In a groundbreaking move toward advancing treatment options for cutaneous sarcoidosis, the Milken Institute Science Philanthropy Accelerator for Research and Collaboration (SPARC), in collaboration with the Ann Theodore Foundation (ATF), has unveiled a novel funding initiative aimed at propelling a Phase 2 clinical trial focusing on mechanistic target of rapamycin (mTOR) inhibitors. This strategic program, designated as the ATF Sarcoidosis Inhibitor of mTOR (SIM), earmarks up to $575,000 over two years to empower researchers independently poised to design and execute clinical investigations assessing the efficacy of mTOR inhibitors in cutaneous sarcoidosis.
Cutaneous sarcoidosis, an inflammatory granulomatous disease, manifests as epidermal and dermal lesions and stands as the second most affected organ system following the pulmonary sphere in sarcoidosis pathology. Characterized by the accumulation of noncaseating granulomas, this disorder imparts significant morbidity due to multisystem involvement and, in the context of cutaneous disease, results in painful rashes, disfiguring lesions, and potentially persistent subcutaneous nodules. Current pharmacotherapy remains palliative and symptom-targeted, mainly utilizing corticosteroids and immunosuppressants, none of which achieve disease modification or halt progression.
The impetus for the ATF-SIM initiative stems from promising preliminary data, including a pivotal 2024 small-scale clinical trial published recently, which highlights the potential of mTOR inhibitors as a viable therapeutic class targeting pathogenetic pathways intrinsic to sarcoidosis granuloma formation and persistence. This trial evaluated sirolimus, a potent mTOR inhibitor approved for other inflammatory and proliferative disorders, on a cohort of ten cutaneous sarcoidosis patients; seven demonstrated significant and sustained amelioration of clinical manifestations following treatment. This efficacy signal paves the way for rigorous phase 2 exploration to substantiate these outcomes across a broader population.
mTOR inhibitors target the mTOR signaling pathway, a central regulator of cell growth, metabolism, and immune responses implicated in various inflammatory conditions. Dysregulation of this pathway in sarcoidosis contributes to the pathological activation and maintenance of granulomas, thus making it a rational and innovative pharmacologic target. By impeding downstream mTOR activity, these agents may disrupt granuloma cellular proliferation and cytokine milieu, potentially translating into clinical remission and tissue healing.
One of the strategic goals behind funding ATF-SIM is rectifying the historic underinvestment in sarcoidosis research. Despite sarcoidosis’s debilitating disease burden and often progressive course leading to organ dysfunction, research funding has lagged substantially compared to other inflammatory diseases. The Milken Institute and ATF have committed to changing this landscape through their complementary funding schemes, of which ATF-SIM is the latest installment. Previous initiatives, including ATF-LOMAS and ATF-BSI, have already funneled over $11 million into sarcoidosis research, fostering scientific discovery and clinical innovation.
Melissa Stevens, Executive Vice President of Strategic Philanthropy at the Milken Institute, underscored the urgency of filling therapeutic gaps for sarcoidosis patients. She emphasized that advancing mTOR inhibitors through late-stage clinical trials is critical to securing regulatory approval and integrating these agents into standard treatment algorithms. The multipronged philanthropic approach by ATF and Milken Institute epitomizes a strategic model of translational science support — bridging early-stage molecular insights to tangible clinical interventions.
Moreover, the ongoing accumulation of evidence regarding mTOR inhibition in sarcoidosis intersects with biomolecular research that elucidates immune dysregulation patterns. Emerging genomic and proteomic data underscore altered signaling pathways central to immune cell metabolism and granuloma sustainability, giving context to mTOR’s translational relevance. These mechanistic revelations propel the compelling rationale for targeted intervention with mTOR inhibitors, which could redefine treatment paradigms for sarcoidosis and related granulomatous diseases.
Lisa Spalding, spokesperson for the Ann Theodore Foundation, highlighted the foundation’s steadfast commitment to a continuum of research funding — from early scientific discovery through to pragmatic clinical trials such as the ATF-SIM. She affirmed that aggressive pursuit of new therapies for cutaneous sarcoidosis mirrors the foundation’s ethos of community-driven impact, aiming to alleviate the chronic, often unpredictable symptoms patients endure daily.
The competitive grant application window for ATF-SIM remains open until April 20, 2026, inviting submissions from biomedical investigators with the competence and vision to undertake this critical clinical trial. Recipients will be announced in June 2026, marking a pivotal point in accelerating the clinical validation of mTOR-based therapies for cutaneous sarcoidosis.
This emerging therapeutic focus not only has the potential to transform clinical outcomes but also represents a significant advance in precision medicine approaches within inflammatory dermatology. By moving beyond nonspecific anti-inflammatory treatments toward mechanistically-grounded interventions, the ATF-SIM initiative champions a paradigm shift that could inspire analogous efforts across complex immune-mediated diseases.
The Milken Institute’s leadership in orchestrating SPARC’s efforts situates them at the forefront of medical philanthropy that prioritizes scalable, impactful research investments. Their integrative strategy leverages scientific rigor and philanthropic capital effectively to catalyze breakthroughs with tangible patient benefits. Aligning with partner foundations such as ATF, Milken underscores how cross-sector collaboration is instrumental in overcoming entrenched barriers to drug development for neglected diseases like sarcoidosis.
As further insights emerge from the planned Phase 2 trial, the sarcoidosis research community anticipates a critical influx of robust clinical data that may support regulatory submissions in the near future. Such progress holds promise to substantially alter the treatment landscape and enhance quality of life for thousands affected by cutaneous and systemic sarcoidosis worldwide.
For additional details and application guidelines for the ATF Sarcoidosis Inhibitor of mTOR funding opportunity, interested researchers and stakeholders can visit the Milken Institute’s dedicated platform, which offers comprehensive resources and contact information.
Subject of Research: Cutaneous sarcoidosis and therapeutic evaluation of mTOR inhibitors in clinical trials
Article Title: Milken Institute and Ann Theodore Foundation Initiate Novel mTOR Inhibitor Funding Program to Transform Cutaneous Sarcoidosis Treatment
News Publication Date: February 20, 2026
Web References:
– https://milkeninstitute.org/content-hub/rfps/request-proposals-ann-theodore-foundation-sarcoidosis-inhibitor-mtor-sim-trial
– https://pubmed.ncbi.nlm.nih.gov/38267106/
– https://milkeninstitute.org/content-hub/research-and-reports/reports/sarcoidosis-giving-smarter-guide
References: See linked clinical trial and Milken Institute resources above
Keywords: Clinical studies, Cutaneous sarcoidosis, mTOR inhibitors, Phase 2 clinical trial, Sarcoidosis research funding, Inflammatory diseases, Mechanistic target of rapamycin, Sirolimus, Biomedical research philanthropy, Disease-modifying treatments
