New chemical probe provides tool to investigate role of malignant brain tumor domains in chromatin structure and regulation

Researchers lead by assistant professor in the lab of Stephen Frye, Fred Eshelman Distinguished Professor in the UNC School of Pharmacy and member of the UNC Lineberger Comprehensive Cancer Center, announced the discovery of a chemical probe that can be used to investigate the L3MBTL3 methyl-lysine reader domain. The probe, named UNC1215, will provide researchers with a powerful tool to investigate the function of malignant brain tumor (MBT) domain proteins in biology and disease.....

New chemical probe provides tool to investigate role of malignant brain tumor domains in chromatin structure and regulation

High-Fiber Diet Helps Heart Too, Expert Says - Drugs.com MedNews

Eating a high-fiber diet does more than promote digestive well-being; it's also good for your heart, an expert says.

 

Dietary fiber from fruits, vegetables, whole grains and beans "has been shown in research to help lower cholesterol," Jody Gilchrist, a nurse practitioner at the Heart and Vascular Clinic at the University of Alabama, Birmingham.

"Most nutrition experts say that a person needs at least 25 grams of fiber a day as part of a balanced diet," Gilchrist said. "The American Heart Association recommends that a good rule of thumb is 14 grams of fiber for every 1,000 calories consumed, and at least 10 grams should come from soluble fiber."

Soluble fiber makes you feel full quickly, which helps control how much you eat. Research has shown that soluble fiber also helps lower "bad" LDL cholesterol by interfering with how the body absorbs cholesterol from foods.

High-Fiber Diet Helps Heart Too, Expert Says - Drugs.com MedNews

Pernix Therapeutics Receives FDA Approval for Vituz

Pernix Therapeutics Receives FDA Approval for Vituz

FDA Approves Stivarga for Advanced Gastrointestinal Stromal Tumors

In continuation of my update on  Regorafenib (BAY 73-4506)

FDA Approves Stivarga for Advanced Gastrointestinal Stromal Tumors

Positive health indicators associated with avocado consumption

We know that avocado (Persea americana) is a tree native to Central Mexico, classified in the flowering plant family Lauraceae along with cinnamon, camphor and bay laurel. Avocado or alligator pear also refers to the fruit (botanically a large berry that contains a single seed) of the tree.

Avocados are commercially valuable and are cultivated in tropical and Mediterranean climates throughout the world. They have a green-skinned, fleshy body that may be pear-shaped, egg-shaped, or spherical. Commercially, it ripens after harvesting. Trees are partially self-pollinating and often are propagated through grafting to maintain a predictable quality and quantity of the fruit.

The fruit of horticultural cultivars has a markedly higher fat content than most other fruit, mostly monounsaturated fat, and as such serves as an important staple in the diet of various groups where access to other fatty foods (high-fat meats and fish, dairy products, etc.) is limited.
A ripe avocado yields to gentle pressure when held in the palm of the hand and squeezed. The flesh is prone to enzymatic browning; it turns brown quickly after exposure to air. To prevent this, lime or lemon juice can be added to avocados after they are peeled.

Positive health indicators associated with avocado consumption

Tranylcypromine may also hold promise in treating sickle cell disease

In continuation of my update on Tranylcypromine

An antidepressant drug used since the 1960s may also hold promise for treating sickle cell disease, according to a surprising new finding made in mice and human red blood cells by a team from the University of Michigan Medical School.

The discovery that tranylcypromine, or TCP, can essentially reverse the effects of sickle cell disease was made by U-M scientists who have spent more than three decades studying the basic biology of the condition, with funding from the National Institutes of Health.

Tranylcypromine may also hold promise in treating sickle cell disease

Unequivocal evidence that omega-3 fatty acids reduce breast cancer risk

In continuation of my update on omega-3-fatty-acids

Unequivocal evidence that omega-3 fatty acids reduce breast cancer risk

TIR-199 shows promise in fighting renal cancer

TIR-199 shows promise in fighting renal cancer

Ref : http://ucrtoday.ucr.edu/12020

Meloxicam recognizes and treats osteoarthritis in cats

Meloxicam is a nonsteroidal anti-inflammatory drug (NSAID) with analgesic and fever reducer effects. It is a derivative of oxicam, closely related to piroxicam, and falls in the enolic acid group of NSAIDs. It was developed by Boehringer-Ingelheim.

Meloxicam recognizes and treats osteoarthritis in cats

Resistant starch kills pre-cancerous cells and reduces inflammation

Resistant starch (RS) is starch and starch degradation products that escape digestion in the small intestine of healthy individuals. Resistant starch is considered the third type of dietary fiber, as it can deliver some of the benefits of insoluble fiber and some of the benefits of soluble fiber.

Some carbohydrates, such as sugars and most starch, are rapidly digested and absorbed as glucose into the body through the small intestine and subsequently used for short-term energy needs or stored. Resistant starch, on the other hand, resists digestion and passes through to the large intestine where it acts like dietary fiber.

More...

Resistant starch kills pre-cancerous cells and reduces inflammation

Study describes pharmacological action of TZDs directly on pancreas

Study describes pharmacological action of TZDs directly on pancreas

New Antibiotic May Treat Skin Infections in Shorter Time - Drugs.com MedNews

Torezolid (also known as TR-701 and now tedizoli) is an oxazolidinone drug being developed by Trius Therapeutics(originator Dong-A Pharmaceuticals) for complicated skin and skin-structure infections (cSSSI), including those caused by Methicillin-resistant Staphylococcus aureus (MRSA). 

New Antibiotic May Treat Skin Infections in Shorter Time - Drugs.com MedNews

Common chemicals linked to osteoarthritis

Common chemicals linked to osteoarthritis

Study describes pharmacological action of TZDs directly on pancreas

Study describes pharmacological action of TZDs directly on pancreas

Copper can protect against Alzheimer's disease

Copper can protect against Alzheimer's disease

Breakthrough in ovarian cancer: Selumetinib

In continuation  of my update on Seumetinab

We know that, Selumetinib (AZD6244) is a drug being investigated for the treatment of various types of cancer, for example non-small cell lung cancer (NSCLC). 

Mode of action : The gene BRAF is part of the MAPK/ERK pathway, a chain of proteins in cells that communicates input from growth factors. Activating mutations in the BRAF gene, primarily V600E (meaning that the amino acid valine in position 600 is replaced by glutamic acid), are associated with lower survival rates in patients with papillary thyroid cancer. Another type of mutation that leads to undue activation of this pathway occurs in the gene KRAS and is found in NSCLC. A possibility of reducing the activity of the MAPK/ERK pathway is to block the enzyme MAPK kinase (MEK), immediately downstream of BRAF, with the drug selumetinib. More specifically, selumetinib blocks the subtypes MEK1 and MEK2 of this enzyme....

The study was initially developed in 2007, with 52 patients enrolled for the Phase II clinical trial between December 2007 and November 2009. Patients were given 50 milligrams of selumetinib orally twice daily. Of those participants, eight had a measurable decrease in tumor size, seven had partial responses and 34 patients saw their tumors stabilize. The findings suggest that inhibitors of the MAPK pathway warrant further investigation in patients with low-grade ovarian cancer.

"There just aren't very good treatments for low-grade ovarian cancer, so this discovery opens up a lot of new exciting possibilities for us," Dr. Farley said. He added that Phase III of this trial is scheduled to begin in the next few weeks, with that trial to be the "definitive test" before the treatment becomes available to the general population.

Ref : http://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(12)70572-7/fulltext



Breakthrough in ovarian cancer: Selumetinib

Avastin May Help Boost Survival With Aggressive Cervical Cancer: Study - Drugs.com MedNews

In continuation of my update on Avastin
Avastin May Help Boost Survival With Aggressive Cervical Cancer: Study - Drugs.com MedNews

Nitric oxide: A little molecule's remarkable feat -- prolonging life, worm study shows

Nitric oxide: A little molecule's remarkable feat  prolonging life, worm study shows

FDA Approves Pomalyst for Advanced Multiple Myeloma

We know that, Pomalidomide (INN, originally CC-4047 or 3-amino-thalidomide, marketed as Pomalyst by Celgene), is a derivative of thalidomide that is anti-angiogenic and also acts as an immunomodulator. Pomalidomide was approved on February 8, 2013 by the U.S. Food and Drug Administration (FDA) as a treatment for relapsed and refractory multiple myeloma....

FDA Approves Pomalyst for Advanced Multiple Myeloma

FDA Approves Ravicti for the Chronic Management of Some Urea Cycle Disorders

The U.S. Food and Drug Administration today approved Ravicti (glycerol phenylbutyrate) for the chronic management of some urea cycle disorders (UCDs) in patients ages 2 years and older.

UCDs are genetic disorders that involve deficiencies of specific enzymes involved in the urea cycle, a series of biochemical steps normally required to remove ammonia from the blood. When protein is absorbed and broken down by the body, it produces nitrogen as a waste product. The urea cycle removes nitrogen from the blood and converts it to urea, which is removed from the body through urine. In people with UCDs, nitrogen accumulates and remains in the body as ammonia, which can travel to the brain and cause brain damage, coma or death.

FDA Approves Ravicti for the Chronic Management of Some Urea Cycle Disorders

Diabetes Drug Byetta May Offer 'Modest' Weight Loss for Very Obese Teens: Study - Drugs.com MedNews

In continuation of my update Exenatide

Diabetes Drug Byetta May Offer 'Modest' Weight Loss for Very Obese Teens: Study - Drugs.com MedNews

'Clot-Buster' Drug May Still Be Best Stroke Treatment - Drugs.com MedNews

'Clot-Buster' Drug May Still Be Best Stroke Treatment - Drugs.com MedNews

Top-line results from Vanda's tasimelteon Phase IIb/III study on major depressive disorder

We know that, Tasimelteon (BMS-214,778) is a drug which is under development for the treatment of insomnia and other sleep disorders. It is a selective agonist for the melatonin receptors MT₁ and MT₂ in the suprachiasmatic nucleus of the brain, similar to older drugs such as ramelteon. It has been through Phase III trials successfully and was shown to improve both onset and maintenance of sleep, with few side effects.

A year-long (2011-2012) study at Harvard is testing the use of tasimelteon in blind subjects with non-24-hour sleep-wake disorder.

Now Vanda Pharmaceuticals Inc. (NASDAQ: VNDA),  announced top-line results of the Phase IIb/III clinical study (MAGELLAN) in Major Depressive Disorder (MDD), investigating the efficacy and safety of tasimelteon as a monotherapy in the treatment of patients with MDD.  The clinical study did not meet the primary endpoint of change from baseline in the Hamilton Depression Scale (HAMD-17) after 8 weeks of treatment as compared to placebo.  Both tasimelteon and placebo treated patients had an approximately 40% reduction of their MDD symptoms from baseline.  Tasimelteon was shown to be safe and well-tolerated, consistent with observations in prior studies.  Given these current proof of concept clinical study results, Vanda has decided to discontinue all activities in this indication.

"These results are disappointing, as there is still a significant unmet medical need for patients with Major Depression," said Mihael H. Polymeropoulos , M.D., President and CEO of Vanda.  "Tasimelteon's application in the treatment of blind individuals with Non-24 remains our top priority as we pursue our planned NDA submission this year."     

Vanda has recently reported positive results in two phase III clinical studies of tasimelteon in Non-24-Hour Disorder (Non-24) and plans to submit a New Drug Application to the U.S. Food and Drug Administration in mid-2013......

Top-line results from Vanda's tasimelteon Phase IIb/III study on major depressive disorder

Hops, Key to Flavor in Beer, May Prove Useful in New Drugs - Drugs.com MedNews

We know that, Humulone (α-lupulic acid) is a bitter-tasting chemical compound found in the resin of mature hops (Humulus lupulus). Humulone is a prevalent member of the class of compounds known as alpha acids, which collectively give beer its characteristic bitter flavor.

In a  new study, researchers determined the precise configuration of humulones, substances derived from hops that give beer its unique flavor.

"Now that we have the right results, what happens to the bitter hops in the beer-brewing process makes a lot more sense," study lead author Werner Kaminsky, a University of Washingto.

Previous research has suggested that moderate consumption of beer  and therefore its bitter compounds might have positive effects on diseases such as diabetes and some types of cancer, as well as aiding weight loss and decreasing inflammation. 

The new findings could help scientists determine which humulones might prove useful in efforts to develop new drugs, Kaminsky said.

The authors wrote in their report that while "excessive beer consumption cannot be recommended to propagate good health  isolated humulones and their derivatives can be prescribed with documented health benefits."

Ref : http://onlinelibrary.wiley.com/doi/10.1002/anie.201208450/abstract

  

Hops, Key to Flavor in Beer, May Prove Useful in New Drugs - Drugs.com MedNews

FDA Approves New Orphan Drug Kynamro (mipomersen ) to Treat Inherited Cholesterol Disorder

In continuation of my update on mipomersen

FDA Approves New Orphan Drug Kynamro to Treat Inherited Cholesterol Disorder

Messenger RNA–Based Vaccines for Cancer | Articles | Drug Discovery and Development Magazine

Nucleic acids are being extensively investigated for use in gene therapy and in genetic vaccinations in which foreign nucleic acid is translated into proteins by the host cells. Vaccines based on DNA and messenger RNA (mRNA) are able to stimulate all effectors of the adaptive immune response: B lymphocytes, cytotoxic T cells, and T helper cells. This makes them a useful tool in the creation of prophylactic vaccines for infectious diseases and for cancer immunotherapy.

Messenger RNA–Based Vaccines for Cancer | Articles | Drug Discovery and Development Magazine

Phenformin decreases size of lung tumors and increases survival in mice

In continuation of my update on metformin and phenformin
In a new study in the journal Cancer Cell, Shaw and a team of scientists at the Salk Institute for Biological Studies found that phenformin, a derivative of the widely-used diabetes drug metformin, decreased the size of lung tumors in mice and increased the animals' survival. The findings may give hope to the nearly 30 percent of patients with non-small cell lung cancer (NSCLC) whose tumors lack LKB1 (also called STK11).

The LKB1 gene turns on a metabolic enzyme called AMPK when energy levels of ATP, molecules that store the energy we need for just about everything we do, run low in cells. In a previous study, Shaw, an associate professor in Salk's Molecular and Cell Biology Laboratory and researcher in the Institute's new Helmsley Center for Genomic Medicine, demonstrated that cells lacking a normal copy of the LKB1 gene fail to activate AMPK in response to low energy levels. LKB1-dependent activation of AMPK serves as a low-energy checkpoint in the cell. Cells that lack LKB1 are unable to sense such metabolic stress and initiate the process to restore their ATP levels following a metabolic change. As a result, these LKB1-mutant cells run out of cellular energy and undergo apoptosis, or programmed cell death, whereas cells with intact LKB1 are alerted to the crisis and re-correct their metabolism.

"The driving idea behind the research is knowing that AMPK serves as a sensor for low energy loss in cells and that LKB1-deficient cells lack the ability to activate AMPK and sense energy loss," says David Shackelford, a postdoctoral researcher at Salk who spearheaded the study in Shaw's lab and is now an assistant professor at UCLA's David Geffen School of Medicine.

That led Shaw and his team to a class of drugs called biguanides, which lower cellular energy levels by attacking the power stations of the cell, called mitochondria. Metformin and phenformin both inhibit mitochondria; however, phenformin is nearly 50 times as potent as metformin. In the study, the researchers tested phenformin as a chemotherapy agent in genetically-engineered mice lacking LKB1 and which had advanced stage lung tumors. After three weeks of treatment, Shaw and his team saw a modest reduction in tumor burden in the mice.

Ref : https://www.cell.com/cancer-cell/abstract/S1535-6108%2812%2900518-1

Phenformin decreases size of lung tumors and increases survival in mice

Can Brightly Colored Fruits, Veggies Protect Against ALS? - Drugs.com MedNews

Researchers from Harvard School of Public Health have,  found that increasing consumption of carotenoids, particularly beta-carotene and lutein, might reduce the risk for this progressive neurological disease, which attacks nerve cells in the brain and spinal cord.

Carrots, yams and mangoes are rich in beta-carotenes, and spinach, collard greens and egg yolks are good sources of lutein. The study found, however, that diets rich in the antioxidants lycopene, beta-cryptoxanthin and vitamin C do not apparently reduce the risk for ALS, which causes the muscles to waste away and eventually results in paralysis.

"ALS is a devastating degenerative disease that generally develops between the ages of 40 and 70, and affects more men than women," senior study author Dr. Alberto Ascherio, a professor of epidemiology and nutrition at Harvard School of Public Health, said in a journal news release. "Understanding the impact of food consumption on ALS development is important."

Analyzing information on more than 1 million people, the researchers identified nearly 1,100 cases of ALS. The researchers found that increased overall carotenoid intake -- especially among those who ate diets rich in beta-carotene and lutein -- seemed to be linked to a lower risk for the devastating condition. 

Those who ate more carotenoids daily also were more likely to exercise, have an advanced degree, have increased vitamin C intake and take vitamin C and E supplements.

The researchers pointed out, however, that long-term vitamin C supplements did not lower people's risk for this degenerative disease.

"Our findings suggest that consuming carotenoid-rich foods may help prevent or delay the onset of ALS," Ascherio concluded. "Further food-based analyses are needed to examine the impact of dietary nutrients on ALS."

 Ref : http://onlinelibrary.wiley.com/doi/10.1002/ana.23820/abstract

Can Brightly Colored Fruits, Veggies Protect Against ALS? - Drugs.com MedNews

Meta-analysis links increased magnesium intake with fasting glucose and insulin reductions...

"Evidence from cross-sectional and longitudinal observational studies suggests that diets higher in magnesium are associated with reduced risk of insulin resistance and type 2 diabetes, whereas in intervention studies, supplemental magnesium improves measures of glucose and insulin metabolism in generally healthy adults, as well as in those with insulin resistance and type 2 diabetes," Adela Hruby and colleagues write. "However, little is known about potential interaction between magnesium intake and genetic variability on glycemic traits, in which genetic variants related to either magnesium transport and homeostasis or glucose and insulin metabolism may modify the pathways through which magnesium exerts its effects."

The researchers analyzed data from up to 52,684 nondiabetic men and women who participated in 15 studies included in the Cohorts for Heart and Aging Research in Genomic Epidemiology (CHARGE) Consortium. Dietary questionnaire or interview responses, or food diary entries were analyzed for magnesium content from food and beverages. Participants were genotyped for up to 25 single nucleotide polymorphisms related to fasting glucose, insulin or magnesium.  Average magnesium intake ranged from 224.7 milligrams to 479.7 milligrams per day. Reductions in both fasting glucose and fasting insulin were observed in association with increased magnesium. While a nominal association was found between one of the genetic variants examined in this study and fasting glucose and two variants showed nominal interactions with magnesium intake on fasting glucose and fasting insulin, no significant effects for the variations were observed......... 

Ref : http://jn.nutrition.org/content/early/2013/01/22/jn.112.172049.abstract?sid=0219d7a7-cc12-4709-8167-805f0663e750

More..

HSS involving resiniferatoxin receives U.S. patent to alleviate intractable pain

We know that, Resiniferatoxin (RTX) is a naturally occurring, ultrapotent capsaicin analog that activates the vanilloid receptor in a subpopulation of primary afferent sensory neurons involved in nociception (the transmission of physiological pain). RTX causes an ion channel in the plasma membrane of sensory neurons — the transient receptor potential vanilloid 1 — to become permeable to cations, most particularly the calcium cation; this evokes a powerful irritant effect followed by desensitization and analgesia. Research is being conducted at the National Institutes of Health and the University of Pennsylvania to design a novel class of analgesics from the latex of resin spurge (Euphorbia resinifera), a cactus-like plant commonly found in Morocco that contains high concentrations of RTX. Resiniferatoxin has a rating of 16,000,000,000 on the Scoville Scale making it one of the highest rated substances known.

Now U. S. Patent and Trademark Office recently issued a patent to the U. S. Department of Health and Human Services involving resiniferatoxin, or RTX, an experimental compound that represents a potential new class of drugs to alleviate the intractable pain that can occur in people with advanced cancer, severe arthritis, and other extremely chronic conditions.

HSS involving resiniferatoxin receives U.S. patent to alleviate intractable pain

Sanofi Canada announces new option for emergency treatment of anaphylaxis

We know that, Epinephrine (also known as adrenaline/adrenalin) is a hormone and a neurotransmitter. Epinephrine has many functions in the body, regulating heart rate, blood vessel and air passage diameters, and metabolic shifts; epinephrine release is a crucial component of the fight-or-flight response of the sympathetic nervous system. In chemical terms, epinephrine is one of a group of monoamines called the catecholamines. It is produced in some neurons of the central nervous system, and in the chromaffin cells of the adrenal medulla from the amino acids phenylalanine and tyrosine.

Now, Sanofi Canada announces a new option for the emergency treatment of anaphylaxis. Allerject™ is the first and only 'talking' epinephrine auto-injector in Canada. 

 

Sanofi Canada announces new option for emergency treatment of anaphylaxis

Intravenous iron dextran effective for restless leg syndrome

Intravenous low molecular weight iron dextran appears to provide effective long-lasting treatment for some patients with restless leg syndrome (RLS), even for those with normal serum ferritin levels, research indicates.

The results showed that 68% of 25 patients with RLS showed moderate or complete improvement of all RLS symptoms after treatment according to a Korean-translated version of the International RLS Severity (K-IRLS) scale and the PAM-RL device, which records periodic leg movements.

However, as reported in Sleep Medicine, the researchers saw no correlation between increases in cerebrospinal fluid (CSF) ferritin in response to therapy and clinical improvements.

"Although a relatively accessible measure, CSF ferritin is at best a crude measure of CNS [central nervous system] iron status and not necessarily reflective of regionally specific changes in iron status that are assumed to account for the symptom development," say Yong Won Cho, from Keimyung University School of Medicine in Daegu, South Korea, and colleagues.

"In addition, the narrow range of symptom severity in this study may have limited our ability to find any differences between CSF ferritin and subjective measures of the disease."

Intravenous iron dextran effective for restless leg syndrome

Everolimus treatment option for tuberous sclerosis patients

In continuation of my update on Everolimus

Everolimus treatment option for tuberous sclerosis patients

FDA Approves Gleevec for Children with Acute Lymphoblastic Leukemia

In continuation of my update on Gleevec

FDA Approves Gleevec for Children with Acute Lymphoblastic Leukemia

Exjade Approved for Inherited Blood Disorder - Drugs.com MedNews

We know that, Deferasirox (marketed as Exjade) is a rationally-designed oral iron chelator. Its main use is to reduce chronic iron overload in patients who are receiving long-term blood transfusions for conditions such as beta-thalassemia and other chronic anemias. It is the first oral medication approved in the USA for this purpose.

It was approved by the United States Food and Drug Administration (FDA) in November 2005. According to FDA (May 2007), renal failure and cytopenias have been reported in patients receiving deferasirox oral suspension tablets. It is approved in the European Union by the European Medicines Agency (EMA) for children 6 years and older for chronic iron overload from repeated blood transfusions.

Now  Exjade (deferasirox) has been approved by the U.S. Food and Drug Administration to remove excess iron in the blood among people with a genetic blood disorder called non-transfusion-dependent thalassemia (NTDT). Too much iron in the blood can damage vital organs, the agency said Wednesday in a news release. 

Thalassemia typically leads to the production of fewer red blood cells and less hemoglobin, a protein that carries oxygen throughout the body. NTDT is a milder form of thalassemia that unlike other forms, does not require frequent blood transfusions. Thalassemia affects about 1,000 people in the United States, the FDA said............

Exjade Approved for Inherited Blood Disorder - Drugs.com MedNews

NIH clinical trial begins for treatment of rare, fatal neurological disorder, January 23, 2013 News Release - National Institutes of Health (NIH)

 We know that, Cyclodextrins (sometimes called cycloamyloses) are a family of compounds made up of sugar molecules bound together in a ring (cyclic oligosaccharides). Cyclodextrins are produced from starch by means of enzymatic conversion. They are used in food, pharmaceutical,drug delivery, and chemical industries, as well as agriculture and environmental engineering. Hydroxypropyl Beta Cyclodextrin (HPβCD) is the chief active compound found in Procter and Gamble's deodorizing product "Febreze" under the brand name "Clenzaire".Cyclodextrins are composed of 5 or more α-D-glucopyranoside units linked 1->4, as in amylose (a fragment of starch). The 5-membered macrocycle is not natural. Recently, the largest well-characterized cyclodextrin contains 32 1,4-anhydroglucopyranoside units, while as a poorly characterized mixture, at least 150-membered cyclic oligosaccharides are also known. Typical cyclodextrins contain a number of glucose monomers ranging from six to eight units in a ring, creating a cone shape.

A clinical trial to evaluate a drug candidate called cyclodextrin as a possible treatment for Niemann-Pick disease type C1 (NPC), a rare and fatal genetic disease has already started on 23rd Jan, 2012. Scientists from the NIH’s National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) will conduct the clinical trial at the NIH Clinical Center. Reaching this trial stage required collaboration among government, industry, patient advocacy groups and academic researchers.

NIH clinical trial begins for treatment of rare, fatal neurological disorder, January 23, 2013 News Release - National Institutes of Health (NIH)