The U.S. Food and Drug Administration, through its Office of Orphan Products Development (OOPD), facilitates the development of drugs and biologics which have the potential to diagnose, treat, or prevent rare diseases that affect fewer than 200,000 Americans or that affect more than 200,000 individuals, but where treating these persons is not expected to recover the costs of research, development, and marketing.
Since 1985, the OOPD has enabled the introduction of more than 350 such “orphan” drugs to the U.S. market, in many cases providing therapies that would otherwise be unavailable. Although the Orphan Drug Act provides tax credits and market incentives to drug developers – and sometimes grants monetary assistance for research – orphan drug designation does not alter the requirements for proving a new agent’s safety and efficacy in clinical trials.
Neuralstem Stem Cells Get FDA Nod
Neuralstem, a Rockville, Maryland-based biotherapeutics company, has developed a technology which allows them to produce commercial quantities of human neural stem cells and to control the differentiation of these cells into various types of neurons and glial cells.
This technology was developed by Dr. Karl Johe when he was with the NIH’s National Institute of Neurological Disorders and Stroke. The Neuralstem line of cells is capable of sustained division for up to 60 generations, which provides ample supplies of stem cells for both research and therapeutic applications.
On February 9, 2011, Neuralstem announced that the FDA had designated its neural stem cell line as an orphan drug.
Phase I Trials for Orphan Drug Treatment Have Already Begun
One of Neuralstem’s current projects involves the use of spinal cord-derived stem cells to treat ALS, or Lou Gehrig’s disease. After demonstrating benefit in animal models, Neuralstem recruited its first human ALS patients in 2010 for spinal implantations of their stem cell line.
The first patients enrolled in this clinical trial – a joint venture between Neuralstem and Emory University’s ALS Center – were already suffering from advanced ALS and were unable to walk. Non-ambulatory patients were specifically chosen first, because Neuralstem’s scientists wanted to ensure the treatment’s safety before implanting the cells into ambulatory individuals.
Neuralstem has since included ambulatory patients in this safety trial – classified as a Phase I trial by the FDA – and will soon begin treating other ALS patients with less severe disabilities.
Future Possibilities for Neural Stem Cells
Possible uses for neural stem cells include the treatment of traumatic spinal cord injuries, Huntington’s disease, Parkinson’s disease, stroke, traumatic head injury, Alzheimer’s disease, and a host of other traumatic or neurodegenerative conditions. As scientists learn more about the causes of major depression and schizophrenia, stem cells could find new applications for refractory cases of these illnesses, too.
As Neuralstem moves forward with its clinical trials, ALS sufferers may be granted a boon that will reverse, or at least forestall, their terrible disease. If so, an array of other maladies may soon be the targets of similar advances in technology. Thus, the granting of “orphan drug” status to a collection of neural stem cells may open doors to new and more effective ways for treating diseases that are commonplace.
Resources
- U.S. Department of Health and Human Services: U.S. Food and Drug Administration. Developing Products for Rare Diseases and Conditions.
- U.S. Food and Drug Administration: Orphan Drug Act
- RedOrbit. Neuralstem Receives FDA Orphan Designation for Spinal Cord Stem Cells. 9 February 2011
Stephen Allen Christensen - Dr. Steve Christensen's writing has appeared in magazines, professional journals, poetry anthologies, and children's books since 1976.
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