Orphan Drug Program Benefits People With Epilepsy
Orphan Drug Program Benefits People With Epilepsy
The Center for Orphan Drug Research (CODR) at the College of Pharmacy, University of Minnesota, Minneapolis, Minnesota, was created in 2005. Directed by James Cloyd, PharmD, Professor and Lawrence C. Weaver Endowed Chair-Orphan Drug Development, the center's stated mission is to "improve the care of individuals suffering from rare diseases through research on new drug therapies; education of health professionals and health profession students; and contributions to the discussion and formulation of public policy relating to rare diseases and orphan drugs." Researchers at CODR include laboratory scientists, a research associate, a postdoctoral fellow, graduate students, residents, and undergraduate students. To speed development, CODR partners with pharmaceutical and biotechnology companies. Its primary research focus is rare pediatric neurologic disorders.
Several projects are currently under way at CODR that may potentially help people with epilepsy. These include:
Dr. Cloyd commented,
These 4 projects address our interest in treating seizure emergencies in a vulnerable population. Neonatal seizures are a type of seizure emergency, and we are developing an injectable form of topiramate for that. We plan to determine whether it is safe and effective in neonatal seizures and whether it has the potential for neuroprotection in those babies.
The other 3 projects are directed at finding a better way of treating seizure emergencies outside the hospital. At the moment, Diastat, another orphan drug, is the only alternative. Although Diastat is effective, because it must be administered rectally, there are problems with patient and caregiver acceptance. We think that the nasal delivery of diazepam would be at least as effective and far more practical. At least one other company is working on nasal diazepam and another company on nasal midazolam. Intramuscular benzodiazepines are another option under development for controlling breakthrough seizures outside the hospital.
Dr. Cloyd explained that the orphan drug designation can be applied to a new molecule or to an old drug that is repurposed for an orphan indication. In either case, patients with rare diseases could benefit. Projects at CODR include collaborations with the FDA, Mayo Clinic, and University of Pennsylvania as well as companies such as NeuroVista, which is developing seizure prediction software; Ligand Pharmaceuticals, which is participating in the development of intravenous topiramate; and Neurelis, which is supporting the development of intranasal diazepam.
Dr. Cloyd concluded, "At CODR, we are trying to act like a small drug company and leverage resources across the University of Minnesota and elsewhere to get this work done in a way that is responsible and rigorous. We are filling a niche that really isn't being filled by anyone else. Our goal is to get new products out so that clinicians can prescribe them and patients can use them. Alternatively, we want to generate high quality information about old drugs so that they can be used safely and effectively for rare disorders."
In addition to CODR, other centers that focus on orphan drug research include The Center for Rare and Neglected Diseases at the University of Notre Dame, Notre Dame, Indiana; The Manton Center for Orphan Disease Research at Children's Hospital in Boston, Massachusetts; The Center for Rare Disease Therapies at the Keck Graduate Institute of Applied Life Sciences, Claremont, California; and The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.
Specialized Centers Advancing Research
The Center for Orphan Drug Research (CODR) at the College of Pharmacy, University of Minnesota, Minneapolis, Minnesota, was created in 2005. Directed by James Cloyd, PharmD, Professor and Lawrence C. Weaver Endowed Chair-Orphan Drug Development, the center's stated mission is to "improve the care of individuals suffering from rare diseases through research on new drug therapies; education of health professionals and health profession students; and contributions to the discussion and formulation of public policy relating to rare diseases and orphan drugs." Researchers at CODR include laboratory scientists, a research associate, a postdoctoral fellow, graduate students, residents, and undergraduate students. To speed development, CODR partners with pharmaceutical and biotechnology companies. Its primary research focus is rare pediatric neurologic disorders.
Several projects are currently under way at CODR that may potentially help people with epilepsy. These include:
Use of topiramate for neuroprotection and seizure control in neonates;
Development of responsive pharmacotherapy in canine partial epilepsy;
Intranasal benzodiazepines for seizure emergencies; and
Development of water-soluble benzodiazepine prodrugs to treat seizure emergencies.
Dr. Cloyd commented,
These 4 projects address our interest in treating seizure emergencies in a vulnerable population. Neonatal seizures are a type of seizure emergency, and we are developing an injectable form of topiramate for that. We plan to determine whether it is safe and effective in neonatal seizures and whether it has the potential for neuroprotection in those babies.
The other 3 projects are directed at finding a better way of treating seizure emergencies outside the hospital. At the moment, Diastat, another orphan drug, is the only alternative. Although Diastat is effective, because it must be administered rectally, there are problems with patient and caregiver acceptance. We think that the nasal delivery of diazepam would be at least as effective and far more practical. At least one other company is working on nasal diazepam and another company on nasal midazolam. Intramuscular benzodiazepines are another option under development for controlling breakthrough seizures outside the hospital.
Dr. Cloyd explained that the orphan drug designation can be applied to a new molecule or to an old drug that is repurposed for an orphan indication. In either case, patients with rare diseases could benefit. Projects at CODR include collaborations with the FDA, Mayo Clinic, and University of Pennsylvania as well as companies such as NeuroVista, which is developing seizure prediction software; Ligand Pharmaceuticals, which is participating in the development of intravenous topiramate; and Neurelis, which is supporting the development of intranasal diazepam.
Dr. Cloyd concluded, "At CODR, we are trying to act like a small drug company and leverage resources across the University of Minnesota and elsewhere to get this work done in a way that is responsible and rigorous. We are filling a niche that really isn't being filled by anyone else. Our goal is to get new products out so that clinicians can prescribe them and patients can use them. Alternatively, we want to generate high quality information about old drugs so that they can be used safely and effectively for rare disorders."
In addition to CODR, other centers that focus on orphan drug research include The Center for Rare and Neglected Diseases at the University of Notre Dame, Notre Dame, Indiana; The Manton Center for Orphan Disease Research at Children's Hospital in Boston, Massachusetts; The Center for Rare Disease Therapies at the Keck Graduate Institute of Applied Life Sciences, Claremont, California; and The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.