Category Archives: Stem Cell Treatments

Unregulated clinics worldwide are offering stem cell therapies that may not live up to the hype, and can worsen a patients health.

The injections of stem cells into his spine were supposed to help Jim Gass, 66, recover from a stroke he had six years ago.

Gass traveled to clinics in Mexico, China, and Argentina to undergo these unproven procedures. Including travel, he spent close to $300,000, according to a story in The New York Times.

After the final round of shots, he was able to walk better. But his hope for a full recovery was cut short. While on vacation in Thailand six months after his treatments, he developed low back pain and difficulty walking and standing.

Back in Boston, doctors at Brigham and Womens Hospital did an MRI scan of his spine, and found a large mass filling the entire lower part of his spinal column.

Genetic testing revealed that the abnormal, primitive cells of the mass did not come from Gass, but from stem cells injected into his spine.

Radiation treatments seemed to slow the growth of the mass and improve Gass symptoms. But another scan done later in San Diego showed that the mass was growing again.

The doctors involved wrote about his case in a letter published June 22 in the New England Journal of Medicine.

Despite the outcome of this case, experts familiar with this kind of stem cell tourism say that some good may still come of it.

It is a really sad case, but its good that its causing discussion around both the potential harm of these therapies and the lack of evidence regarding the benefits, Timothy Caulfield, research director of the Health Law Institute at the University of Alberta, who wrote a recent commentary on stem cell hype, told Healthline.

Read more: Get the facts on stem cell research

This is not the first time that stem cell treatments have led to bad outcomes such as tumors or lesions.

There have been other reports of adverse events as a result of these kinds of therapies, said Caulfield. There have even been reports of adverse events when the procedure is less extreme such as people getting stem cell therapy for anti-aging, anti-wrinkle procedures.

Caulfield is quick to point out that therapy should be in quotes because with the exception of a few approved treatments the use of stem cells to treat illnesses has not reached the point where it is ready for widespread use in clinics.

There are very few stem cell therapies that have been proven, at this point, to be efficacious, said Caulfield. Lots of exciting work is going on theyre in clinical trials right now but for most conditions we simply arent there yet.

Although there are a few documented cases like Gass, many more may go unreported, resulting from treatments at unregulated stem cell clinics around the world.

We dont know exactly how many people are having these procedures, Dr. Jaime Imitola, a neurologist and stem cell researcher at The Ohio State University Wexner Medical Center, who has written about the dangers of stem cell tourism and how to counsel patients, told Healthline.

There are so many diseases that these clinics are often treating for from diabetes to ALS and some of these treatments may involve more risk than others, Imitola said.

Theres a big difference in risk between taking cells from your own body and putting them back in your blood, and injecting foreign cells into your spine, as was done in Gass case.

Also, these clinics are not part of a clinical research program, so there are a lot of unknowns about what happens during the procedures.

Are they actually using stem cells? How are they getting the stem cells into people? said Caulfield. Those are all open questions, because it is such an unregulated field.

While Gass traveled outside the United States for injections, unproven stem cell therapies show up much closer to home.

A paper published online Thursday in the journal Cell Stem Cell found that at least 351 businesses in the United States are marketing stem cell therapies that have not gone through the rigorous clinical trial process, or been approved by the Food and Drug Administration (FDA).

These businesses marketed stem cells as treatment for a wide range of conditions from spinal cord injuries and immune system problems to heart disease or even cosmetic fixes.

Read more: Stem cell treatments offering hope for MS patients

With few treatments available for many diseases, stem cell clinics step in to fill the void, many overhyping the actual research being done in this area.

[Clinics] are leveraging excitement around legitimate stem cell research and the pop culture footprint Ill put it that way of stem cells, said Caulfield.

Some of this hype has been generated when high-profile athletes undergo stem cell therapy and see improvements, like Peyton Manning did in Germany for a neck injury.

The company that Gass contacted had been involved in the treatment of former NFL quarterback John Brodie.

These remarkable success stories offer people hope. But because they happened outside a clinical trial, its impossible to know if the athletes health would have improved on their own.

Imitola compares this to using acupuncture alongside proven treatments.

If I give you acupuncture after a stem cell treatment, I cannot make the distinction whether what happens is a result of the acupuncture or the treatment, said Imitola, because this is not a clinical trial.

Researchers, universities, and the media also have a hand in stem cell hype. The time element, in particular, can be misrepresented.

I think that the scientific community really needs to be careful how they talk about stem cell research, said Caulfield. We did a study that showed, for example, that the time from doing basic research to getting into the clinic is often exaggerated when people talk about stem cell research. Our study found that it was often portrayed as if the research was going to be in the clinic in 5 to 10 years, or sooner, which is really, really fast. It creates unrealistic expectations.

Read more: Stem cells as a possible treatment for rheumatoid arthritis

Patients with spinal cord injuries or diseases are often anxious for new treatments to be approved quickly. But stem cell researchers have good reason to be cautious.

One characteristic that stem cells share with cancer cells is that they both multiply rapidly. This is why stem cell researchers have long been concerned that stem cells could form tumors.

Thats why there are so many years of testing in the lab, in animal models, and finally in clinical trials.

It is unethical to offer a procedure or a drug that is unproven, said Imitola.

When clinics skip ahead and offer treatments that have not been properly tested, they may end up hurting people instead of helping them.

Its interesting because [Gass] case, and others, is generating a new disease, a new complication, an iatrogenic tumor, said Imitola.

Of course, bad outcomes can happen during a clinical trial. But these are tracked, and clinical trials can be shut down if unforeseen side effects happen.

A recent stem cell clinical trial in Japan was stopped, because when the researchers looked at whether the cells were clean from a genetic point of view, the cells had some problems, some changes, said Imitola, So the researchers said, We cant do that, we cant inject the cells.

Imitola recently co-authored a paper in JAMA Neurology calling on doctors to educate patients with neurological diseases about stem cell tourism.

But he admits that cases like Gass can serve as an even more effective warning.

This patient, in particular, is important because he put a human face to this tragedy, said Imitola. We need more patients to come forward. Most likely, this is not an isolated case.

Read more: Stem cell treatment for COPD

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Unproven Stem Cell Treatments Offer Hope & Risks - Healthline

Problems soon after transplant

Many of the problems that can happen shortly after the transplant come from having the bone marrow wiped out by medicines or radiation just before the transplant. Others may be side effects of the conditioning treatments themselves.

Your transplant team can help you cope with side effects. Some can be prevented, and most can be treated to help you feel better. This is not a complete list and you should tell your doctor or transplant team about any problems you have or changes you notice. Some of these problems can be life-threatening, so its important to be able to reach your doctor or transplant team at night, on weekends, and during holidays. Ask for their after hours contact numbers to makesure you will be able to do this.

Mucositis (inflammation or sores in the mouth) is a short-term side effect that can happen with chemo and radiation. It usually gets better within a few weeks after treatment, but it can make it very painful to eat and drink.

Good nutrition is important for people with cancer. If mouth pain or sores make it hard to eat or swallow, your transplant team can help you develop a plan to manage your symptoms.

Because chemotherapy drugs can cause severe nausea and vomiting, doctors often give anti-nausea medicines at the same time as chemo to try to prevent it. As much as possible, the goal is to prevent nausea and vomiting, because its easier to prevent it than it is to stop it once it starts. Preventive treatment should start before chemo is given and should continue for as long as the chemo is likely to cause vomiting, which can be up to 7 to 10 days after the last dose.

No one drug can prevent or control chemo-related nausea and vomiting 100% of the time. In many cases, two or more medicines are used. Youll need to tell your transplant team how well the medicines are controlling your nausea and vomiting. If they arent working, they will need to be changed.

For at least the first 6 weeks after transplant, until the new stem cells start making white blood cells (engraftment), you can easily get serious infections. Bacterial infections are most common during this time, but viral infections that were controlled by your immune system can become active again. Fungal infections can also be an issue. And even infections that cause only mild symptoms in people with normal immune systems can be quite dangerous for you. This is because right after the transplant you don't have many white blood cells that are working well, and they are the primary immune cells that fight off infections.

You may be given antibiotics to try to prevent infections until your blood counts reach a certain level. For instance, pneumocystis pneumonia (often called PCP) is a common infection thats easy to catch. Even though the germ doesnt harm people with normal immune systems, for others it can cause fever, cough, and serious breathing problems. Antibiotics are often used to keep transplant patients from getting this.

Your doctor may check you before the transplant for signs of certain infections that may become active after transplant, and give you special medicines to keep those germs under control. For example, the virus called CMV (cytomegalovirus) is a common infection that many adults have or had in the past. Adults with healthy immune systems may not have any symptoms because their immune system can keep the virus under control. But, CMV can be a cause of serious pneumonia in people who have had transplants, because the transplant lowers the amount of white blood cells they have. Pneumonia from CMVmainly happens to people who were already infected with CMV, or whose donor had the virus. If neither you nor your donor had CMV, the transplant team might follow special precautions to prevent this infection while you are in the hospital.

After engraftment, the risk of infection is lower, but it still can happen. It can take 6 months to a year after transplant for the immune system to work as well as it should. It can take even longer for patients with graft-versus-host disease (GVHD, see below). It's important to talk to your cancer care team about your risk for infection during this time.

Because of the increased risk, you will be watched closely for signs of infection, such as fever, cough, shortness of breath, or diarrhea. Your doctor may check your blood often, and extra precautions will be needed to keep you from being exposed to germs. While in the hospital, everyone who enters your room must wash their hands well. They may also wear gowns, shoe coverings, gloves, and masks.

Since flowers and plants can carry bacteria and fungi, theyre not allowed in your room. For the same reason, you may be told not to eat certain fresh fruits and vegetables. All your food must be well cooked and handled very carefully by you and family members. You might need to avoid certain foods for a while.

You may also be told to avoid contact with soil, feces (stool, both human and animal), aquariums, reptiles, and exotic pets. Your team may tell you to avoid being near disturbed soil, bird droppings, or mold. You will need to wash your hands after touching pets. Your family may need to move the cats litter box away from places you eat or spend your time. Also, you should not clean pet cages or litter boxes during this time. Instead, give this task to a family member or friend.

Your transplant team will tell you and your family in detail about the precautions you need to follow. There are many viruses, bacteria, and fungi that can cause infection after your transplant. You may be at risk for some more than others.

Despite all these precautions, patients often develop fevers, one of the first signs of infection. In fact, sometimes fever is the only sign of infection, so it's very important to contact your cancer care team if you have one or if you have any other signs of infection. You'll probably be asked to take your temperature by mouth every day or twice a day for a while. And your cancer care team will let you know when you should call in your temperature to them. If you get a fever, tests will be done to look for possible causes of the infection (chest x-rays, urine tests, and blood cultures) and antibiotics will be started.

After transplant, youre at risk for bleeding because the conditioning treatment destroys your bodys ability to make platelets. Platelets are the blood cells that help blood to clot. While you wait for your transplanted stem cells to start working, your transplant team may have you follow special precautions to avoid injury and bleeding.

Platelet counts are low for at least several weeks after transplant. In the meantime, you might notice easy bruising and bleeding, such as nosebleeds and bleeding gums. If your platelet count drops below a certain level, a platelet transfusion may be needed. Youll need to follow precautions until your platelet counts stay at safe levels.

It also takes time for your bone marrow to start making red blood cells, and you might need red blood cell transfusions from time to time as you recover.

For more information on the transfusion process, see Blood Transfusion and Donation.

Pneumonitis is a type of inflammation (swelling) in lung tissue thats most common in the first 100 days after transplant. But some lung problems can happen much later even 2 or more years after transplant.

Pneumonia caused by infection happens more often, but pneumonitis may be caused by radiation, graft-versus-host disease, or chemo rather than germs. Its caused by damage to the areas between the cells of the lungs (called interstitial spaces).

Pneumonitis can be severe, especially if total body irradiation was given with chemo as part of the pre-transplant (conditioning) treatment. Chest x-rays will be taken in the hospital to watch for pneumonitis as well as pneumonia. Some doctors will do breathing tests every few months if you have graft-versus-host disease (see next section).

You should report any shortness of breath or changes in your breathing to your doctor or transplant team right away. There are many other types of lung and breathing problems that also need to be handled quickly.

Graft-versus-host disease (GVHD) can happen in allogeneic transplants when the immune cells from the donor see your body as foreign. (Remember: The recipients immune system has mostly been destroyed by conditioning treatment and cannot fight back, so the new stem cells make up most of the immune system after transplant.) The donor immune cells may attack certain organs, most often the skin, gastrointestinal (GI) tract, and liver. This can change the way the organs work and increase the chances of infection.

GVHD reactions are very common and can range from barely noticeable to life-threatening. Doctors think of GVHD as acute or chronic. Acute GVHD starts soon after transplant and lasts a short time. Chronic GVHD starts later and lasts a long time. A person could have one, both, or neither type of GVHD.

Acute GVHD can happen 10 to 90 days after a transplant, though the average time is around 25 days.

About one-third to one-half of allogeneic transplant recipients will develop acute GVHD. Its less common in younger patients and in those with closer HLA matches between donor and the patient.

The first signs are usually a rash, burning, and redness of the skin on the palms and soles. This can spread over the entire body. Other symptoms can include:

Doctors try to prevent acute GVHD by giving drugs that suppress the immune system, such as steroids (glucocorticoids), methotrexate, cyclosporine, tacrolimus, or certain monoclonal antibodies. These drugs are given before acute GVHD starts and can help prevent serious GVHD. Still, mild GVHD will almost always happen in allogeneic transplant patients. Other drugs are being tested in different combinations for GVHD prevention.

The risk of acute GVHD can also be lowered by removing immune cells called T-cells from the donor stem cells before the transplant. But this can also increase the risk of viral infection, leukemia relapse, and graft failure (which is discussed later). Researchers are looking at new ways to remove only certain cells, called alloactivated T-cells, from donor grafts. This would reduce the severity of GVHD and still let the donor T-cells destroy any cancer cells left.

If acute GVHD does occur, it is most often mild, mainly affecting the skin. But sometimes it can be more serious, or even life-threatening.

Mild cases can often be treated with a steroid drug applied to the skin (topically) as an ointment, cream, or lotion, or with other skin treatments. More serious cases of GVHD might need to be treated with a steroid drug taken as a pill or injected into a vein. If steroids arent effective, other drugs that affect the immune system can be used.

Chronic GVHD

Chronic GVHD can start anywhere from about 90 to 600 days after the stem cell transplant. A rash on the palms of the hands or the soles of the feet is often the earliest sign. The rash can spread and is usually itchy and dry. In severe cases, the skin may blister and peel, like a bad sunburn. A fever may also develop. Other symptoms of chronic GVHD can include:

Chronic GVHD is treated with medicines that suppress the immune system, much like those used for acute GVHD. These drugs can increase your risk of infection for as long as you are treated for GVHD. Most patients with chronic GVHD can stop the immunosuppressive drugs after their symptoms improve.

Hepatic veno-occlusive disease (VOD) is a serious problem in which tiny veins and other blood vessels inside the liver become blocked. Its not common, and it only happens in people with allogeneic transplants, and mainly in those who got the drugs busulfan or melphalan as part of conditioning, or treatment that was given before the transplant.

VOD usually happens within about 3 weeks after transplant. Its more common in older people who had liver problems before the transplant, and in those with acute GVHD. It starts with yellowing skin and eyes, dark urine, tenderness below the right ribs (this is where the liver is), and quick weight gain (mostly from fluid that bloats the belly). It is life-threatening, so early diagnosis of VOD is very important. Researchers continue to find ways to try to measure a person's chances of getting VOD so that treatment can start as soon as possible.

Grafts fail when the body does not accept the new stem cells (the graft). The stem cells that were given do not go into the bone marrow and multiply like they should. Graft failure is more common when the patient and donor are not well matched and when patients get stem cells that have had the T-cells removed. It can also happen in patients who get a low number of stem cells, such as a single umbilical cord unit. Still, its not very common.

Graft failure can lead to serious bleeding and/or infection. Graft failure is suspected in patients whose counts do not start going up within 3 to 4 weeks of a bone marrow or peripheral blood transplant, or within 7 weeks of a cord blood transplant.

Although it can be very upsetting to have this happen, these people can get treated with a second dose of stem cells, if they are available. Grafts rarely fail, but if they do it can result in death.

The type of problems that can happen after a transplant depend on many factors, such as the type of transplant done, the pre-transplant chemo or radiation treatment used, the patients overall health, the patients age when the transplant was done, the length and degree of immune system suppression, and whether chronic graft-versus-host-disease (GVHD) is present and how bad it is. The problems can be caused by the conditioning treatment (the pre-transplant chemotherapy and radiation therapy), especially total body irradiation, or by other drugs used during transplant (such as the drugs that may be needed to suppress the immune system after transplant). Possible long-term risks of transplant include:

The medicines used in transplants can harm the bodys organs, such as the heart, lungs, kidneys, liver, bones/joints, and nervous system. You may need careful follow-up with close monitoring and treatment of the long-term organ problems that the transplant can cause. Some of these, like infertility, should be discussed before the transplant, so you can prepare for them.

Its important to find and quickly treat any long-term problems. Tell your doctor right away if you notice any changes or problems. Physical exams by your doctor, blood work, imaging tests, lung/breathing studies, and other tests will help look for and keep tabs on organ problems.

As transplant methods have improved, more people are living longer and doctors are learning more about the long-term results of stem cell transplant. Researchers continue to look for better ways to care for these survivors to give them the best possible quality of life.

The goal of a stem cell transplant in cancer is to prolong life and, in many cases, even cure the cancer. But in some cases, the cancer comes back (sometimes called relapse or recurrence depending on when it might occur after a transplant). Relapse or recurrence can happen a few months to a few years after transplant. It happens much more rarely 5 or more years after transplant.

If cancer comes back, treatment options are often quite limited. A lot depends on your overall health at that point, and whether the type of cancer you have responds well to drug treatment. Treatment for those who are otherwise healthy and strong may include chemotherapy or targeted therapy. Some patients who have had allogeneic transplants may be helped by getting white blood cells from the same donor (this is called donor lymphocyte infusion) to boost the graft-versus-cancer effect. Sometimes a second transplant is possible. But most of these treatments pose serious risks even to healthier patients, so those who are frail, older, or have chronic health problems are often unable to have them.

Other options may include palliative (comfort) care, or a clinical trial of an investigational treatment. Its important to know what the expected outcome of any further treatment might be, so talk with your doctor about the purpose of the treatment. Be sure you understand the benefits and risks before you decide.

Along with the possibility of the original cancer coming back (relapse) after it was treated with a stem cell transplant, there is also a chance of having a second cancer after transplant. Studies have shown that people who have had allogeneic transplants have a higher risk of second cancer than people who got a different type of stem cell transplant.

A cancer called post-transplant lymphoproliferative disease (PTLD), if it occurs, usually develops within the first year after the transplant. Other conditions and cancers that can happen are solid tumor cancers in different organs, leukemia, and myelodysplastic syndromes. These other conditions, if they occur, tend to develop a few years or longer after the transplant.

Risk factors for developing a second cancer are being studied and may include:

Successfully treating a first cancer gives a second cancer time (and the chance) to develop. No matter what type of cancer is treated, and even without the high doses used for transplant, treatments like radiation and chemo can lead to a second cancer in the future.

Post-transplant lymphoproliferative disorder (PTLD) is an out-of-control growth of lymph cells, actually a type of lymphoma, that can develop after an allogeneic stem cell transplant. Its linked to T-cells (a type of white blood cell that is part of the immune system) and the presence of Epstein-Barr virus (EBV). T-cells normally help rid the body of cells that contain viruses. When the T-cells arent working well, EBV-infected B-lymphocytes (a type of white blood cell) can grow and multiply. Most people are infected with EBV at some time during their lives, but the infection is controlled by a healthy immune system. The pre-transplant treatment given weakens the immune system, allowing the EBV infection to get out of control, which can lead to a PTLD.

Still, PTLD after allogeneic stem cell transplant is fairly rare. It most often develops within 1 to 6 months after allogeneic stem cell transplant, when the immune system is still very weak.

PTLD is life-threatening. It may show up as lymph node swelling, fever, and chills. Theres no one standard treatment, but its often treated by cutting back on immunosuppressant drugs to let the patients immune system fight back. Other treatments include white blood cell (lymphocyte) transfusions to boost the immune response, using drugs like rituximab to kill the B cells, and giving anti-viral drugs to treat the EBV.

Even though PTLD doesnt often happen after transplant, its more likely to occur with less well-matched donors and when strong suppression of the immune system is needed. Studies are being done to identify risk factors for PTLD and look for ways to prevent it in transplant patients who are at risk.

Most people who have stem cell transplants become infertile (unable to have children). This is not caused by the cells that are transplanted, but rather by the high doses of chemo and/or radiation therapy used. These treatments affect both normal and abnormal cells, and often damage reproductive organs.

If having children is important to you, or if you think it might be important in the future, talk to your doctor about ways to protect your fertility before treatment. Your doctor may be able to tell you if a particular treatment will be likely to cause infertility.

After chemo or radiation, some women may find their menstrual periods become irregular or stop completely. This doesnt always mean they cannot get pregnant, so birth control should be used before and after a transplant. The drugs used in transplants can harm a growing fetus.

The drugs used during transplant can also damage sperm, so men should use birth control to avoid starting a pregnancy during and for some time after the transplant process. Transplants may cause temporary or permanent infertility for men as well. Fertility returns in some men, but the timing is unpredictable. Men might consider storing their sperm before having a transplant.

For more information on having children after being treated for canceror sexual problems related to cancer treatment, see Fertility and Sexual Side Effects.

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Stem Cell or Bone Marrow Transplant Side Effects - American Cancer Society

It was a $137 million day for the Golden States stem cell agency no small event even for an enterprise that is backed by billions.

The scientific scope covered by the $137 million was impressive. It ranged from bolstering the vauntedAlpha Clinic Networkinitiated around the state by theCalifornia Institute for Regenerative Medicine (CIRM), as the agency is legally known, to raising the number ofCIRMs clinical trials to 83. Plus, CIRM directors gave the go-ahead to a $50 million program to finance shared labs around the state.

CIRMs cash comes from $5.5 billion that voters approved in 2020. The money is borrowed by the state via state bonds. The agency, however, does not have all its boodle lying around in a vault in its South San Francisco headquarters. CIRM can only receive $540 million in bond funding annually. But the cash carries over from year to year.

CIRMs $137-million-day came on Oct. 27, a few days before CIRM officially turned 18.

Nonetheless, awarding the money sooner rather than later is in CIRMs best interest. No research is done without cash. CIRM needs to generate results that will convince voters to approve more billions in about 10 years when its funding runs out. Given the slow pace of therapy development, a decade may span only the initial steps in the process.

CIRMs $137-million-day came on Oct. 27, a few days before CIRM officially turned 18. The ballot measure that gave birth to the agency, the largest such state enterprise in the country, was approved on Nov. 2, 2004. Voters awarded CIRM an initial $3 billion, hoping for quick development of miraculous stem cell therapies available to the general public. CIRM is still working on that promise.

The biggest chunk of last months $137 million went for the Alpha Clinics $72 million on top of the $40 million already invested in the network. Continued CIRM funding of the existing Alpha Clinic sites does raise questions about the initial rationale behind network

The first request for applicationsfor Alpha clinic was posted in 2013. It said the applications would be judged on whether they present a feasible and compelling business/fundraising proposal, and the likelihood that implementation of the plan would support the Alpha Stem Cell Clinics beyond the 5-year funding provided by this RFA. Last months awards are also for five years.

One criterion considered by reviewers was whether the (UCSD) application met the needs of underserved and disproportionately affected communities.

The clinics are aimed at expanding existing capacities for delivering stem cell, gene therapies and other advanced treatment to patients, according to CIRM. They also serve as a competency hub for regenerative medicine training, clinical research, and the delivery of approved treatments.

At last months meeting, CIRM directors expanded the program to includeStanford UniversityplusCedars-Sinaiand theUniversity of Southern California, both in Los Angeles. The network already includedUCLA, UC Davis, UC San Francisco, UC Irvine, UC San Diegoand theCity of Hope.

UC San Diegos bid for $8 million more hit a roadblock, however, when it was rejected prior to the Oct. 27 board by CIRMs application reviewers, who make the de facto decisions on grants while meeting behind closed doors. The board almost never overturns a positive decision by the reviewer on applications.

The reviewers found significant flaws in the UC San Diego application (number INFR4-13597). They included criticism of the diversity plan and problems with training. One criterion considered by reviewers was whether the application met the needs of underserved and disproportionately affected communities.

In other awards, the sole clinical trial application for $12 million went toJana Portnowat theBeckman Research InstituteofCity of Hope

The review summary said that was an underdeveloped portion of the proposal. Ability to effectively increase DEI (diversity, equity, inclusion) in enrollment seems to be there but so many aspects of recruitment, retention, etc were missing.

The review summary also cited the limited number of patients enrolled clinically in stem cell and gene therapy trials.

Catriona Jamieson, director of the current Alpha program at UC San Diego, successfully appealed the rejection of the application by reviewers ina five-page, single-spaced letterto CIRM directors.

CIRM ChairmanJonathan Thomastold the board that the San Diego program, which will extend into rural Imperial County, is absolutely first rate and has produced many excellent projects.

In other awards, the sole clinical trial application for $12 million went toJana Portnowat theBeckman Research InstituteofCity of Hopefor a phase one trial involving the development of a delivery vehicle for a cancer-killing virus that targets brain tumor cells (application number CLIN2-13162 #2). It was the second try by Portnow for CIRM funding.

Another $3 million was awarded toBoris MinevofCalidi Biotherapeuticsof La Jolla, Ca., for work to initiate a clinical trial involving skin cancer (application number CLIN1-14080).

The $50 million shared labs planwas approved by directors but does not immediately involve individual awards. CIRM plans a deadline of next spring for applications. They are scheduled to be approved in late 2023.

The aim of the labs effort is to overcome hurdles in stem cell research. Not all research laboratorieshave local access to relevant infrastructure and training, nor do all have the opportunity to collaborate with a stem cell-based modeling laboratory, CIRM said in the plan proposal.

Laboratories well-versed in stem cell-based modeling that share their expertise and/or provide models collaboratively cant meet demand, as it is time-consuming and costly to divert resources to educating and supporting other researchers.

Regarding the Alpha awards, below are the names of the recipient institutions and principal scientists, along with their application numbers. The numbers are needed to locate the specific application review summaries, which do not identify the applicants. The review summaries include both positive and negative comments about the applications. All of the awards are for $8 million.All of the review summaries can be found at this link.

Cedars Sinai Michael Lewis, INFR-13586

City of Hope Leo Wang, INFR4-13587

Stanford University Matthew Porteus,INFR4-13579

UC Davis Mehrdad Abedi, INFR4-13596

UC Irvine Daniela Bota,INFR4-13952

UC Los Angeles Noah Federman, INFR4-13685

UC San Diego Catriona Jamieson,INFR4-13597

UC San Francisco Mark Walters, INFR4-13581Editors Note: David Jensenis a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report,where this story first appeared.He has published thousands of items on California stem cell matters.

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Stem cell: $137 million buys more clinical trials, shared labs ...

How are new treatments developed?

If you have seen a stem cell treatment advertised, featured in the media, or mentioned to you by a friend or fellow patient, it can be hard to work out if it may be an option for you.

Although there is a lot of attention surrounding the potential of stem cells, in reality, the range of diseases for which there are current proven stem cell treatments is quite small. Within Australia the only proven treatments available involving stem cells are corneal and skin grafting, and blood stem cell transplants for the treatment of some blood disorders, inherited immune and metabolic disorders, cancer and autoimmune diseases. There are many other potential treatments, but these are still in the research phase or in clinical trials and are yet to be proven as safe and effective.

This page provides a breakdown of the steps that should occur before a stem cell treatment makes it to you in a clinic, and identifies who should be looking after your interests.

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Stem Cells Australia | Australian research, stem cell treatments and ...

A bone marrow transplant is a medical treatment that replaces your bone marrow with healthy cells. The replacement cells can either come from your own body or from a donor.

A bone marrow transplant is also called a stem cell transplant or, more specifically, a hematopoietic stem cell transplant. Transplantation can be used to treat certain types of cancer, such as leukemia, myeloma, and lymphoma, and other blood and immune system diseases that affect the bone marrow.

Stem cells are special cells that can make copies of themselves and change into the many different kinds of cells that your body needs. There are several kinds of stem cells and they are found in different parts of the body at different times.

Cancer and cancer treatment can damage your hematopoietic stem cells. Hematopoietic stem cells are stem cells that turn into blood cells.

Bone marrow is soft, spongy tissue in the body that contains hematopoietic stem cells. It is found in the center of most bones. Hematopoietic stem cells are also found in the blood that is moving throughout your body.

When hematopoietic stem cells are damaged, they may not become red blood cells, white blood cells, and platelets. These blood cells are very important and each one has a different job:

Red blood cells carry oxygen throughout your body. They also take carbon dioxide to your lungs so that it can be exhaled.

White blood cells are a part of your immune system. They fight pathogens, which are the viruses and bacteria that can make you sick.

Platelets form clots to stop bleeding.

A bone marrow/stem cell transplant is a medical procedure by which healthy stem cells are transplanted into your bone marrow or your blood. This restores your body's ability to create the red blood cells, white blood cells, and platelets it needs.

There are different types of bone marrow/stem cell transplants. The 2 main types are:

Autologous transplant. Stem cells for an autologous transplant come from your own body. Sometimes, cancer is treated with a high-dose, intensive chemotherapy or radiation therapy treatment. This type of treatment can damage your stem cells and your immune system. That's why doctors remove, or rescue, your stem cells from your blood or bone marrow before the cancer treatment begins.

After chemotherapy, the stem cells are returned to your body, restoring your immune system and your body's ability to produce blood cells and fight infection. This process is also called an AUTO transplant or stem cell rescue.

Allogenic transplant. Stem cells for an allogenic transplant come from another person, called a donor. The donor's stem cells are given to the patient after the patient has chemotherapy and/or radiation therapy. This is also called an ALLO transplant.

Many people have a graft-versus-cancer cell effect during an ALLO transplant. This is when the new stem cells recognize and destroy cancer cells that are still in the body. This is the main way ALLO transplants work to treat the cancer.

Finding a donor match is a necessary step for an ALLO transplant. A match is a healthy donor whose blood proteins, called human leukocyte antigens (HLA), closely match yours. This process is called HLA typing. Siblings from the same parents are often the best match, but another family member or an unrelated volunteer can be a match too. If your donors proteins closely match yours, you are less likely to get a serious side effect called graft-versus-host disease (GVHD). In this condition, the healthy transplant cells attack your cells.

If your health care team cannot find a donor match, there are other options.

Umbilical cord blood transplant. In this type of transplant, stem cells from umbilical cord blood are used. The umbilical cord connects a fetus to its mother before birth. After birth, the baby does not need it. Cancer centers around the world use cord blood. Learn more about cord blood transplants.

Parent-child transplant and haplotype mismatched transplant. Cells from a parent, child, brother, or sister are not always a perfect match for a patient's HLA type, but they are a 50% match. Doctors are using these types of transplants more often, to expand the use of transplantation as an effective cancer treatment.

The information below tells you the main steps of AUTO and ALLO transplants. In general, each process includes collecting the replacement stem cells, the patient receiving treatments to prepare their body for the transplant, the actual transplant day, and then the recovery period.

Often, a small tube may be placed in the patient's chest that remains through the transplant process. It is called a catheter. Your health care team can give you chemotherapy, other medications, and blood transfusions through a catheter. A catheter greatly reduces the amount of needles used in the skin, since patients will need regular blood tests and other treatments during a transplant.

Please note that transplants are complex medical procedures and sometimes certain steps may happen in a different order or on a different timetable, to personalize your specific care. Ask your health care whether you will need to be in the hospital for different steps, and if so, how long. Always talk with your health care team about what to expect before, during, and after your transplant.

Step 1: Collecting your stem cells. This step takes several days. First, you will get injections (shots) of a medication to increase your stem cells. Then your health care team collects the stem cells through a vein in your arm or your chest. The cells will be stored until they are needed.

Step 2: Pre-transplant treatment. This step takes 5 to 10 days. You will get a high dose of chemotherapy. Occasionally, patients also have radiation therapy.

Step 3: Getting your stem cells back. This step is your transplant day. It takes about 30 minutes for each dose of stem cells. This is called an infusion. Your health care team puts the stem cells back into your bloodstream through the catheter. You might have more than one infusion.

Step 4: Recovery. Your doctor will closely monitor your cells' recovery and growth and you will take antibiotics to reduce infection. Your health care team will also treat any side effects. Read more details below about recovering from a bone marrow transplant.

Step 1: Donor identification. A matched donor must be found before the ALLO transplant process can begin. Your HLA type will be found through blood testing. Then, your health care team will work with you to do HLA testing on potential donors in your family, and if needed, to search a volunteer registry of unrelated donors.

Step 2: Collecting stem cells from your donor. Your health care team will collect cells from either your donors blood or bone marrow. If the cells are coming from the bloodstream, your donor will get daily injections (shots) of a medication to increase white cells in their blood for a few days before the collection. Then, the stem cells are collected from their bloodstream. If the cells are coming from bone marrow, your donor has a procedure called a bone marrow harvest in a hospital's operating room.

Step 3: Pre-transplant treatment. This step takes 5 to 7 days. You will get chemotherapy, with or without radiation therapy, to prepare your body to receive the donor's cells.

Step 4: Getting the donor cells. This step is your transplant day. Your health care team puts, or infuses, the donors stem cells into your bloodstream through the catheter. Getting the donor cells usually takes less than an hour.

Step 5: Recovery. During your initial recovery, you will get antibiotics to reduce your risk of infection and other drugs, including medications to prevent and/or manage GVHD. Your health care team will also treat any side effects from the transplant. Read more details below about bone marrow transplant recovery.

Recovery from a bone marrow/stem cell transplant takes a long time. Recovery often has stages, starting with intensive medical monitoring after your transplant day. As your long-term recovery moves forward, you will eventually transition to a schedule of regular medical checkups over the coming months and years.

During the initial recovery period, it's important to watch for signs of infection. The intensive chemotherapy treatments that you get before your transplant also damage your immune system. This is so your body can accept the transplant without attacking the stem cells. It takes time for your immune system to work again after the transplant. This means that you are more likely to get an infection right after your transplant.

To reduce your risk of infection, you will get antibiotics and other medications. If you had an ALLO transplant, your medications will include drugs to prevent and/or manage GVHD. Follow your health care team's recommendations for how to prevent infection immediately after your transplant.

It is common to develop an infection after a bone marrow transplant, even if you are very careful. Your doctor will monitor you closely for signs of an infection. You will have regular blood tests and other tests to see how your body and immune system are responding to the donor cells. You may also get blood transfusions through your catheter.

Your health care team will also develop a long-term recovery plan to monitor for late side effects, which can happen many months after your transplant. Learn more about the possible side effects from a bone marrow transplant.

Your doctor will recommend the best transplant option for you. Your options depend on the specific disease diagnosed, how healthy your bone marrow is, your age, and your general health. For example, if you have cancer or another disease in your bone marrow, you will probably have an ALLO transplant because the replacement stem cells need to come from a healthy donor.

Before your transplant, you might need to travel to a center that does many stem cell transplants. Your doctor may need to go, too. At the center, you will talk with a transplant specialist and have a medical examination and different tests.

A transplant will require a lot of time receiving medical care away from your daily life. It is best to have a family caregiver with you. And, a transplant is an expensive medical process. Talk about these questions with your health care team and your loved ones:

Can you describe the role of my family caregiver in taking care of me?

How long will I and my caregiver be away from work and family responsibilities?

Will I need to stay in the hospital? If so, when and how long?

Will my insurance pay for this transplant? What is my coverage for my follow-up care?

How long will I need medical tests during my recovery?

A successful transplant may mean different things to you, your family, and your health care team. Here are 2 ways to know if your transplant worked well.

Your blood counts are back to safe levels. A blood count measures the levels of red blood cells, white blood cells, and platelets in your blood. At first, the transplant makes these numbers very low for 1 to 2 weeks. This affects your immune system and puts you at a risk for infections, bleeding, and tiredness. Your health care team will lower these risks by giving your blood and platelet transfusions. You will also take antibiotics to help prevent infections.

When the new stem cells multiply, they make more blood cells. Then your blood counts will go back up. This is one way to know if a transplant was a success.

Your cancer is controlled. Curing your cancer is often the goal of a bone marrow/stem cell transplant. A cure may be possible for certain cancers, such as some types of leukemia and lymphoma. For other diseases, remission of the cancer is the best possible result. Remission is having no signs or symptoms of cancer.

As discussed above, you need to see your doctor and have tests regularly after a transplant. This is to watch for any signs of cancer or complications from the transplant, as well as to provide care for any side effects you experience. This follow-up care is an important part of your recovery.

It is important to talk often with your health care team before, during, and after a transplant. You are encouraged to gather information, ask questions, and work closely with your health care team on decisions about your treatment and care. In addition to the list above, here are some possible questions to ask. Be sure to ask any question that is on your mind.

What type of transplant would you recommend? Why?

If I have an ALLO transplant, how will we find a donor? What is the chance of finding a good match?

What type of treatment will I have before the transplant?

How long will my pre-transplant treatment take? Where will this treatment be given?

Can you describe what my transplant day will be like?

How will a transplant affect my life? Can I work, exercise, and do regular activities?

What side effects could happen during treatment, or just after?

What side effects could happen years later?

What tests will I need after the transplant? How often?

Who can I talk to if I am worried about the cost?

How will we know if the transplant worked?

What if the transplant does not work? What if the cancer comes back?

Side Effects of a Bone Marrow Transplant (Stem Cell Transplant)

Bone Marrow Aspiration and Biopsy

Donating Bone Marrow is Easy and Important: Here's Why

Bone Marrow Transplants and Older Adults: 3 Important Questions

Why the Bone Marrow Registry Needs More Diverse Donors and How to Sign Up

Be the Match: About Transplant

Be the Match: National Marrow Donor Program

Blood & Marrow Transplant Information Network (BMT InfoNet)

National Bone Marrow Transplant Link (nbmtLINK)

U.S. Department of Health and Human Services: Learn About Transplant as a Treatment Option

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What is a Bone Marrow Transplant (Stem Cell Transplant)? - Cancer.Net

Cameron Mathison Is Undergoing Stem Cell Treatments - Soaps In Depth

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Cameron Mathison Is Undergoing Stem Cell Treatments

A federal judge in Riverside declared a California stem cell treatment firm to be exempt from Food and Drug Administration regulations, opening the door to the further proliferation of clinics offering therapies the FDA says are scientifically unproven and potentially dangerous.

In the ruling issued late Tuesday, Judge Jesus G. Bernal of Riverside declined to block California Stem Cell Treatment Center from continuing to offer purported stem cell treatments to customers.

Bernal accepted the centers position that its treatments qualified for an exception from FDA regulations, in part because they were tantamount to surgical procedures.

Stem cells have been called everything from cure-alls to miracle treatments. But dont believe the hype.

Food and Drug Administration warns public against unproven stem cell treatments

Bernals ruling, which came more than a year after a seven-day trial in May 2021 and closing arguments last August, potentially undermines a years-long FDA crackdown on clinics claiming that stem cells can treat or cure conditions including orthopedic injuries, Alzheimers and Parkinsons diseases, multiple sclerosis, and erectile dysfunction.

The defendants are engaged in the practice of medicine, Bernal ruled, not the manufacture of pharmaceuticals.

The FDA regulations, however, define drugs much more broadly than the manufacture of pharmaceuticals rather, as any article that is intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease. Any such articles, the agency says, must have agency approval and the California centers treatments do not.

To me, the language of his ruling sounded almost like it was written by the defendants, Paul S. Knoepfler, a UC Davis stem cell biologist who has been tracking the proliferation of such clinics for years, observed in his laboratory blog.

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The Bernal ruling will not be a good thing for careful oversight of stem cell clinic practices, Knoepfler said. Its concerning to think about more people being put at risk.

Knoepfler suggested that the FDA would feel duty-bound to appeal Bernals ruling in order to protect its regulatory campaign, and because it conflicts with federal court rulings in a nearly identical case the FDA brought against Florida clinics in Miami federal court.

The agency told me by email that it is reviewing the courts decision and does not have further comment at this time.

Bioethicist Leigh Turner of UC Irvine, who has collaborated with Knoepfler on tracking the growth of the stem cell clinic industry, agreed that Bernals ruling is an enormous setback for the FDA in an area where theyve struggled for many years.... For people who work in this industry, its great news. But not for anyone concerned about patient safety, misinformation or disinformation.

As part of its crackdown, the FDA has written hundreds of letters warning stem cell clinics that theyre violating the law, and has pursued some in court.

After issuing regulations in 2017 declaring that treatments using unproven stem cell therapies were illegal, the FDA suspended its enforcement for more than three years to give clinic operators time to comply with FDA rules. The agencys forbearance, however, opened the door to a further proliferation of suspect clinics.

By March 2021, according to a survey by Turner, nearly 1,500 U.S. businesses were pitching the suspect treatments at more than 2,700 clinics, reflecting a torrent of openings since 2016, when Turner and Knoepfler jointly started tracking the field.

More than four times as many businesses than were identified five years ago are selling stem cell products that are not FDA-approved and lack convincing evidence of safety and efficacy, Turner wrote last year.

Some clinics have charged customers more than $10,000 for the unproven treatments, plying the customers with unsupported claims of medical success. The fees are seldom, if ever, covered by health insurance. Some treatments resulted in serious medical complications.

The FDA also has warned the public that some patients seeking cures and remedies are vulnerable to stem cell treatments that are illegal and potentially harmful.

The FDA has approved stem cell treatments only for disorders of the blood-producing, or hematopoietic, system. No other stem cell treatment claims have been scientifically validated, the agency says.

Stem cells have been called everything from cure-alls to miracle treatments, the agency says in its public warning. But dont believe the hype.

Bernals ruling conflicts with a 2019 decision by U.S. District Judge Ursula Ungaro of Miami, who ordered a Florida clinic shut down after the FDA asserted that its purported stem cell therapies were scientifically unproven and illegal.

Ungaros decision was upheld last year by the 11th Circuit U.S. Court of Appeals, which found that the clinics claim of exemption from FDA regulation on grounds similar to those claimed by the California center, didnt apply. No reasonable fact-finder could disagree, the three-judge appellate panel ruled.

As it happens, in its finding the appellate panel specifically rejected an earlier ruling by Bernal, in which he denied the FDAs request for a preliminary injunction against the California center and set the case for trial. The appellate ruling isnt binding precedent for federal courts outside the 11th Circuit, which covers parts of the Southeast.

The FDAs lawsuits against the Florida clinic and the California center were almost identical and filed on the same day, May 9, 2018. In both cases, the agency asserted that the clinics were in effect purveying illegal drugs as defined by federal law.

The lawsuits were part of an agency effort to clamp down on the burgeoning trade in stem cell-related therapies. The California lawsuit named the centers founders, Elliott Lander and Mark Berman, as defendants. Berman died in April

The California Stem Cell Treatment Center has operated clinics in Beverly Hills and Rancho Mirage, and provides treatment protocols and other assistance to other clinics around the country.

In its Florida and California lawsuits, the FDA targeted a widespread practice utilized by both clinics in which fat cells are extracted from a customer by liposuction. The extraction is treated to produce a fluid purportedly rich in stem cells known as a stromal vascular fraction or SVF, which is injected back into the same subject.

Both sets of defendants asserted in their defense that their activities qualified for exceptions from FDA drug regulations afforded to surgical procedures involving the use of a patients own tissues as well as the use of minimally manipulated tissues. The agencys position is that the fat extractions are so heavily treated before the reinjections that the treatments fall outside those exceptions.

In his ruling, Bernal drew extensively and verbatim from California Stem Cell Treatment Centers proposed findings of facts and scarcely at all from the FDAs proposal. His ruling incorporated several scientific errors, according to Knoepfler.

For example, Bernal accepted the centers assertion that unlike manufactured drugs, the SVF Surgical Procedure does not create any cellular or tissue-based product that did not previously exist within the patient.

In fact, Knoepler says, there is no equivalent of SVF already in the body.

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Hiltzik: A judge undermines the FDA on stem cells - Los Angeles Times

NEW YORK, Oct. 02, 2022 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, announced today that it has submitted to the U.S. Food and Drug Administration (FDA) substantial new information on clinical and potency assay items identified in the Complete Response Letter (CRL) received from FDA in September 2020 to the Biologics License Application (BLA) for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD).

Mesoblast has maintained an active dialog with the FDA since receiving the CRL, and the substantial new information submitted to the Investigational New Drug (IND) file for remestemcel-L in the treatment of children with SR-aGVHD, as guided by FDA, represents a major milestone in the Companys complete response to the FDA. Remestemcel-L has been granted Fast Track Designation and BLA Priority Review from the FDA.

Survival outcomes have not improved over the past two decades for children or adults with the most severe forms of SR-aGVHD.1-3 The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children.

The submission summarizes controlled data providing further evidence of remestemcel-Ls ability to save lives, said Dr. Silviu Itescu, Chief Executive of Mesoblast. Additionally, the improved process controls we have put in place to assure robust and consistent commercial product, together with a potency assay that predicts consistent survival outcomes, makes remestemcel-L a compelling treatment for these children.

About Steroid-refractory Acute Graft Versus Host Disease Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, including about 20% in pediatric patients.4,5 SR-aGVHD is associated with mortality as high as 90% and significant extended hospital stay costs.6,7 There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Mesoblast Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio of late-stage product candidates which respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. The Companys proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, biologic-resistant inflammatory bowel disease, and acute respiratory distress syndrome. Rexlemestrocel-L is in development for advanced chronic heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

References / Footnotes

Forward-Looking StatementsThis press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblasts preclinical and clinical studies, and Mesoblasts research and development programs; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblasts ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals (including BLA resubmission), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblasts product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblasts product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblasts ability to enter into and maintain established strategic collaborations; Mesoblasts ability to establish and maintain intellectual property on its product candidates and Mesoblasts ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblasts expenses, future revenues, capital requirements and its needs for additional financing; Mesoblasts financial performance; developments relating to Mesoblasts competitors and industry; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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Mesoblast Submits New Information to FDA IND File in Response to Items in the CRL to the Remestemcel-L BLA for SR-aGVHD - GlobeNewswire

Newswise LOS ANGELES (Oct. 3, 2022) --Pioneering heart care is a tradition at Cedars-Sinai. Its a tradition that took root in 1924, when Cedars-Sinai became home to the first electrocardiogram machine in Los Angeles.

The roots grew stronger in the 1970s, when two Cedars-Sinai cardiologists invented the game-changing Swan-Ganz catheter. And stronger still in 2007, when Cedars-Sinai founded what is today theSmidt Heart Institute.

On the occasion of the institutes 15thanniversary, the Cedars-Sinai Newsroom sat down withEduardo Marbn, MD, PhD, executivedirector of theSmidt Heart Institute and the Mark Siegel Family Foundation Distinguished Professor, to get his views on the state of cardiac medicine.

Newsroom: What are the advances youve seen in heart medicine over the past 15 years?Marbn:We've been able to really address programs from the point of view of the need of the patient. One thing we've excelled at, for example, is helping our patients with advanced heart diseaseheart failure so severe that they're facing either highly experimental stem cell treatments or heart transplantation. And these are two areas in which we've excelled, become the world leader in the number and also in the outcomes of heart transplants for those patients who need them. And we've also made quite a lot of progress in the area of regenerative medicine.

When the Heart Institute was established 15 years ago, almost all valvular problems in the heart or major structural problems required opening the chest surgically. Putting a patient on cardiopulmonary bypass. Fixing the problem in an operating room. And then keeping the patient in the hospital for about a week. In the past 15 years, the management of valvular heart disease has been completely transformed so that things that used to take that long and were that complicated can now be done as overnight therapy, using just a catheter inside the heart, and no opening of the chest.

We've led the way in the development of these technologies. We've done more such procedures than any other center in the United States.

Newsroom: How much has the Smidt Heart Institute grown?Marbn:Over the last 15 years, the Heart Institute has seen an incredible increase in volume. We have gone from doing 25 thoracic transplants a year, meaning either lung or heart or both, to doing over 200 now, with outstanding outcomes. We have gone from doing about 3,000 catheter-based procedures a year, to 13,000 a year.

We have gone up in our U.S. News and World Report (Best Hospitals) ranking nationally, from #17 Heart Program and #5 in the western U.S. to now being #1 in the western U.S. for the past 10 years and to being the third-ranked program nationally for the last four years.

Newsroom: Also during that time, theBarbra Streisand Womens Heart Centerin the Smidt Heart Institute has pioneered a lot of work on sex differences in heart disease.Marbn:The Smidt Heart Institute has come to be known as the world leader in a number of areas. One of those is in women's heart disease. In the last 15 years, we've really become recognized as a go-to destination for teaching, research and training. We've trained generations of researchers and physicians who are going into this field, and we've discovered that women are not the same as men and the way they manifest their heart disease. Nor are the treatments necessarily likely to work equally in men and in women.

Newsroom: What lessons were learned from the COVID-19 pandemic?Marbn:When the pandemic of COVID-19 started early in 2020, we were among the first to codify the cardiac complications that are associated with the disease. For example, myocarditis and other forms of acute manifestations and the long-running complications that have to do with long COVID. We were among the first to describe these situations. And since then, we've been at the forefront of studying the cardiac complications of COVID-19 in communities.

For example, we have a large ongoing study looking at a large number of community-based participants that then develop COVID-19 and some of whom go on to develop heart disease. We're asking questions like, how does the heart disease manifest itself? Who's at highest risk? What can we do about it? And of course, we're in the data collection stage more than in the answer formulation stage, but we've really tried to get ahead of it rather than just being observers.

Newsroom: What kind of progress do you expect in the next 15 years?Marbn:It's mind boggling to imagine what might happen over the next 15. But for sure, among the trends that we've seen there will continue miniaturization. We believe that it's very likely that major equipment that is now required, for example, to sustain the heart artificiallyluggage sized pumps that need to be plugged in periodically to batteriesfor example, as an external pump for the heart. These things will be miniaturized and implanted.

We're going to see an enormous rise in the maturation and ability of biological therapies, gene-based and cell-based therapies, to address heart disease in a very fundamental way that actually changes the disease rather than just reacts to it. And of course, we're getting better at prevention and also the understanding of how disease is manifested differently in men and in women, as well as in various socioeconomic groups.

Newsroom: Is there a single achievement of which you are most proud?Marbn:The signature accomplishment of the Smidt Heart Institute has been proof of principle that by bringing together diverse specialists from different backgrounds into one setting, that we can improve patient care and our understanding of disease. What we've done here is created a model for how cardiology and cardiac surgery should work together to transform the care and the knowledge about illness as we move forward.

Read more on the Cedars-Sinai Blog:Matters of the Heart: C. Noel Bairey Merz, MD

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15 Years of Heart - Newswise