Sentences with phrase «therapy trial which»

Twelve patients, about half of them women, will take part in Britain's second gene therapy trial which began last week at two centres in Oxford and Cambridge.

Not exact matches

On the logistics end, it is easier to manufacture certain stem - cell therapies, which will be key for human trials like the heart - muscle - regeneration program.

Amgen unveiled some promising news during the American Heart Association's annual meeting involving its next - gen cholesterol - busting medication Repatha, which has been shown to significantly reduce the level of «bad» cholesterol in patients, announcing that more than 60 % of patients who were receiving the therapy in a trial had a reduction in arterial plaque.

The idea for the database is to create a single repository for both patients» clinical trial data and the genetic specifics of their cancers, including which therapies they've been given and how effective they were.

In May, Kite disclosed that one person had died in a clinical trial for its late - stage CAR - T therapy from cerebral edema, a condition in which excessive fluid causes the brain to swell.

These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical trials involving investigational compounds; Gilead's ability to initiate clinical trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all, for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).

Dr. Newman added that rival CAR T cell therapy developer Kite Pharma was still likelier to generate more revenue than Juno with KTE - C19, which last week completed patient enrollment in the Phase II portion of the ZUMA - 1 trial in patients with diffuse large B - cell lymphoma.

Revenue from selling Celgene's therapies will fund the company's late - stage pipeline, which boasts three new cancer therapy candidates in pivotal trials.

• A Randomised Controlled Trial in which depressed pregnant women received twice weekly massage therapy from their partners found those who received the massage reporting less depressed mood, anxiety and anger and better relationship quality than women in the control group (Field et al, 2008).

«There are currently no therapies which successfully reverse the damage seen in the more than 12,000 individuals who suffer a spinal cord injury each year in the United States alone,» says Dr. Richard G. Fessler, professor of neurological surgery at Rush University Medical Center and principal investigator for the Phase 1 clinical trial involving AST - OPC1 (oligodendrocyte progenitor cells).

The trial he conducted tested the safety of a therapy for ornithine transcarbamylase (OTC) deficiency, a rare disorder in which the liver lacks a functional copy of the OTC gene.

Strikingly, the treatment approach didn't make a difference to 10 - year survival: Ninety - nine percent of the more than 1600 men in the trial were alive after a decade, whether they had initially received radical surgery, radiation, or active monitoring, which meant no immediate therapy, but intervention as needed.

The Phase I clinical trial of OMP - 54F28 (FZD8 - Fc) is an open - label dose escalation study in patients with advanced solid tumors for which there was no remaining standard curative therapy.

It typically takes many years to initiate such trials because of the stringent safety testing that must be done before testing in humans begins, but Reynolds said it may be possible to move faster as the therapy only involves modifying a patient's dietary intake and supplementing with a medium - chain triglyceride oil, both of which have no known side effects.

One clinical trial involves the drug CGF166, a one - time gene therapy, which, if proven successful in humans, could regenerate new hair cells within the cochlea that can signal the part of the brain that processes sound.

Another candidate, AVXS - 101 (AveXis), is a gene therapy approach to increase SMN levels, which is in Phase I clinical trials.

Pain therapies are highly subject to placebo effects, which often boost initial results and then whither away during later trials.

The experimental therapy, which involves administering a drug currently used in cancer therapy trials, treats cocaine addiction by inhibiting memories responsible for cravings.

He was catching potential investors at an inauspicious time: Many were smarting from disappointing trials of RNA interference therapies, which use short, double - stranded RNA to disrupt the production of disease - causing proteins.

Many point to the Jesse Gelsinger case, in which a teenager died during a gene therapy clinical trial.

He added: «The most difficult decision was whether or not to have a period of initial triple therapy after stent implantation, given the findings of the WOEST trial which implied an adverse effect of triple therapy on bleeding and death.

The endpoint is a proof - of - mechanism clinical trial, after which Pfizer has the first option to develop the potential therapy.

Meanwhile, his research teams are moving several other novel therapies toward clinical trials, each of which has potential for use in the synergistic model of cancer treatment that Black believes will offer the best hope for his patients.

The trial investigated the drug in two separate cohorts of patients: Cohort A, which included 170 patients with heavily pretreated metastatic triple negative breast cancer (mTNBC) regardless of PD - L1 expression, and Cohort B, which included 52 patients with PD - L1 - positive tumors who received it as first - line therapy.

The proposed clinical trial, in which researchers would use CRISPR to engineer immune cells to fight cancer, won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health, a panel that has traditionally vetted the safety and ethics of gene therapy trials funded by the U.S. government and others.

ACT, which has almost gone under several times in recent years, may now be on the verge of conducting the nation's first trial of an embryonic stem cell therapy, says Chief Scientific Officer Robert Lanza.

After seeing promising results in phase 1 of the Pediatric Leukemia Adoptive Therapy (PLAT - 02) trial with 93 percent of patients with relapsed or refractory acute lymphoblastic leukemia (ALL) achieving complete initial remission, researchers at Seattle Children's are continuing their quest to improve the experimental therapy and reduce the rate of relapse, which is about 50 pTherapy (PLAT - 02) trial with 93 percent of patients with relapsed or refractory acute lymphoblastic leukemia (ALL) achieving complete initial remission, researchers at Seattle Children's are continuing their quest to improve the experimental therapy and reduce the rate of relapse, which is about 50 ptherapy and reduce the rate of relapse, which is about 50 percent.

The lab is also working with the Breast Oncology Program at UCSF to make this data part of an adaptive clinical trial called I - SPY, which lets researchers identify the most effective therapies based on patient molecular profiling, and is collaborating with members of the UCSF Institute for Computational Health Sciences (ICHS) to put these and other public data into a centralized database that clinicians can access through an app to help make the most appropriate treatment decisions.

Most gene - therapy trials use viruses to deliver genes to a patient's cells, and most of those viruses are retroviruses, which have the ability to neatly splice their genes — and the human gene they're carrying — into a cell's chromosomes.

«The good news is that this finding predicts that patients missing either gene should be sensitive to new therapies targeting focal adhesion enzymes, which are currently being tested in early - stage clinical trials,» says Shaw, who is also a member of the Moores Cancer Center and an adjunct professor at the University of California, San Diego.

Commenting on the trial, Dr Alice Shaw, director of thoracic oncology at the Massachusetts General Hospital Cancer Centre in Boston, US, said: «This is the first randomised study to examine how a second generation ALK inhibitor compares to standard second line chemotherapy in ALK positive patients who failed the standard first line therapy, which currently is crizotinib.»

At a meeting last week of the International Society for Biological Therapy of Cancer in San Diego, California, Oncolytics Biotech of Calgary in Alberta, Canada, reported positive results from two trials of its drug Reolysin, which contains reovirus.

Dr. Levine directs the Clinical Cell and Vaccine Production Facility (CVPF), which develops, manufactures, and tests novel cell and gene therapies in clinical trials at Penn and collaborating institutions.

The FDA's medical reviewer's recommendation for approval says, in part, that more trials before approval would «significantly delay effective therapy,» which not only reduces body weight but exerts favorable effects on blood pressure and myocardial oxygen for «patients with a serious disease condition with few treatment options.»

The phase II trial, which involves 28 kids from 16 countries, is the first to evaluate a therapy in humans against this disease.

The objective of the «SOSTA - net Trial,» led by Christine Freitag and coordinated by Hannah Cholemkery and in which six university hospitals in Germany participated, was to examine whether the social responsiveness of children and adolescents with ASD could be raised by means of group - based behavioural therapy.

Over the past two years, investigators from the Perelman School of Medicine at the University of Pennsylvania have reported results from a human trial in GBM using chimeric antigen receptor (CAR) T cell therapy, through which patients» own T cells were engineered to track down and kill cancer cells that express a tumor - specific protein known as EGFRvIII.

He says that if future trials are successful, the experimental vaccine, which can be made relatively quickly and cheaply, could become a short - term therapy administered immediately after diagnosis to try to keep tumors in check.

All patients also received standard medical therapy throughout the trial, which in a vast majority of cases included treatment with statins or other cholesterol - lowering drugs.

The LCA2 trials are a rare success for the field of gene therapy, which has also cured children with the immune disorder known as bubble boy disease.

The Phase 2 trial — which will test an existing anti-inflammatory's ability to block inflammation and pyroptosis in HIV - infected people — promises to validate a variety of expected advantages to this therapy.

The small feasibility trial, which involved 12 people with treatment - resistant depression, found that psilocybin was safe and well - tolerated and that, when given alongside supportive therapy, helped reduce symptoms of depression in about half of the participants at 3 months post-treatment.

Formerly a research leader at CHOP, High pursued groundbreaking preclinical investigations in hemophilia B gene therapy and provided scientific expertise to previous gene therapy trials in hemophilia and other genetic disorders at CHOP before moving to Spark Therapeutics, which was spun off from CHOP in 2013.

The results drew attention in part because they come from the lab of James Wilson at the University of Pennsylvania, who led a 1999 trial in which a teenager died from an immune reaction to a different gene therapy vector.

Stricter safety procedures and new ways to weaken pathogens to reduce their risks are leading investigators in industry, universities, and government to take a new look at human challenge trials, which offer a powerful tool for studying diseases and potential therapies.

Women were not included in the first trial of gene therapy, which was mainly concerned with proving the safety of the technique.

His story inspired a book, The Forever Fix, which heralded the LCA trials as part of the wave of successes for gene therapy, which was recovering from several setbacks — including the death of a patient and cancer in others — in the late 1990s and early 2000s.

Spark Therapeutics in Philadelphia, Pennsylvania, which is testing the Bennett group's therapy in a phase III clinical trial needed to seek regulatory approval, also maintains that the NEJM results may not be relevant to their product.

Gene therapy experts are heartened by the new results, which may counteract a wave of negative publicity after the death last year of a volunteer in a gene therapy trial at the University of Pennsylvania (ScienceNOW, 9 December 1999).

But many projects come under public and federal scrutiny — the recent fracas over gene therapy trials is a case in point (Science, 24 March, p. 2163), often because the manner in which they were conducted led to concerns over the safety of the recruited participants or the protection of their confidentiality.