Hope to fill the blanks! Anti-epileptic "old medicine" to establish "new work"

we all know that new drug research and development investment is large, long cycle, high risk, and "old drug" new research and development cheap, short cycle, high success rate, with "new drugs" incomparable advantages. Valproic acid is widely used to treat epilepsy and bipolar disorder drugs, but also to prevent migraines, its clinical application has been nearly 60 years, is still "active" in the clinical front line.
February 11th, a study published in the national scientific review, one of the world's top journals, found that valproic acid, an anti-epileptic "old drug", has new "functions".
The results come from Xiamen University's National Key Laboratory of Cell Stress, Xiamen University School of Neuroscience Research Institute Professor Wang Xin team, researchers through a series of studies, confirmed that the clinical anti-epileptic drug valproic acid, can greatly improve a new type of resymnant disorder and small brain atrophy disease, the discovery has strong clinical conversion application value, is expected to fill the gap in the field of genetic co-eclampsia treatment.disorder is a kind of neurological symptoms, which are clinically manifested as gait instability and loss of balance. Among them, excessive drinking can trigger the symptoms of the disorder, belonging to the access to the disorder, such conditions generally do not require treatment can be alleviated or cured.
" another category belongs to the genetic disorder, caused by congenital gene mutations, clinical manifestations are different, the rate of death and disability is high, accounting for about 10% to 15% of the neurological genetic diseases, there is no special drug, the treatment of this disease is still a world problem. Wang Xin said.
with the widespread use of gene sequencing, a new genetic disorder, SCAR20, was reported in the journal Nature-Genetics in 2015, and Wang Xin is the co-author of the paper. "SCAR20" case families are mainly close relatives married, patients show early oncercular brain atrophy, severe motor imbalance and intellectual disabilities, life can not take care of themselves, life expectancy is greatly shortened.
lack of special-effects drugs, clinicians are helpless, which creates a huge financial and spiritual burden on patients' families. So what is the cause of "SCAR20"? Is there a drug to relieve the patient's pain? With these scientific questions in hand, Wang Xin returned to China in 2015 to set up a laboratory, immediately began the "SCAR20" research.
Because patient samples were difficult to obtain, the team first constructed a "SCAR20" model mice, and behavioral analysis confirmed the presence of progressive co-effect disorders and small brain atrophy in mice, which is very similar to clinical patients and is suitable for follow-up mechanisms and drug evaluation studies.cells are arranged according to a certain pattern to form tissues, and tissue atrophy is usually caused by a decrease in the size or number of the cells that make up. Through an autopsy, the researchers confirmed that a large number of deaths of Pokenno's cells caused the patient's brain to atrophy and co-dysfunction.
is one of the largest neurons in the brain of vertebrates. Precision structure foreshadows the power of function, Pukenno cells are important cell types that control the body's motion coordination, and impaired function usually leads to co-ordination disorders.
, how did Poken wild cells die? "There are no more factors that cause cell death than internal and external causes, and through further study, we found significant deficiencies in mitochondrial function in Puken wild cells." Wang Xin said.
As the "energy factory" of cells, mitochondrials are essential to maintain cell survival and, if damaged, can be fatal to cells, the most complex neurons in the brain, with an extremely high demand for energy. Like a leafy tree, once the supply of nutrients is lost, all that awaits it is withering and dying.
researchers further found that the original Puken wild cells responsible for transporting mitochondrial "orbit" problems, resulting in mitochondrials can not reach the designated location to function, resulting in cell energy supply disorders, and ultimately caused the death of Puken wild cells.
to find a drug for "SCAR20," the researchers screened several potentially therapeutic drugs in mouse models. After several attempts, they found that valproic acid had a significant effect on "SCAR20" mice. After giving the model mice a month of treatment with valproic acid, the common disorder phenotype in the mice improved significantly (74%), the death of Puken wild cells was significantly curbed (contained 52%), nerve inflammation decreased significantly, and mitochondrial transport and function were restored.
is encouraged by the good therapeutic effect of valproate on the 'SCAR20' model mice, and we are eager to bring it to clinical use as soon as possible to realize the mission of 'new use of the old drug' of valproate, so that 'SCAR20' patients can benefit at an early time. Wang Xin revealed that the next step, the team will explore whether valproic acid is suitable for other types of hereditary resonance disorders (especially characterized by the death of Puken wild cells) to determine whether valproic acid has greater potential in treating other types of resonant disorders.