• Universitat de València
• Fundación INCLIVA

• Piotr Tadeusz Konieczny

Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease manifested by a progressive loss of muscle strength. This occurs mainly due to the involvement of the motor neurons in the spinal cord, which means that the nerve impulse cannot be transmitted correctly to the muscles and that the muscles atrophy. The disease is caused by an alteration in the gene coding for the SMN (surviving motor neuron) protein. It is considered the second leading cause of neuromuscular diseases, with a prevalence of 4 per 100,000 people. Although SMA is considered a neuromotor disease, recent evidence shows that the pathology affects not only muscles but also the brain, heart, pancreas and even blood vessels.

The only therapeutic approach already available for SMA patients is based on the use of oligonucleotides to increase the amount of functional SMN protein. However, the high cost of the therapy, the type of administration (lumbar puncture) and the relatively weak improvement of patients are still important limitations. Therefore, the search for new effective and systemically deliverable drugs is an urgent need in the search for SMA treatments.

Research staff from the UV together with the Fundación INCLIVA have identified a new valid molecule to develop a new therapy for SMA. This new approach involves the use of moxifloxacin, a drug already approved as an antibiotic and widely used to treat bacterial infections. Moxifloxacin has been found to significantly increase SMN protein levels, one avenue of treatment for spinal muscular atrophy.

Moxifloxacin has demonstrated efficacy as an antibiotic when administered by oral, injectable and ophthalmic routes, offering for SMA the possibility of less invasive administration than currently used treatments. In addition, its lower cost would allow the development of a treatment with distribution of the drug throughout the body, which would help to alleviate not only the neurological effects, but also the other alterations that also occur in this disease.

The main application of the technology is in the pharmaceutical sector as an active ingredient for the treatment of Spinal Muscular Atrophy.

The main advantages provided by the invention are:

• Non-invasive administration: oral, injectable, ophthalmic.
• Systemic distribution, which makes it possible to combat not only neurological signs but also other problems associated with the disease.
• Lower cost compared to existing treatments.

• Patent granted