Not exact matches
The
treatment was approved for the devastating rare disease Duchenne muscular dystrophy but created major divisions within the FDA because it simply hadn't demonstrated a whole lot of efficacy, meaning the pricey
therapy is essentially being subjected to a real - world clinical
test.
Just this year, over 50 U.S. genetics and gene
therapy startups raised at least $ 1 million to support genetics - based
treatments, including speeding trails, improving accuracy of
tests, and providing better platforms.
This is the second high - profile
therapy to
test out the fledgling pay - for - performance model in the U.S. behind Novartis» blood cancer
treatment Kymriah.
Though it's not a perfect
treatment, Eli Lilly's solanezumab — a drug 15 years in the making — will pass muster in clinical
tests and be well on its way to FDA approval, as a
therapy for mild Alzheimer's, by year's end.
«Precision medicine is already positively affecting providers (for example, by reducing ED visits by 30 % through application of molecular profiling
treatment strategy); payers ($ 25 billion expected annual spending on genetic
tests by 2021, and 45 % of FDA approvals were geared toward targeted
therapies in 2013); and pharma (the pharmacogenomics market is expected to be $ 7.5 billion by 2017).»
This active IND enables Aura to begin initial clinical
testing of AU - 011, a unique targeted
therapy that could transform the primary
treatment of patients with OM, a rare and life - threatening disease.
Nathan's
treatment included oxygen
therapy, antibiotics to treat infection, nitric oxide inhalation, and frequent blood
tests to see if he was getting enough oxygen.
If
treatments become available for childhood disorders such as spinal muscular atrophy, it will mean children should be
tested for the condition at birth so they can begin
therapy as soon as possible.
He and his lab, for example, along with the company Neurologix based in Fort Lee, N.J., are conducting a clinical trial to
test a similar gene -
therapy treatment for Parkinson's disease.
These human genes were also protective against alpha - synuclein - induced death, suggesting that they could be worth
testing as gene
therapy treatments for Parkinson's disease, Lu says.
In another ongoing study, psychiatrist Michael Mithoefer of Charleston, South Carolina, is
testing MDMA (3,4 - methylenedioxymethamphetamine) on people suffering from severe post-traumatic stress disorder (PTSD), including rape victims and Iraq War veterans who have not gotten any relief from conventional
treatments such as antidepressants and
therapy.
The Wyss team believes the ability of the human gut - on - a-chip to culture the microbiome with human gut cells also holds promise for the field of precision medicine, where a patient's own cells and gut microbiota could one day be cultured inside a gut - on - a-chip for
testing different
therapies and identifying an individualized
treatment strategy.
Some
treatments for mitochondrial diseases aim to boost ATP production, says Michio Hirano, a neurologist at Columbia University, who is running the North American Mitochondrial Disease Consortium, which aims to characterize and
test new
therapies for mitochondrial diseases.
Luznik says that the researchers» next step will be to
test the short course
therapy in a phase III randomized clinical trial that would directly compare results in patients who receive the cyclophosphamide
treatment with those who receive either a separate, experimental approach to prevent GVHD or the more traditional six - month immunosuppressive
therapy.
GABA and related
therapies would have to be
tested in human clinical trials, a process that could take several years, the researchers said, noting that many
treatments that work in mice do not always translate into effective human
therapies.
Dr. Rassool says that a clinical trial is planned to
test whether low doses of a DNMT inhibitor, decitabine, and an investigational PARP inhibitor, talazoparib, can be safely combined and whether this
therapy shows efficacy for AML patients, especially those who can not receive intensive chemotherapy, whose leukemia is resistant to
treatment, or who have experienced a relapse after
treatment.
While many trials
test new drugs, trials can also
test treatment approaches (medical
therapy vs surgical
therapy) or lifestyle modifications (one diet vs another).
Dr Sian Clarke from the London School of Hygiene & Tropical Medicine, also a principal investigator in the research, said: «This study shows that rapid diagnostic
tests can improve the use of artemisinin - based combination
therapies — the most effective
treatment for malaria — in drug shops, but it's not without its challenges.
In the past year, the South Korean Food and Drug Administration (FDA) has approved the world's first three stem - cell
treatments — Hearticellgram - AMI, Cupistem and Cartistem — which followed on the heels of clinical
tests for human embryonic stem - cell
therapies approved in 2010, according to the health ministry.
To that end, in collaboration with the University of Zurich and MD Anderson Cancer Center, the researchers
tested melanoma tumor samples from human patients undergoing
treatment with the same targeted
therapies.
In addition to providing insights regarding selection of
treatment, this blood
test may also prove helpful in assessing the presence of residual disease, recurrence, or relapse and detection of
therapy - resistant cancer cells more quickly than conventional tissue - based methods.
The study was a randomized phase II trial
testing whether the addition of local
treatment, in the form of consolidative radiation
therapy, to the standard
treatment of systemic
therapy improved progression - free survival for patients with limited metastatic NSCLC.
Updating the 2008 guidelines, the new guidelines for the diagnosis and
treatment of aspergillosis highlight the increased evidence for antifungal
therapy recommendations as well as diagnostic
tests.
We hope this leads to the ability to design, study and
test new
therapies for every patient on their own cells in the lab, leading to new
treatments and breakthroughs in personalized medicine for individuals with a variety of lung diseases, including cystic fibrosis,» explained lead author Katherine McCauley, a PhD student at BUSM.
Epigenetic
therapies are rapidly being developed as cancer
treatments, opening the possibility to also
test these drugs for antiviral activity.
The nanotechnology approach that is being
tested for inhibitor control could also improve the haemophilia
treatment that is now at the cutting edge of clinical research: gene
therapy.
The emergency
treatment would be the first
test of their gene
therapy approach over such a large and severely damaged area.
«We can also use these models to
test the efficacy and safety of new cancer
treatment therapies and cancer drugs.»
Although there remains no cure for Alzheimer's, researchers hope that
testing potential drugs or
therapies prior to mental decline will prove to be more successful than previous attempts at finding the most effective
treatment in the battle against the debilitating disease.
It is not about cost or spending, but about weighing risks and benefits of
tests and
therapies, so that people receive the best available
treatment.
The clinical trial named METOCARD - CNIC is the first to
test metoprolol
therapy, at a cost less than three dollars (or less than two euros), in heart attack patients undergoing standard angioplasty
treatment procedures.
Currently, Doody is
testing in preclinical models whether combining this agent with other, known rheumatoid arthritis
treatments results in more efficacious
therapies.
Deploying a point - of - care
test for infant diagnosis of HIV using a portable, battery - operated device may result in more timely initiation of drug
therapy, decreasing the number of children who are diagnosed with HIV weeks or months after birth and never receive
treatment.
Other
therapies need to be
tested for the
treatment of patients at risk for lymph node metastasis,» says Padera, who is an assistant professor of radiation oncology at Harvard Medical School.
Reference materials are critical to properly evaluate the next - generation of gene sequencing and genetic
testing methods that will increase the reliability and effectiveness of precision medicine (also known as «personalized medicine»), in which a person's genetic profile is used to create
treatments and
therapies unique to that individual.
«These results are powerful in that early diagnosis of mental illness could possibly happen with a simple blood
test, leading to better interventions,
therapy and
treatment options.»
Training lay volunteers or «feeding buddies,» to provide information about breastfeeding, infant health and HIV
treatment protocols may diminish the damaging impact of stigma on an HIV positive mother's ability to safely feed her child and adhere to World Health Organization (WHO) guidelines, which recommend exclusive breastfeeding, antiretroviral
therapy and early infant
testing.
Although doctors have long incorporated personal information like family history into
treatment plans, personalized medicine holds the promise of revolutionizing medical care by using knowledge of molecular biology and genetics that will allow more precise diagnoses, better diagnostic
tests, greater predictability of disease course, more successful
therapies by targeting the right
treatments to the right patients, and improved patient safety by selecting drugs and their proper dosage to reduce adverse side effects.
This
therapy is a leading example of precision medicine, where
treatment is based on a
test for genetic mutations.
«We urgently need markers to predict which
therapies are going to be effective and which will not be effective in individual patients with advanced prostate cancer,» said lead study author Emmanuel Antonarakis, MD, an assistant professor of oncology and urology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins in Baltimore, Md. «AR - V7
testing may be extremely valuable in guiding
treatment decisions for men with hormone - resistant disease in the near future.»
The research, part of a phase I clinical trial to
test the safety of the
treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human Gene
Therapy.
Alley said there are already plans for trials that will
test combination
therapies, which will utilize pembrolizumab in conjunction with other
treatments.
Platinum - based
therapies are being
tested in clinical trials for triple - negative breast cancer, and evaluation of tumor - infiltrating lymphocytes is an important factor in determining response to this
treatment.
A new blood
test using gold nanoparticles could soon give oncologists an early and more accurate prognosis of how cancer
treatment is progressing and help guide the on - going
therapy of patients.
Gene
therapy: Généthon starts a clinical trial to
test a gene
therapy treatment for a rare liver disease, Crigler - Najjar Syndrome
Thus, the results open the way to discover and
test potential ALS
therapies targeted at the early stages of the disease, when
treatment is most likely to be effective.
UC San Diego Health joins national clinical trial
testing a gene
therapy treatment for individuals with hemophilia B.
A new blood
test using gold nanoparticles could soon give oncologists an early and more accurate prognosis of how cancer
treatment is progressing and help guide the ongoing
therapy of patients.
Through strategic partnerships with industry and nonprofit organizations, we gain access to a portfolio of priority drugs for
testing within our expert focus groups, including checkpoint inhibitors, therapeutic vaccines, innate immune stimulants, targeted
therapies, and many other promising
treatments and technologies with high therapeutic potential.
The Food and Drug Administration (FDA) approved nivolumab as second - line
therapy for these patients this past July, and clinical trials are underway to
test the drug as frontline
treatment.