Mouse Stand-ins Leading The Way Towards Cures, Treatment

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A mouse in a lab is nothing new. But what if that mouse had a duplicate immune system of the researcher working on it? Or what if a mouse could carry the exact disease that a patient was suffering from?

The latest research in autoimmune disease and cancer treatment is doing just that: using mice as stand-ins to study exactly how an individual’s cells work, and how and why they respond to certain medicine. They are now being called mouse avatars, and they are the future of medical technology.

Dr. Megan Sykes of Columbia University has led the research initiative to use mice to replicate individuals' immune systems, in order to see how autoimmune diseases develop. Dr. Sykes named her own immune system replica mouse "Mini-Me."

"There are different types of what we call 'humanized mice,'" says Dr. Sykes. Some researchers use mice to grow human tumors, in order to test different treatments, and determine which one is the most effective on a particular cancer. But what Dr. Sykes and her team have done is create from scratch a human immune system in mice, in order to study an autoimmune disease: Type 1 Diabetes. 

"The problem is, when you have a patient who has an immunological disease," Dr. Skyes says, "you don't know what's cause and effect." Often the patient has had the disease for some time, and has undergone several treatments, so it is not clear what is driving the illness. "What we're able to do with these mice is go back to the beginning." By recreating the immune system from scratch, the researchers are able to determine what is fundamentally different about that immune system, before the disease even develops. 

This research has a lot of therapeutic possibilities. While it would be very harmful for a patient to be experimented upon with a number of different drugs and therapies, a mouse with an identical immune system can provide a guinea pig for the treatments. Those that go well can then be used on the patient themselves. But Dr. Sykes and her team are even thinking beyond that. The ultimate goal would be to find a way of using the mice to produce the human immune cells, and manipulate them in a way that they could be given back to the patient. 

Dr. Paul Haluska, of the Mayo Clinic, is also using mouse avatars — in this case, to develop therapies for ovarian cancer patients. Dr. Haluska says the avatars could change the way drugs are tested by allowing the researchers to determine which mice are responding to the drugs and which are not. "We can do a hundred avatar trial with the new drug, and find out what the characteristics are of those responders, and then when we actually go to the clinical trial, look for those characteristics molecularly with the patients."