Is It a Wonder Drug or Snake Oil?

Sufferers of a slew of debilitating diseases claim that low doses of naltrexone is healing them

Photo: Björn Forenius/Getty

In October 2003, Linda Elsegood was diagnosed with secondary progressive multiple sclerosis, and told that doctors could do nothing more for her.

“That was devastating,” she says. “I was living, but it couldn’t be called living.”

Then she found out about a treatment using a low dose of a drug called naltrexone (LDN) from other MS sufferers. Elsegood found a prescribing doctor, and began taking it. “After three weeks, LDN tuned in that television set. I could see properly. I could begin to hear better. Cognitively, I could start to understand. It was amazing. Totally, totally amazing.”

Annabel Ince was diagnosed with Hailey-Hailey disease (HHD) 45 years ago. HHD is a genetically inherited disease that causes breaks and blisters in the skin. Traditionally treated by antibiotics and steroids, Ince found that, after a number of years, her treatment was causing more side effects than benefits.

“After nearly losing it with a life-destroying outbreak, going on for five years, and getting hopeless treatment from all the specialists that I went to, my son persuaded me to be more proactive,” she says. Ince studied research by an Italian rare disease team, who found that magnesium and vitamin D-3 helped some HHD sufferers, and then joined a Facebook HHD group where she found out about LDN. “After five months on LDN — and I did have a severe reaction at the start — I have been free of HHD for over a month and a half.”

After her own experience, Linda Elsegood set up the LDN Research Trust, hoping that if she spread the word, other people could reap the benefits. The Trust now holds international conferences for medical doctors, maintains a database of prescribing doctors, and hosts online forums for users. Yet despite being a low-cost, potentially high impact drug, why have so few doctors heard of it?

Naltrexone is in a class of drugs called antagonists, which block the activity of other drugs, some naturally occurring hormones, catecholamines, peptides, and neurotransmitters. It emerged as part of research to try and synthesize a non-morphine opiate in the 1940s, when two scientists from the pharmaceutical company Merck discovered a substance — which they called nalorphine — that seemed to reverse the effects of morphine. In 1963, a pure morphine-blocking drug called naloxone was patented, and in 1967, scientists discovered an oral analog now called naltrexone.

Naltrexone works by blocking opiate receptors. At low doses, naltrexone tricks the body into producing more endorphins, which can kill pain in the peripheral and central nervous systems, as well as elevate and regulate mood. Research conducted in 1985 showed that endorphins could also regulate and balance immune systems, and in recent years LDN has been found to reduce inflammation.

The first clinician to explore the immunomodulatory effects of naltrexone was Dr. Bernard Bihari in 1985 in New York. When he used LDN on advanced cases of AIDS, none of the patients taking it developed opportunistic infections. In a larger patient group, he demonstrated that LDN could prevent the destruction of the immune system and be used alongside antiretroviral drugs. From this point on, a few clinicians began prescribing LDN.

While naltrexone has been approved for opiate addiction by regulatory bodies in the U.S. and the U.K., it can only be used “off-label” for other conditions. This means that individual medical practitioners may decide to prescribe it in low doses, but it has not been officially approved for other uses. But because of the huge variety of diseases that have some immune or inflammatory-related component to them, LDN potentially has a wide-ranging impact. Elsegood created the LDN Research Trust in 2004 to help clinicians and users share information as the drug began to treat more conditions.

“Initially, we were just saying it’s for autoimmune disease,” says Elsegood, “but now we know it’s for chronic pain, infertility, autism, depression, post-traumatic stress and other mental health conditions, and cancers.” LDN is often used to help infectious diseases like Lyme and Epstein-Barr, both of which can trigger autoimmunity. Evolving medical research and clinical experience show that its effects on autoimmune diseases appears to only work at low doses, typically between .5mg and 4.5mg — and each patient needs to experiment to find the right dose.

Dr. Phil Boyle, who runs the Neo Fertility clinic in Dublin, gives LDN to about 50 percent of couples who see him for infertility and recurrent miscarriage. He says that: “If a patient has clinical endorphin deficiency with fatigue, PMS and painful periods, clinically they commonly improve with LDN treatment and they are then more likely to achieve a successful pregnancy.” He says he has used LDN with patients for about 10 years, and argues it is both safe and improves pregnancy outcomes.

Dr. Brian Udell, Medical Director of the Child Medical Center in Florida, uses LDN to treat autism. Predicated on a body of evidence which posits that excessive opioid peptides are found in autism, Udell and user groups suggest improvements in clarity and mood. Udell argues that “70 percent of parents either love it or like the treatment, 25 percent say that they don’t see a difference (but may have better immune functioning — less illness — so it is difficult to judge). Five percent of patients do not tolerate LDN due to increased agitation or negative behaviors.”

Clinical observation, small-scale trials and user feedback show that, while LDN has pretty good odds, it doesn’t work for everyone. Many users report improvements in mood, energy, and even pain relief with continued LDN use. Some have also observed that abnormalities, such as antibodies in their blood, have been resolved. Two patient surveys by Skips Pharmacy showed that approximately 80 percent of patients had not experienced any deterioration of their symptoms and 60 to 95 percent had some symptom relief.

Researchers simply don’t yet know why LDN doesn’t work for 20 to 40 percent of users, or why it improves pain and fatigue in some users, but not others. The answer could be that LDN only works in combination with other factors such as lifestyle and diet, and an additional regime of pharmaceutical medications might also be needed. Some users report unpleasant side effects, including more pain, insomnia, anxiety, and flare-ups of their disease, though it can be unclear whether these symptoms are due to the drug or the progress of their disease.

But with a success rate of anything from 50 percent to 80 percent, particularly for hard to treat systemic conditions, why isn’t LDN attracting more interest from mainstream medicine?

Medically, we simply do not yet know if LDN is a wonder drug or snake oil. The FDA offers a mild caution with regards to “off-label” use, saying that clinicians use drugs for other conditions if other medications don’t exist for specific conditions or haven’t worked. And often, people with complex autoimmune, reproductive, or genetic conditions are in this position, so they’re willing to take the risk.

The doctors and specialists I spoke to either hadn’t heard of it, or know of Naltrexone only as a treatment for alcohol and opiate addiction. “Due to the medication being readily available and inexpensive, there seems to be a lack of interest on the part of pharma to study it,” says Udell.

But individual clinicians and the LDN Research Trust are campaigning for large-scale medical trials to establish its efficacy. LDN seems less “alternative medicine snake oil” and more a conventional medicine that needs systematic testing. A low-cost, relatively safe, and possibly effective drug for many different diseases has to merit greater attention. Maybe it’s time for government and pharma to step up.