Boron-based formulations for the treatment of muscular dystrophies and muscle injuries

Summary

A Spanish biomedical research network center has developed a new therapeutic strategy for the treatment of pathophysiological conditions which affect skeletal muscle, and is looking to establish license, research cooperation, or joint venture agreements.

Description

This research center gathers some of the main Spanish research groups in biomedicine, located in more than 100 institutions such as universities, hospitals and technological centers distributed around the country.
Boron is an essential metalloid that plays a key role in the metabolisms of plants and animals. It has been seen that boron is involved in bone mineralization. Among other uses it has been employed in synthesis chemistry and has been recently exploited in medical-chemistry. At present, little is known about homeostasis and boron function in animal cells; however, in the latter, boron has been reported to be involved in myogenic differentiation in mice and muscular dystrophy.
There are different types of dystrophies. For instance, Duchenne muscular dystrophy (DMD) is a progressive and lethal disease, caused by mutations associated with the X chromosome of the gene that encodes dystrophin. Lack of dystrophin leads to weakness, degeneration and consequent fibrosis in the skeletal and cardiac muscles. Currently, there is no cure for DMD patients. Some of pre-clinical and clinical approaches in development, include exon omission, genetic editing through viral vectors, and stem cell transplants.
All of these approaches will take some time to become available, and even when approved they may only be suitable for selected patients and/or have the ability to improve symptoms but not completely relieve them. Therefore, therapies are needed to, either independently or in combination with other treatments, slow or ease the disease progression.
A recent strategy is based on the soluble combination of some basic boron salts such as borax, which has experimental results in flies and a potential use in the treatment of muscular dystrophies. While the aforementioned combination allow the use of lower dosages of borax when combined with fibronectin, these still need to be further reduced for the use of boron compounds in pharmaceutical compositions for the treatment of muscular diseases or other medical treatments.
The invention of this profile refers to new combinations of boron compounds and adjuvants to induce myotube formation and suppress cell mortality in a mammal in need.
This innovative technology helps to minimize the physiological effect of defective mRNA to provide a quality of life for patients with rare disease such as muscular dysthropies, as well as minimizing and reducing the dosage of the suitable boron compounds to non-toxic dosages, to ensure effectivity while reducing or eliminating side effects.
The research center is looking for Biotech or Pharma companies to cooperate under license, research cooperation, or joint venture agreements.

Advantages & innovations

This innovative technology comprises new combinations of boron compound, adjuvants and compositions thereof, which are suitable to be used in a methodology to induce myotube formation and suppress cell mortality in a mammal in need for it. Furthermore, it is related to compositions comprising boron compounds, adjuvants and support platforms, which enhance the effect of synergistic activation of the boron cell membrane transporter (NaBC1) and adhesion receptors in cells. The invention refers to the aforementioned combinations and compositions for the treatment of sport injuries that affect skeletal muscle, such as acute strain and tear injuries of muscles, a relevant factor in lost time and performance efficiency in professional athletes. The invention also refers to the treatment of pathophysiological conditions that affect skeletal muscle, in particular muscular dystrophies such as Myotonic Dystrophy or Duchenne Dystrophy. The next step towards clinical studies is to assess optimal formulations that can provide its therapeutic effect in humans and minimize toxicity. In their research, the center has shown that toxicity is negligible, due to the ultra-low doses boron compounds at which the invention works.

Stage of development

Prototype available for demonstration

Contact/ source: Enterprise Europe Network (europa.eu)