See Who Inspires Us | Meet Brian & Brandon

When Paul and Liliana Rojas talk about their life, they describe it the way it was before their sons, 10-year-old Brandon and 8-year-old Brian, were diagnosed with adrenoleukodystrophy (ALD), and the way it is after. Their story is one of heartbreak — but also hope, in the form of a clinical trial for this debilitating brain disease.

The first signs of ALD

In May 2014, Brandon was showing signs of fatigue and an inability to focus. Based on some other neurological symptoms, he was referred to a local pediatric neurologist. Weeks later, the Rojases learned Brandon had ALD. This genetic mutation causes a buildup of very long chain fatty acids that destroy the protective sheath around nerve cells, responsible for thought and muscle control. Physicians explained that it primarily affects young boys between the ages of 4 and 10, leading to permanent disability or death within four to eight years. Then, they asked the Rojases, “Do you have any other children?” “It was a nightmare,” Paul says. “In one sit-down, they give us a diagnosis on Brandon, and then we learn it’s possible Brian has the same thing.”   

A race against time

As time passed, the Rojases began to notice more changes in Brandon. By October, he was in a wheelchair and no longer able to speak. In November, the family was devastated to discover that Brian also had ALD. Then they learned that Brian qualified for a study at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. In this experimental treatment for asymptomatic boys, their blood cells are removed, treated with a virus carrying the gene for the ALD protein and then reinfused. The added gene would enable Brian to make the protein needed to process certain fats and halt the disease’s progression. But the Rojases would have to move quickly. Although Brian was not exhibiting any symptoms, ALD progresses rapidly. They headed to Boston.

Surviving ALD

After undergoing chemotherapy to prepare him to accept the newly gene-corrected blood stem cells, Brian received the single infusion aimed at preventing ALD from affecting his brain. Remarkably, there haven’t been any signs of disease activity since then. The joy the Rojases feel for Brian is tempered by the sorrow they feel for Brandon. “I cry when I see pictures of when Brandon was healthy,” admits Liliana. “I ask myself, why can’t I have him back like that? But I know he is still with us.”