Diseases come in two flavors
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James Sabry
Global Head, Roche Pharma Partnering

The human body is an organism that is designed to continue to live. That's the way every biological system is. It's known now that diseases come in really two flavors. They come in a disease where something from the outside infects us – and those are bacterial diseases and viral diseases. Aside from that, all the other diseases are just disordered regulation of normal behavior.

So all of cardiac disease, all of cancer, all of renal disease, all of respiratory disease, all of neurologic disease, it’s normal systems gone awry – systems that don't work properly anymore. That's why there's no specific Alzheimer's disease gene. There are genes that are involved in Alzheimer's, but they turn out to be genes that are normal genes that are just dysregulated.

This idea that disease-causing genes were normal genes that were unregulated or dysregulated in cancer is what got Varmus and Bishop the Nobel Prize in 1989. They found that oncogenes, genes that cause cancer, turned out to be normal genes that just were hyperactive. And that's cancer.

I mean, it changed the field forever because before that we thought, why would you get cancer? There's no evolutionary reason why you would get it. Some cancers are caused by viruses, but very few.

So outside of viruses, outside of infectious disease, everything is normal stuff that's gone awry, and the goal of treatment is to reestablish that normal regulation. So how do you do that?

We reestablish it by either inhibiting something with a small molecule or a protein, or enhancing something, although we don't have very many drugs that enhance things. There are a few out there. Or you can regenerate tissue that has normal regulation in it that replaces the dysregulated tissue. That would be a type of cellular therapy, for instance.

So imagine being able to take a human cell out of the patient, reprogram it so that it's a normal cell, and then put it back in the patient. Let’s replace, for instance, the neurons that are dysregulated in Alzheimer's disease.

I'm talking way into the future now, but then you could eliminate Alzheimer's disease. Basically you supply the proper regulated cellular system as a treatment. In the end, that's all our therapies are doing, is trying to reestablish normal regulation of genes that we all have in these diseases.

Read more about James Sabry and his thoughts on the future of medicine.

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