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BioRestorative Therapies in Substantive Discussions for Potential License Agreement for ThermoStem Metabolic … – GlobeNewswire
Posted: June 14, 2024 at 2:42 am
- Brown adipose derived stem cells with brown adipogenic potential would represent a new modality for the treatment of obesity and related metabolic disorders
- ThermoStem is an off-the-shelf platform, well-protected by a comprehensive portfolio of issued patents that cover both the U.S. and international markets
- Potential license partner is a leading commercial stage regenerative medicine company
MELVILLE, N.Y., June 13, 2024 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative, BRTX or the Company) (NASDAQ:BRTX), a clinical stage company focused on stem cell-based therapies, today reported that it has had substantive discussions with an undisclosed commercial stage regenerative medicine company with regard to a license of BioRestoratives allogeneic, off-the-shelf ThermoStem metabolic intellectual property.
Previously published peer-reviewed preclinical data from a study conducted in collaboration with the University of Utah School of Medicine demonstrated a clonogenic population of metabolically active brown adipose tissue (BAT) stem cells residing in adult humans that: (i) can be expanded in vitro; (ii) exhibit multilineage differentiation potential (osteogenetic, chondrogenic and adipogenic); and (iii), functionally differentiate into metabolically active brown adipocytes. In addition, the preclinical results confirmed that multipotent brown adipose derived stem cells (BADSCs) induced to differentiate into brown adipocytes exhibit the mature functional properties of these cells, including increased mitochondrial activity, an important functional characteristic of BAT.
BADSCs with brown adipogenic potential would represent an exciting new modality for the treatment of obesity and related metabolic disorders. To explore this possibility, BioRestorative developed a 3D Porous Extracellular Matrix-Derived Scaffold for effective cell delivery. Data obtained in high-fat fed NOD-SCID mice transplanted with differentiated BADSCs supported by the 3D scaffold showed significant reductions in weight, triglyceride and blood glucose levels compared to saline-only injected controls.
No assurances can be given that a license agreement will be entered into whether on commercially reasonable terms or otherwise. The Company does not intend to make any further announcements with regard to the license unless and until an agreement is entered into.
It has been captivating to watch the scale, scope and speed with which potential license partners have begun to show interest in our proprietary off-the-shelf ThermoStem platform, said Lance Alstodt, BioRestoratives Chief Executive Officer. We are particularly pleased that our pioneering animal studies, which demonstrated that BADSCs show promise in correcting the weight gain and hyperglycemia associated with high fat feeding, and that our 3D scaffolds, which are capable of retaining viable transplanted cells for at least five weeks post-implantation, have attracted the caliber of commercial stage regenerative medicine company with which we are now engaged in substantive licensing discussions.
About BioRestorative Therapies, Inc.
BioRestorative (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. As described below, our two core clinical development programs relate to the treatment of disc/spine disease and metabolic disorders, and we have also recently begun offering BioCosmeceutical products:
Disc/Spine Program (brtxDISC): Our lead cell therapy candidate,BRTX-100,is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. TheBRTX-100production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure,BRTX-100is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have commenced a Phase 2 clinical trial usingBRTX-100to treat chronic lower back pain arising from degenerative disc disease.
Metabolic Program (ThermoStem): We are developing cell-based therapy candidates to target obesity and metabolic disorders using brown adipose (fat) derived stem cells (BADSC) to generate brown adipose tissue (BAT), as well as exosomes secreted by BADSC. BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.BADSC secreted exosomes may also impact weight loss.
BioCosmeceuticals: We operate a commercial BioCosmeceutical platform. Our current commercial product, formulated and manufactured using our cGMP ISO-7 certified clean room, is a cell-based secretome containing exosomes, proteins and growth factors. This proprietary biologic serum has been specifically engineered by us to reduce the appearance of fine lines and wrinkles and bring forth other areas of cosmetic effectiveness. Moving forward, we also intend to explore the potential of expanding our commercial offering to include a broader family of cell-based biologic aesthetic products and therapeutics via Investigational New Drug (IND)-enabling studies, with the aim of pioneering U.S. Food and Drug Administration (FDA) approvals in the emerging BioCosmeceuticals space.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K, as amended, filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.
CONTACT: Email:ir@biorestorative.com
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BioRestorative Therapies in Substantive Discussions for Potential License Agreement for ThermoStem Metabolic ... - GlobeNewswire
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IV infusion enables editing of the cystic fibrosis gene in lung stem cells – Ars Technica
Posted: June 14, 2024 at 2:42 am
The development of gene editing tools, which enable the specific targeting and correction of mutations, hold the promise of allowing us to correct those mutations that cause genetic diseases. However, the technology has been around for a while nowtwo researchers were critical to its development in 2020and there have been only a few cases where gene editing has been used to target diseases.
One of the reasons for that is the challenge of targeting specific cells in a living organism. Many genetic diseases affect only a specific cell type, such as red blood cells in sickle cell anemia, or specific tissue. Ideally, to limit potential side effects, we'd like to ensure that enough of the editing takes place in the affected tissue to have an impact, while minimizing editing elsewhere to limit side effects. But our ability to do so has been limited. Plus, a lot of the cells affected by genetic diseases are mature and have stopped dividing. So, we either need to repeat the gene editing treatments indefinitely or find a way to target the stem cell population that produces the mature cells.
On Thursday, a US-based research team said that they've done gene editing experiments that targeted a high-profile genetic disease: cystic fibrosis. Their technique largely targets the tissue most affected by the disease (the lung), and occurs in the stem cell populations that produce mature lung cells, ensuring that the effect is stable.
The foundation of the new work is the technology that gets the mRNAs of the COVID-19 mRNA vaccines inside cells. The nucleic acids of an mRNA are large molecules with a lot of charged pieces, which makes it difficult for them to cross a membrane to get inside of a cell. To overcome that problem, the researchers package the mRNA inside a bubble of lipids, which can then fuse with cell membranes, dumping the mRNA inside the cell.
This process, as the researchers note, has two very large advantages: We know it works, and we know it's safe. "More than a billion doses of lipid nanoparticlemRNA COVID-19 vaccines have been administered intramuscularly worldwide," they write, "demonstrating high safety and efficacy sustained through repeatable dosing." (As an aside, it's interesting to contrast the research community's view of the mRNA vaccines to the conspiracies that circulate widely among the public.)
There's one big factor that doesn't matter for vaccine delivery but does matter for gene editing: They're not especially fussy about what cells they target for delivery. So, if you want to target something like blood stem cells, then you need to alter the lipid particles in some way to get them to preferentially target the cells of your choice.
There are a lot of ideas on how to do this, but the team behind this new work found a relatively simple one: changing the amount of positively charged lipids on the particle. In 2020, they published a paper in which they describe the development of selective organ targeting (SORT) lipid nanoparticles. By default, many of the lipid particles end up in the liver. But, as the fraction of positively charged lipids increases, the targeting shifts to the spleen and then to the lung.
So, presumably, because they know they can target the lung, they decide to use SORT particles to send a gene editing system specific to cystic fibrosis, which primarily affects that tissue, and is caused by mutations in a single gene. While it's relatively easy to get things into the lung, it's tough to get them to lung cells, given all the mucus, cilia, and immune cells that are meant to take care of foreign items in the lung.
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IV infusion enables editing of the cystic fibrosis gene in lung stem cells - Ars Technica
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Positive Psychology Intervention Aids Allogeneic Stem Cell Transplant Survivors – HealthDay
Posted: June 14, 2024 at 2:42 am
THURSDAY, June 13, 2024 (HealthDay News) -- A telephone-delivered positive psychology intervention (Positive Affect for the Transplantation of Hematopoietic stem cells intervention [PATH]) is beneficial for allogeneic hematopoietic stem cell transplantation (HSCT) survivors, according to a study published online June 11 in the Journal of the National Comprehensive Cancer Network.
Hermioni L. Amonoo, M.D., M.P.H., from the Dana-Farber Cancer Institute in Boston, and colleagues randomly assigned HSCT survivors who were 100 days post-HSCT for hematologic malignancy to either PATH or usual care. PATH entailed nine weekly phone sessions on gratitude, personal strengths, and meaning and was delivered by a behavioral health expert.
Seventy-two of 105 eligible patients were enrolled. The researchers found that 91 percent of those randomly assigned to PATH completed all sessions and reported that the positive psychology exercises were easy to complete and subjectively useful. PATH participants reported greater improvements in gratitude, anxiety, and physical function compared with usual care at nine weeks and in gratitude, positive affect, life satisfaction, optimism, anxiety, depression, posttraumatic stress disorder, quality of life, physical functioning, and fatigue at 18 weeks.
"Cancer care providers should consider the potential benefits of psychosocial resources and interventions like PATH that focus on enriching positive emotions to bolster their patients' well-being," Amonoo said in a statement. "Encouraging patients to engage in simple, structured, and systematic exercises aimed at fostering positive thoughts and emotions, such as gratitude, has the potential to enhance well-being as well."
One author disclosed ties to the biopharmaceutical industry.
Abstract/Full Text
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School of Dentistry to launch new Center for Regenerative Sciences – UT Health San Antonio
Posted: June 14, 2024 at 2:42 am
The University of Texas Health Science Center at San Antonio School of Dentistry is preparing to launch its Center for Regenerative Sciences, a new research initiative that aims to position the university at the forefront of regenerative dentistry and medicine.
The center will provide new avenues for interdisciplinary collaborations to accelerate the translation of preclinical discoveries into therapeutic benefit for patients suffering from dental, oral and craniofacial diseases, said Yong-Hee Chun, DDS, PhD, MS, associate professor of periodontics.
Chuns research focuses on understanding the molecules essential for tooth development and mineralization. When teeth and their support structures are malformed, such as with molar-incisor hypomineralization, or are damaged by diseases like periodontal disease, new treatments are needed. These treatments aim to regenerate the affected tissues and restore normal function, she said.
Anibal Diogenes, DDS, PhD, professor and chair of the Department of Endodontics, also highlighted the centers potential.
We have the capability to advance the field rapidly due to our ability to translate biological advancements into clinical applications faster than many other medical fields, he said. This center represents a huge opportunity to bootstrap the field, diversify our research and enhance collaboration among our scientists.
The Center for Regenerative Sciences will focus on several key areas, including the development of advanced tissue regeneration strategies, 3D printing of tissues and acellular scaffolds, the encapsulation of growth factors, morphogens and chemotactic molecules for targeted drug delivery.
Our goal is to create an environment within the body where biological cues guide cells to achieve their full potential, Diogenes said. By integrating basic sciences with clinical applications, we can bridge gaps and push the field of regenerative dentistry forward.
The center intends to accomplish this by leveraging and expanding the schools existing strengths in regenerative endodontics, dental and craniofacial development and the advancement of reliable sources of potent stem cells.
Diogenes research, for example, includes work on stem cell-based root canal therapies for children.
When a childs dental pulp is damaged or infected, we can activate their own stem cells to reestablish the pulp and allow the tooth to continue developing, which is crucial for their overall growth and development, he noted. This has significant implications not only for a childs dental health but also for their nutrition, speech development and psychosocial well-being.
Brij B. Singh, PhD, associate dean of research at the school, spoke to the broader impact of the new center.
This initiative will not only enhance our research capabilities but will attract top talent from various fields, creating a collaborative environment that can lead to breakthroughs in both dental and medical sciences, he said. The vision is to become a global leader in regenerative sciences, ultimately improving patient care outcomes everywhere.
The body has amazing regenerative capacity and the oral cavity is very special, Diogenes said. Its very rich in stem cells that are extremely potent with a high regenerative capacity. They are also unique because oral stem cells come from an origin where they can even become neurons. This means they have the potential to be used as therapies in many neurodegenerative diseases such as Alzheimers or Parkinsons therapies and for spinal cord injuries. There is so much potential there that extends far beyond the oral and craniofacial structures.
The Center for Regenerative Sciences will be formally launched by the school later this fall. It will be one of three centers of dental research, joining the Center for Global and Community Oral Health and the Center for Pain Therapeutics and Addiction Research. Recruitment for the centers director is currently underway.
For more information, visit the School of Dentistrys website.
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Human adipose-derived stem cells genetically programmed to induce necroptosis for cancer immunotherapy | Cancer … – Nature.com
Posted: June 14, 2024 at 2:42 am
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Human adipose-derived stem cells genetically programmed to induce necroptosis for cancer immunotherapy | Cancer ... - Nature.com
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Efficient bone marrow irradiation and low uptake by non-haematological organs with an yttrium-90-anti-CD66 antibody … – Nature.com
Posted: June 14, 2024 at 2:42 am
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Efficient bone marrow irradiation and low uptake by non-haematological organs with an yttrium-90-anti-CD66 antibody ... - Nature.com
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A cell therapy to heal a broken heart – Drug Discovery News
Posted: June 14, 2024 at 2:42 am
For many people, surviving a heart attack is just the beginning. Within minutes after one or more areas of the heart stop receiving oxygen, cardiac muscle cells begin to die. Given the limited regeneration potential of the heart, its response to this destruction is to replace the lost cells with scar tissue.
Matthieu de Kalbermatten, CEO of CellProthera, said that their product, ProtheraCytes, mimics one of the natural responses of the body after heart attack by mobilizing progenitor blood CD34+ stem cells towards the cardiac tissue.
Credit: Studio Chlorophylle
This scar is just there to save the patient, said Matthieu de Kalbermatten, chief executive officer of the biotechnological company CellProthera. The heart wont pump blood as efficiently as before, and if the damage is severe, it can result in chronic heart failure. The [organ] becomes weaker and weaker, [leading to] a high mortality after three to five years, plus bad quality of life, he explained.
Drugs and therapies prescribed after a heart attack may improve patient survival rates, but they do not repair the injured cardiac tissue, said de Kalbermatten. His team at CellProthera aims to prevent this long-term damage by injecting patients with their own, lab-expanded, stem cells.
The promise of this cell therapy, called ProtheraCytes, is to intervene early within a month after the heart attack and inject these cells in the hope that they will help regenerate the tissue, reducing the scar area and regaining partial heart function.
The researchers at CellProthera focus their efforts on the regenerative potential of CD34+ stem cells, which give rise to all types of blood cells in the body as well as the endothelial cells that line the insides of blood vessels. Since the early 2000s, studies have shown that CD34+ cells mobilize from the bone marrow into peripheral blood circulation shortly after a heart attack (1,2). These observations suggest that the human body naturally calls for these cells to come and help after such an event, but de Kalbermatten hypothesized that the migration might not be sufficient to heal after a severe heart attack. With Protheracytes, he said, We are trying to mimic [this] natural phenomenon, but just making it bigger and stronger.
To achieve this goal, the team first obtains CD34+ cells from the patient a few weeks to a month after the heart attack. After administering a growth factor to the patient to stimulate the bone marrows production of these cells, doctors take a blood draw from the patient and isolate the CD34+ cells. The team use their own cell expansion protocol and technology for in vitro proliferation to increase the number of these cells. Finally, nine days after the blood draw, there is a CD34+ suspension ready to be injected back into the patient, de Kalbermatten said. The cells are maintained fresh during that period. He noted, We dont freeze them. Keeping the cells fresh allows for higher cell viability and potency, he explained.
A doctor then injects the stem cell suspension via a catheter directly into the left ventricle muscle wall of the patient. CellProthera partnered with the biotech company BioCardia, which designed a specialized catheter known as the Helix Transendocardial Biotherapeutic Delivery System. The goal was to deliver therapeutic agents cells, genes, or proteins directly into the heart muscle to offer better results than injecting them into the coronary arteries, while also avoiding cardiac surgery, explained Peter Altman, chief executive officer of BioCardia.
Injecting them into the myocardium as opposed to just sending them down the capillaries [might be] better, concurred Robb MacLellan, a practicing cardiologist and physician scientist studying regenerative therapies at the University of Washington who is not associated with CellProthera or BioCardia. With gene therapy, doing that leads to better delivery amounts.
Using a patients own cells for transplant comes with advantages and disadvantages. The alternative option, an allogeneic transplant, might be more efficient since the production of cells does not rely on the patient, and cell quantities may be less limited. Yet, using foreign cells poses rejection risks.
We are trying to mimic [this] natural phenomenon, but just making it bigger and stronger. - Matthieu de Kalbermatten, CellProthera
Autologous transplantation, on the other hand, is very safe, de Kalbermatten said. Since cells are from the patient, rejection is unlikely, and there is no need for immunosuppressive drugs. However, using the patients own cells has other requirements, such as a well-designed logistic bench-to-bedside process. We have developed a technology that is totally automated, he said. You take a kit; you take the blood; you put it in the machine; you get a product. That standardization also reduces costs, he added.
The benefits from the therapy do not rely on the stem cells differentiating into cardiomyocytes, but the secretion of factors makes the difference. The release of these factors may modulate endogenous repair processes (3). Its the beauty about the cell as a drug, because the cell is a small factory that is able to react to the environment, de Kalbermatten said.
This idea that cells can impact scar formation and scar resolution has been around for decades ... in cardiology, said MacLellan. Yet, he noted that while researchers have tried to use cell therapies to modulate the healing process post injury in the heart and other organs, none of them have translated into standard of care.
Translating preclinical studies of stem cell therapies to successful clinical trials to treat acute myocardial infarction has proved challenging. One reason for this is the lack of rigor and standardized protocols in many preclinical studies (4).
The various drugs beta blockers, angiotensin-converting enzyme (ACE) inhibitors, aspirin administered to patients after a heart attack may also account for this difference, said MacLellan. If you get on that cocktail of medicines, your prognosis is then very good, he said. That has really frustrated these cell therapy trials, he added. [Most] preclinical trials never use the same medication background that we use in patients. Researchers need to prove that cell therapies add to these existing therapies, and thats a high bar, he added.
Differences in the delivery methods between animal and human cell therapy protocols may also explain the inconsistencies between preclinical and clinical outcomes for acute myocardial infarction. Researchers often deliver the cells surgically into the heart muscle in small animal models, while for humans, they mostly use catheters that go into the coronary arteries. Using the BioCardia Helix catheter may help bring cell therapies in humans closer to achieving the positive results reported in preclinical studies, according to Altman.
Once the stem cell suspension is ready, scientists at CellProthera ship it from the manufacturing site to the clinical site where doctors prepare the patient for the cell injection.
Credit: CellProthera
In addition to the delivery system, MacLellan acknowledged that ProtheraCytes has two more primary differences that stand out from what researchers have previously done, namely, the process for obtaining and expanding the CD34+ cells and the timing of the infusion.
CellProthera is currently conducting a clinical trial to reveal whether these variations in their protocol result in more successful clinical translation than previous attempts. Already in the 2000s, the founder of CellProthera, Philippe Henon, led a pilot study on seven patients who had suffered a severe heart attack. That first trial was nonrandomized, and the surgeons injected the cells directly into the cardiac tissue by open heart surgery, explained de Kalbermatten. The outcome for six of the patients was promising. Thats why we decided to start this adventure.
Now, the teams Phase 1/2b randomized clinical trial evaluates the safety and efficacy of their therapy in 33 patients. For assessing the efficacy, they use primarily magnetic resonance imaging (MRI). This is the most precise imaging system that you can have these days, said de Kalbermatten. They compare, for instance, visible damage after the heart attack versus six months after injection of ProtheraCytes. The aim is to determine whether the therapy reduces the area in the heart that became nonviable after the heart attack. The interim data based on this parameter is already very compelling, said de Kalbermatten. The team also measures other markers that are predictive of the future outcome of the disease, he added.
Completing this assessment will provide enough information to potentially advance to the next stage and design a Phase 3 trial. In this study, they plan to assess survival rate and hospitalization for worsening heart failure.
There is a lot of history to overcome in this field, MacLellan said, but he is optimistic about the future. The scientific community may be emerging from the period of disappointment regarding cell therapies, he suggested, and well-designed randomized controlled trials will add important information about their value.
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Q&A: New Frontiers in ALS Research – Newswise
Posted: June 14, 2024 at 2:42 am
Newswise LOS ANGELES (June 12, 2024) --Clive Svendsen, PhD, executive director of theBoard of Governors Regenerative Medicine Instituteand professor of Medicine and Biomedical Sciences at Cedars-Sinai, is developing new treatments and models for amyotrophic lateral sclerosis (ALS) using stem cells.
ALS progressively destroys nerve cells in the brain and spinal cord, causing people to lose muscle movement required for walking, talking, swallowing and breathing. Currently, the disease is irreversible and has no cure, but a potential therapy that combines gene and stem cell technologies is progressing well, according to Svendsen.
Svendsen has been selected as an inaugural recipient of a grant from the Tambourine ALS Breakthrough Research Fund, funded by Tambourine and administered in partnership with the Milken Institute. The grant will fund research that combines advanced stem cell modeling with artificial intelligence to learn more about the cause of ALS. Svendsen sat down with theCedars-Sinai Newsroomto discuss his ALS research and new projects that the grant will help fund.
It uses a protein called GDNF (glial cell line-derived neurotrophic factor), which is a nutrient that brain cells can use to mature and survive. It promotes the growth of axons, the threadlike structures brain cells use to communicate, and also helps damaged cells rejuvenate.
In ALS, motor neurons are dying. And there is evidence that GDNF can help protect them. The problem is that GDNF is a large protein and it cannot pass through the barrier that protects the brain by preventing potentially harmful substances from entering it.
We've developed a clever technique, like a Trojan horse. We've engineered a human stem cell called a neural progenitor to release GDNF, and we're transplanting those cells into the spinal cords and brains of patients with ALS. The idea is that the progenitor cells mature into healthy support cells and release GDNF that will help heal diseased motor neurons.
Our first trials are to ensure the therapy is safe for patients. We have completed a Phase I/IIa clinical trial to treat the lower motor neuron, which controls leg movement, in 18 patients with ALS. We had good safety data in every one of those patients.Thoseresultswere published inNature Medicine.
We only put the cells on one side of each patients spinal cord, so we could compare the treated leg to the nontreated leg. While the focus of this initial study was safety, we did see a trend toward improvement in the treated limb after 12 months, even though the patients were at an advanced disease stage. We plan to perform an additional trial with patients who are earlier in the disease process and with optimized cell targeting, and hope to significantly improve limb function.
As the upper motor neuron in the brain is also affected in ALS, weve started an additional safety trial where we will treat the upper motor neurons, specifically those that control hand movement. We've treated five patients andthe trial is still open. Our next step will be to treat both the upper and lower motor neurons, and at that point, we will also measure the effectiveness of the therapy. If our technique is working, we should see a slowing of disease progression in the treated hand and leg. Research Project Advisor Pablo Avalos, MD; Adam Mamelak, MD, director of the Functional Neurosurgery Program at Cedars-Sinai; and Richard Lewis, MD, director of the Electromyography Laboratory at Cedars-Sinai, are key contributors to this work.
With the help of theCedars-Sinai Biomanufacturing Center, as part of a large consortium calledAnswer ALS, we have generated induced pluripotent stem cells from 1,000 patients with ALS. We have full clinical records and genomic sequencing for these patients. Induced pluripotent stem cells are adult human cells taken back in time to a stage where they are capable of developing into any cell in the body. Were then differentiating those cells into motor neurons that are known to die in ALS patients.
In collaboration with MIT [Massachusetts Institute of Technology], we will use the Tambourine grant to fund an advanced imaging project, called cell painting, on these motor neurons. Michael Workman, a project scientist in my laboratory, has been a key contributor to these studies. Up to six fluorescent dyes are used to label different components of the cells. With the help of an advanced, high-powered microscope and massive cloud computing, each individual neuron will be analyzed.
We will use artificial intelligence to attempt to distinguish ALS patients from healthy patients, and then look for molecular signatures within ALS patients that define subgroups. This data can be used to develop new targeted drugs to treat specific subgroups of ALSa truly personalized medicine approach leveraging the latest medical technology. This could be a big boost for clinical trials and drug development.
Read more on the Cedars-Sinai Blog:Regenerative Medicine: A New Path for ALS Treatment
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Q&A: New Frontiers in ALS Research - Newswise
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UC San Diego Develops First-In-Kind Protocol for Creating ‘Wired Miniature Brains’ – University of California San Diego
Posted: June 14, 2024 at 2:42 am
Other research possibilities for the brain organoids include disease modeling, understanding human consciousness and additional space-based experiments. In March, Muotri in partnership with NASA sent to space a number of brain organoids made from the stem cells of patients with Alzheimers disease and ALS (amyotrophic lateral sclerosis, also known as Lou Gehrigs disease). The payload returned in May, and results, which will eventually be published, are being reviewed.
Because microgravity mimics an accelerated version of Earth-based aging, Muotri should be able to witness the effects of several years of disease progression while studying the month-long missions payload, including potential changes in protein production, signaling pathways, oxidative stress and epigenetics.
Were hoping for novel findings things researchers havent discovered before, he said. Nobody has sent such a model into space, until now.
Co-authors of the study include Michael Q. Fitzgerald, Tiffany Chu, Francesca Puppo, Rebeca Blanch and Shankar Subramaniam, all of UC San Diego, and Miguel Chilln, of the Universitat Autnoma de Barcelona and the Instituci Catalana de Recerca i Estudis Avanats, both in Barcelona, Spain. Blanch is also affiliated with the Universitat Autnoma de Barcelona.
This work was supported by the National Institutes of Health R01MH100175, R01NS105969, MH123828, R01NS123642, R01MH127077, R01ES033636, R21MH128827, R01AG078959, R01DA056908, R01HD107788, R01HG012351, R21HD109616, R01MH107367, California Institute for Regenerative Medicine (CIRM) DISC2-13515 and a grant from the Department of Defense W81XWH2110306.
Disclosures: Muotri is a cofounder and has equity interest in TISMOO, a company dedicated to genetic analysis and brain organoid modeling focusing on therapeutic applications customized for autism spectrum disorder (ASD) and other neurological disorders with genetic origins. He is also the inventor of several patents related to human functional brain organogenesis, including the protocol described in the new publication.
The UC San Diego Sanford Stem Cell Institute (SSCI) is a global leader in regenerative medicine and a hub for stem cell science and innovation in space. SSCI aims to catalyze critical basic research discoveries, translational advances and clinical progress terrestrially and in space to develop and deliver novel therapeutics to patients.
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Pilot Study in JNCCN Explores New Approach for Reducing Anxiety and Improving Quality of Life after Stem Cell … – PR Newswire
Posted: June 14, 2024 at 2:42 am
Researchers at Brigham and Women's Hospital and Dana-Farber Cancer Institute found significant uptake and scalability in phone-based "PATH" intervention to improve psychological well-being in blood cancer patients.
PLYMOUTH MEETING, Pa., June 11, 2024 /PRNewswire/ -- New research in the June 2024 issue of JNCCNJournal of the National Comprehensive Cancer Networkhighlights a promising approach for alleviating distress, enhancing quality of life, improving physical function, and reducing fatigue in patients with blood cancers who undergo hematopoietic stem cell transplantation (HSCT). The study used a randomized clinical trial to evaluate the feasibility of a nine-week, phone-delivered, positive psychology program called Positive Affect for the Transplantation of Hematopoietic stem cells intervention (PATH), that was specifically tailored to the needs of this population. The findings indicate that the PATH intervention is both feasible and well-received by this patient population, as most of the patients (91%) who received the PATH intervention completed all of the intervention sessions and found them easy and helpful.
"The active identification and treatment of psychological distress, like anxiety, in patients with cancer are crucial."
"Having 9 out of 10 people complete all the sessions is great," explained lead researcherHermioni L. Amonoo, MD, MPP, MPH, Brigham and Women's Hospital/Dana-Farber Cancer Institute. "We designed PATH with the needs of HSCT survivors in mind. First, PATH is accessible to patients, as they can learn the skills and engage with the intervention over phone from wherever they areeliminating the need to travel to the cancer center. Second, the weekly exercises can be completed by patients at their convenience using the PATH manual, which guides patients on how to use the exercises and skills. This means that the actual phone sessions only last 15-20 minutes, in contrast to other well-established psychotherapies like cognitive behavioral therapy, which typically last 60-90 minutes per session. Third, we carefully curated the intervention sessions based on which activities patients can safely engage in while their immune system recovers following the transplant. For instance, unlike in other medical populations, we did not include exercises that focus on community service, which might unnecessarily expose patients to risks."
The pilot study was conducted at the Brigham and Women's Hospital/Dana-Farber Cancer Institute from August 2021 to August 2022. A total of 70 adult patients with blood cancers who have received HSCT, were randomized into two groups, with the intervention beginning about 100 days after HSCT. Those randomized into the PATH arm participated in a variety of weekly positive psychology exercises focused on gratitude, personal strengths, and meaning. Not only was participation high94% completed at least six of the nine sessions and 91% completed all ninethe intervention had promising effects on patient-reported outcomes immediately after completion of the program and again at week 18.
Dr. Amonoo added: "Cancer care providers should consider the potential benefits of psychosocial resources and interventions like PATH that focus on enriching positive emotions to bolster their patients' well-being. While the active identification and treatment of psychological distress, like anxiety, in patients with cancer are crucial, encouraging patients to engage in simple, structured, and systematic exercises aimed at fostering positive thoughts and emotions, such as gratitude, has the potential to enhance well-being as well."
"This positive psychology intervention highlights the importance of not only screening for distress but the promise of creating mechanisms that enhance well-being and reduce distress in our patients," commented Jessica Vanderlan, PhD, Manager, Siteman Psychology Service, Licensed Clinical Psychologist, Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, Vice Chair of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) Panel for Distress Managementwho was not involved in this research. "Development of clinical interventions that are brief (15-20 minutes) and delivered by phone could greatly improve patient access to care. This type of accessibility is important in an oncology population, especially in acute recovery periods with many competing demands and physical symptoms."
To read the entire study, visit JNCCN.org. Complimentary access to "A Positive Psychology Intervention in Allogeneic Hematopoietic Stem Cell Transplantation Survivors (PATH): A Pilot Randomized Clinical Trial" is available until September 10, 2024.
AboutJNCCNJournal of the National Comprehensive Cancer Network More than 25,000 oncologists and other cancer care professionals across the United States readJNCCNJournal of the National Comprehensive Cancer Network. This peer-reviewed, indexed medical journal provides the latest information about innovation in translational medicine, and scientific studies related to oncology health services research, including quality care and value, bioethics, comparative and cost effectiveness, public policy, and interventional research on supportive care and survivorship.JNCCNfeatures updates on the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines), review articles elaborating on guidelines recommendations, health services research, and case reports highlighting molecular insights in patient care.JNCCNis published by Harborside/BroadcastMed. VisitJNCCN.org. To inquire if you are eligible for aFREEsubscription toJNCCN, visitNCCN.org/jnccn/subscribe. Follow JNCCN at x.com/JNCCN.
About the National Comprehensive Cancer NetworkThe National Comprehensive Cancer Network (NCCN) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, equitable, and accessible cancer care so all patients can live better lives. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) provide transparent, evidence-based, expert consensus recommendations for cancer treatment, prevention, and supportive services; they are the recognized standard for clinical direction and policy in cancer management and the most thorough and frequently-updated clinical practice guidelines available in any area of medicine. The NCCN Guidelines for Patients provide expert cancer treatment information to inform and empower patients and caregivers, through support from the NCCN Foundation. NCCN also advances continuing education, global initiatives, policy, and research collaboration and publication in oncology. Visit NCCN.org for more information.
Media Contact:Rachel Darwin267-622-6624[emailprotected]
SOURCE National Comprehensive Cancer Network
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Pilot Study in JNCCN Explores New Approach for Reducing Anxiety and Improving Quality of Life after Stem Cell ... - PR Newswire
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