Precision Medicine will need to get out of the pharma silo that is based on symptoms


Welcome to the digital era of biology (and to this modest blog I started in early 2005).

To cure many diseases, like cancer or cystic fibrosis, we will need to target genes (mutations, for ex.), not organs! I am convinced that the future of replacement medicine (organ transplant) is genomics (the science of the human genome). In 10 years we will be replacing (modifying) genes; not organs!


Anticipating the $100 genome era and the P4™ medicine revolution. P4 Medicine (Predictive, Personalized, Preventive, & Participatory): Catalyzing a Revolution from Reactive to Proactive Medicine.


After low-cost airlines (Ryanair, Easyjet ...) comes "low-cost" participatory medicine. Some of my readers have recently christened this long-lasting, clumsy attempt at e-writing of mine "THE LOW-COSTE INNOVATION BLOG". I am an
early adopter of scientific MOOCs. My name's Catherine Coste. I've earned myself four MIT digital diplomas: 7.00x, 7.28x1, 7.28.x2 and 7QBWx. Instructor of 7.00x: Eric Lander PhD.

Upcoming books: Airpocalypse, a medical thriller (action taking place in Beijing) 2017; Jesus CRISPR Superstar, a sci-fi -- French title: La Passion du CRISPR (2018).

I love Genomics. Would you rather donate your data, or... your vital organs?

Audio files on this blog are Windows files ; if you have a Mac, you might want to use VLC (http://www.videolan.org) to read them.

Concernant les fichiers son ou audio (audio files) sur ce blog : ce sont des fichiers Windows ; pour les lire sur Mac, il faut les ouvrir avec VLC (http://www.videolan.org).


"Big Data, Genetics and Translation"

http://genomeinformatician.blogspot.co.uk/2014/03/big-data-genetics-and-translation.html

"Drugs act on these targets. Drugs are often small molecules and sometimes proteins, like antibodies, that will change the activity of a biological target. A drug has to be able to enter the body, do what it was designed to do and not mess around doing other things. Pharmaceutical companies are really good at making these.
Clinical trials involve giving a new (or repurposed) drug to a group of consenting people. They are jaw-droppingly expensive. Unfortunately, the vast majority (90%) of drugs ultimately fail to make it through clinical trials. A large proportion of those fail because the information on which they were based, from right at the start – the target protein– was not quite right.
What’s the problem? In short, it is that a billion-dollar phase III clinical trial is a very expensive way to discover that your drug wasn't changing the right target."

How do you make it better?

"Clearly, validating those biological targets is extremely important and it can be done a lot better. What the CTTV is aiming to do is change the landscape for the initial phase of drug discovery by pooling our knowledge and resources to improve target validation.
GSK realised that this is not something that they (or any other commercial organisation) can do easily in house. Wisely, they decided that this work is best carried out pre-competitively, in the public domain. Around a year ago, members of GSK’s senior leadership came to visit the Genome Campus to explore a way forward, and the CTTV concept was born."
(...)
"At what point can one say, definitively, that this protein is a good target for a drug to act on to change the course of a disease?
To resolve this you would ideally create a specific perturbation that changes a specific molecule, verify its safety in humans and give it to people with the disease. In short, develop a drug. But the CTTV aims to get a good handle on target validation without actually making drugs, so what information are we working with, and what are we hoping to deliver?"
 
http://genomeinformatician.blogspot.co.uk/2014/03/big-data-genetics-and-translation.html


"Not to get too geeky, but I’m particularly excited about the CRISPR/Cas9 technology (like most experimentalists) because they make it possible to introduce specific mutations into cell lines. This should be particularly powerful in oncology, where systematic cancer sequencing efforts are giving rise to a number of robust targets. Cancer has been transformed by the ability to more systematically find oncogenes and tumour supressors via sequencing, leading to targeted therapies such as BRAF inhibitors. The question is: which ones in a now established cancer cell do you need to target to slow (or stop) its growth?"

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