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Osteosarcoma – A Rare but Dangerous Pediatric Cancer Osteosarcoma is a form of bone cancer, occurring in all age groups, but most often in teenagers and young adults. Although rare, osteosarcoma is the most common of all childhood bone cancers, with approximately 450 new cases occurring each year in the U.S. in adolescents and very young adults. The primary site is predominantly in one of the long bones, typically the femur. Prior to diagnosis, most patients complain of pain and swelling at the tumor site, but is usually and initially attributed to “growing pains� or mild trauma. On average, diagnosis is made approximately 4 months after the onset of symptoms. . Modern treatment of osteosarcoma requires both systemic chemotherapy and surgery. Until the introduction of modern, limb-sparing, surgical techniques, amputation of the affected limb was necessary. Even with modern surgical procedures, very wide margins are required, sometimes including amputation of a joint. Patients usually receive major reconstructive surgery at the time of the tumor excision or within a few weeks. Patients receiving surgery alone, without chemotherapy, have an 80-90% rate of recurrence, usually in only 3-6 months. New chemotherapy treatments introduced in the 1970s and 1980s, in combination with surgery, has resulted in significantly lower rates of recurrence and longer survivals. Chemotherapy is provided prior to surgery, typically for ten weeks. Once patients have sufficiently recovered from the surgery, patients receive an additional twenty weeks of chemotherapy. The standard chemotherapy regimen consists of cisplatin, doxorubicin, and high-dose methotrexate, and is highly toxic with significant serious side effects. Among patients with non-metastatic disease, the combination of surgery and systemic chemotherapy has improved five-year survival to approximately 70%. Patients experiencing a recurrence are often treated with additional surgery and ifosfamide in monotherapy or ifosfamide in combination with etoposide. Unfortunately, this second-line therapy is very often ineffective. Osteosarcoma patients experiencing a recurrence have a five year survival of only 25%. Approximately 25% of patients already have overt metastatic disease at the time of initial diagnosis. These patients will also receive surgery and chemotherapy, but the prognosis is very poor, with five year survival of only 20%. Since the 1980s, there has been little if any improvement in patient outcomes and survival rates in osteosarcoma. Multiple new agents have been evaluated in the past 20 years, but none have demonstrated significant efficacy, and no new agents have been approved by the FDA. While many cancer types metastasize to the lungs, osteosarcoma has a particular tendency to metastasize to the lungs and only the lungs. It is this tendency that is the basis for the clinical study now being conducted by Eleison Pharmaceuticals. Eleison is evaluating ILC, a nanoscale lipid-complexed cisplatin which is administered via inhalation. ILC, once inhaled, dissolves slowly, releasing cisplatin and resulting in high concentrations of cisplatin in the lung tissues. In prior clinical studies, ILC was shown to have minimal toxicity and evidence of clinical benefit. In the current clinical trial, ILC is being evaluated to determine if ILC can delay or perhaps prevent lung metastases of osteosarcoma.

AUTHOR Edwin Thomas is CEO and founder of Eleison Pharmaceuticals, LLC. A biotechnology executive and entrepreneur, Mr. Thomas has been principal and founder of several successful life science companies, creating value for investors, and achieving timely and profitable exits.

RESOURCES Eleison Pharmaceuticals, LLC. Osteosarcoma Clinical Trials Slideshow Osteosarcoma Clinical Trials Video

Osteosarcomaclinicaltrials branded  

Osteosarcoma Studies, Eleison Pharm, Orphan Drugs for Cancer Treatment

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