BrainStorm Cell Therapeutics has opened an expanded access program (EAP) in the U.S. to allow certain amyotrophic lateral sclerosis (ALS) patients to gain access to its investigational cell-based therapy NurOwn.
EAPs, also known as compassionate use programs, are intended to make investigational therapies available outside of a clinical trial to people whose serious or life-threatening conditions have few or no adequate treatments, when the therapys benefits are thought to outweigh potential risks.
Developed in partnership with the U.S. Food and Drug Administration (FDA), the program will allow clinicians to prescribe NurOwn, at no cost, to ALS patients who completed the therapys pivotal, placebo-controlled Phase 3 trial (NCT03280056) and who meet specific eligibility criteria.
Those with less advanced disease as measured by the ALS Functional Rating Scale(ALSFRS-R) will be the first to receive the treatment. This decision was based on the trials top-line data, which showed a superior treatment response in people in earlier stages of ALS.
Detailed, full data are expected to be presented at upcoming scientific conferences, and published in a peer-reviewed scientific journal.
We are pleased to have the opportunity to treat additional patients with NurOwn through this Expanded Access Program, which was strongly advocated for by members of the ALS advocacy community, Chaim Lebovits, BrainStorms CEO, said in a press release.
We recognize the urgency with which people with ALS want and need access to new potential treatments. This EAP is an important next step to providing an immediate option for some patients, Lebovits added.
Fred Fisher, president and CEO of The ALS Associations Golden West Chapter, said that enabling early access, while the data review continues, is an extraordinary gesture of support and compassion for those living with ALS, and reflects an understanding of the ALS communitys urgent need for an effective therapy. The Golden West Chapter represents the largest ALS community in the U.S.
I applaud Brainstorm for taking this important step, and I look forward to learning the results of their full data analysis, Fisher added.
Lebovits emphasized that BrainStorm remains committed to rapidly advancing NurOwn through clinical development and regulatory review in the hope that the greatest number of people living with ALS may benefit. The EAP will not interfere with data or regulatory timelines.
NurOwn involves collecting mesenchymal stem cells(MSCs) from a patientsown bone marrow, and expanding and maturating them into cells that produce high levels of neurotrophic factors molecules that promote nervous tissue growth and survival. MSCs are stem cells that can generate a variety of other cell types.
The mature cells called MSC-NTF cells are then injected into the patients spinal canal to promote and support nerve cell repair. Using a patients own cells minimizes the risk of an immune reaction, as might occur with cells from a donor.
NurOwn has been designated an orphan drug in both the U.S. and European Union, and given to fast track designation in the U.S.; all help to speed its clinical development and review.
Afterpromising Phase 2 resultsin people with fast-progressing ALS, BrainStorm launched a Phase 3 trial to confirm NurOwns benefits in a larger patient population.
The Phase 3 study evaluated the therapys safety and effectiveness in 189 people with rapidly progressing ALS, who wererandomly assigned to a total of three injections of either NurOwn or a placebo, given directly into the spinal canal, every other month.
Patients were recruited at six clinical sites in the U.S.: three in California, two in Massachusetts, and one in Minnesota. NurOwn will be available under the EAP at these six centers.
Top-line data showed that a greater proportion of NurOwn-treated patients (34.7%) had a slower disease progression as assessed with the ALSFRS-R compared with those given a placebo (27.7%).
However, this difference did not reach statistical significance. This was mainly due to unexpectedly good placebo group responses, exceeding those reported in other ALS trials, the company reported.
Also, no significant group differences were observed in ALSFRS-R score mean changes over the seven months of treatment (-5.52 in the NurOwn group vs. -5.88 in the placebo group), meaning that the trial failed to meet both its main and secondary effectiveness goals.
However, greater treatment responses were seen in a pre-specified group of participants with less advanced disease.
In this group, 34.6% of those given NurOwn showed a slower disease progression, compared with 15.6% of those in the placebo group. In addition, the mean decline in the ALSFRS-R total score was 1.77 with NurOwn and 3.78 with a placebo reflecting a 2.01-point improvement with the cell-based therapy.
Differences between these groups were also not statistically significant, but they were considered clinically meaningful. Based on these positive findings, BrainStorm is actively working with the FDA to identify regulatory pathways that may support NurOwn approval as an ALS treatment.
Biomarker analyses also confirmed that NurOwn was driving its intended biological effects. Its use significantly increased the levels of neurotrophic factors, and dropped those of neurodegenerative and neuroinflammatory biomarkers, in patients cerebrospinal fluid a finding not observed among placebo patients. (The cerebrospinal fluid is the liquid that surrounds the brain and spinal cord.)
This expanded access program is an appropriate and welcome next step in following up the exciting results of the Phase 3 study; it is widely anticipated and deeply appreciated by our ALS patients, said Robert Brown, MD, PhD, one of the trials principal investigators.
Brown is also the Leo P. and Theresa M. LaChance chair in medical research, and chair of the neurology department atUniversity of Massachusetts Medical Schooland UMass Memorial Medical Center.
NurOwn will initially be manufactured by the Dana Farber Cancer Institute, assisted by on-site BrainStorm personnel.
BrainStorm also is evaluating NurOwn as a potential therapy for other neurodegenerative diseases, such as multiple sclerosis, Parkinsons disease, Huntingtons disease, as well as for autism spectrum disorder.
Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling 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.
Read this article:
NurOwn May Be Given to Early ALS Patients in US Who Finished Phase... - ALS News Today
- Mass. Law About Stem Cell Research - Massachusetts Trial ... [Last Updated On: August 22nd, 2014] [Originally Added On: August 22nd, 2014]
- Theres a New Anti-ALS Association Ice Bucket Challenge [Last Updated On: August 23rd, 2014] [Originally Added On: August 23rd, 2014]
- Expert: Catholic objections wont leave ALS fundraiser all wet [Last Updated On: August 23rd, 2014] [Originally Added On: August 23rd, 2014]
- Gene expression patterns in pancreatic circulating tumor cells revealed [Last Updated On: September 24th, 2014] [Originally Added On: September 24th, 2014]
- Inner Cell Mass - Embryonic Development & Stem Cells ... [Last Updated On: September 27th, 2014] [Originally Added On: September 27th, 2014]
- Mass production of placenta stem cells gets FDA approval ... [Last Updated On: September 27th, 2014] [Originally Added On: September 27th, 2014]
- Siamab Therapeutics Appoints Robert Mashal to Board of Directors [Last Updated On: October 9th, 2014] [Originally Added On: October 9th, 2014]
- Brain Tumor Stem Cell Lab - Massachusetts General Hospital ... [Last Updated On: October 9th, 2014] [Originally Added On: October 9th, 2014]
- 107.23 /$ (5 p.m.) [Last Updated On: October 15th, 2014] [Originally Added On: October 15th, 2014]
- Stem Cell Eye Treatment May Restore Vision [Last Updated On: October 16th, 2014] [Originally Added On: October 16th, 2014]
- Scientists Engineer Cancer-Killing Stem Cells [Last Updated On: October 26th, 2014] [Originally Added On: October 26th, 2014]
- Stem Cell Therapy, Stem Cell Biology at Mass General Hospital [Last Updated On: October 31st, 2014] [Originally Added On: October 31st, 2014]
- Harvard researchers genetically 'edit' human blood stem cells [Last Updated On: November 6th, 2014] [Originally Added On: November 6th, 2014]
- Human blood stem cells genetically 'edited' [Last Updated On: November 7th, 2014] [Originally Added On: November 7th, 2014]
- Critical Mass of Stem Cells Sets Off Embryonic Development ... [Last Updated On: November 12th, 2014] [Originally Added On: November 12th, 2014]
- Reprogramming cells, long term [Last Updated On: November 18th, 2014] [Originally Added On: November 18th, 2014]
- Pain and itch neurons grown in a dish [Last Updated On: November 25th, 2014] [Originally Added On: November 25th, 2014]
- Blood test could pick up risk of cancer five years in advance, say Harvard scientists [Last Updated On: November 28th, 2014] [Originally Added On: November 28th, 2014]
- The obesity pill that could replace exercise by turning 'bad' fat to 'good' [Last Updated On: December 9th, 2014] [Originally Added On: December 9th, 2014]
- Stem cells: The black box of reprogramming [Last Updated On: December 11th, 2014] [Originally Added On: December 11th, 2014]
- Canadian scientists crack stem cell reprogramming code [Last Updated On: December 11th, 2014] [Originally Added On: December 11th, 2014]
- Proteins drive cancer cells to change states [Last Updated On: December 16th, 2014] [Originally Added On: December 16th, 2014]
- Massachusetts (Stem Cell) - what-when-how [Last Updated On: February 3rd, 2015] [Originally Added On: February 3rd, 2015]
- New Organ Liver Prize Gathers Momentum [Last Updated On: February 4th, 2015] [Originally Added On: February 4th, 2015]
- MIT researchers develop glucose-responsive diabetes treatment [Last Updated On: February 11th, 2015] [Originally Added On: February 11th, 2015]
- Scientists develop novel technique for finding drugs to combat malaria [Last Updated On: February 11th, 2015] [Originally Added On: February 11th, 2015]
- Could we soon have man-made blood? [Last Updated On: March 10th, 2015] [Originally Added On: March 10th, 2015]
- Protein that repels immune cells protects transplanted pancreatic islets from rejection [Last Updated On: March 10th, 2015] [Originally Added On: March 10th, 2015]
- Researchers identify a vital protein that can determine head and brain development [Last Updated On: March 13th, 2015] [Originally Added On: March 13th, 2015]
- Biomedical engineer developing nanomaterial for healing broken bones [Last Updated On: March 18th, 2015] [Originally Added On: March 18th, 2015]
- Scientists engineer toxin-secreting stem cells to treat ... [Last Updated On: October 19th, 2015] [Originally Added On: October 19th, 2015]
- Cloning/Embryonic Stem Cells - Genome.gov [Last Updated On: October 19th, 2015] [Originally Added On: October 19th, 2015]
- Stem Cell Facts - University of Massachusetts Medical School [Last Updated On: July 27th, 2016] [Originally Added On: July 27th, 2016]
- Stem Cell FAQ - Massachusetts General Hospital, Boston, MA [Last Updated On: July 27th, 2016] [Originally Added On: July 27th, 2016]
- Stem-cell-based therapy promising for treatment of breast ... [Last Updated On: September 26th, 2016] [Originally Added On: September 26th, 2016]
- What Are Stem Cells? - Massachusetts General Hospital ... [Last Updated On: November 22nd, 2016] [Originally Added On: November 22nd, 2016]
- New England Cord Blood Bank - Cord Blood and Cord Tissue ... [Last Updated On: November 22nd, 2016] [Originally Added On: November 22nd, 2016]
- Stem cell controversy - Wikipedia [Last Updated On: December 1st, 2016] [Originally Added On: December 1st, 2016]
- From Stem Cells to Human Development - September 2016 ... [Last Updated On: December 4th, 2016] [Originally Added On: December 4th, 2016]
- X4 joins hands with Yale on rare disease program - FierceBiotech [Last Updated On: August 6th, 2017] [Originally Added On: August 6th, 2017]
- TGF-1: ALS Astrocytes' Secret Sauce? - ALS Research Forum [Last Updated On: August 6th, 2017] [Originally Added On: August 6th, 2017]
- CRISPR fixes disease gene in viable human embryos - Nature.com [Last Updated On: August 6th, 2017] [Originally Added On: August 6th, 2017]
- Current humanized mice not good models for studying stem cell transplants, say researchers - Scope (blog) [Last Updated On: August 27th, 2017] [Originally Added On: August 27th, 2017]
- Mouse Model of Human Immune System Inadequate for Stem Cell ... - Technology Networks [Last Updated On: August 27th, 2017] [Originally Added On: August 27th, 2017]
- ORGANOID - Science Magazine [Last Updated On: August 27th, 2017] [Originally Added On: August 27th, 2017]
- Asymmetrex Introduces New Contract Service For Producing ... - PR Web (press release) [Last Updated On: August 29th, 2017] [Originally Added On: August 29th, 2017]
- For Lowell native, stem cell match becomes a match as friends - Lowell Sun [Last Updated On: September 2nd, 2017] [Originally Added On: September 2nd, 2017]
- Lymphoma Patient's Brain Tumor Disappeared After She Received JCAR017, Study Reports - Lymphoma News Today [Last Updated On: September 2nd, 2017] [Originally Added On: September 2nd, 2017]
- Doubts raised about CRISPR gene-editing study in human embryos - Nature.com [Last Updated On: September 2nd, 2017] [Originally Added On: September 2nd, 2017]
- Alternate Methods for Preparing Pluripotent Stem Cells ... [Last Updated On: September 25th, 2017] [Originally Added On: September 25th, 2017]
- Cloning/Embryonic Stem Cells - National Human Genome ... [Last Updated On: July 5th, 2018] [Originally Added On: July 5th, 2018]
- Boston, MA, Stem Cell Transplant, Weston, Nantucket ... [Last Updated On: January 10th, 2019] [Originally Added On: January 10th, 2019]
- Chronic variable stress activates hematopoietic stem cells ... [Last Updated On: April 12th, 2019] [Originally Added On: April 12th, 2019]
- Massachusetts Stem Cells | Stem Cell TV [Last Updated On: September 10th, 2019] [Originally Added On: September 10th, 2019]
- Comparison of Merus N.V. (MRUS) and Sage Therapeutics Inc. (NASDAQ:SAGE) - MS Wkly [Last Updated On: October 23rd, 2019] [Originally Added On: October 23rd, 2019]
- Reviewing Cellectis S.A. (CLLS)'s and Magenta Therapeutics Inc. (NASDAQ:MGTA)'s results - MS Wkly [Last Updated On: October 23rd, 2019] [Originally Added On: October 23rd, 2019]
- New study reveals why breast cancer spreads to the brain - USC News [Last Updated On: October 23rd, 2019] [Originally Added On: October 23rd, 2019]
- Arkuda bags $44M to target progranulin and head off inherited dementia - FierceBiotech [Last Updated On: November 9th, 2019] [Originally Added On: November 9th, 2019]
- New Podcast Sponsored by Asymmetrex Increases Awareness to the Need for Stem Cell Dose in Stem Cell Treatments - PR Web [Last Updated On: November 9th, 2019] [Originally Added On: November 9th, 2019]
- Exercise found to block chronic inflammation in mice - Harvard Gazette [Last Updated On: November 9th, 2019] [Originally Added On: November 9th, 2019]
- Top Emerging Technologies of the Year - Technowize [Last Updated On: December 29th, 2019] [Originally Added On: December 29th, 2019]
- Firm adds a new wrinkle to anti-aging products - Williamson Daily News [Last Updated On: January 5th, 2020] [Originally Added On: January 5th, 2020]
- Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 - BioSpace [Last Updated On: January 10th, 2020] [Originally Added On: January 10th, 2020]
- 10 Of The Biggest World Revelations In The 21st Century - World Atlas [Last Updated On: February 29th, 2020] [Originally Added On: February 29th, 2020]
- Infrared Laser Treatment of TBI, PTSD, and Depression: An Expert Perspective - Psychiatry Advisor [Last Updated On: April 3rd, 2020] [Originally Added On: April 3rd, 2020]
- Startup targets glioblastoma tumors with CAR-T therapy - FierceBiotech [Last Updated On: May 28th, 2020] [Originally Added On: May 28th, 2020]
- Massachusetts Eye and Ear Enters Licensing Agreement with Biogen to Develop Treatment for Inherited Retinal Disorder - Newswise [Last Updated On: July 2nd, 2020] [Originally Added On: July 2nd, 2020]
- Targeted Therapy for Non-Hodgkin Lymphoma: Current Progress and Future Plans - Cancer Therapy Advisor [Last Updated On: September 5th, 2020] [Originally Added On: September 5th, 2020]
- Be Biopharma debuts with $52M to advance engineered B-cell therapies - FierceBiotech [Last Updated On: October 24th, 2020] [Originally Added On: October 24th, 2020]
- Orchard Therapeutics Outlines Comprehensive Presence at 2021 WORLDSymposium - GlobeNewswire [Last Updated On: February 1st, 2021] [Originally Added On: February 1st, 2021]
- How Coronavirus Damages Lung Cells Within Mere Hours And What Drugs Could Halt COVID-19 Infection - SciTechDaily [Last Updated On: February 1st, 2021] [Originally Added On: February 1st, 2021]
- On systemic sources of early life stress, and empathetic responses - MIT News [Last Updated On: June 6th, 2021] [Originally Added On: June 6th, 2021]
- Asymmetrex Will Present a New Test for Therapeutic Stem Cell Potency at the ISSCR 2021 Annual Meeting - PRNewswire [Last Updated On: June 23rd, 2021] [Originally Added On: June 23rd, 2021]
- Infertility: Men account for at least half of cases. So why have women shouldered the blame? - The Irish Times [Last Updated On: June 23rd, 2021] [Originally Added On: June 23rd, 2021]
- cGVHD Paradigm Gains Systemic Options Beyond Steroids, But Real-World Data Are Required - OncLive [Last Updated On: October 28th, 2021] [Originally Added On: October 28th, 2021]
- Stem cells: Sources, types, and uses - Medical News Today [Last Updated On: December 24th, 2021] [Originally Added On: December 24th, 2021]
- Liso-Cel Outperforms Standard Therapy in Improving QoL in Relapsed/Refractory LBCL - www.oncnursingnews.com/ [Last Updated On: January 5th, 2022] [Originally Added On: January 5th, 2022]
- January 2022: 2021 Papers of the year - Environmental Factor Newsletter [Last Updated On: January 5th, 2022] [Originally Added On: January 5th, 2022]
- MorphoSys and Incyte Announce Swissmedic Temporary Approval of Minjuvi(R) (tafasitamab) in Combination with Lenalidomide for the Treatment of Adults... [Last Updated On: March 25th, 2022] [Originally Added On: March 25th, 2022]
- Takeda and the New York Academy of Sciences Announce 2022 Innovators in Science Award Winners - The New York Academy of Sciences [Last Updated On: April 6th, 2022] [Originally Added On: April 6th, 2022]