Stem Cells Market is expected to reach US$270.5 billion by 2025, TMR – BioSpace

The global stem cells market is predicted to witness players seeking funds from international organizations for developing new therapies. With a view to cement their position in the market, players could take to the adoption of partnerships and collaborations with pharmas. This could also help them to expand their product portfolios. While these factors are expected to enhance market growth, there could be a few others helping with strong demand for stem cells.

Transparency Market Research (TMR) foresees the global stem cells market to earn a US$270.5 bn by the completion of 2025 while registering a 13.8% CAGR for the forecast tenure 2017-2025. In a research led by University of Singapores (NUS) Professor G.V. Shivashankar and the FIRC Institute of Molecular Oncology (IFOM), it has been found that mature cells can be confined to reprogram them into re-deployable stem cells. Interestingly, this could be achieved without direct genetic modification.

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Induced Pluripotent Stem Cells as Emerging Segment of Market

There are various types of products available in the global stem cells market: induced pluripotent, human embryonic, and adult stem cells. Among these, the market could find adult stem cells collecting a larger share in the coming years. Their demand could increase due to their potential to multiply into trillions of specialized cells capable of repairing tissue damage and lowering the risk of rejection.

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North America Banks on Massive Investments to Lead Market

On the regional front, the global stem cells market is prognosticated to find North America taking a leading position in the near future. By the end of the forecast period, the region could earn US$167.3 bn. The following factors are anticipated to augur well for the regional market.

North America could be trailed by Europe during the course of the forecast period. Medical tourism in European countries such as Germany could push the growth of the market in the region. Germany welcomes patients from the U.S., Canada, and even other countries. On the other hand Asia Pacific is expected to grow at a faster CAGR of 14.6%.

Some of the key players operating in the global stem cells market are STEMCELL Technologies Inc., Astellas Pharma Inc., Cellular Engineering Technologies Inc., BioTime Inc., Takara Bio Inc., U.S. Stem Cell, Inc., BrainStorm Cell Therapeutics Inc., Cytori Therapeutics, Inc., Osiris Therapeutics, Inc., and Caladrius Biosciences, Inc.

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Stem Cells Market is expected to reach US$270.5 billion by 2025, TMR - BioSpace

New sickle cell disease treatments are helping people live longer and giving them a higher quality of life – PhillyVoice.com

Treatment for sickle cell disease has come a long way since the 1970s when the life expectancy of people living with it was less than 20 years.

People with sickle cell disease are not only living longer life expectancy is now 42 to 47 years of age but are enjoying a better quality of life, too.

"In the Philadelphia area, there has been great pediatric care for sickle cells disease and because of that people who have it are living very well," said Dr. Farzana Sayani, a hematologist at Penn Medicine.

Sayani is the director of a comprehensive sickle cell program focusing on adults living with the disease. Penn also has an active transition program for youth transitioning from a pediatric institution to adult care.

Sickle cell disease is an inherited red blood cell disorder that affects about 100,000 Americans.It is most often found in people of African or Hispanic descent.About 1 in 365 African-American babies are born with sickle cell disease, according to Sayani.

People who have the disease inherit an abnormal type of hemoglobin in their red blood cells, called Hemoglobin S, from both their mother and father.When only one parent has the hemoglobin S gene, a child will have the sickle cell trait, but usually does not develop the disease. But they may pass it on to their children.

Hemoglobin is the protein in the blood responsible for carrying oxygen to the rest of the body. Hemoglobin S causes red blood cells to become stiff and sickle-shaped. Instead of being round in shape, they look like crescent moons.

Sickle cells are sticky and can bind together, blocking the flow of blood and preventing oxygen from getting where it needs to go in the body. This causes sudden attacks of pain referred to as a pain crisis.

There are severaldifferent types of sickle cell disease.Hemoglobin SS, also known as sickle cell anemia, is the most common and most severe type of sickle cell disease.

Anemia occurs when red blood cells die at a rate faster than the body can replace them. Normal red blood cells generally live for 90 to 120 days. Sickled cells only live for 10 to 20 days. This shorter life-to-death cycle is harder for the body to sustain.

Another form,Hemoglobin SC, is not as severe as sickle cell anemia, but it can still cause significant complications, Sayani said.Other forms include Hemoglobin S0 thalassemia, Hemoglobin S+ thalassemia, Hemoglobin SD and Hemoglobin SE.

Sickle cell disease screening is a mandatory part of newborn screenings in Pennsylvania.

If the screening is positive, the family is informed and plugged into the health care system in order to receive the proper care.

If the disease is not diagnosed at birth, a blood test can confirm it at any age in which symptoms start to surface.

The severity of sickle cell disease can vary.

Each individual is affected differently, making it difficult to predict who will get what complications, Sayani said. That is why a comprehensive sickle cell program is so important.

Early signs include a yellowish tint to the skin or jaundice, fatigue and a painful swelling of the hands and feet.

"Young children with sickle cell disease may be tired, not eat very well and have delayed growth," Sayani said. "They may also develop anemia, be at greater risk of infection and start to experience pain crises."

Acute pain crises, also known as vaso-occlusive crises, can lead to long stays in the hospital to manage the crippling pain. Children with sickle cell disease also tend to experience delayed growth and puberty.

As a person with sickle cell disease grows older, the sickled red blood cells start to affect various organs, bones and joints.

This can lead to acute chest syndrome, which occurs when damaged lung tissues makes it difficult to breathe. Brain complications, including stroke, are possible.People with sickle cell disease are also prone to heart damage, eye problems, and infections like chlamydia, salmonella and staphylococcus. Chronic and acute pain is common.

There are different types of medicine that can help manage sickle cell disease.

Last year, an oral medicine was approved that makes sickle cells less likely to sickle. So was an intravenous medicine that has been shown to reduce pain crises and hospitalizations by 50%. Some people living with sickle cell disease also may need regular blood transfusions.

Hydroxyurea has also been used successfully for many years to reduce pain crises and the need for blood transfusions and hospitalizations.

Currently, blood and bone marrow transplant is the only way to cure the disease. But it is not an option for everyone because of the difficulty of finding a well-matched stem cell donor.

A related donor is best but only about a third of sickle cell patients have a donor that is related and fully-matched, Sayani said.

While these transplants have a 85% or more success rate, they also are associated with significant risks, including organ dysfunction, infection and graft vs. host disease which can be quite debilitating.

Transplants completed in children have the best results, Sayani said. But because of the risks involved, doctors only suggest it for patients with severe forms of the disease.

Early clinical trials with gene therapy are also showing promise, she added.

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New sickle cell disease treatments are helping people live longer and giving them a higher quality of life - PhillyVoice.com

Olive Oil Consumption Could Lead to a ‘Younger Brain’ in the Elderly, Researchers Say – Olive Oil Times

As people age, the more their brains could benefit from the action of an important component in olive oil.

New research has revealed the deeper effects of hydroxytyrosol (HTyr): Not only does it protect brain functions from aging, but it may even restore the vitality of brain neurons, and multiply them.

Italian scientists at the National Research Center (CNR) investigated how hydroxytyrosol works in those portions of the brain that generate new neurons throughout life. They discovered that HTyr impacts brain activities far beyond its well-known neuroprotective effects.

Researchers at the CNR Biochemistry and Cellular biology Lab (CNR-Ibbc) were able to show how the administration of the compound in the elderly may reverse neuronal aging, combining the protection of the active neurons and the generation of new ones.

Hydroxytyrosol oral consumption by young and older animals within amonth shows not only how the new neurons generated by the brain in that timeframe are protected, but it also hints how in older animals it stimulates the multiplication of stem cells, said Felice Tirone, chief scientist and author of the study published in Faseb Journal. It is from those cells that new neurons are generated.

Tirone and his colleagues explained that HTyr activates neurogenesis in the dentate gyrus of an adult, where new neurons are generated, by increasing survival of new neurons and decreasing apoptosis. The neurons multiplication effect is only found in aged brains.

We also found how the antioxidant activity of hydroxytyrosol activates asort of cleaning treatment for nervous cells, in the sense that it washes away several byproducts of brain aging like the lipofuscin, which are debris found in neuron cells, Tirone explained.

The whole array of these effects hints at the possibility of reversing some of the most known effects of brain aging.

Olive oil is at the core of the Mediterranean diet and, as often reported in these pages, its regular consumption has been scientifically linked to health improvements in humans of any age at every latitude.

The new research hints at anew series of products that could help to relieve the reduction of cognitive abilities in aging humans.

CNR scientists underlined in apress statement that hydroxytyrosol is not only found in olive oils people usually consume but also in the byproducts of olive oil production.

That process impacts on the environment, but the biological waste it produces holds great quantities of hydroxytyrosol. By upgrading procedures and means, by splitting the good compounds from the other byproducts, oil mills could obtain hydroxytyrosol while also reducing the environmental impact of their activities, said Tirone.

The CNR statement explained that scientists could verify how the new neurons produced in the brain of the aged individuals actually enter the neuronal circuits and fuel neuronal functionality.

The daily intake of the compound in our study is similar to the dose ahuman could ingest with an enriched diet or food integrators. Still, the most efficient intake of hydroxytyrosol would happen through olive oil consumption, said Laura Micheli, one of the CNR-Ibbc researchers who signed the study.

Scientists also explained that future research will have to verify the behavioral effects of the increased neuron production and the extent of the HTyr treatment of neural aging.

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Olive Oil Consumption Could Lead to a 'Younger Brain' in the Elderly, Researchers Say - Olive Oil Times

Proteostasis Therapeutics and CF Europe Announce Completion of Patient Enrollment for CHOICES, the First-Ever Personalized Medicine-Based Study in…

Ex Vivo Testing Phase Underway with Tissue Samples Collected from More than 500 Adult CF Patients; Enrollment Target Exceeded

Clinical Testing Phase to Begin in 2H 2020

Company Expects CHOICES to Potentially Serve as the Basis for an MAA in 2021

BOSTON, Feb. 24, 2020 /PRNewswire/ --Proteostasis Therapeutics, Inc. (NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and CF Europe, the federation of 48 national CF Associations in Europe, today announced the completion of enrollment of 502 patients with CF for HIT-CF, a European-based initiative that is paving the path to personalized medicine through the CHOICES clinical trial. CHOICES will test PTI drug combinations in an ex vivo study and then in a clinical trial to assess the predictability of the organoid assay for clinical benefit.

For the ex vivo portion, organoids derived from tissue samples provided by patients enrolled in the study are evaluated for responsiveness to investigational CFTR modulators, including Proteostasis' CFTR potentiator, corrector and amplifier, dirocaftor (DIR), posenacaftor (POS) and nesolicaftor (NES), respectively. Based on an individual's organoid response, patients will be invited to progress to the next portion of the study which is a placebo controlled, double blind, crossover study known as the CHOICES trial (Crossover trial based on Human Organoid Individual response in CF - Efficacy Study).

The results from CHOICES may serve as the basis for a potential Marketing Authorization Application with the European Medicines Agency (EMA) in 2021 through a novel regulatory pathway. This strategic initiative is led by the HIT-CF consortium, funded through the European Commission's Horizon 2020 program. The CHOICES clinical study is part of PTI's broader clinical development strategy for its CFTR modulator candidates that also includes the MORE trial in CF subjects with the most common F508del homozygous genotype.

"The enrollment of more than 500 patients across Europe in the first phase of the HIT-CF project is a testament to the strategic imperative this program holds for both the patient and treatment community," said Geoffrey Gilmartin, M.D., M.M.Sc., Chief Medical Officer of Proteostasis Therapeutics. "With the successful translation of activity from organoids to patients, this study has the potential to usher in a personalized medicine approach to CF. This approach would begin with patients who have less common mutations, but could ultimately serve the broader CF community by delivering personalized treatment choices that maximize benefit based on each patient's responsiveness to therapy."

"We are excited that Proteostasis is participating in the HIT-CF project and supporting our efforts to bring CF treatment to more people across Europe," said Jacquelien Noordhoek, President of CF Europe and representative of the Netherlands Cystic Fibrosis Society (NCFS). "Enrolled individuals are a portion of the approximately 2,300 adults in the European patient registry who are not eligible for any currently approved modulator due to their genotype and the HIT-CF project represents the only option to explore potential benefit of disease modifying drugs for this group. Putting patients with CF first is our highest priority. We are looking forward to continuing our partnerships and providing Europeans with CF the best possible care."

About Organoids

Organoids are cell cultures that grow in a culture dish with properties similar to those of the organ from which they are derived. Because organoids are made from stem cells, they contain the same mutations as the person from whom the biopsies are derived. Investigational drugs which target the basic defect of CF can be used in an organoid system to evaluate rare mutations where the drugs may have a positive effect.

Unlikein vitrosystems such as human bronchial epithelial (HBE) cells, which are derived from lungs that have been removed from CF patients, or the engineered rat-derived FRT cell line (which has had false positive clinical results), rectal organoids are cultured from tissues obtained through a minimally invasive and painless procedure from donors who then become eligible to participate in a clinical study. Organoids can provide valuable insights for donors, including their likelihood of achieving improvements in pulmonary function and reductions in sweat chloride concentration with CFTR modulators based on theex vivoresponse to those drugsi.

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About HIT-CF Europe

HIT-CF Europe is a research project which aims to provide better treatment and better lives for people with cystic fibrosis (CF) and rare mutations. To achieve this, drug candidates are first tested on patient-derived organoids in qualified laboratories acrossEurope. Subsequently, based on the measured signal in the organoids, a smaller group of patients will be invited to participate in clinical trials with investigational molecules from participating pharmaceutical companies.

All participating centers are part of theEuropean Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN). The project has received funding from theEuropean Union'sHorizon 2020 research and innovation program under grant agreement number 755021. For more information, visitwww.hitcf.org.

About Proteostasis Therapeutics, Inc.

Proteostasis Therapeutics, Inc.is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. Headquartered inBoston, MA, theProteostasis Therapeuticsteam focuses on identifying therapies that restore protein function. For more information, visitwww.proteostasis.com.

Safe Harbor

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including but not limited to statements regarding the potential of PTI drug combinations, expectations regarding ex vivotesting of our proprietary combinations in organoids and clinical evaluation in CF patients, the expected timing for enrollment, completion and reporting of results of our CHOICES Phase 3 clinical trial, our commitment to expanding available therapeutic options for CF patients and the intended goals of the CHOICES trial and the ability to serve as a potential basis for future marketing approval. Words such as "aim," "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the potential of our proprietary combination therapies for the treatment of CF, the potential benefit of our proprietary combination therapies to patients, expected completion of our clinical studies and cohorts for our clinical programs, initiation of a pivotal or registrational study, the possibility final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported by the Company (noting that these results are based on a small number of patients and small data set), uncertainties inherent in the execution and completion of clinical trials (including, without limitation, the possibility that FDA or other regulatory agency comments delay, change or do not permit trial commencement, or intended label, or the FDA or other regulatory agency requires us to run cohorts sequentially or conduct additional cohorts or pre-clinical or clinical studies), in the enrollment of CF patients in our clinical trials in a competitive clinical environment, in the timing of availability of trial data, in the results of the clinical trials, in possible adverse events from our trials, in the actions of regulatory agencies, in the endorsement, if any, by therapeutic development arms of CF patient advocacy groups (and the maintenance thereof). For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, unless required by law.

CONTACTS:

Investors:David Pitts / Claudia StyslingerArgot Partners212.600.1902david@argotpartners.com/ claudia@argotpartners.com

Media:David RosenArgot Partners212.600.1902david.rosen@argotpartners.com

HIT-CF Project Coordination:HIT-CF ConsortiumUniversity Medical Centre Utrecht (The Netherlands)HITCF@umcutrecht.nl

i Berkers et al, Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis Cell Reports 26, 17011708,February 12, 2019

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SOURCE Proteostasis Therapeutics, Inc.

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Proteostasis Therapeutics and CF Europe Announce Completion of Patient Enrollment for CHOICES, the First-Ever Personalized Medicine-Based Study in...