Novartis is well ahead of the pact as it announced the positive results of its experimental chimeric antigen receptor T cell (CART) therapy CTL019 in two forms of lymphomas or blood cancer.
The results came in early and is said to most likely make it to the U.S. regulatory submission in 2017.
The two types of lymphomas, which CTL019 is being tested for, are diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL). Researchers from the University of Pennsylvania's Perelman School of Medicine conducted the study in patients with advanced diseases that are not responsive to conventional treatments anymore.
"We remain consistent again with the data set," said Usman Azam, the global head of cell & gene therapies at Novartis. He added that the set of participants are attractive consumers, who persist to have unmet needs and serve as the cornerstone of the company's investments.
CTL019 is unlike any other biological or small molecular treatments such that it is made for each individual patient. A type of white blood cells, called T lymphocytes, is extracted from a patient using a special procedure called leukapheresis. The cells are then transferred to a Novartis facility in Morris Plains, New Jersey.
In Novartis, the cells are reprogrammed to detect cancer cells and other B cells (another type of white blood cells) expressing CD19. The altered cells will then be returned to a treatment facility where it will be injected to the patient again.
"The company's investment in our state-of-the-art manufacturing facility has given us the capacity and scalability needed to support our growing global clinical trial program," said Azam.
For the Phase II trial, patients with DLBCL showed a 47 percent overall response rate at three months, while patients with FL presented with 73 percent.
Six patients (three with DLBCL and three with FL) who attained partial response at three months progressed to have complete response by six months.
After six months, DLBCL progressed in one patient with partial response at three months. The disease of one patient with FL progressed about 12 months after treatment amid maintaining partial response at three and nine months.
In the clinical trial, four participants developed grade three or higher cytokine release syndrome (CRS), which is characterized by fever, muscle pain, nausea, difficulty of breathing and low blood pressure. The said condition was observed following administration of CTL019 when the modified cells were activated and spread throughout the body.
Two other patients developed neurologic toxicity, one of which had grade three delirium and the other possible grade five encephalopathy.
The accurate safety and efficacy of CTL019 has not yet been confirmed as it is still under investigation. With this, CTL019 is not yet guaranteed to hit commercial markets anywhere in the world.
Photo: Ed Uthman | Flickr
