For a range of unmet medical needs, India offers a fantastic opportunity to push cell and gene therapies: B .. – ETHealthWorld

Shahid Akhter, editor, ETHealthworld, spoke to B N Manohar, MD and CEO, Stempeutics Research, to know more about the challenges and opportunities associated with stem cell therapy.

Medical Therapies: ChallengesThe increased prevalence of cardiovascular diseases, metabolic disorders, and cancer and the emergence of a more virulent form of the existing indications have posed many challenges to the existing medical therapies. As a result, the industry and medical community are looking for something novel that can reverse the disease's trend and determine whether it is possible to repair and regenerate diseased tissues, organs, or cells.

Cellular Therapy: Market and OpportunitiesThe Indian bioeconomy was about $80 billion in 2021. The bioeconomy consists of revenues generated from various segments like vaccines, therapeutics, diagnostics, Covid vaccine and Covid testing, which was about 14.5 billion dollars in 2021. In biotherapeutics, the revenue was about $10 billion. But these revenues are coming from biosimilars and biologics. The revenue generated from cell and gene therapies in India is very small.

The landscape of cellular therapies outside India has changed dramatically over the past few years and is likely to continue to do so over the next decade. There is a big opportunity for India to showcase cell and gene therapies for many unmet medical needs, and I strongly feel cell and gene therapy will be an emerging area of science and stem cells will be a major branch of medical treatments and will be a standard of cure and practise in the years to come. India has the opportunity to become a leading global player for stem cell products and services, and the government needs to react with speed in implementing a clear policy for cell therapy. Also, India can become a good manufacturing hub for cellular therapy.

Stem Cell Therapy: Care to Cure The current medicines available today only address the symptoms, making it only a symptomatic treatment. The medicines are not addressing the root cause of the disease. For example, in diabetes, the beta islet cell degenerates due to genetic defects or other issues, resulting in insufficient insulin production and the need for insulin injections.But we are not fixing the root cause of the disease, where we had to regenerate the degenerated cells. Here, cell and gene therapies tried to address the root cause of the disease, thereby regenerating the beta islet cells so that we do not have to take repeat insulin injections.

So far, we have been using stem cells like MSCs (mesenchymal stromal cells), which are available in various parts of the body. Currently, Stempeutic's major focus is on bone marrow-derived MSCs. We have got a patented technology for isolating MSCs from bone marrow. We have the technology to multiply, expand, and ultimately produce the end product. But the current trend is moving towards engineered cells. So far, we are working on non-engineered stem cells or on god-given stem cells and figuring a way to use them for multiple indications. Now, to further improve the overall efficacy of the cell therapy product, we are working on engineering MSCs so that they can overexpress the relevant growth factors and cytokines, which are required for treating the required indications.

Stempeutics: Journey so farStempeutics is a leading regenerative medicine company with a stem cell platform technology for the treatment of immune-mediated and inflammatory diseases. We have been working in this field for the last 14 years. We are an MSC company. We focus on mesenchymal stem cells or mesenchymal stromal cells. In the last 14 years, we have been working on R&D, preclinical studies, and clinical studies to see what the potency of these MSCs is. Also, we have undertaken numerous studies in terms of testing the safety profile of this compound. We tried many indications, and ultimately, we succeeded on 2 of them: one is the peripheral vascular disease called clinical limb ischemia (CLI), and the other is knee osteoarthritis (OA).

After going through multiple phases of DCGI-approved clinical trials, the Stempeucel product has been granted manufacturing and marketing approval for the treatment of CLI and OA by the Drug Controller General of India (DCGI). Stempeucel becomes the first allogeneic cell therapy product to be approved for commercial use in India and the first stem cell product to be approved globally for CLI indications. Stempeucel product has been licenced to Cipla for CLI indication (marketed under the brand name Regenacip) and to Alkem Labs for OA indication (marketed under the brand name StemOne) for the India territory.

Stempeutics has put India on the world map of regenerative medicine. The first company to develop a drug, which was designed and developed by Indian scientists, wasStempeucel has strong patent protection, with more than 20 granted across the globe. The core Stempeucel patent has been granted in the USA, Australia, New Zealand, South Africa, Japan, China, Singapore, the UK, France, Germany, Italy, and the Philippines. Stempeucel has been classified as an "advanced therapy medicinal product" by the European Medicinal Agency and designated as an orphan drug for the treatment of CLI due to Buergers Disease. Stempeutics is the first company to come up with a cell-based drug that has gone from bench to bedside. Additionally, it is the first company to have taken the product from concept to being commercially available for treating patients.

Now that we have commercialised two indications in India (CLI and knee OA), Stempeutics is seeking partners for further clinical development and commercialization of Stempeucel in the US, Europe, and Japan for the knee OA product, whose prevalence is huge globally. In knee OA, we are taking patients who are grades 2 or 3 on the X-ray or MRI scale. Existing treatment options focus on providing temporary pain relief and reducing inflammation during the early stages of the disease without affecting its course. Stempeucel-OA has the potential to provide best-in-class pain reduction, improve stiffness, improve physical function, improve quality of life, and have the potential to regenerate or maintain cartilage and stall further disease progression for Grade 2 and Grade 3 osteoarthritis patients based on the Kellgren and Lawrence radiographic criteria.

We are happy that we could commercialise our product, Stempeucel, in India. Now our goal is to take this product from India to developed markets like the US, Europe, and Japan. In that context, we have initiated conversations with the US FDA, the EMA in Europe, and the PMDA in Japan. There are a few challenges in terms of commercialising the product in developed markets, but we are trying to improve our product based on the inputs shared by these regulators on our chemistry, manufacturing, and control.

Stem Cell: ChallengesThe three key challenges we are facing are:

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For a range of unmet medical needs, India offers a fantastic opportunity to push cell and gene therapies: B .. - ETHealthWorld

Experts discuss innovative advances driving healthcare transformation at ‘Future of Medicine 2023’ – Devdiscourse

Bengaluru (Karnataka) [India], March 10 (ANI/NewsVoir): Health and wellness knowledge platform, Happiest Health, hosted the 'Future of Medicine 2023', the first annual summit that shared insights from leading minds in medicine, research, and technology, to foster innovation in the healthcare industry. Happiest Health brought together expert speakers who discussed emerging trends in healthcare such as Artificial Intelligence (AI) and Machine Learning (ML), human gut microbiome theories, stem cell genomics, bioinformatics and other fundamental pillars of healthcare on the cusp of shaping the future of health and medicine. Speaking at the inaugural event, Ashok Soota, Chairman, of Happiest Health said, "Our vision with 'Future of Medicine' is to create an interactive experience that encourages industry leaders to explore the potential at the intersection of cutting-edge science and technology to improve human health. If we are to work towards a healthier tomorrow, the healthcare industry needs to collaborate to redefine the notion of wellness and medicine, the healthcare delivery model and the way various fields align to improve health outcomes and save more lives."

"Healthcare and medicine are entering a completely new sphere. Technology like AI/ML and the availability of data from medical devices will create a paradigm shift in healthcare from disease-focused to a health-care-centric model," said Dr Paul Salins, Managing Director, Narayana Hrudayalaya Multispecialty Hospital & Mazumdar Shaw Medical Foundation, and Vice Chairman, Skan Research Trust in his keynote address. "AI, ML, and data will now do the heavy lifting of diagnosis, taking much of the burden off doctors. This shift will enable doctors to focus on giving patients the level of compassion and care they need over long term. The model of wellness and healthcare will transform from treating illnesses to managing wellness. Hospitals will rely more on physicians than specialists, and holistic modes of treatment will play a stronger role, over symptomatic treatment." Dr Jyoti Nangalia, Clinician Scientist and Consultant Haematologist, Cambridge Stem Cell Institute, UK explained, "The mutations in blood cells make each blood cell unique, leaving us with a clear way to study age-related markers. This can help to detect onset of cancer or possibly find undetected cancer. Understanding the breadth of trajectories to adult blood cancers is still unknown, so studies like this can help improve the outcomes for blood cancers and blood disorders."

With the paradigm shift of healthcare, as more universal data is available through AI/ ML and medical devices, there will also be a shift in health-seeking behaviour-people will look more towards managing their own health as compared to how to treat their illnesses. This increases the relevance of India's Ayurveda medicine as a new modern healthcare ecosystem. Dr Narendra Pendse, Member BOG, Trans-disciplinary University, Bangalore, Medical Advisor, Institute of Ayurveda and Integrative medicine (I-AIM), Bangalore called for synergy and dialogue to break silos between all fields of medicine to explore whether the emerging field of Ayurveda biology can contribute to the advancement of Indian health science. "In the current world, where we can factor in macro-environment into health using technology and tools, Ayurveda can deliver personalised care at a mass scale, accounting for variations in geography and unique genetic make-up of each person. We need to build bridges between pure science, allopathy, naturopathy and Ayurveda to give everyone a sense of health and balance," he said.

AI and ML capabilities are already shaping the future of medicine and are at the centre of many innovative offerings that will soon become a mainstay in the not-so-distant future. The summit concluded with an interactive panel discussion on the usage of AI and medical devices for smart healthcare. Among the speakers were Graeme Brown, (Chief Business Officer, Quadram Institute Bioscience), Prof. Arjan Narbad (Transactional Microbiome Group Leader, Quadram Institute Bioscience, Norwich, England), Dr Yogesh Schouche (Principal Investigator of National Centre for Microbial Resource, National Centre for Cell Science), Dr Jyoti Nangalia (Clinician Scientist and Consultant Haematologist, Cambridge Stem Cell Institute, UK), Dr Narendra Pendse (Rheumatologist, Ayurveda, /Member BOG, Trans-disciplinary University, Bangalore, Medical Advisor, Institute of Ayurveda and Integrative medicine (I-AIM), Bangalore), Prof. Sanjeev Jain (Principal Investigator, SKAN projects at NIMHANS) and Vishal Bali, (Executive Chairman, Asia Healthcare Holdings). The discussions focused on how advancements in various fields of research are pointing to a new future for medicine, such as how next-gen sequencing is providing a better understanding of psychiatry and mental wellness, how mutations in blood can help predict diseases such as blood cancer, and how the human microbiome is emerging as a new dimension to understand the role of gut health for both body and mind. Another point of emphasis was on how inter-disciplinary collaboration across the globe and across sectors and fields will be crucial for the future of medicine. While the medical sector is familiar with collaboration, but there are issues such as intellectual property rights, which are contentious.

In line with the intent to become the leading forum for the public to hear first-hand from leading healthcare experts, The Future of Medicine will be an annual summit. "To make a tangible difference to the healthcare industry, industry leaders must keep a pulse on emerging trends. Collaboration and conversation around offering integrated solutions spanning modern science and traditional therapies drive path-breaking advancements. Our hope is for 'Future of Medicine' to become an avenue for industry leaders to regularly connect and share new knowledge that enhances the healthcare ecosystem for a healthier tomorrow." said Anindya Chowdhury, President & CEO, of Happiest Health.

Happiest Health is a global health & wellness knowledge enterprise promoted by Ashok Soota. Happiest Health provides credible and trustworthy health and wellness knowledge with views from globally renowned experts and doctors. The primary knowledge platforms are the daily newsletter, knowledge website, and newly launched monthly print magazine, and knowledge app. Happiest Health embraces scientific knowledge with a keen focus on medical breakthroughs providing kinder, gentler therapies including cell-based treatments. It also has deep coverage of integrated medicine including Ayurveda, homeopathy and naturopathy. Happiest Health's focus on wellness is holistic and energizing. They live by their Mission Statement: "Better Knowledge. Better Health." and convey its benefits to all.

For more information please visit: http://www.happiesthealth.com. This story has been provided by NewsVoir. ANI will not be responsible in any way for the content of this article. (ANI/NewsVoir)

(This story has not been edited by Devdiscourse staff and is auto-generated from a syndicated feed.)

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Experts discuss innovative advances driving healthcare transformation at 'Future of Medicine 2023' - Devdiscourse

New Solutions Continue to Show Up in the Lymphoma Treatment … – Targeted Oncology

The field of treatment for patients with lymphoma diseases like follicular lymphoma or diffuse large B-cell lymphomas [DLBCL] has undergone some significant changes with the advent of chimeric antigen receptor (CAR) T-cell therapy and bispecific antibodies, but challenges and questions remain.

In an interview with Targeted OncologyTM, Matthew Matasar, MD, chief of the division of blood disorders at the Rutgers Cancer Institute and professor at the Rutgers Robert Wood Johnson Medical School, discussed the major highlights of treatment for this patient population in newer treatments like mosunetuzumab [Lunsumio], which was approved by the FDA for patients with relapsed/refractory follicular lymphoma.1 Moreover, he breaks down recent data for patients with non-Hodgkin lymphoma and what new studies mean for this patient population now, and into the future.2

What is a top highlight for the field of follicular lymphoma?

For follicular lymphoma, we have important updates on the activity and durability of responses with the treatment with mosunetuzumab. Mosunetuzumab is a novel bispecific antibody targeting CD20 and CD3, creating an immune synapse with healthy T cells. This has been described in prior [meetings] and publications and clearly is an active medicine in patients with multimodal follicular lymphoma, with overall response rates [ORR] of 80% and complete response [CR] rates of 60%.

The important question, however, is how durable are these responses? We now have maturing data, with a median follow up now of 27 months, we see that the median duration of response, CR, and even median progression-free survival [PFS] have not yet been reached, which for me, emphasizes the point that not only is this agent highly active in relapsed FL, but the responses are quite durable.We need further follow up and certainly we have a lot more to learn, but we're looking forward to it's likely FDA approval and our ability to prescribe it in the clinic.

What about for patients with non-Hodgkins lymphoma?

In the area of non-Hodgkin lymphoma aggressive B-cell lymphoma, I presented work that we conducted with colleagues at the Dana Farber and City of Hope. Looking at improving treatments [in the] second line [for patients with] large cell lymphoma that are eligible for stem cell transplant. We know that even in this era of CAR T-cell therapy there are a subset of patients who should receive and benefit from platinum based chemoimmunotherapy with planned auto transplant in consolidation. [This is for those] with later relapsing large cell lymphoma those who ever relapse greater than 12 months beyond completion of first line therapy. That being said, the standard of care treatment has a sub optimal ORR to CR rate and we clearly need to do better for such patients.

What new data is there for this patient population?

We presented our phase 2 study called Pola-R-ICE, which incorporates the use of polatuzumab vedotion [and RICE (rituximab [Rituxan], ifosfamide, carboplatin and etoposide)], the antibody drug conjugate targeting CD79B, in combination with RICE for patients with relapsed large cell lymphoma who were eligible for subsequent consolidated stem cell transplant.2 Briefly, in the study patients received 2 or 3 cycles of the Pola-R-ICE treatment and those who had chemo-sensitive disease went on to receive a standard of care auto transplant, and then after transplant would receive polutuzumab monotherapy to complete a total of 6 doses. So, 3 doses if they got 3 cycles beforehand and 4 if they only required 2.

We showed high activity for this combination in this patient population despite a high-risk patient population putting many patients that currently would be shunted towards the CAR-T program, because of primary refractory or early relapsing disease. We nonetheless showed high ORR and CR rates and the ability to get patients to transplant. The data are still maturing, and we're looking to see what the impact of the consolidative treatment is going to look like in terms of durability of response, but very encouraging results that we were able to present.

What are your hopes for the future of treatment for patients with lymphoma?

In follicular lymphoma, clearly, we are coming upon the era of bispecific antibodies, with mosunetuzumab and competitive molecules that are being developed in this space as well. We're likely to find ourselves in a situation where we have multiple options in the treatment of [patients with] follicular lymphoma with bispecific antibodies. Important questions will remain, however, including both the durability of these agents as well as the optimal sequencing and which patients should be getting CAR T-cell therapy, which patients be getting bispecific antibodies, and how do these agents work in sequence? And how can we optimize the course of care for a patient navigating through follicular lymphoma?

In DLBCL, there's clearly still a lot of work to be done in clarifying the optimal second line treatment program for patients who should be receiving the stem cell transplant, as well as further innovation that's required in terms of novel ways of assessing response. [Other questions remain such as] should we be incorporating [minimal residual disease] decision making into the transplant decision making apparatus? And how can we best incorporate other novel therapies into multi agent programs, again, with the intention of improving outcomes for our patients with highest risk disease.

REFERENCES

1. FDA approves Genentechs Lunsumio, a first-in-class bispecific antibody, to treat people with relapsed or refractory follicular lymphoma. News release. December 23, 2022. Accessed March 1, 2023. https://bit.ly/3WJw6B3

2. Herrera A, Chen L, Crombie J, et al. Polatuzumab vedotin combined with r-ice (polar-ice) as second-line therapy in relapsed/refractory diffuse large b-cell lymphoma.. Blood(2022) 140 (Supplement 1): 10651067. doi.org/10.1182/blood-2022-165699

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New Solutions Continue to Show Up in the Lymphoma Treatment ... - Targeted Oncology

Temporal evolution and differential patterns of cellular reconstitution … – Nature.com

Childhood cancer and its treatment compromise immune functions bearing implications for the risk of infections and effectiveness of revaccinations. A plethora of studies describe the immune reconstitution after allogeneic HSCT and provide recommendations for infection prophylaxis and revaccination strategies. According to the frequency of the disease, analyses of the immune recovery after the end of therapy in pediatric patients who did not undergo HSCT focused on ALL. In this context, an early report by Alanko et al. described the recovery of blood B-lymphocytes and serum immunoglobulins7. The authors found that, based on these parameters, a sufficiently functioning immune system was established 6months after cessation of chemotherapy and concluded that prophylactic antibiotics can be withdrawn and immunizations started. In a subsequent publication, the same group later described a differential and age-dependent recovery of blood T cell subsets posttherapy9. Although their analysis showed a mean reversion to normal values by 6months, some individual patients continued to have subnormal values for up to 1year after therapy, some of whom exhibited increased susceptibility to infections. Such observations were largely confirmed in later studies in ALL11,14,19,20. Some of these analyses showed that in comparison, B-cells were reduced more significantly and for longer periods than T-cells. In a long-term follow-up, van Tilburg and colleagues showed that whereas naive B and T cells exhibited a relatively fast recovery, memory B-cells regenerated significantly slower and memory T-cells did not fully recover during the entire 5-year follow-up12. In a more recent large study including 116 participants, Williams and colleagues underlined that immune reconstitution differs between lymphocyte compartments21. Concerning the influence of treatment intensity, Ek et al. found that immune reconstitution after childhood ALL was most severely affected in the high-risk group10.

The immune reconstitution after therapy for solid tumors in children and adolescents has been characterized less extensively as compared to ALL.

In an early orienting pilot study, Cranendonk et al. retrospectively determined the effects of various chemotherapeutic drug regimens on the numbers of different blood cell types in 131 children treated for solid tumors. Apart from the general cytoreductive effects during treatment, they observed that, in the majority of the children, the lymphocyte count became normal between 1 and 12months after cessation of therapy22. Later, Alanko and coworkers focused on the hematologic and immunologic recovery in a small analysis including 11 children who had been treated for HD (n=3), Nephroblastoma (n=4), Burkitt`s Lymphoma (n=2), clear cell sarcoma (n=1) and rhabdomyosarcoma (n=1). The group described that the lymphocyte counts of most patients normalized during the first 12months after therapy. The recovery in patients with HD or Burkitt`s lymphoma was slower than in patients with nephroblastoma and radiotherapy appeared to prolong immune reconstitution15. In a later investigation, Kovacs and colleagues assessed immune recovery in 88 children receiving chemotherapy for ALL (n=43), lymphoma (n=15), bone tumors (n=20), and other solid tumors (n=10). The group determined serum immunoglobulin levels (Ig), natural killer activity (NK), antibody-dependent cellular cytotoxicity (ADCC), and T and B cell proliferation 1year after cessation of therapy. They found, that cytotoxic therapy can lead to long-term depression of the immune system, which was most prominent in patients with ALL.

For adults with HD, a treatment-associated marked long-term dysregulation of T-cell subset homeostasis has long been described17 and it had been shown that radiation therapy decreases the absolute CD4 T-cell counts23. As a consequence, antibody responsetopneumococcal vaccinewas profoundly impaired in patients who had received intensive treatment for HD24.

Comparable larger studies in pediatric cohorts have not been performed.

To illustrate varying characteristics of the cellular reconstitution after completion of chemotherapy in pediatric patients with ALL and other disease entities, we here present a comparative analysis of the course of total leukocyte, neutrophil and lymphocyte counts from 4months before until twelve months after therapy in a large pediatric cohort of n=132 patients who had been treated for ALL, HD and ES. Depending on the underlying disease and the resulting treatment regimens and modalities, we describe significant differences mainly affecting the total lymphocyte counts.

In contrast to earlier reports10, we found no differences in the cellular reconstitution between patients with ALL belonging to the HR group and those belonging to the MR or SR group. This might be explained, however, by the relatively small proportion of patients with HR in this study.

In particular, we saw a marked and prolonged post-therapeutic lymphocyte depression in patients with HD and ES as compared to patients with ALL. Lymphopenia was most distinct in individuals with HD who had received radiation therapy. This appears obvious because in the vast majority of cases the radiation field comprised the mediastinum and thus the thymic region with direct influence on T lymphocyte regeneration. In patients with HD, these findings are in line with earlier investigations in adult cohorts17,23,24.

In addition to these entity, treatment, and modality specific differences in cellular reconstitution, we observed a clear age dependency with patients aged11years showing an earlier recovery of the total lymphocyte count than patients aged 1218years. An age dependency of the post-therapeutic immune reconstitution has been proposed in different contexts in some earlier studies9,25 whereas others did not report comparable differences12. Considering the lower median age of patients with ALL compared to patients with HD and ES, this finding could contribute to the differences in lymphocyte reconstitution described between the disease entities in this study.

Overall, our study underlines that immune reconstitution after chemotherapy for childhood cancer highly depends on the underlying disease entity, the therapeutic regimen, and treatment modality as well as patient age and differs significantly between ALL and solid tumors. These discrepancies might at least in part be a consequence of the less intensive maintenance therapy applied in ALL but not in HD or ES. However, at the same time, current recommendations for infection prophylaxis and revaccination in childhood cancer have been mainly derived from pediatric patients with ALL4,26,27,28, are still rather uniform, and do not consider potential differences for patients with solid tumors. In pediatric patients, who have been treated for childhood cancers without autologous or allogeneic HSCT, infection prophylaxis is generally withdrawn between 3- and 6months2,3, and inactivated vaccines are administered from 3months and live vaccines from 6months after cessation of conventional antineoplastic therapy4.

The results of our study could potentially contribute to discussions about adjusting the recommendations and establishing differentiated disease-, therapy- and modality-specific guidelines considering the differences in cellular reconstitution and in particular the clear and long-lasting effect of irradiation on lymphocyte total counts.

We are aware that our study bears some limitations. Above all, due to the retrospective design, we could not investigate any additional immunological parameters and therefore focused on absolute leukocyte, neutrophil, and lymphocyte blood counts as surrogate parameters of immunological recovery. Moreover, we could not draw any clinical correlations, especially between the evolution of cell counts and infectious complications. In addition, the recent implementation of the bispecific T cell engager blinatumomab in the treatment protocols might further influence and thus alter cellular reconstitution after therapy in patients with ALL.

As a consequence, future joint large studies are essential to cover these aspects in a disease- and treatment-specific manner. For a few treatment protocols, such investigations have already been initiated.

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Temporal evolution and differential patterns of cellular reconstitution ... - Nature.com

Multiple Myeloma Month: A Ceres woman’s journey to treatment – Olean Times Herald

WELLSVILLE March is designated Multiple Myeloma Month MM, the second most prevalent form of blood cancer, causes malignant plasma cells to accumulate in bone marrow.

To raise awareness and put a face behind the statistics, the theme for Multiple Myeloma Month this year is #MYelomaStory.

The National Cancer Institute estimates that more than 34,470 people living in the United States were diagnosed with multiple myeloma in 2022. This number includes Kimberly Miller of Ceres, who is the receptionist at the Wilmot Cancer Institute Oncology and Infusion Center at Jones Memorial Hospital.

Millers story began in August 2022, when she started experiencing intense pain in her lower back, hip and groin. After a phone call to her Olean-based primary care provider (PCP), she was told it sounded like diverticulitis and she was advised to go to the emergency room.

That was frustrating because even with my limited medical knowledge, I knew I did not have diverticulitis, she said. I decided to wait a week and see if it got better.

The pain did not get better; it continued to get worse. When it reached the point that she could barely lift her leg to get up into the car, she called her primary care provider again, requesting an appointment to discuss her symptoms. She was again advised that she had diverticulitis; however, her PCP ordered an x-ray of her hips and spine. When the results came back, the nurse told her the images showed no acute findings.

Seeking relief from the agonizing back pain, Miller decided to seek out a chiropractor. She took the x-ray results to her appointment, and was surprised when the doctor explained in detail that what was stated as no acute findings, was actually more acute than was reported.

The chiropractor would not treat her until she had an MRI, so she called her PCP and finally got the appointment she had been requesting. She would see her primary care provider on Sept. 29.

However, on Sept. 25, the pain became so unbearable that her husband, Ed, took her to the closest emergency room, where she had an abdominal CT and a spinal CT. With no family history of cancer, she was shocked when the results came back and she was diagnosed with multiple myeloma.

It has settled in my spine, she said, noting that the cancer eats holes in bone.

From Olean, she was taken by ambulance to Buffalo General Hospital where she met with the Great Lakes Cancer team. To make her treatment more convenient, Miller was referred to Dr. Yasar Shad, an oncologist at the Wilmot Olean Oncology and Infusion Center on West State Street.

Millers treatment so far has included four cycles of chemotherapy, five sessions of radiation, and a spinal surgery.

I began radiation treatment in Olean, in mid-October, and chemotherapy on Nov. 9, she said. Dr. Shad and his staff have been a huge blessing to me through chemotherapy, and countless doctor appointments in Rochester.

She also met with a neurosurgeon, Dr. Pierre Girgis, to deal with the brittle bones and spine fractures. She had kyphoplasty surgery on her spine on March 1.

Now in her fifth cycle of chemo, Miller is anticipating an appointment with the UR Medicine Stem Cell Transplant Team to prepare for a stem cell transplant in April.

Because stem cell transplants take the immune system back to that of a newborn baby, recovery includes at least 90 days of quarantine at home, she said. I have heard very good things about the SCT process and how many patients go into remission afterward.

She said she is thankful for all of the support she has received from staff at Jones Memorial Hospital, Strong Memorial Hospital and the Wilmot Cancer Center at Strong.

I know I have many, many people praying for me, and being able to get the treatment I need closer to home has been a blessing, she said.

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Multiple Myeloma Month: A Ceres woman's journey to treatment - Olean Times Herald

Dinutuximab Beta and Interleukin-2 After Haploidentical Stem Cell … – The ASCO Post

By Matthew StengerPosted: 3/6/2023 11:51:00 AM Last Updated: 3/6/2023 12:16:04 PM

In a phase I/II trial reported in the Journal of Clinical Oncology, Flaadt et al found that treatment with the anti-GD2 antibody dinutuximab beta plus low-dose interleukin-2 (IL-2) following haploidentical stem cell transplantation (haplo-SCT) is feasible in patients with relapsed high-risk neuroblastoma. The treatment had a low risk of inducing graft-vs-host disease and led to long-term remission in some patients.

As stated by the investigators, Patients with relapsed high-risk neuroblastoma have a poor prognosis. We hypothesized that graft-vs-neuroblastoma effects could be elicited by transplantation of haploidentical stem cells exploiting cytotoxic functions of natural killer cells and their activation by the anti-GD2 antibody dinutuximab beta.

Study Details

In the study, 68 patients aged 1 to 21 years enrolled between November 2010 and November 2017 at four European centers. Patients underwent T-cell/B-celldepleted haplo-SCT from haploidentical siblings followed by treatment with dinutuximab beta and subcutaneous low-dose IL-2. Dinutuximab beta was given at 20 mg/m2 once per day on 5 consecutive days in a total of six cycles given every 4 weeks. IL-2 at 1 106 IU/m2 was added in cycles four to six on days 6, 8, 10.

The primary endpoint was defined as patients receiving six cycles; being alive 180 days after the end of trial treatment; and having no progressive disease, unacceptable toxicity, acute 3 graft-vs-host-disease, or extensive chronic graft-vs-host-disease.

Key Points

The primary endpoint was met by 37 (54.4%) of 68 patients. Median follow-up was 7.8 years. Five-year event-free survival and overall survival from the start of study treatment were 43% (95% confidence interval [CI] = 31%55%) and 53% (95% CI = 41%65%), respectively.

Five-year event-free survival among patients with complete remission (52%, 95% CI = 31%69%) or partial remission (44%, 95% CI = 27%60%) before immunotherapy were significantly improved (overall P = .26) vs patients with nonresponse, mixed response, or progressive disease (13%, 95% CI = 1%42%). Similar outcomes were observed for overall survival (overall P = .001).

The cumulative incidence of relapse or disease progression at 5 years was 49% (95% CI = 37%61%). Among 43 patients with evidence of disease after haplo-SCT, complete or partial remission was achieved in 22 (51%), with complete remission in 15 (35%).

Hematologic grade 3 or 4 adverse events occurred in 42.6% of patients. The majority of nonhematologic grade 3 or 4 adverse events were fever, pain, hypersensitivity reactions, capillary leak syndrome, elevated liver enzymes, and central neurotoxicity. Late-onset acute graft-vs-host-disease during dinutuximab beta treatment occurred in five patients (7.4%): two patients developed grade 2 and 3 acute graft-vs-host-disease of the gut, and three developed grade 1/2 skin graft-vs-host-disease.

The investigators concluded, Dinutuximab beta therapy after haplo-SCT in patients with relapsed high-risk neuroblastoma is feasible, with low risk of inducing graft-vs-host-disease, and results in long-term remissions likely attributable to increased antineuroblastoma activity by donor-derived effector cells.

Tim Flaadt, MD, of the Department of Hematology and Oncology, University Childrens Hospital, Eberhard Karls University Tuebingen, Germany, is the corresponding author for the Journal of Clinical Oncology article.

Disclosure: The study was supported by Aktion Nils, Gesellschaft fuer Kinderkrebsforschung Geltendorf, and others. For full disclosures of the study authors, visit ascopubs.org.

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Dinutuximab Beta and Interleukin-2 After Haploidentical Stem Cell ... - The ASCO Post

Cell Cryopreservation Market is Estimated to Surpass USD 52.5 Billion by 2030 at a CAGR of 22.28% from 2023-2030; Rising Incidence of Infertility…

SkyQuest Technology Consulting Pvt. Ltd.

SkyQuest, a renowned market research firm, has recently released a detailed report on the cell cryopreservation market. The report offers valuable insights and analysis on the industry, such as growth projections, segment breakdowns, emerging trends, and lucrative business prospects. This report is a reliable source of information for companies and individuals looking to invest in the market. The analysis provides a comprehensive view of the industry's current state, along with predictions for future growth and potential areas of opportunity.

Westford, USA, March 10, 2023 (GLOBE NEWSWIRE) -- The cell cryopreservation market is witnessing significant growth in North America, primarily due to the increasing cases of infertility among the population. Besides, major key players in the market for stem cells are investing heavily in research and development activities, fueling the growth of this market. In addition, the government's recent support for developing novel medical equipment also contributes to the market's growth. Furthermore, the growing geriatric population worldwide, coupled with the rising awareness about the benefits of cryopreservation cell lines and the high focus on drug development, are further expected to drive the market's growth.

SkyQuest's global research has revealed that a staggering 45 million couples and 178 million individuals of reproductive age worldwide struggle with infertility. This startling figure indicates a significant need for advanced medical treatments and technologies, including the emerging field of cell cryopreservation. Given the growing demand for infertility treatments and the increasing success rates of cell cryopreservation techniques, the market is poised for significant growth in the coming years.

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Cryopreservation is a vital technique in the medical field that involves preserving biological materials at extremely low temperatures. This process is critical in maintaining the viability of cells, tissues, and other biological constructs over extended periods. Cryopreservation is commonly used to store sperm, eggs, embryos, and tissues for transplant purposes or research studies.

Prominent Players in Cell Cryopreservation Market

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Cryopreservation Media Segment to Witness Swift Growth due to Rising Use of Cell Cryopreservation in Research, Drug Development, and Cell-Based Therapies

According to recent market research, the cryopreservation media segment emerged as the leading contributor to the growth of the cell cryopreservation market in 2022. This trend is expected to continue from 2023 to 2030. One of the key factors driving this growth is the increasing use of cryopreservation media in preserving cells used in cell therapy development. According to SkyQuest, more than 48% of all deaths globally are attributable to chronic diseases, including cardiovascular disease, type 2 diabetes, cancer, and chronic respiratory disease. This alarming statistic indicates the urgent need for innovative solutions to prevent and treat these conditions. One promising solution is cell cryopreservation, which involves freezing living cells to preserve them for future use.

The North American cell cryopreservation market is expected to hold the largest market share by the end of 2030 compared to other regions. This is due to the region's increasing healthcare expenditure on research and development activities by pharmaceutical and biotech companies, which is estimated to impact the market in the coming years significantly. In addition, the pharmaceutical and biotech companies in the region are investing heavily in R&D activities to develop new and innovative drugs and therapies for various diseases, driving the demand for cell cryopreservation.

Pharmaceutical and Biotechnology Segment to Drive High Growth as Cell Cryopreservation Widely Used to Store and Transport Living Cells and Tissues

The pharmaceutical and biotechnology sector was the frontrunner in the cell cryopreservation market in 2022, and it is expected to maintain its dominant position in the future. The increasing demand for cell-based therapies and regenerative medicines has been a major factor driving the growth of this segment. The pharmaceutical and biotechnology industry has been at the forefront of these applications, relying heavily on cell-based assays and models for drug development and testing.

The Asia Pacific region is poised to witness the highest growth rate in the cell cryopreservation market between 2023 and 2030. This growth is due to various factors, including improving healthcare infrastructure and increasing product management operations. The optimization of cryopreservation is a rapidly developing segment in this region, which is expected to boost the demand for cryopreservation cell lines in Asia Pacific countries. In addition, the acceleration of product management operations is anticipated to boost market growth further, enabling more efficient and effective distribution of cryopreservation cell lines.

To succeed in the highly competitive field of the cell cryopreservation market, companies must stay updated with the latest industry trends and capitalize on emerging opportunities. SkyQuest's report offers valuable insights and recommendations to help businesses expand their operations and make informed decisions that can lead to success in this dynamic market. By constantly monitoring and analyzing the market, companies can differentiate themselves from their competitors.

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Key Developments in Cell Cryopreservation Market

FUJIFILM Irvine Scientific, Inc. has recently made an exciting announcement regarding the use of its cryopreservation medium in a clinical trial conducted by Vitro Biopharma. The world-renowned company specializes in developing and manufacturing serum-free and chemically defined cell culture media, specifically for bioproduction and cell therapy manufacturing. The use of this medium in a clinical trial is expected to advance the field of regenerative medicine by providing a viable alternative to traditional cryopreservation methods that utilize DMSO, a toxic substance.

BioPharma Dynamics, a leading player in the cell and gene therapy market, has recently announced the expansion of its product portfolio. The company now offers chemically defined T-Cell media, DMSO-free cryopreservation solutions, and recombinant growth factors. These products are suitable for cell therapy applications at any stage. In addition, these products are designed to provide researchers and clinicians with the necessary tools to improve the efficiency, consistency, and safety of their cell therapy protocols.

LifeCell and Cellutions BioStorage have allied to provide high-quality cryopreservation services. Cryopreservation is preserving biological materials at extremely low temperatures, typically below -150C, to maintain their viability and functionality for future use. The alliance between LifeCell and Cellutions BioStorage will offer advanced cryopreservation services to various clients, including research institutions, biotech and pharmaceutical companies, and individuals.

Key Questions Answered in Cell Cryopreservation Market Report

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Cell Cryopreservation Market is Estimated to Surpass USD 52.5 Billion by 2030 at a CAGR of 22.28% from 2023-2030; Rising Incidence of Infertility...

Scherrer, Linnstaedt Earn NIH HEAL Initiative Honors | Newsroom – UNC Health and UNC School of Medicine

Gregory Scherrer, PharmD, PhD, associate professor of cell biology and physiology and member of the UNC Neuroscience Center, received a Directors Award for Excellence in Research, and Sarah Linnstaedt, PhD, associate professor of Anesthesiology, earned an honorable mention for the Directors Trailblazer award.

CHAPEL HILL, N.C. The Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative, Directors Awards recognize researchers for excellence in Research, Mentorship, Interdisciplinary Collaboration, and Community Partnership. Researchers in the early to middle stages of their careers are recognized with a Trailblazer Award or honorable mention.

Two faculty members in the UNC School of Medicine were recognized. Gregory Scherrer, PharmD, PhD, associate professor in the Department of Cell Biology and Physiology and member of the UNC Neuroscience Center, earned a Directors Award for Excellence in Research. Sarah Linnstaedt, PhD, associate professor in the Department of Anesthesiology, earned an honorable mention for the Directors Trailblazer award.

Dr. Rebecca G. Baker, director of the NIH HEAL Initiative, presented the inaugural Directors Awards at the 4th Annual NIH HEAL Initiative Investigator Meeting in February.

The NIH HEAL Initiative Directors Award for Excellence in Research recognizes HEAL-funded investigators who exemplify research excellence and have had a major, transformative impact on the pain and addiction research fields, support broad dissemination of key research, and demonstrate leadership in the scientific community.

Scherrer investigates the mechanisms that underlie pain perception and its modulation by opioids. More specifically, he studies the sensory, emotional, and cognitive dimensions of pain, and how opioids act in neural circuits to produce pain relief and their harmful side effects such as tolerance, addiction, and respiratory depression. Armed with a better understanding of the underlying biology, the Scherrer lab is pursuing new ways to block pain without causing significant side effects, and novel treatments against opioid addiction and overdose death.

In 2020, Scherrers lab received a $2.8-million, four-year NIH grant to investigate mechanisms responsible for generating the unpleasant quality of pain in the brain and the emotional suffering that play significant roles in chronic pain, a condition that affects about 100 million people in the United States alone.

For its work on opioids, the lab received another NIH grant in 2021 $7 million over five years for an interdisciplinary project to generate and make publicly available an exceptional resource for the opioid research field: a comprehensive accounting of the various brain cell types that express each of the opioid receptors and make them sensitive to opioids, as well as the cell-type-specific molecular changes that occur when these brain cells are exposed to opioids during pain treatment or during opioid abuse and addiction.

In addition to NIH support, Scherrers research is also funded by awards from foundations such as the New York Stem Cell Foundation Robertson Neuroscience Investigator award, the McKnight Endowment Fund for Neuroscience award, the Brain Research Foundation Scientific Innovator award, as well as by UNCs Yang Family Biomedical Scholar Program and Eshelman Institute for Innovation.

Together research projects ongoing in the Scherrer lab may lead to the development of a novel type of pain killers that will be more efficient against chronic pain and safer than opioids, and of treatments that can prevent or treat opioid use disorders.

The NIH HEAL Initiative Directors Trailblazer Award recognizes HEAL-funded researchers in the early to middle stages of their careers, across all disciplines, who are applying an innovative approach or creativity in their research or are expanding HEAL research into addressing the pain and opioid crisis in new directions. The 2023 awardees demonstrate the ability to develop or apply novel techniques, approaches, models, or methodologies to HEAL research.

The research in Dr. Linnstaedts lab focuses on the identification of novel therapeutic targets for the prevention and treatment of a common and morbid type of pain, posttraumatic chronic pain. Her lab uses translational research methods that include human cohort-based discovery, behavioral model work, and molecular/cell culture studies. These focused discovery efforts have identified several new therapeutic strategies with promise for safe and non-addictive chronic pain treatment.

In 2020, Dr. Linnstaedts lab received a 2.5 million, four year NIH HEAL grant to investigate a key mediator of the physiological stress response as a novel therapeutic target for the prevention of posttraumatic chronic pain. Her lab has also received multiple additional public and private grant awards to fund other areas of discovery related to chronic pain prevention.

Read about other NIH HEAL award recipients here.

About the NIH HEAL Initiative: The Helping to End Addiction Long-term Initiative, or NIH HEAL Initiative, is an aggressive, trans-NIH effort to speed scientific solutions to stem the national opioid public health crisis. Launched in April 2018, the initiative is focused on improving prevention and treatment strategies for opioid misuse and addiction, and enhancing pain management. For more information, visit: https://heal.nih.gov.

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Scherrer, Linnstaedt Earn NIH HEAL Initiative Honors | Newsroom - UNC Health and UNC School of Medicine

Cyborg technology analyzes the functional maturation of stem-cell … – Harvard School of Engineering and Applied Sciences

Using these techniques in in vitro experiments, the team discovered that the blood vessel lining cells that regulate blood flow between vessels and surrounding tissues (called endothelial cells) play a previously underestimated but crucial role in the rapid and functional maturation of stem-cell derived cardiomyocytes. When cultured together in a 3D cardiac tissue matrix, cardiomyocytes underwent extraordinary electrical maturation in the presence of endothelial cells.

Over the course of seven weeks of monitoring the developing organoids, the team observed that proximity to endothelial cells had a direct impact. Cardiomyocytes cultured next to endothelial cells matured faster compared to cardiomyocytes located farther away from endothelial cells, and they also displayed electrical characteristics typically found in healthy heart tissue.

The new insight is a leap forward for engineering stem-cell derived cardiac tissues. Experimental preclinical research in animals with human-like hearts has proven its difficult to engineer and transplant stem-cell derived cardiomyocytes that can beat in tandem with surrounding heart tissue for extended periods of time. Immature cardiomyocytes transplanted into an animals heart tend to beat to their own drum, and this electrical misfire can cause dangerous irregular heartbeats. Thats why the discovery that co-culturing stem-cell-derived cardiomyocytes with endothelial cells can create more functionally mature cardiomyocytes is so significant.

In their new paper, the team also describes using a novel machine-learning-based analysis to interpret the electrical activity captured by the tissue-embedded nanoelectronic devices, enabling continuous monitoring of the electrical waves generated by maturing cardiomyocytes of interest and enabling a better understanding of how the tissue microenvironment influences electrical stability.

Liu says the nanoelectronic devices and machine-learning-based analysis represent new platform technologies for monitoring and managing stem-cell derived tissue implants enabling scientists to culture cyborgs made from both living tissues and electronics that can be controlled with a high degree of specificity. In cardiac tissues, he envisions that someday these cyborgs could even be used in a sophisticated, real-time feedback system to detect abnormal electrical activity in cardiomyocytes and provide highly targeted voltage, acting like a nanoscale pacemaker, to help correct implanted cells and ensure they continue to beat in rhythm with the rest of the heart.

If we have both nanoelectronic sensors and stimulators, we can monitor electrical activity and use feedback to pace implanted tissues into the same frequency as surrounding tissues, Liu says. This approach could be adapted to so many other types of stem-cell-derived tissues, such as neuronal tissues and pancreatic organoids.

He also says this nanoelectronics platform approach could be used in drug screening, providing single-cell-level, continuous analysis of how tissues respond to different compounds and therapies.

Harvards Office of Technology Developmenthas protected the intellectual property arising from this study and is exploring commercialization opportunities.

Additional authors include Zuwan Lin, Jessica C. Garbern, Ren Liu, Qiang Li, Estela M. Juncosa, Hannah L.T. Elwell, Morgan Sokol, Junya Aoyama, Undine-Sophie Deumer, Emma Hsiao, and Hao Sheng.

This work was supported by the National Institutes of Health (K08 HL150335, HL151684, HL137710, and 1DP1DK130673), National Science Foundation (NSF ECCS-2038603), the William Milton Fund, a Harvard Stem Cell Collaborative Seed Grant, a Blavatnik Biomedical Accelerator Pilot Grant, an American Heart Association Career Development Award, a Boston Childrens Hospital Office of Faculty Development Career Development Award, and an Aramont Fellowship for Emerging Science Research.

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Cyborg technology analyzes the functional maturation of stem-cell ... - Harvard School of Engineering and Applied Sciences

New facility boosts UKs cell and gene therapy manufacturing capacity – Pharmaceutical Technology

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UKs ability to develop and manufacture cell and gene therapies. A 9.3 million ($11 million) grant is being used to build the facility in Bristol where therapies for currently incurable diseases, such as some forms of cancer, sickle cell disease, and cystic fibrosis can be manufactured. Personalised medicines will also be developed at the centre.

In an email to Pharmaceutical Technology, an NHSBT spokesperson said the centre will support early phase clinical trials and preclinical work, providing a route to eventual commercial scale production. The representative expected the facility to open more treatment opportunities for UK citizens saying, It will help give patients quick access to the latest treatments by increasing the number of UK patients with incurable diseases who are able to take part in clinical trials and bring new treatments into the NHS faster.

This is important due to the UKs currently limited short-scale manufacturing capacity. The NHSBT representative explained that a lack of manufacturing spaces led to delays in production slots for many gene therapy developers. In the past, researchers have often had to seek help from outside the UK, thus delaying clinical trials and patient access. The NHSBT hopes the CBC will increase the UKs competitiveness within the market.

The opening of this new facility plays into the UK governments Life Sciences Industrial Strategy. The strategy, underlines one of the governments goals of increasing the UKs manufacturing capacity for DNA-based therapeutics. Operations at the CBC will focus on building plasmids, viral vectors and recombinant proteins for the production of cell and gene therapies.

This facility will join the NHSBTs five other sites throughout the UK, which provide a combination of stem cell and immunotherapy support services, GMP cell therapy manufacturing services, and advanced therapy medicinal products (ATMP) storage and distribution. The representative said NHSBT plans to continue increasing manufacturing capacity in this way and said, We are actively pursuing collaborations with external partners.

In a statement released along with the centres launch, Steve Barclay, the UKs Health and Social Care Secretary said, The investment in this new centre will continue to develop this area of research and help provide patients the best possible care. Dr Lilian Hook, NHSBTs Director of Cell, Apheresis and Gene Therapies affirmed this sentiment saying, This will enable cutting edge research with the potential to develop cures for some critical diseases which can currently only be treated and often ultimately prove fatal. Well be supporting delivery of these curative treatments into the NHS, so patients can access them more quickly.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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New facility boosts UKs cell and gene therapy manufacturing capacity - Pharmaceutical Technology