BelSU Scientists Contribute to Development of New Drug to Enhance Physical Performance in Muscular Dystrophies

Muscular dystrophy encompasses a range of hereditary disorders characterized by progressive muscle weakness and loss of muscle mass. Although these diseases are considered orphan conditions due to their rarity, they can lead to irreversible damage to muscle cells. Patients suffering from dysferlinopathies often find daily tasks increasingly challenging, and in severe cases, they may struggle to control their bodily movements.

The drug was developed through a collaboration between Genotarget LLC and Artgen-Biotech PJSC. Together with these developers, BelSU specialists are making significant strides toward creating an effective treatment to enhance physical performance in individuals with muscular dystrophies linked to dysferlin gene dysfunction.

Under the leadership of Professor Mikhail Pokrovsky, Director of the Research Institute of Pharmacology of Living Systems at BelSU, a dedicated team of researchers conducted comprehensive studies on the drug, which utilizes an adeno-associated viral vector for the treatment of dysferlinopathies. This research team included postgraduate students from the Department of Pharmacology and Clinical Pharmacology: Elena Kuzubova, Vladimir Pokrovsky, Nikita Zhunusov, Anastasia Krayushkina, and Alexandra Radchenko.

"Our scientific group is investigating a drug based on an adeno-associated virus that carries codon-optimized complementary DNA of the dysferlin gene, regulated by a muscle tissue-specific linker," explained Professor Mikhail Korokin, head of the study and a faculty member at the BelSU Medical Institute. "This approach enhances the expression of dysferlin while mitigating the drawbacks associated with existing analogs and their contraindications."

During effectiveness trials conducted at the BelSU Research Institute of Pharmacology of Living Systems, researchers observed significant improvements in physical performance among dysferlin-deficient mice. The innovative methods developed for enhancing physical performance in animal models are protected by three patents from the Russian Federation.

One of the methods proposed by the researchers involves a single intravenous administration of the genetically engineered drug. The impact was measured using a "grip strength" test, revealing that thirty days post-injection, the peak traction force in dysferlin-deficient mice increased by 27.3% compared to untreated animals.

Another method involved a single intramuscular injection of the drug at a dosage of 1 x 10^12 viral units containing the DYSF gene. In this case, physical performance was assessed through forced swimming tests with added weight. Remarkably, three months after receiving the injection, treated laboratory mice swam 92.3% longer than their untreated counterparts.

Additionally, BelSU scientists confirmed the drug’s effectiveness using the “animal holding on a slippery vertical rod” test. In this experiment, dysferlin-deficient mice received the drug twice, one month apart. Three months following the initial injection, treated mice were able to hold onto the slippery rod for 23% longer than those that did not receive the drug.