Kadimastem Finishes Treating Second Group of ALS Patients with AstroRx in Phase 1/2a Trial – ALS News Today

Kadimastem has finished treating its second group of participants in a Phase 1/2a clinical trial testing the safety and preliminary efficacy of AstroRx, aninvestigational stem cell therapy for amyotrophic lateral sclerosis (ALS).

AstroRx is an off-the-shelf cell therapy consisting of fully mature astrocytes star-shaped cells derived from human embryonic stem cells that are injected into the fluid surrounding the spinal cord to support damaged motor neurons.

Astrocytes usually help maintain a healthy environment in the brain, but often malfunction in ALS, contributing to disease progression. AstroRx was designed to compensate for diseased astrocytes and prevent the loss of motor nerve cells. The therapys goal is to potentially slow disease progression, improve quality of life, and extend life expectancy.

The U.S. Food and Drug Administration grantedAstroRx orphan drug status in November 2018 for the treatment of ALS, a designation meant to accelerate the development of AstroRx for this rare condition.

Earlier preclinical studiesshowed that AstroRx delayed disease onset, maintained muscle function, and prolonged survival in mice and rat models of ALS. The treatment also was found to be safe, with no signs of toxicity.

The ongoing, open-label, Phase 1/2a trial (NCT03482050) is testing the safety and preliminary signs of efficacy of AstroRx in people with ALS. Underway at the Hadassah Ein-Kerem Medical Center in Israel, the trial is expected to enroll 21 patients, ages 18-70, with early stage disease. Recruitment is ongoing.

The trial was originally designed to test three doses of AstroRx delivered into the spinal canal: a low dose of 100 million cells, a medium dose of 250 million cells, and a high dose with two consecutive administrations of 250 million cells.

However, after promising early efficacy results from the low-dose group (cohort A), Kadimastem is seeking to amend the trial protocol. It wants the third group of patients (cohort C) to receive two consecutive injections of the low dose, instead of the originally planned medium dose. Pending positive safety and efficacy results from the first three cohorts, a fourth group (cohort D) will receive two injections of the medium dose.

Safety and tolerability are the studys primary outcome measures. Secondary goals include changes in patients ALS Functional Rating Scale revised (ALSFRS-R) scores, respiratory muscle strength, hand grip strength, limb muscle strength, and quality of life.

In cohort A, the low-dose group, the therapy was found to be safe, with no serious side effects or dose-limiting toxicities. Participant had increased ALSFRS-R scores in the three months after treatment, suggesting a gain in functional abilities.

The trial has now treated all five ALS patients in cohort B, the second group, with the medium dose. Participants will be monitored for six months to evaluate the therapys safety and preliminary efficacy. The company expects to report the results for this group in August 2020.

Completing treatment for the additional 5 ALS patients in Cohort B, for a total of 10 patients treated with our product in our clinical trial, serves as an additional demonstration of our ability to develop and produce high quality clinical grade cells and takes us a significant step forward in our path to bringing innovative cure to ALS, Rami Epstein, CEO of Kadimastem, said in a press release.

The expected completion of cohort B 6-months follow-up period will allow us to assess the safety and preliminary efficacy of [250 million]cells, compared to that of the lower dose administered in cohort A, he added.

Kadimastem has recruited the first patient of cohort C, who all will receive two AstroRx injections of 100 million cells, separated by 2-3 months. Results from this group are expected during the first half of 2021.

The results of the next treatment group, Cohort C, in which each patient will be treated with two consecutive injections separated by an interval of 2-3 months, will allow us to assess the possible prolonged efficacy of the repeated dose, compared to the single dose treatment provided in cohorts A and B, said Michel Revel, founder and chief scientific officer of Kadimastem.

The results that will be obtained from the different cohorts, will support us in the process of defining the dose and treatment regimen that will lead to most favorable results for patients over time, Revel said.

Alejandra has a PhD in Genetics from So Paulo State University (UNESP) and is currently working as a scientific writer, editor, and translator. As a writer for BioNews, she is fulfilling her passion for making scientific data easily available and understandable to the general public. Aside from her work with BioNews, she also works as a language editor for non-English speaking authors and is an author of science books for kids.

Total Posts: 6

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.

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Kadimastem Finishes Treating Second Group of ALS Patients with AstroRx in Phase 1/2a Trial - ALS News Today

Global Stem Cell Therapies Overview Report 2019: The Highs & Lows, Off-The-Shelf Solutions, New Technologies, and Safety, Efficacy & Logistical…

DUBLIN, Jan. 8, 2020 /PRNewswire/ -- The "Highs and Lows of Stem Cell Therapies: Off-The-Shelf Solutions" report has been added to ResearchAndMarkets.com's offering.

The report includes:

Key Topics Covered

Chapter 1 Sources and Characteristics of Stem Cells

Chapter 2 New Technologies Driving Stem Cell Development

Chapter 3 Safety, Efficacy and Logistical Challenges

Chapter 4 Off-the-Shelf Solutions

Chapter 5 Companies and Technologies to Watch

Chapter 6 References

List of TablesTable 1: Stem Cell SourcesTable 2: Comparison of Stem CellsTable 3: Stem Cell Types Versus Cell PropertiesTable 4: Recent Advances in Genome Modulation TechnologiesTable 5: FDA-Approved Cell-Based TherapiesTable 6: Acute and Delayed Adverse Events Associated with Proven and Unproven Stem Cell-Based InterventionsTable 7: Companies Leading the Field in Cell-Based Therapies

List of FiguresFigure 1: Stem Cell Sources Figure 2: Two-Dimensional Versus Three-Dimensional Cell CultureFigure 3: Schematic of Approaches Used for Three-Dimensional Stem Cell Culture Figure 4: Patient Management Strategies to Decrease Side Effects Related with CAR-T Cell Therapy

For more information about this report visit https://www.researchandmarkets.com/r/15o8p9

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million – Business Wire

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, today announced the expansion of its Series A financing with an additional investment of $14.1 million led by Roche Venture Fund and with participation from other investors, bringing the total company financing to more than $50 million to date. The initial Series A round was led by Abingworth LLP and Qiming Venture Partners USA, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC.

Jasper plans to use the proceeds to advance and expand the study of its lead clinical asset, JSP191. A humanized antibody targeting CD117 on hematopoietic stem cells, JSP191 is designed to replace toxic chemotherapy and radiation therapy as conditioning regimens to prepare patients for curative stem cell and gene therapy. JSP191 is the only antibody of its kind in clinical development as a single conditioning agent for people undergoing curative hematopoietic cell transplantation.

This investigational agent is currently being evaluated in a Phase 1/2 dose-escalation and expansion study as a conditioning agent to enable stem cell engraftment in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that resulted in poor outcome. Initial positive results from this ongoing clinical trial were presented in an oral session at the American Society of Hematology (ASH) Annual Meeting in December 2019. Jasper plans to expand the Phase 1/2 clinical study to include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) receiving hematopoietic cell transplant. The development of JSP191 is supported by a collaboration with the California Institute for Regenerative Medicine (CIRM).

About Hematopoietic Cell TransplantationBlood-forming, or hematopoietic, stem cells are rare cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow by transplantation and engraftment of healthy stem cells is the only cure for most of these life-threatening conditions. Successful transplantation is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy followed by transplantation of a donor or gene-corrected hematopoietic cell graft. These toxic regimens cause DNA damage and lead to short- and long-term toxicities, including unwanted damage to organs and prolonged hospitalization. As a result, many patients who could benefit from a hematopoietic cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191JSP191 (formerly AMG 191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant. IND-enabling studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About Jasper TherapeuticsJasper Therapeutics is a biotechnology company focused on hematopoietic cell transplant therapies. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at https://jaspertherapeutics.com.

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Jasper Therapeutics Announces Expansion of Series A Financing, Bringing Total Corporate Fundraising to More than $50 Million - Business Wire

Q&A: Cancer Death Rates Are Falling Nationally. Here’s What’s Happening at UVA – University of Virginia

This week, the American Cancer Society released some very welcome news: the cancer death rate in the U.S. dropped by 2.2% from 2016 to 2017, the largest single-year drop ever recorded.

The drop, which the report attributes to plummeting smoking rates as well as new screening and treatment methods, continues a decades-long trend, as cancer death rates have fallen by nearly 30% since 1991 about 2.9 million fewer deaths.

Dr. Thomas Loughran, director of the University of Virginia Cancer Center, said UVA is in step with this national trend.

The UVA Cancer Center is one of 71 National Cancer Institute-designated treatment centers nationwide and ranked among the nations top 50 cancer centers over each of the past four years (No. 26 last year). The center serves approximately 4 million people in Virginia and West Virginia.

We spoke with Loughran about what he is seeing at UVA and beyond, new treatments and research helping to eradicate cancer, and where he sees cancer treatment in five years.

Q. Why have cancer death rates dropped so significantly?

A. As reports of this latest drop have said, a large part of the decline can be attributed to declining rates of lung cancer. The importance of preventing cancer particularly behavioral interventions like stopping smoking has become more prominent, and there have been remarkable declines in smoking across the United States.

This is a very important focus for us at UVA. We serve a large geographical area 90 contiguous counties in Virginia and West Virginia, including rural Appalachia. Southwest Virginia in Appalachia still has high smoking rates, and as a result, high rates of lung cancer. Education, screening and tobacco cessation programs are critically important, especially in those areas.

Q. What advances in treatment have contributed to falling cancer death rates, nationally and at UVA?

A. Screening technology, especially for the more common cancers like lung, colorectal, prostate and breast cancer, has improved. The latest report probably doesnt fully reflect recent implementation of lung cancer screening using a low-dose CT scan, recommended for high risk individuals and especially those with a history of heavy smoking. That has only been around a few years, and its impact will likely show up in future reports.

The second big factor is the development of immunotherapy [cancer treatments that utilize and help the patients immune system]. UVA has invested quite a lot of institutional resources in becoming a state-of-the-art immunotherapy center, and I am proud to say we are a leader in the field.

We have created a Cancer Therapeutics Program to support the development of new therapies. Dr. Craig Slingluff, who leads that program, is a surgical oncologist internationally famous for immunotherapy treatments for melanoma. To strengthen this program, we have recruited a cadre of leading physician scientists from across the country. Dr. Karen Ballen came here to lead our stem cell and bone marrow transplant program. Dr. Lawrence Lum, the scientific director of the transplant program, has developed a novel therapy using antibodies that bind to both T-cells [patient cells that can kill cancer cells] and tumor cells, forming a bridge between the two that helps the T-cells kill the cancer cells. Dr. Trey Lee is a leader in CAR-T cell therapy.

I could keep going; there are so many great people working on this. We also have a new Good Manufacturing Practice lab, supported by a grant from the commonwealth, that will help us grow and modify T-cells as needed and give them to patients under sterile conditions. That just opened and we are very excited about that program.

Q. What other areas of research have shown great promise?

A. Some of our work in nanotechnology is really unique and exciting. [Biomedical engineering professor] Mark Kester directs UVAs nanoSTAR Institute, which is working on delivering cancer therapies by nanotechnology basically, engineering at a very small scale. For example, nanoliposomes a sort of delivery system for cancer therapy are actually smaller than individual cells and can therefore penetrate cancer cells and release treatment from inside those cells.

We are very excited about early phase trials testing this technology on solid tumors, and we also hope to use it to treat patients with acute leukemia over the next few years.

Q. Looking ahead, where do you see the next big gains coming from?

A. Immunotherapy has revolutionized cancer treatment, but why some patients respond well and some dont remains puzzling. I hope that we can begin to discover why some patients are reacting to these newer treatments differently than others. Once we figure out why some patients respond to immunotherapy, we can begin to make improvements that could benefit a larger percentage of patients with these deadly cancers.

CAR T Cell therapy one method of immunotherapy is very effective against leukemia, lymphoma and cancers of the blood, but not yet against solid tumors. Over the next five years, I hope we can determine how to deliver these T-cells to solid tumors such as those found in lung, colorectal and other common cancers again to make this advance more widely applicable to a larger number of patients.

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Q&A: Cancer Death Rates Are Falling Nationally. Here's What's Happening at UVA - University of Virginia

SVC112 molecule reduces effects of cancer stem cells with little toxicity – Drug Target Review

A study has shown that the SVC112 molecule can prevent cancer stem cells from producing more tumour cells in cell lines and mice, with no impact on healthy cells.

Researchers have found that SVC112, a molecule based on the chemical bouvardin, can act specifically against head and neck cancer stem cells (CSCs).

Bouvardin can be extracted from the Bouvardia ternifolia bush, native to Central America. Research from the University of Colorado, US has previously shown that bouvardin can slow a cancers ability to make proteins which allow it to grow and metastasise.

As CSCs manufacture cells that become the bulk of cancer tissue, they are a viable drug target. However, they often resist treatments like radiation and chemotherapy.

when the population of stem cells was reduced to under one percent of the tumour cells, the tumour began to shrink

The current study, conducted by scientists at SuviCa, synthesised SVC112 in order to control tumours more effectively than current FDA-approved protein synthesis inhibitors and with less toxicity.

The groups work showed that SVC112 acts specifically against proteins like Myc and Sox2 which are required by CSCs, while leaving healthy cells unharmed. They demonstrated this by comparing the effects of the drug in matched pairs of cancer cells and healthy cells grown from samples donated by five head and neck cancer patients.

Many groups have linked the production of transcription factors to the survival and growth of CSCs, but inhibitors have just been too toxic they come with too many side effects. Definitely our studies suggest that this drug could be an advantage over existing drugs. It inhibits protein synthesis in a way that no other drug does and thats why were excited, says lead researcher Dr Tin Tin Su.

For further comparison, the researchers conducted the same experiment with the FDA-approved protein synthesis inhibitor omacetaxine mepesuccinate (also called homoharringtonin, or HHT).

Having cancer cells along with matched non-cancer cells from the same patient is pretty unique. When we tested these matched pairs with SVC112 and with HHT, what we saw is the approved drug eliminated both cancer and normal cells, whereas SVC112 had selectivity it affected cancer cells but not healthy cells so theoretically the effects on the normal tissue will be less, Su says.

Next, the team used SVC112 to treat head and neck tumours in mouse models, grown from samples of human tumours. Earlier work showed that SVC112 sensitised previously radiation-resistant CSCs to radiation treatment, so the group tested the molecule and radiation alone and in combination.

They found that when the population of stem cells was reduced to under one percent of the tumour cells, the tumour began to shrink. They concluded that the CSCs act as controllers for the growth of tumours, so impairing this group of stem cells will slow down or stop cancer.

The researchers hope that their pre-clinical results will lay the groundwork for human clinical trials of SVC112 in head and neck cancer patients.

The findings were published in Cancer Research.

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SVC112 molecule reduces effects of cancer stem cells with little toxicity - Drug Target Review