Category Archives: Stem Cell Treatments

Houston, TX - (NewMediaWire) - October 03, 2022 - Joint pain is one of the leading causes of discomfort among the American populace. It is estimated that more than 65 million American adults suffer from low back pain, while over 16 million deal with acute or chronic joint pain due to arthritis. The number seems to be growing at an alarming rate annually, with the problem spreading to more young adults due to their lifestyle choices, occupation, accidents, and other reasons. QC Kinetix (The Heights) estimates that a whopping number of Americans will be battling chronic joint pain in years to come if no real step is taken.

As a pain control clinic focusing on health and wellness, QC Kinetix (The Heights) understands the health problems associated with heavy medication use to combat joint pain. The lead treatment provider at the clinic noted that more people who depend on medication for pain relief are growing tired and abandoning it because of its negative side effects on their bodies and organs. Others are too scared to consider surgical interventions which offer no real promises of long-term health benefits.

In the face of these health challenges, QC Kinetix (The Heights) is offering a better solution with better healing potential to patients suffering from joint pain and related problems. Speaking on their natural treatment therapies, the clinic's spokesperson maintained that they offer a minimally invasive and natural treatment solution that helps patients avoid the costs, risks, and complications associated with surgeries. He added that their treatment plan is also devoid of medication use, which means patients can significantly dump the unhealthy habit of medication consumption while improving their health and wellness through other natural means.

The clinic's spokesperson noted that their sports and regenerative medicine treatment focuses on each patient's challenges and finds a way to improve their body's natural healing abilities. Speaking on the treatment modality's effectiveness for Houston Heights back pain treatment, Scott Hoots said: "In our QC Kinetix The Heights regenerative medicine clinics, we serve our patients by providing leading regenerative therapies that reduce joint pain associated with direct trauma, a sports-related injury, or a degenerative medical condition. These techniques target the impacted area to reduce inflammation and repair damaged tissue. Our team of medical professionals receives the highest quality training and research to provide our clients with the latest therapies to alleviate their ailments. In addition, we pride ourselves on providing the highest level of respect and care for our clients, as returning their ability to live with pain relief is our primary goal."

Each patient coming into the clinic will have access to a personalized health and wellness service centered around their needs. The treatment providers offer an initial consultation service where they get to learn more about the patient's health, symptoms, past treatments, allergies, and other information. Patients will also have access to a comprehensive examination and physical assessment for diagnostic purposes. Once diagnosed, the team will determine the patient's suitability for the treatment and develop a personalized treatment plan to alleviate their pain and discomfort while equipping the body with the needed building blocks for long-term health and wellness.

Welcoming Houston residents to schedule an appointment, the lead treatment provider at the clinic noted that regenerative medicine offers a wide range of benefits to patients. He maintained that several of their patients have been able to save money and avoid the complications of surgical intervention through their natural treatment plans. He further noted that their minimally invasive treatments are low risks and require a shorter recovery time compared to surgeries. Using stem cell therapy, platelet-rich plasma therapy, Class IV laser therapy, and others, patients will also enjoy positive health benefits like reduced inflammation, pain relief, improved healing, and a better range of motion from the affected joints.

QC Kinetix (The Heights) is currently taking new patients as its appointment slots are filling up fast. The pain control clinic can be reached via phone at (713) 913-5285 or via its website. The clinic is located at 1900 North Loop West, Suite 300, Houston, TX, 77018, US.

Media Contact:

Company Name: QC Kinetix (The Heights)

Contact Person: Scott Hoots

Phone: (713) 913-5285

Address: 1900 North Loop West, Suite 300

City: Houston

State: TX

Postal Code: 77018

Country: USA

Website: https://qckinetix.com/houston/the-heights/

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QC Kinetix (The Heights) Helping Patients Heal Better from Joint Pain Through Houston Heights Sports Medicine - Yahoo Finance

Minnesota Timberwolves star Karl-Anthony Towns was hospitalized last week with a throat infection that caused him trouble breathing and forced him to be on bed rest for days, sources told ESPN.

Towns spoke to the media Monday for the first time since falling ill and said his weight was down to 231 pounds (he's listed at 248), but he didn't reveal the nature of the non-COVID-19 illness.

Towns, who missed all of the Timberwolves' training camp, said he didn't get clearance to walk again until Saturday, when he attended a team event.

ESPN's countdown of the league's best players returns for its 12th season. See which stars made the cut, which vaulted to the top and which are sliding down the list.

NBArank 1-5: International stars on the rise NBArank 6-10: How far LeBron and KD fell NBArank 11-25: L.A. duo and rising Wolves NBArank 26-100: Russ, Ben and a host of Qs Debate! LeBron's ranking and top-10 tweaks

"I'm still recovering. I'm still getting better," Towns told reporters in Minneapolis before the team left on a 10-day preseason road trip. "There was more drastic things to worry about than basketball [during the illness]."

Timberwolves coach Chris Finch said Monday that Towns wouldn't play in Tuesday's preseason opener against the Heat in Miami as he works on ramping up his conditioning. During the offseason, Towns had stem-cell treatments and platelet-rich plasma injections in both knees, his left ankle, left wrist and right finger. He also signed a four-year, $224 million contract extension, tying him to the team for the next five seasons.

Towns averaged 24.6 points, 9.8 rebounds and shot 41% on 3-pointers last season, when he was named to the All-NBA team.

ESPN Reporter Ramona Shelburne contributed to this story

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Sources - Minnesota Timberwolves' Karl-Anthony Towns was on bed rest for days due to throat infection - ESPN

Editor's Note

This article is brought to you by Children's Hospital New Orleans.

While Childrens Hospital New Orleans has long been home to a nationally-known pediatric hematology/oncology program, it now has the state-of-the-art facilities to match those services.

The hospitals $300 million campus transformation included an expansion of its Center for Cancer and Blood Disorders. The center treats more than 1,100 children each year with leukemia, lymphoma, sickle cell anemia, hemophilia and other childhood cancers and blood disorders. That is more pediatric patients that all other Louisiana facilities combined. The expanded unit encompasses 10,880 square feet, 16 spacious exam rooms and a cohesive space that combines all specialty clinics in one area.

There is plenty of evidence architecture and interior design make a difference for people, said Dr. Charles Hemenway, who helps lead Childrens Hospital New Orleans pediatric hematology and oncology services. An open and sunny space is more conducive to making someones attitude bright and sunny. The medical care doesnt change, but I think peoples overall outlooks are shaped in part by their surroundings not just for children and families, but also for the health care providers. It's a space where people can feel good about themselves and the work they are doing.

The Center for Cancer and Blood Disorders is home to a care team comprised of the Gulf Souths largest group of hematology and oncology physicians and nurses dedicated exclusively to pediatrics. Most patients are from Louisiana, although they have treated children from Mississippi and Alabama, as well as international countries.

Lynn Winfield, MBA, BSN, RN, CPHON, NEA-BC, Childrens Hospital New Orleans senior director of patient care services, said that when a new patient arrives for cancer or blood disorder treatment, the staff meets with the family to outline the care plan and answer questions.

Its a multidisciplinary team that includes physicians, residents and nurse practitioners. We have nurse navigators who help educate the family. We have great social workers, and one of them is usually in that conference, Winfield said. Families are overwhelmed and they dont retain every piece of information right away. Thats why we continue to meet with them as time goes on. Its a team that provides not only medical support, but financial and psychological support as well.

That makes a big difference to patients like Ashlynn and her family. After being diagnosed with cancer, Ashlynn underwent several rounds of radiation, chemotherapy and stem cell treatments. But, today she can live like a kid again.

I am grateful for our Childrens Hospital family who, every day, give us something extraordinary to celebrate," Ashlynns mom Kim wrote in a letter to the staff. When I became a mother, I never imagined that this would be my story to share. But I am grateful Ashlynns story is one of hope and promise, thanks to the extraordinary caregivers at Childrens Hospital and friends in our community.

Dr. Hemenway said a child may remain at Childrens Hospital New Orleans anywhere from a few days to several weeks, depending on their condition and associated complications. Regardless of the length of their stay, patients also enjoy support from the community. For example, as a patient named Cam recovered from a transplant at Childrens Hospital New Orleans, his room overlooked the Audubon Park baseball fields where the Isidore Newman School team plays. After inspiration from Cams dad and a few phone calls, the team decided to turn around and wave at Cam before a game. Later, team moms installed an All In For Cam sign behind the Newman dugout.

The Center for Cancer and Blood Disorders also allows physicians to conduct cutting edge research and house a dedicated clinical trials team. Last year, this team worked on more than 90 cancer and blood disorder studies.

There are all kinds of ways to conduct research, starting with understanding cancer at its molecular origins all the way to finding the best medicines and dosages to achieve a good outcome, Dr. Hemenway said. With childhood cancer, we have made tremendous progress using medications that were developed many years ago. Because of clinical research, we are able to conclude every few years that some combination of therapies works better than what we were using in the past.

Winfield has seen the impact of this research firsthand. When she first started working at Childrens Hospital New Orleans 30 years ago, Winfield said the staff always knew they would lose more than a dozen children each year to disease. Today, that figure is greatly reduced and often is in the single digits.

However, because three out of five children who survive pediatric cancer experience late-developing side effects, Childrens Hospital New Orleans created Louisianas first and only dedicated pediatric cancer survivorship clinic, The Treatment After Cancer and Late Effects Clinic. Services include promoting follow-up appointments and routine tests and education on the long-term effects of cancer treatment. There are also emotional, psychological and social support services to help patients cope with the aftermath of a cancer diagnosis.

This job has allowed me to meet and be inspired by people that I never would have encountered, Winfield said. Its not an atmosphere of sad children sitting around and crying. Theres a lot of joy in it and people are so appreciative no matter the outcome. The beauty of this work lies in the relationships we form.

For more information, visit https://www.chnola.org/our-services/hematology-oncology/.

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Innovative spaces and an expert team help Childrens Hospital New Orleans set the standard in cancer care - NOLA.com

OverviewWhat is a stem cell transplant?

Healthcare providers use stem cell transplants to treat people who have life-threatening cancer or blood diseases caused by abnormal blood cells. A stem cell transplant helps your body replace those blood cells with healthy or normal blood cells. If you receive a stem cell transplant, your provider may use your own healthy stem cells or donor stem cells.

Your blood cells come from stem cells in your bone marrow. Your bone marrow constantly creates new stem cells that become blood cells. Stem cell transplants can involve stem cells taken from bone marrow or from blood. Providers sometimes refer to stem cell and bone marrow transplants as haematopoietic stem cell transplants (HSCT). This article focuses on stem cells taken from blood.

Healthcare providers use stem cells to replace unhealthy blood cells that cause conditions such as several types of leukemia, lymphoma and testicular cancer. They also use transplanted stem cells to treat several types of anemia. Some people who have multiple sclerosis may benefit by receiving healthy stem cells. Researchers are investigating ways to treat other autoimmune diseases with stem cell transplants.

Healthcare providers typically use stem cell transplants to treat life-threatening cancer or blood diseases. Unfortunately, not everyone who has those conditions can have the procedure. Here are factors providers take into consideration:

Recently data reported nearly 23,000 people had stem cell transplants in 2018.

To understand how stem cell transplants work, it may help to know more about stem cells and their role in your body:

Healthcare providers obtain stem cells from several sources:

If youre a candidate for a stem cell transplant, your healthcare provider will perform the following tests to confirm youre physically able to manage transplantation processes, including pre-treatment chemotherapy called conditioning and transplantation side effects:

Before your blood tests, your provider may place a central venous catheter (CVC) in one of the large veins in your upper chest. CVCs are tubes that serve as central lines that providers use to take blood and provide medication and fluids. CVCs eliminate repeated needle sticks to draw blood or insert intravenous tubes throughout the transplantation process.

Transplant conditioning is intensive chemotherapy and/or radiation therapy that kills cancer cells in your bone marrow. Conditioning also kills existing blood cells.

If youre receiving your own stem cells, your provider may give you medication to boost your stem cell production. Theyll do follow-up blood tests to check on stem cell production.

If youre receiving your own stem cells, your providers will take blood so they can remove healthy stem cells for transplant. . To do that, they connect veins in both of your arms to a cell separator machine. The machine pulls your blood from one arm, filters the blood and then returns it to through your other arm. This process doesnt hurt. Providers may need to take blood more than once to ensure they have enough stem cells to transplant. The actual transplantation involves receiving your stem cells via your CVC.

Just like someone receiving their own cells, youll receive healthy stem cells via your CVC.

Your new stem cells will need time to produce new blood cells. If you received donor stem cells, your transplanted stem cells will replace unhealthy stem cells and begin to build a new immune system. This process is engraftment.

Either way, you may need to stay in or close to the hospital for several months so your healthcare providers can support your recovery and monitor your progress. Heres what you can expect after your stem cell transplant:

Successful stem cell transplants may help people when previous treatments dont slow or eliminate certain cancers.

The greatest risk is that youll go through the procedure and your transplanted stem cells cant slow or eliminate your illness.

Allogenic and autologous stem cell transplants have different complications. Allogenic stem cell transplants can result in graft versus host disease. This happens when your immune system attacks new stem cells. Potential complications will vary based on your overall health, age and previous treatment. If youre considering a stem cell transplant, your healthcare provider will outline potential complications so you can weigh those risks against potential benefits.

It can take several weeks to several months to recover from a stem cell transplant. Your healthcare provider may recommend you stay in or near the hospital or transplant center for the first 100 days after your procedure.

Its difficult to calculate an overall success rate. That said, the most recent data show the highest number of stem cell transplants involved people with multiple myeloma or Hodgkin and non-Hodgkin lymphoma who received autologous stem cell transplants. Here is information on three-year survival rates:

A successful stem cell transplant can change your life, curing your condition or slowing its growth. But its not an overnight transformation. It can take a year or more for you to recover. Here are some challenges and ways to overcome them:

You may have days when you feel exhausted and days when you feel fine. A hard day doesnt mean youre not doing well. It means you need to give yourself a break and take it easy.

Youll have regular follow-up appointments with your provider. But its important to remember your immune system likely will be weak for a year or so after your transplantation. Contact your provider right away if you develop any of the following symptoms:

A note from Cleveland Clinic

If youve been coping with cancer or a blood disease, a stem cell transplant can be a new lease on life. It can mean hope for a cure or remission when other treatments havent worked. But stem cell transplants come with demanding physical challenges and significant risks. Not everyone who has cancer or blood conditions is a candidate for a stem cell transplant. Unfortunately, not everyone who is a candidate but needs donor stem cells finds a donor. If youre considering a stem cell transplant, talk to your healthcare provider about potential risks and benefits. Theyll evaluate your situation, your options and potential outcomes.

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Stem Cell Transplantation: What it Is, Process & Procedure

LOS ANGELES Cedars-Sinai researchers say a new stem cell therapy procedure allows them to protect patients with ALS, or Lou Gehrigs disease, and helps block muscle deterioration which normally occurs as a result of the fatal neurological disorder.

The Cedars-Sinai team successfully engineered and embedded protective proteins through the blood-brain barrier of patients with amyotrophic lateral sclerosis (ALS). The procedure increases hope that similar one-time treatments will greatly slow the diseases degenerative effects, including limb paralysis and the loss of ones ability to move, speak, or breathe. Researchers did not encounter any negative side-effects, while patients avoided leg paralysis following the transplant and replication of protein-producing stem cells from patients central nervous systems.

This breakthrough investigational therapy promotes the survival of motor neurons which typically degenerate in the spinal cord of patients with ALS. The researchers highlighted that none of the 18 people who underwent the stem cell gene therapy endured any serious side-effects after the transplantation.

Using stem cells is a powerful way to deliver important proteins to the brain or spinal cord that cant otherwise get through the blood-brain barrier, says corresponding author Clive Svendsen, PhD, professor of Biomedical Sciences and Medicine and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, in a media release.

The safety of the trial and a lack of side-effects among patients is receiving praise from numerous scientists in the ALS and neurological research community.

We were able to show that the engineered stem cell product can be safely transplanted in the human spinal cord. And after a one-time treatment, these cells can survive and produce an important protein for over three years that is known to protect motor neurons that die in ALS, Svendsen says.

The researchers had several primary goals in this trial, which sought to show that stem cells engineered in Svendsens laboratory could produce the glial cell line-derived neurotrophic factor (GDNF), which help motor neurons pass signals between the brain and spinal cord. This process ultimately allows ALS patients to continue muscle movement which the disease typically destroys.

Prior to this recent study, neurological researchers feared there could be dire side effects or an inability to successfully bypass ALS patients blood-brain barrier using this therapeutic procedure. The blood-brain barrier, or BBB, serves as a structural and functional roadblock to potentially harmful microorganisms including parasites, viruses or bacteria in a persons bloodstream.

Because they are engineered to release GDNF, we get a double whammy approach where both the new cells and the protein could help dying motor neurons survive better in this disease, Svendsen added.

Researchers say they will soon build on the findings, published in the peer-reviewed journal Nature Medicine, including tests which target lower areas on the spinal cord and enrolling ALS patients in the study much earlier in their diagnosis.

We are very grateful to all the participants in the study, Svendsen concludes. ALS is a very tough disease to treat and this research gives us hope we are getting closer to finding ways to slow down this disease.

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New stem cell therapy provides long-term brain protection against ALS - Study Finds

From left, T. Denny Sanford, Catriona Jamieson, MD, PhD, and Chancellor Pradeep K. Khosla celebrate the establishment of the UC San Diego Sanford Stem Cell Institute, made possible by a historic gift from Sanford.

Noted businessman and philanthropist T. Denny Sanford has committed $150 million in new funding to expand and, in some ways, quite literally launch stem cell research and regenerative medicine at University of California San Diego into new spaces and endeavors.

The gift will fund the new UC San Diego Sanford Stem Cell Institute and builds upon a $100 million gift in 2013 from Sanford that boldly established UC San Diego as a leader in developing and delivering the therapeutic promise of human stem cells special cells with the ability to develop into many different cell types and which, when modified and repurposed, have the potential to treat, remedy or cure a vast array of conditions and diseases.

Dennys previous generosity spurred discoveries in stem cell research and medicine at UC San Diego that are already benefiting countless patients around the world, said Chancellor Pradeep K. Khosla. His most recent gift adds to our portfolio of stem cell research conducted in Earths orbit that will help us better understand the progression of cancer cells and aging.

Sanfords gift to establish the Sanford Stem Cell Institute is the largest single gift to UC San Diego. This investment enables the team to dream beyond what is possible, said Sanford. The sky is no longer the limit.

In addition to his investment to create the Sanford Stem Cell Clinical Center at UC San Diego Health in 2013, Sanford established the T. Denny Sanford Institute for Empathy and Compassion in 2019, which focuses on research into the neurological basis of compassion, with application toward developing compassion and empathy-focused training for future generations of medical professionals. He also recently made a $5 million gift to support the Epstein Family Alzheimers Research Collaboration, a partnership between UC San Diego and the University of Southern California to spark new collaborative efforts to discover effective therapies for Alzheimers disease.

Sanford was also honorary co-chair of the Campaign for UC San Diego, which concluded in June 2022 having raised more than $3 billion exceeding its initial $2 billion goal. He was honored as a recipient of the 2014 Chancellors Medal, one of the universitys highest honors, in recognition of his exceptional service in support of the campus mission.

Stem cell research will be conducted in a laboratory bay located aboard the International Space Station, pictured here, in low-Earth orbit. Credit: NASA

The new UC San Diego Sanford Stem Cell Institute, under the direction of Catriona Jamieson, MD, PhD, Koman Family Presidential Endowed Chair in Cancer Research in the UC San Diego School of Medicine, will continue three existing stem cell programs at UC San Diego with three new programs.

The new programs to be established with Sanfords gift include:

Existing stem cell programs at UC San Diego in the Sanford Stem Cell Institute include:

We are thrilled to announce the establishment of the UC San Diego Sanford Stem Cell Institute with Denny Sanfords generous support, said Jamieson. This will allow us to keep pace with the growing need for regenerative and stem-cell based therapies and accelerate translational stem cell research and discoveries that will transform human health for years to come.

With three new programs established as part of the Sanford Stem Cell Institute, a key focus of the institute will be leveraging space as a new frontier for stem cell science. Exposure to radiation and microgravity in low-Earth orbit can simulate and speed up aging in stem cells, as well as their transformation into cancer cells. Space-related research may have applications that create better treatments for various cancers and diseases on earth, including blood cancers, as well as neurodegenerative diseases such as Alzheimers and Parkinsons.

To fuel sustained research and education in this promising area, Sanfords gift will establish the Sanford Stem Cell Institute STELLAR Endowed Chair in Regenerative Medicine, the Sanford Stem Cell Institute Endowed STELLAR Exploration Faculty Scholars and Fellows Fund, and the Sanford Stem Cell Institute STELLAR Exploration Discovery Fund.

UC San Diego already has expanded its research capacity in stem cell science to space efforts that will be further amplified with the recent gift.

In late 2021, UC San Diego worked with NASA, Space Tango and the JM Foundation to launch stem cells into space aboard a SpaceX Falcon 9 rocket to study stress-induced aging and how stem cells and their progeny transform into pre-cancer and cancer stem cells associated with leukemia and other blood cancers.

Allyson Muotri, PhD, with human organoid samples

In 2019, Alysson Muotri, PhD, professor of pediatrics and cellular and molecular medicine, and colleagues sent a payload of stem cell-derived human brain organoids to the International Space Station (ISS) orbiting almost 250 miles above Earth to study how these masses of cells organize into the beginnings of a functional brain in microgravity. The first-ever project of its type was dedicated to Sanford, a longtime supporter of Muotris work and others.

When I was designing these experiments, I realized how innovative and cutting edge they were, said Muotri. I thought Denny would be proud of this project, and that I should dedicate this first mission to him. Denny has been a cheerleader for the stem cell community. He is pushing all of us to speed discovery and translate it to help millions of people who suffer from different conditions that could be treated with stem cell-based therapies.

Since its inception in 2013, the Sanford Stem Cell Clinical Center at UC San Diego has yielded a three-fold return on investment by obtaining more than $312 million in funding, including $253.6 million in grants, $15.8 million in clinical trial contracts, $2.7 million in Advanced Cell Therapy Lab (ACTL) service charges and more than $40.2 million in philanthropy all with the goal of discovering new treatments to benefit patients.

Key successes include new pharmaceutical treatments Fedratinib, which was approved by the FDA for the treatment of myelofibrosis in 2019, and Glasdegib, FDA approved for acute myeloid leukemia in 2018.

Meanwhile, clinical trials are ongoing for Cirmtuzumab, a monoclonal antibody-based drug developed by Thomas Kipps, MD, PhD, Distinguished Professor of Medicine and deputy director of research at Moores Cancer Center at UC San Diego Health, and colleagues. Cirmtuzumab targets cancer stem cells and is being tested, alone and in combination with other drugs, to treat chronic lymphocytic leukemia and other blood cancers.

Stem cell research at UC San Diego has been a substantial beneficiary of the California Institute for Regenerative Medicine (CIRM), the states stem cell agency, created in 2004 with the approval of Proposition 71. UC San Diego researchers have garnered 116 awards totaling more than $227 million. Cirmtuzumab is named as a nod to CIRM and its support. In 2020, California voters passed Proposition 14 to continue CIRM operations and funding.

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$150 Million Gift Takes Stem Cell Research to New Heights - University of California San Diego

Immusofts steps to delivering a treatment using engineered B cells. (Screen grab from Immusofts website)

Seattle biotech startup Immusoft has received approval to begin clinical trials of its novel strategy for treating genetic disease, the company announced Thursday. Immusoft says its the first to get permission to use engineered immune system cells called B cells in a human study.

The U.S. Food and Drug Administration (FDA) approved Immusofts Investigational New Drug Application for testing its immunotherapy for a rare, lethal childhood disease called MPS I.

An immune response includes a suite of players, and the B cells are responsible for producing the antibodies that stick to invading bacteria and viruses. The company is able to modify B cells into biofactories that instead crank out missing or non-functioning enzymes and proteins in the cells of patients.

This is a huge achievement for the company and a historic moment in the field of cell and gene therapies, said Sean Ainsworth, Immusofts CEO and chairman, in a statement.

The approach has potential advantages to current strategies for delivering treatments. Therapies that use a virus as its delivery mechanism can trigger immune responses that limit their effectiveness. Treatments using stem cells can have difficulties associated with chemotherapy and stem cell transplants.

Immusofts technology, called ISP-001, uses a patients own B cells, reprogramming them to make needed proteins. Other companies working on B cell therapies include Be Biopharma and Walking Fish Therapeutics.

I dont know if they are going to be successful, but its exciting for all of us that they have gotten permission to start a trial, researcher Richard James told MIT Technology Review. Jamess lab at the University of Washington is also working on B cell engineering.

The trial will be done at the University of Minnesota Medical School and led by Dr. Paul Orchard, a professor in the universitys Division of Pediatric Bone Marrow Transplantation.

Children with MPS I are not able to produce an essential enzyme that helps break down long-chain sugars inside cells. The sugars then build up in cells, causing progressive damage. Severe MPS1 occurs in about 1 in 100,000 births, and symptoms appear within a year.

Immusoft is interested in expanding its therapy to other rare diseases, as well as cardiovascular, autoimmune and central nervous system diseases.

In October 2021, the company announced a collaboration with pharma giant Takeda to develop treatments targeted to the nervous system in a deal worth potentially more than $900 million.

Immusoft was founded in 2009 and has raised more than $50 million in venture capital, according to PitchBook. In 2018, Ainsworth took over leadership from founder Matthew Scholz.

Scholz is now CEO of Oisn Biotechnologies, a startup developing preclinical therapies that target and kill damaged zombie cells. He is still on Immusofts board of directors.

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Seattle biotech company is the first to receive approval to test B cell gene therapy in humans - GeekWire

SANTA MONICA, Calif.--(BUSINESS WIRE)--Kite, a Gilead Company (Nasdaq: GILD), today announced that the European Commission (EC) has approved its CAR T-cell therapy 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).

This approval makes Tecartus the first and only CAR T-cell therapy indicated for this population of patients, addressing a significant unmet medical need, said Christi Shaw, CEO, Kite. This is also the fourth indication in Europe for which a Kite cell therapy is approved, clearly demonstrating the benefits they offer to patients, especially those with limited treatment options.

ALL is an aggressive type of blood cancer; the most common form is B-cell precursor ALL. Globally, approximately 64,000 people are diagnosed with ALL each year. Half of adults living with ALL will relapse, and median overall survival (OS) with current standard-of-care treatments is approximately just eight 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. Patients in Europe now have a meaningful advancement in treatment. Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.

The approval is supported by data 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. This study 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 pivotal dosed patients (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) safety results were consistent with the known safety profile for Tecartus. Grade 3 or higher cytokine release syndrome (CRS) and neurologic adverse reactions 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, Europe), 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.

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 92% (72/78) of patients with ALL, including Grade 3 (Lee grading system 1) CRS in 26% of patients. Three patients with ALL had ongoing CRS events at the time of death. The median time to onset of CRS was five days (range: 1 to 12 days) and the median duration of CRS was eight days (range: 2 to 63 days) for patients with ALL.. 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 June 30, 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.

Excerpt from:
Kite's CAR T-cell Therapy Tecartus Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia...

The findings suggest stem cell-based replacement therapy has the potential to manage blood sugar levels and may eventually replace the requirement for external insulin injections or dosing.

A clinical study presented at ENDO 2022, the Endocrine Societys annual meeting in Atlanta, Georgia, suggests that an investigational stem cell-based therapy called PEC-Direct, intended to function as a replacement pancreas, has the potential to provide blood sugar control in patients with high-risk type 1 diabetes.

The research discovered that numerous individuals receiving the new treatment showed clinically significant increases in C-peptide, a substance produced in the pancreas alongside insulin. Measuring C-peptide can reveal how much insulin the body is producing since they are both released from the pancreas at the same time and insimilar quantities.

This research represents the first instance in multiple patients of clinically relevant increases in C-peptide, indicative of insulin production, with a stem cell-based therapy delivered in a device, according to Manasi Sinha Jaiman, M.D., M.P.H., Chief Medical Officer of ViaCyte, Inc., in San Diego, Calif., the company that makes PEC-Direct.

People with type 1 diabetes gradually lose the ability to produce insulin on their own, which is necessary for blood sugar regulation. Patients must check those levels on a regular basis using finger sticks, administer repeated insulin injections, or carry around cumbersome devices. Additionally, there is a chance that the insulin injection can inadvertently drop blood sugar to unsafe levels.

The PEC-Direct device is intended to provide a consistent, long-term sourceof insulin to control blood sugar levels. The system consists of a pouch containing pancreatic cells produced from stem cells that, when implanted in the body, develop into cells that produce insulin. The devices open membrane enables blood vessels to grow into it to contact the cells. Patients use immunosuppressive drugs to prevent an immune reaction.

The treatment is meant for patients with high-risk type 1 diabetes, who may be especially vulnerable to acute complications due to factors such as recurrent severe low blood sugar, or frequent and extreme blood sugar fluctuations that are difficult to control.

The study included 10 adults with type 1 diabetes who had received their diagnosis at least 5 years prior to the start of the study and were not able to tell when their blood sugar went too low (called hypoglycemia unawareness). Initial data from one patient showed clinically relevant levels of stimulated C-peptide and corresponding improvements in blood glucose control within six months after implantation of PEC-Direct.

Since then, increased C-peptide levels were seen in multiple patients, along with decreases in HbA1C (a blood test that measures average blood sugar levels over the past three months) by as much as 1.5%, and decreases in the amount of insulin patients needed to administer by as much as 70%.

The results suggest stem cell-based replacement therapy has the potential to provide blood glucose control and could one day eliminate the need for injecting or dosing insulin externally, Jaiman said. The study provides further proof-of-concept that continued optimization of PEC-Direct has promise as a functional cure for type 1 diabetes.

Meeting: ENDO 2022

Read more:
Could Stem-Cell Based Therapy Treat Type-1 Diabetes? A New Study Demonstrates the Treatments Potential - SciTechDaily

BOSTON and LONDON, Aug. 29, 2022 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced seven presentations from across its neurometabolic portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, taking place from August 30 to September 2, 2022, in Freiburg, Germany.

Featured presentations include an oral presentation on Libmeldy (atidarsagene autotemcel) from clinical development through approval by the European Commission and treatment of the first patients in a commercial setting in Europe, several accepted abstracts highlighting newborn screening efforts to support the timely and accurate diagnosis of metachromatic leukodystrophy (MLD), as well as an encore clinical data presentation from the companys investigational hematopoietic stem cell (HSC) gene therapy OTL-203 for MPS-IH.

The oral presentation details are as follows:

The poster presentation details are as follows:

About Libmeldy / OTL-200Libmeldy (atidarsagene autotemcel), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies of Libmeldy, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard TherapeuticsAt Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S. headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

Availability of Other Information About OrchardInvestors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (TwitterandLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-looking StatementsThis press release contains forward-looking statements, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards most recent annual or quarterly report filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

See the original post:
Orchard Therapeutics Announces Multiple Presentations at 2022 SSIEM Annual Symposium Highlighting Neurometabolic Disease Portfolio - GlobeNewswire