Health and Medical News and Resources

General interest items edited by Janice Flahiff

[Press release] NIH researchers tackle thorny side of gene therapy

NIH researchers tackle thorny side of gene therapy 

From the 20 January 2015 press release

 

NIH researchers tackle thorny side of gene therapy

Pre-clinical studies in mice reveal ways to reduce cancer risk with modified treatment

NHGRI researchers conduct laboratory investigations to advance gene therapy. Watch the video featuring Dr. Charles Venditti and Dr. Randy Chandler: YouTube video Methylmalonic Acidemia (MMA) Gene Therapy
Lab technnician with a pipette

Bethesda, Md., Tues., Jan. 20, 2015 – National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute (NHGRI), part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.

“Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children,” said NHGRI Scientific Director Dan Kastner, M.D., Ph.D. “This study is an important step in developing gene therapies that can be safely used to benefit patients.”

Toxic side effects actually are rarely observed by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a specific point in the cell’s DNA. AAVs are small viruses that infect humans but do not cause disease. A vector is a DNA molecule of AAV used as a vehicle to carry corrected genetic material into a cell. AAV viruses are uniquely suited for gene therapy applications.

But one prior study did find an association between AAV and the occurrence of liver cancer. The present research addresses this problem in gene therapy for an inherited disease in children called methylmalonic acidemia, or MMA. For 10 years, NHGRI researchers have worked toward a gene therapy to treat MMA. The condition affects as many as 1 in 67,000 children born in the United States. Affected children are unable to properly metabolize certain amino acids consumed in their diet, which can damage a number of organs and lead to kidney failure. MMA patients also suffer from severe metabolic instability, failure to thrive, intellectual and physical disabilities, pancreatitis, anemia, seizures, vision loss and strokes. The most common therapy is a restrictive diet, but doctors must resort to dialysis or kidney or liver transplants when the disease progresses.

January 23, 2015 Posted by | Medical and Health Research News | , , , , , | Leave a comment

NIH establishes National Center for Advancing Translational Sciences

diagram.gif

[From http://www.vetmed.vt.edu/news/vs/may06/index.html]

From the 23rd December 2011 National Institutes of Health (NIH) press release

In a move to re-engineer the process of translating scientific discoveries into new drugs, diagnostics, and devices, the National Institutes of Health has established the National Center for Advancing Translational Sciences (NCATS). The action was made possible by Congress’ approval of a fiscal year 2012 spending bill  and the president’s signing of the bill, which includes the establishment of NCATS with a budget of $575 million.

NCATS will serve as the nation’s hub for catalyzing innovations in translational science. Working closely with partners in the regulatory, academic, nonprofit, and private sectors, NCATS will strive to identify and overcome hurdles that slow the development of effective treatments and cures.

“Congressional support for the National Center for Advancing Translational Sciences marks a major milestone in mobilizing the community effort required to revolutionize the science of translation,” said NIH Director Dr. Francis S. Collins, M.D., Ph.D.  “Patients suffering from debilitating and life threatening diseases do not have the luxury to wait the 13 years it currently takes to translate new scientific discoveries into treatments that could save or improve the quality of their lives.  The entire community must work together to forge a new paradigm, and NCATS aims to catalyze this effort.”

A prime example of the type of innovative projects that will be led by NCATS is the new initiative between NIH, the Defense Advanced Research Projects Agency, and the U.S. Food and Drug Administration  to develop cutting-edge chip technology.  This new technology will allow researchers to screen for safe and effective drugs far more swiftly and efficiently than current methods. A great deal of time and money can be saved testing drug safety and effectiveness much earlier in the process.

To meet the goals of NCATS, NIH is reorganizing a wide range of preclinical and clinical translational science capabilities within NIH into an integrated scientific enterprise with new leadership and a bold new agenda.  While the effort to recruit an NCATS director continues, organizational changes and realignment of resources will move forward under the leadership of Acting Director Thomas R. Insel, M.D., and Acting Deputy Director Kathy Hudson, Ph.D.  Dr. Insel is the director of the National Institutes of Mental Health and Dr. Hudson is the deputy director for science, outreach, and policy at the National Institutes of Health.

The following programs will comprise NCATS:

  • Bridging Interventional Development Gaps, which makes available critical resources needed for the development of new therapeutic agents
  • Clinical and Translational Science Awards, which fund a national consortium of medical research institutions working together to improve the way clinical and translational research is conducted nationwide
  • Cures Acceleration Network, which enables NCATS to fund research in new and innovative ways
  • FDA-NIH Regulatory Science, which is an interagency partnership that aims to accelerate the development and use of better tools, standards and approaches for developing and evaluating diagnostic and therapeutic products
  • Office of Rare Diseases Research, which coordinates and supports rare diseases research
  • Components of the Molecular Libraries, which is an initiative that provides researchers with access to the large-scale screening capacity necessary to identify compounds that can be used as chemical probes to validate new therapeutic targets
  • Therapeutics for Rare and Neglected Diseases, which is a program to encourage and speed the development of new drugs for rare and neglected diseases

The budget for NCATS is primarily a reallocation of funds from programs previously located in the NIH Office of the Director, National Human Genome Research Institute, and National Center for Research Resources.  NIH is committed to both basic and applied research and has maintained a relatively stable ratio of funding across these two areas of focus. The funding ratio will not be disturbed by the establishment of this new center.

The formation of NCATS has been a methodical process highlighted by the recommendation of the NIH Scientific Management Review Board in December 2010 to create a new center dedicated to advancing translational science.  This recommendation was followed by a year of intensive feedback and expert insight from all sectors of translational science through advisory meetings and extensive public consultation.

“I am deeply grateful for the expertise and insight provided by the many researchers, industry executives, patients, voluntary organizations, and NIH staff that helped NIH evaluate NCATS’ purpose and crystallize its vision,” said Dr. Collins.

To learn more about the impetus and development of NCATS, go to:

About the National Institutes of Health (NIH): NIH, the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visitwww.nih.gov.

December 24, 2011 Posted by | Medical and Health Research News | , , , , , , | Leave a comment

   

%d bloggers like this: