First trial of new gene therapy for HSP (SPG47) approved

There is hopeful news for people with HSP: the US health authority FDA has approved an initial trial of a new gene therapy for a rare form of hereditary spastic paraplegia, namely SPG47.

What is SPG47?

SPG47 is a rare, hereditary form of HSP where children show symptoms at an early age, such as muscle stiffness, difficulty walking, developmental delays, and speech problems. The cause lies in a mutation in the SPG47 gene, which prevents an important protein in brain cells from functioning properly. Currently, there is no treatment available.

What will happen now?

A biotech company, BlackfinBiohas been granted permission to begin the first study involving children with SPG47. The trial will take place at a specialized hospital in Boston (USA). The goal is to determine whether the treatment is safe.

The treatment involves a one-time injection into the cerebrospinal fluid containing a healthy piece of DNA. This healthy gene should enable the child’s cells to produce the correct protein again.

Why is this important?

• This is the first treatment for SPG47 to be tested in humans.
• The therapy targets the root cause of the disease, not just the symptoms.
• FDA approval is a major step toward possible treatment in the future.

What does this mean for families?

Although this research is still in its early stages, it offers new hope for children with SPG47 and their families. Progress is being made, and scientists and doctors are seriously working on solutions for rare forms of HSP. here more about research