Press Release: Novartis Personalized Cell Therapy CTL019 Receives FDA Breakthrough Therapy Designation

Novartis announced today that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy status to CTL019, an investigational chimeric antigen receptor (CAR) therapy for the treatment of pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The Breakthrough Therapy filing was submitted by the University of Pennsylvania's Perelman School of Medicine (Penn) which has an exclusive global agreement with Novartis to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers.
This is the fifth Breakthrough Therapy designation for Novartis, continuing the company's trajectory as a leader in developing innovative therapies to help treat diseases in which there remains significant unmet medical need. Novartis' Zykadia(TM) (ceritinib, previously known as LDK378), for the treatment of anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC), is one of the first medicines to receive an FDA approval following earlier receipt of Breakthrough Therapy designation by the FDA[5].
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Francis Collins Says Medicine in the Future Will Be Tailored to Your Genes

Given how swiftly the field of medicine is moving, it is impossible to predict where we might stand in 25 years, let alone 125 years. However, one thing is certain: From my vantage point at the helm of the world's leading supporter of biomedical research, I see a broad horizon filled with exciting opportunities—many with the potential to transform medicine.
At the forefront of these revolutionary possibilities is personalized medicine, which is the idea of precisely tailoring each person's medical care to his or her own unique genetic makeup. In fact, if all goes as we envision, today's mostly one-size-fits-all approach to medical care will seem as outdated to future generations as bloodletting leeches and patent-medicine potions are to us.
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First Cancer Immunotherapy for Dogs Developed


Nearly every second dog develops cancer from the age of ten years onward. A few therapies derived from human medicine are available for dogs. A very successful form of therapy by which antibodies inhibit tumor growth has not been available for animals so far. Scientists at the inter-university Messerli Research Institute of the Vetmeduni Vienna, the Medical University of Vienna, and the University of Vienna have developed, for the first time, antibodies to treat cancer in dogs. The scientists published their research data in the journal Molecular Cancer Therapeutics.
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Renewed Hope for Gene Therapy in Rare Disease


Between 30 and 40 million people in Europe suffer from rare diseases—many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure. Until now, however, there have been very few successful trials. Now, the EU-funded project AIPGENE, due to be completed in 2014, may have made significant progress in a gene therapy approach. The project focussed on the genetic liver disorder, Acute Intermittent Porphyria (AIP). Through an early stage clinical trial, in phase I, it demonstrated the viability of a new approach, based on a so-called, adeno-associated vector (AAV). This is a 'DNA transporter' derived from a type of virus and carries the therapeutic gene to liver cells, known as hepatocytes.
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DNA Vaccines Database Available Online

DNAVaxDB is the first web-based DNA vaccine database and analysis system that curates, stores, and analyzes DNA vaccines and DNA vaccine plasmid vectors. DNAVaxDB includes only those DNA vaccines that have been verified to induce protection in at least a laboratory animal model. The DNAVaxDB website includes the following programs:
  • DNAVax Query: Query DNA vaccines that have been experimentally verified and stored in DNAVaxDB
  • DNAVax Plasmids: Query DNA vaccine plasmids
  • DNAVax Antigens: Query protective antigens that have been used in experimentally verified DNA vaccines
  • DNAVaxAg BLAST: BLAST DNA or protein sequence similarity search against the protective antigens used in experimentally verified DNA vaccines
  • Data Exchange: Exchange of data stored in DNAVaxDB
  • Data Submission: A web-based interactive program for submitting DNA vaccine data.
  Racz R, Li X, Patel M, Xiang Z, and He Y. DNAVaxDB: the first web-based DNA vaccine database and its data analysis. BMC Bioinformatics. 2014, 15(Suppl 4):S2.
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Increase in DNA Vaccine Efficacy by Virosome Delivery and Co-Expression of a Cytolytic Protein

DNA vaccines are appealing vaccine candidates as they are simple and inexpensive to manufacture and also highly stable. These characteristics make DNA vaccines an ideal technology particularly for use in developing countries where inexpensive vaccines are required most urgently. In practical terms, vaccines may be required to elicit systemic or mucosal immune responses.

This study aims to identify new techniques to improve DNA vaccination. The efficacy of DNA vaccination after IN delivery by influenza virosomes to protect DNA from degradation and ID delivery of vaccines that encode an immunogen and a cytolytic or toxic protein to increase the efficacy of DNA vaccination by inducing cell death in vaccine-targeted cells, resulting in cross presentation of the immunogen.
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Scientists Find Potential New Use for Cancer Drug in Gene Therapy for Blood Disorders

In a new study led by Associate Professor Bruce Torbett at The Scripps Research Institute (TSRI), a team of researchers report that the drug rapamycin, which is commonly used to slow cancer growth and prevent organ rejection, enables delivery of a therapeutic dose of genes to blood stem cells while preserving stem cell function. These findings, published recently online ahead of print by the journal Blood, could lead to more effective and affordable long-term treatments for blood cell disorders in which mutations in the DNA cause abnormal cell functions, such as in leukemia and sickle cell anemia.
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Madison Vaccines Expands Phase 2 Trial Of Prostate Cancer Vaccine

Clinical stage biopharmaceutical company Madison Vaccines Incorporated (MIV) announced that it has begun additional patient enrollment in the expansion of a Phase II clinical trial of its lead candidate MVI-816 (pTVG-HP). The Phase II trial is investigating whether MVI-816 can delay the onset of metastatic disease. Fifty patients will be added to the original group of 56 patients in the trial. Eligible patients include those with non-metastatic prostate cancer and who have rising PSA after primary treatment prior to necessitating androgen deprivation therapy or castration therapy. Current patient recruitment is ongoing at the University of Wisconsin and future enrollment is expected at the University of California in San Francisco and at least another site later this 2014.
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Frederic Bushman gets Pioneer Award from Human Gene Therapy

Frederic D. Bushman, PhD's early pioneering work in understanding how HIV reproduces by inserting its genetic material into the DNA of a host cell led to key advances in the ability to move pieces of DNA and whole genes between cells. In recognition of his scientific achievements and leadership in the field, Dr. Bushman is the recipient of a Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Human Gene Therapy is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon panel and publishing a Pioneer Perspective by each of the award recipients.
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Vaccine ‘Reprograms’ Pancreatic Cancers to Respond to Immunotherapy

Researchers at the Johns Hopkins Kimmel Cancer Center have developed and tested a vaccine that triggered the growth of immune cell nodules within pancreatic tumors, essentially reprogramming these intractable cancers and potentially making them vulnerable to immune-based therapies.
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