In Case of Nuclear War - Pluristem Gets Orphan Drug Designation for its ARS Drug
The U.S. Food and Drug Administration (FDA) has granted Pluristem Therapeutics Inc. an orphan drug designation for its PLX-R18 cell therapy to prevent and treat acute radiation syndrome.
Acute radiation syndrome is the result of exposure to extremely high levels of radiation. Such exposure would be due to a nuclear attack or accident.
Per the Centers for Disease Control and Prevention (CDC
), Acute radiation syndromes can be categorized according to the system involved.
1. Bone marrow syndrome (or hematopoietic syndrome) usually occurs with a dose between 0.7 and 10 Gy (70 – 1000 rads). Survival will be dependent on the dose but the primary cause of death is infection and hemorrhage due to the destruction of the bone marrow.
2. Gastrointestinal (GI) syndrome usually occurs with a dose above 10 Gy (1000 rads). Destructive of the GI tract and bone marrow lead to infection, dehydration, and electrolyte imbalance. Death usually occurs within 2 weeks.
3. Cardiovascular / Central Nervous System syndrome usually occurs with a dose greater than approximately 50 Gy (5000 rads). Death typically occurs within 3 days.
PLX-R18 cells are designed to restore the bone marrow that is destroyed in people with acute radiation syndrome. According to the company, PLX-R18 cells secrete proteins that trigger the regeneration of bone marrow hematopoietic cells. A Phase I trial testing PLX-R18 in incomplete bone marrow recovery following hematopoietic cell transplantation (HCT) is currently underway and a pivotal trial acute radiation syndrome is in development.
Earlier this year, Pluristen reported
that PLX-R18 improved survival in animals exposed to high radiation levels. In a study with the National Institute of Allergy and Infectious Diseases (NIAID) the survival rates in monkeys given high doses of radiation was 50% in the placebo group compared to 85% in the PLX-R18-treated group.
The Orphan Drug Designation provides numerous incentives for Pluristem in the development and potential marketing of PLX-R18, including orphan drug grants, tax credits, and 7-year market exclusivity if approved.
In a news release
, Zami Aberman, Co-CEO and Chairman of Pluristem reminded everyone one why drugs like these are important to have stockpiled. “Receiving Orphan Drug Designation brings us one step closer to providing a next-generation medical countermeasure against ARS, which is especially important given today’s volatile political climate.”
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