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Novel Use of Acthar Gel and (Purported) Oremepermin For Pharmacologic Control and Reversal of Total Limbal Stem Cell Defifiency Limbsl

John C Affeldt, MD


John C Affeldt, MD



Loma Linda University Eye Institute

Purpose: The successful commercial introduction of Oxervate for pharmacologic treatment of neurotrophic keratitis (NK) has generated a wave of potential next generation NK agents. One of these is topical epitheliotropic Oremepermin-α, which is currently in Phase 1/2 clinical trials.

Total Stage III limbal stem cell deficiency (TLSCD) is the most serious and extreme of the 6 clinically defined stages of LSCD. Control, reversal and ultimate visual rehabilitation of TLSCD is extremely difficult, and the available interventions are all surgical with no primary pharmacologic option.

Acthar gel, a corticotropin immunomodulator, has been FDA approved since 1952 for a broad spectrum of severe systemic and ocular inflammatory diseases.

We now report for the first-time successful pharmacologic control and reversal of both Stage II NK and TLSCD with the novel combination of Acthar Gel and (purported) Oremepermin-α.

Methods: Retrospective observational case report

Results: A 39 yo CF was referred for acute vision loss OU x5 weeks. Her past ocular history was significant for ~20 years of soft contact lens (SCL) wear OU. Presenting BCVA was 20/100 OD and 20/250 OS. Corneal sensation by Cochet Bonnet Esthesiometry measured 15mm OU. Initial diagnosis included (1) severe contact lens related keratitis OU, (2) Stage I NK OD (3) Stage II NK OS (4) LSCD OU; OS>OD, and (5) significant, associated ocular inflammation OU.

Forwarding treatment included immediate cessation of all SCL wear, aggressive lubrication, prophylactic topical antibiotics OS, a full 8-week course of Oxervate OU, and initiation of Acthar Gel 80u 2x/week. The NK and LSCD quickly resolved OD, but OS remained unchanged and refractory to all treatment for the next 17 months.

On 4/11/22 OS was entered into the Claris Bio Oremepermin-α NK clinical trial, and exited 10 weeks later with full resolution of stage II NK. Unfortunately, the TLSC remained persistent and unchanged. Follow up over the next 4 months however revealed progressive regression of the TLSCD to Stage I LSCD (no central corneal involvement) with a concomitant 7-line BCVA improvement to 20/40.

Conclusion: The unexpected reversal of a long-standing Stage III TLSCD to Stage I LSCD using the novel combination of Acthar Gel and (purported) Oremeparin-α suggests that for the first time a nonsurgical pharmacologic treatment option for TLSCD may exist.


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