The future of neurological research at Texas Children’s Hospital: 5 things to know

Brain disorders affect more than 3 billion people worldwide. In the U.S. alone, nearly 5% of all newborns will have autism, intellectual disability, behavioral problems or epilepsy. On the other end of the lifespan, more than 55 million people around the world live with dementia — a figure that researchers expect to balloon to roughly 150 million by 2050.

In 2010, Texas Children’s Hospital set out to change this reality. Since then, the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital has aimed to chart new paths into brain discovery to solve the unsolvable and improve the lives of patients impacted by debilitating conditions from autism and epilepsy to Alzheimer’s and OCD.

Here are five things to know:

1. The Duncan NRI at Texas Children’s has already discovered the causes of more than 100 rare neurogenetic diseases that were once believed unsolvable. Some of these breakthroughs have turned into clinical trials that are helping children and families today — including those suffering from Batten disease, Rett syndrome and Angelman syndrome.
   
2. Today, 50% of neurological conditions remain unsolved. To change this, Duncan NRI researchers are now extending their focus to tackle adult diseases like Alzheimer’s, Parkinson’s and psychiatric disease in addition to childhood disorders. They’ve already pinpointed promising genes, including ITSN1 in autism and Parkinson’s, that will serve as potential drug targets.
   
3. The Duncan NRI is home to one of the largest fruit fly lab in the world, powered by custom robots. Fruit flies share 75% of human disease-causing genes, so studying them accelerates the discovery of underlying disease drivers.
   
4. The Duncan NRI’s computational scientists created an AI-based tool, AI MARRVEL, that dramatically speeds the ability of researchers to pinpoint the genetic diagnosis of rare and undiagnosed conditions. This tool helps narrow the potential genetic mutations responsible for different diseases. Then, geneticists in the fruit fly lab engineer those mutations in fruit flies to determine which changes cause damage.
   
5. Psychiatric diseases present particularly complex challenges, as an array of genes often contribute to illness. Duncan NRI researchers are increasingly learning that these diseases may be more connected to other neurological disorders than previously understood. In fact, have found that some of the same genes implicated in childhood neurologic disease may, when present in milder form, be related to psychiatric disease. And so, as they look forward to the institute’s next 15 years, the Duncan NRI’s leaders have a moonshot: By harnessing new technology and evolving neurogenetic understanding, their goal is to identify the complex genetic causes that make an individual vulnerable to psychiatric disorders — and, as a result, help these patients early, before the disease sets in.

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