calcitriol (BPM 31543) / BPGbio - LARVOL DELTA

A Phase I safety study of topical Calcitriol (BPM 31543) for the prevention of chemotherapy-induced alopecia (CIA) (ESMO 2017) - P1; "Patients were managed with adequate safety monitoring and pharmacokinetic (PK) analysis in order to determine levels of exposure. The potential efficacy (secondary objective) of the topical calcitriol was evaluated by photographic assessment using a Canon digital camera system (to ensure standardization and uniformity among all enrolled patients) in addition to patient self-assessments."

HEOR • P1 data • Breast Cancer • Gynecologic Cancers • Sarcoma

A phase I safety study of topical calcitriol (BPM 31543) for the prevention of chemotherapy-induced alopecia (CIA). (ASCO 2017) - P1; "Data have shown that the twice daily application of BPM 31543 in patients receiving taxane-based chemotherapy was safe and well-tolerated. The Maximum Tolerated Dose (MTD) was not detected. No Dose Limiting Toxicities (DLTs) or severe treatment-related adverse events occurred in any of the dose cohort."

HEOR • P1 data • Biosimilar • Breast Cancer • Gynecologic Cancers • Pain • Sarcoma

[VIRTUAL] Parkinsonism Due to Basal Ganglia Calcifications Due to Pseudohypoparathyroidism and Fahr’s Disease: A Case Report (AAPMR 2021) - "Given parkinsonian symptoms, he was also started on Sinemet, which improved his bradykinesia and rigidity... Individualized care consisting of appropriate medication management and tailored rehabilitation are needed to care for patients with Fahr’s disease. Additionally, discussing goals of care and understanding family dynamics in these patients serve to improve overall functional outcomes and improve patient quality of life"

Clinical • CNS Disorders • Developmental Disorders • Endocrine Disorders • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Psychiatry • Rare Diseases • Renal Disease

A phase I safety study of topical calcitriol (BPM31543) for the prevention of chemotherapy-induced alopecia (CIA): Final study results (ESMO 2018) - P1; "BPM31543 applied topically twice daily to the scalp in patients receiving taxane-based chemotherapy is safe and well-tolerated with no apparent differences in safety between doses. Here, no DLT was observed at up to 80 µg/mL and MTD was not reached. Some efficacy was detected at each dose."

Clinical • P1 data • Breast Cancer • Gynecologic Cancers • Sarcoma

Recurrent drug sensitivity patterns in myelodysplastic syndrome patients are recapitulated by ex vivo drug response profiling (AACR 2018) - "...Furthermore, while the resistant cluster showed increased resistance to most drugs, some drugs seemed to have increased activity in this cluster, including calcitriol and poly ADP ribose polymerase (PARP) inhibitor rucaparib. This unique platform, applied to predict ex vivo therapeutic response of MDS patient samples to various classes of drugs, recapitulates known clinical and molecular predictors of therapeutic response, and possible new therapeutic targets. These data suggest the possible utility of using this methodology to aid decision making for therapeutic selection in the management of MDS patients."

PARP Biomarker • Myelodysplastic Syndrome

A phase I safety study of topical calcitriol (BPM31543) for the prevention of chemotherapy-induced alopecia. (PubMed, Breast Cancer Res Treat) - "BPM31543 applied topically twice daily to the scalp is safe and well tolerated in patients receiving taxane-based chemotherapy. No DLT was observed at up to 80 µg/mL, and MTD was not reached. Based on the data from this trial, BPM31543 represents a promising therapy and warrants further investigation in Phase 2/3 trials."

Clinical • Journal • P1 data • Alopecia • Breast Cancer • Gynecologic Cancers • Oncology • Pain • Sarcoma • Solid Tumor

"First time a therapy has shown overall survival benefit in second line #bladdercancer. Awesome work! @OncoBellmunt @cnsternberg @DrChoueiri" - apolo_andrea

Biosimilar • Bladder Cancer • Oncology • Urothelial Cancer

A Feasibility Study of Biologically Focused Therapy for Myelodysplastic Syndrome Patients Refractory to Hypomethylating Agents (ASH 2019) - "MTB recommendations varied widely among patients and encompassed various drug classes including targeted therapies (venetoclax, sorafenib, lenalidomide, ruxolitinib, midostaurin, everolimus), cytotoxic agents (cytarabine, fludarabine), differentiative agents (calcitriol, ATRA), HMAs, and androgens (danazol) as single agents or in combination...Personalized treatment recommendations accurately predicted clinical responses in vivo and enabled some patients to be bridged to allogeneic HCT. Randomized prospective trials are needed to determine whether this approach may improve outcomes for patients with HMA-refractory MDS."

Clinical • DNMT3A • SRSF2

Effects of vitamin D3 and its chemical analogs on the growth of Hodgkin's lymphoma, in vitro. (PubMed, BMC Res Notes) - "nuVDR for DMSO control and VD3 was comparable. These results suggest that VD3 or VDAs may affect growth of HL."

Journal • Preclinical

Unusual Presentation of Large Parathyroid Adenoma with Acute Cerebellar Ataxia in a Pediatric Patient (ENDO 2019) - "...On exam, she was not acutely ill, she had normal deep tendon reflexes, but sustained tetany of the quadriceps noted when reflexes were elicited; gait was wide-based and stiff-legged, favoring the left side.Laboratories:Calcium: 16.5mg/dlPhosphorus: 3.1 mg/dl (3.1-5.9)PTH: 2417 pg/ml (12-72)Vit D 25 OH: 19 ng/ml (≥30)Urine Ca/Cr: 0.93Neck and parathyroid ultrasound: a 1.9 x 1.7 x 1.5 cm solid mass was noted at left inferior thyroid lobe suggestive of a parathyroid adenoma.Sestamibi (99mTc) scan: intense focus of radiotracer uptake inferior to the left lobe of thyroid gland.Hospital Course: Patient admitted to ICU and treated with IV fluids, calcitonin and zoledronic acid... This case illustrates a rare neurological presentation of hypercalcemia in a child. The diversity and subtle development of symptoms contributed to the delay in diagnosis. Although mechanisms of this association are unknown, hyperparathyroidism mimics motor neuron disease and skeletal muscle is a..."

Clinical • Late-breaking abstract

Atypical Parathyroid Adenoma- Is There More Than What Meets the Eye ? (ENDO 2019) - "This is an unusual presentation of severe hypercalcemia in a young woman due to primary hyperparathyroidism. Concerning features for possible parathyroid carcinoma included the patient’s young age, a corrected calcium level >14 mg/dL, a markedly elevated PTH, a parathyroid mass >3 cm, and the combination of renal and bone involvement.1 These factors prompted the resection of adjacent lymph nodes with the parathyroid mass. Pathology was suggestive of an atypical parathyroid adenoma."

Atypical Parathyroid Adenoma- Is There More Than What Meets the Eye ? (ENDO 2019) - "This is an unusual presentation of severe hypercalcemia in a young woman due to primary hyperparathyroidism. Concerning features for possible parathyroid carcinoma included the patient’s young age, a corrected calcium level >14 mg/dL, a markedly elevated PTH, a parathyroid mass >3 cm, and the combination of renal and bone involvement.1 These factors prompted the resection of adjacent lymph nodes with the parathyroid mass. Pathology was suggestive of an atypical parathyroid adenoma."