Navigation Links
To treat rare disease, NIH scientists repurpose FDA-approved drug
Date:9/2/2011

WHAT: A new study reports that a drug already approved by the Food and Drug Administration for use in patients undergoing a bone marrow transplant may also have promise for treating people who have a rare immune deficiency known as WHIM syndrome. People with the syndrome are more susceptible to potentially life-threatening bacterial and viral infections, particularly human papillomavirus infections, which cause skin and genital warts and can lead to cancer. The study was conducted by investigators at the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH).

Boys and girls are equally at risk of inheriting the genetic mutation that causes WHIM syndrome, and the disorder frequently affects multiple family members. Approximately 60 patients worldwide have been diagnosed with WHIM syndrome, 10 of whom are currently receiving care at NIH.

As a result of the inherited genetic mutation, the function of a molecule, called CXC chemokine receptor 4 (CXCR4), increases. This in turn inhibits migration of neutrophils and other types of white blood cells from the bone marrow into the bloodstream. With fewer circulating immune cells, those with the disorder are less able to fight off infections.

Patients with WHIM syndrome are currently treated with intravenous immunoglobulin, a blood product containing purified human infection-fighting antibodies, and granulocyte colony-stimulating factor, a molecule that stimulates production and maturation of neutrophils. But both therapies are difficult to administer, costly and only partially effective in treating the disease.

The drug tested in the study, known as plerixafor, blocks the activity of CXCR4 and could provide a targeted therapy for this disease. Over a seven-day period, three adult patients with WHIM syndrome were given six injections of increasing doses of plerixafor. The NIAID team observed that the numbers of nearly all immune cell deficiencies in the three patients increased to normal levels, with only minimal adverse side effects at the highest doses.

The investigators say the next step is to determine if long-term use of plerixafor, which is manufactured by Genzyme Corporation (Cambridge, Mass.), is safe and effective in adults. If it is, they will consider conducting clinical studies of plerixafor in children with WHIM syndrome.


'/>"/>

Contact: Julie Wu
niaidnews@niaid.nih.gov
301-402-1663
NIH/National Institute of Allergy and Infectious Diseases
Source:Eurekalert

Related medicine news :

1. Enhancing arrest of cell growth to treat cancer in mice
2. Despite Treatment, Employees with Depression Generate Higher Absentee Costs, According to Thomson Reuters Study
3. Women More Likely to Fail Treatment for Atrial Fibrillation
4. Social Anxiety and Panic - Alternative Treatment to Drugs and Therapy
5. Neuroimaging study may pave way for effective Alzheimers treatments
6. Nanofilm Introduces Clarity Defog It™ Anti Fog Treatment and EcoClens™ Eco-Friendly Lens Cleaner at Vision Expo East
7. Many veterans not getting enough treatment for PTSD
8. Scott & White Memorial Hospital uses device to revolutionize treatment of traumatic aortic injury
9. Parents often wait too long to treat childrens asthma symptoms
10. Allegheny General Hospital Study Demonstrates Safety and Potential Efficacy of Oral Allergy Treatment
11. Minorities Not Treated at Higher-Quality Centers
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/13/2017)... ... October 13, 2017 , ... ProVest ... the greater Dallas, Miami, and Raleigh regions, is organizing an extended charity drive ... rare and deadly chromosome abnormality. , After struggling since birth with several health ...
(Date:10/13/2017)... ... October 13, 2017 , ... ... process to promote standards of excellence for the field of eating disorders, announces ... 22 – 25, 2018 in Orlando, Florida at the Omni Resort at ChampionsGate. ...
(Date:10/13/2017)... ... October 13, 2017 , ... Many families have long-term insurance that covers care ... have a waiver for care if the client has a cognitive impairment diagnosis. ... for care, is often waived, so the benefits from their insurance start immediately,” said ...
(Date:10/13/2017)... ... October 13, 2017 , ... ... giving viewers the lowdown on sciatica in a new episode of "Success Files," ... on current events and innovation and investigates each subject in-depth with passion and ...
(Date:10/12/2017)... ... October 12, 2017 , ... ... sleep apnea using cutting-edge Oventus O2Vent technology. As many as 18 ... characterized by frequent cessation in breathing. Oral appliances can offer significant relief to ...
Breaking Medicine News(10 mins):
(Date:9/25/2017)...  EpiVax, Inc., a leader in the fields ... announced the launch of EpiVax Oncology Inc., a ... cancer vaccines. EpiVax has provided $500,000 in seed ... technologies to the new precision immunotherapy venture. Gad ... as Chief Executive Officer. Gad brings over 25 ...
(Date:9/22/2017)... , Sept. 22, 2017  As the latest ... Senators Bill Cassidy (R-LA) and Lindsey ... notes that the medical device industry is in an ... device tax, the 2.3% excise tax on medical device ... they also want covered patients, increased visits and hospital ...
(Date:9/19/2017)... -- HistoSonics, Inc., a venture-backed medical device company developing a non-invasive, robotically assisted, platform therapy ... team developments today:   ... ... Tom Tefft ... Veteran medical device executive Josh Stopek , PhD, who has led R&D ...
Breaking Medicine Technology: