Document text
Principal Investigator: Joshua Alfaro
Organization: PHARMAIN CORPORATION
Fiscal Year: 2024
Award: $995,099
Funding agency: National Cancer Institute
Osteosarcoma (OSA) is the most common primary malignancy of bone in children and
adolescents, accounting for approximately 5% of all childhood tumors (www.cancer.gov).
Osteosarcoma occurs primarily in the long bones of the limbs, including most commonly the
femur, tibia, and humerus1. The current standard of care therapy for human patients diagnosed
with OSA includes multi-agent neoadjuvant chemotherapy (doxorubicin + cisplatin +/-
methotrexate) followed by surgical resection of the primary lesion and adjuvant chemotherapy,
curing approximately 60 to 70% of patients 1–3. However, up to 40% of newly diagnosed patients
presenting with localized disease eventually develop metastases to the lung, indicating that
many patients already have micrometastases present in the lung at the time of diagnosis or
develop them during the perioperative period 4. The 5-year survival rate for children with
metastatic OSA is extremely low, hovering around 20%-30%, a rate that has not improved in
over 30 years 2. Clearly, novel therapies are in critical need, specifically molecules capable of
inhibiting the process of OSA metastasis. PharmaIN is developing immunomodulating peptide
with unique anti-tumor and anti-fibrotic properties for the treatment of osteosarcoma and other
cancer types. The peptide’s anti-tumor activity has been demonstrated as a monotherapy and
combination with other immuno/chemotherapies in multiple cancer models, including OSA
metastatic model. Pilot tox data has been obtained and PharmaIN proposes to conduct a clinical
trial in canine OSA patients as a translation tool for human and concurrent product development
for veterinary applications. The additional proposed work is IND enabling for a Phase 1 human
clinical trial and consists of manufacturing drug substance and drug product needed for GLP-tox
and clinical studies, completion of GLP-tox studies and submission of the IND for the OSA
indication.
Terms: <0-11 years old><14-Hydroxydaunomycin><Abscission><Accounting><Acute><Adjuvant Chemotherapy><Adjuvant Drug Therapy><Adolescent><Adolescent Youth><Adriamycine><Assay><Bioassay><Biological Assay><Bone Sarcoma><C-Type Natriuretic Peptide><CDDP><CNP-22><Cancer Model><CancerModel><Cancers><Canine Species><Canis familiaris><Chest><Child><Child Youth><Childhood Neoplasm><Childhood Tumor><Children (0-21)><Cis-diammine-dichloroplatinum><Cis-diamminedichloridoplatinum><Cis-diamminedichloro Platinum (II)><Cis-dichloroammine Platinum (II)><Cis-platinous Diamine Dichloride><Cis-platinum II><Cis-platinum II Diamine Dichloride><Cisplatin><Cisplatina><Cisplatinum><Clinical><Clinical Research><Clinical Study><Clinical Trials><Common Rat Strains><Cyclic GMP><Cysplatyna><Data><Data Set><Development><Diagnosis><Dichlorodiammineplatinum><Disease><Disorder><Dogs><Dogs Mammals><Dose><Doxorubicin><Doxorubicina><Drugs><Engineering><Excision><Extirpation><Feedback><Femur><Formulation><Freeze Drying><Freeze Dryings><Freezing><Funding><Guanosine Cyclic Monophosphate><Human><Hydroxyl Daunorubicin><Hydroxyldaunorubicin><Immunomodulation><In Vitro><Induction Therapy><Lead><Life><Liquid substance><Localized Disease><Lung><Lung Respiratory System><Lyophilization><MICMET><Malignant Neoplasms><Malignant Tumor><Measures><Medication><Metastasis><Metastasis to the Lung><Metastasize><Metastatic Lesion><Metastatic Mass><Metastatic Neoplasm><Metastatic Neoplasm to the Lung><Metastatic Osteogenic Sarcoma><Metastatic Osteosarcoma><Metastatic Tumor><Metastatic Tumor to the Lung><Methods><Methotrexate><Methotrexate Methylaminopterin><Methotrexatum><Metotrexato><Micrometastasis><Micromets><Modeling><Modern Man><NEOADJ><Neoadjuvant><Neoadjuvant Therapy><Neoadjuvant Treatment><Neoplasm Metastasis><Newly Diagnosed><Operative Procedures><Operative Surgical Procedures><Osseous Sarcoma><Osteogenic Sarcoma><Osteosarcoma><Patients><Pb element><Pediatric Neoplasm><Pediatric Tumor><Peptides><Perioperative><Peyrone's Chloride><Peyrone's Salt><Pharmaceutical Preparations><Phase><Platinum Diamminodichloride><Population Control><Primary Lesion><Process><Progressive Disease><Property><Rat><Rats Mammals><Rattus><Removal><Reporting><SBIR><Sampling><Secondary Neoplasm><Secondary Tumor><Skeletal Sarcoma><Small Business Innovation Research><Small Business Innovation Research Grant><Specific qualifier value><Specified><Surgical><Surgical Interventions><Surgical Procedure><Surgical Removal><Survival Rate><Thorace><Thoracic><Thorax><Time><Translations><Work><analytical method><antifibrotic agent><antifibrotic medication><antifibrotic therapy><antifibrotic treatment><bone><cGMP><cancer metastasis><cancer type><canine><chemotherapy><cis dichlorodiammineplatinum><cis platinum compound><cis-Diaminedichloroplatinum><cis-Diamminedichloroplatinum><cis-Diamminedichloroplatinum(II)><cis-Dichlorodiammineplatinum(II)><cis-Platinum><developmental><domestic dog><drug/agent><extremity bone><fluid><heavy metal Pb><heavy metal lead><humerus><immune modulation><immune regulation><immunologic reactivity control><immunomodulatory><immunoregulation><immunoregulatory><improved><induction therapies><juvenile><juvenile human><kids><limb bone><liquid><long bone><lung metastasis><malignancy><manufacture><meeting><meetings><metastasize to the lung><neoplasm/cancer><new drug treatments><new drugs><new pharmacological therapeutic><new therapeutics><new therapy><next generation therapeutics><novel drug treatments><novel drugs><novel pharmaco-therapeutic><novel pharmacological therapeutic><novel therapeutics><novel therapy><osteochondrosarcoma><osteoid sarcoma><product development><pulmonary><pulmonary metastasis><resection><standard of care><success><surgery><tibia><tool><translation><tumor><tumor cell metastasis><tumors in children><youngster>