Czech scientists are working on research that could help patients with amyotrophic lateral sclerosis (ALS) in the future.
A team led by Martin Maršala of the University of California in San Diego has developed a surgical method that, combined with gene therapy, aims to slow the onset of the disease.
The surgical technique called “subpial application” involves the administration of gene therapy capable of stifling the mutated ALS-inducing gene directly under the soft tissue of the central nervous system without disrupting the brain or spinal cord.
“Animal testing on rodents has shown that a single subpial injection of gene therapy in the cervical and lumbar spinal area just before the expected outbreak of ALS causes a long-term delay in disease symptoms. Gene therapy administered after the outbreak of the disease then prevents further aggravation of the disease,” explained Maršala, who hails from Slovakia.
Czech scientists, who collaborated in the development of the method, work for the PIGMOD Center at the Institute of Animal Physiology and Genetics of the Czech Academy of Sciences in Liběchov.
These experts also carry out the testing on miniature pigs who have spinal cords that are similar to human ones.
According to PIGMOD Center director Jan Motlík, testing should prove that with the help of this method, it is also possible to precisely deliver the therapeutic substance in much larger biomedical models.
However, he noted that miniature pigs do not have the mutation that causes ALS. Therefore, it is not possible to observe the possible stifling of the problematic gene. Prior to the tests, it was also necessary to suppress the pig’s immune response.
Amyotrophic lateral sclerosis attacks the nervous system and causes a gradual paralysis of the body. Its causes have not yet been sufficiently explained, and the treatment focuses on the mitigation of the patients’ issues.
The rate, at which the disease progresses, varies.
Among the well-known personalities who suffered from the ALS were British physicist Stephen Hawking, historian and essayist Tony Judt, and former Czech Prime Minister Stanislav Gross.