Gene editing makes possible new therapies and actual cures (not just treatments) that were previously not. But, one thing that doesn’t get discussed a great deal is how these new gene editing-based therapies throw the “take two and call me in the morning” model out the window.
This interesting piece in BioPharmaDive gives a fascinating look at all the steps for a gene editing therapy for sickle cell disease that Vertex Pharmaceuticals is awaiting FDA approval for. The steps include:
- referral by hematologist (not to mention insurance approval!)
- collection of cells (probably via bone marrow extraction)
- (partial) myeloablation of the patient
- shipping the cells to a manufacturing facility
- manufacturing facility applies gene editing on the cells
- shipping of cells back
- infusion of the gene edited cells to the patient (so they hopefully engraft back in their bone marrow)
Each step is complicated and has their own set of risks. And, while there are many economic aspects of this that are similar to more traditional drug regimens (high price points, deep biological understanding of disease, complicated manufacturing [esp for biologicals], medical / insurance outreach, patient education, etc.), gene editing-based therapies (which can also include CAR-T therapy) now require a level of ongoing operational complexity that the biotech/pharmaceutical industries will need to adapt to if we want to bring these therapies to more people.
To make and administer the therapy is laborious, first requiring a referral from a hematologist. If the patient is eligible, their cells are collected and shipped to a manufacturing facility where they’re genetically edited to express a form of an essential protein called hemoglobin.
The cells are then shipped back to a treatment facility that infuses them into the patient’s bone marrow. But to make sure there’s enough room for these new cells, patients first undergo myeloablation — a chemotherapy regimen that can be very difficult on their bodies and comes with the risk of infertility. Older patients may not be healthy enough to receive this treatment.
“This is an extensive and expensive process,” Arbuckle said.
Vertex gearing up for launch as sickle cell therapy review advances
Jacob Bell | BioPharma Dive