How effective is stem cell therapy for liver cirrhosis? – Times of India

Lifestyle diseases are gradually moving up the ranks causing deaths globally. Liver diseases appear to be one such condition, with a significantly increased number of patients being diagnosed each year. According to the WHO data published in 2017, liver disease was responsible for around 2.95% of the total deaths in India, accounting for one-fifth of all cirrhosis-associated deaths globally. Earlier, infections such as hepatitis B and C were the main causes; however, alcohol consumption and obesity are now becoming bigger contributors to liver disease. We know that the liver is responsible for detoxifying alcohol and drugs, but also performs multiple other functions, including glucose supply to the brain, food digestion, producing blood during foetal development, storing nutrients, etc. We also know that the liver can regenerate, but that does not mean a fully damaged liver can grow back on its own. Therefore, it is important to pay attention to signs of liver disease and initiate treatments promptly. Liver cirrhosis Cirrhosis is a condition where scars form in the liver causing the normal liver tissue to harden, thereby preventing the effective functioning of the organ. Cirrhosis and liver cancer are the prime causes of death due to liver disease globally. Regenerative medicine researcher Dr Pradeep Mahajan shares that alcohol consumption, viral hepatitis, autoimmune diseases, non-alcoholic fatty liver disease, and several inherited metabolic disorders can cause cirrhosis. The disease process begins as inflammation (swelling) in the liver tissue followed by scar formation and ultimately liver failure. Considering that there is no cure for cirrhosis per se, symptom and lifestyle management remain the mainstay of conventional treatment. Liver transplantation is the only curative option for severe cases; however, the issue of organ shortage is a chief and ever-growing concern. How can one get treated? Dr Mahajan says: Since we know that the liver can regenerate itself, the way ahead is to diagnose the liver disease as early as possible and find ways to enhance its regenerative potential. This is where cell and growth factor-based therapy can be beneficial. Stem cells in our body are capable of differentiating into liver (and various other) cells. In addition, they are also capable of regulating the immune system, reducing inflammation, enhancing blood supply, and stimulating other cells to perform their functions more efficiently. Similarly, growth factors can be isolated from blood/platelets, which serve as nutrition for cells of the body. These can help in cirrhosis by stabilising the internal environment of the liver making it more conducive to healing and regeneration. We are simply trying to find ways to capitalise on the healing potential of the liver before issues like scarring happen. Of course, lifestyle modification will be required to enhance the outcomes, but the end goal is to prevent the need for (or at least delay) liver transplantation, which can significantly affect a patients quality of life, adds Dr Mahajan.

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How effective is stem cell therapy for liver cirrhosis? - Times of India

Kite’s CAR T-cell Therapy Tecartus Receives Positive CHMP Opinion in Relapsed or Refractory Acute Lymphoblastic Leukemia (r/r ALL) – Gilead Sciences

Tecartus (Brexucabtagene Autoleucel) First and Only CAR T in Europe to Receive Positive CHMP Opinion to Treat Adults 26+ with r/r ALL

If Approved, it will Address a Significant Unmet Need for a Patient Population with Limited Treatment Options

SANTA MONICA, Calif.--(BUSINESS WIRE)-- Kite, a Gilead Company (Nasdaq: GILD), today announces that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Tecartus (brexucabtagene autoleucel) for the treatment of adult patients 26 years of age and above with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia (ALL). If approved, Tecartus will be the first and only Chimeric Antigen Receptor (CAR) T-cell therapy for this population of patients who have limited treatment options. Half of adults with ALL will relapse, and median overall survival (OS) for this group is only approximately eight months with current standard-of-care treatments.

Kites goal is clear: to bring the hope of survival to more patients with cancer around the world through cell therapy, said Christi Shaw, CEO, Kite. Todays CHMP positive opinion in adult ALL brings us a step closer to delivering on the promise that cell therapies have to transform the way cancer is treated.

Following this positive opinion, the European Commission will now review the CHMP opinion; the final decision on the Marketing Authorization is expected in the coming months.

Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy and stem cell transplant, creating a significant burden on a patients quality of life, said Max S. Topp, MD, professor and head of Hematology, University Hospital of Wuerzburg, Germany. If approved, patients in Europe will have a meaningful advancement in treatment. Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.

Results from the ZUMA-3 international multicenter, single-arm, open-label, registrational Phase 1/2 study of adult patients (18 years old) with relapsed or refractory ALL, demonstrated that 71% of the evaluable patients (n=55) achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) with a median follow-up of 26.8 months. In an extended data set of all patients dosed with the pivotal dose (n=78) the median overall survival for all patients was more than two years (25.4 months) and almost four years (47 months) for responders (patients who achieved CR or CRi). Among efficacy-evaluable patients, median duration of remission (DOR) was 18.6 months. Among the patients treated with Tecartus at the target dose (n=100), Grade 3 or higher cytokine release syndrome (CRS) and neurologic events occurred in 25% and 32% of patients, respectively, and were generally well-managed.

About ZUMA-3

ZUMA-3 is an ongoing international multicenter (US, Canada, EU), single arm, open label, registrational Phase 1/2 study of Tecartus in adult patients (18 years old) with ALL whose disease is refractory to or has relapsed following standard systemic therapy or hematopoietic stem cell transplantation. The primary endpoint is the rate of overall complete remission or complete remission with incomplete hematological recovery by central assessment. Duration of remission and relapse-free survival, overall survival, minimal residual disease (MRD) negativity rate, and allo-SCT rate were assessed as secondary endpoints.

About Acute Lymphoblastic Leukemia

ALL is an aggressive type of blood cancer that develops when abnormal white blood cells accumulate in the bone marrow until there isnt any room left for blood cells to form. In some cases, these abnormal cells invade healthy organs and can also involve the lymph nodes, spleen, liver, central nervous system and other organs. The most common form is B cell precursor ALL. Globally, approximately 64,000 people are diagnosed with ALL each year, including around 3,300 people in Europe.

About Tecartus

Please see full FDA Prescribing Information, including BOXED WARNING and Medication Guide.

Tecartus is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME and NEUROLOGIC TOXICITIES

Cytokine Release Syndrome (CRS), including life-threatening reactions, occurred following treatment with Tecartus. In ZUMA-2, CRS occurred in 91% (75/82) of patients receiving Tecartus, including Grade 3 CRS in 18% of patients. Among the patients who died after receiving Tecartus, one had a fatal CRS event. The median time to onset of CRS was three days (range: 1 to 13 days) and the median duration of CRS was ten days (range: 1 to 50 days). Among patients with CRS, the key manifestations (>10%) were similar in MCL and ALL and included fever (93%), hypotension (62%), tachycardia (59%), chills (32%), hypoxia (31%), headache (21%), fatigue (20%), and nausea (13%). Serious events associated with CRS included hypotension, fever, hypoxia, tachycardia, and dyspnea.

Ensure that a minimum of two doses of tocilizumab are available for each patient prior to infusion of Tecartus. Following infusion, monitor patients for signs and symptoms of CRS daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility, and for four weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

Neurologic Events, including those that were fatal or life-threatening, occurred following treatment with Tecartus. Neurologic events occurred in 81% (66/82) of patients with MCL, including Grade 3 in 37% of patients. The median time to onset for neurologic events was six days (range: 1 to 32 days) with a median duration of 21 days (range: 2 to 454 days) in patients with MCL. Neurologic events occurred in 87% (68/78) of patients with ALL, including Grade 3 in 35% of patients. The median time to onset for neurologic events was seven days (range: 1 to 51 days) with a median duration of 15 days (range: 1 to 397 days) in patients with ALL. For patients with MCL, 54 (66%) patients experienced CRS before the onset of neurological events. Five (6%) patients did not experience CRS with neurologic events and eight patients (10%) developed neurological events after the resolution of CRS. Neurologic events resolved for 119 out of 134 (89%) patients treated with Tecartus. Nine patients (three patients with MCL and six patients with ALL) had ongoing neurologic events at the time of death. For patients with ALL, neurologic events occurred before, during, and after CRS in 4 (5%), 57 (73%), and 8 (10%) of patients; respectively. Three patients (4%) had neurologic events without CRS. The onset of neurologic events can be concurrent with CRS, following resolution of CRS or in the absence of CRS.

The most common neurologic events (>10%) were similar in MCL and ALL and included encephalopathy (57%), headache (37%), tremor (34%), confusional state (26%), aphasia (23%), delirium (17%), dizziness (15%), anxiety (14%), and agitation (12%). Serious events including encephalopathy, aphasia, confusional state, and seizures occurred after treatment with Tecartus.

Monitor patients daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility and for four weeks following infusion for signs and symptoms of neurologic toxicities and treat promptly.

REMS Program: Because of the risk of CRS and neurologic toxicities, Tecartus is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta and Tecartus REMS Program which requires that:

Hypersensitivity Reactions: Serious hypersensitivity reactions, including anaphylaxis, may occur due to dimethyl sulfoxide (DMSO) or residual gentamicin in Tecartus.

Severe Infections: Severe or life-threatening infections occurred in patients after Tecartus infusion. Infections (all grades) occurred in 56% (46/82) of patients with MCL and 44% (34/78) of patients with ALL. Grade 3 or higher infections, including bacterial, viral, and fungal infections, occurred in 30% of patients with ALL and MCL. Tecartus should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after Tecartus infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 6% of patients with MCL and 35% of patients with ALL after Tecartus infusion and may be concurrent with CRS. The febrile neutropenia in 27 (35%) of patients with ALL includes events of febrile neutropenia (11 (14%)) plus the concurrent events of fever and neutropenia (16 (21%)). In the event of febrile neutropenia, evaluate for infection and manage with broad spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, life-threatening and fatal opportunistic infections have been reported. The possibility of rare infectious etiologies (e.g., fungal and viral infections such as HHV-6 and progressive multifocal leukoencephalopathy) should be considered in patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

Prolonged Cytopenias: Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and Tecartus infusion. In patients with MCL, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 55% (45/82) of patients and included thrombocytopenia (38%), neutropenia (37%), and anemia (17%). In patients with ALL who were responders to Tecartus treatment, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 20% (7/35) of the patients and included neutropenia (12%) and thrombocytopenia (12%); Grade 3 or higher cytopenias not resolved by Day 60 following Tecartus infusion occurred in 11% (4/35) of the patients and included neutropenia (9%) and thrombocytopenia (6%). Monitor blood counts after Tecartus infusion.

Hypogammaglobulinemia: B cell aplasia and hypogammaglobulinemia can occur in patients receiving treatment with Tecartus. Hypogammaglobulinemia was reported in 16% (13/82) of patients with MCL and 9% (7/78) of patients with ALL. Monitor immunoglobulin levels after treatment with Tecartus and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement.

The safety of immunization with live viral vaccines during or following Tecartus treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least six weeks prior to the start of lymphodepleting chemotherapy, during Tecartus treatment, and until immune recovery following treatment with Tecartus.

Secondary Malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

Effects on Ability to Drive and Use Machines: Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following Tecartus infusion. Advise patients to refrain from driving and engaging in hazardous activities, such as operating heavy or potentially dangerous machinery, during this period.

Adverse Reactions: The most common non-laboratory adverse reactions ( 20%) were fever, cytokine release syndrome, hypotension, encephalopathy, tachycardia, nausea, chills, headache, fatigue, febrile neutropenia, diarrhea, musculoskeletal pain, hypoxia, rash, edema, tremor, infection with pathogen unspecified, constipation, decreased appetite, and vomiting. The most common serious adverse reactions ( 2%) were cytokine release syndrome, febrile neutropenia, hypotension, encephalopathy, fever, infection with pathogen unspecified, hypoxia, tachycardia, bacterial infections, respiratory failure, seizure, diarrhea, dyspnea, fungal infections, viral infections, coagulopathy, delirium, fatigue, hemophagocytic lymphohistiocytosis, musculoskeletal pain, edema, and paraparesis.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Gilead and Kite to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving Tecartus; the risk that physicians may not see the benefits of prescribing Tecartus for the treatment of blood cancers; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead and Kite, and Gilead and Kite assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Tecartus including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com .

Kite, the Kite logo, Tecartus and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies .

View source version on businesswire.com: https://www.businesswire.com/news/home/20220722005258/en/

Jacquie Ross, Investors investor_relations@gilead.com

Anna Padula, Media apadula@kitepharma.com

Source: Gilead Sciences, Inc.

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Kite's CAR T-cell Therapy Tecartus Receives Positive CHMP Opinion in Relapsed or Refractory Acute Lymphoblastic Leukemia (r/r ALL) - Gilead Sciences

Stem cells: Could we gain the power to induce cell regeneration? Dr Catherine Berry – The Scotsman

In short, stem cells initiate the production of new tissue cells, which can then replace their diseased counterparts.

Mesenchymal stem cells (MSCs) are adult stem cells found in many areas of the body such as bone marrow. The unique thing about these cells is their compatibility with a range of tissues such as bone, cartilage, muscle, or fat. MSCs respond to injury or disease by migrating to these damaged areas, where they restore tissue function by replacing the damaged cells.

It has recently been shown that the success of MSCs relies on their ability to release cell signals their mechanism to initiate tissue regeneration. These signals are packaged into extracellular vehicles (EVs) which are essentially bubbles of information. These are released by MSCs and taken up by the injured or diseased tissue cells to kickstart their inbuilt process of regeneration.

Through funding from the Royal Society of Edinburgh, research has started into the development of artificial EVs as a viable alternative to cell therapy. These EVs will contain the key molecules released by stem cells when they are responding to injury cues in the body.

The power to induce tissue regeneration would provide a significant new tool in biomedical treatment, such as incorporating EVs into synthetic hydrogels within a wound dressing to encourage and accelerate healing.

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Within the lab setting, we have been able to manipulate stem cell cultures to produce EVs with different signal make-ups, and accurately identify their properties.

Controlling and identifying the different make-ups contained in EV signals which in turn induce different cell responses is crucial if we want to operationalise their use in medicine.

We now aim to synthesise artificial vesicles, or bubbles, for different clinical problems, such as, for example, bubbles with potent wound-healing properties that would help our ability to use new artificial stem cell therapy.

The research is underway and it is showing promise that we may be able to harness the regenerative power of stem cells in the near future.

An artificial EV-based approach also has several advantages over stem cell-based therapies, such as having increased potency and greater consistency in treatment, and at a lower cost to carry out.

Both inside and on the surface of the body, we would have the ability to induce a process vital to medical treatment we work with every day and, in turn, open a whole new avenue of possibilities in biomedical science.

Dr Catherine Berry is a reader in the Centre for the Cellular Microenvironment at the University of Glasgow, and a recipient of the Royal Society of Edinburghs personal research fellowship in 2021. This article expresses her own views. The RSE is Scotland's national academy, bringing great minds together to contribute to the social, cultural and economic well-being of Scotland. Find out more at rse.org.uk and @RoyalSocEd.

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Stem cells: Could we gain the power to induce cell regeneration? Dr Catherine Berry - The Scotsman

S’porean doctor, a sought-after top expert in cell therapy, appointed to WHO expert panel – The Straits Times

SINGAPORE - A Singaporean doctorwho is one of the top cell therapy experts in the worldhas been appointed to a World Health Organisation (WHO) expert panel.

Dr Mickey Koh is so sought-after in his field that for the past 15 years, he has been holding two jobs in two different countries.

The 56-year-old shuttles between England and Singapore, spending six weeks at a time in London, where he oversees the haematology department and looks after bone marrow transplant patients at St George's University Hospital, before returning to Singapore for a week and a half to head the cell therapy programme at the Health Sciences Authority.

Cell therapy is a growing field of medicine that uses living cells as treatment for a variety of diseases and conditions. This is an increasingly important therapeutic area and both his employers have agreed to his unusual schedule.

Over in London, Dr Koh is head of the Haematology Department at St George's Hospital and Medical School. In Singapore, he is the programme and medical director of the cell and gene therapy facility at the Health Sciences Authority.

In May, Dr Koh was selected to be on the WHO Expert Advisory Panel on Biological Standardisation.

Individuals on the panel have to be invited by WHO to apply, and are well recognised in their respective scientific fields. Eminent names on the panel include the current president of the Paul-Ehrlich-Institut in Germany, which is the country's federal agency, medical regulatory body and research institution for vaccines and biomedicine.

The WHO panel, which is made up of about 25 members, provides detailed recommendations and guidelines for the manufacturing, licensing and standardisation of biological products, which include blood, monoclonal antibodies, vaccines and, increasingly, cell-based therapeutics.

The recommendations and advice are passed on to the executive board of the World Health Assembly, which is the decision-making body of WHO.

Dr Koh's role had to be endorsed by the British government and was a direct appointment by the director-general of WHO.

His appointment as a panel expert will last for a term of four years.

Speaking to The Straits Times, Dr Koh shared his thoughts about the importance of regulation: "We are well aware that there is a very lucrative worldwide market peddling unproven stem cell treatments, where side effects are often unknown, and such unregulated practice can result in serious harm.

"This is already happening. People are claiming that you can use stem cells to treat things like ageing, and even very serious conditions like strokes, without any evidence."

With many medications now taking the form of biologics - a drug product derived from biological sources such as cells - the next wave of treatment would be the utilisation of these cells for the treatment of a wide range of diseases, Dr Koh said.

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S'porean doctor, a sought-after top expert in cell therapy, appointed to WHO expert panel - The Straits Times

No Stone Unturned: Seattle Children’s High-Risk Leukemia Experts Specialize in the Toughest Cases – On the Pulse – On the Pulse

Josh, Harper and Meagan in June 2022

Two years ago, Meagan stood in a hospital room at Seattle Childrens cradling her 1-year-old daughter, Harper, against her chest. Her fianc, Josh, huddled close to them and kissed the thinning hair on top of their babys head.

A feeding tube was routed through Harpers nose and her eyes were brimming with tears. Exhausted, she snuggled into her moms arms as a photographer took their picture.

Meagan and Josh feared those would be the last photos taken of their baby girl.

Six months before, Harper became seriously ill. After multiple visits to their pediatrician in Yakima, Meagan took her to an emergency room where blood tests revealed Harper had leukemia.

It was shocking, Meagan says. Thirty minutes later we were on an emergency flight to Seattle Childrens.

The family didnt return home for nearly two years.

The type of leukemia Harper had acute lymphoblastic leukemia (ALL) is typically harder to treat and has lower survival rates when it occurs in infants who are less than a year old.

Harpers case was exceptionally challenging. She didnt respond to standard chemotherapy, even after providers added a medication designed to sensitize her leukemia to the treatment.

Her care team, which included Seattle Childrens High-Risk Leukemia Program, believed a stem cell transplant would give Harper the best chance of surviving, but they had to eliminate the majority of her leukemia cells first.

Drs. Kasey Leger and Brittany Lee, Harpers primary oncologists, started her on a novel immunotherapy medication, called blinatumomab, which effectively destroyed many of her ALL cells.

Unfortunately, two weeks later, the team discovered some of Harpers ALL cells had morphed into a different blood cancer acute myeloid leukemia (AML). This rare occurrence, called lineage switch, occurs in less than 5% of infant ALL cases.

It was a roller coaster, Josh says. She didnt do anything they expected her to do. It felt like every day we had to come up with a new plan.

Drs. Leger and Lee gave Harper a different kind of chemotherapy that destroyed the new AML cells. Still, some of her ALL cells remained, so the team gave Harper blinatumomab again which finally suppressed her cancer enough for her to have a stem cell transplant just before her first birthday.

Harper and her mom, Meagan, celebrating Harpers first birthday shortly after her stem cell transplant

The team had done everything they could to get Harper healthy enough for a stem cell transplant, hopeful it would be the treatment that finally cured her. Tragically, Harpers leukemia was back less than a month later.

When leukemia comes back so soon after transplant, patients have very few treatment options, if any, says Dr. Corinne Summers, Harpers stem cell transplant specialist. Many patients will not survive long term.

Harpers parents were terrified they were going to lose her.

Her bone marrow was packed with leukemia, Josh remembers. You could tell the life was slipping out of her and she just looked like it was going to be the end.

After Harpers stem cell transplant failed, the family met with end-of-life specialists and scheduled a special photo session to create memories that they would carry forward

They struggled to decide if they should continue treatment.

How do you know when enough is enough? Meagan says. When do you say, We cant do this to her anymore? Harper couldnt tell us how she was feeling, so it was all our decision.

Meagan and Josh worked closely with the care team to decide what to do next.

Those conversations were emotional for all of us, says Dr. Lee. Thankfully, we had a close, trusting relationship with their family and were able to give recommendations that reflected what they wanted for their daughter and what they felt was most important.

After much consideration, Meagan and Josh decided Harper was strong enough to continue treatment.

Drs. Leger and Lee filed a compassionate use request with the Food and Drug Administration to give Harper an investigational chemotherapy drug called venetoclax. Unfortunately, the treatment didnt work.

Collaborating with the family, the team decided to try giving Harper blinatumomab one more time. There was no evidence suggesting the medication would work so soon after a bone marrow transplant and with such a high burden of leukemia, but within a week it eliminated 98% of Harpers cancer cells.

Family is a critical piece of the team, Dr. Leger says. And Harper is fortunate to have amazing parents who were at her bedside 24/7 and had a beautiful way of advocating for her. They challenged us to leave no stone unturned and partnered with us throughout her treatment to keep figuring out a way forward.

With Harpers leukemia under control, the team searched for a way to wipe out any remaining cancer cells and keep her disease from coming back. Doctors in Childrens Cancer and Blood Disorders Center lead national research groups such as the Childrens Oncology Group, so they have access to trials around the world. However, Harpers care team found the best treatment for her was at Seattle Childrens Hospital, in partnership with Seattle Childrens Therapeutics.

Harpers T-cells were removed through a process called apheresis before they were reprogrammed to target her cancer cells and infused back into her blood

Harper was enrolled in one of Childrens T-cell immunotherapy clinical trials. The treatment involves re-programming a patients T cells (a type of white blood cell) to target and destroy their cancer cells.

After her T-cell therapy, Harper was finally in remission.

Meagan cried with relief when she found out. Harper would not be here right now if it wasnt for everybody at Seattle Childrens, she says. From day one, theyve been comforting and compassionate. They bend over backwards to keep families involved and helped us fight for our child.

To keep her in remission, Harper was given six antigen-presenting cell boosters, which kept her reprogrammed T cells circulating through her blood longer. She received the last booster earlier this year and is still in remission today.

Harper had a very unique disease in that her leukemia manifested as both ALL and AML, says Dr. Leger. Thankfully, we have team members with deep expertise in each of those diseases. Having internationally recognized chemotherapy, transplant and immunotherapy specialists on our team allowed us to be creative with her care when she needed to go beyond the standard pathways.

Today, Harper is a joyful, boisterous 3-year-old who loves experimenting with musical toys and splashing around in her bath or kiddie pool. One of her favorite things to do is grab Meagan by the hair and squish their faces together.

Because of the treatments Harper received at such a young age and the extended time she spent in the hospital, Harper is behind on some developmental milestones like speaking and walking. Still, Meagan and Josh say shes catching up.

Shes starting to bloom and take off and its so nice to see, Meagan says. At the same time, we cant get too comfortable. We know how relentless her disease is and that it could come back one day.

Harper plays in a pool, one of her favorite activities, in June 2022

Harpers family encourages community members to support cancer research at Childrens so that new treatments can be developed for Harper and other kids like her.

Without donors, Harper probably wouldnt be alive right now, Josh says. The treatments she had were developed in just the last few years. If people dont step up and donate, those programs arent there. Those drugs arent invented. Cancer treatment has come a really long way and thats because of donors stepping up to make that happen.

Learn more about Seattle Childrens High-Risk Leukemia Program and Cancer and Blood Disorders Center.

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No Stone Unturned: Seattle Children's High-Risk Leukemia Experts Specialize in the Toughest Cases - On the Pulse - On the Pulse

Cryopreservation Equipment Market Report 2022-2028: Importance of Cryopreservation for Success of Cell-Based Therapies Presents Opportunities -…

DUBLIN--(BUSINESS WIRE)--The "Cryopreservation Equipment Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type, Cryogen Type, Application, and End User" report has been added to ResearchAndMarkets.com's offering.

The cryopreservation equipment market is expected to reach US$ 12,489.84 million by 2028 from US$ 6,358.65 million in 2022; it is estimated to grow at a CAGR of 11.9% from 2022 to 2028.

The factors such as growing acceptance for regenerative medicine and increasing need of biobanking practices are contributing to the market growth. However, the stringent regulatory requirements hinder the cryopreservation equipment market growth.

Cryopreservation is a technique employed to minimize cell damage caused during freezing and storage of biological materials such as tissue, bacteria, fungi, virus, and mammalian cells. Tissues and genetically stable living cells preserved via cryopreservation can be used in research and other biomedical applications. The equipment required for cryopreservation includes cryopreservation systems, cryoware, accessories, and cryogen.

Cryopreservation plays an important part in the field of regenerative medicine as it facilitates stable and secure storage of cells and other related components for a prolonged time. Regenerative medicine enables replacing diseased or damaged cells, tissues, and organs by retrieving their normal function through stem cell therapy.

Owing to the advancements in the medical technology, stem cell therapy is now being considered as an alternative to traditional drug therapies in the treatment of a wide range of chronic diseases, including diabetes and neurodegenerative diseases.

Type Insights

Based on type, the global cryopreservation equipment market is segmented into freezers, sample preparation systems, and accessories. In 2021, the freezers segment held the largest share of the market, and it is expected to register the highest CAGR in the market during 2022-2028. In ultracold freezers, liquid nitrogen is used for the successful preservation of more complex biological structures by virtually seizing all biological activities.

Cryogen Type Insights

Based on cryogen type, the global cryopreservation equipment market is segmented into liquid nitrogen, oxygen, liquid helium, argon, and others. In 2021, the liquid nitrogen segment held the largest share of the market; the market for this segment is further expected to grow at the highest CAGR during 2022-2028. Liquid nitrogen is a nonmechanical method of cells preservation. Large thermos-like containers are used to house either racks or shelves that hold cryogenic vials.

Application Insights

Based on application, the global cryopreservation equipment market is segmented into cord blood stem cells, sperms, semen & testicular tissues, embryos and oocytes, cell and gene therapies, and others. In 2020, the cord blood stem cells segment held the largest share of the market. Moreover, the market for the sperms segment is expected to register the highest CAGR in the market during 2022-2028. In recent years, public cord banking has been promoted over private cord banking. Various centers across the world are performing cord blood stem cell transplantation as a part of the management of genetic, hematologic, immunologic, metabolic, and oncologic disorders, among others, which is bolstering the growth of the market for the public cord banking segment.

End User Insights

Based on end user, the cryopreservation equipment market is segmented into stem cell banks, biotechnology and pharmaceuticals organizations, stem cell research laboratories, and others. The biotechnology and pharmaceuticals organizations segment held the largest market share in 2020, and it is further expected to be the largest shareholder in the market by 2028. Cryopreservation has become an integral part of the manufacturing process of many cellular therapies as it sometimes precedes cell culture (by preserving the starting cellular material before beginning large-scale manufacturing) and generally follows cell expansion.

Key Topics Covered:

1. Introduction

2. Cryopreservation Equipment Market - Key Takeaways

3. Research Methodology

4. Global Cryopreservation Equipment Market - Market Landscape

5. Cryopreservation Equipment Market - Key Market Dynamics

5.1 Market Drivers

5.1.1 Growing Acceptance for Regenerative Medicine

5.1.2 Increasing Needs of Biobanking Practices

5.2 Market Restraints

5.2.1 Stringent Regulatory Requirements

5.3 Market Opportunities

5.3.1 Importance of Cryopreservation for Success of Cell-Based Therapies

5.4 Future Trends

5.4.1 3D Printing to Provide Customizable Probes for Sensing and Monitoring in Cryobiology Applications

5.5 Impact Analysis

6. Cryopreservation Equipment Market- Global Analysis

7. Global Cryopreservation Equipment Market Revenue and Forecasts To 2028- by Type

10. Cryopreservation Equipment Market Revenue and Forecasts To 2028 - End User

11. Cryopreservation Equipment Market Revenue and Forecasts to 2028 - Geographical Analysis

12. Impact Of COVID-19 Pandemic on Cryopreservation Equipment Market

13. Cryopreservation Equipment Market- Industry Landscape

14. Company Profiles

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/l7syls

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Cryopreservation Equipment Market Report 2022-2028: Importance of Cryopreservation for Success of Cell-Based Therapies Presents Opportunities -...