A Phase 3 clinical trial is recruiting adults with amyotrophic lateral sclerosis(ALS) to evaluate the safety and effectiveness of NeuroNata-R (lenzumestrocel), a stem cell therapy approved to treat ALS in South Korea.
Enrollment is currently ongoing in South Korea, with additional sites expected to open in the U.S.,Corestem, the therapys developer, stated in an email to ALS News Today.
With U.S. Food and Drug Administration (FDA) approval, one patient was treated with NeuroNata-R atMassachusetts General Hospital in November 2020 under an expanded access program, the company also stated. Such programs make investigational therapies available to people with serious conditions and few or no treatments, when the therapys benefits are thought to outweigh potential risks.
ALS is a progressive neurodegenerative disordercausedby the gradual loss of motor neurons the specialized nerve cells responsible for controlling voluntary movements in the spinal cord and brain, leading to muscle weakness and shrinkage.
Stem cell therapy is emerging as a potential way of treating ALS. Taking advantage of stem cells unique ability to give rise to many types of specialized cells, the approach is expected to help regenerate damaged or dying neurons and key cells that support them.
NeuroNata-R, Corestems lead product, makes use of a patients own mesenchymal stem cells (MSCs), which are capable of maturing into many other cell types that may help to promote nerve cell repair.
The treatment involves isolating MSCs from a persons bone marrow, growing them in the lab, and returning them to the patient, mixed with their cerebrospinal fluid (CSF), via an injection directly into the spinal canal. CSF is the liquid that surrounds the brain and spinal cord.
The first NeuroNata-R injection typically takes place one month after bone marrow extraction, followed by a second injection about a month later.
Prior studies highlighted the immunosuppressive, anti-inflammatory, and neuroprotective properties of NeuroNata-R. As such, the therapy is expected to promote motor neuron growth and prevent the death of these cells, slowing ALS progression.
The therapy was previously tested as an add-on to riluzole (marketed asRilutek, Tiglutik, and Exservan) in a Phase 1/2 clinical trial (NCT01363401) conducted in South Korea.
Results showed that NeuroNata-R, given as two intrathecal (into the spinal canal) injections, safely and effectively slowed disease progression as assessed with the ALS Functional Rating Scale(ALSFRS-R) in 32 ALS patients for at least six months.
These findings supported the conditional approval of NeuroNata-R in South Korea in 2014 as the first stem cell therapy for ALS. Its continued use, and full approval, requires further verification and description of clinical benefit in a larger, longer-term confirmatory study.
It was designated an orphan drug as a potential ALS treatment in the U.S. in 2018, followed by a similar designation in Europe about six months later. Both designations are meant to accelerate its development and review.
Corestem also reports that the FDA has approved its request to open a Phase 3 clinical trial of NeuroNata-R in the U.S.
This study, called ALSummit (NCT04745299) and already underway in Korea, is designed to evaluate the long-term safety and effectiveness of NeuroNata-R in up to 115 adults, ages 2575, diagnosed with ALS within the previous two years.
Participants will be randomly assigned to either NeuroNata-R followed or not by three injections, given three months apart or to a placebo injection (CSF without stem cells). All enrolled will also receive riluzole.
The trials main goal is to assess changes in a combined measure of function, based on the ALSFRS-R score, and survival benefits. Secondary goals include other measures of function and quality of life.
These assessments will be conducted at six months for patients receiving one treatment cycle, and at one year for those given three additional injections.
Eligible patients may also be asked to enter a supplementary study that will continue to week 48 (nearly a year), and long-term efficacy and safety will be evaluated during an observation period of 36 months from first NeuroNata-R injection.
According to Corestem,more than 300 ALS patients have been treated with NeuroNata-R in South Korea as of December, including at least 49 people who traveled from their home countries for the treatment.
Not many patients are aware of the existence of Neuronata-R, so we are eager to let the community know its existence, Eunsan Cho,the assistant manager of Corestems marketing team, said in the email.
Marta Figueiredo holds a masters in evolutionary and developmental biology and a PhD in biomedical sciences from the University of Lisbon, Portugal. Her research is focused on the role of several signaling pathways in thymus and parathyroid glands embryonic development.
Total Posts: 45
Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
Visit link:
Phase 3 Trial of Stem Cell Therapy NeuroNata-R Likely to Expand to US - ALS News Today
- Signal of Benefit for Stem Cell Therapy in Progressive MS - Medscape - March 7th, 2024
- The Controversies Surrounding Stem Cell Therapy for Autism - The Portugal News - February 24th, 2024
- Benefits of Stem Cell Therapy - News Channel 5 Nashville - February 7th, 2024
- What is Stem Cell Therapy & How It Helps Others - Publicist Paper - January 31st, 2024
- A guide to stem cell therapy in Thailand - Thaiger - January 4th, 2024
- Half of pediatric patients with aHUS benefit from Soliris after... - AHUS News - December 21st, 2023
- Real world analysis on the determinants of survival in primary ... - Nature.com - December 5th, 2023
- The Best Beauty Gifts According To People Who Really Know Skin Care - HuffPost - December 5th, 2023
- The Eyepopping Factory Construction Boom in the US - WOLF STREET - December 3rd, 2023
- Benefit of Neoadjuvant Therapy Illustrated During ESMO Congress ... - Targeted Oncology - December 1st, 2023
- Benefits Outweigh Risks as FDA Inspects CAR-T Cell Therapy ... - Curetoday.com - December 1st, 2023
- Review What Real Cavityn Customers Say About Benefits and Side ... - Seattle Weekly - December 1st, 2023
- 'There is a Scientific Fraud Epidemic' - Slashdot - Slashdot - December 1st, 2023
- 15 Best Hydrating Serums to Soothe Any Skin Type 2023 - Town & Country - December 1st, 2023
- Innovations in Cosmetic Dermatology: A Glimpse into the Future - APN News - December 1st, 2023
- FDA Probes New Cases of Cancer That May Stem From Cancer Cell ... - MedCity News - November 29th, 2023
- Use of plant stem cells in topical formulations on the rise - CosmeticsDesign.com USA - November 29th, 2023
- Stem cell-based treatment controls blood sugar in people with Type ... - EurekAlert - November 29th, 2023
- Biologics Market is projected to grow at a CAGR of 8.5% by 2034: Visiongain - Yahoo Finance - November 29th, 2023
- Scientists devise new technique that can pinpoint the causes and ... - EurekAlert - November 29th, 2023
- Global Advanced Therapy Medicinal Products Market is on the brink ... - PharmiWeb.com - November 29th, 2023
- Introducing Orgavalue - The 2023 EIT InnoStars Awards winner - EU-Startups - November 29th, 2023
- Episode 160: Euan Ashley discusses precision medicine and the ... - IHMC - November 29th, 2023
- Is Phenq Right For You? Review The Facts Before Making a ... - Vashon-Maury Island Beachcomber - November 27th, 2023
- Cavityn Reviews - Does It Work? Cheap Scam or Effective ... - The Daily World - November 25th, 2023
- Trend Hunting With Beautystreams At Cosmoprof Asia 2023 - BeautyMatter - November 25th, 2023
- FDA Considers First CRISPR Gene Editing Treatment That May ... - Slashdot - November 25th, 2023
- How Do Plants Determine Where the Light Is Coming From? - Lab Manager Magazine - November 25th, 2023
- Deaths From Coal Pollution Have Dropped, But Emissions May be ... - Slashdot - November 25th, 2023
- Stem cell-based therapies for stroke in newborns - Cochrane - November 23rd, 2023
- Dr Leslie on Outpatient CAR T-Cell Therapy Programs for ... - OncLive - November 23rd, 2023
- Human Milk-Based Therapy May Be Beneficial in Blood Cancers - Curetoday.com - November 23rd, 2023
- Oncological Horizons: The Synergy of Medical and Surgical ... - Cureus - November 23rd, 2023
- Human and environmental safety of carbon nanotubes across their ... - Nature.com - November 23rd, 2023
- Immix Biopharma to Host KOL Event to Discuss its BCMA-Targeted ... - BioSpace - November 23rd, 2023
- #HearTheMarsdenKids call on NHS London to reconsider cancer ... - Metro.co.uk - November 23rd, 2023
- Diagnosis and Management of Cardiovascular Risk in Patients with ... - Dove Medical Press - November 23rd, 2023
- BioLineRx Reports Third Quarter 2023 Financial Results and ... - PR Newswire - November 21st, 2023
- Why our Award Winners Beauty Box is the ultimate beauty gift (for ... - Yahoo Singapore News - November 21st, 2023
- SightCare Reviews - Is Official Website for Sight Care Legit to Try or ... - Vashon-Maury Island Beachcomber - November 21st, 2023
- Legend Biotech Reports Third Quarter 2023 Results and Recent ... - StreetInsider.com - November 21st, 2023
- Lorenzo's Oil: Can It Really Treat ALD? - Verywell Health - November 19th, 2023
- Doctor Says Living To 120 By End Of Decade Is Possible What's the Secret? - Benzinga - November 19th, 2023
- The week in pharma: action, reaction and insight week to ... - The Pharma Letter - November 19th, 2023
- Race Cannot Be Used To Predict Heart Disease, Scientists Say ... - Slashdot - November 19th, 2023
- SpaceX's Starship Reaches Outer Space Before Intentional ... - Slashdot - November 19th, 2023
- Mesenchymal Stem Cells Market is projected to reach USD 3,319 million by 2030 according to a new research r... - WhaTech Technology and Markets News - November 17th, 2023
- LMRUK: Protecting the next generation through cord blood - Charity Today News - November 17th, 2023
- Anti-aging molecule discovered that extends lifespan - Earth.com - November 17th, 2023
- Montana State researcher envisions living building materials that ... - Montana State University - November 17th, 2023
- OxyHelp Explores the Benefits of Hyperbaric Oxygen Therapy for ... - StreetInsider.com - November 15th, 2023
- Health benefits of bone marrow - Pakistan Observer - November 15th, 2023
- Evolving the standard of care: What is next for functional ... - NeuroNews International - November 15th, 2023
- Affimed Reports Third Quarter 2023 Financial Results and ... - BioSpace - November 15th, 2023
- Meet the World's First Whole-Eye Transplant Recipient - TIME - November 15th, 2023
- Calidi Biotherapeutics Reports Third Quarter 2023 Operating and ... - Business Wire - November 15th, 2023
- 10 Asia-Pacific biotech companies leading innovation in the region - Labiotech.eu - November 15th, 2023
- Fitspresso Reviews (November Update) Natural Ingredients That ... - Bainbridge Island Review - November 15th, 2023
- 2seventy bio Reports Third Quarter Financial Results and Recent ... - Business Wire - November 15th, 2023
- Japan To Create $6.6 Billion Fund To Develop Outer Space Industry ... - Slashdot - November 15th, 2023
- Be The Match, Healthcare Marketing Impact Awards 2023 - Ad Age - November 13th, 2023
- Medicare coverage saves lives. Enrolling shouldn't be this ... - Kevin MD - November 13th, 2023
- Future Ocean Foods: 36 Global Alt-Seafood Startups Form Association To Tackle Overfishing, Microplastics & More - Green Queen Media - November 13th, 2023
- Orchard Therapeutics Reports Third Quarter 2023 Financial Results ... - StreetInsider.com - November 13th, 2023
- Celebrity doctor reveals Hollywoods hidden trend of stem cell antiaging therapies - Longevity.Technology - November 11th, 2023
- Amazon Expands Healthcare Push With One Medical Benefits For ... - Slashdot - November 11th, 2023
- Bene Meat Technologies becomes the first to receive EU approval ... - Pet Food Processing - November 11th, 2023
- FDA Grants Orphan Drug Designation to MAIA Biotechnology for THIO as a Treatment for Glioblastoma - Yahoo Finance - November 11th, 2023
- MaaT Pharma Provides Third Quarter 2023 Business Update and ... - Business Wire - November 11th, 2023
- IN8bio Reports Third Quarter 2023 Financial Results and Provides ... - GlobeNewswire - November 11th, 2023
- Fast Lean Pro Reviews - Overpriced or Optimal Ingredients That ... - Journal of the San Juan Islands - November 11th, 2023
- Fate Therapeutics Reports Third Quarter 2023 Financial Results and ... - GlobeNewswire - November 11th, 2023
- Worlds first whole-eye and partial face transplant gives Arkansas man new hope - CNN - November 9th, 2023
- Glowing Fingertips And Green Eyes: First-of-Its-Kind Monkey ... - ScienceAlert - November 9th, 2023
- 10 Natural Body Scrubs That Exfoliate And Invigorate My Skin - PINKVILLA - November 9th, 2023
- CRISPR Therapeutics Provides Business Update and Reports Third ... - StreetInsider.com - November 9th, 2023
- Fate Therapeutics Reports Third Quarter 2023 Financial Results and ... - StreetInsider.com - November 9th, 2023
- Sight Care Reviews - Overpriced or Worth the Hype? SightCare Pills ... - Journal of the San Juan Islands - November 9th, 2023
- 6 Amazing Health Benefits Of Giloy (Tinospora cordifolia) - WION - November 9th, 2023
- The Global Market for Bispecific Antibody Therapeutics Contract Manufacturing 2023-2030: Driven by Demand for Innovative Therapies and Promising... - November 7th, 2023
Recent Comments