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Καταχωρήθηκε: Τρίτη 11 Δεκ 2018

We presented Treatments that made headlines in hematology in 2018. From  Nature Medicine 24,17851787 (2018)

Presenter: Prof. Spyridonidis
Paricipants: Dr Liga, Dr Spyridis, Dr Lekka, Dr Bountouris

 

Green light

CD19 CAR therapy

Chimeric antigen receptor (CAR) therapy using the CD19 antigen continued to show its benefits after a long-term follow-up study found that patients with B cell acute lymphoblastic leukemia (ALL) had favorable remission and survival rates. Historically, 18–45% of patients with ALL have gone into remission, but in a follow-up study at Memorial Sloan Kettering Cancer Center conducted about two and a half years after patients received an infusion of CD19 CAR T cells, the patients were found to have a remission rate of 83% (N. Engl. J. Med. 378, 449–459, 2018). The median survival time of patients with ALL has typically been less than nine months, but in this study, that time was a little over a year, although patients who had a low disease burden (indicated by less than 5% of bone marrow being blast cells) had a median survival time of more than 20 months.

Onpattro

Onpattro (patisiran) became the first RNA interference drug to win US Food and Drug Administration (FDA) approval. The drug works by silencing the gene that is responsible for a rare condition known as hereditary transthyretin-mediated amyloidosis, in which mutated forms of the transthyretin protein accumulate in the body and sometimes cause problems with heart and nerve function. The drug is produced by Massachusetts company Alnylam and is approved only for the treatment of nerve damage associated with the disease. Another drug for the same condition, called Tegsedi (inotersen) and produced by Akcea Therapeutics, was approved by the FDA just two months after Onpattro’s approval in August. A third drug, tafamidis, was found to be effective in a clinical trial in reducing deaths and cardiovascular events associated with the same condition, regardless of whether it was caused by heredity or other factors. In a study of 441 patients, 78 of the 264 patients who received tafamidis died, when compared with 76 of the 177 patients who received placebo (N. Engl. J. Med. 379, 1007–1016, 2018). Pfizer, the company behind tafamidis, is already offering the drug through an expanded-access program.

 

 

Yellow light

Beta-thalassemia drugs

Two different therapies against beta-thalassemia, a condition that affects roughly 1 in 100,000 people around the world, made headway this year. Celgene and Acceleron Pharma, the makers of luspatercept, announced that a phase 3 trial testing the drug against transfusion-dependent beta-thalassemia had achieved its primary endpoint in two different trials. In one trial, called MEDALIST, those on luspatercept were able to achieve the endpoint of not needing a red blood cell transfusion for at least two months during the first half year of treatment when compared with placebo. In another trial, patients who received luspatercept required fewer transfusions during the first three months of treatment and less blood per transfusion during the second three months of treatment when compared to placebo. The second beta-thalassemia treatment to show promise this year was CRISPR Therapeutics and Vertex Pharmaceuticals’ CTX001. The companies launched a phase 1/2 trial in Germany to test CRISPR-based treatment, making the trial the first from US companies to test a therapy that is based on the gene-editing technology. The companies also have plans to launch a US trial to test the technology in patients with sickle cell anemia. However, following the announcement of such a plan in April, the FDA placed a clinical hold on the study in May to get more questions answered about the application submitted by the companies.

 

Bacteriophage therapy

The FDA gave approval to Baltimore-based company Intralytix to begin phase 1/2 trials of bacteriophage therapy against inflammatory bowel disease (IBD). The idea behind bacteriophage therapy is to use a virus that is capable of targeting and infecting problem bacteria, in this case the Escherichia coli bacteria that are especially problematic in those with Crohn’s disease. The viruses are not thought to pose a problem to human hosts, but no phage therapy has yet been approved by the FDA. Intralytix is developing the phages along with Ferring Pharmaceuticals and will be conducting the trials at Mount Sinai Hospital in New York.

 

 

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