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AAV-hAADC-2 (was AV-201)


Treatment of Motor Symptoms

Gene Therapy

"In the therapy being studied, the gene coding for the enzyme that converts L-dopa to dopamine (AADC) is inserted into a common, non-pathogenic virus (AAV) to which > 90% of humans have been exposed.; The AAV will help to transport the AADC into the brain cells.; AAV-hAADC-2, the investigational drug being studied, is injected into the striatum during a surgical procedure. " (clinicaltrials.gov)


Patients who undergo the procedure would continue to take L-dopa; AAV-hAADC-2 is intended to provide, directly to the brain, the missing enzyme needed to convert L-dopa to dopamine.

In Clinical Trials (US)

Trial is not recruiting Patients

AV201 was initially developed by Avigen. On April 5, 2005 Avigen announced that it will focus on the development of traditional pharmaceutical products, particularly small molecules and biologics, and indicated it would divest its proprietary AAV technology but was committed to ensuring the continuation of the ongoing clinical programs, including the AV201 Program in Parkinson's disease.
Clinical development was taken over by Genzyme. The treatment name was changed to AAV-hAADC-2.
Sponsor(s)
Sponsor (click for details) URL
Primary Sponsor Genzyme Corporation (GENZ) http://www.genzyme.com/corp/structure/corp_home.asp
Regulatory Information

Active
Not Available

Not Yet Filed
Treatment Phase Details
Click on Phase Number for DETAILED INFORMATION on that phase
Phase Status Title of Trial Start Date Duration Weeks
Preclinical Phase Completed   Unknown   Avigen, Inc.
Safety Review Not Started      
Phase 1 Phase Completed A Phase 1 Open Label Safety Study of Intrastriatal Infusion of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase (AAV-hAADC-2) in Subjects With Parkinson's Disease [AAV-hAADC-2-003] December 2004   Genzyme Corporation (GENZ)
Phase 2 Not Started       Genzyme Corporation (GENZ)
Phase 3 Not Started       Genzyme Corporation (GENZ)
Phase 4 Not Started       Genzyme Corporation (GENZ)
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Partnerships and Agreements
Description Notes Url1 Url2
Genzyme Corp. Goal: Product/Outcome: Importance: "In January 2006 Avigen sold its AAv gene therapy assets to Genzyme of Cambridge Mass.Genzyme will make an upfront cash payment of $12 million to Avigen with additional milestone payments and royalty payments on all products developed under Avigen"
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Citations for this treatment information
Authors Title Source Citation Date Online Ret Date VolumeSort by this column Pages URL
CW Christine, PA Starr, P Larson, R Mah, J Eberling, W Jagust, D McGuire, MJ Aminoff Aromatic L-amino acid decarboxylase gene transfer therapy for Parkinson's disease: Initial results of an open-label, dose-escalation, safety and tolerability study Neurology 2006. Supplement on 58th Annual Meeting of American Academy of Neurology. Poster session April 2006   66 (supplement 2) S23.005, A185
Christine, CW, et.al. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease Neurology November 17, 2009 17-DEC-09 73 1662-9.
  Avigen Announces Encouraging Early Data from Parkinson's Disease Clinical Trial Avigen press releases July 19, 2005       http://www.avigen.com/non_financial_release/2005/2005_Avigen_EarlyData_PDClinicalTrial_071805.htm
  Phase I Safety Study AAV-hAADC-2 in Subjects With Parkinson's Disease clinicaltrials.gov June 30, 2006 11-SEP-06     http://www.clinicaltrials.gov/ct/show/NCT00229736?order=2
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Related information
Description Information
FDA reviewing all ongoing trials involving any use of adeno-associated virus (AAV) - announced July 26, 2007.

Excerpts from FDA Statement on Gene Therapy Clinical Trial (July 26, 2007)

"On July 24, 2007 the U.S. Food and Drug Administration (FDA) was informed by Targeted Genetics Corporation of Seattle about the death of a patient who received an investigational gene therapy product in a clinical trial for the treatment of active inflammatory arthritis...

The product that was being studied uses a particle called a vector that is designed to deliver treatment genes to target cells. The vector used is a recombinant adeno-associated virus (AAV) derived vector and delivers the gene for Tumor-Necrosis Factor -Receptor, with the intent to inhibit a key mediator of inflammation. In the study, the gene therapy was administered into the joint affected by the disease to reduce inflammation and disease in patients with active inflammatory arthritis. ...

FDA is not aware of similar adverse events occurring in other gene therapy trials either with this specific product or with those that use other genes in AAV vectors. However, as a precaution, the agency is further reviewing all ongoing trials involving any use of AAV. "

Full FDA statement at:

http://www.fda.gov/bbs/topics/NEWS/2007/NEW01672.html

  

 
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