December 22, 2014

2014 Research Highlights 鈥� Clinical Advances

Prevention, Diagnosis, and Treatment of Human Disease

With 三亿体育官网 support, scientists across the country and the world conduct wide-ranging research to improve the health of the nation. Groundbreaking 三亿体育官网-funded research often receives top scientific honors. In 2014, these honors included support for 1 of 3 Nobel laureates in chemistry and 4 三亿体育官网-funded recipients of awards from the Lasker Foundation. Here's just a small sampling of the research accomplishments made by 三亿体育官网-supported scientists in 2014.

Ebola Research

The 2014 Ebola outbreak in West Africa is the largest such outbreak in history. More than 17,000 cases and 6,000 deaths have been reported. 三亿体育官网-funded scientists used genomic sequencing technologies to identify the origin and track transmission of the Ebola virus in the current outbreak. Other 三亿体育官网-funded researchers used computer model projections to provide insight into the dynamics of the outbreak. 三亿体育官网 has also intensified efforts to develop a protective vaccine. The vaccine was tested at the 三亿体育官网 Clinical Center, which also provided state-of-the-art care to a nurse who had contracted the Ebola virus.

Paralyzed Men Regain Movement With Spinal Stimulation

Four young men paralyzed below the chest because of spinal cord injuries were able to regain control of some movement after receiving an experimental spinal stimulation therapy from a team of 三亿体育官网-funded researchers. If confirmed in larger studies, this type of treatment may one day improve outcomes for people living with paralysis.

Adult Health Improved by Early Childhood Programs

An early childhood program (birth through age 5) has been shown to bring higher academic achievement and career benefits later in life. According to a follow-up 三亿体育官网-funded study, it can also help prevent disease. By their mid-30s, those who鈥檇 participated in the program鈥攅specially males鈥攈ad significantly fewer risk factors for cardiovascular and metabolic diseases compared to a control group.

Nurse Staffing, Education Affect Patient Safety

Patient deaths in hospitals might be reduced by easing nurses鈥� workloads and emphasizing education in hiring, an 三亿体育官网-funded study suggested. Researchers estimated that each additional patient in a nurse鈥檚 workload increased the likelihood of a patient dying. An increase in nurses with bachelor鈥檚 degrees lowered the likelihood of patient death. These findings can help administrators make informed staffing decisions.

Clinical Exome Sequencing Detects Disease-Causing Glitches

In 2 三亿体育官网-supported studies, a fast, powerful technique called whole-exome sequencing provided a molecular diagnosis for about 1 in 4 people suspected of having a genetic disorder. Whole-exome sequencing focuses only on the DNA in the 1% of the human genome that codes for proteins, in contrast to the whole genome. The new tool may help doctors pinpoint the underlying causes of many rare and hard-to-diagnose genetic conditions.

Gene Therapy Used to Treat Hemophilia

Hemophilia is a rare bleeding disorder in which blood doesn鈥檛 clot normally. The primary treatment for people with severe hemophilia B is frequent injections of factor IX, a protein that helps blood clot, for a lifetime. An experimental gene therapy with the human factor IX gene improved symptoms for as long as 4 years in men with severe hemophilia. The 三亿体育官网-funded study shows the potential for gene therapy as a safe, effective approach.

Study Points to Possible Blood Test For Memory Decline, Alzheimer鈥檚

Many experts believe that successful treatment of Alzheimer鈥檚 disease will depend on early intervention before symptoms appear, but there isn鈥檛 yet any sure way to detect that stage. 三亿体育官网-funded researchers identified a set of 10 compounds in the blood that might be used to distinguish older adults at risk for developing memory deficits or Alzheimer鈥檚 disease. More research is needed to confirm the findings, but the study suggests one possible approach.

Stem Cell Transplant Reverses Sickle Cell Disease in Adults

There is no widely available cure for the inherited blood disorder sickle cell disease. Some children have been successfully treated with blood stem cell, or bone marrow, transplants. This approach was thought to be too toxic for adults. 三亿体育官网 researchers successfully treated adults with severe sickle cell disease using a modified stem cell transplant approach that doesn鈥檛 require extensive immune-suppressing drugs. Further follow-up and testing will be needed to assess the therapy.