It's not often that I read study abstracts and am wowed, but in a moment you'll understand why a new study out of the Scripps Research Institute this past week is so exciting.
But before we dive right in, how about a little background on one of the world's most dangerous yet underserved disease indications: diabetes.
The silent killer
Diabetes comes in two forms. Type 1 diabetes is a genetic disease caused by a person's immune system attacking and destroying the pancreatic beta-cells responsible for creating insulin in the body. Type 2 diabetes, a disease that can occur at any age whereby not enough insulin is produced by the body, or the body doesn't properly utilize insulin, is largely preventable and/or manageable through proper diet and exercise.
Within the United States, according to the Centers for Disease Control and Prevention, some 29.1 million people have diabetes, with 8 million of those cases currently undiagnosed, earning it the reputation of being a silent killer. A majority of these cases are the type 2 variety, with somewhere between 5% and 10% being the genetic type 1 diabetes variety. Regardless of type, diabetes can lead to a number of co-morbidities over a patient's lifetime, potentially resulting in death.
Per the American Diabetes Association, the cost to treat diagnosed diabetes in 2012 was practically a quarter of a trillion dollars, with $176 billion being spent on direct medical care associated with the disease, and $69 billion representing the economic cost of lost productivity associated with the disease. Because this is a disease that is so widely skewed toward type 2 patients, those who have no say in whether or not they get this disease (i.e., type 1 diabetics) are left wondering when they're going to receive the next major therapeutic advancement.
This week the Scripps Research Institute (TSRI) unveiled what could one day represent not just a symptom-abating therapy, but a genuine cure for type 1 diabetes.
Forget the symptoms! Is this a cure for type 1 diabetes?
According to a study published in the March issue of Endocrinology, TSRI researchers tested an experimental compound known as SR1001 in non-obese but diabetic animal models and observed that it completely stopped type 1 diabetes from developing.
Specifically, researchers noted that SR1001 completely eliminated the incidence of diabetes, it maintained insulin levels, and it reduced inflammation associated with insulin-producing cells, which is a common occurrence in type 1 diabetic patients. By targeting a specific pair of receptors with SR1001, researchers were able to suppress a targeted immune response that creates Th17 cells, which have been linked to a handful of autoimmune diseases, including type 1 diabetes.
Furthermore, researchers also note that SR1001 led to an increased expression of the Foxp3 protein in T-cells. T-cells play a role in the development of T-regulatory cells, which act as the balancer of the immune system. In effect, researchers appear to, in animal models, have discovered a way to reduce the immune system's incorrect response to destroy insulin-producing cells, and to protect those cells over the long term from destruction by the immune system.
Researchers believe this research could lead to drugs targeted at Th17 cells, since it would appear Th17 plays a role in the development of type 1 diabetes.
Major advances on the way for type 1 diabetics
The results from TSRI's study are incredible and certainly hold encouraging promise for the future of type 1 diabetes treatments. Unfortunately, considering the long-winded studies that developing drugs have to be shuffled through prior to approval by the Food and Drug Administration, it could be years before a compound like SR1001, or any other Th17-targeting drug for that matter, sees the light of day in human clinical trials.
But on the flipside, there is one brand new therapy on the market poised to make a big difference in the lives of type 1 diabetes patients, as well as another therapy in the works which also aims to cure diabetes, though through a different pathway than what was described in TSRI's study.
The relatively new drug gracing pharmacy shelves that could make a difference for both type 1 and type 2 diabetics is MannKind's (NASDAQ:56400P706) inhaled therapeutic Afrezza. There are a number of benefits that diabetics could really appreciate with Afrezza, including its rapidly acting formulation and the fact that it leaves the body quicker than traditional insulin, thus implying that it could reduce instances of hypoglycemia, or low blood sugar, which can be just as dangerous as high blood sugar. Also, the convenience of an inhaled and painless product cannot be overlooked.
As with any new therapy, MannKind's biggest challenge will be in securing insurance coverage and in informing physicians and the public of this new option. To that end it's enlisted the licensing assistance of Sanofi (NYSE:SNY). The next quarterly report will be Afrezza's first full quarter on pharmacy shelves, and it should give us an indication of how well the drug has been accepted by the medical community. It certainly has a shot at being successful, but it's possible MannKind's stock price already reflects that implied success.
The other big name to watch here is privately held Perle Biosciences, which is developing PRL001, a drug designed to regenerate the pancreatic beta cells that produce insulin and protect those cells from a patient's immune system. With an ultimate clinical goal of insulin independence, Perle's drug would be nothing short of an effective cure if successful.
The drug itself is a combination of two already-approved oral therapeutic agents, meaning no injections for type 1 diabetics. A late-stage clinical study is expected to begin this year and could yield results in the not-so-distant future. As I've previously noted, Perle is a privately held company, so no investments can be made here as of yet. However, positive results in its phase 3 study could prompt Perle to rethink going public, since a drug launch can be expensive. It's certainly a study worth closely monitoring.
Working toward a cure
Although type 2 diabetes patients far outnumber type 1 diabetics, I'm encouraged to see that progress, no matter how small, is being made in the fight against type 1 diabetes. Without a means to prevent diabetes from the get-go, having a potential cure coming down the pipeline in the next few years is something millions of Americans, including myself, would love to see. I've been hoping that a diabetes cure would be discovered during my lifetime, and studies such as TSRI's indicate we may not be too far off.
Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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