Tumors shrank in one quarter of patients with advanced lung cancer who received a bespoke cell therapy made by doctors at H. Lee Moffitt Cancer Center in Tampa, Fla. In two patients, the researchers found, lung tumors disappeared completely.

Though the study was small, the results are impressive because the patients had tumors in their lungs that were not responding to Opdivo, one of the checkpoint-inhibitor immunotherapies that are now standard of care. That makes these patients particularly difficult to treat effectively.

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Custom cell therapy shrinks lung tumors in advanced cancer cases - STAT

On April 14, shares of Iovance Biotherapeutics jumped 17%, thanks to three magic words in the title of an abstract the company said will be presented at this weeks virtual meeting of the American Association for Cancer Research (AACR): durable complete responses.

The study, which will be discussed Tuesday morning, is a phase 1 clinical trial of Iovances highly anticipated tumor-infiltrating lymphocyte (TIL) product in non-small cell lung cancer (NSCLC). How durable were those responses? The abstract, released over the weekend, provides some clues.

Data from the study, being conducted at the H. Lee Moffitt Cancer Center, included results from 18 of 20 NSCLC patients treated with Iovances TILs after having received Bristol Myers Squibbs checkpoint inhibitor Opdivo. Two of the patients had complete responses to the therapy that are ongoing one year later, according to the abstract. Three other patients developed new lesions after undergoing the TIL therapy but went into remission after they received tumor-directed treatments.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

TILs are personalized cell treatments made from immune cells that have a natural ability to invade and kill cancer cells. Iovances technology involves taking T lymphocytes from patients, expanding them into the billions, and then infusing them back into the body.

Iovances most advanced TIL, called lifileucel, is in pivotal trials in melanoma and cervical cancer, with FDA filings expected sometime this year. The company has simultaneously been investigating the potential of TILs in lung cancer and several other tumor types, including cervical cancer and head and neck tumors.

Because Iovance is considered a leading player in TIL technology, its investors tend to jump on any news that hints at the effectiveness of the technology. Last year, its shares rocketed up on news that its TILs produced a 44% response rate and 89% disease control in ovarian cancer. Earlier this year, Iovances stock rose 40% on rumors that it was exploring acquisition offers.

RELATED: Iovance shares jump on buyout rumors

The small study of Iovances TILs released at AACR has already raised some questions that will be hard to answer until data emerge from larger trials. Among the enrolled patients, 13 progressed on Opdivo and were given subsequent treatments combining two chemotherapy drugs with IL-1. Thus, it seems difficult to put any benefit specifically down to Iovances TILs, said an analyst from Evaluate Vantage in a recent posting.

Analysts from Jefferies pointed out in a note issued Monday that the median shrinkage of tumors observed in the study to be presented at AACR was 38%, but that the researchers did not disclose the objective response rate (ORR). "We think this means that least half of the patients observed a partial response, but we caution that we don't know if these are confirmed responses. We think anything greater than 20% ORR would be clinically meaningful," Jefferies said.

Iovances technology was originally developed at the National Cancer Institute and has so far shown the most promise in melanoma. Some patients involved in early trials have continued to respond to the therapy for more than a decade. So its no surprise Iovance has been investing heavily to prepare for the launch of lifileucel in melanoma: Last year, it announced a $75 million investment in a manufacturing facility in Philadelphia.

Meanwhile, Iovance continues to work with research partners to investigate the potential of TILs in other cancers and to improve upon the first generation of the product. Iovance teamed up with the University of Texas MD Anderson Cancer Center in 2017 to study TILs in several tumor types. And in January of this year, it licensed technology from Cellectis that it will use to genetically modify TILs in the hopes of improving their potency, the company said at the time.

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AACR: Iovance details early responses to TIL cell therapy in lung cancer - FierceBiotech

Ventilator-dependent COVID-19 patients with moderate/severe acute respiratory distress syndrome (ARDS) who were treated with intravenous infusions of an allogeneic mesenchymal stem cell product candidate had a survival rate of 83%.

The allogeneic mesenchymal stem cell product candidate remestemcel-L from Mesoblast Limited (Melbourne, Australia) was administered within the first five days under emergency compassionate use at New York Citys Mt Sinai hospital during the period March-April 2020. Patients received a variety of experimental agents prior to remestemcel-L. All patients were treated under an emergency Investigational New Drug (IND) application or expanded access protocol at Mt Sinai hospital. 75% of the patents successfully came off ventilator support within a median of 10 days, in contrast to only 9% of ventilator-dependent COVID-19 patients being able to come off ventilators with standard of care treatment and only 12% survival in ventilator-dependent COVID-19 patients at two major referral hospital networks in New York during the same time period. These poor outcomes are consistent with earlier published data from China where mortality rates of over 80% were reported in patients with COVID-19 and moderate/severe ARDS.

The remarkable clinical outcomes in these critically ill patients continue to underscore the potential benefits of remestemcel-L as an anti-inflammatory agent in cytokine release syndromes associated with high mortality, including acute graft versus host disease and COVID-19 ARDS, said Mesoblast Chief Executive Dr. Silviu Itescu. We intend to rapidly complete the randomized, placebo-controlled Phase 2/3 trial in COVID-19 ARDS patients to rigorously confirm that remestemcel-L improves survival in these critically ill patients.

There is a significant need to improve the dismal survival outcomes in COVID-19 patients who progress to ARDS and require ventilators, said Mesoblast Chief Medical Officer Dr Fred Grossman. We have implemented robust statistical analyses in our Phase 2/3 trial as recommended by the US Food and Drug Administration (FDA) in order to maximize our ability to evaluate whether remestemcel-L provides a survival benefit in moderate/severe COVID-19 ARDS.

Related Links:Mesoblast Limited

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Stem Cell Therapy Shows 83% Survival in COVID-19 Patients Dependent on Ventilator - HospiMedica

27 April 2020

Genome-edited human stem cells from diabetic patientshavebeen shown to successfully reverse diabetes in mice.

Researchers at Washington University School of Medicine in St Louis, Missouri, used CRISPR/Cas9 to correct a genetic defect in human stem cells and then converted them into cells capable of producing insulin. When these edited insulin-producing cells were implanted into diabetic mice, they were able to effectively control blood sugar levels for six months.

'We're excited about the fact that we were able to combine these two technologies - growing beta cells from induced pluripotent stemcells and using CRISPR to correct genetic defects,' said corresponding author Dr Jeffrey Millman. 'This is the first time CRISPR has been used to fix a patient's diabetes-causing genetic defect and successfully reverse diabetes.'

The human cells used were from a patient with a rare genetic type of diabetes called Wolfram syndrome, which develops during childhood and typically requires affected patients to inject insulin multiple times each day.

'For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping-stone toward applying gene therapy to a broader population of patients with diabetes,' said Dr Millman.

The same team previously developed a new technique to more efficiently convert human stem cells into insulin-producing cells, allowing them to 'functionally cure' diabetes in mice for the first time (see BioNews1037). In the current study, they went one step further, adding the genome editing step to make cells from a diabetic personproduce insulin.

'We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation,' said Dr Fumihiko Urano, who co-led the study. 'In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes,' added Dr Millman.

'It's also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration' said Dr Urano.

The study was published in the journal Science Translational Medicine.

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Diabetes reversed in mice using CRISPR and stem cell therapy - BioNews

I dont know why people pay attention only to vaccines and treatments against the new coronavirus. Stem cell therapies are more useful to treat Covid-19.

So claimed Lee Hee-young, president of the Korean Association of Stemcell Therapy, at a news conference in Seoul, Monday. He called for active use of stem cell therapies to treat Covid-19 patients.

Several studies have proved the effects of autologous stem cells in treating acute respiratory distress syndrome (ARDS), which is the leading cause of death in Covid-19 patients, Lee said. The concept of stem cell therapy is the same as that of blood transfusion or bone marrow transplantation. Decades of cell therapies have proved that stem cell therapy is safe.

While the development of a treatment or a vaccine against Covid-19 takes a long time and it may not be able to treat patients immediately because of virus mutation possibilities, stem cell therapies can restore damaged lungs directly, Lee claimed.

It is more important to restore damaged lungs than to fight the virus. Stem cell therapy restores the lungs, giving patients time to beat the virus, he went on to say. However, people are paying attention to vaccine or treatment candidates only. This is why I am holding a news conference.

Lee pointed out that the local environment makes it difficult to use stem cell therapies. Thus, the government should ease regulations on the management and use of cell culture facilities so that doctors can perform stem cell therapies with simple cell culture, he said.

As long as physicians have a positive pressure facility and a culture kit, they can separate and culture cells with simple training, he said. If the authorities allow doctors to perform stem cell therapies with a disposable mobile culture autonomously, the cost of stem cell therapies will go down significantly.

Lee added that he asked related officials to include such rules in the Act on Safety and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals, which is to take effect in the second half of the year.

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'Stem cell therapy more effective on Covid-19' - Korea Biomedical Review - Korea Biomedical Review

CRANFORD, N.J., April 27, 2020 /PRNewswire/ --Citius Pharmaceuticals, Inc.. ("Citius" or the "Company") (Nasdaq: CTXR), a specialty pharmaceutical company focused on developing and commercializing critical care drug products, today announced that it submitted a pre-IND meeting request and supporting briefing documents to the Center for Biologics Evaluation and Research ("CBER") of the FDA under the Coronavirus Treatment Acceleration Program (CTAP) on April 24. The Company has requested the Division's feedback to support the most expeditious pathway into the clinic to evaluate a novel cell therapy in patients suffering from COVID-19-related ARDS.

The cells, called NoveCite Cells or NC-MSCs, are made by Novellus, Inc. ("Novellus"), a Cambridge-based biotechnology company, using its patented mRNA-based cell-reprogramming process. NC-MSCs are mesenchymal stem cells derived from a single donor's fibroblasts that have been dedifferentiated into an induced pluripotent stem cell (iPSC) master cell bank, thereby avoiding the need to source additional donor cells. The iPSCs are then further differentiated into a mesenchymal stem cell (MSC) therapy. Citius and Novellus plan to develop NC-MSCs for the treatment of ARDS, and last month the companies signed an exclusive option agreement.

The Company plans a multi-center randomized placebo-controlled dose-finding study followed by an expansion phase to assess the safety, tolerability, and efficacy of NC-MSCs in patients with moderate to severe ARDS due to COVID-19. The proposed trial, a Phase 1b/2 clinical trial, is titled "A Randomized Placebo-Controlled Dose-Finding Study Followed by a Dose Level Expansion to Assess the Safety and Efficacy of NoveCite MSCs in Subjects with Acute Respiratory Distress Syndrome (ARDS) Due to SARS-CoV-2 Disease (COVID-19)," or "MARCO". The primary objectives of this study are to evaluate the safety and efficacy of NoveCite cells as a treatment for subjects with moderate-to-severe ARDS due to COVID-19 and to identify therapeutic doses.

"MSCs have an established track-record of clinical safety, and have shown promise in the treatment of inflammatory lung disease," said Matt Angel, PhD, co-founder and Chief Science Officer at Novellus, Inc. "Our research has shown that the NoveCite cells, being derived from mRNA-reprogrammed iPSCs, secrete higher levels of immunomodulatory proteins than donor-derived MSCs, and have unique manufacturing advantages."

"We believe we have the key elements in place from a clinical design and manufacturing point of view to evaluate this novel cell therapy approach to deal with the current pandemic," said Myron Holubiak, Chief Executive Officer of Citius. "ARDS is a very serious complication for many patients suffering from COVID-19, and is believed to account for about 80% of the deaths in ventilated patients. There is no proven or FDA-approved treatment for it, other than oxygen therapy, including use of mechanical ventilation, and fluid management. Literature from previous investigational studies with MSCs in the treatment of lung injuries support the idea that MSCs could prove effective in treating COVID-19-related ARDS. We look forward to our FDA discussions and are excited to be at the cusp of what could be a novel and effective therapy for ARDS."

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

About Coronavirus Treatment Acceleration Program (CTAP)In response to the pandemic, the FDA has created an emergency program called the Coronavirus Treatment Acceleration Program (CTAP) to accelerate the development of treatments for COVID-19. By redeploying staff, the FDA is responding to COVID-19-related requests and reviewing protocols within 24 hours of receipt. The FDA said CTAP "uses every available method to move new treatments to patients as quickly as possible, while at the same time finding out whether they are helpful or harmful." In practice, that means developers of potential treatments for COVID-19 will benefit from an unusually faster track at the FDA to shorten wait times at multiple steps of the process.

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Novellus, Inc.Novellus is a pre-clinical stage biotechnology company developing engineered cellular medicines using its non-immunogenic mRNA, nucleic-acid delivery, gene editing, and cell reprogramming technologies. Novellus is privately held and is headquartered in Cambridge, MA. For more information, please visit http://www.novellus-inc.com.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating a license agreement with Novellus within the option period; our need for substantial additional funds; the ability to access the FDA's CTAP program for the MARCO trial; the estimated markets for our product candidates, including those for ARDS, and the acceptance thereof by any market; risks associated with conducting trials for our product candidates, including those expected to be required for any treatment for ARDS and our Phase III trial for Mino-Lok; risks relating to the results of research and development activities; risks associated with developing our product candidates, including any licensed from Novellus, including that preclinical results may not be predictive of clinical results and our ability to file an IND for such candidates; uncertainties relating to preclinical and clinical testing; the early stage of products under development; risks related to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close, and integrate product candidates and companies successfully and on a timely basis; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions, or circumstances on which any such statement is based, except as required by law.

Contact:Andrew ScottVice President, Corporate Development(O) 908-967-6677 x105ascott@citiuspharma.com

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SOURCE Citius Pharmaceuticals, Inc.

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Citius Announces Pre-IND Submission to FDA Under the Coronavirus Treatment Acceleration Program for a Novel Stem Cell Therapy for Acute Respiratory...

Given the debilitating impact of COVID-19 (Coronavirus) on the Animal Stem Cell Therapy market, companies are vying opportunities to stay afloat in the market landscape. Gain access to our latest research analysis on COVID-19 associated with the Animal Stem Cell Therapy market and understand how market players are adopting new strategies to mitigate the impact of the pandemic.

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World coronavirus Dispatch: Animal Stem Cell Therapy Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2036 2017 2025 - Latest Herald