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Archive for the ‘CAR-T’ Category


FDA: CAR-T therapy outweigh its risks tisagenlecleucel, manufactured by Novartis of Basel – 52 out of 63 participants — 82.5% — experienced overall remissions – young patients with Leukaemia [ALL]

 

Reporter: Aviva Lev-Ari, PhD, RN

 

Basel, July 12, 2017 – Novartis announced today that the US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of CTL019 (tisagenlecleucel), an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).

“The panel’s unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need,” said Bruno Strigini, CEO, Novartis Oncology. “We’re very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review.”

Acute lymphoblastic leukemia comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US[1]. Effective treatment options for patients with r/r ALL are limited. In pediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30%[2],[3],[4].

CTL019 was first developed by the University of Pennsylvania (Penn) and uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Children’s Hospital of Philadelphia (CHOP) was the first institution to investigate CTL019 in the treatment of pediatric patients and led the single site trial.

SOURCE

https://www.novartis.com/news/media-releases/novartis-car-t-cell-therapy-ctl019-unanimously-10-0-recommended-approval-fda

RISKS:

During the 2015 tisagenlecleucel trial, 47% of participants experienced an

  • extreme inflammatory reaction known as cytokine release syndrome, severe cases of which are called cytokine storms. The syndrome — characterized by symptoms such as high fevers and organ failure — can be life-threatening. But
  • Novartis says trial clinicians were able to manage the reaction successfully in all cases.
  • Neurological problems such as seizures and hallucinations were also relatively common but temporary,
  • the Novartis team reported. This is in stark contrast to some other CAR-T trials that have,
  • over the past year, reported the deaths of several participants from severe brain swelling.
  • Novartis’s therapy is not identical to the CAR-T cells used in those trials, which were administered in adults, but the deaths cast a pall over the entire field.

To generate a batch of tisagenlecleucel, white blood cells are purified from a sample of a patient’s blood and shipped to a central processing centre. There, staff use a virus to insert into the T cells genes that encode a cellular receptor — called a chimaeric antigen receptor — that will recognize leukaemia cells.

SOURCE

Engineered cell therapy for cancer gets thumbs up from FDA advisers

Treatment shows promise in young people with leukaemia, but safety risks abound.

Heidi Ledford, 12 July 2017

http://www.nature.com/news/engineered-cell-therapy-for-cancer-gets-thumbs-up-from-fda-advisers-1.22304?WT.ec_id=NEWSDAILY-20170713

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