Minimally invasive micro-catheter with an integrated camera (MIMIC) to reach suboccipital space of the cisterna magna via lumbar puncture for drug and gene-therapy delivery to the brain
Open surgery is the standard of care for access to a diseased brain and spinal cord, but such surgeries are invasive, associated with significant morbidity, and can require long recovery times.
Recent advances in gene therapy hold the potential to cure devastating neurological diseases. In order to be effective, circumvention of the blood brain barrier (BBB) is required for gene therapy treatment of neurological disorders such as Parkinson’s disease and glycogen storage diseases. One method to bypass the BBB is to place a needle via suboccipital puncture into the cisterna magna, a relatively large subarachnoid space located in the cranial vault. However, there is hesitation to utilize this technique due to potential for catastrophic injury to the brain and spinal cord.
Bryan Pukenas, M.D., at the Hospital of the University of Pennsylvania, has developed a minimally invasive catheter that enables direct access for focal delivery of gene therapy vectors to the suboccipital space of the cranial vault in a minimally invasive manner.
Traditionally, catheters are navigated using fluoroscopic guidance. However, navigating catheters with only fluoroscopic guidance in the spinal canal poses significant risk of spinal cord or nerve injury since one cannot see these structures using this method. In contrast, the MIMIC catheter utilizes an embedded micro-camera permitting real-time visualization of the spinal canal contents during catheter navigation and gives operators the ability to avoid vital structures like the spinal cord and nerves as the catheter is directed from the lumbar canal to the cranial vault.
In addition to increased safety and simplicity, this approach is expected to reduce the amount of required gene vector material by 10-100 fold since gene transduction is much more efficient when delivered into the cisterna magna. The technology will change the procedure of gene therapy delivery to the brain from an inpatient surgery performed by an interventionalist /neurosurgeon to an outpatient procedure.
Gene therapy may become accessible for patients who otherwise would not clinically qualify for gene therapy treatment due to risks associated with surgery and post-surgical care. Moreover, the cost of care will be dramatically reduced by moving towards an outpatient model and decreasing the amount of vector required for treatment.
This invention has received over $90,000 in support from the Penn Medical Device Accelerator. The device has been tested in bench top spine models and a human cadaver. The technology is being further developed for additional application such as treatment of aneurysms, chemotherapy for brain tumors, epilepsy, laser ablation for brain tumors, etc.