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What is CAR-T Cell Therapy | CAR-T Definition | Bioinformant

March 16th, 2019

CAR-T cell therapy is asa type of immunotherapy that teaches T cells to recognize and destroy cancer.CAR-T cell therapy has demonstrated promising results in a range of patients from young and old. In some patients, this can lead to the total elimination of the cancer. In others, there is a significant improvement of the disease.

For those who are facing cancer, it is important to answer the question What is CAR-T? This guide will answer the most common questions about CAR-T cell therapy for readers who want to understand this novel technology platform for treating cancer.

What you need to know about CAR-T therapy and its role in cancer treatment is described below.

CAR-T is pronounced phonetically, as car tee cell.CAR-T is named after a mythical creature called the chimera. A chimera is an animal made of different parts of different animals attached together.

With CAR-T cell therapy, apatientsTcells are modified within a laboratory, so that they they can find and attack cancer cells. Because CAR-T cells combine different parts from different sources, they are called chimera (meaning, blended or fused) antigen receptor T cells.

T cells are a type of white bloodcell that plays a central role in the immune response within humans.T cell that have been genetically altered into CAR-T cells function as living drugs when they are administered to patients.

To understand CAR-T cell therapy,a brief history of immunologymay prove helpful. An antigen is a foreign substance in the body, either a toxin or disease agent or unhealthy cell (as in cancer), that triggers an immune response. The body then produces white blood cells to attack the agent. It does this by binding to it with the use of antigen receptors on the surface of the white blood cells, or lymphocytes. Only then does the body produce antibodies to destroy the foreign or diseased agent.

The problem is T cells, the white blood cells responsible for destroying tumor cells,are not good enough at recognizing it. Therefore, in order to increase the patient immune levels, medical specialists take blood. From the blood, they harvest T cells and add extra antigen receptors to the surface of the cells. They inject those cells back into the patient via blood transfusion, where they multiply and can then attack cancer, either with or without the aid of additional therapies.

Specifically, the antigens can then recognize the protein CD-19, which forms on the surface of B cells, a type of blood cell that frequently becomes cancerous. By knowing which proteins to look for, the modified T cells can hunt them down, attack, and destroy them throughout the bloodstream.

CAR-T cells are defined as T-cells (immune cells) that have been modified to match markers present on the outside of cancer cells, allowing them to selectively find and attack them. To create CAR-T cells, physicians extract T-cells from a patient, genetically alter them, expand them in quantity, and re-infuse them to the patient so that the engineered CAR-T cell can selectively attack cancer cells.

The patient response is then monitored using a variety of tools.

There are four steps involved with the CAR-T cell therapy process.

These steps include:

The patient is then monitored by the attending physicians to document the therapeutic response.

Cancer is a silent killer. Too often, it has devastating results, because the cells in the human body are not adept at killingit. This is the case with T cells, human immune cells whose responsibility is to fight invasion and disease. These cells, also known as T lymphocytes a special type of white blood cell are not always able to recognize and eliminate cancer.A potential new solution may be CAR-T cell therapy.

As theCancer Treatment Centers of Americapoints to CAR-T treatment as a novel way to treat cancer, it could drastically alter the medical outlook for both children and adults. These patients would otherwise be without the possibility of a cure.

However, CAR-T immunotherapy is not a cure-all for every patient. For some, it only works for a short time before the cancer relapses. Other patients respond to it, but suffer such severe side effects that it does almost nothing to ease the symptoms. While researchers work furiously to determine why some treatments work on cancer cells and others do not, they still have not arrived at a firm answer.

During transport and until ready to administer at bedside CAR-T cells must be stored at least -150 Celsius. @SylvesterCancer is the only center in South Florida certified to treat patients with this novel #immunotherapy pic.twitter.com/1LKm6UHzd8

Sylvester Cancer (@SylvesterCancer) August 7, 2018

In 2017, two experimental CAR-T treatments received approval from the U.S. FDA with more in clinical trials:

Kymriah was approved by FDA in August 2017 to be used in children and adults with ALL. In May 2018, the FDA approved Kymriah for a second indication (diffuse large B-cell lymphoma). The second CAR-T product, Yescarta, was approved by FDA in October 2017 for patients with lymphoma.In August 2018, both Kymriah and Yescarta secured European regulatory approval. In September 2018, Health Canada made Kymriah the first CAR-T therapy to receive regulatory approval in Canada.

Numerous companies are also working to perfect the technology of CAR-T cells. Akron Biotechmodifies many types of cells for use in medical treatments.

CAR-T is a new technology. Not only is it expensive to manufacture antigens in a lab and attach them to T cells, it takes a long time and carries a number of different specifications in order for candidates to gain approval for the treatment. So, exactly which candidates can receive therapy?

Both treatment protocols modify T cells to help them recognize and attack diseased B cells in the blood. Patients with either leukemia or B-cell lymphoma may apply for the clinical trial at this time. However, they cannot do so without first trying at least two other cancer therapies of a more standard nature.

Currently, researchers are experimenting with CAR-T therapies for other types of cancers as well. These include leukemia and lymphoma subtypes, as well as non-blood-borne cancers. Its ability to fight solid tumors, or those that do not spread throughout blood or bone marrow, have thus far proven less than impressive.

Physicians make CAR-T cells via a careful process. First, the patient is set up in the hospital and prepped for a blood draw, followed by a long stay. Most patients are quite ill by the time they start CAR-T cell immunotherapy, necessitating they remain in the hospital until the completion of the treatment.

Doctors then take a patients blood and feed it into anapheresis machine. This device separates out the white blood cells, T cells included. Then it feeds the remaining blood back to the patient. This means they do not lose a lot of blood while physicians now have a healthy supply of cells to transform. Doctors then freeze the harvested cells and send them off to a lab.

Lab workers then take the collected T cells and introduce a gene that manufactures the chimeric antigen receptor into the DNA of each cell. Lab workers then grow millions of versions of these cells. Once they have enough, they harvest the cells, freeze them once more and deliver them back to the patient via transfusion.

Both these T cells, plus the ones subsequently manufactured by the patients body, can then bind to and attack the cancer cells.

Because transforming T cells is such a complex process, the treatment is typically a long one for the patient. From beginning to end, the transformation and reintroduction of cells may take up to 3 weeks. During that time, the patient is compromised even more than usual due to the reduction in their T cell population. Thats why they usually stay in the hospital during the entire process. This way, doctors can monitor them and make sure their immunity stays as robust as possible.

Before introducing the modified T cells to the patient, physicians typically give them a round of chemotherapy. This helps to weaken their immune system further, which reduces the chances that existing T cells will outnumber the new ones. Counterintuitively, by depressing the immune system in the short run, doctors give patients the best chance of engineered T cells multiplying and doing their job.

The transfusion itself is typically short and painless, lasting only about an hour. After staying in the hospital for monitoring, patients must come in regularly for a few weeks afterward.

The huge benefit of a treatment like this is the T cell modifications will last for life. Each time a bodys T cells encounter a toxin or disease agent and develop antigen receptors and antibodies to fight it, the person has that ability forever. That means patients who receive modified T cells now have the tools to fight their particular cancer for the remainder of their days.

This makes CAR-T cell therapy more than a treatment. For example, while chemotherapy and radiation are effective, their curative effects end when the treatment ends (or, more accurately, a few days or weeks after the last course). In contrast, modified T cells hang aroundforever, turning this type of immunotherapy into a living drug.

While CAR-T therapies are long-lasting, making them more affordable over a lifetime, it is expensive to access these therapies.Currently, Kymriah and Yescarta are offered at the following prices:

Moreover, possible side effects do exist. These include:

Finally, while the process is very beneficial to some patients, it is extremely time-consuming. Some question where it can actually serve the broader population, considering the necessary time and specialization required.

Do you need a visual look at how CAR-T therapy works? Watch this video from Associated Press.

CAR-T companies are on the rise, supported by growing investment flowing into CAR-T product development and landmark approvals of CAR-T cell therapies by the U.S. FDA, European Medicines Agency (EMA), and Health Canada.

Are you interested to know the identities of the companies developing CAR-T therapies worldwide?

For a limited-time, you can claim the Global Database of CAR-T Cell Therapy Companies and get the CAR-T Funding Brief ($49 value) for FREE:

Overall, T-cell therapy has proven a promising new treatment approach. As its manufacture, administration, and safety profile improve, it will become an important tool in the cancer treatment toolkit.

Do you know anyone in need of a cancer cure? What role could CAR-T therapy play in their treatment? Let us know in the comments below.

What is CAR-T Cell Therapy? | CAR-T Definition

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Crude versus defined CAR T-cell therapy product

March 8th, 2019

In the race for the most potent CAR T-cell therapy, there is a big interest to the issue of purity and composition of the final cell product. In this post, Ill try to summarize the current knowledge about defined CAR T-cell products, based on two clinical studies, published this week.

What is defined product and why it is important?We can roughly divide CAR T-cell products on bulk T-cell or crude and defined composition. Crude CART products are not purified and contain: different ratios of CD4/CD8 T-cells and their subsets, other than T- contaminating cells and non-CAR cells. Defined composition products could be the following:

There are few good reasons for development of defined CART products:

The later is single most important reason, which begs the question Will defined CAR T-cell products deliver superior therapeutic benefit? As of today, vast majority of CART developers manufacture crude cell products. Manufacturing process usually does not include sorting of T-cells on day 0 or purification of T-cells/ CAR+ cells in-process or on a harvest day. Most of developers release final CAR T-cell product with CD3+ cells >80-90%, highly variable CD4/CD8 ratio and % of CAR+ cells.

Preclinical dataAt least one group of researchers have done a lot of experimental and clinical work on defined composition of CAR T-cell products. Defined product/ process development has been done by Michael Jensen lab, initially at City of Hope and then further adapted and modified at Fred Hutchinson Cancer Center and Seattle Childrens Hospital. Stanly Riddells lab from Hutch did a lot of experimental work to demonstrate potential value of defined CAR T-cell product. All together they have tried all versions of defined composition CAR T-cell products, mentioned above. One of recent and the most comprehensive studies by Riddells lab, showed that (1) combination of both CD4 and CD8 T-cells has superior therapeutic potency and (2) naive CD4 cells and central memory (T-CM) CD8 T-cell subsets were the most potent in vivo. Long-term persistence of human memory T-cells was demonstrated by Riddell and Jensen earlier in mouse and primate models. Importance of CD4+CD8 combo rather than use of potent cytotoxic CD8+ cells alone was also demonstrated in numerous studies (check here, here and here). Therefore, experimental studies created a strong rational for favoring T-CM, naive T-cells (T-N) and CD4+CD8 combo in adoptive cell therapy trials.

Feasibility of manufacturing defined T-CM CAR T-cell productBefore I get to the first clinical results, Id like to look at manufacturing process of defined CAR T-cell product. Jensen started with purified CD8 T-CM or CD4 + CD8 T-CM manufacturing process, which described in details here. It includes sequential 2-step CliniMACS procedure for negative selection of CD14/CD45RA/CD4 or CD14/CD45RA-negative cells, positive selection for CD62L (marker of T-CM) and culture with IL2/IL15 for up to 30 days in bags. After two CliniMACS procedures, cell recovery was as low as 0.4% (in contrast to expected 1.4%). If input cell number was usually 5 billions PBMCs, average output cell number before starting a culture was ~19 millions. T-CM recovery efficiency was 26%. Even though, they typically started culture from 7-15 millions of CD8 T-CM cells, generation of ~3 billions of cells in 3-6 weeks was feasible. This manufacturing process was used in 2 clinical trials: NHL1 and NHL2.

In the modified manufacturing process, designed for NCT01865617 trial, included CliniMACS selection of CD4+ bulk population and 2-step CD8 T-CM (see above) or CD8 bulk selection with 2 parallel 15-20 days cultures and mixing CD4:CD8 as 1:1 before infusion. Importantly, CAR+ cells were selected before infusion by a marker (EGFRt). Interestingly, either CD8 T-CM purified on day 0 or CD8 bulk cells yielded only ~40-50% of CD8+/CAR+ cells with T-CM phenotype (CD45RA-CD62L+).Now, feasibility of manufacturing in NCT01865617. 16/30 (53%) patients have passed threshold of 20 T-CM cells/ ul in screening assay for feasibility of manufacturing. From selected products, T-CM were successfully manufactured in all, but 1 cases. 3 out of 30 infused products were not formulated as 1:1 (10%), due to lack of expansion.Id summarize some of my thoughts of defined CAR T-cell product manufacturing feasibility as the following:

Clinical outcomes of using defined CAR T-cell productsResults of 3 clinical trials (NCT01318317, NCT01815749, NCT01318317), using defined CAR T-cell products have been published so far (here and here). However, the therapeutic benefit of using defined versus crude CAR T-cell product remains unclear. Ideally, defined CAR T-cells should be compared with crude product within one trial settings, because even for the same conditions, clinical protocols are very very different between sites. Also, ideally, CD8 bulk vs. CD T-CM or CD8 alone vs. CD8+CD4 combo should be compared within one trial in exactly the same settings. Unfortunately, none of these ideal comparison conditions were met in 3 published trials, mentioned above.

Even though, it seem like CD4 + CD8 T-CM combo performed better in NHL2 trial (75% progression-free survival at 1-year) than CD8 T-CM alone in NHL1 (50% progression-free survival at 1-year), the difference is not significant, due to low number of patients (n=8 in each trial). On top of it, different CAR vectors were used between these trials, culture duration was shorter and CD25+ T-regs were depleted in NHL2 trial manufacturing protocol. So, data cannot be compared. If we look at results of other CAR T-cell lymphoma trials (narrowing to DLBCL), City of Hope results are not much better than reported from other centers (for example, from Penn). CD8 T-CM persistence was not beneficial, compare to data from other centers. Two excerpts from the study, which demonstrate that assessment of defined CAR T-cell product benefit is impossible:

CD19-CAR T cell activity is difficult to assess by disease response, since 9/16 patients were in CR at start of study, and HSCT can also produce CRs.Thus, a T cell product derived from central-memory enrichment as described in these studies, does not persist longer than what is observed in trials with conventional bulk T cells transduced with CARs bearing CD28 co-stimulatory domains.

Now, moving to B-ALL study, published this week in JCI. First of all, B-ALL is not the best condition to assess a difference by clinical outcome between crude and defined CAR T-cell products, because response rate is too high (close to 90%) across the centers no matter what. The authors about outcome:

The 93% remission rate by flow cytometry and 86% MRD-negative CR rate in our study compares very favorably to that reported by others in which CART cells of undefined composition were manufactured using CD19 CARs that incorporate either a 4-1BB costimulatory domain (children and young adults, 79%) or a CD28 costimulatory domain (adults, 75%; children and young adults, 60%) (1-4)

What about persistence? Theoretically, based on experimental work, CD8 T-CM should have superior long-term persistence. But it was not the case. The study showed persistence only at 1 month time point. What about relapses? Maybe application of T-CM will reduce the rate of relapses? No, 9/30 patients in the study relapsed, half of them (5/9) received CD8 T-CM product. Clinical outcome was significantly improved in the study after implementation of different conditioning regiment (with fludarabine). This change significantly complicates and even make impossible data comparison between CD8 bulk and CD8 T-CM groups:

The high overall rate of BM remission of 93% by flow cytometry in this study and differences in lymphodepletion regimens and infused cell doses do not allow comparison of the efficacy of CART cell products manufactured from CD8+ TCM cells or from bulk CD8+ T cells. Analysis of differences in long-term persistence of cell products that were selected for CD8+ TCM or bulk CD8+ T cells in our study was further complicated by our findings that immune-mediated rejection of CART cells occurs in some patients, which may provide an explanation for the loss of CART cells observed in a subset of patients in other studies

To conclude: Despite the strong experimental evidence and very attractive idea behind of defined CAR T-cell products, it is too early to conclude about their therapeutic benefit and superior potency. With greater number of patients and technical improvements in manufacturing (more efficient clinical cell sorting, IL7+IL15 in culture and other), potential benefit of defined CAR T-cell product may become more obvious. Such benefits as dropping a therapeutic dose, better correlation between dose and in vivo expansion dynamics, decreasing donor variability in manufacturing, we can see today already.

Tagged as:CART, cell product, manufacturing

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Japan Approves iPS Cell Therapy Trial for Spinal Cord …

February 21st, 2019

The Japanese governments health ministry has given the go-ahead for a trial of human induced pluripotent stem cells to treat spinal cord injury, Reutersreports today (February 18).Researchers at Keio University plan to recruit four adults who have sustained recent nerve damage in sports or traffic accidents.

Its been 20 years since I started researching cell treatment. Finally we can start a clinical trial, Hideyuki Okano of Keio University School of Medicine told a press conference earlier today, The Japan Timesreports. We want to do our best to establish safety and provide the treatment to patients.

The teams intervention involves removing differentiated cells from patients and reprogramming them via human induced pluripotent stem cells (iPSCs) into neural cells. Clinicians will then inject about 2 million of these cells into each patients site of injury. The approach has been successfully tested in a monkey, which recovered the ability to walk after paralysis, according to the Times.

Its not the first time Japan has approved the use of iPSCs in clinical trials. Last year, researchers at Kyoto University launched a trial using the cells to treat Parkinsons disease. And in 2014, a team at the RIKEN Center for Developmental Biology led the first transplant of retina cells grown from iPSCs to treat a patients eye disease.

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Stem Cell Therapy For Knees | What You Need To Know …

February 12th, 2019

The main conditions treated by stem cell injections include knee osteoarthritis, cartilage degeneration, and various acute conditions, such as a torn ACL, MCL, or meniscus. Stem cell therapy may speed healing times in the latter, while it can actually rebuild tissue in degenerative conditions such as the former.

Thats a major breakthrough. Since cartilage does not regenerate, humans only have as much as they are born with. Once years of physical activity have worn it away from joints, there is no replacing it. Or at least, there wasnt before stem cell therapy.

Now, this cutting-edge technology enables physicians to introduce stem cells to the body. Thesemaster cells are capable of turning into formerly finite cell types to help the body rebuild and restore itself.

Although it may sound like an intensive procedure, stem cell therapy is relatively straightforward and usually minimally invasive. These days, physicians have many rich sources of adult stem cells, which they can harvest right from the patients own body. This obviates the need for embryonic stem cells, and thereby the need for moral arguments of yore.

Mesenchymal stem cells (MSCs) are one of the main types used by physicians in treating knee joint problems. These cells live in bone marrow, butincreasing evidence shows they also exist in a range of other types of tissue.This means they can be found in places like fat and muscle. With a local anesthetic to control discomfort, doctors can draw a sample of tissue from the chosen site of the body. The patient usually doesnt feel pain even after the procedure. In some cases, the physician may choose to put the patient under mild anesthesia.

They then isolate the mesenchymal stem cells. Once they have great enough numbers, physicians use them to prepare stem cell injections. They insert a needle into the tissue of the knee and deliver the stem cells back into the area. This is where they will get to work rebuilding the damaged tissue. Although the mechanisms arent entirely clear, once inserted into a particular environment, mesenchymal stem cells exert positive therapeutics effectsinto the local tissue environment.

Mechanisms of action of mesenchymal stem cells appear to include reducing inflammation, reducing scarring (fibrosis), and positively impacting immune system function.

Thats not quite enough to ensure a successful procedure, however. Thats why stem cell clinics may also introduce growth factors to the area. These are hormones that tell the body to deliver blood, oxygen,and nutrients to the area, helping the stem cells thrive and the body repair itself.

Physicians extract these growth factors from blood in the form of platelet-rich plasma (PRP). They take a blood sample, put it in a centrifuge and isolate the plasma, a clear liquid free of red blood cells, but rich in hormones needed for tissue repair.

So, what can a patient reasonably expect when it comes to stem cell therapy, in terms of time and cost outlay?

The answers to both of these questions differ depending on the clinic doing the procedure and the patients level of knee degradation. Some clinics recommend a course of injections over time. Meanwhile, others prepare the injection and deliver it back to the patient in only a matter of hours. Either way, the treatment is minimally invasive, with fast healing times and a speedy return to normal (and even high-intensity) activity.

Some quotes for stem cell knee treatment are as low as $5,000. Others cost up to $20,000 or more. Again, this depends on how many treatments a patient needs, as well as how many joints theyre treating at the same time. Because its easier to batch prepare stem cells, a patient treating more than one knee (or another joint) can address multiple sites for far less. The procedure would only cost an addition of about $2,000 or so per joint.

No treatment proves effective every time. However, insofar as patients reporting good results for stem cell injections, the overall evidence does lean in a beneficial direction.Studies at the Mayo Clinic, for instance, indicate that while further research is needed, it is a good option for arthritis in the knee. Anecdotal reports are positive as well. Patients report it as an effective alternative to much more invasive solutions, such as arthroscopic or knee replacement surgery.

Other studies point to the need for caution. Stem cell therapy and regenerative medicine, in general, are only now exiting their infancies. There arent enough high-quality sources from which to draw at this point, so hard and fast conclusions remain elusive. Of the studies that do exist, some contain unacceptably high levels of bias.

Of course, any new treatment will face these kinds of challenges in the beginning. For those who need an answer to knee pain, and havent yet found one that works, its likely worth the risk that it wont prove as effective as they hoped. But what about other risks?

The good news about this form of stem cell therapy is that the patient uses their own cells. That means they completely skip over the dangers that accompany donor cells. The main one of which is graft-versus-host disease (in which the donor cells initiate an immune response against the patients body). Because the cells have all the same antibodies, neither the body nor the reintroduced cells will reject one another.

Also, the relatively low-stakes outpatient nature of the procedure (versus, say, a bone marrow transplant) means that the chances of something going wrong are much reduced.

However, there do exist some risks wherever needles come into play. It is possible to get an infection at the site of the blood draw as well as at the injection site, but these risks are quite low. Other risks include discoloration at theinjection site or soreness. While some people fear the possible growth of stem cells at the site of injection into a tumor, it is unlikely for this to happen, because physicians utilize adult stem cells for these procedures that have a low proliferative capacity.

These adult stem cells tend to be much safe than pluripotent stem cell types. Examples of pluripotent stem cells are embryonic stem cells (derived from embryos) and a type of lab-made stem cell known as induced pluripotent stem cell (iPS cell).

For those who think stem cell therapy could prove beneficial, its time to set up a consultation with your doctor. Multiple factors will influence whether or not its a good idea. These include age, health, andseverity of the condition and other available treatments. However, overall, this form of regenerative medicine is reasonably affordable, very low-risk, and typically effective.

Are you seeking a stem cell treatment for your knees or other joints?To support you,we have partnered withOkyanosa state-of-the-art facility providing patients with advanced stem cell treatments.

The group offers treatments for arange of chronic conditions, includingosteoarthritis and degenerative joint disease, which are leading causes of knee pain.

If you are seeking a stem cell treatment for knee pain or other chronic condition,contact Okyanos for a Free Medical Consultation.

What questions do you still have about stem cell therapy for knees? Ask them below and we will get you answers.

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The Cost Of Stem Cell Therapy And Why It’s So Expensive …

February 10th, 2019

How much is stem cell therapy? As stated by CBC Canada,the cost of stem cell therapy is $5,000 to $8,000per stem cell treatment for patients. According to a Twitter poll by BioInformant, the cost can be even higher. Our May 2018 poll found that stem cell treatments can cost as much as $25,000 or more. This article explores the key factors that impact the cost of stem cell therapy, including the type of stem cells used within the protocol, the number of treatments required, and the site of theclinic. It also provides pricing quotes from stem cell clinics within the U.S. and worldwide.

In this article:

Stem cell therapy is the use of living cells as therapeutics to treat disease or injury. Read on to learn about the cost requirements of these procedures.

CBC Canadas pricing involves Cell Surgical Network (CSN) following its protocol to remove fat tissue and process it before re-injecting [adipose-derived stem cells] either directly or intravenously into the same patient. Unfortunately, the U.S. FDA and Department of Justice (DOJ) sent this network of stem cell treatment providers a permanent injunction notice in May 2018. Therefore, patients should not seek treatments from the group at this time.Although Cell Surgical Network (CSN) is based in California, it has a network of approximately 100 U.S. treatment centers. They also have three Canadian clinics located in Vancouver, Sudbury,andKamloops.

The controversy such as the one above stirs up questions about the safety of stem cell procedures. Anyone considering stem cell therapy from any tissue or source will benefit from understanding the possible consequences of stem cell therapy and the factors driving costs.

For the patient, a stem cell transplant involves multiple steps, including:

There are also real costs for the doctors who provide stem cell treatments. They have overhead costs, including:

There is also time and expertise required toperform the procedure and offer post-operative care. In some cases, the physician must pay licensing fees to access stem cell sourcing, processing, or delivery technologies.

Stem cell treatment has gained more and more traction over the last decade. It has been helped along by considerable advances in research. In 2017, the number of scientific publications about stem cells surpassed 300,000. The number of stem cell clinical trials has also surpassed4,600 worldwide.

However, stem cell therapy is still expensive. Among the cheapest and easiest options is to harvest adipose-derived stem cells (ADSCs) those that exist in adult fat layers and re-deliver them to the patient. Unlike harvesting from bone marrow or teeth, providers can feasibly remove fat, separate stem cells, then re-inject them into a patient the same day. This approach is typically less expensive than those that require more invasive procedures for harvesting. Because of its practicality in terms of cost, it has become a common approach to stem cell treatment.

Relatively easy harvesting stilldoesnt translate to inexpensive cost, although some are certainly more affordable than others. For orthopedic conditions, the costof stem cell therapy is typically lower, averaging between $5,000 and $8,000. Examples of these types of medical conditions include:

Note that these prices are typically out-of-pocket costs paid by the patientbecause most insurance companies will not cover them. They are considered experimental and unapproved by the FDA. This means patients needing stem cell treatment will need to use their own savings.

Although fat is a frequently utilized source for stem cells, it is also possible for physicians to utilize stem cells from bone marrow. Regenexx provides this service in the U.S. and Cayman Islands. With theRegenexxstem cell injection procedure, a small bone marrow sample is extracted through a needle, and blood is drawn from a vein in the arm. These samples are processed in a laboratory, and the cells it contains are injected into an area of the body that needs repair. On June 19, 2018, ACAP Health, a leading provider in innovative, clinical-based solutions partnered with Regenexx to reduce high-cost musculoskeletal surgeries.ACAP Health is a national leader in employer healthcare expense reduction. It is one of the first healthcare groups to partner with a stem cell treatment group to support insurance coverage to patients.

A recent Twitter poll conducted by BioInformant reported that, on average, patients can expect to spend $25,000 or more on stem cell therapies. According to the poll,

Most likely, those paying lower stem cell treatment costs under $5,000 were pursuing treatment for orthopedic or musculoskeletal conditions. In contrast, those paying higher treatment costs were likely getting treated for systemic or more complex conditions, such as diabetes, multiple sclerosis (MS), neurodegenerative diseases (such as Alzheimers disease or dementia), psoriatic arthritis, as well as the treatment for autism.

In the U.S., treatment protocols vary depending on the clinic and the treating physician. A one-time treatment that utilizes blood drawn from a patient can cost as little as $1,500. However, protocols that utilize a bone marrow or adipose (fat) tissue extraction can run as much as $15,000 $30,000. This is because bone marrow extraction is an invasive procedure that requires a penetrating bone and adipose tissue extraction requires a medical professional trained in liposuction.

For treatments that require a systemic or whole-body approach, the cost tends to be in the higher range, often averaging from $20,000 to $30,000. Examples of the diseases or conditions requiring this type of stem cell treatment include:

These higher costs reflect the complexity of treating these patients and the fact that multiple treatments are often required.

Founded by Dr. Neil Riordan, a globally recognized stem cell expert, theStem Cell Institutein Panama is one of the worlds most trusted adult stem cell therapy centers. Over the past 12 years, the center has performed more than10,000 procedures, making it a widely recognized destination for stem cell treatments.

Working in collaboration with universities and physicians worldwide, its stem cell treatment protocols utilize combinations of allogeneic human umbilical cord blood stem cells and autologous bone marrow stem cells to treat a wide variety of conditions.

A reader of BioInformant was recently treated for psoriatic arthritis at the Stem Cell Institute in Panama in early 2018. The price of his stem cell treatment was $22,000. With travel and lodging included, the total expenses were approximately $30,000.

Because of its proximity to the U.S., Mexico is increasingly becoming a destination for medical tourism.Before choosing a stem cell treatment provider in Mexico, ensure the clinic is fully authorized by COFEPRIS, the Mexican equivalent to the FDA.

One patient who recently shared stem cell treatment quotes with BioInformant found that the treatment for glycogen storage disease, a metabolic disorder that often onsets in infancy and continues into adulthood, would cost $23,900 throughGIOSTAR Mexico.

In contrast, the patient was quoted$33,000 throughCelltex, a U.S.-based company that treats patients in Cancun, Mexico.Celltex follows FDA regulations concerning the export of cells to Mexico and is compliant with the standards and procedures of COFEPRIS. Celltex also has an alliance with a certified hospital in Mexico, which is approved to receive cells and administer them to patients by a licensed physician.

In contrast, the patient was quoted $10,000 from Stem Cell Therapy of Las Vegas and Med Spa, an American clinic. This price difference may reflect regulatory restrictions that prevent U.S. providers from expanding cells. It may also reflect the therapeutic approach used by the clinic, as well as the quality of their expertise.

In Mexico, where certain types of stem cell expansion are allowed that are restricted within the U.S., treatment protocols vary depending on the clinic and the treating physician. A one-time treatment that utilizes peripheral blood from a patient can cost as little as $1,000. In contrast, protocols that utilize more invasive sources of stem cells can run as much as $15,000 $35,000. Examples of invasive procedures includebone marrow and adipose tissue extraction. In some cases, hospitalization may be required, which raises costs. The location of a stem cell facility can factor heavily into thecost of the procedure.

Not every cost associated with treatment gets billed to the patient at the time of the procedure. Hidden costs such as reactions to the treatment, graft-versus-host disease, or disability derived from the treatment can all result in more money to the patient, to insurance, or to the government.

For example, in the case of someone with cancer, it frequently isnt viable to harvest the patients own stem cells because they may contain cancerous cells that can reintroduce tumors to the body. Instead, the patient would receive stem cells by transplant. Treatments that involve cells from another person are called allogeneic treatments. The danger here is that the body may see those cells as invaders and attack them via the immune system, a condition known as graft-versus-host disease (GvHD). The body (host) and the introduced stem cells (graft) then battle rather than coexist.

Transplanted cells often face the risk of being rejected by their host; this article discusses the effect of plasma exchange on acute graft vs. host diseasehttps://t.co/cA3nzFntew

Katie Bunde (@kbuns76) May 29, 2018

In addition to making the stem cell treatments less effective or ineffective, GvHD can be deadly. Roughly30 to 60 percent ofhematopoieticstem cell and bone marrow transplantationpatients sufferfrom it, and of those, 50 percent eventually die. The hospital costs associated with it are substantial.

Another hidden cost is the potential to disrupt a system that formerly functioned adequately. The best current example of this isthe case of Doris Tyler, who received bilateral stem cell injections in her eyes from Drs.RobertHalpernand JamieWalraven of Stem Cell Center of Georgia. According to her, while her vision was failing, it was still good enough to perform various tasks, and now it is not. That means the cost increases for her, as well as potential insurance or disability claims (though again, insurance is unlikely to cover the specific consequences of this action).

Because of tight regulations surrounding stem cell procedures performed in the United States, many stem cell treatment providers provide both on-shore (U.S.-based) and offshore (international) treatment options.Depending on where a treatment is received, patients may have to pay travel, lodging,and miscellaneous expenditures.

For example, Regenexx offers treatments at a wide range of U.S. facilities using non-expanded stem cells. However, it also offers a laboratory-expanded treatment option at a site in the Cayman Islands, which can administer higher cell doses to patients by expanding the cells in culture within a laboratory.

Similarly, Okyanos (pronounced Oh key AH nos) offers treatments to patients at its Florida location and provides more involved stem cell procedures at its offshore site inGrand Bahama. It was founded in 2011 and is a stem cell therapy provider specializing in treatments for congestive heart failure (CHF) and other chronic conditions. It is fully licensed under the Bahamas Stem Cell Therapy and Research Act and adheres to U.S. surgical center standards.

Similarly, Celltex is headquartered in Houston, Texas, but offers stem cell treatments in Cancun, Mexico. Celltex specializes in storing a patients mesenchymal stem cells (MSCs) for therapeutic use.

While no hard evidence yet points to stem cell clinics raising their rates as a result of lawsuits, that is a typical response in industries whose products or services the public perceives as a high risk.

An additional danger to stem cell treatment providers,points out Nature, is the reduction of bottom-line profits through former patients winning suits. If clinics have to pay out the money they earned and then some to individuals suing for damages, they may soon become faced with an unviable business model. That is a definite concern for those hoping to leverage these treatments now and in the future.

As with any other area of medicine, patient evaluations of stem cell providers and treatments run the gamut from extremely satisfied to desolately unhappy. Those like Doris Tyler who have lost their eyesight exist at the negative end of the spectrum. However, many others praise stem cell treatments for their power to heal diseases, boost immunity, fight cancer, and more.

For example, BioInformants Founder and President, Cade Hildreth, had a favorable experience with stem cell therapy. Cade had bone marrow-derived stem cells collected and then had them re-injected into the knee to treat a devastating orthopedic injury. Cade was able to reverse pain, swelling, and scarring to reclaim an elite athletic ability.

As of now, this much is clear. There exists enough interest in America and across the world that stem cell providers are continuing to offer a wide range of treatments. Stem cell treatments also offer the potential to reverse diseases that traditionally had to be chronically managed by drugs. Like most medical practices, stem cell treatments will require further testing to reveal merits and faults. Until then, the public will likely continue to pursue services when medical needs arise.

Although the cost of stem cell therapy is pricey, some patients choose to undergo the treatment because it is more economical than enduring the costs associated with chronic diseases.

Although most stem cell therapy providers do not provide FDA-approved procedures, the Food and Drug Administration (FDA) continues to encouragepatients to pursue approved therapies, even if there is a higher associated treatment cost.

Providers rarely post their prices for stem cell treatments in print or digital media because they want patients to understand the benefits of therapy before making a price decision. Additionally, the price of stem cell treatments varies by condition, the number of treatments required, and the complexity of the procedure, factors that can make it difficult for medical providers to provide cost estimates without a diagnostic visit for the patient. However, in many cases, it is not in the patients best interest to make treatment decisions based on the cost of stem cell therapy. The best way to know whether to pursue stem cell therapy is to explore patient outcomes by condition and compare the healing process to other surgical and non-surgical treatment options.

The cost of stem cell therapy is indeed expensive, especially because the procedures are rarely covered by health insurance. However, with the right knowledge and a clear understanding of the treatment process, the risk of undergoing stem cell therapy can be worth it, especially if it removes the requirement for a lifetime of prescription medication. Although stem cell therapy has associated risks, it has improved thousands of lives and will continue to play in a key role in the future of modern medicine.

Download this infographic for your reference:

Are you seeking a stem cell treatment? If so, we have partnered with GIOSTAR to help you acccess medical guidance and advice.

In alignment with what we believe at BioInformant, GIOSTARs goal is to offer cutting-edge, extensively researched stem cell therapy options designed to rejuvenate and improve a patients quality of life.

Click here to Schedule a Consultation or ask GIOSTAR a question.

If you found this blog valuable, subscribe to BioInformants stem cell industry updates.

As the first and only market research firm to specialize in the stem cell industry, BioInformant research is cited by The Wall Street Journal, Xconomy, AABB, and Vogue Magazine. Bringing you breaking news on an ongoing basis, we encourage you to join more than half a million loyal readers, including physicians, scientists, executives, and investors.

Do you think the cost of stem cell therapy is too much? Share your thoughts in the comments section below.

Up Next: Japan to Supply Human Embryonic Stem Cells (hESC) for Clinical Research

Cost Of Stem Cell Therapy And Why Its So Expensive

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Somatic cell nuclear transfer – Wikipedia

February 8th, 2019

In genetics and developmental biology, somatic cell nuclear transfer (SCNT) is a laboratory strategy for creating a viable embryo from a body cell and an egg cell. The technique consists of taking an enucleated oocyte (egg cell) and implanting a donor nucleus from a somatic (body) cell. It is used in both therapeutic and reproductive cloning. Dolly the Sheep became famous for being the first successful case of the reproductive cloning of a mammal.[1] In January 2018, a team of scientists in Shanghai announced the successful cloning of two female crab-eating macaques (named Zhong Zhong and Hua Hua) from fetal nuclei.[2] "Therapeutic cloning" refers to the potential use of SCNT in regenerative medicine; this approach has been championed as an answer to the many issues concerning embryonic stem cells (ESC) and the destruction of viable embryos for medical use, though questions remain on how homologous the two cell types truly are.

Somatic cell nuclear transfer is a technique for cloning in which the nucleus of a somatic cell is transferred to the cytoplasm of an enucleated egg. When this is done, the cytoplasmic factors affect the nucleus to become a zygote. The blastocyst stage is developed by the egg which helps to create embryonic stem cells from the inner cell mass of the blastocyst.[3] The first animal that was developed by this technique was Dolly, the sheep, in 1996.[4]

The process of somatic cell nuclear transplant involves two different cells. The first being a female gamete, known as the ovum (egg/oocyte). In human SCNT (Somatic Cell Nuclear Transfer) experiments, these eggs are obtained through consenting donors, utilizing ovarian stimulation. The second being a somatic cell, referring to the cells of the human body. Skin cells, fat cells, and liver cells are only a few examples. The nucleus of the donor egg cell is removed and discarded, leaving it 'deprogrammed.' What is left is a somatic cell and an denucleated egg cell. These are then fused by inserting the somatic cell into the 'empty' ovum.[5] After being inserted into the egg, the somatic cell nucleus is reprogrammed by its host egg cell. The ovum, now containing the somatic cell's nucleus, is stimulated with a shock and will begin to divide. The egg is now viable and capable of producing an adult organism containing all the necessary genetic information from just one parent. Development will ensue normally and after many mitotic divisions, this single cell forms a blastocyst (an early stage embryo with about 100 cells) with an identical genome to the original organism (i.e. a clone).[6] Stem cells can then be obtained by the destruction of this clone embryo for use in therapeutic cloning or in the case of reproductive cloning the clone embryo is implanted into a host mother for further development and brought to term.

Somatic cell nuclear transplantation has become a focus of study in stem cell research. The aim of carrying out this procedure is to obtain pluripotent cells from a cloned embryo. These cells genetically matched the donor organism from which they came. This gives them the ability to create patient specific pluripotent cells, which could then be used in therapies or disease research.[7]

Embryonic stem cells are undifferentiated cells of an embryo. These cells are deemed to have a pluripotent potential because they have the ability to give rise to all of the tissues found in an adult organism. This ability allows stem cells to create any cell type, which could then be transplanted to replace damaged or destroyed cells. Controversy surrounds human ESC work due to the destruction of viable human embryos. Leading scientists to seek an alternative method of obtaining stem cells, SCNT is one such method.

A potential use of stem cells genetically matched to a patient would be to create cell lines that have genes linked to a patient's particular disease. By doing so, an in vitro model could be created, would be useful for studying that particular disease, potentially discovering its pathophysiology, and discovering therapies.[8] For example, if a person with Parkinson's disease donated his or her somatic cells, the stem cells resulting from SCNT would have genes that contribute to Parkinson's disease. The disease specific stem cell lines could then be studied in order to better understand the condition.[9]

Another application of SCNT stem cell research is using the patient specific stem cell lines to generate tissues or even organs for transplant into the specific patient.[10] The resulting cells would be genetically identical to the somatic cell donor, thus avoiding any complications from immune system rejection.[9][11]

Only a handful of the labs in the world are currently using SCNT techniques in human stem cell research. In the United States, scientists at the Harvard Stem Cell Institute, the University of California San Francisco, the Oregon Health & Science University,[12] Stemagen (La Jolla, CA) and possibly Advanced Cell Technology are currently researching a technique to use somatic cell nuclear transfer to produce embryonic stem cells.[13] In the United Kingdom, the Human Fertilisation and Embryology Authority has granted permission to research groups at the Roslin Institute and the Newcastle Centre for Life.[14] SCNT may also be occurring in China.[15]

In 2005, a South Korean research team led by Professor Hwang Woo-suk, published claims to have derived stem cell lines via SCNT,[16] but supported those claims with fabricated data.[17] Recent evidence has proved that he in fact created a stem cell line from a parthenote.[18][19]

Though there has been numerous successes with cloning animals, questions remain concerning the mechanisms of reprogramming in the ovum. Despite many attempts, success in creating human nuclear transfer embryonic stem cells has been limited. There lies a problem in the human cell's ability to form a blastocyst; the cells fail to progress past the eight cell stage of development. This is thought to be a result from the somatic cell nucleus being unable to turn on embryonic genes crucial for proper development. These earlier experiments used procedures developed in non-primate animals with little success.

A research group from the Oregon Health & Science University demonstrated SCNT procedures developed for primates successfully using skin cells. The key to their success was utilizing oocytes in metaphase II (MII) of the cell cycle. Egg cells in MII contain special factors in the cytoplasm that have a special ability in reprogramming implanted somatic cell nuclei into cells with pluripotent states. When the ovum's nucleus is removed, the cell loses its genetic information. This has been blamed for why enucleated eggs are hampered in their reprogramming ability. It is theorized the critical embryonic genes are physically linked to oocyte chromosomes, enucleation negatively affects these factors. Another possibility is removing the egg nucleus or inserting the somatic nucleus causes damage to the cytoplast, affecting reprogramming ability.

Taking this into account the research group applied their new technique in an attempt to produce human SCNT stem cells. In May 2013, the Oregon group reported the successful derivation of human embryonic stem cell lines derived through SCNT, using fetal and infant donor cells. Using MII oocytes from volunteers and their improved SCNT procedure, human clone embryos were successfully produced. These embryos were of poor quality, lacking a substantial inner cell mass and poorly constructed trophectoderm. The imperfect embryos prevented the acquisition of human ESC. The addition of caffeine during the removal of the ovum's nucleus and injection of the somatic nucleus improved blastocyst formation and ESC isolation. The ESC obtain were found to be capable of producing teratomas, expressed pluripotent transcription factors, and expressed a normal 46XX karyotype, indicating these SCNT were in fact ESC-like.[12] This was the first instance of successfully using SCNT to reprogram human somatic cells. This study used fetal and infantile somatic cells to produce their ESC.

In April 2014, an international research team expanded on this break through. There remained the question of whether the same success could be accomplished using adult somatic cells. Epigenetic and age related changes were thought to possibly hinder an adult somatic cells ability to be reprogrammed. Implementing the procedure pioneered by the Oregon research group they indeed were able to grow stem cells generated by SCNT using adult cells from two donors aged 35 and 75, indicating that age does not impede a cell's ability to be reprogrammed.[20][21]

Late April 2014, the New York Stem Cell Foundation was successful in creating SCNT stem cells derived from adult somatic cells. One of these lines of stem cells was derived from the donor cells of a type 1 diabetic. The group was then able to successfully culture these stem cells and induce differentiation. When injected into mice, cells of all three of the germ layers successfully formed. The most significant of these cells, were those who expressed insulin and were capable of secreting the hormone.[22] These insulin producing cells could be used for replacement therapy in diabetics, demonstrating real SCNT stem cell therapeutic potential.

The impetus for SCNT-based stem cell research has been decreased by the development and improvement of alternative methods of generating stem cells. Methods to reprogram normal body cells into pluripotent stem cells were developed in humans in 2007. The following year, this method achieved a key goal of SCNT-based stem cell research: the derivation of pluripotent stem cell lines that have all genes linked to various diseases.[23] Some scientists working on SCNT-based stem cell research have recently moved to the new methods of induced pluripotent stem cells. Though recent studies have put in question how similar iPS cells are to embryonic stem cells. Epigenetic memory in iPS affects the cell lineage it can differentiate into. For instance, an iPS cell derived from a blood cell will be more efficient at differentiating into blood cells, while it will be less efficient at creating a neuron.[24] This raises the question of how well iPS cells can mimic the gold standard ESC in experiments, as stem cells are defined as having the ability to differentiate into any cell type. SCNT stem cells do not pose such a problem and continue to remain relevant in stem cell studies.

This technique is currently the basis for cloning animals (such as the famous Dolly the sheep),[25] and has been theoretically proposed as a possible way to clone humans. Using SCNT in reproductive cloning has proven difficult with limited success. High fetal and neonatal death make the process very inefficient. Resulting cloned offspring are also plagued with development and imprinting disorders in non-human species. For these reasons, along with moral and ethical objections, reproductive cloning in humans is proscribed in more than 30 countries.[26] Most researchers believe that in the foreseeable future it will not be possible to use the current cloning technique to produce a human clone that will develop to term. It remains a possibility, though critical adjustments will be required to overcome current limitations during early embryonic development in human SCNT.[27][28]

There is also the potential for treating diseases associated with mutations in mitochondrial DNA. Recent studies show SCNT of the nucleus of a body cell afflicted with one of these diseases into a healthy oocyte prevents the inheritance of the mitochondrial disease. This treatment does not involve cloning but would produce a child with three genetic parents. A father providing a sperm cell, one mother providing the egg nucleus, and another mother providing the enucleated egg cell.[10]

In 2018, the first successful cloning of primates using somatic cell nuclear transfer, the same method as Dolly the sheep, with the birth of two live female clones (crab-eating macaques named Zhong Zhong and Hua Hua) was reported.[2][29][30][31][32]

Interspecies nuclear transfer (iSCNT) is a means of somatic cell nuclear transfer used to facilitate the rescue of endangered species, or even to restore species after their extinction. The technique is similar to SCNT cloning which typically is between domestic animals and rodents, or where there is a ready supply of oocytes and surrogate animals. However, the cloning of highly endangered or extinct species requires the use of an alternative method of cloning. Interspecies nuclear transfer utilizes a host and a donor of two different organisms that are closely related species and within the same genus. In 2000, Robert Lanza was able to produce a cloned fetus of a gaur, Bos gaurus, combining it successfully with a domestic cow, Bos taurus.[33]

Interspecies nuclear transfer provides evidence of the universality of the triggering mechanism of the cell nucleus reprogramming. For example, Gupta et al.,[34] explored the possibility of producing transgenic cloned embryos by interspecies somatic cell nuclear transfer (iSCNT) of cattle, mice, and chicken donor cells into enucleated pig oocytes. Moreover, NCSU23 medium, which was designed for in vitro culture of pig embryos, was able to support the in vitro development of cattle, mice, and chicken iSCNT embryos up to the blastocyst stage. Furthermore, ovine oocyte cytoplast may be used for remodeling and reprogramming of human somatic cells back to the embryonic stage.[35]

SCNT can be inefficient. Stresses placed on both the egg cell and the introduced nucleus in early research were enormous, resulting in a low percentage of successfully reprogrammed cells. For example, in 1996 Dolly the sheep was born after 277 eggs were used for SCNT, which created 29 viable embryos. Only three of these embryos survived until birth, and only one survived to adulthood.[25] As the procedure was not automated, but had to be performed manually under a microscope, SCNT was very resource intensive. The biochemistry involved in reprogramming the differentiated somatic cell nucleus and activating the recipient egg was also far from understood. However, by 2014, researchers were reporting success rates of 70-80% with cloning pigs[36] and in 2016 a Korean company, Sooam Biotech, was reported to be producing 500 cloned embryos a day.[37]

In SCNT, not all of the donor cell's genetic information is transferred, as the donor cell's mitochondria that contain their own mitochondrial DNA are left behind. The resulting hybrid cells retain those mitochondrial structures which originally belonged to the egg. As a consequence, clones such as Dolly that are born from SCNT are not perfect copies of the donor of the nucleus. This fact may also hamper the potential benefits of SCNT-derived tissues and organs for therapy, as there may be an immunoresponse to the non-self mtDNA after transplant.

Proposals to use nucleus transfer techniques in human stem cell research raise a set of concerns beyond the moral status of any created embryo. These have led to some individuals and organizations who are not opposed to human embryonic stem cell research to be concerned about, or opposed to, SCNT research.[38][39][40]

One concern is that blastula creation in SCNT-based human stem cell research will lead to the reproductive cloning of humans. Both processes use the same first step: the creation of a nuclear transferred embryo, most likely via SCNT. Those who hold this concern often advocate for strong regulation of SCNT to preclude implantation of any derived products for the intention of human reproduction,[41] or its prohibition.[38]

A second important concern is the appropriate source of the eggs that are needed. SCNT requires human egg cells, which can only be obtained from women. The most common source of these eggs today are eggs that are produced and in excess of the clinical need during IVF treatment. This is a minimally invasive procedure, but it does carry some health risks, such as ovarian hyperstimulation syndrome.

One vision for successful stem cell therapies is to create custom stem cell lines for patients. Each custom stem cell line would consist of a collection of identical stem cells each carrying the patient's own DNA, thus reducing or eliminating any problems with rejection when the stem cells were transplanted for treatment. For example, to treat a man with Parkinson's disease, a cell nucleus from one of his cells would be transplanted by SCNT into an egg cell from an egg donor, creating a unique lineage of stem cells almost identical to the patient's own cells. (There would be differences. For example, the mitochondrial DNA would be the same as that of the egg donor. In comparison, his own cells would carry the mitochondrial DNA of his mother.)

Potentially millions of patients could benefit from stem cell therapy, and each patient would require a large number of donated eggs in order to successfully create a single custom therapeutic stem cell line. Such large numbers of donated eggs would exceed the number of eggs currently left over and available from couples trying to have children through assisted reproductive technology. Therefore, healthy young women would need to be induced to sell eggs to be used in the creation of custom stem cell lines that could then be purchased by the medical industry and sold to patients. It is so far unclear where all these eggs would come from.

Stem cell experts consider it unlikely that such large numbers of human egg donations would occur in a developed country because of the unknown long-term public health effects of treating large numbers of healthy young women with heavy doses of hormones in order to induce hyperovulation (ovulating several eggs at once). Although such treatments have been performed for several decades now, the long-term effects have not been studied or declared safe to use on a large scale on otherwise healthy women. Longer-term treatments with much lower doses of hormones are known to increase the rate of cancer decades later. Whether hormone treatments to induce hyperovulation could have similar effects is unknown. There are also ethical questions surrounding paying for eggs. In general, marketing body parts is considered unethical and is banned in most countries. Human eggs have been a notable exception to this rule for some time.

To address the problem of creating a human egg market, some stem cell researchers are investigating the possibility of creating artificial eggs. If successful, human egg donations would not be needed to create custom stem cell lines. However, this technology may be a long way off.

SCNT involving human cells is currently legal for research purposes in the United Kingdom, having been incorporated into the Human Fertilisation and Embryology Act 1990.[42][5] Permission must be obtained from the Human Fertilisation and Embryology Authority in order to perform or attempt SCNT.

In the United States, the practice remains legal, as it has not been addressed by federal law.[43] However, in 2002, a moratorium on United States federal funding for SCNT prohibits funding the practice for the purposes of research. Thus, though legal, SCNT cannot be federally funded.[44] American scholars have recently argued that because the product of SCNT is a clone embryo, rather than a human embryo, these policies are morally wrong and should be revised.[45]

In 2003, the United Nations adopted a proposal submitted by Costa Rica, calling on member states to "prohibit all forms of human cloning in as much as they are incompatible with human dignity and the protection of human life."[46] This phrase may include SCNT, depending on interpretation.

The Council of Europe's Convention on Human Rights and Biomedicine and its Additional Protocol to the Convention for the Protection of Human Rights and Dignity of the Human Being with regard to the Application of Biology and Medicine, on the Prohibition of Cloning Human Being appear to ban SCNT of human beings. Of the Council's 45 member states, the Convention has been signed by 31 and ratified by 18. The Additional Protocol has been signed by 29 member nations and ratified by 14.[47]

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Somatic cell nuclear transfer - Wikipedia

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Parkinson’s Glossary: The Michael J. Fox Foundation …

February 8th, 2019

Acetylcholinesterase inhibitors

A class of drugs used to treat mild to moderate dementia in Parkinson's disease. These drugs increase brain levels of a neurotransmitter called acetylcholine, which helps neurons communicate with each other and is involved in memory, learning and thinking.

Cellular Therapies Section Subsections – AABB

February 8th, 2019

Groups called subsections allow members of the CT Section to focus on specific topic areas. Subsections work to identify challenges and develop materials to meet the needs of the field. Section participation is open to all AABB individual members who may enroll in any subsection(s) they choose.

CT Spanish LanguageCord BloodCT Asia Pacific GroupCT ManagementCT Product Collection and Clinical PracticesCT Product Manufacturing and TestingCT Quality OperationsCT Regulatory AffairsNovel Therapies and CT Product Development

This group is for Spanish-speaking members or those members located in Spanish-speaking regions. Members will be able to join live discussions and participate with fellow SLS members in regular subsection meetings held at 'convenient' times for the zones encompassed in the Latin America region. The SLS will address CT issues related to cord blood; donor qualification; manufacturing; storage and transport challenges; quality operations; regulatory issues; and development. The group will also address specific regional issues for developing programs. All interested individual AABB members who speak Spanish and would like to share their CT interests, insights and expertise in may join. (Meets 3rd Wednesdays at 10:00am ETmonthly)

La Subseccin en Espaol (SLS) ofrece a todos sus miembros la oportunidad de conocer e interconectarse con otros profesionales hispanohablantes. Los miembros pueden formar parte de discusiones estimulantes con otros colegas de la subseccin durante las reuniones regulares. Los convenientes horarios de estas reuniones han sido establecidos para acomodar a miembros localizados en Latinoamrica. El SLS abarcar temas de Terapia Celular (CT) relacionados con cordn umbilical; requisitos para donacin de productos celulares; retos en la manufactura, almacenamiento y transporte de productos de terapia celular; operaciones de calidad y temas especficos para el desarrollo de otros programas regionales. Todos los miembros hispanohablantes de AABB que deseen compartir sus intereses, visiones y experiencias pueden ser parte de esta subseccin. (Se rene el tercer mircoles de cada mes a las 10:00 am ET)

This group works on topics such as donor issues for public banking (recruitment, consent, screening/testing), manufacturing, storage and transport challenges, licensure, international issues, and private and family banking issues. (Meets 1st Thursdays at 1:00pm ET monthly)

For members located in the Asia-Pacific region, a designated group called the Asia Pacific Group or APG is available. Members will be able to join live discussions and directly participate with fellow APG members in regular subsection meetings held at 'convenient' times for the zones encompassed in this region. Countries in the region include Australia, China, Guam, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Qatar, Singapore, South Korea, Sri Lanka, Taiwan, Thailand and Vietnam.

CT issues related to cord blood, donor qualification, manufacturing, storage and transport challenges, as well as quality operations, regulatory, development and specific regional issues will be addressed. The APG meeting time is tailored to those in the Asia-Pacific; however, all interested individual AABB members who would like to share their CT interests, insights and expertise may join. (Meets 2nd Wednesdays, monthly at 0400 UTC coordinated universal time)

This group works on topics such as reimbursement issues (Centers for Medicare/Medicaid (CMS), Food and Drug Administration (FDA)), funding sources for cell therapy development and clinical trials - federal and other public sources, venture capital, charitable donations (disease advocacy groups), as well as the administrative business (e.g. budgets, human resources , workload recording, cost accounting, job descriptions, staffing models, personnel management/project management, strategic planning, Lean/process engineering tools, expense reduction initiatives and cost containment) of cell therapy production. (Meets 2nd Tuesdays at 12:00pm ET bimonthly)

This group focuses primarily on clinical topics associated with the collection, transport, utilization and outcomes of cellular therapy products obtained from peripheral blood by apheresis, bone marrow, cord blood and other sources by the use of new technologies. Clinical topics include donor and recipient screening, eligibility, mobilization and collection, informed consent, product administration and infusion-related adverse events. (Meets 2nd Mondays at 1:00pm ET monthly)

This group works on technical topics and operational aspects related to the manufacturing and testing of CT products such as cryopreservation, cell separation and selection, automation, product characterization, assay development, validation and implementation. (Meets 3rd Thursdays at 11:00am ET monthly)

This group works on topics such as Quality Program design, risk assessment and risk management, vendor and supply qualification, facility, environmental and operational controls. (Meets 3rd Thursdays at 2:00pm ET monthly)

This group works on US and international topics involving regulations, guidance and policies from a variety of sources. Examples include FDA, Health Canada, European Medicine Evaluation Agency (EMEA), Office for Human Research Protections (OHRP), NIH, Regulatory Affairs Certification (RAC), and Health Resources and Services Agency (HRSA). (Meets3rd Tuesdays at 11:00am ET monthly)

This group works on topics such as 'new' research and preclinical studies, new devices for manipulating cells as well as later-stage cellular product development, validation, and technology transfer for clinical production. Examples include developments in the areas of induced pluripotent cells (iPS cells), tissue-derived cells, genetic engineering, structural materials, and biomaterials to name a few. (Meets 2nd Thursdays at 12:00pm ET monthly)

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Cellular Therapies Section Subsections - AABB

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Stem Cell Therapy in Thailand – Beike Biotech – Hospitals

February 5th, 2019

TREATMENT:hRPE stem cells implantation (human Retinal Pigment Epithelial cells, (adult stem cells) by stereotactic brain injection + nutritious stem cell cocktail treatment (intravenous).

START OF TREATMENT:March 6, 2007.

BEFORE THE TREATMENT: Lindas main symptoms were rigidity and stiffness in the left side of her body. She had mild tremors mainly in her left hand and had difficulty grasping small objects or holding things with her fingers. She would drag her left leg while walking and while at rest the

muscles in her leg and tows would contract. During the night her muscles would contract constantly keeping her regularly from having more than few hours sleep. Her muscles were very weak and she would tire very quickly, her posture was stooped and she suffered from a general tenseness and stiffness in her face, neck and back.

Without the affect of the medications she could not turn her neck and should turn her whole body in order to look back. Every morning, before the medications started to influence, it was difficult getting dressed, getting out of bed or taking a shower.

Before the treatment Linda took her medications every 2-3 hours (Contam 250mg x 8 times a day). One hour after taking the medications Lindas symptoms were hardly noticed, but the medications influence wear out quickly and Lindas every activity was dependant on her next dose of medications.

During the last few years Lindas short term memory was affected up to a level that she quit her job in human resources. Her hand writing was affected too even after taking the medications, it was still very scratchy and hard to read.

Linda also suffered from general anxiety and depression.

AFTER THE TREATMENT:

Lindas first notable change after the surgery was a full night sleep - the first one in 5 years. Within 5 weeks after the stem cell implantation most of Lindas symptoms were gradually gone. Her fingers got their flexibility back and the tremors were gone she could now grasp things, open a door and articulate more precise movements with her fingers.

The cramps in her leg were gone and she stopped dragging her left leg.

I dont need to think anymore about every movement, as I did before she says.

Her muscle tension was significantly reduced, she felt more relaxed and stronger than before.

Her posture became more open and she could now turn her neck more easily. Before leaving the hospital Linda still had some weakness in her muscles but she felt that she is getting stronger every day.

Linda also noticed that her sense of smell and taste that were greatly weakened during the last years were coming back.

A major change in her quality of life was that now her symptoms were unnoticeable with almost half the dosage of the medications she used to take before. Linda is now taking medications 4 times a day (Sinemet 200mg X4 times a day) instead of 8 times of double dosage that she used to take before the treatment.

I was a watch keeper, I used to watch at the clock all the time, I stopped swimming riding bicycle and other activities because I never knew when the medications affect will wear out she says.

Linda hopes that her medications could be gradually reduced even more, and she will keep a close contact with her doctors in China in order to follow up with her condition.

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Stem Cell Therapy in Thailand - Beike Biotech - Hospitals

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Stem Cell Therapy for ALS Patients

February 3rd, 2019

Learn about what stem cells are, why they are important and how they are going to revolutionize healing and medical care in Canada.

Not all conditions are effectively treated by PRP injections or stem cell therapy, and with ongoing clinical trials its important to realize what stem cells can and cannot help with. Weve built a comprehensive list of the different types of conditions that stem cell therapy shows promise for, however if you dont find it listed wed recommend checking outDanish health website Doc24.dk. Regular maintenance of health is key to making sure long-term issues dont arise as we age, and part of that is a rich, balanced diet and careful supplementation.

Research on human embryos in general, and stem cell research in particular, has been the subject of public debate in Canada since the late 1980s. In 2002, the Canadian Institute of Health Research (CIHR) issued guidelines for research on human embryonic stem cell lines, which have been revised and reissued several times since 2005 (most recently in 2007). These guidelines regulate the allocation of state funds in the field of research on human embryonic stem cells and concern both the handling of existing stem cell lines and the establishment of new stem cell lines.

The guidelines specify a number of important conditions that must be fulfilled in order for research projects to be eligible for funding. These include, but are not limited to:

The Stem Cell Oversight Committee (SCOC) was set up to ensure that research projects comply with the provisions of the Directive and to address the complex ethical issues surrounding research projects. Any project applying for government funding in the field of stem cell research must first be positively evaluated by the SCOC.

In addition to the regulation of state funding, the Assisted Human Reproduction Act came into force in 2004, which broadly regulates the field of reproductive medicine. Unlike the guidelines of the CIHR, it is not merely a guideline for state funding of certain research activities, but a law that places certain activities under state control and generally prohibits others. Research on human embryos is one of the controlled activities of the Assisted Human Reproduction Act. According to 8 Para. 3, the approval according to 10 Para. 2 requires the consent of the donor after clarification of the intended use. The Assisted Human Reproduction Agency of Canada (AHRAC), established by law, is responsible for granting authorisations and monitoring research activities.

The extraction of ES cells also falls under this section and is therefore permitted in Canada. The use of in vitro embryos for research purposes, including the derivation of stem cells, is subject to the following conditions under the Assisted Human Reproduction Act:

The production of a human clone is prohibited according to 5 a Assisted Human Reproduction Act. This provision also includes so-called therapeutic cloning by nuclear transfer. According to 5 b, the creation of embryos for purposes other than the creation of a human being or the improvement of artificial reproduction procedures is also prohibited. The law does not apply to the handling of already established human embryonic stem cell lines.

The CBC news network and other media responded to Twitter posts and a YouTube live video about unapproved treatments that lately came up. Patients that suffer from chronic pain or disease could benefit from stem-cell therapies. Canadians who have been treated more open by their federal and other regulatory laws about unlicensed stem cell therapies are asking for the legalization or this procedure.

A new company now made it their mission to offer direct-to-customer opportunities for trainees and people in general which can mean a big advantage for a patient. Unproven stories about this training in marketing and science services are offering support for approved stem-cell professionals.

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Stem Cell Therapy for ALS Patients

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Stem Cell Therapy Has a Lot to OfferIt Just May Take Some …

January 24th, 2019

Stem Cell Therapy Has a Lot to OfferIt Just May Take Some Time to Get There

By: Ashwini Nagappan

In conversation with the New York Times, Dr. Shinya Yamanaka, the director of Kyoto Universitys Center for iPS Cell Research and Application and researcher at the Gladstone Institutes, illuminates the complexities and future of stem-cell research. Yamanaka was jointly awarded the 2012 Nobel Prize in Physiology or Medicine for reconfiguring adult cells back to their pluripotent states. These induced pluripotent stem cells, or iPS cells, have been used as treatments for conditions such as macular degeneration.

However, Yamanaka mentions that these treatments are temporarily suspended because of the possibility of mutations developing in the patients iPS cells. Cancer could be a potential outcome because the production of iPS cells increases the chance of mutations. Researchers are rigorously testing to make sure that there are no cancer-causing mutations and that the cells function as they should. In order to be certain that these cells are safe, they are transplantedinto mice or rats for about a year. Yamanaka approximates that only 100 lines would be needed to cover the Japanese population and 200 lines for the US population.

Yamanaka acknowledges that the potentialfor stem cells may have been too eagerlyanticipated as they can only remedy the small portion of diseases that are caused by a single cell failure such as heart failure. Stem cell therapy cannot target diseases caused by multiple types of cell failures. He mentions an alternative to iPS known as direct cellular reprogramming, which would be beneficial if the patient in question was elderly instead of a younger person, and if the area targeted was larger instead of a small wound.

In essence, Yamanaka highlights the need for an ethical consensus in order to understand how to move forward with advancing stem cell technology. Further, iPS cells are fairly young they are only tenyears old. For patients to be able to receive these treatments requires money and time. In the mean time, Yamanaka recommends arrivingat an ethical consensus onthe use of stem cells.

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FUJIFILM Cellular Dynamics to Establish New Facility for …

January 9th, 2019

FUJIFILM Cellular Dynamics to Establish New Facility for Production of

Human iPS Cell Therapy Applications

Fujifilm is investing in the cGMP-compliant facility located in Madison, Wisconsin with the goal to begin operations by March 2020

MADISON, Wis., January 3, 2019 -- FUJIFILM Cellular Dynamics, Inc. (FCDI), a US subsidiary of FUJIFILM Corporation (President: Kenji Sukeno) and a leading global developer and manufacturer of human induced pluripotent stem (iPS) cell technologies, has announced an investment of about $21 million to open a new cGMP-compliant*1 production facility with the goal of industrializing iPS cell manufacturing for regenerative medicine therapies. The facility will support FCDIs internal cell therapeutics pipeline and will also serve as a Contract Development and Manufacturing Organization (CDMO) for iPS cell products.

Regenerative medicine is a highly advanced treatment modality with the potential to improve the quality of life for patients. The field is quickly growing due to scientific and engineering advancements that can harness the potential of iPS cells.

To meet the growing demand for FCDIs iPS cell platform, the state-of-the-art production facility will have a flexible cell culturing design to serve production requirements of both industrial quantities of cells, and small, diverse batches, said Seimi Satake, Chairman and Chief Executive Officer of FCDI. By combining Fujifilms experience gleaned from the intricate process of manufacturing photographic film along with FCDIs knowledge of cell reprogramming, genetic engineering and cell differentiation, the facility is poised to address the complex manufacturing processes of cell therapies.

With the facility operational by March 2020, FCDI intends to accelerate the development of its internal pipeline to address unmet medical needs in areas such as age-related macular degeneration, retinitis pigmentosa, Parkinsons disease, heart diseases, and cancer.

To fulfill the promise of cell therapy, sophisticated techniques and expertise are required to culture, differentiate, and control the quality of cells. Fujifilm has broad expertise across the regenerative medicine field through its group companies including Japan Tissue Engineering Co., Ltd., FUJIFILM Wako Pure Chemical Corporation, and FUJIFILM Irvine Scientific, Inc.

FCDI will continue to leverage its technologies and knowledge of iPS cells -- working together with academic institutions and corporations around the world to advance the field of regenerative medicine with the hope of providing new therapies for patients. A recent milestone achievement in the field includes Fujifilms partnership with Cynata Therapeutics Limited*2 for its product candidate CYP-011, which utilized FCDIs iPS cells, and represents the first-ever time a clinical trial using an iPS cell-derived therapy has been completed.

In addition to its advancements in regenerative medicine, FCDI manufactures iPS cell products for public institutions, major pharmaceutical companies, and academia for the purpose of life science research.

About Fujifilm:

FUJIFILM Cellular Dynamics, Inc. (FCDI), is a leading developer and supplier of human cells used in discovery, toxicity testing and regenerative medicine applications. Leveraging technology that can be used to create induced pluripotent stem cells (iPSCs) and differentiated tissue-specific cells from any individual, FCDI is committed to advancing life science research and transforming the therapeutic development process in order to fundamentally improve human health. The companys inventoried iCell products and donor-specific MyCell Products are available in the quantity, quality, purity and reproducibility required for drug and cell therapy development. For more information, please visit: http://www.FujifilmCDI.com

FUJIFILM Holdings Corporation, Tokyo, Japan brings cutting-edge solutions to a broad range of global industries by leveraging its depth of knowledge and fundamental technologies developed in its relentless pursuit of innovation. Its proprietary core technologies contribute to the various fields including healthcare, graphic systems, highly functional materials, optical devices, digital imaging and document products. These products and services are based on its extensive portfolio of chemical, mechanical, optical, electronic and imaging technologies. For the year ended March 31, 2018, the company had global revenues of $23.0 billion, at an exchange rate of 106 yen to the dollar. Fujifilm is committed to environmental stewardship and good corporate citizenship. For more information, please visit: http://www.fujifilmholdings.com.

###

All product and company names herein may be trademarks of their registered owners.

Media Contact:

Christine Jackman

Fujifilm

(914) 789-8523

christine.jackman@fujifilm.com

*1 An abbreviation of current Good Manufacturing Practice. Refers to the most recent rules and regulations for manufacturing and quality control of pharmaceuticals and quasi-drugs as determined by the US Food and Drug Administration (FDA).

*2 In January 2017 Cynata executed a license option agreement with FUJIFILM Corporation of Japan for the development and commercialization of certain Cynata technology, including Cynatas lead induced pluripotent stem cell(iPSC)-derived therapeutic mesenchymal stem cell (MSC) product, CYP-001, forgraft-versus-host disease (GvHD). As part of the transaction, Fujifilm acquired an equity position in Cynata through the purchase of 8,088,403 ordinary shares in Cynata, leading to Fujifilm becoming the largest shareholder in the Company with an approximate 9% stake.

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FUJIFILM Cellular Dynamics to Establish New Production …

January 9th, 2019

- Accelerating the practical application of treatments that apply iPS cells towards the early industrialization of regenerative medicine- Making the high quality and highly efficient production of iPS cells a reality

January 4, 2019FUJIFILM Cellular Dynamics, Inc.

FUJIFILM Cellular Dynamics, Inc. (FCDI), a US subsidiary of FUJIFILM Corporation (President: Kenji Sukeno) and a leader in the development and manufacture of human induced pluripotent stem (iPS) cells and tissue-specific cells differentiated from iPS cells, will establish a new cGMP-compliant* production facility with an investment of about 21 million US dollars in order to enhance its production of iPS cells for cell therapy. The facility is scheduled to begin operations during fiscal year ending March 2020.FCDI will use the iPS cells produced at this facility to accelerate development of its regenerative medicine products. In addition, by also conducting contract development and manufacturing of iPS cells and iPS cell-derived differentiated cells, it will expand its business and scale to the industrial stage.

Regenerative medicine is drawing interest as a solution for unmet medical needs. There are high expectations for the practical application of treatments that utilize iPS cells, as these cells possess totipotency and the capacity for infinite reproduction, making it possible to produce a large volume of diverse cells. To fulfill the promise of cell therapy, sophisticated techniques and know-how are required to culture, induce differentiation in, and control the quality of cells.

FCDI will be establishing a new production facility equipped with cell culture facilities appropriate for the production of a large volume of cells, as well as culture facilities appropriate for small-scale, diverse production, and a system capable of highly precise cell quality analyses. By also harnessing world-class technologies for the initialization and induction of differentiation in iPS cells and Fujifilm's advanced engineering technology and image analysis technology, the facility will be capable of efficiently producing high-quality iPS cells.Going forward, FCDI will use the high-quality iPS cells produced at this facility to accelerate the development of regenerative medicine products in the areas of age-related macular degeneration, retinitis pigmentosa, Parkinson's disease, heart diseases, and cancer. FCDI will also contribute to the realization and spread of treatments that utilize iPS cells by widely conducting the contract development and manufacturing of iPS cells and iPS cell-derived differentiated cells.

Currently, FCDI provides iPS cells and iPS cell-derived differentiated cells to public institutions, major pharmaceutical companies, and academia including the California Institute for Regenerative Medicine** and the National Heart, Lung, and Blood Institute*** while accelerating the development of its regenerative medicine products. FCDI will continue to harness its accumulated data, technologies, and know-how related to iPS cells, working together with academic institutions and corporations around the world and utilize the technologies and know-how of Fujifilm group companies including Fujifilm, Japan Tissue Engineering Co., Ltd., FUJIFILM Wako Pure Chemical Corporation, and Irvine Scientific Sales Company, Inc. to further expand its iPS cell-based business and contribute to the elevation of regenerative medicine business to the industrial stage.

Overview of the New Facility

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Cellular Therapy – fujifilmcdi.com

October 19th, 2018

iPSCells Represent a Superior Approach

iPS cell-derived cardiomyocyte patch demonstrates spontaneous and synchronized contractions after 4 days in culture.

One of the greatest promises of human stem cells is to transform these early-stage cells into treatments for devastating diseases. Stem cells can potentially be used to repair damaged human tissues and to bioengineer transplantable human organs using various technologies, such as 3D printing. Using stem cells derived from another person (allogeneic transplantation) or from the patient (autologous transplantation), research efforts are underway to develop new therapies for historically difficult to treat conditions. In the past, adult stem and progenitor cells were used, but the differentiation of these cell types has proven to be difficult to control. Initial clinical trials using induced pluripotent stem (iPS) cells indicate that they are far superior for cellular therapy applications because they are better suited to scientific manipulation.

CDIs iPS cell-derived iCell and MyCell products are integral to the development of a range ofcell therapyapplications. A study using iCell Cardiomyocytesas part of a cardiac patch designed to treat heart failure is now underway. This tissue-engineered implantable patch mayemerge as apotential myocardial regeneration treatment.

Another study done with iPS cell-derived cells and kidney structures has marked an important first step towards regenerating, and eventually transplanting, a functioning human organ. In this work, iCell Endothelial Cellswere used to help to recapitulatethe blood supply of a laboratory-generated kidney scaffold. This type of outcome will be crucial for circulation and nutrient distribution in any rebuilt organ.

iCell Endothelial Cells revascularize kidney tissue. (Data courtesy of Dr. Jason Wertheim, Northwestern University)

CDI and its partners are leveraging iPS cell-derived human retinal pigment epithelial (RPE) cells to develop and manufacture autologous treatments for dry age-related macular degeneration (AMD). The mature RPE cells will be derivedfrom the patients own blood cells using CDIs MyCell process. Ifapproved by the FDA, this autologous cellular therapy wouldbe one of the first of its kind in the U.S.

Learn more about the technologybehind the development of these iPScell-derived cellular therapies.

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Cellular Therapy - fujifilmcdi.com

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Kotton Lab – Boston University Medical Campus | Boston …

October 2nd, 2018

The Kotton labs goal is advancing our understanding of lung disease and developmental biology with a focus on stem cell biology and gene therapy. We believe that novel treatments for many lung diseases can be realized based on a better understanding of how the lung develops as well as regenerates after lung injury.

We are particularly interested in understanding how lung cells decide and remember who they are. To this end, one focus of our group is defining the genomic and epigenomic programs that regulate lung cell fate. A longer term goal is the de novo generation of the full diversity of lung lineages and transplantable 3D lung tissues from pluripotent stem cells. Our Principal Investigator, Dr. Darrell Kotton, also serves as the founding Director of the Center for Regenerative Medicine (CReM). Take a full tour of the CReM by clicking on our logo above.

Click on the menu to learn more about our research areas and our team

Have forty five minutes for an overview of our last decade? Listen here to Darrells ATS Discovery Series Lecture, Lung Regeneration: An Achievable Mission.

Open Source Works! Click here to access our:iPS Cell Lines, Lentiviral Vectors, Bioinformatics Datasets, or Detailed Protocols!

or read more about our Open Source Biology Philosophyor a recent interview on Darrells approach to sharing our cells

More photos of lab life

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Market Players Developing iPS Cell Therapies – BioInformant

September 17th, 2018

1. Cellular Dynamics International, Owned by FujiFilm Holdings

Founded in 2004 and listed on NASDAQ in July 2013, Cellular Dynamics International (CDI) is headquartered in Madison, Wisconsin. The company is known for its extremely robust patent portfolio containing more than 900 patents.

According to the company, CDI is the worlds largest producer of fully functional human cells derived from induced pluripotent stem (iPS) cells.[1] Their trademarked, iCell Cardiomyocytes, derived from iPSCs, are human cardiac cells used to aid drug discovery, improve the predictability of a drugs worth, and screen for toxicity. In addition, CDI provides: iCell Endothelial Cells for use in vascular-targeted drug discovery and tissue regeneration, iCell Hepatocytes, and iCell Neurons for pre-clinical drug discovery, toxicity testing, disease prediction, and cellular research.[2]

Induced pluripotent stem cells were first produced in 2006 from mouse cells and in 2007 from human cells, by Shinya Yamanaka at Kyoto University,[3] who also won the Nobel Prize in Medicine or Physiology for his work on iPSCs.[4] Yamanaka has ties to Cellular Dynamics International as a member of the scientific advisory board of iPS Academia Japan. IPS Academia Japan was originally established to manage the patents and technology of Yamanakas work, and is now the distributor of several of Cellular Dynamics products, including iCell Neurons, iCell Cardiomyocytes, and iCell Endothelial Cells.[5]

Importantly, in 2010 Cellular Dynamics became the first foreign company to be granted rights to use Yamanakas iPSC patent portfolio. Not only has CDI licensed rights to Yamanakas patents, but it also has a license to use Otsu, Japan-based Takara Bios RetroNectin product, which it uses as a tool to produce its iCell and MyCell products.[6]

Furthermore, in February 2015, Cellular Dynamics International announced it would be manufacturing cGMP HLA Superdonor stem cell lines that will support cellular therapy applications through genetic matching.[8] Currently, CDI has two HLA super donor cell lines that provide a partial HLA match to approximately 19% of the population within the U.S., and it aims to expand its master stem cell bank by collecting more donor cell lines that will cover 95% of the U.S. population.[9] The HLA super donor cell lines were manufactured using blood samples and used to produce pluripotent iPSC lines, giving the cells the capacity to differentiate into nearly any cell within the human body.

On March 30, 2015, Fujifilm Holdings Corporation announced that it was acquiring CDI for $307 million, allowing CDI to continue to run its operations in Madison, Wisconsin, and Novato, California as a consolidated subsidiary of Fujifilm.[14] A key benefit of the merger is that CDIs technology platform enables the production of high-quality fully functioning iPSCs (and other human cells) on an industrial scale, while Fujifilm has developed highly-biocompatible recombinant peptides that can be shaped into a variety of forms for use as a cellular scaffold in regenerative medicine when used in conjunction with CDIs products.[15]

Additionally, Fujifilm has been strengthening its presence in the regenerative medicine field over the past several years, including a recent A$4M equity stake in Cynata Therapeutics and an acquisition of Japan Tissue Engineering Co. Ltd. in December 2014. Most commonly called J-TEC, Japan Tissue Engineering Co. Ltd. successfully launched the first two regenerative medicine products in the country of Japan. According to Kaz Hirao, CEO of CDI, It is very important for CDI to get into the area of therapeutic products, and we can accelerate this by aligning it with strategic and technical resources present within J-TEC.

Kaz Hirao also states, For our Therapeutic businesses, we will aim to file investigational new drugs (INDs) with the U.S. FDA for the off-the-shelf iPSC-derived allogeneic therapeutic products. Currently, we are focusing on retinal diseases, heart disorders, Parkinsons disease, and cancers. For those four indicated areas, we would like to file several INDs within the next five years.

Finally, in September 2015, CDI again strengthened its iPS cell therapy capacity by setting up a new venture, Opsis Therapeutics. Opsis is focused on discovering and developing novel medicines to treat retinal diseases and is a partnership with Dr. David Gamm, the pioneer of iPS cell-derived retinal differentiation and transplantation.

In summary, several key events indicate CDIs commitment to developing iPS cell therapeutics, including:

Australian stem cell company Cynata Therapeutics (ASX:CYP) is taking a unique approach by creating allogeneic iPSC derived mesenchyal stem cell (MSCs) on a commercial scale. Cynatas Cymerus technology utilizes iPSCs provided by Cellular Dynamics International, a Fujifilm company, as the starting material for generating mesenchymoangioblasts (MCAs), and subsequently, for manufacturing clinical-grade MSCs. According to Cynatas Executive Chairman Stewart Washer who was interviewed by The Life Sciences Report, The Cymerus technology gets around the loss of potency with the unlimited iPS cellor induced pluripotent stem cellwhich is basically immortal.

On January 19, 2017, Fujifilm took an A$3.97 million (10%) strategic equity stake in Cynata, positioning the parties to collaborate on the further development and commercialization of Cynatas lead Cymerus therapeutic MSC product CYP-001 for graft-versus-host disease (GvHD). (CYP-001 is the product designation unique to the GVHD indication). The Fujifilm partnership also includes potential future upfront and milestone payments in excess of A$60 million and double-digit royalties on CYP-001 product net sales for Cynata Therapeutics, as well as a strategic relationship for the potential future manufacture of CYP-001 and certain rights to other Cynata technology.

One of the key inventors of Cynatas technology is Igor Slukvin, MD, Ph.D., Scientific Founder of Cellular Dynamics International (CDI) and Cynata Therapeutics. Dr. Slukvin has released more than 70 publications about stem cell topics, including the landmark article in Cell describing the now patented Cymerus technique. Dr. Slukvins co-inventor is Dr. James Thomson, the first person to isolate an embryonic stem cell (ESC) and one of the first people to create a human induced pluripotent stem cell (hiPSC). Dr. James Thompson was the Founder of CDI in 2004.

There are three strategic connections between Cellular Dynamics International (CDI) and Cynata Therapeutics, which include:

Recently, Cynata received advice from the UK Medicines and Healthcare products Regulatory Agency (MHRA) that its Phase I clinical trial application has been approved, titled An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease. It will be the worlds first clinical trial involving a therapeutic product derived from allogeneic (unrelated to the patient) induced pluripotent stem cells (iPSCs).

Participants for Cynatas upcoming Phase I clinical trial will be adults who have undergone an allogeneic haematopoietic stem cell transplant (HSCT) to treat a hematological disorder and subsequently been diagnosed with steroid-resistant Grade II-IV GvHD. The primary objective of the trial is to assess safety and tolerability, while the secondary objective is to evaluate the efficacy of two infusions of CYP-001 in adults with steroid-resistant GvHD.

Using Professor Yamanakas Nobel Prize-winning achievement of ethically uncontentious iPSCs and CDIs high-quality iPSCs as source material, Cynata has achieved two world firsts:

Cynata has also released promising pre-clinical data in Asthma, Myocardial Infarction (Heart Attack), and Critical Limb Ischemia.

There are four key advantages of Cynatas proprietary Cymerus MSC manufacturing platform. Because the proprietary Cymerus technology allows nearly unlimited production of MSCs from a single iPSC donor, there is batch-to-batch uniformity. Utilizing a consistent starting material allows for a standardized cell manufacturing process and a consistent cell therapy product. Unlike other companies involved with MSC manufacturing, Cynata does not require a constant stream of new donors in order to source fresh stem cells for its cell manufacturing process, nor does it require the massive expansion of MSCs necessitated by reliance on freshly isolated donations.

Finally, Cynata has achieved a cost-savings advantage through its unique approach to MSC manufacturing. Its proprietary Cymerus technology addresses a critical shortcoming in existing methods of production of MSCs for therapeutic use, which is the ability to achieve economic manufacture at commercial scale.

On June 22, 2016, RIKEN announced that it is resuming its retinal induced pluripotent stem cell (iPSC) study in partnership with Kyoto University.

2013 was the first time in which clinical research involving transplant of iPSCs into humans was initiated, led by Masayo Takahashi of the RIKEN Center for Developmental Biology (CDB) in Kobe, Japan. Dr. Takahashi and her team were investigating the safety of iPSC-derived cell sheets in patients with wet-type age-related macular degeneration. Although the trial was initiated in 2013 and production of iPSCs from patients began at that time, it was not until August of 2014 that the first patient, a Japanese woman, was implanted with retinal tissue generated using iPSCs derived from her own skin cells.

A team of three eye specialists, led by Yasuo Kurimoto of the Kobe City Medical Center General Hospital, implanted a 1.3 by 3.0mm sheet of iPSC-derived retinal pigment epithelium cells into the patients retina.[196] Unfortunately, the study was suspended in 2015 due to safety concerns. As the lab prepared to treat the second trial participant, Yamanakas team identified two small genetic changes in the patients iPSCs and the retinal pigment epithelium (RPE) cells derived from them. Therefore, it is major news that the RIKEN Institute will now be resuming the worlds first clinical study involving the use of iPSC-derived cells in humans.

According to the Japan Times, this attempt at the clinical study will involve allogeneic rather than autologous iPSC-derived cells for purposes of cost and time efficiency. Specifically, the researchers will be developing retinal tissues from iPS cells supplied by Kyoto Universitys Center for iPS Cell Research and Application, an institution headed by Nobel prize winner Shinya Yamanaka. To learn about this announcement, view this article from Asahi Shimbun, a Tokyo- based newspaper.

In November 2015 Astellas Pharma announced it was acquiring Ocata Therapeutics for $379M. Ocata Therapeutics is a biotechnology company that specializes in the development of cellular therapies, using both adult and human embryonic stem cells to develop patient-specific therapies. The companys main laboratory and GMP facility are in Marlborough, Massachusetts, and its corporate offices are in Santa Monica, California.

When a number of private companies began to explore the possibility of using artificially re-manufactured iPSCs for therapeutic purposes, one such company that was ready to capitalize on the breakthrough technology was Ocata Therapeutics, at the time called Advanced Cell Technology. In 2010, the company announced that it had discovered several problematic issues while conducting experiments for the purpose of applying for U.S. Food and Drug Administration approval to use iPSCs in therapeutic applications. Concerns such as premature cell death, mutation into cancer cells, and low proliferation rates were some of the problems that surfaced. [17]

As a result, the company shifted its induced pluripotent stem cell approach to producing iPS cell-derived human platelets, as one of the benefits of a platelet-based product is that platelets do not contain nuclei, and therefore, cannot divide or carry genetic information. While the companys Induced Pluripotent Stem Cell-Derived Human Platelet Program received a great deal of media coverage in late 2012, including being awarded the December 2012 honor of being named one of the 10 Ideas that Will Shape the Year by New Scientist Magazine,[178]. Unfortunately, the company did not succeed in moving the concept through to clinical testing in 2013.

Nonetheless, Astellas is clearly continuing to develop Ocatas pluripotent stem cell technologies involving embryonic stem cells (ESCs) and induced pluripotent stem cells (iPS cells). In a November 2015 presentation by Astellas President and CEO, Yoshihiko Hatanaka, he indicated that the company will aim to develop an Ophthalmic Disease Cell Therapy Franchise based around its embryonic stem cell (ESC) and induced pluripotent stem cell (iPS cell) technology. [19]

What other companies are developing iPSC derived therapeutics and products? Share your thoughts in the comments below.

BioInformant is the first and only market research firm to specialize in the stem cell industry. BioInformant research has been cited by major news outlets that include the Wall Street Journal, Nature Biotechnology, Xconomy, and Vogue Magazine. Serving Fortune 500 leaders that include GE Healthcare, Pfizer, and Goldman Sachs. BioInformant is your global leader in stem cell industry data.

Footnotes[1] CellularDynamics.com (2014). About CDI. Available at: http://www.cellulardynamics.com/about/index.html. Web. 1 Apr. 2015.[2] Ibid.[3] Takahashi K, Yamanaka S (August 2006). Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126 (4): 66376.[4] 2012 Nobel Prize in Physiology or Medicine Press Release. Nobelprize.org. Nobel Media AB 2013. Web. 7 Feb 2014. Available at: http://www.nobelprize.org/nobel_prizes/medicine/laureates/2012/press.html. Web. 1 Apr. 2015.[5] Striklin, D (Jan 13, 2014). Three Companies Banking on Regenerative Medicine. Wall Street Cheat Sheet. Retrieved Feb 1, 2014 from, http://wallstcheatsheet.com/stocks/3-companies-banking-on-regenerative-medicine.html/?a=viewall.%5B6%5D Striklin, D (2014). Three Companies Banking on Regenerative Medicine. Wall Street Cheat Sheet [Online]. Available at: http://wallstcheatsheet.com/stocks/3-companies-banking-on-regenerative-medicine.html/?a=viewall. Web. 1 Apr. 2015.[7] Cellular Dynamics International (July 30, 2013). Cellular Dynamics International Announces Closing of Initial Public Offering [Press Release]. Retrieved from http://www.cellulardynamics.com/news/pr/2013_07_30.html.%5B8%5D Investors.cellulardynamics.com,. Cellular Dynamics Manufactures Cgmp HLA Superdonor Stem Cell Lines To Enable Cell Therapy With Genetic Matching (NASDAQ:ICEL). N.p., 2015. Web. 7 Mar. 2015.[9] Ibid.[10] Cellulardynamics.com,. Cellular Dynamics | Mycell Products. N.p., 2015. Web. 7 Mar. 2015.[11]Sirenko, O. et al. Multiparameter In Vitro Assessment Of Compound Effects On Cardiomyocyte Physiology Using Ipsc Cells.Journal of Biomolecular Screening 18.1 (2012): 39-53. Web. 7 Mar. 2015.[12] Sciencedirect.com,. Prevention Of -Amyloid Induced Toxicity In Human Ips Cell-Derived Neurons By Inhibition Of Cyclin-Dependent Kinases And Associated Cell Cycle Events. N.p., 2015. Web. 7 Mar. 2015.[13] Sciencedirect.com,. HER2-Targeted Liposomal Doxorubicin Displays Enhanced Anti-Tumorigenic Effects Without Associated Cardiotoxicity. N.p., 2015. Web. 7 Mar. 2015.[14] Cellular Dynamics International, Inc. Fujifilm Holdings To Acquire Cellular Dynamics International, Inc.. GlobeNewswire News Room. N.p., 2015. Web. 7 Apr. 2015.[15] Ibid.[16] Cyranoski, David. Japanese Woman Is First Recipient Of Next-Generation Stem Cells. Nature (2014): n. pag. Web. 6 Mar. 2015.[17] Advanced Cell Technologies (Feb 11, 2011). Advanced Cell and Colleagues Report Therapeutic Cells Derived From iPS Cells Display Early Aging [Press Release]. Available at: http://www.advancedcell.com/news-and-media/press-releases/advanced-cell-and-colleagues-report-therapeutic-cells-derived-from-ips-cells-display-early-aging/.%5B18%5D Advanced Cell Technology (Dec 20, 2012). New Scientist Magazine Selects ACTs Induced Pluripotent Stem (iPS) Cell-Derived Human Platelet Program As One of 10 Ideas That Will Shape The Year [Press Release]. Available at: http://articles.latimes.com/2009/mar/06/science/sci-stemcell6. Web. 9 Apr. 2015.[19] Astellas Pharma (2015). Acquisition of Ocata Therapeutics New Step Forward in Ophthalmology with Cell Therapy Approach. Available at: https://www.astellas.com/en/corporate/news/pdf/151110_2_Eg.pdf. Web. 29 Jan. 2017.

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IPS and G-CON Launch iCON Cell Therapy Facility Platform

September 10th, 2018

BERcellFLEX24

COLLEGE STATION, Texas (PRWEB) September 05, 2018

Following up on the launch of the iCON Turnkey Facility Platform for a mAb manufacturing facility late last year, IPS-Integrated Project Services, LLC and G-CON Manufacturing have successfully designed and delivered the first BERcellFLEX PODs for the manufacturing of autologous cell therapies. The iCON solution provides a pre-fabricated modular cleanroom infrastructure for the drug manufacturers requirements for both clinical and commercial manufacture of critical therapies. Following the iCON model, IPS provided the engineering design while G-CON built, tested and delivered the BERcellFLEX CAR-T processing suites in both twelve (12) foot and twenty-four (24) foot wide POD configurations.

This is an exciting time for our companies as the iCON platform is being adopted by clients who recognize that new innovative approaches are needed to meet the growing demand for cell and gene therapy manufacturing said Dennis Powers, Vice President of Business Development and Sales Engineering at G-CON Manufacturing Inc. We believe that the iCON platform approach with its faster and more predictable project schedules for new facility construction are essential for supplying life changing therapies to the patients that need them.

The gene therapy industry needs standardized solutions to meet its speed to market requirements, said Tom J. Piombino, Vice President & Process Architect at IPS. In addition to our larger 2K mAb facility platform that we rolled out earlier this year, the BERcellFLEX12 and 24 represent a line of gene/cell therapy products that operating companies can buy today, ready-to-order, in either an open or closed-processing format with little to no engineering time we start fabricating almost immediately after URS alignment. Multiple cellFLEX units can be installed to scale up/out from Phase 1 Clinical production to Commercial Manufacturing and serve the needs of thousands of CAR-T patients per year. Being able to meet this critical need is consistent with our vision; were thrilled to be able to offer this modular solution to help our clients get therapies to their patients.

About iCONThe iCON platform, the collaborative efforts of IPS and G-CON Manufacturing, Inc., is redefining facility project execution for the biopharma industry where there is a growing need for more rapidly deployable and flexible manufacturing capability. iCON has launched turnkey designs for monoclonal antibody facilities and autologous cell therapies, and is developing platforms for cell and gene therapies, vaccines, OSD, and aseptic filling. An iCON solution can be deployed for:

About G-CONG-CON Manufacturing designs, produces and installs prefabricated cleanroom PODs. G-CONs cleanroom POD portfolio encompasses a variety of different dimensions and purposes, from laboratory environments to personalized medicine and production process platforms. The POD cleanroom units are unique from traditional cleanroom structures due to the ease of scalability, mobility and the ability to repurpose the PODs once the production process reaches the end of its lifecycle. For more information, please visit the Company's website at http://www.gconbio.com.

About IPSIPS is a global leader in developing innovative facility and bioprocess solutions for the biotechnology and pharmaceutical industries. Through operational expertise and industry-leading knowledge, skill and passion, IPS provides consulting, architecture, engineering, construction management, and compliance services that allow clients to create and manufacture life-impacting products around the world. Headquartered in Blue Bell, PA-USA, IPS is one of the largest multi-national companies servicing the life sciences industry with over 1,100 professionals in the US, Canada, Brazil, UK, Ireland, Switzerland, Singapore, China, and India. Visit our website at http://www.ipsdb.com.

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Induced Pluripotent Stem Cell (iPS Cell): 2018-2022 …

August 4th, 2018

Dublin, Aug. 02, 2018 (GLOBE NEWSWIRE) -- The "Global Induced Pluripotent Stem Cell (iPS Cell) Industry Report 2018-19" report has been added to ResearchAndMarkets.com's offering.

Groundbreaking experimentation in 2006 led to the introduction of induced pluripotent stem cells (iPSCs). These are adult cells which are isolated and then transformed into embryonic-like stem cells through the manipulation of gene expression, as well as other methods. Research and experimentation using mouse cells by Shinya Yamanaka's lab at Kyoto University in Japan was the first instance in which there was a successful generation of iPSCs.

In 2007, a series of follow-up experiments were done at Kyoto University in which human adult cells were transformed into iPSCs. Nearly simultaneously, a research group led by James Thomson at the University of Wisconsin-Madison accomplished the same feat of deriving iPSC lines from human somatic cells.

Since the discovery of iPSCs a large and thriving research product market has grown into existence, largely because the cells are non-controversial and can be generated directly from adult cells. While it is clear that iPSCs represent a lucrative product market, methods for commercializing this cell type are still being explored, as clinical studies investigating iPSCs continue to increase in number.

iPS Cell Therapies

2013 was a landmark year in Japan because it saw the first cellular therapy involving the transplant of iPS cells into humans initiated at the RIKEN Center in Kobe, Japan. Led by Masayo Takahashi of the RIKEN Center for Developmental Biology (CDB). Dr. Takahashi was investigating the safety of iPSC-derived cell sheets in patients with wet-type age-related macular degeneration.

Although the study was suspended in 2015 due to safety concerns, in June 2016 RIKEN Institute announced that it would resume the clinical study using allogeneic rather than autologous iPSC-derived cells, because of the cost and time efficiencies.

In a world-first, Cynata Therapeutics received approval in September 2016 to launch the world's first formal clinical trial of an allogeneic iPSC-derived cell product, called CYP-001. The study involves centers in the UK and Australia. In this trial, Cynata is testing an iPS cell-derived mesenchymal stem cell (MSC) product for the treatment of GvHD.

On 16 May 2018, Nature News then reported that Japan's health ministry gave doctors at Osaka University permission to take sheets of tissue derived from iPS cells and graft them onto diseased human hearts. The team of Japanese doctors, led by cardiac surgeon Yoshiki Sawa at Osaka University, will use iPS cells to create a sheet of 100 million heart-muscle cells. From preclinical studies in pigs, the medical team determined that thin sheets of cell grafts can improve heart function, likely through paracrine signaling.

Kyoto University Hospital in Kobe, Japan also stated it would be opening an iPSC therapy center in 2019, for purposes of conducting clinical studies on iPS cell therapies. Officials for Kyoto Hospital said it will open a 30-bed ward to test the efficacy and safety of the therapies on volunteer patients, with the hospital aiming to initiate construction at the site in February of 2016 and complete construction by September 2019.

iPS Cell Market Competitors

In 2009 ReproCELL, a company established as a venture company originating from the University of Tokyo and Kyoto University was the first to make iPSC products commercially available with the launch of its human iPSC-derived cardiomyocytes, which it called ReproCario.

Cellular Dynamics International, a Fujifilm company, is another major market player in the iPSC sector. Similar to ReproCELL, CDI established its control of the iPSC industry after being founded in 2004 by Dr. James Thomson at the University of Wisconsin-Madison, who in 2007 derived iPSC lines from human somatic cells for the first time ever (the feat was accomplished simultaneously by Dr. Shinya Yamanaka's lab in Japan).

A European leader within the iPSC market is Ncardia, formed through the merger of Axiogenesis and Pluriomics. Founded in 2001 and headquartered in Cologne, Germany, Axiogenesis initially focused on generating mouse embryonic stem cell-derived cells and assays. After Yamanaka's groundbreaking iPSC technology became available, Axiogenesis was the first European company to license and adopt Yamanaka's iPSC technology in 2010.

Ncardia's focus lies on preclinical drug discovery and drug safety through the development of functional assays using human neuronal and cardiac cells, although it is expanding into new areas. Its flagship offering is its Cor.4U human cardiomyocyte product family, including cardiac fibroblasts.

In summary, market leaders have emerged in all areas of iPSC development, including:

iPS Cell Commercialization

Key Findings

Key Topics Covered

1. SCOPE AND METHODOLOGY

2. EXECUTIVE SUMMARY

3. BACKGROUND - iPSC RESEARCH

4. MARKET ANALYSIS BY PRODUCT CATEGORY

5. MARKET ANALYSIS BY APPLICATION

6. MARKET ANALYSIS BY GEOGRAPHY

7. PATENTS

8. COMPANIES

9. COMPANY PROFILES

10. CONCLUSIONS

For more information about this report visit https://www.researchandmarkets.com/research/njhzjc/induced?w=12

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CTERP International Conference – 2018: About

July 11th, 2018

CTERP INTERNATIONAL CONFERENCEApril 11-13, 2018Moscow, Russia

In recent years there have been rapid advances in applying the discoveries in cell technologies field into medical practice. Cell technologies are progressing as the result of multidisciplinary effort of scientists, clinicians and businessmen,with clinical applications of manipulated stem cells combining developments in transplantation and gene therapy.Challenges address not only thetechnology itself but also compliancewith safety and regulatory requirements.

The Conference will provide a platform for scientists from basic and applied cell biology fields, practical doctors, and biotech companies to meet and share their experience, to discuss the research associated with developing biomedical clinical products and translating this research into novel clinical applications, challenges of such translational efforts and foundation of bioclusters assisting further developments in cell technology.

The official language of the conference is English.

Conference materials will be published in the Russian Journal of Developmental Biology.

Please download your abstracts in accordance with the journal guidelines (english, russian) for authors provided on their website.

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CTERP International Conference - 2018: About

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Stem Cell Treatment/Therapy COST in India| DheerajBojwani.Com

June 25th, 2018

Get your Stem Cell Treatment in India with Dheeraj Bojwani Consultants

Stem Cell treatment is an intricate process. Stem Cell transplant patients need utmost care with respect to both emotionally and physically. Dheeraj Bojwani Consultants is a prominent medical tourism company in India making world-class medical facilities from best surgeons and hospitals accessible for international patients looking for budget-friendly treatment abroad.

Mrs. Marilyn Obiora - Nigeria Stem Cell Therapy For her Daughter in India

Hi, my name is Mrs. Marilyn Obiora, and I am from Nigeria. I came to India for my daughter's Stem Cell Therapy in India. My daughter had her first stroke in 2011. She couldn't sit, talk and had lost control of her neck. We could not find suitable help for her condition and searched for treatment in India.

We sent a query to the dheerajbojwani.com and received fast reply. Within no time we were in India for my daughter's treatment. We are very pleased with the treatment offered and there has been serious improvement in her condition in just two weeks. Thanks to the Dheeraj Bojwani Consultants, my daughter is regaining proper body functions and recuperating well.

Medical science has come a long way since its practice began thousands of years ago. Scientists are finding superior and more resourceful ways to cure diseases of different organs. Stem cells are undifferentiated parent cells that can transform into specialized cell types, divide further and produce more stem cells of the same group. Stem Cell therapy is performed to prevent or treat a health condition. Stem Cell Treatment is a reproductive therapy where nourishing tissues reinstate damaged tissues for relief from incurable diseases. Stem cell treatment is one of the approaches with a potential to heal a wide range of diseases in the near future. Science has always provided ground-breaking answers to obdurate health conditions, but the latest medical miracle that the medical fraternity has gifted to mankind is the Stem Cell Therapy.

Stem cell therapy is an array of techniques intended to replace cells damaged or destroyed by disease with healthy functioning ones. Even though the techniques are relatively new, their applications and advantages are broad and surprising the medical world with every new research. Stem cells are obtained from bone marrow or human umbilical cord. They are also known as the fundamental cells of our body and have the power to develop into any type of tissue cell in the body. Stem cell treatment is based on the principle that the cells move to the site of injury and transform themselves to form new tissue cells to replace the damaged ones. They have the capacity to proliferate and renew themselves indefinitely and can form mature muscle cells, nerve cells, and blood cells. In this type of therapy, they are derived from the body, kept under artificial conditions where they mature into the type of cells that are required to heal a certain part of the body or disease.

Stem cells are being studied and used to treat different types of cancers, disorders related to the blood, immune disorders, and metabolic disorders. Some other diseases and health conditions that may be healed using stem cell treatment are,

Recently, a team of researchers successfully secured the peripheral nerves in the upper arms of a patient suffering peripheral nerve damage, by using skin-derived stem cells (SDSCs) and a previously developed collagen tube, premeditated to successfully bridge gaps in injured nerves.

A research has found potential in bone marrow stem cell therapy to treat TB. Patients injected with new mesenchymal stromal cells derived from their own bone marrow showed positive response against the TB bacteria. The therapy also didnt show any serious adverse effects.

Stem cells are also used to treat hair loss. A small amount of fat is taken from the waist area of the patient by a mini-liposuction process. This fat contains dormant stem cells, and is then spun to separate the stem cells from the fat. An activation solution is added to the cells, and may be multiplied in number, depending on the size of the bald area. Once activated, the solution is washed off so that only cells remain. Now, the stem cells are injected into the scalp. One can find some hair growth in about two to four weeks.

Damaged cones in retinas can be regenerated and eyesight restored through stem cell. Stem cell therapy could regenerate damaged cones in people, especially in the cone-rich regions of the retina that provide daytime/color vision.

Kidney transplants have become more common and easier thanks stem cell therapy. Normally patients who undergo organ transplants need a lifetime of costly anti-rejection drugs but the new procedure may negate this need, with organ donors stem cells. Unless there is a perfect match donor, patients have to wait long for an organ transplant. Though still in early stages, the stem cell research is being considered as a potential player in the field of transplantation.

Transplanted stem cells serve as migratory signals for the brain's own neurogenic cells, guiding the new host cells towards the injured brain tissue. Stem cells have the potential to give rise to many different cell types that carry out different functions. While the stem cells in adult bone marrow tend to develop into the cells that make up the organ system from which they originated. These multipotent stem cells can be manipulated to take up the characteristics of neural cells.

Experts are using Stem cell Transplant to treat the symptoms of spinal cord injury by transplantation of cells directly into the gray matter of the patients spinal cord. Expectedly, the cells will integrate into the patients own neural tissue and create new circuitry to help transmit nerve signals to muscles. The transplanted cells may also promote reorganization of the spinal cord segmental circuitry, possibly leading to improved motor function.

Stem cells are capable of differentiating into a variety of different cell types, and if the architecture of damaged tendon is restored, it would improve the management of patients with these injuries significantly.

A promising benefit of stem cell therapy is its potential for cardiac tissue regeneration to reverse tissue loss underlying the development of heart failure after cardiac injury. Possible mechanisms of recovery include generation of heart muscle cells, stimulation of new blood vessels growth, secretion of growth factors.

It is a complex and multifarious procedure, with several risks and complications involved and is thus recommended to a few patients when other treatments have failed. Stem Cell therapy is recommended when other treatments fail to give positive results. The best candidates for Stem cell Treatment are those in good health and have stem cells available from a sibling, or any other family member.

India has been recognized as the new medical destination for Stem Cell therapies. Hundreds of international patients from around the world visit to India for high quality medical care at par with developed nations like the US, UK, at the most affordable costs. The Hospitals in India have the most extensive diagnostic and imaging facilities including Asias most advanced MRI and CT technology. India provides services of the most leading doctors and Stem Cell Therapy professionals at reasonable cost budget in the following cities

India offers outstanding Stem Cell Treatment at rates far below that prevailing in USA or other Western countries. Even with travel expenses taken into account, the comprehensive medical tourism packages still provide a savings measured in the thousands of dollars for major procedures. A cost comparison can give you the exact idea about the difference:

There are many reasons for India becoming a popular medical tourism spot is the low cost stem cell treatment in the area. When in contrast to the first world countries like, US and UK, medical care in India costs as much as 60-90% lesser, that makes it a great option for the citizens of those countries to opt for stem cell treatment in India because of availability of quality healthcare in India, affordable prices strategic connectivity, food, zero language barrier and many other reasons.

The maximum number of patients for Stem Cell Treatment comes from Nigeria, Kenya, Ethiopia, USA, UK, Australia, Saudi Arabia, UAE, Uzbekistan, Bangladesh

Below are the downloadable links that will help you to plan your medical trip to India in a more organized and better way. Attached word and pdf files gives information that will help you to know India more and make your trip to India easy and memorable one.

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iPS Cell Therapy: Is Japan the Market Leader?

June 18th, 2018

Although there are key players in markets like the U.S., Australia, and the EU, Japan continues to accelerates its position as a hub for induced pluripotent stem cell (iPS cell) therapy with generous funding, acquisitions, and strategic partnerships.

Pluripotent stem cells are cells that are capable of developing into any type of cell or tissue in the human body. These cells have the capability to replicate and help in repairing damaged tissues within the body. In 2006, the Japanese scientist Shinya Yamanaka demonstrated that an ordinary cell can be turned into a pluripotent cell by genetic modification. These genetically reprogrammed cells are known as induced pluripotent cells, also called iPS cells or iPSCs.

An induced pluripotent stem cell (iPS cell) is a type of pluripotent stem cell that has the capacity to divide indefinitely and create any cell found within the three germ layers of an organism. These layers include the ectoderm (cells giving rise to the skin and nervous system), endoderm (cells forming gastrointestinal and respiratory tracts, endocrine gland, liver, and pancreas), and mesoderm (cells forming bones, cartilage, most of the circulatory system, muscles, connective tissues, and other related tissues.).

iPS cells have significant potential for therapeutic applications. For autologous applications, the cells are extracted from the patients own body, making them genetically identical to the patient and eliminating the issues associated with tissue matching and tissue rejection.

iPS cells have the potential to be used to treat a wide range of diseases, including diabetes, heart diseases, autoimmune diseases, and neural complications, such as Parkinsons disease, Alzheimers disease.

Over the past few years, Japan has accelerated its position as a hub for regenerative medicine research, largely driven by support from Prime Minister Shinzo Abe who has identified regenerative medicine and cellular therapy as key to the Japans strategy to drive economic growth.

The Prime Minister has encouraged a growing range of collaborations between private industry and academic partners through an innovative legal framework approved last fall.

He has also initiated campaigns to drive technological advances in drugs and devices by connecting private companies with public funding sources. The result has been to drive progress in both basic and applied research involving induced pluripotent stem cells (iPS cells) and related stem cell technologies.

2013 was a landmark year in Japan, because it saw the first cellular therapy involving transplant of iPS cells into humans initiated at the RIKEN Center in Kobe, Japan.[1]Led by Masayo Takahashi of theRIKEN Center for Developmental Biology (CDB).Dr. Takahashi and her team wereinvestigating the safety of iPSC-derived cell sheets in patients with wet-type age-related macular degeneration.

To speed things along, RIKEN did not seek permission for a clinical trial involving iPS cells, but instead applied for a type of pretrial clinical research allowed under Japanese regulations.The RIKEN Center is Japans largest, most comprehensive research institution, backed by both Japans Health Ministry and government.

This pretrial clinical research allowed the RIKEN research team to test the use of iPS cells for the treatment of wet-type age-related macular degeneration (AMD) on a very small scale, in only a handful of patients.Unfortunately, the study was suspended in 2015 due to safety concerns. As the lab prepared to treat the second trial participant, Yamanakas team identified two small genetic changes in the patients iPSCs and the retinal pigment epithelium (RPE) cells derived from them.

However, in June 2016 RIKEN Institute announced that it would be resuming the clinical study involving the use of iPSC-derived cellsin humans.According to theJapan Times, this second attempt at the clinical studyis using allogeneic rather than autologous iPSC-derived cells, because of the greater cost and time efficiencies.

Specifically,the researchers will be developing retinal tissues from iPS cells supplied by Kyoto Universitys Center for iPS Cell Research and Application, an institution headed by Nobel prize winner Shinya Yamanaka.

Japan has a unique affection for iPS cells, as the cells were originally discovered by the Japanese scientist, Shinya Yamanaka of Kyoto University. Mr. Yamanaka was awarded the Nobel Prize in Physiology or Medicine for 2012, an honor shared jointly with John Gurdon, for the discovery that mature cells can be reprogrammed to become pluripotent.

In addition, Japans Education Ministry said its planning to spend 110 billion yen ($1.13 billion) on induced pluripotent stem cell research during the next 10 years, and the Japanese parliament has been discussing bills that would speed the approval process and ensure the safety of such treatments.[3]

In April, Japanese parliament even passed a law calling for Japan to make regenerative medical treatments like iPSC technology available for its citizens ahead of the rest of the world.[4] If those forces were not enough, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan, who is heading the worlds first clinical research using iPSCs in humans, was also chosen by the journal Natureas one of five scientists to watch in 2014.[5]

Clearly, Japan is the global leader in iPS cell technologies and therapies. However, progress with stem cells has not been without setbacks within Japan, including a recent scandal at the RIKEN Institute that involved falsely manipulated research findings and a hold on the first clinical trial involving transplant of an iPS cell product into humans.

Nonetheless, Japan has emerged from these troubles to become the most liberalized nation pursuing the development of iPS cell products and services.

iPS cells represent one of the most promising advances within the field of stem cell research, because of their diverse ability to differentiate into any of the approximately 200 cell types that compose the human body.

Even though there is growing evidence to support the safety of iPS cells within cell therapy applications,some people remain concerned that patients who receive implants of iPS derived cells might be at risk of cancer, as genetic manipulation is required to create the cell type.

In a world-first, Cynata Therapeutics (ASX:CYP) received approval in September 2016 to launch a clinical trial in the UK with the worlds first first formal clinical trial of an allogeneic iPSC-derived cell product, which it calls CYP-001.The study involves centers in both the UK and Australia.

In this landmark trial, the Australian regenerative medicine company is testing an iPS cell-derived mesenchymal stem cell (MSC) product for the treatment of Graft-vs-Host-Disease (GvHD).Not surprisingly, the Japanese conglomerate Fujifilm is also involved with this historic trial.

Headquartered in Tokyo, Fujifilm is one of the largest players in regenerative medicine field and has invested significantly into stem cells through their acquisition of Cellular Dynamics International (CDI). Additionally, Fujifilm has invested in Japan Tissue Engineering Co. Ltd. (J-Tec), giving it a broad base in regenerative medicine across multiple therapeutic areas.

For a young company like Cynata, having validation from an industry giant like Fujifilm is a huge boost. As stated by Cynata CEO, Dr. Ross Macdonald, The decision by Fujifilm confirms that our technology is very exciting in their eyes. It is a useful yardstick for other investors as well. Of course, the effect of the relationship with Fujifilm on our balance sheet is also important.

If Fujifilm exercises their option to license Cynatas GvHD product, then the costs of the product and commercialization will become the responsibility of Fujifilm. Cynata would also receive milestone payments from Fujifilm of approximately $60M AUS and a double-digit royalty payment.

Cynata was also the first to scale-up manufacture of an allogeneic cGMP iPS celll line. It sourced the cell line from Cellular Dynamics International (CDI) when CDI was still an independent company listed on NASDAQ. In April 2015, CDI was subsequently acquired by Fujifilm, who as mentioned, is a major shareholder in Cynata and its strategic partner for GvHD.

Although Cynata is showing promising early-stage data from its GvHD trial, methods for commercializing iPS cells are still being explored and clinical studies investigating iPS cells remain extremely low in number.

Footnotes[1] Dvorak, K. (2014).Japan Makes Advance on Stem-Cell Therapy [Online]. Available at: http://online.wsj.com/news/articles/SB10001424127887323689204578571363010820642. Web. 14 Apr. 2015.[2] Note: In the United States, some patients have been treated with retina cells derived from embryonic stem cells (ESCs) to treat macular degeneration. There was a successful patient safety test for this stem cell treatment last year that was conducted at the Jules Stein Eye Institute in Los Angeles. The ESC-derived cells used for this study were developed by Advanced Cell Technology, Inc, a company located in Marlborough, Massachusetts.[3] Dvorak, K. (2014).Japan Makes Advance on Stem-Cell Therapy [Online]. Available at: http://online.wsj.com/news/articles/SB10001424127887323689204578571363010820642. Web. 8 Apr. 2015.[4] Ibid.[5] Riken.jp. (2014).RIKEN researcher chosen as one of five scientists to watch in 2014 | RIKEN [Online]. Available at: http://www.riken.jp/en/pr/topics/2014/20140107_1/. Web. 14 Apr. 2015.

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Cell Therapy Companies – BioInformant

June 16th, 2018

Cell therapy companies have been rapidly populating over the past few years, making the cell therapy market a high-value, fast-growth market. Key drivers for the market include high rates of cell therapy clinical trials, accelerated pathways for cell therapy product approvals, new technologies to support cell therapy manufacturing, and the potential for cell therapies to revolutionize healthcare.

Additionally, the market gained recent momentum when the Swiss pharmaceutical giant Novartis made history as the first company to win FDA approval for a CAR-T cell therapy in the U.S. in August 2017 (Kymriah).In October 2017, Kite Pharma became the second company to get FDA approval of a CAR-T cell therapy (Yescarta).

These historic events demonstrate to investors, the public and funding providers alike that cell therapy is a market that has emerged, no longer one that is evolving in the future.Today, there are nearly 40 companies developing redirected T cells or NK cells for therapeutic use. There are nearly 70 companies developing stem cell therapeutics (45% of all cell therapy companies). Finally, direct cell reprogramming is gaining popularity as a therapeutic strategy, because of its safety and efficacy advantages.

Because of this rapid market growth, BioInformant has released a global database featuring 150+ cell therapy companies worldwide. It was originally developed in-house for our own purposes, but we have had more and moreclients requesting access to it. For this reason, we updated and expanded it with additional company details. Now, we have officially launched it to the public.

Cell Therapy Companies CAR-T, CAR-NK, Stem Cells, Direct Reprogramming

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iPS Cell Therapy – Parent Project Muscular Dystrophy

June 15th, 2018

iPS Cells and Therapeutic Applications for Duchenne

We are currently in the optimization/validation phase of pre-clinical development.

This research is being done in the lab of Dr. Rita Perlingeiro at the University of Minnesota, in partnership with the University of Minnesota Center for Translational Medicine and the Molecular and Cellular Therapeutics Facility. This work is currently funded by the Department of Defense (DoD).

Induced pluripotent stem cells (iPS) are adult cells that have been reprogrammed to an embryonic stem cell-like state.There has been tremendous excitement for the therapeutic potential of iPS cells in treating genetic diseases. Our current research builds on our successful proof-of-principle studies for Duchenne performed with mouse wild-type and dystrophic iPS cells as well as control (healthy) human iPS cells. These studies demonstrate equivalent functional myogenic engraftment to that observed with their embryonic counterparts following their transplantation into dystrophic mice.

Our goal now is to apply this technology to clinical grade GMP-compliant iPS cells, and generate a cell product, iPS-derived myogenic progenitors, that can be delivered to muscular dystrophy patients.

Optimization of methodology, characterization of cell product, scalability with GMP-compliant method, followed by safety and efficacy studies. Once these have been achieved, we will be ready to move into a clinical trial.

2-3 years (it depends largely on how much funding we have available to conduct these studies).

University of Minnesota

In the first phase, adults with confirmed diagnosis of Duchenne (> 18 years old).

You can learn more about this research at the website for Dr. Perlingeiros lab: http://www.med.umn.edu/lhi/research/PerlingeiroLab/index.htm

http://www.ClinicalTrials.gov will post all clinical trials once they are actively recruiting patients.

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New Jersey Stem Cell Therapy – Stem Cell Center Of NJ

April 15th, 2018

COPD

Over 32 million Americans suffer from chronic obstructive pulmonary disease (also known as COPD). COPD is a progressive lung disease, however regenerative medicine, such as lung regeneration therapies using stem cells are showing potential for COPD by encouraging tissue repair and reducing inflammation to the diseased lung tissue.

Following up with stem cell therapy and exome therapy immediately in the first 36 to 48 hours after stroke symptoms surface has proven to be crucial to long-term recovery and regaining mobility again. Cell therapy also calms post-stroke inflammation in the body, and reduces risk of serious infections.

Parkinsons is a neurodegenerative brain disorder caused by the gradual loss of dopamine-producing cells in the brain. It afflicts more than 1 million people in the U.S., and currently, there is no known cure. Stem cell therapies have been showing incredible progress. Using induced pluripotent stem (iPS) cells, a mature cell can be reprogrammed into an embryonic-like, healthy and highly-functioning state, which has the potential to become a dopamine-producing cell in the brain.

A thick, full head of hair is possible, naturally! Stem cell and exosome therapy promotes healing from within to naturally stimulate hair follicles, which encourages new hair growth. Using your own stem cells, Platelet Rich Plasma (PRP) and exosomes, you can regrow your own healthy, thick hair naturally and restore your confidence!

Erectile Dysfunction (ED) is the inability to achieve or maintain an erection sufficient for satisfactory sexual intercourse. Regenerative medicine offers a non-surgical option that commonly uses the patients own stem cells, exosomes, and other sources of growth factors to regenerate healthy tissue to improve performance and sensation.

If chronic joint pain is derailing your active lifestyle, then youre not alone. Regenerative medicine offers a non-surgical option that commonly uses the patients own stem cells, exosomes, and other sources of growth factors to reduce inflammation, promote natural healing and regenerate healthy tissue surrounding the joint for relief.

Multiple Sclerosis (MS) affects 400,000 people in the U.S., and occurs when the body has an abnormal immune system response and attacks the central nervous system. Regenerative medicine now offers treatment for MS with stem cell therapy, which is an exciting and rapidly developing field of therapy. Stem cells work to repair damaged cells these new cells can become replacement cells to restore normal functionality.

Spinal cord injuries are as complex as they are devastating. Today, cellular treatments, usually a combination of therapies, such as stem cell, Platelet Rich Plasma (PRP) and exosome therapy with growth factors are showing promise in contributing to spinal cord repair and reducing inflammation at the site of injury.

If you have chronic nerve injury pain that doesnt fade, your health care provider may recommend surgery to reverse the damage. However, regenerative medicine offers a non-surgical option to repair damaged tissue and reduce inflammation at the site of injury. Stem cell therapy commonly uses the patients own stem cells, exosomes, and other sources of growth factors to regenerate healthy tissue.

Neuropathy also called peripheral neuropathy occurs when nerves are damaged and cant send messages from the brain and spinal cord to the muscles, skin and other parts of the body. Simply put, the two areas stop communicating. Stem cell and exosome therapies treat damaged nerves affected by neuropathy, and they have the ability to replicate and create new, healthy cells, while repairing damaged tissue.

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Stem Cell Therapy for Duchenne Muscular Dystrophy …

March 16th, 2018

Duchenne muscular dystrophy (DMD) is the most common and serious form of muscular dystrophy. One out of every 3500 boys is born with the disorder, and it is invariably fatal. Until recently, there was little hope that the widespread muscle degeneration that accompanies this disease could be combated.

However, stem cell therapy now offers that hope. Like other degenerative disorders, DMD is the result of loss of cells that are needed for correct functioning of the body. In the case of DMD, a vital muscle protein is mutated, and its absence leads to progressive degeneration of essentially all the muscles in the body.

To begin to approach a therapy for this condition, we must provide a new supply of stem cells that carry the missing protein that is lacking in DMD. These cells must be delivered to the body in such a way that they will engraft in the muscles and produce new, healthy muscle tissue on an ongoing basis.

We now possess methods whereby we can generate stem cells that can become muscle cells out of adult cells from skin or fat by a process known as reprogramming. Reprogramming is the addition of genes to a cell that can dial the cell back to becoming a stem cell. By reprogramming adult cells, together with addition to them of a correct copy of the gene that is missing in DMD, we can potentially create stem cells that have the ability to create new, healthy muscle cells in the body of a DMD patient. This is essentially the strategy that we are developing in this proposal.

We start with mice that have a mutation in the same gene that is affected in DMD, so they have a disease similar to DMD. We reprogram some of their adult cells, add the correct gene, and grow the cells in incubators in a manner that will produce muscle stem cells. The muscle stem cells can be identified and purified by using an instrument that detects characteristic proteins that muscles make.

The corrected muscle stem cells are transplanted into mice with DMD, and the ability of the cells to generate healthy new muscle tissue is evaluated. Using the mouse results as a guide, a similar strategy will then be pursued with human cells, utilizing cells from patients with DMD. The cells will be reprogrammed, the correct gene added, and the cells grown into muscle stem cells. The ability of these cells to make healthy muscle will be tested by injection into mice with DMD that are immune-deficient, so they will accept a graft of human cells.

In order to make this process into something that could be used in the clinic, we will develop standard procedures for making and testing the cells, to ensure that they are effective and safe. In this way, this project could lead to a new stem cell therapy that could improve the clinical condition of DMD patients. If we have success with DMD, similar methods could be used to treat other degenerative disorders, and perhaps even some of the degeneration that occurs during normal aging

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Stem Cell Therapy for Duchenne Muscular Dystrophy ...

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