heavy emphasis on digital in their commercial models — but how can mobile technologies assist pharmaceutical and biotech firms in this transition?   Tablets to support the sales force – While sales forces are shrinking, they still play a vital role in educating prescribers on new medications. The pharma industry, taking the lead of companies like GSK, is starting to incent sales reps based on quality of service versus amount of sales (read more here in the WSJ). One of the tools that is helping deliver better service is the tablet. Reps with an iPad can deliver more interactive and engaging product information, capture signatures for compliance and make the most of a few quick minutes with a doctor in the time it would take a laptop to boot up.   Online and mobile drug sampling programs – Companies now have the ability to leverage PDMA-compliant mobile apps and websites that allow physicians to request free product samples that they can distribute to their patients to gauge efficacy and assist with adherence. Because the Internet never sleeps, physicians can do this no matter what shifts they are working, independent of time zone or location, 24 hours a day.   Direct-to-HCP mobile advertising – It used to be that most online and mobile advertisements for drugs were placed only in industry magazines, blogs and online communities geared toward healthcare professionals and general consumer websites. We see this changing, with emergence of mobile networks focused on healthcare such as Tomorrow Networks, which is comprised of more than 50 medical apps. Pharma companies can now buy ad placements in mobile apps made exclusively for physicians and other healthcare professionals. A physician can be looking up treatment information at the point of care and see an ad for a medication that is relevant to their patient’s ailment. That’s incredibly powerful for the physician and advantageous for the advertiser.   mDetails – Physicians want to learn about the best drugs and treatments for their patients. mDetails are multimedia mobile product presentations that provide information about drugs in a way that allows physicians to absorb detailed information at their own pace — and in their own time. Because mDetails are distributed on smartphones – it lets physicians fit pharma product education into ‘found time’ at any point during their day that’s convenient for them.   By employing a multi-channel approach and by helping healthcare professionals do their jobs better instead of just selling to them, pharmaceutical companies can reach their target audiences and develop deeper value-based relationships. The aforementioned examples are just a few of the ways that pharmaceutical companies can leverage the ever-growing mobile channel; there are many more evolving every day.
Via http://www.mhimss.org

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Via Scoop.it – inPharmatics
UK Government launches G-Cloud store with 257 cloud computing suppliers. Offering the public sector around 1,700 cloud computing services for year-long contracts. The G-Cloud initiative, dubbed CloudStore, aims to bring a broader range of cloud computing suppliers to the government market and increase the flexibility in procurement contracts
Via http://www.govstore.net

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Thank God that Muin Khoury is blogging! I would have totally missed this!


When the CDC held a conference to evaluate Family History, they found "insufficient evidence" that this was a useful tool.


At that time I was quite dismayed about this, because I know that I have helped SAVE LIVES by integrating family history in our daily personalized medicine practice.


So I knew in my clinical heart of hearts that soon we would find "sufficient data" to prove that family history should be integrated into every encounter clinically!


Here is some of the first data and evidence that integrating family history helps clarify high risk individuals


Some of this evidence suggests that for every 5000 patients in a practice we would find an extra 200-250 patients at risk for heart disease!


From Dr Berg:


Dr Berg further states: “The complex design and analysis were meticulously planned and rigorously executed… That this study was done at all speaks to the better support for high-quality research in primary care in the United Kingdom; finding support for a study like this would be extraordinarily difficult in the United States … I look forward to further research and quality improvement evaluations documenting clinical results, and I hope that more work in this clinical domain can be done in the United States.“


I am happy that despite massive cuts in the budget Dr Khoury is all over this!


The Sherpa Says: Still much cheaper than 23andMe or Navigenics or any fly by night Direct to Consumer Genetic testing company! 


P.S. I will be hosting a panel at the Consumer Genetics Conference in June. Any ideas on who I should invite????

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