Lacosamide is a medicinal product that is being developed for the treatment of Primary Generalised Tonic-Clonic Seizures (PGTCS). Epilepsy is a neurological disorder that is characterised by an imbalance in the excitation and inhibition of the brain and this imbalance causes a phenomenon known as a seizure. Seizures are brief increases in electrical activity within the brain and there are many types. One such type is PGTCS, which occur when the seizure happens all over the brain and affects both sides of the brain from the start, causing muscles to stiffen and convulsions to occur. They can last for a few seconds or minutes and patients can suffer multiple seizures every day.
Lacosamide is a currently licensed drug for partial onset seizures with or without secondary generalisation. It works by stabilising electrical activity in the brain. One of the ways in which it is thought to work is by preventing sodium from entering the nerve cells when they begin to fire rapid and repetitive electrical signals. An accumulation of sodium in the nerve cells is necessary for the electrical signal to build up and be passed on. Lacosomide is in clinical development to supplement the current anti-epileptic drugs (AEDs) used to treat patients with PGTCS, particularly in patients for whom the current combinations are ineffective.
Tofersen (BIIB067) is in clinical development for the treatment of amyotrophic lateral sclerosis (ALS – also known as motor neurone disease) caused by mutations in the SOD1 gene (SOD1-ALS). ALS is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. ALS is a debilitating and life-threatening disease. The gradual loss of neurons leads to a paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.