Rare neurogenerative diseases

In the Institute the following rare neurodegenerative diseases are treated:

  • Multiple System Atrophy (MSA)
  • Progressive Supranuclear Palsy (PSP)
  • Progressive Bulbar Palsy (PBP)
  • Kennedy's disease bulbar -spinal muscular atrophy
  • Huntington's disease
  • Frontotemporal dementia (FTD)
  • Dementia with Lewy bodies (DLB).

Neurodegenerative diseases are a large group of diseases with various aetiologies, but common pathomechanism involving damage of neurons within central motor neuron. Nerve cells degeneration is most often the result of storage phenomena leading to impaired metabolism or/and transport or the damage of intracellular structures. It also occurs as a result of the processes of early aging and apoptosis.
Genetic conditioning of many neurodegenerative diseases is associated with the presence of proteins containing improper sequences, which due to their occurrence or harmful spatial structure constitute metabolic blocks, or pose a direct material that loads nerve cells.
However, not all the mechanisms and causes of these diseases have been ultimately determined.
Persistant development and course of neurodegenerative diseases result in deficits associated with damages induced in the nervous system. Their spectrum is related to the location of lesions and includes movement disorders in the form of paresis, motor ataxia, spasticity, involuntary movements, stiffness and cognitive impairment, dementia, psychotic or personality symptoms.
Apart from a few promising cases of gene therapy (e.g. in spinal muscular atrophy), in neurodegenerative diseases a causative treatment is not possible. Supplements or neuromodulatory systems modifying agents as well as symptomatic treatment are used.
Stem cells offer a new hope for therapy in neurodegenerative diseases. The goal of stem cell transplantation is to trigger trophic factors and to stimulate immunomodulatory and antiapoptotic mechanisms, and in the case of pluripotent cells – to provide undifferentiated cells that are able to divide and differentiate into neurons and glial cells. Mesenchymal cells (MSCs) are of greatest importance in the treatment of neurodegenerative diseases, due to the fact that they are capable of producing cytokines and trophic factors, interleukins and other substances. Due to the tendency to migrate towards damaged cells, MSCs have the ability to influence these cells by stimulating growth, decreasing the level of free radicals and inhibiting apoptosis.