Sarcoidosis Research: $575K Funding for mTOR Inhibitor Clinical Trial
- A new funding program aimed at accelerating research into cutaneous sarcoidosis has been launched by the Milken Institute Science Philanthropy Accelerator for Research and Collaboration (SPARC), in partnership...
- Cutaneous sarcoidosis, an inflammatory disease, affects the skin and represents the second most commonly affected organ system in sarcoidosis, following the lungs.
- The focus on mTOR inhibitors stems from promising preliminary data, including a small clinical trial conducted in 2024.
A new funding program aimed at accelerating research into cutaneous sarcoidosis has been launched by the Milken Institute Science Philanthropy Accelerator for Research and Collaboration (SPARC), in partnership with the Ann Theodore Foundation (ATF). The program, called ATF Sarcoidosis Inhibitor of mTOR (SIM), will award a grant of up to $575,000 over two years to a biomedical researcher or team to design and conduct a Phase 2 clinical trial investigating the effectiveness of mechanistic target of rapamycin (mTOR) inhibitors in treating the condition.
Cutaneous sarcoidosis, an inflammatory disease, affects the skin and represents the second most commonly affected organ system in sarcoidosis, following the lungs. It impacts approximately a quarter of individuals diagnosed with sarcoidosis, manifesting as painful rashes, skin lesions, and subcutaneous nodules. The launch of ATF-SIM reflects a growing effort to address the historical underfunding of sarcoidosis research.
The focus on mTOR inhibitors stems from promising preliminary data, including a small clinical trial conducted in 2024. That trial demonstrated that seven out of ten participants with cutaneous sarcoidosis experienced sustained improvement in their symptoms after treatment with sirolimus, an mTOR inhibitor already approved for other inflammatory conditions. This suggests a potential for targeted therapy addressing the underlying mechanisms of the disease.
“People living with sarcoidosis, which can be extremely painful and unpredictable, currently lack the treatment options they deserve. In order to advance this potential treatment toward late-stage clinical trials and regulatory approval for use in cutaneous sarcoidosis, researchers must continue to demonstrate that an mTOR inhibitor has a significant effect in a new trial with more participants. We are excited to work with ATF to ensure the field accomplishes this next step as quickly as possible,”
Melissa Stevens, Executive Vice President of Strategic Philanthropy, Milken Institute
Currently, treatment for sarcoidosis primarily relies on general anti-inflammatory medications, such as corticosteroids and immunosuppressants, which can alleviate symptoms but do not address the root causes of the disease. The ATF-SIM program aims to validate the potential of mTOR inhibitors as a disease-modifying therapy.
This initiative builds upon previous collaborations between the Milken Institute and the ATF. Since 2020, the organizations have launched two other funding programs: ATF-LOMAS (Learning Opportunities in Medicine and Sarcoidosis) and ATF-BSI (Breakthrough Sarcoidosis Initiative). The ATF-BSI program has already committed over $11 million to sarcoidosis research. They have also published a “Giving Smarter Guide” identifying strategic opportunities for philanthropic investment in sarcoidosis.
“At ATF, we believe we owe it to the sarcoidosis community to support promising research directions at all stages of development, including this timely opportunity to accelerate a potential near-term treatment,” said Lisa Spalding, a spokesperson for the Ann Theodore Foundation. “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 yield benefits for decades to come. We must pursue all possible avenues to improve daily symptom management toward long-term symptom relief.”
Sarcoidosis is characterized by the formation of granulomas – abnormal clusters of immune cells – in various organs. While the lungs are most frequently affected, the skin is a common secondary site. The disease can cause significant morbidity due to multisystem involvement.
The ATF-SIM Trial is accepting applications until , at 11:59 PM Eastern Time. Grant recipients will be selected in . Applications will be prioritized from research teams that can demonstrate a clear plan for enrolling sufficient participants and have access to clinical trial networks or consortia.
The awarded team will be eligible for up to $250,000 in funding for direct costs per year, plus 15 percent in indirect costs, totaling $287,500 per year for a total of $575,000 over two years. This Phase 2 trial is designed to provide the scientific support needed for future Phase 2b/3 or Phase 3 trials, ultimately paving the way for potential regulatory approval of mTOR inhibitors for the treatment of cutaneous sarcoidosis.