May 2017 Newsletter
Breaking New Ground in Medicine
In This Issue:
Healthcare is back in the news again as the House votes on their ACA “repeal and replace” bill, and the focus is squarely on who’s covered, what’s covered, and where the money comes from to pay for it all. It is a very logistical and bureaucratic conversation. Far less media attention gets directed to the actual care being provided and the tremendous work being done by researchers and doctors around the country and the world. This month we highlight just a few of these studies and breakthroughs that are advancing the frontiers of medicine and quality of life.
Diabetes ‘Cure’ Under Study in San Antonio
At best, an average diabetic leads a life of constant disruption, one where blood sugar levels must be monitored, insulin must be injected, and where it can be difficult to take the mind off the condition. At worst, it can lead to kidney disease, blindness, heart disease and death.
Research being conducted at UT Health San Antonio has potentially crafted a technique to end the painful daily procedure of insulin injections and finger pricks for type I diabetes patients by making the body produce insulin on its own again. Two of the scientists behind the research, Ralph DeFronzo, MD and Bruno Doiron, Ph.D., believe that their gene transfer method could be an effective cure for millions of people.
Figure 1: Diabetes Growth 1980-2014
Diabetes is one of the fastest growing conditions as a percent of population and has seen significant increases in prevalence in every region of the world. This research could be a game changer for millions.
Figure 2: Projected Diabetes Growth Rates by Region through 2035
Following the current trends, worldwide diabetes cases are expected to continue growing rapidly.
The procedure involves creating sections of DNA in the lab, attaching those molecules to a virus as a delivery mechanism, and injecting the cocktail into the pancreas of a diabetic. The virus then infects the cells and delivers the genetic information necessary to spark the production of insulin again. In a recent NPR article DeFronzo was quoted as saying “Basically, what we’re going to do is we’re going to give you a runny pancreas.”
According to Doiron, the results exceeded expectations saying “We’re able to cure a mice model for more than one year without any side effects, we’re thinking that it could be permanent.”
The scientists will now seek to raise funding for tests in larger animals and predict that human trials on South Texas diabetics could start in the next 3 years.
Solving Some of Alzheimer’s Mysteries
In the quiet rolling hills of the Colombian countryside lives the largest known family with inherited Alzheimer’s disease. For 25 years, Kenneth Kosik, MD and Dr. Francisco Lopera have studied this family, uncovering the gene responsible for causing this iteration of the disease in the process, called presenilin 1 (PSEN1). The family tree stretches back centuries and hundreds of people in the family already have or will certainly start to show symptoms between the ages of 45-50. Knowing the gene mutation that causes the disease in this family means that the doctors can predict who will get the disease, and they have never seen someone with the PSEN1 gene that has not developed Alzheimer’s.
For years, researchers have associated Alzheimer’s with the buildup of a protein called amyloid, which abnormally clusters in the brains of those affected. Several treatments and medications are currently in trial to clear the brain of these plaques, some exhibiting results that stabilize the progression of cognitive impairment. But once the amyloid plaque is detected, the person is often already showing significant symptoms.
What makes the Colombian research unique is that they know who is going to get Alzheimer’s in the future, and can use that knowledge to work on a preventative treatment, rather than waiting until symptoms develop and attempting to stem the decline.
The bad news is that naturally, this research will take years as the patients undergoing preventative treatments will have to age until we know if they are working. But any progress made on one of the world’s most confounding diseases is certainly welcome as our population is living longer and the number of cases are expected to drastically increase over the next several decades.
Figure 3: Recent Statistics on Alzheimer’s Disease
Graphic credit to the Alzheimer’s Association
Figure 5: Projected Alzheimer’s Growth Rates 2015-2025
Currently more than 5 million Americans are living with Alzheimer’s. All 50 states are expected to see increases.
Grad Student Develops Cure for Multiple Sclerosis in Paralyzed Mice
With a single injection of an immunotherapeutic cocktail, graduate student Lisa Tostanoski at the University of Maryland, College Park restored movement in the hind legs and tails of paralyzed mice in her lab. Multiple Sclerosis is an autoimmune disease in which a person’s immune system T-cells mistake healthy cells in the body for a foreign threat and attacks them, degrading the fatty myelin sheaths coating nerve cells and leading to weakness and paralysis. Doctors typically have treated MS with medicine that suppresses the entire immune system, which can slow the progress of the disease but can also have catastrophic side effects.
But Tostanoski took a different approach, attempting to transform the T-cells themselves. In a Baltimore Sun article, Bruce Bebo, executive vice president for research at the National Multiple Sclerosis Society in Waltham, Mass was quoted “It’s the Holy Grail of MS. Finding the exact T-cells that are causing the disease and getting rid of them could be close to a cure, by selectively targeting the T-cells, you might be able to stop MS in its tracks.”
Her research centers on “teaching” the T-cells not to attack myelin in the lymph nodes, where T-cells are developed. She injected the nodes with myelin, and discovered the T-cells that matured in the presence of myelin wouldn’t attack it, and would in fact defend it against other bad T-cells in the body.
To support this breakthrough research, the National Multiple Sclerosis Society has awarded the team more than $600,000 in research funding.
Figure 6: Multiple Sclerosis Progression
While it is not a particularly prevalent disease, only 0.1% of the US population is affected, it is one of the most feared, because of the limited treatment options and slow but steady degradation of a previously healthy body.
These examples are just a few highlights from the many fronts on which doctors and researchers are progressing. So while the news is dominated by the bureaucratic wrangling in Washington, keep in mind that there are people working tirelessly in labs and hospitals worldwide to provide new and exciting treatments for some of our civilizations most challenging health problems.
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