Progress on Theme 5

Advancing Safe and Effective Therapeutics and Devices for Pregnant and Lactating Women, Children, and People with Disabilities

Highlighted Programs and Activities

  • NEW: Home and community-based physical activity interventions to improve the health of wheelchair users
    NICHD, the National Institute of Neurological Disorders and Stroke and the NIH Office of Disease Prevention launched a Pathways to Prevention (P2P) initiative titled “Can Physical Activity Improve the Health of Wheelchair Users?” This multipronged effort included a Systematic Evidence Review, a workshop held in December 2020, and a meeting of federal agencies that conduct research or other activities in this field. NICHD is now requesting research applications to develop/adapt and test physical activity interventions for individuals who use wheelchairs due to physical disability. Interventions that could be applied or easily adapted to large populations of wheelchair users and used in multiple settings are a priority. Learn more: RFA-HD-22-017; NIH Pathways to Prevention Workshop.
  • Maternal and Pediatric Precision in Therapeutics (MPRINT) Hub
    The MPRINT Hub will serve as a national resource, providing expertise in maternal and pediatric therapeutics, for conducting and fostering therapeutics-focused research in obstetrics, lactation, and pediatrics, while enhancing inclusion of people with disabilities. The MPRINT Hub will disseminate available knowledge, tools, and expertise in maternal and pediatric therapeutics to the broader research, regulatory science, and drug-development communities. Learn more: RFA-HD-21-025, RFA-HD-21-026.

Selected Recent Advances

  • NEW: High dose of concentrated therapy produces several lasting benefits for children with cerebral palsy (PMID: 34649982)
    Children with hemiparetic cerebral palsy, a movement disorder that affects use of one side of the body, showed improved use of the arm and hand after receiving a high dose of Constraint-Induced Movement Therapy (CIMT). CIMT involves restricting the better functioning arm and hand with a splint or cast, while a highly trained therapist engages the child in activities that reinforce and shape the movement and functional skills in the impaired arm and hand. Researchers found that a more intensive level of CIMT—3-hour sessions, 5 days a week for 4 weeks—produced the most noticeable and longer lasting improvements. A moderate dose—2.5-hour sessions, 3 days a week for 4 weeks—did not produce gains significantly greater than the control group, which received a standard combination of physical and occupational therapy.
  • NEW: Repurposed drug shows promise in mouse model of rare childhood genetic disorder (PMID: 34802899)
    Niemann-Pick disease type C1 (NPC1), a rare genetic disorder affecting children and adolescents, results from an impaired ability to move cholesterol through cells, leading to difficulty controlling movements, liver and lung disease, impaired swallowing, intellectual decline, and death. NICHD researchers found that Riluzole, a drug approved to treat amyotrophic lateral sclerosis, a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in NPC1.
  • MRI technique could reduce need for radiation in measuring tumor response to chemotherapy in children (PMID: 32368961)
    Researchers compared whole-body diffusion-weighted magnetic resonance imaging (DW MRI), which measures the density of tumors by tracking the movement of water molecules in tissue, to fluorine 18 fluorodeoxyglucose positron emission tomography (FDG PET). Both techniques showed significant agreement in tracking tumor response to therapy, raising the possibility that DW MRI might one day be used in place of computerized tomography scanning, either together with FDG PET or alone, without the need to inject radioactive glucose. This new approach could reduce radiation exposure by 80% for combined FDG PET/DW MRI and fully eliminate radiation exposure for tumors that can be evaluated with DW MRI only.
  • Personal Protective Equipment (PPE) does not hinder pediatric Cardiopulmonary Resuscitation (CPR) (PMID: 32483079)
    Studies have shown that wearing PPE while performing CPR on adult manikins results in more fatigue and poorer quality chest compressions. However, researchers found no such effect in pediatric CPR; the quality of the compressions and fatigue levels were no different for PPE compared with usual clothing.
  • Effect of metformin treatment differs in gestational diabetes mellitus (GDM) and type 2 diabetes (PMID: 31742716)
    Researchers showed that the pharmacological response to metformin treatment was significantly different in pregnant women with GDM compared with nonpregnant women with type 2 diabetes. Despite similar metformin exposure, women with GDM had a greater improvement in insulin sensitivity and peak glucose concentrations than nonpregnant women with type 2 diabetes. These results provide evidence for more tailored treatments for GDM.
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