New research on the genetic component

[drew_wymore]

http://www.reuters.com/article/scienceNews/idUSL276667620080629?rpc=64

 

[butt-eze]

Thanks for the info Drew. If they are able to pinpoint the genetic marker what do you suppose they can do to stop others from developing crohn's???

 

[Creepy Lurker]

Sounds interesting.

One way to find out is to conduct genome-wide association studies, in which genetic markers from thousands of volunteers are analyzed in order to identify genetic differences between diseased and healthy individuals.

I wonder if the UKBiobank project will be able to tackle anything like this.

 

[drew_wymore]

Thanks for the info Drew. If they are able to pinpoint the genetic marker what do you suppose they can do to stop others from developing crohn's???

I don't think they can stop it from it from happening but if they can pinpoint the genes involved them they can develop gene specific treatments. I think they're doing something on this level with cancer research right now.

 

[butt-eze]

Ok, I just like to understand what this means for us and our family. Thanks Drew. Good to know.
This disease makes me want to go to medical school just so I can understand all of the lingo and processes involved in the human body. What a complicated system we have.

 

[Jeff D.]

Cool thanks Drew for posting that.

 

[drew_wymore]

I saw it while I was browsing digg and I thought it might be worth mentioning here. It is baby steps but gene therapy isn't that far off from what I've read.

 

[saidinstouch]

The science of gene therapy already exists, however in practice it is pretty much not a major target of pharmaceutical companies due to massive complications in a majority of clinical trials thus far. A number of patients have died from complications related to gene therapies where good copies of a defective gene have been inserted using a few different vectors to deliver the gene. Basically unless the disease is already deadly I doubt it will be a target of gene therapy simply because the risks are too great for something that can be managed in other ways.

As far as the genetic markers go it will be used to diagnose crohns in a similar way as the breast cancer gene, Rb gene (retinoblastoma oncogene), and other similar genetic diseases are diagnosed. Basically they can use the specific markers to easily determine someones risk for crohns using a very simple panel of genetic analysis similar to when you karyotype a fetus in the womb if there is family history for a disease like Huntingtons.

The one good thing knowing the genetic markers is that there is a ton of other information that can be gleaned from the markers. An example would be to know which biological pathway the genes lie in and determining their exact function in the pathway. You can then selectively target parts of the pathway to restore or inhibit function as dictated by the product produced by the defective gene. Often knowing a protein target makes designing a drug a lot easier since finding compounds that bind tightly to a known protein is fairly simple. Its finding safe compounds that are also active that is the hard part after you find a number of hits to begin with.

Basically as you all said a genetic study to find genes linked to crohns would be a huge asset, but only a baby step toward any new treatment. The standard development cycle for a drug takes in the vicinity of 8-10 years from start to finish. If this study isn't complete, then basically we're at least 8-10 year away from even potentially seeing a drug related to these results unfortunately. On the other hand you have to remember there are drugs that are constantly nearing approval that blow anything we currently have out of the water (or at least in most cases this is the case). At some point a new drug will come out that makes a current treatment less toxic or that just completely changes the face of our treatment as has been seen with a number of other diseases. Obviously we can hope for something big out of this, but the reality is the world of pharma just doesn't move that fast.

 

[saidinstouch]

Now that I actually read the article after my lengthy reply on some of the science behind what they are doing I see they basically said what I stated in my third paragraph. You can target the protein of a gene if you know its function and make rational decisions about how to modify the effect of the protein in order to get the desired result. A monoclonal antibody which inhibits the effect of the mutated CCR6 gene would make it an optimal target for developing a drug. It is promising, but one thing to keep in mind is that this is not gene therapy as gene therapy involves attempting to insert a good copy of a gene so that the proper protein product can be made in addition to the malfunctioning product. I know I'm being knit picky as they are using a bioinformatics genome wide approach to this problem and it gets down to some of the basics of genetics, however the results will more likely be drugs and biologics that come out of this rather than a true gene therapy. I guess my interest in drug design and my pursuit of a Ph.D in the field makes me a bit of a pain on this subject and this was definitely a good find and good read because the tools to do this kind of analysis have existed for years now and the only thing holding it back is the cost of sequencing an entire genome.

 

[drew_wymore]

@said

No I think we can appreciate the feedback and proper information on the subject. I'm by no means a doctor or a geneticist. I just hope that someone, somewhere can help us all out *someday* =)

 

[saidinstouch]

I'm not sure I'm going to work for crohns treatments or not but I'm definitely entering the pharmaceuticals industry to help people! Whether it is crohns, cancer, or any other devastating disease, I love the research and the chance to make a difference for people. I still have another 5-6 years of school sadly (5 down 5 to go ), but it's nice to be able to share the knowledge with others when possible.

 

[drew_wymore]

@said Glad to know we've got your knowledge going into a field that could potentially help us or others who have a chronic illness. 5 years will breeze by! =)

 

[BWS1982]

I shall return to this thread in 5 years or so to see you live up to your word Saidinstouch! I want my cure!

Kidding....

 

[AbstractDonut]

The article just exposed that the disease is much more complicated than originally thought. 1/5th of the markers have been discovered. Seems to say there could be multiple causes of the disease, and multiple types of the disease.

Which is something I also read about Schizophrenia the other day.