One single injection as a treatment for Parkinson’s disease. This is what researchers in Lund hope will be a reality within five years. Researcher Tomas Björklund and his colleagues have developed a form of gene therapy that appears much more promising than what has been achieved with previous research in the field. Their therapy is based on transferring genes, which code for the dopamine-producing enzymes, to the patient’s own nerve cells.
The Lund researchers have investigated the treatment in an animal model in a situation corresponding to the final stage of Parkinson’s disease, when no dopamine-producing nerve cells remain. “The animals recovered almost entirely after the treatment”, says Tomas Björklund, who recently presented his doctoral thesis on the subject. The thesis includes the article on the Lund researchers’ gene therapy that has now been published in the respected scientific journal Brain.
Tomas Björklund’s thesis describes the entire journey from understanding the interplay between the two genes that comprise the gene therapy to the creation of a ‘gene vector’ that will now undergo clinical testing. A gene vector is the shell of a virus, into which genes can be inserted to then be delivered by the virus shell to the cells to be treated. The Lund researchers have already submitted a patent for the construction. “The entire thesis builds up to the clinical testing of this method. We hope to find a partner who can help finance the clinical studies. The aim is to have a first clinical trial within three years. If all goes well we can have a treatment on the market in five years’ time”, says Tomas Björklund. And it is not a question of a drawn-out treatment. “The idea is that the patient would receive one injection and that’s it.”
Parkinson’s disease is caused by the death of certain cells in the brain, which produce the transmitter substance dopamine. Usual symptoms are shaking, stiffness and slow movements. The standard treatment is L-DOPA, a precursor to dopamine, but after several years, the treatment often produces side-effects in the form of involuntary movements. An advantage of the gene therapy is that the dopamine is continually produced in the cells. The patient avoids the peaks and troughs in dopamine production that are part of, for example, traditional treatment with L-DOPA.
“The pharmaceuticals industry is interested in this type of treatment and is beginning to see the possibilities of it. We can show that it is safe and effective. This can also open up the way for new treatments for Huntington’s disease and Alzheimer’s disease”, says Tomas Björklund.
For more information please contact Tomas Björklund +46 (0)704 85 21 50 or tomas.bjorklund@med.lu.se Tomas Björklund is a member of the Bagadilico research network at Lund University, which aims to shorten the route from the laboratory to the patient for new treatments for Parkinson’s disease. Link to Bagadilico: http://www.med.lu.se/bagadilico Link to journal Brain: http://brain.oxfordjournals.org/
