Milken Institute and Ann Theodore Foundation Launch $575,000 Grant for mTOR Inhibitor Trial in Cutaneous Sarcoidosis
核心洞察
The Milken Institute (搜索) and Ann Theodore Foundation (搜索) have launched ATF-SIM, a new funding program offering up to $575,000 for a Phase 2 clinical trial evaluating mTOR (搜索) inhibitors in cutaneous sarcoidosis (搜索).
The initiative builds on promising 2024 trial results where seven of 10 participants with cutaneous sarcoidosis (搜索) experienced sustained symptom improvement after treatment with sirolimus, an mTOR inhibitor (搜索).
Cutaneous sarcoidosis (搜索) affects approximately one-quarter of sarcoidosis (搜索) cases and currently lacks disease-modifying treatments, with only general anti-inflammatory medications available for symptom management.
The Milken Institute (搜索) Science Philanthropy Accelerator for Research and Collaboration (SPARC), in partnership with the Ann Theodore Foundation (搜索) (ATF), has launched a new funding program to advance clinical research in cutaneous sarcoidosis (搜索). The ATF Sarcoidosis (搜索) Inhibitor of mTOR (搜索) (SIM) program will award one two-year grant of up to $575,000 to support a Phase 2 investigator-initiated clinical trial evaluating mechanistic target of rapamycin (搜索) (mTOR) inhibitors for treating this challenging inflammatory condition.
Addressing Critical Treatment Gaps
Sarcoidosis (搜索) is a debilitating and sometimes fatal inflammatory condition marked by abnormal clusters of immune cells in various organs. The skin represents the second-most affected organ after the lungs and is involved in approximately one-quarter of sarcoidosis cases. Cutaneous sarcoidosis (搜索) manifests through painful rashes, skin lesions, and subcutaneous growths, significantly impacting patients' quality of life.
Currently, patients with sarcoidosis (搜索) face limited therapeutic options. The only US Food and Drug Administration-approved pharmaceuticals commonly used to treat the condition are general anti-inflammatory medications that may mitigate symptoms but cannot address the disorder's underlying causes. This treatment landscape represents a significant unmet medical need for disease-modifying therapies.
Promising Preliminary Evidence
The funding initiative builds on encouraging results from a small 2024 clinical trial that demonstrated the potential of mTOR (搜索) inhibitors in cutaneous sarcoidosis (搜索). In this preliminary study, seven of the 10 participants with cutaneous sarcoidosis experienced sustained symptom improvement after completing treatment with sirolimus, an mTOR inhibitor (搜索) already approved for use in other inflammatory conditions. These results suggest that mTOR inhibitors, which target a specific sarcoidosis (搜索)-related molecular pathway, may offer meaningful therapeutic benefits.
"People living with sarcoidosis (搜索), which can be extremely painful and unpredictable, don't currently have the treatment options they deserve," said Melissa Stevens, executive vice president of strategic philanthropy at the Milken Institute (搜索). "In order to advance this potential treatment toward late-stage clinical trials and regulatory approval for use in cutaneous sarcoidosis (搜索), researchers must continue to show that an mTOR inhibitor (搜索) has a significant effect in a new trial with more participants."
Comprehensive Research Strategy
ATF-SIM represents the third sarcoidosis (搜索)-related funding program launched by the Milken Institute (搜索) and ATF partnership, reflecting their commitment to addressing the historical precedent of sparse funding for sarcoidosis research. Since 2020, the organizations have established two other programs: ATF-LOMAS (Learning Opportunities in Medicine and Sarcoidosis) and ATF-BSI (Breakthrough Sarcoidosis Initiative), with the latter committing over $11 million to sarcoidosis research to date.
"At ATF, we believe that we owe it to the sarcoidosis (搜索) community to support promising research directions across all stages of development, including this timely opportunity to accelerate a potential treatment in the near term," said Lisa Spalding, spokesperson for the Ann Theodore Foundation (搜索). "ATF-SIM augments—but does not supplant—our parallel efforts to support early-stage biomedical research and long-term scientific community-building through two other grant programs, which will reap benefits for decades to come."
Application Timeline and Next Steps
The funding program is currently accepting applications through April 20, 2026, at 11:59 pm Eastern Time, with grant awardees expected to be selected in June 2026. The initiative aims to validate the therapeutic potential of mTOR (搜索) inhibitors through a larger, more robust clinical trial that could provide the evidence needed to advance toward late-stage clinical development and potential regulatory approval for cutaneous sarcoidosis (搜索) treatment.