Eltrombopag is the first new drug treatment for severe aplastic anemia, shown to be effective in both previously treated patients unresponsive to other therapies and to improve responses in patients given the drug in combination with standard immunosuppressive drugs. Both indications received FDA approval (2014 and 2018, respectively) based on first-in-human clinical trials conducted by the National Heart, Lung, and Blood Institute at the NIH Clinical Center with the participation of Children’s Inn residents.

Researchers at the National Institute of Diabetes and Digestive and Kidney Diseases find that in patients with generalized lipodystrophy, long-term treatment with metreleptin is well tolerated and results in sustained improvements in hypertriglyceridemia, glycemic control and liver volume. Results support the recommendation of metreleptin as a first-line treatment (with diet) for children and adults with generalized lipodystrophy.

First lady Melania Trump visits The Children’s Inn at NIH for Valentine’s Day, starting a three-year tradition. Her visits bring joy and hope to Inn children as they create Valentine’s Day-themed arts and crafts together.

Researchers at the National Institute of Diabetes and Digestive and #INN2025, The Inn’s strategic plan, will accelerate The Inn’s impact on medical discovery and care by developing a state-of-the-art “smart” living environment, strong support programs that better integrate discovery and care, and a diverse group of leaders, supporters and volunteers.

During ongoing buiding renovations, a breach causes The Inn’s roof to partially cave. After safely evacuating all families and staff, The Inn’s main wing remains closed for months while the roof is shored up and rebuilt. The Inn makes use of the downtime to refresh family suites with new paint, bedding and other improvements. In March 2019, families and staff move back to the main wing.

A National Institute of Allergy and Infectious Diseases-led expanded access program shows the rheumatoid arthritis drug baricitinib improves symptoms of rare autoinflammatory diseases, including CANDLE and SAVI. Participating children reported symptom improvement, and biomarkers indicated high interferon levels dropped during the study. Researchers also noted improvements in height growth and bone mineral density that surpassed expectations.

Children’s Inn families now can relax on The Inn’s patio, enjoying comfortable outdoor furniture thanks to The Dick & Jody Vilardo Family Foundation. The space is adorned with a whimsical sign, displaying part of their family mantra, “Friends are family, and family is everything.” In 2019, Anthony Wilder Design/Build and its trade partners collaborate to design and build an outdoor cook station for families to grill out and prepare meals on the patio.

The National Institute of Mental Health’s focus on differentiating irritability from bipolar disorder in children has led to ongoing studies testing new treatment approaches for irritability.

The National Human Genome Research Institute launches a first-in-human gene therapy trial for GM1 gangliosidosis, a fatal disease that affects the brain and nervous system. The gene therapy is the only hope for children with the neurodegenerative disease.

In a race against the clock and the coronavirus pandemic, Jessica and Niclas leave Sweden to have their three children participate in the NIH GM1 gene therapy trial.

FDA approves the first treatment for neurofibromatis type 1, a tumor predisposition disorder that can cause tumors to grow along nerves anywhere in the body and cause deformities, pain, bring about vision and hearing loss and cause serious neurological complications. National Cancer Institute trials showed that selumetinib can shrink tumors in sometimes dramatic ways, benefiting many children for whom surgery is not an option.

Autumn, a frequent Inn resident, has been an important partner in research for the National Cancer Institute. Autumn’s clinical trial participation culminated in a major medical breakthrough: U.S. Food and Drug Administration approval of the first treatment for inoperable tumors caused by neurofibromatis type 1.

The COVID-19 pandemic continues to impact all areas of The Children’s Inn’s operations, including family visits, volunteering, family activities and fundraising events. Working closely with NIH epidemiology, The Inn implements and continuously adjusts strict pandemic safety measures to keep vulnerable children and families as well as staff safe, while doing everything possible to preserve the magic of The Inn. NIH Clinical Center safety measures mean only children and young adults with the most difficult diagnoses that require immediate treatment stay at The Inn, reducing Inn occupancy to 20-30 patients at a time, plus one accompanying family member. While occupancy is low, The Inn takes the chance to renovate the E-wing kitchen and dining area and refresh family suites.

A phase 1 National Cancer Institute trial finds that children with high-grade glioma or diffuse intrinsic pontine glioma not only tolerate much higher doses of the immunomodulatory agent lenalidomide than adults do when administered daily during six weeks of radiation therapy but also can achieve exceptional responses at higher dose levels and at higher steady-state plasma concentrations.

With fewer families staying at The Inn during the COVID-19 pandemic, The Inn uses this time to undertake needed building renovations. These include a complete makeover of the E-wing kitchen, dining room and living area thanks to generous support from Merck. Renovations also turn the former live-in resident services manager apartment into the 60th family suite, functioning as a concept room to test out new technology and materials. Due to a significantly lower occupancy rate during the pandemic, The Inn reduces its number of community apartments but keeps three to continue to provide lodging for families with children on isolation due to community-acquired infections.

FDA grants the rare pediatric disease designation for CAR T-cell therapy, an experimental immunotherapy for young patients with acute lymphoblastic leukemia.

Felix of Vienna, Austria, makes the long journey to The Inn to participate in NIH’s CAR T-cell therapy. Felix’s leukemia had returned multiple times, but since undergoing CAR T-cell therapy, he has been cancer-free.

Since the year 2000, the National Institute of Dental and Craniofacial Research has been doing research to better understand and treat children with fibrous dysplasia/McCune-Albright syndrome. Over two decades of research, NIDCR:

As part of a natural history study, NCI researchers administered neuropsychological assessments to children ages 6-18 with NF1 three times over the course of six years. Study findings show that children’s scores in math, inhibitory control and working memory decreased over time.

Patients staying at The Children’s Inn are instrumental in helping the National Eye Institute identify how likely it is that molecular diagnostic testing in children with largely non-syndromic coloboma will show genetic errors as a cause of the disorder. These patients are also critical in achieving a greater understanding of the role of systemic testing in this condition.

Late in 2023, The Inn broke ground on a new Young Adult Residence to be housed in a residential building on NIH’s campus, just across West Drive from The Children’s Inn.