Experimental medications for metachromatic leukodystrophy registered by KFU’s Laboratory of Gene and Cell Technologies

The medications and treatment procedures have been patented.

"Metachromatic leukodystrophy is found in 1 of every 50-70 thousand newborns," says Lab Head, Director of the Center for Precision and Regenerative Medicine Albert Rizvanov. "One of the genes doesn't generate arylsulfatase, and the organism starts amassing excess sulfatide, which is a non-degradable lipid. This leads to demyelination of the central and peripheral nervous systems. As of now, there are no effective treatments for this lethal ailment. Since there is a very small number of patients, pharmaceutical companies are often not interested in developing drugs for this market. The only hope is on the research conducted by leading university clinics."

The Laboratory has conducted preclinical trials, and the results have proven the safety and efficacy of novel medications. Research of patented drugs and therapies is now underway.

"The drugs are based on adeno-associated viruses whose genome has a built-in gene the defect in which causes this syndrome. After entering a target cell, the medication launches a genetic program to synthesize the absent protein. Because of their ability to cross the blood-brain barrier our drugs can restore the enzyme in the nervous system and prevent further neurodegeneration," explains Lead Research Associate Valeriya Solovyova.

As Dr. Rizvanov notes, KFU is also working on other medications for neurodegenerative diseases, "We are researching gene and gene-cell drugs for other orphan diseases, such as Tay-Sachs disease, Sandhoff disease, lamellar ichthyosis, lateral amyotrophic sclerosis, and others. The existing treatments are symptomatic and cannot extirpate the underlying causes of these ailments. Gene therapy can be an effective way to treat hereditary diseases."

KFU is now in negotiations with potential partners to launch a pilot GMP production of gene medications and further preclinical and clinical trials.

Provided by Kazan Federal University