A protein to repair the brain: Italy's Ngf study begins

Intranasal trial started at Policlinico Gemelli: will involve 60 young patients with cerebral palsy in ten Italian centres

by Francesca Cerati

30 March 2026

3' min read

Translated by AI

Versione italiana

Key points

Cerebral palsy cases
Possible therapies
What the study involves

3' min read

Translated by AI

Versione italiana

A Nobel Prize-winning protein discovered over seventy years ago and linked to nerve cell growth is now back at the centre of medical research with an ambitious goal: to try to change the fate of children with infantile cerebral palsy.

At the Agostino Gemelli University Hospital in Rome, the first patient has been enrolled in a phase 2 clinical trial that will evaluate the efficacy of Nerve Growth Factor (Ngf) administered intranasally. The trial is promoted by the Italian biopharmaceutical company Dompé, which is studying the therapeutic potential of the molecule - cenegermin, an orphan drug already approved to treat neurotrophic keratitis - to improve motor skills and neurological development in children suffering from spastic cerebral palsy.

The cases of infantile cerebral palsy

Infantile cerebral palsy is the most common motor disability of childhood. It is a group of permanent disorders of movement and posture development, caused by non-progressive lesions or abnormalities of the brain early in life. The most common form is the spastic form, characterised by muscle rigidity and difficulty in movement. In Europe, an estimated 712,000 people live with this condition, while in the United States it is about 764,000. In Italia, approximately 740 children with cerebral palsy are born each year.

Despite the impact of the disease, there are currently no therapies that can intervene in the underlying brain damage. Available treatments mainly aim at alleviating symptoms and include physiotherapy, occupational therapy and speech therapy to improve coordination, muscle strength and autonomy in daily activities.

It is precisely on this therapeutic limit that the new trial fits in. Ngf is a protein naturally present in the body and plays a crucial role in the survival, growth and repair of nerve cells. The researchers' hypothesis is that its administration may promote the processes of recovery and communication between neurons in damaged brain areas.

Possible therapies

"Infantile cerebral palsy is a complex challenge for children and families, and there are currently no proven therapies available other than supportive care," explained Marcello Allegretti, Chief Scientific Officer of Dompé. With our innovative platform based on intranasally administered Ngf, we hope to offer a treatment for infantile cerebral palsy and pave the way for a better future for patients and their families.

The intranasal route represents one of the most innovative aspects of the project. For the first time, a biotech molecule such as recombinant human Ngf is administered in a clinical study with the aim of directly reaching the central nervous system. The protocol is also based on previous studies conducted on children with severe head trauma or cardiovascular arrest by Antonio Chiaretti, paediatrician at the Catholic University of the Sacred Heart and director of the paediatric emergency room at Gemelli.

According to Ahmed Enayetallah, Dompé's chief development officer, the scientific rationale for the trial is sound: 'The disability associated with infantile cerebral palsy is closely linked to the physiological role of Ngf in the body, i.e. supporting the growth, survival and repair processes of developing nerve cells.

What the study involves

The study involves the enrolment of 60 children between the ages of two and six, with different levels of severity of the disease according to the international Gmfcs classification. After starting at Gemelli, the trial will also be activated at the Bambino Gesù Paediatric Hospital and progressively in other Italian centres, up to a total of ten facilities involved.

The aim is not only to verify the safety and tolerability of the treatment, but also to assess any improvements in daily activities, quality of life, motor skills and neurodevelopmental indicators.

If the results are positive, the molecule that won Rita Levi-Montalcini and biochemist Stanley Cohen a Nobel Prize could open a new chapter in research into neurological diseases of childhood. A perspective that today starts with a first small patient and an open question: whether it is possible to help the growing brain repair itself.