CLINICAL NEWS
European Commission Approves First Gene Therapy for “ Bubble Boy Disease ”
The European Commission approved the first gene therapy for children with severe combined immunodeficiency due to adenosine deaminase deficiency ( ADA-SCID ), also known as “ bubble boy disease .” The therapy is made up of autologous CD34 + cells transduced to express ADA and is the first ex vivo stem cell gene therapy for patients with ADA-SCID for whom no suitable human leukocyte antigen-matched related stem cell donor is available .
The approval was based on results from a pivotal study of 18 children with ADA-SCID that was recently published in Blood . The survival rate after a median of 6.9 years ( range = 2.3-13.4 years ) was 100 percent , and intervention-free survival in the evaluable population ( n = 17 ) was 82 percent .
All 18 patients reported adverse events ( AEs ), the most frequent of which were upper respiratory tract infection , gastroenteritis , and rhinitis . Of the 39 serious AEs reported , 62 percent were infections , with the most common being device-related . Patients also experienced neurologic , central nervous system , or hearing impairments that continued post-treatment . The safety findings were noted as similar to those expected in this patient population who received low-dose chemotherapy and who are undergoing immune recovery . A significant reduction in severe infections was documented , with no leukemic events observed to date .
Source : GSK press release , May 27 , 2016 .
Researchers Identify 11 Different Subtypes of Acute Myeloid Leukemia
In an analysis of more than 100 genes known to cause acute myeloid leukemia ( AML ), researchers from the Memorial Sloan Kettering Cancer Center in New York and Britain ’ s Wellcome Trust Sanger Institute have identified 11 different subtypes of AML . The research suggests that AML is not one disorder , but many , and could change the way patients are diagnosed and the ways drugs are developed .
“ For the first time , we untangled the genetic complexity seen in most AML cancer genomes into distinct evolutionary paths ,” said first author of the study , Elli Papaemmanuil , PhD , from the Cancer Genome Project at the Wellcome Trust Sanger Institute .
The study – published in the New England Journal of Medicine – analyzed 1,540 AML patients , including more than 100 genes known to cause leukemia in an effort to seek out common genetic themes related to the development of the disease . They found that patients could be categorized into at least 11 major groups , each with genetic changes and distinctive clinical features . The researchers also found that most patients had a unique combination of genetic changes , which explains why AML patients have such variable survival rates , they wrote .
Source : Papaemmanuil E , Gerstung M , Bullinger L , et al . Genomic classification and prognosis in acute myeloid leukemia . N Engl J Med . 2016 June 15 . [ Epub ahead of print ]
First Trial to Use CRISPR in Humans Approved by Federal Biosafety and Ethics Panel
A federal biosafety and ethics panel approved the first-in-human study of the genome-editing technology CRISPR-Cas9 to create genetically altered immune cells to attack three kinds of cancer .
The early-stage clinical trial was proposed by scientists at the University of Pennsylvania with plans to enroll 15 patients with multiple myeloma , melanoma , and sarcoma to gauge the safety of the experimental therapy and test the feasibility of manufacturing genetically engineered and CRISPR T cells . The scientists plan to use CRISPR to genetically modify the T cells so that , when infused back into a patient , they can target and destroy tumor cells . Specifically , this trial is targeting PD-1 and TCR .
The trial would be conducted at the University of Texas MD Anderson Cancer Center , the University of California , San Francisco , and the University of Pennsylvania , pending further approval from the other medical centers and the U . S . Food and Drug Administration .
Source : Science , June 21 , 2016 .
National Institutes of Health Funds Study of Zika Virus Exposure in Olympic Athletes
The National Institutes of Health ’ s ( NIH ) Eunice Kennedy Shriver National Institute of Child Health and Human Development ( NICHD ) is funding a research initiative to monitor potential Zika virus exposure in at least 1,000 athletes , coaches , and other U . S . Olympic Committee staff who are attending the 2016 Summer Olympics and Paralympics in Rio de Janeiro , Brazil , which is currently at the center of the Zika virus outbreak .
Carrie L . Byington , MD , from the University of Utah in Salt Lake City , leads the study , which seeks to improve the understanding of how the virus persists in the body and to identify potential factors that influence the course of infection . With this study , Dr . Byington and colleagues hope to :
• determine the incidence of Zika virus infection
• identify potential risk factors for infection
• detect where the virus persists in the body ( blood , semen , vaginal secretions , or saliva )
• evaluate how long the virus remains in these fluids
• study the reproductive outcomes of Zika-infected participants for up to one year
Participants will complete health surveys and provide samples of bodily fluids for the detection of Zika and similar flaviviruses . Zika virus testing kits and training will be provided by the Centers for Disease Control and Prevention .
“ Monitoring the health and reproductive outcomes of members of the U . S . Olympic team offers a unique opportunity to answer important questions and help address an ongoing public health emergency ,” said Catherine Y . Spong , MD , acting director of NICHD .
Source : National Institutes of Health press release , July 5 , 2016 .
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