Parkinson's Disease: The promising future of a Disease Modifying Therapy (DMT).

Parkinson’s disease (PD) is a degenerative disorder that largely affects the brain and for which the only available treatments, at best, can dampen some of the symptoms experienced by the patients. There is therefore no medication that can stop or slow down the progression of the disease.

 

As such, the entire Parkinson community is facing numerous unmet medical needs that require urgent attention. In the last decades, Dr. Francesca Cicchetti has been working on a drug already approved for the treatment of the rare disease Cystinosis and referred to as cysteamine.

 

This compound has demonstrated robust and promising neuroprotective and neurorestorative effects in various animal models and human cells relevant to PD. Moreover, cysteamine has shown benefits in patients with another brain degenerative disorder called Huntington’s disease (HD) and similar, in some ways, to PD.

Our Research

Cysteamine

15 years of preclinical work on an existing molecule called Cysteamine, which has demonstrated the following 3 advantages in animal and human models:

  1. easily crosses the blood-brain barrier (brain)
  2. has neuroprotective properties
  3. neuro-restorative effect on affected cells.

Blood Biomarkers

Currently under validation in over 357 patients, our preliminary findings have demonstrated that our blood biomarkers in Parkinson’s Disease could become a world first to support clinicians with a more precise diagnostic while being able to stratify the disease over time.

Clinical Programs

Disease Modifying Therapy program (DMT) in Parkinson Disease

SYN-001 (novel formulation of cysteamine)

Blood biomarkers in Parkinson’s disease

Ongoing validation program with over 357 patients