Category Archives: Stem Cell Treatments

The FDA has granted approval for Kites Tecartus (brexucabtagene autoleucel) for the treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Tecartus is the first and only chimeric antigen receptor (CAR) T-cell therapy approved for adults (18 years and older) with ALL. The drug had received FDA Breakthrough Therapy Designation and a priority review for this indication.

In July 2020, the FDA approved Tecartus for treatment of adult patients diagnosed with mantle cell lymphoma (MCL) who have not responded to or who have relapsed following other kinds of treatment. Tecartus was the first cell-based gene therapy approved by the FDA for the treatment of MCL.

Tecartus has already transformed outcomes for adults living with mantle cell lymphoma, and we look forward to offering the hope for a cure to patients with ALL, said Christi Shaw, Chief Executive Officer of Kite, which is a Gilead company.

The drugs current approval for ALL is based on results from ZUMA-3, a global, multicenter, single-arm, open-label study in which 65% of the evaluable patients (n=54) achieved complete remission (CR) or CR with incomplete hematological recovery (CRi) at a median actual follow-up of 12.3 months. The duration of CR was estimated to exceed 12 months for more than half the patients. Among efficacy-evaluable patients, median duration of remission (DOR) was 13.6 months.

There were side effects. Among the patients treated with Tecartus at the target dose (n=78), Grade 3 or higher cytokine release syndrome (CRS) and neurologic events occurred in 26% and 35% of patients, respectively. Kite reports these side effects were generally well-managed.

Median overall survival (OS) from ALL is only approximately eight months with current standard-of-care treatments

Adults with ALL face a significantly poorer prognosis compared to children, and roughly half of all adults with B-ALL will relapse on currently available therapies, said Bijal Shah, MD, ZUMA-3 investigator and medical oncologist, Moffitt Cancer Center, Tampa, Florida. We now have a new meaningful advancement in treatment for these patients. A single infusion of Tecartus has demonstrated durable responses, suggesting the potential for long-term remission and a new approach to care.

Adults with relapsed or refractory ALL often undergo multiple treatments including chemotherapy, targeted therapy, and stem cell transplant. Instead, CAR T-cell therapy works by harnessing a patients own immune system to fight cancer. With CAR T, the patients blood is drawn and the T cells are separated. Then the T cells are genetically engineered with a specific receptor that enables them to identify and attack cancer cells, and put back into the patients body.

Roughly half of all ALL cases actually occur in adults, and unlike pediatric ALL, adult ALL has historically had a poor prognosis, said Lee Greenberger, PhD, Chief Scientific Officer of The Leukemia & Lymphoma Society (LLS). Developing new therapies that would be life-changing for people with cancer has been a dream of LLS. We are proud to see the potential of CAR T realized for even more people with this approval for brexucabtagene autoleucel. Patients can accessTecartus through 109 authorized treatment centers across the US.

Tecartus is also currently under review in the European Union and United Kingdom for the treatment of adult patients with relapsed or refractory B-cell precursor ALL.

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Kite's Tecartus is First CAR T Therapy Approved for Adults with B-Cell ALL - Clinical OMICs News

Prolymphocytic leukemia (PLL) is a very rare subtype of chronic leukemia. Although most forms of chronic leukemia progress slowly, PPL is often aggressive and can be difficult to treat.

Well walk you through what you need to know about PLL, including the symptoms, how its diagnosed, current treatment options, and more.

PLL is a rare and aggressive type of chronic leukemia.

The American Cancer Society estimates that more than 60,000 people will receive a diagnosis of leukemia in the United States in 2021.

Less than 1 percent of all people with chronic leukemia have PLL. Its most often diagnosed in people between ages 65 and 70 and is slightly more common in men than in women.

Like all types of leukemia, PLL affects blood cells. PLL is caused by the overgrowth of cells called lymphocytes. These cells usually help your body fight infection. In PLL, large immature lymphocyte cells called prolymphocytes are produced too quickly and overwhelm the other blood cells.

There are two subtypes of PLL:

PLL, like other chronic leukemias, is often found on lab work before any symptoms develop. When symptoms develop, they might include:

There are a few additional symptoms that are specific to T-PLL, which include:

Many of these are general leukemia symptoms and are also found in less serious conditions. The presence of any of these symptoms doesnt always indicate PLL.

In fact, since PLL is rare, its unlikely that its causing your symptoms.

However, its a good idea to see a healthcare professional if youve been experiencing any of these symptoms for more than a week or two.

Because PLL is very rare, it can be hard to diagnose. PLL sometimes develops from existing chronic lymphocytic leukemia (CLL) and is found during lab work when monitoring CLL.

PLL is diagnosed when more than 55 percent of the lymphocytes in your blood sample are prolymphocytes. Blood work can also be checked for antibodies and antigens that can signal PLL.

If PLL isnt found during routine blood work, a healthcare professional will order more tests if you have symptoms that might indicate PLL. These tests may include:

Currently, theres no one specific treatment for either type of PLL. Your treatment will depend on how fast your PLL progresses, the type you have, your age, and your symptoms.

Since PLL is rare, your doctor will likely come up with a treatment plan specific to your case. Healthcare professionals may often encourage people with PLL to sign up for clinical trials to try new medications.

Treatments you might receive for PLL include:

PLL is an aggressive form of chronic leukemia. Therefore, the outlook is generally poor due to how quickly it may spread. But outcomes and survival rates can vary greatly between people.

As mentioned earlier, one potential cure for PLL is a stem cell transplant, although not all people with PLL are eligible to receive stem cell transplants.

Newer treatments have improved survival rates in recent years, and research into new therapies is ongoing.

PLL is a rare type of chronic leukemia. Its most commonly diagnosed in people between 65 and 70 years old. It often progresses more quickly and is treatment-resistant than other forms of chronic leukemia.

Treatment options depend on your overall health, age, symptoms, and the type of PLL you have. People are often encouraged to take part in clinical trials to take advantage of new therapies.

Originally posted here:
Prolymphocytic Leukemia: What Is It and How Is It Treated? - Healthline

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About 250 miles above sea level, a unique laboratory circles the planet. Here in low-Earth orbit on the International Space Station (ISS), gravitys tug is faint enough that objects appear to be weightless. These microgravity conditions cause living things from people to organs and cells to function differently than on terra firma. Researchers from UPMC and the University of Pittsburgh aim to use the ISS U.S. National Laboratory (ISSNL) to conduct biomedical research not possible on Earth and advance space-based biomanufacturing.

In recent years, microgravity research has become more accessible through the ISSNL. At the same time, breakthroughs in gene editing, stem cell research and development of mini lab-grown organs have helped advance regenerative medicine, the discipline that focuses on replacing or repairing damaged or diseased body parts. Progress in these two areas means that now is the right moment to consider the future of biomanufacturing in space, according to Dr. William Wagner, director of the University of Pittsburghs McGowan Institute for Regenerative Medicine and distinguished professor of surgery, chemical engineering and bioengineering at Pitt.

Dr. William Wagner and Gary Rodrigue discuss the future of biomanufacturing in space.

We are truly blessed to be at a point in humankind where access to low-Earth orbit and access to microgravity is available, Wagner said during a fireside chat with Gary Rodrigue, director of programs and partnerships at the Center for the Advancement of Science in Space (CASIS), at the 10th annual International Space Station Research and Development Conference. The question is, how are we going to leverage the unique aspects of this environment?

To help answer this question, the McGowan Institute and CASIS, the organization that manages the ISSNL, brought together more than 100 experts in regenerative medicine, tissue engineering and aerospace for the Biomanufacturing in Space symposium last year. Originally planned as an in-person event, the symposium pivoted to a series of virtual meetings that took place weekly over several months.

In a review article recently posted to Preprints, symposium attendees identified areas that hold the greatest promise for space-based biomanufacturing for tissue engineering and regenerative medicine.

Its an incredible privilege to try to answer the question of what makes sense to do in microgravity, said Wagner. Its a similar feeling to being an explorer on a ship and heading to some uncharted land. I think many people who were on those calls shared that sense of exploration.

The researchers identified organs-on-a-chip miniature devices that mimic human organs as one important subject for future research in space. Because disease-like characteristics develop in these systems under microgravity, insight into disease pathways and development of drugs that block these pathways can be accelerated in low-Earth orbit.

Stem cell research is another area that could be advanced in microgravity, according to the article. Stem cells, which can be turned into any type of cell, have many potential applications for treating diseases, injuries and birth defects. Because these cells behave differently in low gravity, research on the ISS could improve stem cellbased treatments and enhance fundamental understanding of processes such as aging and organ development.

The third target area that the researchers identified is 3D printing of tissues for transplantation and disease modeling. The near absence of gravity on the ISS could enable more consistent deposition of complex molecules and reduce the need for structural support, which have been hurdles in the production of these 3D tissues.

The researchers also outline commercial opportunities and potential business models for developing a sustainable market for space-based biomanufacturing. According to Wagner, these considerations are important for ensuring that new therapies make it to patients.

We cant just run experiments; we cant just publish papers. We have to think about how it is going to get to the patient, said Wagner. If we dont get it to the patient, we always say that we havent succeeded in our mission.

Originally posted here:
Researchers Gaze into Space to Envision Future of Regenerative Medicine - UPMC & Pitt Health Sciences News Blog - UPMC

The umbrella term of leukemia encompasses several distinct types of leukemia, including acute myeloid leukemia (AML).

In 2021, its estimated that over 20,000 new cases of AML will be diagnosed, according to the National Cancer Institute (NCI). Since treatment varies depending on the specific kind of leukemia present, an accurate diagnosis is crucial.

There are a variety of treatments for AML. Your doctor will explain them and help choose a treatment plan based on the type of cancer you have and your individual situation.

Read on to learn more about the various treatment options for AML.

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It affects white blood cells (WBCs), making them abnormal. In some forms of AML, they may also multiply very quickly.

Other names for AML include:

Read this for more information about AML.

Once the diagnosis is confirmed, your healthcare team will develop a plan to treat AML. Depending on the specific type and stage of AML, you may receive one or more of these treatments:

Chemotherapy is the main form of treatment for AML. Its divided into two phases:

Since AML can progress quickly, treatment is usually started as soon as possible after diagnosis. Other treatments may be used as well.

Chemotherapy, also called chemo, is the use of anti-cancer drugs to treat cancer. This is the main treatment for AML.

These drugs can be injected into a vein or under the skin, allowing the chemotherapy to travel through the bloodstream to attack cancer cells throughout the body. If leukemia has been found in the brain or spinal cord, chemo medication may be injected into the cerebrospinal fluid (CSF).

Chemo medications most often used to treat AML include:

Other chemo medications may include:

Side effects of chemotherapy can vary depending on the drug, dosage, and duration. They can include:

While chemotherapy is the main treatment for AML, for a subtype of AML called acute promyelocytic leukemia (APL), other non-chemotherapy drugs are more effective.

APL is caused by a specific gene mutation that affects WBCs. Some medications work better than chemo to help those cells develop normally. Two of these medications are:

ATRA can be given with chemotherapy or with ATO for the initial treatment of APL. Both drugs can also be given during consolidation.

Side effects of ATRA include:

Side effects of ATO can include:

Radiation therapy uses high-energy radiation to kill cancer cells. While its not the main treatment for those with AML, it can be used in treating AML. In AML, the radiation used is external beam radiation, which is similar to an X-ray.

Radiation can be used in AML to treat:

Side effects of radiation can include:

Surgery is rarely used in AML treatment. Leukemia cells are spread through the bone marrow and blood, making the condition impossible to improve with surgery. On rare occasions, a tumor or mass related to leukemia may form that may be treated with surgery.

Prior to chemotherapy, a small surgery to place a central venous catheter (CVC) or a central line, is often done. During this procedure, a small flexible tube is placed into a large vein in the chest. The end of it is either right under the skin or sticks out in the chest or upper arm.

Having a central line installed allows the treatment team to give intravenous medication and chemotherapy through the CVC, and to draw blood from it, reducing the number of needle sticks an individual has to have.

While chemotherapy is the main treatment for AML, it has its limits. Since high doses of these medications are toxic, the dosage must be limited. A stem cell transplant allows for higher doses of chemotherapy medications.

In a stem cell transplant, very high doses of chemotherapy medications, sometimes combined with radiation, are given. All of the individuals original bone marrow is destroyed on purpose.

Once this stage of therapy is over, blood-forming stem cells are given. These stem cells will grow, rebuilding the bone marrow. Healthy, cancer-free stem cells replace the destroyed bone marrow.

Read this article for more information about a stem cell transplant.

Targeted therapy drugs are medications that target only certain parts of cancer cells. They can be very effective for some people with AML. Most targeted therapy drugs are taken orally, except for gemtuzumab ozogamicin (Mylotarg), which is given as an intravenous infusion.

Talk with your treatment team about the potential side effects of each drug and what you should watch for when taking it. Some targeted therapy medications include:

One type of targeted therapy medication called FLT3 inhibitors targets the FLT3 gene. In some people with AML, a mutation in the FLT3 gene causes the creation of a protein, also called FLT3, that enables cancer cells to grow. Drugs in this category include:

Side effects of these drugs may include:

In some people with AML, there is a mutation in the IDH2 gene. These mutations stop bone marrow cells from maturing in a normal way. Medications called IDH inhibitors block IDH proteins produced by these mutated genes, allowing these bone marrow cells to grow normally and remain healthy.

Drugs in this category include:

Side effects can include:

AML cells contain a protein called CD33. A medication called gemtuzumab ozogamicin (Mylotarg) attaches to this CD33 protein and helps deliver chemotherapy medications directly to cancer cells so that these drugs are more effective.

Common side effects include:

There are less common but serious side effects like:

Venetoclax (Venclexta) is a BCL-2 inhibitor. This drug targets BCL-2, which is a protein that helps cancer cells live longer. The drug stops the BCL-2 protein from helping cancer cells survive so that these cancer cells die sooner. This medication can be used along with other chemotherapy drugs.

Side effects include:

AML can cause cellular mutations that prevent cells like bone marrow cells from developing and functioning normally. These mutations may affect the pathway cells use to send necessary signals. This pathway is called hedgehog. For some people with AML, especially those over age 75, strong chemo medications may be so harmful that chemo is not an option. For these individuals, a medication called, Glasdegib (Daurismo), may help them live longer. This medication helps stop the mutations and allows bone marrow cells to function normally.

Side effects of this medication may include:

Refractory AML happens when an individual is not in remission even after one to two cycles of induction chemotherapy, which means they have a blast count of 5 percent or more. Ten to 40 percent of people with AML have refractory AML.

If treatment isnt successful with one course of chemo, another one may be done. If a person is still not in remission after the second course of chemo, they may be given other medications or an increased dose of their current chemotherapy medications.

Other treatment options include stem cell transplant or a clinical trial of new therapies.

When an individual has no evidence of disease after treatment, its called remission or complete remission. Remission means these three criteria are met:

If there is no evidence at all of leukemia cells in the bone marrow, using highly sensitive tests, its called complete molecular remission. Minimal residual disease (MRD) occurs when, after treatment, leukemia cells cannot be seen in the bone marrow with standard tests but more sensitive tests like PCR tests do find leukemia cells.

Even after an individual has entered remission, they will likely need follow-up care and will need to be monitored by their doctor and healthcare team. This may mean additional tests, more frequent physical exams, and other care.

Although chemotherapy is the main treatment for AML, there are a variety of treatment options, depending on the AML subtype or whether you have a specific mutation. Treatment also depends on your response to initial treatment and whether or not remission is sustained.

Your treatment team will explain all of your treatment options and help you choose the treatment plan that is best for you and your individual situation.

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Acute Myeloid Leukemia Treatment: What You Need to Know - Healthline

When utilizing a validated antibody assay against the SARS-CoV-2 spike protein, investigators revealed a high seroconversion rate of 94% among 200 patients with cancer in New York City who had received a full dose of 1 of the FDA-authorized COVID-19 vaccines.

Patients with solid tumors experienced an impressive seroconversion rate of 98% compared with a rate of 85% in those with hematologic malignancies, 70% in those who had received highly immunosuppressive therapies like anti-CD20 agents, and 73% in those who had previously undergone stem cell transplantation. Notably, patients who received treatment with immune checkpoint inhibitors or hormonal therapies experienced seroconversion rates of 97% and 100%, respectively, following vaccination.

We saw very encouraging [data] showing that most patients with a cancer diagnosis have a really high chance of responding to vaccinationsas long as the vaccinations are done in an appropriate manner, [with] both doses administered, Balazs Halmos, MD, MS, study author, director of Thoracic Oncology, and director of Clinical Cancer Genomics at Montefiore Medical Center, told OncLive in an exclusive interview on the research. This was [true] even for [patients who were receiving] active treatment with chemotherapy, targeted therapy, or immunotherapy.

Halmos and colleagues launched this study to develop a better understanding with regard to the immunogenicity of vaccines in a group of patients with a cancer diagnosis in New York City by examining the rates of anti-spike immunoglobulin G (IgG) antibody positivity after receiving 1 of the 3 authorized COVID-19 vaccines.

A total of 213 patients were enrolled to the study through an informed-consent process. Twenty-nine additional patients with cancer who received the SARS-CoV-2 spike IgG testing were identified through retrospective chart review.

A total of 18 patients did not have this test conducted following consent, and thus, they were excluded from the analysis. Twenty additional patients were excluded because they had their test done before having received full vaccination in accordance with FDA guidance. Four additional patients were excluded for other reasons.

As such, 233 patients with cancer were noted to have received all required doses of their COVID-19 vaccine; all these patients were included in the safety analysis. A subset of 200 patients received the IgG test and were included in the immunogenicity analysis. Serological information from these patients were utilized in association studies between cancer subtypes and therapies.

Investigators also examined the link between the quantitative titer of SARS-CoV-2 spike IgG and cancer subtypes and therapies. If the 200 patients, 185 had available IgG titers that were at least 2 days following the last vaccine dose. A total of 15 patients were excluded from the vaccination cohort with titers; these patients had received the vaccine, but titers were checked less than 1 week from their last dose.

Among those included in the efficacy analysis (n = 200), the median age was 67 years (range, 27-90), 58% were female, and 42% were male. The study population was noted to be representative of the diverse population that resides in the Bronx, New York, with 32% of patients identifying as African American, 39% as Hispanic, 22% as Caucasian, 5% as Asian, and 3% as other ethnicities.

Additionally, 67% of patients had a solid tumor diagnosis and 33% had a hematologic malignancy. Among those with solid tumors, 26% had breast cancer, 14% had gastrointestinal cancer, 9% had genitourinary cancer, 5% had gynecologic cancer, 13% had thoracic or head and neck cancer, 1% had skin or musculoskeletal cancer, and 1% had carcinoma of an unknown primary. Among those with hematologic malignancies, 13% had lymphoid disease, 9% had myeloid disease, and 11% had plasma cell disease.

Seventy-five percent of patients had an active malignancy and 67% were receiving active treatment at the time that they received the COVID-19 vaccine. Fifty-six percent of patients were on active chemotherapy. Moreover, 19% of patients were on active chemotherapy within 48 hours of receiving at least 1 of their COVID-19 vaccine doses.

Fifty-four percent of patients completed vaccination with the Pfizer vaccine, 31% with the Moderna vaccine, and 10% with the Johnson & Johnson vaccine. A total of 3 patients had received a complete mRNA vaccination series but the information regarding the type of vaccine (Pfizer vs Moderna) are not yet available.

Additional findings from the study showed that significantly higher titer values were observed in solid tumors vs hematologic malignancies among a subgroup of 185 patients with available IgG titers longer than 7 days post vaccination, at a median of 7858 AU/mL vs a median of 2528 AU/mL, respectively (P = .013).

When comparing patients who were receiving active cancer treatment vs those who were not, no significant differences in seroconversion were reported, at 96% and 93%, respectively. However, investigators did report lower seropositivity rates in those who were on active cytotoxic chemotherapy versus other treatments, at 92% vs 99%, respectively (P = .04). Moreover, significantly lower seroconversion rates were also noted in those who received immunosuppressive therapies like stem cell transplant (73%; P = .0002), CD20 antibody therapy (70%; P = .0001), or CAR T-cell therapy (all seronegative; P = .0002).

Significantly lower titer levels were observed in patients who received CD20 antibody therapy vs the overall patient population, which underscored the susceptibility of patients receiving these treatments during the pandemic.

No statistically significant associations between age, ethnicity, time since immunosuppressive therapy, steroid use, or treatment within 48 hours of a vaccine dose, and seropositivity were reported.

Although all patients who were receiving CDK4/6 inhibitor treatment demonstrated positive anti-spike IgG test results, notably antibody titers were noted to be very low in this subset (n = 5), at a median of 1242 AU/mL vs a median of 6887 AU/mL in the overall cohort. Given the known involvement of the CDK4/6 pathway in immune activation, this might be biologically plausible and warrants further studies into the impact of CDK4/6 inhibitors on vaccine efficacy, the study authors noted.

A trend to lower titers were also reported among subsets of patients who received BCL-2 or BTK inhibitors.

Among a subset of 22 patients with cancer who had previously been infected with COVID-19, the seroconversion rate was 95%. Notably, antibody titers in those who had prior infection with the virus were found to be significantly higher than those who did not have a known prior infection, at a median of 46,737 AU/mL and a median of 5296 AU/mL, respectively (P < .001).

Our study, along with other emerging data, strongly highlights the continued need to vaccinate patients with a cancer diagnosis urgently and broadly, as vaccinations are likely to be highly effective, the study authors concluded. On the other hand, our study highlights at-risk cohorts of patients, in particular patients with hematologic malignancies following receipt of immunosuppressive therapies such as stem cell transplantation, anti-CD20 therapies, and CAR T-cell treatments. These cohorts of patients could potentially benefit from passive immunization with anti-COVID antibodies in the face of the ongoing pandemic.

Read more from the original source:
Study Calls for COVID-19 Vaccination in Patients With Cancer to Enable Optimal Treatment Delivery During Pandemic - OncLive

As you may have heard, the Centers for Disease Control and Prevention (CDC)has recommended a third dose of the COVID-19 vaccine for people who are immunocompromised. This includes some but not all people with cancer.

Mini Kamboj

Mini Kamboj, Memorial Sloan Kettering Cancer CentersChief Medical Epidemiologist, has answers to your questions about who is eligible and how you can schedule an appointment to receive your third shot.

For a vaccine to protect you, it must activate your immune system. In some immunocompromised patients, this ability is impaired, so a third dose can boost the immune response.

According to the CDC, among severely immunocompromised people who had undergone solid organ transplant and had virtually no protection after receiving two doses of the Pfizer-BioNTech or Moderna vaccine, 30 to 50% developed antibodies protecting them from COVID-19 after getting an additional dose.

People who have moderate to severe immunosuppression qualify to receive an additional dose, usually because of an organ or stem cell transplant, HIV infection, steroid therapy, or certain cancer treatments that impair the bodys ability to fight infections.

Its important to know that not all cancer patients have a weakened immune system. Those cancer patients who are considered immunocompromised include:

These eligibility criteria cover the most common indications. Your provider will be able to order the third vaccine dose for other immunosuppressive treatments or conditions if they decide that the extra dose will benefit you.

If you meet the criteria, you can receive a third dose 28 days or later after completing your first vaccine series.

Only patients who completed their primary immunization with either Pfizer-BioNTech or Moderna vaccines can receive the third dose. MSK will offer the same vaccine brand to patients as they previously received. Mixing vaccines is not permitted at this time.

The CDC has not made any recommendations yet for people who received the Johnson & Johnson vaccine. We are closely following their guidance and will communicate any changes.

To find out if you should get a third dose, call your MSK doctors office or send a message through the MyMSK patient portal. If you are eligible for an additional vaccine, your doctor will schedule an appointment for you.

On Wednesday, August 18, MSK will begin offering the additional vaccines at the David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center, located at 530 East 74th Street.

Starting Monday, August 23, we will be scheduling appointments at:

These clinics will be open 9:00 a.m. to 5:00 p.m., Monday through Friday.

Additional dates and locations, including our New Jersey locations, will be added shortly.

If you think you meet the criteria for getting a thirdvaccine dose, you should call your providers office to confirm your eligibility, and a vaccine appointment will be scheduled for you. You should be prepared to share your vaccination card or a photo of it. Please present information from your card, rather than the Excelsior pass, which does not have the details about what vaccine brand you received and on what dates.

Yes, the Food and Drug Administration (FDA) hasgranted emergency use authorization for patients 12 and older to receive the Pfizer-BioNTech vaccine and 18 and older to receive the Moderna vaccine.

The side effects from a third COVID-19 vaccine are similar to those experienced after receiving the original vaccines. Scientists in Israel recently began giving a third dose of the Pfizer-BioNTech vaccine to people with compromised immune systems. Side effects were reported by 31% of people, the most common being soreness at the injection site. Other side effects included fatigue, headache, body aches, and fever. These symptoms dont last long about one to three days.

The safety of a third dose in people whove had COVID-19 breakthrough infections is not known, therefore an additional dose for those patients is not recommended at this time. Some patients in whom initial vaccine responses are expected to be severely blunted, such as stem cell transplant orCAR-T recipients or those treated with B-cell depleting therapies, may benefit from a third dose after breakthrough infection. Discuss your situation with your clinical care team.

Even after the third dose, people with weakened immune system must take precautions to protect themselves from COVID-19. You should:

If you develop symptoms of COVID-19, contact your clinical care team and get tested.

Not at this time.The vaccines remain very effective against severe disease for those who do not have compromised immune systems. In the future, third doses may be recommended for more people because immune protection tends to weaken over time. In addition, as new variants of COVID-19 emerge, it may be necessary to design new vaccines to protect against them.

August 16, 2021

See the rest here:
A Third Dose of the COVID-19 Vaccine Recommended for Some Cancer Patients With Weakened Immune Systems - On Cancer - Memorial Sloan Kettering

Stem cells: What they are and what they do

Stem cells and derived products offer great promise for new medical treatments. Learn about stem cell types, current and possible uses, ethical issues, and the state of research and practice.

You've heard about stem cells in the news, and perhaps you've wondered if they might help you or a loved one with a serious disease. You may wonder what stem cells are, how they're being used to treat disease and injury, and why they're the subject of such vigorous debate.

Here are some answers to frequently asked questions about stem cells.

Stem cells: The body's master cells

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Stem cells are the body's master cells. All other cells arise from stem cells, including blood cells, nerve cells and others.

Stem cells are the body's raw materials cells from which all other cells with specialized functions are generated. Under the right conditions in the body or a laboratory, stem cells divide to form more cells called daughter cells.

These daughter cells either become new stem cells (self-renewal) or become specialized cells (differentiation) with a more specific function, such as blood cells, brain cells, heart muscle cells or bone cells. No other cell in the body has the natural ability to generate new cell types.

Researchers and doctors hope stem cell studies can help to:

Generate healthy cells to replace diseased cells (regenerative medicine). Stem cells can be guided into becoming specific cells that can be used to regenerate and repair diseased or damaged tissues in people.

People who might benefit from stem cell therapies include those with spinal cord injuries, type 1 diabetes, Parkinson's disease, amyotrophic lateral sclerosis, Alzheimer's disease, heart disease, stroke, burns, cancer and osteoarthritis.

Stem cells may have the potential to be grown to become new tissue for use in transplant and regenerative medicine. Researchers continue to advance the knowledge on stem cells and their applications in transplant and regenerative medicine.

Test new drugs for safety and effectiveness. Before using investigational drugs in people, researchers can use some types of stem cells to test the drugs for safety and quality. This type of testing will most likely first have a direct impact on drug development first for cardiac toxicity testing.

New areas of study include the effectiveness of using human stem cells that have been programmed into tissue-specific cells to test new drugs. For the testing of new drugs to be accurate, the cells must be programmed to acquire properties of the type of cells targeted by the drug. Techniques to program cells into specific cells continue to be studied.

For instance, nerve cells could be generated to test a new drug for a nerve disease. Tests could show whether the new drug had any effect on the cells and whether the cells were harmed.

Researchers have discovered several sources of stem cells:

Embryonic stem cells. These stem cells come from embryos that are three to five days old. At this stage, an embryo is called a blastocyst and has about 150 cells.

These are pluripotent (ploo-RIP-uh-tunt) stem cells, meaning they can divide into more stem cells or can become any type of cell in the body. This versatility allows embryonic stem cells to be used to regenerate or repair diseased tissue and organs.

Adult stem cells. These stem cells are found in small numbers in most adult tissues, such as bone marrow or fat. Compared with embryonic stem cells, adult stem cells have a more limited ability to give rise to various cells of the body.

Until recently, researchers thought adult stem cells could create only similar types of cells. For instance, researchers thought that stem cells residing in the bone marrow could give rise only to blood cells.

However, emerging evidence suggests that adult stem cells may be able to create various types of cells. For instance, bone marrow stem cells may be able to create bone or heart muscle cells.

This research has led to early-stage clinical trials to test usefulness and safety in people. For example, adult stem cells are currently being tested in people with neurological or heart disease.

Adult cells altered to have properties of embryonic stem cells (induced pluripotent stem cells). Scientists have successfully transformed regular adult cells into stem cells using genetic reprogramming. By altering the genes in the adult cells, researchers can reprogram the cells to act similarly to embryonic stem cells.

This new technique may allow researchers to use reprogrammed cells instead of embryonic stem cells and prevent immune system rejection of the new stem cells. However, scientists don't yet know whether using altered adult cells will cause adverse effects in humans.

Researchers have been able to take regular connective tissue cells and reprogram them to become functional heart cells. In studies, animals with heart failure that were injected with new heart cells experienced improved heart function and survival time.

Perinatal stem cells. Researchers have discovered stem cells in amniotic fluid as well as umbilical cord blood. These stem cells also have the ability to change into specialized cells.

Amniotic fluid fills the sac that surrounds and protects a developing fetus in the uterus. Researchers have identified stem cells in samples of amniotic fluid drawn from pregnant women to test for abnormalities a procedure called amniocentesis.

More study of amniotic fluid stem cells is needed to understand their potential.

Embryonic stem cells are obtained from early-stage embryos a group of cells that forms when a woman's egg is fertilized with a man's sperm in an in vitro fertilization clinic. Because human embryonic stem cells are extracted from human embryos, several questions and issues have been raised about the ethics of embryonic stem cell research.

The National Institutes of Health created guidelines for human stem cell research in 2009. The guidelines define embryonic stem cells and how they may be used in research, and include recommendations for the donation of embryonic stem cells. Also, the guidelines state embryonic stem cells from embryos created by in vitro fertilization can be used only when the embryo is no longer needed.

The embryos being used in embryonic stem cell research come from eggs that were fertilized at in vitro fertilization clinics but never implanted in a woman's uterus. The stem cells are donated with informed consent from donors. The stem cells can live and grow in special solutions in test tubes or petri dishes in laboratories.

Although research into adult stem cells is promising, adult stem cells may not be as versatile and durable as are embryonic stem cells. Adult stem cells may not be able to be manipulated to produce all cell types, which limits how adult stem cells can be used to treat diseases.

Adult stem cells also are more likely to contain abnormalities due to environmental hazards, such as toxins, or from errors acquired by the cells during replication. However, researchers have found that adult stem cells are more adaptable than was first thought.

A stem cell line is a group of cells that all descend from a single original stem cell and are grown in a lab. Cells in a stem cell line keep growing but don't differentiate into specialized cells. Ideally, they remain free of genetic defects and continue to create more stem cells. Clusters of cells can be taken from a stem cell line and frozen for storage or shared with other researchers.

Stem cell therapy, also known as regenerative medicine, promotes the repair response of diseased, dysfunctional or injured tissue using stem cells or their derivatives. It is the next chapter in organ transplantation and uses cells instead of donor organs, which are limited in supply.

Researchers grow stem cells in a lab. These stem cells are manipulated to specialize into specific types of cells, such as heart muscle cells, blood cells or nerve cells.

The specialized cells can then be implanted into a person. For example, if the person has heart disease, the cells could be injected into the heart muscle. The healthy transplanted heart muscle cells could then contribute to repairing defective heart muscle.

Researchers have already shown that adult bone marrow cells guided to become heart-like cells can repair heart tissue in people, and more research is ongoing.

Yes. Doctors have performed stem cell transplants, also known as bone marrow transplants. In stem cell transplants, stem cells replace cells damaged by chemotherapy or disease or serve as a way for the donor's immune system to fight some types of cancer and blood-related diseases, such as leukemia, lymphoma, neuroblastoma and multiple myeloma. These transplants use adult stem cells or umbilical cord blood.

Researchers are testing adult stem cells to treat other conditions, including a number of degenerative diseases such as heart failure.

For embryonic stem cells to be useful in people, researchers must be certain that the stem cells will differentiate into the specific cell types desired.

Researchers have discovered ways to direct stem cells to become specific types of cells, such as directing embryonic stem cells to become heart cells. Research is ongoing in this area.

Embryonic stem cells can also grow irregularly or specialize in different cell types spontaneously. Researchers are studying how to control the growth and differentiation of embryonic stem cells.

Embryonic stem cells might also trigger an immune response in which the recipient's body attacks the stem cells as foreign invaders, or the stem cells might simply fail to function normally, with unknown consequences. Researchers continue to study how to avoid these possible complications.

Therapeutic cloning, also called somatic cell nuclear transfer, is a technique to create versatile stem cells independent of fertilized eggs. In this technique, the nucleus, which contains the genetic material, is removed from an unfertilized egg. The nucleus is also removed from the cell of a donor.

This donor nucleus is then injected into the egg, replacing the nucleus that was removed, in a process called nuclear transfer. The egg is allowed to divide and soon forms a blastocyst. This process creates a line of stem cells that is genetically identical to the donor's cells in essence, a clone.

Some researchers believe that stem cells derived from therapeutic cloning may offer benefits over those from fertilized eggs because cloned cells are less likely to be rejected once transplanted back into the donor and may allow researchers to see exactly how a disease develops.

No. Researchers haven't been able to successfully perform therapeutic cloning with humans despite success in a number of other species.

However, in recent studies, researchers have created human pluripotent stem cells by modifying the therapeutic cloning process. Researchers continue to study the potential of therapeutic cloning in people.

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Excerpt from:
Stem cells: What they are and what they do - Mayo Clinic

Theres been a lot of talk about stem cell therapy in recent yearsbut what is it, exactly? How does it work, and is it an effective treatment for knee pain?

Knee pain affects over 25% of adults in the US, and can affect anyone of any age. While physical therapy and prescription medications can be used to effectively treat mild pain, those with more severe pain may require surgery.

For patients who are struggling to manage knee pain but would like to delay knee replacement surgery, stem cell therapy may be an option. Dr. Daniel E. Murphy, an orthopedic specialist here at Florida Medical Clinic wants to help patients understand stem cell treatment, its uses and limitations, and how it may help some find relief from knee pain caused by injury or arthritis.

Stem cell therapy, also called orthobiologic treatment when talking about treating orthopedic conditions, is a special kind of medical treatment that uses stem cells and growth factors to reduce pain.

In the human body, most cells have one job. For example, a red blood cell cant do the same job as a skin cell. However, stem cells are a special kind of cell that have the potential to divide into any other type of cell found in the body, like a liver cell or a heart muscle cell. That means they can perform many different jobs.

In some laboratories, scientists have been able to help some patients rebuild damaged tissue using stem cells. While that success has been limited to lab settings, Dr. Murphy says doctors are still studying if it can be used to help everyday patients regenerate lost tissue. Thats because theres still a lot we dont know about stem cells and how they can be manipulated to work in different parts of the body.

Scientists arent sure if or how stem cell treatment can help patients regrow lost cartilage or bone density, which may be at the root of many cases of knee pain. However, we do know that patients suffering from knee pain may find relief with treatment.

You may be wary of stem cells if youve heard about some of the controversy surrounding how theyre sourced. Its true that some kinds of stem cells are sourced from donated blastocysts (early-stage embryos) that are just a few days old, but these kinds are not used for orthobiologic treatment.

Instead, orthobiologic therapy for knee pain most commonly uses stem cells from adult patients themselves by taking samples of bone marrow and other tissues.

During treatment, a doctor will take samples of stem cells from other parts of your body and reimplant them into an injured area. For knee pain, that may involve taking sample cells from bone marrow or fat tissue and injecting them into knee joints.

Your doctor will always discuss the source of your stem cells with you before starting treatment and will not inject you with any substance you do not consent to.

Stem cell therapy may help reduce pain but is not a magic solution for any disease or condition. Be wary of any physician or clinic that claims stem cell therapy can completely reverse or heal an orthopedic condition.

As of 2020, the FDA has only approved stem cell treatment for a few kinds of diseases, including some cancers and blood disorders. However, reinjecting a patients own stem cells back into their body is permitted as a therapy for orthopedic purposes.

Many patients do find relief from pain and stiffness caused by knee injuries or osteoarthritis with orthobiologic treatment. It may also be a good alternative to knee replacement surgery in some patients. There has been some evidence that orthobiologic injections reduce knee pain in patients by as much as 75%, which was supported by a follow-up study. Scientists believe this may be because injected stem cells can help reduce inflammation.

Stem cell therapy and orthobiologic treatment are still burgeoning medical fields. Theres a lot were still learning about their uses, benefits, and drawbacks. We dont know for certain if it works for things like cartilage regeneration or healing spinal cord injuries.

Despite this, Dr. Murphy reports that many patients do find orthobiologic treatment to be helpful for their knee paineven if it doesnt reverse bone or cartilage loss. Some of the benefits may include:

The majority of patients who receive stem cell treatment for knee pain experience quick recovery times and little to no adverse side effects. That being said, its important to know the potential drawbacks:

Your doctor should fully discuss the pros and cons of this treatment with you before you receive any injections. Some patients may not be eligible based on certain risk factors.

In combination with lifestyle changes and other treatments, many patients find relief from knee pain with stem cell therapy. That being said, its important to discuss your options and risk factors with your doctor to determine what kind of treatment path is right for you.

If youre considering a stem cell injection, make sure your doctor is a board-certified orthopaedic surgeon before you proceed. Your physician should be listed on the American Academy of Orthopaedic Surgeons website at aaos.org.

To learn more about stem cell therapy for knee pain, schedule an appointment with Dr. Murphy today. Virtual appointments are also available.

Disclaimer: This blog is not intended to substitute professional medical advice. Always talk with your doctor before starting or stopping medications or treatments.

Read this article:
Stem Cell Therapy for Knee Pain: What Patients Should Know

By shifting mouse elderly hematopoietic stem cells (aged HSCs) to the environment of young mice (bone marrow niche), it was shown that the pattern of stem cell gene expression was rejuvenated to that of young hematopoietic stem cells. On the other hand, the function of elderly HSCs failed to recover in the young bone marrow niche. The epigenome (DNA methylation) of aged HSCs didnt change significantly even in the young bone marrow niche, and DNA methylation profiles were found to be a better index than the gene expression pattern of aged HSCs.

A research group headed by Professor Atsushi Iwama in the Division of Stem Cell and Molecular Medicine, The Institute of Medical Science, The University of Tokyo (IMSUT) declared these world-first Outcomes and was published in the Journal of Experimental Medicine (online) on November 24th.

The results will contribute to the development of treatments for age-related blood diseases.

Professor Atsushi Iwama, Lead Scientist, IMSUT

The research group investigated whether rejuvenating aged HSCs in a young bone marrow market environment would rejuvenate.

Tens of thousands of elderly hematopoietic stem/progenitor cells gathered from 20-month-old mice were transplanted into 8-week-old young mice without pretreatment like irradiation. After two months of follow-up, they collected bone marrow cells and performed flow cytometric analysis.

The research team also transplanted 10-week-old young mouse HSCs for comparison. In addition, engrafted aged HSCs were fractionated and RNA sequence analysis and DNA methylation analysis were conducted.

They discovered that engrafted elderly HSCs were less capable of producing hematopoietic cells compared to younger HSCs. They also showed that differentiation of aged HSCs into multipotent progenitor cells was persistently impaired even in the young bone marrow market, and that the direction of differentiation was biased. It was found that the transfer of aged HSCs into the young bone marrow market does not enhance their stem cell function.

A more detailed analysis may reveal mechanisms that irreversibly affect aged HSC functionAging studies focusing on HSCs have been chased in mice with a bone marrow transfer model. However, the effect of aging on HSCs remains to be clarified.

Professor Iwama says as follows. This analysis has a substantial impact because it clarified the effect of aging on HSCs. Our results are expected to contribute to further elucidation of the mechanism of aging in HSCs and comprehension of the pathogenic mechanism of age-related blood disorders.

Source:

Journal reference:

Kuribayashi, W.,et al.(2020) Limited rejuvenation of aged hematopoietic stem cells in young bone marrow niche.Journal of Experimental Medicine.doi.org/10.1084/jem.20192283.

Read more here:
Study clarifies the impact of getting old on hematopoietic stem cells - Microbioz India

Lesser or stable disability over two years was evident in most progressive multiple sclerosis (MS) patients given a stem cell treatment in a small Phase 1 clinical trial, supporting a larger study now underway, researchers report.

These results suggest that a treatment using mesenchymal stem cell-derived neural progenitors (MSC-NPs) can safely and effectively ease inflammation in progressive MS.

But for a subset of patients, particularly those with more advanced disease and greater disability, this treatment did not sufficiently counter a continued inflammatory response in the brain.

The study, Mesenchymal stem cell-derived neural progenitors in progressive MS: Two-year follow-up of a phase I study, was published in the journal Neurology: Neuroimmunology and Neuroinflammation.

MSC-NPs are seen as a possible way of treatingpeople with progressive MS, who have few effective disease-modifying treatments available. They are essentially stem cells collected from a patients bone marrow that are expanded and matured to produce factors involved in modulating the immune response and innervous tissue growth and survival.

An open-label Phase 1 trial (NCT01933802) investigated this stem cell treatment in 20 adults with stable primary(four PPMS patients) or secondary progressive MS(16 SPMS patients) and significant disability.

All received a total of three injections of MSC-NPs, given directly into the spinal canal three months apart. They were then evaluated at three and six months, and again at two years, after the final treatment to determine its long-term safety and tolerability, and for signs of potential effectiveness.

An initial analysisat six months post-treatment found lesser disability in most trial participants (15 of the 20) andbetter muscle strength in 14 of them. Greater exercise capacity was also seen in four of the 10 patients able to walk at the studys start, and two nonambulatory patients gained an ability to walk using assistive devices.

Researchers now reported clinical findings at two years after treatment. All 20 completed two-year follow-up assessments, buttwo who were severely disabled could not do a final in-person visit. They were examined via telemedicine and did not provide biomarker samples.

Disability was evaluated using the Expanded Disability Status Scale (EDSS), in which a higher score indicates more severe disability. The two who moved to telemedicine had EDSS scores of 8.0.

At six months, eight participants had an EDSS reduction of at least 0.5 points, including four with disability reductions of two or more points. At the two-year follow-up, seven of these eight people continued to show improvements in their EDSS scores, including two who showed a sustained 2.0 or more point reduction.

The eighth patient, whose disability had initially improved by one point, showed a worsening in disability at two years.

Of the 10 patients without initial improvements in EDSS scores, six had no evidence of disease progression throughout the study and follow-up. Two others worsened at each follow-up, and two showed worsening disease between the six-month and two-year examinations.

Of the 10 nonambulatory patients at the trials start, four showed improvements in walking speed greater than 20% at three months post-treatment. At two years, three had maintained these walking speed gains, while one fell just below the 20% improvement mark.

One of the two people unable to walk at the beginning of the study completed the walking test at both the three-month and two-year exams. One other patient, with an initial normal walking speed, maintained that speed throughout the trial and follow-up periods.

These results indicate that multiple MSC-NP treatments led to disability reduction for most progressive patients with long-standing disease. But those who sustained these gains at two years after treatment had lower EDSS and ambulatory status at baseline or the studys start, the researchers wrote.

A subset of patients with initial improvement failed to maintain shown benefits, while others showed no disease progression throughout the follow-up.

Cerebrospinal fluid (CSF) levels of CCL2, a pro-inflammatory factor, were lower following treatment, while levels of the anti-inflammatory TGF beta 2 rose post-treatment, consistent with previous studies of similar treatments.

Interestingly, no difference here was observed between patients whose disability improved in response to the treatment (responders) and those who failed to improve (non-responders).

However, some inflammatory factors were seen to rise after treatment in non-responders, but not among those who responded to treatment. This suggests that a continued inflammatory response may hinder clinical response to MSC-NP use.

Neurofilament light chain (NfL) levels in the CSF, a marker of nerve cell degeneration and damage, can be elevated in MS patients. Among a small number of trial patients with high NfL levels prior to treatment, these levels rose further in nonresponders after treatment while they declined among responders.

We observed that most subjects who received repeated [MSC-NP] injections exhibited either a reversal in disability or lack of disease progression that was sustained for 2 years after treatment, the researchers wrote.

The impact of any efficacy conclusion, however, are severely limited by the very small number of patients in the study and the lack of blinding and placebo controls, they added.

An ongoing and placebo-controlled Phase 2 clinical trial (NCT03355365), which opened last year, is now investigating the safety and efficacy of repeat MSC-NP injections in progressive MS patients. The study is expected to have enrolled50 adults with progressive MS (40 SPMS and 10 PPMS), being given a total of six injections of either MSC-NPs or a placebo every other month for a first year.

In its second year, those in the MSC-NP group cross to the placebo group and those previously on a placebo move to treatment again for six total injections given every two months. This single-site trial at the Tisch MS Research Center of New Yorkwill run for three years, and is expected to finish in late 2023.

Aisha Abdullah received a B.S. in biology from the University of Houston and a Ph.D. in neuroscience from Weill Cornell Medical College, where she studied the role of microRNA in embryonic and early postnatal brain development. Since finishing graduate school, she has worked as a science communicator making science accessible to broad audiences.

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Read more:
Stem Cell Therapy Shows 2-year Benefit for Progressive MS Patients in Phase 1 Trial - Multiple Sclerosis News Today