Improving Access to Bone Marrow Transplant and Cellular Therapies – Targeted Oncology

Hematopoietic stem cell transplantation (HCT) and cellular therapies offer life-saving potential for individuals facing hematologic malignancies, bone marrow failure syndromes, some autoimmune disorders, and certain solid tumors. However, various social vulnerabilities significantly influence access to these critical procedures, including socioeconomic status, race, culture, and geography.1-8 Other vital factors that affect access include insurance coverage, associated costs (co-payments, travel, housing, and time away from work), the knowledge of referring oncologists, donor availability, caregiver support, patient comorbidities, health literacy, and personal beliefs. There is an expanding interest in the HCT and cellular therapy community in abrogating these potential barriers to transplant.

Notably, recent strides in access to allogeneic (allo) HCT have focused on refining alternative donor transplantation methods, including haploidentical and mismatched unrelated transplants. The integration of posttransplant cyclophosphamide has yielded outcomes comparable to those of matched unrelated donor transplants.9 This is extremely important due to the uneven distribution of potential donors in the National Marrow Donor Program (NMDP) registry, with significant discrepancies based on ethnicity. Evidence from one study revealed a 75% likelihood of finding a full (8/8) human leukocyte antigen (HLA) match in the NMDP registry for patients of White European descent, contrasting with only 16% for Black people of South or Central American ancestry.10 With the use of alternative donors, over 95% of patients have at least 1 HLA-haploidentical first-degree donor.11

An additional obstacle to alloHCT is tied to the location of transplant centers, especially given the prolonged stay of patients and their caregivers during the transplant process. Despite the presence of 229 transplant facilities in the United States (185 for adults and 111 for pediatric care), the concentration of these centers, particularly on the East Coast, poses a signifi cant challenge for patients residing in more rural areas.7 Although 66% of the population lives within 60 minutes of travel time and 94% within 3 hours, the burden of frequent posttransplant visits, coupled with unaddressed housing and caregiver expenses not covered by insurance, creates substantial financial barriers for many.

Some patients may have potential donors and easy access to a transplant center, but another critical issue is the lack of referral for a transplant evaluation. Some existing databases used to analyze disparities, whether institutional or national (such as the Center for International Blood and Marrow Transplant Research), focus primarily on those who have undergone HCT, neglecting individuals with conditions who could benefit from transplantation but who have not been evaluated.1 A recent prospective study by Scott et al assessed transplantation referral patterns among 778 patients with high-risk myelodysplastic syndromes or acute monocytic leukemia (AML) in the Connect Myeloid Disease Registry undergoing treatment at community versus academic sites.12 Data from this study showed that more patients at academic sites were considered potentially eligible for transplant compared with the community sites (43.9% vs 27.9%; P<.0001), with multivariate analysis demonstrating ineligibility was based on age or comorbidities. In this study, 20% of patients were not assessed at all for transplant eligibility.

The aging population has become a particular focus in the transplant community. Over 40% of patients receiving allogeneic transplants in the United States are 60 years or older based on Center for International Blood and Marrow Transplant Research (CIBMTR) data.13,14 Artz et al examined the preferred post remission consolidation therapy (alloHCT vs consolidative chemotherapy) for older adults with AML in first complete remission (CR1). Results were worse initially in the alloHCT group, with an improvement over time. The overall survival rate of those who received an HCT was inferior during the first 9 months after transplant compared with those who did not receive HCT (HR, 1.52; P = .02). Conversely, after year 5, those who received an alloHCT had improved overall survival (28.6% vs 13.8%; HR, 0.53; P<.0001).14,15 These data highlight the importance of a transplant referral for patients of all ages with potentially eligible diagnoses.

Recent efforts have also aimed at expanding access to patients with active disease or measurable residual disease.

These efforts include clinical trials with novel targeted conditioning approaches, manipulated cellular therapy products, and targeted radiation approaches.

Favorable outcomes are achievable, and referral to a transplant center should be considered.

Barriers to accessing bone marrow transplants and cellular therapies are complex and multifaceted. Addressing them requires collective efforts to ensure equitable access and improved outcomes for all eligible patients, considering social, economic, and geographic factors.

Amanda Blackmon, DO, is assistant clinical professor, Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, City of Hope Comprehensive Cancer Center, Duarte, California. Idoroenyi Amanam, MD, is Assistant Professor, Division of Leukemia, Department of Hematology & Hematopoietic Cell Transplantation, City of Hope Comprehensive Cancer Center, Duarte, California.

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Improving Access to Bone Marrow Transplant and Cellular Therapies - Targeted Oncology

Neurona Raises $120M, Buoyed by Early-Stage Data for Epilepsy Cell Therapy – BioSpace

Pictured: 3D illustration of stem cells used for cell therapy/iStock,Maksim Tkachenko

Neurona Therapeutics on Thursday announced that it has secured $120 million in funding, which will help it advance its pipeline of investigational off-the-shelf cell therapies.

The California-based biotech will use the money to advance its lead candidate NRTX-1001, a regenerative neural cell therapy candidate being assessed in a Phase I/II trial for drug-resistant mesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy that can trigger ongoing seizures despite medical treatment.

Neuronas financing on Thursday was co-led by Viking Global Investors and Cormorant Asset Management. Other institutional supporterssuch as UC Investments, UCB Ventures, Euclidean Capital, The Column Group and Alexandria Venture Investmentsalso participated in the funding round.

Neurona has pioneered development of a fully-differentiated cell therapy for drug-resistant focal epilepsy that is designed to be disease-modifying, repairing the affected neural network, and is yielding very promising initial clinical data, Raymond Kelleher, managing director at Cormorant, said in a statement, adding that NRTX-1001 could be a game-changer with its potential to control seizures and preserve neurocognitive function.

According to the biotechs website, NRTX-1001 is a cell therapy comprised of human inhibitory GABAergic interneurons, which can potentially address the underlying hyperactive neural networks in epileptic seizures.

In December 2023, at the annual meeting of the American Epilepsy Society, Neurona presented data from two patients enrolled in the Phase I/II study of NRTX-1001, who saw at least a 95% drop in overall seizure frequency more than one year after treatment. NRTX-1001 was also safe overall, with adverse events categorized as mild to moderate in severity.

Neurona is also testing NRTX-1001 in Alzheimers disease, for which it is currently in the preclinical evaluation stage.

Beyond NRTX-1001, Neurona will also channel some of Thursdays haul into its other pipeline projects including investigational myelinating glial cells and gene-edited cells, both for yet-undisclosed indications.

Neuronas funding comes nearly a year after the biotech laid off 18 employees, or 25% of its total headcount, to streamline its budget in the face of the current tight funding environment and extend available cash to support the ongoing clinical trial of NRTX-1001, a spokesperson told Fierce Biotech at the time.

The financing could also be a sign of an uptick in the industrys fundraising activities. This week alone, there have been two initial public offeringsKyverna and Metagenomiwhich follow the earlier debuts of Alto Neuroscience, Fractyl Health, ArriVent Biopharma and CG Oncology.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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Neurona Raises $120M, Buoyed by Early-Stage Data for Epilepsy Cell Therapy - BioSpace

Stem Cell Transplant and CAR T-cell Therapy: The Patient and Care Partner Experience | BMT Infonet – BMT Infonet |

How to Support theUnique Psychosocial Challenges of Stem Cell Transplantand CAR T-cell Therapy Patients and Their Care Partners Mental Health Professionals: Earn 7 continuing education credits Why Take this Course? "I learned, as a transplant recipient, that a therapist is not a therapist is not a therapist. Working with a therapist who understands stem cell transplant is a completely different animal than working with an excellent therapist who doesnt have that knowledge base. I didnt have to explain the transplant experience. We could hit the ground running with our therapy. Nancy, stem cell transplant recipient.

Hematopoietic stem cell transplantation (HCT), also calledbone marrow, stem cell and cord blood transplantation, andCAR T-cell therapy, are two intensive therapies used to treat patients with advanced hematologic malignancies and certain genetic/immune system disorders. The intensity of the treatment, compared to standard chemotherapy for cancer patients, generates a unique set of medical and psychosocial consequences that are critical for mental health professionals (MHPs) to understand in order to most effectively support and treat these patients and their care partners.

This course is intended for psychologists, social workers, mental health counselors, and therapists. The instructional level isintermediate. We assume that participants already have psycho-oncology educational background and experience.

The primary format is a non-interactive distance-learning continuing education course presented through a series of videos.

Module 1: The Nuts and Bolts of Hematopoietic Cell Transplantation (HCT)John Wingard, MD; Michelle Bishop, PhD;Justin Regan, RN A broad overview of HCT including the rationale for its use; eligible patients; short-term, long-term, and late effects of treatment; and the impact on patients and care partners overall quality of life (QOL) and health behaviors.Includes an in-depth review of the psychosocial issues experienced by HCT patients and care partnersand concludes with a brief presentation by an HCT patient discussing the challenges both he and his family faced while undergoing and surviving two stem cell transplants.

Module 2: Autologous HCT: A Deeper Dive R. Gregory Bociek MD, MSc An in-depth review of the medical processes and the short- and long-term consequences of undergoing an autologous HCT. Autologous HCT is a transplant that uses the patients own stem cells. It is a treatment option for some patients diagnosed with multiple myeloma, non-Hodgkin lymphoma, Hodgkin disease and some solid tumors.

Module 3: Allogeneic HCT: A Deeper Dive Minoo Battiwalla MD, MS An in-depth review of the medical processes and the short-and long-term consequences of undergoing an allogeneic HCT. Allogeneic HCT uses stem cells provided by a donor, rather thanthe patient. It is a treatment option forsome patients diagnosed with leukemia, myelodysplastic syndrome, a myeloproliferative disorder, severe aplastic anemia, or an inherited disorder such as sickle cell disease.

Module 4: Unique Psychosocial Challenges for Autologous and Allogeneic HCT Patients and Care PartnersElizabeth M. Muenks, PhD A comparison of the psychosocial issues experienced by autologous HCT patients,allogeneic HCT patients, and their care partners during preparation for HCT, the day of transplant and early recovery, the first 100 days after HCT, and long-term.

Module 5: Graft-versus-Host Disease (GVHD): Trading an Acute Disease for a Chronic Disease Amin M. Alousi, MD;Michelle Bishop, PhD An overview of GVHD, a major complication that affects approximately 50%of patients who undergo an allogeneic HCT.This module includes an in-depth discussion of how GVHD affects multiple organs and tissues, treatment options and their potential side effects, and the psychosocial consequences of livingwith GVHD in the short- and long-term for both patients and care partners.

Module 6: Psychiatric Disorders in Patients Undergoing HCTMaria Adelaida Rueda-Lara, MD A review of common psychiatric issues that occur during and after HCT, and their management.

Module 7: CAR T-cell Therapy: The Medical and Psychosocial Experience Areej El-Jawahri, MD An in-depth review of the medical and psychosocial consequences of CAR T-cell therapy - a treatment option for some patients with non-Hodgkin lymphoma, acute lymphoblastic leukemia, and multiple myeloma.

At the conclusion of this course, participants will be able to:

Describe the rational for using HCT to treat patients with hematological disorders and certain genetic/immune system disorders, and the psychosocial issues associated with treatment in the short-and long-term.

Describe the steps involved in autologous HCT,andthe short- and long-term medical side effectsand psychosocial issues.

Describe the steps involved in an allogeneic HCT,and the short- and long-term medical side effects and psychosocial issues.

Describe how psychosocial issues associated with autologous HCT compareto those associated with an allogeneic HCT for both patients and care partners.

Explain what graft-versus-host disease (GVHD) is, differentiate acute vs chronic GVHD, describe the unique medical and psychosocial challenges that GHVD patients and care partnersexperience, and discuss psychotherapeutic treatment modalities to address those challenges.

Describe the incidence of and risk factors associated with common psychiatric disorders thatoccur during each phase of the HCT process and identify the psychiatric sequelae of common medications used to treat patients during HCT.

Describe the types of hematological disorders most frequently treated with CAR T-cell therapy and the unique stressors and medical and psychosocial sequelae that come with this treatment.

Describepsychological and psychiatric treatment modalities that have proven beneficial for these patient populations.

Amin M. Alousi MD

Professor of Medicine,Medical Director of theStem Cell Transplant and Cellular Therapy ProgramandDirector of the Multi-Discipline GVHD Clinic and Research Program,MD Anderson Cancer Center

Minoo Battiwalla, MD, MS

Director of Blood Cancer Outcomes Research,Sarah Cannon Transplant and Cellular Therapy Network

Michelle Bishop, PhD

Coping with Cancer & Caregiving, LLC Co-Director of Mental Health Projects andGVHD Support Group Program Lead, BMT InfoNet

R. Gregory Bociek, MD, MSc

Associate Professor, Oncology & Hematology,University of Nebraska Medical Center

Areej E-Jawahri, MD

Associate Professor of Medicine andDirector of the Bone Marrow Transplant Survivorship Program,Massachusetts General Hospital

Elizabeth M. Muenks, PhD

Assistant Professor,Psychiatry and Behavioral Sciences, andDirector of Psychology Services, University of Kansas Medical Center

Justin Regan, RN

Recipient of two allogeneic stem cell transplants

Maria Adelaida Rueda-Lara, MD

Assistant Professor, Department of Psychiatry & Behavioral Sciences andMedical Director of Psycho-Oncology,University of Miami Miller School of Medicine

John Wingard, MD

Professor of Medicine, Retired Deputy Director of theUF Health Cancer Center andDirector of the Bone Marrow Transplant Program,University of Florida Health Cancer Center

ENROLL in this course consisting of seven modules.Course fee - $199.(includes all seven modules)

COMPLETE COURSE CONTENT by finishing all modules.

TAKE THE POST-TEST assessment and achieve a passing grade of at least 75%

Complete the COURSE EVALUATION

Full attendance is required for all CE activities. This means you will be asked to certify that you watched all the videos included in this course.Once you have completed the above steps, your course certificate will automatically populate for download. It can be downloaded immediately, emailed, and will be available for future download.

Click to Enroll Now

There was no commercial support provided for this CE program, the content, or to the instructor(s).

It is absolutely critical that our program exemplifies accessibility for all participants. We seek to ensure all participants are able to fully, equally, and independently access the educational content. Our goal is to have a website that is perceivable, operable, understandable, and robust. If you have any difficulty navigating the website, we want to know! Please contact us atinfo@findempathy.com.

CE credits require a post-test assessment. The assessment is untimed and provided in writing on the course website. However, for individuals with visual impairments, or those that may benefit from an orally presented post-test and program evaluation, contact us. We will work with you to find an alternative option for completing the course post-test and program evaluation.We remain available and open to ANY additional requests for format modification or additional assistance.

If any accommodations are needed to enroll or participate in the course, or complete the post-test and course evaluation, please contact us atinfo@findempathy.com.

Course Access::Once enrolled in a course, you have 30 days to complete the post-test and course evaluation. If your access lapses before completing all tasks, you may need to re-enroll.

Certificate Access: Once you have completed the post-test and program evaluation, your certificate will be immediately available for download. Find Empathy will keep course certificates on file for 7 years. If a certificate is needed log into your Find Empathy account and download it, or contact:info@findempathy.com.

Post-test Retake Policy:You are allotted 3 retries to achieve the acceptable 75% passing grade.

This course is intended for psychologists, social workers, mental health counselors, and therapists. The instructional level is intermediate. We assume that participants already have psycho-oncology educational background and experience. The primary format is a non-interactive distance-learning continuing education course presented through a series of videos. There was no commercial support provided for this CE program, the content, or to the instructor(s).

Contact us atinfo@findempathy.com. We will do our best to return your email within 24-48 hours during business hours: Monday through Friday, 9am to 5pm eastern.If you have any questions, concerns, or grievances please email us atinfo@findempathy.com.You can also see our grievance policy via the following link:findempathy.com/grievance.

PSYCHOLOGISTS

Empathie, LLC is approved by the American Psychological Association to sponsor continuing education for psychologists. Empathie, LLC maintains responsibility for this program and its content.

COUNSELORS: Find Empathy has been approved by NBCC as an Approved Continuing Education Provider, ACEP No. 7257. Programs that do not qualify for NBCC credit are clearly identified. Find Empathy is solely responsible for all aspects of the program

SOCIAL WORKERS

Find Empathy, #1817, is approved to offer social work continuing education by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program. Organizations, not individual courses, are approved as ACE providers. State and provincial regulatory boards have the final authority to determine whether an individual course may be accepted for continuing education credit. Find Empathy maintains responsibility for this course. ACE provider approval period: 5/14/2023-5/14/2026. Social workers completing this course receive 7 continuing education credits.

Stem Cell Transplant and CAR T-cell Therapy: The Patient and Care Partner Experience,[course number],is approved by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program to be offered by Find Empathy as an individual course. Individual courses, not providers, are approved at the course level. State and provincial regulatory boards have the final authority to determine whether an individual course may be accepted for continuing education credit.ACE course approval period: [dates]. Social workers completing this course receive 7 continuing education credits.

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Stem Cell Transplant and CAR T-cell Therapy: The Patient and Care Partner Experience | BMT Infonet - BMT Infonet |

BioRestorative Therapies to Present Preliminary BRTX-100 Clinical Data at the Orthopaedic Research Society (ORS … – OrthoSpineNews

Blinded data from the ongoing Phase 2 clinical trial of BRTX-100 to be described in poster presentation

Company to host webcasted conference call to review data on February 5, 2024 at 8:30am EST

MELVILLE, N.Y., Feb. 01, 2024 (GLOBE NEWSWIRE) BioRestorative Therapies, Inc. (BioRestorative, BRTX or the Company) (NASDAQ:BRTX), a clinical stage company focused on stem cell-based therapies, today announced that preliminary 2652 week blinded data from the ongoing Phase 2 clinical trial of BRTX-100 in subjects with chronic lumbar disc disease (cLDD) will be presented by Francisco Silva, Vice President of Research and Development, at the Orthopaedic Research Society (ORS) 2024 Annual Meeting, taking place February 2-6, 2024 in Long Beach, California.

BRTX-100, a novel cell-based therapeutic engineered to target areas of the body that have little blood flow, is the Companys lead clinical candidate. The safety and efficacy of BRTX-100 in treating cLDD is being evaluated in a Phase 2, prospective, randomized, double-blinded and controlled study. A total of up to 99 eligible subjects will be enrolled at up to 16 clinical sites in the United States. Subjects included in the trial will be randomized 2:1 to receive either BRTX-100 or placebo.

The presentation, titled Autologous Stem Cell Therapy for Chronic Lumbar Disc Disease, Initial Phase 2 Clinical Safety and Feasibility Data of Intradiscal Injections of Hypoxic Cultured Mesenchymal Stem Cells, is scheduled for Sunday, February 4, 2024 between 10:15am-11:15am PST.

We are excited by this opportunity to share, for the very first time, preliminary data from the ongoing Phase 2 clinical trial of BRTX-100 in the treatment of cLDD, said Lance Alstodt, Chief Executive Officer of BioRestorative. As interest in the potential clinical benefits of BRTX-100 grows, it is important that we continue to drive awareness of this novel therapeutic candidate amongst researchers, regulators and clinicians through clinical presentations at important meetings like ORS.

Conference Call & Webcast Details

BioRestorative management will host a webcasted conference call with an associated slide presentation at 8:30am EST on Monday, February 5, 2024 to review the BRTX-100 poster. To join the conference call via phone and participate in the live Q&A session, please dial 888-506-0062 (United States) or 973-528-0011 (International), participant access code 234972. The live webcast and audio archive of the presentation may be accessed on the investor section of the BioRestorative website athttps://www.biorestorative.com/investor-relations/. An archived replay will be available for approximately 90 days following the event.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

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 a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). 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.

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 Companys latest Form 10-K 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 to Present Preliminary BRTX-100 Clinical Data at the Orthopaedic Research Society (ORS ... - OrthoSpineNews

New FDA-approved sickle cell gene editing therapies offer hope for a pain-free life to patients some living in Colorado – Colorado Public Radio

Growing up, I really didnt have as many crises with sickle cell, Hymes said in a recent virtual interview from Arkansas. When I was getting older, I started having more and more sickle cell crises. But Ive always had this dream of becoming a physician, and as I got older, the more crises I had, the harder it made that journey.

A report from the Centers for Disease Control and Prevention (CDC) stated the origins of the pain: Sickled cells traveling through small blood vessels can get stuck and block blood flow throughout the body, causing pain. A pain crisis (vaso-occlusive episode or VOE) can start suddenly, be mild to severe, and can last for any length of time.

If you got hit by a bus and survived, all that excruciating pain that you would feel, thats how Id describe it. Id spend a week, two weeks, in the hospital, and then you have to recover, thats another week, and Id miss two weeks of class, Hymes said when explaining what living through a pain crisis has been like for him.

So, he looked for a solution by doing some online research that led him to McKinneys work.

We shot a few emails back and forth and it just felt like the right place to be I knew that the life I wanted and how it was going couldnt really co-exist, he said. Leading up to around the time I reached out to Dr. McKinney, I started to have crises almost every other month or sometimes even every month.

After he and McKinney developed an email relationship, Hymes came to Colorado for some blood work and testing and discovered he was a match for the trial.

Once it was determined that I could, I became a little more hopeful in the journey, said Hymes, who didnt dwell on the challenges of that journey, and instead honored the people who helped him through it: his mother, his girlfriend, and his grandparents all of whom came to visit him in Colorado, while he spent months coping with the complicated procedure.

We evaluate the patient to ensure their organs are healthy enough to tolerate the gene therapy process; we then start putting them on chronic transfusions where they get a monthly transfusion to decrease how much sickle hemoglobin they have," explained McKinney. "We give them medications to help them release their stem cells from their bone marrow into their blood and we hook them up to machines to collect those stem cells.

Its those cells that then get the gene therapy, they are edited to stop forming the sickle shape.

And that may take several months to get enough stem cells ... once weve collected those stem cells, we have to send those stem cells off to manufacture and to change the genes so that they make non-sickling hemoglobin. And that process can take about 12 weeks, McKinney said.

Patients then return for high doses of chemotherapy, which gets rid of the old bone marrow and makes room for the new.

Once they get that chemotherapy, we then infuse the stem cells, and it can take about four weeks for those cells to grow, McKinney said. The patient remains in the hospital while the new stem cells grow. Upon discharge, they have years of follow-up monitoring. Ultimately, patients who receive gene therapy need to be followed for about 15 years after the infusion to make sure theyre doing well."

For Hymes, things went according to plan. Because he was from out of town, he had to stay in Colorado for about six months in total. He said part of it was to have my edited genes put back in my body, and thats when I would have the chemo and stay in the hospital for an extended period of time.

Recovering from the chemotherapy, Hymes said, was one of the hardest parts.

I was still having to deal with the chemo and all the problems that it can cause. Its just this really bad taste you could have from the chemo and it could knock out your taste and you have a sore throat ... I guess Im a big eater, so food was the important part to me. But I drank a lot of Ensures just recovering from the chemo, said Hymes.

Now, two years since his journey began and one year after the transplant, Hymes said he feels like a new man.

It was a total change in my energy level and how I was able to just go about life! I didnt have to worry about having pain crises every other day or every month, said Hymes.

Regarding his pain crisis, Hymes said, Its almost non-existent at this point. I dont say almost non-existent. Its been non-existent at this point.

Like about 90 percent of McKinneys other trial participants, its all over now.

I havent had any crises since I had the stem cell transplant, and sickle cell-related problems since the transplant. So I would say it has been extremely beneficial in my life, said Hymes.He said it's also been good for his mother.

I only seen my mom cry one or two times, Hymes explained. One time was seeing him struggle with the disease, the other time, Hymes said, was seeing him leave it behind.

The two FDA-approved treatments are slightly different from what Hymes received but they work the same way, editing cells, and then returning them to the patient.

Now that the drug therapy has been approved, another component of McKinneys job is to let the rest of his patients the ones who werent part of a trial know about it. Some of those who werent in any of his trials experience pain from the disease serious enough to make the therapy worth considering.

A few weeks after the FDA announced its approval of the drugs in early December, McKinney met with Mia Hilton, a 20-year-old esthetician from Green Valley Ranch in Denver. Hilton has been McKinneys patient for the past 10 years. Vivacious and upbeat, she spent most of the meeting making funny comments. After a quick overall exam, he asked if shed heard about the newly approved gene therapies.

They are meant to be curative, he explained. Youd no longer have sickle cell after administration of them.

For her, that would be a big change. Like Hymes, sometimes, her pain got bad enough to go to the ER, where shed sometimes receive intravenous drugs.

Usually a pain crisis feels kind of like burning or stabbing a little bit. It can feel pretty hot internally in your muscles. I'll take a Benadryl and then they'll give me morphine through my IV, and that usually works, said Hilton. If not, she gets a nasal dose of fentanyl. In the ER, medical staff monitor her progress.

They usually do a chest X-ray to make sure that I'm not going to have a chest crisis, explained Hilton. And after a few hours, if the pain was under control, shed get discharged. I'll have somebody come and pick me up from the ER just because of the heavy medicines, they don't want me to drive.

Sometimes, when she didnt bounce back, she would be admitted from the ER into the hospital.

Usually my stay can range from three to seven days, so it does get pretty strenuous, she said.

She told McKinney that shed heard some pieces of information about the new gene therapies, but wanted to know more. In a cautious, gentle voice, he told her, The side effects of the chemo could cause infertility and hair loss.

Hilton had a quick answer to that.

Ive never wanted babies, she said, So its a free form of birth control.

I'm very into make-up and hair, so I already have 40 wigs in my closet, the bubbly woman added while already wearing eyelashes nearly an inch long. When McKinney mentioned that painful sores could form in her mouth, she didnt care about that either.

I bite my jaws a lot, so Im already there. I bit my tongue and my jaw four times in the elevator on the way up here, said Hilton.

Her reservations focused on the time shed lose working, and concerns about whether her case was severe enough to warrant the sacrifice.

I love science, so to hear that you're going to take something out, fix it, and put it back, it's like, Oh my gosh, that's kind of cool. Personally, I would definitely look into it, but ... I don't know if I would have the energy to do that. You know what I mean? I would be pretty much down to do it because I feel like in the long run, it could really help because having sickle cell, if you're not on top of it and you're not maintaining it, it can really slow you down ... So to get rid of crises altogether, that would be pretty ideal, said Hilton.

Although the side effects didnt bother Hilton, the cost could if her insurance wont cover it. One therapy costs $2 million, and the other is $3 million. But, according to Dr. David Rind, chief medical officer at the Institute for Clinical and Economic Review, a nonprofit that evaluates the prices and effectiveness of medications, that price wouldnt come out of the patients pocket. He said he expects insurance companies to cover the gene therapy.

I dont think any manufacturer would charge a price like that thinking only the patients who can afford it will get it, Rind said. I think the assumption is that insurance will pay for this ... I think this will be paid for, for almost anyone who wants it.

The rationale for why insurers should cover the gene therapies relates to slavery, he said.

The reason there is sickle cell in the U.S. is because we brought Africans over as slaves, Rind said. He explained that the disease is a gene mutation that fights malaria, a disease common in Africa. Thats why the people who are here have sickle cell . This is a population obligation in the United States to get this right.

Hymes has a sense of obligation, too, to other people born, like him, with the disease. Now that he has gotten rid of his crises as a clinical trial participant, what he thinks would be ideal is to become a doctor caring for people with sickle cell.

My entire life, Ive been extremely interested in hematology-oncology, of course, because that is what [sickle cell disease] is grouped as. But lately, Ive been interested in emergency medicine as well, because I know a lot of times we get seen on the front end in the emergency room, and thats where a lot of chronic pain patients get perceived as opioid-seeking, said Hymes.

He is now participating in an academic program for aspiring medical students while waiting to hear if hes been accepted to medical school. Once he has completed his medical degree, he said, he knows hed have the sensitivity to understand a sickle cell disease patients experience as real, not one of faking pain in the ER.

So either helping on that side or helping in the hematology and oncology area is what Im interested in. If I could do both or figure out a way to help all across the board, then Id probably choose that, too, said Hymes.

McKinney looks forward to offering his patients the choice of a different ending.

Now, when those families come in for their first visit with us to talk about what its going to look like for their child to live with sickle cell disease, we can at least provide them some hope that this is something that is very, very treatable, McKinney said. Something that if theyre having a lot of problems related to this, that we can potentially cure and get rid of.

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New FDA-approved sickle cell gene editing therapies offer hope for a pain-free life to patients some living in Colorado - Colorado Public Radio

Stem cells used to successfully treat arthritis in gorilla at Budapest zoo – University of Sheffield News

Stem cell therapy has been used to treat osteoarthritis in a gorilla for the first time, by scientists at the University of Sheffield.

Scientists at the University of Sheffield have used mesenchymal stem cells to treat arthritis in a gorilla at Budapest Zoo

The use of stem cells for the treatment of arthritis and regeneration of the damaged cartilage has been successfully piloted in several animal species, such as dogs and horses, in recent years

Liesel the gorilla is believed to be the first primate in the world to benefit from this joint work

Stem cell therapy has been used to treat osteoarthritis in a gorilla for the first time, by scientists at the University of Sheffield.

Liesel, the elderly matriarch at the Budapest Zoo has been finding it difficult to walk on her left leg for some time now, suggesting that she may be suffering from arthritis.

An international team, led by Endre Ss, Chief Vet and Acting Director General at the Budapest Zoo and Professor Mark Wilkinson, an Orthopaedic Surgeon and leading international expert in the treatment of human arthritis from the University of Sheffield, carried out a comprehensive assessment of Liesels major joints and used mesenchymal stem cells to treat alterations in her left hip and knee joints.

Osteoarthritis is a progressive degenerative process of the joint. Once the cartilage is worn and damaged, the process is irreversible and current treatments focus on symptomatic control but not to treat the disease itself.

The use of stem cells for the treatment of arthritis and regeneration of the damaged cartilage has been successfully piloted in several animal species in recent years, such as dogs and horses and small-scale clinical trials in humans have also proven to be a promising treatment for this condition. Liesel is thought to be the first primate in the world to receive the treatment and successfully benefited from the work of the research team.

Following previous successful research trials on arthritis-affected dogs, Stem CellX - a company made up of ateam of international scientists working in the field of stem cells, regenerative medicine, and genetics - was established to develop new technologies for the formulation of stem cell-based products for arthritis treatment in animals.

Stem CellX founder and Professor of Cell Signalling at the University of Sheffield, Endre Kiss-Tth has collaborated with Professor Mark Wilkinson for a number of years to explore novel treatment options for human arthritis. They now jointly lead a preclinical programme to test Stem CellX technologies for the development of a similar stem cell treatment in human patients.

The company recently partnered with Budapest Zoo to provide this treatment for animals in need, as well as supplying zoos globally.

The mesenchymal stem cells used for the procedure on Liesel were isolated from a piece of fat tissue donated by N'yaounda, a young female gorilla who underwent a planned minor operation in 2022. A specialist team at Stem CellX then isolated, purified and cultured these cells at their R&D base in Hungary to formulate a cell suspension that could be kept deep-frozen until the treatment.

Professor Kiss-Tth and Professor Wilkinson are now jointly leading a preclinical programme to test Stem CellX technologies for the development of a similar stem cell treatment in human patients.

Professor Endre Kiss-Toth, from the University of Sheffield and Founder of Stem CellX said:It has been a great privilege to be part of this word-first collaboration and bring together Stem CellX expertise in stem cell technologies, with the internationally leading clinical skills and knowledge in osteoarthritis pathogenesis of the University of Sheffield to provide a novel treatment option for Liesel to improve her quality of life in her golden years.

We are now following her recovery closely, in the hope to see marked improvement in her movements and in the use of her osteoarthritis affected leg.

Professor Mark Wilkinson, from the University of Sheffield and Leader of Clinical Orthopaedic Team, said:I was delighted to be part of the team doing this ground-breaking work and having the opportunity to treat Liesels arthritis. We are currently developing a similar treatment for humans. This work is in its very early stages but hopefully will lead to a real solution for patients to the pain and suffering that arthritis causes.

Honorary Associate Professor, Endre Ss, Leader of the Zoo Team said:The advanced husbandry and veterinary practices in modern zoos result inincreased longevity in many species, including apes. Our task is to providethe best medical care and best quality of life for these animals, despite theirage-related conditions. Stem-cell therapy hopefully brings in a new era in thisfield as well.

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Stem cells used to successfully treat arthritis in gorilla at Budapest zoo - University of Sheffield News

Could Treatments for HIV and Sickle Cell Open the Gene Therapy Floodgates? – BioSpace

Pictured: Illustration of gloved hands using tools to manipulate a DNA double helix/iStock, MicrovOne

In the early 1990s, Mike McCune and his colleagues thought theyd found a path to a one-time HIV treatment for babies: genetically modifying hematopoietic stem cells to suppress the virus and then transplanting them into the bone marrow. But the treatment never made it to clinical trials.

It was not considered to be a good business plan, he explained, because companies would make more money by selling these patients antiretroviral therapies for a lifetime than to deliver a one-time treatment.

Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy, which involves a process much like McCunes HIV treatmentex vivo gene editing of blood stem cells for the treatment of sickle cell disease (SCD) followed by transplantation back into the patientdid make it to market, having been recently approved by the FDA as the first CRISPR-based therapy. But Casgevy is pricy: it costs a whopping $2.2 million dollars. Its competitor Lyfgenia, another ex vivo gene therapy for SCD approved at the same time, carries a sticker price of $3.1 million. The high cost may limit access even for patients in the U.S., and will likely be a far more formidable barrier in sub-Saharan Africa, where 79% of babies with SCD are born.

Now McCune and others are looking to bring gene therapy to the masses by developing treatments that involve treating cells in vivo, which could be far less costly than the ex vivo variety. Requiring only a single injection, the in vivo approach would use vectors to deliver DNA-changing machinery into cells. In the case of SCD, the target would be blood-making stem cells and the tweaks would induce them to make healthy hemoglobin, McCune explained; for HIV, viral DNA could be snipped inside infected cells to prevent viral replication and spread, as well as reinfection.

While an existing in vivo gene therapies for rare genetic diseases also have eye-popping sticker prices, McCune argued that over time, costs will fall.

McCune, now head of the Gates Foundations HIV Frontiers program, is helping to forge agreements between the philanthropy and multiple private companies with the aim of making gene therapy an accessible reality as a treatment for both HIV and SCD. And at least one biotech, Excision BioTherapeutics, is pursuing a similar approach to HIV treatment independently of Gates.

As McCune sees it, there will be both a market and payers for a one-shot HIV treatment, both in the U.S. and in sub-Saharan Africa. He points out that the healthcare system is currently paying tens of thousands of dollars annually for each HIV patient in the U.S., money that could be redirected to paying for a cure. And in sub-Saharan Africa, he added, the U.S. is spending about $7 billion each year on antiretroviral treatment for people with HIV under a program called PEPFARfunding that could likely cover the cost of a one-shot treatment instead.

Theres no comparable payer for an SCD treatment, he said. We have to work on that. And I think thats going to become really important because the inequities of healthcare, so poignantly highlighted during COVID, are going to become even more glaringly obvious now that there are multimillion-dollar treatments that are likely to remain inaccessible to many people with SCD.

Gates has partnered with companies including Guide Therapeutics, bluebird bio, GreenLight Biosciences, Intellia Therapeutics, CRISPR Therapeutics, Immunocore, BioNTech, Vir, Ensoma, Emmune, Addition and Novartis, as well as nonprofits and academic labs, to work on aspects of in vivo gene therapy. The foundation has also partnered with the National Institutes of Health, which committed to kicking in $100 million toward the effort.

Agreements with partners, McCune said, include a non-exclusive right for the Gates Foundation to make sure that the technologies are available to people living with HIV and SCD in low- and middle-income countries. Why would they give us those rights? he asked. Part of the answer is that the applications of this platform are diverse, outside of the realm of HIV and sickle [cell disease] and into the realm of the more remunerative diseases that companies seem to focus on in the context of gene therapy, including cancer. They would see this as a pathway for return on investment, which [could] be huge, McCune said.

Intellia, a former Gates grantee, is in the preclinical stages of developing an in vivo CRISPR treatment for SCD, company spokesperson Ian Karp wrote in an email to BioSpace. The benefit of a one-time treatment certainly has applicability to patients across the globe, he said. Additionally, our technology platform is modular, such that we do hope to leverage it across multiple indications / diseases. Oftentimes the only change we need to make from one investigational product to the next (particularly when targeting the same cell type and edit type) is in the targeting region of the guide RNA which serves to direct the CRISPR machinery to the gene of interest.

Similarly, Christine Silverstein, chief financial officer at Excision BioTherapeutics (which has not received Gates funding), said that the technology behind the companys candidate CRISPR HIV treatment, the Fast Tracked EBT-101, may be applied to other chronic viral infectious diseases including herpes and hepatitis B. In fact, our work in HIV is setting the foundation for advancements of Excisions pipeline which unites next-generation CRISPR nucleases with a novel, multiplexed gene editing approach to develop potentially curative therapies, she said in a statement.

But success is by no means assured. Despite the current excitement over CRISPR, hurdles to its therapeutic use remain, including not-insignificant safety concerns, and safety issues have also cropped up with other gene therapies. The Gates Foundation itself is hedging its bets, working in parallel to drive the development of a therapeutic vaccine for HIV.

Still, McCune is dreaming big. Even for conditions where effective treatments exist, these sorts of in vivo treatments might be something that takes pills off the shelves and sets the course for a different kind of medicine.

Shawna Williams is a senior editor at BioSpace. She can be reached at shawna.williams@biospace.com or on LinkedIn.

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Could Treatments for HIV and Sickle Cell Open the Gene Therapy Floodgates? - BioSpace

AI Biotech Secures Funding to Speed Up Stem Cell Therapy Development – BioPharm International

CellVoyant has raised 7.6 million in seed funding to speed up the development of novel cell therapies.

CellVoyantan artificial intelligence (AI) first biotechnology company spun out of the University of Bristolhas raised 7.6 million in seed funding to speed up the development of novel cell therapies. According to a Jan. 16, 2024 press release, the funding was led by Octopus Ventures and included Horizon Ventures, Verve Ventures, and Air Street Capital as participants of the funding round.

The biotechs primary aim is to use its platform, which combines advanced AI with live cell imaging, to develop novel stem cell therapies in a cost-effective way while also improving reliability and speed of development. While there are promising applications of stem cell therapies, the science behind these options is complex and unpredictable and development tends to have high attrition rates.

Cell therapies have the potential to revolutionize the way we treat diseases that affect millions of people every year, said Rafael E. Carazo Salas, CEO and founder of CellVoyant, in the press release. By combining the latest advances in AI and live cell imaging, we can help bring these transformative treatments to the market quickly, reliably, and cost-effectively. [This funding] milestone validates the potential of our approach and will help us to accelerate our R&D capabilities.

University spin-outs like CellVoyant are at the heart of the UKs thriving biotech ecosystem, added Uzma Choudry, lead biotech investor at Octopus Ventures, in the press release. CellVoyant sets a new standard in precision and reliability for predicting and controlling stem cell behavior, which will make cell therapies more accessible to those who need them. We are thrilled to invest into a company that is transforming how patients can benefit from life-changing treatments.

With the seed funding raised, CellVoyant will scale up its operations, including doubling the team size over the next two years, expanding laboratory and experimental infrastructure, and advancing AI capabilities.

Source: CellVoyant

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AI Biotech Secures Funding to Speed Up Stem Cell Therapy Development - BioPharm International

LVHN announces opening of new stem cell transplant center. Here’s what that means for the Lehigh Valley – The Morning Call

Lehigh Valley Health Networks Lehigh Valley Topper Cancer Institute celebrated the opening of its new Stem Cell and Transplant Therapy Program Tuesday.

Stem cell transplants are a life-saving treatment option for certain blood disorders and cancers, including numerous forms of leukemia. LVHN first announced two years ago that it would expand what was at the time an infusion suite to include stem cell transplant and cellular therapy, thanks to donations from Tom and Karin Hall. Karin Hall was treated for breast cancer at LVHN.

The program is led Dr. Amir Toor, who has more than 20 years of experience with stem cell transplants and vast expertise with cellular therapies for hematologic malignancies.

Toor said the network has spent the last few years preparing for the program by bringing together a specialized team of physicians, clinicians, nurses, social workers, financial counselors and transplant coordinators specially trained to care for and support patients undergoing stem cell transplants. He added that LVHN has also developed designated spaces where stem cells are collected for transplants and an inpatient unit designed to keep patients safe during treatment.

It is an honor to bring stem cell transplantation to people in all the communities LVHN serves, Toor said.

Stem cells are produced in bone marrow and many other tissues of the body. These cells can divide into more stem cells or turn into certain specialized cells throughout the body, such as nerve cells, cardiac muscle cells and blood cells, replacing older cells. Stem cells are essential, but some peoples bodies may not produce enough of them or they may have lost stem cells to cancer or cancer treatment.

The goal of stem cell transplants, also called bone marrow transplants, is to restore blood-forming stem cells to the body, often using a patients own preserved cells or cells from a donor, such as a family member.

Treatment through LVHNs new program will be available for people with disorders and cancers such as:

There are roughly 200 blood stem cell transplant centers that report data to the U.S. Health Resources & Services Administration, of which 11 are in Pennsylvania. Patients will often travel for this level of care, spending weeks far from home, Dr. Brian A. Nester, president and CEO of LVHN, said.

Stem cell transplants are a lifesaving treatment option, but they are also incredibly taxing for those receiving them and their loved ones, said Nester.

There are other uses of stem cells in medicine, including regenerative medicine, which uses stem cells to repair diseased, dysfunctional or injured tissue in the body. This technique can even be used to grow new organs to replace damaged ones using a patients own stem cells.

Dr. Suresh Nair, physician in chief of the Topper Cancer Institute, said that for now, LVHN will not offer regenerative medicine treatments, but this new center puts LVHN at the forefront of opportunities in future stem cell research technologies.

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LVHN announces opening of new stem cell transplant center. Here's what that means for the Lehigh Valley - The Morning Call

CellVoyant secures 7.6 million seed funding to develop stem cell therapies with AI – BioPharma-Reporter.com

Led by Octopus Ventures, with participation from Horizons Ventures, Verve Ventures, and Air Street Capital, this funding marks the first stage in commercialising CellVoyant's AI and imaging platforms.

The company aims to enable the cost-effective and rapid development of novel cell therapies. The proceeds from this funding round will be used to increase headcount, expand laboratory and experimental infrastructure, and support R&D.

Rafael E. Carazo Salas, CEO and founder of CellVoyant, said: Cell therapies have the potential to revolutionise the way we treat diseases that affect millions of people every year. By combining the latest advances in AI and live cell imaging, we can help bring these transformative treatments to the market quickly, reliably, and cost-effectively. Todays milestone validates the potential of our approach and will help us to accelerate our R&D capabilities.

Stem cells unlock a range of treatment possibilities that have not previously been available to patients. Current treatment options act on the bodys existing cells, while stem cells can develop into many different cell types in the body - regenerating damaged tissues and organs or replacing dysfunctional and dead cells.

There are already promising potential applications in chronic diseases, such as cancer, and metabolic diseases like diabetes and age-related macular degeneration. In future, stem cells could be used to alleviate currently untreatable conditions like dementia, fertility, and spinal cord injury.

Stem cell biology is complex and unpredictable, and producing pure, viable populations of stem cell derivatives at clinical scale can be slow and challenging.

In fact, 84% of cell therapy assets fail within two years of development, and 47% of cell therapy assets still fail after the five-year point in development.

CellVoyants platform combines advanced AI with live cell imaging to address these concerns.

Its technology can extract spatial and temporal information in real-time from hundreds of millions of cells within complex cell mixtures, helping scientists understand their composition and forecast how they will evolve.

This allows them to forecast stem cell behaviour with unprecedented precision and find optimal pathways for differentiating cells into specific cell types, such as nerve, cardiac or blood, facilitating more effective development of cell therapies.

According to the company, its technology can improve target cell yields, reduce time, and can offer potential savings of $10 100 million per therapy.

Uzma Choudry, Lead Biotech Investor at Octopus Ventures, added: University spin-outs like CellVoyant are at the heart of the UKs thriving biotech ecosystem. CellVoyant sets a new standard in precision and reliability for predicting and controlling stem cell behaviour, which will make cell therapies more accessible to those who need them. We are thrilled to invest into a company that is transforming how patients can benefit from life-changing treatments.

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CellVoyant secures 7.6 million seed funding to develop stem cell therapies with AI - BioPharma-Reporter.com