Diabetes: Silence the Messenger, Don’t Shoot It!
July 27, 2017 12:09 pm ET
Diabetes is a leading cause of death in the developed world, killing one person every six seconds around the world. In addition to $827 billion in costs last year, analysts expect global gross domestic product losses to exceed $1.7 trillion between 2011 and 2030, according to the World Health Organization. There is no curative therapy for diabetes at the moment, but new techniques like gene suppression could dramatically change the game over the coming years.
In this article, we will look at PreveCeutical Medical Inc. (CNSX: PREV) and its ground breaking research targeting a gene therapy for Type 2 diabetes and obesity through silencing of the protein, PTP-1B.
Targeting PTP-1B Proteins
Type 2 diabetes (T2D) is characterized by insulin resistance, which is a pathological condition that occurs when cells fail to respond normally to the blood sugar lowering hormone, insulin. Normally, the body produces insulin when glucose enters the bloodstream from carbohydrate consumption and its levels are elevated beyond safe, homeostatic levels, triggering cells/tissues to ‘absorb’ the glucose, so bringing it back down to normal levels. Insulin resistance means that these cells never receive this important trigger so blood sugar levels remain elevated beyond safe levels.
The protein tyrosine phosphatase 1B – or PTP-1B – is a target that specifically addresses insulin resistance. By silencing the gene encoding for the protein, patients could see improved insulin sensitization and weight loss as excessive PTP-1B levels have also been implicated in adversely affecting the processing of fat, which is an important component of T2D pathology. Unfortunately, efforts by drug companies to develop small molecule drugs to inhibit PTP-1B have been wholly unsuccessful due to their unfavorable pharmacokinetic properties and off-target (adverse) effects.
The good news is that RNA-based therapies that inhibit production of PTP-1B may provide an alternative mechanism for therapeutic intervention. In fact, researchers have already tailor-made siRNA therapeutics that target the gene, although the biggest remaining challenge is finding ways to safely and efficiently deliver siRNAs into target cells. siRNA are rapidly degraded in the circulation, much more so than small molecules, and so they need carriers that package, deliver and release their payload, which in this case includes the liver, skeletal muscle and adipose (fat) tissue.
Developing a Program
PreveCeutical Medical is commencing ground breaking research targeting a gene therapy for T2D and obesity using PTP-1B. By taking a gene-silencing approach, the company aims to selectively target the gene encoding for the protein PTP-1B only, which sidesteps many of the safety and efficacy concerns associated with small molecule drugs. Reducing PTP-1B levels is expected to reinstate signalling pathways that promote glucose uptake by our cells/tissues, while also facilitating improved processing of fat.
The company will first engineer a panel of novel, tissue-targeted bio-responsive gene carrier and release systems to address the biggest issue – delivering and releasing the payload to intended targets. In parallel the company will synthesize a library of chemically-modified siRNA’s targeting PTP-1B and conduct toxicity and efficacy studies in cell and animal models of diabetes and obesity, paving the way for further pre-clinical evaluation in the coming years.
Dr Harendra (Harry) Parekh, Ph.D at the University of Queensland’s Pharmacy Australia Centre of Excellence is directing the research program. Dr. Parekh heads the Drug/Gene Delivery Group with his team developing highly innovative and translational medicine delivery systems in conjunction with physicians whose expertise span cancer, diabetes, macular disease, infectious disease, and traditional medicine.
Diverse Product Pipeline
PreveCeutical Medical is also involved in several other businesses aside from its gene therapy efforts, which investors may want to consider.
The company’s CellB9® is a homeopathic medicine that contains blue scorpion venom, which may be beneficial in regulating and preventing cancer progression. The venom is extremely rich in a variety of proteins that abnormal cells consume and research has shown promise across a wide range of potential medical indications.
In the cannabis industry, the company is developing the first cannabidiol (CBD) nose-to-brain delivery system via its Sol-Gels, which may provide relief across a range of indications, including pain, inflammation, seizures, and mental illnesses. These Sol-Gels have the potential to deliver improved bioavailability relative to many other cannabinoid delivery systems, while enabling an extended release feature that enables once or twice weekly administration.
PreveCeutical Medical Inc. (CNSX: PREV) represents a compelling investment opportunity in the gene therapy space. With a unique target protein, the company is rapidly advancing a gene silencing siRNA therapeutic that could have tremendous implications in a multi-billion dollar market. Investors may want to take a closer look at the company given these developments and its other programs involving scorpion venom and cannabinoids.