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ThermoGenesis : The History of Cell and Gene Therapy – marketscreener.com

By daniellenierenberg

Cell and gene therapies are overlapping fields of research and treatments. While both aim to treat and potentially cure diseases, they have slightly differing approaches and have different historical backgrounds. Due to growing interest surrounding this field, the general public still has much to learn and understand about each of these potentially life-saving therapies.

Below, we provide a general overview and brief historical context for each type of therapy.

Cell therapyis the process of replacing damaged or dysfunctional cells with new, healthy ones by transferring live cells into a patient. These can be autologous (also known as self-to-self, using cells from the patient receiving the treatment) or allogeneic (using cells from a donor for the treatment). While this field of treatment has recently begun to expand, some forms of cell therapy like the cancer-treating hematopoietic stem cell transplantation(HSCT) have been in practice for decades.

While many people have heard of bone marrow transplants, few realize that this procedure is a stem cell therapy. While stem cells can be derived from many sources, such as umbilical cord blood and mobilized peripheral blood, bone marrow derived stem cell therapy is the most commonly used today and has been for more than 50 years.

The first transfusion of human bone marrow was given to a patient with aplastic anemia in 1939. After World War II researchers diligently worked to restore bone marrow function in aplasia patients caused by exposure to radiation produced by the atomic bomb. After a decade of work they were able to show, in a mouse model, that aplasia could be overcome by bone marrow treatment.

The first allogeneic HSCT, which led the way to current protocols, was pioneered by E. Donnall Thomas and his team at the Fred Hutchinson Cancer Research Center and reported in the New England Journal of Medicine in 1957. In this study six patients were treated with radiation and chemotherapy and then received intravenous infusion of bone marrow rich stem cells from a normal donor to reestablish the damaged or defective cells. Since then the field has evolved and expanded worldwide. While almost half of HSCT are allogeneic, the majority of HSCT are autologous, the patient's own stem cells are used for treatment, which carries less risk to the patient.

In 1988, scientists discovered that they could derive stem cells from human embryos and grow the cells in a laboratory. These newly derived stem cells, referred to as embryonic stem cells (hESCs), were found to be pluripotent, meaning they can give rise to virtually any other type of cell in the body. This versatility allows hESCs cells to potentially regenerate or repair diseased tissue and organs. Two decades after they were discovered, treatments based on hESCs have been slow in coming because of controversy over their source and concerns that they could turn into tumours once implanted. Only recently, testing has begun as a treatment for two major diseases: heart failure and type 1 diabetes.

In 2006, researchers made a groundbreaking discovery by identifying conditions that would allow some cells to be 'reprogrammed' genetically. This new type of stem cell became known as induced pluripotent stem cells (iPSCs). Since this discovery, the field has expanded tremendously in the past two decades. Stem cell therapies have expanded in use and have been used to treat diseases such as type 1 diabetes, Parkinson's and even spinal cord injuries.

There has also been a growing focus on using other immune cells to treat cancer. Therapies such as CAR T-cellare dependent upon a patient's T-cells, which play a critical role in managing the immune response and killing cells affected by harmful pathogens. These cells are then reengineered to target and kill certain cancerous cells. Several CAR T-cell therapies have been FDA approved, with the first approval being given in 2017 for Yescarta and Kymriah, to be used for the treatment of B-cell leukemia in children and young adults.

Gene therapyis a process that modifies the expression of a gene or alters the biological process of living cells for therapeutic use. This process can take the form of replacing a disease-causing gene with a new, healthy one, inactivating the mutated gene, or introducing a new gene to help the patient's body fight a disease.

While the use of gene therapy to treat humans is fairly new, the science behind it has been used in science for decades. Farmers and geneticists have collaborated for years on crop improvement using cross pollination, genetic engineering and microinjection techniques to create stronger, more resilient crops.

The first human patient to be treated with gene therapy was a four-year old girlsuffering from severe combined immunodeficiencyin 1990. She received treatment for a congenital disease called adenosine deaminase (ADA). Since then, gene therapies have been used to treat diseases such as cancer, cystic fibrosis and hemophilia.In 2017, the FDA gave its first approval of a gene therapy called Luxturna, which is used to treat patients with established genetic vision loss that may result in blindness. Gene therapies are still being studied and developed, with over 1,000 clinical trialscurrently underway.

ThermoGenesis Holdings Inc., is a pioneer and market leader in the development and commercialization of automated cell processing technologies for the cell and gene therapy fields. We market a full suite of solutions for automated clinical biobanking, point-of-care applications and large-scale cell processing and manufacturing with a special emphasis on the emerging CAR-T immunotherapy market. We are committed to making the world a healthier place by creating innovative solutions for those in need.

For more information on the CAR-TXpress multi-system platform, please contact our Sales team.

Disclaimer

Thermogenesis Holdings Inc. published this content on 13 April 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 13 April 2021 07:10:03 UTC.

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Stem cell treatment needed to fight the good fight – Victoria Lookout

By daniellenierenberg

LCol Laura Laycock on deployment.

LCol Laura Laycock

It was Oct. 7, 2019, and life was not just good, it was amazing.

My career in the Royal Canadian Air Force was going great. I loved my job and was getting promoted. Throughout my Canadian Armed Forces career of over 20years, I had represented Canada around the world with NORAD, NATO and the UN. I had married the most incredible man. We relocated to Ottawa, started to travel the world together, and were ready to start a family.

Then, on Oct. 8, 2019, everything changed.

I was diagnosed with Chronic Myeloid Leukemia(CML) after blood work for vertigo showed extremely elevated white blood cell counts. CML is a blood cancer where the bone marrow overproduces white blood cells, which eventually impairs the development of white and red blood cells and platelets. Its usually caused by a spontaneous mutation in DNA, which contains our genetic code.

LCol Laycock

Twenty years ago, researchers developed a new line of drugs that combat this overproduction of white blood cells. These targeted oral chemotherapy pills have been revolutionary in the fight against CML. Most people who take them do so for the rest of their lives and have good survival rates; however, a stem cell transplant remains the only actual cure. But its risky and not needed for most people.

Its now been about 17months since my diagnosis and my body has not tolerated this targeted chemotherapy. I fall into that small fraction of people who get debilitating or life-threatening side effects from this medication. My doctors are discussing other treatment options, one of which is a stem cell transplant, but my mixed ethnicity (European/Middle Eastern) has made it difficult to find a donor match.

My journey since my diagnosis has been to slow down and educate myself so that I can heal and advocate for my care; to appreciate every little moment of joy; and to do my best to overcome each challenge that arises. I have found strength in the extraordinary support Ive received from my family, my friends and my community, both old and new.

With the help of family and friends, I recently began a social media campaign to increase stem cell donor education and registration in Canada and around the world. Many people are unaware of the potentially lifesaving role they can play by registering to become stem cell donors. Stem cell transplants are vital treatment options for people with a range of medical conditions including spinal cord injuries, heart disease, diabetes, and some cancers.

The process to donate is simple. First, you register online with Canadian Blood Services or Hma-Qubec and do a mail-in cheek swab., and then you wait. It could be months or years before you are identified as a match. During this waiting period, you should update your contact information with the registry if it changes.

When you are matched, you will be contacted to continue with the donation process. This process is similar to giving blood, but it has its differences. The cells are usually collected intravenously from peripheral blood in a non-surgical procedure but, in rare cases, they are collected directly from the bone marrow in a surgical procedure. In either case, the risks associated with donating are minor.

In Canada, individuals aged17 to 35 can register to become stem cell donors (ages18 to 35 in Quebec). Both CBS and Hma-Qubec are part of an international network of donor registries from over 50countries. This network has a pool of over 38million donors but, unfortunately, matches are rare.

Your stem cells could potentially help others around the world, and throughout this process donor privacy is assured at all times.

LCol Laycock on her wedding day.

Stem cell matching relies on Human Leukocyte Antigen typing, which is highly influenced by ethnicity. This means that a patients best chance of finding a matching donor is from those who share similar ethnic backgrounds. Research conducted by Gragert et al.(2014) has shown that the likelihood of finding a match for certain ethnic groups can be as low as 16 percent and as high as 75 percent for others. This disparity highlights the need for more ethnically diverse stem cell donors in our registries.

Today, I am calling on my DND and CAF families to register as stem cell donors to help people, like me, who are fighting for our lives. If you arent able to register, please share this call with those who can. You, or someone you know, could be the match that saves a life a simple swab is all it takes to be a hero.

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The Governments Watchful Eye on Fraud Stemming from Stem Cell Therapy – JD Supra

By daniellenierenberg

Stem cell therapy, also known as regenerative medicine, has been around for decades, but in recent years, the use of and interest in stem cell therapy has increased exponentially. The dramatic utilization of stem cell therapy, and the increasing government spend related to these novel techniques, have now caught the eye of federal regulators and prosecutors. In this client alert, we profile some brief context of stem cell therapy, the governments regulations governing these techniques, and some of the best practices for those interested in this emerging space.

Stem cells are cells from which all other cells with specialized functions are generated (i.e., the bodys raw materials). Stem cells may duplicate themselves to create more stem cells or they may generate cells with a specific function like blood or brain cells.

Stem cell therapy is used to repair or replace damaged tissue or cells within the body. Many in the medical community are hopeful that stem cell therapy can be used to treat a wide array of conditions and diseases from multiple sclerosis to vision loss to traumatic spinal cord injuries to Lou Gehrigs disease just to name a few.

The Food and Drug Administration (FDA) oversees and regulates stem cell therapy treatments. While the FDA has acknowledged that stem cell therapy has the potential to treat diseases or conditions for which few treatments exist, there are still only a few treatments that have actually been approved by the FDA. Many treatments are still only in early investigatory stages.

The FDA has recognized the massive potential that stem cell therapy has in allowing patients treatments for various conditions. Consequently, in 2017, the FDA issued guidance indicating its intent to exercise enforcement discretion as a means to support and expedite the development of regenerative medicine products. This enforcement discretion period was to allow innovators time to determine whether to submit an Investigational New Drug (IND) or marketing application and, if such an application is needed, to prepare and submit the application as appropriate. The FDA, however, has made clear its enforcement discretion policy only applies to products that do not raise potential significant safety concerns. What the FDA considers significant is debatable, creating uncertainty and ambiguity for those who might be relying on the FDAs enforcement discretion period.

Initially, the FDA stated that its enforcement discretion period would last through November 2020. But in July 2020, the FDA extended its enforcement discretion period through May 2021 a fast-arriving date. It remains unclear whether the FDA intends to extend the time period of its enforcement discretion any further, but either way, stem cell therapy providers would be well-served by planning for and expecting enforcement efforts to ramp up in the near future.

In 2019, the FDA went to great lengths to warn consumers of the potential fraud that may arise from what it called stem cell therapy hype, and encouraged consumers to make sure any stem cell therapy treatments were either approved or being studied as an IND. The FDAs concerns have led to multiple enforcement actions, including one just last month. On February 1, 2021, for example, the government announced the indictment of Ashton Derges, a healthcare provider in Missouri, who marketed stem cell shots as a successful treatment for various conditions, including COVID-19. According to the indictment, Derges was paid nearly $200,000 by patients for the stem cell shots, none of which actually contained stem cells at all. While this alleged fraud was not particularly sophisticated, it nonetheless marked a significant development: the governments first criminal prosecution of those touting stem cell therapies.

But blatant fraud is not the only type of stem cell therapy case the government has expressed interest in investigating. A primary concern of the government is the marketing and use of unproven stem cell treatments as miracle cures. A good case study of the risks associated with aggressive marketing of stem cell therapy is a case out of Florida involving US Stem Cell Clinic Inc. The clinic was marketing stem cell therapy to treat conditions and diseases such as Parkinsons disease, stroke, and brain injuries none of which were approved by the FDA. And, much of the marketing that US Stem Cell Clinic used promised almost miraculous results. As a result, last year, the FDA successfully permanently enjoined the US Stem Cell Clinic from selling or providing those stem cell therapy treatments. Notably, this case was pursued by the FDA despite the FDA explicitly stating its intent to be lenient with emerging stem cell therapy treatments.

Stem cell therapy is a groundbreaking medical tool with great possibilities to treat a plethora of diseases and conditions. As the industry continues to expand, so will the governments interest. Our firm continues to see an uptick in cases involving stem cell therapy treatments. And we have successfully assisted clients in avoiding unnecessary scrutiny by the FDA and other government regulators.

If you are in the stem cell therapy industry or are considering offering stem cell therapy treatments, we recommend that you:

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The Governments Watchful Eye on Fraud Stemming from Stem Cell Therapy - JD Supra

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Multiple sclerosis: Recent research on causes and treatments – Medical News Today

By daniellenierenberg

Multiple sclerosis (MS) causes a wide range of symptoms involving the brain, optic nerves, and spinal cord. Research is only just beginning to reveal who is at risk and what causes the condition.

MS is a chronic condition affecting 2.8 million people worldwide. While treatment options are currently limited, trials of several new approaches are underway.

Researchers believe that MS is an autoimmune disorder. This type of illness involves the immune system attacking healthy cells, much as it would attack viruses or bacteria.

In the case of MS, the immune system attacks the myelin sheath that surrounds nerve cells. The attack causes lesions to form, and over time, these cause scarring, which leads to nerve damage and reduced function.

As a result of this damage, a person with MS may experience numbness and tingling sensations, fatigue, muscle weakness, dizziness and vertigo, memory issues, and vision problems, among other symptoms.

There are four types of MS: clinically isolated syndrome (CIS), relapsing-remitting MS, primary progressive MS, and secondary progressive MS.

CIS is a single episode of MS-like symptoms that lasts for at least 24 hours. People with CIS do not necessarily have MS, but experiencing an episode can be the first sign of the condition.

Treating MS involves interdisciplinary care, including rehabilitation, disease-modifying drugs (DMARDs), and complementary and alternative therapies.

Scientists do not fully understand the risk factors for MS and the mechanisms of the condition. However, they are making new headway in the search for answers and improvements in treatment.

What does the latest research show about the risk factors, mechanisms, and treatments of MS? In this Special Feature, Medical News Today takes a closer look.

French neurologist Jean-Martin Charcot first described the features of MS in 1868. He noted the differences between this condition and the tremor of paralysis agitans, a symptom of the neurological condition later named Parkinsons disease.

The three symptoms associated with MS at the time were called Charcots triad. They included a characteristic tremor, involuntary eye movements, also known as nystagmus, and scanning speech, which some call staccato or explosive speech.

Decades later, the invention of MRI scans helped doctors diagnose MS. Treatment with steroids became commonplace, and doctors then began to use medications in a class of drugs called interferons. The Food and Drug Administration (FDA) first approved interferons for use in people with MS in 1993.

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Although scientists and healthcare professionals understand the defining features of MS, several aspects of the condition remain a mystery.

While researchers recognize that MS is an autoimmune condition, they do not understand why immune cells attack myelin.

Also, diagnosing MS is still an ambiguous process because its symptoms are similar to those of many other health conditions.

In addition, experts do not know why women are 23 times more likely to be diagnosed with MS than men.

Research suggests that risk factors of MS include a lack of vitamin D or sunlight, smoking, obesity, a history of infection with the Epstein-Barr virus, being female, and possibly having inherited specific genes, as well as environmental factors.

More recently, the gut microbiota has emerged as a possible risk modulator.

A recent overview of clinical research found that people with MS had larger populations of Pedobacteria, Flavobacterium, Pseudomonas, Mycoplana, Acinetobacter, Eggerthella, Dorea, Blautia, Streptococcus, and Akkermansia bacteria in their intestines than people without MS.

People with MS also had reduced populations of Prevotella, Bacteroides, Parabacteroides, Haemophilus, Sutterella, Adlercreutzia, Coprobacillus, Lactobacillus, Clostridium, Anaerostipes, and Faecalibacterium bacteria.

Researchers speculate that balancing out the populations of gut bacteria in people with MS may reduce inflammation and the overactivation of the immune system.

Research from the MS Society Edinburgh Centre for MS Research found that people with MS had reduced numbers of inhibitory neurons, compared with people who did not have the condition.

However, people with MS had as many stimulating neurons as those without the condition. This was true even for people who had received their MS diagnoses many years earlier.

These findings help reveal the types of neurons affected by MS, shedding more light on how the condition evolves within the body. The research may also offer insight into treatments that could protect the targeted neurons.

DMARDs that health authorities have recently approved as MS treatments include cladribine (Mavenclad) and siponimod (Mayzent) for relapsing-remitting and active secondary progressive forms of the condition.

Cladribine targets lymphocytes, white blood cells responsible for attacks on myelin. Siponimod harnesses specific white blood cells that attack myelin and prevents them from circulating in the body.

However, due to their interactions with the immune system, these drugs may lead to a reduction in lymphocytes, making a person vulnerable to infections.

The medicines actions may also contribute to reduced responses to vaccines in people who receive routine vaccinations. With the introduction of COVID-19 vaccines, scientists have investigated whether people with MS who take medications such as cladribine can have adequate responses to vaccines.

The latest research indicates that people taking cladribine do produce protective antibodies to other common vaccines, despite having decreased lymphocyte levels induced by the medication.

This result gives scientists and others in the medical community hope that people who take these drugs for MS will have similarly adequate responses to COVID-19 vaccines.

Some scientists are currently investigating the potential for stem cell therapy for MS. In a phase 1 study conducted at the Karolinska Institute, in Stockholm, Sweden, seven people with progressive MS received infusions of stem cells derived from each participants own bone marrow.

As early as 7 days after administration of the stem cell therapy, researchers found evidence of positive changes in the participants immune systems. At 12 weeks, five out of six participants had no new characteristic lesions on follow-up MRI brain scans.

As their understanding of the condition evolves, many scientists are investigating the root cause of MS.

An analysis of the current data has revealed a possible connection between gut health and the condition. Data revealing relationships between the gut microbiota and the brain continually emerge, and scientists are hopeful that diet modifications, probiotics, and certain drugs that balance the gut microbiome will play a role in MS treatment.

Also in development are remyelination and neuroprotection therapies. The latter aim to protect the axons and myelin from further damage, while the former could restore lost function for people with MS.

Meanwhile, immunotherapy drugs would protect the nerves from destruction and rebuild neurons that have already sustained damage.

Another potential treatment in phase 1 trials is a tumor necrosis factor-alpha (TNF-alpha) inhibitor called MYMD-1. TNF-alpha is a type of cytokine produced by white blood cells that regulates some aspects of the immune system.

Overproduction of this cytokine is associated with several autoimmune conditions, including MS. MYMD-1 is a new type of TNF-alpha blocker that shows promise as a treatment for MS and other conditions.

Trials for therapies involving the gut microbiome, stem cells, neuroprotective treatments, remyelination, and MYMD-1 are still in the earliest stages. However, the possibilities provide hope that ongoing research will lead to effective ways to prevent MS and better methods of treatment.

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Multiple sclerosis: Recent research on causes and treatments - Medical News Today

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Stem Cell Therapy Market Research Reveals Enhanced Growth During The Forecast Period 2017 2025 FLA News – FLA News

By daniellenierenberg

Stem cells are found in all human beings, from the initial stages of human growth to the end of life. All stem cells are beneficial for medical research; however, each of the different kinds of stem cells has both limitations and promise. Embryonic stem cells that can be obtained from a very initial stage in human development have the prospect to develop all of the cell types in the human body. Adult stem cells are found in definite tissues in fully developed humans. Stem cells are basic cells of all multicellular animals having the ability to differentiate into a wide range of adult cells. Totipotency and self-renewal are characteristics of stem cells. However, totipotency is seen in very early embryonic stem cells. The adult stem cells owes multipotency and difference flexibility which can be exploited for next generation therapeutic options. Recently, scientists have also recognized stem cells in the placenta and umbilical cord blood that can give rise to several types of blood cells. Research for stem cells is being undertaken with the expectation of achieving major medical inventions. Scientists are attempting to develop therapies that replace or rebuild spoiled cells with the tissues generated from stem cells and offer hope to people suffering from diabetes, cancer, spinal-cord injuries, cardiovascular disease, and many other disorders.

The stem cell therapy market is segmented on the basis of type, therapeutic applications, cell source, and geography. On the basis of type, the stem cell therapy market is categorized into allogeneic stem cell therapy and autologous stem cell therapy. Allogeneic stem cell therapy includes transferring the stem cells from a healthy person (the donor) to the patients body through high-intensity radiation or chemotherapy. Allogeneic stem cell therapy is used to treat patients who do not respond fully to treatment, who have high risk of relapse, and relapse after prior successful treatment. Autologous stem cell therapy is a type of therapy that uses the persons own stem cells. These type of cells are collected earlier and returned in future. The use of stem cells is done to replace damaged cells by high doses of chemotherapy, and to treat the persons underlying disease. On the basis of therapeutic applications, the stem cell therapy market is segmented into cardiovascular diseases, wounds and injuries, musculoskeletal disorders, gastrointestinal diseases, surgeries, neurodegenerative disorders, and others. On the basis of cell source, stem cells therapy is segmented into bone marrow-derived mesenchyme stem cells, adipose tissue-derived mesenchyme stem cells, and cord blood or embryonic stem cells

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By geography, the market for stem cell therapy is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America leads the stem cell therapy market owing to rising awareness among people, early treatment adoption, and new product innovations. Europe is the second leading market for stem cell therapy due to development and expansion of more efficient and advanced technologies. The Asia Pacific stem cell therapy market is also anticipated to grow at an increasing rate owing to increasing healthcare spending, adoption of western lifestyles, and growth in research and development. Asia Pacific is the fastest growing region for stem cell therapy as several players have invested in the development of new stem cell technologies. These factors are expected to drive the growth of the stem cell therapy market globally during the forecast period.

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The major player in the stem cell therapy market are Regenexx, Takara Bio Company, Genea Biocells, PromoCell GmbH, CellGenix GmbH, Cellular Engineering Technologies, BIOTIME, INC., Astellas Pharma US, Inc., AlloSource, RTI Surgical, Inc., NuVasive, Inc., JCR Pharmaceuticals Co., Ltd., Holostem Terapie Avanzate S.r.l., PHARMICELL Co., Ltd, ANTEROGEN.CO., LTD., The Future of Biotechnology, and Osiris Therapeutics, Inc. Rising demand for advanced stem cell therapies will increase the competition between players in the stem cell therapy market.

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PM Modi Waives off Rs 6 Crore Tax on Imported Medicine for 6-month-old Baby Girl from Mumbai – News18

By daniellenierenberg

For baby Teera Kamat, who has been on the earth for a mere six months, every day has been a struggle for existence and a grim reminder to her parents about the fragile little being that needed a miracle to be saved. Mumbai-born Teera is suffering from Spinal Muscular Atrophy, a very rare medical condition that often does not let children live beyond 5 months of age and her condition requires a lot of money for the treatment.

On Wednesday, Prime Minister Narendra Modi, in a humanitarian move, decided to waive off Rs 6 crore as a GST amount against Rs 16 crore of imported medicines that are required to treat Teera. Baby Teera's parents Priyanka and Mihir Kamat have raised Rs 16 crore through crowdfunding for their daughter who needs a surgery to be cured. It also includes the cost of the medicine Zolgensma which has to be imported from the US. The tax exemption for baby's treatment amounts to at least Rs 6.5 crore and it includes 23 percent import duty and 12 percent Goods Services Tax.

The infant's parents had earlier appealed to PM Modi in October last year about Teeras medical condition and in January this year. The Leader of Opposition Devendra Fadnavis also wrote to the Prime Minister and Finance Minister Nirmala Sitaraman reiterating the request to exempt taxes on the medicine import.

It is a type of genetic disorder and a motor neuron disease that results in a person not having any control over movement of their muscles due because of the lack of nerve cells, in their spinal cord and/or brain stem.

Spinal muscular atrophy (SMA) results in muscle wasting and weakness. For someone suffering from SMA, it is very difficult to stand, walk and control their movements. Some intense forms of the SMA can also result in inability to breathe and swallow.

SMA can either occur at birth or even appear at stages of life and they can affect one's life expectancy depending upon the seriousness and the type of the SMA.

So far, there has been no cure of SMA, but certain medicines do help, such as nusinersen (Spinraza) and onasemnogene abeparvovec-xioi (Zolgensma), that help slow the disease's progress.

The types of SMA depend on when they start showing up in a patient and how the symptoms vary in them. There are basically four kinds of SMA, as National Institute of Neurological Disorders and Stroke list, which affects symptoms and life expectancy.

The first type of SMA, or Werdnig-Hoffmann disease appears before the infant is even 6 months of age. The child might be born with difficulty in breathing and the serious condition can turn fatal if there's no treatment.

Those with SMA type II will start showing symptoms of the disease usually when they are between 6 and 18 months of age. These children can sit but will not be able to walk or stand without helped and without treatment, they might just lose their power to sit as well.

Children with SMA type III or Kugelberg-Welander disease start showing symptoms after they are 18 months of age and can walk on their own. They however, experience difficultly in walking or running and other such physical exercises related to legs.

Those with SMA type IV usually develop the symptoms after they are over 21 years of age ad have minor muscle weakness and other issues. It doesn't affect one's life expectancy.

The USA Food and Drug Administration has approved the Zolgensma gene therapy for children who show the signs of the disease and are less than 2 years. Last year in August, the FDA also gave its nod to the orally-administered drug risdiplam (Evrysdi) for patients who are older than two months of age and are diagnosed with SMA.

Physical therapy, occupational therapy, and rehabilitation are some measures that can be taken to help improve posture, stop joint immobility and help in case of muscle weakness and atrophy.

You can find the link to the crowdfunding page for baby Teera here.

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PM Modi Waives off Rs 6 Crore Tax on Imported Medicine for 6-month-old Baby Girl from Mumbai - News18

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Activin A promotes the development of acquired heterotopic ossification and is an effective target for disease attenuation in mice – Science

By daniellenierenberg

Endogenous activin A in ectopic bone formation

Heterotopic ossification (HO) is the formation of ectopic bone in soft tissues at sites of injury-induced inflammation. Similar to the development of normal endochondral bone, HO is initiated by a local mass of chondrocytes that progress through chondrogenesis, osteogenesis, and mineralization to form bone tissue. Using mouse models of both subcutaneous and intramuscular HO formation and single-cell RNA sequencing, Mundy et al. found that inflammatory cells and skeletal progenitor cells initially recruited to sites of HO formation expressed Inhba, which encodes the TGF- superfamily member activin A. Treating mice with an activin Aneutralizing antibody reduced the number of chondrogenic cells at HO sites and inhibited HO formation. These results demonstrate that this ligand plays an important role in the physiological progression in these mouse models of HO and suggest that interfering with activin A signaling may be effective in patients.

Heterotopic ossification (HO) is a common, potentially debilitating pathology that is instigated by inflammation caused by tissue damage or other insults, which is followed by chondrogenesis, osteogenesis, and extraskeletal bone accumulation. Current remedies are not very effective and have side effects, including the risk of triggering additional HO. The TGF- family member activin A is produced by activated macrophages and other inflammatory cells and stimulates the intracellular effectors SMAD2 and SMAD3 (SMAD2/3). Because HO starts with inflammation and because SMAD2/3 activation is chondrogenic, we tested whether activin A stimulated HO development. Using mouse models of acquired intramuscular and subdermal HO, we found that blockage of endogenous activin A by a systemically administered neutralizing antibody reduced HO development and bone accumulation. Single-cell RNA-seq analysis and developmental trajectories showed that the antibody treatment reduced the recruitment of Sox9+ skeletal progenitors, many of which also expressed the gene encoding activin A (Inhba), to HO sites. Gain-of-function assays showed that activin A enhanced the chondrogenic differentiation of progenitor cells through SMAD2/3 signaling, and inclusion of activin A in HO-inducing implants enhanced HO development in vivo. Together, our data reveal that activin A is a critical upstream signaling stimulator of acquired HO in mice and could represent an effective therapeutic target against forms of this pathology in patients.

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Activin A promotes the development of acquired heterotopic ossification and is an effective target for disease attenuation in mice - Science

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I got stung by a stingray, and all I got was this deeper understanding of venom medicine – NOVA Next

By daniellenierenberg

Three years ago, wading in the sun-warmed waters of the Florida Keys, I felt a sharp pinch and looked down at my feet in surprise. My friend Jen and I had driven down from Miami for a weekend full of strong Cuban coffee and Hemingways six-toed cats. Tempted by water so warm and aquamarine it was almost a cliche, we had stopped to swim at a roadside beach on Bahia Honda Key. I had eased in, careful to drag my feet slowly across the seafloor in a dance known to beachgoers as the stingray shuffle, hoping to alert any local sealife to my approach. But not careful enough.

My foot throbbing, I stumbled back to the beach toward Jen, who wondered if I might have stepped on some glass. But in the next half hour, as my ankle and foot ballooned and the pain ratcheted upfrom stinging to aching, from aching to bone breakingit became clear I must have been stung by something. Then my foot started to turn blue, and we drove to the hospital.

Bahia Honda? the nurse said. Youre the fourth person to come in with a stingray sting from there today.

The pain didnt subside until the next day, when my foot had returned to its normal color. That was the start of a six-week recovery, which also involved crutches, painkillers, heavy-duty antibiotics, and a horrible rash. I wouldnt wish the experiencewhich involves a level of discomfort that some have compared to a gunshot woundon anyone. But in retrospect, its an interesting one to consider. Because, it turns out, animal venoms like the one coursing through my veins on Bahia Honda Key are sought after for drug development, with seven FDA-approved drugs derived from venom toxins on the market so far. Harnessing their power to hurt opens up a world of possibilities for healing.

The Bahia Honda beach where the author had a run-in with a stingray. Image Credit: Giuseppe Milo, Flickr (CC BY 2.0)

Chemical biologist Mand Holford, who studies venom science at her lab at Hunter College, compares what was happening in my foot in the moment after the sting to a cluster bomb. The toxins in animal venom have been engineered by evolution over many millennia to incapacitate by affecting some component in the blood, brain, or cell membranes, she says. Youre getting invaded with 200 to 300 different toxins, all trying to figure out how to reach their target, moving through and rupturing cell membranes, doing all sorts of damage.

The nurse at the emergency room told me stingrays were migrating through the area, their path bringing them close to the cove where I went wading. Stingrays deliver their venom through one or more serrated barbs that lie along their tails. While at rest, a stingray keeps its barb tucked away, immunologist Carla Lima told me in an email. But when it feels threatenedsay, by the feet of a clueless human out for a swimit pushes its tail perpendicular to its body, puncturing that humans flesh with its venom-laden spine.

Lima studies toxins in venomous fish at the Butantan Institute in So Paolo, Brazil. Her research into stingray venom has shown that whats in that venom actually changes as a stingray matures. In the freshwater species she studieswhose venom properties are better explored than the marine stingray that got methe venom of young rays tends to contain toxins that cause pain to the target. Lima hypothesizes this may be to chase predators away. In contrast, the toxins in adult venom have a necrotizing effect, meaning they destroy tissue, which would be helpful for hunting.

Peptides, short chains of amino acids that play key roles in the biological functions of all kinds of organisms, make up a large part of most animal venomsand some are only found in those venoms. Lima and other researchers have identified the peptides porflan and orpotrin as two of the elements in the freshwater stingrays toxic cocktail, along with a number of different proteases, which are enzymes that break down peptides.

As I sat cradling my foot on the beach in Bahia Honda, similar proteases and related proteins worked to break down the structure of cells in my heel, helping the venom spread further, and to prompt an inflammatory reaction that led to the swelling I saw. The peptides, on the other hand, likely caused the arteries to constrict and blood to pool, creating more inflammation and blocking circulationperhaps the cause of my foot turning blue.

A southern stingray (Dasyatis americana) cruises the ocean floor off Grand Turk Island in the Caribbean. Image Credit: Nate Madden, Shutterstock

That a substance that causes so much pain and wreaks so much biological havoc can be used in medicine is what Holford calls the yin and yang of nature. And the fact that damage and healing are, at least in this case, two sides of the same coin forms the basis for the work she does in her lab, identifying new drug applications for various components of animal venom.

Venoms have great potential to contribute to drug development because they are both potent and highly targeted, Holford says, with peptides that fit physically into cell receptors and change how those cells function. Thanks to this dynamic, venom-based drugs can work almost instantaneously. And theyre not what people in the pharmaceutical business call leaky, meaning they tend to only act on the intended cell component and dont stop at other spots along the way causing side effects.

Most stingray venom research, like Limas, takes place in areas where stingrays pose a threat to people: tropical spots like Brazil and Australia. On a drug-development level, we still dont know much about it, Lima says. But we do know a lot about other venomsin particular those created by cone snails and snakes.

For one thing, not all venom toxins cause pain. Some peptides present in snake venom focus on manipulating proteins in the wound so blood flows freely, acting as natural anticoagulants. Other peptides in Gila monster venom promote insulin production, helpful for a hungry lizard that hasnt eaten for awhile. And yet other peptides in cone-snail venom do the opposite of what stingray venom does: paralyze and suppress pain, keeping the snails prey from going into fight-or-flight mode and slowing it down until the (also slow) snail can come nab it for a snack.

This last type of venom is one of the focuses at Holfords lab. Many cone-snail venom peptides are rich in cysteine amino acids, whose structure she compares to Velcro. That makes it relatively easy for them to stick in the hourglass-shaped pores on the surface of cells that let important minerals like sodium, calcium, and potassium flow in and out. The free movement of those minerals is part of how cells talk to each other.

With those channels shut down, neurons cant communicate with one another to indicate pain. Thats what makes Prialt, the commercial version of the cone snails ziconotide peptide, an effective pain medication. Holford and her colleagues are also exploring the potential of other related cone-snail peptides to help dampen signals firing too fast in someone having a heart attack or an epileptic seizure.

She even sees possible applications here for cancer treatment. Current chemotherapy regimens dont discriminate between normal cells and tumor, she says. But because venom peptides work on specific receptorsreceptors that some tumors grow too many of as part of their developmentthey could help create a cancer drug that specifically starves cancer cells of essential minerals, stopping their growth.

The saw-scaled viper (Echis carinatus) is one of the deadliest snakes in India, and its venom is the basis of the blood-thinning drug Tirofiban. Image Credit: Sagar Khunte, Wikimedia Commons (CC BY-SA 4.0)

The venom that nearly ruined my Florida Keys vacation (though I still got to enjoy some beautiful sunsets, and the seafood was fantastic) was incredibly sophisticated, honed by evolution to inflict pain and physiological damage with laser precision. It was almost comforting to learn this in the weeks after, as I hobbled around on my crutches and watched with fascinated disgust as the wound developed a stingray-shaped blister. (My boyfriend said it was a sign I was developing superpowers, but sad to say none appeared.)

We know from nature that these peptides work, Holford says. What we dont know is massive: where they work, how they work, how effective they are. And thats a huge game of Wheres Waldo. Holford and her colleagues have come up with a protocol for finding new venom components that have potential in drug applications, then figuring out how to get them there. The first step is a practical look at the natural world: identifying which animal species are creating venom, especially venom that can be extracted manually. Next, the team uses new technologies that Holford refers to as the omics genomics, transcriptomics, proteomicsto identify the toxins within those venoms, by examining the instructions the animals' DNA and RNA contain and the proteins built by following those instructions.

From there, the team is able to use that genetic code to manufacture more of a chosen peptide in the lab, which is especially useful when it comes to studying venoms that are produced in small quantities in nature. They then test the synthetic toxin on the animals natural prey to make sure its effective and further tweak it to ensure its as specifically targeted as it can be for humans. And finally, they start to think about drug delivery. Does this drug need to cross the blood-brain barrier? Would it work if administered orally? These are essential questions, since potential drugs that cant be delivered effectively cant really be drugs at all.

Much like the experience of the sting itself, the possibilities for new drugs here are dizzying. Most venom-based drugs on the market are derived from a single peptide. But my stingrays venom (just like other naturally occurring venoms) featured hundreds of peptides. And with the advent of the omics, drug development with venom has become more efficient. Time- and resource-intensive experiments can now be run much more quickly using computer modeling, making the whole process more viable and opening up a whole world of drug prospects.

Lima and her colleagues in Brazil, for example, are continuing to explore the realm of fish venom. One synthetic peptide derived from the venom of a species of toadfish shows particular promise. A 2017 study suggested that peptide, known as TnP, has powerful anti-inflammatory and therapeutic effects in mice. Effects that could potentially help stem the autoimmune reactions that lead to spinal cord damage in patients with multiple sclerosis.

As Holford and her team navigate the new technological landscape, theyre also looking for ways to simplify their process. One innovation Holford is excited about is organoids, in this case, venom glands grown independently in a laboratory. Growing organoids would make acquiring venom samples much easier, she says, and would not require sacrificing an animal for the initial sample.

Thats especially important with climate change and habitat loss fueling a looming biodiversity collapse that could take with it undiscovered venoms with the capacity to heal. In 10 years were heading toward this major shift thats coming if we dont change our attitudes and lifestyle, she says. We could lose a lot of things on the planet that are potentially lifesaving.

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Alberta Health to cover $2.8-million gene therapy treatment on case-by-case basis – Edmonton Journal

By daniellenierenberg

And for some families this wait is excruciating, he said at a Wednesday news conference.

Shandro said there is no specific budget or cap on how much the government will spend, but that it was working with drug manufacturer Novartis to provide access. Nearly 70 per cent of children with spinal muscular atrophy type 1 do not live past age two. The drug is typically only approved for children under two.

We just dont want kids to fall through the cracks, said Shandro.

Susi Vander Wyk, executive director of Cure SMA Canada, thanked the province for making a decision she said is saving lives.

Its a fairly new treatment, so we dont know the long-term future of it, but we sure know that it has an astounding impact on these babies, she said at the news conference.

The earlier they receive the treatment, the better their prognosis, Vander Wyk said.

Time is ticking for them.

For Lana Martin, whose two-year-old son Kaysen Martin received the Zolgensma treatment in December after fundraising and an anonymous $1.4 million donation, the news was a huge step towards more kids accessing the drug.

Its still early after Kaysens treatment, but hes already more confident in his movements and doesnt tire as easily, said Martin.

He can now officially completely roll from one side of the room to the other side of the room, and he was not able to do that before, she said.

Martin said it was difficult for her and her husband, normally private people, to advocate publicly for the drugs coverage, but shes glad they did.

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Transforming optimism: finding new ways to treat rare cancers – Cancer Research UK – Science Blog

By daniellenierenberg

Cancer is an extremely complex disease. There are over 200 different types, some of which are considered common and others which are classified as rare cancers. But what exactly does it mean if a cancer is rare?

Usually, it means it only affects a small handful of people, but doctors might also call a cancer rare if it starts in an uncommon place in the body, or if the cancer is an unusual type and requires special treatment.

For secondary central nervous system (CNS) lymphoma, its an incredibly rare cancer for a combination of these reasons.

Secondary CNS lymphoma is a type of lymphoma thats spread to the brain and spinal cord nervous system after originating elsewhere in the body. And as well as being a rare cancer, secondary CNS lymphoma is an aggressive cancer, which has relatively low survival rates.

However, the latest results from the Stand Up To Cancer-funded MARIETTA clinical trial, which details a new potentially transforming treatment, has shed a glimmer of hope for patients and doctors alike.

We spoke to Dr Kate Cwynarski, who led the study in the UK, about what the latest results could mean for patients with secondary CNS lymphoma.

With a rare cancer such as secondary CNS lymphoma, finding a large enough group of patients can be a real challenge. And in cases like this, researchers have to think on a global scale.

Its a rare disease. So the reality of it is that you would not get this information if we just performed a trial in the UK, says Cwynarski. International collaboration is the only way to do it.

The MARIETTA trial is the largest study focused on patients with secondary CNS lymphoma, involving 24 centres across 4 countries and recruiting a total of 79 patients. It involved the International Extranodal Lymphoma Group (IELSG) lead by Professor Andres Ferreri in Italy and it built on the success of prior research with this group. In the UK, the trial was managed by CRUK Southampton CTU.

In particular, findings from a previous clinical trial partly funded by us, which tested treatments for primary CNS lymphoma, a lymphoma thats only found in the brain, helped inform the design of this clinical trial.

The IELSG-32 trial tested the benefits of an intensive chemotherapy regimen known as MATRIX, followed by either whole brain radiotherapy or a stem cell transplant using the patients own cells.

Cwynarski describes the IELSG-32 trial as practice changing, and its from these impressive results that the MARIETTA trial was developed. So we adapted a strategy that was successful in treating primary CNS lymphoma in the IELSG-32 trial and added another chemotherapy regimen, called R-ICE, to help treat the systemic disease on top of the secondary brain disease.

Cwynarski specialises in lymphoma, so she has treated SCNSL patients both on and off the trial. One of the big benefits of this trial, she describes, is that the inclusion criteria for the cohort more accurately reflected the patients she sees in her clinic and referral practice.

This trial included patients up to 70 years of age. And it wasnt just focused on fit, young people. So I have to say I think it was meaningful, because it included the kind of patients that we actually see.

The trial also included people regardless of when their secondary CNS lymphoma was diagnosed, whether that was when someone was originally diagnosed with lymphoma, during treatment, or after their cancer had come back.

And the results look promising. A total of 49 patients (65%) responded to the treatment in some way, with 37 people going on to have a stem cell transplant. 100% of the patients who had the stem cell transplant had not seen their cancer recur a year after registering onto the trial. We are optimistic many will be cured of this aggressive lymphoma.

But the trial also picked up differences between groups. While the regime was effective to an extent in every sub-group, the most significant results were seen in patients whose CNS disease was discovered at initial lymphoma diagnosis. Within this group, 71% of patients had lived for 2 years without their cancer growing.

A result which has never been seen before.

The results of the trial have completely transformed the teams optimism when meeting new patients. We really have identified a regimen which is intensive, but its potentially curative, concludes Cwynarski, and the word cure is not something weve really used before when talking about this disease.

Recently, Cwynarski has been busy filling out a cohort of her patients DVLA forms, confirming they are fit to drive again after being 2 years treatment free. So thats an amazing success and it was very symbolic as a reminder that these people have been alive and off all treatment for 2 years.

Moments like this are a reflection of the huge impact the MARIETTA trial has had for real people, like Maureen Brewster.

Maureen was diagnosed with lymphatic cancer of the liver in 2011 and was under the watchful eyes of a consultant during her treatment. But in the summer of 2016, I started to have very extreme headaches, says Maureen.

After getting an emergency appointment, she was taken to A&E and admitted to hospital straight away. I was transferred to the National Neurology hospital in Russell Square for a biopsy. They thought I might have had a stroke. But it wasnt. Instead, Maureen was diagnosed with a secondary cancer in her brain.

When Maureen was transferred to UCLH, she was told about the MARIETTA trial. I could have chosen not to go on the trial, but being part of it meant that I would get more examinations and monitoring. So it was more reassuring to be on the trial, she says.

Maureen during treatment.

Maureen went through 8 tough months of chemotherapy before having a stem cell transplant in the summer of 2017. During one round of chemo in the hospital I became ill with an infection and really thought I was going to die. The last chemo prior to me having stem cell transplant was so strong it really had an impact on me and I couldnt eat I felt very poorly for a few weeks.

Maureens stem cell transplant went smoothly and prior to COVID-19, she was having regular check-ups and scans in hospital.

Prior to the first lockdown in March 2020, Maureen was able to do some volunteering and also go back to work, teaching a course on Project Management at a local adult college. In April 2019 I also secured a part-time job as a User Involvement Co-ordinator. It was great to get back to that level.

Dr Cwynarski emphasises that while the trial was a great success for some, it also exposed a group of patients who didnt do so well on the treatment.

The results threw up a real disparity and uncovered an unmet need in a particular group of patients. For the group of patients whose cancer had already failed to respond to a chemotherapy treatment, known as R-CHOP, at the follow up of 2 years, only 20% had not experienced their cancer progressing or getting worse.

We need to target this cohort of patients in a different way, says Cwynarski. So really the challenge is, can we identify experimental agents be it different biological agents or immunotherapies such as CAR T cell therapy in the patients who have relapsed, and maybe bringing these therapies into the frontline.

Lilly

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Genetic Energy Boost Reverses Cellular Effects of Motor Neuron Disease – Technology Networks

By daniellenierenberg

A study examining the damage caused to nerve cells by motor neuron disease (MND) has shown that by targeting these cells energy centers, called mitochondria, neuronal function can be restored.

The research, conducted by a team at the University of Edinburgh, has been published in the journal Acta Neuropathologica.1MND is a broad term for a group of rare, progressive and sometimes fatal neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS), progressive bulbar palsy (PBP) and progressive muscular atrophy (PMA).

The research team, led by Dr Arpan Mehta, alongside Dr Bhuvaneish Selvaraj and Professor Siddharthan Chandran, all based at the University of Edinburghs Euan MacDonald Centre for MND Research, focused their work on the axon of human motor neurons. This is the region of a motor neuron that conducts electrical signals released by the brain and carries them to the body part they are intended for. In some human motor neurons, the axon can be over a meter long. These processes are energy-guzzling, and that power is provided by mitochondria, known to generations of long-suffering biology students as the powerhouses of the cell.

The scientists noted that the axons of MND-affected neurons were shorter than normal, and their mitochondria were not as easily able to move around the cell as they were in healthy neurons.

Using stem cells taken from people who have a mutation in a gene called C9orf72 that is known to play a causal role in both the MND subtype ALS and frontotemporal dementia, Mehta and colleagues created a stem cell model of MND, and sought to repair these stricken neurons.

In their stem cell models of MND, the team showed that by increasing the levels of a protein named PGC1 that regulates mitochondrial energy metabolism, the motor neurons function could be returned to healthy levels.

Dr Arpan Mehta (right), alongside Euan MacDonald MBE, co-founder of the Euan MacDonald Centre.

Our data provides hope that by restoring the cells energy source we can protect the axons and their connection to muscle from degeneration. Work is already underway to identify existing licensed drugs that can boost the mitochondria and repair the motor neurons. This will then pave the way to test them in clinical trials.

The team focused solely on the most common genetic form of ALS in their study and acknowledge that MNDs such as ALS are caused by a range of genetic and environmental factors. Nevertheless, they hope that their findings can be applied to other forms of the disease.

Reference:Mehta AR, Gregory JM, Dando O, et al. Mitochondrial bioenergetic deficits in C9orf72 amyotrophic lateral sclerosis motor neurons cause dysfunctional axonal homeostasis. Acta Neuropathol. Published online January 4, 2021. doi:10.1007/s00401-020-02252-5

Correction: This article was updated on January 25, 2021 to amend a quote from Dr Mehta.

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Novel Treatment Leads to Dog’s Recovery – The Bark

By daniellenierenberg

Life for Miro, a 5-year-old German shepherd, has been what his owner describes as an emotional roller coaster over the past two years. Several peaks and valleys have dotted his metaphorical landscape as he has gone from premiere fitness to dealing with injuries and disease. But a clinical trial at the UC Davis veterinary hospital may have put him back on a positive track.

Working as a patrol dog with his handler/owner Martin Gilbertson, a ranger with California State Parks, Miro spent three years performing duties that required him to be at the top of his game. In early 2019, he was just that, having won the top dog award for his department.

By that summer, however, things started declining for Miro. He was diagnosed with lumbosacral intervertebral disc disease that caused spinal cord compression. UC Davis veterinary neurosurgeons performed a surgical decompression, and Miro eventually recovered after a lengthy recuperation period.

Miro with his handler Martin Gilbertson

Life was great, said Gilbertson. By early December 2019, Miro was cleared to return to work. I thought all the troubles were behind us.

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It only took a few weeks, though, until the roller coaster cleared a peak and started to descend.

In late December 2019, Miro collapsed for no apparent reason and started shaking in a way Gilbertson had never seen. So, the pair returned to UC Davis where Miro was diagnosed with myasthenia gravis, a disease in which there is a malfunction in the transmission of signals between the nerves and muscles. This causes muscle weakness, and an inability to walk or run properly, as well as potentially devastating neuromuscular disorders.

Gilbertson was devastated.

To go from the pinnacle of our profession to potentially being a couch potato at best for the rest of his life was a real gut check, he said.

But hope appeared a few weeks later when Neurology/Neurosurgery Service faculty members Drs. Pete Dickinson and Bev Sturges informed Gilbertson of a myasthenia gravis clinical trial they were beginning with the help of the schools Center for Companion Animal Health (CCAH) and the Veterinary Institute for Regenerative Cures.

I thought, What do we have to lose? stated Gilbertson. Dr. Dickinson told me that Miro would be the first dog to ever receive this new treatment. We were excited and grateful to be able to participate.

A computer program shows Miro's stride pattern on the Tekscan Strideway pressure walkway.

Over the next few months, Miro received three stem cell treatments, as well as traditional medications to treat myasthenia gravis. Additionally, part of Miros recovery involved examining his gait, which utilized a new piece of equipment aimed at better analyzing a dogs stride pattern. Thanks to CCAH funding, the school recently acquired a Tekscan Strideway pressure walkway that allows clinicians and researchers to better gauge a patients step pattern and make decisions about their optimal care and recovery. To fully understand a patients gait abnormalities associated with injuries or neuromuscular diseases, veterinarians and researchers rely on objective, quantitative ways to assess locomotor function. The Strideway system complements the force plates in the schools J.D. Wheat Veterinary Orthopedic Research Laboratory, which captures extensive information, but only for one gait step. The new pressure walkway expands the capabilities to quantify pressure, vertical force, and stride parameters (timing and spacing) on all limbs for several strides during walking, trotting or landing. Miros progress was able to be tracked with pinpoint accuracy throughout his recovery.

Before the trial, Miro could only walk about 10 steps before falling down. After the trial, he seemed fully recovered, and blood tests revealed no trace of antibodies to the disease. While the disease may not be completely gone from his system, the clinical trial seems to have repressed the disease to a point where it no longer inhibits Miro from his normal activities. Retired from his job, Miro now enjoys life as a family pet.

It is true that Miro is now in remission, but until more analysis of data is completed, it is still too early to determine if the stem cells were the driving force behind his recovery, since they were administered at the same time as standard-of-care medications. Miros results are being closely examined, along with the results of two other dogs that have completed the trial, to see if this stem cells treatment truly can be considered a cure for myasthenia gravis. Regardless of the final outcome of the study, Miros recovery, in one way or another, came from a novel combination of treatments pioneered at UC Davis.

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Scientists find uncommon hereditary disorder that influences the brain, heart and facial highlights – Microbioz India

By daniellenierenberg

Researchers at the National Institutes of Health have discovered a new genetic disorder characterized by developmental delays and malformations of the brain, heart and facial features. Named linkage-specific-deubiquitylation-deficiency-induced embryonic defects syndrome (LINKED), it is caused by a mutated version of the OTUD5 gene, which interferes with key molecular actions in embryo development.

The findings indicate that the newly identified pathway may be essential for human growth and may also underlie other disorders that are present at birth. The information will help scientists better understand such diseasesboth common and rareand enhance patient care. The results were reported Jan. 20, 2021 at Science Advances.

The project began when David B. Beck, M.D., Ph.D., a clinical fellow in the laboratory of Dan Kastner M.D., Ph.D., at the National Human Genome Research Institute (NHGRI) and co-first author, was asked to consult on a male baby who had been born with severe birth defects that included abnormalities of the brain, craniofacial skeleton, heart and urinary tract.

Our discovery of the dysregulated neurodevelopmental pathway that underlies LINKED syndrome was only possible through the teamwork of geneticists, developmental biologists and biochemists from NIH,. This collaboration provided the opportunity to pinpoint the likely genetic cause of disease, and then take it a step further to precisely define the sequence of cellular events that are disrupted to cause the disease.

Achim Werner, Ph.D., Investigator, National Institute of Dental and Craniofacial Research (NIDCR) and Lead Author

An in-depth examination of siblings and family members genomes, combined with hereditary bioinformatics analyses, revealed a mutation in the OTUD5 gene as the possible cause of the problem. Through outreach to other researchers working on similar problems, Beck found seven additional males ranging from 1 to 14 years of age who shared symptoms with the first patient and had varying mutations in the OTUD5 gene.

The gene comprises instructions for making the OTUD5 enzyme, which is involved in ubiquitylation, a process which molecularly alters a protein to change its purpose. Ubiquitylation plays a part in governing cell fate, where stem cells are taught to turn into specific cell types in the early stages of embryo development.

According to the genetic evidence, I was pretty sure OTUD5 mutations caused the disease, but I did not understand how this enzyme, when mutated, led to the symptoms seen in our patients, said Beck. For this reason we sought to work with Dr. Werners group, which specializes in using biochemistry to comprehend the functions of enzymes such as OTUD5.

To begin, the NIH team analyzed cells taken from patient samples, which were processed in the NIH Clinical Center. Usually, OTUD5 edits or eliminates molecular tags on particular proteins (substrates) to modulate their function. However, in cells from patients with OTUD5 mutations, this activity was diminished.

Using a method to reunite mature human cells into the stem cell-like state of embryo cells, the scientists discovered that OTUD5 mutations were linked to abnormalities in the development of neural crest cells, which give rise to tissues of the craniofacial skeleton, and of neural precursors, cells that eventually give rise to the brain and spinal cord.

In additional experiments, the team discovered that the OTUD5 enzyme acts on a few protein substrates called chromatin remodelers. This class of proteins alters the closely packed strands of DNA in a cells nucleus to make sure genes accessible for being turned on, or expressed.

With help from collaborators led by Pedro Rocha Ph.D., an investigator in the National Institute of Child Health and Human Development (NICHD), the group found that chromatin remodelers targeted by OTUD5 help enhance expression of genes that control the cell fate of neural precursors during embryo development.

Taken together, the investigators reasoned, OTUD5 normally keeps these chromatin remodelers from being tagged for destruction. However, while OTUD5 is mutated, its protective function is lost and the chromatin remodelers are destroyed, leading to abnormal development of neural precursors and neural crest cells. Ultimately, these changes can lead to some of the birth defects seen in LINKED patients.

This implies that the mechanism we discovered is a portion of a common developmental pathway that, when mutated at different points, will result in a spectrum of disease.

We were amazed to discover that OTUD5 elicits its effects via multiple, functionally related substrates, which shows a new principle of cellular signaling during early embryonic development, said Mohammed A. Basar, Ph.D., a postdoctoral fellow in Werners lab and co-first author of this study. These findings lead us to believe that OTUD5 may have far-reaching effects beyond those identified in LINKED patients.

In future work, Werners team plans to fully investigate the role which OTUD5 and similar enzymes play in development. The researchers hope the study can serve as a guiding framework for unraveling the causes of other undiagnosed diseases, ultimately helping clinicians better evaluate and care for patients.

Were finally able to provide families with a diagnosis, bringing an end to what is often a long and exhausting search for answers, said Beck.

Source:

Journal reference:

Beck, D.B.,et al.(2021) Linkage-specific deubiquitylation by OTUD5 defines an embryonic pathway intolerant to genomic variation.Science Advances.doi.org/10.1126/sciadv.abe2116.

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New Genetic Disorder Discovered That Affects Brain and Craniofacial Skeleton – Technology Networks

By daniellenierenberg

Researchers at the National Institutes of Health have discovered a new genetic disorder characterized by developmental delays and malformations of the brain, heart and facial features.

Named linkage-specific-deubiquitylation-deficiency-induced embryonic defects syndrome (LINKED), it is caused by a mutated version of theOTUD5gene, which interferes with key molecular steps in embryo development. The findings indicate that the newly identified pathway may be essential for human development and may also underlie other disorders that are present at birth. The information will help scientists better understand such diseases both common and rare and improve patient care. The results were reported Jan. 20, 2021 inScience Advances.

Our discovery of the dysregulated neurodevelopmental pathway that underlies LINKED syndrome was only possible through the teamwork of geneticists, developmental biologists and biochemists from NIH, said Achim Werner, Ph.D., an investigator at the National Institute of Dental and Craniofacial Research (NIDCR) and lead author. This collaboration provided the opportunity to pinpoint the likely genetic cause of disease, and then take it a step further to precisely define the sequence of cellular events that are disrupted to cause the disease.

The project began when David B. Beck, M.D., Ph.D., a clinical fellow in the laboratory of Dan Kastner M.D., Ph.D., at the National Human Genome Research Institute (NHGRI) and co-first author, was asked to consult on a male infant who had been born with severe birth defects that included abnormalities of the brain, craniofacial skeleton, heart and urinary tract. An in-depth examination of siblings and family members genomes, combined with genetic bioinformatics analyses, revealed a mutation in theOTUD5gene as the likely cause of the condition. Through outreach to other researchers working on similar problems, Beck found seven additional males ranging from 1 to 14 years of age who shared symptoms with the first patient and had varying mutations in theOTUD5gene.

The gene contains instructions for making the OTUD5 enzyme, which is involved in ubiquitylation, a process that molecularly alters a protein to change its function. Ubiquitylation plays a role in governing cell fate, where stem cells are instructed to become specific cell types in the early stages of embryo development.

Based on the genetic evidence, I was pretty sureOTUD5mutations caused the disease, but I didnt understand how this enzyme, when mutated, led to the symptoms seen in our patients, said Beck. For this reason we sought to work with Dr. Werners group, which specializes in using biochemistry to understand the functions of enzymes like OTUD5.

To start, the NIH team examined cells taken from patient samples, which were processed at the NIH Clinical Center. Normally, OTUD5 edits or removes molecular tags on certain proteins (substrates) to regulate their function. But in cells from patients withOTUD5mutations, this activity was impaired.

Using a method to return mature human cells to the stem cell-like state of embryo cells, the scientists found thatOTUD5mutations were linked to abnormalities in the development of neural crest cells, which give rise to tissues of the craniofacial skeleton, and of neural precursors, cells that eventually give rise to the brain and spinal cord.

In further experiments, the team discovered that the OTUD5 enzyme acts on a handful of protein substrates called chromatin remodelers. This class of proteins physically alters the tightly packed strands of DNA in a cells nucleus to make certain genes more accessible for being turned on, or expressed.

With help from collaborators led by Pedro Rocha Ph.D., an investigator at the National Institute of Child Health and Human Development (NICHD), the team found that chromatin remodelers targeted by OTUD5 help enhance expression of genes that control the cell fate of neural precursors during embryo development.

Taken together, the researchers concluded, OTUD5 normally keeps these chromatin remodelers from being tagged for destruction. But when OTUD5 is mutated, its protective function is lost and the chromatin remodelers are destroyed, leading to abnormal development of neural precursors and neural crest cells. Ultimately, these changes can lead to some of the birth defects seen in LINKED patients.

Several of the chromatin remodelers OTUD5 interacts with are mutated in Coffin Siris and Cornelia de Lange syndromes, which have clinically overlapping features with LINKED syndrome, said Werner. This suggests that the mechanism we discovered is part of a common developmental pathway that, when mutated at various points, will lead to a spectrum of disease.

We were surprised to find that OTUD5 elicits its effects through multiple, functionally related substrates, which reveals a new principle of cellular signaling during early embryonic development, said Mohammed A. Basar, Ph.D., a postdoctoral fellow in Werners lab and co-first author of the study. These findings lead us to believe that OTUD5 may have far-reaching effects beyond those identified in LINKED patients.

In future work, Werners team plans to more fully investigate the role that OTUD5 and similar enzymes play in development. The researchers hope the study can serve as a guiding framework for unraveling the causes of other undiagnosed diseases, ultimately helping clinicians better assess and care for patients.

Were finally able to provide families with a diagnosis, bringing an end to what is often a long and exhausting search for answers, said Beck.

Reference: Beck DB, Basar MA, Asmar AJ, et al. Linkage-specific deubiquitylation by OTUD5 defines an embryonic pathway intolerant to genomic variation. Sci Adv. 2021;7(4):eabe2116. doi:10.1126/sciadv.abe2116.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Motor neurone disease: Researchers dare to hope existing drugs can reverse the deadly nerve damage – The New Daily

By daniellenierenberg

Have researchers in Scotland truly found a way to reverse the damage done to nerve cells by motor neurone disease (MND)?

A new study suggests that it all comes down to boosting the energy output from the mitochondria, the cells power supply.

How can that be done? Possibly with drugs that already exist.

The researchers, from the University of Edinburgh, are already looking for existing drugs that boost mitochondrial function and may be able to be repurposed to treat MND.

Overall, this is a startling development, albeit one that has to be tempered with wait-and-see caution. There are thousands of people around the world, sufferers and their families, desperate for even a glimmer of good news.

People with MNDprogressively lose the use of their limbs and ability to speak, swallow and breathe, whilst their mind and senses usually remain intact.

The average life expectancy is two and a half years.

Motor neuron cells are nerve cells that control movement. An axon is the long part of the motor neuron cell that connects to the muscle and it can be up to a metre long.

The starting point in the study was examining axonal dysfunction a common problem in neurodegenerative disorders, including in amyotrophic lateral sclerosis (ALS), a form of MND.

For axons to function properly, they need a lot of energy. The scientists wondered if the production of energy in the cells is put out of whack by MND. And if that was true, could it be fixed and if so, could the axon be restored to full function?

The researchers from the Euan MacDonald Centre for MND Research at the University of Edinburgh used stem cells taken from people with the C9orf72 gene mutation that causes both MND and frontotemporal dementia.

They discovered that, in these human stem cell models of MND, the axon was shorter than in healthy cells.

The study then found the back-and-forth movement along the axon was impaired.

And then, bingo! Once the mitochondrial performance was boosted, the axon think of a stunted tree suddenly getting water and nourishment regained its healthy function. Back to normal.

This then allowed the mitochondria to travel freely along the axon.

The study also examined human post-mortem spinal cord tissue from people with MND. The tissue was obtained from the Medical Research Council Edinburgh Brain and Tissue Bank.

These examinations supported the findings from the stem cells.

The first film shows mitochondria travelling along an axon in a healthy motor neuron:

data-s="video/mp4">

This second film shows how the mitochondria becomes stalled as it attempts to travel along damaged motor neuron with the C9orf72 gene:

data-s="video/mp4">

In this third film we see a damaged motor neuron with the C9orf72 gene restored to function after boosting the mitochondria:

data-s="video/mp4">

Dr Arpan Mehta, the Lady Edith Wolfson Fellow and a PhD student at the University of Edinburgh, led the study. In a prepared statement he said:

The importance of the axon in motor nerve cells cannot be understated. Our data provides hope that by restoring the cells energy source we can protect the axons and their connection to muscle from degeneration.

Work is already underway to identify existing licensed drugs that can boost the mitochondria and repair the motor neurons. This will then pave the way to test them in clinical trials.

Although the research focused on the people with the commonest genetic cause of the ALS (amyotrophic lateral sclerosis) type of MND, researchers are hopeful that the results will also apply to other forms of the disease.

By the way: Frontotemporal dementia is a consequence of progressive damage to the frontal and/or temporal lobes of the brain.The right and left frontal lobes at the front of the brain are involved in mood, social behaviour, attention, judgement, planning and self-control.

Hence, damage can lead to reduced intellectual abilities and changes in personality, emotion and behaviour.

More than 2000 people have MND in Australia, of whom 60 per cent are male and 40 per cent female, according to figures from MND Australia.

The mean time from onset to confirmation of diagnosis is 10 to 18 months, and approximately 58 per cent of people with MND are under 65.

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Stem cells on the ballot – Science Magazine

By daniellenierenberg

California's ballot measures often reveal much about the broader U.S. policy environment. This is particularly true of the approval by the state's voters in November of Proposition 14, The California Stem Cell Research, Treatments, and Cures Initiative of 2020. Proposition 14 extends the 2004 ballot Proposition 71, which established the California Institute for Regenerative Medicine (CIRM) and authorized $3 billion in state-issued bonds for CIRM to fund stem cell and regenerative research and medicine (restricted to California). Proposition 14, which authorizes $5.5 billion over the next 10 years to continue CIRM's work, succeeded in part by informing voters of CIRM's successes and that its conflict-of-interest provisions are extremely strong. This state-level action is critical because, contrary to opponents' opinions, the overall policy environment for human stem cell research in the United States is in some ways worse now than when Proposition 71 passed.

Since 2004, CIRM has funded groundbreaking work on immune disorders, cancer, spinal cord injury, diabetes, and more. The result has been more than 90 stem cellrelated clinical trials (directly or indirectly supported by CIRM), almost 3000 scientific papers, and contributions to two cancer therapies approved by the U.S. Food and Drug Administration. The lives of many patients have improved because of CIRM. Notably, many CIRM-funded clinical trials rely on human embryonic or fetal stem cells, whereas the federal government currently does not fund any clinical trials using these types of cells.

Proposition 71 was motivated largely in response to restrictions on human embryonic stem cell research in the United States in 2004. However, although research was limited to a small number of human embryonic stem cell lines, there was no formal ban on federal funding of research on such stem cells. In addition, in 2004 there were no restrictions on federal funding of human fetal stem cell and tissue research; however, there is now near-complete blockage of federal funding for such research. And federal funding for human embryonic stem cell research is again at risk. On 4 September 2020, 22 Republican senators and 72 Republican House members wrote to President Trump requesting an end to all federal funding of human embryonic stem cell research. Could President Trump impose a ban that would be difficult to revoke? Or, could Republican senators manufacture a ban by legislative maneuvering on a budget reconciliation vote, which requires 60% support? Such maneuvering created the effectively permanent 1995 Dickey-Wicker amendment, which prohibits federal funding of any research in which human embryos are created or destroyed. Dickey-Wicker has limited research on in vitro fertilization methods and stalled progress on understanding early human development. It has not solved the problem of the many, perhaps 1 million frozen embryos in the United States that will not be used for in vitro fertilization and will be destroyed without benefit if not used for research. Vital long-term research is greatly harmed by the U.S. policy environment, with the likely outcome that many young scientists will avoid research using human embryonic stem cells and human fetal tissue.

Restrictions on valuable, ethical research appear particularly fool-hardy during a deadly pandemic. Research on viruses such as HIV and SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2) can benefit greatly from work using mice that utilize human fetal stem cells and tissues to generate a human-like immune system. These mice allow evaluation of a human immune system in the contexts of infection mechanisms, generation of immunity, and drug response. These studies can be supported with Proposition 14 funds in California, but not with federal funds. It is crucial for the incoming Biden administration to evaluate the need for federal funding in these important areas with high-quality scientific input and evidence.

California's vote on Proposition 14 should also help the rest of the country appreciate the need to increase investments in biomedical research at the U.S. National Institutes of Health and other federal agencies. Current biomedical research expenditures amount to only a tiny fraction of the costs of disease, so an objective evaluation of appropriately increased research funding relative to disease costs is warranted. Once again, California is showing the way.

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New gene therapy methods deliver promise – Spectrum

By daniellenierenberg

Special delivery: Stem cells can be modified to produce a therapeutic protein in the brain.

Laguna Design / Science Photo Library

Two unpublished studies detail improved methods for delivering gene therapies to the brain: One involves a type of stem cell that can produce gene-altering proteins on-site; the other taps an engineered virus to target neurons efficiently and noninvasively.

Researchers presented the work virtually on Monday and Tuesday at the 2021 Society for Neuroscience Global Connectome.

One of the biggest hurdles for targeted gene therapy is getting enough treatment to the right spot. In the first study, researchers overcame this obstacle by developing stem cells that produce a therapeutic protein inside the brain.

The team is using the approach to develop a treatment for Angelman syndrome, which is caused by mutation in or deletion of the maternal copy of the gene UBE3A. Because the paternal copy of the gene is typically silent, loss of the maternal copy results in an absence of UBE3A protein. People with Angelman syndrome usually have intellectual disability and motor impairments, and many are autistic.

The researchers had previously used modified stem cells to produce a protein that can activate the paternal copy of UBE3A. Transplanting the cells into the brains of Angelman syndrome model mice boosts levels of UBE3A protein, they found. However, the treatment required multiple direct injections into the animals brains.

In the new work, they instead tried injecting the cells into a pocket of cerebrospinal fluid at the base of the skull an approach that is less invasive and can be performed multiple times. They compared the results with direct injection into the animals hippocampus. In both cases, the mice had UBE3A expression in the brain for up to three weeks.

Mice that received direct injection of the stem cells had fewer Angelman syndrome traits than controls, as measured by their motor skills.

This suggests that though the new route is effective, it may not provide a high enough dosage, says Peter Deng, a postdoctoral researcher inKyle Finkslab at the University of California, Davis, who presented the work. And because the transplanted cells produce protein for only a limited period of time, the effects are temporary a limitation the team is addressing.

Deng and his colleagues also found that monkeys treated with the stem cells had the therapeutic protein throughout their brain and spinal cord three weeks after injection, which suggests the approach has potential for treating people.

The second approach presented at the conference improves the delivery of a more permanent form of gene therapy that uses adeno-associated viruses (AAVs).

Researchers typically inject these viruses directly into the brain, and the viruses usually only affect cells immediately surrounding the injection site.

Youre required to use a ton of the virus to penetrate the whole brain, says Jerzy Szablowski, assistant professor of neuroengineering at Rice University in Houston, Texas, who presented the work.

One potential workaround is to inject the AAV into the blood and use focused ultrasound to temporarily open up the blood-brain barrier, allowing the AAV to cross into the brain. Sometimes with this approach, however, the virus also inserts itself into other organs.

In their new work, the team developed AAVs that more easily cross the blood-brain barrier and more selectively target neurons than previous versions do. As a result, the new AAVs can be given in lower doses, reducing the amount of tissue affected outside the brain, Szablowski says.

To identify the most efficient AAV, Szablowski and his colleagues designed 2,100 new viruses, injected them all into the bloodstream of mice and applied focused ultrasound to the animals skulls. The mice had been engineered so that AAVs that successfully inserted themselves into a neuron got tagged with a marker. The team performed genomic sequencing on the mouse brains a few weeks later and read out the levels of viruses.

Compared with the previously most effective AAV, the top five newly identified AAVs targeted twice as many cells in the brain (including more neurons), and nearly half as many cells outside the brain, the researchers found.

The approach could be used to more efficiently deliver treatments for conditions such as Angelman syndrome or Parkinsons disease, the team says.

Read more reports from the 2021 Society for Neuroscience Global Connectome.

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Glioblastoma Tumors Triggered by the Healing Process of Brain Injury – Science Times

By daniellenierenberg

The growth ofglioblastoma tumors can be linked to the healing process that follows a brain injury, the researchers in Canada said. They believe that mutations derailed the new cells generated during the healing process in brain injuries, such as trauma and infection or stroke, that were supposed to replace the lost cells.

The researchers said that their data suggest that right mutations in particular brain cells could be modified by injury, which would create tumors. Dr. Peter Dirks, Head of the Division of Neurosurgery and a Senior Scientist in the Developmental and Stem Cell Biology program at SickKids, said that glioblastoma could be thought of as a wound that never heals.

They think that by studying the origins of glioblastoma, they can know how cancer originates and grows as it opens up new ideas about cancer treatment. Thestudywas headed by Dirks, molecular genetics professor Dr. Gary Bader, and senior scientist Dr. Trevor Pugh.

(Photo: Wikimedia Commons)Glioblastoma multiforme - MRT T1 axial mit Kontrastmittel. Histologie bioptisch gesichert.

According to Mayo Clinic, glioblastoma is an aggressive type of cancer that can either be found in the brain or the spinal cord that forms cells called astrocytes that support nerve cells. Although it can occur at any age, glioblastoma more common in older adults.

Unfortunately, options for treating glioblastoma are still limited, and patients have an average lifespan of 15 months after diagnosis. The researchers said that it is mostly because of the extensive heterogeneity observed within tumors as they harbor diverse cells, such as the glioblastoma stem cells.

Dirk's team believes that glioblastoma stem cells are responsible for tumor growth and recurrence after treatment. After a series of tests, they confirmed that each tumor contains multiple subpopulations of cancer stem cells, making its recurrence more likely that existing therapies cannot wipe away.

Moreover, they found that glioblastoma stem cells were comingled with the cancer stem cells within the tumors, which indicates that glioblastoma is starting to form when the healing process begins as new cells replace the lost cells due to injury. Dirks said that once the mutant cell becomes involved in the healing process, it can no longer stop multiplying, spurs tumor growth.

ALSO READ: Mobile Phones Caused Brain Tumour: Italian Court Rules

Further in their study, the researchers also classified two distinct molecular states that the tumors exhibited, Genetic Engineering & Biotechnology Newsreported. These are the "Developmental" and "Injury Response" states.

The Developmental state represents a hallmark of the glioblastoma stem cells, which is similar to the rapidly dividing stem cells in the growing brain of an infant after birth. On the other hand, the Injury Response state showed an increase in the number of immune pathways and inflammation makers that indicate a healing process.

Moreover, additional experiments established that the two states are at risk of various types of gene knockouts, which means that there are many therapeutic targets linked to inflammation that had not been previously linked to glioblastoma cells' growth.

The researchers found that the two states were patient-specific, which could lean toward the Developmental state of Injury Response state. Researchers are looking into these biases to make tailored therapies that are effective on different points of the two states.

READ MORE: Brain Tumor Vaccine: Combination Therapy Offers Promising Survival Results For Glioblastoma Brain Cancer Patients

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Hemostemix steps into the new year with capital and its critical clinical study data in hand – InvestorIntel

By daniellenierenberg

With a new management team spearheading Hemostemix Inc. (TSXV: HEM | OTC: HMTXF), the Company started 2021 with its critical clinical study data in hand. Raising over $4 million in 2020 and then in December adding an additional $4 million to the coffers ($2.75 million at a 50% premium), Hemostemix completed a 1-for-20 share consolidation as it charges into the New Year.

Receiving a copy of its entire clinical trial database relating to the clinical trial for Critical Limb Ischaemia (CLI) using its ACP-01 therapy (Angiogenic Cell Precursors) in November 2020 was a key event for Hemostemixs management team and it garnered real interest from the market.

Hemostemix Platform for Stem Cell Therapies

Based in Calgary and founded in 2006, Hemostemix is a clinical-stage biotechnology company specializing in blood-derived stem cell therapeutics with its lead product (ACP-01) in Stage 2 clinical trials for the treatment of CLI.

CLI is a disease caused by the narrowing of arteries in the limbs, particularly the legs, hands, and feet, causing chronic pain and soreness. Untreated CLI can sometimes require the amputation of the specific limb.

Stem cell treatments have been used for over 30 years to treat people with cancer conditions such as leukemia and lymphoma.

There are two main types of stem cell transplants: allogeneic and autologous. In an allogeneic stem cell transplant procedure, the patient receives stem cells from a donor. In an autologous stem cell transplant procedure, the patient provides themselves the stem cells for the procedure from various sources, including bone marrow or blood.

Hemostemixs autologous stem cell therapy platform uses the patients own blood to harvest the stem cells and the treatment helps to restore circulation in the damaged tissues.

Hemostemix has a strong intellectual property (IP) portfolio of 91 patents and has treated more than 500 patients with clinical results showing an improvement in 83% of the patients receiving its ACP-01 stem cell therapy.

Advantages with Hemostemixs process include the use of blood, which is safer and less invasive than extracting bone marrow, and since you are using the patients own blood, there is no immune rejection.

The clinical trials have shown that ACP-01 is safe and effective in the treatment of CLI. Now that Hemostemix has received the entire clinical trial database, it has entered into a contract with a new Clinical Research Organization (CRO) to complete the midpoint statistical analyses of the efficacy of ACP-01 and expects to publish the results this quarter.

Hemostemix Not a 1-Trick Pony Company

ACP-01 has the potential to treat other conditions such as Angina, Ischemic & Dilated Cardiomyopathy, and Peripheral Artery Disease (PAD). Currently, Hemostemix is preparing for Phase 2 trials for the treatment of Angina and is seeking joint-venture partners to fund the other Phase 2 trials.

Hemostemix has also developed NCP-01 (Neural Cellular Precursor) from blood with the potential, through building new neuronal lineage cells in a patient, to treat Alzheimers disease, Amyotrophic Lateral Sclerosis (ALS), Parkinsons disease, spinal cord injuries, and stroke-related issues. NCP-01 is currently in the R&D phase and is pre-clinical.

Market Size

According to the American Heart Association, Cardiovascular disease (CVD) accounted for approximately 1 of every 3 deaths in the United States in 2019.

Factors that increase the risk of CLI include diabetes, high cholesterol levels, high blood pressure, obesity, or smoking, all risk factors also associated with CVD.

Unfortunately, most of these factors are increasing at an alarming rate a study by the Centers for Disease Control and Prevention (CDC) in the United States, showed the prevalence of diagnosed diabetes has more than doubled from 3.3% in 1995 to 7.40% in 2015, affecting 23.4 million Americans.

According to a market research report released in 2019, the value of just the global CLI treatment market is projected to reach US$5.39 billion by 2025, up from US$3.13 billion in 2018, at an annual growth rate of 8%.

Competitive Landscape and Market Cap Comparisons

Even with Hemostemixs recent market surge, its market cap is only C$32.5 million. Similar-sized biotech companies focusing on CLI trade much higher.

Cynata Therapeutics Limited (ASX: CYP) is an Australian biotechnology company with a Phase 2 clinical-stage trial for its stem cell therapy for CLI using bone marrow and has a market cap of C$93.6 million.

Pluristem Therapeutics Inc. (NASDAQ: PSTI) is a Phase 3 bio-therapeutics company, based in Israel, that also has an allogeneic cell therapy for the treatment of CLI using the placenta and has a market cap of C$231.9 million.

In November 2020, Bristol-Myers Squibb Company (NYSE: BMY) bought MyoKardia, Inc. for US$13.1 billion. MyoKardia was a clinical-stage biopharmaceutical company that developed therapies for the treatment of cardiovascular diseases and its lead product was a Phase III clinical trial drug used in the treatment of hypertrophic cardiomyopathy (HCM).

As a company shifts from Phase 2 to Phase 3 clinical trials, the market cap often has a step-function shift higher, making it an ideal time to look at Hemostemix.

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Stem Cell Assay Market Competitive Landscape Analysis with Forecast by 2025 – SoccerNurds

By daniellenierenberg

Stem Cell Assay Market: Snapshot

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues andtumors, wherein their toxicity, impurity, and other aspects are studied.

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With the growing number of successfulstem cell therapytreatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

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Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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