Stem cell transplant – NHS

A stem cell or bone marrow transplant replaces damaged blood cells with healthy ones. It can be used to treat conditions affecting the blood cells, such as leukaemia and lymphoma.

Stem cells arespecial cells produced bybone marrow (aspongytissue found in the centre of some bones) that can turn into different types of blood cells.

The 3 maintypes of blood cellthey can become are:

A stem cell transplant involves destroying any unhealthy blood cells and replacing them with stem cells removed from the blood or bone marrow.

Stem cell transplants are used to treat conditions in which the bone marrow is damaged and is no longer able to produce healthy blood cells.

Transplants can also be carried out to replace blood cells that are damaged or destroyed as a result of intensive cancer treatment.

Conditions that stem cell transplants can be used to treat include:

A stem cell transplant will usually only be carried out if other treatments have not helped, the potential benefits of a transplant outweigh the risks and you're in relatively good health, despite your underlying condition.

A stem cell transplant can involve taking healthy stem cells from the blood or bone marrow of one person ideally a close family member with the same or similar tissue type and transferring them to another person. This is called an allogeneic transplant.

It's also possible to remove stem cells from your own body and transplant them later, after any damaged or diseased cells have been removed. This is called an autologous transplant.

Astem celltransplant has 5 main stages. These are:

Having a stem cell transplant can be an intensive and challenging experience. You'll usually need to stay in hospital forat least a few weeks until the transplant starts to take effect and itcan take up toa year or longer to fully recover.

Read more about what happens during a stem cell transplant.

Stem celltransplants arecomplicated procedures with significant risks. It's important that you're aware of both the risks and possible benefits before treatment begins.

Possible problems you can have during or after the transplant process include:

Read more about the risks of having a stem cell transplant.

Ifit is not possible to use your own stem cells for the transplant, stem cells will need to come from a donor.

To improve the chances ofthetransplant being successful, donated stem cells need tocarry a special genetic marker known as a human leukocyte antigen (HLA) that'sidentical or very similar to that of the person receiving the transplant.

The best chance of getting a match is from a brother or sister, or sometimes another close family member. If there are no matches in your close family,a search of theBritish Bone Marrow Registry will be carried out.

Most peoplewill eventually find a donor in the registry,although a small number of people may find it very hard or impossibleto find a suitable match.

The NHS Blood and Transplant website and the Anthony Nolan website have more information about stem cell and bone marrow donation.

Page last reviewed: 07 September 2022 Next review due: 07 September 2025

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

Pain.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

June 27, 2020

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

ABOUT ANDREWS MEDICINE OF PENSACOLA, FLORIDA

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

ABOUT CELLTEX THERAPEUTICS CORPORATION

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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Its a Shame to See Him, How He is Today: Tyson Furys Father Calls Out Mike Tyson Following Deteriorating Health Condition – EssentiallySports

Former world heavyweight champion Mike Tyson was one of the greatest athletes to ever step foot inside the boxing ring. Tysons iconic performances inside the ring and eccentricity outside the ring have only further elevated his status as one of the Titans of boxings very own Tartarus. Meanwhile, Tyson Furys father, John Fury, had a few choice words to say about Tyson. Especially with regard to him undergoing stem cell treatments.

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Fury was in an interview JOE. And, he was talking about his desire to fight Tyson. In conversation, he was initially all praise for the legendary heavyweight. Fury said, Mike Tyson was tricky. He had plenty of movement. Plenty of head movement. He was in and out quick feet. And, he had bombs in each hand and a great fighter.

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Tyson had previously undergone stem cell treatments in preparation for his return to boxing. Talking about the same, Fury stated, You know its a shame to see him, how he is today. You with all you hear about stem cells, blood transfusions to make him younger. Whatever they call them. You know what Im saying? But hes a great fella and I do like him.

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However, Fury added that he likes Tyson and that he would be honored to step inside the ring with the legend. Moreover, it is worth mentioning that Furys statement came at a time when Tysons health and well-being were under scrutiny.

Mike Tyson retired from professional boxing in the year 2005. And he completely moved away from the sport for almost 15 years, even gaining weight in the process.

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Tyson was in conversation with NBA legend Shaquille ONeal. And this was when he opened up about undergoing stem cell treatments. Talking about the same, he said, I feel like a different person, but I cant comprehend why I feel this way. Its really wild what scientists can do.

He mentioned that his body was badly out of shape and that the treatment has been helping him return to the sport. However, it is not known which condition he has been treating with stem cell therapy.

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WATCH THIS STORY Movies and Documentaries About Mike Tyson Every Fan Must Watch

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3 Revolutionary Stocks I’d Buy Right Now Without Hesitation – The Motley Fool

Regeneron Pharmaceuticals (REGN 0.60%), Vertex Pharmaceuticals (VRTX 1.08%) and Biogen (BIIB 1.19%) are developing remarkable therapies that, instead of merely treating disorders, have the potential to cure them or at least make an impact that no other drug has to date.

At the same time, all three companies are profitable, and their successes in the lab have been paying off in the market, with their shares up nearly 10% this year. Let's see why.

Regeneron's shares are up about 10% this year, despite a second quarter that saw its revenue fall 44% year over year. The reason that's not a big cause for alarm is that the company's second-quarter revenue of $2.86 billion would be up 20% over the same period in 2021 if its COVID-19 therapy, REGEN-COV, was excluded from total revenue. The company's sales of the treatment ended late last year when government contracts for the therapy ended. Even without REGEN-COV, the company had earnings per share (EPS) of $7.47 in the quarter.

The biotech company's revolutionary concept is its process of using genetic research to develop specific antibodies that bind to two different antigen proteins. The company has medicines that treat atopic dermatitis, high cholesterol, retinal disease, prostate cancer, Ebola, rheumatoid arthritis, certain skin cancers, and non-small cell lung cancer. That doesn't include the 35 therapies it has in its pipeline.

It just reported positive data for its phase 1 study on ALN-HSD, which it is developing with Alnylam Pharmaceuticals to treat non-alcoholic steatohepatitis (NASH), a form of fatty liver disease which can lead to liver failure, cancer, and death.

Its current blockbuster drug, Dupixent, is approved alone or paired with other drugs to treat atopic dermatitis, asthma, eosinophilic esophagitis (a chronic immune system disorder), and chronic rhinosinusitis with nasal polyps. In the second quarter, it brought in $2.09 billion, up 40% year over year. Overall, the company reported $2.86 billion in revenue and earnings per share of $7.47.

Regeneron is well on its way to developing another blockbuster drug. Eylea (aflibercept), an anti-blindness treatment the company is developing with Bayer, showed effectiveness and safety at high dosages in late-stage trials. The potential label expansion would help a drug that brought in $1.6 million in the second quarter, up 14% over the same period last year.

BIIB data by YCharts.

Vertex Pharmaceuticals' shares are up more than 33% this year. That may be due to several late-stage-trial successes that have investors excited. Most prominent is Exa-cel (CTX001), which the company is developing with CRISPR Therapeuticsand is seen as a revolutionary potential cure for two genetic blood disorders -- transfusion-dependent thalassemia and severe sickle cell disease.

It's also expecting big things this year from VX-880, a stem-cell treatment to cure type 1 diabetes that is in Phase 1/2 trials.

Vertex is already profitable, thanks to cystic fibrosis drug Trikafta. In the second quarter, the company reported product revenue of $2.2 billion, up 22% year over year. The company reported EPS of $3.13 compared to $0.26 in the same period a year ago.

Trikafta alone was responsible for $1.89 billion in sales in the quarter, up 50.7%, year over year. The company raised its full-year revenue guidance to be between $8.6 billion to $8.8 billion, compared to $7.5 billion last year.

Biogen, founded in 1978, is one of the older biotech companies. It focuses on fighting neurological disorders and has a diverse portfolio. The company's stock is up some 36% over the past month, due to a promising Phase 3 trial for Lecanemab, an Alzheimer's drug that Biogen is developing with Japanese pharmaceutical company Eisai. Biogen said that the drug, after 18 months, slowed cognitive decline in Alzheimer's patients by 27%, compared to a placebo, based on the Clinical Dementia Rating Sum of Boxes scale (CDR-SB).

It's the second bite of the apple for Biogen, as another Alzheimer's drug it developed with Eisai, Aduhelm, had mixed clinical trials. Though Aduhelm was approved by the FDA, The Centers for Medicare and Medicaid Services said it would allow Medicare to pay for Aduhelm treatment only for Alzheimer's patients enrolled in qualifying clinical trials. As a consequence, Biogen hasn't been able to profit much from the drug.

Finding therapies that help slow the progression of Alzheimer's has been difficult because many drugs are rendered ineffective by the blood-brain barrier. Unlike Aduhelm and other Alzheimer's therapies, Lecanemab is the first to show a strong connection between reducing amyloid-related plaque in the brain associated with Alzheimer's and slowing the progression of the disease.

Biogen is already on solid financial ground to market Lecanemab. While revenue in the second quarter was a reported $2.589 billion, down 6% year over year, the company had EPS of $7.24, compared to EPS of $2.99 in the same period in 2021. Biogen also released improved guidance based on better top-line performance and cost-cutting measures. It now expects annual revenue of between $9.9 billion to $10.1 billion, up from an earlier range of between $9.7 billion to $10 billion. It also raised non-GAAP (adjusted) EPS from a range of $14.25 to $16.00 up to an estimate $15.25 to $16.75.

Jim Halley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Alnylam Pharmaceuticals, CRISPR Therapeutics, and Vertex Pharmaceuticals. The Motley Fool recommends Biogen. The Motley Fool has a disclosure policy.

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Century Therapeutics Announces Appointment of Daphne Quimi and Timothy Walbert to its Board of Directors – Yahoo Finance

Century Therapeutics, Inc.

PHILADELPHIA, Oct. 03, 2022 (GLOBE NEWSWIRE) -- Century Therapeutics (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology, today announced the appointment of Daphne Quimi and Timothy Walbert to the Companys Board of Directors. Ms. Quimi is currently Chief Financial Officer of Amicus Therapeutics and brings experience in public accounting and financial reporting to Century. Mr. Walbert is currently Chairman, President, and Chief Executive Officer of Horizon Therapeutics, and brings expertise in product portfolio building and commercialization. In conjunction with these new appointments, Century also announced that Eli Casdin, Chief Investment Officer of Casdin Capital, has resigned from the Board of Directors, effective as of October 1, 2022.

I am thrilled to welcome both Daphne and Tim to our Board. They will each play critical roles as we accelerate our next-generation cell therapy platform, said Lalo Flores, Ph.D., Chief Executive Officer, Century Therapeutics. Daphnes strong financial background and experience at both biotechnology and large pharmaceutical companies will be a tremendous asset as we enter the next transformative years for Century. Tims background, which includes numerous product launches, will be instrumental to our continued evolution, particularly as we progress our pipeline candidates with the ultimate goal of delivering innovative cancer therapies. Additionally, on behalf of the management, Board and all of our employees, we would like to thank Eli for his contributions to Centurys rapid growth and his leadership in our early formative years, where he was a key strategic thought partner.

Before serving as Amicuss Chief Financial officer, Ms. Quimi was Amicuss Senior Vice President, Finance and Corporate Controller. Ms. Quimi is currently a member of the Board of Directors at Amylyx Pharmaceuticals. Prior to Amicus, Ms. Quimi served as Director of Consolidations and External Reporting at Bristol-Myers Squibb. She also held roles of increasing responsibility in the finance department at Johnson & Johnson. Earlier in her career she worked for KPMG. Ms. Quimi received a B.S. in Accountancy from Monmouth University and an M.B.A from the Stern School of Business of New York University.

In addition to his current role of President and Chief Executive Officer of Horizon Therapeutics, Mr. Walbert has served as Chairman of Horizons Board of Directors since 2010. Before joining Horizon, Mr. Walbert served as President, Chief Executive Officer and Director of IDM Pharma Inc., and also held prior senior roles at NeoPharm Inc., Abbott (AbbVie), G.D. Searle & Company, Merck & Co. Inc. and Wyeth. Mr. Walbert received a B.A. in Business from Muhlenberg College.

About Century Therapeutics

Century Therapeutics (NASDAQ: IPSC) is harnessing the power of adult stem cells to develop curative cell therapy products for cancer that we believe will allow us to overcome the limitations of first-generation cell therapies. Our genetically engineered, iPSC-derived iNK and iT cell product candidates are designed to specifically target hematologic and solid tumor cancers. We are leveraging our expertise in cellular reprogramming, genetic engineering, and manufacturing to develop therapies with the potential to overcome many of the challenges inherent to cell therapy and provide a significant advantage over existing cell therapy technologies. We believe our commitment to developing off-the-shelf cell therapies will expand patient access and provide an unparalleled opportunity to advance the course of cancer care. For more information on Century Therapeutics please visit http://www.centurytx.com.

Century Therapeutics Forward-Looking Statement

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, The Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as may, might, will, should, expect, plan, aim, seek, anticipate, could, intend, target, project, contemplate, believe, estimate, predict, forecast, potential or continue or the negative of these terms or other similar expressions. These statements are not guarantees of future performance These risks and uncertainties are described more fully in the Risk Factors section of our most recent filings with the Securities and Exchange Commission and available at http://www.sec.gov. You should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

For More Information:

Company: Elizabeth Krutoholow investor.relations@centurytx.com

Investors: Melissa Forst/Maghan Meyers century@argotpartners.com

Media: Joshua R. Mansbach century@argotpartners.com

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Century Therapeutics Announces Appointment of Daphne Quimi and Timothy Walbert to its Board of Directors - Yahoo Finance