Cancer Lab · DeCure for X

DeCure for Mixed Cell Uveal Melanoma

DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for Mixed Cell Uveal Melanoma — screening already-approved drugs against its 34-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module34 genesLead labCancer
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CancerDOID:6522$DeCureCancer

The disease map

Disease moduleMixed Cell Uveal Melanoma maps to a 34-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.

Research record

01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash

Current lead

No approved-drug candidate for mixed cell uveal melanoma is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.

Molecular view

protein tyrosine phosphatase receptor type D (PTPRD)PTPRD is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.

Loading structure…
helix sheet flcdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 2YD6 · 1.35 Å · ligand CITRATE ANION (FLC). Experimental structure, not a prediction.

What the evidence adds up to

Uveal melanoma accounts for 5% of all melanoma diagnoses in the United States, with an age-adjusted risk of 5 per 1 million population. Once metastatic, therapy options are limited and are often extrapolated from cutaneous melanoma therapies despite the routine exclusion of uveal melanoma patients from clinical trials. In a European registry study of 5,788 patients diagnosed between 1983 and 1994, five-year relative survival was 68.9% overall and remained stable with the period of diagnosis. Relative excess risk of death was 2.45 in patients aged 75 years or older compared with patients aged 54 years or younger, and was slightly higher in male patients (relative excess risk 1.10) than in female patients.

A prospective study of 167 patients treated with preenucleation radiotherapy (8 Gy in 2 fractions) between 1978 and 1992, compared with 108 historical controls treated by enucleation only, reported melanoma-related death in 32.3% of the PER-treated group and 40.7% of the enucleation-only group. Estimated 15-year survival rates for patients with melanoma were 63.7% in the PER group and 51.0% in the enucleation-only group. For patients dying of all causes, these percentages were 47.5% and 25.2%, respectively. A significant difference in survival became evident after 48 months of follow-up in favour of the PER group.

Approximately 50% of patients will develop metastatic disease, facing poor survival outcomes due to a lack of effective treatment options. A phase II study of nivolumab plus ipilimumab in 35 patients with metastatic uveal melanoma (33 evaluable) reported an overall response rate of 18%, including one confirmed complete response and five confirmed partial responses, with median progression-free survival of 5.5 months and median overall survival of 19.1 months. A separate phase II trial of the same combination in 52 treatment-naïve patients reported an ORR of 11.5%, median progression-free survival of 3.0 months, and median overall survival of 12.7 months. The 2021 review notes that there is no current standard of care for metastatic uveal melanoma and that expert guidelines recommend starting treatment on a clinical trial if possible.

What is still missing are prospective clinical trials that include uveal melanoma cohorts from the start rather than relying on retrospective series or case studies, and a deeper understanding of which patients might benefit from existing immune-based combinations. The intrinsic differences between uveal and cutaneous melanoma mean that therapies targeting skin melanoma genes are unlikely to work, and the tumour environment in uveal melanoma has been described as immune-paralysed rather than an immune desert, suggesting that different strategies for turning on an immune response are needed.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Cancer · 2016 · 446 citations · open access

Uveal melanoma: From diagnosis to treatment and the science in between

AbstractMelanomas of the choroid, ciliary body, and iris of the eye are collectively known as uveal melanomas. These cancers represent 5% of all melanoma diagnoses in the United States, and their age-adjusted risk is 5 per 1 million population. These less frequent melanomas are dissimilar to their more common cutaneous melanoma relative, with differing risk factors, primary treatment, anatomic spread, molecular changes, and responses to systemic therapy. Once uveal melanoma becomes metastatic, therapy options are limited and are often extrapolated from cutaneous melanoma therapies despite the routine exclusion of patients with uveal melanoma from clinical trials. Clinical trials directed at uveal melanoma have been completed or are in progress, and data from these well designed investigations will help guide future directions in this orphan disease. Cancer 2016;122:2299-2312. © 2016 American Cancer Society.

https://doi.org/10.1002/cncr.29727
Archives of Ophthalmology · 2008 · 119 citations · open access

Survival in Patients With Uveal Melanoma in Europe

AbstractOBJECTIVE: To estimate survival in patients in whom uveal melanoma was diagnosed between January 1, 1983, and December 31, 1994, in Europe. METHODS: Survival analysis of data from 32 cancer registries in 16 European countries adhering to the European Cancer Registry for 5788 patients with uveal melanoma diagnosed between January 1, 1983, and December 31, 1994, with follow-up to 1999. RESULTS: Five-year relative survival was 68.9% overall and remained stable with the period of diagnosis. Relative excess risk of death was 2.45 (95% confidence interval [CI], 2.10-2.86) in patients aged 75 years or older compared with patients aged 54 years or younger and was slightly higher in male patients (relative excess risk, 1.10; 95% CI, 1.02-1.19) than in female patients. Survival was similar in Nordic countries (relative excess risk, 1.03; 95% CI, 0.87-1.21) compared with the United Kingdom (reference country) and was lower in eastern and western European countries (1.26; 1.05-1.52, and 1.25; 0.90-1.60, respectively) compared with the reference country. CONCLUSIONS: In this large series of patients with uveal melanoma, 5-year relative survival remained stable with the introduction of conservative treatment in individuals in whom uveal melanoma was diagnosed between 1983 and 1994. We found differences in survival between sexes and in European areas that should be investigated in studies that consider tumor characteristics at the individual level.

https://doi.org/10.1001/archopht.126.10.1413
Archives of Ophthalmology · 2005 · 10 citations · open access

Reduced Melanoma-Related Mortality in Uveal Melanoma by Preenucleation Radiotherapy

AbstractBACKGROUND: Radiotherapy of an eye before enucleation, so called preenucleation radiotherapy (PER), of patients with uveal melanoma was initiated to reduce enucleation-induced systemic metastasis. Earlier studies with a short follow-up period have not demonstrated a significant effect on survival. OBJECTIVE: To study the effect of PER on melanoma-related mortality after more than 9 years of follow-up. DESIGN: In a prospective study, 167 patients with uveal melanoma were treated between 1978 and 1992 by irradiation with 800 rad (8 Gy) given in 2 fractions 2 days before enucleation. A group of 108 patients with uveal melanoma treated between 1971 and 1992 by enucleation only in the same hospital served as a historical control group. Patients were followed up until December 2002 or death. RESULTS: Melanoma-related death occurred in 32.3% of the PER-treated group and in 40.7% of the enucleation only group. Mean follow-up was 9.25 years. After 48 months of follow-up, a significant difference in survival became evident in favor of the PER group. The estimated 15-year survival rates for patients with melanoma in the PER group and enucleation only group were 63.7% and 51.0%, respectively. For patients dying of all causes, these percentages were 47.5% and 25.2%, respectively. In both groups, women had a better prognostic outcome than men. CONCLUSION: This study suggests that PER improves long-term survival in patients with uveal melanoma.

https://doi.org/10.1001/archopht.123.10.1363
Ophthalmic Research · 2022 · 7 citations · open access

Outcome Measures of New Technologies in Uveal Melanoma: Review from the European Vision Institute Special Interest Focus Group Meeting

AbstractUveal melanoma (UM) is the most common primary intraocular tumor in adults. New diagnostic procedures and basic science discoveries continue to change our patient management paradigms. A recent meeting of the European Vision Institute (EVI) special interest focus group was held on "Outcome Measures of New Technologies in Uveal Melanoma," addressing the latest advances in UM, starting with genetic developments, then moving on to imaging and treatment of the primary tumor, as well as to investigating the most recent developments in treating metastases, and eventually taking care of the patient's well-being. This review highlights the meeting's presentations in the context of the published literature.

https://doi.org/10.1159/000524372
Oncology Times · 2021 · 1 citations

Uveal Melanoma

AbstractUveal Melanoma: Uveal MelanomaUveal melanoma—a rare cancer that forms in the eye—remains a challenge for the field of oncology. Approximately 50 percent of patients will develop metastatic disease, facing poor survival outcomes due to a lack of effective treatment options. With no current standard of care, metastatic uveal melanoma represents a significant area of unmet need. However, there is reason for hope, according to Sapna Patel, MD, Associate Professor and Director of the Uveal Melanoma Program in the Department of Melanoma Medical Oncology at The University of Texas MD Anderson Cancer Center, Houston. “Outcomes are improving,” noted Patel, a specialist in uveal melanoma with an active clinical practice and research program. “The most recent clinical trial data and even some aggregate data of individuals who do not go on clinical trials show that patients are living past a year, which is a striking improvement from just a decade ago. “We're not at the point where we are giving patients 2 or 5 years of survival, but this extra time is invaluable to patients and progress continues,” she said. Current Approaches & Challenges Since there is currently no standard of care, expert guidelines recommend that patients with uveal melanoma start treatment on a clinical trial, if possible, according to Patel. However, therapeutic choices remain limited for patients who do not have access to this option. “Today, a lot of what we use to treat melanoma of the eye is borrowed from melanoma of the skin, but it's not a good fit,” explained Patel, noting there are two reasons for this. “For one, uveal melanoma patients have been excluded from all of the Phase III studies in skin melanoma that have led to or are in the process of being evaluated for approval. “However, when these drugs receive approval, the language doesn't necessarily exclude these patients from receiving treatment,” she elaborated. “That leaves us with a caveat noting that the effect of this therapy in uveal melanoma is unknown.” Patel emphasized the importance of including uveal melanoma in these larger studies, pointing out the FDA Oncology Center of Excellence is attuned to the efforts in rare cancers. “This involves including cohorts of rare cancers such as uveal melanoma in registrational study efforts that can be separated from the main skin melanoma cohort at the end for evaluation without penalizing the endpoint. “However, this message has not translated over to pharmaceutical companies, and large trials continue to be run without these patient cohorts,” Patel said. “As a result, we have the uveal melanoma community wondering if a treatment will work for this patient population. Without inclusion in these larger trials, it is a challenge to amass data other than inherently biased retrospective series or case studies.” Another problem with borrowing treatments from skin melanoma are the intrinsic differences between the two diseases. “Skin melanoma and uveal melanoma have different pathobiology,” Patel noted. “Therefore, any therapies that target cancer-specific genes in skin melanoma would not work for patients with uveal melanoma.” Recent Research Despite these ongoing challenges, researchers continue to dedicate significant time and effort to find a standard of care and improve outcomes for this rare disease. Patel and colleagues performed the only Phase II study in the U.S. exploring nivolumab plus ipilimumab for metastatic uveal melanoma. The findings were recently published in the Journal of Clinical Oncology (2020; doi:10.1200/JCO.20.00605). The study included 35 patients with metastatic uveal melanoma; 33 were evaluable for efficacy. Patients received nivolumab (1 mg/kg) and ipilimumab (3 mg/kg) for 4 cycles, followed by nivolumab maintenance therapy for up to 2 years. Overall response rate (ORR) was the primary outcome and data showed an ORR of 18 percent, including one confirmed complete response and five confirmed partial responses. The researchers reported a median progression-free survival of 5.5 months and a median overall survival of 19.1 months. Another trial, led by the Spanish Multidisciplinary Melanoma Group, recently released their Phase II data on nivolumab plus ipilimumab among treatment-naïve metastatic uveal melanoma patients (J Clin Oncol 2021; doi:10.1200/JCO.20.00550). Fifty-two patients were included in the study and the ORR was 11.5 percent. The median progression-free survival and overall survival were 3.0 months and 12.7 months, respectively. “These two studies validate each other,” Patel noted. “We now have prospective clinical trial data using these immune agents, and that is a start. We can now comfortably say that, if a patient cannot join a clinical trial, nivolumab plus ipilimumab is a standard of care combination that has some efficacy. While I don't think anybody would say 18 percent ORR is where we want to be, it shows promise.” Patel and colleagues are currently taking a deeper look at the responding patients in their trial, as well as the study out of Spain. “What differentiates the responders? Did their tumors at the start of treatment have a different characteristic signature?” she queried. “A deeper understanding will help us identify future patients who may or may not benefit from this treatment approach.” Ongoing Avenues of Exploration While continued study of cancer genes and the immune system are crucial to ongoing treatment advances, Patel noted that there are two other areas that should not be overlooked. First is the use of a patient's autologous T cells, according to Patel. “This comes in many different flavors, such as tumor infiltrating lymphocytes,” she said, noting the promising research in this area. At the University of Pittsburgh Medical Center, researchers are recruiting patients for a Phase II study exploring the adoptive transfer of tumor infiltrating lymphocytes for metastatic uveal melanoma (NCT03467516). Intratumoral therapy is another interesting area of study. “Improvements over the years have generated a pretty effective response in the injected tumor, but that doesn't help the patient who might have tumor in multiple locations or multiple tumors in an organ,” Patel explained. “And so, there are strategies to enhance intratumoral therapy by either changing how you deliver it, such as with a side-hole or multi-pronged needle or pressure-enabled delivery device to enhance vascular drug delivery to a tumor, or giving it with systemic checkpoint blockade or an immune-stimulating agent to enhance the benefit beyond the treated tumor(s).” These two strategies, Patel told Oncology Times, “are very exciting and can turn on that immune response for cancers like uveal melanoma where the tumor environment is not exactly an immune desert, but rather immune paralyzed.” Looking to the future, Patel is optimistic. “I've been working in uveal melanoma for over a decade, and where you used to get no attention at meetings or from pharmaceutical companies, we are now seeing more focus on this area of study,” she said, noting that the uveal melanoma TCGA has helped raise awareness of this disease and potential drug targets. “I am very optimistic and hopeful that we are on a path to make meaningful progress for these patients.” Catlin Nalley is a contributing writer. Read This Article & Earn CME or NCPD! Earn continuing education credit by completing a quiz about this article. You may read the article here or on our website, then complete the quiz, answering at least 70 percent of the questions correctly to earn credit. CONTINUING MEDICAL EDUCATION INFORMATION FOR PHYSICIANS Visit http://CME.LWW.com for more information about this educational offering and to complete the CME activity. This enduring material is available to physicians in all specialties. Lippincott Continuing Medical Education Institute, Inc., is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians. Lippincott Continuing Medical Education Institute, Inc., designates this enduring material for a maximum of 1 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. This activity expires February 28, 2023. The cost of the exam is $10. The payment covers processing and certificate fees. PROVIDER ACCREDITATION INFORMATION FOR NURSES Lippincott Professional Development (LPD) will award 1.0 contact hour for this Nursing Continuing Professional Development (NCPD) activity. LPD is accredited as a provider of NCPD by the American Nurses Credentialing Center's Commission on Accreditation. This activity is also provider approved by the California Board of Registered Nursing, Provider Number CEP 11749 for 1.0 contact hour. LWW is also an approved provider by the District of Columbia, Georgia, and Florida CE Broker #50-1223. Visit www.nursingcenter.com for more information and to complete the NCPD activity. Fee: $12.95. Deadline: March 3, 2023 For nurses who wish to take the test for NCPD contact hours, visit www.nursingcenter.com. Learning Objectives for This Month's Activity: After participating in this activity, readers should be better able to: 1. Explain research challenges to establishing a standard of care for the treatment of patients with uveal melanoma. 2. Analyze results from two phase II clinical trials evaluating the use of nivolumab and ipilimumab in the treatment of uveal melanoma. Disclosure: The author(s), faculty, staff, and planners, including spouses/partners (if any), in any position to control the content of this activity, have disclosed that they have no financial relationships with, or financial interests in, any commercial companies relevant to this educational activity.

https://doi.org/10.1097/01.cot.0000737688.11182.56

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.

DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.