Navigation Links
Phase II study of an oral therapy for Gaucher disease yields positive results

(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
American Society of Hematology

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. 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:
(Date:10/13/2017)... , ... October 13, 2017 , ... ... throughout Arkansas that offers insurance and financial preparation services, is providing an update ... City Rescue organization. , Rock City Rescue is a locally recognized nonprofit that ...
(Date:10/13/2017)... Vegas, Nevada (PRWEB) , ... October 13, 2017 , ... ... 7® Hemp CBD Oil utilizing Purzorb™ technology. Applying the Purzorb™process to full spectrum CBD ... dose required and providing a CBD form that can be easily incorporated into liquid ...
(Date:10/13/2017)... RIDGE, N.J. (PRWEB) , ... October 13, 2017 , ... ... annual Holly Day Market. Featuring a collection of specialty vendors and unique items from ... of personalized and quality-focused health and wellness services offered by the VNA. The ...
(Date:10/13/2017)... ... October 13, 2017 , ... ... Software Development, has been awarded a contract by the Center for Medicare and ... aims to accelerate the enterprise use of Agile methodologies in a consistent and ...
(Date:10/12/2017)... ... October 12, 2017 , ... Planet Fitness, one of the ... today its plans to open a flagship location in Covington, LA at 401 N. ... To Go store next to Office Depot in the Holiday Square shopping center. Its ...
Breaking Medicine News(10 mins):
(Date:10/2/2017)... -- The Rebound mobile app is poised to become a ... of prescription drug addiction. The app empowers users to develop ... down their dosage in a safe, controlled manner while maximizing ... first 100,000 people to sign up will enjoy 3 months ... ...
(Date:9/25/2017)... , Sept. 25, 2017   Montrium , ... File solutions, today—from the IQPC Trial Master Files ... , NL)—announced that EastHORN Clinical Services has selected ... programs and TMF management. EastHORN, a leading European ... platform to increase transparency to enable greater collaboration ...
(Date:9/19/2017)... a venture-backed medical device company developing a non-invasive, robotically assisted, platform therapy that uses pulsed ...   ... Jim Bertolina, ... Tom Tefft ... medical device executive Josh Stopek , PhD, who has led R&D and business development ...
Breaking Medicine Technology: