Holloway Discusses Later-Line Treatment in Recurrent or Metastatic … – Targeted Oncology

Targeted OncologyTM: What is the recommended next-line systemic therapy for this patient with recurrent/metastatic endometrial cancer?

HOLLOWAY: The NCCN [National Comprehensive Cancer Network] guidelines preferred regimens for recurrent or metastatic endometrial cancer are carboplatin/paclitaxel or carboplatin/paclitaxel/trastuzumab. For patients with uterine serous carcinoma, there is a role for HER2 testing, IHC, [and] next-generation sequencing, and phase 3 data indicate an improved outcome with carboplatin, paclitaxel, and trastuzumab.1

Robert W. Holloway, MD

Medical Director,

Gynecologic Oncology Program

AdventHealth Cancer Institute

Orlando, FL

Other recommended options include carboplatin/ docetaxel, cisplatin/doxorubicin, and cisplatin/doxorubicin/paclitaxel, which [raises] the toxicity. There is carboplatin/paclitaxel/bevacizumab [Avastin]. My nurse practitioner told me they wouldnt approve these combinations. She said only single agent was [available], which surprised me. Now Ill have to circle back with her on that. Albumin-bound paclitaxel and topotecan [are also options]. Temsirolimus has been used with some limited success, and ifosfamide [has been used] for carcinosarcomas, which can be single agent or combined.

For biomarker-directed systemic therapy, there [are] lenvatinib [Lenvima] and pembrolizumab [Keytruda] as category 1 for nonMSI-H, non-MRD tumors and pembrolizumab for tumor mutational burdenhigh, MSI-H tumors as a single agent. Other regimens [such as] nivolumab [Opdivo] and dostarlimab [Jemperli] recently received the pan-tumor indication. For NTRK gene fusions, [you can use larotrectinib (Vitrakvi) or entrectinib (Rozlytrek)]. Avelumab [Bavencio] has been approved as a secondline option [for MRD/MSI-H tumors], and cabozantinib [Cabometyx] [has been approved] as well.

What data support the use of single-agent pembrolizumab as the next-line therapy in patients with recurrent or metastatic endometrial cancer?

The KEYNOTE-158 study [NCT02628067] is an ongoing, international, open-label, phase 2 study of patients with select solid tumors that have progressed on standard-ofcare therapy. Patients with previously [managed] MSI-H, MRD advanced endometrial cancers enrolled in cohorts D and K in the study. They had progression after a standard line of carboplatin plus paclitaxel and werent curable by other means. They received pembrolizumab once every 3 weeks. They were treated for 2 years or until progression of disease [PD] or intolerable toxicity, and then survival follow-up was done. The overall response rate [ORR] was the primary end point per RECIST version 1.1 [criteria]. The secondary end points were duration of response [DOR], progression-free survival [PFS], overall survival [OS], and safety. The median time from the first dose to data cutoff, which was January 2022, was 54.5 months, a good long-term follow-up in the study. The data are rather mature.2

The mean age in this endometrial [cancer] population was not surprising at 64 years. For ECOG PS, about 50% of them had PS of 1. Disease stage was M0 in 4% and M1 in 96%. For number of prior lines of therapy, about half of them had 1 line, but it was greater than 4 lines in about 10%. Prior adjuvant or neoadjuvant therapies were [used in] 27%, so it looks like the majority had not had neoadjuvant or adjuvant therapy. Prior radiation therapy was [noted] in 67% and prior surgery in 86%. So some of these patients did [have previous operations].

How have patients in KEYNOTE-158 responded to treatment so far?

All patients treated in the study had about a 50% response rate. Patients who had prior neoadjuvant and/or adjuvant therapy, only 10 patients, had a 40% response rate, so I dont know [whether] thats different. Patients who had 1 prior line of therapy had a 59% response rate. I think these numbers are relatively small to discriminate. Patients who had [more] than 1 prior line of therapy had a 44% response rate. Its all clustering in the 40% to 50% [range], so [its] an extraordinarily high response rate if a patient has MSI-H disease on checkpoint inhibitors, even though they had carboplatin, paclitaxel, and/or pelvic radiotherapy before in many cases. The complete response [CR] rate was 16%, partial response [PR] rate was 34%, and stable disease [SD] [rate] was 18%.2 Theres no difference in terms of survival between PR and SD. They both are benefiting.

The median DOR was 63.2 months. The DOR at 1 year was 87%, 2 years was 71%, 3 years was 66%, and 4 years was 66%. The duration and recurrence start to flatten out. If you make it to 3 years, youre going to have an ongoing response.

The PFS and OS plots plateau, which weve seen in other solid tumors [during] immunotherapy. A percentage of patients get a long-lasting DOR and benefit tremendously from the therapy. The median OS was 65.4 months [(95% CI, 29.5-not reached) and the median PFS was 13.1 months (95% CI, 4.3-25.7)].

Prior to pembrolizumab coming on the market, patients had a shortened and significantly toxic environment [for] survival. The treatment options were not good, and patients [experienced pain and died from] this disease in a short period once theyd had carboplatin, paclitaxel, and radiation. Doxorubicin was thrown in the mix there, which isnt a pleasant treatment. So this is a tremendous advance in endometrial cancer management.

Have you used dostarlimab as the next-line therapy in patients with recurrent or metastatic endometrial cancer?

I have used [dostarlimab] in a clinical trial with carboplatin and paclitaxel, and I can say anecdotallyno direct comparisonI dont think theres any significant difference in the AEs compared with any other checkpoint inhibitor. Ive treated a large number of patients on the clinical trial, which has been ongoing for over 2 years, and Ive seen some amazing responses in patients who I would have expected to [die] from their disease within a short time. So based on the OS and PFS curves, some patients do get a tremendous response.3,4

The GARNET study [NCT02715284] is the prospective study of dostarlimab, which received its first approval in endometrial cancer. It is a 2-part phase 1 trial. Part 1 is for dose finding, part 2A is for the fixed-dose and safety run-in, and part 2B is for expansion cohorts. Its another example of an early-phase study that presented data to the FDA, leading to a quick approval because of the significant response rates and benefits. The FDA did not require a large prospective phase 3 randomized trial. Pembrolizumab was already labeled in this setting, and the data points looked like the outcomes with other checkpoint inhibitors. So it probably helped lead to its approval, with no unusual signals of toxicity.

In this study, dostarlimab was dosed at 500 mg every 3 weeks for 4 cycles, then 1000 mg every 6 weeks until PD. The primary end points were the ORR and DOR per RECIST version 1.1 criteria by blinded independent central review assessment. The key inclusion criteria were progression during or after platinum doublet therapy and 2 or fewer prior lines of treatment for advanced or recurrent disease. The patients could have had hormonal therapy and carboplatin, paclitaxel or carboplatin, paclitaxel, or doxorubicin [and join] this trial. They had to have measurable disease. They had not received prior checkpoint inhibitors and had MRD or MSI-H disease by approved laboratory testing.

They had 2 scans demonstrating PD on their last systemic therapy, so they had [experienced progression]. These were not patients with stable disease. The median time from the first dose to data lock was 27.6 months. The efficacy and safety were assessed in all patients with measurable disease.

For the study characteristics, the median age was 65 to 66 years. Slightly less than half of these patients originally had stage I or stage II disease and then [experienced] relapse, and 56% to 62% had stage III or IV disease and then [experienced] relapse. Low-grade endometrioid carcinoma accounted for 64% of the MSI-H [disease] and only 23% of nonMSI-H [disease]. Serous carcinomas flipped the other way. A small percentage were MRD or MSI-H [disease], whereas a larger percentage were MR-proficient. Grade 3 carcinomas, clear cell, and other histology types had relatively small numbers after endometrioid carcinomas and serous carcinomas. All patients had prior anticancer therapy, and the majority had 1 prior line. About 70% of the patients had prior radiation therapy.

What was the efficacy demonstrated in the GARNET trial?

The ORR was 45.5%, very similar to the [ORR from] KEYNOTE158. The CR was also very similar to [the CR from] KEYNOTE158 at 16.1%. The PR was approximately double the CR at 29.4%, and SD was about 15%. Approximately one-third of patients [experienced progression]. The duration of the CR was 60.1%. Response was ongoing in 83%. The median DOR had not been reached. These data were reported without the longterm follow-up of the KEYNOTE-158 study, but the results were so similar at this point that it got FDA approval in this setting.

The estimated probability of maintaining response at 6 months was 97%, 12 months was 93%, and 24 months was 83%. What I see in these data is that if you see a patient whos getting a response, overwhelming odds are theyre going to continue to get a response. The question is, are they going to get a response? About one-third of patients will [experience progression]. Im not intimately familiar with all the research going on in immunotherapy for solid tumors, but it would be a key clinical question about whats different about these patients [with] MSI-H [disease who experience progression] on therapy. What do you do about it? Hopefully, well see more answers about that in the coming years.

As of November 2021, the data cutoff, the median duration of follow-up was about 28 months. The estimated PFS rate at 12 months was 46% and 40% at 24 months. So about 40% of the patients get a long-lasting benefit from checkpoint inhibition.

How do the KEYNOTE-158 and GARNET studies compare?

The KEYNOTE-158 study with 94 patients and GARNET [study] with 143 patients were phase 2 and phase 1 studies. They were done in patients with previously [managed] MRD recurrent or advanced endometrial cancer. The primary end points were ORR in both along with DOR in the GARNET study. The follow-up was longer with KEYNOTE-158 at 54.5 months vs 27.6 months for GARNET.2-4

The ORR was 50% in KEYNOTE-158 and 46% in GARNET. The median DOR was 63.2 months in KEYNOTE-158 and was not reached in GARNET. The 1-year DOR in GARNET was about 93% vs 87% in KEYNOTE-158 and 84% at 2 years vs 71%, respectively. So they were relatively similar, perhaps numerically slightly better in GARNET. But comparisons cannot be looked at in that manner because they are slightly different populations. The median PFS was 13.1 months in KEYNOTE-158 and 6 months in GARNET. Im not sure why there was the big difference in PFS between them because the DOR was similar. The median OS was over 65 months in KEYNOTE-158 and not reached in GARNET. The 1-year OS look[s] rather identical.2-4

I think therell be more follow-up reporting on GARNET as we get more data. My anticipation would be outcomes on these studies are going to be rather identical, and these checkpoints are probably interchangeable with the expected outcomes and AEs.

What is the safety profile of pembrolizumab and dostarlimab based on data from both studies?

For treatment-related AEs [TRAEs] in KEYNOTE-158, grade 3 and grade 4 AEs were a relatively small percentage at 12%, and treatment discontinuations were only 7%. Some AEs that were familiar with were pruritis, fatigue, diarrhea, arthralgia, nausea, hypothyroidism (which is not uncommon), rashes, decreased appetite, and myalgia. ASCO [American Society of Clinical Oncology] has covered it with detailed advice about managing AEs with checkpoint inhibitors.

When these drugs came out, we were all concerned about the AEs, but weve all seen in practice that theyre easily managed. Rarely do we get life-threatening AEs because we are monitoring these patients. So most of these things are manageable. But you do need to pay attention. The percentage of immune-related colitis was very small at 3%. For diabetes, hepatitis, [and] pneumonitis, you get down to 1%.

The GARNET safety data, compared with KEYNOTE-158 data, for AEs grade 3 or [above] had a [slightly] higher percentage. TRAEs of any grade [occurred in] 70% and grade 3 [occurred in] 17%. Treatment-related serious AEs were 11.8% in the population with MSI-H [disease] and 8.7% in the population with nonMSI-H [disease]. TRAEs leading to discontinuation were very similar at about 8%, and those leading to death were 0%. They were all managed appropriately, without toxicity or death.4 I didnt see anything unique in the safety summary of both studiesjust a small percentage of laboratory abnormalities with liver function tests, mild anemia, and low percentage of diarrhea overall.

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Holloway Discusses Later-Line Treatment in Recurrent or Metastatic ... - Targeted Oncology

Mucopolysaccharidosis Treatment Market is projected to reach US … – Future Market Insights

The globalMucopolysaccharidosis Treatment Marketis estimated to be worth aroundUS$ 2.38 Bnin 2022. With increasing prevalence of mucopolysaccharidosis (MSP) worldwide, the overall market is projected to grow at a robust CAGR of5.9%between 2022 and 2029, reaching a valuation ofUS$ 3.37 Bnby 2029.

A market study presented by FMIMucopolysaccharidosis Treatment Market Global Industry Analysis 2014-2018 and Opportunity Assessment 2022-2029, explains the significant factors influencing the current market structure.

According to research, the mucopolysaccharidosis treatment is still emerging, and different geographies have implemented standard treatment options for the condition. However, there is no universally accepted treatment pattern for mucopolysaccharidosis. Mucopolysaccharidosis falls under rare diseases, which is a complex, diverse, constantly evolving field, and there is a significant shortage of medical and scientific data related to it. Mucopolysaccharidosis treatment and diagnosis involves complex managing requirements, which include long-term care, rehabilitation support, and a continuous treatment plan.

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Research Activities Uplift Stem Cell Therapy Application in MPS Treatment

In the present scenario, the drugs that are considered for mucopolysaccharidosis treatment, such as aldurazyme, naglazyme, vimizim, elaprase, mepsevii and hunterase, are the only regulated and recommended drugs present in the market. These drugs fall under enzyme replacement therapies, however, with present research initiatives for stem cell therapies, the latter is considered a prominent mucopolysaccharidosis treatment. Clinical research shows that stem cell transplantation covers a large area that is not covered with the more frequently recommended practice of enzyme replacement therapies, which changes the current market structure for mucopolysaccharidosis treatment, prioritising the latter.

Stem cell therapy is a therapeutic option for mucopolysaccharidosis patients suffering from a severe phenotype, as research shows the method can preserve neurocognition or can even help break the progressive neurodegeneration. The method is provided with strict selection criteria, which is followed by maintained regulations. Research shows that stem cell therapy as a treatment option is gaining popularity among healthcare professionals for mucopolysaccharidosis treatment, which can be attributed to the relation of its better reach towards a normal health condition for the patient.

Enzyme Replacement Therapy Remains Lucrative for Market Investors

As per further assessments of the mucopolysaccharidosis treatment market, it has been difficult to collect epidemiological data pertaining to rare diseases, especially mucopolysaccharidosis treatment, which is likely to impede the actual estimation of the economic burden associated with the condition. The cost estimation for each mucopolysaccharidosis treatment type is affecting and, in turn, creating a more vulnerable situation for the businesses and disrupting research & development activities for each company.

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Enzyme replacement therapy is the most attractive segment for investors, however, the unavailability of reimbursement plans and precise treatment plans is encouraging most patient in developing regions to opt for symptomatic treatments instead of enzyme replacement therapy, which is still considered to be the standard mucopolysaccharidosis treatment.

Key Players Focus on Clinical Research of Treatment Models

The report segments the mucopolysaccharidosis treatment market into seven regions to elaborate on the regional trends pertaining to the treatment plan. Major players are focusing on increasing their market share in the Asia Pacific market through strategic collaborations with regional research institutes. There is a lack of awareness about rare diseases among the general public as well as in medical healthcare facilities in several emerging economies. According to a survey and industry report, it takes patients in the US an average of 7.6 years and patients in the UK an average of 5.6 years to actually receive a proper diagnosis. Moreover, it involves a team of healthcare professionals to actually get the right mucopolysaccharidosis treatment and diagnosis pattern for reported cases.

Manufacturers are in the process of introducing a considerable number of mucopolysaccharidosis treatment options, which are currently under clinical trials. An estimated 160 and above clinical trials are being performed for mucopolysaccharidosis treatment. Thus, ensuring that the manufacturers take this mucopolysaccharidosis treatment market to be lucrative and potential rich in terms of revenue. Medical research institutes play an important role in this particular market. They are expected to be the bridge between treatment plans and economical solutions for manufacturers, thus leaving an explicable and lucrative model for mucopolysaccharidosis treatment.

The mucopolysaccharidosis treatment market includes companies such as BioMarin Takeda Pharmaceutical Company Limited, Sanofi S.A., and Ultragenyx Pharmaceutical Inc. BioMarin accounts for a significant value share in the present mucopolysaccharidosis treatment market. The currently existing promising drug types are expected to face competition from emerging candidates. In addition the mucopolysaccharidosis treatment products of other companies, such as Sangamo Therapeutics, Inc., REGENXBIO Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc., and others, are also in the pipeline.

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LGMD type 2A mutation corrected by CRISPR-Cas9 gene editing |… – Muscular Dystrophy News

A new gene editing technique was able to fix the mutation that most commonly causes type 2A limb-girdle muscular dystrophy (LGMD), a new study reports.

We show an effective and precise repair to correct the most frequent [LGMD type 2A] mutation Our approach is a promising source for autologous [self-derived] cell replacement therapies for LGMD type 2A, the researchers wrote in the study, Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation, which was published in Molecular Therapy Nucleic Acids.

LGMD encompasses more than two dozen genetic disorders that feature muscle weakness that mainly affects the shoulders and hips. The most common form of LGMD is type 2A (also called type R1), which affects about 80,000 people worldwide.

LGMD type 2A is caused by mutations in the geneCAPN3. Hundreds of disease-causing mutations in CAPN3 have been documented. The most common, a deletion variant called c.550delA, originates from the eastern Mediterranean.

Scientists in Germany created a system using CRISPR-Cas9 technology to correct the c.550delA mutation. CRISPR-Cas9, adapted from molecular mechanisms that bacteria use to defend themselves against infecting viruses, works by using enzymes that can be programmed to cut DNA at a specific genetic sequence.

DNA molecules form as two strands, which gives them the famous double helix shape. The system works by cutting the two strands unevenly so one strand sticks out a bit further than the other. When the cells normal DNA repair machinery comes in to fix the cut, it fills in an extra nucleotide one of the building blocks of DNA so both strands are the same length. Essentially, the DNA repair mechanism is tricked into inserting an additional nucleotide where one had been deleted, repairing the mutation.

After testing to optimize the gene editing system and test it in research models, the researchers examined whether it could correct the mutation in muscle satellite cells with LGMD type 2A. Satellite cells are a special subset of muscle stem cells being explored as a potential avenue for cell therapy. The idea is to collect these cells, edit them to correct the mutation, then transplant them back into the patient to grow into healthy muscles.

Satellite cells are remarkable as they are quiescent [inactive] but remain able to regenerate skeletal muscle even in old age, the researchers wrote.

The system was tested on satellite cells from three LGMD type 2A patients. In two of them, both CAPN3 copiescarried the c.550delA mutation. After editing, a substantial number of genes were corrected 55% in one patient and 33% in the other.

The third patient harbored the 550delA mutation in only oneCAPN3 gene. In this patients satellite cells, the number of correct sequences rose from 50% to 79% after the CRISPR-Cas9 system editing.

Further profiling of the cells indicated that gene editing led to the increased function of the CAPN3 protein thats encoded by the gene and indicated the editing didnt induce any off-target changes in other unintended genes.

The researchers also injected patient-derived satellite cells into the muscle tissue of mice in a model of LGMD type 2A. Results showed the cells that had been corrected via gene editing were better able to grow and form new muscle tissue.

Edited and healthy control cells successfully integrate into the muscle, build new muscle fibers, and repopulate the stem cell niche, wrote the researchers, who noted that a limitation of this approach is that technical constraints mean there are usually not many cells available for any given patient. This may limit how effective cell-based therapies using this technique could be, especially when targeting large muscles in the hips. The researchers said more research is needed to improve its technical capacities and to explore cell-based strategies for treating muscular dystrophy.

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LGMD type 2A mutation corrected by CRISPR-Cas9 gene editing |... - Muscular Dystrophy News

VetStem, Inc. Announces Major Expansion of New Manufacturing and R&D Facilities – EIN News

VetStem, Inc. has acquired three new GMP manufacturing and R&D facilities in the San Diego Biopharma corridor.

Dr. Bob Harman, VetStem CEO

VetStem CEO, Dr. Bob Harman, stated, This next expansion brings on very substantial GMP manufacturing capacity under our in-house control supporting both commercial and R&D programs and provides clinical supplies for our upcoming Canine Orthopedic Phase III trial. We expect this new manufacturing capacity will help sustain the continued growth of VetStems products. Anne Hale, President and Chief Development Officer, added, It will enable us to continue to fulfill our long-term commitment to deliver our life-changing treatments to patients around the world. This expansion allows VetStem to hire more employees experienced in the cell therapy field, further accelerating the VetStem allogeneic and autologous stem cell clinical programs. These new facilities will be used for VetStem internal programs, manufacturing for its sister company, Personalized Stem Cells (PSC), and for contract manufacturing of new veterinary biologics.

As the first company to provide adipose-derived stem cell services to veterinarians in the United States and Canada, VetStem has processed over 15,000 patient samples resulting in over 35,000 stem cell treatments for animals. VetStem Cell Therapy has been primarily used for the treatment of orthopedic conditions but importantly also in a myriad of systemic diseases in neurology, cardiology, and immune-mediated conditions. Dr. Harman stated, The data derived from treating these real disease conditions is immediately available to help translate the discoveries into human medicine in our sister company, PSC. In a first-ever translational medicine achievement, PSC used VetStem dog and horse stem cell data to go directly from the company founding in 2018 to a Phase 2a clinical study in human knee arthritis in less than 7 months. In addition to domestic animals, VetStem has worked with multiple exotic animal organizations (including Sea World, the San Diego Zoo, and the San Diego Zoo Safari Park) to provide stem cell therapy for exotic species such as Dolphins, Sea Lions, Cheetahs, Elephants, multiple species of Rhinoceros, and 30 others.

About VetStem, Inc. VetStem is a veterinarian-led Company that was formed in 2002 to bring regenerative medicine to the profession. This privately held biopharmaceutical enterprise, based near San Diego, California, currently offers veterinarians an autologous stem cell processing service (from patients own fat tissue) among other regenerative modalities. With a unique expertise acquired over the past 20 years and thousands of treatments by veterinarians for joint, tendon and ligament issues, VetStem has made regenerative medicine applications a therapeutic reality. The VetStem team is focused on developing new clinically practical and affordable veterinary solutions that leverage the natural restorative abilities present in all living creatures. In addition to its own portfolio of patents, VetStem holds exclusive global veterinary licenses to a large portfolio of issued patents in the field of regenerative medicine.

Kristi Hauta, Director of Commercial OperationsVetStem, Inc.+1 858-748-2004email us here

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VetStem, Inc. Announces Major Expansion of New Manufacturing and R&D Facilities - EIN News

Stem Cell Therapy: NeuroGen director in soup for allegedly violating NMC direction – Medical Dialogues

As per various media accounts, the doctor allegedly violated the Indian Council of Medical Research (ICMR) guidelines by providing Stem Cell Therapy (SCT) treatment to more than 12500 patients of autism and cerebral palsy from 75 countries that costed between Rs 5 lakh to 10 Lakh.

Taking action, the civic health department revoked his registration after carrying out a number of inspections between December 26 and February 22, on complaints from patients that false promises were made by the NeuroGen Brain and Spine Institute in Seawoods for treatment of Autism, and many parents lost lakhs of rupees on treatment, but the result was not as promised.

Moreover, several irregularities in maintaining files and advertising their services, paved the way for the action against NeuroGen.

NMMC medical officer health Dr Pramod Patil stated, "Our action was taken after detection of violations followed by complaints and after serving notice. More action is likely to follow with regards to misuse of stem cell therapy and professional misconduct."

"The hospital will soon be sealed and face other actions as per law," added civic officials.

On December 06, 2022, the National Medical Commission (NMC) directed that Stem cell therapy is not recommended as a treatment for Autism Spectrum Disorder (ASD) in clinical practice. ln view of the above recommendation, the use of Stem cells in ASD, its promotion and advertisement will be considered as professional misconduct. Further research needs to be conducted and encouraged in terms of well-designed Double-blind RCT's to explore the safety and efficacy of Stem Cell Therapy in ASD.

Also Read: NMC Slams Stem Cell Therapy For Treating Autism Spectrum Disorder, Declares It As Professional Misconduct

In response to the matter, the director of NeuroGen has decided to approach the Ministry of Health for clarification on using the therapy for the treatment of autistic patients.

The decision has been taken on the recommendation of the Ethics & Medical Registration Board (EMRB) of the National Medical Commission (NMC), Dr Sharma told FPJ.

He further claimed that there is a list of practices and procedures of the Ministry of health which says Stem cell therapy can be used in Autism.

Moreover, commenting on the action taken by the NMMC, Dr Sharma told TOI that the action was hasty and "incorrect". He added that there is widespread confusion over the term "stem cell therapy in India and few realise that he offers cell therapy for autism and other neurological conditions.

However, Sion hospital dean Dr Mohan Joshi stated, "Dr Sharma is with our hospital, but medical professionals are allowed to practice elsewhere as per law. The complaints against him will be checked. Appropriate legal action will be taken after the government authorities in their respective areas act."

Sources told TOI that the doctor, who is due to retire from Sion Hospital, could face action soon.

Media report claims that the doctor had also conducted a clinical trial for stem cell therapy in 2016 in Sion hospital and had faced opposition. As per medical experts, stem cell therapy is still a grey zone and its commercial use is prohibited.

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Stem Cell Therapy: NeuroGen director in soup for allegedly violating NMC direction - Medical Dialogues

Cell therapy first for chronic heart failure – European Pharmaceutical Review

In a Phase III trial, a new cell therapy has shown potential for the first time to treat inflammation, a major contributor to heart failure.

The largest cell therapy trial to date for chronic heart failure due to low ejection fraction (EF), has shown that mesenchymal precursor cells (MPCs) have potential for the first time to address inflammation.

This study examined the effects of [MPCs] a specific type of stem cell obtained from the bone marrow of healthy adult donors, the studys lead author, Dr Emerson C Perin, PhD, FACC, Medical Director at The Texas Heart Institute shared with EPR.

For the first time, weve discovered that stem cells can successfully treat the inflammation that causes heart failure.

For the first time, weve discovered that stem cells can successfully treat the inflammation that causes heart failure. In the simplest terms, the stem cells are injected directly into the heart muscle and shut down inflammation and begin to heal the cells, he summarised.Based on the findings, Dr Perin explained to EPR that the trial is significant because the condition currently is not addressed with medications, which treat the symptoms.

In findings published in the Journal of the American College of Cardiology, the stem cell therapy reduced the risk of heart attack and stroke and improved the hearts pumping ability, especially in patients who have high levels of inflammation. The treatment, Perin observed, gave patients a 58 percent reduced risk of heart attack or stroke, and the benefits rose to 75 percent in patients with high inflammation.

the MPCs can protect cardiac muscle cells from dying and can improve blood flow and energetics. In large blood vessels throughout the body, the reduced inflammation resulting from the activation of MPCs may decrease plaque instability, which is what leads to heart attacks and strokes, explained Dr Perin.

The Phase III trial, DREAM-HF (Double-Blind Randomized Assessment of Clinical Events With Allogeneic Mesenchymal Precursor Cells in Heart Failure), assesed the effects of MPCs on the number of hospitalisations and major adverse cardiovascular events in heart failure.

The 565 patients were also on standard-of-care heart failure treatment. Therefore, the results suggest that the effect of the cell therapy was synergistic with and additive to state-of-the-art heart failure medications.

MPC-treated patients showed significant strengthening of the left ventricular muscle within the first 12 months. This was determined by an increase in left ventricular ejection fraction, which measured the hearts pumping abilityone of the metrics used to assess overall heart function.

The results help to identify those heart failure patients with inflammation at greatest risk and most likely to benefit from MPC therapy. The findings will be confirmed in future studies.

As the largest cell therapy trial in patients with heart failure to date, this study provides new insights into how cell therapy works, including both local and systemic effects, and can serve as a roadmap for other uses for conditions caused by inflammation, such as atherosclerosis, Dr Perin told EPR.

He concluded that the trail results revealed long-term improvements in outcomes for patients with heart failure and are an important milestone in the field of cardiac cell therapy.

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Cure for Parkinson’s? First patient receives stem cell transplant to treat disease – Study Finds

LUND, Sweden Stem cell transplants may finally provide patients with Parkinsons disease with hope of a cure. Doctors at Skne University Hospital successfully transferred a stem cell-derived nerve cell to a Parkinsons patient for the first time ever in February 2023. It represents a major breakthrough in the treatment of the neurological disorder, according to researchers at Lund University, who developed the procedure.

More specifically, scientists generate the transplantation product using embryonic stem cells. They serve as a replacement for the dopamine nerve cells those with Parkinsons typically lose as the disease progresses. The initial patient is just the first of eight with Parkinsons disease who will receive the transplant.

This is an important milestone on the road towards a cell therapy that can be used to treat patients with Parkinsons disease. The transplantation has been completed as planned, and the correct location of the cell implant has been confirmed by a magnetic resonance imaging. Any potential effects of the STEM PD-product may take several years. The patient has been discharged from the hospital and evaluations will be conducted according to the study protocol, says Gesine Paul-Visse, principal investigator for the STEM-PD clinical trial, consultant neurologist at Skne University Hospital, and adjunct professor at Lund University, in a media release.

Roughly eight million people live with Parkinsons disease worldwide. The main characteristic of the disease is a loss of dopamine nerve cells deep in the brain, resulting in issues with movement control such as tremors in the hands. Current standard treatments for Parkinsons disease usually include meds that replace the lost dopamine, but over time, these medications often end up becoming less and less effective and may cause harmful side-effects.

Currently, there are no treatments capable of repairing the damaged structures within the brain or replacing the lost nerve cells.

Now, the STEM-PD trial is testing a new investigational therapy developed to replace the lost dopamine cells with healthy cells created via stem cells. Using the Swedish Medical Products Agencys quality standards, researchers subjected their cell product to rigorous pre-clinical tests. After transplantation, scientists expect the cells to mature into new and healthy dopamine-producing nerve cells in the mind.

With this trial, we hope to demonstrate that the cell product works as expected in patients. Over time, this creates the opportunity to help many more people with Parkinsons in the future. adds Lund University professor Malin Parmar.

Parmar leads the STEM-PD team in close collaboration with their colleagues at Skne University Hospital, Cambridge University, Cambridge University Hospitals NHS Foundation Trust, and Imperial College London.

Further studies are required to move STEM-PD from this first in human trial all the way to a global treatment, and we have therefore worked in close collaboration with the pharmaceutical company Novo Nordisk A/S.Their input to the study, as well as operational and regulatory guidance, have been fundamentally important toinitiatethisfirst in human studyand we look forward to future collaborations.

All in all, eight patients from Sweden and the United Kingdom will undergo this transplantation at Skne University Hospital, which just so happens to have a long tradition of this type of surgery. The surgical instrument surgeons use in the current trial was originally developed by the hospital for cell transplantations during the 1980s. Back then, stem cells werent readily available, so neurosurgeons transplanted nerve cells taken from fetal tissue.

The brain region that the cells are transplanted into in this trial can be as narrow as four millimeters. The surgical instrument has a very high level of precision, and we are greatly helped by modern imaging techniques says consultant neurosurgeon Hjlmar Bjartmarz, who actually carried out the transplantation surgery.

All patients in this trial having been living with Parkinsons for at least a decade. The team considered each of them to be at a moderate stage in their disease progression. The researchers will continue following these patients closely, assessing cell survival rates and potential effects over the coming years.

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Cure for Parkinson's? First patient receives stem cell transplant to treat disease - Study Finds

ROSALIND FRANKLIN UNIVERSITY GRANTS TO TACKLE … – PR Newswire

NORTH CHICAGO, Ill., March 2, 2023 /PRNewswire/ -- New funding for Rosalind Franklin University researchers will help revolutionize therapeutic approaches for HIV-related cancers and for brain repair.

Dr. Neelam Sharma-Walia, associate professor of microbiology and immunology and investigator in the Center for Cancer Cell Biology, Immunology and Infection, has been awarded a two-year, $401,115 grant from the National Cancer Institute to investigate treatments for Kaposi Sarcoma (KS)-Associated Herpes Virus (KSHV).

"KSHV, a human oncogenic herpesvirus, is the underlying cause of KS tumor (lesions) and a rare B cell malignancy called primary effusion lymphoma (PEL)," Dr. Sharma-Walia said. "KS is the most common vascular malignancy causing high morbidity and mortality in HIV-infected patients."

Her study, "Anti-nucleolin Aptamer AS1411: Applications in Kaposi's Sarcoma Associated Herpes Virus (KSHV) Biology," aims to transform current therapeutic approaches to treat KS and PEL. Knowledge obtained from the study can be tested and applied to cancers caused by other human tumor viruses.

Dr. Daniel Peterson, professor of neuroscience and director of RFU's Center for Stem Cell and Regenerative Medicine, has been awarded a one-year, $90,000 grant from the regional Walder Foundation to investigate how non-neuronal glial cells can be reprogrammed to replace lost neurons. The project represents a collaboration with Illinois Institute of Technology's (IIT) Dr. Ali Cinar, professor of chemical engineering, who received a similar Walder grant and will contribute expertise in artificial intelligence and engineering to optimize glial cell progenitor culture processes.

Dr. Peterson's project will culture human skin cells and use gene therapy to reprogram them into human glial progenitor cells that, once generated, can target cells in the human brain and convert them to new neurons to ultimately repair the brain.

"Recent progress in understanding the biology of stem/progenitor cells in these tissues suggests that stem cells could be a potential source of cells for replacing lost neurons or heart muscle," Dr. Peterson said.

Dr. Ronald Kaplan, RFU executive vice president for research, commended both studies, stating: "These translational research efforts are key to discovering new therapies for cancer and brain diseases and are core to our research mission."

ROSALIND FRANKLIN UNIVERSITYencompasses six colleges and more than 10 research centers and institutes. Learn more atrosalindfranklin.edu.

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Life-Changing Stem Cell Treatment Shaw Local – The Herald-News

Stem cell therapy has the potential to significantly change peoples lives, as it did for one woman. Darniece experienced a spinal cord injury in 2015 that left her dependent on others and struggling with constant pain. To manage her symptoms, she tried medications, chiropractic care, and holistic approaches, but nothing worked until Chicago Stem Cell Therapy & Pain Management Institute offered her an opportunity to try stem cell treatment.

Stem cells can repair and regenerate healthy tissue. Through exosome infusionssignaling proteins derived from stem cellsdirectly into the bloodstream, Darniece was able to reduce inflammation and pain and rejuvenate aging cells.

Over three months of treatment, she gradually saw a transformation in her life. She could speak more easily and clearly, eat on her own, think with greater clarity, and engage more actively in conversations.

As you can see, there are more than physical benefits that come with stem cell therapy. Darnieces story highlights how this kind of treatment can effectively restore independence and dignity to patients who have suffered neurological damage or degenerative disorders. It offers improved physical health and mental clarity. Furthermore, these treatments allow families affected by such illnesses to feel reconnected.

She is living her life again - its like she is back and really with us!

Stem cell treatment is a second chance for people struggling with pain and degenerative disorders. It has the potential to change peoples lives for the better, and we still have much to learn about its effectiveness.

Stem cell therapy is emerging as one of the most promising treatments for neurological diseases today. Its use continues to expand across different medical fields thanks to its regenerative capabilities. Every day, it brings more hope and optimism to those coping with traumas or age-related issues, offering them a brighter future.

At Chicago Stem Cell Therapy & Pain Management Institute, our mission is to provide the highest quality, comprehensive care and maximize the potential of stem cell treatment for each patient we serve. Contact Dr. Anwar at 815-412-6187 to learn how you can benefit from this life-changing treatment.

Chicago Stem Cell Therapy & Pain Management Institute

10181 W Lincoln Hwy

Frankfort, IL 60423

http://www.ChicagoStemCellTherapy.com

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Life-Changing Stem Cell Treatment Shaw Local - The Herald-News

New hydrogel stem cell treatment repairs injured brain tissue in mice – Science Daily

A new 'hybrid' hydrogel, which allows clinicians to safely deliver stem cells to the site of a brain injury in mice, has been developed by researchers from the University of Melbourne and the Australian National University.

A hydrogel is a water-based gel that can be used to deliver substances into the body and can be used to promote the effective growth of new cells.

Published in Nature Communications, the proof-of-concept breakthrough solves a major challenge faced by stem cell researchers since the 1980s -- keeping stem cells alive for long enough to allow them to evolve into the cells required to create new tissue when they are inserted into a damaged part of the body.

The hydrogel supplies both the stem cells and oxygen needed to keep stem cells alive during the injection process and to ensure the stem cells evolve into the type of cells needed to create new tissue to repair damage. Researchers believe this advance will benefit stem cell treatments in many other parts of the body beyond the brain and central nervous system.

The team that developed the hydrogel is co-led by University of Melbourne Professor David Nisbet, Director of The Graeme Clark Institute for Biomedical Engineering; and The Australian National University (ANU) Professor Colin Jackson, a member of both the Innovations in Peptide and Protein Science, and Synthetic Biology Australian Research Council Centres of Excellence.

Professor Nisbet said: "After an injury such as a stroke, there is a dead area in the brain, including the blood system. So, we need a temporary blood supply to support cells until the blood system repairs. This patented hydrogel provides that.

"Very few drug treatments can treat conditions like stroke or Parkinson's Disease and they have little efficacy. There are currently no treatments that can reverse these conditions."

Professor Jackson said the breakthrough will interest researchers and clinicians globally and is likely to lead to many revolutionary medical treatments.

"Proof of concept has now been demonstrated within the brain of mice, but the research represents a generalisable strategy for developing injectable nanomaterials for a diverse range of applications, including cell transplantation, gene and drug delivery, 3D in vitro disease models and organ-on-a-chip technology," Professor Jackson said.

Over five years of research, the team discovered that a synthetic protein based on myoglobin -- a natural protein found in high concentrations in the heart muscles of sperm whales and horses -- added to their hydrogel provided the sustained oxygen release needed to ensure stem cells survive the delivery process and develop into the type of cells needed to repair brain tissue.

Whales and other deep-diving animals are thought to have evolved high concentrations of myoglobin in their muscle tissue so they could slowly absorb as much oxygen as possible while diving. Similarly, horses are thought to have evolved higher concentrations of myoglobin so they could run over longer distances.

University of Melbourne Professor Clare Parish conducted the mouse studies and said the results were achieved in injured brain tissue, raising the possibility for growing new tissue for future human treatment.

"We saw that the hydrogel incorporating myoglobin and stem cells repaired injured brain tissue. Analysis at 28 days after delivery of the hydrogel revealed significantly enhanced survival and growth of the new stem cells that are needed for healthy brain functioning, compared with a hydrogel without myoglobin," Professor Parish said.

"We observed that the new tissue could be stimulated in a similar way to healthy brain tissue, providing the first evidence of the benefits of including oxygen delivery within a hydrogel to achieve the long-term survival and integration of stem cell transplants."

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New hydrogel stem cell treatment repairs injured brain tissue in mice - Science Daily