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Veru Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)

By Global News Feed

MIAMI, Oct. 04, 2022 (GLOBE NEWSWIRE) -- Veru Inc. (NASDAQ: VERU), a biopharmaceutical company focused on developing novel medicines for COVID-19 and other viral and ARDS-related diseases and for the management of breast and prostate cancers, today announced the granting of inducement awards to seven new employees. In accordance with NASDAQ Listing Rule 5635(c)(4), the awards were made as a material inducement to the seven employees’ entry into employment with the Company.

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Veru Reports Inducement Grants Under NASDAQ Listing Rule 5635(c)(4)

Roche to present new data from its expanding neuromuscular disease portfolio at World Muscle Society 2022

By Global News Feed

Basel, 05 October 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its industry-leading neuromuscular portfolio will be presented at the World Muscle Society (WMS) congress, 11th-15th October 2022. These data demonstrate Roche’s commitment to advancing clinical understanding and supporting the development of treatments for people living with neuromuscular disorders.

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Roche to present new data from its expanding neuromuscular disease portfolio at World Muscle Society 2022

Stem Cells Therapy for Autism: Does it Work?

By Somatic Stem Cells

Most of us are familiar with the scientific fact that any living, breathing animal, insect etc. is made up of cells. These cells form tissues and organs that support the existence of the host. Many of us have also heard of stem cells therapy for autism but are unsure about its validity.

Scientists have studied the underlying mechanism of cells, as well as their functioning, and have discovered ways of using the cells to improve the lives of humans and treat diseases. To do so, scientists have discovered stem cells; think of it as the building blocks of a fully differentiated cell.

Stem cells are human cells that can be developed and differentiated into other cell types. These cells can be derived from any part of the body, for example, stem cells from the brain, muscle, bone marrow, etc. Stem cells are versatile in that they can be used to fix damaged tissues. The two essential characteristics of stem cells include: Firstly, the ability to self-renew to create successors identical to the original cell. Secondly, stem cells, unlike cancer cells, are controlled and highly regulated, therefore, stem cells need to be able to give rise to specialized cell types that become part of the healthy body.

The purpose of stem cell therapy is to regenerate and repair damaged tissues and cells in the body. There are two main classes of stem cells. Pluripotent stem cells have the potential to become any cell in the adult body and multipotent stem cells are much more restricted to a specific population or lineage of cells. Other stem cell types include totipotent and unipotent.

Lets look at pluripotent and multipotent stem cells in detail.

Pluripotent stem cells are generated from somatic cells. These mainly come from embryos and, as such, theyre often referred to as embryonic stem cells.

Lets discuss three types of embryonic stem cells that are used to generate pluripotent cells. These include true embryonic stem cells (ES), nuclear transfer of somatic cells (ntES), and parthenogenetic embryonic stem cells (these are stem cells from unfertilized eggs).

The true embryonic stem cells are made from unused embryos, such as those that undergo IVF (in vitro fertilization). The process of IVF is such that the eggs and sperm are fertilized in a lab dish. What then happens is that, through this process, more embryos are generated, usually more than the couple actually need. Those that arent used can be donated to science.

Pluripotent cells made from these unused embryos are not genetically matched to the original hosts. These are mainly used in science for studies to learn how stem cells regenerate.

Every cell contains an organelle called a nucleus. The nucleus contains all the cells genetic information essential to its function. The word somatic refers to any cell in the body.

The process of somatic cell nuclear transfer (SCNT) extracts the nucleus from a somatic cell and transfers it into another cell that has had its own nucleus removed; i.e. the nucleus from the previous cell is being transferred to an egg cell that does not contain a nucleus (unnucleated).

When the nucleus is transferred to another cell, it activates the process of pluripotent cell generation that reprograms the generation of genes in that cell. The egg then becomes a zygote nucleus or a fertilized egg, the cell then replicates and through it embryonic stem cells are created.

Imagine being able to fertilize an egg without fertilization by sperm. Unusual, but science makes crazy things happen.

Parthenogenesis is the process whereby an unfertilized egg develops an embryo without fertilization. This can be achieved through chemical, physical or combined activation methods.

The parthenogenetic embryonic stem cells have the capacity for infinite proliferation and self-renewal, and maintain the ability to differentiate into one or more specialized types of cell or tissue.

pESCs are especially useful for regenerative medicine, and therefore allow the generation of functional cells that could potentially be used as treatment for many incurable diseases in the future.

Multipotent stem cells are unspecialized cell types that have the ability to self-renew and differentiate into specialized cell types. However, these cells are specific to the type of tissue or organ. For example, a multipotent adult stem cell from the bone marrow can become specialized to produce all blood cell types; and cells in the stem cells from neural networks in the brain can specialize to glial and neuronal cells.

When we talk about all blood cell types, we have to get a little scientific, but for the curious mind, all blood cell types refers to platelets, B and T lymphocytes, natural killer cells, dendritic cells.the list goes on.

In addition, for the curious mind, various types of stem cells include hematopoietic stem cells (the ones that make blood cell types), mesenchymal stem cells (differentiate into bone, fat, cartilage, muscle, and skin), and neural stem cells (from neural networks).

Now that weve covered the types of stem cells, the question remains, can stem cell therapy cure autism? Lets have a look.

To answer this question, I refer to the review by Price (2020) as it is the latest up-date data on this subject. It is important to note however, that at the time of reading this article there may be other research data published on this topic.

Several research studies cite immune dysfunction as the cause and effect of autism spectrum disorder (ASD). By virtue of this analogy, it has informed the basis of the stem cell therapy approach for treating autism. This is founded on the properties that regulate the immune system (immuno-regulatory properties).

From the review, it was also found that when exposed to inflammatory stimuli, this may lead to the development of postnatal diagnosis of ASD. Inflammation to the cell describes the process that occurs when the cell is exposed to harmful stimuli such as bacteria, trauma, toxins, heat, and pathogens. The affected cells then release chemicals that cause blood vessels to leak fluid into the tissues, causing swelling.

Therefore, an inflammatory stimuli is that which influences the occurrence of an inflammatory response.

Other bodies of research found an altered level of proteins called cytokines which are essential for interaction and communication between cells in ASD. These may also be the cause of the development of autism spectrum disorder. Some genetic studies propose an association between a genetic loci (a specific point on the genome of the autistic individual) and ASD whose function is related to immune function. While others suggest a possible anomaly in the neuronal signaling pathway that directs communication and information transfer between neurons

All these are proposed reasons that hypothesize the use of stem cell therapy to treat autism biologically. However, all these propositions do not lead to one voice, there are too many hypotheses that make it difficult to narrow down the target area that would potentially treat autism or autism symptoms. Keeping in mind that autism traits are diverse, therefore, narrowing this information down to one plausible pathology is an even greater challenge.

So, is stem cell therapy effective? The answer to this is unknown.

Is ASD caused by genetic, immune dysfunction, or inflammatory stimuli? The answer to this is not clear and theres a vast number of studies that argue different theories.

It is even more disturbing to consider these hypotheses because, for example, each person can experience bacterial or viral infections, or stress that can impact immune functioning and/or lead to inflammation but were not all on the spectrum. Therefore, we cant say that factors which alter our immune functioning lead to the development of neurodevelopmental conditions.

However, according to Price, the study by Riordan et al. (2019) proposes the influence of cytokines for the treatment of autism.The data proposed could be a point in a positive direction to answering whether stem cell therapy could potentially treat autism symptoms.

Unfortunately, there is no data to positively state the effectiveness of stem cell therapy for treating autism. As more research is developed in this field, theres hope that more understanding of autism will arise, and perhaps an alternative form of treatment of autism symptoms can be developed. It is also worth noting the possibility of genetic markers that could help diagnose autism during pregnancy or during the prenatal development stage.

The studies highlighted in this article are simply preliminary assessments. Further research needs to be conducted in order to understand the potential of cell therapies for treating autism.

The findings of these studies vary in hypothesis and this makes generalization hard. Science has developed greatly over years, therefore, for those that believe in the potential of science and all that it could offer, theres a reason to hope that stem cell therapy could potentially be used as treatment for autism in the near future.

Biehl, J. K., & Russell, B. (2009). Introduction to stem cell therapy. The Journal of cardiovascular nursing, 24(2), 98105. https://doi.org/10.1097/JCN.0b013e318197a6a5

Price, J.(2020). Cell therapy approaches to autism: a review of clinical trial data. Molecular Autism, 11, 37 . https://doi.org/10.1186/s13229-020-00348-z

http://stemcell.childrenshospital.org/about-stem-cells/adult-somatic-stem-cells-101/where-do-we-get-adult-stem-cells/

Thermo Fisher Scientific. An Overview of Pluripotent and Multipotent Stem Cell Targets. https://www.thermofisher.com/za/en/home/life-science/antibodies/antibodies-learning-center/antibodies-resource-library/antibody-methods/pluripotent-multipotent-stem-cell-targets.html

Yu, Z., Han, B. (2016). Advantages and limitations of the parthenogenetic embryonic stem cells in cell therapy. Journal of Reproduction and Contraception, 27 (2), Issue 2, 118-124. https://doi.org/10.7669/j.issn.1001-7844.2016.02.0118

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Stem Cells Therapy for Autism: Does it Work?

Long-Lasting Effects: Spaceflight Linked to an Increased Risk of Cancer and Heart Disease – SciTechDaily

By Somatic Stem Cells

The researchers believe it is important to continuously screen the blood of astronauts throughout their careers and during their retirement in order to monitor their health.

A groundbreaking study from the Icahn School of Medicine at Mount Sinai found that astronauts are more likely to develop mutations, potentially connected to spaceflight, that raise astronauts lifelong risk of acquiring cancer and heart disease.

Researchers took blood samples from astronauts who served on space shuttle missions between 1998 and 2001 for the National Aeronautics and Space Administration (NASA). All 14 astronauts were found to have DNA alterations, or somatic mutations, in the blood-forming system (hematopoietic stem cells). Their research, which was recently published in the journalCommunications Biology, raises the possibility that these mutations may be brought on by spaceflight and highlights the importance of routine blood testing for astronauts throughout their careers and during retirement to keep an eye on their health.

Somatic mutations are mutations that occur after conception and in cells other than sperm or egg cells, meaning they cannot be passed on to children. The mutations uncovered in this study were characterized by an excess of blood cells produced from a single clone, a process known as clonal hematopoiesis (CH). Such mutations are commonly brought on by environmental causes, such as exposure to UV radiation or certain chemicals, and may develop as a consequence of chemotherapy or radiation treatment for cancer. There are few symptoms or signs of CH; most individuals are identified via genetic testing of their blood for other disorders. Although CH is not always a sign of disease, it is linked to an increased risk of blood cancer and cardiovascular disease.

An infographic describing the research process. Credit: Communications Biology / Mount Sinai Health System

Astronauts work in an extreme environment where many factors can result in somatic mutations, most importantly space radiation, which means there is a risk that these mutations could develop into clonal hematopoiesis. Given the growing interest in both commercial spaceflights and deep space exploration, and the potential health risks of exposure to various harmful factors that are associated with repeated or long-duration exploration space missions, such as a trip to Mars, we decided to explore, retrospectively, somatic mutation in the cohort of 14 astronauts, said the studys lead author David Goukassian, MD, Professor of Medicine (Cardiology) with the Cardiovascular Research Institute at Icahn Mount Sinai.

The study subjects were astronauts who flew relatively short (median 12 days) space shuttle missions between 1998 and 2001. Their median age was approximately 42 years old; roughly 85 percent were male, and six of the 14 were on their first mission. The researchers collected whole blood samples from the astronauts 10 days before their flight and on the day of landing, and white blood cells only three days after landing. The samples were stored at -80C for approximately 20 years.

Using DNA sequencing followed by extensive bioinformatics analyses, researchers identified 34 mutations in 17 CH-driver genes. The most frequent mutations occurred in TP53, a gene that produces a tumor-suppressing protein, and DNMT3A, one of the most frequently mutated genes in acute myeloid leukemia. However, the frequency of the somatic mutations in the genes that the researchers assessed was less than two percent, the technical threshold for somatic mutations in hematopoietic stem cells to be considered clonal hematopoiesis of indeterminate potential (CHIP). CHIP is more common in older individuals and is associated with an increased risk of developing cardiovascular disease and both hematologic and solid cancer.

Although the clonal hematopoiesis we observed was of relatively small size, the fact that we observed these mutations was surprising given the relatively young age and health of these astronauts. The presence of these mutations does not necessarily mean that the astronauts will develop cardiovascular disease or cancer, but there is the risk that, over time, this could happen through ongoing and prolonged exposure to the extreme environment of deep space, Dr. Goukassian said. Through this study, we have shown that we can determine the individual susceptibility of astronauts to develop disease related to their work without any implications that can affect their ability to do their work. Indeed, our studies demonstrate the importance of early and ongoing screening to assess that susceptibility. Our recommendation is that NASA, and its medical team, screen astronauts for somatic mutations and possible clonal expansion, or regression, every three to five years, and, not less importantly, well into their retirement years when somatic mutations may expand clonally and become CHIP.

The teams research follows previous studies that used the same samples to identify predictive biomarkers in exosomessmall lipid-layered microscopic vesicles of nucleic acids, proteins, lipids, and metabolites that form within the cells of the human body and are subsequently released into the blood circulation, hence carrying the information from their cells of origin that reflects their intercellular condition. This feature of exosomes may qualify them as great biomarkers of health and/or disease, as well as transfer information from one cell to another at a great distance in the body. When they treated human heart cells with exosomes derived from astronauts, the researchers found that the exosomes affected the biology of the vitamin D receptor, which plays a key role in bone, heart, and skeletal muscle health. They also assessed the impact of space flight on mitochondrial DNAthe genome of small organelles that supply energy to cells. In that study, the team found that the amount of cell-free mitochondrial DNA circulating in the blood of astronauts was two to 350 times higher than normal, which may lead to oxidative damage and inflammation elsewhere in the body.

Through these studies, we have demonstrated the potential to assess the health risk of space flight among astronauts. What is important now is to conduct longitudinal retrospective and well-controlled prospective studies involving a large number of astronauts to see how that risk evolves based on continued exposure and then compare that data against their clinical symptoms, imaging, and lab results. That will enable us to make informed predictions as to which individuals are more likely to develop disease based on the phenomena we are seeing and open the door to individualized precision medicine approaches to early intervention and prevention, said Dr. Goukassian.

Reference: Retrospective analysis of somatic mutations and clonal hematopoiesis in astronauts by Agnieszka Brojakowska, Anupreet Kour, Mark Charles Thel, Eunbee Park, Malik Bisserier, Venkata Naga Srikanth Garikipati, Lahouaria Hadri, Paul J. Mills, Kenneth Walsh and David A. Goukassian, 17 August 2022, Communications Biology. DOI: 10.1038/s42003-022-03777-z

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Long-Lasting Effects: Spaceflight Linked to an Increased Risk of Cancer and Heart Disease - SciTechDaily

Stem cell treatment after spinal cord injury: The next steps – Mayo Clinic

By Stem Cell Treatment

June 27, 2020

Following promising phase 1 testing, Mayo Clinic is launching phase 2 of a randomized clinical trial of stem cell treatment for patients with severe spinal cord injury. The clinical trial, known as CELLTOP, involves intrathecal injections of autologous adipose-derived stem cells.

"The field of spinal cord injury has seen advances in recent years, but nothing in the way of a significant paradigm shift. We currently rely on supportive care. Our hope is to alter the course of care for these patients in ways that improve their lives," says Mohamad Bydon, M.D., a neurosurgeon at Mayo Clinic in Rochester, Minnesota.

The first participant in the phase 1 trial was a superresponder who, after stem cell therapy, saw significant improvements in the function of his upper and lower extremities.

"Not every patient who receives stem cell treatment is going to be a superresponder. Among the 10 participants in our phase 1 study, we had some nonresponders and moderate responders," Dr. Bydon says. "One objective in our future studies is to delineate the optimal treatment protocols and understand why patients respond differently."

In CELLTOP phase 2, 40 patients will be randomized to receive stem cell treatment or best medical management. Patients randomized to the medical management arm will eventually cross over to the stem cell arm.

Study participants must be age 18 or older and have experienced traumatic spinal cord injury within the past year. The spinal cord injuries must be American Spinal Injury Association (ASIA) grade A or B.

The initial participant in CELLTOP phase 1 sustained a C3-4 ASIA grade A spinal cord injury. As described in the February 2020 issue of Mayo Clinic Proceedings, the neurological examination at the time of the injury revealed complete loss of motor and sensory function below the level of injury.

After undergoing urgent posterior cervical decompression and fusion, as well as physical and occupational therapy, the patient demonstrated improvement in motor and sensory function. But that progress plateaued six months after the injury.

Stem cells were injected nearly a year after his injury and several months after his improvement had plateaued. Clinical signs of efficacy in both motor and sensory function were observed at three, six, 12 and 18 months following the stem cell injection.

"Our patient also reported a strong improvement with his grip and pinch strength, as well as range of motion for shoulder flexion and abduction," Dr. Bydon says.

Spinal cord injury has a complex pathophysiology. After the primary injury, microenvironmental changes inhibit axonal regeneration. Stem cells can potentially provide trophic support to the injured spinal cord microenvironment by modulating the inflammatory response, increasing vascularization and suppressing cystic change.

"In the phase 2 study, we will begin to learn the characteristics of individuals who respond to the therapy in terms of their age, severity of injury and time since injury," says Anthony J. Windebank, M.D., a neurologist at Mayo's campus in Minnesota and director of the Regenerative Neurobiology Laboratory. "We will also use biomarker studies to learn about the characteristics of responders' cells. The next phase would be studying how we can modify everyone's cells to make them more like the cells of responders."

CELLTOP illustrates Mayo Clinic's commitment to regenerative medicine therapies for neurological care. "Our findings to date will be encouraging to patients with spinal cord injuries," Dr. Bydon says. "We are hopeful about the potential of stem cell therapy to become part of treatment algorithms that improve physical function for patients with these devastating injuries."

Bydon M, et al. CELLTOP clinical trial: First report from a phase I trial of autologous adipose tissue-derived mesenchymal stem cells in the treatment of paralysis due to traumatic spinal cord injury. Mayo Clinic Proceedings. 2020;95:406.

Regenerative Neurobiology Laboratory: Anthony J. Windebank. Mayo Clinic.

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Stem cell treatment after spinal cord injury: The next steps - Mayo Clinic

Regenerative Spine and Pain Institute: Treating Pain with PRP and Stem Cell Therapy – Community News

By Stem Cell Treatment

Pain.

It gnawsat you. It drains you. It becomes the focus of your life.

Experiencing a few pain-free moments can be euphoric; it makes you realize how long youve been living with aches and pain. You might wonder how you can find a solution to relieve the pain and regain your freedom from discomfort.

Regenerative Spine and Pain Institute on how to lesson your pain.

Dr. Ronak Patel at Regenerative Spine and Pain Institute wants you to know there are two new revolutionary answers to pain relief.

Both platelet-rich therapy otherwise known as PRP and stem cell therapy give patients new hope by using the bodys powerful healing power to accelerate the battle against pain. Dr. Patel has seen incredible success implementing these cutting-edge treatments on hundreds of patients suffering from pain-related issues.

So if you are suffering fromany of the ailments below, theres a lifeline.

Heres the best news: Neither PRP or stem cell therapy involves drug use with side effects or any surgical procedures.

Both PRP and stem cell treatments use the bodys own healing resources to repair diseased or damaged tissue and the results are quite remarkable.

PRP therapy involves injecting concentrated platelets and growth factors into damaged tissue to stimulate the faster growth of new healthy cells. Platelets are cells that prevent and stop bleeding. If a blood vessel is damaged, the body sends signals to our platelets to get on the job and start the healing. Some call platelets the bodys natural bandage.

So how does PRP therapy work? Its basically drawing a one small vial of blood from the patient and then using a centrifuge to turn it into a potent and concentrated form of platelets. It is then injected back into the patient. Think of it as a boost of your own blood only superpowered.

Recovery time for PRP therapy is far shorter than for surgery. Patients usually experience soreness for a week or so, but the gradual improvement soon begins. Unlike a steroid shot, which gives you immediate relief and quickly wears off, a PRP patient will see pain symptoms improve over a period of months, and up to 80 percent of patients will see relief for up to two years.

Stem cell therapy can be an even more powerful way to harness the bodys healing power. Stem cells are the building blocks for every cell in our body. These powerful cells can be harvested to produce powerful new cells to fight inflammation and disease.

For those suffering from osteoarthritis, stem cell therapy has proven very effective. Thats because the stem cells may help develop new cartilage cells and suppress inflammation. Stem cells can be harvested through a sample of body fat or bone marrow or be harvested from donated umbilical cord tissue.

And yes, you can even augment PRP therapy with stem cell therapy for an even bigger boost!

Stop wondering if youll have to live with your pain forever. Contact Regenerative Spine and Pain Institute today at 609-269-4451 or go to http://www.njpaindoc.com to book an appointment and learn more.

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Regenerative Spine and Pain Institute: Treating Pain with PRP and Stem Cell Therapy - Community News

Are immunotherapy and chemotherapy the same thing? How cancer treatments work – Nebraska Medicine

By Stem Cell Treatment

As cancer treatments continue to advance and new therapies are introduced, it's easy to get lost in your search for information. To help you better understand the differences between specific cancer treatments and how they work, we spoke with medical oncologist Bhavina Sharma, MD, MPH.

"Chemotherapy are drugs designed to directly attack all rapidly dividing cells in the body, including cancer cells," explains Dr. Sharma. "It relies on the idea that cancer cells reproduce much faster than most healthy cells in our body."

Chemotherapy drugs can be given by infusion or in pill form. Unfortunately, these drugs can't tell the difference between cancerous cells and fast-growing healthy cells like the gastrointestinal tract and hair follicles, leading to side effects such as diarrhea and hair loss. Thankfully, recent advancements in chemotherapy have helped lessen side effects such as nausea, pain and lethargy.

Targeted therapy are special drugs designed to target differences within cancer cells that help them thrive. Unlike chemotherapy, targeted therapy drugs actually change the inner workings of the cancer cell. Because targeted therapy focuses on the part of the cancer cell that makes it different from the normal, healthy cell, it often has fewer side effects than standard chemotherapy treatments.

Immunotherapy is very different than chemotherapy in that it helps our immune system to find and kill cancer cells.

"Cancer cells are abnormal cells that have formed in our body because of cell damage or mutations," explains Dr. Sharma. "Cancer cells hide from your immune system by shutting down certain pathways of the immune response. Immunotherapy unlocks those pathways so your immune system can recognize and remove the cancer cells."

Cellular therapies are treatments that improve the body's ability to fight cancer. "Stem cell therapy falls under the umbrella of cellular therapy," explains Dr. Sharma. "It uses stem cells to mount an immune response to attack your cancer cells."

Stem cells from blood and bone marrow can be used in transplants. These stem cells can either come from a matched donor (allogeneic) or from the patient themselves (autologous).

Chimeric antigen receptor therapy or CAR T-cell, is a type of cellular therapy.

"T cells are white blood cells that help our bodies fight infection and cancer," explains Dr. Sharma. "With CAR T-cell therapy, your own T cells are collected from your blood. These T cells are modified to recognize cancer as a foreign cell and attack it."

CAR T-cell therapy has been approved by the Food and Drug Administration to treat lymphoma, leukemia and multiple myeloma.

Hormone therapy slows or stops the growth of cancer that uses hormones to grow. It is also called hormonal therapy, hormone treatment or endocrine therapy. Hormone therapy is recommended for cancers that are hormone-receptor positive, such as certain breast and prostate cancers. It can't be used in cancers that don't carry hormone receptors.

"Hormone therapy can be used for both early stage and metastatic hormone-receptor positive breast cancers," explains Dr. Sharma. "In patients with early-stage breast cancer, it is used after surgery to help reduce the risk of the cancer coming back."

Chemotherapy, immunotherapy, targeted therapy, and hormone therapy are just a few of the treatments we use to treat cancer. Many of these cancer treatments can be combined with others like cancer surgery and radiation therapy. Every person's journey through cancer is different. Your oncology team will help you sort through the best therapies available to create your treatment plan.

The information in this article is for information purposes only. For specific questions regarding your medical condition or treatment plan, please consult with your doctor directly. To schedule an appointment with a Nebraska Medicine cancer specialist, call 402.559.5600.

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Are immunotherapy and chemotherapy the same thing? How cancer treatments work - Nebraska Medicine

ANDREWS MEDICINE AND CELLTEX THERAPEUTICS CORPORATION JOIN FORCES TO BRING CUTTING EDGE TECHNOLOGIES TO ATHLETIC INJURIES – BioSpace

By Stem Cell Treatment

HOUSTON, Oct. 4, 2022 /PRNewswire/ -- James R. "Jim" Andrews, MD, Chairman and CEO of Andrews Medicine of Pensacola, Florida, and David G. Eller, Chairman and CEO of Celltex Therapeutics Corporationof Houston, Texas, announced today that together they will form a new biotechnology company to bridge the divide between stem cell research and the current treatment of athletic injuries. The jointly owned company will operate under the name of "Andrews Celltex Biologics".

The new company will undertake the operation of a Sports Injury Program that includes the Celltex-sponsored FDA developmental study associated with its Investigational New Drug (IND) for the use of Celltex-produced autologous Mesenchymal Stem Cells (MSCs) in the treatment of orthopedic indications. Andrews Medicine will participate in this FDA observed IND research study and will be responsible for selection of participants and administration for this new study. The new venture promises to bring the best medical orthopedic practices of Andrews Medicine for treatment of sports injuries with best practices of Celltex for production of therapeutic quality autologous MSCsas a regenerative component of the treatment. Andrews Celltex Biologics will break new ground with this combined effort. The goal is an FDA approved Biologic License for this Celltex product.

Dr. Andrews said: "I have always known that stem cells, when properly handled, can add value to already effective treatments of inflammatory conditions caused by injury or disease. I am delighted that the FDA has been working with Celltex for studying the safety and efficacy of this combination of Celltex-produced MSCs with traditional medical treatment of multiple sports injury indications. Our know-how and network of sports teams and physicians will add immediate value to our new joint company. I am convinced that Andrews Celltex Biologics will be a leader in this huge growth sector of health care coupled with regenerative medicine."

"Celltex is excited about the opportunity to join forces with one of the best orthopedic and sports medicine companies in the country," said David G. Eller, Chairman and CEO of Celltex. "It is a perfect match. Surgical treatments coupled with Celltex-produced regenerative MSCs will improve recovery time and remediation. This joint effort of the two leading companies in their field will bring cutting edge regenerative health care for sports injuries and beyond."

David Eller continued, "Over the past 11 years, Celltex has developed unique know-how and proprietary technology to produce, in its specially designed cGMP lab, millions of MSCs from a small extraction of a person's adipose tissue. The integrity and vibrancy of the MSCs are of therapeutic quality. Over one trillion MSCs have been produced by Celltex, in its cGMP lab, from individuals wanting their own stem cells reintroduced into their bodies by medical doctors to fight a variety of diseases, especially those caused by inflammatory conditions. Given the autologous nature of Celltex-produced stem cells, there have been no severe adverse events when these stem cells have been reintroduced into the individuals from whom the small, one-time adipose tissue sample had been extracted."

ABOUT ANDREWS MEDICINE OF PENSACOLA, FLORIDA

Andrews Medicine is an integrated healthcare platform built on five decades of research, innovation, and clinical expertise led by internationally recognized orthopaedic surgeon, Dr. James "Jim" Andrews. Widely known as the surgeon for elite athletes from around the world, Dr. Andrews is also a pioneering thought leader in the field of research, injury prevention, new surgical techniques, and practice management. Andrews Medicine brings this experience and expertise to healthcare systems, medical providers, sports organizations, and the communities they serve, ensuring that every patient has access to exceptional clinical care. Read more at: http://www.andrewsmedicine.com

ABOUT CELLTEX THERAPEUTICS CORPORATION

Founded in 2011, Celltex Therapeutics Corporation is a Houston, Texas-based biotechnology company that specializes in the manufacturing and cryopreservation of Mesenchymal Stem Cells (MSCs).Celltex is a leading commercial provider of autologous MSCs for therapeutic use. The Company uses its proprietary technology to isolate, cryopreserve and culture billions of undifferentiated and genetically stable MSCs in its state-of-the-art current Good Manufacturing Practices (cGMP)-compliant laboratory.The Company is dedicated to pioneering technological breakthroughs in regenerative medicine. For more information see: http://www.celltexbank.com

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SOURCE Celltex Therapeutics Corporation

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ANDREWS MEDICINE AND CELLTEX THERAPEUTICS CORPORATION JOIN FORCES TO BRING CUTTING EDGE TECHNOLOGIES TO ATHLETIC INJURIES - BioSpace

The Second Person in History Has Been Functionally Cured of HIV – Twisted Sifter

By Stem Cell Treatment

Researchers have achieved remission for an HIV patient through stem cell transfusion, only the second person in history to report a functional cure.

Functional cures, as opposed to eradicated cures, mean that strains of the virus could still be present in the body but are inactive and undetectable. In 2007, Timothy Ray Brown, known as the Berlin Patient underwent the same procedure and has remained HIV-free since. The new London Patient received a double stem cell transfusion.

Successful HIV treatments are rare, as drug-resistant strains have become widespread, and while stem cell transfusions have achieved positive results, they are complex and difficult to replicate on a wide scale. Blood donors are rare because they must have inherited the 32 mutation of the CCR5 gene from both parents, as this mutation is naturally resistant to HIV-1. The odds of having two parents with this mutation is like winning the genetic lottery.

There are other reasons for the infrequency of positive results. In Browns case, he already had one 32 mutation. Also, both functionally cured patients had cancer diagnoses in addition to HIV. Doctors believe that chemotherapy aided the blood transfusions because it temporarily destroys fast-dividing cells, leaving room for replacement.

While stem cell transfusions as an HIV treatment can be expensive, dangerous, and rare, the remission results from the London Patient are encouraging for doctors and researchers. And for the 38 million people currently living with HIV, any option for a life-saving cure is a reason for hope.

Categories: SCI/TECH Tags: Berlin Patient, CCR5 gene, chemotherapy, drug-resistant, eradicated cure, functional cure, genetic mutation, HIV, HIV treatment, London Patient, stem cell transfusion, stem cells, Timothy Ray Brown, top

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The Second Person in History Has Been Functionally Cured of HIV - Twisted Sifter

Companies to Seek BLA Approval of Gene-editing Therapy for SCD |… – Sickle Cell Anemia News

By Stem Cell Treatment

Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy exagamglogene autotemcel known as exa-cel seeking its approval for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia.

The U.S. Food and Drug Administration (FDA) cleared Vertexand CRISPR to begin a rolling submission of exa-cels BLA a formal request therapy manufacturers make for regulatory approval to introduce a new biologic product in the market.

That submission is planned to begin this November, with completion likely to occur by the end of the first quarter of 2023.

We are pleased to have concluded our exa-cel pre-submission meetings with regulators and are excited that FDA has granted a rolling review, Nia Tatsis, PhD, executive vice president and chief regulatory and quality officer at Vertex, said in a press release.

A rolling submission means the companies can submit sections of the application for review as soon as they are ready, rather than waiting until each and every section is complete prior to submission, as is typically done.

CRISPR and Vertex gained access to this type of review after they met with the FDA as part of a fast track program that aims to speed a therapys development and get it to patients sooner.

SCD and beta thalassemia are two genetic diseases that occur when the body makes either a faulty version of hemoglobin, or none or too little of it. Hemoglobin is the protein in red blood cells that is responsible for oxygen transport.

Not having enough proper hemoglobin causes anemia, which occurs when there is a shortage of red blood cells in the blood.

Exa-cel, formerly known as CTX001, uses hematopoietic stem cells, or blood cell progenitors, that are taken from a patients own peripheral blood. These cells are modified in the lab in such a way that they make high levels of fetal hemoglobin. This version of hemoglobin, produced during fetal development, is more effective at carrying oxygen than its adult counterpart.

When these cells are given back to the patient, in the form of a stem cell transplant, they are expected to drive the production of fetal hemoglobin. This in turn is expected to help ease anemia, lower the need for blood transfusions, and reduce the frequency of painful vaso-occlusive crises (VOCs) that occur in SCD.

The modification is made with the aid of the gene-editing tool CRISPR/Cas9. It uses an RNA molecule that guides an enzyme to a specific point in the DNA sequence of a gene of interest. The enzyme cuts open the DNA sequence at a specific point and removes some of its building blocks.

In exa-cel, this is done in a gene that provides instructions to make BCL11A, a protein that shuts off the production of fetal hemoglobin some time after birth. The modification stops BCL11A from being made, turning the production of fetal hemoglobin back on.

The therapy is being tested in multiple clinical trials as a potential one-time therapy for patients with either SCD or beta thalassemia.

We continue to work with urgency to bring forward the first CRISPR therapy for a genetic disease, and one that holds potential to transform the lives of patients with sickle cell disease or beta thalassemia, said Tatsis.

Data from an open-label Phase 1/2/3 clinical trial, called CLIMB121 (NCT03745287), showed that a single dose of exa-cel increased the levels of fetal hemoglobin and prevented VOCs in SCD patients. The ongoing trial, now fully enrolled, involves patients ages 1235 with severe SCD.

The reported side effects were consistent with those of busulfan, a medicine that is used as part of a conditioning treatment regimen carried out in preparation for the stem cell transplant.

A similar clinical trial, CLIMB111 (NCT03655678), is evaluating the safety and efficacy of one-time exa-cel in patients with beta thalassemia of about the same age.

Two other open-label Phase 3 clinical trials CLIMB151 (NCT05329649) and CLIMB141 (NCT05356195) are enrolling patients ages 511 with SCD or beta thalassemia at two locations in the U.S. and Italy. There are plans to extend recruitment to patients as young as age 2 at a later date.

All patients who participated and received exa-cel in any of these clinical trials will have the chance to enter CLIMB-131 (NCT04208529), a follow-up study that will evaluate the long-term safety and efficacy of the therapy for up to 15 years.

In addition to fast track status, exa-cel received regenerative medicine advanced therapy (RMAT) and orphan drug designations from the FDA.

By the end of this year, the companies plan to submit an application requesting marketing approval of exa-cel in Europe. The therapy holds orphan drug status from the European Commission and priority medicines (PRIME) designation from the European Medicines Agency.

Read more here:
Companies to Seek BLA Approval of Gene-editing Therapy for SCD |... - Sickle Cell Anemia News

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