Within the caution they ask for when disseminating their assessments, experts in Parkinson's disease (PD) bet on Genetics and molecular pathology, and rule out cell transplants as the solution. They believe that the core topic to find more promising therapies may lie in combining two types of advances: understanding how genetic disorders lead to the loss of neurons in the substantia nigra and measuring the progression of the neurodegenerative process with neuroimaging techniques. This is one of the conclusions of a group of international experts gathered in Navarra, convened by Dr. José Obeso, neurologist at the Clínica Universitaria de Navarra and neuroscientist at research center Applied Medicine (CIMA) of the University of Navarra.

Along with the Spanish scientist, his colleagues Matt Farrer (Mayo Clinic, Florida), Etienne Hirsch (Hospital La Salpetriere, Paris), Christopher Goetz and Jeff Kordower (Rush University Medical Center, Chicago), Glenda Halliday (Prince of Wales Medical Research Institute, Sydney), Juan Carlos Lopez (publisher-head of Nature Medicine, New York), Tony Schapira (University College London, London), Manuel Rodriguez (University of La Laguna, Tenerife), Concepción Marin (Institut d "Investigacions Biomèdiques August Pi i Sunyer, Barcelona) and Maria C. Rodríguez-Oroz (University of Navarra). The meeting was developed at partnership with Lundbeck Laboratories, business pharmaceutical company specialized in research and development therapeutics for diseases of the central nervous system.

Advances, hopes and cautions for 100,000 patients in Spain

PD is a neurodegenerative process that affects 1 million people in the EU and more than 100,000 in Spain. It is typically defined by the loss of neurons in the substantia nigra, which leads to a deficit of dopamine in the brain and causes slowness and scarcity of movements, tremor and muscle stiffness. Over the years of evolution, the neurodegenerative process spreads and affects other regions of the nervous system. Additional symptoms such as balance and gait disorders, sleep and vegetative disturbances (constipation, bladder dysfunction, impotence) or cognitive deficits emerge, representing the progression of the disease.

Experts believe that at present the treatment of dopaminergic deficits is well achieved and is not the main therapeutic challenge .Numerous pharmacological resources (levodopa, rasagiline, dopaminergic agonists, parenteral infusions, rotigotine patch, etc.) are now available toreplenish dopamine action and control the main symptoms. In addition, the widespread application of surgical treatment (deep brain stimulation) makes it possible to improve the quality of life of patients in whom drugs are not sufficiently effective. In this way, the motor manifestations that typify PD are controllable in most patients with the resources available. challenge According to Dr. Obeso, "the most pressing issue today is early diagnosis to halt its progression".

Many questions and 4 conclusions

There are molecules available (rasagiline) or in the process of clinical essay (neurotrophic factors) with a possible neuroprotective action in PD. However, it has not yet been possible to decipher why neuronal loss begins and the mechanisms involved in the progression and extension of the neuronal death process. For the specialist from the University of Navarra, "the difficulty in discerning these key aspects explains why, despite sensationalist prognoses in recent years with stem cells and the human genome, there have been no significant advances in the control of PD. Therefore, the effort researcher should focus on the origin of the problems".

At the scientific meeting held in Navarra, the main questions and unresolved problems were analyzed. The experts reached several conclusions, which are summarized by Dr. Obeso:

1. Better control of PD (halting its progression) is expected in the next decade, although it is not easy to envision a cure.

2. The therapeutic approach should be considered from a double perspective: to restore the dopamine deficit as soon as possible and to protect other brain regions from being affected by the neurodegenerative process. In this sense, it is recognized that the age of presentation and the cellular changes associated with aging play a determining role and an excellent opportunity to act with neuroprotective treatments.

3. Genetic alterations in Parkinson's patients with no family history are higher than assumed so far. In the near future it will be possible to recognize early people at risk of developing PD and to start neuroprotective treatments.

4. Cell transplants (e.g. stem cells) aimed only at replenishing the dopamine deficit do not provide conceptually or at internship any significant advance over existing treatments. Gene therapy or other possibilities at development are more promising to compensate for the effects of neuronal loss in the substantia nigra and, above all, to prevent its spread to other areas of the brain.