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LEAPS Project to Advance Encapsulated Cell Delivery of GDNF

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) announced that it has awarded a grant to a research team led by Olle Lindvall, MD, PhD, of the University of Lund, Sweden to develop a neuroprotective therapy based on the implantation of encapsulated cells that produce the growth factor GDNF directly into the brain.  Awarded under the Linked Efforts to Accelerate Parkinson’s Solutions (LEAPS) program, the project will receive funding totaling approximately $3 million over four years, assuming all milestones are met.  

This project was made possible through a lead gift from Anne and Bernard Spitzer with matching funds provided by members of the Foundation’s Board of Directors.

“This project drives forward potentially breakthrough technologies on two fronts,” stated Deborah W. Brooks, executive director of the Foundation. “First, should encapsulated cell technology prove safe and effective, the result would be a novel delivery mechanism with applications for a myriad of therapies for Parkinson’s patients. In addition, the team will further assess the ability of GDNF to slow, halt or even reverse the degenerative effects of Parkinson’s disease.”

The Lindvall team’s approach plans to take the encapsulated cell technology (ECT) delivery of GDNF from a theoretical concept to a therapeutic reality. GDNF, or glial-derived neurotrophic factor, is recognized by researchers for its potentially protective influence on the dopamine neurons that typically degenerate in Parkinson’s disease. But because GDNF does not cross the blood-brain barrier, it cannot be given orally or by injection. While still experimental, encapsulated cell technology, if successful, would enable localized, long-term sustained delivery of GDNF to the brain with the primary aim of protecting dopamine neurons and stimulating their regeneration.

Prior studies of GDNF in Parkinson’s disease have shown positive effects in non-human models as well as in early clinical trials. Recently, preliminary results from a phase II trial employing another delivery system showed no clinical improvement after six months. However, the ability of GDNF to protect dopamine neurons and stop the progression of the disease was not tested.  This LEAPS project will help resolve and advance understanding of GDNF’s therapeutic potential as well as potential delivery system variables.

Full funding for the project is contingent upon the achievement of predetermined scientific milestones. The team’s first milestone is to generate human cell lines capable of steadily secreting small amounts of GDNF and place these cells in retrievable fiber capsules. Once created, these capsules will be tested to assess cell viability, level and duration of GDNF secretion and diffusion into the brain, along with general safety issues such as inflammation and retrievability. In addition, the neuroprotective and regenerative effect of this type of delivery of GDNF will be studied in disease models.  Should this therapy prove effective, 12 patients will be enrolled in clinical trials in Sweden, Switzerland, Germany and the U.K. and assessed clinically for at least 24 months.

In addition to Dr. Lindvall, members of the team represent seven academic institutions, a research foundation and a private company, including:

  • Patrick Aebischer, MD, PhD, Federal Institute of Technology (EPFL)Lausanne, Switzerland
  • Anders Björklund, MD, PhD, University of Lund, Lund, Sweden
  • David Brooks, MD, DSc, FRCP, FMedSci and Paola Piccini, MRCP, MD, Imperial College, London
  • Wolfgang Oertel, MD, PhilippsUniversity, Marburg, Germany
  • Niall Quinn, MD, FRCP, UniversityCollege, London
  • Jean-Guy Villemure, MD and Francois Vingerhoets, MD, Centre Hospitalier Universitaire Vaudois (CHUV), Lausanne, Switzerland
  • Lars Wahlberg, MD, PhD, NsGene A/S, Ballerup, Denmark.
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