patient story

patient name: Ciro Lavato

age: 3 years old

condition: Canavan disease

seen in: Neurosurgery

provider: Rob Lober, MD, PhD, FAANS

The Lavatos’ story began in Argentina, where Juan was a professional soccer player and Natalia, a successful business owner. In addition to their successful professions, Juan and Natalia were embracing life as first-time parents to their beautiful son, Ciro. 

When Ciro was six months old, Juan and Natalia noticed something wasn’t quite right. Ciro struggled to sit on his own, couldn’t hold his head up and had difficulty eating. Like any mother, Natalia hoped it was nothing serious, but as time went on, it became clear that Ciro needed help. 

finding hope after a devastating diagnosis

Ciro’s journey to a diagnosis was long and uncertain. He saw ten different specialists before they finally discovered the cause of his symptoms: Canavan disease, a rare degenerative condition that damages nerve cells in the brain, affecting vision, speech and motor functions. The Lavatos were devastated when they learned of the diagnosis. But in that same moment, they were given a glimmer of hope – a clinical trial that might offer a path forward. 

At the time of Ciro’s diagnosis, Dayton Children’s Hospital in collaboration with Myrtelle, a gene therapy company, was the first and only to conduct a Phase 1/2 clinical trial for Canavan disease. This First-in-Human gene therapy study utilizes a novel recombinant adeno-associated virus (rAAV) vector, known as rAAV-Olig001-ASPA, to target oligodendrocytes in the brain. At Dayton Children’s, the clinical trial is led by pediatric neurosurgeon, Dr. Robert Lober. 

The Lavato family participated in a video consultation with Dr. Lober and learned that Ciro qualified for the groundbreaking trial. The prospect of a new treatment was enough for Juan and Natalia to make a life-changing decision. They left behind their professional lives and everything they knew to come to Dayton, where Ciro could receive the care he needed. 

Upon arriving at Dayton Children’s, the family felt an immediate sense of comfort and trust. They were not only welcomed into a community of families experiencing the same challenges but they were also welcomed by a community of caregivers with the largest experience and expertise in the world regarding Canavan disease. The cutting-edge care offered hope where there had been none. 

reaching new milestones after gene therapy

Just a month after their arrival, Ciro underwent gene therapy surgery. The results were nothing short of miraculous. Slowly but surely, Ciro began to regain abilities that once seemed impossible. With support, he can now stand, take steps, hold his head up and even transfer objects between his hands.  

“He’s made incredible strides,” Natalia shares. “He says he wants to eat and draw pictures, and he’s very attentive to see what everyone is doing around him.” 

Extensive therapies have played a crucial role in Ciro’s progress. He and his family attend numerous therapies at the hospital each week. Juan and Natalia would tell you that their commitment to helping Ciro develop and progress has been worth every challenge they’ve faced along the way. Each day, they witness Ciro reaching new milestones, bringing renewed hope for their son’s future. 

“We’re very enthusiastic about what we’re seeing,” said Dr. Lober. “There are things we still don’t understand about this disease, but as we go along, we are more and more hopeful about the results.” 

Ciro’s journey is a testament to the exceptional care at Dayton Children’s and the power of hope. For families like the Lavatos, Dayton Children’s isn’t just a hospital – it's a lifeline, a place where the impossible becomes possible, and where every step forward is a victory worth celebrating. 

For more information about the gene therapy trial, visit clinicaltrials.gov.