T H E   N I H    C A T A L Y S T      M A Y   –  J U N E   2002


Organized According to Government Performance and Results Act Goals

Goal A: Add to the body of knowledge about normal and abnormal biological functions and behavior

Identification of disease genes

GNAS1 mutations that underlie McCune-Albright syndrome (MAS) found to underlie non-MAS fibrous dysplasia (NIDCR, NIDDK)

Protocadherin defect found to underlie Usher 1F syndrome (NIDCD)

TMC1, a novel transmembrane protein, found to underlie DFNA36, DFNB7, and DFNB11 (NIDCD)

More prostate cancer susceptibility genes identified using DNA chip technology (NHGRI)

Germline mutations in the Fas immune cell receptor associated with autoimmune lymphoproliferative syndrome also found to increase lifetime lymphoma risk (NIAID, NCI, NHGRI, CC)

Altered COMT gene found to disrupt dopa-mine function in prefrontal cortex and increase risk of schizophrenia (NIMH, NIAAA)

ST7, new tumor suppressor gene found on chromosome 7, is involved in tumors arising from epithelial cells, such as breast, prostate, colon, and ovary (NHGRI)

Novel cytokine receptor-binding protein that regulates receptor shedding and innate immune responses provides new insights into the regulation of airway inflammation (NHLBI)

A macromolecular complex involved in the initiation and regulation of membrane trafficking provides new insight into mechanisms of vesicle formation (NHLBI)

Complete genomic sequence and regulatory elements modulating gene expression have been determined for the ABCA1 transporter, the protein whose gene is defective in Tangier disease (NHLBI)

Analysis of FOXL2, a gene that determines the age of menopause by controlling the number of ovarian follicles formed, is an entry point to understanding the regulation of reproductive lifespan (NIA)

Mutation in a gene normally associated with spinocerebellar ataxia (SCA3) can produce a clinical picture more similar to Parkinson’s disease in different racial groups, showing how ethnic background can affect expression of genetically determined disease (NIA)

Important new animal models

Transgenic mouse model to study relationship between carcinogens and skin cancer using transformation-associated recombination cloning (NIEHS)

Mouse model to assess the toxicity of combination anti-AIDS regimens (NIEHS)

Knockout mouse model of amelogenesis imperfecta sheds light on hereditary dental enamel defect (NIDCR)

Mouse model of b-thalassemia suggests gene therapy is successful if at least 10 percent and ideally 20 percent of stem cells are corrected (NHGRI)

Mouse models to explore diversity of brain signals regulating appetite and body size; mice lacking the M3 muscarinic acetylcholine receptor are hypophagic and lean (NIDDK, NIAAA, NIMH)

PDS knockout mouse model to study inner-ear defects and deafness in Pendred syndrome (NIDCD)

Mouse model for human nonsyndromic hearing impairment (DFNA36/B7/B11)

Mouse model for Usher syndrome type 1D (NIDCD)

Transgenic mouse model of a targeted disruption of a cyclic nucleotide phosphodiesterase gene provides new insights into the regulation of insulin homeostasis (NHLBI)

Animal model of immune-mediated aplastic anemia shows pivotal role of type 1 cytokines in causing severe marrow cell destruction (NHLBI)

Animal model demonstrates that cocaine craving increases progressively over weeks or months of forced cocaine abstinence, in parallel with increases in brain-derived neurotrophic factor in reward-associated brain circuitry (NIDA)

Knockout mouse model demonstrates that animals with a D2 dopamine receptor deletion do not self-administer morphine (NIDA)

Basic discoveries in cell, molecular, and structural biology with implications for the treatment of human disease

A peptide fragment of myelin basic protein was found to have encephalogenetic potential in humans, providing direct evidence of an autoimmune etiology for multiple sclerosis (NINDS)

New insights into how p53 protects health: the relation of the structural biology of p53 to its tumor suppressor effects elucidated by identification of specific phosphorylation sites (NIEHS, NCI)

Elucidation of the mechanisms of mitochondrial DNA disruptions by the various AIDS drugs to inform the development of safer, more effective antiviral medications (NIEHS)

Elucidation of role of p38 kinase signaling in cellular response to cytoxic agents suggests new molecular target for cancer (NCI, NIA)

Many genes differentially expressed in ovarian cancer are coordinately expressed, suggesting the presence of a small number of key pathways in ovarian tumorigenesis (NIA)

Mitochondria from human breast cancer cell lines found to be defective in the repair of one form of oxidative DNA damage, suggesting another factor in breast tumorigenesis (NIA)

Changes in X-chromosome structure, as well as interruption of specific genes, may lead to early menopause (NIA)

High levels of homocysteine, resulting from folic acid deficiency, found to promote dysfunction and degeneration of nerve cells in well-established mouse models of Alzheimer’s and Parkinson’s diseases (NIA)

Discovery that auditory pitch recognition differences are mostly genetic presages identifying the involved genes and how they function (NIDCD)

Wnt-7A signaling protein excess leads to disoriented bundling in embryonic ear and hearing loss (NIDCD)

Identification of induced cell surface channel on red blood cells infected with malaria parasites presents potential target for vaccine or drug (NICHD, NIAID)

b-Amyloid found to bind to mouse immune cell receptor and to induce free radicals, findings that may suggest new therapies to slow Alzheimer’s progress (NIAID, NCI)

b-Amyloid binding to a rat brain receptor blocks signals thought to be involved in learning and memory before the formation of b-amyloid plaques in Alzheimer’s (NIEHS)

CD21 identified on B-cell surface serves as a vehicle for HIV spread throughout body and to CD4 T cells (NIAID)

Removal of cholesterol from cells decreases HIV's ability to produce new virus particles and infect additional cells, suggesting a novel anti-HIV therapy (NIAID)

A single gene change allowed Yersinia pestis, the plague bacterium, to survive in the flea midgut and evolve from transmissibility via contaminated food or water to a flea-borne agent (NIAID)

Antibodies against the cellular receptor for IL-10 completely eliminated leishmania parasites in mice, suggesting a new way to prevent recurring infections in people (NIAID)

Mutations in genes encoding antibodies that enhance response to antigens found to be similar in young and old humans, suggesting that effective response to antigenic challenge persists well into old age (NIA, NCI)

Effect of normal and mutant MARCKS protein on cell adhesion suggests a role for MARCKS in the migration of brain neurons during development (NIEHS)

Discovery of new cytochrome p450 arachidonic acid hydroxylase (CYP2J9) in mouse brain sheds light on brain responses to environmental neurotoxins (NIEHS)

The human Cockayne syndrome protein found to be involved in DNA repair of oxidative damage (NIA)

Neuronal responses to visual stimuli in monkeys suggest process for memory formation (NIMH)

Prefrontal cortex activities in monkeys related to perception, integration of information, and translation into action shed light on brain processes underlying disturbed behavior in schizophrenia and mood disorders (NIMH)

fMRI reveals connectivity among different areas of the brain involved in different aspects of language processing (NIMH, NIDCD)

Learning a motor task requires consolidation of the learning in the motor cortex before subsequent distribution and storage in other cortical areas (NINDS)

Brain imaging identifies human brain region activated by anticipation of reward (NIAAA)

Discovery that marijuana activates the same reward pathways as cocaine and heroin and idenitification of the site of rewarding action of endomorphin-1, an endogenous m-opioid agonist, have implications for treatment of addiction (NIDA)

Blocking natural killer T lymphocytes and interleukin-13 turned off the suppression of immune system tumor surveillance in mice, presenting a promising approach to cancer immunotherapy and vaccine development (NCI)

Altered protein (histone H2AX) found to indicate double-stranded DNA breaks may be useful clinically in establishing optimal ionizing radiation dose and identifying patients with increased sensitivity to radiation and other chemically induced DNA disruptions (NCI, NIDDK)

New understanding regarding the way steroid receptors turn on genes that respond to hormones such as androgen and estrogen may help in developing receptor-targeting drugs (NCI)

Oxyradical-induced mutations in the p53 gene pinpointed early in the development of liver and colon cancer may enable prediction of a person’s risk for these cancers and early interventions to interrupt the cancer-forming process (NCI)

Two bone-related proteins expressed by many primary tumors found to enable tumor cell evasion of complement-mediated attack (NIDCR)

Dynamin-related protein 1, a mediator of mitochondrial fission, found to be required for apoptosis (NINDS)

Insight into the interdependent association of three oral bacteria that play a role in dental plaque formation may lead to preventive interventions (NIDCR)

Receptor differences in the white blood cells of Caucasians, African-Americans, and Asians may help explain differences in response to certain infections among peopleof different racial backgrounds (CC, NCI)

Different Helicobacter pylori strains, with strain-specific differences in genetic content shown by microarray, were found to induce varying degrees of inflammation, ulceration, and cancer susceptibility in a gerbil model (NIAID)

Restoration of vascular blood flow with inhaled nitric oxide has implications for the treatment of atherosclerosis and coronary artery disease (NHLBI, CC)

Aberrant transcriptional control in alveolar macrophages may be a contributing factor in the pathogenesis of pulmonary fibrosis (NHLBI)

Anti-TNF therapy in asthma alters the airway inflammatory response (NHLBI)

Data from an ongoing study of the natural history of lymphangioleiomyomatosis have established clinical, pathological, physiological, and genetic criteria that define disease severity and progression (NHLBI)

New pathway for the transport and removal of intracellular cholesterol by HDL and the specific plasma apolipoproteins in HDL that act as cholesterol acceptors have been identified in ABCA1 transgenic mouse studies (NHLBI)

Despite lower initial HIV viral loads among infected women, women and men were found to progress to AIDS at similar rates, suggesting that initial viral load is not predictive in women as it is in men and that treatment decisions need to be adjusted accordingly (NIAID)

X-ray crystallography determines structure of gd T-cell receptors, which may bind phosphoantigens released from pathogens causing tuberculosis, malaria, and other diseases—findings with implications for vaccine design (NIAID)

Variant of CX3CR1 gene found to protect against atherosclerosis and myocardial infarction (NHLBI, NIAID)

MRI shows structural changes in brains of psychotic, nonschizophrenic children that resemble those in schizophrenic children and differ from findings in normal children (NIMH)

Insight into the synchronous firing of brain cells in response to an object of attention (while ignoring less relevant features of a given visual scene) has implications for managing ADHD (NIMH)

Maintaining levels of the enzyme CYP2J2 and its products protects against the cell injury in the heart and blood vessels that occurs during angioplasty and other hypoxia-reoxygenation scenarios (NIEHS)

Molecular insights into inflammatory disorders: The roles of TNF receptor subtypes in the pathogenesis of tristetraprolin-induced TNF excess suggest that inhibiting the type I receptor or stimulating the type 2 receptor might be therapeutically useful in such conditions as rheumatoid arthritis and Crohn’s disease (NIEHS)

DNA replication errors generated by newly discovered DNA polymerase h found to resemble the hypermutation process in genes that code for immunoglobulin proteins and may shed light on how to enhance immune function (NIEHS, NLM)

Elucidation of how the alloimmune environment reshapes the immune response of the donor after stem cell transplantation and identification of innate T-cell responses to known and putative tumor-specific antigens informs immunotherapeutic strategies (NHLBI)

Elucidation of the metabolic underpinnings of insulin receptor impairment in type 2 diabetes should inform the development of new therapies (NIA)

Development of a theoretical analysis of how water is transported through membranous channels (NIDDK)

K-Opioid actions found to modulate internalization of the dopamine transporter, as well as cocaine effectiveness and cocaine sensitization (NIDA)

Self-administered heroin and amphetamine found to produce brain hyperthermia (NIDA)

Methamphetamine shown to have the potential to lower anti-apoptotic pathways in brain as well as increase pro-apoptotic pathways (NIDA)

Corticotrophin-releasing factor found to be involved in processes underlying stress-induced relapse to heroin and cocaine seeking (NIDA)

Leptin found to be involved in relapse to heroin seeking induced by acute food deprivation (NIDA)


Goal B: Develop new or improved instruments and technologies for use in research and medicine

Advances in imaging

Improved MRI with magnetization transfer contrast for managing cataracts and other lens changes (NINDS, NEI, NHLBI)

New imaging technique—diffusion tensor MRI—to follow stroke in progress (NICHD, CIT)

Transcranial magnetic stimulation to enhance understanding of fMRI of the brain (NINDS)

Development of manganese as a tracer in imaging neuronal activity and connectivity (NINDS)

"Virtual colonoscopy"—automated polyp detection using a computer algorithm to analyze CT scans of the abdomen and pelvis—shown to be feasible for real patients with real polyps (CC, CIT)

Development of a small animal PET scanner to visualize biochemical processes and track biochemical pathways in vivo (CC, CIT, SEIB)

Development of a PET ligand for central nervous system nicotinic cholinergic receptors (NIDA)

Advances in bioinformatics

New software to analyze tumor DNA changes (NCBI)

Improving the accuracy of PSI-BLAST protein database searches for sequence similarities (NLM)

Addition of new genomes and new functionalities to the COGs (Clusters of Orthologous Groups of proteins) database to enhance the classification of proteins from complete genomes (NLM)

New technique to gain insight into three-dimensional protein structure and to correlate structure and sequence information involves the new Conserved Domain Database in conjunction with several other web-based tools (NLM)

MAPS (MicroArray Project System) database to manage and interpret microarray gene expression data (NIEHS)

Developing tools for toxicogenomics: a DNA database for Xenopus laevis expressed sequence tags, a model to evaluate effects of environmental toxins on development (NIEHS)

Internet connectivity and communications network established throughout African sites of the Multilateral Initiative on Malaria (NLM)

Functional atlas of Caenorhabditis elegans, the first comprehensive catalog of protein interactions in a eukaryotic organism (NLM)

Computational research supports a role for "junk DNA" in genomic organization, cellular activities, and gene expression (NCBI)

Genomic analysis of radiation-resistant bacterium to elucidate DNA damage and repair mechanisms; sequence placed in GenBank (NLM)

Evolutionary analyses of two bacterial genomes deposited into GenBank—H. pylori and Chlamydia pneumoniae—provide insight into mechanisms of bacterial pathogenesis (NLM)

A collection of 15,000 sequence-verified and annotated mouse cDNAs, primarily derived from early mouse embryos, has been freely distributed to the research community (NIA)

Completion of the draft genome sequence of the mouse (within the Mouse Sequencing Consortium) and initiation of the collaborative program to sequence the genome of the laboratory rat lays the groundwork for cross-species comparisons of mice, rats, and humans (NHGRI)

Advances in biotechnology

Manipulations of cell-culture conditions induced the formation of pancreas-like cells from mouse embryonic stem cells that survived unchanged when injected into diabetic mice, a promising first step toward developing a treatment (NINDS, NIDDK, NIMH)

Adult mouse bone marrow cells injected into newborn mice migrate to the brain and form cells resembling nerve cells (NINDS)

New evidence of the existence of circulating skeletal stem cells (in adult mice, rabbits, guinea pigs, and humans) and their ability to form bone (NIDCR)

Stem cells isolated from pulp of adult human teeth and grown in lab generate a dentin structure in mice and may be basis for clinical tissue engineering to repair or replace teeth (NIDCR)

New system (MALDI/mass spectroscopy) to rapidly identify and characterize markers of genetic variations (single nucleotide polymorphisms) may hasten discovery of cancer genes (NCI)

Development of new technologies for prevention of nosocomial pneumonia and ventilator-induced injury may reduce patient morbidity and mortality in the intensive care unit (NHLBI)

New cell-culture process to generate large numbers of dendritic cells from cells circulating in the blood for use in cancer immunotherapy (NCI)

Rationale for waiting 28 days between donations established in studies pinpointing the effects on white blood cell donors of the cell collection process and agents used to augment the yield (CC)

Tool to measure a patient’s capacity to repair treatment-induced DNA double-strand breaks may aid in the selection of more successful treatment regimens (CC)

New technique of in vivo site-directed mutagenesis using oligonucleotides to assess large volume of genes rapidly (NIEHS)

Rapid functional analysis of mutations in p53 tumor suppressor gene using yeast as an in vivo test tube (NIEHS)

Use of a biologically active triple helix–forming oligonucleotide to generate gene knockout mice by a novel mechanism (NIA)


Goal C: Develop new or improved approaches for preventing or delaying the onset or progression of disease and disability

Elucidation of the molecular basis of alphavirus assembly to guide the design of agents to prevent viral entry and infection in such conditions as encephalitis and arthritis (NIAID)

Children with hereditary retinoblastoma found at risk for early-onset lung cancer and are candidates for aggressive antismoking efforts (NCI)

Estrogen replacement therapy found to attenuate systolic blood pressure rise in postmenopausal women (NIA, NHLBI)

First evidence found in rats and mice that dietary restriction increases levels of growth factors in brain and stimulates new nerve cell production, with implications for strategies to combat age-related neurodegenerative disorders (NIA)

An observational study of hormone use in postmenopausal women in the Baltimore Longitudinal Study of Aging revealed better memory performance in women receiving hormone therapy than in those who had never been treated (NIA)

Angiotensin converting enzyme inhibitor treatment was associated with a lower decline in muscle strength and walking speed in older hypertensive women (NIA)

Blockade of CB1 receptors found to block acute effects of marijuana in humans (NIDA)

Strategies to prevent kidney cancer suggested in finding that obesity and hypertension are related to increased renal cancer risk in men (NCI)

Newly discovered atherosclerosis-associated protein markers for glaucoma present intervention points to prevent disease progression (NEI)

Tests devised to identify genetic polymorphisms that affect Taxol metabolism and its toxicity in breast cancer patients set the stage to tailor taxol dose to each patient and avert its toxic effects (NIEHS)

In utero exposure to diabetes found to increase offspring’s risk of diabetes and obesity (NIDDK)

The association of visceral adiposity in healthy older adults with aortic stiffness suggests that keeping weight within normal limits and visceral fat low may slow vascular aging and reduce heart disease and stroke risk (NIA)

Silicone breast implants were not linked to increased risk for most cancers nor to increased mortality in a large and continuing epidemiologic study undertaken to address this public health concern (NCI)

Cell phone use was not found to increase the risk of brain tumors in an ongoing case-control study (NCI)

First national prevalence data on bullying among middle school and high school students identifies potential risk and protective factors (NICHD)

First national data on where children drown in the U.S. (by age, race, and sex) establish targets for prevention (NICHD)

Participation in the African-American Hereditary Prostate Cancer Study and the African-American Diabetes Mellitus Study should lead to discovery of genes involved in prostate cancer and susceptibility genes to type 2 diabetes (NHGRI)

Vaccine Development

Candidate vaccines against pediatric otitis media caused by two different pathogens found safe and immunogenic in early trials (NIDCD)

Clinical trial begins of AIDS vaccine encoding core HIV proteins (VRC)

Clinical trial begins of papilloma virus vaccine to prevent cervical cancer (NCI)

Conjugate vaccines against Salmonella typhi and Staphylococcus aureus performing well in early clinical trials (NICHD, NIDDK)

New DNA-based vaccine prevented Ebola virus infection in monkeys (VRC)

A hybrid vaccine formed by removing key genes from dengue virus and replacing them with WNV genes protected mice from West Nile virus infection (NIAID)

Live, attenuated dengue virus vaccine found to be safe and highly immunogenic in early clinical trials (NIAID)

A multiprotein DNA/MVA vaccine protected macaques from illness following mucosal challenge with an extremely pathogenic HIV/SIV virus (NIAID)

A DNA vaccine augmented with IL-2 prevented AIDS in monkeys (VRC)

A vaccine directed against a component of sand fly saliva prevented leishmaniasis in mice (NIAID)

Nasal administration of a respiratory syncytial virus (RSV)/human parainfluenza (HPIV) virus construct stimulated strong RSV- and HPIV-specific immune responses in rhesus monkeys (NIAID)

Transgenic animals can be engineered to secrete malaria proteins in their milk, offering a practical option for large-scale vaccine production in developing countries (NIAID)

Identification of group A Streptococcus (GAS) molecules produced by all human isolates of GAS examined from worldwide sources that may be suitable for human vaccine components (NIAID)

CMV pp65 protein canarypox construct prevents cytomegalovirus reactivation after stem cell transplantation (NHLBI)

Seroconversion studies of B19 parvovirus in sickle cell anemia patients presage a recombinant vaccine trial of baculovirus-engineered empty capsids (NHLBI)


Goal D: Develop new or improved methods for diagnosing disease and disability

Men with the lowest levels of prostate-specific antigen found to be unlikely to benefit from frequent PSA surveillance to detect early prostate cancer; for those whose PSA levels are between 2.0 and 4.0 ng/mL, a PSA velocity greater than 0.1 ng/mL/year was found to be associated with a 6.5-fold increased risk for developing prostate cancer (NIA)

Clinical proteomics initiative combining laser capture microdissection, protein microarrays, and proteomic signatures enhances the diagnosis (and treatment) of an array of cancers (NCI)

Diagnostic approaches based on immunological detection of the Pseudomonas aeruginosa type III secretory apparatus and its associated cytotoxins provide evidence for early colonization and/or infection in children with cystic fibrosis (NHLBI)

Combining three measures (diffusion-weighted MRI of brain, NIH Stroke Scalescore, and time from symptom onset to brain scan) enables early prediction (and tailored management) of stroke recovery (NINDS)

Only one of the brain differences found in boys with ADHD, compared with unaffected boys, has also been found in girls with ADHD (NIMH)

New triple-lipid screening test measures total and HDL cholesterol and triglycerides all at once (CC)

A multicopy gene-based nucleic acid detection system in oral gargles to obviate need for bronchoscopy to diagnose Pneumocystis carinii pneumonia (NIAID, CC)

Gene expression patterns

Identification of genes expressed differently in normal ovary and ovarian cancer cells promises far earlier detection of ovarian cancer than currently possible (NIA)

DNA microarrays combined with artificial neural network analysis yield highly accurate differential diagnoses of four complex childhood cancers—neuroblastomas, rhabdomyosarcoma, non-Hodgkin’s lymphoma, and Ewing tumors (NHGRI)

DNA microarrays yield gene expression profiles that distinguish hereditary (BRCA1 and 2) from sporadic breast cancers, with implications for treatment (NHGRI)

Two distinct subtypes of diffuse large B-cell lymphomas (with different prognoses) identified by gene expression profiling (NCI)

(Note: Diagnostic advances are also inherent in the isolation of the disease genes listed in "Goal A.")


Goal E: Develop new or improved approaches for treating disease and disability

Recombinant immunotoxin BL22 induced complete remissions in majority of chemotherapy-resistant hairy cell leukemia patients and shows promise in the treatment of more common leukemias as well in a Phase I study not anticipated to demonstrate efficacy (NCI)

Successful transplantation of pancreatic islet cells to treat type 1 diabetes (NIDDK)

Phosphocysteamine shows promise in treating an otherwise fatal genetic disorder (infantile neuronal ceroid lipofuscinosis) and, because it can cross the placenta and enter the brain, has potential also as a prenatal treatment (NICHD)

New, less-toxic stem-cell transplantation regimen eliminates the need for radiation in patients with chronic granulomatous disease (NIAID, CC)

New protein, thymosin b4, found to promote corneal wound healing in experimental model (NIDCR)

Minimally invasive, image-guided tumor ablation with radiofrequency current established as a less damaging alternative to open surgery in patients with kidney tumors (NCI, NINDS, CC)

St. John’s wort does not interfere with anti-epileptic drug pharmacokinetics in healthy volunteers and should not compromise treatment in patients with seizure disorder (NINDS, CC)

Rhodopsin effect of isotretinoin in causing night blindness in rats suggests treatment strategy against retinal and macular degeneration (NEI)

Continuing identification of genes encoding cancer antigens in melanoma, breast, ovarian, prostate, and lung cancers contribute to expanding immunotherapeutic anticancer strategies (NCI)

Primate findings that testosterone protects against the proliferative effects of estrogen on breast tissue were put to the test in the first controlled study combining testosterone with estrogen replacement in young women with ovarian failure (NICHD)

High rate of complete remission and voice and speech preservation in patients with head and neck cancer achieved in a Phase I study of Taxol and radiation (NCI, NIDCD)

Clinical trial launched of PS-341, an agent that blocks activation of a pathway needed by head and neck cancer cells to grow and form blood vessels (NIDCD, NCI)

Anthrax toxin modified to selectively kill tumor cells that express plasminogen activator, an enzyme overproduced in a wide array of human tumors, may be effective in preventing cancer invasion and metastasis (NIDCR)

Gene therapy to produce heme oxygenase-1 protects against vascular constriction and excessive growth following injury and may be useful in treating vascular diseases, preventing restenosis postangioplasty, and reducing vasoconstriction during stroke (NHLBI, VRC)

Genotype and phenotype resistance testing may help select the most appropriate highly active antiretroviral therapy drug combination in drug-experienced patients (NIAID, CC)

Enzyme replacement therapy shown to be effective for patients with the hereditary metabolic disorder Fabry's disease (NINDS)

High-dose intravenous immunoglobulin shown to ameliorate Stiff-Person Syndrome (NINDS)

Endogenous marijuana-like substances shown to mediate the vasodilated state that complicates advanced liver cirrhosis, and a cannabinoid receptor antagonist looks promising in reversing this condition (NIAAA)

Improved safety and efficacy of allogeneic stem cell transplantation achieved in the treatment of hematological and nonhematological malignant disease (NHLBI)

Clinical trial of immunosuppression in severe aplastic anemia demonstrates that early robust improvement in blood counts predicts long-term survival without malignant evolution (NHLBI)

Overexpression of the ABCA1 transporter in mice found to markedly decrease diet-induced atherosclerosis, indicating that drugs that upregulate the expression of the ABCA1 transporter may be a unique new approach to treating cardiovascular disease in humans (NHLBI)

Classes of drugs (tropanes, GBR12909) found to bind to the "cocaine site" on the dopamine transporter without interfering with dopamine uptake—and having no abuse potential themselves—offer leads to treatment of cocaine addiction (NIDA)



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