Dr Suzan Hammond from the MDUK Oxford Neuromuscular Centre is developing molecular patches for spinal muscular atrophy (SMA) that can cross from the blood into the central nervous system . This research could lead to potential treatments that are safer and less invasive than Spinraza .

What are the aims of the project?

The aim of this project is to develop a new generation of molecular patches for SMA that are safer and easier to deliver than existing patches like Spinraza.

Why is this research important?

Treatments for SMA must get into the spinal cord, as this is where the affected motor neurons are. However this can be challenging as the spinal cord and the brain are surrounded by a protective wall called the blood-brain barrier. This acts as a defence against bacteria and toxins that may be present in our blood.

Molecular patches such as Spinraza are unable to cross the blood-brain-barrier. This is why Spinraza has to be delivered directly into the spinal cord through a procedure known as an intrathecal injection. This procedure requires an experienced team of healthcare professionals and involves sedation or general anaesthetic, which can be risky for someone with SMA.

This research project will address the delivery challenges of Spinraza by developing new molecular patches that cross the blood-brain-barrier. These would be injected into the bloodstream (systemic injection) rather than intrathecally. If successful, this research could lead to a safer, less invasive and potentially more attractive treatment option than Spinraza.

What will the researchers do?

Dr Hammond and her team will design molecular patches like Spinraza and link them to antibodies. Antibodies are proteins found naturally in our bodies, which have the ability to recognise and bind to specific targets. The researchers will use antibodies that specifically recognise targets within the blood-brain-barrier. This will allow the molecular patch to cross the barrier and enter the spinal cord.

The researchers will test the antibody-linked molecular patches in a mouse model of SMA. They will assess how well they get into the spinal cord, how safe they are and how well they improve symptoms in the mice.

How will the outcomes of this research benefit people with SMA?

If successful, this project will generate molecular patches for SMA with the effectiveness of Spinraza, but which are safer and could be given via a simple injection into the bloodstream. These next-generation molecular patches could then be tested in clinical trials and potentially become a new treatment option for people with SMA.

How might this research benefit other neuromuscular conditions?

The antibody technology developed in this project could also be used to modify and improve molecular patches for other neuromuscular conditions where the blood-brain-barrier is an issue. This includes spinal bulbar muscular atrophy, myotonic dystrophy and Duchenne muscular dystrophy.

Grant information

Project leader: Dr Suzan Hammond

Institute: University of Oxford

Condition: Spinal muscular atrophy

Duration: Three years

Total cost (£): 225,000

Official title: Novel antibody-oligonucleotide drug conjugates target the CNS to treat neuromuscular disease.

For further information

If you would like further details about this research project, please contact the MDUK Research Line on 02078034813 or email research@musculardystrophyuk.org

Find out more about the MDUK Oxford Neuromuscular Centre