Stem cell-derived exosomes have the potential to treat neurological diseases through the BBB (blood brain barrier) and autism using a mild form of nasal inhalation * Preclinical studies have shown success in treating the core symptoms of autism
The Stem Cell Company, a biotechnology company that develops innovative treatments for neurological symptoms based on stem cells, anti-BMP molecules for the treatment of multiple sclerosis and gene therapy based on (Gene Therapy) for neuropathic pain, announces that it has acquired a license for an innovative technology based on exosomes for the treatment of neuronal, neuropsychiatric diseases and in particular autism . The treatment for autism was developed by Prof. Danny Ofen from Tel Aviv University Sklar School of Medicine and the Shem Segol School of Neuroscience and was purchased from Ramot, Tel Aviv University's commercialization company.
Exosomes are tiny particles that appear in the fluid of the cells and are used to communicate with distant cells. Exosomes from mesenchymal stem cells MSC-exosomes are given in a unique way by inhalation through the nose. The first indication developed is for autism. Autism is a neurodevelopmental disorder characterized by three main symptoms that include: severe impairment in social interactions and communication skills, repetitive behaviors and cognitive inflexibility. The number of people suffering from autism has been steadily increasing in recent years, and there is currently no effective treatment, and hence, the unanswered medical need is steadily increasing. More than 3.5 million Americans have autism. According to the US Ministry of Health, the prevalence of autism among children in the US rose to 1 in every 59 children in 2018 compared to 1 in 110 children in 2010, therefore autism is the fastest growing developmental disability without an approved drug. The company expects that after regulatory approval for the drug based on exosomes, the drug has the potential to become a leading drug that sells more than a billion dollars a year, when the peak in sales will be reached five years after the approval.
Based on the impressive efficiency data in preclinical models, the treatment with exosomes of mesenchymal origin leads to a significant improvement in the following parameters: increasing social interaction including vocal, reducing repetitive behaviors and a significant improvement in maternal behaviors of retrieving puppies. No negative symptoms were observed in the pre-clinical trials, thus clinical success in humans is expected.
Prof. Ditza Tzhor, Director of the Autism Center, ALOT, at the Assaf Harofeh Medical Center, an expert in general pediatrics with a specialization in pediatric neurology and child development, said: "Exosomes of mesenchymal origin are an innovative and promising technology that has shown great effectiveness in preclinical studies carried out by the company, intended for the treatment in autism in child and adult populations, with an unmet medical need that is growing at a rapid pace." Ehud Marom, CEO and Chairman of the Stem Cell Company Ltd. said: "With such encouraging clinical results, when it comes to a medicine suitable for children through the nose, we will be pushed for an immediate clinical trial. The development of treatments using cells or cell derivatives in the fields of neurology is at the core of the company's activities, including autism, which is a medical need without a solution today."
Stem cell drugs are rapidly moving from a phase of preclinical trials to clinical trials and the clinical development of the drugs will be financed through the raising of 30 million dollars through strategic collaborations and investments in the company.
A stem cell drug company Established at the beginning of 2010 that combines medicines and biomedicine. The company has a research laboratory center and a GMP-level production site in the Mount Hotzavim Science Park in Jerusalem for product development up to and including clinical trials. The company develops cellular therapy products as independent treatments or in combination with drugs, focusing on neurological indications, including multiple sclerosis, pain and neuromuscular injuries, and autism. The company develops, manufactures, registers and markets its products, was established by a team of entrepreneurs from the field of life sciences and pharmaceuticals.
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When will they find a cure for paranoid schizophrenia?
I would love to find out about my 9.5 year old experiment
Daniel, 10 years old, was diagnosed with Rasmussen syndrome two years ago and is a candidate for surgery to remove the left lobe.
My little one has not been tested before they check that it is true and that it really helps. I will not try it on my daughter before I know it is real and true
How can we get in touch? Can we take the child who suffers from autism for an experiment?
Do you also think that in 15 or less we will be able to repair and renew most areas of the brain and in general when do you think you will see a doctor for autism
very encouraging. How can I get in touch and get details? Thanks
Hagit, on the company's website it is not indicated that the treatment targets all neurological diseases, but it is likely that there are other companies in the world that each focus on their own field. The fact that the development is Israeli does not mean that the Israelis will enjoy its first fruits. The genome of the brain is fully deciphered and everyone agrees that within up to 15 years from now we will be able to repair and renew most parts of the brain.
Are the drugs also aimed at other neurological and neuropsychiatric conditions? For example, Tourette's syndrome, ocd, various movement disorders such as Parkinson's or Alzheimer's?