Afford
Can't tell you how many times I've agreed with you. Not only this point. But, I stay away from posting because it seems as though you must be in the 'In Crowd', much like the cheerleaders back in the day, in order to post! That being said, thought I would just share with you this article in The WSJ this morning. Hope you find it pertinent and not any rah-rah stuff.
Thanks for your perspectives.
Cancer treatments that genetically modify patients’ blood cells to target the disease have shown amazing results in clinical trials. Now drug companies and biotechs must overcome big hurdles to get them into hospitals, including their potential cost.
In two separate clinical trials—sponsored by Novartis AG NOVN.VX -2.92% of Switzerland and Seattle-based biotech Juno Therapeutics Inc.—almost 90% of patients saw their leukemia disappear after being given experimental so-called CAR T-cell therapies. The results were published in December and February, respectively.
Both trials were in small numbers of patients: 22 children in the Novartis trial and 16 adults in the Juno trial. The patients had acute lymphoblastic leukemia—the most common childhood cancer—and had exhausted standard treatments. Both companies are now conducting larger trials.
“CAR T cells are probably one of the most exciting concepts and fields to come out in cancer in a very, very long time,” says Dr. Daniel DeAngelo, a Boston-based hematologist and associate professor of medicine at Harvard Medical School, who wasn’t involved in either study.
Usman Azam, head of cell and gene therapies at Novartis, calls the therapies “critically important” for Novartis. “I think that a cure for cancers such as leukemia and lymphoma through a CAR technology is plausible,” said Dr. Azam in an interview with The Wall Street Journal. “Our job is to get this into patients as soon as we feasibly can.”
Dr. Azam heads a new unit Novartis created partly to speed the therapies’ time to market. Its leading CAR therapy was granted ‘breakthrough’ designation by the U.S. Food and Drug Administration in July, and Novartis wants to file it with regulators in 2016.
CAR therapies are a mixture of genetic tweaking and “immunotherapy,” or using the patient’s own immune system to fight disease. They involve extracting disease-fighting white blood cells called T-cells from a patient’s blood. The T-cells are then genetically modified, grown in a laboratory for around 10 days and reinjected into the patient.
Typically, the T-cells are combined with a disabled virus, which enables them to produce chimeric antigen receptors, or CARs, that recognize and target malignant proteins on a cancer cell’s surface.
Novartis and Juno are developing their treatments in partnership with top academic teams: Novartis with the University of Pennsylvania and Juno with teams at Memorial Sloan-Kettering Cancer Center in New York, Seattle Children’s Hospital and the Fred Hutchinson Cancer Research Center, also in Seattle.
Novartis and Juno lead the pack in a race to bring the therapies to market, but Pfizer Inc., PFE -0.50% Kite Pharma Inc. KITE -1.84% and Celgene Corp. CELG -1.80% —working in collaboration with Bluebird Bio Inc. BLUE -0.40% —all are developing rival approaches.
“Competition will keep all of the companies involved on their toes,” said Hans Bishop, Juno’s chief executive.
There are still big unanswered questions about CAR therapies: one is how long they last.
That is hard to tell because of the small numbers of patients treated so far, and because many of those whose cancer went into remission after the CAR therapies subsequently became eligible for stem-cell transplants—which can themselves prolong survival.
Another concern is a potentially dangerous side effect called “cytokine-release syndrome,” an immune response which shows the therapy is working, but which can cause a sharp drop in blood pressure and surge in the heart rate.
The deaths of two patients in a Juno-backed Sloan-Kettering trial in March caused a temporary halt in the study because of worries over these immune responses.
“Patients need to be healthy enough to combat that side effect,” says Mr. Bishop, who thinks it is now manageable. The trial is recruiting patients again, excluding those with a risk of heart failure, and giving those with very advanced leukemia fewer modified cells.
But the biggest hurdle yet may be the cost of the therapies.
The genetic engineering involved means CAR therapies are very complex to manufacture, and each is a unique personalized treatment using a patient’s own blood cells. The inability to mass-produce them has likely implications for how much companies will charge for them.
“What we’re talking about here is a single, very expensive therapy that’s used once for a specific patient and is not generalizable,” says Dr. Malcolm Brenner, director of the Center for Cell and Gene Therapy at the Texas Children’s Hospital in Houston.
Dr. Brenner signed a deal in March to commercialize his own CAR research with Celgene.
Novartis and Juno say it is too early to speculate on price, although Dr. Usman agrees the challenge is getting the manufacturing process to “a viable level where it’s both affordable and attractive.”
While most analysts think it is too early to estimate potential revenue or price, Citigroup believes CAR therapies could cost in excess of $500,000 per patient, which it notes is roughly in line with the cost of a stem cell transplant.
“This technology needs to be widely developed and accessible to patients,” says Dr. DeAngelo. “If the cost is going to be a hindrance, it’s going to be a really sad day.”
Scalability and cost are one reason Pfizer is taking a different approach to the field.
“We would like to take it to the next level, where CAR therapies become a more standardized, highly controlled treatment,” said Mikael Dolsten, Pfizer’s head of global research and development.
Working with French biotech Cellectis SA, ALCLS.FR -6.14% Pfizer wants to develop a generic CAR therapy for use in any patient, potentially lowering its cost. But its research is still preclinical, and may not work in humans.
Stephen McGarry, global head of health-care research at Société Générale, agrees the treatments being developed by Novartis and Juno could justify “astronomical” prices, although he thinks health-care payers and patients might fight back.
“When you look at the initial data with the Novartis therapy, you’re getting cures in some kids—what do you charge for that?” he asks.
Write to Hester Plumridge at Hester.Plumridge@wsj.com
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