Immunotherapy’s Clinical Trial Impact In Endocrinology Is Still In Flux

Immunotherapy’s whirlwind of clinical trial successes and failures show no signs of abating.[1]

Each passing week finds more immunotherapy trials entered into the ledger, and many of these trials are now exploring new territory: endocrine related disorders. The endocrine system is a challenging environment for immunotherapies for some reasons, ranging from lack of basic research to paradoxical toxic reactions.

Despite these obstacles, there are a few promising trials using antibodies and immune checkpoint inhibitors which have a good shot at providing relief to patients with endocrine disorders or cancers of the endocrine system.


Bringing Immunotherapy To The Endocrine System

Preclinical research has focused on producing endocrine disorder targeted immunotherapies that are monoclonal antibodies, CAR-T cells, and most recently, immune checkpoint inhibitors.[2] There have also been several studies which have sought to include cell therapies with immune activators, though this will likely remain a niche approach because of the complexity of the treatment regimen.[3]

Minimizing complexity is always important in a clinical trial protocol, but immunotherapeutic trials for endocrine disorders must stress simplicity even more than usual due to the number of unknown unknowns regarding endocrine system dysfunction. While that hasn’t stopped trials from proceeding, it has made the record of recent trials quite spotty. 

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Elusive Answers In Endocrine Immunotherapies

Certain trials see inconsistent results due to unprofiled aspects of patient pathology, which makes regulators very uneasy.[4] Other preclinical data sets have also caused regulators to apply extreme scrutiny to endocrine system targeted immunotherapies—in one particularly disturbing study, immunotherapy prompted the spontaneous acquisition of an endocrine disorder.[5] Aggressive basic research to answer questions about the endocrine system will be necessary if prospective clinical trials are to recruit the patients that their immunotherapeutic intervention is the most likely to help.

Like in all immunotherapies, the patient profiles of trial recruits are especially important in immunotherapeutic endocrinology trials. Preclinical data shows that common endocrine disorders like obesity may prevent immunotherapies from working, even if they’re targeted outside the endocrine system.[6] This means that the evolving understanding of immunotherapy’s relationship with the endocrine system is broadly relevant to the entire field of immunotherapeutics.


Critical Clinical Obstacles Surpassed

Clinical trials in immunotherapy for endocrine disorders aren’t all gloomy results and unanswered questions, thankfully. Importantly, recent research and subsequent clinical trials have confirmed that immunotherapies can be effective for endocrine cancers occurring in the brain.[7] Though this is a very general finding, it’s important to remember that the entire field of immunotherapy for endocrine disorders is so new that even basic principles like efficacy at various locales are still in question.

Perhaps the best news is that immunotherapy trials are making promising strides forward in treating obesity.[8][9] Though researchers still have to iron out some pre-clinically identified kinks, there’s reason to hope that immunotherapy could deliver a cure.[10]

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