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Oral DMSO Treatment for Lipoid Proteinosis: Efficacy, Side Effects, and Alternatives

How effective is oral DMSO treatment for lipoid proteinosis. What are the potential side effects and risks of using DMSO. Are there alternative treatments available for lipoid proteinosis. How is lipoid proteinosis diagnosed and managed long-term. What is the prognosis for patients with this rare genetic disorder.

Understanding Lipoid Proteinosis: A Rare Genetic Disorder

Lipoid proteinosis, also known as Urbach-Wiethe disease, is an uncommon autosomal recessive disorder characterized by the deposition of hyaline material in the skin, mucous membranes, and internal organs. This rare condition affects both males and females equally and typically manifests in early childhood.

Key features of lipoid proteinosis include:

  • Thickening and scarring of the skin and mucous membranes
  • Hoarseness due to laryngeal involvement
  • Beaded papules along the eyelid margins
  • Neurological symptoms in some cases

The exact prevalence of lipoid proteinosis is unknown, but it is considered a very rare disorder with only a few hundred cases reported in medical literature. Early diagnosis and appropriate management are crucial for improving patients’ quality of life.

The Role of DMSO in Treating Lipoid Proteinosis

Dimethyl sulfoxide (DMSO) has been explored as a potential treatment option for lipoid proteinosis due to its penetrating and anti-inflammatory properties. However, the efficacy of oral DMSO treatment remains controversial.

Does oral DMSO treatment provide significant benefits for patients with lipoid proteinosis? The study by Ozkaya-Bayazit et al. suggests that long-term oral DMSO treatment may not be effective in managing the symptoms of lipoid proteinosis. The researchers treated three patients (two sisters and an unrelated man) with oral DMSO at a dose of 60 mg/kg/day for an average of 3 years.

Key findings from the study include:

  • No beneficial effects were observed on skin lesions, mucosal involvement, or hoarseness
  • One patient experienced disease progression, with worsening hoarseness and onset of dyspnea
  • The treatment was ultimately discontinued due to lack of efficacy

These results contrast with an earlier case report by Wong and Lin, which reported a remarkable response to oral DMSO treatment in a patient with lipoid proteinosis. This discrepancy highlights the need for further research to determine the true efficacy of DMSO in treating this rare disorder.

Potential Side Effects and Risks of Oral DMSO Treatment

While DMSO is generally considered safe for topical use, oral administration may carry certain risks and side effects. Patients considering DMSO treatment for lipoid proteinosis should be aware of these potential complications.

Common side effects of oral DMSO may include:

  • Garlic-like breath odor and taste
  • Nausea and gastrointestinal discomfort
  • Skin irritation or rash
  • Headache
  • Dizziness

More serious risks associated with long-term DMSO use may include:

  • Liver and kidney damage
  • Changes in blood cell counts
  • Allergic reactions
  • Potential interactions with other medications

Given the limited evidence supporting its efficacy and the potential for side effects, oral DMSO treatment for lipoid proteinosis should be approached with caution and under close medical supervision.

Alternative Treatment Options for Lipoid Proteinosis

As the search for effective treatments for lipoid proteinosis continues, researchers have explored various alternatives to DMSO. Some promising options include:

Retinoids

Oral retinoids, such as acitretin, have shown potential in managing lipoid proteinosis symptoms. A case report by Bakry et al. described successful treatment of two Egyptian patients with acitretin, resulting in improvement of skin lesions and overall quality of life.

Laser Therapy

Carbon dioxide (CO2) laser treatment has been used to address specific skin lesions associated with lipoid proteinosis. This approach may be particularly helpful for managing localized symptoms and improving cosmetic outcomes.

Cryosurgery

Shirani reported the successful use of cryosurgery with liquid nitrogen to remove lip lesions in a patient with lipoid proteinosis. This technique may offer a minimally invasive option for managing specific manifestations of the disorder.

Surgical Interventions

In cases where lipoid proteinosis affects the airways or other vital structures, surgical removal of infiltrates may be necessary. For example, the study by Ozkaya-Bayazit et al. mentioned that one patient required surgical removal of vocal cord infiltrates due to worsening hoarseness and dyspnea.

While these alternative treatments show promise, it’s important to note that their efficacy may vary among individuals, and long-term studies are needed to establish their safety and effectiveness in managing lipoid proteinosis.

Diagnosis and Long-Term Management of Lipoid Proteinosis

Accurate diagnosis and comprehensive long-term management are essential for patients with lipoid proteinosis. The diagnostic process typically involves:

  • Clinical evaluation of characteristic symptoms
  • Skin biopsy to confirm the presence of hyaline deposits
  • Genetic testing to identify mutations in the ECM1 gene
  • Imaging studies to assess internal organ involvement

Long-term management of lipoid proteinosis often requires a multidisciplinary approach, involving dermatologists, otolaryngologists, neurologists, and other specialists as needed. Key aspects of management include:

  • Regular monitoring of disease progression
  • Symptomatic treatment of specific manifestations
  • Addressing complications, such as airway obstruction or neurological symptoms
  • Psychological support and counseling
  • Genetic counseling for affected families

How often should patients with lipoid proteinosis undergo follow-up evaluations? While there is no standardized protocol, most experts recommend regular check-ups every 6-12 months, with more frequent visits if new symptoms arise or complications develop.

Prognosis and Quality of Life for Patients with Lipoid Proteinosis

The prognosis for patients with lipoid proteinosis is generally favorable, as the condition is typically slowly progressive and does not significantly affect life expectancy. However, the impact on quality of life can be substantial, depending on the severity and extent of symptoms.

Factors influencing prognosis and quality of life include:

  • Age of onset and rate of disease progression
  • Extent of skin and mucosal involvement
  • Presence of neurological symptoms
  • Access to appropriate medical care and treatments
  • Psychosocial support and coping mechanisms

While a cure for lipoid proteinosis remains elusive, ongoing research and advancements in treatment options offer hope for improved symptom management and quality of life for affected individuals.

Future Directions in Lipoid Proteinosis Research and Treatment

As our understanding of lipoid proteinosis continues to evolve, researchers are exploring new avenues for treatment and management of this rare disorder. Some promising areas of investigation include:

Gene Therapy

With the identification of mutations in the ECM1 gene as the underlying cause of lipoid proteinosis, gene therapy approaches may offer potential for targeted treatment in the future.

Novel Drug Therapies

Researchers are investigating new compounds that may help prevent or reduce the accumulation of hyaline material in affected tissues. These could potentially offer more effective treatment options than currently available therapies.

Improved Diagnostic Tools

Advancements in imaging techniques and genetic testing may lead to earlier and more accurate diagnosis of lipoid proteinosis, potentially improving outcomes through earlier intervention.

Patient Registries and Collaborative Research

The establishment of international patient registries and collaborative research networks can help pool data on this rare disorder, facilitating larger studies and more rapid advancements in treatment.

What role can patients and families play in advancing lipoid proteinosis research? Participation in clinical trials, patient support groups, and advocacy efforts can contribute significantly to progress in understanding and treating this rare condition.

As research continues, it is hoped that more effective and targeted treatments for lipoid proteinosis will emerge, improving the lives of those affected by this challenging disorder.

[Oral DMSO therapy in 3 patients with lipoidproteinosis. Results of long-term therapy]

Case Reports

. 1997 Jul;48(7):477-81.

doi: 10.1007/s001050050613.

[Article in

German]

E Ozkaya-Bayazit 
1
, G Ozarmağan, C Baykal, T Uluğ

Affiliations

Affiliation

  • 1 Dermatologische Abteilung, Medizinischen Fakultät Istanbul der Universität, Istanbul.
  • PMID:

    9333627

  • DOI:

    10.1007/s001050050613

Case Reports

[Article in

German]

E Ozkaya-Bayazit et al.

Hautarzt.

1997 Jul.

. 1997 Jul;48(7):477-81.

doi: 10.1007/s001050050613.

Authors

E Ozkaya-Bayazit 
1
, G Ozarmağan, C Baykal, T Uluğ

Affiliation

  • 1 Dermatologische Abteilung, Medizinischen Fakultät Istanbul der Universität, Istanbul.
  • PMID:

    9333627

  • DOI:

    10.1007/s001050050613

Abstract

Lipoid proteinosis is a rare autosomal recessive disorder with a chronic, benign course. There is no generally accepted systemic therapy apart from the experimental oral use of dimethyl sulphoxide (DMSO) and etretinate in two single cases. We treated two sisters and an unrelated man with lipoid proteinosis with longterm oral DMSO (60 mg/kg/d). At the end of an average treatment time of 3 years, DMSO was withdrawn because it produced no beneficial effects with regard to their skin, mucosal lesions or hoarseness. Additionally, one patient showed progression of her disease with worsening hoarseness and onset of dyspnea, requiring surgical removal of vocal cord infiltrates. Three patients with lipoid proteinosis failed to show any beneficial response to long term treatment with DMSO.

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  • Acitretin treatment for lipoid proteinosis.

    Gündüz O, Sahiner N, Atasoy P, Senyücel C.

    Gündüz O, et al.
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DMSO (Dimethyl Sulfoxide): Uses, Benefits, Risks, and More

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Medically reviewed by Angelica Balingit, MD — By Jessica DiGiacinto and Joe Bowman — Updated on May 16, 2023

DMSO is a colorless chemical solvent that may have many medical uses but is currently only approved by the FDA to treat interstitial cystitis.

The story of dimethyl sulfoxide (DMSO) is an unusual one. This by-product of the paper making process was discovered in Germany in the late 19th century. It’s a colorless liquid that gained notoriety for its ability to penetrate the skin and other biological membranes.

Scientists discovered that they could use DMSO as a transportation device to pass small molecules through skin in the 1960s. Since then, scientists have researched the potential benefits and risks of using DMSO to treat a variety of conditions. This research is ongoing.

DMSO was approved by the Food and Drug Administration (FDA)to treat interstitial cystitis (a chronic bladder issue) under the brand name RIMSO-50.

The compound has no other approved uses, but it’s been purported to be a treatment for:

  • arthritis
  • cancer
  • chemotherapy side effects
  • general pain

Because it absorbs easily into the skin, it’s also been studied as a vehicle for administering topical drugs.

In the late 70s, the FDA approved DMSO to help treat interstitial cystitis. It remains the only FDA-approved bladder installation (or bladder wash) for this condition. For individuals living with interstitial cystitis, DMSO has been shown to:

  • ease pain due to the condition
  • help relax the bladder
  • increase bladder capacity

When it comes to off-label uses, DMSO is often employed as an alternative treatment to reduce inflammation and pain.

Because it absorbs easily into the skin, DMSO may be a beneficial alternative to other pain medications. However, further investigation into this area is needed before any conclusions can be drawn.

DMSO has also been touted for its ability to reduce the amount of leakage during chemotherapy administration, but more studies, and real-world usage, need to be done before it can be labeled as a trusted method.

Additionally, there has been some research into DMSO’s benefits when it comes to inhibiting cancer cells. A 2020 study published in the Journal of Medical Discovery found evidence of benefit. However, research is just beginning in this area, so many more studies need to be done before any conclusions can be made.

While many of the reported side effects of taking DMSO are mild, the amount of DMSO someone takes is directly correlated to the severity of the reaction.

One common side effect is the taste of garlic in the mouth and throat.

More severe side effects include:

  • headache
  • nausea
  • vomiting
  • stomach ache
  • diarrhea
  • fever
  • chills
  • a lowered heart rate
  • itching
  • rash
  • rough or thickened skin

Risks

Because it’s seen as a more alternative treatment, DMSO is easy to find and buy online. However, buying this product and using it without a healthcare professional’s supervision could increase the likelihood of overuse.

DMSO may also increase the effect of a few medications, which could produce serious reactions in some people. A few medications DMSO may affect include:

  • sedatives
  • blood thinners
  • steroids

DMSO can be administered

  • topically, via a gel or solution
  • as a bladder wash, via a catheter (for interstitial cystitis)

As with any alternative treatment, it’s always advised to talk with a doctor before deciding to purchase any product that contains DMSO. Dosage is directly connected to the severity of possible side effects.

Dimethyl sulfoxide (DMSO) is a chemical solvent that is sometimes used to help reduce inflammation and pain, and may also be beneficial in reducing leakage during chemotherapy treatment.

It has been FDA approved to treat only one condition: interstitial cystitis.

Because of possible interactions with other common medications, and lack of definitive research into its benefits, DMSO should not be used without medical supervision.

Last medically reviewed on February 1, 2022

How we reviewed this article:

Healthline has strict sourcing guidelines and relies on peer-reviewed studies, academic research institutions, and medical associations. We avoid using tertiary references. You can learn more about how we ensure our content is accurate and current by reading our editorial policy.

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  • Understanding unapproved use of approved drugs “off-label.” (2018).
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  • Wengström Y, et al. (2008). European oncology nursing society extravasation guidelines.
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Our experts continually monitor the health and wellness space, and we update our articles when new information becomes available.

Current Version

May 16, 2023

Written By

Jessica DiGiacinto, Joe Bowman

Edited By

Jessica DiGiacinto

Copy Edited By

Delores Smith-Johnson

Feb 1, 2022

Medically Reviewed By

Angelica Balingit, MD

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Medically reviewed by Angelica Balingit, MD — By Jessica DiGiacinto and Joe Bowman — Updated on May 16, 2023

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What does dimexide treat? | Zdorovye

Dimexide has a number of features that will help to consider this remedy as one of the key products in the treatment of oncological diseases. This compound is a true free radical fighter and detox product. Dimexide is able to penetrate through cell tissues, delivering the necessary substances in preparations, has an anti-cancer effect and, in combination with other anti-cancer drugs, gains more strength.

Dimexide (Dimethylsulfoxide, DMSO, (CH₃)₂SO) has a number of features that make it one of the key products in cancer therapy. This compound is a true free radical fighter and detox product. Dimexide is able to penetrate through cell tissues, delivering the necessary substances in preparations. Dimexide has an anti-cancer effect and, in combination with other anti-cancer drugs, gains more strength.

Dimexide is a safe product. It has been used by a large number of patients in the United States and beyond, and not a single case of death or any serious injury has been recorded.

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Side effects

  • if 60-90% dimexide is applied to the palm, the skin may be wrinkled for several days.
  • Garlic-like body odor and mouthfeel are all a specific metabolite of dimexide: dimethyl sulfide, a constituent of onion and garlic flavor. Drinking a significant amount of drinking water will help dissipate unwanted odor.
  • indigestion, headaches, nausea are detox reactions. If dimexide is diluted with water, heat is released. This reaction is not dangerous.

Be careful! Industrial dimethyl sulfoxide solution contains acid and acetone impurities. Similar DMSO is strongly discouraged . Acetone is extremely harmful to the body. Prolonged contact with acetone leads to disruption of the liver and death in some cases. Dimethyl sulfoxide should be purchased for medical manipulations exclusively from trusted manufacturers.

An undiluted solution of dimethyl sulfoxide has a solid state of aggregation if it is in the refrigerator (not in the freezer) for two hours. Undesirable impurities do not have the opportunity to freeze during this period. Turning the vial over, you can see how streams of poisonous liquid will seep through the ice. It is necessary to use only ice in a bottle.

In order to return dimexide to a liquid state, it is necessary to carefully heat the vial with the contents in a water bath (t water – about 60 ° C). When about half of the contents are unfrozen, the vial should be removed from the bath. It is dimethyl sulfoxide, almost odorless. The agent at a concentration of 26% inhibits the development of various microorganisms in the blood of cancer patients. In oncology practice, the ability of DMSO to deliver drugs with an antitumor effect to the tumor and accumulate in it is exploited.

Sulfur (S)

When salt-laden ocean water becomes gaseous, it oxidizes to dimethyl sulfoxide. Further, obeying the water cycle in nature, it passes into the composition of atmospheric moisture and later clouds form from it and from them water falls to the Earth’s surface in the form of precipitation. Dimethyl sulfoxide is present in every cell.

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Sulfur (S) as a chemical element is involved in a wide range of chemical reactions involving the detoxification of drugs and other toxic compounds. Dimexide has up to forty pharmacological possibilities that are potentially useful in the treatment of a number of diseases. Garlic is an active agent precisely because of the presence of sulfur in this plant. Therefore, dimethyl sulfoxide is also effective.

  • The body uses sulfur, constantly generating cells instead of old ones.
  • Sulfur (S) is needed to maintain the permeability of cell membranes.
  • Sulfur (S) works in the formation of connective tissue, victory over inflammatory processes, support of immune defense.
  • Lack of sulfur (S) is a problem because this element is lost during the cooking process.

Sulfur deficiency symptoms are prolonged wound healing, brittle nails and hair, digestive dysfunction, inflammation, lung problems, weakened immune system, arthritis, acne, depression, memory loss.

Therapeutic possibilities of dimethyl sulfoxide

Dimexide has the most diverse therapeutic effect.

Anti-pain, anti-inflammatory, anti-virus, anti-fungal, vasodilator, effective against free radicals, immune defense optimization, diuretic. The agent transports drugs through cell membranes, against blood clots, works as a tranquilizer, activates wound healing, takes part in the synthesis of interferon (proteins that fight viruses), enhances the action of antibacterial substances.

Free radicals and dimethyl sulfoxide

DMSO, as already mentioned, is one of the effective means of fighting free radicals. Certain molecules in the human body are unstable and this causes them to destroy cells. Dimexide is attached to these molecules and removes them from the body. DMSO replaces cellular water and thus heals diseased cells by killing free radicals. All this helps to use dimexide in the treatment of cancer and a number of other diseases.

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Transport and strengthening

Dimethyl sulfoxide acts as a transport for other chemical compounds, medicines, enhances their effect. Some drugs, when diluted with DMSO, can be used at lower dosages without weakening their therapeutic effect.

Immunity and Dimethyl Sulfoxide

DMSO strengthens the immune defense by activating the production of white blood cells and macrophages. The tool stimulates macrophages in such a way that they turn into the most destructive for harmful microorganisms and malignant cells. DMSO reduces allergic manifestations by opening cell membranes and acts to attach antigens to cell receptors that stimulate the production of antibodies. This mechanism forms immunity to infections and the development of neoplasms.

Anti-freeze capability

Dimethyl sulfoxide exhibits anti-freeze properties. It protects against damage caused by low air temperatures. Prior to the discovery of dimexide, there was no way to preserve organs without forming ice crystals that destroyed tissues. Thus, the storage of organs for the purpose of transplantation is a type of Dimexide application.

Detoxification

Once in the body, heavy metals are extremely difficult to remove and pose a threat to health. Dimexide acts in this case as a means of detoxification. Sulfur is a micronutrient, which has already been mentioned, a key component of some amino acids, which binds to heavy metals and removes them with urine, defecation and sweat. DMSO (intravenous) clears amyloid proteins that trigger Alzheimer’s disease.

How to use Dimethyl Sulfoxide

It is recommended to use Dimexide (DMSO) only under the supervision of experienced professionals. Thoroughly wash the area on the skin where the product will be applied. The concentration of the agent and dosage vary. White-skinned, fair-haired people are most sensitive to DMSO. For these individuals, any concentration should be 50% or less, especially in the area around the face and neck. The agent applied to the skin is finally absorbed after 4-8 hours. Taken orally, DMSO has a peak in the blood after 4 hours.

Surface application: Liquid form is the most common form of Dimexide application. It makes sense to start surface manipulations of DMSO from low concentrations. The skin must be prepared: clean, dry and undamaged. Concentrations of about 50% are applied to the face and neck area. In areas with reduced circulation, the concentration of the agent is below 70%.

Oral: Oral dosage is 1-2 teaspoons per day. Dimeksid can be mixed with tomato/grape juice. You need to start taking with 1/2 teaspoon of dimexide and gradually build up.

Intramuscular administration of dimexide solutions, intravenous injections, intravenous drips are also possible.

Good to know! DMSO has properties that make it an indispensable agent in the treatment of persons affected by accidents, sudden heart attacks.