NeoStem Inc., a player in the growing cell therapyindustry, announced Sept. 13 that it has been awarded funds for the second yearof a two-year grant totaling a little over $1.2 million, with $706,682 awarded in the first year and$515,172 for the second year of the project, to cover the cost of anInvestigational New Drug (IND) submission to the U.S. Food and DrugAdministration (FDA) for the product candidate.
The award, for "Repair of bone defects with humanautologous pluripotent very small embryonic-like stem cells (VSEL)," is from the National Institute of Dental andCraniofacial Research of the U.S. National Institutes of Health (NIH). NeoStem'sVSEL technology is an autologous therapy derived from a patient's own stemcells, and it is being developed for use in the regeneration of bone tissuedamaged by periodontitis.
The required preclinical data, cell manufacturingprocesses and clinical protocols necessary for submission of an IND to the FDAare in the final stages of preparation, the company reports, and it anticipatessubmitting its IND filings in late 2013 or early 2014.
"We are very excited about our progress towardsthe IND submission for what we expect to be the first human clinical study forour VSEL technology and for the support of the NIH," said Dr. Robin L. Smith, chairmanand CEO of NeoStem. "We continue to pursue opportunities for non-dilutivefinancing of our programs, such as our recently awarded Phase 1 NIH grant toinvestigate VSEL Technology for the treatment of scleroderma."
Periodontitis is a severe form of periodontaldisease, which is prevalent not just in the United States but which also affectsup to 90 percent of the world population, according to NeoStem. The most severecases of periodontal disease affect between 5 percent and 15 percent of theU.S. population, or between 15 million and 47 million Americans, the companynotes, with the incidence of new cases of periodontal disease estimated to be growingat a 7 percent rate each year. Beyond the dental damage, studies have shownthat periodontal inflammation could have a role in the initiation orprogression of coronary heart disease and stroke.
Market research experts have estimated that severeperiodontal disease represents a market between $1.25 billion and $1.5 billionannually.
NeoStem describes VSEL cells as "a residentpopulation of multipotent stem cells in the bone marrow involved in theturnover and regeneration of tissues." Reportedly, VSEL technology offers thepotential to go beyond the paracrine effect, yielding cells that actually differentiateinto the target tissue and create true cellular regeneration. Recent preclinicaldata in animal models suggest that VSELs may be capable of developing intocells of all three germ layers which, if substantiated by further research,could imply significant potential for restorative healing, according to NeoStem.Unlike in the case of classically defined pluripotent stem cells, the companyadds, it is believed that VSELs do not contribute to teratoma formation.