Good news today! Another company has launched a potential drug for myotonic dystrophy and by extension this may also treat the childhood forms of myotonic dystrophy. The childhood forms of myotonic dystrophy are highly associated with autism spectrum disorder, so it is hopeful that this new drug will have some effects on this as well as the cognitive effects in the adult forms of the disease. For the general autism population reversing the effect in the childhood forms of myotonic dystrophy. May help narrow the mechanism of action and suggest certain treatments in the future. The childhood form of myotonic dystrophy is one of the few single gene causes of autism. Because the drug will work through an RNA mechanism, it is  unlikely this drug will have a direct effect on the general autism population.

The company is SMaRT therapeutics:

SMaRT Therapeutics is a leading biopharmaceutical company focused on the discovery and development of orally administered, proprietary small-molecule drugs that treat a broad range of diseases by targeting and modulating RNA.

We target and modulate RNA during the transcription segment of protein synthesis.  During transcription, genetic information is copied from DNA to RNA.  Mutations can occur during this process.  These RNA mutations are toxic to the human body and cause a number of human diseases, such as heart disease, cancer and stroke.  By targeting and modulating toxic RNA produced during transcription, we are able to reverse the toxic features of the RNA, enabling the RNA to function properly again and resume its role in protein synthesis.

We currently have 15 drug candidates in various stages of development.  With our patented platform technology, we have the ability to identify and develop thousands of additional drug leads that will target such RNA-mediated diseases as cancer (breast cancer, prostate cancer), neurodegenerative diseases (Parkinsons, ALS and Alzheimers), heart disease, viral infections, deafness, Cystic Fibrosis, Hepatitis C, bacterial diseases, stroke, anemia and diabetes.

Our SMaRT technology was invented and developed over the past 10 years by Dr. Matthew D. Disney of The Scripps Research Institute, one of the world’s leading biomedical research institutions.  Through our SMaRT technology, we are attacking a new generation of higher-probability RNA targets rather than focusing on the same protein targets that have been worked on for generations with diminishing returns.  Our SMaRT technology enables us to greatly expedite the drug discovery process and develop drugs that are far more effective and less expensive to produce than traditional drugs.

We are the first and only company to have developed a suite of patent-pending technologies that enables us to effectively employ a Small Molecule approach to RNA Targeting (SMaRT) for the rational design and development of an exciting new class of drugs that treat diseases by selectively targeting and modulating RNA.

Here is the information on the DRUG from their website:

SRT-149 is our drug candidate for the treatment of Myotonic Dystrophy Type 1 (DM1). SRT-149 is one of our most advanced drug candidates. It is currently in the pre-clinical testing stage and is expected to enter Phase I clinical trials within the next 12 months.

This is really all the information that has been released so far. I have not been able to find out other information. The fact that the company has some patents will enable them to raise money and it looks like this is a promising approach. The small molecule approach defined at the end here is just like most drugs that you take.

I’m excited to see if this drug will have an autism effect as well as other effects and affects on the central nervous system. Myotonic dystrophy has brain effects that are well documented in adults such as executive function, excess sleepiness, motivation, apathy as well as the exciting possibility that this may help with the general autism population but that remains to be seen. Hopefully the clinical trials will include the childhood forms as well.

SMALL MOLECULE INFO

a small molecule is a low molecular weight organic compound which is by definition not a polymer. The term small molecule, especially within the field of pharmacology, is usually restricted to a molecule that also binds with high affinity to a biopolymer such as protein, nucleic acid, or polysaccharide and in addition alters the activity or function of the biopolymer. The upper molecular weight limit for a small molecule is approximately 800 Daltons which allows for the possibility to rapidly diffuse across cell membranes so that they can reach intracellular sites of action. In addition, this molecular weight cutoff is a necessary but insufficient condition for oralbioavailability.

Small molecules can have a variety of biological functions, serving as cell signaling molecules, as tools in molecular biology, as drugs in medicine, as pesticides in farming, and in many other roles. These compounds can be natural (such as secondary metabolites) or artificial (such as antiviral drugs); they may have a beneficial effect against a disease (such as drugs) or may be detrimental (such as teratogens and carcinogens).