"The HIll" GOP lawmakers: Spare NIH from cuts

GOP lawmakers: Spare NIH from cuts

By Ian Swanson 

05/27/11

A group of Republican lawmakers has asked appropriators to avoid making deep cuts to the budget of the National Institutes of Health.

The request that NIH be spared highlights the difficult choices facing the GOP as it moves from writing a budget to wringing out cuts from 13 different appropriations measures.

Casting a vote to reduce the nation’s deficit is easy. Cutting programs that are seeking a cure for cancer is more difficult.

“It’s very tough,” said Rep. Brian Bilbray (R-Calif.), one of 12 Republicans pressing for NIH funding to be kept up. 

NIH took a $321 million cut in the 2011 budget process, and health advocates believe it could face a much bigger reduction as lawmakers seek $18 billion in cuts to a spending measure for the departments of Health and Human Services, Labor and Education.

“They’ll have trouble getting the cuts anywhere else,” Atul Grover, chief advocacy officer for the Association of American Medical Colleges, told The Hill this week.

The cuts will feel more severe at NIH after the agency benefited from the 2009 stimulus bill. Those remaining funds will run out at the end of the year.

Given the 40 percent cuts being proposed to discretionary domestic spending programs, Bilbray said a call to keep NIH funding stable or to even increase it means other programs must take bigger hits. 

Faced with a choice between NIH and public broadcasting, Bilbray said, “I’m sorry Big Bird. I’m going with NIH.”

Republicans are already starting to feel the heat as budget talks wade into specifics.

The GOP moved its first two spending measures through committee on Tuesday, chopping funding for the Federal Emergency Management Agency (FEMA) and firefighter training and assistance grants, among other things.

Democrats accused the GOP of cutting disaster aid. Republicans said they were cutting grants that communities get before a disaster, and that FEMA had done a poor job of prioritizing funds.

The fight over Rep. Paul Ryan’s (R-Wis.) budget and its proposed reforms of Medicare is the main event, but Democrats are looking to pounce on Republican cuts to other programs as the appropriations process unfolds.

Republicans supporting the NIH say it represents the nation’s best hope for “finding cures, improving treatment and gaining a better understanding of the diseases and conditions that affect millions of Americans.”

“We urge you to consider the wide-ranging, positive effects of investment in research on health and the economy. We appreciate your consideration of our request,” Bilbray and 11 other Republicans wrote to Rep. Denny Rehberg (R-Mont.), the chairman of the subcommittee that handled NIH’s budget.

Besides Bilbfray, the letter was signed by Reps. Dave Reichert (Wash.), Jim Gerlach (Pa.), Todd Platts (Pa.), Tom Petri (Wis.), Steve Stivers (Ohio), Aaron Schock (Ill.), Judy Biggert (Ill.), Bill Johnson (Ohio), Pete King (N.Y.), Elton Gallegly (Calif.) and Nan Hayworth (N.Y.), along with Democratic Rep. Gerry Connolly (Va.). 

The NIH also has friends in the Senate. It was set to take a $1.6 billion cut in funding for 2011 before Senate negotiators winnowed that down to $320 million.

Winning further reprieves won’t be easy, given the deficit-cutting mood in Washington. Prominent supporters of the NIH such as Sen. Arlen Specter (D-Pa.) are no longer in Congress. And with the rise of the Tea Party, Sen. 

Orrin Hatch (R-Utah) and other GOP backers must worry about being seen as soft on fiscal issues.

“I want to thank you, as members of the Senate, for sparing NIH from deeper cuts in the final fiscal year 2011 continuing resolution,” NIH Director Francis Collins said in testimony earlier this month.

NIH: The Director’s NEW INNOVATOR Awards Program

New Innovator Awards Program:

• Up to $1.5 million in direct costs over 5 years
• For early career stage investigators (ESI), defined as those who have not received an NIH R01 or similar grant and are within 10 years of completing their terminal research degree or medical residency.

The deadline for submitting Award applications is October 14, 2011

See the instructions in the Funding Opportunity Announcement RFA-RM-11-005 (http://grants.nih.gov/grants/guide/rfa-files/RFA-RM-11-005.html) andhttp://commonfund.nih.gov/newinnovator for more information.

Send questions to newinnovator@nih.gov<mailto:newinnovator@nih.gov>.

NIH: The Director’s Pioneer Awards Program

The Director’s Pioneer Awards Program:

NIH welcomes proposals for 2012 NIH Director’s Pioneer Awards for innovative approaches to major challenges inbiomedical or behavioral research.

NIH expects to make at least 7 Pioneer Awards in summer 2012.  To continue its strong record of diversityin these programs, NIH especially encourages women and members of groups that are underrepresented in NIH research to apply.

Pioneer Awards:

• Up to $2.5 million in direct costs over 5 years
• Open to scientists at any career stage.

The deadline for submitting Pioneer Award applications is October 7, 2011.

See the instructions in the Funding Opportunity Announcement RFA-RM-11-004 (http://grants.nih.gov/grants/guide/rfa-files/RFA-RM-11-004.html) andhttp://commonfund.nih.gov/pioneer for more information. Send questions to pioneer@nih.gov<mailto:pioneer@nih.gov>.

The NIH wants to hear from you! NIH Common Fund Program

NIH Wants To Hear from You!

NIH Seeks Comments on Proposed Common Fund Programs

Deadline: September 14

The NIH Common Fund supports exceptionally innovative programs that are inherently high-risk but have the potential for high-payoff by catalyzing research across NIH and in the Biomedical research community. New ideas for Common Fund programs are identified annually by internal and external scientists and stakeholders. NIH is now asking for people’s input on these ideas to help shape new programs for 2013.

To provide comments on the programs, visit http://commonfund.nih.gov/strategicplanning.The feedback period is open until Wednesday, September 14.

You can post comments and discuss posts to help NIH refine broad conceptual topics into programs that more closely address Common Fund criteria.  NIH is looking for your input about how to shape these ideas so that the Common Fund investment will have a catalytic, transformative impact on a broad range of diseases and conditions.

NIH Common Fund programs are intended to be:

• Transformative: Must have high potential to dramatically affect biomedical and/or behavioral research over the next decade
• Catalytic: Must achieve a defined set of high impact goals within a defined period of time
• Synergistic: Outcomes must synergistically promote and advance individual missions of NIH Institutes and Centers to benefit health
• Cross-cutting: Program areas must cut across missions of multiple NIH Institutes and Centers, be relevant to multiple diseases or conditions, and be sufficiently complex to require a
  coordinated, trans-NIH approach
• Unique: Must be something no other entity is likely or able to do

The Daniella Foundation Congratulates Dr. Neal Young! 2012 Science and Environment Medal Recipient!

2012 Science and Environment Medal Recipient

This medal recognizes a federal employee for a significant contribution to the nation in activities related to science and environment (including biomedicine, economics, energy, information technology, meteorology, resource conservation, and space).

Neal A. Young

Position: Chief of the Hematology Branch, National Heart, Lung and Blood Institute

Agency: National Institutes of Health

Location: Bethesda, Maryland

Achievement: Saves lives through cutting-edge research and treatments for patients with bone marrow failure diseases, including the rare and once deadly blood disorder known as aplastic anemia.

Infusing New Life

For years, patients with severe aplastic anemia died within months of developing the rare blood disease. Dr. Neal Young has increased the survival rate to 80 percent—and climbing.

Dr. Neal Young is the world’s leading expert in the field of bone marrow failure, engaging in groundbreaking research that has led to life-saving treatments for a rare and deadly blood disorder known as aplastic anemia.

In his work at the National Institutes of Health (NIH), Young has combined pioneering basic laboratory science, clinical research protocols, and direct patient care to save the lives of thousands of people suffering from a blood disorder that wipes out the cells in the bone marrow, including red blood cells that carry oxygen, white blood cells that fight infection and platelets that help clot the blood.

Young conceived, designed and headed the first multicenter clinical trial in the United States for immunosuppressive therapy for aplastic anemia. The regimen he developed for this blood disease has become standard therapy for patients all over the world. This disease strikes about 600 to 900 people a year in the United States, and thousands across the globe.

“Neal has completed an amazing series of landmark clinical trials and published results that have taken what was a fatal disease in the early 1980s to one that now has a survival rate of 80 percent or more in the long-term,” said Dr. Cynthia Dunbar of the National Heart, Lung and Blood Institute at NIH.

Because of Young’s efforts, his clinic at NIH is today considered one of the world’s major referral centers for patients with bone marrow failure syndromes, including aplastic anemia. His clinic follows many hundreds of patients from North and South America, Asia and Africa.

One of them is Jordan Culbreath, a Princeton University football player diagnosed with aplastic anemia in 2009, during his junior year. Not only was Culbreath cured, but he was able to return to the field his senior year after Young treated him.

As chief of the Hematology Branch of the National Heart, Lung and Blood Institute, Young is continually building on his significant accomplishments. He developed an understanding of how the highly contagious B19 parvovirus infects cells of the bone marrow and showed its role in disease among patients, especially those with sickle cell anemia and others with defective immune systems. He developed ways to test for and diagnose this virus, came up with a treatment and currently has a vaccine in clinical trials that, if successful, will protect vulnerable patients.

Young also has made landmark discoveries linking genetic defects—mutations or mistakes in the DNA sequence—to the break down in cells and as a cause of aplastic anemia. He has determined through laboratory tests that sex hormones can prevent the cell damage, and he is now moving these findings into human clinical trials.

In addition, Young has implicated the same type of genetic mutations linked to aplastic anemia much more broadly to liver cirrhosis, lung fibrosis and leukemia. These findings may offer clues to improving treatment and patient outcomes for these common and devastating diseases.

Dr. Ching-Hon Pui, chairman of the Department of Oncology at St. Jude Children’s Research Hospital in Tennessee, said Young’s work in hematology “has totally transformed the field.”

“His impact has been truly global,” said Pui. “I cannot think of another hematologist with comparable accomplishments.”

Dr. Robert Gallo, a biomedical researcher who played a major role in the discovery of the virus that causes AIDS, said Young is “at the forefront of the field of aplastic anemia.” He said Young has made significant contributions at the “fundamental research level, in the clinical setting and at the therapeutic level.”

In addition to heading the NIH Hematology Branch, Young is the director of the NIH Center for Human Immunology, Autoimmunity and Inflammation. This center is dedicated to gaining a better understanding of how the immune system and inflammation affect a wide variety of common disorders, including cancer, atherosclerosis, rheumatic syndromes and neurologic degeneration. The center pulls in NIH experts from a wide variety of disciplines to share information and find commonalities that might help spur new treatments

Anna Rita Migliaccio, PhD, awarded NIH (R01) for Glucocorticoid Receptor Research!

Anna Rita Migliaccio, PhD, Professor of Medicine, Hematology and Medical Oncology and member of The Tisch Cancer Institute of Mount Sinai School of Medicine, has been awarded a Research Project (R01) grant from the National Institutes of Health (NIH) for her research project “The human glucocorticoid receptor and normal and pathological terminal Erythroid.”

This project, which has received funding for a four-year period, will study the pathobiology of erythropoietin (EPO) resistant anemias, such as Diamond Blackfan Anemia (DBA), and will seek to identify alternative erythroid stimulating agents for their treatments.

Diamond Blackfan Anemia is a congenic form of erythropoietin (EPO) resistant erythroid aplasia often associated with mutations that result in ribosome insufficiency.

Although approximately half of DBA patients have a clinical response to dexamethasone – a steroid drug in the glucocorticoid class that acts on the glucocorticoid receptor (GR) – the reason why only 40-50% of patients respond to these steroids is still unknown.

Clinicians have established important correlations between GR haplotype and variability in patients’ glucocorticoid resistance. Additional studies have revealed that a GR polymorphism may affect the biological consequences of GR activation in the terminal erythroblast (EB) maturation. These studies reflect a paradigm shift in understanding dexamethasone responsiveness in DBA patients.

Dr. Migliaccio’s newest study will characterize the biological activity of different GR isoforms in terminal erythroid maturation; identify the microenvironmental and genetic factors that regulate expression of these GR isoforms during terminal erythroid maturation; and investigate the role exerted by individual GR isoforms in rescuing terminal erythroid maturation in DBA patients. The study, by improving understanding of the biology in GR in normal and DBA erythropoiesis, will hopefully identify more potent ways to improve the treatment of DBA patients and possibly other erythropoietin-resistant anemia.

“I am very excited about this research project,” says Dr. Migliaccio. “As a relative newcomer to the study of Diamond Blackfan Anemia, I owe a great debt to other researchers in the field, such as Jeffrey Michael Lipton, MD, PhD, andAdrianna Vlachos,MDwho have paved the way for my research by creating registries of patients with DBA.” She adds, “I hope to collaborate with other scientists who are researching DBA to discover new and improved treatments for this disease.”

Cohen Children’s Medical Center Announces Leucine Trial for Transfused DBA Patients

Dear DBA Patients and Families:

We are pleased to announce the opening of: “The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance, using Bone Marrow Failure as a Model: A Pilot, Phase II Study of the Amino Acid Leucine in the Treatment of Patients with Transfusion-Dependent Diamond Blackfan Anemia”.

Diamond Blackfan Anemia (DBA) is a rare pure red cell anemia resulting from a failure of the bone marrow to make red blood cells. The anemia may present in infancy, in childhood, or occasionally in adulthood. For some patients a genetic cause for DBA can be found but for others no known genetic mutation is known at this time. Some patients have little or no anemia after treatment with steroids, whereas others may need continuous red blood cell transfusions or steroid therapy for a long time, sometimes for life. Steroids may control DBA in many patients at first, but after some time, the steroids may not work as well anymore. Red cell transfusions have many possible negative side effects, such as iron overload. Iron overload can occur in the liver, heart, and endocrine organs which can cause more complicated health problems such as liver or heart failure, diabetes, thyroid issues, etc.

Purpose of this project

The purpose of this study is to find an alternative treatment for patients with DBA who are dependent on transfusions, to better their quality of life. This study will use an amino acid supplement called leucine. The Department of Defense Telemedicine and Advanced Technology Research Center (TATRC) will fund up to 17 centers throughout the United States to participate in this study. A total of 50 patients with Diamond Blackfan anemia are expected to join this study.

About the study drug, Leucine

Leucine is widely available in health food stores as a food/nutritional supplement. Leucine is the only dietary protein that is able to help the muscles to make more protein. It is also used commonly by athletes to increase muscle mass. It has been used in infants who don’t have normal leucine levels. This medicine comes as in a powder form that is mixed in water or other liquid and swallowed. Mild gastrointestinal upset has been noted in patients who are receiving leucine as a liquid through a vein. This study will include only oral leucine.

Description of procedure

This study is nine months long. The patient will receive a complete physical exam as well as blood tests every 3-4 weeks, during his/her scheduled transfusion. Additionally, blood tests will be monitored every 2 weeks for the first month of the study. The patient will take the leucine 3 times per day, as a capsule, which can be opened so the powder can be mixed in a liquid and will continue the red cell transfusions as routinely scheduled, every 3-4 weeks.

The following are some of the basic inclusion and exclusion criteria for this pilot trial.

Inclusion Criteria
– Diagnostic and supporting criteria for the diagnosis of DBA
– Transfusion dependent
– Age 2 years and older
– Negative pregnancy test
– Signed informed consent

Exclusion Criteria
– Have a know allergy to leucine, isoleucine, norvalie, valine, methylalanine.
– Evidence of kidney or liver dysfunction
– Pregnancy, or plans to become pregnant during duration of trial
Please refer to the website link: http://www.clinicaltrials.gov and enter the search term “Diamond-Blackfan Anemia and leucine” for full eligibility criteria and additional trial information.

CONTACT INFORMATION

Principal Investigator: Dr. Adrianna Vlachos, Cohen Children’s Medical Center of NY

Scientific Co-Investigators:
Monica Bessler, MD, PhD, Children’s Hospital of Philadelphia
George Buchanan, MD, University of Texas Southwestern Medical Center
Steven R Ellis, PhD, University of Louisville
Bertil Glader, MD, PhD, Stanford University
Jeffrey M. Lipton, MD, PhD, Cohen Children’s Medical Center of NY
Zora Rogers, MD, University of Texas Southwestern Medical Center
Colin Sieff, MD, Dana Farber Cancer Institute

Please contact our study nurse, Ellen Muir:

TEL: 1-877-DBA-NURSe (322-6877) or 516-562-1505 FAX: 516-562-1599 Email: emuir@nshs.edu

Also, feel free to contact Dr. Vlachos by email: avlachos@nshs.edu

Dr. Harvey Lodish of Whitehead Institute Publishes New Finds

Click here to read the exciting findings published by Dr. Harvey Lodish of Whitehead Institute titled, “Scientists Identify Potential Drug Target for Treatment-Resistant Anemias.

Exciting DBA Findings from The Children’s Hospital of Philadelphia Published in BLOOD This Week!

For years, physician scientists have been trying to model the abnormal blood cell production that occurs in patients with DBA. These efforts have involved studies on yeasts, flies, fish and mice.  Human models have been difficult to create and previously relied on samples of patient bone marrow. As published this week in BLOOD, a group of collaborating scientists at The Children’s Hospital of Philadelphia show that skin cells from patients with DBA can be reprogramed so that, instead of skin, they start to produce blood cells (http://www.ncbi.nlm.nih.gov/pubmed/23744582). These reprogrammed cells, termed induced pluripotent stem cells (iPSCs) show defects in blood production that are characteristic of DBA when compared to iPSCs from normal individuals.  The work also demonstrates that the abnormal blood cell production is caused by the genetic mutation, because when the reprogramed cells are corrected for the mutation, blood cell production becomes near normal. Thus, for the first time, DBA can be modeled in the culture dish and each model is specific for the patient from whom the skin cells were taken. Moreover, the genetic defect can be corrected in iPSCs, suggesting new approaches for gene therapy.  This new technology is likely to accelerate progress in understanding DBA. For example we can compare reprogramed cells from individual patients who have only mild anemia to those from their relatives who are transfusion dependent or we can test new (and old) medications to identify ones that improve blood cell production with less toxicity than steroids. In the longer term blood cells produced in the culture dish may one day be used for patient specific therapy. Although there still is a long way to go, these are exciting times for DBA patients and research.  Importantly, this progress would not be possible without the support of DBA patients and families, including their participation in research.  (For more information and/or study participation in iPSC research for DBA, contact Weissmi@chop.edu and Besslerm@email.chop.edu).