Personalized Medicine Watch
By John Russell
October 28, 2008 | Diabetes is something of a modern epidemic and there is an army of academic and biopharmaceutical researchers seeking better treatment and curative strategies. Personalized medicine approaches represent a growing portion of those efforts and in the past couple of years, the University of Chicago Medical Center has had success treating genetic-caused type 1 (T1) diabetes with PM approaches.
In fact, UCMC was able to wean more than 30 children off of insulin. The children all presented symptoms of type 1 diabetes as newborns and they also shared a “genetic mutation that, by altering the function of a potassium channel involved in glucose sensing, mimics insulin-dependent diabetes. The damage caused by the mutation can be repaired by giving the children a common, inexpensive, oral drug used to treat type-2 diabetes.”
Part of what interesting here—beyond the obvious benefits of the treatment—is the low cost of treatment and the further demonstration that what we see as “diabetes” may constitute a wider variety of illnesses, perhaps many based on mutations. The developer of this therapy is reportedly an English physician named Andrew Hattersley, of Peninsula University, who focused on these genetically triggered versions of diabetes in the early 2000s.
Until recently this approach gained little attention in the U.S. UCMC’s success produced more publicity and referrals. “An interesting sidelight is that by genetic testing of children with very early onset diabetes, the team has found several other diabetes-causing mutations, although so far no immediately treatable ones,” according to the report. “Some have tiny alterations of their insulin genes. Other forms of monogenic diabetes that can masquerade as either type 1 or type 2, some of them discovered at the University of Chicago, also remain under-diagnosed.”
It seems likely we will uncover more genomic mutations whose symptoms “mimic” other diseases which we thought were well-characterized. It also seems likely that a fair number will be treatable with simple, inexpensive regimens. Write to John_Russell@bio-itworld.com with your thoughts on the opportunities and challenges associated with personalized medicine.
Personalized Medicine for Monogenic Diabetes
The Gene Genie
DNA for All to See
Medical Genetics: Are We Ready to Get Personal with Diagnosis and Treatments?