BREAKING NEWS: Ampligen Slows Melanoma in Mice

The elusive ME/CFS, AIDS & cancer drug continues to stumble toward acceptance

On July 24, a press release from AIM ImmunoTech Inc. announced the publication of new pre-clinical (laboratory) data revealing that the company’s drug Ampligen, along with other therapies, helped slow tumor cell growth and improved survival in a mouse model of the deadly skin cancer melanoma.

The combination therapy used in the study included “dendritic cell-based vaccines” as well as Ampligen-containing chemokine modulators. (1)

Dendritic cells are a type of immune system cell. The job of dendritic cells is to present antigenic material—toxins or foreign substances, including infections—to T-cells so they will stimulate an immune response against it. (2)

In the press release, AIM Medical Officer David Strayer, MD, stated, “This new pre-clinical data further demonstrates Ampligen’s therapeutic potential when used with dendritic-cell vaccines, with anti-PD-L1 checkpoint inhibitors, or in combination with both. The data supports AIM’s belief that we are on the right path in our ongoing development of Ampligen as an anti-tumor therapy.” (1)

The new research paper was published in the journal Vaccines. (3)

Ampligen, at this point, is a very old drug that appears to be efficacious in a wide number of diseases—including ME/CFS, AIDS, Long Covid, and various cancers—but has never attained Food and Drug Administration (FDA) approval as an official treatment for any of them. First developed in the 1960s, this double-stranded RNA compound was observed to inhibit tumor growth. It also interacts with immune complexes known as cytokines, including interferon and several interleukins.

Ampligen is characterized as an immune modulator. It’s a particular type of RNA, the sister molecule to DNA. It appears to be generally safe; the most frequent side effect reported is a mild flushing reaction. Other, rarely reported side effects include chills, fever, fatigue, and disturbances in the ratios of various types of blood cells.

And for the last three decades, the drug has been something of a Holy Grail for ME/CFS patients: Amid published reports of striking clinical improvement in people with ME/CFS during treatment, Ampligen remains largely unavailable outside of extremely limited clinical trials.

Despite those clinical trials consistently showing that the health of ME/CFS patients—and patients with other immune system disorders—improves significantly during Ampligen treatment, the FDA has not approved the drug to treat any of these immune system conditions.

For more than 30 years, Ampligen has been developed and tested by one corporation, the name of which has changed over the decades. First established as HEM Pharmaceuticals, its name subsequently became Hemispherx Biopharma and it’s now known as AIM ImmunoTech Inc.  

One of the first immune diseases Ampligen was tested on was AIDS. In 1986, HEM funded a small pilot study to treat ten patients who tested positive for HIV and the results, subsequently published in the British medical journal The Lancet, generated tremendous excitement. (4)

”Just about everyone was talking about Ampligen,” Kevin Armington, of Gay Men's Health Crisis in New York, told reporter Mindy Kitei. The drug induced surprising energy increases, and treated patients had fewer swollen lymph nodes and night sweats. (5)

One of the first ME/CFS patients treated with Ampligen was Nancy Kaiser in Albuquerque, New Mexico. She’d consulted more than 200 doctors, most of whom told her she was imagining her symptoms or malingering. By the time she found Dr. Daniel Peterson in Nevada—one of the two doctors who first identified the 1984 CFS outbreak in Incline Village, Nevada—Kaiser was so weak she couldn’t walk, she’d experienced a precipitous drop in IQ and was having seizures. After a course of treatment with Ampligen, Kaiser experienced a remarkable recovery: her IQ increased, her seizures ended, and she was able to return home to her family. (6)

The first clinical trial of Ampligen treatment in ME/CFS patients was green-lighted in 1990 by the FDA as a 92-person double-blind study that would last 48 weeks. Hoping to get fast-track approval for Ampligen as several AIDS treatments had, HEM Pharmaceuticals ran afoul of regulators by cutting the trial time in half without the FDA approving the new trial parameters. When HEM submitted the Ampligen data to the FDA, the agency ruled that the data were incomplete and inadequate, requiring more. Even after more data were supplied, FDA refused fast-track approval. And the CFS patients in the trial, who’d been told they’d receive Ampligen free after the trial ended, were left without any treatment at all. (5)

Regulatory and clinical progress stalled. Fast forward to 2020: AIM’s scientists had decided to assess the effectiveness of Ampligen in treating cancer, and in December 2020, Ampligen was awarded Orphan Drug status for treating pancreatic cancer. (7)

This FDA program provides “orphan status” to drugs and biologics that are intended for the treatment, prevention or diagnosis of a rare disease or condition. “Rare” is defined as a disease that affects fewer than 200,000 people in the United States—“or meets cost recovery provisions of the Act,” as AIM’s press release explained. (7)

Pancreatic cancer is deadly; it has a five-year survival rate of only six percent, according to the Pancreatic Cancer Action Network.

When Ampligen was tested against a control group matched for age, gender, stage of disease, and previous treatment, Ampligen-treated pancreatic cancer patients had an overall survival rate about 200% higher than the controls.

“This study data demonstrates that Ampligen has the potential to extend the survival rates of people suffering with pancreatic cancer significantly when compared to the traditional standard of care for this deadly disease,” commented AIM’s CEO Thomas K. Equels when the Orphan Drug status was announced. (7)

It took about two more years, but on April 2, 2022, the Annual International Hepato-Pancreato-Biliary Association World Congress accepted AIM’s abstract describing this pancreatic cancer triumph—an important step in gaining academic respect for Ampligen and, hopefully, spurring more research. (8)

Ampligen’s promising results in the melanoma mouse model will hopefully lead to safety trials in humans. And maybe a cancer researcher or two will circle back to the original immune system diseases treated with Ampligen—ME/CFS, AIDS and more recently, Long Covid—and design clinical trials that will settle the ME/CFS and Ampligen question once and for all.

Author’s Note:  If you want to know more about the history of Ampligen, check out my book Ampligen: The Battle for a Promising ME/CFS Drug on Amazon.

BIBLIOGRAPHY
1.        “AIM Announces New Positive Data on Ampligen’s Anti-Tumor Potential When Used as Part of a Combination Therapy.” AIM corporate press release, July 24, 2024. https://aimimmuno.com/aim-announces-new-positive-data-on-ampligens-anti-tumor-potential-when-used-as-part-of-a-combination-therapy/
2.        Neenyah Ostrom. “Danger to the Defenders of Immunity.” SubStack, November 11, 2022. https://immuneillnessreport.substack.com/p/danger-to-defenders-of-immunity-is
3.        Taylor JL, Kokolus KM, Basse PH, Filderman JN, Cosgrove CE, Watkins SC, Gambotto A, Lowe DB, Edwards RP, Kalinski P, et al. “Therapeutic Anti-Tumor Efficacy of DC-Based Vaccines Targeting TME-Associated Antigens Is Improved When Combined with a Chemokine-Modulating Regimen and/or Anti-PD-L1.” Vaccines. 2024; 12(7):777. https://doi.org/10.3390/vaccines12070777
4.        Carter, William et al.; “Clinical, Immunological, and Viro­logi­cal Effects of Ampligen, a Mismatched Double-Stranded RNA, in Patients with AIDS or AIDS-Related Complex”; The Lancet, p. 1228, 1987.
5.     Kitei, Mindy; “The AIDS Drug No One Can Have”; Philadelphia Magazine, October 1994, pp. 94-105.
6.     Ostrom, Neenyah; Interview with Nancy Kaiser, published in the New York Native, #367, 1990.
7.     “AIM ImmunoTech Inc.’s Drug Ampligen Awarded FDA’s Orphan Drug Designation Status for the Treatment of Pan­creatic Cancer.” AIM ImmunoTech Press Release, Decem­ber 21, 2020. https://aimimmuno.com/press-release/aim-immunotech-inc-s-drug-ampligen-awarded-fdas-orphan-drug-designa­tion-status-for-the-treatment-of-pancreatic-cancer/
8.     “AIM ImmunoTech Announces Abstract Accepted for Presentation at 15^th Annual IHPBA World Congress. https://aimimmuno.com/press-release/aim-immunotech-announces-abstract-accepted-for-presentation-at-15th-an­nual-ihpba-world-congress/