Bellicum is among the flurry of biotechs investing heavily into
cell therapies such as experimental chimeric antigen receptor T - cell (CAR - T) treatments for cancer (this is the next - gen treatment that involves reprogramming immune cells to become cancer killers and has shown promise in blood cancers, which Bellicum specializes in).
Not exact matches
New technologies
such as gene and
cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.
With major clinical successes in areas
such as CAR - T, gene
therapy, immune - oncology,
cell therapy and gene editing, many see 2017 as the year that biotech really came of age.
If ESCR using «excess» embryos from IVE» continues, the next step will likely be the pursuit of
such «therapeutic» cloning — the creation of embryos through somatic
cell nuclear transfer (SCNT) to provide individually tailored stem
cell therapies.
During the sessions, U.S. and Cuban scientists explored
such topics as the molecular mechanisms cancer
cells employ to evade the body's immune system, new tools to image and manipulate that system, and ways to rethink how
such therapies can best be deployed to reach patients where they receive health services.
Tissue engineering provides a more practical means for researchers to study
cell behavior,
such as cancer
cell resistance to
therapy, and test new drugs or combinations of drugs to treat many diseases.
Researchers at the University of Louisville have discovered a mechanism involved in skeletal muscle repair that may enable clinicians to boost the effectiveness of adult stem
cell therapies for diseases
such as muscular dystrophy.
The act of reprogramming
cells to make them as capable as ones from embryos apparently can result in aberrant
cells that age and die abnormally, suggesting there is a long way to go to prove
such cells are really like embryonic stem
cells and can find use in
therapies.
Researchers
such as geneticist Richard King of the University of Minnesota and
cell biologist Vitali Alexeev of Thomas Jefferson University are working on gene
therapies or drugs that would fix albinism - causing mutations.
New products and developments,
such as new drugs for cancer, fresh
therapies for rare diseases, progress in medications for HIV / AIDS, and advances in stem
cell research had the greatest positive impact.
To acquire new insights into the biology and possible
therapy of these tumors, Feigin et al. looked for aberrant expression of G protein — coupled receptors,
cell signaling proteins that have been successfully targeted for treatment of other disorders
such as depression.
Eye diseases —
such as age - related macular degeneration, as well as a genetic condition called Stargardt's macular dystrophy that afflicts young people — are considered excellent candidates for stem
cell therapy because the eye is an immune - privileged site, meaning transplanted
cells are not as likely to be rejected as foreign compared with transplants elsewhere.
These include the ability to bring new, innovative products to the market; progress in oncology,
such as the approval of Genentech's drug Avastin for breast cancer and advances in the use of gene
therapy, despite some setbacks; continuing progress in research on stem
cells; the emergence of treatments for previously untreated diseases; and solutions for food and fuel shortages,
such as biocrops and biofuels.
Being able to acquire new technologies, as well as becoming more innovative internally by venturing into new research areas,
such as stem
cell and gene
therapy research, have allowed Genzyme to maintain its edge.
Two months after
therapy, tissue analysis showed less scarring and higher levels of regenerative agents
such as fibroblast growth factor — which accelerate healing — in rats treated with SIS plus stem
cells compared with those treated with SIS alone.
Still, a few stem
cell therapies have now been approved,
such as a treatment available in India that takes stem
cells from the patient's eye in order to regrow the surface of their cornea, and a US product based on other people's bone stem
cells.
Clinical trials that charge enrollees to participate are ostensibly aimed at giving patients early access to promising
therapies — often in the fields of stem
cells or aging reversal — that are too unusual or have too little profit potential to get funding from traditional sources
such as companies, foundations, or the National Institutes of Health.
Developing safe and effective
therapies for conditions
such as peripheral nerve disorders requires the ability to take investigations from
cells in a petri dish to patients in a clinic.
By varying the compositions of lipids, cues, and diffusible factors in the scaffolds, we engineered a very versatile and flexible platform that can be used to amplify specific T
cell populations from blood samples, and that could be deployed in existing
therapies such as CAR - T
cell therapies,» said Mooney, Ph.D., a Core Faculty member at the Wyss Institute and leader of its Immunomaterials Platform.
«The treatment of multiple myeloma has improved significantly in recent years with the introduction of
therapies such as proteasome inhibitors [which interfere with tumor
cells» protein - disposal system] and potent immuno - modulatory agents,» said the paper's senior author and lead investigator, Paul Richardson, MD, clinical program leader and director of clinical research at Dana - Farber's Jerome Lipper Multiple Myeloma Center, and the R.J. Corman professor at Harvard Medical School.
Lanza says eye disease is a good place to start with
such cell therapies because the eye doesn't reject foreign tissues, so no imunnosuppressive drugs are necessary.
Trapnell and Suzuki were prompted to test the novel macrophage transplantation
therapy by studies showing that resident macrophage populations (
such as those residing in the lung) can self - maintain without the
cells having to regenerate directly from the bone marrow.
On the flipside, targeting this growth factor or BCL - 2 could reduce NK
cell numbers and offer potential
therapies for immune disorders
such as some types of autoimmune diseases, sepsis or graft versus host disease, a side effect of bone marrow transplants.
But many stem
cell scientists in Italy and abroad say it's too early for
such a study because there is little to suggest that the
therapy, whose details remain unpublished, might work.
«Perhaps we can also combine resident memory T
cells with other
therapies such as checkpoint modulators (PD1 blockade) that would ensure a more hospitable environment for the reinfused
cells.»
This makes adipose tissue, in theory, a readily available reservoir for regenerative
therapies such as bone healing if doctors can get enough of those
cells and compel them to produce bone.
Broad claims of success have sparked the ire of scientists, who insist the complexities of stem
cell therapies make
such success unlikely.
He envisions some sort of visual aid «designed to maximize the activity of these
cells but notes that even if
such therapies are possible, they won't be available anytime soon.
Many groups, including Urnov's company, are already using gene - editing tools to develop
therapies that correct genetic defects in people (
such as by editing white blood
cells).
Experimental approaches
such as gene
therapy are also being investigated, but Dr. Rudnicki's research suggests that these approaches will have to be modified so that they target muscle stem
cells as well as muscle fibres.
Genetic
therapies,
such as those made from DNA or RNA, face challenges because of the difficulty in delivering the nucleic acid to the right
cells.
Rivals
such as Pfizer and Sanofi are also investing, and overall financing for gene and gene - modified
cell therapies reached $ 1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.
Stem -
cell experts contacted by Nature insist that
such therapies are not ready for the clinic and say that some may even endanger patients» health.
The
therapy employs a virus to insert a gene for a common ion channel into normally blind
cells of the retina that survive after the light - responsive rod and cone photoreceptor
cells die as a result of diseases
such as retinitis pigmentosa.
A fix for broken rat hearts Scientists this week successfully implanted human embryonic stem
cells into rats that suffered heart attacks, coming a heartbeat closer to realizing the full potential of
such therapy.
Under the proposed law, gene
therapy will be approved only for the treatment of people with genetic diseases
such as cystic fibrosis and will not be allowed in germ
cells, where genetic alterations would be passed on to the next generation.
Ideally,
therapy for autoimmune diseases should eliminate pathogenic autoimmune
cells while sparing protective immunity, but feasible strategies for
such an approach have been elusive.
Because diseases
such as cancer tend to evade detection by T -
cells» receptors, allowing a tumor to grow unchecked, scientists have long sought «intel» on this process as a means of developing
therapies that target malignant
cells, but leave healthy
cells alone.
Because RNA carries the genetic message from DNA to the
cell's proteinmaking factories, or can directly perform acts
such as gene regulation, it, too, is an appealing target for
therapies.
One solution might be, however, using gene
therapy to stimulate
such reprogramming of
cells and thus to strengthen the self - healing abilities of the heart.
The enzyme
therapy also inhibited sunburn symptoms
such as reddening and death of skin
cells.
Such rapid and extensive degradation suggests that conjugates may be able to prevent or hinder cancer
cells from developing resistance to targeted
therapies, the researchers state.
Hydrogels, noted for their biomimetic properties, are the leading materials for biomedical applications,
such as drug delivery and stem
cell therapy.
«Our researchers were able to identify a change in a specific mark in the proteins that wrap the DNA — called histones — in
such a way that makes repair much faster in cancer
cells, increasing their resistance to
therapy.»
B
cell signaling pathways are the current targets of several
therapies used to treat B
cell malignancies
such as CLL.
He hopes that Cas9 - based
therapies for T
cell - related disorders, which include autoimmune diseases as well as immunodeficiencies
such as «bubble boy disease,» will enter the clinic in the future.
She says that even though Sasai's team hasn't yet grown an entire eyeball in a dish, the work shows that it's possible to grow specific eye structures,
such as retinas, from stem
cells in a great enough quantity that they could be used in
therapy.
Technology
such as this, scientists said, may have a promising future in the identification and surgical removal of malignant tumors, as well as using near - infrared light
therapies that can kill remaining cancer
cells, both by mild heating of them and generating reactive oxygen species that can also kill them.
In further studies, they plan to explore the role of autophagy in immune reactions toward other tumor
cell types, to determine whether
such therapies might be effective in a broad range of cancers.
Indeed, inhibiting CXCR4 function systemically with the small molecule antagonist plerixafor results in the peripheral mobilization of hematopoetic stem
cells, thus mitigating the potential of
such therapy for long - term anti-retroviral
therapy.