Celavie Biosciences Application
Celavie Biosciences is committed to relieving suffering across the globe with regenerative stem cell therapies that address the following diseases of the central nervous system and other disorders.
Diseases of the Central Nervous System
According to the World Health Organization, 7.1% of the global burden of disease stems from central nervous system neurological disorders and cerebrovascular disease.
Parkinson’s disease (PD) is a chronic and progressive movement disorder. More than 6 million people worldwide are living with Parkinson’s disease.1 The risk of developing Parkinson’s is about 1% at age 60.2 The cause is unknown, and there is no cure at this time.
PD is characterized by:
- Death of neurons that produce dopamine—a chemical that controls movement and coordination
- Lack of ability to control movement normally, especially as the disease progresses
Huntington’s disease (HD) is a progressive neurodegenerative disorder causing an abnormal expansion of IT-15 gene on chromosome 4. Most people develop Huntington’s disease between 30-54 years old, but HD can manifest as early as 4 years old and as late as 80 years. Individuals with the adult-onset form of HD usually live about 15 to 20 years after signs and symptoms begin. Huntington’s disease is most common in populations of European descent and affects 25,000 to 30,000 individuals in the US.3
Huntington’s disease often includes some or all of the following symptoms:
- Early signs including depression, poor coordination, and trouble learning new information or making decisions
- Involuntary jerking or twitching movements, known as chorea, which become more pronounced as the disease progresses
- Trouble walking, speaking, and swallowing
- Changes in personality and a decline in thinking and reasoning abilities
Friedreich’s ataxia (also called FA or FRDA) is a rare, autosomal, recessive, inherited disease caused by a mutation in a FXN gene. Friedreich’s ataxia affects about 1 in every 50,000 people in the US.4
FA is characterized by:
- Degeneration of spinal cord, peripheral nerves and cerebellum
- Awkward, unsteady movement that becomes progressively worse as the disease progresses
- Impaired sensory functions that become progressively worse as the disease progresses
ALS, or amyotrophic lateral sclerosis, is a progressive neurodegenerative disease that affects nerve cells, particularly motor neurons, in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. More than 1 in 50,000 people in the US live with ALS.5
ALS is characterized by:
- Loss of ability for the brain to initiate and control muscle movement
- Losses of ability to speak, eat, move and/or breathe
Epilepsy is a central nervous system disorder in which nerve cell activity in the brain becomes disrupted, causing seizures. More than 23 million individuals worldwide suffer from epileptic seizures.1 Treatment with medication or surgery can control seizures for approximately 80% of people with epilepsy. Some children with epilepsy may outgrow the condition.
Seizure symptoms vary widely, but can include:
- Staring blankly for a few seconds
- Repeatedly twitching arms or legs
- Loss of consciousness
Celavie is committed to using our allogeneic pluripotent stem cell technology to alleviate suffering for a multitude of other disorders as well.
Diabetes mellitus refers to a group of diseases that affect how the body uses blood sugar (glucose). There are three major types of diabetes: type 1 diabetes, type 2 diabetes and gestational diabetes. High levels of blood glucose can damage blood vessels and lead to heart disease, stroke, kidney disease, blindness and damage to nerves in the feet. In 2014, the World Health Organization (WHO) estimated approximately 420 million people have diabetes mellitus worldwide.
Second-degree burns affect both the epidermis and the second layer of skin, the dermis. Blisters may develop and pain can be severe. Deep second-degree burns can cause scarring. Third-degree burns reach the fat layer beneath the skin. These burns can destroy nerves, causing numbness and often lead to permanent disfigurement. The WHO estimated that, in 2014 alone, nearly 11 million people worldwide required medical attention for burn injuries.
- GBD 2015 Disease and Injury Incidence and Prevalence Collaborators. Global, regional, and national incidence, prevalence, and years lived with disability for 310 diseases and injuries, 1990-2015: a systematic analysis for the Global Burden of Disease Study 2015. Lancet Lond. Engl. 388, 1545–1602 (2016).
- Truong, D. D., Bhidayasiri, R., Lisak, R. P. & Carroll, W. M. International Neurology. (Wiley Blackwell, 2016).
- Frank, S. Treatment of Huntington’s disease. Neurother. J. Am. Soc. Exp. Neurother. 11, 153–160 (2014).
- Pandolfo, M. Friedreich ataxia: the clinical picture. J. Neurol. 256 Suppl 1, 3–8 (2009).
- Kiernan, M. C. et al. Amyotrophic lateral sclerosis. Lancet Lond. Engl. 377, 942–955 (2011).