Batten's disease--A Disorder with No Cure
Batten's disease is a rare, fatal, autosomal recessive inherited disease of the nervous system (neurodegenerative disorder) that begins in childhood. It is named after the British pediatrician Frederick Batten who first described the juvenile form of neuronal ceroid lipofuscinosis (NCL) in 1903. The name is now commonly used to encompass three forms of NCL: infantile, late infantile and juvenile onset. All forms have the same basic cause; the lack of a lysosomal enzyme and have a similar progression and outcome. Batten's disease is caused when defective genes fail to make enzymes needed to dispose of waste made by brain cells. The waste piles up in the brain and kills healthy cells until the patient eventually dies. Children with Batten's disease suffer seizures, progressive loss of motor skills, sight and mental capacity, eventually becoming blind, bedridden and unable to communicate. Today, Batten's disease is always fatal.
Clinical manifestations of Batten's disease:
Early symptoms of this disorder usually appear between the ages of 4 and 10, with gradual onset of vision problems, including eye discoloration in a milky fog gloss over the eyes, and seizures. Early signs may be subtle personality and behavioral changes, slow learning or regression, repetitive speech or echolalia, clumsiness, or stumbling. There may be slowing head growth in the infantile form, poor circulation in the lower extremities (legs and feet), decreased body fat and muscle mass, curvature of the spine, hyperventilation and/or breath-holding spells, teeth grinding, and constipation.
Over time, affected children suffer mental impairment, worsening seizures, and progressive loss of sight, speech and motor skills. Eventually, children with Batten's disease become blind, bedridden, and demented. Batten's disease is often fatal between the ages of 8 and 15.
Stem Cell Treatment:
We have developed a comprehensive program for patients suffering from Batten's disease combining both nerve stem cell activation and stimulation treatment, and stem cells injections.
The treatment includes some medications to adjust the microenvironment of the patient's brain, and stem cell implantation, a "cocktail" of medications and neural nutrition materials given through IV, biofeedback treatment and comprehensive rehabilitation.
By employing all of the above methods we can maximally regulate and repair the patient's nervous system, improve symptoms such as motor disturbance and speech impediment. The curative effect depends on the patient's medical condition. In general, the earlier the patient receives the treatment, the better effect they can achieve. But this does not mean that we can cure the disease completely. Current treatment has only been proven to slow the progression of this terrible disease.
Stem Cells Injections:
1. Before the injection of the stem cells, medications will be used to improve the microenvironment of the brain
The disease is inherited in an autosomal recessive manner. The mutation causes the lack of lysosomal enzymes which can digest food particles and worn-out cell parts and it leads to the build-up of lipfusicins in the body's tissues. These substances consist of fats and proteins and form certain distinctive deposits that cause the symptoms. So, initially the doctor will give the patient medication to clean the waste in the brain and adjust the microenvironment of the brain. That treatment is very important as it can help stem cells take full advantage of the clinical effect. And with the cooperation of the medication and stem cells treatment, the patient can get the greatest improvement.
2. Neural stem cells injection
As an inherited disease (meaning the patient has a gene defect), sufferers from Batten's disease cannot use their autologous stem cells for the stem cells treatment. For this reason, our doctors will advise neural stem cells treatment. Doctors will implant the Mesenchymal Stem Cells (MSC) through the CSF (spinal cord fluid) by a lumbar puncture. Each injection has 1,000,000 stem cells. The cell count involved in the injection is safe to the young patients because it has been calculated accurately beforehand. This procedure is short and simple, and is usually done with local anesthesia. Yet, in order to prevent any risk of infection and to keep a strictly sterile environment, the procedure is done in the operating room by the experienced neurology doctor only.
Neural stem cells are multipotent cells that can differentiate into a variety of cell types, including neurons and neural ganglia cells and the new neurons and neural ganglia cells have normal functions such as secreting some neuro-factors essential to maintaining normal activity in the brain. These neural stem cells can be acquired conveniently, and all the neural stem cells come from Peking University's Stem Cells research Center which is the largest and most qualified stem cell lab in China, where the cells have undergone rigorous testing according to our highly strict regulations. So the advantages are: Firstly, the cells are convenient and timesaving because they do not have to be cultured for a long time. Secondly, the neural stem cells are very safe to be used in clinic therapy. In addition, these stem cells have good biological activity and have little chance of causing an immune response (immunological rejection) within the patients themselves.
The side effects of stem cells treatment:
The procedure can cause minor side effects for 2 to 3 days after the stem cells injection, including fever, sleepiness, headaches, dizziness and fatigue. These side effects are expected and should not alarm or concern you. Some patients do not suffer any side effects at all.
Rehabilitation:
Rehabilitation therapy is a very necessary part of the entire treatment regimen. The stem cell mobilization technique we use is combined with signal induction in the rehabilitation training procedure which can significantly improve the patient's neural function.
In our Rehabilitation Center we use different kinds of rehabilitation methods such as: Physical Therapy, Occupational Therapy, Speech Therapy and Traditional Chinese Medicine (TCM).
Treatment period:
The recommended period of treatment is 5-6 weeks which includes 3-4 stem cells injections.
Contraindication£º
Pituitary tumors and Adrenocortical Hyperplasia are specially treated by the doctors.
Medical plan procedure:
1. Use some medications to clean the waste/toxic substances accumulated in the brain and improve the brain¡¯s internal microenvironment so that it is suitable for the subsistence of stem cells.
2. Neural stem cells implantation. Subarachnoid cavity injection
3. Signal inductive treatment to contribute to the stem cells fixing and planting in a particular location. This is according to the theory of immunology and endocrinology.
4. Prompt stem cells differentiation: To assist the neural precursor cells to differentiate into neurons, oligodendrocytes and neurogliocytes and to repair the damaged brain cells.
5. Form a remolding of the neural network (medication treatment with the assistance of effective rehabilitation) to promote the formation and normal functioning of the new neural network.
Written by Dr. Like Wu and Dr. Xiaojuan Wang
