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New Hope Against ALS: They Manage To Transplant Stem Cells That Could Prevent Paralysis – Nation World News

An experimental therapy developed by researchers at Cedars-Sinai Center in Los Angeles, California (USA) has shown that stem cell transplant Possible and safe for patients suffering from amyotrophic lateral sclerosis (ALS). The results are published in the peer-reviewed scientific journal nature medicine,

By combining gene and cell therapy, scientists have shown that these cells can cross the blood-brain barrier and release a protective protein from the motor neurons of the spinal cord. This could potentially prevent its worsening and paralysis of muscle functions that characterize this neurodegenerative disease.

Stem cells are a powerful method for Transport of important proteins in the brain or spinal cord which otherwise could not cross the blood-brain barrier, explains Clive Svendsen, MD, director of medicine and biomedical sciences at the Cedars-Sinai Institute for Regenerative Medicine and senior author of the current study.

We have been able to show that these laboratory-modified stem cells can be safely transplanted into the spinal cord. And, after a one-time treatment, these cells survive and Protein production for more than three years What we know protects neurons that die as ALS progresses, Svendson continues.

Read Also: Global stocks edge up after Wall St., allaying Omicron's fears. AP News

These cells were intended maintain leg function In patients, one of the first manifestations of paralysis is associated with amyotrophic lateral sclerosis. Subsequently, the disease automatically leads to the inability to walk, speak, and finally breathe. The trial involved 18 volunteers, and none of them experienced serious side effects.

The modified cells in Svendsons lab were designed to produce GDNF. a protein called, stands for glial cell line-derived neurotrophic factor. These glial cells are affected by ALS and stop protecting the neurons responsible for passing the nerve signals that enable muscle function and movement, causing them to deteriorate.

GDNF alone cannot cross the blood-brain barrier But stem cell transplantation is a method that manages to move the protein to where it should be, explains Dr. Pablo Avalos, co-author of this work. In this first therapeutic approach, the technique was to demonstrate that that it did not cause adverse in patients.

ALS often results in a loss of strength in both legs simultaneously, so researchers applied therapy. only one of them Through the half of the cord that controls it, so that the results can be compared with the other untreated leg. After transplantation, patients were followed for one year. During this period, it was found that the implant did not negatively affect the muscle strength of the operated leg.

Read Also: Republican Lawmakers Sued Over COVID Claims At Washington Capitol Building

One of the minor side effects, however, manifested itself when the transplanted cells went to higher levels of the marrow, affecting sensory area and causing instances of pain. there were also cases of benign growth, As Svendson points out, these phenomena should be studied in future trials with a deeper focus and a different surgical approach.

The next trial, he explains, will enroll more patients with earlier ALS cases, to verify the long-term effects. This time they will focus on the lower part of the spine. In addition, these cells will be tested Direct implants in the cerebral motor cortex In hopes of better preserving the neurons responsible for the use of the hand.

We are very grateful to the study participants, Svendsson says. ALS is a very difficult disease to treat, and this research a new Hope To get closer to a way to delay the progression of this disease through therapy.

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New Hope Against ALS: They Manage To Transplant Stem Cells That Could Prevent Paralysis - Nation World News

Great oaks grow from small acorns: Oncology is committed to doing its part for sustainability – EurekAlert

Late-breaking results presented at the ESMO Congress 2022 elucidate the link between air pollution and lung cancer

ESMO partners with EONS to launch the Cancer Prevention across Europe (PrEvCan) campaign

Study confirms accuracy of multi-cancer early detection blood testing, paving the way for a new era in cancer screening

Paris, France, 9 September 2022 Sustainability will be at the heart of many discussions at the ESMO Congress 2022, as illustrated by the new results and initiatives spanning cancer prevention, early detection and treatment that were announced today during the opening press conference to the annual meeting of the international oncology community taking place 9-13 September in Paris, France.

By definition, sustainability is about being able to maintain important, high-quality processes over time. In oncology, seeing the rise in cancer cases, we need to ask ourselves how we can make sure the essential process of caring for patients can be maintained, said ESMO President Prof. Solange Peters. Sustainability encompasses the notion of avoiding degradation, meaning that we also have to look at maintaining the quality which, in cancer, includes the availability of and access to anticancer drugs. It also includes quality of life, which is still dependent on the environment, and as ESMO we need to start looking at the environmental sustainability of everything we do.

Underscoring the multifaceted nature of sustainability as a societal goal, late-breaking results to be presented at the ESMO Congress 2022 offer a deeper understanding of the long-established link between air pollution and non-small cell lung cancer (NSCLC) arising in people who have never smoked, and make clear the link between climate change and human health. Pollution has a known association with lung cancer, but we didnt know if and how it directly causes the disease, said study author and ESMO 2022 Scientific Co-Chair Prof. Charles Swanton, the Francis Crick Institute and Cancer Research UK Chief Clinician, London, UK, explaining the background to this work. (1)

The research, based on human and laboratory studies, showed for a population of nearly half a million people living in England, South Korea and Taiwan that exposure to increasing concentrations of airborne particulate matter (PM) 2.5 micrometres (m) in diameter was linked to increased risk of NSCLC with mutations in the EGFR gene, which are known to be present in about half of people with lung cancer who have never smoked. In laboratory mouse models, the same pollutant particles (PM2.5) were seen to directly cause lung cancer by acting through lung tissue inflammation, driving the release of a molecule known as interleukin-1 that causes epithelial cells to transdifferentiate into cancer stem-like cells. In the presence of mutations in EGFR and in another gene linked to lung cancer called KRAS, these cells can then bloom into a tumour.

These mutations can be found in over half of normal lung tissue biopsies and are a natural process of ageing. They are necessary, but not sufficient to drive cancer: it is in combination with pollution that the cancer stem cells can expand and initiate a tumour. This begins to explain how environmental carcinogens that dont induce DNA mutations can drive cancer, said Swanton, deriving from this discovery a public health mandate to lower the levels of these pollutants, which are produced by the combustion of fossil fuels. We have to achieve a 50% reduction in greenhouse gas emissions by 2030, and by doing so we will naturally reduce levels of PM2.5. We can all play a part here: we need to cycle more, walk more. Its worth bearing in mind that PM2.5 cause 8 million deaths a year, not just due to cancer but also to other diseases like cardiovascular disease, strokes, dementia that is more than the deaths caused by tobacco globally.

In light of the fact that his research confirmed the blockade of interleukin-1 could inhibit lung cancer initiation by blocking the pollution-induced transformation of airway cells into cancer stem cells, Swanton also suggested that targeting interleukin-1 should be further explored in the future as a potential new approach to cancer prevention.

The findings come in a context where the global incidence of respiratory cancers is on the rise, with annual new cases expected to jump by about 70% over the next two decades. In Europe alone, similar trends observed for other malignancies could result in an increase in overall cancer mortality, from 2 million annual deaths in 2020, to as many as 3 million by 2040. (2) As up to half of all cancers are thought to be preventable, prevention is considered by the World Health Organisation to be the most cost-effective, and thus the most sustainable, long-term strategy for cancer control.

The ESMO Vision 2025, made very clear that if we want to succeed in tackling cancer, we need to develop a clear plan for primary and secondary prevention, continue to offer the optimal care for cancers that cannot be prevented and adequately support cancer survivorship. Focusing on only one of these areas and neglecting the others would lead to failure, said ESMO Director of Public Policy Dr. Rosa Giuliani.

In line with the Societys commitment to promoting research-based cancer prevention, the ESMO representatives joined European Oncology Nursing Society (EONS) Executive Board member Dr. Lena Sharp, Regional Cancer Centre, Stockholm, Sweden, in announcing the launch today of the Cancer Prevention across Europe Campaign (PrEvCan).

Led by EONS with ESMO as key partner alongside other international organisations, the campaign will over a one-year period dedicate each month to promoting and explaining the scientific evidence for each one of the 12 recommendations of the European Code Against Cancer (ECAC) to prevent the disease, starting in October with smoking as one of the most important cancer risk factors.

What is new here is that it is the cancer care workforce leading the way, said Sharp, the PrEvCan project leader. We are the ones who meet patients and their families, so we could intervene on a daily basis supporting and advising people to adopt healthier lifestyles to reduce the risk of new cancers but also to reduce negative effects on the current disease.

The campaign will target the general public, including the most vulnerable groups who can be difficult to reach with health promotion and lifestyle advice, but equally healthcare professionals, who according to Sharp can also take a more prominent role in supporting vaccination and screening programmes.

The ESMO President added: We thought for a while that prevention should be in the hands of family doctors, but then we started to learn that preventing the disease must be at least partly in the hands of the specialists of a specific disease, in order to convince people about its importance. Particularly after COVID, a certain degree of suspicion can happen in medicine. You need to make sure that everything you propose has a basis and one of these bases for cancer prevention is the burden of cancer, what it represents not only in terms of lost years of life but also in terms of the sustainability of our societies and healthcare systems. Peters further highlighted that oncologists should view prevention as an integral part of oncology care, also because the science of prevention still requires more data.

For cancers that are not currently avoidable, screening and early detection has the potential to both maximise individuals chances of survival, and alleviate the burden on health systems by reducing the proportion of patients with advanced disease who require costly, chronic therapies and care.

A study to be presented at this years Congress could lead to a major paradigm shift in this field, having confirmed the feasibility of multi-cancer early detection (MCED) blood testing as a method of screening for up to 50 different cancer types simultaneously. (3)

This is one of the very first studies where the detection of cancer DNA in the blood has allowed us to detect cancer at an early stage, said ESMO 2022 Scientific Co-Chair Prof. Fabrice Andr, Institute Gustave Roussy, Villejuif, France. If this test works, in the future, it will be good news for patients, but most cancer centres are not equipped to scale up surgeries for hundreds more patients with, say, early-stage pancreatic cancer. With this landmark study comes a need for a wake-up call for hospitals to see what will happen in 10 years and start now to train fellows and change their infrastructures accordingly.

Highlighting the time and patience required to turn promising research results into meaningful innovation for patients, Swanton observed: ESMO 2022 is a celebration of the collaboration between basic scientists and healthcare professionals to advance care for our patients. Some of the breakthroughs that will be discussed over the next four days have come from biological studies of worms, yeast, bacteria and plants but they took 30 or 40 years of painstaking science from the bench to the bedside. We need our funders to recognise that and to sustain investment in discovery research to generate the medicines of the future.

Among other highly anticipated results to be presented at the ESMO Congress 2022 , Andr drew attention to several examples of novel approaches which could soon become a reality in the clinic: from the phase III trial of gamma secretase inhibitor (GSI) nirogacestat, a first-in-class drug targeting a new molecular alteration in a rare category of cancers known as desmoid tumours, through a landmark trial of cell therapy using tumour-infiltrating lymphocytes (TILs) to improve the outcomes of patients with advanced melanoma, all the way to several late-phase trials of immunotherapy, including for non-small cell lunger cancer patients not eligible to standard platinum chemotherapy. Andr welcomed the presence of studies for underrepresented patient populations in the Congresss scientific programme, concluding: We cannot exclude patients from clinical trials.

In closing, ESMO 2022 Press Officer Dr. Antonio Passaro called for a wide and wise dissemination of the data to be presented: We have here a community of about 25,000 people, with more than 1,900 abstracts and 76 LBAs that will be presented in the coming days. We need to pass these messages to all of our colleagues and the public in order to dramatically improve the future of our patients, which risks being worse than it is today considering current cancer incidence trends.

-END-

Notes to Editors

Please make sure to use the official name of the meeting in your reports: ESMO Congress 2022

Official Congress Hashtag: #ESMO22

Follow the conversation on Twitter, LinkedIn, Instagram, Facebook and watch video material on YouTube

References

1 LBA1 Mechanism of action and an actionable inflammatory axis for air pollution induced non-small cell lung cancer in never smokers will be

presented by Charles Swanton during Presidential Symposium 1 on Saturday, 10 September, 16:30 to 18:00 CEST in Paris Auditorium. Annals of

Oncology, Volume 33 Supplement 7, September 2022

2 Source: Globocan

3 Abstract 903O A prospective study of a multi-cancer early detection blood test will be presented by Deb Schrag during the proffered paper

session Basic science and translational research on Sunday, 11 September, 16:30 to 18:00 CEST in Orlans Auditorium. Annals of Oncology,

Volume 33 Supplement 7, September 2022

4 The ESMO ANMS 2.0 survey about access to cancer medicines will be discussed during the educational session The Universal Health Coverage (UHC) dilemma: Can we afford to pay for what we want? on Saturday, 10 September, 8:30 to 10:00 CEST in Marseille Auditorium.

About the European Society for Medical Oncology (ESMO)

ESMO is the leading professional organisation for medical oncology. With 25,000 members representing oncology professionals from over 160 countries worldwide, ESMO is the society of reference for oncology education and information.Driven by a shared determination to secure the best possible outcomes for patients, ESMO is committed tostanding by those who care about cancerthroughaddressing the diverse needs of#ONEoncologycommunity,offering#educationforLIFE, andadvocating for#accessiblecancerCARE.www.esmo.org

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Great oaks grow from small acorns: Oncology is committed to doing its part for sustainability - EurekAlert

What happens to the brain on prescription steroids? – Medical News Today

Glucocorticoids also known as corticosteroids or just steroids are a class of medications prescribed for a variety of different diseases and conditions. These are different from anabolic steroids that may be used to increase muscle mass.

Concerningly, prescription steroids can sometimes come with harsh side effects, including neurological issues, such as mood disorders and cognitive issues.

Now a team of scientists from Leiden University Medical Center in The Netherlands has found evidence suggesting the use of prescribed steroids causes structural and volume changes in the white and gray matter of the brain.

This study recently appeared in the journal BMJ Open.

Doctors mainly prescribe corticosteroids to help lower inflammation in the body, suppress the bodys immune system, or balance hormone levels

They normally prescribe them in tablet or inhaler form, although sometimes people require prescribed steroid injections. There are also topical corticosteroids in the form of lotions or creams.

A doctor might prescribe steroids for the following conditions:

Using glucocorticoids for an extended time increases a persons risk of developing certain side effects, such as:

According to doctoral researcher Merel van der Meulen, from the Department of Medicine in the Division of Endocrinology at Leiden University Medical Center and lead author of this study, previous research of people with Cushings disease, who have very high levels of the bodys own glucocorticoid cortisol, shows that long-term exposure to glucocorticoids can affect both the function and the structure of the brain.

Correcting cortisol levels can at least partially reverse these changes. But what about people whose steroid levels increase due to other medical needs?

A few small studies in selected populations also showed that long-term systemic glucocorticoid medication use is associated with some differences in the brain, van der Meulen told Medical News Today.

We wondered whether these effects of glucocorticoids on brain structure could also be observed in the large population-based cohort of the UK Biobank, including inhaled glucocorticoid users, she added.

The research team examined data, including questionnaires and MRI scans, from 222 systemic glucocorticoid users meaning they took the prescribed medication orally or through an injection and 557 inhaled glucocorticoid users from the UK Biobank population recruited between 2006 and 2010.

None of the participants had a history of neurological, psychiatric, or hormonal issues. Researchers compared the data from glucocorticoid users to that of 24,106 people who did not use steroids.

The researchers found that participants using either systemic or inhaled prescribed steroids had less intact white matter structure in the brain compared to non-steroid users. However, this observation increased in systemic steroid users compared to inhaled steroid users.

White matter occurs deep in the brain and is made up of bundles of nerve cells. It plays a role in neuronal connections and signaling in the brain.

The scientists moreover found that participants taking systemic steroids had a larger caudate a part of the gray matter of the brain involved in high-level activities like planning the execution of movements, learning, and memory compared to non-users.

And participants using inhaled glucocorticoids had a smaller amygdala compared to those not taking prescribed steroids. The amygdala is also part of the brains gray matter and is linked to the processing and regulation of emotions.

MNT spoke with Dr. Santosh Kesari, a neurologist at Providence Saint Johns Health Center in Santa Monica, CA, and Regional Medical Director for the Research Clinical Institute of Providence Southern California about this study.

I was excited to know someone did this study that really validates what weve known for a long time that steroids cause brain atrophy and a lot of neuropsychiatric symptoms or side effects, he stated.

This study showed that steroids do have an effect on the structure of the brain, Dr. Kesari continued. You do lose white matter, which [makes up] the connections from one neuron to another. Theres also some loss of the gray matter, the actual neurons, that needs to be studied [further].

Dr. Kesari explained that white matter is the conduit for information from one neuron to another:

When you lose white matter, everything slows down, meaning slower response, some memory issues potentially, or cognitive issues. And then there [are] also psychiatric issues, so they [people who take prescription steroids] can get agitated, depressed, mood disorders, things like that.

Adding to the white matter discussion, van der Meulen said that previous research shows that glucocorticoids can have psychiatric side effects, such as depression and anxiety.

In our observational study, we report associations between glucocorticoids and a lower white matter microstructure in the brain, she continued. It is possible that these associations may be related to the psychiatric side effects of glucocorticoids, but more research is needed to confirm this.

MNT also spoke with Dr. Ilan Danan, a sports neurologist and pain management specialist at the Center for Sports Neurology and Pain Medicine at Cedars-Sinai Kerlan-Jobe Institute in Los Angeles, CA.

He cautioned that it is important to note there is a difference between the prescribed steroids discussed in this study compared to those taken by athletes.

As opposed to the steroids that may be prescribed by physicians, the ones that athletes will look into are going to be more for performance enhancement, he explained. Those are anabolic, androgenic-type steroids that dont necessarily apply in this context.

As for the next steps in this research, van der Meulen said that many questions remain unanswered that she hopes to address in the future.

For example, are these effects reversible? she wondered. How do they depend on the dose and duration of glucocorticoid use and the type of glucocorticoid medication used? And could selective glucocorticoid receptor modulators a type of glucocorticoid-like medication that has a more selective effect and therefore potentially [fewer] side effects prevent these effects from happening?

Dr. Danan stated that he would like to see more details regarding how long participants used prescribed steroids and whether the systemic glucocorticoid users took the medication orally or through an injection.

Those are things that as a physician [I] would want to know so that I can tie in whether or not this has a potential impact on my patient base, he added.

And Dr. Kesari said that although this study documents atrophy of the brain, more research is required to understand how that happens.

We need to do more basic science research to understand the mechanisms of how steroids are causing this brain damage, and then how we can mitigate it with other medications or reparative mechanisms in the future, whether its stem cells or growth factors that may stimulate stem cells, he said.

Read more here:
What happens to the brain on prescription steroids? - Medical News Today

Single mutation helped separate human, Neanderthal brains – Big Think

How the modern human brain evolved, and how it differs from the brains of Neanderthals and other extinct hominin species, is an open question. Expansion of the neocortex was a key event in human brain evolution, and now researchers in Germany say they have identified a genetic mutation that drove this process.

Anneline Pinson of the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden and her colleagues examined a gene called TKTL1, which is active in immature neurons in the fetal neocortex, and is also implicated in the proliferation of brain tumor cells.

TKTL1 encodes an enzyme consisting of 596 amino acid residues and is one of just a few genes whose DNA sequence differs between humans and extinct archaic hominins. In the Neanderthal genome, residue 317 is the amino acid lysine, but in humans, this has been substituted with arginine. Such seemingly tiny differences can matter greatly.

Pinson and her colleagues analyzed previously published human fetal transcriptome datasets, revealing TKTL1 is expressed in a specific population of neural stem cells in the developing nervous system from nine weeks of gestation onward, and that its levels then increase in the immature frontal lobe, but not other areas. The human fetal TKTL1 protein is a shorter form, however, containing only 540 residues, with the aforementioned substitution at position 261.

TKTL1 is not expressed in the embryonic mouse cortex, but when the researchers inserted the human gene into mouse embryos, it increased the number of stem cells that give rise to frontal cortex neurons, resulting in more newborn neurons at later stages of development. Insertion of the Neanderthal TKTL1 gene had no such effect.

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The researchers also inserted human TKTL1 into ferret embryos, which normally express the Neanderthal-like, lysine-containing variant of the gene. This, too, increased the number of neural progenitors and newborn neurons, leading to an expansion of the upper layers of the neocortex.

Conversely, deleting TKTL1 from human fetal brain tissue reduced the number of neural stem cells, and inserting the Neanderthal variant into lab-grown cerebral organoids reduced the number of stem cells and neurons derived from them.

Finally, Pinson and her colleagues determined the function of the TKTL1 enzyme. Their experiments showed that it promotes the synthesis of fatty acids that are inserted into the neural stem cell membrane, which are crucial for the outgrowth of their fibers and their proliferation.

These findings show that a mutation in the DNA sequence of the TKTL1 gene, causing a single amino acid substitution in the human protein sequence, is responsible for the observed effects on neural stem cell behavior. The researchers conclude that this simple genomic change may contribute to the differences in size and shape of the human and Neanderthal neocortex.

Brain development is an extremely complex process, however, and it is highly unlikely that a single genetic event was responsible for evolution of the human brain. Indeed, the process of human brain evolution likely involved many hundreds of genes and multiple different types of genetic events, including changes in non-coding DNA sequences, gene deletions and duplications, jumping genes, and other large-scale genomic changes.

Continued here:
Single mutation helped separate human, Neanderthal brains - Big Think

Regenerative Medicine Global Market to Surpass $40.7 Billion by 2030 at a CAGR of 12.75% – PR Newswire

DUBLIN, Sept. 7, 2022 /PRNewswire/ --The "Regenerative Medicine Global Market Opportunities And Strategies To 2031" report has been added to ResearchAndMarkets.com's offering.

This report describes and explains the global regenerative medicine market and covers 2016 to 2021, termed the historic period, and 2021 to 2026 termed the forecast period, along with further forecasts for the period 2026-2031. The report evaluates the market across each region and for the major economies within each region.

The global regenerative medicine market reached a value of nearly $7,282.2 million in 2020, having increased at a compound annual growth rate (CAGR) of 54.1% since 2015. The market is expected to grow from $7,282.2 million in 2020 to $22,373.7 million in 2025 at a rate of 25.2%. The market is then expected to grow at a CAGR of 12.7% from 2025 and reach $40,710.1 million in 2030.

Growth in the historic period in the regenerative medicine market resulted from rising prevalence of chronic diseases, emerging markets growth, implementation of the 21st century cures act, rapid growth in aging population, and the improvement in healthcare awareness and expenditure. The market was restrained by high cost of cell and gene therapies, ethical concerns related to the use of embryonic stem cells in research and development, and inadequate reimbursements.

Going forward, rising demand for organ transplantations, growth in healthcare expenditure, technological advancements in regenerative medicines, rising investments in regenerative medicine research, and changes in lifestyles. Factors that could hinder the growth of the market in the future include rising popularity of alternative therapies and natural remedies, low per capita healthcare expenditure, and tissue-engineered products and biomaterials are lagging in adoption.

The regenerative medicine market is also segmented by end-use into ambulatory surgical centers, hospitals and clinics, and others. The hospitals and clinics segment was the largest segment of the regenerative medicine market segmented by end-use, accounting for 63.8% of the total in 202o. Going forward, hospitals and clinics segment is expected to be the fastest growing segment in the regenerative medicine market segmented by end-use, at a CAGR of 25.3% during 2020-2025.

The regenerative medicine market is also segmented by application into musculoskeletal, oncology, dental, wound care and others. The oncology segment was the largest segment of the regenerative medicine market segmented by application, accounting for 60.0% of the total in 2020. Going forward, musculoskeletal segment is expected to be the fastest growing segment in the regenerative medicine market segmented by application, at a CAGR of 27.4% during 2020-2025.

North America was the largest region in the regenerative medicine market, accounting for 53.3% of the total in 2020. It was followed by the Western Europe, Asia Pacific, and then the other regions. Going forward, the fastest-growing regions in the regenerative medicine market will be Middle East and South America where growth will be at CAGRs of 72.4% and 71.9% respectively during 2020-2025. These will be followed by Eastern Europe and Asia Pacific, where the markets are expected to register CAGRs of 57.8% and 49.7% respectively during 2020-2025.

Market Trends And Strategies

Markets Covered:

Key Topics Covered:

1. Regenerative Medicine Market Executive Summary

2. Table of Contents

3. List of Figures

4. List of Tables

5. Report Structure

6. Introduction

7. Regenerative Medicine Market Characteristics

8. Regenerative Medicine Market Trends And Strategies

9. Impact Of COVID-19 On Regenerative Medicine

10. Global Regenerative Medicine Market Size And Growth

11. Global Regenerative Medicine Market Segmentation

12. Regenerative Medicine Market, Regional And Country Analysis

13. Asia-Pacific Regenerative Medicine Market

14. Western Europe Regenerative Medicine Market

15. Eastern Europe Regenerative Medicine Market

16. North America Regenerative Medicine Market

17. South America Regenerative Medicine Market

18. Middle East Regenerative Medicine Market

19. Africa Regenerative Medicine Market

20. Regenerative Medicine Global Market Competitive Landscape

21. Key Mergers And Acquisitions In The Regenerative Medicine Market

22. Regenerative Medicine Market Opportunities And Strategies

23. Regenerative Medicine Market, Conclusions And Recommendations

24. Appendix

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/bnpkzj

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

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SOURCE Research and Markets

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Regenerative Medicine Global Market to Surpass $40.7 Billion by 2030 at a CAGR of 12.75% - PR Newswire

Global Induced Pluripotent Stem Cells Market (2022 to 2027) – Growth, Trends, Covid-19 Impact and Forecasts – ResearchAndMarkets.com – Business Wire

DUBLIN--(BUSINESS WIRE)--The "Induced Pluripotent Stem Cells Market - Growth, Trends, Covid-19 Impact, and Forecasts (2022 - 2027)" report has been added to ResearchAndMarkets.com's offering.

The Induced Pluripotent Stem Cells Market is projected to register a CAGR of 8.4% during the forecast period (2022 to 2027).

Companies Mentioned

Key Market Trends

The Drug Development Segment is Expected to Hold a Major Market Share in the Induced Pluripotent Stem Cells Market.

By application, the drug development segment holds the major segment in the induced pluripotent stem cell market. Various research studies focusing on drug development studies with induced pluripotent stem cells have been on the rise in recent years.

For instance, an article titled "Drug Development and the Use of Induced Pluripotent Stem Cell-Derived Cardiomyocytes for Disease Modeling and Drug Toxicity Screening" published in the International Journal of Molecular Science in October 2020 discussed the broad use of iPSC derived cardiomyocytes for drug development in terms of adverse drug reactions, mechanisms of cardiotoxicity, and the need for efficient drug screening protocols.

Another article published in the Journal of Cells in December 2021 titled "Human Induced Pluripotent Stem Cell as a Disease Modeling and Drug Development Platform-A Cardiac Perspective" focused on methods to reprogram somatic cells into human induced pluripotent stem cells and the solutions to overcome the immaturity of the human induced pluripotent stem cells derived cardiomyocytes to mimic the structure and physiological properties of adult human cardiomyocytes to accurately model disease and test drug safety. Thus, this increase in the research of induced pluripotent stem cells for drug development and drug modeling is likely to propel the segment's growth over the study period.

Furthermore, as per an article titled "Advancements in Disease Modeling and Drug Discovery Using iPSC-Derived Hepatocyte-like Cells" published in the Multi-Disciplinary Publishing Institute journal of Cells in March 2022, preserved differentiation and physiological function, amenability to genetic manipulation via tools such as CRISPR/Cas9, and availability for high-throughput screening, make induced pluripotent stem cell systems increasingly attractive for both mechanistic studies of disease and the identification of novel therapeutics.

North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period

The rise in the adoption of highly advanced technologies and systems in drug development, toxicity testing, and disease modeling coupled with the growing acceptance of stem cell therapies in the region are some of the major factors driving the market growth in North America.

The United States Food and Drug Administration in March 2022 discussed the development of strategies to improve cell therapy product characterization. The agency focused on the development of improved methods for testing stem cell products to ensure the safety and efficacy of such treatments when used as therapies.

Likewise, in March 2020, the Food and Drug Administration announced that ImStem drug IMS001, which uses AgeX's pluripotent stem cell technology, would be available for the treatment of multiple sclerosis. Similarly, REPROCELL introduced a customized iPSC generation service in December 2020, as well as a new B2C website to promote the "Personal iPS" service. This service prepares and stores an individual's iPSCs for future injury or disease regeneration treatment.

Thus, the increasing necessity for induced pluripotent stem cells coupled with increasing investment in the health care department is known to propel the growth of the market in this region.

Key Topics Covered:

1 INTRODUCTION

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS

4.1 Market Overview

4.2 Market Drivers

4.2.1 Increase in Research and Development Activities in Stem Cells Therapies

4.2.2 Surge in Adoption of Personalized Medicine

4.3 Market Restraints

4.3.1 Lack of Awareness Regarding Stem Cell Therapies

4.3.2 High Cost of Treatment

4.4 Porter's Five Force Analysis

5 MARKET SEGMENTATION

5.1 By Derived Cell Type

5.2 Application

5.3 End User

5.4 Geography

6 COMPETITIVE LANDSCAPE

6.1 Company Profiles

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

For more information about this report visit https://www.researchandmarkets.com/r/ylzwhr

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Global Induced Pluripotent Stem Cells Market (2022 to 2027) - Growth, Trends, Covid-19 Impact and Forecasts - ResearchAndMarkets.com - Business Wire

Nerve Repair and Regeneration Market Size Worth $12,514 – GlobeNewswire

New York, Sept. 02, 2022 (GLOBE NEWSWIRE) -- The Insight Partners published latest research study on Nerve Repair & Regeneration Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Product {Neurostimulation & Neuromodulation Devices [Internal Neurostimulation Devices (Spinal Cord Stimulation, Deep Brain Stimulation, Vagus Nerve Stimulation, Sacral Nerve Stimulation, and Gastric Electrical Stimulation) and External Neurostimulation Devices (Transcutaneous Electrical Nerve Stimulation Devices, Transcranial Magnetic Stimulation Devices, Respiratory Electrical Stimulation Devices)] and Biomaterials [Nerve conduits, Nerve wraps, Nerve Connectors, and Other Biomaterials]} and Application {Neurostimulation & Neuromodulation Applications [Internal neurostimulation & neuromodulation and External neurostimulation & neuromodulation], and Nerve Repair & Regeneration Applications [Direct Nerve Repair/Neurorrhaphy (Epineural Repair, Perineural Repair, and Group Fascicular Repair), Nerve Grafting (Autografts, Allografts, and Xenografts), and Stem cell therapy]}, the global nerve repair & regeneration market size is projected to reach USD 12,514.06 million by 2028 from USD 6,607.71 million in 2021; it is expected to grow at a CAGR of 9.7% from 2022 to 2028.

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Nerve Repair and Regeneration Market Report Scope & Strategic Insights:

Global Nerve Repair & Regeneration Market: Competitive Landscape and Key Developments A few companies in the global nerve repair & regeneration market are Axogen Corporation, Boston Scientific Corporation, Integra LifeSciences, Medtronic, Abbott Laboratories, Stryker Corporation, Neuronetics, LivaNova PLC, Baxter, and Polyganics BV. The companies operating in the global nerve repair & regeneration market implement various growth strategies, such as product launches, expansions, and product upgrades. Similarly, several companies are implementing inorganic strategies such as acquisitions, collaboration, and partnerships.

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In Aug 2021, Checkpoint Surgical, Inc. launched the NeuroShield Chitosan Nerve Wrap to expand its intraoperative nerve care product line. The NeuroShield Wrap marks the company's first product in a planned line of chitosan-based biological implants to support nerve healing. Adding the product complements the existing product portfolio of Checkpoint Surgical, including the companys flagship Checkpoint Nerve Stimulator/Locator, the leading nerve stimulation device for intraoperative nerve protection and repair.

In Mar 2021, Abbott launched NeuroSphere Virtual Clinic in the US, a first-of-its-kind technology enabling patients to interact with physicians, ensure proper functionality and settings, and receive new treatment settings remotely as per requirement. The NeuroSphere Virtual Clinic can extend access to optimal treatment for patients with movement disorders or chronic pain who don't live near a care provider, who can not access care easily or are unable to visit a physician due to circumstances such as COVID-19.

The nerve repair and regeneration market growth is attributed to the growing incidence of nerve injuries and the high prevalence of neurological disorders, the growing geriatric population and rising prevalence of age-associated neurological disorders, and increasing funding by private and government organizations for research in neurology. However, the absence of trained professionals is hampering the growth of the global nerve repair & regeneration market.

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Global Nerve Repair & Regeneration Market: Key Insights Future Trend Nerve repair and regeneration offers products such as neuromodulators & neurostimulation devices, and biomaterials. These devices treat neurodegenerative or traumatic disorders such as Parkinson's disease, Alzheimer's disease, sclerosis, multiple system atrophy, and amyotrophic lateral sclerosis. Therefore, market players are launching various products such as internal neurostimulation devices, external neurostimulation devices, nerve conduits, nerve wraps, nerve connectors, and other biomaterials to enhance the treatment output of nerve repair and regeneration.

A few of the recent developments related to the nerve repair and regeneration market are mentioned below:

In July 2022, Checkpoint Surgical launched the Checkpoint Edge Nerve Cutting Kit, expanding the strength of the intraoperative nerve care portfolio of the company. The Checkpoint Edge Nerve Cutting Kit is the first offering in the planned portfolio of nerve-specific instruments by Checkpoint Surgical, with further expansion planned in the future.

In April 2022, Boston Scientific Corporation received US Food and Drug Administration (FDA) approval for its latest image-guided programming software, Vercise Neural Navigator with STIMVIEW XT. The product is developed in collaboration with Brainlab AG. STIMVIEW XT allows physicians, in real-time, the capacity to visualize both stimulation modeling and lead placement of the brain anatomy of their patients having an essential tremor or Parkinsons disease.

In Mar 2022, Integra LifeSciences Holding Corporation launched the NeuraGen 3D Nerve Guide Matrix, which is a resorbable implant to repair peripheral nerve discontinuities. NeuraGen 3D is a nerve repair product specifically engineered to develop an optimized environment that enables a greater complete functional recovery following mid-gap nerve repair in comparison to hollow nerve conduits alone.

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In Jan 2022, Medtronic plc received US Food and Drug Administration approval for its Intellis rechargeable neurostimulator and Vanta recharge-free neurostimulator treating chronic pain associated with diabetic peripheral neuropathy (DPN).

Thus, the active participation of market players in product innovation and development and increase in approvals of products are likely to fuel the market growth in the coming years.

Global Nerve Repair & Regeneration Market: Product Overview Based on product, the nerve repair & regeneration market is bifurcated into neurostimulation & neuromodulation devices and biomaterials. Neurostimulation & neuromodulation devices segment is further bifurcated into internal neurostimulation devices and external neurostimulation devices. Internal neurostimulation devices segment is further subsegmented into spinal cord stimulation, deep brain stimulation, vagus nerve stimulation, sacral nerve stimulation, and gastric electrical stimulation. External neurostimulation devices is further subsegmented into transcutaneous electrical nerve stimulation devices, transcranial magnetic stimulation devices, respiratory electrical stimulation devices. The neurostimulation and neuromodulation devices segment held the largest share of the market in 2021 and is anticipated to register the highest CAGR in the market during the forecast period. The growing prevalence of neurological disorders, increasing application of neurostimulation and neuromodulation devices, and rapid advancements are driving the market growth of the nerve repair & regeneration market.

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Browse Adjoining Reports: Nerve Repair Biomaterial Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Product (Nerve Protection, Nerve Repair); Application (Epineural Nerve Repair, Perineural Nerve Repair, Group Fascicular Repair); End User (Hospitals and Clinics, Ambulatory Surgery Centers, Other) and Geography

Nerve Repair and Re-generation Biomaterials Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type (Nerve Conduits, Nerve Wraps, Nerve Graft, Others); Application (Neurorrhaphy, Nerve Grafting, Stem Cell Therapy, Others) and Geography

Neurostimulation Devices Market Forecast to 2028 - Covid-19 Impact and Global Analysis - By Product (Deep Brain Stimulation Device, Spinal Cord Stimulation Device, Sacral Nerve Stimulation Device, Vagus Nerve Stimulation Device, and Gastric Electric Stimulation Device), Application (Pain Management, Epilepsy, Depression, Parkinson's Disease, Essential Tremor, Urinary and Fecal Incontinence, and Other Applications), and Geography

Neuromodulation Market Forecast to 2027 - COVID-19 Impact and Global Analysis by Technology (External (Non-Invasive) Neuromodulation, and Internal Neuromodulation); Application (Chronic Pain Management, Failed Back Syndrome, Epilepsy, Tremor, Incontinence, Depression, Dystonia, Gastroparesis, Parkinson

Neuromodulation Devices Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type (Internal, External); Application (SCS, DBS, VNS, SNS, GES, TENS, TMS); Biomaterial (Metallic, Polymeric, Ceramic); End-User (Hospitals, Clinics, Homecare) and Geography

Biomaterials Market Forecast to 2028 - Covid-19 Impact and Global Analysis - by Type (Metallic, Ceramic, Polymers, Natural); Application (Cardiovascular, Orthopedic, Dental, Plastic Surgery, Wound Healing, Neurological disorders, Tissue Engineering, Ophthalmology)

Biomaterials Devices Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type ( Natural Biomaterials, Polymers Biomaterials, Ceramic Biomaterials, Metallic Biomaterials ); Application ( Orthopedic, Cardiovascular, Dental, Ophthalmology, Tissue Engineering, Other ) and Geography

Deep Brain Stimulation Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Product (Single Channel Deep Brain Stimulator, Dual Channel Deep Brain Stimulator.); Application (Parkinson's Disease, Tremor, Depression, Alzheimer's Disease, Dystonia and Tourette Syndrome); End User (Hospitals, Research Centres) and Geography

Transcranial Magnetic Stimulator (TMS) Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type (Single or Paired Pulse TMS, Repetitive TMS (rTMS)); Age Group (Adults, Children); Application (Research, Diagnostic, Therapeutic), and Geography

Implantable Neurostimulation Devices Market Forecast to 2028 - COVID-19 Impact and Global Analysis by Technology ( spinal cord stimulation (SCS), deep brain stimulation (DBS), sacral nerve stimulation (SNS), gastric electric stimulation (GES), vagus nerve stimulation (VNS), others ); Application ( pain management, epilepsy, Parkinson's, urinary and fecal incontinence, others )

Low-Frequency Sine Wave Neurostimulation Therapy Devices Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Product (Percutaneous treatment, Implantation); Application (Urinary impulse, Interstitial cystitis, Idiopathic constipation) and Geography

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Nerve Repair and Regeneration Market Size Worth $12,514 - GlobeNewswire

Regenerative Medicine: An alternative to surgery and pills | Loop Cayman Islands – Loop News Cayman

Surgical methods of repair have long since been touted as the best approach to treatment and healing for patients with long-term, degenerative conditions.

While effective, factors such as high costs, long downtime, surgical complications and more can be a deterrent for patients who are considering surgery for non-life-threatening conditions.

Regenerative Medicine is an approach available for some patients who are seeking pain relief and long-term healing and would like to reduce the need for surgery.

Regenerative medicine can be defined as the process of replacing or regenerating human cells or tissues to facilitate healing and repair. The ability to encourage this repair in the human body reduces the time most patients will remain ill and offers many possibilities in the field of medicine.

In understanding the regenerative process, it is important to note that the potential for repair and the ability to repair are two separate things. We all have the potential to repair but we dont all have the ability. Increasing the ability to repair is considered one of the main benefits of regenerative medicine. This is derived from the ability of cells, particularly stem cells, to promote cell repair by reducing inflammation which promotes faster healing, better circulation and less pain in an injured area.

Stem cells can be taken from a variety of tissues in the body, and they tend to be harvested from areas that are easily accessible. With stem cell therapy, you are able to use your bodys own repair team (cells) to heal an injured area.

In fact, stem cell therapy has been proven to regenerate tissue in the human body without the need for surgery or additional medication in certain conditions. It is useful for patients who are suffering from degenerative and chronic diseases, such as chronic pain, who are in search of medical treatments without surgical interference.

Image via iStock

Regenerative Medicine and Stem Cell Therapy are useful treatment options for:

The typical patient is over 40 years perhaps starting to experience some joint pains, sexual dysfunction or lack of energy aging with an inability to keep up with their demanding lifestyles.

One patient, DG, came in for a list of concerns that are typical in a 60-year-old including knee pain, mild erectile dysfunction and high blood pressure.

One patient reported improved eyesight among other benefits after his treatment. Image via iStock

DG underwent stem cell therapy and reports to us on his experience. He reported an overwhelming relief from pain and the anxiety associated with expecting this pain. His knee, which was injured during an uphill walk, has had improvement and he is able to enjoy his daily walks without expectation of pain. He also marveled at the maintenance of life and health aspect of Stem Cell Therapy which enabled him to bank his extracted Stem Cells for future usage.

Lastly, DG mentioned the unexpected benefits that have come from the treatment such as improved eyesight without glasses, a reduction of graying hairs, a reduction in bags under the eye, a younger appearance and a reduction of Erectile Dysfunction!

BioRegeneration Integrated Medical Centre in Jamaica specialises in stem cell therapy using your own stem cells. This treatment is done locally by Dr Janice Simmonds-Fisher who has pioneered Regenerative Medicine in Jamaica for over a decade providing a life-improving treatment that enjoys over 90 per centsuccess rate in our population.

BioRegeneration is located at 15 Haining Road, Kingston. They can be contacted at 876-552-6484. For any questions, email questions@drjanicefisher.com or visit http://www.stemcelljamaica.com.

For our regional patients, Dr. Fisher offers phone or virtual consultations. To make a virtual consultation appointment, please email questions@drjanicefisher.com or whatsapp 876-565-1168.

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Regenerative Medicine: An alternative to surgery and pills | Loop Cayman Islands - Loop News Cayman

Building Europes gene therapy ecosystem POLITICO

Gene therapy is a transformative field of medicine that could halt diseases before patients become symptomatic, saving lives and improving quality of life for millions of people who suffer from conditions caused by genetic abnormalities1. But these breakthrough treatments require new approaches to pricing and reimbursement2.

While the science behind gene therapy has existed for decades, the first generation of treatments is now emerging for debilitating conditions including spinal muscular atrophy3 and inherited retinal diseases4 5. Although Europe was at the forefront6 of science a decade ago, other regions have invested heavily in research and Europe has work to do to re-gain7 its leadership role.

A cell is the fundamental biological building block of all living things8. Genes, found within cells, are small sections of DNA that carry the cells genetic information. Genes are the cells blueprint for making proteins that support functions such as muscle strength, and mutations in genes contribute to a range of diseases including those caused by recessive gene disorders, like hemophilia and sickle cell anemia, acquired genetic diseases such as certain cancers, and some viral infections like AIDS9.

Gene therapies improve and/or replace mutated genes, most often via the technique of recombinant DNA technology in which a molecular carrier known as a vector is used to carry a healthy copy of the gene and introduce the new genetic material into the cell11. Most commonly, gene therapy allows cells to add an additional genetic unit, but new engineering techniques now exist to switch off, allow modification to or correct the problematic DNA sequence12.

Europes gene therapy leadership

Academic researchers, biotech and pharmaceutical companies in Europe have been instrumental in developing the field of gene therapy13. Between 2017 and 2019, the lead author of 120,000 papers in cell and gene therapy (CGT) publications was affiliated with a European institution, compared to 72,000 and 100,000 in the U.S. and China respectively14. Professor Emmanuelle Charpentier at the Max Planck Unit for the Science of Pathogens in Berlin was a joint winner of the Nobel Prize in Chemistry 2020, a recognition of her contribution to the development of tools for gene editing15.

The European Unions medical regulatory institution, the European Medicines Agency (EMA), has been a world leader in assessing advanced therapy medicinal products (ATMPs), a class of innovative biological products of which gene therapy is part, approving a tissue-engineering product in 2009, a year before the U.S.s first ATMP approval16. It also approved the worlds first gene therapy in 201216. The EMA has granted orphan drug status to the majority of CGT drugs currently in development and performed accelerated assessments for a number of cell and gene therapies14.

But the region needs to build on its momentum to take gene therapy from the lab to patients. The total number of gene-, cell- and tissue-based therapeutic developers worldwide lags behind the North American and Asia-Pacific regions17.

Europes gene therapy sector could be boosted by introducing more flexibility and agility to the methodologies that are used to evaluate and reimburse breakthrough treatments.

Because the diseases that cell and gene therapies are aimed at are rare, trials are conducted in a far smaller number of patients than is normal for a new medicine. Decisions to license the treatment will be based on the data from one small trial7.

Health Technology Assessment (HTA) bodies, who ultimately determine whether a new therapy is reimbursed, expect to review randomized controlled trials against a standard of care18. Yet gene therapies are a new approach that can effectively intercept, rather than manage, a disease and there is therefore no standard of care to compare against19.

Cost-effectiveness is commonly evaluated based on the direct financial burden of a disease on the health system18. Yet many genetic diseases biggest impact is indirect inherited blindness, for instance, is costly for the individual, their family and carers, and most costs are borne in the social and welfare system, rather than in clinics and hospitals20. One review by Deloitte estimated the total cost attributable to inherited retinal diseases at 523.3 million in 2019, across economic and wellbeing costs (see chart). Health system costs were the lowest share20.

Gene therapies that have the potential to intercept a disease entirely, are more difficult to quantify in terms of benefits19. Conventional HTA methods discount a benefit over time as a patients quality of life deteriorates due to aging and its other attendant health consequences21. This makes little sense in the case of a gene therapy that can avoid blindness emerging in a young person, say, thereby allowing them to lead a full and active adult life21.

Unlike rare disease treatments that are regularly administered over decades, gene therapy would be administered only once, providing many years, if not a lifetime, of biological activity and clinical benefit2. Under current reimbursement systems, this therapy would be paid once at the time it is administered. To encourage investment in the development of these therapies, payments in excess of $1 million may be needed2. The fact that one intervention can provide a cure, replacing long-term costs of treatment, makes investment in gene therapy a problem that multiple stakeholders need to work together to solve2. The industry is developing new approaches to pricing and reimbursement, such as outcome-based payment models22, orannuity payments23 based upon duration and efficacy, which could help payers accommodate the one-time nature of gene therapies.

Europe is already moving towards a coordinated response to review the challenges of breakthrough therapies. In December 2021, Europes Regulation on Health Technology Assessment was adopted to help member states to make timely and evidence-based decisions on patient access to innovative health technologies24. Gene therapies are amongst the medical breakthroughs that European reform dialogue is focused on to address unequal access across the continent25.

Building on this momentum can bring forth the required change to value assessment methodologies and help to realize the full potential of gene therapies. It will ensure gene therapy, long confined to research laboratories, can finally reach patients, and open a new chapter in medical history21.

Follow our journey at https://www.politico.eu/evolution-of-health-care/

References

1Mayo Clinic Staff. (2017). Gene Therapy. Available at:https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619

2Brennan, T. and Wilson, J. (2014). The special case of gene therapy pricing. Available at:https://www.nature.com/articles/nbt.3003

3Vamshi K. et al. (2018). Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease. Available at: https://www.jmcp.org/doi/pdf/10.18553/jmcp.2018.24.12-a.s3

4EMA Europe. (2018). Luxturna, European Medicines summary of product characteristics. Available at https://www.ema.europa.eu/en/documents/product-information/luxturna-epar-product- information_en.pdf

5Maldonado, R. et al. (2020). Curative gene therapies for disease. Available at: https://link.springer.com/article/10.1007/s12687-020-00480-6

6Cressey, D. (2012). Europe Nears First Approval for Gene Therapy Treatment. Available at: https://www.scientificamerican.com/article/europe-nears-first-approval-gene-therapy-treatment/

7Collis, H. (2022). Patients with rare diseases hope for Brexit hastened treatments. Available at: https://www.politico.eu/article/uk-gene-cell-therapy-brexit-science-research-rare- diseases/

8NCBI. (1989). The Fundamental Biological Unit of All Living Organisms is the Cell. Available at: https://www.ncbi.nlm.nih.gov/books/NBK217797/

9NCBI. (2017). Gene therapy: advances, challenges and perspectives. Available at:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5823056/

10American Society of Gene + Cell Therapy. (2021). Gene, Cell, & RNA Therapy Landscape. Available at: https://asgct.org/global/documents/asgct-pharma-intelligence-quarterly-report-july-20.aspx?_zs=sisac&_zl=Uu4h2

11Britannica. (2022). Recombinant DNA Gene therapy. Available at:https://www.britannica.com/science/recombinant-DNA-technology/Gene-therapy

12Medline Plus. (2022). What is gene therapy?. Available at:https://medlineplus.gov/genetics/understanding/therapy/genetherapy/#:~:text=A%20newer%20technique%2C%20called%20genome,existing%20DNA%20in%20the%20cell

13AuWerter, T. et al. (2020). Biopharma portfolio strategy in the era of cell and gene therapy. Available at: https://www.mckinsey.com/industries/life-sciences/our-insights/biopharma-portfolio-strategy-in-the-era-of-cell-and-gene-therapy

14Loche, A. et al. (2021). A call to action: Opportunities and challenges for CGTs in Europe. Available at: https://www.mckinsey.com/industries/life-sciences/our-insights/a-call-to-action-opportunities-and-challenges-for-cgts-in-europe

15Rogers, K. (2020). Emmanuelle Charpentier. Available at: https://www.britannica.com/biography/Emmanuelle-Charpentier

16NCBI. (2019). Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States. Available at:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6728416/

17Alliance for Regenerative Medicine. (2022). Regenerative Medicine: Disrupting the status quo. Available at:http://alliancerm.org/wp-content/uploads/2022/03/ARM_AR2021_FINAL-singles.pdf

18EUPATI. (2022). HTA systems in Europe. Available at: https://toolbox.eupati.eu/resources/hta-systems-in-europe/

19Pochopie M et al. (2021). An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8592603/

20Deloitte Access Economics & Retina International. (2019). The socio-economic impact of inherited retinal dystrophies (IRDs) in the United Kingdom. Available at: https://www2.deloitte.com/content/dam/Deloitte/au/Documents/Economics/deloitte-au-economics-cost-illness-irds-uk-030919.pdf

21Salzman, R. et al. (2018). Addressing the value of gene therapy and enhancing patient access to transformative treatments. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6277509/

22Jrgensen, J. and Kefalas, P. (2019). The use of innovative payment mechanisms for gene therapies in Europe and the USA. Available at: https://www.futuremedicine.com/doi/10.2217/rme-2020-0169

23Cook, F. et al. (2018). Regenerative Medicine is Here: New Payment Models Key to Patient Access. Available at: https://alliancerm.org/wp-content/uploads/2018/07/ARM_WhitePaper3_IV1807_LRS.pdf

24HTA Regulation Press. (2021). Health Technology Assessment: Commission welcomes the adoption of new rules to improve access to innovative technologies. Available at: https://ec.europa.eu/commission/presscorner/detail/en/ip_21_6771

25Cornetta, K. et al. (2022). Gene therapy access: Global challenges, opportunities, and views from Brazil, South Africa, and India. Available at: https://www.sciencedirect.com/science/article/pii/S1525001622002301

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Building Europes gene therapy ecosystem POLITICO

FDA approval brings first gene therapy to the United States

For Immediate Release: August 30, 2017

This release was updated on Aug. 30, 2017 to correctly identify the FDA designations granted to Kymriah.

Espaol

The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, said FDA Commissioner Scott Gottlieb, M.D. 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. At the FDA, were committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patients own T-cells, a type of white blood cell known as a lymphocyte. The patients T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year. ALL can be of either T- or B-cell origin, with B-cell the most common. Kymriah is approved for use in pediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.

Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research (CBER). Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.

The safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 percent.

Treatment with Kymriah has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events. Both CRS and neurological events can be life-threatening. Other severe side effects of Kymriah include serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). Most symptoms appear within one to 22 days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.

The FDA today also expanded the approval of Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of Actemra.

Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events. Additionally, the certified health care settings are required to have protocols in place to ensure that Kymriah is only given to patients after verifying that tocilizumab is available for immediate administration. The REMS program specifies that patients be informed of the signs and symptoms of CRS and neurological toxicities following infusion and of the importance of promptly returning to the treatment site if they develop fever or other adverse reactions after receiving treatment with Kymriah.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

CAR T-cell therapy approved to treat certain children and young adults with B-cell acute lymphoblastic leukemia

Andrea Fischer 301-796-0393

Angela Stark 301-796-0397

888-INFO-FDA, OCOD@fda.hhs.gov

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FDA approval brings first gene therapy to the United States