- Among the major players currently in the market, experts expect Novartis's treatment CTL-019, for acute lymphoblastic leukemia (ALL), to come to market first.
- But the challenge for Novartis (as well as Juno and Kite Pharma, which are also heavily involved in the space) will be being able to scale up production techniques to treat 6,000 people with ALL (60% of whom are under the age of 20.)
- The CAR-T treatments are made by extracting immune T-cells from an individual patient, modifying the DNA to create killer targeting cells, and then infusing the therapy back into patients' bodies.
CAR-T has shown tremendous promise for altering the treatment landscape for all types of blood cancer, with cure rates as high as 90% for refractory cancers that are no longer responsive to other therapies. Currently, Novartis, Kite, and Juno are developing therapies for ALL, and Novartis is also hoping to have a therapy on the market for diffuse large B-cell lymphoma (DLBCL) by 2017.
The challenge for all of the companies is production capacity and related cost. However, Novartis has a distinct advantage when it comes to this issue thanks to its size. The cost of production for each patient ranges from $50,000 to $100,000, which pushes the cost of therapy up to at least $200K to $300K for a large company like Novartis, and as high as $450,000 for smaller companies.
At the moment, Kite and Juno are retooling two leased plants to make the cells used in clinical studies. In addition, there is another possible approach which involves taking cells from healthy patients and modifying them for patients with cancer. Experts say that one donor could be the source of cells for 4,000 patients—with the only major downside being the risk of donor rejection.
All told, the challenges associated with production pale in comparison to the tremendous life-saving potential of CAR-T therapy.