Dimethyl Sulfoxide (DMSO)
and Down Syndrome
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by Len Leshin, MD, FAAP
Copyright 1998, All rights reserved
Go to List of Past Abstracts Dimethyl sulfoxide, or DMSO, is an industrial solvent produced during the process of making paper from trees. In 1959, researchers found that it could protect tissues from the damaging effects of freezing, and it became used in animal research. In 1963, Dr. Stanley Jacob, a researcher in a cryogenics lab, announced that it was useful to treat sprains and arthritis. Since then, this product has been promoted for a wide variety of illnesses and conditions, with almost all based merely on anecdotal evidence. Research with DMSO on humans was temporarily halted in 1965 after animals treated with DMSO were found to have developed changes in the lens of the eye. Research was gradually restarted after no evidence was found of eye changes in humans who had received DMSO previously.
Send Me Email DMSO has several interesting properties that may make it useful. For instance, it has the ability to penetrate tissues including intact skin, and carry along with it a variety of chemicals. It has a local analgesic affect, and inhibits certain compounds in the body called prostaglandins, thereby reducing inflammation in tissues. At the present time, the US FDA has approved only a version that is instilled into the bladder for a condition called interstitial cystitis. Current research with DMSO is targeted at head injury, spinal cord trauma, amyloidosis and scleroderma. DMSO is also currently used as a cryopreservative for bone marrow cells and stem cells.
Studies on the toxicity of DMSO in adult humans have shown no serious adverse effect when given at or below 1 g/kg body weight, with the exception of hemolysis (breaking down of red blood cells) during intravenous infusions. At doses above this, liver, kidney and intestinal damage has been noted. (Not enough studies have been done on children to determine whether that dosage is the same for them for toxicity.) The most common side effect of DMSO is the breath and body odor, which takes on a quality similar to garlic or oysters. Skin reactions are very common after application of DMSO directly to the skin. Sedation, headache, dizziness and nausea have all been described as side effects of DMSO therapy. (1,2)

In Down syndrome, the rationale given for using DMSO is as a carrier agent, in that it supposedly carries amino acids or other nutrients directly into the brain to affect cerebral metabolism. One Mexican clinic refers to this treatment as the "Weinstein-Turkel Method," based on two doctors who supposedly developed it. Interestingly enough, the only published study on this treatment doesn't mention either doctor.

In 1975, a group of doctors in Chile published a study (3) in which 31 children with Down syndrome were given DMSO and the amino acids GABA, GABOB, acetylglutamine, and arginine intramuscularly. The injections were given either daily or every other day, depending on the age of the child, for 90 days. The children then went through a "rest period" of 30 days, during which they received the amino acids orally, but no DMSO or injections of any sort. This rotation was repeated a total of 3 to 5 times, again depending on the age of the child. The children were studied with psychometric tests, physical exams and lab studies. The treated children were compared to 24 children with Down syndrome who were not given any treatments at all. It is an important point that it is almost impossible to do a blinded placebo study with DMSO because of the body odor that users invariably develop. The authors stated that the treatments improved various physical attributes of the children less than 3 years old, including the appearance of a nasal bridge, hair becoming thicker and the eyes losing their epicanthal folds. The authors also stated that the treated children had an improved developmental quotient, which included advances in motor and social skills. Complications included transient hard, non-painful nodules at the injection sites, with two reported abscesses.

This study has several methodological problems, the biggest one being that apparently the investigators were able to tell which children were treated and which weren't (that is, it was not a "blinded" study). This situation can cause a bias on the part of the investigators and may influence the results. The authors state that photos were taken, but they were not published, nor were any physical measurements of the children published. To quote the authors: "We are aware that this clinical work includes many variables, which make the exact evaluation of the experience difficult. We also believe that to attain better results it is necessary to increase the number of children treated and the length of treatment to more than two years....We plan to continue our work with DMSO-amino acids for at least two years." Unfortunately, no follow-up study was ever published.

Also in 1975, a group of researchers in Oregon, USA, published a study (4) which looked at the effects of oral DMSO (without amino acids) on a group of children diagnosed with mental retardation. The authors do not give their reasoning behind why DMSO by itself might be helpful in these cases. This study group included children with Down syndrome as well as "other types of retardation." The authors do not state how many children had Down syndrome. As with the Chilean study, the side effect of notable body odor made it impossible to set up a placebo group. Instead the authors set up a high-dose group of 34 children and a low-dose group of 33 children, with the low-dose group being just enough to give the children the characteristic odor. There was also a non-dose comparison group of 22 children. No significant side effects were reported. The authors used several different tests to measure language, motor and behavior skills; it does appear that the testers were blinded in this study. The authors state that when the testing is looked at globally, the trend was for the high-dose children to make the most gains, the low-dose children to make intermediate gains, and the non-dose children to make the fewest gains. However, this conclusion was not arrived at statistically. In fact, in language skills, the non-treated children had the most gains. And it should be stressed that the authors did not break down the results to show if children with Down syndrome had any different results from other children with mental retardation. In fact, the failure to group children by diagnosis is a major failure of this study.

Based on these two studies, treatment of children with Down syndrome with DMSO cannot be justified at this time.

References:

1. Willhite CC and Katz PI. Toxicology Update: Dimethyl sulfoxide. J Appl Toxicol 4(3): 155-160, 1984.

2. Swanson, BN. Medical use of dimethyl sulfoxide. Rev Clin Basic Pharmacol 5: 1-33, 1985.

3. Aspillaga MJ, Morizon, G, Avendano, I. Dimethyl sulfoxide therapy in severe retardation in Mongoloid children. Ann NY Acad Sci 243: 421-431, 1975.

4. Gabourie J, Becker, JW, Bateman, B, Dunn, M, Jacob, S. Oral dimethyl sulfoxide in mental retardation. Ann NY Acad Sci 243: 449-459, 1975.

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