Category Archives: Stem Cell Treatments

Africa July 2022. Currently, umbilical cord blood stem cells are being used to treat blood cancers, inherited blood disorders, and certain metabolic and immune disorders. These diseases are prominent in South Africa, yet access to cord blood stem cells is limited.

Dr. Yvonne Holt is Chief Medical Officer of Netcells Stem Cell Bank the largest established private umbilical cord blood bank in Africa. Dr. Holt acknowledges that great strides have been made internationally with regard to cord blood banking and stem cell treatment. However, its use is still not fully accessible in South Africa.

July is Cord Blood Awareness Month. The company aims to educate the public regarding the importance of umbilical cord blood storage and how it can save lives.

Dr. Holt explains that having access to cord blood provides an important source of stem cells, which can treat many life-threatening illnesses such as blood disorders such as Thalassaemia, sickle cell anemia, aplastic anemia; cancers such as leukemia, lymphoma, and solid tumors of childhood; metabolic and immune disorders.

The umbilical cord blood stem cells are mainly haematopoietic (blood-forming) stem cells. They are therefore used in these illnesses to create new, healthy bone marrow after chemotherapy.

Internationally, cord blood stem cells can be sourced from public banks that collect cord blood from voluntary donations. Since we dont have a local public stem cell bank in South Africa, it puts South African patients at a greater disadvantage, as bringing in cord blood units from overseas is prohibitively expensive.

In the past, the costs associated with private stem cell banking has posed a barrier. However, the launch of Netcells Community Stem Cell Bank in partnership with the South African Bone Marrow Registry (SABMR) the only bank of its kind in the country offers parents the opportunity to store their babys cord blood at a significantly reduced rate.

The cord blood unit can then be used by the family themselves. Alternatively, it can be used by someone else in the community who may need a lifesaving transplant. If the cord blood unit is utilized by someone else, then all costs associated with the storage are refunded to the family.

This will greatly improve transplant options for SAs diverse demographic, as it can be difficult to find a bone marrow stem cell match for patients of African origin given the lack of donors from these communities.

Its important to educate all South Africans regarding the importance of building a donor base that can serve all members of our community, says Dr Holt.

Alternatively, parents can choose to go the private cord blood banking route. They will pay the full price to have their babys cord unit stored exclusively for their child or familys use.

Dr. Holt says the process of collecting blood from a newborns umbilical cord, is easy and painless.

Once collected, it is tested, frozen, and stored in the cord blood bank.

Cord blood stem cells are used to replace dysfunctional blood-producing cells in bone marrow with healthy new cells. Once diseased cells are destroyed by chemotherapy and irradiation, new stem cells are transplanted into the patient to restore normal functioning of the bone marrow.

Netcells also runs a sibling cord blood donor program, called Families of Hope. It aims to improve poor and disadvantaged families access to stem cell banking. This is especially important if they have a child with a stem cell-treatable disease.

If a disadvantaged child requires a bone marrow stem cell transplant, and their mother is expecting a child from the same father, we will facilitate the collection of the cord blood, process, test and store the cord blood until it is needed for transplant in the ill sibling.

In this way, we hope to expand the opportunity to families who are faced with a life-threatening disease but cannot afford to save their babys precious stem cells at birth, she says.

The first cord blood transplant was done in the late 80s. Since then, there have been remarkable advances. In fact, there have been more than 40 000 cord blood transplants done worldwide.

Cord blood stem cells are also proving useful in new areas of regenerative medicine to potentially treat cerebral palsy, autism, and Type 1 diabetes. If the potential of stem cell research is realised, it could lead to breakthroughs in treatments for many diseases we face today.

Researchers are also continuing to look for ways to improve transplant success and toincrease the number of stem cellscollected from each cord, which could potentially reduce the costs associated with cord blood transplants in the future.

Cord blood is by no means regarded as a miracle cure. However, it provides an important source of stem cells to treat several life-threatening illnesses. It also has the potential to treat many more common diseases in the future as technology progresses.

For now, collecting your babys stem cells remains a valuable insurance policy for you and your loved ones.

For more info regarding cord blood banking, visit Next Biosciences.

Dr. Yvonne Holt

Yvonne Holt graduated from the University of the Witwatersrand in 1997 with a Bachelor in Medicine and Surgery (MBBCH Wits). Dr. Holt worked as a Medical Officer in the departments of Paediatrics and Obstetrics and Gynaecology for 4 years after graduation. She obtained a Diploma in Child Health (DCH) during this time.

In 2006, Dr. Holts interest in the fields of Obstetrics and Gynaecology, Paediatrics, and stem cell technology brought her to a new career path. She joined Next Biosciences as Medical Director.

In this capacity, Yvonne is responsible for the medical development of the stem cell program at Next Biosciences. She also highlights the benefits of this new technology. In 2011, Yvonne completed a 2 year, internationally recognized Diploma in Transfusion Medicine through the University of Bloemfontein.

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Stem Cells Are Needed To Treat Life-Threatening Diseases - Longevity LIVE - Longevity LIVE

A stem cell hair transplant is similar to a traditional hair transplant. But rather than removing a large number of hairs to transplant to the area of hair loss, a stem cell hair transplant removes a small skin sample from which hair follicles are harvested.

The follicles are then replicated in a lab and implanted back into the scalp in the areas of hair loss. This allows hair to grow where the follicles were taken from, as well as where theyre transplanted.

Stem cell hair transplants exist only in theory at the moment. Research is ongoing. Its estimated that stem cell hair transplants may be available by 2020.

Stem cells are cells that have the potential to develop into different types of cells found in the body. Theyre unspecialized cells that are unable to do specific things in the body.

However, theyre able to divide and renew themselves to either stay stem cells or become other types of cells. They help repair certain tissues in the body by dividing and replacing damaged tissues.

A stem cell hair transplant was successfully performed by Italian researchers in 2017.

The procedure begins with a punch biopsy to extract stem cells from the person. The punch biopsy is performed using an instrument with a circular blade thats rotated into the skin to remove a cylindrical sample of tissue.

The stem cells are then separated from the tissue in a special machine called a centrifuge. It leaves a cell suspension thats then injected back into the scalp in the areas of hair loss.

There are several different research teams working on stem cell hair loss treatments. While the procedures may vary slightly, theyre all based on growing new hair follicles in a lab using a small skin sample from the patient.

Currently, there are some clinics offering a version of stem cell hair transplants to the public. These arent approved by the U.S. Food and Drug Administration (FDA). Theyre considered investigational.

In 2017, the FDA released a warning about stem cell therapies. The warning advises anyone considering stem cell therapies to choose those that are either approved by the FDA or being studied under an Investigational New Drug Application (IND). The FDA authorizes INDs.

These procedures are performed in-office on an outpatient basis. They entail removing fat cells from the persons abdomen or hip using a liposuction procedure under local anesthesia.

A special process is used to remove the stem cells from the fat so that they can be injected into the scalp. This procedure takes approximately 3 hours.

The clinics that currently offer this procedure cant provide a guarantee for the outcome of the procedure. The results, if any, can vary from person to person. It may require several treatments over many months to see results.

Some research has found stem cell hair transplants can be effective in treating different hair loss conditions, including:

Some pain following the procedure is expected. It should subside within a week.

No recovery time is required, though excessive exercise should be avoided for a week. Some scarring can be expected where the fat has been removed.

You wont be able to drive yourself home following the procedure because of the effects of the local anesthesia.

Theres very little information available about the possible side effects of stem cell hair transplants. As with any medical procedure, theres always the risk of bleeding or infection at the site of the sample and the injection. Scarring is also possible.

Though complications from a punch biopsy are rare, theres a small risk of damage to the nerves or arteries beneath the site. Liposuction can also cause the same side effects and complications.

The research available on the success rate of stem cell hair transplants is very promising. The results of the Italian study showed a 29 percent increase in hair density 23 weeks after the last treatment.

The clinics that currently offer stem cell hair therapies not approved by the FDA dont make any guarantees in regard to results or success rates.

The cost of stem cell hair transplants hasnt been determined since theyre still in the research stages.

Some of the investigational stem cell hair replacement therapies being offered by various clinics range from approximately $3,000 to $10,000. Final cost depends on the type and extent of the hair loss being treated.

The stem cell hair transplant treatments being researched are expected to be available to the public by 2020. Stem cell hair transplants offer options to people who arent candidates for the hair loss treatments currently available.

While some clinics are offering stem cell hair replacement therapies, these are considered investigational and havent been approved by the FDA.

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Stem Cell Hair Transplant: What Is It and When Will It Be Available?

What are stem cells?

Our bodies are made up of about 200 different types of cells, such as muscle cells, skin cells and nerve cells. Each cell type performs a specialised function. Stem cells are cells that are not specialised. Their role is to replace specialised cells that have been lost through injury, diseases, or the normal course of events.

Medical research into stem cells is still at the very early stages. Our understanding of the risks associated with stem cell treatment is limited. Further research is required to create safe and effective treatments.

Are stem cell treatments safe and effective?

In Australia, the only proven safe and effective stem cell treatment is haematopoietic stem cell transplantation (using stem cells from umbilical cord blood or bone marrow) for the treatment of disorders of the blood and immune system such as leukaemia.

Currently, no other stem cell treatment has been demonstrated to be safe and effective. However, some clinics located both in Australia and overseas offer unproven stem cell treatments.

Any unproven stem cell treatment may be unsafe, posing serious risks to your health. The risks include infection, allergic reactions or the development of cancer; complications that can be fatal.

What stem cell treatments are approved by the TGA?

The only established stem cell treatment approved in Australia is haematopoietic stem cell (HPC) transplantation (using stem cells from umbilical cord blood or bone marrow), which is standard practice for the treatment of disorders of the blood and immune system such as leukaemia. The use of fresh bone marrow-derived HPC is standard practice and is exempt from oversight by TGA. Cord-blood derived HPC is usually stored frozen prior to use, so involves some processing, and is approved by TGA.

What are the risks of undergoing unproven stem cell treatments?

An unproven stem cell treatment may also pose serious, potentially fatal, risks to your health including infection, allergic reactions, rejection of the cells by your immune system and the development of cancer.

Unproven stem cell treatments may cause financial hardship, including treatment and ongoing follow-up costs, as well as the cost of emergency medical care in the event that something goes wrong. If you undergo treatment outside Australia, there are additional costs associated with travel.

Importantly, undergoing an unproven stem cell treatment may interfere with proven and potentially beneficial therapies recommended by your general practitioner or specialist. It can also disqualify you from participation in a registered clinical trial.

Does the TGA regulate stem cell treatments?

In Australia, the Therapeutic Goods Administration (TGA) is responsible for the regulation of products for therapeutic use including human cells and tissues (termed 'biologicals'). This includes human stem cell treatments.

However, the TGA does not regulate medical practice. Some products that would otherwise be considered biologicals are excluded from TGA regulation (through the Therapeutic Goods (Excluded Goods) Determination 2018). Under this provision stem cell treatments may not be subject to regulation by TGA if they meet all of the following criteria:

Where one or more criteria are not met, including advertising to consumers, regulation by TGA will apply.

Are there any approved facilities for storing stem cells in Australia?

Cord blood can be stored in Australia. There are approved facilities for storing cord blood stem cells (collected from a newborn's umbilical cord). There are currently no TGA approved facilities for the storage of other stem cells in Australia.

Does the TGA regulate dental pulp stem cells?

Stem cells derived from sources such as dental pulp are subject to the same regulatory requirements as all other human cell and tissue products for therapeutic use.

However, the use of these stem cells to produce a product that could be of therapeutic use is only in the research and development stage. It is possible that in coming years these stem cells will be used in products for clinical trials, but products suitable for full marketing approval by the TGA are still some time away.

What are autologous stem cells?

Autologous cells are those that are removed from, and applied to, the same person, so the donor and recipient are the same.

Is it safe to use autologous stem cells?

Autologous cells are those that are removed from and applied to the same person. These cells may be treated, processed or purified after removal. As the cells come from the same person, there is a lower chance of reaction and rejection of the cells by the patient's immune system.

However, an unproven autologous stem cell treatment may be unsafe, posing serious risks to your health. The risks include infection and growth of specialised tissue in the wrong place (ectopic tissue formation).

Unproven stem cell treatments may also involve significant financial costs, including treatment and follow-up costs, as well as the cost of emergency medical care in the event that something goes wrong. Undergoing an unproven stem cell treatment may interfere with proven and potentially beneficial therapies or treatment plans recommended by your general practitioner or specialist. It can also disqualify you from participation in a registered clinical trial.

Can I use Cord Blood stem cells from my own pregnancy for my own treatment?

The collection and use of cord blood stem cells for a specific patient is known as directed donation. On rare occasions, a hospital will collect and store a baby's cord blood for a family member who has a medical condition that can be treated with cord blood transplantation. This requires a treating doctor's approval.

Should I use a natural therapy that claims to increase stem cells that is not approved by the TGA or available via the Special Access Scheme?

The TGA ensures that where specific therapeutic claims are made about a product or therapy then these are supported and the product is safe. For natural therapies that do not have oversight by TGA there is no guarantee of safety or effective use.

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Stem cell treatments and regulation - a quick guide for consumers

NEW YORK--(BUSINESS WIRE)--The Tisch Multiple Sclerosis Research Center of New York, the worlds largest independent research center focusing on MS, is proud to announce that its FDA-approved stem cell treatment study was featured in an article in BioSpace.

Preliminary analysis of the study results found that stem cell treatments substantially improved muscle strength and disability scores, particularly among patients with lower levels of disability. Interviewing Dr. Saud A. Sadiq, the Director and Chief Research Scientist of the Tisch MSRCNY, as well as Senior Research Scientist Dr. Violaine Harris, the article features the progress and key results of the stem cell treatment study, as well as the broader history and goals of the center.

We are excited to share more about our stem cell treatment research, which is a key study in service of our centers overarching goal of finding the cause of and the cure for MS, said Dr. Sadiq. Furthermore, the study has enormous potential not only for the treatment of MS patients, but also promising potential implications for those with other neurodegenerative diseases. Were delighted to share our analysis with the broader public and scientific community, and look forward to sharing further updates on this groundbreaking research as it progresses.

The full BioSpace article can be accessed here.

About the Tisch MS Research Center of New York

The mission of the Tisch Multiple Sclerosis Research Center of New York is to conduct groundbreaking medical research to ensure unparalleled care and positive outcomes for MS patients. Its integrated relationship with the International Multiple Sclerosis Management Practice (IMSMP) accelerates the pace at which research discoveries translate from lab bench to bedside. The Center aims to identify the cause of MS, understand disease mechanisms, optimize therapies, and repair the damage caused by MS while offering patients access to the best and most advanced treatments possible.

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Tisch Multiple Sclerosis Research Center of New York Study Featured in BioSpace - Business Wire

While historically lacking in foreign investments, Japans biotech scene is thriving with global investors showing increasing interest. Here are five of the hottest Japanese private companies innovating in the healthcare space.

Japan boasts one of the highest life expectancies in the world, and, faced with a rapidly aging population, is witnessing a growing burden of chronic conditions including cardiovascular disease and type 2 diabetes. For this reason, the Japanese healthcare authorities are encouraging research into the treatment and prevention of these diseases, in addition to promoting the potential of regenerative medicine.

In addition to having a roster of healthcare giants including Takeda, Astellas Pharma and Eisai, Japan is also an Asian hotspot for biotech companies. Upcoming startups have historically been limited in foreign funding and reliant on local venture capital players such as Nippon Venture Capital, Shinsei Capital Partners, and the University of Tokyo Edge Capital Partners.

In 2021, however, the amount of foreign investment flowing into the Japanese biotech space rose to $98 million, almost triple the haul of previous years. The most prominent global backers included Newton Biocapital, F-Prime Capital, and SoftBank Group. This trend arose as the COVID-19 pandemic triggered a wave of investor enthusiasm in biotechnology around the world.

With the help of local experts, weve listed five of the hottest private biotech companies in Japan. These firms, shown in alphabetical order, have raised large funding rounds in the last two years and are developing innovative treatments for a range of conditions including cancer, cardiovascular disease and inflammatory disorders.

Source: Shutterstock

Founded: 2017

Headquarters: Fujisawa

Chordia Therapeutics derives its name from the English term chord referring to a collection of musical notes normally played in harmony. In a similar way, the company aims to work in harmony with stakeholders and collaborators to develop first-in-class small molecule treatments for cancer.

Chordias lead program is a drug that disrupts the processing of RNA in tumor cells. In a healthy cell, RNA molecules are typically transcribed from a DNA template and spliced together to guide the production of new proteins. Some cancer cells accumulate mutations in the RNA splicing machinery and become vulnerable to Chordias drugs that interfere with this process.

Chordia raised $31 million (4 billion yen) in a Series C round in May 2022. The aim of the round was to push the companys lead drug through phase I testing and fund the preclinical development of the rest of its pipeline.

This month, the company announced interim results from the phase I trial of its lead candidate, with four of the recruited patients so far showing signs of responding to the treatment.

Founded: 2015

Headquarters: Tokyo

Heart failure occurs when the heart muscle is irreparably damaged and is unable to pump blood. While this deadly condition can be treated with a heart transplant, there is a general shortage of donors available, making a pressing need for alternatives.

In June 2021, the stem cell therapy developer Heartseed raised $36.5 million (4 billion yen) in a Series C round. The mission is to provide a regenerative route to saving the heart via stem cell therapy.

In the lab, Heartseed reprograms skin cells from the patient into a type of stem cell called induced pluripotent stem cells and grows these stem cells into heart muscle cells. The company then injects the muscle cells as a small cluster, or seed, into heart tissue to repair the muscle.

The proceedings from its Series C round will allow Heartseed to take its lead candidate into clinical development, including a phase I/II trial scheduled for later this year. Last year, Heartseed also licensed its treatment to Novo Nordisk in Denmark to co-develop the treatment outside of Japan.

Founded: 2018

Headquarters: Tokyo

LUCA Science hit the headlines in the last week for raising an impressive $30.3 million (3.86 billion yen) in a Series B round. The company is developing an unusual approach for treating a wide range of diseases: delivering a therapy based on mitochondria, the energy production plants in human cells.

One example where the technology could work well is in strokes and heart attacks, where blood flow is blocked to critical tissue in the brain and heart respectively. The reperfusion of blood to these tissues after the blockage can kill the tissue by damaging its mitochondria. Delivering healthy mitochondria could keep the tissue working properly and protect it from harm.

LUCA Science plans to use its recent Series B winnings to accelerate the preclinical development of its mitochondrial therapies and establish its manufacturing process. In May 2022, the firm also inked a collaboration deal with compatriot pharmaceutical company Kyowa Kirin Co., Ltd. to co-develop a mitochondrial therapy for rare genetic diseases.

Founded: 2016

Headquarters: Boston, U.S., and Tokyo

Modulus Discovery is a preclinical-stage drug discovery specialist. The company focuses on developing small molecule treatments for conditions such as cancer, inflammatory disorders and rare genetic conditions.

The firm uses a mixture of strategies to speed up the drug discovery process. These include simulating target proteins using a supercomputer; structural protein biology; forming collaborations such as with the peptide drug expert PeptiDream; and tapping into global networks for biological expertise. Modulus most advanced drug program is in late-stage preclinical testing for the treatment of chronic inflammatory diseases.

In March 2022, Modulus bagged $20.4 million (2.34 billion yen) in a Series C round. The cash is earmarked to advance the companys R&D programs by expanding its infrastructure, collaborations and headcount.

Founded: 2015

Headquarters: Tokyo

The name Noile-Immune is derived from blending together the phrases no illness and no immunity, no life. This company is developing CAR-T cell therapies for the treatment of cancer, which traditionally consist of extracting the patients immune T cells, engineering them in the lab to hunt down cancer cells, and reinfusing them into the patient.

Unlike approved CAR-T cell therapies, which are limited to treating forms of blood cancer, Noile-Immune aims its therapies at treating solid tumors. The company does this by engineering immune T cells to produce proteins that cause immune cells to migrate into the tumor site.

Noile-Immune is testing its lead candidate in a phase I in patients with solid tumors. The firm is also co-developing therapies with partners including Takeda and the European cell therapy specialists Adaptimmune and Autolus. Additionally, Noile-Immune has an allogeneic version of its cell therapy in the pipeline where immune T cells are sourced from healthy donors rather than the patient.

To finance the clinical development of its lead candidate, Noile-Immune raised $21.8 million (2.38 billion yen) in a Series C round in early 2021. The company hit a setback in January 2022 when a collaboration deal fell through with the U.S. player Legend Biotech. Nonetheless, other external companies remain interested in Noile-Immunes offering, including Japan-based Daiichi Sankyo Company Ltd., which opted to assess Noile-Immunes technology in late 2021.

Cover image via Elena Resko. Inline images via Shutterstock.

Thanks to feedback from Shiohara Azusa, VC investor at the University of Tokyo Edge Capital Partners, and Hironoshin Nomura, Chief Financial Officer at Sosei Group Corporation.

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Japan's five hottest biotech companies in healthcare - Labiotech.eu

MATFORS, Sweden, June 21, 2022 (GLOBE NEWSWIRE) -- Sweden-based NorthX Biologics (NorthX) is expanding into cell therapy manufacturing at its existing GMP-facility, as well as in premises at the Karolinska University Hospital campus in Stockholm. This initiative is part of NorthXs Innovation Hub, an Innovation Track designed to provide development and GMP-manufacturing services to the next generation of drug development companies and innovative research groups in need of NorthXs Good Manufacturing Practice (GMP) expertise.

NorthX has one of Northern Europes largest clinical-grade manufacturing capacities for plasmid DNA, recombinant proteins, cell banking and associated gene therapy services and this expansion into cell therapy is a major step to complete our offering for innovative clients. We are especially excited to work with Dr. Kristian Tryggvason, a leader in cell therapy technologies, and his team, said Dr. Ted Fjllman, CEO of NorthX.

After having built up BioLamina and its cell culture reagents that are used worldwide both in academia and industry, Dr. Tryggvason recently launched his latest venture: Alder Therapeutics. In addition to its own product development, the company now entered into an agreement to help NorthX expand its cell culture services to many new different cell types, including pluripotent stem cells. The Alder team will also help to design and validate NorthXs new process development and GMP-manufacturing labs in Matfors, alongside those being established at the Karolinska University Hospital campus in Stockholm.

Our goal is to be able to offer synergies to both cell and gene therapy clients and to collaborate with them through our Innovation Track, in which we work hand in hand with our clients regarding process development, manufacturing, and analytics to progress clinical programs and bring life-saving treatments to patients, added Aaron Small, NorthX VP of Global Sales and Corporate Development

Universities and cell therapy companies worldwide need GMP-grade development and manufacturing capacity, as it is complex and outside the scope of most biotech companies to build themselves. NorthX Biologics is already helping to translate cutting-edge gene therapy research into clinical development and now we will together build upon the existing broad cell therapy know-how in Sweden to do the same for cell therapies, said Dr. Kristian Tryggvason, CEO Alder Therapeutics.

About NorthX Biologics:

NorthX Biologics provides process development and manufacturing services with expertise in plasmids, proteins and other advanced biologics. NorthX Biologics sits in the heart of Sweden, and the team has been manufacturing biologics to GMP since 1988. In 2021 NorthX was recognized as a national innovation hub for advanced therapeutics and vaccines. NorthX has the ambition to become a leading cell and gene therapy manufacturer and partner of choice for innovative drug development companies. For more information see http://www.nxbio.com

About Alder Therapeutics:

Headquartered inStockholm, Alder Therapeutics AB is a novel development stage, cell therapy platform company aiming to develop and manufacture the best functional cell therapy products based on the most simple and robust processes. Alder Therapeutics unique cell therapy platform will allow manufacturing of better cells at a lower cost, which will make pluripotent cell therapy treatment available for ever more patients. Alder Therapeutics will play an important role in opening the next era in medicinal treatment. For more information and important updates, please visit. http://www.aldertx.com

For further information please email NorthX at:contact@nxbio.com

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NorthX Biologics expands to Cell Therapy: Partnership with Alder Therapeutics and new manufacturing site on Karolinska campus - GlobeNewswire

Sickle cell beta-thalassemia refers to an inherited condition that impacts hemoglobin. People with the condition have different changes in each copy of their hemoglobin gene. One causes red blood cells (RBCs) to form a sickle shape and another reduces the amount of hemoglobin.

Sickle cell beta-thalassemia is a type of RBC disorder known as a hemoglobinopathy. These are conditions that cause abnormal hemoglobin production or a change in its structure. Hemoglobin is the protein in RBCs responsible for carrying oxygen around the body.

Both sickle cell disease and beta-thalassemia are genetic conditions that affect hemoglobin. A person with sickle cell beta-thalassemia inherits a trait for both conditions, impacting the shape and number of hemoglobin.

In this article, we will discuss the causes and risk factors for sickle cell beta-thalassemia, as well as symptoms and treatment options for the condition.

Sickle cell beta-thalassemia is a genetic condition and a type of sickle cell disease that features symptoms of both sickle cell disease and beta-thalassemia. It causes RBCs to take on a sickle shape, making them unable to flow through the blood vessels as smoothly. It also affects the amount of normal hemoglobin a person has in their blood.

It is an inherited condition, meaning that parents pass it on to their children. Researchers estimate that 57% of the global population are carriers of a significant hemoglobin mutation. People with sickle cell beta-thalassemia inherit a sickle trait from one parent and a beta-thalassemia trait from the other.

There are two types of sickle cell beta-thalassemia: plus (HbS beta+) and zero (HbS beta0). The former is the milder variant. The plus indicates that the blood contains a lower-than-average amount of normal hemoglobin. This differs from the latter, in which a person has no normal hemoglobin.

Sickle cell beta-thalassemia results from a change in the beta-hemoglobin (HBB) gene. The beta-hemoglobin gene is responsible for forming the hemoglobin subunit beta component of the hemoglobin protein.

A person develops sickle cell beta-thalassemia when they inherit one sickle cell trait from one parent and one beta-thalassemia trait from the other. The beta-thalassemia gene can be either beta+, which results in a lower production of normal hemoglobin, or beta0, which leads to a complete absence of normal hemoglobin.

Risk factors for a person developing sickle cell beta-thalassemia include having parents that may be carriers of the sickle cell, HbS beta+, or HbS beta0 gene. The condition follows an autosomal recessive inheritance pattern. This means that if a person has a beta-thalassemia trait and their partner has a sickle cell trait, there is a 25% chance with each pregnancy that their child will have sickle cell beta-thalassemia.

The symptoms a person may experience will depend on whether they have HbS beta+ or HbS beta0. The amount of normal hemoglobin a person can produce largely determines the severity of the condition.

A person with HbS beta+ will likely have milder symptoms, as while they produce less functioning hemoglobin than is typical, they can still produce normal hemoglobin. Individuals with either type will produce sickle-shaped RBCs that can block circulation and cause cell damage and pain.

A person with HbS beta+ may experience:

The symptoms of HbS beta0 are typically similar but with a more severe case of anemia.

Some newborn screenings include testing for sickle cell disorders such as sickle cell beta-thalassemia. Screening involves taking a blood sample and looking at the types of hemoglobin present in the newborns blood. However, newborn screenings only suggest the possibility of sickle cell beta-thalassemia and require further confirmatory tests. This may include the parents and any siblings having tests.

These tests may include:

Treatment aims to prevent complications from occurring and treat the symptoms a person may experience. This generally involves continuous care to prevent and manage potential problems.

In addition to the treatments below, a person may receive pain relief medications and antibiotics to help reduce pain and infections. The spleen usually helps prevent infections, but many people with sickle cell beta-thalassemia may lose spleen function. As such, children may receive penicillin prophylaxis to prevent pneumococcal sepsis.

Treatment options for sickle cell beta-thalassemia may include:

People may require hydroxyurea if they experience frequent periods of pain. Hydroxyurea is a drug that makes RBCs bigger and changes their shape to the typical round and flexible composition. This can help slow or prevent complications.

Hydroxyurea increases the level of fetal hemoglobin (HbF) in the body. HbF is present in higher quantities in newborns and can help protect against sickle cell complications. With higher levels of HbF, RBCs are less likely to become sickle-shaped.

According to the American Society of Hematology, people with sickle cell beta-thalassemia who take hydroxyurea also have fewer:

Some people with sickle cell beta-thalassemia may require blood transfusions. This is when a healthcare professional infuses healthy donor blood into the body of a person with sickle cell beta-thalassemia via a tube. The donor blood will need to have matching antigens to the blood of the person receiving the transfusion. These antigens, known as human leukocyte antigens, are proteins present on the surface of RBCs.

If the antigens do not match, the immune system of the person receiving the blood donation is more likely to reject the transfusion, and it may lead to a reaction that can cause health problems.

The bone marrow in the body produces blood cells. A person with dysfunctional bone marrow, such as in sickle cell beta-thalassemia, may receive hematopoietic stem cells from a healthy donor. This may help improve bone marrow function and reduce the symptoms of sickle cell beta-thalassemia. A hematopoietic stem cell transplant is currently the only cure for this condition.

Sickle cell beta-thalassemia is a type of sickle cell disease. Some evidence suggests the life expectancy of a person living with sickle cell disease is reduced by 2030 years compared with a healthy individual. Similarly, a 2019 study suggests a person with sickle cell disease may live roughly 22 fewer years than a person without the condition.

Advances in therapy and treatment options are helping to improve the outlook of people with sickle cell disease. However, statistics like these highlight the necessity to further develop approaches to improve the underlying morbidity and mortality of individuals with the condition.

Most often, a person will receive a diagnosis of sickle cell beta-thalassemia shortly after birth. Later in life, if they believe their current treatment regime is not suitable or notice worsening symptoms, they should contact their doctor.

If a person is aware of a family history of sickle cell disease, they may wish to consider undergoing genetic screening before attempting to have children.

Sickle cell beta-thalassemia is a type of sickle cell disease. It occurs when a person inherits a sickle cell trait and a beta-thalassemia trait from their parents. It results in a person having sickle-shaped RBCs and either producing a low amount of hemoglobin or none at all.

Symptoms of the condition can include mild to severe anemia, tiredness, weakness, pain, and possible organ damage. Treatment may involve pain relief medications, antibiotics, hydroxyurea, and blood transfusions. Some people may also receive a bone marrow transplant to help them produce healthy hemoglobin.

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Sickle cell beta thalassemia: Causes, symptoms, and treatments - Medical News Today

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SEATTLE, June 16, 2022 (GLOBE NEWSWIRE) -- Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo to treat patients with solid and hematologic malignancies, announced today that it will have a poster presentation at the 2022 International Society for Stem Cell Research (ISSCR) Annual Meeting, to be held June 15-18, 2022 in San Francisco, California.

On Wednesday, June 15th, Principal Scientist & iPSC Team Lead, Teisha Rowland, Ph.D., will give a poster presentation titled, A Synthetic Cytokine Receptor Platform for Producing Cytotoxic Innate Lymphocytes as Off-the-Shelf Cancer Therapeutics. The presentation will discuss Umojas engineered induced pluripotent stem cell (iPSC) platform, that incorporates the synthetic cytokine receptor system rapamycin-activated cytokine receptor (RACR) platform. Umojas engineered iPSCs that are modified to express RACR, called RACR-induced cytotoxic innate lymphoid (iCIL) cells, drive differentiation and expansion of the cells while eliminating the need for expensive cytokines and other raw materials. The RACR platform has the potential to enable cytokine-free manufacturing and engraftment of the engineered cells in the patient without the need for toxic lymphodepletion.

Despite the advances chimeric antigen receptor T cell therapies have provided to the oncology space, we continue to battle significant challenges that these therapies cannot address, like limited expansion capacity and scalability, manufacturing complexity, variability among patients, and the need for toxic chemotherapy administration to combat patients anti-allograft response, said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. We are developing an engineered iPSC platform, including the RACR platform, to address these challenges by enabling a scalable, virtually unlimited, and simplified manufacturing of engineered, cancer-fighting cytotoxic innate lymphocytes.

About Umoja Biopharma

Umoja Biopharma, Inc. is an early clinical-stage company advancing an entirely new approach to immunotherapy. Umoja Biopharma, Inc. is a transformative multi-platform immuno-oncology company founded with the goal of creating curative treatments for solid and hematological malignancies by reprogramming immune cells in vivo to target and fight cancer. Founded based on pioneering work performed at Seattle Childrens Research Institute and Purdue University, Umojas novel approach is powered by integrated cellular immunotherapy technologies including the VivoVec in vivo delivery platform, the RACR/CAR in vivo cell expansion/control platform, and the TumorTag targeting platform. Designed from the ground up to work together, these platforms are being developed to create and harness a powerful immune response in the body to directly, safely, and controllably attack cancer. Umoja believes that its approach can provide broader access to the most advanced immunotherapies and enable more patients to live better, fuller lives. To learn more, visit http://umoja-biopharma.com/.

About RACR

CAR T cells generated by the body with VivoVec can be expanded and sustained with the rapamycin activated cytokine receptor (RACR) system, an engineered signaling system designed to improve chimeric antigen receptor (CAR) T cell persistence and produce durable anti-tumor responses. The RACR/CAR payload is integrated into the genomic DNA of a patients T cells. Rapamycin activates the RACR system resulting in preferential expansion and survival of cancer-fighting T cells. The RACR technology enables a patients cells to expand in a manner that resembles a natural immune response that does not require lymphodepletion, promoting durable T cell engraftment. RACR/CAR technology can also be used to enhanceex vivomanufacturing in support of more traditional autologous or allogeneic cell therapy manufacturing processes. To learn more about Umojas RACR platform please visit https://www.umoja-biopharma.com/platforms/

Media Contact:Darren Opland, Ph.D.LifeSci Communications[emailprotected]

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Umoja Biopharma Presents Data on its Engineered Induced Pluripotent Stem Cell Platform at the 2022 International Society for Stem Cell Research Annual...

Stem cell treatment is an alternative medical approach for various health conditions. It involves using stem cells to prevent, treat, or manage different diseases. But before you consider stem cell treatment as a therapy option, here are six things to know about this approach.

Different types of stem cells have their own purposes

There are four types of stem cells, and each has a unique application in regenerative medicine. Embryonic stem cells and pluripotent stem cells, for example, are commonly used not for treatment purposes but for studying disease processes and testing new drugs. In contrast, tissue-specific and mesenchymal stem cells are adopted for therapy use.

Tissue-specific stem cells are known to differentiate into cells of the certain tissues, e.g., hematopoietic stem cells (HSCs) that act as the progenitor of the different blood cells of the body. These stem cells are supposed only to be used to treat health conditions affecting the tissues in which they are found.

Mesenchymal stem cells (MSCs) differ from other stem cells in that it gives rise to their kind of cells and a variety of body cells. They are used in regenerative medicine to treat various medical conditions. Mesenchymal stem cells can be used in stem cell treatment for strokes, spinal cord injury, Crons disease, arthritis, and other conditions. You can visit website for a further list of diseases and for more information.

Stem cells cannot treat multiple diseases at the same time

Although there is stem cell treatment for diabetes, Crohn's disease, fibromyalgia, and several other medical conditions, stem cells cannot be used to treat comorbidities at the same time. For example, you can't use stem cell treatment for diabetes as the same stem cell treatment for knee problems. One stem cell treatment cannot be used to treat two or more unrelated medical conditions due to different routes of administration of a cell-based product, depending on the disease, as well as different treatment programs that may involve additional therapies. Be careful when choosing a clinic for stem cell treatment, and be sure to visit an accredited stem cell therapy clinic that offers various stem cell treatments instead of just one.

An experimental treatment offered for sale is not the same as a clinical trial

Many alternative treatment approaches have not been subjected to clinical trials. So, although a procedure can be an experimental treatment, it doesn't mean it has been researched or placed in clinical trials. When clinical trials are done successfully, it leads to the development of new treatment procedures that conforms to health regulations. Before signing up for any medical treatment, verify that it has passed through clinical trials.

Cells from your own body are not automatically safe when used in treatments

Stem cell treatments procedures involve harvesting some stem cells from a part of your body, manipulating these cells in the lab, and then reintroducing them into your body. Normally, because doctors take these stem cells from your body, there shouldn't be any problem reintroducing them back into your body.

Some scenarios can make them unsafe. If the cells were contaminated before being injected into your body, it could lead to severe microbial infection. If doctors inappropriately manipulate the cells and unintentionally alter their functioning and growth, they could become tumor cells. Be aware of these risks; abstain from clinics that do not lessen the risks associated with stem cell treatments.

Patient testimonials and other marketing provided by clinics may be misleading

One of the ways clinics market their services is through patient testimonials. Although patient testimonials are great for reviewing feedback from people who have adopted a particular medical treatment, they can be misleading.

Some clinics promote all the benefits of their treatments while lessening the potential risks involved. Others offer unproven treatments and unreliable patient testimonials to convince people to opt-in for their services. You want to ensure that the treatment is science-based, approved for use after successful trials, and provided by an accredited clinic.

There is something to lose when you try an unproven treatment

Most unproven experimental treatments have no solid scientific evidence detailing their effectiveness and benefits. Subscribing to such unproven treatments places you at risk of complications resulting in short to long-term problems.

Asides from the health risks attached, the costs of these procedures can be enormous and might amount to waste as the procedure might be ineffective. If you believe the potential benefits outweigh the presented risks, discuss with your family and healthcare providers to assess the treatment before properly making your final decision.

Conclusion

Stem cell treatments are proven medical therapy for several health conditions. For managing selected diseases, stem cell-based therapy has been subjected to clinical trials successfully. However, not all clinics are accredited to provide this type of treatment. The best stem cell therapy center offers various treatment programs for different medical conditions, does not treat multiple diseases with the same option, and provides you with adequate information about the benefits and risks of stem cell treatment.

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Six Things to Know About Stem Cell Treatment - HealthTechZone

An existing center on the University of Colorados Anschutz Medical Campus that helps develop treatments based on patients own cells is getting a $200 million boost, with the hope of getting those treatments to the public faster.

Chancellor Don Elliman said the Anschutz campus and the Gates Frontiers Fund will each invest $20 million per year over the next five years to turn the existing Gates Center for Regenerative Medicine into the significantly larger Gates Institute.

The Gates Frontiers Fund is affiliated with the Gates Family Foundation, a Colorado-based nonprofit,and is not connected to Bill Gates foundation.

The fund and the campus in Aurora also have partnered on a manufacturing facility that reprograms patients immune cells to fight certain cancers. Elliman said they dont expect to need a new building for the institutes expanded work.

Regenerative medicine is a broad term for treatments that try to harness the bodys ability to fix itself. That could involve reprogramming cells to replace dying tissue or fight cancer, or therapies that insert a healthy gene to replace a defective version thats causing disease.

Its early enough in the process that the institutes leadership hasnt chosen specific focus areas under the regenerative medicine umbrella.

Most of the $200 million will go toward hiring scientists, as well as support personnel to help both the new researchers and those already working on campus, Elliman said.

Once the institute is up and running, it will bring in new federal grants to support research and investment from biotech firms that can bring the treatments to market, he said.

This investment is really a seed investment, he said.

Dr. Terry Fry, the institutes executive director, said its meant to help scientists with ideas that show promise in the lab to take the steps toward testing them in humans.

The process of manufacturing treatments and getting trials approved is more complex for biologic therapies than for standard drugs, he said.

Theres a stage in the development of that sort of project where investigator-scientists run up against a brick wall, he said. A lot of the role that I see the institute playing is removing those barriers.

Fry, a pediatric oncologist and head of T-Cell therapeutics at Sana Biotechnology, was one of the first researchers who worked on chimeric antigen receptor T-cell (CAR-T) therapy the immune cell reprogramming therapy. It was approved first for children with leukemia, but now is also used for adults and for other blood cancers, like lymphoma and myeloma. He declined to name specific projects the institute would work on, but said potential improvements to CAR-T could be within its scope.

The therapy takes a kind of T-cell that kills cells infected with viruses or bacteria, and gets it to attack cancerous cells instead. While it has improved survival for people with certain blood cancers, it doesnt work well against solid tumors at this point. It also requires taking T-cells from each patient to produce their own treatment, which is expensive and slows down the process. Researchers are working on how to make CAR-T work for more people, and to create an off the shelf option, Fry said.

Another general area the institute could work on is growing cells to replace ones that have died or are defective, Fry said. Much of that work involves adult stem cells that have been coaxed back into an earlier form, when they could become almost any type of cell under the right conditions.

For example, if the stem cells can be primed to turn into cells producing insulin, that could help patients with Type 1 diabetes, which is caused when the insulin-producers die, he said.

It is really remarkable technology, he said.

The institute wont take down every hurdle to bringing new treatments to patients, Fry said. Manufacturing and distributing at a large scale will require partnerships with biotech firms, which fortunately are setting up in the Denver area in increasing numbers, he said.

I think this is the right time and exactly the right part of the country for this type of institute, he said.

Diane Gates Wallach, one of the Gates funds co-trustees, said the new institute will further her fathers goal of speeding up the process of getting new discoveries into clinical practice, so patients can benefit. Since the Anschutz campus includes researchers and two hospitals, it made sense to invest there, she said in a news release.

It takes a dynamic, innovative medical ecosystem for an institute like this to thrive and be successful, she said.

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CU Anschutz center for cell-based therapy gets $200 million expansion - The Denver Post