Orphazyme A/SCompany announcement                                                                                       No. 66/2020                                                                                                           Company Registration No. 32266355

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Stabilization measures taken

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VANCOUVER, British Columbia, Oct. 16, 2020 (GLOBE NEWSWIRE) -- MYDECINE INNOVATIONS GROUP, INC., (CSE: MYCO) (OTC: MYCOF) (FSE: 0NFA) (“Mydecine” or the “Company”) is pleased to announce the appointment of UK-based Boustead Capital Markets LLP (“Boustead”) to commence the dual listing process on the London Stock Exchange (“LSE” or the “Exchange”) for the admission of the Company’s common shares to the Standard Segment of the Official List’s Main Market.

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Mydecine Innovations Group Appoints Boustead Capital Markets LLP as Financial Advisor for its Planned Dual Listing on the London Stock Exchange

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– Agios Continues to Advance Two Phase 3 Combination Trials of TIBSOVO® in Newly Diagnosed AML Patients –

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Agios Announces Withdrawal of European Marketing Authorization Application for TIBSOVO® as a Treatment for Relapsed or Refractory IDH1-mutant Acute...

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DEER PARK, Ill., Oct. 16, 2020 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the closing of its previously announced offering of 3,220,000 shares of common stock at a public offering price of $7.00 per share. The total offering included 420,000 shares sold as a result of the underwriter’s exercise of its overallotment option in full.

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Eton Pharmaceuticals Announces Closing of Public Offering

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A one-year-old girl donated her bone marrow to her brother. (Representational)

A one-year-old girl, conceived by her parents through IVF technology specifically for the purpose of donating her bone marrow to their thalassemic son, has succeeded in saving her six-year-old brother's life.

Baby Kavya was born a year ago through In-Vitro Fertilisation (IVF) technique in Ahmedabad, under the concept known as "saviour sibling".

Her bone marrow was successfully transplanted to her brother Abhijeet Solanki in March this year and the boy is now "risk-free", doctors said on Thursday.

Abhijeet, the second child of Sahdev Singh Solanki and Alpa Solanki, was diagnosed with Thalassemia-major, a blood disorder, and was dependent on blood transfusion every month, they said.

Thalassemia-major patients require frequent blood transfusions and their life expectancy is also less.

His parents were advised bone marrow transplant as the last resort to treat the child, but they could not find the required HLA (human leukocyte antigen) match.

"Due to unavailability of matching HLA donors for the transplant, we opted for IVF with HLA matching," city-based Nova IVF Fertility's medical director Dr Manish Banker told PTI.

This process of HLA typing is an established method for conceiving a child, who may donate cord blood or hematopoietic stem cells for transplantation to save a sibling with a critical illness.

Abhijeet's father approached Mr Banker after he found that the bone marrow of his family members, including the boy's elder sister, was not matching.

"Bone marrow transplant from an HLA-identical donor is the best therapeutic option for thalassemia major patients. We took the challenge and created a healthy savior sibling to save her elder brother," Mr Banker said.

With the help of IVF, Abhijeet's mother delivered a healthy baby girl Kavya a year ago, who was found to be the HLA match for the sibling.

In March this year, after Kavya gained the required weight, a successful bone marrow transplant was done for Abhijeet at the CIMS Hospital, Mr Banker said.

"Now, Abhijeet is risk-free and doesn't require blood transfusion," Mr Banker said.

"This is the first case in India when an HLA matching baby was born through IVF specifically to save the thalassemia-major sibling," he said.

The siblings' father, who himself researched well about this technique, thanked Mr Banker and other expert doctorsfor saving his son.

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Child Conceived To Donate Bone Marrow Saves 6-Year-Old Brother's Life - NDTV

Bone Marrow Stem Cells Comments Off on Child Conceived To Donate Bone Marrow Saves 6-Year-Old Brother’s Life – NDTV | October 17th, 2020

India just conducted its first successful experiment with savior sibling therapy, in which a baby was conceived through in-vitro fertilization for the purposes of donating bone marrow to an older ailing brother struggling with thalassemia, a condition characterized by low levels of hemoglobin in the blood that requires frequent blood transfusions. While the doctors involved in the therapy celebrated their success this week, some on social media challenged the ethics of such a therapy, in which a baby was essentially birthed to save her sibling.

In this case, the child with the genetic disorder needed a bone marrow transplant to cure his disease, and the chances of a successful cure are higher if coming from a person whose proteins (human leukocyte antigens, or HLA) exactly match those of the child. None of the childs existing family members was a match, further complicating the process of getting a bone marrow transplant an already difficult process to execute. The parents, in an effort to create a perfect bone marrow match for their child, underwent three cycles of in-vitro fertilization, out of which 18 embryos were created, and one perfectly matched that of the child, and was disease-free, using a technique called pre-implantation genetic diagnosis (PGD). The embryo was then implanted in the mothers uterus, carried to term, and a baby girl was born.

We had to wait for the baby to grow. She had to weigh 10 kg before we could draw bone marrow, Deepa Trivedi, program director of Sankalp Bone Marrow Unit in Ahmedabad, told The Hindu. Its been approximately seven months since the transplant, and the older sibling has not needed any more blood transfusions, indicating he has been cured of his thalassemia, his doctors announced.

Savior sibling therapy has already been used in countries such as the United Kingdom, the U.S.A., New Zealand, and France. Its mainly used to cure genetic blood disorders in children, such as sickle cell anemia or as seen in the Indian case, thalassemia major. The main way this is done, which is a departure from the Indian case, is by harvesting stem cells from a newborns umbilical cord, which are then injected into the bone marrow of the sibling with the disease, a practice that works 90% of the time. In case it doesnt, doctors can take bone marrow from the savior sibling as they grow, in a process that is painful but not known to be dangerous.

Related on The Swaddle:

Designer Babies Are Far From Reality, Even After the Gene-Edited Babies in China

The first ethical concern with this practice is treating a baby as a source for spare parts, as a means to an end, as a commodity. A study of the bioethics of savior sibling therapy, published in the Journal of Medical Ethics, surmised that treating a baby as a means to an end was not by itself a concern that devalued the utility of savior sibling therapy, as long as theyre also treated as human beings. Bioethicists surmise that using cord blood, something that is frequently discarded after birth, cannot endanger a newborn, or prove to be an ethical quandary used against the therapy.

But what has happened in the most recent case in India actually complicates the issue, because its not the umbilical cord blood that was harvested from the savior sibling at birth, but bone marrow 10 months into her life, which makes her an organ donor. This traverses thorny territory, as governments strictly regulate organ donation by minors due to issues related to consent. Can a baby consent to donating bone marrow to their sibling, or a 10-year-old consent to donating a kidney to their parent? It depends on where the individual resides, and how old the person being asked to donate is. In India, for example, it was only recently that the Delhi High Court ruled that minors could donate organs or tissues, as long as the procedure didnt pose a danger to their lives, and only in exceptional circumstances. However, where minors are mostly dependent upon their families, an element of coercion can also manifest. Also, determining whether a child rationally consented to donate an organ to their parent, for example, becomes difficult when we factor in the emotional element of their relationship that can perhaps override their judgments about their own safety.

Another concern is the well-being of the savior sibling throughout their life, both physical and psychological. Whats to stop a parent from asking the savior sibling to be on standby for their entire lives for their siblings health, available to be tapped for tissues and organs at any point in their lives? This is the plot of Jodi Picoults My Sisters Keeper (also turned into a film of the same name starring Cameron Diaz), but it is an unlikely scenario in real life, ethics experts have said. The aforementioned organ donation rules can prevent such an exploitative situation from arising, they say, with governments around the world tasked with ensuring the consent of the donor remains at the forefront of organ donation.

The third issue with savior sibling therapy arises out of the process itself if a parent can select an embryo that perfectly matches their child, whats to stop them from selecting an embryo for intelligence, or athleticism? This wades into the territory of the production of designer babies, which is an ethical slippery slope that critics have said goes against the natural reproductive order. However, the bioethics study asserts that the connection between savior sibling therapy and the production of designer babies is less of a slippery slope and more of a reach, as the technology might be similar, but the utility of both poles apart the former is used to save childrens lives, while the latter is a superficial, hypothetical fantasy.

For now, the world of savior sibling therapy, and its perception, remains similar to when parents first selected an embryo to create a savior sibling in the U.S. in 2000. As appeared in a New York Times article at the time, It is the kind of talk heard with every scientific breakthrough, from the first heart transplant to the first cloned sheep. We talk like this because we are both exhilarated and terrified by what we can do, and we wonder, with each step, whether we have gone too far. But though society may ask, How could you? the only question patients and families ask is, How could we not? 20 years later, savior sibling therapy still centers the children that can be saved, while government stipulations around the world try to ensure the savior siblings are protected, cared for, and treated as human beings, like any other child.

While a few critics argue for a ban, the bioethics study sums up the dilemma, and perhaps a solution to this ethical debate given that a ban will be fatal for a section of the population, the onus of proof rests clearly with the prohibitionists who must demonstrate that these childrens deaths are less terrible than the consequences of allowing this particular use of PGD.

You have got to have a very powerful reason to resist the means by which a childs life can be saved.

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An Indian Baby 'Savior Sibling' Just Gave Her Brother Bone Marrow. But Is It Ethical? - The Swaddle

Bone Marrow Stem Cells Comments Off on An Indian Baby ‘Savior Sibling’ Just Gave Her Brother Bone Marrow. But Is It Ethical? – The Swaddle | October 17th, 2020

Cecelia Ahern, the author of P.S. I love you, once beautifully said: Moments are precious; sometimes they linger and other times theyre fleeting, and yet so much could be done in them; you could change a mind, you could save a life and you could even fall in love.

Helping save lives is what we decided to dedicate some of our lives to at Manchester Marrow.More specifically, we are the student-ran arm of the charity Anthony Nolan, which signs up students/young people (aged 16-30 years) to the stem cell register. This is required in finding matches for patients suffering from blood cancers and blood disorders who desperately need transplants. The more people we sign up for this register, the higher the chance of finding a blood stem cell or bone marrow match.

Anthony Nolan was initially founded by Shirley Nolan in 1974, realising the hardships associated with requiring an urgent bone marrow transplant. This was due to her three-year-old son suffering from a rare blood disorder known as Wiskott-Aldrich Syndrome. This inspired her to set up the worlds first register to match donors with people in desperate need. Today, there are over 800,000 people on Anthony Nolans UK register list, and each of these people could be a potential donor and save a life.

Although there are many resources at hand, without you, theres no cure! In Marrow, we have three important missions: raise awareness of Anthony Nolan and blood cancer within UK universities through our events, encourage every student to join stem cell register through our donor recruitment opportunities, and lastly, raise funds to help support this vital work.

As a student, in addition to signing up to the register, you have the amazing opportunity of volunteering for us and to save a life! One of our most outstanding achievements is signing up over 100,000 people to the Anthony Nolan register and raising over 92,000 in a year. Additionally, 1 in 4 people who go on to donate stem cells is recruited via Marrow!

Being a volunteer for us is no hard work. You could do many things, including spreading the word and talking to people about why they should sign up to the register. Furthermore, you need to inform them what the donation involves if they ever found a match, checking medical backgrounds for donor eligibility, assisting them with cheek swabs, and filling out an application form.

If youre interested in this opportunity, there will be several volunteer training sessions held throughout the year. Unfortunately, due to the current situation, all these events will be held online. We can assure you, however, that were doing our best to make the most of it.

To sign up to the register visit the Anthony Nolan website!

Make sure to follow Manchester Marrows social media accounts to keep updated with all the news and events:

Facebook: @ManchesterMarrow

Facebook: Manchester Marrow Volunteers Group

Instagram: @manchestermarrow

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Looking to save lives? Here's how - The Mancunion

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DetailsCategory: DNA RNA and CellsPublished on Friday, 16 October 2020 14:20Hits: 301

First therapy recommended for full marketing authorization in the EU for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants

One-time treatment with Libmeldy has been shown to preserve cognitive and motor function in most patients

Libmeldy is backed by data across 35 patients with follow-up of up to 8 years post-treatment, demonstrating the potential durability of HSC gene therapy

BOSTON, MA, USA and LONDON, UK I October 16, 2020 I Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene), an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

The CHMPs positive opinion will now be reviewed by theEuropean Commission(EC), which has the authority to grant marketing authorization for Libmeldy in theEuropean Union(EU). A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

MLD is a very rare, severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them. A majority of these patients pass away in childhood, with palliative care often as their only option.

Todays positive CHMP opinion for marketing authorization of Libmeldy is a remarkable achievement that we share with the MLD community, as it brings us closer to delivering a one-time, potentially transformative therapy for eligible children suffering from this devastating disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Data from the Libmeldy clinical program have demonstrated the potential for long-term positive effects on cognitive development and maintenance of motor function, translating to individual preservation of motor milestones such as the ability to sit, stand and/or walk without support, as well as attainment of cognitive skills like social interactions and school attendance, at ages at which untreated patients show severe motor and cognitive impairments.

Libmeldy is designed as a one-time gene therapy, developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, in which the patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying genetic condition with a single treatment.

This is an important milestone toward making the availability of HSC gene therapy a reality for more patients, and it also is extremely rewarding for our multi-disciplinary team at SR-Tiget who has worked relentlessly along this 15-year journey to move the seminal proof of principle studies to the first in-human testing of this therapy, said SR-Tiget director Luigi Naldini, M.D, Ph.D. The robust and durable clinical benefits observed in early-onset MLD patients who received HSC gene therapy are compelling, especially when compared to the natural history of the disease. These results also further illustrate our view that the HSC gene therapy approach has the potential to deliver transformative effects in other storage diseases as well, especially when the cells are designed to overexpress the functional enzyme and provide an enhanced supply of it to the affected tissues.

As a parent, watching your child start down a seemingly normal developmental path only to suddenly and rapidly lose some or all of his or her abilities is heart-wrenching, and the agony is even more acute knowing no approved therapies currently exist for MLD, said Georgina Morton, Chair of ArchAngel MLD Trust. Todays decision to advance Libmeldy to the final EC approval stage represents a huge step forward for the parents of these young children and for all of us in the MLD community.

We are extremely appreciative of the EMAs expedited and thorough review of Libmeldys marketing authorization application, considering the severity of MLD coupled with the limited treatment options available today for young patients, said Anne Dupraz, chief regulatory officer, Orchard Therapeutics. The Agencys collaboration on this assessment is a testament to their broader public health commitment to ensure timely evaluation of new medicines for diseases where a pressing unmet need exists.

Data Supporting the Clinical Profile of Libmeldy

The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Early-onset MLD encompasses the disease variants traditionally referred to as late infantile (LI) and early juvenile (EJ).

Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation:

Clinical safety was evaluated in 35 patients with early-onset MLD:

Co-primary endpointsThe co-primary endpoints of the integrated efficacy analysis were Gross Motor Function Measure (GMFM) total score and ARSA activity, both evaluated at 2 years post-treatment. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Pre-symptomatic LI and EJ patients treated with Libmeldy experienced significantly less deterioration in motor function at 2 years and 3 years post-treatment, as measured by GMFM total score, compared to age and disease subtype-matched untreated patients (p0.008). The mean difference between treated pre-symptomatic LI patients and age-matched untreated LI patients was 71.0% at year 2 and 79.8% at year 3. Similarly, the mean difference between treated pre-symptomatic EJ patients and age-matched untreated EJ patients was 52.4% at year 2 and 74.9% at year 3. Although not statistically significant, a clear difference in GMFM total score was also noted between treated early-symptomatic EJ patients and age-matched untreated EJ patients (28.7% at year 2; p=0.350 and 43.9% at year 3; p=0.054).

A statistically significant increase in ARSA activity in peripheral blood mononuclear cells was observed at 2 years post-treatment compared to pre-treatment in both pre-symptomatic patients (20.0-fold increase; p<0.001) and early-symptomatic patients (4.2-fold increase; p=0.004).

At the time of the integrated data analysis, all treated LI patients were alive with a follow-up post-treatment up to 7.5 years and 10 out of 13 treated EJ patients were alive with a follow-up post-treatment of up to 6.5 years. No treatment-related mortality has been reported in patients treated with Libmeldy.

Key secondary endpointsFor EJ patients who were early-symptomatic when treated with Libmeldy, meaningful effects on motor development were demonstrated when these patients were treated before entering the rapidly progressive phase of the disease (IQ85 and Gross Motor Function Classification (GMFC)1). By 4 years post-disease onset, an estimated 62.5% of treated, early-symptomatic EJ MLD patients survived and maintained locomotion and ability to sit without support compared with 26.3% of untreated early-symptomatic EJ MLD patients, representing a delay in disease progression following treatment with Libmeldy.

A secondary efficacy endpoint that measured cognitive and language abilities as quantified by Intelligence Quotient/Development Quotient (IQ/DQ) found:

Clinical safetySafety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in 5 out of 35 patients. Antibody titers in all 5 patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. Treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

About MLD and Investigational Libmeldy

Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in thearylsulfatase-A(ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. Currently, there are no approved treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), formerly OTL-200, is being studied for the treatment of MLD in certain patients. Libmeldy was acquired from GSK inApril 2018and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their jointSan Raffaele-Telethon Institute for Gene TherapyinMilan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

SOURCE: Orchard Therapeutics

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Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) | DNA RNA and...

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) | DNA RNA and… | October 17th, 2020

In KEYNOTE-021 (Cohort G), first-line treatment with KEYTRUDA in combination with chemotherapy (n=60) demonstrated a significant improvement in objective response rates (58% vs. 33%), progression-free survival (HR=0.54 [95% CI, 0.35-0.83]) and a sustained, long-term survival benefit (HR=0.71 [95% CI, 0.45-1.12]) versus chemotherapy alone (n=63) in patients with advanced nonsquamous non-small cell lung cancer (NSCLC) regardless of PDL1 expression (Featured Poster #OFP01.02). Patients in Cohort G had no EGFR or ALK genomic tumor aberrations. These findings represent the longest follow-up data for an anti-PD-1/PDL1 therapy in combination with chemotherapy for the first-line treatment of NSCLC. Additionally, updated follow-up data from a Phase 1/2 study of quavonlimab in combination with KEYTRUDA showed encouraging anti-tumor activity and an acceptable safety profile as first-line treatment in patients with advanced NSCLC (Poster #TS01.02).

Over the last five years, KEYTRUDA has become foundational in the treatment of metastatic lung cancer. The long-term data from KEYNOTE-021 (Cohort G) reinforce the use of KEYTRUDA in combination with chemotherapy in certain advanced lung cancer patients, while data from our oncology pipeline reflect our commitment to exploring a number of new combinations with KEYTRUDA that we believe could have a meaningful impact for more lung cancer patients, said Dr. Vicki Goodman, vice president, oncology clinical research, Merck Research Laboratories. Updated data from our anti-CTLA-4 antibody quavonlimab in combination with KEYTRUDA support the continued development of this new combination and a Phase 3 study of quavonlimab coformulated with KEYTRUDA in advanced non-small cell lung cancer is planned.

Results from both studies were presented at the IASLC 2020 North America Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer on Friday, Oct. 16. Follow Merck on Twitter via @Merck and keep up to date with NACLC news and updates by using the hashtag #NACLC20.

KEYTRUDA in Combination With Chemotherapy: Long-Term Data in Advanced NSCLC From KEYNOTE-021 (Cohort G) (Featured Poster #OFP01.02) New data from Cohort G of KEYNOTE-021 (NCT02039674) demonstrated a significant improvement in objective response rates (ORR), progression-free survival (PFS) and a sustained, long-term survival benefit with KEYTRUDA in combination with pemetrexed (ALIMTA) and platinum chemotherapy versus pemetrexed and platinum chemotherapy alone after four years of median study follow-up (49.4 months; range, 43.5 to 55.4). Cohort G of the Phase 1/2, multi-cohort, multi-center, open-label trial evaluated KEYTRUDA in combination with chemotherapy (n=60) versus chemotherapy alone (n=63) as first-line treatment in patients with advanced nonsquamous NSCLC. Patients in Cohort G had no EGFR or ALK genomic tumor aberrations.

Findings from KEYNOTE-021 (Cohort G) showed that 50% of patients treated with KEYTRUDA in combination with chemotherapy were alive at three years versus 37% of patients who received chemotherapy alone. KEYTRUDA in combination with chemotherapy also reduced the risk of death by 29% (HR=0.71 [95% CI, 0.45-1.12]) versus chemotherapy alone, with a median overall survival (OS) of 34.5 versus 21.1 months. The OS benefit was observed despite a 70% (n=43/61) effective crossover rate from chemotherapy to antiPD1/PDL1 therapy, including 28 patients who were treated with KEYTRUDA as part of the on-study crossover.

The ORR was 58% for KEYTRUDA in combination with chemotherapy versus 33% for chemotherapy alone. KEYTRUDA also reduced the risk of disease progression or death by 46% (HR=0.54 [95% CI, 0.35-0.83]) versus chemotherapy, with a median PFS of 24.5 months (range, 9.7 to 36.3) versus 9.9 months (range, 6.2 to 15.2). The estimated three-year PFS rate was 37% for patients who received KEYTRUDA in combination with chemotherapy versus 16% for those who received chemotherapy alone. The median duration of response (DOR) was more than one year longer with KEYTRUDA in combination with chemotherapy (36.3 months; range, 1.4+ to 49.3+) versus chemotherapy alone (22.8 months; range, 2.8+ to 47.2+). Additionally, 51% of patients treated with KEYTRUDA in combination with chemotherapy had responses lasting three years versus 47% with chemotherapy alone.

Notably, 92% of patients who completed two years of treatment with KEYTRUDA were alive at three years (n=11/12). All 12 patients experienced an objective response and the estimated three-year DOR rate was 100% (median DOR not reached [NR]; range, 11.7+ to 49.3+ months).

No new safety signals for KEYTRUDA in combination with chemotherapy were identified with long-term follow-up. Among all those treated, 39% of those who received KEYTRUDA in combination with chemotherapy and 31% of those who received chemotherapy alone experienced Grade 3-5 treatment-related adverse events (TRAEs). Grade 3-5 TRAEs that led to discontinuation occurred in 17% of patients who received KEYTRUDA in combination with chemotherapy and 16% of those who received chemotherapy alone. Grade 3-5 TRAEs that led to death occurred in 2% (n=1) of patients who received KEYTRUDA in combination with chemotherapy and 3% (n=2) of those who received chemotherapy alone.

The KEYNOTE-021 (Cohort G) trial was conducted in collaboration with Eli Lilly and Company, the makers of pemetrexed (ALIMTA).

Quavonlimab (anti-CLTA-4) in Combination With KEYTRUDA: Phase 1/2 Results in Advanced NSCLC (Poster #TS01.02) In this first-in-human, open-label, multi-arm Phase 1/2 study (NCT03179436), quavonlimab, Mercks novel anti-CTLA-4 therapy, was evaluated in combination with KEYTRUDA as a first-line treatment in patients with advanced NSCLC. In the dose-confirmation phase, patients received quavonlimab (25 mg or 75 mg) every three weeks (Q3W) or every six weeks (Q6W) in combination with KEYTRUDA (200 mg Q3W for up to 35 cycles). The primary objective of the study was safety and tolerability; secondary and exploratory objectives included ORR per RECIST v1.1 by blinded independent central review (BICR), PFS, OS and DOR. Response based on PD-L1 status was retrospectively evaluated using tumor proportion score (TPS) as a continuous variable.

Findings showed that quavonlimab in combination with KEYTRUDA had an acceptable safety profile with no unexpected toxicities and suggested encouraging anti-tumor activity. Any-grade adverse events occurred in 98% of patients; TRAEs occurred 85% of patients. Grade 3 TRAEs occurred in 36% of patients across all treatment arms and the most common TRAEs (>10% in any arm) were increased alanine aminotransferase (8%), pneumonitis (8%) and increased aspartate aminotransferase (6%).

With 16.9 months of median follow-up (range, 7.0 to 21.3), results from the study showed the effect of quavonlimab in combination with KEYTRUDA across secondary and exploratory endpoints, including ORR, PFS, OS and DOR. Responses to quavonlimab in combination with KEYTRUDA were observed regardless of PD-L1 expression with higher TPS scores significantly associated with better response (one-sided p=0.015). These safety and efficacy data support the 25 mg Q6W dose as the recommended Phase 2 dose of quavonlimab when used in combination with KEYTRUDA.

Quavonlimab25 mg Q6W + KEYTRUDAn=40

Quavonlimab25 mg Q3W + KEYTRUDAn=40

Quavonlimab75 mg Q6W + KEYTRUDAn=40

Quavonlimab75 mg Q3W + KEYTRUDAn=14

TotalN=134

ORR, %(95%, CI)

37.5(22.7-54.2)

40(24.9-56.7)

27.5(14.6-43.9)

35.7(12.8-64.9)

35.1(27.0-43.8)

PFS, median(95%, CI), mo

7.8(4.2-14.8)

6.0(2.0-8.3)

6.0(3.5-8.1)

3.4(1.8-NE)

6.1(4.2-7.3)

OS, median(95%, CI), mo

18.1(14.2-NE)

18.1(9.1-21.8)

17.1(9.0-NE)

13.7(3.5-NE)

16.5(14.2-21.8)

DOR, median(95%, CI), mo

NR(4.0 to 21.6+)

7.9(2.8 to 21.4+)

15.9(3.4 to 21.4+)

NR(8.8+ to 16.3+)

13.6(2.8 to 21.6+)

About Lung Cancer Lung cancer, which forms in the tissues of the lungs, usually within cells lining the air passages, is the leading cause of cancer death worldwide. Each year, more people die of lung cancer than die of colon and breast cancers combined. The two main types of lung cancer are non-small cell and small cell. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for about 85% of all cases. Small cell lung cancer (SCLC) accounts for about 10% to 15% of all lung cancers. Before 2014, the five-year survival rate for patients diagnosed in the U.S. with NSCLC and SCLC was estimated to be 5% and 6%, respectively.

About KEYTRUDA (pembrolizumab) Injection, 100 mg KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

About Quavonlimab (MK-1308) Quavonlimab is a novel humanized IgG1 monoclonal antibody that binds to CTLA-4 and blocks interaction with its ligands, CD80 and CD86. Quavonlimab is currently being evaluated in combination with KEYTRUDA across multiple solid tumors as part of ongoing Phase 1 and 2 trials. A Phase 3 trial of quavonlimab coformulated with KEYTRUDA in advanced non-small cell lung cancer is planned.

Selected KEYTRUDA (pembrolizumab) Indications Melanoma KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Link:
Merck Presents Three-Year Survival Data for KEYTRUDA (pembrolizumab) in Combination With Chemotherapy and Updated Phase 1/2 Data for Investigational...

Cardiac Stem Cells Comments Off on Merck Presents Three-Year Survival Data for KEYTRUDA (pembrolizumab) in Combination With Chemotherapy and Updated Phase 1/2 Data for Investigational… | October 17th, 2020

If you've suffered a spinal cord injury, it is only natural for you to search out the latest breakthroughs in medicine and technology to find a treatment that can get you back to the way things used to be. And one promising branch of current medical research is in the direction of stem cell therapy. But it's important to understand the scope of this relatively new science and to have realistic expectations about the outcomes.

We often get this question, in various forms, from people who suffer from spinal cord injuries:

Is stem cell therapy a cure?

Well, as of today, the sad answer is no. There is no evidence, so far, that stem cell therapy can cure a spinal cord injury. But I'd suggest that the real question they should be asking is this:

Does stem cell therapy have the ability to help after a spinal cord injury?

The answer to this question isn't that firm, but it is a lot more hopeful than the other question. The answer is maybe, sometimes, and we don't know. The reason for this ambiguity is that stem cell therapy for spinal cord injuries is in its infancy as a treatment. In fact, as of January of 2020, the FDA hasn't approved any stem cell therapies for this purpose. So, these treatments are not available in the mainstream medical market. The U.S. Food and Drug Administration has even expressed concerns that patients seeking cures and remedies are vulnerable to stem cell treatments that are currently illegal and potentially harmful.

So, the good news is that there have been some newsworthy and amazing stories of recoveries after stem cell treatments. The unfortunate news is that you won't be able to get stem cell treatment for your SCI through traditional hospital care.

While commercially available stem cell therapies are not available, there are plenty of existing clinical trials out there for which you might qualify. And, a lot of progress is being made in this area. These ongoing trials are being held at various locations around the United States.

Some of these trials focus on individuals who are in the acute stage of their SCI. That generally means they are patients still within 72 hours of the initial injury. Given the short time range on these tests, it is not generally possible to volunteer for them. Rather, the doctors administering the clinical trials will generally seek out patients in the hospital as participants.

There are other trials, however, that are researching the effects of stem cell therapy on patients months or even years after an injury. These are the kinds of trials that patients can apply for and have any real hope of participating.

So, the main point is that, when you're researching trials, it's very important to consider the qualifications. If you don't meet the criterea, you're wasting your time.

With that in mind, if you've decided to pursue clinical trials as a source of treatment, there are a couple of really great resources that can help you to find the right study for you. There are two websites that you can use. They are scitrials.org and clinicaltrials.gov.

This website can really help you to narrow down the search when you're looking for different experimental therapies that could be helpful for your treatment. On the site, you can search for trials based on geographic location, the level of injury, the age of the injury, and you can even use a keyword search.

This is a much larger website, and a much larger resource. And, it's run by the government. It is definitely worth reviewing, especially if you couldn't find what you were looking for at scitrials.org. On the downside, the sheer breadth of information can be overwhelming. Clinicaltrials.gov lists virtually every clinical trial that's going on in the United States. So, that's an enormous amount of information to sift through. But, you can never have too much information, and you're often better off starting with a large amount of information and narrowing it down.

When applying for clinical trials, you will probably be submitting more than one application. Be sure to keep a spreadsheet or some kind of list to keep track of the trials you've already researched, the ones you've applied for, and the responses that you get. Understanding the responses, especially, helps you to improve the quality of future applications. And, it can help you to avoid wasting precious time effort applying for trials that you're not even qualified for.

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Stem Cell Therapies for Spinal Cord Injuries

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Sixteen years ago, voters were promised that $3 billion of bonds for embryonic stem-cell research would deliver cures for diseases such as diabetes, Alzheimers, Parkinsons and heart disease.

Instead, weve gotten cures or potential treatments for a very different and unexpected set of afflictions, such as a deadly immune disorder, spinal cord injury, a type of cancer and a form of blindness.

The moral of the story as Californians decide whether to continue support by approving Proposition 14s nearly doubled research budget of $5.5 billion is this: Science marches to its own beat and on its own clock, awe-inspiring but oblivious to political pledges.

We got things, but not necessarily what we expected, said Hank Greely, director of Stanfords Center for Law and the Biosciences. Its saving lives, but not in the way most people thought.

Without Proposition 71, the ambitious 2004 ballot measure that first paid for the stem-cell research, 8-year-old Evangelina Vacarro of Corona might be in a casket, rather than skateboarding, horseback riding and playing in the dirt with her pet terrier Daisy.

The engaging hazel-eyed child was born with a rare disease that left her unable to fight off infections. In a clinical trial funded by the state initiative, scientists corrected the deadly genetic flaw that disabled her immune system and restored her to health.

Jake Javier, a biomedical engineering student at Cal Poly, might be unable to live independently. Paralyzed in a diving accident, the 22-year-old gained some function in his arms and hands after the introduction of specialized neural cells in a clinical trial funded by Proposition 71.

It completely changed the trajectory of my life, said Javier, of San Ramon.

Sandra Dillon, a San Diego graphic designer diagnosed at age 28 with a rare form of blood cancer called myelofibrosis, is now in remission after treatment with an FDA-approved drug that was identified through Prop. 71 funding. No longer hospitalized, shes backpacking and surfing.

Before injection with stem cells to combat progressive blindness caused by retinitis pigmentosa, Rosie Barrero of Los Angeles could only see shadows. Now she can discern the time on her cell phone, the colors of kitchen cups and the faces of her family.

In addition to these and more than 60 other clinical trials, the California Institute for Regenerative Medicine (CIRM) created under Proposition 71 has led to about 3,000 peer-reviewed research papers and 974 patents or patent applications.

It has helped bankroll 12 elegant research facilities, donating $43 million to Stanford for the Lorry I. Lokey Stem Cell Research Building, $20 million to the Buck Institutes Regenerative Medicine Research Center in Novato, $20 million to UC Berkeleys Li Ka Shing Center and $35 million to UCSFs Regeneration Medicine Building. It funded five stem cell-focused clinics at hospitals to accelerate the delivery of therapies.

It has generated $293.6 million in direct income and taxes on corporate profits and sales of equipment and supplies, according to an estimate by research professors Dan Wei and Adam Rose of the University of Southern California.

And it has vaulted California to a leadership role in the nations stem-cell science.

CIRM has supported some really superb research and researchers and built a powerful infrastructure, said Robert Cook-Deegan of the School for the Future of Innovation in Society at Arizona State University. In a field where there arent as many other sources of funding, thats almost certainly, in the long run, a good thing.

This is stunning progress for an effort that faced bleak prospects after then-President George W. Bushs federal funding restrictions on embryo research.

Still, it falls far short of Proposition 71s breathless rhetoric from the 2004 campaign.

Stem Cell Research: Breakthrough cures for diseases that affect millions of people, asserted the campaign literature. In a 30-second commercial, actor Michael J. Fox, diagnosed with Parkinsons disease, urged voters to please support the effort to find cures, to save the life of someone you love. Other A-list celebrities, as well as more than 20 Nobel Prize-winning scientists, also promoted it.

Prop 71 will help reduce skyrocketing health care costs, the campaign promised.

The measure was tied up by litigation and the effort got off to a late start. Now, one-third of the way through the bonds 35-year payback period, its nowhere near yielding the $2.2 to $4.4 billion in projected state revenues or $1.1 billion in royalty revenues. To date, only $517,989 in royalties has been paid to the state general fund.

And with only two products FDA-approved and no therapies yet in widespread use, theres so far no evidence that Proposition 71 has delivered the anticipated $3.4 to $6.9 billion state health care savings and $9.2 to $18.4 billion in savings for other payers.

Initial CIRM funding created fewer California jobs than expected: 27,208 jobs per year, according to the USC report, rather than the estimated 47,000 jobs per year.

There have been clinical challenges. A cure for diabeteshas been tougher than expected: A promising approach has lagged because the bodys immune system rejects the pouch that holds implanted, insulin-producing cells. A leukemia cure, seemingly around the corner, has been stymied because blood-forming stem cells are stubbornly reluctant to multiply.

If it all worked, it wouldnt be research, said Stanfords Greely. Politics has a corrupting influence on everything it pushes toward exaggeration.

There have also been business failures. More than $5 million was invested in a promising brain cancer treatment called ICT-107, but efforts were abandoned when company ImmunoCellular Therapeutics ran out of money. Another $3 million was for naught when company Neostem couldnt find funding and swapped CEOs, dropping its melanoma treatment CLBS20.

Even some home runs, such as Gileads recent $4.9 billion deal for the CIRM-supported immuno-oncology biotech Forty Seven, offer taxpayers relatively little payoff. While CIRM expects royalties from Gileads future cancer cure, it didnt benefit from an explosive jump in share prices because the state Constitution bars state agencies from holding stock in private companies.

I think the big downside of CIRM has been the overpromising of how fast things would happen and the form that the return would take, said Cook-Deegan. The commercial potential may be real in the long runbut it was oversold with shorter time horizons than are actually practical.

Proposition 14 will cost far more: $7.8 billion $5.5 billion in principal and $2.3 billion in interest by the time the bonds are repaid. The total cost of Proposition 71 is $3.54 billion, or more than $4 billion when adjusted for inflation. The increase is necessary due to the soaring cost of clinical trials, said Robert Klein, the author of both measures

As voters consider whether to support the new proposition, its unfair to measure it against the prior propositions current trajectory, because medicine reaps its greatest rewards many years after the initial investment, said Klein. It takes 12 to 15 years, on average, to go from discovery to therapy.

It would be similar to judging the Apollo 11 mission when the capsule was a little past a third of the way to the moon and find it coming up short because not one of the three astronauts had set foot on the lunar surface, said Klein.

But critics say Proposition 14 commits California to spending money it does not have. It adds future debt, while education, health care and housing are underfunded.

While I think CIRM has done good work, and I support stem-cell research, the state is facing a huge budget deficit, said Jeff Sheehy, a CIRM board member. And the new measure fails to ensure that the state gets a return on its investment. Instead, it is a giveaway to pharma and biotech.

Theres no longer a compelling rationale for California to support the research because the federal ban has been lifted, with NIH spending about $300 million a year on embryonic stem-cell research, he said.

If the new proposition is rejected by voters, CIRM will begin closing its doors this winter and ongoing research may crash, said Dr. Larry Goldstein, director of the UC San Diego Stem Cell Program.

If these trials get killed because of lack of funding, there is no guarantee that we will get them back up and running again, even if they look really promising, Goldstein said. It will be hard to find financing for them.

Proposition 71 launched the beginning of embryonic stem-cell research and Proposition 14, despite the steep price tag, should continue that momentum, say proponents.

There is no dollar amount you can put on having a healthy child, said Alysia Vaccaro, Evangelinas mother. There is no price for that.

Prop 71, by the numbers:

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Should California invest $5.5 billion more into promising ...

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Immuno-oncology and CAR T cells energized the field of regenerative medicine, but for cell and gene to deliver on their promises, new, disruptive technologies and new modes of operation are needed. Specifically, that entails improving manufacturing to control variables and thus ensure product consistency, and maturing the regulatory environment to improve predictability.

Manufacturing cells is not like manufacturing small molecules, Brian Culley, CEO of Lineage Cell Therapeutics, told BioSpace. For cell therapy products to mature into real products that deliver on the promises of 10 years ago, they must be scalable which drives affordability and they must solve their purity issues.

On the clinical side, cell and gene therapies must find places where small molecules, antibodies or other traditional approaches may not be the best option.

For example, The era of transplant medicine is unfolding before us, Culley said. Because of the transplant component, cell therapy may enable changes the body never could do alone.

Lineage is addressing dry AMD and spinal cord injuries with two of its therapeutics.

Our approach is fundamentally different from traditional approaches. We replace the entire cell rather than modulate a pathway. There is a rational hypothesis where cell therapy can win, but first we need to fix the operational hurdles, Culley said.

To address the manufacturing challenges, Culley said, We work only with allogeneic approaches. For us, not being patient-specific is a huge advantage.

Not long ago, the industry was focused on 3D manufacturing in bioreactors.

Were beyond that, Culley said. For our dry AMD product, we can manufacture 5 billion retinal cells in a three liter bioreactor. The advantage is that the cells exist in a very homogenous space and are 99% pure.

As a result, they are more affordable and can be harvested with little manipulation.

Manual manipulation affects gene expression, he pointed out, so minimizing that, as well as the vast quantities of plastics typically required, results in a more controlled process and a more consistent product.

Additionally, Lineage introduced a thaw and inject formulation, so the cell therapy can be thawed in a water bath, loaded into a chamber and injected, all within a few minutes. Traditional dose administration requires washing, plating and reconstituting the cells the before they are administered to a patient.

Getting rid of the prior day dose prep is one example of the maturation of the field, which we are deploying today to help usher in a new branch of medicine, Culley said.

At Lineage, were tackling problems that largely were intractable. For dry AMD, theres nothing approved by the FDA. No one know why the retinal cells die off, so we manufacture brand new retinal cells (OpRegen) and implant them, Culley said. Were seeing very encouraging clinical signs, including the first-ever case of retinal restoration.

Retinal cells compose a thin layer in the back of the eye, Culley explained.

They start to die off in one spot, and that area grows outward. When we inject our manufactured cells where the old ones died, weve seen the damaged area shrink and the architecture in previously damage areas completely restored, Culley said. Weve treated 20 patients for dry AMD in, ostensibly, safety trials, but you cant help but notice efficacy when a patient reads five more lines on an eye chart. Its hard to imagine our intervention wasnt responsible for that, especially when humans cant regenerate retinal tissue.

The spinal injury program (OPC1) may represent an even greater breakthrough. As with dry AMD, there is no FDA-approved therapy.

We manufacture oligodendrocytes and transport them into the spinal cord, to help produce the myelin coating for axons, he told BioSpace. Because of the oligodendrocytes, the axons grow, become myelinated, and begin to function. Small molecule and antibody therapies havent been able to do that.

So far, 25 people have been treated in a Phase I/II trial. Culley reported cases in which a quadriplegic man, after OPC1 therapy, is now typing 30 to 40 words per minute, and another who now can throw a baseball. Its not unusual for patients who initially were completely paralyzed to now schedule their treatments around college classes, Culley said.

Humans can have varying degrees of recovery from spinal cord injury, but these are higher than we would expect, Culley said.

Other cell and gene companies are advancing solutions, too.

Many companies with induced pluripotent stem cells (iPSCs) are trying to figure out how to get scalability, purity, and reproducibility to work for them. Its not a quick fix, he said.

One of the challenges is balancing the clinical and manufacturing aspects of development.

If you have a technology thats not yet commercially viable, but you have clinical evidence, its tempting to focus on the clinical side, Culley said.

Too many companies do that, and then find their candidate must be reworked for scale up. Therefore, consider scale up and manufacturing early.

Theres a need for balance at a more granular level, too. For example, he asked, How many release criteria do you need? Just because you know a cell expresses a certain surface marker, does that add to your process? Ive seen companies ruined by trying to be perfect, and others by rushing headlong, seeing evidence where evidence doesnt exist.

As Lineage matures its processes to support larger clinical trials, the greatest challenges have been time It takes 30 to 40 days to grow cells, Culley said and regulatory uncertainty. Often, there is no regulatory precedence so there are holes to be addressed. For example, cell and gene therapies sometimes have a delivery component such as a scaffold or delivery encapsulation technology that also must be considered. Real-time regulatory feedback isnt available, so you proceed, presuming that what youre doing will be acceptable to regulators.

The FDA recognizes that new, disruptive technologies and approaches are being used, and must be used, for cell and gene therapy to reach patients.

The FDA is responsive and is trying to push guidance out, Culley said, but it takes time.

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Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation - BioSpace

Spinal Cord Stem Cells Comments Off on Disruptive Technologies and Mature Regulatory Environment Vital for Cell Therapy Maturation – BioSpace | October 17th, 2020

President Trump has benefitted from decades of medical research using human fetal tissue, and so have you.

When he got sick with the coronavirus in September, Trump's Regeneron antibody treatment had been developed with the use of HEK 293T cells, which have been a workhorse material on biomedical lab benches around the world, since they were first cultured from an embryonic kidney cell in the Netherlands in the early 1970s.

Even so, Trump and his administration have cracked down on new fetal tissue research being done today in the US dampening hopes the same kinds of cells that helped create his treatment may continue being used in new research, to pave the way toward future treatments and cures for millions of people.

The ban could have a crippling effect on the hunt for treatments for neurodegenerative diseases, and new drug therapies for cancers and HIV. It's also affecting the way scientists study viruses that cause disease in humans, a critical impairment during the coronavirus pandemic.

The truth is, there is no great alternative (yet) for human fetal tissue in medical research. (Humans, and their development, are kind of complicated.) So, for now, we're missing out on an unknown number of medical advances, as the raw materials for this research get discarded after miscarriages and abortions.

"Scientists, such as myself, think that it's better for all of that tissue to go to research, than to just destroy it," Professor Lawrence Goldstein, a neuroscientist at UC San Diego, who has used fetal tissue to study Alzheimer's in the past, said.

Here's why.

Dr. Scott Kitchen, associate professor of medicine and Director, UCLA CFAR/JCCC Humanized Mouse Core Laboratory, in the vivarium mice room at the UCLA campus in Los Angeles, California on November 15, 2019. Philip Cheung for The Washington Post via Getty Images

"Most drugs and most vaccines have, at some point in their movement towards the clinic, passed through a stage in which they've been either developed or tested using cell lines that came from human fetal tissue," said Dr. Mike McCune, an HIV researcher who, in the 1980s, developed the first mice engineered to study human diseases using fetal tissue.

In McCune's lab, fetal tissue has been used to test out drug treatments that have turned HIV from a death sentence into a livable, chronic disease. Today, many branches of medical research, treating everything from cancer, to spinal cord injuries, Alzheimer's, and organ transplants have all, in some way, benefitted from fetal tissue research.

One of the most basic ways that fetal tissue has been used is in the creation of prolific cell lines (like the ones Regeneron's used in its labs) which can be dispatched indefinitely to test out how well treatments or vaccines might work, before they go inside people.

Such cell lines are also sometimes used not just to test, but to create treatments. Take the advent of the polio vaccine, which has prevented millions of cases of paralysis, and saved hundreds of thousands of lives. That was once grown inside fetal cells, as were many other vaccines.

Fetal tissue is also used as a gold standard comparison tool. For example, in organ development, it's used to make sure that stem cells being developed into artificial organs are mimicking real, human, cells in the proper way.

Studying human fetal tissue also helps researchers better understand the reasons why birth defects arise, and glean insights into how these congenital issues may be better prevented in the future.

During the coronavirus pandemic, fetal tissue could be used to develop precise human immune system models which could then be used to quickly try out drugs, determining within weeks which might work, and which are duds.

Today, there is no reliably good alternative material to use for fetal tissue, the very makings of our humanness. Fetal cells are less specialized than fully-formed human cells, and as such, they are much more flexible tools than our own cells for using in the lab, and studying all kinds of diseases that only affect us.

Donating fetal tissue is a lot like organ donation. There are strict rules in place to ensure no exchange of money or favor occurs. Instead of being incinerated, then, that fetal tissue may be used in a lab.

"You have a family, a mother, or parents, who make the decision that they want something good to come out of this tragedy of losing, or terminating, a pregnancy," virologist Alexander Ploss, who does work on fetal stem cells at Princeton University, told Insider of fetal tissue donation. "We're basically now potentially restricting this option, and taking this option away."

Dr. Lindsey Baden, right, bumps elbows with COVID-19 vaccine trial participant Anthony Shivers at Brigham and Women's Hospital in Chestnut Hill, Massachusetts on October 8, 2020. Craig Walker/The Boston Globe via Getty Images

Fetal tissue was already given the Congressional thumbs up by a bipartisan group of lawmakers in 1993.

The vote in the Senate that year was near-unanimous: 93 to 4, with anti-abortion senators Mitch McConnell and Chuck Grassley both voting "yes". They acknowledged there are clear benefits to humanity in using this tissue, just as the National Institutes of Health did, as recently as 2018.

The National Catholic Bioethics Center has for years, likewise, agreed that there are benefits to using fetal cell lines and tissues.

The NCBC said as recently as May 2020 on its website that "one is morally free to use the vaccine, despite its historical association with abortion, if there is a proportionately serious reason for doing so."

"This is especially important for parents," the NCBC added, "who have a moral obligation to protect the life and health of their children, and those around them."

But on June 5, 2019, the Department of Health and Human Services stopped funding all new human fetal tissue research, effectively shutting down the last private labs (in California and Montana) that were using federal dollars for fetal tissue work.

"Many of the researchers who may be most affected by this policy change study pathways and processes associated with disease in infants and children," professor Carolyn Coyne, who studies viruses that affect fetal and neonatal health, wrote in the Washington Post at the time. "As such, the only certain consequence of the new policy is that it will impede medical discoveries that could advance new treatments to save the lives of infants, the very lives those in favor of this policy claim they are trying to protect."

Juan Duran-Gutierrez kisses his newborn baby girl Andrea for the first time in his home after bringing her home from the hospital, August 5, 2020. Duran-Gutierrez's wife and Andrea's mother, Aurora, died from COVID-19 in July. Elizabeth Flores/Star Tribune via Getty Images

The NIH can technically still fund work on fetal tissue outside its own walls, but the agency has been hamstrung by a new fetal tissue advisory board, largely comprised of members with strong antiabortion group ties. At a recent meeting, the group approved just one of 14 fetal tissue grant proposals up for review, The Washington Post reported. It was a grant to study whether an alternative to fetal tissue works as well.

"The administration has developed a policy that the evangelical and hardcore pro-life community wants, which is a complete ban on the use of any federal funds for new fetal tissue," Goldstein, who sat on the recent NIH committee, told Insider. "You know, they're okay with the old stuff."

Antiabortion groups have largely shrugged off the fact that Trump's coronavirus treatment benefitted from medical research on fetal tissue, decades ago.

Goldstein sees this as pure hypocrisy.

"The claim they're making is that, 'well, it was done a long time ago, so it's okay now,'" he said. "Well, you know, that's not really morally very consistent. You're going to block us from developing new therapies with fetal tissue, but you're going to be okay using the ones that are already here?"

Goldstein says there are three main reasons to continue using fetal stem cells: One, there's no evidence that this research incentivizes anyone to have an abortion. Two: "it's really valuable research" which has saved and improved countless lives. And three: "The alternative is throwing it in the trash," he said. "How is that a dignified use of the material?"

"I don't know if that's going to persuade anybody, but those are the factors I'd cite," he added.

Dr. Mustafa Gerek is vaccinated in volunteer in trials of a COVID-19 vaccine from China at Ankara City Hospital in Ankara, Turkey on October 13, 2020. Aytac Unal/Anadolu Agency via Getty Images

The coronavirus isn't the only area where scientists now have a blind spot.

"There's a whole bunch of genetic diseases that we don't understand very well, neuro-developmental disorders that we don't understand very well, for which actually having access to, let's say, human neuronal tissue, or whatever it may be, is absolutely critical," Ploss said.

"I'm not particularly optimistic that it's possible right now to obtain any serious funding, federal funding, for this kind of research," he said. "It's pretty much impossible right now to get any kind of funding for research that involves human fetal tissue."

But, he says, the stakes are so high that he'll still try, in the coming months. He, and all the other scientists Insider spoke to for this story are already worried about the US losing some of its competitive edge in biotech.

"The reason we are a world leader is because we have been innovative, we have rewarded innovation, and for the most part, the government has stayed out of the way, except funding high-quality research, that's been competitively reviewed," Goldstein said.

"We are always at risk of losing our advantage to an aggressive competitor, and I don't want to see that happen. I'm a loyal American, and I want to see us be the best."

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What is human fetal tissue research and why is it done - Business Insider - Business Insider

Spinal Cord Stem Cells Comments Off on What is human fetal tissue research and why is it done – Business Insider – Business Insider | October 17th, 2020

Skin is a complex, living organ made up of several structural components that (quite literally) touches a lot of different parts of us. When you look at the makeup, there's collagen, elastin, hyaluronic acid, ceramides, lipids, and so forth; from there, it's also affected by the muscle composition and bone underneath. This is all to say, when wrinkles formit's hardly ever due to a singular reason.

So to start, there is the fact that many components of our skin structure decline with age. "As we age, we lose collagen, fat, and start to resorb bone. These changeslead to volume loss, thin, saggy skin. As a result, our nasolabial folds become more pronounced," says Lolis.

It's also important to note that these lines are exacerbated with movement, the same way crow's feet can come from squinting and the "11's" can come from scrunching yourbrow. "Laugh lines are formed by constant use of the orbicularis orbis muscle which allows us the ability to speak," says Masur. "Over time as we age the skin protecting this muscle becomes stretched creating laxity increasing the appearance of these folds. The region around the mouth known as the peri-oral area is one of the thinnest-skinned on the face, making us more susceptible to fine lines or wrinkles forming the 'laugh lines.'"

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Laugh Lines Are Totally Normal How To Ease Them Sans Injections - mindbodygreen.com

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The healthcare industry has been focusing on excessive research and development in the last couple of decades to ensure that the need to address issues related to the availability of drugs and treatments for certain chronic diseases is effectively met. Healthcare researchers and scientists at the Li Ka Shing Faculty of Medicine of the Hong Kong University have successfully demonstrated the utilization of human induced pluripotent stem cells or hiPSCs from the skin cells of the patient for testing therapeutic drugs.

The success of this research suggests that scientists have crossed one more hurdle towards using stem cells in precision medicine for the treatment of patients suffering from sporadic hereditary diseases. iPSCs are the new generation approach towards the prevention and treatment of diseases that takes into account patients on an individual basis considering their genetic makeup, lifestyle, and environment. Along with the capacity to transform into different body cell types and same genetic composition of the donors, hiPSCs have surfaced as a promising cell source to screen and test drugs.

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In the present research, hiPSC was synthesized from patients suffering from a rare form of hereditary cardiomyopathy owing to the mutations in Lamin A/C related cardiomyopathy in their distinct families. The affected individuals suffer from sudden death, stroke, and heart failure at a very young age. As on date, there is no exact treatment available for this condition.

This team in Hong Kong tested a drug named PTC124 to suppress specific genetic mutations in other genetic diseases into the iPSC transformed heart muscle cells. While this technology is being considered as a breakthrough in clinical stem cell research, the team at Hong Kong University is collaborating with drug companies regarding its clinical application.

The unique properties of iPS cells provides extensive potential to several biopharmaceutical applications. iPSCs are also used in toxicology testing, high throughput, disease modeling, and target identification. This type of stem cell has the potential to transform drug discovery by offering physiologically relevant cells for tool discovery, compound identification, and target validation.

A new report by Persistence Market Research (PMR) states that the globalinduced pluripotent stem or iPS cell marketis expected to witness a strong CAGR of 7.0% from 2018 to 2026. In 2017, the market was worth US$ 1,254.0 Mn and is expected to reach US$ 2,299.5 Mn by the end of the forecast period in 2026.

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Customization to be the Key Focus of Market Players

Due to the evolving needs of the research community, the demand for specialized cell lines have increased to a certain point where most vendors offering these products cannot depend solely on sales from catalog products. The quality of the products and lead time can determine the choices while requesting custom solutions at the same time. Companies usually focus on establishing a strong distribution network for enabling products to reach customers from the manufacturing units in a short time period.

Entry of Multiple Small Players to be Witnessed in the Coming Years

Several leading players have their presence in the global market; however, many specialized products and services are provided by small and regional vendors. By targeting their marketing strategies towards research institutes and small biotechnology companies, these new players have swiftly established their presence in the market.

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To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

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Induced Pluripotent Stem Cells Market To Grow At 7% YOY In Forecast Years 2026 - The Think Curiouser

Skin Stem Cells Comments Off on Induced Pluripotent Stem Cells Market To Grow At 7% YOY In Forecast Years 2026 – The Think Curiouser | October 17th, 2020

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CC cream stands for color corrector or complexion corrector cream. It's lighter weight than foundation but provides more coverage than BB cream. All you need to do is substitute it for your foundation in your makeup routine, or use it as a primer. Start with a cleansed, toned, and moisturized face. Then using a beauty blender or your fingertips, apply the liquid all over your face, ensuring you're fully smoothed out around the edges, building up the coverage as you desire.

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15 Best CC Creams for a Flawless Complexion - The Trend Spotter

Skin Stem Cells Comments Off on 15 Best CC Creams for a Flawless Complexion – The Trend Spotter | October 17th, 2020

In summary

Proposition 14 asks voters to spend nearly $8 billion to continue the stem cell research program at a time when the coronavirus pandemic has decimated the state budget.

For the second time in 16 years, California voters will decide the fate of the states multi-billion dollar stem cell research program that established the state as a worldwide leader.

How the times have changed.

In November, as the pandemic drags on, Proposition 14 asks voters to spend nearly $8 billion to continue the program during a period when the research environment has significantly evolved and coronavirus has battered the states budget.

The bond measure would approve $5.5 billion in bonds to keep the states stem cell research agency running and grants flowing to California universities and companies.

At least $1.5 billion would be earmarked for brain and central nervous system diseases like Alzheimers and Parkinsons. The overall cost of the bonds and their interest totals about $7.8 billion, according to the state legislative analyst. The state would pay about $260 million annually for 30 years, or about 1 percent of Californias annual budget.

Proposition 14 is essentially a repeat with a bigger price tag and a few tweaks of Proposition 71, which California voters approved in 2004 after then-President George W. Bush prohibited, on religious grounds, all federal funding of any stem cell research using human embryos.

The bond measure would approve $5.5 billion in bonds to keep the states stem cell research agency running and grants flowing to California universities and companies.

That groundbreaking measure authorized $3 billion in state bonds to create the states stem cell research agency, the California Institute for Regenerative Medicine, and fund grants for research into treatments for Alzheimers disease, cancer, spinal cord injuries and other diseases.

The institute has nearly used up its original funding, so Prop. 71s author, real estate investor and attorney Robert N. Klein II, led a new effort to get Prop. 14 on the November ballot.

This time, embryonic stem cell research is in a much different place, with federal funding no longer blocked and more funding from the biotech industry.

Voters will want to consider what Californias previous investment in stem cell research has accomplished. Its a nuanced track record.

While many scientific experts agree that Prop 71 was a bold social innovation that successfully bolstered emerging stem cell research, some critics argue that the institutes grantmaking was plagued by conflicts of interest and did not live up to the promises of miracle cures that Prop. 71s supporters made years ago. Although the agency is funded with state money, its overseen by its own board and not by the California governor or lawmakers.

The agency had done a very good job of setting priorities for stem cell research, including research using human embryos, and doling out $300 million annually to build up California as a regenerative medicine powerhouse, according to a 2013 evaluation by the National Academies of Science, Engineering and Medicine.

But the report also found that because the institutes board is made up of scientists from universities and biotech firms likely to apply for grants, board members had almost unavoidable conflicts of interest.

Because human stem cells can develop into many types of cells, including blood, brain, nerve and muscle cells, scientists have long looked to them for potential treatments for currently incurable diseases and injuries. Researchers use two types of stem cells: embryonic stem cells, derived from unused human embryos created through in vitro fertilization, and adult stem cells, which are harder to work with but in some cases can be coaxed in a lab into behaving more like embryonic stem cells.

From the start, stem cell research has been ethically charged and politically controversial because human embryos are destroyed in some types of studies. Federal restrictions on the research have waxed and waned, depending on which political party holds power. While former President Bush restricted federal money for embryonic stem cell research, former President Obama removed those restrictions.

The Trump administration has restricted government research involving fetal tissue but not embryonic stem cells. However, anti-abortion lawmakers have called on the President to once again end federal funding for embryonic stem cell research.

California-funded research has led to one stem cell treatment for a form of Severe Combined Immunodeficiency known as the bubble baby disease. Children with the rare disease dont make enough of a key enzyme needed for a normal immune system. Without treatment, they can die from the disease if not kept in a protective environment. The U.S. Food and Drug Administration is now reviewing the treatment but has not yet approved it for widespread use.

Although many of the agencys early grants were for basic science, the institute also has supported 64 clinical trials of treatments for many types of cancer, sickle cell disease, spinal cord injuries, diabetes, kidney disease and amyotrophic lateral sclerosis, commonlyknown as Lou Gehrigs disease.

A June 2020 analysis by University of Southern California health policy researchers estimated that taxpayers initial $3 billion investment in the research institute helped create more than 50,000 jobs and generated $10 billion for the states economy.

Gov. Gavin Newsom has endorsed Proposition 14, and other supporters include the Regents of the University of California, the California Democratic Party, the Juvenile Diabetes Research Foundation, patient advocacy groups like the March of Dimes, and some local politicians and chambers of commerce.

Supporters have raised more than $8.5 million, including about $2 million from billionaire Dagmar Dolby, to pass the measure, according to California Secretary of State campaign finance reports.

The passage of Proposition 71 helped save my life, Sandra Dillon, a blood cancer patient, wrote in a San Diego Union-Tribune commentary supporting Proposition 14. She wrote that she had benefited from a drug developed with Institute-funded research that has been designated by the FDA as a breakthrough therapy.

It is unimaginable to think that Californians would vote to discontinue this amazing effort I dont know where I would be or what condition I would be in if it wasnt for the investment Californians made nearly two decades ago.

I think the agencys done good work, but this was never planned to be funded forever with debt.

Lawrence Goldstein, a UC San Diego professor of cellular and molecular medicine and stem cell researcher, said the grants were instrumental in furthering his research on treatments for Alzheimers disease and that Prop. 14 will help create new jobs. The agency has funded a great deal of very important stem cell medical research thats already produced terrific results and has the prospect of saving many more lives in the decade to come, he said.

Opponents include one member of the institutes board and a nonprofit that advocates for privacy in genetic research. They contend that the proposition seeks too much money and does not sufficiently address the conflicts of interest that cropped up after Prop. 71 was passed. They also note that private funding, including venture capital, for stem cell research has grown in recent years. Opponents had raised only $250 by late September, from a single contribution by the California Pro Life Council.

The editorial boards of some of Californias biggest newspapers also have opposed the measure, including the Los Angeles Times, the Orange County Register, the San Francisco Chronicle and the San Jose Mercury News/East Bay Times. The Fresno Bee, Modesto Bee, and San Luis Obispo Tribune newspaper editorial boards support Prop 14.

Jeff Sheehy, the only institute board member not to support Proposition 14, told CalMatters that the research environment has changed since voters initially approved state funding for stem cell research in 2004 and that California should prioritize other needs like education, health care, and housing.

I think the agencys done good work, but this was never planned to be funded forever with debt, Sheehy said. At this point the state cant afford it; were looking at a huge deficit.

Read the rest here:
Prop. 14: In the COVID age, can California still afford its stem cell research program? - CALmatters

Spinal Cord Stem Cells Comments Off on Prop. 14: In the COVID age, can California still afford its stem cell research program? – CALmatters | October 16th, 2020

By Michael Brown, CP Op-Ed Contributor | Thursday, October 15, 2020

Have you heard the latest? Trump cannot be pro-life since he used and is promoting the anti-COVID drug Regeneron, which was allegedly developed with the help of fetal tissue. And pro-life organizations are being hypocritical by refusing to condemn the drug. Is there any truth to these charges?

As reported by the UK Metro, Trump faces hypocrisy allegations after it was revealed Regeneron is made from stem cells originally taken from an embryonic kidney. That kidney was taken during an elective abortion performed in the Netherlands during the 1970s.

More bluntly, the MIT Technology Review claimed, Trumps antibody treatment was tested using cells originally derived from an abortion.

The Trump administration has looked to curtail research with fetal cells. But when it was life or death for the president, no one objected.

As for pro-life organizations, a lengthy headline on Business Insider stated, Antiabortion groups say they stand behind Trump's use of a drug tested on cells derived from an aborted fetus because the president was not involved with that abortion.

How should pro-life Trump voters respond to these concerns?

Lets say that Regeneron was actually developed with the help of fetal tissue. Does anyone actually think that doctors came to Trump and said, Mr. President, we have an experimental drug that was tested and developed using tissue from an aborted baby from the 1970s. How do you feel about using this?

Only the most hardened anti-Trumper could imagine such a scenario. In the world of reality, the very thought of it is absurd. And, even if the fetal tissue charges are true, who knew about this? Was this something that any of the doctors would have been aware of? I very seriously doubt it, especially when, as we shall see, Regeneron itself denies the charge.

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It is therefore completely ridiculous to claim that Trump is being hypocritical in using Regeneron, as if he knew the alleged history of the drug.

But that leads us back to the big question: Did Regeneron use fetal tissue from a baby aborted in the 1970s?

Another headline on Business Insider stated, One of Trump's COVID-19 treatments was developed using tissue that originally came from an abortion. He's tried to ban this kind of research.

This is being widely reported as established fact. But is it true?

In an official statement for the pro-life Charlotte Lozier Institute, David Prentice, Ph.D. and Tara Sander Lee, Ph.D., wrote:

The Regeneron therapy given to the president was made in Velocimmune humanized mice, a novel platform that uses genetically modifiedmouse embryonic stem (ES)cells to generate antibodies describedhereandhere.Development of Regenerons antibody cocktail is detailed in the journalScience, describing how they identified their antibodies made from Velocimmune miceandblood from recovered patientspreviously infected with SARS-CoV-2.The final antibody pair used in theREGN-COV2 therapycocktail was then produced inChinese hamster ovary (CHO) cells.Results from Regenerons REGN-COV2 clinical trials to treat COVID-19 patients are reportedhere.

Mouseembryonic stem cells and genetic modifications to make such mice date back to 1981, have been extensively studied, and were instrumental in the discovery of induced pluripotent stem (iPS) cells, another fully ethical alternative to fetal material, as discussed in thisNaturereview.iPSCs are much easier to use thanhuman embryonic stem cells,more flexible in their uses, and are not ethically controversial. No one has ever advocated against using mouse embryonic stem cells for development of therapies only against destruction of human lives.

In a statement made to The Christian Post, Alexandra Bowie, a spokesperson for Regeneron, explained, We did not use human stem cells or human embryonic stem cells in the development of REGN-COV2. We did use the HEK293T cell line to test our antibodies ability to neutralize the SARS-CoV-2 virus (they were used to make pseudovirus that looks like the Spike protein).

HEK293s are considered immortalized cells and are a common and widespread tool in research labs. The cell line was originally derived by adenovirus transformation of human embryonic kidney cells in 1977.

After this, it was further transformed at Stanford in the 80s with SV40 T-antigen (hence the T). HEK293T wasnt used in any other way and fetal tissue was not used in this research.

Im not a medical doctor or a scientist, but it seems clear from these descriptions (and from what I could glean from Regenerons technical article published in the journal Science, also cited above) that there is hardly a direct connection between the drug and an aborted baby.

Still, there is so much misinformation going viral online that Dr. Tara Lee started a Twitter account just to rebut the misconceptions. (Lees Twitter bio identifies her as Researcher, Clinical Scientist, Science Policy Advisor. PhD. Senior Fellow & Director of Life Sciences @lozierinstitute. For LIFE in this world and the next.)

She summarized the evidence in simple and clear terms: NO human embryonic stem cells or human fetal tissue were used to produce the treatments President Trump received period.

So, Regeneron denies that it used embryonic cells and other scientists deny it. This completely undermines the charge that pro-life groups are being hypocritical for not condemning the use of the drug (and the presidents promotion of it).

That being said, some pro-life groups have, in fact, opined that, even if testing for the drug could be traced back to an abortion in the 1970s, Trump had nothing to do with any of this and is therefore not being hypocritical in promoting the drug.

Is there some hypocrisy in this response?

Thats a fair question, and its the type of question that ethicists debate. If you could save the lives of many people using a drug that was developed with the help of aborted fetal cells, is that justifiable?

My answer to those accusing the president with hypocrisy is threefold. First, as stated here, its unlikely in the extreme he had any idea of the alleged abortion connection. Second, scientists from Regeneron deny any connection to human fetal cells, and Trump would presumably take them at their word. Third, if Regeneron had been developed with the help of an aborted baby, there would then be a serious ethical debate as to its use.

Still, with all that being said, it is grasping at straws to question the pro-life commitment of these organizations (along with that of President Trump). Thats because we are comparing the willful killing of more than 60 million babies in the womb, often out of convenience, with the possible, distant connection of a life-saving drug to a baby aborted in the 1970s. Who would seriously make such a comparison?

When it comes to the question of the benefit of embryonic cells in the development of medical cures, Dr. Lee noted that, Fetal tissue from aborted babies is not required for medical advancements. After 100+ years of research, no therapies have been developed that needed aborted fetal tissue to begin with. Newer & better ethical alternatives are available & being used now to make humanized mice.

There is, therefore, no justification for using embryonic fetus cells in medical research, and the Regeneron drug does not contradict this at all.

Of course, as readers here know well, I have no problem identifying President Trumps many, evident shortcomings, some of which can be quite destructive. And I continue to shake my head over some of the things he says.

But to call him a hypocrite or to question his pro-life commitment because he used Regeneron is to reveal ones anti-Trump animus. It indicts the accuser far more than it indicts the accused.

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Dr. Michael Brown(www.askdrbrown.org) is the host of the nationally syndicatedLine of Fireradio program. His latest book isEvangelicals at the Crossroads: Will We Pass the Trump Test?Connect with him onFacebook,Twitter, orYouTube.

Excerpt from:
Regeneron, Trump, and the alleged hypocrisy of the pro-life movement - Christian Post

IPS Cell Therapy Comments Off on Regeneron, Trump, and the alleged hypocrisy of the pro-life movement – Christian Post | October 16th, 2020

DUBLIN--(BUSINESS WIRE)--The "Cell Based Assay & High Content Screening Markets Market Forecasts by Application, With Executive and Consultant Guides and including Customized Forecasting and Analysis 2020 to 2024" report has been added to ResearchAndMarkets.com's offering.

This updated report will bring the entire management team up to speed, on both the technology and the opportunity.

Cell Based Assays are a mainstay of drug development and scientific research, but growth is now accelerating as new immuno-oncology markets create unprecedented investment in the race to cure cancer. On top of this new technology is allowing Cell Based Assays to be used to measure any aspect of cell function. This market just keeps on growing with no end in sight. The workhorse of the pharmaceutical industry is becoming a central player in biotechnology.

The technology is moving faster than the market. Genomics and Immunology are playing a role too. Find opportunities and pitfalls. Understand growth expectations and the ultimate potential market size.

Key Topics Covered:

1. Introduction and Market Definition

1.1 What are Cell Based Assays?

1.2 Clinical Trial Failures

1.2.1 Immuno-oncology Plays a Leading Role in Cell Based Assays

1.3 Market Definition

1.4 Methodology

1.5 U.S. Medical Market and Pharmaceutical Research Spending - Perspective

1.5.1 U.S. Expenditures for Pharmaceutical Research

2. Cell Based Assays - Guide to Technology

2.1 Cell Cultures

2.1.1 Cell Lines

2.1.2 Primary Cells

2.1.3 Stem Cells

2.1.3.1 iPSC's - The Special Case

2.2 Cell Assays

2.3 Cell Viability Assays

2.3 Cell Proliferation Assays

2.4 Cytotoxicity Assays

2.5 Cell Senescence Assays

2.6 Apoptosis

2.7 Autophagy

2.8 Necrosis

2.9 Oxidative Stress

2.10 2D vs. 3D

2.11 Signalling Pathways, GPCR

2.12 Immune Regulation & Inhibition

2.13 Reporter Gene Technology

2.14 CBA Design & Development

2.15 Cell Based Assays - The Takeaway

3. Industry Overview

3.1 Players in a Dynamic Market

3.1.1 Academic Research Lab

3.1.2 Contract Research Organization

3.1.3 Genomic Instrumentation Supplier

3.1.5 Cell Line and Reagent Supplier

3.1.6 Pharmaceutical Company

3.1.7 Audit Body

3.1.8 Certification Body

4. Market Trends

4.1 Factors Driving Growth

4.1.1 Candidate Growth

4.1.2 Immuno-oncology

4.1.3 Genomic Blizzard

4.1.4 Technology Convergence

4.1.5 The Insurance Effect

4.2 Factors Limiting Growth

4.2.1 CBA Development Challenges

4.2.2 Instrument Integration

4.2.3 Protocols

4.3 Technology Development

4.3.1 3D Assays

4.3.2 Automation

4.3.3 Software

4.3.4 Primary Cells

4.3.5 Signalling and Reporter Genes

4.3.6 The Next Five Years

5. Cell Based Assays Recent Developments

5.1 Recent Developments - Importance and How to Use This Section

5.1.1 Importance of These Developments

5.1.2 How to Use This Section

6. Profiles of Key Cell Based Assay Companies

7. Global Market Size

8. Global Market by User Type

8.1 Pharmaceutical Market

8.2 Basic Research Market

8.3 Industrial/Cosmetic Market

9. Cell Based Assay by Product Class

9.1 Instrument Market

9.2 Reagent Market

9.3 Services Market

9.4 Software Market

10. Appendices

10.1 FDA Cancer Drug Approvals by Year

10.2 Clinical Trials Started 2010 to 2016

10.3 Share of Pharma R&D by Country

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Global Cell Based Assay & High Content Screening Markets to 2024: Updated Report - Understand Growth Expectations and the Potential Market Size -...

Cardiac Stem Cells Comments Off on Global Cell Based Assay & High Content Screening Markets to 2024: Updated Report – Understand Growth Expectations and the Potential Market Size -… | October 16th, 2020

Indias first saviour sibling experiment is a success, say doctors.

A one-year-old sibling has saved her brothers life by donating her bone marrow. Kavya was conceived by her parents through invitro fertilisation to save her brother, Abhijeet Solanki, who was born with Thalassemia.

Thalassemia is a disorder where the haemoglobin count is low in blood and such persons require frequent blood transfusions.

Abhijeet was born in November 2013 but unlike normal babies he did not achieve the growth milestones. The parents learned that Abhijeet had Thalassemia major. Abhijeet required blood transfusions every 25 days and the gap between two transfusions reduced as he grew. By the age of six Abhijeet had undergone 80 transfusions, recalled his father Sahdev Singh Solanki. The only way to save him was through a bone marrow transplant.

The family was willing to donate their bone marrow but the human leukocyte antigen (HLA) of the family, including that of his older sister, did not match.

The Solanki family consulted many doctors. Mr. Solankis research led him to the saviour sibling concept following which he sought out Manish Banker, medical director of Nova IVF Fertility in Ahmedabad.

Dr. Banker said Mr. Solankis research and the science behind it was known but nobody had approached him with such a request before.

Dr. Banker started the assisted reproductive therapy, called pre-implantation genetic testing, for monogenic disorder with HLA matching. The couple underwent three cycles of IVF and 18 embryos were created. Of this only one perfectly matched Abhijeets HLA. The embryo was implanted in Apla Solanki, who delivered a baby girl a year ago.

We had to wait for the baby to grow. She had to weigh 10 kg before we could draw bone marrow, said Deepa Trivedi, programme director of Sankalp Bone Marrow Unit, CIMS Hospital, Ahmedabad.

Pointing out that the best therapeutic option for Thalassemia major patients is bone marrow transplant from an HLA-identical donor, Dr. Banker said, We are extremely thrilled to be part of reproductive history in India to create the first-ever saviour-sibling through ART. We used pre-genetic diagnosis and screening test, an established method for conceiving a child who may donate cord blood or hematopoietic stem cells for transplantation to save a critically ill sibling.

Mr. Solanki said the transplant was done on March 17. Since then Abhijeet has not needed any blood transfusion, indicating that he had been cured of the disorder. His haemoglobin count was 11.3, Dr. Trivedi said.

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A sister born to save ailing brother - The Hindu

Bone Marrow Stem Cells Comments Off on A sister born to save ailing brother – The Hindu | October 16th, 2020