A recent phase one clinical trial in the area of Parkinson’s disease, in which a growth factor was pumped directly into the human brain, has given promising results. Clive Svendsen, University of Wisconsin professor of anatomy and neurology and neuroscientist of the Stem Cell Research Program at the Waisman Center, co-authored the study along with an international team of surgeons and scientists.
Parkinson’s disease is characterized by involuntary muscle spasms due to the death of brain cells that produce dopamine, which controls voluntary muscular movement. The disease is degenerative and is fatal.
The trial’s main purpose was to test the safety of administering a protein known as the glial cell line-derived neurotrophic factor (GDNF) to the human brain. GDNF has been shown in animal studies to inhibit the death of dopamine brain cells.
Svendsen’s trial got similar results: patients’ brains were found to have greater control over storing dopamine, allowing better management of muscle movements. In fact, those administered GDNF showed a 39 percent increase in motor skills and a 61 percent increase in daily-life activities over those not given GDNF.
Also, patients showed no clinical side effects as a result of GDNF.
Although results have so far been promising, Svendsen stressed that the trial, which focused on five patients, was very small and is only in the beginning stages.
“GDNF seems to have some clinical effect in patients. AMGEN (a biotech company) is now doing a larger trial,” he said, noting that if that trial, with 32 patients, shows similar beneficial results, another larger study will probably be conducted.
Nonetheless, researchers say the results of this study have raised hopes that GDNF will one day help relieve the symptoms of Parkinson’s disease.
Svendsen became involved in this study after meeting British neurosurgeon Steve Gill several years ago.
“I have a long experience in growth factors and cell death in the brain; [Gill] was a great neurosurgeon,” Svendsen said, adding that after that point, the two designed a trial to get GDNF infusions into the brain, where the infusions were most needed.
As a part of this designed trial, patients were given the protein every day for 18 months, during which a catheter pumped 40 micrograms of GDNF to a region of the brain called the putamen.
Although the study was conducted at the Frenchay Hospital, Institute of Neurosciences, in England, Svendsen hopes to one day bring his studies to the Waisman Center.
“We have a great team together which will try to bring GDNF to the clinic … Collaborations between the PET imaging group of Andy Roberts and Jim Holden here at Waisman will be vital to the success of this pre-clinical trial,” he said. Studies are already underway to genetically modify human neural stem cells to produce GDNF.
The study and its team have received much recognition since publishing its results March 31, gaining coverage in news sources such as CNN and The Guardian in the United Kingdom.
“Basically, it has been picked up around the world,” Svendsen said.
Dana G. Longstreth, clinical instructor of communicative instructors, sees this study as one of many that have been making strides in the area of Parkinson’s disease. She is hopeful that the current technological advances will one day improve the quality of life for its victims.
“I perceive in the current research that there are going to be strives made,” she said, specifically in the areas of mobility, independence and communication.
