Featured Studies

Treatment of severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) usually involves a bone marrow transplant. Researchers at the National Institutes of Health (NIH) are testing ways to make transplant using unrelated donor cells from umbilical cord blood safer and more effective. We hope these procedures will reduce post-transplant complications and improve transplant outcomes.

Fanconi anemia is a rare genetic disorder that mostly affects children and often leads to bone marrow failure. Researchers at the National Institutes of Health (NIH) are investigating a new drug to improve blood counts in individuals diagnosed with Fanconi anemia. Eltrombopag is an oral drug that mimics a special protein that causes the body to make more platelets. Currently eltrombopag is used to treat low platelet counts in patients with hepatitis C and chronic immune thrombocytopenic purpura (ITP). Researchers are evaluating if this new drug will improve blood cell counts in individuals diagnosed with Fanconi anemia.

African-American women, ages 25-75, who are obese or overweight and pre-diabetic, are needed to participate in a research study at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland. Researchers want to explore if the use of mobile technology and coaching can help increase physical activity.

Crohn's disease is a chronic bowel illness that is a lifelong condition characterized by relapsing inflammation. Although there is no cure for Crohn's disease, researchers at the National Institutes of Health (NIH) are investigating if Vorinostat is safe for people with Crohn's Disease. Participants will receive maintenance therapy with Stelara® after successful treatment with Vorinostat.

Researchers at the National Institutes of Health are conducting a research study to learn more about the motor skill and brain development of infants who are at high-risk for or diagnosed with Cerebral Palsy (CP) and Autism Spectrum Disorder (ASD) compared to infants with typical development. The results of this study may assist with better methods for early diagnosis as well as improved treatment for children with CP and ASD. The results of this study may eventually assist with better methods for early diagnosis as well as improved treatment for children with autism.

Researchers at the National Institutes of Health (NIH) are studying if the medication danazol can be used to treat people with short telomere disease who also have bone marrow failure, liver, or lung disease. In recent studies, danazol, at high doses, showed a positive influence on telomere length. Additional research is needed to learn more about the role of different doses of danazol on telomere disease as well as its effects on secondary diseases.

Doctors at the National Institutes of Health (NIH) seek healthy volunteers (with or without a sickle cell trait) and patients with sickle cell for a research study. Sickle cell disease is a genetic blood disorder that occurs mainly in people of African descent. Study participants will have a one-time visit to the NIH to provide blood samples. Researchers hope this study will help us better understand sickle cell disease.

National Cancer Institute (NCI) researchers are conducting a study of patients with IDH-mutated gliomas. This study will test whether stimulating the immune system using the drug nivolumab can shrink recurrent IDH-mutant gliomas with and without hypermutator phenotype or increase the time it takes for them to grow or spread.