Spain introduces first ALS drug, offering potential for improved disease management
In recent developments, Tofersen, a drug based on antisense oligonucleotides, has shown promising results in the management of Amyotrophic Lateral Sclerosis (ALS). This treatment, marketed under the name Qalsody and developed by Biogen, is currently available in Spain, Germany, Luxembourg, Slovenia, the Netherlands, and Poland.
The Phase III study involving 108 patients demonstrated that 25 percent of them experienced improvements in functionality and strength with Tofersen. The therapy has been observed to stabilize and improve respiratory parameters in some patients, offering hope for those living with this debilitating disease.
Tofersen is designed to alter RNA stability and protein expression for the correction of genetic defects. It is indicated for ALS patients with a mutation in the SOD-1 gene, which represents approximately 2% of affected individuals in Spain.
The drug is administered once a month via a lumbar puncture. Pilar García-Lorda, the medical director of Biogen Iberia, and Monica Povedano, head of the Functional Unit of Motor Neuron Disease at the Neurology Service of the Hospital Universitari Bellvitge in Barcelona, are among those using Tofersen to treat ALS patients.
Alberto García Redondo, principal investigator of the Genetic Diagnosis and ALS Research Laboratory at the Hospital Universitario 12 de Octubre in Madrid, emphasized the importance of research for different types of ALS patients. Genetic studies should be conducted on patients, even without family cases, for obtaining more information about the disorder and early control.
The treatment has been shown to be safe and well-tolerated by patients. A case study shows a 30-year-old woman who regained some motor capabilities with the treatment, allowing her to dance again.
Spain is financing the only available treatment for ALS in Europe. Biogen, as a committed partner in innovative science for severe diseases with many unmet needs, is taking on high-risk clinical research challenges with determination to improve the lives of patients. The ultimate goal is to move towards a preclinical phase in which the patient has no symptoms.
It is important to note that ALS is characterized by a high degree of heterogeneity, adding complexity to the disease. The role of genetic studies in precision medicine and prevention for ALS is emphasized. Biogen remains dedicated to this cause, working tirelessly to advance our understanding of this disease and to develop effective treatments.
