More Links to Equipment, and Some to Experimental Treatments

http://www.child-disability.co.uk/Index.html

For anyone in the UK, the link above goes to a fantastic website aimed at giving a range of suggestions on how to make life easier and richer for a child with a disability. From things like fun at home to days out and holidays, as well as a brilliant page about equipment types. It gives the names, photos and sometimes links as well to different pieces of equipment. The tiltrite chair, portable hoists and moulded mattresses designed to help with a twisted posture particularly spring to mind.

Here are some links to more experimental treatments I have read about. Some of these may be dated but appeared to produce small improvements in the child. These reports were purely anecdotal, and so I can't validate or invalidate them. I am also not a doctor, and doctors would be more up to date about where this research has led, or at least point you in the right direction to somebody who would know.

Clorazepate - This drug belongs to a group of drugs called the benzodiazepines, and has been used experimentally in the treatment of metachromatic leukodystrophy. Its brand names are tranxene and novo-clopate, and it is also known as clorazepate dipotassium. It is a skeletal muscle relaxant, meaning it can make a person relaxed and supple. It is also an anxiolytic (reduces anxiety), anticonvulsant (reduces seizures) and sedative. It can't be used in people with impaired kidney or liver function. You may need to wait a few months to see the effects if you do end up using this drug. Tolerance and dependence do occur with frequent use, and withdrawal symptoms can occur if use is decreased suddenly. However, clorazepate has breakdown products that are still active in the body, and tolerance to these is slower. If the person takes any other sedatives these are likely to enhance the sedative effects of clorazepate. If the person having clorazepate is a child or takes any other medicines, special precautions should be taken.

Gamma globulin - This a blood protein, given in the form of injections normally to people who have been exposed to measles or hepatitis A, but has been used as an experimental treatment for metachromatic leukodystrophy. It can also be given through an IV drip. The injections or infusions temporarily boost immunity to disease which is very important in people with leukodystrophy, since small infections can quickly become life-threatening. Gamma globulin injections are also used to treat immune disorders including an auto-immune disease where the body's immune system attacks its own blood cells. It has been postulated that demyelination (which is what happens leading to loss of movement in leukodystrophy) may occur because abnormal accumulation of chemicals in the nerve insulation causes the person's immune system to attack and destroy it. Some leukodystrophies also appear to be due to the loss of the cells themselves that insulate nerves. Since gamma globulin also causes the spleen to ignore antibody tagged blood cells, it may be possible to prevent auto-immune attack of the cells that insulate nerves using gamma globulin injections, if they have been tagged by the immune system in the same way.

Hydrocortisone - This might be useful in some cases as a short term way of reducing inflammation in the brain. This may relieve to some degree, problems such as convulsions, fevers and nerve pain.

Potential Curative and Palliative Treatments, Scientific and Non-Scientific

I am not actively advocating any of these, and you should definitely talk to a doctor (or even more than one - it is your right to be able to do this), and/or homeopathic expert, and do more research before considering pursuing any of these. The purpose of these being here is to give parents, carers and in some cases patients, the widest range of choices. Leukodystrophy and the secondary problems arisinf from it are tricky to treat, and quality rather than quantity is the most important thing - but that does not always have to mean just taking a person home and loving them.

Potentially Curative
- Bone Marrow Transplant: This may be an option for very mildly symptomatic or asymptomatic metachromatic or adrenoleukodystrophy. Involves killing all the bone marrow in the body using aggressive chemotherapy (this will cause hair loss, vomiting, diahorrea, possibly fevers and rashes, immune reactions, and severe susceptibility to infections). Some young children may be at risk from just the chemotherapy. Then the donor bone marrow is dripped in through an IV line in the chest, and encouraged to grow. Infection susceptibility, graft vs. host disease, and rejection of the donor marrow can be risks for months afterwards, and the child's appearence will be very much altered (although those constantly nearby will not notice so much). Furthermore, the disease may progress for some months afterwards until the marrow cells have grown more, and in some cases transplant will fail to stop disease progression. A specific bone marrow match must also be found, either from within the family, or in the general population. However, if the procedure works, the disease will be slowed or even halted for the time being. The long term effects are not known.

- Cord blood transplant: This is in many ways similar to bone marrow transplant and is used to treat asymptomatic Krabbes Disease. The main difference is that instead of bone marrow being donated, stem cells and blood from the umbillical cord of a newborn infant donor are used. Most children who undergo this kind of transplant have been diagnosed with Krabbes Disease prenatally.

- Lorenzo's Oil: This is used to treat asymptomatic adrenoleukodystrophy, and is a blend of purified oleic acid (found in olive oil) and erucic acid (found in rapeseed oil). It stops the body producing the fatty acids it can't break down, because the same chemical that produces them breaks down oleic and erucic acid - in effect the chemical is 'distracted'. The long term effects of the oil are not known and usually if symptoms of the disease have already begun, the oil does not work. However its efficacy in treating asymptomatic ALD has been fully proven scientifically.



Palliative

- Dimethyl Sulphoxide: Studied Scientifically and reported as somewhat protecting nerves and decreasing inflammation in Krabbes disease

- Nitrizepam: Studied Scientifically and reported as being helpful in reducing irritability and increasing comfort in Krabbes disease

- Gelsemium 30: This is a homeopathic medication sometimes used in the treatment of unspecified leukodystrophies.

- Equal volumes of tincture of myrrh and olive oil, mixed in a teaspoon, heated over a small flame, and once cooled to body temperature, rubbed immediately down the muscles either side of the spine every day. It can't be stored and must be mixed fresh every time. This a supposed treatment for neurological dysfunction, but its efficacy has in no way been proven. However myrrh has long been used as a painkiller, and olive oil rubs easily into skin, because the skin is very fatty. Therefore some insulation of nerves may occur - though the effect, if any, would be extremely slight.