National Center for Advancing Translational Sciences (NCATS)

As we welcome the astronauts back to Earth, we’re reflecting on our #ChipsInSpace activities. The microgravity environment has allowed astronauts working in the International Space Station National Laboratory to use tissue chips to better understand diseases and conditions in ways that may be difficult to achieve or would take longer to do on Earth. Our first program offered insights into the effects of spaceflight and microgravity on the heart, bone, kidneys and other organs. ? We’re excited about what’s on the horizon! ?? We’re actively collaborating with NASA - National Aeronautics and Space Administration, the FDA?and the Biomedical Advanced Research and Development Authority (BARDA) to adapt existing #TissueChips to better understand the effects of space on humans during long-duration spaceflights and to support longer-term studies on the ground, which could be important for studying acute versus chronic exposures. ?? Also, plans are well underway for Tissue Chips in Space 2.0, which will support the development of more complex organ-on-a-chip platforms to better model microgravity-induced physiological changes associated with aging and related diseases. ? Learn about Tissue Chips in Space: https://go.nih.gov/Xy7NKrh

We develop platform approaches to streamline the development of gene-targeted therapies for many diseases at a time through programs like #PaVeGT, #BGTC and #SCGE. We’re also thinking about platform approaches to early diagnosis when #GeneTherapies can greatly improve disease outcomes. The NIH-funded Newborn Screening by Whole Genome Sequencing (#NBSxWGS) Collaboratory will explore the feasibility of integrating whole genome sequencing for #RareDiseases with available interventions into United States public health newborn screening programs. View the initiative page for details and learn more about the first #FundingOpportunity: https://lnkd.in/efc2RAad

Researchers are tackling the challenge of predicting adolescent #MentalHealth risks in youth. Using data from over 11,000 children as part of an ongoing study, they developed an #ArtificialIntelligence model that accurately predicted those at high risk for mental illness within the next year, with sleep disturbances being a key predictor. This study — published in Nature Medicine — highlights the potential for AI to improve mental health screening and early intervention strategies in primary care settings. Learn more about this NIH-supported study: https://lnkd.in/gvNs6GeM

Children with Aicardi-Goutières syndrome (AGS), a rare genetic disorder affecting the brain, spinal cord and immune system, can have a range of intellectual and language limitations. A recently published study supported by scientists from the Global Leukodystrophy Initiative — part of the NCATS-led Rare Diseases Clinical Research Network (RDCRN) — and funded by the National Institute of Neurological Disorders and Stroke (NINDS) found that a pair of objective assessment tools measuring non-verbal learning skills can provide insights into the cognitive capabilities of children with AGS. “These results are a validation for parents saying, ‘My child can understand more than you are capturing in your tests,’” said Laura Adang, M.D., Ph.D., a clinician at Children's Hospital of Philadelphia who co-led the work. “We have to listen to families.” Read more: https://go.nih.gov/8yfchaK #RareDiseases #RDCRN

#Glioblastoma (GBM) is a rare brain cancer with no adequate treatments and a five-year survival rate of less than 5%. #NCATSIntramural scientists developed an innovative approach to find existing drugs that may work against GBM. This new #DrugRepurposing approach found two drugs that outperformed the current standard GBM treatment in cell-culture experiments. Learn more: https://go.nih.gov/iUnV8lu #RareDiseases

Check out this research partially supported by NCATS’ #CTSAProgram???

Do you want to learn how to bridge the gap between preclinical research and the delivery of novel therapeutics? Join us for the #AssayGuidanceManual webinar on March 7 at 11 a.m. EST with #GeneTherapy pioneer Katherine High, M.D.! Dr. High will discuss the relationship of preclinical studies to the clinical development of Luxturna — the first FDA-approved gene therapy for a genetic #RareDisease. Register today: https://lnkd.in/dt3KR5dX

A new study from Penn State Clinical and Translational Science Institute researchers reveals that an irregular #sleep schedule can have long-term effects on young people’s heart health, increasing risks later in life. These findings suggest that maintaining a regular sleep cycle may be key to protecting adolescent #HeartHealth. This research was supported by NCATS’ #CTSAProgram, the National Institute of Mental Health (NIMH) and the National Heart, Lung, and Blood Institute. Learn more: https://lnkd.in/e4YcD8R8

??Researchers with the Platform Vector Gene Therapy (PaVe-GT) Program use a common gene delivery vehicle, adeno-associated virus (#AAV), to create gene therapies for four #RareDiseases and incorporate drug development efficiencies. In a new Mary Ann Liebert, Inc. publication, #PaVeGT researchers share the key attributes of a Target Product Profile and their process of preparing for and participating in the FDA INTERACT meeting — important steps in establishing a viable regulatory path to start a #ClinicalTrial. The team has also made available the complete INTERACT package with FDA feedback and an example target product profile. Sharing this information and future learnings from PaVe-GT will help inform other #GeneTherapy development programs. Read more: https://lnkd.in/gwcVWvfJ?#NCATSLabs?

#ClinicalTrials are key to developing and evaluating new treatments for #RareDiseases. We worked with the Eunice Kennedy Shriver?National Institute of Child Health and Human Development (NICHD) to fund #ClinicalTrialReadiness for Rare Diseases, Disorders and Syndromes grants. These grants will help bridge gaps in our understanding of rare diseases and address other obstacles scientists face. Read more: https://go.nih.gov/i6PaEqN