In a typical stem cell transplant for cancer very high doses of chemo are used, sometimes along with radiation therapy, to try to kill all the cancer cells. This treatment also kills the stem cells in the bone marrow. Soon after treatment, stem cells are given to replace those that were destroyed. These stem cells are given into a vein, much like a blood transfusion. Over time they settle in the bone marrow and begin to grow and make healthy blood cells. This process is called engraftment.

There are 2 main types of transplants. They are named based on who gives the stem cells.

In this type of transplant, your own stem cells are removed, or harvested, from your blood before you get treatment that destroys them. Your stem cells are removed from either your bone marrow or your blood, and then frozen. (You can learn more about this process at Whats It Like to Donate Stem Cells?) After you get high doses of chemo and/or radiation, the stem cells are thawed and given back to you.

One advantage of autologous stem cell transplant is that youre getting your own cells back. You dont have to worry about the new stem cells (called the engrafted cells or the graft) attacking your body (graft-versus-host disease) or about getting a new infection from another person. But there can still be graft failure, which means the cells dont go into the bone marrow and make blood cells like they should. Also, autologous transplants cant produce the graft-versus-cancer effect.

This kind of transplant is mainly used to treat certain leukemias, lymphomas, and multiple myeloma. Its sometimes used for other cancers, like testicular cancer and neuroblastoma, and certain cancers in children. Doctors are looking at how autologous transplants might be used to treat other diseases, too, like systemic sclerosis, multiple sclerosis (MS), Crohn's disease, and systemic lupus erythematosis (lupus).

A possible disadvantage of an autologous transplant is that cancer cells may be collected along with the stem cells and then later put back into your body. Another disadvantage is that your immune system is the same as it was before your transplant. This means the cancer cells were able to escape attack from your immune system before, and may be able to do so again.

To help prevent this, some centers treat the stem cells before theyre given back to the patient to try to kill any remaining cancer cells. This may be called purging. It isnt clear that this really helps, as it has not yet been proven to reduce the risk of cancer coming back. A possible downside of purging is that some normal stem cells can be lost during this process. This may cause your body to take longer to start making normal blood cells, and you might have very low and unsafe levels of white blood cells or platelets for a longer time. This could increase the risk of infections or bleeding problems.

Another treatment to help kill cancer cells that might be in the returned stem cells involves giving anti-cancer drugs after transplant. The stem cells are not treated. After transplant, the patient gets anti-cancer drugs to get rid of any cancer cells that may be in the body. This is called in vivo purging. For instance, rituximab (Rituxan), a monoclonal antibody drug, may be used this way in certain lymphomas and leukemias; lenalidomide (Revlimid) may be used for multiple myeloma. The need to remove cancer cells from transplanted stem cells or transplant patients and the best way to do it is being researched.

Doing 2 autologous transplants in a row is known as a tandem transplant or a double autologous transplant. In this type of transplant, the patient gets 2 courses of high-dose chemo, each followed by a transplant of their own stem cells. All of the stem cells needed are collected before the first high-dose chemo treatment, and half of them are used for each transplant. Usually, the 2 courses of chemo are given within 6 months. The second one is given after the patient recovers from the first one.

Tandem transplants are most often used to treat multiple myeloma and advanced testicular cancer. But doctors dont always agree that these are really better than a single transplant for certain cancers. Because this involves 2 transplants, the risk of serious outcomes is higher than for a single transplant. Tandem transplants are still being studied to find out when they might be best used.

Sometimes an autologous transplant followed by an allogeneic transplant might also be called a tandem transplant. (See Mini-transplants below.)

Allogeneic stem cell transplants use cells from a donor. In the most common type of allogeneic transplant, the stem cells come from a donor whose tissue type closely matches the patients. (This is discussed later in Matching patients and donors.) The best donor is a close family member, usually a brother or sister. If you dont have a good match in your family, a donor might be found in the general public through a national registry. This is sometimes called a MUD (matched unrelated donor) transplant. Transplants with a MUD are usually riskier than those with a relative who is a good match.

Blood taken from the placenta and umbilical cord of newborns is a newer source of stem cells for allogeneic transplant. Called cord blood, this small volume of blood has a high number of stem cells that tend to multiply quickly. But there are often not enough stem cells in a unit of cord blood for large adults, so most cord blood transplants done so far have been in children and smaller adults. Researchers are now looking for ways to use cord blood for transplants in larger adults. One approach is to find ways to increase the numbers of these cells in the lab before the transplant. Another approach is the use of the cord blood from 2 infants for one adult transplant, called a dual-cord-blood transplant. A third way cord blood is being used is in a mini-transplant (see below). Other strategies to better use cord blood transplants are being actively studied.

Pros of allogeneic stem cell transplant: The donor stem cells make their own immune cells, which could help kill any cancer cells that remain after high-dose treatment. This is called the graft-versus-cancer effect. Other advantages are that the donor can often be asked to donate more stem cells or even white blood cells if needed, and stem cells from healthy donors are free of cancer cells.

Cons to allogeneic stem cell transplants: The transplant, or graft, might not take that is, the transplanted donor stem cells could die or be destroyed by the patients body before settling in the bone marrow. Another risk is that the immune cells from the donor may not just attack the cancer cells they could attack healthy cells in the patients body. This is called graft-versus-host disease. There is also a very small risk of certain infections from the donor cells, even though donors are tested before they donate. A higher risk comes from infections you had previously, and which your immune system has had under control. These infections may surface after allogeneic transplant because your immune system is held in check (suppressed) by medicines called immunosuppressive drugs. Such infections can cause serious problems and even death.

Allogeneic transplant is most often used to treat certain types of leukemia, lymphomas, multiple myeloma, myelodysplastic syndrome, and other bone marrow disorders such as aplastic anemia.

For some people, age or certain health conditions make it more risky to wipe out all of their bone marrow before a transplant. For those people, doctors can use a type of allogeneic transplant thats sometimes called a mini-transplant. Your doctor might refer to it as a non-myeloablative transplant or mention reduced-intensity conditioning (RIC). Patients getting a mini transplant get less chemo and/or radiation than if they were getting a standard transplant. The goal is to kill some of the cancer cells (which will also kill some of the bone marrow), and suppress the immune system just enough to allow donor stem cells to settle in the bone marrow.

Unlike the standard allogeneic transplant, cells from both the donor and the patient exist together in the patients body for some time after a mini-transplant. But slowly, over the course of months, the donor cells take over the bone marrow and replace the patients own bone marrow cells. These new cells can then develop an immune response to the cancer and help kill off the patients cancer cells the graft-versus-cancer effect.

One advantage of a mini-transplant is that it uses lower doses of chemo and/or radiation. And because the stem cells arent all killed, blood cell counts dont drop as low while waiting for the new stem cells to start making normal blood cells. This makes it especially useful for older patients and those with other health problems. Rarely, it may be used in patients who have already had a transplant.

Mini-transplants treat some diseases better than others. They may not work well for patients with a lot of cancer in their body or people with fast-growing cancers. Also, although side effects from chemo and radiation may be less than those from a standard allogeneic transplant, the risk of graft-versus-host disease is the same.

This procedure has only been used since the late 1990s and long-term patient outcomes are not yet clear. There are lower risks of some complications, but the cancer may be more likely to come back. Ways to improve outcomes are still being studied.

Studies have looked at using an allogeneic mini-transplant after an autologous transplant. This is another type of tandem transplant being tested in certain types of cancer, such as multiple myeloma and some types of lymphoma. The autologous transplant can help decrease the amount of cancer present so that the lower doses of chemo given before the mini-transplant can work better. And the recipient still gets the benefit of the graft-versus-cancer effect of the allogeneic transplant.

This is a special kind of allogeneic transplant that can only be used when the patient has an identical sibling (twin or triplet) someone who has the exact same tissue type. An advantage of syngeneic stem cell transplant is that graft-versus-host disease will not be a problem. Also, there are no cancer cells in the transplanted stem cells, as there might be in an autologous transplant.

A disadvantage is that because the new immune system is so much like the recipients immune system, theres no graft-versus-cancer effect. Every effort must be made to destroy all the cancer cells before the transplant is done to help keep the cancer from coming back.

Some centers are doing half-match (haploidentical) transplants for people who dont have closely matching family members. This technique is most often used in children, usually with a parent as the donor, though a child can also donate to a parent. Half of the HLA factors will match perfectly, and the other half typically dont match at all, so the procedure requires a special way to get rid of a certain white blood cells that can cause graft-versus-host disease. Its still rarely done, but its being studied in a few centers in the US. Researchers are continuing to learn new ways to make haploidentical transplants more successful.

Depending on the type of transplant thats done, there are 3 possible sources of stem cells to use for transplants:

Bone marrow is the spongy liquid tissue in the center of some bones. It has a rich supply of stem cells, and its main job is to make blood cells that circulate in your body. The bones of the pelvis (hip) have the most marrow and contain large numbers of stem cells. For this reason, cells from the pelvic bone are used most often for a bone marrow transplant. Enough marrow must be removed to collect a large number of healthy stem cells.

The bone marrow is harvested (removed) while the donor is under general anesthesia (drugs are used to put the patient into a deep sleep so they dont feel pain). A large needle is put through the skin on the lower back and into the back of the hip bone. The thick liquid marrow is pulled out through the needle. This is repeated until enough marrow has been taken out. (For more on this, see Whats It Like to Donate Stem Cells?)

The harvested marrow is filtered, stored in a special solution in bags, and then frozen. When the marrow is to be used, its thawed and then put into the patients blood through a vein, just like a blood transfusion. The stem cells travel to the bone marrow, where they engraft or take and start to make blood cells. Signs of the new blood cells usually can be measured in the patients blood tests in about 2 to 4 weeks.

Normally, not many stem cells are found in the blood. But giving shots of hormone-like substances called growth factors to stem cell donors a few days before the harvest causes their stem cells to grow faster and move from the bone marrow into the blood.

For a peripheral blood stem cell transplant, the stem cells are taken from blood. A special thin flexible tube (called a catheter) is put into a large vein in the donor and attached to tubing that carries the blood to a special machine. The machine separates the stem cells from the rest of the blood, which is returned to the donor during the same procedure. This takes several hours, and may need to be repeated for a few days to get enough stem cells. The stem cells are filtered, stored in bags, and frozen until the patient is ready for them. (For more on this, see Whats It Like to Donate Stem Cells?)

When theyre given to the patient, the stem cells are put into a vein, much like a blood transfusion. The stem cells travel to the bone marrow, engraft, and then start making new, normal blood cells. The new cells are usually found in the patients blood in about 10 to 20 days.

A large number of stem cells are normally found in the blood of newborn babies. After birth, the blood thats left behind in the placenta and umbilical cord (known as cord blood) can be taken and stored for later use in a stem cell transplant. The cord blood is frozen until needed. A cord blood transplant uses blood that normally is thrown out after a baby is born. More information on donating cord blood can be found in Whats It Like to Donate Stem Cells?

A possible drawback of cord blood is the smaller number of stem cells in it. But this is partly balanced by the fact that each cord blood stem cell can form more blood cells than a stem cell from adult bone marrow. Still, cord blood transplants can take longer to take hold and start working. Cord blood is given into the patients blood just like a blood transfusion.

It is very important that the donor and recipient are a close tissue match to avoid graft rejection. Graft rejection happens when the recipients immune system recognizes the donor cells as foreign and tries to destroy them as it would a bacteria or virus. Graft rejection can lead to graft failure, but its rare when the donor and recipient are well matched.

A more common problem is that when the donor stem cells make their own immune cells, the new cells may see the patients cells as foreign and attack their new home. This is called graft-versus-host disease. (See Stem Cell Transplant Side Effects for more on this). The new, grafted stem cells attack the body of the person who got the transplant. This is another reason its so important to find the closest match possible.

Many factors play a role in how the immune system knows the difference between self and non-self, but the most important for transplants is the human leukocyte antigen (HLA) system. Human leukocyte antigens are proteins found on the surface of most cells. They make up a persons tissue type, which is different from a persons blood type.

Each person has a number of pairs of HLA antigens. We inherit them from both of our parents and, in turn, and pass them on to our children. Doctors try to match these antigens when finding a donor for a person getting a stem cell transplant.

How well the donors and recipients HLA tissue types match plays a large part in whether the transplant will work. A match is best when all 6 of the known major HLA antigens are the same a 6 out of 6 match. People with these matches have a lower chance of graft-versus-host disease, graft rejection, having a weak immune system, and getting serious infections. For bone marrow and peripheral blood stem cell transplants, sometimes a donor with a single mismatched antigen is used a 5 out of 6 match. For cord blood transplants a perfect HLA match doesnt seem to be as important, and even a sample with a couple of mismatched antigens may be OK.

Doctors keep learning more about better ways to match donors. Today, fewer tests may be needed for siblings, since their cells vary less than an unrelated donor. But to reduce the risks of mismatched types between unrelated donors, more than the basic 6 HLA antigens may be tested. For example, sometimes doctors to try and get a 10 out of 10 match. Certain transplant centers now require high-resolution matching, which looks more deeply into tissue types and allow more specific HLA matching.

There are thousands of different combinations of possible HLA tissue types. This can make it hard to find an exact match. HLA antigens are inherited from both parents. If possible, the search for a donor usually starts with the patients brothers and sisters (siblings), who have the same parents as the patient. The chance that any one sibling would be a perfect match (that is, that you both received the same set of HLA antigens from each of your parents) is 1 out of 4.

If a sibling is not a good match, the search could then move on to relatives who are less likely to be a good match parents, half siblings, and extended family, such as aunts, uncles, or cousins. (Spouses are no more likely to be good matches than other people who are not related.) If no relatives are found to be a close match, the transplant team will widen the search to the general public.

As unlikely as it seems, its possible to find a good match with a stranger. To help with this process, the team will use transplant registries, like those listed here. Registries serve as matchmakers between patients and volunteer donors. They can search for and access millions of possible donors and hundreds of thousands of cord blood units.

Be the Match (formerly the National Marrow Donor Program)Toll-free number: 1-800-MARROW-2 (1-800-627-7692)Website: http://www.bethematch.org

Blood & Marrow Transplant Information NetworkToll-free number: 1-888-597-7674Website: http://www.bmtinfonet.org

Depending on a persons tissue typing, several other international registries also are available. Sometimes the best matches are found in people with a similar racial or ethnic background. When compared to other ethnic groups, white people have a better chance of finding a perfect match for stem cell transplant among unrelated donors. This is because ethnic groups have differing HLA types, and in the past there was less diversity in donor registries. However, the chances of finding an unrelated donor match improve each year, as more volunteers become aware of registries and sign up for them.

Finding an unrelated donor can take months, though cord blood may be a little faster. A single match can require going through millions of records. Also, now that transplant centers are more often using high-resolution tests, matching is becoming more complex. Perfect 10 out of 10 matches at that level are much harder to find. But transplant teams are also getting better at figuring out what kinds of mismatches can be tolerated in which particular situations that is, which mismatched antigens are less likely to affect transplant success and survival.

Keep in mind that there are stages to this process there may be several matches that look promising but dont work out as hoped. The team and registry will keep looking for the best possible match for you. If your team finds an adult donor through a transplant registry, the registry will contact the donor to set up the final testing and donation. If your team finds matching cord blood, the registry will have the cord blood sent to your transplant center.

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Types of Stem Cell Transplants for Cancer Treatment ...

What is Regenerative Medicine?

Regenerative Medicine is a new field of medicine with one goal in mind: to heal and restore normal function of damaged tissues and organs. Just like the human body itself, regenerative medicine utilizes stem cells to replace damaged cells and tissues. Stem Cells are, therefore, considered to be one of the most powerful tools in treating diseases. They go beyond conventional methods to repair and regenerate disease-related damage, by returning tissues and organs to a healthier state.

Stem cells exist in many different types as they have been identified in various tissues and organs. Each type of stem cell is classified by: their origin in the body, and their potential (potency) to differentiate (transform) into other cell types. This potential varies among stem cell types.

Some stem cells are capable of differentiating themselves into any cell type of body (pluripotent). Others, on the other hand, are able to transform into many cell types (multipotent), while some are only able to differentiate themselves into few (oligopotent) or one cell type (unipotent).

Having this in mind, it is important to note that not all stem cell types are suitable for treating patients. For example, the use of Embryonic Stem Cells (ESCs) for treating patients is restricted due to ethical issues, and their potential to grow into tumors.

One of the safest and most effective forms of stem cell treatments, which we employ at ANOVA, make use of autologous stem cells, i.e. stem cells derived from the patient themselves. By using autologous stem cells for the treatment of patients, there is very minimal to no risk of tumor formation, the transmission of infectious diseases or adverse immune reactions.

Bone Marrow Concentrate (BMC) and Mesenchymal Stem Cells (MSCs) are the most abundant form of autologous adult stem cells that are well suited for clinical use. They are relatively easily harvested from the bone marrow (BMCs) or from the subcutaneous ("under the skin") fat (MSCs).

Currently, most therapies at ANOVA are based on cell-free secretion of MSCs. We are also offering combination therapies with Platelet Rich Plasma (PRP), a medium that is rich in growth factors and other cytokines (molecules from the immune system) that stimulates healing, as well as BMC in the near future. Both can additionally be supplemented with our stem cell treatments as they seamlessly synergize together.

ANOVA offers individualized stem cell therapies that are best suited for the particular condition of the patient, and for the patient only. The application of these therapies depends entirely on the patients medical condition.

Overview

Numerous types of stem cell therapies are available at ANOVA. Stem cell research brought insights that allowed for technological advancements in therapies and expanding the knowledge of the underlying mechanisms of stem cells. This has allowed for more effective therapies to be developed.

BMC

Bone Marrow Concentrate (BMC) is one of the most commonly applied source of stem cells. Despite the fact that the actual number of stem cells in BMC is biologically limited, several other (regenerative) factors in BMC have been shown to deliver promising results in the treatment of numerous diseases.

Stem Cell Secretome

Stem Cell Therapies 2.0: The ANOVA Stem Cell Secretome Therapy is the next generation of stem cell-based therapies. It was designed to harness and mass produce the healing essences of stem cells (paracrine and regenerative factors, extracellular vesicles, exosomes) in a uniquely designed laboratory process.

MSCs

Fat (adipose) derived Mesenchymal Stem Cells (MSC | adMSCs) are a commonly used source of stem cells, because of their availability and robustness. They communicate to other cells with a broad spectrum of secreted paracrine and regenerative factors. They are our favorite source of stem cells for the production of the secretome.

Platelet Rich Plasma (PRP)

Platelet Rich Plasma (PRP) is a blood-derived, cellular product with concentrated supply of regenerative growth factors and cytokines. Its efficacy has been proven in some orthopedic conditions. When used in combination with our stem cell therapies, its efficacy is synergistically enhanced.

Stem Cells, as explained previously, have the power to differentiate into any cell type, all the way from: bone cells to brain cells, heart cells, nerve cells, kidney cells, etc. This is what defines Stem Cells. However, the potency to differentiate into any body cell type is not what defines their healing powers, as not all stem cell types are able to transform into any cell type. Only a selected few, such as Bone Marrow (BMC) and Mesenchymal Stem Cells (MSCs), have been identified to differentiate into most cells type and have been successfully used in medicine to treat diseases. They have been shown to hold several major therapeutic effects, such as:

After initial damage to tissues or organs, such as mechanical forces in trauma or the lack of blood supply in strokes and heart attacks, further damage is caused by immune processes and inflammation. Subcritically injured cells, which are usually found in the vicinity of the damaged tissue or organ, primarily commit suicide instead of repairing themselves. This process further increases the damaged tissue volume. To repair this damage, which is (potentially) possible in most organs by the specific stem cells residing in them, is very slow or does not happen at all without external stimulation. In such cases, stem cells therapies have been demonstrated to be extremely effective in stimulating repair and limiting further damage.

Early stem cell research indicated that stem cells heal by replacing damaged cells in injured organs. Now, it has become evident that the major effects of tissue repair are not entirely based on direct stem cell implantation, but rather by the secretion of soluble (paracrine) factors from the stem cells themselves.

This discovery has prompted the Anova scientists to explore a completely new therapeutical approach in regenerative medicine, which has ultimately lead to the development of our novel, safe, cell-free treatment: The ANOVA's Stem Cell Secretome Therapy.

ANOVA's Stem Cell therapy method utilizes autologous stem cells, i.e. cells that are derived from the patient itself, to mass produce the secretory factors (that retain the regenerative powers of stem cells), At Anova, a minimally invasive mini-liposuction procedure is performed, which allows for the isolation of stem cells from the subcutaneous fat (adipose tissue) of the patient.

This method does not rely on direct stem cell transplantation to treat numerous diseases and support anti-aging. Latest scientific research has shown that stem cell-free therapies, such as at Anova's, offer the same efficacy as traditional stem cell transplantation therapies, with higher safety and minimized risk for the patient.

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Stem Cell Therapy | ANOVA IRM

Stem cells are special cells inside your body that can develop into many different types of tissue. When injected into your body, stem cells may have the ability to grow into healthy new cells. (You may have heard of stem cells being used alongside chemotherapy during cancer treatment).

In a similar way, doctors use stem cells to treat bone and joint problems. Doctors extract (remove) stem cells from your bone marrow. Your doctor will then re-inject these stem cells into areas of your body that are in pain (for example, your knee or shoulder).

The stem cells then may grow into healthy new tissue.

Just like PRP, stem cell therapy is a new, experimental treatment. This means doctors and researchers are still studying how it works and how effective it is at treating different types of pain or dysfunction.

Stem cell therapy may not work for everyone. You may also need multiple injections of stem cells before your pain gets better.

Stem cell therapy may be a good option if other treatments (like steroid injections) haven't worked.

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PRP & Stem Cell Therapy | University of Utah Health

BE YOUNGER & STRONGER FOR LONGER

Stem cell therapy (SCT) and regenerative medicine are growing topics of conversation in the search for longevity and preventative wellness measures, but along with this curiosity and promise, there are also questions and myths surrounding the topic. This class goal is to inform our community around stem cells, stem cell therapy, and address the common questions around this growing treatment.

So, how does stem cell therapy work and what does it do? Where do the cells come from? Are there are negative side effects or contraindications with Stem Cell Therapy?

Stem cells are our bodys natural internal repair system. In short, stem cells seek out damage in the body and work to regenerate damaged tissue.

In SCT practiced byAdvanced Integrative Medicine, they only use human umbilical cord stem cells that are collected from hospitals across the US. The mother signs a consent form, donating the umbilical cord blood to the hospital after the live birth of a healthy baby. Only cord blood cells from healthy mothers and babies are accepted.

In a very small percentage of patients, they occasionally see a minor reaction of flu-like symptoms. This does not last more than 24-28 hours maximum. This actually demonstrates the cells anti-inflammatory and immune-privilege potential.

Are you curious if you are a good candidate for stem cell therapy? If youre looking for a more natural solution towards regenerating the body, your best option is to consult with a Regenerative Medicine Expert, such asDr. Shawn Pallotti, to discuss your specific symptoms and candidacy.

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REGENERATIVE MEDICINE: Stem Cell Therapy 101 | Events Calendar - Richmond.com

LOUISVILLE, Colo. andNEW ORLEANS andSAN ANTONIO andCHICAGO, March 2, 2020 /PRNewswire/ -- GID BIOannounced today that The American Journal of Sports Medicinepublished resultsof its FDA-approved multi-site, randomized, placebo-controlled Phase IIb clinical trial measuring the safety and efficacy of its SVF-2 device and point-of-care (POC) therapy intended to treat pain and function associated with knee osteoarthritis.

The Phase IIb clinical study was approved by the FDA under an IDE and is the first regenerative cell therapy for osteoarthritis to meet study endpoints using autologous stromal cells from adipose tissue. The cellular therapy for osteoarthritis procedure showed no serious adverse events at two years and a significant reduction in pain at one year. A Phase III pivotal study begins soon at Tulane University School of Medicinewith additional trial sites participating nationwide.

"Publishing this data signifies real science and a breakthrough in regenerative medicine. We've completed a prior safety trial, an FDA-approved Phase IIb trial, and are now beginning a Phase III pivotal trial. Physicians will be able to use the SVF-2 technology to provide a cellular therapy option for patients," said principal investigator for the Phase III trial, Jaime R. Garza, MD, DDS, FACS, Professor of Orthopedic Surgery and Center for Stem Cell Research and Regenerative Medicineat Tulane University School of Medicine. "I am very proud to collaborate with my alma mater, Tulane University, and the School of Medicine's outstanding orthopedic department led by Dr. Felix Savoie, and its worldclass Center for Stem Cell Research and Regenerative Medicine directed by expert cell scientist Dr. Bruce Bunnell," said Dr. Garza.

Dr. Garza is a former NFL player and a Tulane University Athletic Hall of Fame inductee. He is also a clinical professor of plastic surgery and otolaryngology at the University of Texas Health Science Center.

Treatments by clinics using stem cells are under scrutiny by the FDA as its discretionary enforcement period expires in November of this year. The intent is that hundreds of stem cell clinicsnationwide become compliant with FDA regulations, leading to clinical data support of safety and efficacy.

"Our randomized, controlled clinical trial is the first cellular therapy study for osteoarthritis to meet study endpoints using autologous adipose stromal cells for a point-of-care therapy.Eighty-eight percent of subjects responded greater than placebo at one year and reported a median 87% improvement in pain, stiffness and function," said William W. Cimino, Ph.D., CEO of GID BIO. "We are able to isolate and concentrate the right types and numbers of cells to create an effective therapy. We are pleased to begin Phase III trials with Dr. Garza, and to be at the forefront for a cellular therapy option for osteoarthritic knees."

About GID SVF-2 and POC TherapyGID technology has reduced a Good Manufacturing Practice (GMP) cell-processing facility to a single-use disposable device for scalable point-of-care cell processing. The technology uniquely harvests and isolates stromal cells from a patient's own adipose tissue that is then reimplanted by injection in a physician's office in less than two hours. Stromal cells play an essential role in the body's natural healing response, with a dynamic and reactive ability to participate in the healing process. The American Medical Associationgranted GID two new CPT class III codesthat became effective January 2020 as a step toward Medicare reimbursement.

About GID BIOGID BIO develops next-generation cellular therapies for degenerative musculoskeletal, dermal, and organ-specific diseases, with the goal of making cellular medicine available to as many people as possible. GID's SVF-2 device and POC therapy harnesses the innate healing power of a patient's own stromal cells. Information on GID's SVF-2 device, biologic cellular implants, POC therapy, osteoarthritis clinical program and GID's pipeline for treating degenerative disease in musculoskeletal conditions includes other indications including, dermal and organs, specifically, wound care and diabetes. Learn more: https://www.HealingIntelligently.com.

AboutTulane University School of MedicineOne of the nation's most recognized centers for medical education,Tulane University School of Medicineis a vibrant center for education, research and public service.Tulane School of Medicineis the second-oldest medical school in the Deep South and the 15th oldest medical school inthe United States.Tulane School of Medicinerecruits top faculty, researchers and students from around the world, and pushes the boundaries of medicine with groundbreaking medical research and surgical advances.Tulaneremains in the forefront of modern medical innovation and is equipping the next generation of medical professionals with the tools to succeed in the rapidly changing future of health care.

About American Journal of Sports MedicineAglobal organization with 3,000 members that generates evidence-based knowledge and promotes emerging research to educate orthopaedic surgeonsand a resource for the orthopaedic sports medicine community, American Journal of Sports Medicine is a peer-reviewed scientific journal, first published in 1972. It is the official publication ofAOSSMfeaturing 14 issues per year. The journal acts as an important forum for independent orthopaedic sports medicine research and education, allowing clinical practitioners the ability to make decisions based on sound scientific information.

Contact:Kellee Johnson, 312-751-3959 or kjohnson@ballastgroup.com

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SOURCE GID BIO

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"American Journal of Sports Medicine" publishes results of an FDA-approved clinical trial for treating osteoarthritis knee pain - P&T Community

Ozzy Osbourne is banking on a stem cell treatment and Pilates to help him manage his Parkinson's disease.

The Paranoid hitmaker has been laid up since badly injuring himself and suffering from pneumonia last year, revealing he had a type of Parkinson's in January, and scrapping his U.S. tour last month, to head to Switzerland for treatment.

In a joint interview with U.K. TV show Good Morning Britain, Ozzy and his wife and manager Sharon opened up about the rocker's recovery - revealing he is undergoing stem cell treatment to lessen the effects of Parkinson's and to boost his immune system.

"There's a professor there (in Switzerland)," Sharon said. "He hasn't got a cure for Parkinson's, no one has but what he can do is... he can get Ozzy's imune system to here (points high), so now, if Ozzy was to catch a cold it would turn into pneumonia.

"This professor has come up with a way of doing stem cells where it helps with the pain. He could hopefully get rid of Ozzy's pain and then Ozzy will be healthier to deal with the Parkinson's."

The 71-year-old is not the first person in his family to undergo stem cell treatment, as his son Jack flew to Germany to receive similar therapy to help with his multiple sclerosis.

Meanwhile, the former Black Sabbath frontman has also been working hard to get fit again - but thinks he will only truly feel himself again when he's back performing.

"I exercise as much as I can. I've got a trainer, I do Pilates, nurses 24/7, but the best medication I can get is being in front of an audience, which is breaking my heart, to be honest," he added. "I will (perform again). Absolutely. I will be up there. I have to say that. I know you're going to say what will you do if you can't do it again, that's not an option because I will do it."

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Ozzy Osbourne banking on stem cell treatment to get him back onstage - Music News

TMRR, in its recent market report, suggests that the Stem Cell Therapy market report is set to exceed US$ xx Mn/Bn by 2029. The report finds that the Stem Cell Therapy market registered ~US$ xx Mn/Bn in 2018 and is spectated to grow at a healthy CAGR over the foreseeable period.

The Stem Cell Therapy market research focuses on the market structure and various factors (positive and negative) affecting the growth of the market. The study encloses a precise evaluation of the Stem Cell Therapy market, including growth rate, current scenario, and volume inflation prospects, on the basis of DROT and Porters Five Forces analyses. In addition, the Stem Cell Therapy market study provides reliable and authentic projections regarding the technical jargon.

In this Stem Cell Therapy market study, the following years are considered to project the market footprint:

The content of the Stem Cell Therapy market report includes the following insights:

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On the basis of solution, the global Stem Cell Therapy market report covers the following solutions:

Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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The Stem Cell Therapy market study answers critical questions including:

All the players running in the global Stem Cell Therapy market are elaborated thoroughly in the Stem Cell Therapy market report on the basis of R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, the report examines, legal policies, and comparative analysis between the leading and emerging Stem Cell Therapy market players.

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Stem Cell Therapy Market Opportunity Analysis and Industry Forecast up to 2017 2025 - Jewish Life News

Reported Initial Clinical Data from FT500 Phase 1 Study in Advanced Solid Tumors, Supporting Safety and Tolerability of Multi-dose Treatment Paradigm for Off-the-shelf, iPSC-derived NK Cells

First Patients Treated with FT516, the First-ever Engineered iPSC-derived Cellular Immunotherapy, for AML and for B-cell Lymphoma in Combination with Rituximab

Initiated Enrollment of First-in-human Clinical Trial of FT596, the First-ever Cellular Immunotherapy Engineered with Three Active Anti-tumor Modalities

Ended Quarter with $261 Million in Cash, Cash Equivalents and Marketable Securities

SAN DIEGO, March 02, 2020 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, today reported business highlights and financial results for the fourth quarter ended December 31, 2019.

In 2019, we made tremendous progress in pioneering the clinical development of off-the-shelf, iPSC-derived cancer immunotherapy. Our FT500 program demonstrated that multiple doses of iPSC-derived NK cells can be delivered off-the-shelf to a patient in a safe manner without patient matching. Additionally, our FT516 program provided initial clinical evidence that engineered iPSC-derived NK cells may confer anti-tumor activity and deliver clinically meaningful benefit to patients. We also showed the unmatched scalability of our proprietary iPSC product platform, having manufactured hundreds of cryopreserved, infusion-ready doses of our iPSC-derived NK cell product candidates at a low cost per dose in our new GMP manufacturing facility, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. In 2020, we look forward to additional clinical data from our FT500 and FT516 programs, and initial clinical data from FT596, our ground-breaking iPSC-derived CAR NK cell product candidate for the treatment of B-cell malignancies designed to overcome many of the limitations inherent in current CAR T-cell immunotherapies. We also expect to begin clinical investigation of our off-the-shelf, iPSC-derived NK cell programs in multiple myeloma with planned IND submissions for FT538, the first-ever CRISPR-edited, iPSC-derived cell therapy, and for FT576, our multi-antigen targeted, CAR-BCMA product candidate. Finally, under our collaboration with Memorial Sloan Kettering, we strive to be the first group in the world to bring off-the-shelf, iPSC-derived CAR T-cell therapy to patients.

Clinical Programs

Preclinical Pipeline

Fourth Quarter 2019 Financial Results

Today's Conference Call and Webcast

The Company will conduct a conference call today, Monday, March 2, 2020 at 5:00 p.m. ET to review financial and operating results for the quarter ended December 31, 2019. In order to participate in the conference call, please dial 877-303-6229 (domestic) or 631-291-4833 (international) and refer to conference ID 9879730. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company's website at http://www.fatetherapeutics.com. The archived webcast will be available on the Company's website beginning approximately two hours after the event.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 300 issued patents and 150 pending patent applications.

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About FT500

FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of advanced solid tumors (NCT03841110). The study is designed to assess the safety and tolerability of three once-weekly doses of FT500 as a monotherapy and in combination with one of three FDA-approved immune checkpoint inhibitor (ICI) therapies nivolumab, pembrolizumab or atezolizumab in patients that have failed prior ICI therapy. Despite the clinical benefit conferred by approved ICI therapy against a variety of tumor types, these therapies are not curative and, in most cases, patients either fail to respond or their disease progresses on these agents. One common mechanism of resistance to ICI therapy is associated with loss-of-function mutations in genes critical for antigen presentation. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill tumor cells with these mutations.

About FT516

FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, the FDA has allowed investigation of FT516 in an open-label, multi-dose Phase 1 clinical trial in combination with monoclonal antibody therapy, including PDL1-, PD1-, EGFR- and HER2-targeting therapeutic antibodies, across a broad range of solid tumors.

About FT596FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology, which contains a NKG2D transmembrane domain, a 2B4 co-stimulatory domain and a CD3-zeta signaling domain, that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that promotes enhanced NK cell activity. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and hnCD16 targeting receptors, in combination with IL-15RF signaling, convey synergistic anti-tumor activity. Increased degranulation and cytokine release were observed upon dual receptor activation in lymphoma cancer cells as compared to activation of each receptor alone, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a humanized mouse model of lymphoma, FT596 in combination with the anti-CD20 monoclonal antibody rituximab showed enhanced killing of tumor cells in vivo as compared to rituximab alone. FT596 is being investigated in an open-label Phase 1 clinical trial as a monotherapy, and in combination with rituximab, for the treatment of advanced B-cell lymphoma and in combination with obinutuzumab for the treatment of chronic lymphocytic leukemia (NCT04245722).

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Companys results of operations, financial condition and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to its product candidates, clinical studies and preclinical research and development programs, the Companys progress, plans and timelines for the manufacture and clinical investigation of its product candidates, the timing for the Companys receipt of data from its clinical trials and preclinical studies, the Companys development and regulatory strategy, and the therapeutic and market potential of the Companys product candidates. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that results observed in prior studies of the Companys product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Companys product candidates or in the initiation of, or enrollment of patients in, any clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties or delays in patient enrollment in current and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that the Companys expenditures may exceed current expectations for a variety of reasons. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Availability of Other Information about Fate Therapeutics, Inc.

Investors and others should note that the Company routinely communicates with investors and the public using its website (www.fatetherapeutics.com) and its investor relations website (ir.fatetherapeutics.com) including, without limitation, through the posting of investor presentations, SEC filings, press releases, public conference calls and webcasts on these websites. The information posted on these websites could be deemed to be material information. As a result, investors, the media, and others interested in Fate Therapeutics are encouraged to review this information on a regular basis. The contents of the Companys website, or any other website that may be accessed from the Companys website, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except share and per share data)(unaudited)

Condensed Consolidated Balance Sheets(in thousands)(unaudited)

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Reports Fourth Quarter 2019 Financial Results and Operational Progress with 2020 Outlook - Yahoo Finance

Introduction

CRISPR Therapeutics is a gene-editing company focused on the development of CRISPR (Clustered Regulatory Interspaced Short Palindromic Repeats)/CAS9(CRISPR-associated protein 9)-based therapeutics. The company is focused on translating revolutionary CRISPR/Cas9 technology into transformative therapies in therapeutics areas such as hemoglobinopathies, immuno-oncology, regenerative medicine and in vivo applications.

The Need for Gene Editing

Aberrant DNA sequences cause thousands of diseases that have not been treated by traditional small molecule and biologics as such treatments do not address the underlying genetics causes. Gene editing has the potential to provide curative therapies to many genetic diseases by precisely altering DNA sequences within the genomes of cells, which is done with the aid of enzymes cutting the DNA at specific locations. After a cut is made, natural cellular processes repair the DNA to either silence or correct undesirable sequences, potentially reversing their negative effects. As the genome itself is modified in this process, the change is permanent in the patient.

Gene editing also has other applications beyond treating genetically-defined diseases. It can also be applied to the engineering of genomes of cell therapies to make them more efficacious and safer. Cell therapies have been making a meaningful impact in certain therapeutics areas, such as oncology. An example of that is the approval of the CAR-Ts by Novartis (NVS) and Gilead (GILD).

The CRISPR/Cas9 Technology

As its name suggests, the company is utilizing CRISPR/Cas9 as its method of gene editing. Their technology is based on the work of their co-founder, Dr. Emmanuelle Charpentier, who is acknowledged as one of the key inventors of CRISPR-Cas9, and her collaborators.

Figure 1 Applications of CRISPR/Cas9 (Source)

The CRISPR/Cas9 technology is a versatile technology that can be used to disrupt, delete, correct or inset genes. It is used to make cuts in DNA at specific sites of targeted genes, and once the DNA is cut, the cell uses naturally occurring DNA repair mechanisms to rejoin the cut ends. If a single cut is made, a process called non-homologous end joining can result in the addition or deletion of base pairs, disrupting the original DNA sequence and causing gene inactivation. A larger fragment of DNA can also be deleted by using two guide RNAs that target separate sites. After cleavage at each site, non-homologous end joining unites the separate ends, deleting the intervening sequence. Alternatively, if a DNA template is added alongside the CRISPR/Cas9 machinery, the cell can correct a gene or even insert a new gene through a process called homology-directed repair.

Clinical Pipeline

CRISPRs lead product candidate is CTX001 which is being evaluated in -thalassemia and Sickle-Cell Disease (SCD). Both -thalassemia and SCD result from mutations in a gene that encodes a key component of hemoglobin, the molecule that carries oxygen in the blood. Both diseases require lifetime treatment that can result in the need for regular transfusion, painful symptoms and ultimately reduced life expectancy.

The companys approach to treat both diseases is to increase the levels of fetal hemoglobin (HbF), which is a naturally-occurring form of hemoglobin present in all people before birth. The company believes that HbF can substitute for the diseased hemoglobin in -thalassemia and SCD patients, therefore reducing or eliminating symptoms.

CTX001 first isolates a patients own blood stem cells, which is then edited with CRISPR/Cas9 to increase HbF expression, and then returned to the patient. The company believes that over time these edited blood stem cells will generate red blood cells that have increased levels of HbF, which may reduce or eliminate patients symptoms. CTX001 is co-developed and co-commercialized in an agreement with Vertex Pharmaceuticals (VRTX).

In November 2019, both companies announced interim data from the first 2 patients treated in CTX001. 1 patient with transfusion-dependent -thalassemia (TBT) received the treatment in the first quarter of 2019 and the other patient was treated for SCD in mid-2019. The safety and efficacy follow-up of both patients was 9 months and 4 months approximately.

The patient with TDT required 16.5 transfusions per year before enrolling in the clinical study. At nine months after the CTX001 infusion, the patient was transfusion independent. There were 2 serious adverse events (SAEs), although they were assessed to be not related to the administration of CTX001.The patient with SCD experienced seven vaso-occlusive crises (VOCs) per year before enrolling in the clinical study. Three SAEs occurred, none of which were considered related to CTX001. At four months after CTX001 infusion, the patient was free of VOCs. Both the TDT and SCD studies are ongoing and all patients will be followed for approximately two years following the infusion of CTX001. The Company has also mentioned that several additional patients have been enrolled in both trials.

The company is also working on allogeneic CAR-Ts with its gene-editing technology. Current generations of CAR-Ts such as Kymirah from Novartis (NVS) and Yescarta from Gilead (GILD) are autologous and derived from the patients own immune cells. Such treatments have several limitations and healthy-donor based allogeneic CAR-Ts have the potential to improve on the current generation of CAR-Ts.

CRISPR believes that CRISPR-edited allogeneic CAR-Ts has the potential to improve cell persistence as well as overall safety and potency. Its first 2 programs target well-validated targets with the potential to be best-in-class. CTX100 is an anti-CD19 CAR T targeting B-cell malignancies while CTX120 is an anti-B-Cell Maturation Antigen (BCMA) targeting multiple myeloma. Both trials are currently enrolling patients, although no interim data has been released.

A third allogeneic CAR-T, CTX130 is planned to eventually be advanced to clinical trials. CTX130 targets CD70 and will be used to treat both solid tumors, such as renal cell carcinoma, as well as T-cell and B-cell hematologic malignancies. Beyond immunology-oncology, the company also plans to utilize CRISPR/Cas9 in both Regenerative Medicines and In Vivo applications, although such efforts are still limited to preclinical development. Figure 2 illustrates the full clinical pipeline of the company.

Figure 2 CRISPR Therapeutics Clinical Pipeline (Source)

Financials and Competition

As of 31 Dec 2019, cash and equivalents were $943.8M, compared to $435.6 a year prior. The increase in cash was driven by several public offerings, as well as cash received from Vertex for milestone and option payments. The healthy cash pile should take them well into 2021 at the very least.

As the company is working in the gene therapy and cell therapy space, there are several notable competitors. They are often compared to Bluebird Bio (BLUE) who has received the approval of Zynteglo to treat TDT in Europe and in the process of filing a BLA with the FDA for US approval. Bluebird is also evaluating Lentiglobin in SCD. As both of Bluebirds product candidates are more advanced in terms of clinical development, they currently hold a competitive advantage unless CRISPR can prove that their treatments are best-in-class. Notably, Bluebird has faced several challenges with its pricing of Zynteglo as well as regulatory delays due to complex manufacturing and it remains to be seen whether CRISPR can overcome such challenges. Bluebird is also partnering with Bristol-Meyers Squibb (BMY) to develop bb2121 and bb21217 which are both autologous anti-BCMA CAR-T against multiple myeloma.

In the Allogeneic CAR-T space, there also several prominent names that include but are not limited to Allogene Therapeutics (ALLO), Cellectis (CLLS) and Precision Biosciences (DTIL). The main difference among these companies is primarily the choice of gene-editing tools with Allogene and Cellectis using TALEN while Precision is using ARCUS. All these companies are currently in a similar stage of clinical development, with multiple programs in Phase 1 and it remains to be seen who will emerge as a clear frontrunner, even though interestingly, Allogene is trading at a premium market cap compared to the other 2 companies.

In addition to healthy donors derived allogeneic therapies, Fate Therapeutics (FATE) is developing allogeneic therapies from induced pluripotent stem cells (iPSCs) as a renewable cell source. The advantage of this is that product consistency and potency will be improved, and the manufacturing process will be akin to the well-established biologics where they are produced from a single cell line. It is notable to note that Allogene is also investigating using iPSCs as a renewable cell source.

Also, Atara Biotherapeutics (ATRA) is developing an Epstein-Barr Virus (EBV)-based allogeneic T cell therapy platform. Their lead program is in Phase 3 and a BLA filing is expected by the second half of the year. That should put them in the lead position of commercializing an allogeneic T cell therapy and the company is gradually moving into CAR T space as well.

Lastly, there are also other companies such as Editas Medicine (EDIT) and Intellia Therapeutics (NTLA) which are focused on using CRISPR/Cas9 as a gene-editing tool. While both companies are also working on treatments for TDT and SCD, these are not their lead programs and CRISPR is further along than both companies in both therapeutics areas.

Conclusion

CRISPR Therapeutics is a gene-editing company utilizing CRISPR/cas9 to develop therapies in hemoglobinopathies, immuno-oncology, regenerative medicine and in vivo applications. While I consider the company to be a pioneer in CRISPR/Cas9, its market cap of around $3B seems generous for a company that has so far reported only interim data from 2 patients.

Also, there is a long ongoing-argument over the patents of CRISPR/Cas9 between the University of California, which CRISPR license their technology from, and the Broad Institute and Harvard College, of which Editass technology is based on.

With the uncertainty over the patent claims as well as the limited clinical data available, I am inclined to avoid investing in the company for now, although I would be keeping a keen eye on further clinical data, especially on allogeneic CAR-Ts.

As always, investors should do their due diligence before taking up any positions and consider their risk profiles and time horizon. I have covered several companies working on cell therapies and will continue to do so in the coming weeks and months.

Disclosure: I am/we are long ATRA, BLUE. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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CRISPR Therapeutics: A Review Of Its Clinical Pipeline And Progress - Seeking Alpha