Groundbreaking Gene Therapy Trial Begins in Philadelphia

Today Spark Therapeutics announced the start of the first U.S. Human Clinical Trials to treat Choroideremia ("CHM"), an inherited and currently incurable genetic condition that progressively leads to blindness by damaging the retina of impacted individuals. Were it not for the persistent efforts of a determined few in the patient community, like Jeff, this day might never have come. "Spark's groundbreaking announcement today brings real hope of a treatment for blindness caused by Choroideremia, and further paves the way for treatments of other retinal diseases impacting people around the world," said Dr. Chris Moen, President of the Choroideremia Research Foundation (, the leading advocacy and fundraising organization focused on finding a treatment for CHM. "The Choroideremia Research Foundation is proud to have provided key preclinical funding to Jean Bennett, MD, PhD and her team at the Perelman School of Medicine at the University of Pennsylvania, that has helped bring us to the gene therapy human clinical trials being announced today."
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Merck, Bristol Heat up Immunotherapy Race in Lung Cancer

Bristol-Myers Squibb and Merck & Co broke further away from rival drugmakers in the race to treat lung cancer with a new generation of immune system therapies, adding pressure on the likes of Roche and AstraZeneca to play catch-up. Merck said it would file an application with U.S. regulators in the middle of this year seeking expanded use of its new Keytruda treatment for non-small cell lung cancer, the most common form of the deadly disease.
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Tailor-Made Vaccine set to Banish Africa’s Meningitis Epidemics

The website of a global partnership formed to wipe out deadly meningitis epidemics in sub-Saharan Africa is closing down with a simple message: "Thank you and goodbye!". Barely five years after the team began rolling out a tailor-made vaccine in Africa's "meningitis belt", the disease has all but disappeared there and the Meningitis Vaccine Project (MVP) is closing down after pioneering what may be a model for tackling infectious diseases in developing countries.
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Immunotherapy Trials Show Renewed Application for Breast Cancer

Douglas Yee, MD, director of the Masonic Cancer Center, as well as professor of medicine and pharmacology at the University of Minnesota, and a HemOnc Today Editorial Board member, outlines clinical data presented at the San Antonio Breast Cancer Symposium demonstrating potential for the use of immunotherapy drugs for the treatment of breast cancer.

“I think one of the more interesting and exciting themes around this program has been immunotherapy,” Yee told HemOnc Today. “Many of our colleagues in other diseases, particularly melanoma and renal cell cancer, have understood that by actually dealing with immune checkpoints, patients can have a more robust response to treatment. In breast cancer, I think we have been a little slow to address this concept; however, some data presented at this meeting have been particularly exciting.”
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Scientists Tackle Deadly Ebola Virus with Two Experimental DNA Vaccines

Scientists may have come up with a way to tackle the deadly Ebola virus. They've created two experimental DNA vaccines to prevent Ebola and the closely related Marburg virus that are not only safe, but generated a similar immune response in healthy Ugandan adults as reported in healthy U.S. adults earlier this year.

In the phase 1 trial, the researchers enrolled 108 adults between the ages of 18 and 50 from Uganda. Each volunteer was randomly assigned to receive an intramuscular injection of either the Ebola vaccine, Marburg vaccine, both vaccines or placebo. In the end, the researchers found that the vaccines given separately and together were safe and stimulated an immune response in the form of neutralizing antibodies and T-cells against the virus proteins. In addition, four weeks after the third injection, just over half of the volunteers had an antibody response to the Ebola Zaire protein.
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Inovio Pharmaceuticals Initiates DNA Immunotherapy Trial for Breast, Lung and Pancreatic Cancers

Inovio Pharmaceuticals, Inc. announced it has initiated a phase I trial of its hTERT DNA immunotherapy (INO-1400) alone or in combination with Inovio's IL-12 immune activator (INO-9012) in adults with breast, lung, or pancreatic cancer at high risk of relapse after surgery and other cancer treatments. Because high levels of hTERT (human telomerase reverse transcriptase) expression are found in 85% of human cancers, Inovio's cancer candidate holds the potential as a broad spectrum cancer therapeutic.

This human trial is an open label, dose escalation study in subjects with breast, lung, or pancreatic cancer at high risk of relapse after surgery and other treatments including chemotherapy and radiation. Approximately 54 subjects will be enrolled into one of six treatment groups and receive INO-1400 alone or in combination with INO-9012, Inovio's immune activator. The study will be conducted at the University of Pennsylvania's Abramson Cancer Center, which will fund all site-specific clinical study costs.
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Researchers Treat Heart Attacks with New Gene Therapy Based on Telomerase Enzyme

The enzyme telomerase repairs cell damage produced by aging, and has been used successfully in therapies to lengthen the life of mice. Now it has been observed that it could also be used to cure illnesses related to the aging process. Researchers at the Spanish National Cancer Research Centre (CNIO) have for the first time treated myocardial infarction with telomerase by designing a very innovative strategy: a gene therapy that reactivates the telomerase gene only in the heart of adult mice, thus increasing survival rates in those animals by 17% following a heart attack.
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Key Discovery in Understanding Successes and Failures of Immunotherapy

A key discovery has advanced the understanding of why some patients respond to ipilimumab, an immunotherapy drug, while others do not. The report, published in the New England Journal of Medicine (2014;371[23]:2189-2199), indicated that patients who respond to ipilimumab have cancer cells that carry a high number of gene mutations. Some of these mutations make tumors more visible to the immune system, and therefore easier to fight.

"We are learning that there are few treatments that don't have some footprint in the cancer genome," said lead researcher Timothy Chan, MD, PhD, vice chair of Radiation Oncology at Memorial Sloan Kettering Cancer Center in New York, New York. "For the first time, it might be feasible to develop a reliable diagnostic test to help guide treatment decisions by predicting who will respond."
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Hodgkin Lymphoma Responds to Immunotherapy

Patients with classical Hodgkin lymphoma whose cancer had failed to respond to other treatments showed improvement in studies that tested the effect of two PD-1 inhibitors - immunotherapy drugs that help the immune system recognize and attack cancer. The results of the two phase 1 trials are being presented at the 56th Annual Meeting of the American Society of Hematology in San Francisco, CA.
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New Anti-Malaria Compound Destroys Parasite Quickly

One of the challenges in the fight against malaria - a disease that threatens half the world's population and kills a child in Africa every minute - is that it rapidly becomes resistant to new drugs. To sustain their effectiveness, new drugs need to act fast and eliminate the parasite before it has a chance to develop resistance. Now, a new study offers such a compound - in mice it removed all traces of malaria parasite within 48 hours.
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