Impact of Radiation Therapy on Survivors of Pediatric Abdominal and Pelvic Tumors – Cancer Network

A study published in Cancer Epidemiology, Biomarkers & Prevention suggested that among survivors of childhood abdominal or pelvic tumors, abdominal or pelvic radiotherapy is associated with body composition changes that can adversely influence metabolic outcomes and performance status.1

The impacts of radiation therapy on metabolic health have previously been reported for survivors of pediatric leukemia, brain tumors, and hematopoietic stem cell transplants, according to Carmen Wilson, PhD, assistant member in the Epidemiology and Cancer Control department at St. Jude Childrens Research Hospital, though the impacts on survivors of pediatric abdominal and pelvic tumors were unclear.2

Body composition abnormalities and cardiometabolic impairments are of concern among survivors given that in the general population, these conditions increase the risk of developing life-threatening diseases including cardiovascular disease and type 2 diabetes, Wilson said in a press release.

In total, the study cohort included 431 survivors of abdominal or pelvic tumors. Relative lean mass and fat mass were assessed in participants using dual X-ray absorptiometry and metabolic outcomes, including insulin resistance, high-density lipoprotein, low-density lipoprotein, and triglycerides, were based on laboratory values and medication usage.

Lean mass was found to be lower than values from the National Health and Nutrition Examination Survey (NHANES) in both males (Z-score = -0.67 1.27; P < .001) and females (Z-score = -0.72 1.28; P < .001). Higher cumulative abdominal and pelvic radiation doses were also associated with lower lean mass among males (abdominal: b = -0.22 [SE] 0.07; P = .002 and pelvic: b = -0.23 0.07; P = .002) and females (abdominal: b = -0.30 0.09; P = .001 and pelvic: b = -0.16 0.08; P = .037).

Collectively, ours and others data suggest that survivors may be at risk of developing diabetes through multiple pathways, either from direct damage to the pancreas following radiotherapy, and following [insulin resistance] as a result of alterations in function and secretions of adipocytes and from increased adiposity, the authors explained.

Moreover, the prevalence of insulin resistance (40.6% vs. 33.8%; P = .006), low HDL (28.9% vs. 33.5%; P = .046), and high triglycerides (18.4% vs. 10.0%; P < .001) was increased among survivors relative to NHANES. Compared with survivors with normal to high lean mass and normal to low fat mass, those with normal to high lean mass and high fat mass had an increased risk of insulin resistance (P < .001), low HDL (P < .001), reduced quadriceps strength at 60 per second (P < .001) and 300 per second (P < .001), and reduced distance covered in a 6-minute walk (P < .01).

High adiposity and associated reductions in strength, mobility, and flexibility among survivors are of concern because these measures are markers of physical fitness; higher levels of fitness are associated with decreased risk of cardiovascular disease, hypertension, and noninsulin-dependent diabetes mellitus in the general population, the authors noted.

Notably, given that the researchers used data from NHANES for comparisons of prevalence, differences in the measurement of cardiometabolic outcomes among the survivor cohort and NHANES may have adversely influenced comparisons of prevalence. Even further, though the investigators were unable to examine the influence of abdominal or pelvic surgeries on functional performance among survivors, survivors with serious, severe, or disabling chronic musculoskeletal and neurologic conditions were excluded from analyses.

Moving forward, though it may not be possible to avoid radiotherapy as a key treatment for many solid tumors, the researchers suggested that further research is required to evaluate whether interventions in both child- and adulthood could meliorate abnormalities in body composition and cardiometabolic impairments. In addition, Wilson suggested she is interested in exploring how interventions directed at lifestyle behaviors could improve lean mass and decrease fat mass among survivors of pediatric cancers.

While it may not be possible to avoid radiation therapy as a key treatment for many solid tumors, early research suggests that resistance training interventions in survivors increase lean mass, said Wilson. Further work is needed to see if training would also impact cardiometabolic impairments in this population.

References:

1. Wilson CL, Liu W, Chemaitilly W, et al. Body Composition, Metabolic Health, and Functional Impairment among Adults Treated for Abdominal and Pelvic Tumors during Childhood. Cancer Epidemiology, Biomarkers & Prevention 2020. doi: 10.1158/1055-9965.EPI-19-1321.

2. Radiation Therapy for the Treatment of Pediatric Cancers May Have Long-term Impacts on Cardiovascular and Metabolic Health [news release]. Published August 13, 2020. Accessed August 17, 2020.

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Impact of Radiation Therapy on Survivors of Pediatric Abdominal and Pelvic Tumors - Cancer Network

Patients at Hackensack Meridian Childrens Cancer Institute Graduate in a Virtual Commencement Ceremony Celebrating the Class of 2020 – Newswise

Newswise HACKENSACK, NJ (August 25, 2020) Hackensack Meridian Joseph M. Sanzari Childrens Hospital is pleased to announce that 29 patients who have undergone treatment at Hackensack Meridian Health Childrens Cancer Institute graduated in a virtual commencement ceremony celebrating the Class of 2020 earlier this summer. These graduates were honored by Hackensack University Medical Center leadership, sports figures, and celebrities who recognized their academic achievements and graduation from high school.

The Childrens Cancer Institute provides treatment to children through age 22 who suffer from all types of cancers and blood diseases. At its inception in 1987, it was the first pediatric cancer program in New Jersey and has since grown to become the largest pediatric program in the state. The Childrens Cancer Institute offers innovative clinical trials, groundbreaking research initiatives, and cutting-edge therapies to target diseases, as well as personalized care to meet the different needs of each child. Services provided include hematology, comprehensive sickle cell treatment, pediatric stem cell transplant programs and the Cure and Beyond Survivorship program.

The 2020 graduates celebrated in the ceremony include those who were treated at the Childrens Cancer Institute as young children, as well as those who have been treated during their last year of high school. The commencement ceremony acknowledged these students for their accomplishments both inside and outside the classroom.

The virtual commencement ceremony, organized by Sarah Donnangelo, school liaison, Joseph M. Sanzari Childrens Hospital, was the first of its kind. Having worked closely with patients and families for more than 20 years at the Childrens Hospital, Donnangelo recognized the need to join families and children who were treated at the Institute in celebrating their accomplishments.

This is a celebration Ive had the pleasure of organizing for the past 22 years, and I really did not think it would happen this year due to social distancing and COVID-19, remarked Donnangelo. But surprise, with a little help from some friends, we came together virtually to celebrate all of you. This is truly one of my favorite things to do each year because it reminds me that despite all of the obstacles we are sometimes forced to deal with in our lives, they do not prevent us from achieving our goals or living our dreams.

This celebration of life is held in honor of those kids who persevered, said Mark D. Sparta, FACHE, president and chief hospital executive, Hackensack University Medical Center, executive vice president, Population Health, Hackensack Meridian Health. This years group includes graduates who come from all over northern New Jersey. Some have been followed since birth while others range from still being on active treatment, to being many years off therapy and followed in our Cure and Beyond program."

I am incredibly humbled and delighted to be a part of your celebration and congratulate each and every one of you on your accomplishments, said Sparta during the ceremony. Although this years celebration is somewhat of a modified version due to the social distancing requirements of COVID-19, it is still a very important celebration to us and should be to you, as well.

Sparta also paid tribute to physician Frances Flug, M.D. and read a line from Dr. Seuss Oh the Places Youll Go, as Dr. Flug did year after year in honor of graduates journey ahead. Id be remiss if I didnt also pay tribute to one of our beloved physicians at the Childrens Cancer Institute, who was a staple at this celebration year after year. Dr. Flug passed away in April 2019, but had provided care for many of the graduates over their multi-year course of treatment.

Corporate and community scholarships awarded to the Class of 2020 were announced, including the Tom Coughlin Jay Funds 2020 Barry Family Scholarship to Sean OMalley and the 2020 Tom Coughlin Jay Fund Dell Scholarship awarded to William Coyle. A Radio City Rockette also offered words of praise to Gabrielle Peko on earning the Garden of Dreams Inspire Scholarship.

Three graduates received the Kyle Egan Scholarship: Ashanti Columbie, Caylin Batt, and Jesus Sanchez. A special tribute in memory of Kyle Egan, who would have joined his fellow Childrens Cancer Institute graduates, also took place. Kyle was diagnosed with osteosarcoma in January 2018 and he passed away at age 17 in November 2019. Paige Egan, his mother, shared encouraging words with the graduates.

High school graduation is one of the most important milestones in our life, said Alfred Gillio, M.D., director, Childrens Cancer Institute, Hackensack Meridian Childrens Health. You have struggled with many hematologic and oncologic diseases, and received many types of therapy and today is your day. I commend you for your great effort to finish your work and graduate from high school during your illness in these very difficult times.

I also want to applaud the families of the graduates, Dr. Gillio continued. Many of you have had a big role in continuing the education process. The medical staff at the hospital is very excited about your graduation. We have two missions at Hackensack University Medical Center first to deliver the best, most up-to-date care and second, to preserve the quality of the life of our patients. And for those of you in school, that means continuing your high school education.

Graduations are typically filled with speeches trying to inspire young people heading out on the next phase of life to achieve great things, said Judy Aschner, M.D., physician-in-chief of the Joseph M. Sanzari Childrens Hospital. This graduation is different, because it is all of you who have inspired me, and all of us celebrating your graduations today. You have inspired us with your bravery, your resilience, and your ability to see beyond hard times to a better future. You are extraordinary.

Bruce Buffer, renowned boxing personality, announced the start of the video montage, which highlighted all of the graduates names and photos. I know youve been treated for cancer and blood disorders at some point during your life, but now you all are truly the champions, Buffer declared. You can do this! Today is truly the first day of the rest of your lives.

Pre-recorded commencement remarks from former New York Giants football player Mark Herzlich, dancer Valentin Chmerkovskiy of ABCs Dancing with the Stars, and NBC 4 New York reporter David Ushery were also shared with the graduates and their families.

I wish you congratulations and all the best in your future plans, whatever that may be, and know that you have come out of a special program and an institution that really cares about you, said Herzlich. You are in a brotherhood and sisterhood with me, with survivors, and with people who not only have survived, but are striving after.

This group specifically has gone through so much already, said Chmerkovskiy. You have overcome so many obstacles. Beyond just academics, which are hard enough, if anyone can handle this, it is you guys. You are the definition of strength and resilience, with a never give up attitude. I am so proud you, and I dont even know you personally, but I am so proud of you because you didnt defy the odds, you didnt just persevere, you got an education, and developed.

I had a chance to read a short bio of each one of you and I am amazed, not by only what you have endured and by what youve learned, but what you teach us about perseverance and strength, shared Ushery. For some kids, graduating from high school is their greatest challenge, but we know you have all been confronted with real challenges long before today. You faced the challenge of a life threatening disease. You know how to be a strong. Youre fighters. Youve all learned how powerful you are, and with that power, you now have the will to do anything you want in this world. Congratulations to all of the graduates, parents and everyone from the Childrens Cancer Institute who helped you along the way. From our family at NBC 4 New York to yours, we honor you and congratulate you.

About Hackensack Meridian Childrens Health

Hackensack Meridian Childrens Health provides the most comprehensive and highest level of quality care to young patients in the state of New Jersey. The childrens network is comprised of two childrens hospitals Joseph M. Sanzari Childrens Hospital at Hackensack University Medical Center in Hackensack and K. Hovnanian Childrens Hospital at Jersey Shore University Medical Center in Neptune and a large network of pediatric subspecialists and pediatricians. Hackensack Meridian Childrens Health hospitals are the only two in New Jersey to be ranked among the top 50 in the nation for pediatric cancer care by U.S. News and World Report. Joseph M. Sanzari Childrens Hospital also ranks among the top 50 in the nation for pediatric neurology and neurosurgery. Visit http://www.hackensackmerdianhealth.org/kids for more information about Hackensack Meridian Childrens Health.

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Patients at Hackensack Meridian Childrens Cancer Institute Graduate in a Virtual Commencement Ceremony Celebrating the Class of 2020 - Newswise

Cell Therapy Market Technologies Overview, Global Share, Company Profiles, Business Opportunity and Forecast 2025 | JCR Pharmaceuticals Co., Ltd.,…

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By Use & Type Outlook, (Clinical-use,By Cell Therapy Type,,Non-stem Cell Therapies,Stem Cell Therapies,BM, Blood, & Umbilical Cord-derived Stem Cells,Adipose derived cells,Others), By Therapeutic Area, (Malignancies,Muscoskeletal Disorders,Autoimmune Disorders,Dermatology,Others,Research-use), By Therapy Type, (Allogenic Therapies,Autologous Therapies)

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Besides all the information presented in above references, this elaborate research offering by has been thoroughly assessed and evaluated based on thorough primary and secondary research approaches to arrive at logical conclusions of the Cell Therapy market. Besides presenting notable insights on Cell Therapy market factors comprising above determinants, the report further in its subsequent sections of this detailed research report on Cell Therapy market states information on regional segmentation, as well as thoughtful perspectives on specific understanding comprising region specific developments as well as leading market players objectives to trigger maximum revenue generation and profits in the near future according to elaborate speculations.

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Adroit Market Research is an India-based business analytics and consulting company incorporated in 2018. Our target audience is a wide range of corporations, manufacturing companies, product/technology development institutions and industry associations that require understanding of a Markets size, key trends, participants and future outlook of an industry. We intend to become our clients knowledge partner and provide them with valuable Market insights to help create opportunities that increase their revenues. We follow a code- Explore, Learn and Transform. At our core, we are curious people who love to identify and understand industry patterns, create an insightful study around our findings and churn out money-making roadmaps.

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Ninlaro Found to Be Effective and Safe for Treatment of Newly Diagnosed Multiple Myeloma – Curetoday.com

In addition to improving progression-free survival (PFS) in patients with multiple myeloma who have undergone an autologous stem cell transplant, Ninlaro (ixazomib) is also well-tolerated, with low-grade, manageable side effects, according to results of the TOURMALINE-MM3 study.

After earlier phase 3 results of the TOURMALINE-MM1 study led to the approval of Ninlaro in combination with Revlimid (lenalidomide) and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy, the safety and efficacy of the oral proteasome inhibitor were further studied in the TOURMALINE-MM3 trial.

Using the findings from this study, researchers then published additional safety data in the Annals of Hematology, focusing on adverse event (AE) management recommendations for clinical practice, which doctors can use to help patients adhere to treatment and maintain their quality of life.

In the MM3 study, 656 patients were randomized 3:2 to receive either oral Ninlaro at a dose of 3 mg (395 patients) or placebo (261 patients) on days one, eight and 15 in 28-day cycles. If the drug was found to be tolerable, the dosage was increased to 4 mg at cycle five and continued up to 26 cycles (around 2 years) or until disease progression or unacceptable toxicity.

Measuring PFS, which is the time from treatment to disease worsening, was the main goal of the study. Assessing safety was another goal.

At a median follow-up of 30.9 months in the Ninlaro arm and 31.1 months in the placebo arm, 97% of patients experienced AEs of any severity, including thrombocytopenia, neutropenia, gastrointestinal toxicities, peripheral neuropathy, thromboembolic events, pneumonia, and herpes zoster.

While the rate of serious or severe AEs was higher in the Ninlaro arm compared to the placebo arm (19% versus 5%), the rate of treatment discontinuation due to AEs was similar between both arms (7% for Ninlaro versus 5% for placebo).

The most frequently reported AEs in the Ninlaro arm were nausea, diarrhea, and vomiting, which researchers noted were expected and consistent with previous studies evaluating the drug. The overall rates of AEs of clinical interest were higher in the treatment arm than in placebo, as seen with:

According to the study, the majority of AEs experienced were low-grade, non-serious, manageable with supportive therapy or dose reduction, and did not result in cumulative or long-term/late-onset toxicity. The researchers also noted that the AEs were reversible, and did not result in discontinuation, leading them to conclude that single-agent Ninlaro is a safe and effective option for this newly diagnosed patient population.

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Ninlaro Found to Be Effective and Safe for Treatment of Newly Diagnosed Multiple Myeloma - Curetoday.com

First Woman May Be Cured of HIV Without a Bone Marrow Transplant – POZ

A California woman may be the first person to be cured of HIV without a bone marrow transplant, according to a recent report in Nature. More than 60 other so-called elite controllers, who have unusually potent immune responses to HIV, were found to have their virus sequestered in parts of their genome where it is unable to replicate.

The unusual case involves Loreen Willenberg, who acquired HIV in 1992. Her immune system has maintained control of the virus for decades without the use of antiretroviral treatment, and researchers have been unable to find any intact virus in more than 1.5 billion of her cells. Elite controllers are thought to make up less than half a percent of all people living with HIV.

I believe Loreen might indeed meet anyones definition of a cure, study coauthor Steven Deeks, MD, of the University of California at San Francisco, told POZ. Despite heroic efforts, we just could not find any virus that is able to replicate. Her immune system seems completely normal. Even her HIV antibodies levels are low, which is unprecedented in an untreated person.

Although antiretroviral therapy can keep HIV replication suppressed, the virus inserts its genetic material into the chromosomes of human cells, making it very difficult to eradicate. HIV can lie dormant in a reservoir of resting immune cells indefinitely, but when antiretrovirals are stopped and the cells become activated, they can start churning out new virus.

Previously, only two people were known to have been cured of HIV: Timothy Ray Brown, formerly know as the Berlin Patient, and a man in London. Both received bone marrow stem cell transplants from a donor with a rare genetic mutation that makes cells resistant to HIV entry. But this procedure is far too dangerous for people who dont need it to treat advanced cancer.

The new research suggests that Willenberg and some five dozen other people with long-term untreated HIV have their virus hidden away in their cells genomes in such as way that the viral genetic blueprint (known as a provirus) cant be used to produce new viral particles that can go on to infect other cells.

Xu Yu, MD, of the Ragon Institute of Massachusetts General Hospital, MIT and Harvard analyzed integrated HIV in millions of cells from 64 elite controllers and 41 typical HIV-positive people on antiretroviral therapy recruited at Mass General and San Francisco General Hospital.

In both groups, about 20% were women, the average age was approximately 56, they had been living with HIV for an average of 17 years and had undetectable virus according to standard tests for nine years. Overall, the elite controllers had a higher average CD4 count (about 900 versus 70, respectively).

The researchers used next-generation gene sequencing to characterize the participants viral blueprints, including where they were inserted into human chromosomes. They found that the elite controllers had fewer integrated proviruses, but a larger proportion of them were intact, or potentially capable of replicating. The virus in these individuals was highly consistent, without the wide variety of mutations seen in most people with HIV.

Whats more, their proviruses were integrated at distinct sites in the human genome, farther away from elements that enable viral replication. Specifically, the integrated DNA was not located near sites that switch on transcription or close to accessible chromatin, which contains histone proteins that package long DNA strands into a more compact form. The DNA must then be unwound from these proteins before it can be used to produce new virus.

These data suggest that a distinct configuration of the proviral reservoir represents a structural correlate of natural viral control, and that the quality, rather than the quantity, of viral reservoirs can be an important distinguishing feature for a functional cure of HIV-1 infection, Yu and colleagues wrote.

In a commentary accompanying the report, Nicolas Chomont, PhD, of the University of Montreal, characterized the proviruses in these elite controllers as being in a state of deep sleep compared with latent virus in typical people with HIV. This has only become apparent now because researchers have more sophisticated tools to pinpoint the location of proviruses within the genome.

It is unclear why this block and lock phenomenon happens in only a small proportion of people with HIV. Its possible that the virus ends up sequestered in these locations by chance. But the researchers think its more likely that the integrated proviruses at these sites are evolutionarily selected over time as the ones in locations more conducive to viral replication are eliminated by the immune system.

In Willenbergs case, the research team analyzed more than 1.5billion of her peripheral blood immune cells, including samples from gut tissue, where the virus often hides. Theycould not find any intact proviruses that could be used to produce new HIV. Given her absence of intact proviruses, the researcherswere unable to determine whether she ever fit the pattern of having latent HIV locked away in inaccessible locations.

Another 11 people, dubbed exceptional controllers, only had detectableprovirusesatremote sites in the genome where it could not replicate.Since this study, the researchers have discovered a couple more elite controllers who may qualify as additional cures, according to the New York Times.

This raises the possibility that a sterilizing cure of HIVmeaning complete eradicationmay be feasible in rare instances, the study authors suggested. A similar but less complete process may be at play in the subset of about 10% of people with HIV who maintain viral suppression after stopping antiretroviral therapy but who still have detectable proviruses (known as post-treatment controllers).

This research was first presented at the International AIDS Society Conference on HIV Science last summer, where Willenberg was referred to as the San Francisco Patient. Willenberg later went public with her status, and she and Yu discussed the study findings during a webinar with HIV cure advocates last November.

I broke out in tears when saw Dr. Yus final slide, said Willenberg, who over the years has participated in more than a dozen studies. I can only hope and pray that with continued dedication we can figure out how I have dumped the virus into the DNA junkyard.

The question now is whether its possible to develop treatments that could enable the millions of typical people with progressive HIV to become elite controllers like Willenberg. Chomont suggested that immune-based therapiesincluding CAR-T cellsmight be able to shrink the viral reservoir until it consists only of deeply latent proviruses that are unable to replicate.

The key question is how did her immune system achieve this remarkable state, Deeks said. We do not know. We need to find more people who are exceptional controllers like Loreen and get to work on figuring out the mechanism.

In this video fromamfAR, the Foundation for AIDS Research,Loreen Willenbergtalks about living as an elite controller of HIV.

Click here to read the Nature article. Click here for more news about HIV cure research.

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First Woman May Be Cured of HIV Without a Bone Marrow Transplant - POZ

Study: Brain cells of people with autism differ even before theyre born – Study Finds

PHILADELPHIA Autism is a neurodevelopmental disorder which creates difficulties with social interactions, communication, and repetitive behaviors. According to the Centers for Disease Control and Prevention, autism affects one in 54 children in the United States. Although autism typically isnt diagnosed until children are at least 18 months-old, a new study finds that abnormal brain cell development likely occurs much earlier.

Researchers say this even occurs while babies are still in the womb.

To study developing brain cells, a team from Kings College London and Cambridge University use a type of cell known as an induced pluripotent stem cell (or iPSC). Essentially, iPSCs are adult cells that scientists force to become immature embryonic-like stem cells. Scientists can program iPSCs to become one of many different cell types, including neurons. Since iPSCs are forced to restart their cellular development, they mimic the processes occurring in the womb. This allows them to serve as a useful means of studying early brain development.

Using iPSCs from hair samples is the most ethical way to study early brain development in autistic people, explains study author Dwaipayan Adhya in a media release. It bypasses the need for animal research, it is non-invasive, and it simply requires a single hair or skin sample from a person.

Adhya is a molecular biologist at the Autism Research Centre in Cambridge and Department of Basic and Clinical Neuroscience at Kings College London.

To create the iPSCs, the study analyzes hair samples from nine adults with autism and six neurotypical adults. The hair cells were then treated with growth factors (naturally occurring bodily substances that regulate cell division and survival). The authors looked at the cells appearance and genetic makeup at different phases of development.

The scientists results reveal iPSCs from neurotypical people look different from those participants with autism. At day nine, neurons from neurotypical people develop a characteristic pattern of neural rosettes, which have a dandelion-like shape. In contrast, cells from people with autism have smaller rosettes or dont form them at all. Cells from autistic individuals also express lower levels of important developmental genes.

The use of iPSCs allows us to examine more precisely the differences in cell fates and gene pathways that occur in neural cells from autistic and typical individuals, co-author Deepak Srivastava explains. These findings will hopefully contribute to our understanding of why there is such diversity in brain development.

The brain has been the ultimate black box. Here, the authors have used nerve cells derived from peripheral stem cells to peek inside this box. This important study suggests that this is possible and is deepening our understanding of autism, says John Krystal, Editor-in-Chief of the journal Biological Psychiatry.

The study is published in the June 22 edition of Biological Psychiatry.

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Study: Brain cells of people with autism differ even before theyre born - Study Finds

Regulating advanced therapies: Q&A with patent lawyer David Silverstein – Pharmaceutical Technology

Axinn partner and patent attorney David Silverstein. Credit: Axinn. What challenges do cell and gene therapies pose to regulators? Credit: Shutterstock.

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Innovation is a key focus of the pharma industry. This has led to dramatic improvements in the treatment options available to at-need patients. Due to technological innovation and the development of cell and gene therapies, it is now possible to use CAR-T treatments to cure certain types of cancers and particular patient populations. in

Although these innovations drive medicine forward, they pose a significant challenge to regulators. These bodies need to stay abreast of the many new, emerging trends in the industry and figure out how best to assess these novel products for safety, efficacy and value.

Axinn partner and patent attorney with more than 15 years of experience looking at regulatory matters related to the pharma market David Silverstein discusses why cell and gene therapies are such an important development for the industry. However, he also notes how advanced therapies have challenged regulators, particularly linked to the rise in unlicensed clinics administering unapproved regenerative therapies.

Allie Nawrat: What makes cell and gene therapies such an exciting and important development for the pharma industry?

David Silverstein: If you had to analogise it as martial arts, there are traditional ways of treating diseases with small molecules, for example, and that is more like karate it is force against force, forcing the body to do something.

Whereas cell and gene therapies are more like judo, where you use the opponents momentum to your advantage.

To me, these therapies use the strength of the body; evolution has given us the fantastic cellular mechanisms, and cell and gene therapies tap into them.

Its just a much more targeted and efficient way to bring about the result youre after, rather than forcing some foreign small molecule into the cell, and triggering effect.

We are better off for small molecules, but the next step in the evolution of medicine is selling gene therapy.

AN: What challenges have cell and gene therapies created for regulators?

DS: It is a paradigm shift for regulators. Originally, regulators emerged out of the need to ensure that tonics being sold on roadside stands were safe and actually effective, so they are accustomed to dealing with that type of industry. Those products are much easier to describe, characterise and make, than these large, more complicated biological products.

The obvious challenges are around how do you categorise them? You cant just put them all in the same bucket; stem cells are different than gene therapy delivered by a virus. So [there are issues] with the agency just getting its head around that. What are the critical differences that need to be established so that we can evaluate these appropriately? So were not just painting with a broad brush and treating them all the same.

The other challenges [relate to] all the unapproved regenerative therapies. It is a significant [problem]. Studies have shown that [unqualified] people have been discovered administering unlicensed stem cell therapies. [This situation has arisen from] patients who either have exhausted traditional therapies and theyre just desperate to try anything to patients who dont trust what the government and regulators are telling them.

The FDA is struggling with enforcement actions against them; the lack of oversight and regulation on these can lead to fatal results.

AN: How have regulators, and particularly the FDA, responded to the emergence of cell and gene therapies?

DS: In the past several years, the FDA has gotten very motivated. In 2016 and 2017, key FDA guidances came out about the regulation of these products. The FDA had designated 2020 as the year of increased enforcement activity to get under control these unapproved labs that are administering unapproved and potentially dangerous preventative therapies but, unfortunately, Covid-19 has thrown a wrench in their gears.

Well see how priorities shift after this year currently Covid-19 is rightfully in the centre but I dont know if the FDA is going to lose interest or shift its priorities away from regulating these products and engaging in enforcement.

AN: What are the special intellectual property considerations around these advanced therapies?

DS: The challenges are that as these are products of nature you have to be careful and make sure the products different enough so you dont fall afoul of the Supreme Courts case law on patentable subject matter.

Another challenge is the importance of trade secrets. A lot of people when they think about how do you protect these products and manufacture these products, they jump right to patents. But with these kind of products, its important for a stakeholder to take into account trade secret protection. It is an alternative to patent protection. It, of course, complements patent portfolio; but you can protect things with trade secrets that you couldnt protect with patents.

Often I advise clients to segment their manufacturing workflow, so that there isnt one single group or individual who knows every step of the manufacturing process. This is a highly competitive industry and theres a lot of mobility with people changing jobs. Even without getting to malfeasance of people trying to steal trade secrets, you need to think about what knowledge is in peoples heads. You can control it by breaking up your work group so that theyre collaborating together, but no one group knows every step of the process.

AN: What impact has the regulators reactions had on advancing cell and gene therapy innovation?

DS: In the last 10 years, [there has been] an uptick in industry emphasis on them and innovation. This is in part because the regulatory landscape is becoming clearer. If companies know what rules they need to play by, then they can properly assess the risks and rewards of investing in research projects.

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Regulating advanced therapies: Q&A with patent lawyer David Silverstein - Pharmaceutical Technology

FINNCAP’S LIFE SCIENCES REPORT INDICATES CELL AND GENE THERAPY SECTOR IS DRIVING THE NEXT WAVE OF INNOVATION IN PHARMA – PharmiWeb.com

Breakthrough in delivery for cell and gene therapy products has led to a wave of M&A activity as big pharma aims not to miss out on the future of medicine

AIM healthcare index at the centre of innovation, has risen 6% YTD compared with the AIM all share, which has declined 7%

finnLife 50 index has also risen 6% in 2020 led by gains in Synairgen (+2,930%), Avacta (+654%), Omega Diagnostics (+322%) and Tiziana Life Sciences (+283%)

London 25 August 2020 Healthcare companies employing and developing cell and gene therapy (C>) are driving the next wave of innovation in the pharmaceutical industry, leading to increased M&A activity as big pharma aims not to miss out on the future of medicine. The AIM healthcare index has been at the centre of this innovation, rising 6% YTD compared with the AIM all share, which has declined 7%.

These are the findings of finnCaps new quarterly Life Sciences sector report, Rude Health.

Rather than just treating a disease and its symptoms, C> can target the underlying cause of a disease, with long-term benefits and curative potential. C> is now being realised on an applicable level, with many products already approved and the FDA expects to approve 10-20 products a year by 2025.

The financials of the sector are reflective of this rapid progress. In 2018, the market value of C> was $536 million - $1.07 billion; but by 2026 it is set to have a valuation of up to$35.4 billion. Given the high proportion of start-ups in the sector, M&A activity is on the rise, as evidenced by the $3 billion Astellas spent in January 2020 to acquire Audentes Therapeutics, specialists in genetics medicines.

In 2014/2015, M&A activity in the sector was $5 billion; by 2018/2019 it had surged 880% to $49 billion. Much of this is driven by big pharma firms not wanting to fall behind their smaller, more versatile competition, as they did with monoclonal antibody technology. Consequently, they have engaged with M&A to speed up and enhance their own R&D efforts.

The report notes that innovators in C> will be well placed to take part in the land grab that will follow as a result of continued advancements in the sector, and highlights now as a good time for investors and pharmaceutical companies to become involved as the sector is rapidly maturing past its high potential research and development stage with an established pipeline of therapies already being developed.

Some of the key reasons why the report considers the C> sector to be an attractive one for investment are:

Pharmas next wave of innovation. C>s can be potentially curative treatment options as they usually target the underlying cause of disease. In the long term, these therapies could become the backbone of treatment regimens, and solutions to various unmet needs.

Deals. Big Pharma had to play catch-up with monoclonal antibody technology and seems determined not to make the same mistake with C>, as reflected in the high deal activity and high deal values seen within this space.

Sector maturation. Advances in the sector mean that the C> sector is beginning to mature beyond the R&D stage and into commercialisation, with some products already approved, and with a very large future pipeline of therapies.

Revenue.Therapies in this space can command high prices, allowing for high revenue generation, even from rare diseases and limited patient populations.

Despite its vital role in the future of medicine, C> also comes with challenges. The report highlights that the manufacture of C>s is difficult given they are, by definition, personalised for the patient. This means they cannot be batch produced for distribution to multiple patients as more traditional medicines can. For example, Zolgensma, which treats those with motor neurone disease, is priced at $2.1 (1.6) million per therapy, making it the most expensive drug treatment ever.

The report also notes how the payment process for C> requires a reworking of reimbursement systems not used to outlaying so much money up front for a treatment with long-term benefits/curative potential versus continuous, and lower payments for ongoing medicine treatment.

The technologies the report shines a spotlight include CAR-T therapy, stem cell therapy, CRISPR, RNA therapies, among various others.

Arshad Ahad, Research Analyst, Life Sciences, at finnCap, commented:Few technologies in the life sciences sector hold as much promise as Cell and Gene Therapy, with its ability to provide long-term benefits and curative potential. These technologies have been seen as the future of medicine for many years, and now we are closer than ever to that future becoming a reality. If Cell and Gene therapy does become the backbone of treatment regimes in the future, similar to the rise of monoclonal antibodies, then the companies involved are developing expertise in a critical part of the life sciences industry, which should confer a significant competitive advantage as the sector matures further. Now is therefore a good time to invest in the future.

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FINNCAP'S LIFE SCIENCES REPORT INDICATES CELL AND GENE THERAPY SECTOR IS DRIVING THE NEXT WAVE OF INNOVATION IN PHARMA - PharmiWeb.com

The Road To Scale: Challenges Facing the Implementation of Animal-Free Recombinant Proteins Into Stem Cell Supply Chains – Technology Networks

Protein engineering techniques can be used to produce more potent animal-free growth factors as highlighted by the higher level of Nanog expression in iPSCs cultured with an optimized form of the key growth factor TGF-1 (RHS), when compared with cells cultured with the mammalian-cell expressed protein (LHS). Credit: Stemnovate

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Most industries today are under pressure to switch to more ethical and sustainable animal-free alternatives, and now the trend is coming to stem cell labs. As stem cell applications accelerate towards the clinic, novel drug discovery platforms are rapidly scaled, and new transformative stem cell-based technologies such as cultured meat arise, there is a drive to switch to animal-free cell culture media. This move is essential to facilitate future regulatory approval for advanced therapies, and enable pharma and biotech companies to ethically, reproducibly and cost-effectively scale stem cell-based innovations. Most stem cell scientists today use recombinant growth factor and cytokine proteins in their chemically-defined media to supply their cultures with the necessary biological signals required for maintenance of pluripotency, cell proliferation and differentiation into specific cell lineages. However, the fundamental biochemistry and manufacturing processes of these protein messengers can often be overlooked. But as scientists are trying to establish new animal-free systems to support the scale-up of their stem cell applications, the properties and challenges inherent in these proteins are becoming more prominent and frankly a headache for many.

Highlighted here are three key challenges facing pharma and biotech companies as they embark on the path to implementing animal-free systems, from the perspective of two protein scientists. 1.Why batch consistency is kingAs stem cell therapies gear up to make the leap from bench to clinic and the promise of stem cell biology in drug discovery and other industrial applications is realized, more subtle and still largely inexplicable challenges in optimizing growth factor and cytokine supply chains for defined media are being identified why when you change from one supplier or even batch of a recombinant protein do stem cells need weaning onto that protein, or dont tolerate the change? Is this a fundamental lack of batch consistency across the supply chain or is there an underlying biological basis?

At the minute, there is simply not enough data to answer this definitively. While we are starting to tease apart these questions, it highlights the need for greater innovation within the recombinant protein supply chain to bring best practice and innovation from other areas to improve the robustness of the global supply chain and encourage great openness and scrutiny of fundamental biochemical quality early in process development.

Questions we should ask include: are we seeing heterogeneity in post-translational modifications, which is well documented in monoclonal antibody manufacturing? Can synthetic biology or protein engineering be used to optimize proteins and engineer out features contributing to this variation?

For now, and until we have answers, its a good idea to source proteins from reputable suppliers that have rigorous standards for batch quality testing and meticulously scrutinize all biochemical and bioactivity data provided. 2.Cost of goods as a barrier to scaleTo bring innovative stem cell applications to market, pharma and biotech companies need to be able to seamlessly scale their cultures, meet regulatory requirements and achieve a sensible and sustainable process cost. Where recombinant proteins are needed in cell culture media, they are usually the greatest contributor to cost of goods.

Well-defined industry challenges catalyze change and the stem cell field is seeing renewed focus on much needed innovation in complex bioactive protein production to meet the needs for animal-free, highly reproducible proteins. Protein engineering technology, enhanced cell-based and cell-free expression systems, such as bacteria, yeast and even plants, coupled with improvements to downstream processing systems are just some of the latest innovations in this space. Previously, there have been concerns over the ability of simpler systems to form correctly folded and bioactive recombinant proteins. However, it is clear that many of these barriers can be overcome to produce highly pure growth factors and cytokines at scale. Others are striping back chemically-defined media protocols to determine the essential growth factors and cytokines needed for their cell type. For example, homebrews of key growth factors to reduce costs - Paul Burridge and his team at Northwestern University Feinberg School of Medicine have pioneered a cost-effective B8 chemically-defined media for weekend-free hiPSC culture at just 3% of the price of commercially available media. Now the challenge is to take the learnings from academic studies such as these and translate them into industrial processes. Meating the price of lab-grown steaksYou cannot discuss the cost of growth factors without mentioning the daunting step-change and barrier facing the fast-evolving cultured meat market. Here, highly optimized animal-free growth factor production systems will be required to provide the economies of scale needed to deliver kilogram-ton quantities at a fraction of the price in order to bring these lab-grown meat alternatives to consumers. After all, it just isnt viable for companies to be spending hundreds of dollars on each liter of culture media - instead, this needs to be reduced to ~$1/liter. 3.Animal-free or ADCF? Now that is the questionDespite animal-free/animal-derived component free (ADCF) growth factors and cytokines becoming increasing important, there are no standard definitions for these terms across the industry, with many protein manufacturers supporting the sector by defining their own internal standards. For the clinical space, the United States Pharmacopeia and International Organization for Standardization have published a framework for classifying raw materials used in cell therapy manufactureinto four different tiers based on their risk. Under this classification, ancillary materials used in the manufacture of cell-based therapies and tissue-engineered products, such as recombinant growth factors and cytokines, are considered low risk so fall into tiers 1 and 2 with an ADCF level of manufacturing defined as all components, sub-components and consumables do not contain materials derived from animals. To support the wider sector, not just those at the transition to the clinic, clarity over definitions and transparency from manufacturers will help to define and overcome the challenges faced and allow the promise of stem-cell derived innovations to be delivered.

Article Authors:

Beata Blaszczyk, Senior Scientist, Qkine

Dr Catherine Elton, CEO and Founder, Qkine

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The Road To Scale: Challenges Facing the Implementation of Animal-Free Recombinant Proteins Into Stem Cell Supply Chains - Technology Networks

Demand for Myelofibrosis Treatment Market to Witness Rapid Surge During the Period 2016 2022 – Scientect

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

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The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

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As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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Demand for Myelofibrosis Treatment Market to Witness Rapid Surge During the Period 2016 2022 - Scientect