Monash Health has been providing a new treatment for a form of Motor Neuron Disease (MND), which has the potential to reduce nerve damage and slow disease progression.
The treatment received accelerated approval in the U.S in 2023, where it was the first approved treatment for a genetic cause of MND. The new medication is called Tofersen, but is not yet approved in Australia.
As reported by Star News, Monash Health is the first hospital in Victoria to offer compassionate access to the medication as part of an Early Access Program.
Around 2,000 people in Australia are living with MND. There is currently no cure for the disease and 2 Australians die every day from MND.
Dr Yenni Lie, a neurologist who leads this program at Monash Health describes MND as “a relentlessly progressive and fatal degenerative disease that causes the loss of nerve cells in the brain and spinal cord that are responsible for controlling voluntary muscle movement,”
“People with MND experience progressive muscle weakness and wasting, causing them to lose independence as they steadily lose the ability to move, speak, eat, and eventually breathe.”
“Survival is only around two to five years from the onset of symptoms.”
Current treatments aim to control symptoms, prevent complications, and improve quality of life.
Around 2 per cent of all MND patients have inherited a SOD1 gene mutation, which causes the body to create a toxic form of the SOD1 protein, which leads to nerve cell death. Tofersen, the new medication from Biogen, is designed to reduce the toxic SOD1 protein in the body. It is given as an injection into the spinal fluid through lumbar puncture.
Clinical trials of Tofersen showed reduced neurofilament, a marker of nerve cell damage, as well as small trends of reduced decline in clinical function, muscle strength, breathing function, and quality of life. Ongoing research aims to verify Tofersen’s clinical benefit.
Dr Lie says “Tofersen brings a critical treatment option for families facing SOD1 MND, with a potential benefit of slowing disease progression”.
Those suffering with SOD1 MND can enrol in this program, at any stage of their illness.