Cellular and gene therapies are now on the cusp of realizing the early promise of treating, and perhaps curing diseases that previously had little or no available therapeutic options. However, the journey from the laboratory to the clinic has been bumpy, with a history of disappointments, especially in the early 1990's where gene therapy had received much hype and publicity.
Discuss an example of knockout mouse model used for disease modelling (Metast...SaniikaRenganadan
Title: Discuss an example of knockout mouse model used for disease modelling
Disease: Metastatic Bladder Cancer
Module: Gene and Tissue Culture Technology
Discuss an example of knockout mouse model used for disease modelling (Metast...SaniikaRenganadan
Title: Discuss an example of knockout mouse model used for disease modelling
Disease: Metastatic Bladder Cancer
Module: Gene and Tissue Culture Technology
Induced Pluripotent Stem Cells and Somatic Cardiac Regeneration— An Explorato...Robert Chen
In the exploratory analysis, the functional pathway networks of nucleostemin and iPS interact via TP53 (tumor protein P53) and Fgf (fibroblast growth factor), which could be further investigated to provide clues for the future research of postnatal cardiac regeneration.
These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.
Role of cancer stem cells in cancer therapyniper hyd
Cancer is one of the leading cause of death, till to date there is no perfect treatment due to its stem cell relapse which is not responding to conventional therapies like chemo, radio, surgery. so we should target the cancer stem cell with novel targets like nano, and immunotherapies.
Journal of Stem Cells Research, Reviews & Reports is a peer-reviewed, open access journal published by Austin Publishers. It provides easy access to high quality Manuscripts in all related aspects covering Stem cell research that focuses on stem cells, which have a capacity to regenerate and develop into other types of cells namely, like kidney cells, liver cells, heart cells, etc. These circulate and function to replace dysfunctional cells, naturally maintaining optimal health. The Journal encourages all the current medical research that is focused on two particular types of stem cells -- adult and embryonic stem cells that are used in various stem cell therapies against many dreadful diseases.
Austin Publishing Group is a successful host of more than hundred peer reviewed, open access journals in various fields of science and medicine with intent to bridge the gap between academia and research access.
Journal of Stem Cells Research, Reviews & Reports accepts original research articles, review articles, case reports, mini reviews, rapid communication, opinions and editorials on all the related aspects of Stem Cells and Cell-Based Therapies.
Induced Pluripotent Stem Cells and Somatic Cardiac Regeneration— An Explorato...Robert Chen
In the exploratory analysis, the functional pathway networks of nucleostemin and iPS interact via TP53 (tumor protein P53) and Fgf (fibroblast growth factor), which could be further investigated to provide clues for the future research of postnatal cardiac regeneration.
These slides discusses on cellular and gene therapy: the use of cells and genes to treat disease. These therapies can be effective on a wide range of previously untreated diseases, such as hematological, ocular, neurodegenerative diseases, and several types of cancers.
Role of cancer stem cells in cancer therapyniper hyd
Cancer is one of the leading cause of death, till to date there is no perfect treatment due to its stem cell relapse which is not responding to conventional therapies like chemo, radio, surgery. so we should target the cancer stem cell with novel targets like nano, and immunotherapies.
Journal of Stem Cells Research, Reviews & Reports is a peer-reviewed, open access journal published by Austin Publishers. It provides easy access to high quality Manuscripts in all related aspects covering Stem cell research that focuses on stem cells, which have a capacity to regenerate and develop into other types of cells namely, like kidney cells, liver cells, heart cells, etc. These circulate and function to replace dysfunctional cells, naturally maintaining optimal health. The Journal encourages all the current medical research that is focused on two particular types of stem cells -- adult and embryonic stem cells that are used in various stem cell therapies against many dreadful diseases.
Austin Publishing Group is a successful host of more than hundred peer reviewed, open access journals in various fields of science and medicine with intent to bridge the gap between academia and research access.
Journal of Stem Cells Research, Reviews & Reports accepts original research articles, review articles, case reports, mini reviews, rapid communication, opinions and editorials on all the related aspects of Stem Cells and Cell-Based Therapies.
Stem Cell, a raw material to be used in tissue engineering unit to have the solution against any of the ailments. Stem cell therapy may be used in treating any multi cellular organism (MCO).
Stem cell therapy may be the solution against most of ailments of multi cellular organism (MCO). It can be worked as a raw material for tissue engineering unit
In conclusion, both cell strains and primary cells have played vital roles in advancing our understanding of cellular processes, disease mechanisms, and drug discovery. While cell strains provide a continuous and convenient supply of cells, primary cells offer a more authentic representation of human biology, enabling researchers to study specific tissues and diseases in a physiologically relevant context. The growth of cell strains and the significance of primary cells have propelled biomedical research forward, leading to breakthroughs in various fields and fostering the development of innovative therapeutic strategies. As technologies continue to evolve, primary cells will remain indispensable in further unraveling the complexities of human health and disease.
Stem-cell therapy in medicine–how far we came and what we can expect?Apollo Hospitals
The name ‘stem-cell’ is making the news in recent times both for good and not. The current articles tries to give a snap shot of the scientific and clinical picture of stem-cells in medicine as of today and discuss what it have to offer in the to the mankind. The article discusses the characters and types of stem-cells, their current indication in therapeutics (both established and upcoming), as well as their use in research. It also gives a brief overview of the current laws guiding its use in clinical practice and the various cultural beliefs associated with the use of same.
For better view, press F5.
As we go through our lives each of us will have very different needs for our own healthcare.
Scientist's are constantly researching to make medical care treatment more personalized.
One way they are doing this is by-
Stem Cells therapy
Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition.
It is also known as regenerative medicine, promotes the reparative response of diseased, dysfunctional or injured tissue using stem cells or their derivatives.
It is the next chapter of organ transplantation and uses cells instead of donor organs, which are limited in supply.
What are Stem cells?
Stem cells are called “master cells”
Stem cells are cells that are undifferentiated.
What are Stem cells?
Steam cells have the potential to become all other kinds of cells in our body.
What are Stem cells?
Types of Stem cells
How stem cell therapy works?
Disease cured by stem cell therapy.
Spinal Cord Injuries
Stem cell treatment of Diabetes mellitus type 1 & 2
Stem cell treatment of Stroke
Cancer treatment
Heart damage
Baldness
Tooth implanting
Deafness and blindness
Have stem cells already been used to treat diseases?
Ethical Consideration of Stem Cell Therapy
As the research method mainly focused on Embryonic Stem Cells, which involves taking tissue from an aborted embryo to get proper material to study. This is typically done just days after conception or between the 5th and 9th week.
Since then, researchers have moved on to more ethical study methods, such as Induced Pluripotent Stem Cells (iPS). iPS is artificially derived from a non-pluripotent cell, such as adult somatic cells.
Nowadays stem cell treatment has been spreaded throughout the world. It has also been grown commercially in developed countries.
It is thought that one day it may be the major key to treat various diseases.
Using stem cells to conduct medical research and treat disease is acceptable?
Don’t know
No
Yes
Do you approve of the extraction of stem cells from human embryos for medical research?
Don’t know
No
Yes
Genomics Solutions - Single Target to Whole Genome AnalysisCovance
With applied Genomics expertise, global co-location with Central Labs and solutions from biomarker discovery to CDx, our genomics solutions will help make your Precision Medicine drug development a reality.
Medical Device and Diagnostics Solutions for Every Stage of Your Product's De...Covance
Getting a medical device to the patient takes more than good technology in today's environment. Compelling evidence and convincing value proposition matter. Competing priorities and differing stakeholder definitions of value are driving the need for creative, connected strategies to get the most from each step in the development process. And - as evidence is gathered - it should be used to inform and iterate regulatory, reimbursement and clinical post-market strategies.
Pharmacovigilance Risk Management for BiosimilarsCovance
This paper focuses on pharmacovigilance (PV) and risk management for biosimilars, the issues and challenges faced in monitoring their safety and possible solutions.
Cell & Gene Therapy post-approval solutions to reduce scientific, regulatory and commercial risks, and accelerate development wherever you are in your product's development.
Cell & Gene Therapy enterprise development solutions to reduce scientific, regulatory and commercial risks, and accelerate development wherever you are in your product's development.
Cell & Gene Therapy Clinical Development SolutionsCovance
Cell & Gene Therapy clinical development solutions to reduce scientific, regulatory and commercial risks, and accelerate development wherever you are in your product's development.
Inhalation Technology - The Future of Effective Respiratory TreatmentsCovance
Nonclinical development channels: technical strategies, formulations and devices. Conventionally, inhaled drugs have been used to deliver medicines targeted at the most widespread respiratory diseases - specifically, chronic obstructive pulmonary disease (COPD) and asthma.
The Challenges Associated with Evaluating the Cost Benefit of Gene Therapies ...Covance
Despite the growing availability of approved gene therapies, decision-makers face significant challenges when evaluating pricing and reimbursement of these novel therapeutics. From determining cost-benefit ratios, setting out patient access criteria and designing reimbursement plans, this white paper explores some of the complex aspects of value assessment for gene therapies, and discusses results from a survey of key decision-makers across Germany, Sweden and the UK responsible for making pricing and reimbursement decisions.
Environmental Risk Assessment for Pharmaceutical DrugsCovance
Understanding the Evaluation and Implications of Findings to the Regulatory Review of Human Medicines in the Environment. Pharmaceutical drugs are intended for the treatment of human disease, therefore the risk of their environmental exposure in clinical use needs to be evaluated. Environmental risk assessment (ERA) is part of the requirements when applying for marketing approval in many geographic regions throughout the world.
Getting Investigators Onboard: Lab Preferences Make a Difference in Trial Par...Covance
Clinical trials are becoming increasingly complex and competitive, so attracting the best investigator sites to participate in a trial is a crucial step in meeting patient enrollment targets. Learn more about how investigator preference can help meet trial recruitment milestones.
Putting the Patient First: Launching a Comprehensive Patient-Centric ProgramCovance
Following a successful sponsor/CRO partnership in which Covance supported a large sponsor's reimbursement call center, a solid partnership had formed, founded on trust and a shared culture. The sponsor expanded this partnership with Covance with the desire to seamlessly transition another reimbursement support program from their current vendor. With the additional successful execution of this project, the sponsor selected Covance as their partner to consolidate all existing hub programs, build a tailored customer relationship management (CRM) tool and develop programs focused on the sponsor's specific needs and enhanced new therapeutic areas and markets.
Top 15 Pharma Gains an Edge in a Highly Competitive Specialty MarketCovance
Due to the complex nature of patient access and reimbursement process for specialty products, the client required a customized hub program to support multiple products, across several therapeutic areas. The unique intricacies of the initiative required a trusted strategic partner who could help advise, inform and support an innovative patient-centric program. In addition to possessing the right expertise and processes, the vendor needed to have a flexible technology platform that could be tailored to support the multiple brand requirements and deliver a seamless experience to various stakeholders including patients, healthcare professionals (HCPs) and specialty pharmacies.
Field Services: Providing On-Site, Field-Based Assistance to Support Customer...Covance
Ensuring access for patients can be complex and impacted by ever-changing factors that require an adaptable and responsive holistic solution to help provider and patients. Relying on a consultative approach, policy expertise and decades of experience, Covance Market Access deploys a variety of different filed-based teams that are tailored to meet your objectives and simplify access for your product.
Overcome the unique challenges of late-phase product development and generate the right data to support your products' objective. Products in late-phase development have complex needs and demands. Programs at this phase support new indications, value communication, adherence strategies, safety and efficacy and more.
Optimizing Each Patient's Product Access ExperienceCovance
Relying on a consultative approach, deep expertise and decades of experience, we work collaboratively with clients to deliver a successful product launch, program transition or program enhancement. Clients benefit from our market insights, strategic and unique approach and ability to continuously refine our processes.
Covance, in partnership with Oracle, offers a full-service, validated, private cloud, single-tenancy solution based on Argus technology, which enables faster and better safety decisions. This automated and integrated solution allows for easy scientific querying and analytics, which improves the quality and efficiency of safety operations. It also enhances compliance with E2B exchange for expedited and periodic reporting, allowing the organization to conduct global case processing, which can scale to tens of thousands of annual cases.
Plant Metabolism Studies: Options for Plant CultivationCovance
Regulators across the world are concerned with ensuring that any residues left in or on a crop after application of a plant protection product (PPP), present minimal risk to the health of humans and animals. To achieve this, regulators need information on the identity of the residues and the levels of residues remaining in or on a crop, in order to assess dietary risk and set maximum residue levels (MRLs). The testing approaches used are harmonized across most countries worldwide, focusing on the Organization for Economic Co-operation and Development (OECD) Test Guidelines (TGs) for pesticide residue chemistry. This e-book paper focuses on the laboratory-based plant cultivation methods that underlie the success of OECD crop metabolism studies, namely TG 501 and 502.
Earliest Galaxies in the JADES Origins Field: Luminosity Function and Cosmic ...Sérgio Sacani
We characterize the earliest galaxy population in the JADES Origins Field (JOF), the deepest
imaging field observed with JWST. We make use of the ancillary Hubble optical images (5 filters
spanning 0.4−0.9µm) and novel JWST images with 14 filters spanning 0.8−5µm, including 7 mediumband filters, and reaching total exposure times of up to 46 hours per filter. We combine all our data
at > 2.3µm to construct an ultradeep image, reaching as deep as ≈ 31.4 AB mag in the stack and
30.3-31.0 AB mag (5σ, r = 0.1” circular aperture) in individual filters. We measure photometric
redshifts and use robust selection criteria to identify a sample of eight galaxy candidates at redshifts
z = 11.5 − 15. These objects show compact half-light radii of R1/2 ∼ 50 − 200pc, stellar masses of
M⋆ ∼ 107−108M⊙, and star-formation rates of SFR ∼ 0.1−1 M⊙ yr−1
. Our search finds no candidates
at 15 < z < 20, placing upper limits at these redshifts. We develop a forward modeling approach to
infer the properties of the evolving luminosity function without binning in redshift or luminosity that
marginalizes over the photometric redshift uncertainty of our candidate galaxies and incorporates the
impact of non-detections. We find a z = 12 luminosity function in good agreement with prior results,
and that the luminosity function normalization and UV luminosity density decline by a factor of ∼ 2.5
from z = 12 to z = 14. We discuss the possible implications of our results in the context of theoretical
models for evolution of the dark matter halo mass function.
Richard's aventures in two entangled wonderlandsRichard Gill
Since the loophole-free Bell experiments of 2020 and the Nobel prizes in physics of 2022, critics of Bell's work have retreated to the fortress of super-determinism. Now, super-determinism is a derogatory word - it just means "determinism". Palmer, Hance and Hossenfelder argue that quantum mechanics and determinism are not incompatible, using a sophisticated mathematical construction based on a subtle thinning of allowed states and measurements in quantum mechanics, such that what is left appears to make Bell's argument fail, without altering the empirical predictions of quantum mechanics. I think however that it is a smoke screen, and the slogan "lost in math" comes to my mind. I will discuss some other recent disproofs of Bell's theorem using the language of causality based on causal graphs. Causal thinking is also central to law and justice. I will mention surprising connections to my work on serial killer nurse cases, in particular the Dutch case of Lucia de Berk and the current UK case of Lucy Letby.
Seminar of U.V. Spectroscopy by SAMIR PANDASAMIR PANDA
Spectroscopy is a branch of science dealing the study of interaction of electromagnetic radiation with matter.
Ultraviolet-visible spectroscopy refers to absorption spectroscopy or reflect spectroscopy in the UV-VIS spectral region.
Ultraviolet-visible spectroscopy is an analytical method that can measure the amount of light received by the analyte.
This pdf is about the Schizophrenia.
For more details visit on YouTube; @SELF-EXPLANATORY;
https://www.youtube.com/channel/UCAiarMZDNhe1A3Rnpr_WkzA/videos
Thanks...!
Nutraceutical market, scope and growth: Herbal drug technologyLokesh Patil
As consumer awareness of health and wellness rises, the nutraceutical market—which includes goods like functional meals, drinks, and dietary supplements that provide health advantages beyond basic nutrition—is growing significantly. As healthcare expenses rise, the population ages, and people want natural and preventative health solutions more and more, this industry is increasing quickly. Further driving market expansion are product formulation innovations and the use of cutting-edge technology for customized nutrition. With its worldwide reach, the nutraceutical industry is expected to keep growing and provide significant chances for research and investment in a number of categories, including vitamins, minerals, probiotics, and herbal supplements.
THE IMPORTANCE OF MARTIAN ATMOSPHERE SAMPLE RETURN.Sérgio Sacani
The return of a sample of near-surface atmosphere from Mars would facilitate answers to several first-order science questions surrounding the formation and evolution of the planet. One of the important aspects of terrestrial planet formation in general is the role that primary atmospheres played in influencing the chemistry and structure of the planets and their antecedents. Studies of the martian atmosphere can be used to investigate the role of a primary atmosphere in its history. Atmosphere samples would also inform our understanding of the near-surface chemistry of the planet, and ultimately the prospects for life. High-precision isotopic analyses of constituent gases are needed to address these questions, requiring that the analyses are made on returned samples rather than in situ.
Richard's entangled aventures in wonderlandRichard Gill
Since the loophole-free Bell experiments of 2020 and the Nobel prizes in physics of 2022, critics of Bell's work have retreated to the fortress of super-determinism. Now, super-determinism is a derogatory word - it just means "determinism". Palmer, Hance and Hossenfelder argue that quantum mechanics and determinism are not incompatible, using a sophisticated mathematical construction based on a subtle thinning of allowed states and measurements in quantum mechanics, such that what is left appears to make Bell's argument fail, without altering the empirical predictions of quantum mechanics. I think however that it is a smoke screen, and the slogan "lost in math" comes to my mind. I will discuss some other recent disproofs of Bell's theorem using the language of causality based on causal graphs. Causal thinking is also central to law and justice. I will mention surprising connections to my work on serial killer nurse cases, in particular the Dutch case of Lucia de Berk and the current UK case of Lucy Letby.
A brief information about the SCOP protein database used in bioinformatics.
The Structural Classification of Proteins (SCOP) database is a comprehensive and authoritative resource for the structural and evolutionary relationships of proteins. It provides a detailed and curated classification of protein structures, grouping them into families, superfamilies, and folds based on their structural and sequence similarities.
1. A Covance White Paper
Cellular & Gene Therapy Defined
Cell therapy is defined as the administration of live
whole cells or maturation of a specific cell population
in a patient for the treatment of a disease.
Gene therapy is defined as a set of strategies that
modify the expression of an individual’s genes or that
correct abnormal genes. Each strategy involves the
administration of a specific DNA (or RNA).
However…several diseases benefit most from
treatments that combine the technologies of cell and
gene therapy.
STEM CELLS:
A DYNAMIC LANDSCAPE
Cellular and gene therapies are now on the cusp of realizing the early promise of treating, and
perhaps curing diseases that previously had little or no available therapeutic options. However,
the journey from the laboratory to the clinic has been bumpy, with a history of disappointments,
especially in the early 1990’s where gene therapy had received much hype and publicity – both of
which exceeded scientific reality(1)
. With the death
of a patient in the 1990’s, gene therapy received
a major setback. These early setbacks with gene
therapy enabled a better appreciation of the
challenges of conducting clinical trials, and in 2012
the first gene therapy product was approved for use
in Europe. However, this advance was met with
further disappointment when in 2017 the therapy
was removed from the market. Nevertheless, in the
last few years there have been notable advances in
cellular and gene therapies, including marketing
approvals for stem cells, CAR-T Cells, and
gene therapies.
So, one might ask, “what has gene therapy got
to do with the development landscape for stem cells?” The answer is that the definitions between cellular and
gene therapies are somewhat blurred and advancing technology (e.g., CRISPR) will only increase the melding of
cell and gene therapies. Furthermore, the number of promising clinical studies completed or currently ongoing
indicates that many more advanced products will be forthcoming. This white paper focuses on unique clinical and
commercial aspects for developing investigational stem cell therapy products within the framework of the regulated
biotechnology and pharmaceutical industries.
Stem Cells Within the Construct of Advanced Therapies
Given the properties of stem cells to differentiate into specific cell types, and our increasing ability to genetically
modify these cells, it comes as no surprise that these cells are the focus of multiple investigational therapeutic areas.
With properties that give stem cells the potential to repair and renew damaged tissue, they have the potential for
use in multiple medical conditions. Furthermore, by placing stem cells on, or in, biological scaffold constructs, it is
often possible to generate new tissues that can be used in transplants or to augment damaged cellular
activity – entering into the realm of tissue engineering and regenerative medicine.
2. For global health authorities, development of
cellular and gene therapies has necessitated
a rapid progression of dynamic constructs to
best regulate their path to marketing approval.
In the U.S., passage of the 21st Century Cures
Act in December 2016 ushered in a number of
measures to accelerate development of advanced
therapies, including stem cell therapies. In
Japan, the Modernization Act dramatically
lowered the bar for marketing approval for
regenerative therapies, and in the EU there is a
mechanism for faster development through an
Advanced Therapy Medicinal Product (ATMP)
designation. Most other geographies have
similarly adopted pathways to accommodate the
rapidly growing fields of advanced therapies.
Manufacturing & Quality
Issues for Stem Cells
Production of stem cells falls under stringent quality control requirements. These requirements include validated
measurements of purity, potency, efficacy and stability. For stem cells these measures are not well defined.
Regulators continue to tweak manufacturing guidelines, but the industry as a whole sees these issues as major
financial burdens. Scale up from laboratory to commercial/clinical trial use is also a challenge. This is particularly so
for manufacturing differences for allogeneic cells between different geographies, but also raises serious commercial
issues for autologous stem cells.
Advancing Stem Cell Therapies to Market – Maximizing ROIC
Interestingly, health authorities on a global basis have already developed expedited development pathways for
important and lifesaving therapies. Such pathways include Fast Track and Breakthrough Therapy in the U.S.,
adaptive pathways in Europe, as well as opportunities to market the product using accelerated and conditional
approvals, followed by post-marketing commitments to attain full marketing approval. Such pathways have been
supplemented for regenerative medicine with such initiatives as the “Regenerative Medicine Advanced Therapy
(RMAT)” designation in the U.S. and the ATMP designation in Europe. All of these pathways are designed to provide
effective stem cell therapies to patients as quickly as possible – potentially they also enable generation revenue at an
earlier stage that will maximize the return on invested capital (ROIC). This can then be used to encourage further
research and development. For example, a number of stem cell products have been approved for marketing in Japan
under a conditional marketing approval with an expectation of confirmatory clinical studies during the post-approval
period. Interestingly, reimbursement for these stem cell therapies does not seem to be an issue following these
conditional marketing approvals.
Clinical Studies with Stem Cells
As with all biological therapies, a great deal of effort is applied to ensuring the purity, viability and stability of the
product. Furthermore, shipment of these products for clinical investigation usually requires refrigeration and
temperature measurements along the way to rule out excursions from the safe storage requirements. Many of these
Examples Stem Cell Sources
Stem cells are cells that can duplicate themselves and
differentiate into cell types found in the body.
Embryonic stem (ES) cells can transform into any
cell type found in the body and this gives rise to the
term, “pluripotent” cells. Human ES cells are obtained
from pre-implantation embryos, created using in vitro
fertilization.
Completely different pluripotent stem cells can be
derived from fully differentiated cells. These pluripotent
cells are called induced pluripotent stem cells (iPSC).
There are yet other cell types that only differentiate into
specific cell types, e.g. mesenchymal adult cells from
bone marrow, which differentiate into cartilage and other
connective tissues.
3. products require shipment at very cold temperatures (e.g., -80°C) so the logistics of providing clinical
trial material can be complex. In addition, stem cells product requires the overlap of GCP and GMP at
the site of administration. The impact of combining these two highly regulated activities becomes an
integral clinical protocol inclusion and requires careful training of clinical trial investigator.
Aside from ensuring the viability of the cells prior to administration, there is also an additional emphasis
on tracking the cells after administration. The ability of the cells to transform has on some occasions led to
tumor growth, although for cardiovascular studies (one of the major uses of stem cells) there appears to be
no evidence of this phenomenon. Fortunately, imaging technology and fluorescent biomarkers have enabled
substantial advances in this area. There can also be additional safety issues related to stem cell distribution
following administration (e.g., mesenchymal cells being trapped in lungs following intravenous administration).
Administration of stem cells to the desired site of action also appears to be an evolving issue in stem cell studies.
Many clinical studies have been conducted where the stem cells have been administered via a peripheral vein with the
expectation that the cells will migrate to the damaged tissue (2, 3)
. This now appears to have been an overly optimistic
approach. Indeed, a recent editorial suggested that this approach has led to many failures within the stem cell
therapeutic space. However, injection of stem cells directly into tissues (e.g., cardiac injection, or via portal vein in
damaged livers) appears to also be fraught with challenges. While detailed evidence is difficult to quantify, it seems that
many stem cells simply pour back through the needle track of cardiac tissue upon withdrawal of the needle, or in the
case of the liver, simply distribute throughout the body. The inability to place cells at the site of action with any degree
of consistency might be one reason that results have been positive in some studies and neutral in others. The advent of
bio-gels and scaffolds to keep stem cells where they are supposed to be is likely to be a useful development.
Another approach to confining stem cells within a location is by the use of bioprinting. With 3-D printing technology,
tissues are created outside of the body and then surgically implanted. Examples of 3-D printing with stem cells include
cartilage and bone, but there are many more tissues under investigation. These types of tissue-engineered products
derived from stem cells can be classified as biologics, devices, or combinations of both – a challenge for both regulators
and sponsors.
Pricing, Commercialization & Valuations
Stem cell therapy has the potential to treat the underlying cause of the disease – regrowth of neurons or repair of
damaged myocardium – which is very unlike many traditional drug products. But, what price for “cure”? To date this
has not proven to be a quantifiable paradigm since “cure” has not been achieved with stem cells. Perhaps we can take
examples from conditional approvals of stem cell products in Japan where efficacy has yet to be proven in post-approval
studies. However, even in these cases treatments are costing around $10,000 for a bag of infused cells or around $75,000
for a complete cycle of treatment. Another example would be from the recently approved CAR-T cell therapies in
oncology. While not stem cells, this form of cell therapy commands a cost of around $475,000 in the U.S.
Until we accumulate experience with more commercial stem cell products, it will be difficult to determine where
pricing will ultimately fall. However, until then, valuations for stem cell companies will continue to be approximated
based on contractual values from mergers and acquisitions(4)
. Needless to say, the market for stem cell companies with
promising products remains active, with large pharma and private equity investing billions of dollars. This trend seems
likely to continue.