Indeed, the find suggests that
testing human therapies on mice and other mammalian species may have sent us down the garden path.
Not exact matches
Strikingly for Agios and partner Celgene, the
therapy was in
human clinical
testing not so long ago (there was just a three year gap between the first
human studies and Agios» regulatory submission to the FDA).
Of course, there is still a long way to go before this particular method will be
tested on
humans (it was
tested on mice), and an even longer way to go before it'll be used in medical
therapies (if it ever will translate into
therapies), but one thing is becoming clear: We need not compromise our moral principles and rush into government - funded embryo - destructive research.
The researchers caution that the booster
therapy used in their new study will not be available on the market or even for use in
human trials anytime soon; it must await years of animal
testing for safety and effectiveness first.
These allusions to the past aren't surprising considering how drastically the clinical trial changed gene
therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for Human Gene Therapy, where the test took
therapy and, in particular, the career of James M. Wilson, the medical geneticist who headed Penn's Institute for
Human Gene
Therapy, where the test took
Therapy, where the
test took place.
Geron was bigger and better funded than ACT, and it was the first company to be approved by the US Food and Drug Administration (FDA) to
test a
therapy in
humans based on embryonic stem (ES) cells.
Although gene
therapy research has made great strides in recent years, it has yet to be widely deployed, and no CRISPR - edited genes have yet been
tested for safety or efficacy in
human clinical trials.
This clinical study, published in the Proceedings of the National Academy of Sciences,
tested the possibility of imaging inflammation in the pancreas of
human volunteers using ferumoxytol, a coated iron nanoparticle approved by the FDA as an iron replacement
therapy, and MRI.
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.
Human testing is years away, but gene therapy has already become a controversy in professional and amateur sports, where steroids, human growth hormone, and other performance - enhancing drugs have been a problem for y
Human testing is years away, but gene
therapy has already become a controversy in professional and amateur sports, where steroids,
human growth hormone, and other performance - enhancing drugs have been a problem for y
human growth hormone, and other performance - enhancing drugs have been a problem for years.
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.
In the 1990s scientists such as himself, he explains, were too caught up in the promise of gene
therapy to realize that they did not know enough about it to warrant
human testing.
«The mouse is one of the most utilised models for studying
human biology and we use it for creating models of
human illnesses and
testing new drugs and
therapies.
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.
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.
For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights into
human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and
test a «curative
therapy» for the first molecular disease: sickle cell disease.
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.
The lack of discrimination in the viruses used to carry therapeutic genes has been a major obstacle to
testing gene
therapy in
humans.
The new epidermis, grown from
human pluripotent stem cells, offers a cost - effective alternative lab model for
testing drugs and cosmetics, and could also help to develop new
therapies for rare and common skin disorders.
In the new study, to
test whether the same observation was true in
humans, Dr. Rostami and colleagues
tested blood samples of patients with MS who had not yet received
therapy, and those currently being treated with INF - β, a commonly used
therapy.
Such research is not currently eligible for NIH funding, and is still years from producing
therapies that regulators would have to green light before they could be
tested in
humans.
Mini-organ models promise enormous advantages for understanding basic
human biology, teasing apart
human disease processes, and offering an accurate
testing ground for finding or vetting drug
therapies.
The first
human trials are underway to
test whether antibody
therapy can treat schizophrenia and multiple sclerosis.
Human testing is years away, but gene therapy has already become a controversy in professional and amateur sports, where steroids, human growth hormones, and other performance - enhancing drugs have been a problem for y
Human testing is years away, but gene
therapy has already become a controversy in professional and amateur sports, where steroids,
human growth hormones, and other performance - enhancing drugs have been a problem for y
human growth hormones, and other performance - enhancing drugs have been a problem for years.
«First, it would allow us to understand what DUX4 does in muscle to cause muscle loss, and second, it would provide a system in which efficacy of potential
therapies could be evaluated before they are
tested in
humans.»
If successful, it could eventually pave the way for gene -
therapy tests of
humans with MS.
Although the gene / cell
therapy strategy was highly successful in laboratory mice, the authors stressed that additional research and
testing are needed before the
therapy could be
tested in
humans.
DFMO is being investigated in
human clinical trials to treat some types of cancer, but it hasn't been
tested as a potential
therapy for Alzheimer's.
Now, the work has brought the researchers close to a new
therapy for obesity, which they report works well in monkeys and is edging toward
human testing.
When
tested in laboratory samples of leukemia cells and in animals with
human - like leukemia, the approach caused cancer cells to die much more quickly than with conventional targeted
therapies.
Dr. Hashino said these findings are «a real game changer, because up until now, potential drugs or
therapies have been
tested on animal cells, which often behave differently from
human cells.»
«Dish - grown
human inner ear tissues offer unprecedented opportunities to develop and
test new
therapies for various inner ear disorders.»
Gene
therapy has been rhapsodized and vilified in its nearly two decades of
human testing, helping some and making others sicker.
A valuable result of this work, they both agree, is that it sets up a way to
test the effects of microbial
therapies on
human gut bacteria (even though the bugs are living in a mouse).
With miglustat
therapy available off - label in the U.S. and potential
therapies in clinical trials, the researchers said their goal is to see the
test added to the recommended uniform screening panel defined by the U.S. Department of Health and
Human Services (HHS).
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.
These powerful ASOs are identified by the Krainer team as viable for
testing in mouse models — the next step in the process of developing new
human therapies.
His major research interests are
human neuroanatomy and neuropathology and the use of animal models to
test novel
therapies for neurodegenerative diseases including Alzheimer's disease.
All this information is necessary before preclinical safety
testing and product development for an AMD
therapy in
humans can take place.
Specific Aim 2: Having utilized mutant mice to identify the key players in AMD pathology from Specific Aim 1, we will develop and
test molecules that target either the anaphylatoxins or the MAC, or both, in the context of practical
therapies that can be utilized in
humans.
Still, BioViva has pressed ahead with telomerase gene
therapy, and factions within the research community are also aiming for the same outcome of
human tests, though through more conventional channels.
At its conclusion, we will have a candidate gene
therapy for AMD; however, such
therapy will need to be
tested for safety before being able to advance into
humans.
The third Objective (IRF3) brings together promising scientists and clinicians from the University of Pennsylvania and the Rockefeller University to combine gene
therapy strategies, independently
tested in
humans, with the goal of engineering, growing, and administering killer cells that are uniquely empowered to find and kill HIV - infected cells.
Now two more such
therapies have entered
human testing.
This «humanized» mouse is susceptible to
human liver infections and responds to
human drug treatments, providing a new way to
test novel
therapies for debilitating
human liver diseases and other diseases with liver involvement such as malaria.
For only the second time, the Food and Drug Administration approved a company's request to
test an embryonic stem cell - based
therapy on
human patients.
Gene and cell
therapies have made important medical advances over the past three decade, developing technologies and
testing novel
therapies in multiple
human clinical trials of many diseases.
To build upon the encouraging early discoveries, Helmsley renewed and expanded its Crohn's funding for the Institute in 2013 to begin new work with three major aims: 1) continue studies of individual genes to determine how genetic differences between Crohn's patients and healthy individuals contribute to the disease; 2) evaluate promising small molecules in disease - relevant studies and prioritize insights from genetics to help develop novel therapeutics; and 3) begin basic experimentation in animal models with Crohn's disease to provide the data necessary to begin
testing new
therapies in
humans.
Microscopic models — half living, half not — may prove more reliable than animals in explaining
human disease and
testing therapies.