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Description of the Project
Project Description – Corporate and Investors
WHAT IS ALS (EN)
ALS (Amyotrophic lateral sclerosis) is also referred to as Lou Gehrig's disease, Maladie de Charcot or Motor Neuron Disease.
ALS is a very serious disease. Average age at diagnosis is around 55 years, with the disease progressing more rapidly the older the person is at diagnosis. Most people with ALS die from respiratory failure within 3 to 5 years after the disease’s onset, though about 10% of ALS patients survive 10 years or more. Currently, there is no cure for the disease. The only approved drug (Rilutek) modestly improves patients' survival by an average of 3 months.
The process is rapidly degenerative and attacks the nerve cells responsible for voluntary movements, called motor neurons. Once the motor neurons die, the muscles that they command do not receive orders, and they weaken, twitch (fasciculation) and finally waste away (atrophy). Little by little patients lose the ability to carry things, to walk, to speak, to eat and finally to breathe without ventilatory support.
It differs from Alzheimer’s Disease in that it usually does not attack parts of the brain responsible for the "mind" of a person (cognition) and memory or only at the latter stages of the disease. ALS also differs in that it does not impair people’s ability to see, smell, taste or recognize touch.
Despite enjoying a considerably smaller profile and level of understanding, more people die of ALS on a yearly basis than Multiple Sclerosis.
About the MitoTarget Consortium
The MitoTarget Consortium is a group of experts with a proven record of achievements and discovery within the field of neurodegeneration. MitoTarget aims at demonstrating that mitochondrial dysfunction, leading to a critical failure of the cell’s power plant, is a major common feature of neurodegenerative diseases.
MitoTarget aims at providing:
- the clinical 'proof-of-concept' needed to market a olesoxime-TRO19622, the first of a new class of mitochondrial targeted drugs that may address mitochondrial dysfunction and achieve neuroprotection, or protection of the nervous system, in humans, and therefore greatly affect the lives of those affected by diseases like ALS
- the preclinical proof-of-concept of mitochondrial dysfunction in neurodegenerative diseases, and a new class of mitochondrial targeted drugs, that include the following underserved (incurable) diseases:
- Alzheimer’s disease
- ALS
- Hereditary Spastic Paraplegia
- Huntington's disease
MitoTarget aims at deciphering involvement of the mitochondria in neurodegenerative diseases beyond the mechanism of action, (or how the drug works), of the drug called olesoxime-TRO19622. Effectively, the discovery will allow the design of new and more potent analogs of the drug. Mitochondria have already been proved to be valid targets for neuroprotection as seen with the drug Dimebon, clinically used since 1983. Dimebon was licensed by Medivation to Pfizer after a successful phase II trial in Alzheimer's Disease. Under the licensing agreement, Medivation will receive $225 million up front and is eligible for up to $500 million in development and regulatory milestones, plus undisclosed commercial milestones.
By using and combining the above information, MitoTarget will allow the improvement and/or the design of a new class of drugs targeting mitochondria in a wide range of neurodegenerative conditions.
