Brain Leukodystrophy Stem Cell Therapy Development
Leukodystrophy is a group of rare genetic disorders that affect the white matter of the brain, which affects the production or maintenance of myelin. Without proper myelin, nerve signals are not transmitted effectively, leading to neurological symptoms such as muscle weakness, developmental delays, seizures, loss of vision or hearing, and problems with coordination and balance. Stem cell therapy holds promise as a potentially curative treatment option for leukodystrophies. Therefore, CD BioSciences has launched a stem cell therapy development platform for leukodystrophy with our advanced technology, dedicated to advancing the treatment of this disease.
Overview of Leukodystrophies
Leukodystrophies are a group of rare genetic disorders that affect the white matter of the brain, which is responsible for the proper functioning of nerve signals. In these conditions, the myelin, which is the protective covering around nerve fibers, is progressively destroyed, leading to neurological symptoms such as developmental delays, seizures, movement disorders, and cognitive decline.
Diverse mechanisms to hypomyelination in leukodystrophies (Nowacki, et al. 2022)
Stem Cell Therapy in Leukodystrophies
Stem cell therapy is an emerging approach that holds promise for the treatment of leukodystrophies. The idea behind stem cell therapy is to replace the damaged or lost myelin by transplanting healthy cells that can differentiate into myelin-forming cells, called oligodendrocytes. One potential source of these cells is mesenchymal stem cells, which can be obtained from bone marrow or other tissues and have been shown to differentiate into oligodendrocytes in vitro and in animal models. Followings are some potential therapeutic approaches for treating leukodystrophies.
| Therapy | Description |
|---|---|
| Hematopoietic Stem Cell Transplantation (HSCT) | In patients who received HSCT before the symptom onset or at a very early symptomatic stage, the disease stabilized, the rate of loss of gross motor and cognitive functions and central nervous system demyelination decreased. SCT can provide remyelination. |
| Mesenchymal Stem Cells Transplantation (MSCT) | An improvement in the speed of nerve conduction was observed. MSCs provide normal enzyme activity in or around peripheral nerves, or that they differentiate into Schwann cells in vivo, that provide a supportive growth environment for nerve cells that allows them to regenerate to some extent and to improve the nerve conduction velocity. |
| Cord Blood Cell Transplantation (CBCT) | Cord blood cells more efficiently migrate to the CNS, allowing rapid selection of suitable donor and reducing the time between diagnosis and transplantation. It has been shown that the effectiveness of CBCT does not significantly differ from the effectiveness of HSCT |
One of the challenges of stem cell therapy for leukodystrophies is delivering the stem cells to the affected areas of the brain. This can be accomplished by direct injection into the brain, or by using techniques such as intravenous infusion or nasal administration.
Our Services
While stem cell therapy for leukodystrophies is still in the early stages of development, it holds promise as a potential treatment option for these rare and debilitating disorders. CD Biosciences offers leukodystrophy stem cell therapy development services included but not limited as follows:
As a pioneer in biotechnology, CD BioSciences has grown into one of the largest independent biotechnology companies in the world. CD BioSciences is committed to providing professional and efficient service to our customers around the world. If you are interested in our service, please contact us.
Reference
- Nowacki, Joseph C et al. "Emerging cellular themes in leukodystrophies." Frontiers in cell and developmental biology vol. 10 902261. 8 Aug. 2022.
For research use only, not for clinical use.
