5 most unusual treatments approved by the FDA

Here’s a fun fact: Maggots have been used to treat wounds practically since the beginning of time. But did you know that maggot therapy is actually approved by the FDA?

As you are probably aware, the FDA was established way back when—in 1930—as the gatekeeper for the promotion of safe and effective drugs. Amendments to the Federal Food, Drug, and Cosmetic Act later established well-controlled trials as the standard for evidence to evaluate new drugs. In recent years, faster approval of antineoplastic drugs and biologics has been a trend at the FDA.

But some of the drugs and devices approved at the FDA may not be what you’d expect. The following are five unusual FDA approvals: 

MDMA

The nightclub drug MDMA (3-4 methylenedioxymethamphetamine) may have found a home in conventional medicine. In early 2020, the FDA granted MDMA, commonly known as ecstasy or molly, an emergency-use authorization for clinical use in those with treatment-resistant post-traumatic stress disorder (PTSD), with results of phase III trials expected in 2021. MDMA-assisted psychotherapy involves the administration of up to three doses of the drug in a controlled clinical setting to boost the efficacy of psychotherapy in people with PTSD. 

“The resurgence of research into using drugs such as MDMA to catalyze psychotherapy is the most promising and exciting development I’ve seen in my psychiatric career,” stated Michael Mithoefer, MD, acting medical director for the Multidisciplinary Association for Psychedelic Studies (MAPS) Public Benefit Corporation. “Combining the powerful effects of pharmacology with the potential depth of psychotherapy is a compelling model for harnessing advances in neuroscience and psychopharmacology without ignoring the complexity, richness and innate capacity of the human psyche.”

Of note, MAPS is a nonprofit aimed at promoting the therapeutic use of psychedelics and medical marijuana.

CBD

In 2018, the FDA approved Epidiolex (cannabidiol) [CBD] oral solution to treat seizures secondary to Lennox-Gastaut syndrome and Dravet syndrome, both rare forms of epilepsy. The drug can be taken in patients aged 2 years or above, and is the first FDA-approved drug containing a purified compound derived from marijuana. Unlike tetrahydrocannabinol (THC), CBD doesn’t cause euphoria.

FDA Commissioner Scott Gottlieb, MD, was hopeful about the future of marijuana derivatives to treat disease, while striking a cautionary tone. “We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high quality products. But, at the same time, we are prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims. Marketing unapproved products, with uncertain dosages and formulations can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases,” he stated.

Poliovirus

Poliovirus has been eradicated in North and South America since 1994, but it’s making a comeback—albeit in a clinical sense. 

In May 2016, the FDA granted a breakthrough therapy designation for oncolytic polio/rhinovirus recombinant (PVSRIPO), which is a modified poliovirus, in patients with recurrent malignant glioblastoma (GBM). Currently, the drug is in phase II trials at four sites.

In a study expanding on phase I clinical trials published in the New England Journal of Medicine, researchers found that after treatment, survival in participants plateaued at 21% at 24 months, with the result sustained at 36 months. “Intratumoral infusion of PVSRIPO in patients with recurrent WHO grade IV malignant glioma confirmed the absence of neurovirulent potential. The survival rate among patients who received PVSRIPO immunotherapy was higher at 24 and 36 months than the rate among historical controls,” they concluded.

In plain terms, researchers found the outcomes outstanding for the treatment of this notoriously lethal cancer. 

“We’ve never seen survival rates like this before. The long-term survival is very exciting,” said Andrew E. Sloan, MD, Department of Neurosurgery at Case Western Reserve University School of Medicine, Cleveland.

SER-109 BLA

The mere mention of Clostridium difficile (CDI) will run a shiver down any clinician’s spine. But, new hope comes in the form of SER-109 BLA, which was granted breakthrough therapy designation in 2020 by the FDA. 

This drug is a biologically sourced microbiome that, according to manufacturer Seres Therapeutics, allows “patients to achieve a sustained clinical response by breaking the vicious cycle of CDI recurrence and restoring the diversity of the gastrointestinal microbiome.”

In phase III results, researchers found that within 8 weeks of administration, 11.1% of patients given SER-109 experienced C. difficile reinfection compared with 41.3% of those receiving placebo. 

Maggots

Maggots have been used to treat non-healing wounds for centuries, a therapy that was approved as a “medical device” by the FDA in 2004. Better yet, it is even reimbursable by the Centers for Medicare and Medicaid.

Although maggot debridement therapy (MDT) is an age-old alternative treatment, “it has re-emerged as an effective option for many wound types,” according to the authors of a review published in Chronic Wound Care Management and Research. “Even with vast advances in today’s healthcare technology, the management of chronic wounds still poses a significant challenge to the medical world.”

Medicinal maggots work by debriding the wound in order to remove tissue that can hamper the healing process. The maggots eat the dead skin and are also thought to secrete substances that fight infection.  

This treatment is considered cost-effective, well-tolerated, and efficacious, according to another review published in the Journal of Pharmacy Practice. “While maggot therapy demonstrated effectiveness in necrotic wounds, not all wound types respond well to maggot therapy,” the authors wrote.

The Chronic Wound Care Management and Research article noted that nearly all of the literature published supports the use of MDT, “however, level 1 evidence is sparse, and more work is needed to further establish the evidence base. Recent research into the cellular mechanisms of action of MDT and genetic engineering techniques may result in novel and innovative therapies in the future that have the potential to revolutionize wound healing.”