Category Archives: Gene Therapy

Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month's issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments. "Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit," says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. "Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. We're proposing a different approach that spreads payments out and only keep coming if the patient continues to do well." Wilson and co-author Troyen A. Brennan, MD, JD, MPH, chief medical officer of CVS Health, note that while large single payments for gene therapy may be the … Continue reading →

Contact Information Available for logged-in reporters only Newswise PHILADELPHIA - Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this months issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments. Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit, says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. Were proposing a different approach that spreads payments out and only keep coming if the patient continues to do well. Wilson and co-author Troyen A. Brennan, MD, JD, MPH, chief medical officer of CVS Health, note that … Continue reading →

PUBLIC RELEASE DATE: 9-Sep-2014 Contact: Karen Kreeger karen.kreeger@uphs.upenn.edu 215-349-5658 University of Pennsylvania School of Medicine http://www.twitter.com/PennMedNews PHILADELPHIA - Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month's issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments. "Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit," says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. "Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. We're proposing a different approach that spreads payments out and only keep coming if the patient continues to do well." Wilson and co-author Troyen A. Brennan, MD, JD, MPH, … Continue reading →

PUBLIC RELEASE DATE: 9-Sep-2014 Contact: Evy Vierstraete info@vib.be 32-924-46611 VIB (the Flanders Institute for Biotechnology) For the first time the Jeffrey Modell Foundation is giving a research grant to a Belgian laboratory. The team of Adrian Liston from VIB-KU Leuven will use the grant to develop a gene therapy to cure children that suffer from IPEX syndrome, a rare and fatal autoimmune disorder in which the immune system attacks the body's own tissues and organs. At the moment, the only successful therapy to treat the syndrome is a bone marrow transplantation, which is not available for all children. "This is a real chance for a cure", said group leader Adrian Liston. "The gene responsible for this disease was identified 13 years ago, but for the first time we may have learned enough about the basic biology to solve it. We should know within a year whether the gene therapy works in mice, after which we can move to patients at top speed." The Jeffrey Modell Foundation (JMF) JMF is a global non-profit organization for patients who suffer from Primary Immunodeficiency (PI) and their relatives. The organization is devoted to early and precise diagnosis, meaningful treatments and, ultimately, cures. Through clinical … Continue reading →

Contact Information Available for logged-in reporters only Newswise ANN ARBOR, Mich. University of Michigan Health System doctors have started testing a unique new approach to fighting brain tumors -- one that delivers a one-two punch designed to knock out the most dangerous brain cancer. The experimental approach, based on U-M research, delivers two different genes directly into the brains of patients following the operation to remove the bulk of their tumors. The idea: trigger immune activity within the brain itself to kill remaining tumor cells -- the ones neurosurgeons cant take out, which make this type of tumor so dangerous. Its the first time this gene therapy approach is being tried in humans, after more than a decade of research in experimental models. One of the genes is designed to kill tumor cells directly, and is turned on when the patient takes a certain drug. The other gene spurs the bodys own immune system to attack remaining cancer cells. Both are delivered into brain cells via a harmless virus. The Phase I clinical trial has already enrolled two patients who have tolerated the gene delivery without complications. All patients in the study must have a presumptive diagnosis of WHO grade … Continue reading →

ABC Erin Griffin was an advocate for childhood cancer awareness. A 14-year-old girl who campaigned to raise awareness about childhood cancer has died after making history through her participation in a gene therapy trial. Erin Griffin died at the Children's Hospital in Adelaide with her parents, brother and grandmother by her side. In 2013, Erin received a Children's Week Award for her advocacy work in raising childhood cancer awareness. Erin was born in Scotland and moved to Australia when she was six. She was diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG), an incurable brain cancer, on February 18, 2012. "We're all very sad to lose this special girl," Erin's specialist oncologist Dr Geoff McCowage told the ABC. Dr McCowage, who runs a gene therapy trial at the Children's Hospital at Westmead in Sydney, said Erin made history. "She was only the second child in the world to take part [in the trial]," he said. "Throughout her ordeal she stayed courageous, she took two trips to the United States, one to Scotland and she was active on the internet telling the story of what she was going through and offering support to others going through the same thing. See the rest … Continue reading →

The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities. New research led by Charles Shoemaker, Ph.D., professor in the Department of Infectious Disease and Global Health at the Cummings School of Veterinary Medicine at Tufts University, shows that gene therapy may offer significant advantages in prevention and treatment of botulism exposure over current methods. The findings of the National Institutes of Health funded study appear in the August 29 issue of PLOS ONE. Shoemaker has been studying gene therapy as a novel way to treat diseases such as botulism, a rare but serious paralytic illness caused by a nerve toxin that is produced by the bacterium Clostridium botulinum. Despite the relatively small number of botulism poisoning cases nationally, there are global concerns that the toxin can be produced easily and inexpensively for bioterrorism use. Botulism, like E. coli food poisoning and C. difficile infection, is a toxin-mediated disease, meaning it occurs from a toxin that is produced by a microbial infection. Shoemaker's previously reported antitoxin treatments use proteins produced … Continue reading →

Gene Therapy EDTECH By: Krittamet Tatsanapornsatith … Continue reading →

Released: 29-Aug-2014 10:00 AM EDT Embargo expired: 29-Aug-2014 2:00 PM EDT Source Newsroom: Tufts University Contact Information Available for logged-in reporters only Newswise NORTH GRAFTON, Mass. (August 29, 2014, 2 PM US Eastern Time)The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities. New research led by Charles Shoemaker, Ph.D., professor in the Department of Infectious Disease and Global Health at the Cummings School of Veterinary Medicine at Tufts University, shows that gene therapy may offer significant advantages in prevention and treatment of botulism exposure over current methods. The findings of the National Institutes of Health funded study appear in the August 29 issue of PLOS ONE. Shoemaker has been studying gene therapy as a novel way to treat diseases such as botulism, a rare but serious paralytic illness caused by a nerve toxin that is produced by the bacterium Clostridium botulinum. Despite the relatively small number of botulism poisoning cases nationally, there are global concerns that the toxin can be produced easily and inexpensively for bioterrorism use. Botulism, like … Continue reading →

Contact Information Available for logged-in reporters only Newswise PHILADELPHIA Treating the rare disease MPS I is a challenge. MPS I, caused by the deficiency of a key enzyme called IDUA, eventually leads to the abnormal accumulation of certain molecules and cell death. The two main treatments for MPS I are bone marrow transplantation and intravenous enzyme replacement therapy, but these are only marginally effective or clinically impractical, especially when the disease strikes the central nervous system (CNS). Using an animal model, a team from the Perelman School of Medicine at the University of Pennsylvania has proven the efficacy of a more elegant way to restore IDUA levels in the body through direct gene transfer. Their work was published this week online in Molecular Therapy. The study provides a strong proof-of-principle for the efficacy and practicality of intrathecal delivery of gene therapy for MPS patients, said lead author James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine and director of the Penn Gene Therapy Program. This first demonstration will pave the way for gene therapies to be translated into the clinic for lysosomal storage diseases. This family of diseases comprises about 50 rare inherited disorders marked by defects in … Continue reading →