Getting a Stem Cell or Bone Marrow Transplant

When the decision is made to have a stem cell or bone marrow transplant, there are several steps in theprocess. The steps are much the same, no matter what type of transplant youre going to have.

You will first be evaluated to find out if you are eligible for a transplant. A transplant is very hard on your body. For many people, transplants can mean a cure, but for some people, problems can lead to severe complications or even death. Youll want to weigh the benefits and risks before you start.

Transplants can also be hard emotionally. They often require being in the hospital, being isolated, and theres a high risk of side effects. Many of the effects are short-term, but some problems can go on for years. This can mean changes in the way you live your life. For some people its just for a while, but for others, the changes may be lifelong. Some of the side effects are really unpleasant and can be serious. Your cancer care team will do everything they can to make you comfortable, but some of the side effects may not be completely controlled or relieved.

Before you have a transplant, you need to discuss the transplant process and all its effects with your doctors. It also helps to talk with others who have already had transplants.

Its also very hard going through weeks and months of not knowing how your transplant will turn out. This takes a lot of time and emotional energy from the patient, caregivers, and loved ones. Its very important to have the support of those close to you. For example, youll need a responsible adult who will be with you to give you medicines, help watch for problems, and stay in touch with your transplant team after you go home. Your transplant team will help you and your caregiver learn what you need to know. The team can also help you and your loved ones work through the ups and downs as you prepare for and go through the transplant.

Many different medical tests will be done, and questions will be asked to try to find out how well you can handle the transplant process. These might include:

You will also talk about your health insurance coverage and related costs that you might have to pay.

You may have a central venous catheter (CVC) put into a large vein in your chest. This is most often done as outpatient surgery, and usually only local anesthesia is needed (the place where the catheter goes in is made numb). Nurses will use the catheter to draw blood and give you medicines.

If youre getting an autologous transplant, a special catheter can be placed that can also be used when your stem cells are being removed or harvested.

The CVC will stay in during your treatment and for some time afterward, usually until your transplanted stem cells have engrafted and your blood counts are on a steady climb to normal.

Younger people, people who are in the early stages of disease, or those who have not already had a lot of treatment, often do better with transplants. Some transplant centers set age limits. Some people also may not be eligible for transplant if they have other major health problems, such as serious heart, lung, liver, or kidney disease. A mini-transplant, described under Allogeneic stem cell transplant in Types of Stem Cell Transplants for Cancer Treatment may be an option for some of these people.

The hospitals transplant team will decide if you need to be in the hospital to have your transplant, if it will be done in an outpatient center, or if you will be in the hospital just for parts of it. If you have to be in the hospital, you will probably go in the day before pre-transplant chemo or radiation treatment begins (see the next section), the transplant team makes sure you and your family understand the process and want to go forward with it.

If you will be having all or part of your transplant as an outpatient, youll need to be very near the transplant center during the early stages. Youll need a family member or loved one to be a caregiver who can stay with you all the time. You and the caregiver will also need reliable transportation to and from the clinic. The transplant team will be watching you closely for complications, so expect to be at the clinic every day for a few weeks. You may still need to be in the hospital if your situation changes or if you start having complications.

To reduce the chance of infection during treatment, patients who are in the hospital are put in private rooms that have special air filters. The room may also have a protective barrier to separate it from other rooms and hallways. Some have an air pressure system that makes sure no unclean outside air gets into the room. If youre going to be treated as an outpatient, you will get instructions on avoiding infection. Usually, people who have transplants are in a separate, special part of the hospital to keep as many germs away as possible.

The transplant experience can be overwhelming. Your transplant team will be there to help you prepare for the process physically and emotionally and to discuss your needs. Every effort will be made to answer questions so you and your family fully understand what will be happening to you as you go through transplant.

Its important for you and your family to know what to expect, because once conditioning treatment begins (see the next section), theres no going back there can be serious problems if treatment is stopped at any time during transplant.

Having a transplant takes a serious commitment from you and your caregiver and family, so it is important to know exactly what to expect.

Conditioning, also known as pre-transplant treatment,bone marrow preparation, or myeloablation, is usually treatment with high-dose chemo and/or radiation therapy. Its the first step in the transplant process and typically takes a week or two. Its done for one or more of these reasons:

The conditioning treatment is different for every transplant. Your treatment will be planned based on the type of cancer you have, the type of transplant, and any chemo or radiation therapy youve had in the past.

If chemo is part of your treatment plan, it will be given in your central venous catheter and/or as pills. If radiation therapy is planned, its given to the entire body (called total body irradiation or TBI). TBI may be given in a single treatment session or in divided doses over a few days.

This phase of the transplant can be very uncomfortable because very high treatment doses are used. Chemo and radiation side effects can make you sick, and it may take you months to fully recover. A very common problem is mouth sores that will need to be treated with strong pain medicines. You may also have nausea, vomiting, be unable to eat, lose your hair, and have lung or breathing problems.

Conditioning can also cause premature menopause in women and often makes people sterile (unable to have children). (See Stem Cell Transplant Side Effects.)

After the conditioning treatment, youll be given a couple of days to rest before getting the stem cells. They will be given through your central venous catheter, much like a blood transfusion. If the stem cells were frozen, you might get some drugs before the stem cells are given. These drugs are used to help reduce your risk of reacting to the preservatives that are used when freezing the cells.

If the stem cells were frozen, they are thawed in warm water then given right away. There may be more than 1 bag of stem cells. For allogeneic or syngeneic transplants, the donor cells may be harvested (removed) in an operating room, and then processed in the lab right away. Once they are ready, the cells are brought in and given to you theyre not frozen. The length of time it takes to get all the stem cells depends on how much fluid the stem cells are in.

You will be awake for this process, and it doesnt hurt. This is a big step and often has great meaning for patientsand their families. Many people consider this their rebirth or chance at a second life. They may celebrate this day as they would their actual birthday.

Side effects from the infusion are rare and usually mild. The preserving agent used when freezing the stem cells causes many of the side effects. For instance, you might have a strong taste of garlic or creamed corn in your mouth. Sucking on candy or sipping flavored drinks during and after the infusion can help with the taste. Your body will also smell like this. The smell may bother those around you, but you might not even notice it. The smell, along with the taste, may last for a few days, but slowly fades away. Often having cut up oranges in the room will offset the odor. Patients who have transplants from cells that were not frozen do not have this problem because the cells are not mixed with the preserving agent.

Other side effects you might have during and right after the stem cell infusion include:

Again, side effects are rare and usually mild. If they do happen, they are treated as needed. The stem cell infusion must always be completed.

The recovery stage begins after the stem cell infusion. During this time, you and your family wait for the cells to engraft, or take, after which they start to multiply and make new blood cells. The time it takes to start seeing a steady return to normal blood counts varies depending on the patient and the transplant type, but its usually about 2 to 6 weeks. Youll be in the hospital or visit the transplant center daily for a number of weeks.

During the first couple of weeks youll have low numbers of red and white blood cells and platelets. Right after transplant, when your counts are the lowest, you may be given antibiotics to help keep you from getting infections. You may get a combination of anti-bacterial, anti-fungal, and anti-viral drugs. These are usually given until your white blood cell count reaches a certain level. Still, you can have problems, such as infection from too few white blood cells (neutropenia), or bleeding from too few platelets (thrombocytopenia). Many patients have high fevers and need IV antibiotics to treat serious infections. Transfusions of red blood cells and platelets are often needed until the bone marrow starts working and new blood cells are being made by the infused stem cells.

Except for graft-versus-host disease, which only happens with allogeneic transplants, the side effects from autologous, allogeneic, and syngeneic stem cell transplants are much the same. Problems may include stomach, heart, lung, liver, or kidney problems. (Stem Cell Transplant Side Effects goes into the details.) You might also go through feelings of distress, anxiety, depression, joy, or anger. Adjusting emotionally after the stem cells can be hard because of the length of time you feel ill and isolated from others.

You might feel as if you are on an emotional roller coaster during this time. Support and encouragement from family, friends, and the transplant team are very important to get you through the challenges after transplant.

The discharge process actually begins weeks before your transplant. It starts with the transplant team teaching you and your primary (main) caregiver about:

For the most part, transplant centers dont send patients home until they meet the following criteria:

(Why Are Stem Cell Transplants Used as Cancer Treatment? has more information about neutrophils, platelets, and hematocrit).

If you do not meet all of these requirements, but still dont need the intensive care of the transplant unit, you might be moved to another oncology unit. When you do go home, you might need to stay near the transplant center for some time, depending on your condition.

The process of stem cell transplant doesnt end when you go home. Youll feel tired, and some people have physical or mental health problems in the rehabilitation period. You might still be taking a lot of medicines. These ongoing needs must now be managed at home, so caregiver and friend/family support is very important.

Transplant patients are followed closely during rehab. You might need daily or weekly exams along with things like blood tests, and maybe other tests, too. During early rehab, you also might need blood and platelet transfusions, antibiotics, or other treatments. At first youll need to see your transplant team often, maybe even every day, but youll progress to less frequent visits if things are going well. It can take 6 to 12 months, or even longer, for blood counts to get close to normal and your immune system to work well. During this time, your team will still be closely watching you.

Some problems might show up as much as a year or more after the stem cells were infused. They can include:

Other problems can also come up, such as:

Your transplant team is still there to help you, even though the transplant happened months ago. Its important that you tell them about any problems you are having they can help you get the support you need to manage the changes that you are going through. They can also help you know if problems are serious, or a normal part of recovery. The National Bone Marrow Transplant Link helps patients, caregivers, and families by providing information and support services before, during, and after transplant. They can be reached at 1-800-LINK-BMT (1-800-546-5268) or online at http://www.nbmtlink.org.

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Getting a Stem Cell or Bone Marrow Transplant

What is Stem Cell Research? | The Benefits of Stem Cell …

Stem cell research is one of the most innovative research methods being used in modern society. However, it is also not very well known in todays society. Most people are either introduced to it when exposed to the treatment themselves or through a loved one.I was exposed to stem cell research in the ninth grade. Since I went to a STEM(science, technology, engineering, and mathematics) high school, my biology teacher assigned us a report on stem cell research. Since doing that report, I have become passionate about stem cell research and have followed closely the advancements made in the field. Current research can easily be tracked on the National Institute for Health website.

So what is a stem cell? Astem cell is simply a cell that can either reproduce another stem cell or a specialized cell an infinitive amount of times. The specialized cells that can be produced have specific function related to where they are produced in the human body.Stem cell research is also categorized by the stem cell typeused.

Adult stem cells, or somatic cells, are produced from the human body once an individual is born. It can renew itself or become a specialized cell within the body just like the stem cells I explained above. They have no known origin, but they are used in most of the groundbreaking research that is currently happening.

Embryonic stem cells are more controversial since they are produced in the embryo stage of development. All embryonic stem cells originate from an embryo. They are usually grown in a laboratory within a cell culture. There are many misconceptions of embryonic stem cells which will be addressed in the next blog post.

Both types of stem cells have advantages and disadvantages, but the advantage of stem cell research heavily outweigh the disadvantages. It is slowly becoming the future of medicine.

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What is Stem Cell Research? | The Benefits of Stem Cell ...

Is Stem Cell Therapy Right for You? – Health Essentials from Cleveland Clinic

Few of us know what they are or exactly how they work. But many of us have heard about the healing powers of stem cells, as well as the controversy surrounding them. Stem cells are well-debated and highly complex with promises ranging from fixing damaged knees to regenerating receding hairlines.

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But what are stem cells? And, whats all the fuss all about?

Director of the Center for Regenerative Medicine and Surgery, Amy Lightner, MD, shares the differences between stem cell types, how stem cells can be used and when to be cautious of claims that might be too good to be true.

When most of us think of stem cells, we probably recall images of Dolly the cloned sheep. While its true that Dolly was born of stem cells, her place in science history is just one of many advancements in the field.

In fact, there are many different types of stem cells, each of which has different responsibilities and abilities. What unifies them is their ability to regenerate into new cells.

Regenerative medicine is an emerging field that uses innovative treatments to help regenerate or heal cell function thats lost due to aging, disease or injury, Dr. Lightner explains. The way we achieve this is by using stem cells in large quantities, targeted to a certain area, that the body uses to promote healing.

Adult stem cells are the only type of stem cells that are currently approved for medical use in the United States by the U.S. Food and Drug Administration (FDA). The term adult stem cells is a little confusing because theyre actually found in infants, children and adults. These cells live in a variety of tissue in our bodies including bone marrow, muscles, your brain, your intestines and more.

Think of adult stem cells as a little army of cells that can regenerate themselves into new cells to maintain and repair the tissue or muscle where theyre found. The catch with adult stem cells is that they cant become different types of cells (for example, blood stem cells can only become new blood cells, not skin or brain cells).

Unlike adult stem cells, embryonic stem cells have many more possibilities. Harvested during an embryos blastocyst stage (about five or six days after an embryo has been fertilized in a lab), embryonic stem cells have the potential to become any type of cell (called pluripotent cells). For these reasons, embryonic stem cells are the type of stem cells that generate the controversy most people associate with the topic.

Stem cell therapy has been around since the 1970s, when the first adult bone marrow cells were used to treat blood disease. A bone marrow transplant allows a recipient whose bone marrow cells have been damaged by chemotherapy or disease to receive healthy bone marrow stem cells from a donor.

Those stem cells have the potential to mature within the blood system into different immune cells that recognize and fight off different types of blood cancer. And they also have the ability to heal, says Betty Hamilton, MD, Department of Hematology and Medical Oncology.

Bone marrow transplants are currently used to treat diseases including:

While you may have heard about the use of stem cell therapy for knees, back pain, arthritis, hair loss, diabetes and more, no other types of stem cell therapy beyond bone marrow transplants have yet been approved by the FDA. But thousands of clinical trials are available ranging from treatments for Crohns disease to multiple sclerosis and more. The common link between all these trials is the ability of the stem cells to reduce inflammation and repair damage to your body.

Dr. Hamilton and Dr. Lightner agree that were only just beginning to scratch the surface of stem cell therapy. In recent years, during the height of the COVID-19 pandemic, many clinical trials were underway to explore whether stem cells could be used to help treat the damaged lungs in people severely affected by the disease.

I think potential is the perfect word to describe stem cells, says Dr. Hamilton. We know they have these anti-inflammatory and regenerative properties where they can provide a significant improvement to someone suffering from a certain disease. There are so many diseases where inflammation happens, and something needs to be repaired, and so any help the immune system can get provides a lot of potential.

Scientists are also researching whether adult stem cells can turn into pluripotent stem cells, which would allow the cells to change into any cell type without involving the use of embryonic stem cells.

While the potential for stem cell therapy is great, doctors caution that were not quite there yet.

I always tell patients that ask about stem cell therapy clinics or traveling overseas for stem cell therapy treatment that if its not something that is a clinical trial with FDA oversight, then they have no real way of knowing whats being given to them, advises Dr. Lightner.

This means more harm can come than good if you dont know exactly whats being given to you. Or, in some cases, youre just spending thousands of dollars for what ends up being saline, Dr. Lightner says.

The best way to know that youre receiving sound medical treatment is to make sure the one youre considering is approved by the FDA on its Clinical Trials database.

Dr. Lightner cautions against treatments that sound too good to be true. While stem cell therapy has helped improve and save millions of lives, its best to know what exactly youre signing up for by seeking out a qualified medical provider offering an FDA-approved clinical trial.

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Is Stem Cell Therapy Right for You? - Health Essentials from Cleveland Clinic

What is the potential stem cell ‘cure’ for HIV/AIDS all about? | In Focus podcast – The Hindu

Dr Akhil C Banerjea speaks to us on the rare mutation that naturally blocks HIV from entering cells and does the mRNA platform, first used for a COVID-19 vaccine, offer hope. Dr Akhil C Banerjea speaks to us on the rare mutation that naturally blocks HIV from entering cells and does the mRNA platform, first used for a COVID-19 vaccine, offer hope.

Up until January this year, only two people were ever reported cured of HIV/AIDS. And now, researchers have said there may be a third case -- an African American woman, who was diagnosed with HIV in 2013, and started on anti-retroviral therapy. In 2017, she was diagnosed with leukemia and received embryonic stem cells, in the form of cord blood, from a donor who had a rare mutation that naturally blocks HIV from infecting the bodys cells. She also received adult blood stem cells from a relative. Now, doctors say, the woman shows no signs of HIV in her blood and has no detectable antibodies either, making the 60-year-old woman possibly the third case of a person who has been cured of the virus -- the other two cases, both men, received bone marrow transplants as well, from donors with the rare mutation. Unlike the other two cases, however, the woman did not develop graft vs host disease -- a condition where the donor stem cells attack the recipient. Could this be because of the use of embryonic stem cells with adult stem cells? Her doctors believe this may possibly be a factor.

Stem cell therapy, exciting as it is in the field of medicine, is not accessible or possible in the case of a vast majority of persons living with HIV/AIDS in the world. Anti-retroviral therapy or ART however, has ensured that those with access to the medicines now have long lifespans, comparable to those without HIV/AIDS. A vaccine against the virus would be an ideal solution, offering a potential cure, but close to 40 years since researchers first began to study it, the world still does not have a vaccine for this virus, though there are recent reports of a potential vaccine based on the mRNA platform.

In India, as of 2019, an estimated 23.48 lakh people live with HIV/AIDS -- the prevalence among adult males is estimated at 0.24% of the population and among adult females, the prevalence is 0.20%. Worldwide, over 37 million people live with HIV/AIDS.

So what is the potential stem cell cure all about? What is the rare mutation that naturally blocks HIV from entering cells? Why is a vaccine so hard to make, and does the mRNA platform, first used for a COVID-19 vaccine, offer hope?

Guest: Dr Akhil C Banerjea, emeritus professor, National Institute of Immunology and former director, Institute of Advanced Virology, Kerala

Host: Zubeda Hamid

Edited by Reenu Cyriac

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What is the potential stem cell 'cure' for HIV/AIDS all about? | In Focus podcast - The Hindu

Stem cell therapy shows promise in aiding equine wound healing – Horsetalk.co.nz – Horsetalk

WCVM PhD student Dr Suzanne Mund. Rigel Smith

A team of researchers at the Western College of Veterinary Medicine (WCVM) in Saskatchewan has published the first equine study to demonstrate changes in wound healing following stem cell therapy.

The findings of the study, which received funding from the Mark and Pat DuMont Equine Orthopedic Fund and the WCVMs Townsend Equine Health Research Fund (TEHRF), were recently published online in Cells, an international open-access journal.

Team members include PhD student Dr Suzanne Mund along with WCVM faculty members Drs Daniel MacPhee, John Campbell, Ali Honaramooz, Bruce Wobeser and Spencer Barber.

The Canadian researchers used intravenous (IV) treatments of multipotent mesenchymal stromal cells (MSCs) that were extracted from other horses. These stem cells have potential for improving wound healing because they can alter the bodys inflammatory response, which is involved in healing. They can also influence other local cells to produce growth factors that could enhance the speed and quality of wound healing.

MSC therapy is a promising treatment for limb wounds, a common injury in horses that often develops complications, which can include the production of an excess amount of granulation tissue, commonly known as proud flesh.

There are risks associated with IV administration of MSC, and so far, the therapys effectiveness in improving cutaneous wound healing is unknown.

The WCVM research team was successful in administering the highest dose of MSCs ever administered to horses enrolled in the study (using any type of delivery). Contrary to the teams hypothesis, the treated horses did not experience accelerated wound closure or improved histologic healing. However, the horses healed wounds did have smaller immature scar sizes, which may signal a better repair in terms of cosmetics and function.

The stem cell therapy also appeared to alter the cytokine profile within the horses wounds. Cytokines are small proteins that play a role in controlling the growth and activity of other immune system cells and blood cells. After treatment, there was less expression of all measured cytokine types except for antifibrotic mediators.

This finding is contrary to researchers understanding that more acute inflammation followed by rapid resolution improves limb wound healing.

Another concern was that several of the horses in the treatment group temporarily developed minor reactions after receiving stem cell therapy. Since one horse in the control group also experienced similar transient reactions, the cause may be related to the cell suspension solution used or to other external factors rather than to the cells themselves.

While MSC intravenous therapy has the potential to decrease the size of limb wounds in horses, researchers need to do further studies before this therapy can be recommended as an effective wound healing tool for veterinarians in the field. More work also needs to be done to understand the clinical relevance of adverse reactions that were observed in the studys horses.

Reprinted with permission from the Western College of Veterinary Medicines Townsend Equine Health Research Fund.

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Stem cell therapy shows promise in aiding equine wound healing - Horsetalk.co.nz - Horsetalk

Early Treatment Matters More Than Ever in Multiple Myeloma, Kumar Says – AJMC.com Managed Markets Network

Whether a patient is refractory to initial treatment dictates the future course of care.

The multitude of options to treat multiple myeloma (MM) doesnt change an important fact: the duration of a patients response to the first treatment will define the disease biology going forwardhow well the disease is managed in the early going matters, according to Shaji K. Kumar, MD, of the Mayo Clinic Cancer Center, who gave an update on MM management during Fridays National Comprehensive Cancer Center (NCCN) annual meeting.

Fortunately, results from the GRIFFIN trial are showing whats possible. Results presented at the December 2021 meeting of the American Society of Hematology showed positive outcomes after 24 months for newly patients who took quadruplet therapy after an autologous stem cell transplant (ASCT). The combination, which added daratumumab to the usual combination of lenalidomide, bortezomib, and dexamethasone (RVd) had better stringent complete responses (sCR, 66.0% vs 47.4%), along with higher minimal residual disease (MRD) negativity rates.

This clearly appears to be translating into an improvement in progression-free survival (PFS), Kumar said. Its too soon to start treating every patient with newly diagnosed, transplant-eligible MM this way, but given the high rates of MRD negativity that we see with Dara-RVd, this regimen is definitely one to consider for patients with high risk multiple myeloma.

What about patients who are not transplant eligible, or need to wait? The IFM 2009 study compared giving ASCT right away with additional doses of therapy. Although ASCT clearly offered better PFS, there was not improvement in overall survival (OS), Kumar noted. Thus, it is very reasonable to delay stem cell transplant to the time of first relapse.

For these patients, daratumumab with lenalidomide and dexamethasone should be considered the standard, based on the MAIA study, he said.

Ongoing treatment. After initial treatment and lenalidomide maintenance, treatment choices are driven by whether patients are refractory to lenalidomide, Kumar explained. He shared a slide with multiple doublet and triplet options, and explained that triplets are now preferred, with one drug being dexamethasone. Prior treatments, age, comorbidities, frailty, and any lingering toxicity should be considered.

In general, the approachespecially in the earlier lines of therapyis to treat patients to maximum response, and then maintain them on at least one of the drugs from the combination until disease progression, Kumar said. This is easier in the early lines of therapy, he acknowledged. Whether a patient is refractory on their initial therapy is a key differentiator is a key differentiator that guides treatment going forward.

Selinexor, an XP01 inhibitor, was approved in December 2020 for use with bortezomib and dexamethasone in patients who have had at least one prior therapy. Belantamab mafodotin, is an antibody drug conjugate that targets B-cell maturation antigen (BCMA), and could be used to treating patients that have been refractory to other major drug classes, including protease inhibitors. Long-term data from the DREAMM-2 study found that median duration of response, OS, and PFS were 11.0 month, 13.7 months, and 2.8 months.

A recent highlight is the FDA approval last month of a second chimeric antigen receptor (CAR) T-cell therapy for MM, ciltacabtagene autoleucel (cilta-cel) which also targets BCMA. In the CARTITUDE trial, results at 2 years showed median PFS and OS were not reached and sCR was 82.5%.

Kumar also reported on several clinical trials involving investigational therapies and new uses of existing therapies, including:

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Early Treatment Matters More Than Ever in Multiple Myeloma, Kumar Says - AJMC.com Managed Markets Network

Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting – Yahoo…

Jasper Therapeutics

REDWOOD CITY, Calif., March 31, 2022 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (NASDAQ: JSPR), a biotechnology company focused on hematopoietic cell transplant therapies, today announced that updated data from the Companys ongoing study of JSP191 as single agent conditioning prior to allogeneic hematopoietic stem cell (HSC) re-transplant in patients with severe combined immunodeficiency (SCID) has been accepted for presentation as a late-breaking poster at the 2022 Clinical Immunology Society (CIS) Annual Meeting, to be held in Charlotte, North Carolina from March 31 to April 3, 2022.

Title: Update: Single-Agent Conditioning with Anti-CD117 Antibody JSP191 Shows Donor Engraftment, Nave Lymphocyte Production, and Clinical Benefit in Patients with Severe Combined Immunodeficiency (SCID) Date and Time: Friday, April 1, 2022, 1:00-2:00 p.m. ET

This updated data indicates that JSP191 at 0.6mg/kg can deplete blood stem cells, leading to long-term donor cell engraftment, immune reconstitution which positively affects the clinical status of SCID patients who suffer from poor T cell and negligible B cell immunity because they failed their first transplant, said Wendy Pang, MD, Ph.D., Senior Vice President of Research and Translational Medicine of Jasper Therapeutics. This population of SCID patients is largely without treatment options and rely on supportive therapies like life long IVIG to provide some level of immune protection. JSP191 based conditioning may provide these patients with the best chance of a safe and successful transplant and reconstituted immune system.

CIS attendees are the primary caregivers for the immune deficient patient population, we are pleased to be able to present this data at the 2022 CIS annual meeting, Ronald Martell, CEO of Jasper. We believe that with our successful clinical efforts, we are one step closer, and uniquely positioned to deliver a targeted non-genotoxic conditioning agent to patients with SCID.

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About JSP191

JSP191 is a humanized monoclonal antibody in clinical development as a conditioning agent that blocks stem cell factor receptor signaling leading to clearance of hematopoietic stem cells from bone marrow, creating an empty space for donor or genetically modified transplanted stem cells to engraft. To date, JSP191 has been evaluated in more than 100 healthy volunteers and patients. Three clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML), severe combined immunodeficiency (SCID) and Fanconi anemia are currently enrolling. The Company plans a new study of JSP191 as a second-line therapeutic in lower risk MDS patients in 2022 as well as to a pivotal study in MDS/AML transplant in early 2023. Enrollment in additional studies are planned in patients with sickle cell disease, chronic granulomatous disease and GATA2 MDS who are undergoing hematopoietic cell transplantation.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The company is advancing two potentially groundbreaking programs. JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing hematopoietic cell transplantation. It is designed to enable safer and more effective curative allogeneic hematopoietic cell transplants and gene therapies. In parallel, Jasper Therapeutics is advancing its preclinical mRNA engineered hematopoietic stem cell (eHSC) platform, which is designed to overcome key limitations of allogeneic and autologous gene-edited stem cell grafts. Both innovative programs have the potential to transform the field and expand hematopoietic stem cell therapy cures to a greater number of patients with life-threatening cancers, genetic diseases and autoimmune diseases than is possible today. For more information, please visit us at jaspertherapeutics.com.

Forward-Looking Statements

Certain statements included in this press release that are not historical facts are forward-looking statements for purposes of the safe harbor provisions under the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements are sometimes accompanied by words such as believe, may, will, estimate, continue, anticipate, intend, expect, should, would, plan, predict, potential, seem, seek, future, outlook and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These forward-looking statements include, but are not limited to, statements regarding the potential long-term benefits of hematopoietic stem cells (HSC) engraftment following targeted single-agent JSP191 conditioning in the treatment of severe combined immunodeficiency (SCID) and Jaspers ability to potentially deliver a targeted non-genotoxic conditioning agent to patients with SCID. These statements are based on various assumptions, whether or not identified in this press release, and on the current expectations of Jasper and are not predictions of actual performance. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as, and must not be relied on by an investor as, a guarantee, an assurance, a prediction or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Jasper. These forward-looking statements are subject to a number of risks and uncertainties, including general economic, political and business conditions; the risk that the potential product candidates that Jasper develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all; risks relating to uncertainty regarding the regulatory pathway for Jaspers product candidates; the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release; the risk that Jasper will be unable to successfully market or gain market acceptance of its product candidates; the risk that Jaspers product candidates may not be beneficial to patients or successfully commercialized; patients willingness to try new therapies and the willingness of physicians to prescribe these therapies; the effects of competition on Jaspers business; the risk that third parties on which Jasper depends for laboratory, clinical development, manufacturing and other critical services will fail to perform satisfactorily; the risk that Jaspers business, operations, clinical development plans and timelines, and supply chain could be adversely affected by the effects of health epidemics, including the ongoing COVID-19 pandemic; the risk that Jasper will be unable to obtain and maintain sufficient intellectual property protection for its investigational products or will infringe the intellectual property protection of others; and other risks and uncertainties indicated from time to time in Jaspers filings with the SEC. If any of these risks materialize or Jaspers assumptions prove incorrect, actual results could differ materially from the results implied by these forward-looking statements. While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Jaspers assessments of any date subsequent to the date of this press release. Accordingly, undue reliance should not be placed upon the forward-looking statements.

Contacts: John Mullaly (investors) LifeSci Advisors 617-429-3548 jmullaly@lifesciadvisors.com

Jeet Mahal (investors) Jasper Therapeutics 650-549-1403 jmahal@jaspertherapeutics.com

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Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting - Yahoo...

Rheumatoid Arthritis Stem Cell Therapy Market Assessment, With Major Top Companies Analysis, Geographic Analysis, Growing Opportunities Data By…

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By Product Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant and Adipose Tissue Stem Cells)

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Sana On Track to Remove Barriers for Cell & Gene Therapy – BioSpace

A Sana scientist works in the lab/Photo courtesy of Sana Biotechnology

One of the most important transformations of 21st-centurymedicine will be the ability to reengineer cells as medicines. Before cell and gene therapy can truly become mainstream medicine, the challenges of allogeneic rejection and targeted delivery must be solved. Sana Biotechnology is on the path to do that, with notable strides that may, just possibly, change medicine as we know it.

Our aspirations are to be able to repair or control genes and gene expression of every cell type in the body thats broken, and to transplant or replace whats missing or too far damaged, Steve Harr, M.D., Sanas president and CEO, told BioSpace.

The scope of that mission is huge. The breadth of our aspirations set Sana apart from most biotech companies, he said. And, Sana is approaching the challenge like any other great endeavor: one step at a time. In this case, that means with ex vivo and in vivo cell engineering platforms.

Sanas ex vivo hypoimmune platform (HIP) is designed to prevent allogeneic rejection, in which the immune system recognizes modified cells as foreign and attacks them. This is perhaps the greatest issue that has held cell therapy back from reaching its potential for patients, Harr said.

The hypoimmune platform appears to solve that hurdle, he said. We have shown in preclinical models that we have the potential to prevent allogeneic rejection.

As Sonja Schrepfer,M.D., Ph.D., head of Hypoimmune Platform, elaborated, The human leukocyte antigens (HLA) on a cells surface are like fingerprints for proteins on the cell. They tell the immune system what is inside your cell. We recognized HLA as a major hurdle (in overcoming rejection) and so prevented expression of the HLA. Now the cells fingerprint is missing.

Of course, viruses figured out long ago that removing HLA can hide cells, so the immune system evolved to be smarter, she continued. NK cells and macrophages recognize cells without HLA (missing self) and attack them. To protect those cells from this attack, we overexpress CD47, which basically is a dont attack me molecule. It protects these cells from being recognized by NK cells and macrophages, allowing them to survive.

These cellular modifications can be performed on pluripotent stem cells, which can then be differentiated into essentially any hypoimmune cell, and also on allogeneic donor cells. We intend to derive hypoimmune pancreatic islet cells to treat type 1 diabetes, for example. Our goal is to provide off-the-shelf cells to anyone, anywhere, at any time that can function without requiring the patient to take immunosuppressive medications, Schrepfer said. Sana also is working to develop T cells, glial progenitor cells and cardiomyocytes as allogeneic therapies.

In preclinical models, We have seen that without immunosuppression, the HIP-edited induced pluripotent stem cells (iPSCs) survived for many weeks without activating the immune system or being rejected, Schrepfer said. That was very exciting! The company is planning to file its first IND to test this system in patients as early as this year.

Targeted gene delivery is the second big challenge Sana is addressing. With antiviral vectors (AAVs), you cant control where the vectors go and, because the cellular DNA is not changed, they have less utility in dividing cells. Lipid nanoparticles (LNPs), another popular delivery vehicle, at this time mainly go to the liver and deliver only RNA and proteins, not DNA, Harr said.

Sanas fusogen platform has the potential to overcome those limitations with in vivo cell and gene modification. You can make just about any modification to genes and gene expression in a petri dish, Harr pointed out, but it is very difficult to deliver the gene modification payload in vivo. Fusogens offer a unique delivery capability for gene editing and gene modification machinery. We have a number of programs in preclinical development, including four that modify T cells for multiple oncology indications, one that modifies hepatocytes to address liver-related disorders and one that modifies hematopoietic stem cells to address hemoglobinopathies.

Sanas science has the potential to disrupt the cell and gene therapy sector of the industry, but its effects on the practice of medicine are still at least several years away. As Harr said, Our key competitive advantage, as a smaller company, needs to be decision-making, which is possible because of greater focus and having the right people.

Harr defines the right people as program heads who are global leaders in their fields. Thats really important, Harr explained, because this isnt as easy as normal drug development (which, itself, isnt easy). For example, Richard Mulligan, Ph.D., head of SanaX, is instrumental in Sanas work to deliver genomic material. SanaX is a distinct research arm within Sana focused around long-term, disruptive improvements in cell and gene therapy. Mulligan was among the first to discover how to insert genes into cells in the 1980s. Terry Fry, M.D., SVP and head of T Cell Therapeutics, is a renowned expert in chimeric antigen receptor T cell (CAR T) therapies and was critical in the development of a number of CAR T cell therapies to date, including Yescarta. Chuck Murry, M.D., Ph.D., SVP and head of cardiometabolic cell therapy, pioneered the use of human pluripotent stem cells for heart regeneration.

The executive leaders also bring a wealth of experience. Some, including Harr, joined Sana after senior positions at Juno Therapeutics. Others honed their skills at Genentech, Amazon, Amgen, Sangamo and the U.S. Food and Drug Administration, amongst others.

Sanas staff of approximately 400 is divided among its facilities in South San Francisco, Seattle and Cambridge, Massachusetts.

The company is expanding, so there are opportunities throughout Sana both geographically and in terms of business and technical specialties. Harr said one hiring priority is in the area of process and analytical development for manufacturing. Another area includes hiring experts in analytic genomics and computational biology to enable in-depth interrogation of the genome, both when youre inserting things into the cells and as stem cells divide and differentiate, he added.

Our goal is to make important medicines that matter for patients, and these roles are essential in doing that. That means the Sana team must also be flexible and resilient.

The world is constantly changing, and we live on the tip of the innovation spear, Harr said. We need people who are intellectually curious and who have grit. Being a pioneer is hard. We will have great days, and we also will have setbacks. We need people who are resilient and who can collaborate effectively on teams. The things we are doing are complex enough that none of us have all the answers. It always takes some of us.

Harr said Sana is committed to ensuring we do all we can to increase individuals chances to succeed and rewarding them fairly. Biotech tries to help people from all over the world. To do that, we need an environment where people can thrive.

Harr continued, Inclusion is the soil from which all great cultures grow. To truly feel included, you need diversity. It helps to know that there are people just like me whove succeeded here. Succeeding is easier when you have that example.

With that philosophy, Sanas Inclusion, Diversity and Equity program is robust. The measure of a great culture is whether it helps you succeed or holds you back. Our measure is to help you be your best self, your true self and to accept people for who they are. This is an exciting time to be at Sana, Harr said.

Sana is transforming from a research company to a research and development company. The movement into development is an important next step, Harr said. Its the chance to prove things work.

Sana is working towards developing important cell and gene therapies for patients. As Harr said, This is a dynamic company with complex science. We have chosen to be audacious.

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Are COVID-19-Linked Arrhythmias Caused by Viral Damage to the Heart’s Pacemaker Cells? – Weill Cornell Medicine Newsroom

The SARS-CoV-2 virus can infect specialized pacemaker cells that maintain the hearts rhythmic beat, setting off a self-destruction process within the cells, according to a preclinical study co-led by researchers at Weill Cornell Medicine, NewYork-Presbyterian and NYU Grossman School of Medicine. The findings offer a possible explanation for the heart arrhythmias that are commonly observed in patients with SARS-CoV-2 infection.

In the study, reported March 8 in Circulation Research, the researchers used an animal model as well as human stem cell-derived pacemaker cells to show that SARS-CoV-2 can readily infect pacemaker cells and trigger a process called ferroptosis, in which the cells self-destruct but also produce reactive oxygen molecules that can impact nearby cells.

This is a surprising and apparently unique vulnerability of these cellswe looked at a variety of other human cell types that can be infected by SARS-CoV-2, including even heart muscle cells, but found signs of ferroptosis only in the pacemaker cells, said study co-senior author Dr. Shuibing Chen, the Kilts Family Professor of Surgery and a professor of chemical biology in surgery and of chemical biology in biochemistry at Weill Cornell Medicine.

Arrhythmias including too-quick (tachycardia) and too-slow (bradycardia) heart rhythms have been noted among many COVID-19 patients, and multiple studies have linked these abnormal rhythms to worse COVID-19 outcomes. How SARS-CoV-2 infection could cause such arrhythmias has been unclear, though.

In the new study, the researchers, including co-senior author Dr. Benjamin tenOever of NYU Grossman School of Medicine, examined golden hamstersone of the only lab animals that reliably develops COVID-19-like signs from SARS-CoV-2 infectionand found evidence that following nasal exposure the virus can infect the cells of the natural cardiac pacemaker unit, known as the sinoatrial node.

To study SARS-CoV-2s effects on pacemaker cells in more detail and with human cells, the researchers used advanced stem cell techniques to induce human embryonic stem cells to mature into cells closely resembling sinoatrial node cells. They showed that these induced human pacemaker cells express the receptor ACE2 and other factors SARS-CoV-2 uses to get into cells and are readily infected by SARS-CoV-2. The researchers also observed large increases in inflammatory immune gene activity in the infected cells.

The teams most surprising finding, however, was that the pacemaker cells, in response to the stress of infection, showed clear signs of a cellular self-destruct process called ferroptosis, which involves accumulation of iron and the runaway production of cell-destroying reactive oxygen molecules. The scientists were able to reverse these signs in the cells using compounds that are known to bind iron and inhibit ferroptosis.

This finding suggests that some of the cardiac arrhythmias detected in COVID-19 patients could be caused by ferroptosis damage to the sinoatrial node, said co-senior author Dr. Robert Schwartz, an associate professor of medicine in the Division of Gastroenterology and Hepatology at Weill Cornell Medicine and a hepatologist at NewYork-Presbyterian/Weill Cornell Medical Center.

Although in principle COVID-19 patients could be treated with ferroptosis inhibitors specifically to protect sinoatrial node cells, antiviral drugs that block the effects of SARS-CoV-2 infection in all cell types would be preferable, the researchers said.

The researchers plan to continue to use their cell and animal models to investigate sinoatrial node damage in COVID-19and beyond.

There are other human sinoatrial arrhythmia syndromes we could model with our platform, said co-senior author Dr. Todd Evans, the Peter I. Pressman M.D. Professor of Surgery and associate dean for research at Weill Cornell Medicine. And, although physicians currently can use an artificial electronic pacemaker to replace the function of a damaged sinoatrial node, theres the potential here to use sinoatrial cells such as weve developed as an alternative, cell-based pacemaker therapy.

Many Weill Cornell Medicine physicians and scientists maintain relationships and collaborate with external organizations to foster scientific innovation and provide expert guidance. The institution makes these disclosurespublic to ensure transparency. For this information, see profiles for Dr. Todd Evans, and Dr. Robert Schwartz.

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Are COVID-19-Linked Arrhythmias Caused by Viral Damage to the Heart's Pacemaker Cells? - Weill Cornell Medicine Newsroom