Stem Cell Therapy for Hereditary Ataxia
By Like Wu, Xiaojuan Wang, Pearly Liu, Bo Cheng and Susan Chu
Wu Medical Center, Bejing, China
The patient is 33-year-old female. She was presented with gait abnormalities and unstable walking for the past 3 years without any apparent reasons. The disease onset slowly and progressed gradually. This was accompanied with speech disorders and swallow difficulty for the past 1 year. She had gene analysis and was diagnosed with Hereditary Ataxia: Spinocerebellar Ataxia type I (SCAI) 4 months ago at a local hospital. The patient’s mother, uncle, aunt and cousin had the same disease.
The vital signs were normal, the skin with no yellow stains. The heart, liver and spleen were all normal. Her speech was not clear and slower than before. Both eyeballs had an equal level of nystagmus. There was diplopia when she stared to left side or right side. The ability to view things in pairs was a distance of 10 cm. She couldn’t raise the soft palate normally. The other examination of cranial nerves was normal. The muscle power of her four limbs was at level 4. Bilateral patella tendon reflex were normal, Bilateral patellar tendon reflex was active, Left side Hoffmann sign was positive. Left side Rossilimo sign was positive. The deep and shallow sensation of her trunk and lower limbs were slow down. She completed the finger-to-nose test, rapid rotation test and heel-knee test in unstable manner. The rombergs sign was positive whenever she closed or opened her eyes. She was unable to stand with single leg and unable to walk on a straight line. Gene analysis showed: Spinocerebellar Ataxia type I(SCAI).
Diagnosis: Hereditary Ataxia: Spinocerebellar Ataxia type I(SCAI)
Treatment target: Replace the heredodegeneration nerve cells with normal stem cells to repair the nerves, improve the nerve function, equilibrium function and motor function.
Treatment procedure and results:
We gave the patient 4 times neural stem cells (NSCs) and 4 times mesenchymal stem cells (MSCs) implantation treatment. The stem cells were activated in the patient’s body to repair the nerve damage. Together with nourishment of the neurons, improve circulation; regulating the immune, daily rehabilitation training was incorporated. During the treatment, the patient was happy, had a regular eating and sleeping pattern. With our doctor’s help, she completed the treatment. After the treatment, the patient had significant improvement, Binocular abduction diplopia distance reduced to 2cm, her pronunciation was clear, her speech was normal. Her four limbs had muscle power and it was at level 5-. The coordinate movement was better. Bilateral rapid rotation test and finger-to-nose test was more flexible, Bilateral ankle-knee-tibia test was steadier, the rombergs sign was negative when she opened her eyes, and it’s probable positive when she closed her eyes. She could stand on one foot for about 10-15 seconds, she could walk on a straight line and the quality of her life had been noticeably improved.
Case analysis: The Hereditary Ataxia is a group of genetic degenerative disease. Its characteristic is chronic progressive cerebellar ataxias. And the main three characteristics are: Hereditary, ataxia behaviors and cerebellar lesion. Besides cerebella and conducting fibers, this disease also affect columna posterior medullae spinalis, pyramidal tract, nuclei pontis, basal nuclei, nuclei nervorum cranialium, spinal ganglion, autonomic nerve system etc. There was no method of treatment, the patients will be worse and worse, and finally the patients will lose motor function and can’t walk. Finally they will die of complications.
The patient had this disease in his youthful age. There was a history of it in her family. She was presented with speech disorders, diplopia and coordinate movement problems. She had received DNA test and diagnosed as SCAI. Our center uses advanced NSCs transplantation technique to treats patients. The NSCs is used together with MSCs to make the imbedded NSCs increase the number of brain and spinal cord nerve cells, promotes nerve restoration and differentiation. At the same time, NSCs produces growth factors to protect damaged nerve cells. This technique was used in this patient and she recovered well, her life quality was improved. When the conventional therapy could not treat the disease, stem cell treatment is a new way to treat Hereditary Ataxia and this brings hope to patients with this disease.
According to full emergence of clinical symptoms and pathological features our center classified the Hereditary Ataxia patients in mode of inheritance. It’s necessary for directing cellular therapy to individualize the program. The nerve cells had gene mutation which leads to cells death in this disease. The number of nerve cells decreased gradually. At the end of this disease lots of nerves died. The cerebella and spinal cord start to atrophy. The nerves decreasing could not be revived, so there is no method of treatment in later stages.
In recent years, the results of cytology and molecular genetics are changing our knowledge in treating this disease. The new method used in our center with regard to the use of NSCs, make them differentiate into the functional nerve cell, replace the degenerative and necrotic nerve cell. This method needs the newest clinical technology to control procedure.This include: 1. NSCs locate accurately, through diseased region signals induce gradient and get the lesion areas with neurotrophic factor concentration. In the experiments on animals under the same conditions, we found that there are NSCc groups in the cerebellar and spinal cord. In recent years, there were researchers who found out the same situation in ALS research. This proved that cells could live in tissues but no cell functions.2. The cells differentiate into targeting cells. In this disease, we need neural precursor cells to differentiate into purkinje cells and granular cells in cerebella. This can definitely improve clinical symptoms. It can get the target with the drugs we have. 3. The expressive function of cells: even stem cell differentiates into that cell; it can’t express function in pathology environment. We must use cell control technology, for example 5-hydroxy tryptamine. This can promote purkinje cells expressing functions. GABA can promote granular cells growing and expressing functions. According to the nature of the disease, there are numbers of cell control technologies. With this disease, gene mutation in 6p atrophied the olive bridge cerebellar, and 12p, dentate pallidoluysian atrophy. It is different in clinical control locating and differentiating under the cell treatment. We will get a good result, get the right point of treatment and research only if we take subtle individual treatment plan.