First, a process specific to each patient is conducted where millions of immune system T-cells are removed and frozen at an approved medical center.
The altered T-cells are then re-injected into the patient's body, where they multiply and attack cancer. Novartis's therapy commandeers the body's immune system to destroy the cells where the blood cancer begins.
A Food and Drug Administration panel on Wednesday unanimously recommended the agency approve the use of the treatment known as CTL019, or tisagenlecleucel, for use on patients with relapsing ALL. The one-time leukemia treatment would be for children and young adults with the most common form of childhood cancer, known as ALL.
Analysts predict that these individualised treatments could cost more than US$300,000 (S$413,000), but a spokesman for Novartis, Ms Julie Masow, declined to specify a price.
Close on Novartis' heels is a team from Kite, a CAR-T-focused biotech which had hoped to be the first to the market.
The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis. Despite the side effects, the expert committee still felt that the benefits of the therapy outweighed the risks.
Part of the meeting was spent discussing Novartis' risk mitigation plans to address the severe cytokine response syndrome (CRS) most patients encountered after the CTL-019 infusion, along with potential neurotoxicities. "Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world". This study was the first of its kind. The scientists also figured out how to embed a "suicide gene" that shuts the cells down if they start to trigger cytokine release syndrome-a risky inflammatory response that has raised concerns during CAR-T clinical trials conducted by Novartis and its rivals. The ELIANA Trial originally enrolled 88 patients with relapsed or refractory B-cell ALL.
In this pivotal study informing the committee's decision, roughly half of 68 patients receiving the drug experienced this complication, though none died from it. That is because of the nature of the disease, and not because the treatment is toxic.
Juno scrapped its initial CAR-T candidate for ALL after five patient deaths. Not just to patients, but to the physicians who are evaluating these new therapies for their patients. What does a 95% confidence interval mean? Of 52 patients who results were analyzed, 83 percent had complete remission.
There are quite a few risks with the CTL019 treatment by Novartis. Drug company officials said they can now produce a treatment and get it to a patient in about three weeks. Those safety issues did not arise during the phase II trial, but the patients have relatively limited follow-up.
CAR-Ts can also pose serious risks, including a potentially life-threatening inflammatory condition. The committee's unanimous positive vote will go a long way towards that approval. Many of these patients required treatment in the intensive care unit (ICU), but all of them survived.
The news is not only good for Novartis, which expects peak sales of at least $1Bn.
Although the FDA is not bound to follow recommendations from its advisers when making decisions on treatments it usually does, and is due to make a decision on CTL019 in September following a six-month priority review.
The vast majority of people with ALL recover through other treatments, including chemo, radiation and stem-cell transplantation. I have no business relationship with any company whose stock is mentioned in this article.