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: Bioprinting tissue technology for obstetric, gynecologic, and pediatric applications
    NICHD’s new initiative encourages translational research with 3D bioprinting technologies to support development of tissue constructs for using in obstetric, gynecologic, and pediatric populations. In children, long-term use of these technologies will need to adapt to the needs of growing children; likewise, in obstetric and gynecological conditions, the technology will need to adapt to changing hormone levels and possible pregnancy. Overall, this initiative aims to promote the clinical translation of bioprinted tissue constructs by helping to identify and overcome barriers to preclinical product development, validation, and verification. Evidence generated through this initiative will allow for regulatory approval and human testing of mature bioprinted tissue constructs, addressing the unmet needs of NICHD mission-relevant gynecologic, obstetric, and pediatric populations. Learn more: RFA-HD-23-004.
  • NEW: Clinical trials and clinical trial readiness for co-occurring conditions in Down syndrome
    A primary goal of the NIH-wide INCLUDE (Investigation of Co-occurring conditions across the Lifespan to Understand Down syndromE) Project is to conduct clinical trials on critical and co-occurring conditions in Down syndrome that involve people with Down syndrome. To that end, the project is requesting applications to not only support development of clinical trials, but also improve the efficient and effective movement of candidate therapeutics or diagnostics toward clinical trials, such as by developing and testing biomarkers and clinical outcome assessment measures, creating and evaluating novel trial methods and recruitment strategies, and defining the presentation and course of the co-occurring conditions in individuals with Down syndrome. A separate solicitation offers support to individual scientists or groups of scientists proposing groundbreaking, exceptionally innovative, original, and/or unconventional research with the potential to create new scientific paradigms, establish new and improved clinical approaches, or develop transformative technologies related to Down syndrome. Learn more: RFA-OD-22-007, RFA-OD-22-009, and RFA-OD-22-010.
  • NEW: Clinical trial readiness for rare diseases, disorders, and syndromes
    NICHD and other NIH components are working to support clinical projects that address critical needs for clinical trial readiness in rare diseases; among these needs are adequate understanding of the course of the disease, and sensitive and reliable biomarkers and outcome measures for use during a clinical trial. These projects will help enable efficient and effective movement of candidate therapeutics or diagnostics toward clinical trials and increase their likelihood of success. Learn more: PAR-22-100 and PAR-22-101.
  • NEW: NICHD Neonatal Research Network (NRN)
    The purpose of the NRN is to improve healthcare and outcomes for newborns, such as by finding ways to improve the chances for survival without neurodevelopmental impairment for infants born preterm, low-birth weight, or with other serious conditions. NICHD is updating the NRN to be its primary and first-line infrastructure involved in implementing multisite neonatal clinical trials. The NRN will conduct definitive, rigorous, and reproducible, multisite clinical trials and observational studies in newborns and lactating people, providing evidence to guide neonatology, pediatric pharmacology, and lactation clinical practice. The network infrastructure will include clinical sites, a data coordinating center, and, for the first time, separate grants awarded under separate funding opportunity announcements. Learn more: RFA-HD-23-001 and RFA-HD-23-002.
  • NEW: NICHD Maternal-Fetal Medicine Units (MFMU) Network
    NICHD is updating the MFMU Network program to better achieve its goals of improving obstetric care, pregnancy health, and outcomes for pregnant and lactating people and their babies. Updates include finding ways to: reduce maternal mortality, complications, and morbidities related to pregnancy, labor, and postpartum recovery; reduce prematurity, low-birth weight, infant mortality, and morbidities; and expand the evidence base about the safety and efficacy of therapeutic products used during pregnancy and lactation. NICHD expects the MFMU Network to be its primary and first-line infrastructure involved in implementing multisite obstetric clinical trials. The network infrastructure will include clinical sites, a data coordinating center, and, for the first time, separate grants awarded under separate funding opportunity announcements. Learn more: RFA-HD-23-016 and RFA-HD-23-017.
  • 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? in 2020. 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.

Selected Recent Advances

  • NEW: Hydrocortisone does not prevent lung complication in preterm infants (PMID: 35320643)
    In recent years, hydrocortisone was used as a replacement for the widely used drug dexamethasone for prevention of bronchopulmonary dysplasia (BPD). Both drugs inhibit the inflammation thought to contribute to BPD, but animal studies suggested that hydrocortisone had fewer effects on the developing brain. Infants born preterm have higher rates of death and disability. Researchers studied 800 infants born before the 30th week of pregnancy who had been on a ventilator for at least 7 days. From 14 days to 28 days, in addition to receiving standard care and ventilator therapy, infants were randomly assigned to receive either hydrocortisone or a placebo. Of the hydrocortisone-treated infants, about 17% survived to 36 weeks without moderate or severe BPD, a figure that did not differ significantly from 13% in the placebo group. The rate of neurodevelopmental impairment was about 37% in both groups. The data indicated that hydrocortisone did not prevent this lung complication in extremely preterm infants.
  • NEW: Placental genetic material in maternal blood could potentially diagnose gestational diabetes mellitus (GDM) early (PMID: 35613088)
    Typically, GDM, diabetes that develops during pregnancy when there was no prior history of diabetes, is not diagnosed until the late second trimester or the early third trimester. However, researchers identified a new method to detect genetic material from the placenta in the blood of pregnant people. This method could potentially identify the risk for GDM in the first trimester, allowing some patients to benefit from preventive treatments before diabetes develops.
  • 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. More intensive 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.
  • Repurposed drug shows promise in mouse model of rare childhood genetic disorder (PMID: 34802899)
    Niemann-Pick disease type C1 (NPC1), a rare genetic disorder, 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 (sometimes called Lou Gehrig's disease) that affects 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. The findings suggest that DW MRI might one day replace computerized tomography scanning, either together with FDG PET or alone, without the need to inject radioactive glucose. The new approach could reduce radiation exposure by 80% for combined FDG PET/DW MRI and eliminate radiation exposure for tumors 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.
  • Metformin treatment outcomes differ 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 women with GDM.
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