Batten Disease

by Drs.Like Wu, Xiaojuan Wang, Bo Cheng, Susan Chu, Xiaoling Ren and Xinrui Xi

Wu Medical Center, Bejing, China

About BD

Batten's Disease (BD) is a rare, fatal, autosomal recessive inherited disease affecting 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; 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 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

Early symptoms of this disorder usually appear at age 4-10, with a gradual onset of vision problems, including eye discoloration as a milky fog gloss over the eyes, or seizures. Early signs may be subtle personality and behavior changes, slow learning or regression, repetitive speech or echolalia, clumsiness, or stumbling. There may be slow 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, grinding of the teeth, 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 by ages 8 to 15.


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 medicines 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 patients' 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 patients receive the treatment, the better effect they can get. 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 Cell Therapy

1. Before the injection of 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 building-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 at first doctors will give patients a 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 make full advantage of the clinical effect. And with the cooperation of the medication and the stem cells treatment, the patients can get the greatest improvement. 

2. Neural stem cell injection

As an inherited disease (meaning the patient has a genetic defect), patients suffering from Batten's disease cannot use their autologous stem cells to do the stem cells treatment. Our doctors will advise neural stem cells treatment. The doctor will implant the neural stem cells through the SCF (spinal cord fluid) by means of a lumbar puncture. Each injection has 1 million stem cells. The cells involved in the injection are safe to young patients because it has been calculated accurately beforehand. This procedure is short and simple, and it usually is done with local anesthesia. Yet, in order to prevent any risk of infection we maintain a strictly sterile environment. The procedure is done in the operating room by the experienced neurology doctor only.

Neural stem cells are multipotent stem cells that can differentiate and develop into a variety of cell types, including neurons and neural ganglia cells and the new neurons and neural ganglia cells have normal functioning such as secreting some neuro-factors essential to maintaining normal activity of the brain. Those 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 tests according to our highly strict regulations. So the advantages are: Firstly, they are convenient and timesaving because they do not have to be cultured for a long time. Secondly, the neural stem cells are safe and secure in clinical therapy. In addition, those stem cells have good biological activity and have little chance of bringing an immune response (immunological rejection) to the patients.

The possible side effects of stem cells treatment

The procedure can cause minor side effect for 2 to 3 days after the stem cells injection, such as 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.


Rehabilitation therapy is very much a 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 functions.

In our Rehabilitation center we use different kind of rehabilitation methods such as: Physical Therapy, Occupational Therapy, Speech Therapy and Traditional Chinese Medicine (TCM).

Treatment period

The recommended period of treatment is 4 weeks which includes 4 stem cells injections.


Pituitary tumor and Adrenocortical Hyperplasia are pointed specially by our doctors.


1. Use some medicines to clean the waste/toxic substance accumulated in the brain and improve the brain 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 stem cells fixed planting in a particular location. This is according to the theory of immunology and endocrinology.

4. Prompt stem cells differentiation.  Help the neural precursor cells differentiate into neurons, oligodendrocytes and neurogliocytes and repair the damaged brain cells.

5. Form a remolding of neural networks (medical treatment with assistance of effective rehabilitation), to promote normal functioning of the new neural network.

Related Information:

Case Analysis for BD

BD Patient Stories

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