Navigation Links
Phase II study of an oral therapy for Gaucher disease yields positive results
Date:5/3/2010

(WASHINGTON, May 3, 2010) Gaucher disease, a rare enzyme deficiency disorder, is one of many conditions with few approved treatment options for patients. In a study published online today in Blood, the journal of the American Society of Hematology, researchers present positive results of a Phase II clinical trial of eliglustat tartrate, an oral therapy in development to treat Gaucher disease.

Gaucher disease is a genetic disorder affecting an estimated 1 in 50,000 to 1 in 100,000 people in the U.S. population. Gaucher disease occurs when a mutation of the glucocerebrosidase gene causes low activity of that enzyme in the body. As a result, harmful fatty substances accumulate in the liver, spleen, bones, and bone marrow, preventing cells and organs from working properly. The primary treatment option is enzyme replacement therapy, which is given intravenously, to break down the accumulated fatty substances.

Eliglustat tartrate is an oral drug currently in development for Gaucher disease type 1 (GD1). As a substrate reduction therapy, the drug decreases the body's production of the fatty substances so they do not accumulate in cells. Based on Phase I trials in healthy volunteers that demonstrated positive initial safety results, the research team initiated a multinational, open-label, single-arm Phase II study of 26 GD1 patients to evaluate the efficacy, safety, and pharmacokinetics (how the body absorbs and processes the drug) of eliglustat tartrate administered orally at 50mg or 100mg doses twice daily. Eligible patients had confirmed Gaucher disease, characterized as enzyme deficiency and a spleen volume at least 10 times greater than normal, plus abnormal values of either platelet count or hemoglobin levels.

"As with many other rare conditions, we strive to develop new options with improved efficacy and safety for our patients, but we also look for options that may be easier on the provider and the patient.In this case, we were hoping an oral alternative would be viable," said lead study author Elena Lukina, MD, of the Hematology Research Center at the Russian Academy of Medical Sciences in Moscow, Russia.

The study endpoint (improvement in at least two of the three main efficacy parameters: spleen volume, hemoglobin level, and platelet count) was met by three-fourths (77%) of all patients and nearly all (91%) of the 22 patients who completed the full 52 weeks, with the greatest overall improvements seen in hemoglobin level and spleen volume. The research team found statistically significant improvements across many disease symptoms, including mean hemoglobin level, platelet count, spleen volume, liver volume, and lumbar spine bone mineral density. Furthermore, the patients' glucosylceramide plasma levels normalized. Disease symptoms seemed to respond rapidly, and improvement was seen especially in bone mineral density. This may have resulted from the drug's small molecular size, which allows it to diffuse quickly within affected cells.

"Eliglustat tartrate provides promise for a safe, effective, and convenient oral therapy for patients with Gaucher disease type 1," said Dr. Lukina.

Further clinical development of eliglustat tartrate is already proceeding with larger, controlled Phase III studies in untreated patients and in patients previously stabilized with imiglucerase an intravenously administered medication currently used to treat Gaucher disease.


'/>"/>

Contact: Wendy Stokes
webmaster@hematology.org
202-776-0544
American Society of Hematology
Source:Eurekalert

Related medicine news :

1. New phase II study supports potential of gs-9450 as new treatment option for steatohepatitis
2. Pilot Testing Phase Ends; Expansion Of Sales Force Begins
3. Southampton scientists begin Phase II patient trial for new asthma treatment
4. Intellicate Announces New “Phased Return to Work” (PRtW) Service
5. Askthedoctor.com Makes Medical Information More Accessible to the Public by Completing Phase 1 of US Expansion
6. Nuvo Research enrolls first patient in Phase 2 WF10 allergic rhinitis trial
7. Poniard Pharmaceuticals Presents Positive Survival Data from a Phase 2 Clinical Study of Picoplatin in Metastatic Prostate Cancer at the 2010 American Society of Clinical Oncology Genitourinary Cancers Symposium
8. New High Purity Immunoglobulin Enters Phase III in Europe and the US
9. Medical Imaging Northwest Completes Phase I of Its Healthcare IT Integration to Maintain Patient Care Improvements
10. NASA selects DNA Medicine Institutes Nanoscale Diagnostics Technology for Phase II SBIR
11. PRA International Rolls out Client Access to Oracle(R) RDC for Early Phase Studies
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/9/2016)... ... December 09, 2016 , ... Sober College, the game-changing ... grand opening of the Sober College Robert Pfeifer Memorial Learning Center at its ... and was attended by an overwhelming amount of alumni, family, colleagues and friends ...
(Date:12/9/2016)... ... December 09, 2016 , ... Mediaplanet today announces distribution of the latest edition ... to sign up as an organ donor for the 123,000 people in the United ... donor can save up to 8 saves through organ donation and enhance many others ...
(Date:12/9/2016)... ... December 09, 2016 , ... MEDI+SIGN®, a ... today that a new solution for Emergency Departments (ED) has been added to ... in Emergency Department examination rooms, and with a simplified pallet of information available ...
(Date:12/9/2016)... Harrisburg, PA (PRWEB) , ... December 09, 2016 ... ... concussion education program through the Pennsylvania Cable Network (PCN) during the summer of ... Brain Injury Implementation Grant provided by the United States Department of Health and ...
(Date:12/8/2016)... ... December 08, 2016 , ... The Florida ... Undersea and Hyperbaric Medical Society (UHMS), the leading authority in hyperbaric medicine. This ... Only a few hospitals and facilities have earned this distinction. This is the ...
Breaking Medicine News(10 mins):
(Date:12/9/2016)... DUBLIN , Dec 9, 2016 ... "High-Throughput Screening Market - Forecast to 2021" report to ... ... adoption of open innovative models in the pharmaceutical and biotechnology ... spending and rising government funding. Emerging markets and growing research ...
(Date:12/9/2016)... , Dec 9, 2016 Research and Markets ... 2016-2020" report to their offering. ... The global travel vaccines market to grow ... The report covers the present scenario and the growth prospects of ... size, the report considers the revenue generated from the sales of ...
(Date:12/8/2016)... LONDON , Dec. 8, 2016 Australia ... GlobalData,s new report, "Australia Ophthalmic Lasers Market Outlook to ... Lasers market. The report provides value, in millions of ... within market segements - Excimer Lasers, Femtosecond Lasers and ... and distribution shares data for each of these market ...
Breaking Medicine Technology: