Category Archives: Cell Therapy

SAN DIEGO, Feb. 27, 2020 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento"), announced today that Dr. Henry Ji, Chairman and CEO, will present at the 9th Annual LEERINK Partners Global Healthcare Conference (Lotte New York Palace Hotel, New York) on Thursday Feb 27 at 11:00-11:25 AM (Presentation Room Adams).

Key updates:

DAR-T specific developments:

Sorrentos proprietary DAR structure offers potential advantages over the conventional CAR (chimeric antigen receptor) structure by:

In addition to the proprietary DAR structure, Sorrentos proprietary non-viral knockout-knockin (KOKI) manufacturing approach offers several potential benefits over virus-based transduction currently used for CAR-T therapies:

Combining those approaches, the allogeneic DAR-T products could potentially have the following major advantages over current autologous CAR-T therapy technologies:

Beyond targeting cancer related indications, which is the primary focus of Sorrentos immuno-oncology business, Sorrento believes it can apply its KOKI DAR-T platform against pandemic infectious pathogens, including but not limited to coronaviruses, HBV, HIV, malaria, measles and fungi.

The potential spread of the COVID-19 coronavirus from Wuhan, China is only one example of how quickly a local infection can become a pandemic. Sorrentos agile KOKI DAR-T and DAR-NK cell therapy platforms, coupled with in-house cGMP manufacturing facilities, should allow for emergency adoptive T cell and innate NK cell therapies being produced rapidly for clinical testing.

COVID_DAR_001 has been added to the Sorrento development pipeline and allocated internal priority both to demonstrate the maturity, agility and development readiness of the proprietary platform and to address an emergency and potentially critical patient need in the upcoming months.

About Sorrento Therapeutics, Inc.

Sorrento is a clinical stage, antibody-centric, biopharmaceutical company developing new therapies to turn malignant cancers into manageable and possibly curable diseases. Sorrento's multimodal multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as fully human antibodies (G-MAB library), clinical stage immuno-cellular therapies (CAR-T), development stage DAR-T cell therapies, intracellular targeting antibodies (iTAbs), antibody-drug conjugates (ADC), and clinical stage oncolytic virus (Sephrevir).

Sorrento's commitment to life-enhancing therapies for cancer patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule in Resiniferatoxin (RTX) and ZTlido. Resiniferatoxin is completing a Phase 1b trial for intractable pain associated with cancer and a Phase 1b trial in osteoarthritis (OA) patients. ZTlido was approved by the FDA on February 28, 2018.

For more information visit http://www.sorrentotherapeutics.com

Forward-Looking Statements

This press release and any statements made for and during any presentation or meeting contain forward-looking statements related to Sorrento Therapeutics, Inc., under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995 and subject to risks and uncertainties that could cause actual results to differ materially from those projected. Forward-looking statements include statements regarding the expectations for Sorrento's and its subsidiaries' technologies and product candidates, including, but, not limited to, resiniferatoxin (RTX) and DAR-T, the clinical potential of RTX, potential advantages of Sorrentos DAR-T program and NK and CAR gene editing technologies, potential benefits of the KOKI manufacturing approach, potential advantages of allogeneic DAR-T products over current autologous CAR-T therapy and potential applications for the KOKI DAR-T and DAR-NK platforms. Risks and uncertainties that could cause our actual results to differ materially and adversely from those expressed in our forward-looking statements, include, but are not limited to: risks related to Sorrento's and its subsidiaries' technologies and prospects, including, but not limited to, RTX, DAR-T, KOKI and DAR-NK; risks related to seeking regulatory approvals and conducting and obtaining results of clinical trials, including, but not limited to, the Phase 1b studies and any prior RTX studies in animals; costs associated with clinical trials, the clinical and commercial success of RTX; the viability and success of using RTX for treatments in certain therapeutic areas, including for the treatment of intractable pain associated with cancer, Sorrentos DAR-T, KOKI and DAR-NK programs and drug products; risks related to seeking regulatory approvals and conducting clinical trials and other risks that are described in Sorrento's most recent periodic reports filed with the Securities and Exchange Commission, including Sorrento's Annual Report on Form 10-K for the year ended December 31, 2018, and subsequent Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission, including the risk factors set forth in those filings. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release and we undertake no obligation to update any forward-looking statement in this press release except as required by law.

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Media and Investor Relations

Contact: Alexis Nahama, SVP Corp Development

Telephone: 1.858.203.4120

Email: mediarelations@sorrentotherapeutics.com

Sorrento and the Sorrento logo are registered trademarks of Sorrento Therapeutics, Inc.

ZTlido and G-MAB are trademarks owned by Scilex Pharmaceuticals, Inc. and Sorrento, respectively.

Seprehvir, is a registered trademark of VirttuBiologics Limited, a wholly owned subsidiary of TNK Therapeutics, Inc. and part of the group of companies owned by Sorrento Therapeutics, Inc.

All other trademarks are the property of their respective owners.

2020 Sorrento Therapeutics, Inc. All Rights Reserved.

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Sorrento to Provide Corporate Update, Including DAR-T Cell Therapy Advances, at LEERINK Global Healthcare Conference - Yahoo Finance

Theres a rapid expansion going on in CAR T cell therapy, the remarkable living drug treatment that involves genetically engineering a patients own immune cells to fight cancer.

As of right now, CAR T cell therapy is approved by the U.S. Food and Drug Administration (FDA) for widespread use in:

Despite there only being two official CAR T cell approvals, however, Dr. Sid Ganguly, Deputy Director of Hematologic Malignancies and Cellular Therapeutics at the University of Kansas Medical Center, tells SurvivorNet that there are many more cancers expected to benefit from the treatment going forward.

You will be seeing many more diseases where the CAR T will become commercially available, Dr. Ganguly says, adding that patients with cancers that do not fall into the two FDA approval categories may still be able to access the treatment by enrolling in clinical trials.

Multiple myeloma is on of these diseases, as MD Andersons Robert Orlowski has previously told SurvivorNet.

Clinical trials which the FDA requires before it can approve a treatment are abundant in CAR T cell therapy. At Dr. Gangulys cancer center alone, he shares that at this moment there are 38 different trials taking place.

Because CAR T cell therapy is a highly specialized, intense procedure, its important to discuss the possibility of a referral with your oncologist.

When a patient is diagnosed with difficult-to-control lymphoma [for example], they should be referred to a center that is equipped with cellular therapeutics and technology, Dr. Ganguly says, adding that, right now at least, most of these specialized centers are located at academic institutions.

If your oncologist gives you a referral to one of these specialized centers, you will then meet with a cellular therapeutics specialist to discuss the next steps.

CAR T cell therapy is a highly technical treatment that carries the risk of several serious side effects, which explains why it requires a team of specialized professionals to administer.

Id say a village is required to take care of one patient [undergoing CAR T cell therapy], Dr. Ganguly says, adding that in his own institution, this village or team of specialists is collectively referred to as a CAR T hub. The team of specialists includes, among others:

So its a big team approach where holistically we take care of the patients and we take them through the whole process of identification to insurance approval trial accrual, collection of the cells, Dr. Ganguly says. And then post-treatment, taking care of the patient is also vitally important.

Originally, Dr. Ganguly says, there were only 16 centers in the country that had the expertise and experience to administer CAR T cell therapy. Now, however, that number is expanding.

With the explosion of CAR T, more and more centers are now involved in cellular therapeutics, he says. But we have a lot of work to do. There are a lot of patients that could benefit from this novel, exciting technology. But the knowledge is very important.

The knowledge that Dr. Ganguly references meaning the awareness that CAR T cell therapy is even an option in the first place, and the know-how needed to find a center offering it is not always a given. Thats why specialists are working hard to bring this knowledge to community oncologists.

We need to go to the grassroots, he says. We need to go to community oncologists and educate and talk about this technology so that the patients who are in dire need of this technique should be promptly referred.

The types of cancers that can benefit from CAR T cell therapy are often aggressive, fast-growing cancers, making it extremely important that oncologists refer their patients promptly in the cases where CAR T cell therapy is an option.

These cancers dont wait, Dr. Ganguly says. They grow exponentially, and sometimes, if we cannot get them on time, then the CAR T cannot be manufactured, and valuable time may be lost.

The importance of timing with CAR T cell therapy occasionally prompts patients to consider the option even before they need it, so that if and when they do need it, they will have access right away.

Sometimes, I have seen patients who do not even need CAR T right now, but [are] on the waiting list in case the disease does not respond to the standard-of-care treatment, Dr. Ganguly says. This way, he adds, the patients are already in the system so the referring doctor picks up the phone and we get them quickly, and we get the CAR T hub activated, and we transition the patient through the whole process.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Siddhartha Ganguly is aMultiple Myeloma Specialist at the University of Kansas. Read More

Theres a rapid expansion going on in CAR T cell therapy, the remarkable living drug treatment that involves genetically engineering a patients own immune cells to fight cancer.

As of right now, CAR T cell therapy is approved by the U.S. Food and Drug Administration (FDA) for widespread use in:

You will be seeing many more diseases where the CAR T will become commercially available, Dr. Ganguly says, adding that patients with cancers that do not fall into the two FDA approval categories may still be able to access the treatment by enrolling in clinical trials.

Multiple myeloma is on of these diseases, as MD Andersons Robert Orlowski has previously told SurvivorNet.

Clinical trials which the FDA requires before it can approve a treatment are abundant in CAR T cell therapy. At Dr. Gangulys cancer center alone, he shares that at this moment there are 38 different trials taking place.

Because CAR T cell therapy is a highly specialized, intense procedure, its important to discuss the possibility of a referral with your oncologist.

When a patient is diagnosed with difficult-to-control lymphoma [for example], they should be referred to a center that is equipped with cellular therapeutics and technology, Dr. Ganguly says, adding that, right now at least, most of these specialized centers are located at academic institutions.

If your oncologist gives you a referral to one of these specialized centers, you will then meet with a cellular therapeutics specialist to discuss the next steps.

CAR T cell therapy is a highly technical treatment that carries the risk of several serious side effects, which explains why it requires a team of specialized professionals to administer.

Id say a village is required to take care of one patient [undergoing CAR T cell therapy], Dr. Ganguly says, adding that in his own institution, this village or team of specialists is collectively referred to as a CAR T hub. The team of specialists includes, among others:

So its a big team approach where holistically we take care of the patients and we take them through the whole process of identification to insurance approval trial accrual, collection of the cells, Dr. Ganguly says. And then post-treatment, taking care of the patient is also vitally important.

Originally, Dr. Ganguly says, there were only 16 centers in the country that had the expertise and experience to administer CAR T cell therapy. Now, however, that number is expanding.

With the explosion of CAR T, more and more centers are now involved in cellular therapeutics, he says. But we have a lot of work to do. There are a lot of patients that could benefit from this novel, exciting technology. But the knowledge is very important.

The knowledge that Dr. Ganguly references meaning the awareness that CAR T cell therapy is even an option in the first place, and the know-how needed to find a center offering it is not always a given. Thats why specialists are working hard to bring this knowledge to community oncologists.

We need to go to the grassroots, he says. We need to go to community oncologists and educate and talk about this technology so that the patients who are in dire need of this technique should be promptly referred.

The types of cancers that can benefit from CAR T cell therapy are often aggressive, fast-growing cancers, making it extremely important that oncologists refer their patients promptly in the cases where CAR T cell therapy is an option.

These cancers dont wait, Dr. Ganguly says. They grow exponentially, and sometimes, if we cannot get them on time, then the CAR T cannot be manufactured, and valuable time may be lost.

The importance of timing with CAR T cell therapy occasionally prompts patients to consider the option even before they need it, so that if and when they do need it, they will have access right away.

Sometimes, I have seen patients who do not even need CAR T right now, but [are] on the waiting list in case the disease does not respond to the standard-of-care treatment, Dr. Ganguly says. This way, he adds, the patients are already in the system so the referring doctor picks up the phone and we get them quickly, and we get the CAR T hub activated, and we transition the patient through the whole process.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Siddhartha Ganguly is aMultiple Myeloma Specialist at the University of Kansas. Read More

Read this article:
CAR T Cell Therapy Where and How to Access the Lifesaving Living Drug - SurvivorNet

Revolutionary Car T-cell therapy is set to become the fifth pillar of cancer treatment and is already showing dramatic results in the successful treatment of blood cancers.

Prof Owen Smith is a consultant paediatric haematologist who has dedicated his 35-year career to researching and treating cancer in children and young adolescents. Smith, who is based at Childrens Health Ireland at Crumlin hospital, is a specialist in stem cell transplantation. Over the years, he has seen big advances in cancer treatments but few have excited him as much as Car T-cell therapy which is showing huge potential as a pioneering treatment for blood and other challenging cancers.

In a nutshell, Car (chimeric antigen receptor) T-cell therapy is a form of immunotherapy that uses the patients own immune cells to recognise and attack cancer. It involves drawing blood from the patient, isolating the T-cells within it and genetically altering and multiplying them in a lab. The end product is transfused back into the patient where the Car T-cells bind to an antigen or structural molecule on the cancer cells and kills them.

Car T-cells or Cars for short, are like sniper fire that target the cancer rather than blanket bombing it, explains Prof Smith. They cause much less toxicity than chemotherapy and have much better results and remission rates.

Cars therapy has been in gestation since the early 1980s with the first-generation treatment becoming available around 2010.

To date, about 2,200 Cars have been completed in the US, while in Europe the number is about 800 and growing fast. There are now two second generation Car T products licensed for three types of blood cancer and Prof Smith says: We now have hope with these relapse/refractory blood cancers where only a few years ago, we had none.

Prof Smith was working in Royal Free Hospital in London (one of the UKs major Car T therapy centres today) in the 1980s when the therapeutic potential of T-cells, which was initially discovered in Israel, was first being realised. It became clear through some good connected thinking that it was the T-cells that were having a very important effect on cure in patients with CML (chronic myeloid leukaemia), he says. Around the same time there were papers coming in from the States with similar results so the concept was beginning to gather momentum.

The question became: could you take T-cells from a donor and give them to people who were relapsing and put them into remission? Things began to crystallise in the late 80s/early 90s as T cells were recognised as immune effector cells that were very good at killing cancer, especially in patients with CML who were relapsing after stem cell transplantation.

Then people began wondering if we could use the end of the molecule the so-called antigen binding part of an antibody and link it to the T-cell receptor and put it into T-cells to see if that would give an even better result. What eventually followed between 2010 and 2014 were a number of first generation Car T-cell studies that showed it was very effective in some patients with haematological malignancies and in those with acute lymphoblastic and chronic lymphocytic leukaemia in particular. Since then there have been 10 big studies with second generation Cars that have also proved its efficacy, says Prof Smith.

Acute lymphoblastic leukaemia is the most common form of blood cancer in children and Prof Smith sees about 50 cases a year here. Unfortunately, if this cancer relapses, particularly during or shortly after chemotherapy, it usually becomes resistant to further treatment and the children can also no longer tolerate it. The only option up to now has been palliative care, but Cars is a lifeline that can alter this outcome.

Second generation Car T-cell activity can push around 90 per cent of children into remission and once you get them into remission you have time to get them into prime clinical condition to give them a stem cell transplant and cure them that way, says Prof Smith. In the adult population, about 20 cases a year of non-Hodgkins lymphoma would be suitable for this therapy.

Weve known for a long time that the immune system is important in someone developing cancer, Prof Smith adds. So, for example when a persons T-cells are knocked out by something like HIV, they typically have an increased risk of developing lymphoid malignancies. The immune system can also be tricked into not recognising when a cancer develops and undergoes metastatic spread. This immune activation therapy can unmask this camouflaging of the cancer resulting in regression.

One of the challenges with the Cars is that they can become lost in the body and lose their persistence. When this happens the person usually relapses. We are now looking at different combinations of Cars and using two or three of them on the one cell to prevent relapse, says Prof Smith.

This is a massively evolving field and one of these exponential technologies you hear about from time to time. Whats really encouraging it that its now being pushed out to treat other types of high-risk solid tumours that currently have dismal survival rates such as metastatic non-small cell lung cancer, pancreatic cancer, triple negative breast cancer, ovarian cancer, brain tumours and neuroblastomas in children. Its opening up a totally different treatment pathway for cancer.

Prof Smith acknowledges that Cars, like many innovative cancer treatments, is expensive at about 300,000 per commercially produced treatment. But he points out that as its use becomes more widespread prices will drop and that if the total costs of conventional treatments are added up the economic argument for its use is compelling. Yes, its costly but some recent health economic data from the USA is suggesting it may not be as costly as it first appears, he says. Specifically, repeated chemotherapy, the cost of regular hospital admissions due to relapse, trips to intensive care, blood product support and anti-bacterial, anti-fungal and anti-viral therapies all with very little return in terms of clinical outcome.

Prof Smith adds that Car T therapy is set to become the fifth pillar of cancer treatment alongside surgery, chemotherapy, radiation and targeted therapy and that whats happening now is just the start of the Car T revolution. The next phase will be using them in combination with checkpoint inhibitors (another arm of immunotherapy) and the third generation of Cars will have greater efficacy and less toxicity, he says.

Prof Smith is low key about his personal achievements but in addition to saving the lives of countless children under his care, he also has a distinguished research record and his work has been widely published and acknowledged internationally. He was to the forefront of a major breakthrough in the intervention and treatment of meningitis in the 1980s and he is a strong believer in robust peer-reviewed clinical trials, which he believes are one of the most effective ways of establishing a cure for any disease.

Owen Smith is professor of Paediatric and Adolescent Medicine at UCD, honorary Regius Professor of Physic (1637) at Trinity College and academic lead to the Childrens Hospital Group. He is also a principal investigator at the National Childrens Research Centre and Systems Biology Ireland at University College Dublin and national clinical programme lead for children and adolescents/young adults with cancer. In 2015 he was awarded a CBE by Queen Elizabeth for his life-long dedication to treating cancer in children.

Prof Smith is a speaker at the international BioPharma Ambition 2020 event which is being held in Dublin Castle on March 3rd and 4th.

Go here to read the rest:
Car T-cell therapy: The future fifth pillar of cancer treatment - The Irish Times

Research has shown that the CAR-T therapies in development in Asia-Pacific will make the worldwide market more competitive and drive down prices.

According to new research, there arecurrently 445 CAR T-cell therapies under development by companies headquartered across the Asia-Pacific (APAC) region. With research in progress and increased understanding of cancer biology, the new therapies are expected to make the global market more competitive and cost effective, says GlobalData.

The research reveals that the majority of the cellular modalities are in the early stages of clinical development. Acute lymphocytic leukaemia (ALL), B-cell acute lymphocytic leukaemia and refractory multiple myeloma are the three major indications for which CAR T-cell therapies are under development in APAC.

Gowri Prasad Gutti, Director of Pharma Intelligence at GlobalData, commented: Cancer is becoming a serious health concern across the APAC region, because of an ageing population, changes in lifestyle associated with economic development and epidemiologic transition. Clinical outcomes of CAR T-cell therapies have been impressive and have shown remarkable results in relapsed/refractory patients. However, the evidence of efficacy is still being gathered with more clinical trials underway.

The outlet highlights that in 2019, Japans Ministry of Health, Labor and Welfare (MHLW) approved the first CAR T-cell therapy Kymriah (tisagenlecleucel) for the treatment of ALL and diffuse large B-cell lymphoma. The drug received approval for the treatment of Acute Lymphocytic Leukemia (ALL) in Australia in 2018.

According to the researchers, as of 10 February 2020, there are 369 trials that are ongoing in the APAC region. Most of these trials are in Phase I, followed by Phase I/II.

Gutti explained: This suggests that the CAR-T modalities are still under clinical evaluation phase and a considerable amount of data evaluation is required for these to be successful as a new generation of anticancer drugs.

China, by far, leads in the number of CAR T-cell clinical trials, accounting for about 95 percent of the total clinical trials in the APAC region, says the report.

Gutti concluded: Even though China leads the CAR T-cell therapy development, the approval of CAR-T therapy in China will likely take some time as the majority of the programs are in early stages of development.

See more here:
Innovations in CAR-T therapies in APAC will make market competitive - European Pharmaceutical Review

A growing number of patients with advanced cancer are able to benefit from treatment with CAR T cell therapy, a living drug that involves genetically modifying a patients own immune T cells then infusing them back into the body to fight off cancer. The treatment is highly technical, and while it can be a lifesaving option, it also carries with it the risk of potentially serious side effects. Dr. Sid Ganguly,Deputy Director of Hematologic Malignancies and Cellular Therapeutics at the University of Kansas Medical Center, explains the risks associated with one of these side effects called cytokine release syndrome.

During CAR T cell therapy, when patients receive an infusion of their own immune T cells that have been genetically altered, these T cells will usually begin to attack the cancer cells straightaway. In the process of doing so, they release signaling molecules called cytokines. The release of cytokines can cause a number of symptoms and reactions that vary in terms of severity. Because symptoms can range from mild flu-like symptoms to life-threatening reactions, doctors will often refer to different grades of cytokine release syndrome.

Often, if [cytokine release syndrome] is mild, we call it grade 1, Dr. Ganguly says. Many of the patients stay in grade 1, and we use supportive measures like Tylenol and antibiotics, and they get better. The symptoms of grade 1 cytokine release syndrome may mirror that of the flu think fever and elevated heart rate.

If the Tylenol and antibiotics dont help the patient get better, the cytokine release syndrome may be elevated to grade 2. With grade 2, patients may experience low blood pressure and require more specialized treatment measures, such as oxygen, fluid resuscitation, and low doses of pressors that maintain blood pressure.

Beyond grade 2 cytokine release syndrome, patients often need to be transferred to the intensive care unit and monitored extremely closely.

When cytokine release syndrome reaches stage 4, Dr. Ganguly says, it becomes really life-threatening and serious.

When cytokine release syndrome advances beyond the supportive measures, then we have to use specific treatment, Dr. Ganguly says, explaining that this may entail an anti-cytokine, which is a medication that counteracts the cytokines.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Siddhartha Ganguly is aMultiple Myeloma Specialist at the University of Kansas. Read More

A growing number of patients with advanced cancer are able to benefit from treatment with CAR T cell therapy, a living drug that involves genetically modifying a patients own immune T cells then infusing them back into the body to fight off cancer. The treatment is highly technical, and while it can be a lifesaving option, it also carries with it the risk of potentially serious side effects. Dr. Sid Ganguly,Deputy Director of Hematologic Malignancies and Cellular Therapeutics at the University of Kansas Medical Center, explains the risks associated with one of these side effects called cytokine release syndrome.

During CAR T cell therapy, when patients receive an infusion of their own immune T cells that have been genetically altered, these T cells will usually begin to attack the cancer cells straightaway. In the process of doing so, they release signaling molecules called cytokines. The release of cytokines can cause a number of symptoms and reactions that vary in terms of severity. Because symptoms can range from mild flu-like symptoms to life-threatening reactions, doctors will often refer to different grades of cytokine release syndrome.

If the Tylenol and antibiotics dont help the patient get better, the cytokine release syndrome may be elevated to grade 2. With grade 2, patients may experience low blood pressure and require more specialized treatment measures, such as oxygen, fluid resuscitation, and low doses of pressors that maintain blood pressure.

Beyond grade 2 cytokine release syndrome, patients often need to be transferred to the intensive care unit and monitored extremely closely.

When cytokine release syndrome reaches stage 4, Dr. Ganguly says, it becomes really life-threatening and serious.

When cytokine release syndrome advances beyond the supportive measures, then we have to use specific treatment, Dr. Ganguly says, explaining that this may entail an anti-cytokine, which is a medication that counteracts the cytokines.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Siddhartha Ganguly is aMultiple Myeloma Specialist at the University of Kansas. Read More

Read the rest here:
Cytokine Release Syndrome and CAR T Cell Therapy: Understanding the Risks - SurvivorNet

While not yet approved by the FDA, clinical trials testing CAR T-cell therapy have better numbers than weve seen ever in the history of multiple myeloma, says Dr. Robert Orlowski, chair of the Department of Lymphoma and Myeloma at the MD Anderson Cancer Center.

With the living drug treatment CAR T-cell therapy already approved by the Food and Drug Administration for certain non-Hodgkins lymphomas, doctors are now looking at its tremendous promise for multiple myeloma.

The response rate has been around 90%, without about half of those patients ending up in complete remission, Dr. Orlowski says.

CAR T-cell therapy is a one-time treatment that is both a drug and procedure. A patients immune systems T cells are extracted from their body, genetically modified in a lab to identify and attack cancer cells, and then put back into the body to do their work.

For multiple myeloma,CAR T-cell therapy most commonly targets the B-cell maturation antigen, or BCMA.

One of the nice advantages is that, so far, CAR T is a one-and-done therapy, Dr. Orlowski explains. When the CAR Ts are reinfused, you dont have any additional chemotherapy afterward and many of these patients who were quite sick at the beginning feel better after this than after any prior therapy theyve had.

Plus, he says, their quality of life after CAR T-cell therapy, if people have a great response, is much better, it seems, than with standard chemotherapies.

While it takes time to carefully execute the clinical trials, Dr. Orlowski shares that the results so far have been extremely positive. Researchers have learned that BCMA is a really good target because its expressed only on myeloma cells and normal plasma cells, meaning the CAR T-cells genetically engineered to attack it will not attack other tissues in the body.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Orlowski is a Professor of Medicine in the Departments of Lymphoma/Myeloma and Experimental Therapeutics, Division of Cancer Medicine, where he is board-certified in medicaloncology. Read More

While not yet approved by the FDA, clinical trials testing CAR T-cell therapy have better numbers than weve seen ever in the history of multiple myeloma, says Dr. Robert Orlowski, chair of the Department of Lymphoma and Myeloma at the MD Anderson Cancer Center.

With the living drug treatment CAR T-cell therapy already approved by the Food and Drug Administration for certain non-Hodgkins lymphomas, doctors are now looking at its tremendous promise for multiple myeloma.

CAR T-cell therapy is a one-time treatment that is both a drug and procedure. A patients immune systems T cells are extracted from their body, genetically modified in a lab to identify and attack cancer cells, and then put back into the body to do their work.

For multiple myeloma,CAR T-cell therapy most commonly targets the B-cell maturation antigen, or BCMA.

One of the nice advantages is that, so far, CAR T is a one-and-done therapy, Dr. Orlowski explains. When the CAR Ts are reinfused, you dont have any additional chemotherapy afterward and many of these patients who were quite sick at the beginning feel better after this than after any prior therapy theyve had.

Plus, he says, their quality of life after CAR T-cell therapy, if people have a great response, is much better, it seems, than with standard chemotherapies.

While it takes time to carefully execute the clinical trials, Dr. Orlowski shares that the results so far have been extremely positive. Researchers have learned that BCMA is a really good target because its expressed only on myeloma cells and normal plasma cells, meaning the CAR T-cells genetically engineered to attack it will not attack other tissues in the body.

Learn more about SurvivorNet's rigorous medical review process.

Dr. Orlowski is a Professor of Medicine in the Departments of Lymphoma/Myeloma and Experimental Therapeutics, Division of Cancer Medicine, where he is board-certified in medicaloncology. Read More

View post:
The Promise of CAR T-Cell Therapy for Multiple Myeloma - SurvivorNet

Scientists say they have used the gene editing tool CRISPR inside someones body for the first time, a new frontier for efforts to operate on DNA, the chemical code of life, to treat diseases.

A patient recently had it done at the Casey Eye Institute at Oregon Health & Science University in Portland for an inherited form of blindness, the companies that make the treatment announced Wednesday. They would not give details on the patient or when the surgery occurred.

It may take up to a month to see if it worked to restore vision. If the first few attempts seem safe, doctors plan to test it on 18 children and adults.

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We literally have the potential to take people who are essentially blind and make them see, said Charles Albright, chief scientific officer at Editas Medicine, the Cambridge, Massachusetts-based company developing the treatment with Dublin-based Allergan. We think it could open up a whole new set of medicines to go in and change your DNA.

Dr. Jason Comander, an eye surgeon at Massachusetts Eye and Ear in Boston, another hospital that plans to enroll patients in the study, said it marks a new era in medicine using a technology that makes editing DNA much easier and much more effective.

Doctors first tried in-the-body gene editing in 2017 for a different inherited disease using a tool called zinc fingers. Many scientists believe CRISPR is a much easier tool for locating and cutting DNA at a specific spot, so interest in the new research is very high.

The people in this study have Leber congenital amaurosis, caused by a gene mutation that keeps the body from making a protein needed to convert light into signals to the brain, which enables sight. Theyre often born with little vision and can lose even that within a few years.

Scientists cant treat it with standard gene therapy supplying a replacement gene because the one needed is too big to fit inside the disabled viruses that are used to ferry it into cells.

So theyre aiming to edit, or delete the mutation by making two cuts on either side of it. The hope is that the ends of DNA will reconnect and allow the gene to work as it should.

Its done in an hour-long surgery under general anesthesia. Through a tube the width of a hair, doctors drip three drops of fluid containing the gene editing machinery just beneath the retina, the lining at the back of the eye that contains the light-sensing cells.

Once the cell is edited, its permanent and that cell will persist hopefully for the life of the patient, because these cells dont divide, said one study leader not involved in this first case, Dr. Eric Pierce at Massachusetts Eye and Ear.

Doctors think they need to fix one tenth to one third of the cells to restore vision. In animal tests, scientists were able to correct half of the cells with the treatment, Albright said.

The eye surgery itself poses little risk, doctors say. Infections and bleeding are relatively rare complications.

One of the biggest potential risks from gene editing is that CRISPR could make unintended changes in other genes, but the companies have done a lot to minimize that and to ensure that the treatment cuts only where its intended to, Pierce said. He has consulted for Editas and helped test a gene therapy, Luxturna, thats sold for a different type of inherited blindness.

Some independent experts were optimistic about the new study.

The gene editing approach is really exciting. We need technology that will be able to deal with problems like these large genes, said Dr. Jean Bennett, a University of Pennsylvania researcher who helped test Luxturna at the Childrens Hospital of Philadelphia.

In one day, she had three calls from families seeking solutions to inherited blindness.Its a terrible disease, she said. Right now they have nothing.

Dr. Kiran Musunuru, another gene editing expert at the University of Pennsylvania, said the treatment seems likely to work, based on tests in human tissue, mice and monkeys.

The gene editing tool stays in the eye and does not travel to other parts of the body, so if something goes wrong, the chance of harm is very small, he said. It makes for a good first step for doing gene editing in the body.

Although the new study is the first to use CRISPR to edit a gene inside the body, another company, Sangamo Therapeutics, has been testing zinc finger gene editing to treat metabolic diseases.

Other scientists are using CRISPR to edit cells outside the body to try to treat cancer, sickle cell and some other diseases.

All of these studies have been done in the open, with government regulators approval, unlike a Chinese scientists work that brought international scorn in 2018. He Jiankui used CRISPR to edit embryos at the time of conception to try to make them resistant to infection with the AIDS virus. Changes to embryos DNA can pass to future generations, unlike the work being done now in adults to treat diseases.

Marilynn Marchione

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In first use inside human body, CRISPR tested as blindness therapy - STAT

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Stem Cell Therapy Market 2020 To 2027-Expanding Worldwide with Top Players Future Business Scope and Investment Analysis Report - Monroe Scoop

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Animal Stem Cell Therapy Market: Growth Trends, Sales Outlook, Revenue, Market Toppers, End-Users, Major Regions - ReportsPioneer

XconomyNational

Cell and gene therapies are becoming more mainstream, but an essential aspect of the drug development process is talking with clinicians about how it would fit into their practice, according to Joe Dupere, CEO of UK-based regenerative medicine company Rexgenero.

Rexgenero is advancing an experimental cell therapy for critical limb ischemia, a chronic condition that causes severe burning pain in the feet or toes as a result of blocked arteries in the lower limbs. Patients often get ulcers that can lead to amputation of the affected limb.

The company, headquartered in London, has R&D and manufacturing operations in Spain. Dupere talked with journalist Mike Ward at BIO-Europe 2019 in Hamburg, Germany, about the companys progress and plans.

Theres a lot of dialogue with the physicians and the surgeons that we cooperate with in our trial so they understand our product, its place in the treatment of this disease, and understanding when they can bring in patients and potential benefits, Dupere said.

When it comes to involving clinicians in its studies, the earlier, the better, Dupere said he learned as the company advanced its investigational treatments.

We live and breathe our design of our study, but surgeons are coming into this new, and to really understand how it works and really assist them in that journey with us so that they can bring patients in, we make it as easy as possible for them to participate, he said.

Although the type of treatment isnt as unusual as it once was, detailing how Rexgeneros cell therapies could impact how care is currently provided is essential to getting buy-in from the physicians who would use it, he said.

The challenge for us is to really demonstrate the superiority, in terms of the efficacy that we can show, and how that fits in with their practice, he said. Thats also marrying what they do already, which is a series of surgical and in endovascular procedures, [with] where the cell therapies can come in and really turbocharge that and have a really substantial impact on their patients.

As Rexgenero focuses on its Phase 3 trials, its priorities have included thinking through how the product would be used if it were to make it to commercialization.

Theres a whole range of other activities that we need to focus on as we get nearer to the market, he said.One is scaling up the manufacture, so thats a truly commercial manufacturing process that can really deliver the product to as many patients as possible. The second is really understanding how this would be used in practice, so from a commercial perspective, how would you sell this product, how would you position it with the physicians, and how would you get the surgeons to understand where this treatment would work in their general practice.

Duprere said the company also aims to expand into supplementary indications and establish a presence in the US.

Talking with investors as BIO-Europe, Duprere said he noticed more of an appreciation for biotechs that are doing cell therapy work outside of immune-oncology, a hot area for life sciences financings.

I think when you talk about [strategic investors], the core value proposition of the disease and the treatment of the disease and the unmet need of the disease has been understood from the beginning, but utilization of a cell therapy to be able to address that market is something that theyre getting much more familiar with, certainly around things like the manufacture, some of the regulation thats specific to cell therapies, and also the commercial path, when youre dealing with a curative product where you give a single dose of the product and you expect a cure for the patient.

Sarah de Crescenzo is an Xconomy editor based in San Diego. You can reach her at sdecrescenzo@xconomy.com.

Link:
Rexgenero CEO Talks Importance of Dialogue in Cell Therapy Conversations - Xconomy