Dr. Steven Cyr has been treating patients using growth factors and stem cells contained in amniotic tissue and bone marrow aspirate to provide a potential for improved success with fusion procedures, when treating herniated discs, and for arthritic or damaged joints, with remarkable success. "The goal of any medical intervention is to yield improved outcomes with the ideal result of returning a patient to normal function, when possible," states Dr Cyr. He went on to elaborate that there are times when only a structural solution can solve problems related to spinal disorders, but even in that scenario, the use of stem cells or growth factors derived from stem cell products can possibly improve the success of surgical procedures. "I have patients previously unable to jog or run return to normal function and athletic ability after injections of growth factors and stem cell products into the knee joints, hip joints, and shoulder joints," he said. "This includes high-level athletes, professional dancers, and the average weekend warrior."

There may be promise in treating patients with spinal cord injury as well. SASpine CEO, LeAnn Cyr, states, "There are reports of patients gaining significant neurological improvement after being treated with stem cells." Dr Cyr continues, "Most patients with spinal cord injuries resulting from trauma also have mechanical pressure on the nerves that result either from bone fragments or disc material compressing the spinal cord that needs to be removed along with surgical stabilization of the spinal bones. There's significant potential that stem cells bring to the equation when treating these types of patients, and I am excited about the potential that these products offer to the host of treatments to address spinal conditions and arthritic joints."

For more information about SASpine's Stem Cell Treatment Program, visit http://www.saspine.com or call (210) 487-7463 in San Antonio or (832) 919-7990 in Houston.

Related Linkswww.facebook.com/saspinewww.instagram.com/surgical.associates.in.spine

SOURCE SASpine

https://www.saspine.com

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SASpine to offer Stem Cell Therapy - PRNewswire

Arthritis is an umbrella term for a number of conditions that cause swelling and tenderness of one or more of the joints. Osteoarthritis and rheumatoid arthritis are the two most common types of arthritis. There's no cure for arthritis, but there are many treatments that can help slow it down and maintain quality of life, including this unusual one.

Osteoarthritis mainly affects the hands, spine, knees and hips, whereas rheumatoid arthritis usually affects the hands, feet and wrists.

Experts are researching ways to use stem cells therapy to help treat arthritis in the knee and other joints.

Many doctors already use stem cell therapy to treat arthritis, but it is not considered standard practice, with some even calling the treatment controversial.

READ MORE: Joe Swash health: I had to learn everything again Actors scary virus

There is a lot of debate around stem cell treatment and it is helpful for potential patients to understand what stem cells are and the issues surrounding their use in arthritis therapy.

How the treatment could help is by reducing inflammation in the body.

In arthritis, the immune system mistakenly attacks the tissue that lines the joints, which causes pain, inflammation, swelling and stiffness.

By reducing inflammation, stem cell therapy increases the presence of healthy cells in the body.

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And it would seem that stem cell therapy has sparked some interest from the royal family.

Princess Michael of Kent, the wife of the Queens cousin Prince Michael, recently appeared in a slick promotional video for a clinic in the Bahamas that offers similar joint therapy.

Look at that, the Princess declares proudly in the five-minute film, holding her right arm high above her head after stem-cell injections for a painful shoulder.

"Its amazing I think stem cells are the future, theres no doubt in my mind."

A stem cell is a type of cell not specialised to perform a specific role.

Instead, it has the unique ability to develop into one of many different types of cell. Stem cell therapy uses stem cells to replace dead and diseased cells within the body.

The human body contains over 200 different types of cell. Usually, each type has certain characteristics that allow it to preform a specific role.

Cells with similar roles group together to form tissues, which then organise to form the bodys organs.

Scientists source the stem cells from body tissue, either from an embryo or an adult human and isolate them in the laboratory.

After manipulating the cells to develop into specific types, they then inject the cells into the recipients blood or tissue.

Researchers are still investigating ways of using stem cells to control inflammation and regenerate damaged tissues.

Mesenchymal stem cells (MSCs) are types of stem cell that can develop into cartilage and bone. Synovial MSC therapy involves injecting these cells directly int the tissues surrounding the affected joints.

Some research shows that MSCs are also able to suppress the immune system and reduce the bodys inflammatory response.

This makes MSC therapy a promising treatment option for autoimmune conditions such as arthritis.

But for all the positive evidence supporting the treatment, it remains hugely controversial.

Most specialists say there is little robust evidence to show it works and it has not been approved by the UK medicines regulator, the National Institute for Health and Care Excellence. Leading health experts most standard approaches to treating arthritis is advising patients to lose weight and get more exercise.

In the US, Google has banned all advertising for products that contain stem cells or another regenerative therapy which uses blood cells, known as platelet-rich plasma, or PRP, because regulators describe the treatments as "new and exploratory".

And in the UK, experts have rubbished claims that Lipogems can treat arthritis.

Professor Chinmay Gupte, consultant orthopaedic surgeon and senior lecturer in knee surgery at Imperial College London, warns: These are extremely expensive treatments, and largely unproven. The problem is when youre desperate, youll try anything.

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Arthritis treatment: A controversial treatment promoted by a certain royal could help - Express

Natural Killer cells have been engineered to attack blood cancers with excellent provisional results ... [+] from a small clinical trial.

A new cell-based immunotherapy for some types of blood cancer has posted promising initial results in a small clinical trial on patients who had exhausted all other treatment options.

The new study, led by researchers from MD Anderson Cancer Center was published yesterday in the New England Journal of Medicine and used a type of immune cell, called a Natural Killer (NK) cell. The NK cells were engineered to target a protein called CD19 found on B-lymphoblasts and which can become cancerous and cause several types of blood cancer. The study tested the treatment on 11 patients with either chronic lymphocytic leukemia (CLL) or non-Hodgkins lymphoma (NHL), finding a 73% response rate. Of the 8 people who responded, 7 maintained a complete response over a year after the initial treatment.

All our patients had failed conventional therapies and therefore there was no alternative treatment available for them, said said Katy Rezvani, M.D., Ph.D.,lead author of the paper and professor of Stem Cell Transplantation & Cellular Therapy at MD Anderson. We are encouraged by the results of the clinical trial, which will launch further clinical studies to investigate allogeneic cord blood-derived CAR NK cells as a potential treatment option for patients in need, she added.

Most cell based therapies use a different type of modified immune cell; CAR T-cells. These therapies have shown initial promise in some types of blood cancer, but there have been several setbacks with side-effects in patients and the scaleability and cost of the technology. Importantly, the NK cells used on the patients in the new study were made from donated umbilical cord blood, whereas most CAR T-cell therapies currently rely on a long and expensive process of extracting T-cells from the patient themselves, genetically modifying them and expanding the cells before the therapy is ready for use.

This means the new NK cell therapy can theoretically be produced in bulk and doesn't rely on extracting T-cells from the patient, which can be incredibly difficult, especially if the patient has received a lot of previous therapies which can affect T-cell numbers.

Strictly speaking, the manufacturing and engineering steps for CAR T and CAR NK cells are very similar. The main difference is that unlike commercial CAR T-cells, where one product is used to treat one patient (an autologous product),CAR NK cells are not patient specific, allowing for multiple doses to be manufactured from one donor that can then be used to treat multiple patients, said Rezvani.

CAR T-cell therapies, although posting some wonderful results, particularly in children with hard-to-treat, relapsed leukemias, do come with a lot of side effects, particularly neurotoxicity and cytokine release syndrome, which is life-threatening if not quickly treated. These toxicities were not seen in this initial, small trial, giving the researchers hope that perhaps this therapy may have fewer serious side-effects than other similar approaches.

As well as CAR T-cells, there is also another therapy already available which targets CD19, a drug called blinatumomab (Blincyto). What potential advantages does the new NK cell therapy have over this approach?

These are living cells that persist after infusion and will potentially continue to protect the patient from their cancer over time unlike blinatumomab that needs to be given as a continuous infusion and in multiple cycles, said Revzani. In addition, with the caveat of the small number of patients that we have treated so far and the relatively short follow up time, our approach appears to be less toxic, she added.

MD Anderson have licenced the development of this therapy and other similar B-cell targeting therapies to company Takeda Pharmaceuticals.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly and with minimal toxicities, said Rezvani, adding that the team also plans to expand the trial to encompass other CD19-expressing malignancies such as B-cell leukemias.

Continued here:
New Immunotherapy Had Positive Results In Cancer Patients After Other Treatments Failed - Forbes

A retired Texan had endured 12 years of chemotherapy for blood cancer only to see the disease come back stronger and meaner each time.

It was long, hard and terrible, J.C. Cox, 66, said.

So when he was told that he could take part in a clinical trial of a newly modified form of immune therapy, he signed on.

In that small preliminary trial, the results of which were published Wednesday in the New England Journal of Medicine, nearly two-thirds of the patients, all of whom had cancer so advanced that just a decade ago there would have been no hope for them, went into complete remission. Cox was among that two-thirds.

The new treatment involves tweaking a type of therapy called CAR-T that helps the immune system home in on cancer cells. Those tweaks appear to have made it more effective than its predecessor while also leading to fewer side effects, the study found.

In CAR-T therapy, doctors equip a patients own T-cells with a sensor that essentially sniffs out a protein on cancer cells, allowing them to glom onto the protein and then destroy the diseased cells. CAR-T therapy has been approved by the U.S. Food and Drug Administration to treat several types of blood cancer.

The altered T cells end up working like a heat-seeking missile, said study co-author Dr. Katayoun Rezvani, a professor of stem cell transplantation and cellular therapy at The University of Texas MD Anderson Cancer Center.

There were several drawbacks to CAR-T, including the time it took to make the revved up T cells which needed to be harvested from patients and then sent to a lab, their cost, and most important, the possibility of life-threatening side effects.

To try to make a cheaper, safer therapy that would potentially work for all patients, Rezvani and her colleagues switched from T-cells to a different type of immune cell, called natural killer cells.

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Natural killer cells are the best killers of virally infected and abnormal cells, she said. They can continue to patrol and recognize abnormal cells.

There were several advantages to the natural killer cells, not the least of which was that, unlike T cells, they wouldnt make the patients sick by spewing out a flood of inflammatory proteins, leading to a severe condition called a cytokine storm. Another big advantage was that the natural killer cells from one patient could be given to another without any tissue matching. That meant that such cells from healthy donors or from donated umbilical cord blood could be banked and ready to use at any time.

Presumably, this would cut down on the wait time and the costs of the therapy, because the treatments werent being tailor-made for each individual patient.

Rezvani and her colleagues genetically modified the natural killer cells to have a receptor (the R in CAR) for a protein that is on the surface of the cancer cells they were targeting. The receptor would lock onto the protein and then the cell would do its work.

The researchers also tweaked the natural killer cells in two other ways. Unlike T cells which live for a long time, natural killer cells normally have a lifespan of just a couple of weeks, so the researchers added a growth factor that would keep them around for a lot longer. And as a precaution, they also inserted a switch that would allow the researchers to kill off the altered cells if they became too abundant.

Rezvani and her colleagues tested the new treatment in 11 blood cancer patients. When the patients were checked two months after treatment, seven had no signs of cancer while one other showed improvement but not complete remission. The other three had no response to the treatment.

Cox was the eighth patient to receive the new treatment, and initially had misgivings.

I didnt have any other options, Cox, who received the treatment for non-Hodgkin lymphoma, said. But it was scary knowing I would be No. 8 and would be getting the biggest dose.

The trial had been set up to start with a low dose, and then wait to see if there were any serious side effects. If not, the plan was to increase the dose in later patients.

Coxs years of chemotherapy made him worry about possible side effects. But it was probably the easiest thing Ive ever done, he said.

The researchers themselves werent sure what to expect. We were amazed at the safety, Rezvani said. And it didnt seem to matter what dose we gave. This truly is a living drug. It gets inside of the patients body and starts growing and attacking the cancer cells.

Larger studies are needed, but if the treatment which has been licensed to Takeda Pharmaceutical Co. lives up to its early promise, Rezvani hopes to try it on other cancers, such as ones that affect the brain and the breasts.

The response of the patients in the new study is impressive, said Dr. David Porter, the director of cell therapy and transplantation at the University of Pennsylvania Health System. I think this is a major advance in the field of targeted cellular therapy.

Moreover, the natural killer treatments dont seem to have the same life-threatening complications as the original CAR-T therapy, Porter said in an email. Porter was involved with previous CAR-T research, but was not involved with this trial.

But, Porter cautioned, the study included a very small number of patients.

Cox wasnt sure what to expect when he went to be checked two months after receiving his treatment. The news was better than he could have imagined: there was no sign of his cancer.

I did a lot of crying, but they were happy tears, he said. I still get emotional when I talk about it.

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Immune therapy tweak offers new hope to blood cancer patients - NBC News

Early results from a phase I/II trial, published inThe New England Journal of Medicine, found that a majority of patients with either relapsed or refractory non-Hodgkins lymphoma (NHL) or chronic lymphocytic leukemia (CLL) treated with CAR NK cells had a response without the development of cytokine release syndrome, neurotoxicity, or graft-versus-host disease.1

Additionally, there was no increase in the levels of inflammatory cytokines, including interleukin-6, over baseline; however, the maximum tolerated dose was not reached.

We are encouraged by the results of the clinical trial, which will launch further clinical studies to investigate allogeneic cord blood-derived CAR NK cells as a potential treatment option for patients in need, corresponding author Katy Rezvani, MD, PhD, professor of stem cell transplantation and cellular therapy at The University of Texas MD Anderson Cancer Center, said in a press release.2

In this study cohort of 11 patients with relapsed or refractory CD-19 positive cancers, participants were given a single dose of cord blood-derived CD19 CAR NK cells at 1 of 3 dose levels. Five of the patients had CLL and the remaining 6 had NHL. All of the patients were treated with a minimum of 3 and a maximum of 11 lines of prior therapy. The first 9 participants treated were given CD19 CAR NK cells that were partially matched according to the individuals HLA type, but protocol allowed the last 2 patients to be treated with no HLA matching.

Eight (73%) of the participants had a response, and of those, 7 (4 with NHL and 3 with CLL) had a complete remission, while 1 had remission of the Richters transformation component, but had persistent CLL. The responses were rapid and observed within 30 days after infusion at all dose levels. The infused CAR NK cells expanded and persisted at low levels for at least 12 months.

According to the researchers, a proportion of patients treated with anti-CD19 CAR T cells have a subsequent relapse, with a 1-year progression-free survival of approximately 30% observed among patients with CLL and 45% seen among those with NHL.

In view of these outcomes, our study allowed for remission consolidation therapy with an immunomodulatory agent, anticancer drug, or hematopoietic stem-cell transplantation at the discretion of the treating physician, the authors wrote. However, the use of post-remission therapy in this study limits our assessment of the durability of response after CAR NK therapy.

Notably, researchers did observe high-grade transient myelotoxicity in the cohort, of which they attributed to the lymphodepleting chemotherapy. However, they were unable to assess whether the CAR NK cells contributed to the myelotoxicity.

Side effects experienced by the patients were primarily related to the conditioning chemotherapy given before cell infusion and were resolved within 1 to 2 weeks, according to Rezvani. Additionally, no patient required admission to an intensive care unit for management of treatment side effects.

Due to the nature of the therapy, weve actually been able to administer it in an outpatient setting, Rezvani said. We look forward to building upon these results in larger multi-center trials as we work with Takeda to make this therapy available more broadly.

References:

1. Liu E, Marin D, Banerjee P, et al. Use of CAR-Transduced Natural Killer Cells in CD19-Positive Lymphoid Tumors.The New England Journal of Medicine. doi:10.1056/NEJMoa1910607.

2. CD19 CAR NK-cell therapy achieves 73% response rate in patients with leukemia and lymphoma [news release]. Houston, Texas. Published February 5, 2020. app.bronto.com/public/viewmessage/html/6781/hemm7h92mwb6849npf2js0r4mhnrl/0bd003eb00000000000000000000000df096. Accessed February 6, 2020.

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Early Findings Show Promise of CAR NK-Cell Therapy in Leukemia, Lymphoma - Cancer Network

Stem cell bank Cyro-Save moved the umbilical cords it stores on behalf of parents to Poland, but part of that material has gone missing, according to newspaper AD. That left thousands of parents worried about the storage of their children's stem cells.Many parents have no idea where they stand. They are receiving unclear emails from different parties, and are unable to reach the company itself, the newspaper writes.

Cyro-Save has around 230 thousand customers worldwide, including several thousand in the Netherlands. Customers sent the companyblood and pieces of umbilical cord immediately after the birth of their child, to freeze and store in the event of the child becoming sick in future, so that it could be used for stem cell therapy.

According to AD, Cyro-Save's activities are scientifically controversial and even prohibited in some European countries. Under pressure from governments and financial problems, Cyro-Save moved 330 thousand samples of genetic material to the Polish company Famicord last year. This happened without the permission of the parents or the authorities. And a number of tanks are missing, the newspaper writes.

Authorities in multiple countries, including Belgium and Switzerland, are currently investigating the relocation of the material to Poland.

Famicord contacted parents, asking the to sign a contract with the company. But Dubai-based companyCSG Bio, which took over Cyro-Save, also claims that it has taken over Cyro-Save's stem cell activities and are advising parents not to go with the Polish company. Meanwhile, parents have no idea where their children's stem cells actually are.

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Missing stem cells: Thousands of NL parents potentially affected - NL Times

Verified Market Research offers its latest report on the Stem Cell Therapy Market that includes a comprehensive analysis of a range of subjects such as market opportunities, competition, segmentation, regional expansion, and market dynamics. It prepares players also as investors to require competent decisions and plan for growth beforehand. This report is predicted to assist the reader understand the market with reference to its various drivers, restraints, trends, and opportunities to equip them in making careful business decisions.

Global Stem Cell TherapyMarketwas valued at USD 86.62 million in 2016 and is projected to reach USD 221.03million by 2025, growing at a CAGR of 10.97% from 2017 to 2025.

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The top manufacturer with company profile, sales volume, and product specifications, revenue (Million USD) and market share

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The chapter on competitive landscape covers all the major manufacturers in the global Smart Cameramarket to study new trends and opportunities. In this section, the researchers have used SWOT analysis to study the various strengths, weaknesses, opportunities, and trends the manufacturers are using to expand their share. Furthermore, they have briefed about the trends that are expected to drive the market in the future and open more opportunities.

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The researchers have analyzed various factors that are necessary for the growth of the market in global terms. They have taken different perspectives for the market including technological, social, political, economic, environmental, and others. The drivers have been derived using PESTELs analysis to keep them accurate. Factors responsible for propelling the growth of the market and helping its growth in terms of market share are been studied objectively.

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Table of Contents

Introduction: The report starts off with an executive summary, including top highlights of the research study on the Stem Cell Therapy industry.

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Our research studies help our clients to make superior data-driven decisions, capitalize on future opportunities, optimize efficiency and keeping them competitive by working as their partner to deliver the right information without compromise.

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Stem Cell Therapy Market Booming by Size, Revenue, Trends and Top Growing Companies 2026 - Instant Tech News

Testing a new therapeutic in human subjects for the first time is a major step in the translation of any novel treatment from the laboratory bench to clinical use.

When the therapeutic represents a paradigm shift, reaching this milestone is even more significant.

After years of planning, preparation and hard work to establish a base camp, starting human clinical trials is the first step towards the summit itself: gaining regulatory approval for product sales.

Exosomes tiny packets of proteins and nucleic acids (e.g. mRNA and miRNA) released by cells, that have powerful regenerative properties ranging from promoting wound healing to stimulating brain injury recovery following stroke represent just such a paradigm-shifting potential advance in human medicine.

The first commercial exosome therapeutics conference was held in Boston in September 2019 and over 15 companies participated.

This conference signals the emergence of exosomes as a new class of regenerative medicine products.

So far, just one or two of the companies working in the novel field of exosome-based therapies have reached the pivotal point and transitioned into human clinical trials. In this article we survey the field, starting with the pace-setters.

During the past few years, a handful of universities and research hospitals have carried out small scale, first-in-human Phase I clinical trials using exosomes. In each case where the study results are available, the exosome treatment was found to be safe and well-tolerated.

But the field has hotted up in the past few months, with the first companies reaching the pivotal point of testing exosome-based products in people.

On 28th January 2020, Melbourne-based Exopharm announced the first dosing under its first human clinical trial, becoming the first company to test exosomes potential for healing wounds in people.

The PLEXOVAL Phase I study will test Exopharms Plexaris product, a cell-free formulation of exosomes from platelets, which in preclinical animal studies have shown a regenerative effect, improving wound closure and reducing scarring.

The main readouts of the PLEXOVAL study the results of which are expected to be available sometime after mid-2020 will be safety, wound closure and scarring.

Joining Exopharm at the front of the pack is Maryland-based United Therapeutics.

Founded in 1996, United Therapeutics specialises in lung diseases and has a portfolio of FDA-approved conventional small molecule and biologic drugs on the market for a range of lung conditions.

On 26th June 2019, United Therapeutics announced approval for a Phase I trial (NCT03857841) of an exosome-based therapy against bronchopulmonary dysplasia (BDP), a condition common in preterm infants that receive assisted ventilation and supplemental oxygen.

Recruitment has commenced but dosing has not been announced. The study is due to conclude by December 2021. BDP is characterised by arrested lung growth and development, with health implications that can persist into adulthood.

Human clinical trials of a stem cell therapy for BDP, by Korean stem cell company Medipost, are already underway. However as with many stem cell therapies recent animal studies have shown that is the exosomes released by stem cells that are responsible for the therapeutic effect.

United Therapeutics therapy, UNEX-42, is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells. The company has not released any information about how its exosomes are produced or isolated.

A little behind the two leaders, three other companies have announced their aim to initiate their first clinical trials of exosome therapeutics within the next 12 months.

Launched in 2015, Cambridge, Massachusetts-based Codiak has long been considered among the leaders in developing exosome-based therapies.

Rather than exploiting the innate regenerative potential of select exosome populations, Codiak is developing engineered exosomes that feature a defined therapeutic payload. The companys initial focus has been to target immune cells, leveraging the immune system to combat cancer.

The company plans to initiate clinical trials of its lead candidate, exoSTING, in the first half of 2020. The therapeutic is designed to trigger a potent antitumor response from the patients own immune system, mediated by T cells. A second immuno-oncology candidate, exoIL-12, is due to enter clinical trials in the second half of 2020, the company says.

In nearby New Jersey, Avalon Globocare is also developing engineered exosomes. Its lead product, AVA-201, consists of exosomes enriched in the RNA miR-185, which are produced using engineered mesenchymal stem cells.

In animal tests, miR-185 suppressed cancer cell proliferation, invasion and migration in oral cancer. In July 2019, the company announced plans to start its first exosome clinical trial before the close of 2019. As of February 2020, however, no further announcement regarding this clinical trial has been made.

Avalon has also made no further announcement on a second planned clinical trial, also intended to start during the fourth quarter of 2019, of a second exosome candidate, AVA-202.

These angiogenic regenerative exosomes, derived from endothelial cells, can promote wound healing and blood vessel formation, the company says. The planned Phase I trial was to test AVA-202 for vascular diseases and wound healing.

Meanwhile, Miami-based Aegle Therapeutics plans to begin a Phase I/IIa clinical trial of its exosome therapy, AGLE-102, during 2020. AGLE-102 is based on native regenerative exosomes isolated from bone marrow mesenchymal stem cells.

After initially focussing on burns patients, in January 2020 to company announced had raised the funds to commence an FDA-cleared clinical trial of AGLE-102 to treat dystrophic epidermolysis bullosa, a rare paediatric skin blistering disorder. The company says it plans to commence this clinical trial in the first half of 2020.

A number of companies are in the preclinical phase of exosome therapy research.

Some of these companies have been set up specifically to develop exosome-based products. In the UK, Evox co-founded by University of Oxford researcher Matthew Wood in 2016 is developing engineered exosomes to treat rare diseases.

The company has developed or sourced technology that allows it to attach proteins to exosomes surface, or to load proteins or nucleic acids inside the exosome, to deliver a therapeutic cargo to a target organ.

Its lead candidate targets a lysosomal storage disorder called Niemann-Pick Disease type C, using exosomes that carry a protein therapeutic cargo. Evox says it plans to submit the Investigational New Drug (IND) application to the FDA during 2020, paving the way for the first clinical trial. It currently has five other candidates, for various indications, at the preclinical stage of development.

In Korea, Ilias and ExoCoBio are developing exosome therapeutics. Ilias founded by faculty from the Korean Advance Institute of Science and Technology specialises in loading large protein therapeutics into exosomes.

It is currently carrying out preclinical research toward treating sepsis, preterm labour and Gauchers disease. ExoCoBio is focusing on the native regenerative capacity of exosomes derived from mesenchymal stem cells, including to treat atopic dermatitis.

New companies continue to enter the exosome space. In August 2019, Carmine Therapeutics was launched, with the aim to develop gene therapies that utilize exosomes from red blood cells to deliver large nucleic acid cargoes. The company is targeting the areas of haematology, oncology and immunology.

Meanwhile, a wave of companies originally set up to develop live stem cell therapies are diversifying into stem cell derived exosome production and research.

It is now generally acknowledged that stem cell exosomes are the main therapeutically active component of stem cells, and that medical products based on exosomes will be safer to apply, and easier and cheaper to make and transport, than live cell therapies.

Originally established to produce neural stem cells for other research organisations, Aruna Bio has developed proprietary neural exosomes that can cross the blood brain barrier.

The company is now developing an exosome therapy for stroke. In October 2019, the Athens, Georgia-based company said had raised funding to support the research and development to enable its first IND application to the FDA in 2021.

In the UK, ReNeuron has also focussed on stroke, and has several clinical trials underway assessing its CTX stem cells to promote post stroke rehabilitation. The company is also working with third parties to investigate the drug- and gene therapy delivery potential of exosomes derived from CTX stem cells.

Switzerland-based Anjarium is also developing an exosome platform to selectively deliver therapeutics.20 The company is focussing on engineering exosomes loaded with therapeutic RNA cargo and displaying targeting moieties on its surface.

California-based Capricor has commenced clinical trials of a cardiosphere-derived stem cell therapy for the treatment of Duchenne muscular dystrophy (DMD).

At an earlier phase, its regenerative exosome therapy CAP-2003 is in pre-clinical development for a variety of inflammatory disorders including DMD.

A number of other stem cell companies, including TriArm, Creative Medical, AgeX Therapeutics and BrainStorm Cell Therapeutics, are reported to be investigating exosome-based therapies derived from their stem cell lines.

Exopharms position as a frontrunner in bringing exosomes into humans is no lucky accident. The companys operations are based around its unique, proprietary method for manufacturing and isolating exosomes, known as LEAP technology.

As academics and observers of the exosome field have pointed out, reliable and scalable exosome manufacture has threatened to be a major bottleneck that limits the translation of exosome therapeutics into clinical use. The standard laboratory-scale method for collecting the exosomes produced by cultured cells has been to spin the liquid cell culture medium in an ultracentrifuge, or pass it through a fine filter.

The most common technique used so far, the ultracentrifuge, has major scalability limitations. Issues include the high level of skill and manual labour required, the time-intensive nature of the process, and the associated costs of reagents and equipment. It is impossible to imagine collecting enough exosomes for a late stage clinical trial this way.

Another issue is the low purity of the exosomes collected. These techniques sort the contents of cell culture medium by their mass and/or size. Although the exosomes are concentrated, they could be accompanied by other biological components present in the cell culture medium that happen to be a similar size or mass to the exosome.

Importantly, a biotechnology company needs a proprietary step in the process to make a proprietary product over which it has exclusivity. Exopharms LEAP technology is a good example of a proprietary manufacturing step. Ultracentrifuge is not a proprietary process.

So the big players in the emerging exosome field have generally placed a strong emphasis on developing their manufacturing and purification capability.

Exopharm developed a chromatography-based purification method, in which a patent-applied-for inexpensive functionalised polymer a LEAP Ligand is loaded into a chromatography column. The LEAP Ligand sticks to the membrane surface of exosomes passed through the column. Everything else in the cell culture medium mixture is simply washed away. The pure exosome product is then eluted from the column and collected for use. As well as being very scalable, the technique is versatile. LEAP can be used to produce a range of exosome products, by isolating exosomes from different cell sources.

Codiak, similarly, says it has developed scalable, proprietary chromatography-based methods to produced exosomes with comparable identity, purity, and functional properties as exosomes purified using methods such as ultracentrifugation. Chromatography is a flow-based technique for separating mixtures. In an April 2019 SEC filing, the company said it is establishing its own Phase 1/2 clinical manufacturing facility, which it is aiming to have fully-operational by first half 2020.

Avalon GloboCare teamed up with Weill Cornell Medicine to develop a standardised production method for isolating clinical-grade exosomes. Aegle also says it has a proprietary isolation process for producing therapeutic-grade exosomes. And Evox emphasises the GMP compliant, scalable, commercially viable manufacturing platform it has developed.

At Exopharm, the manufacturing technique that has allowed the company to leap ahead of the pack and into human clinical trials is its proprietary LEAP platform. Overcoming the exosome production and isolation bottleneck was exactly the problem the companys scientists set out to solve when Exopharm formed in 2013.

In addition to the Plexaris exosomes, isolated from platelets, currently being tested in human clinical trials, Exopharm is progressing toward human clinical trials of its second product, Cevaris, which are exosomes isolated from stem cells.

Exosomes are now under development by around 20 companies across the world. The leaders in the field are now entering clinical trials with both nave exosome products and engineered exosome products. A number of cell therapy companies are also moving across into the promising exosome product space.

The coming years promise dynamic changes, with partnerships and eventually product commercialization. Exopharm is a clear leader in this emerging field.

(Featured image by Darko Stojanovic from Pixabay)

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Biotech companies leading the way with exosome human clinical trials - Born2Invest