Stem Cell Clinic

Exploring Increase of Point Mutations Associated with High-Dose Melphalan in Multiple Myeloma – Targeted Oncology

By adminDecember 16, 2020Stem Cell Treatment

High-dose melphalan (Evomela) followed by autologous stem cell transplant (ASCT) has demonstrated significant efficacy for the treatment of patients with multiple myeloma. The agent has improved progression-free survival (PFS) in patients when administered as upfront therapy to patients aged 66 years or younger.

The median PFS shown with melphalan plus ASCT in a phase IFM/DFCI 2009 study was 50 months versus 26 months when compared with the standard of care treatment regimen lenalidomide (Revlimib), bortezomib (Velcade), and dexamethasone (RVD) alone hazard ratio for disease progression or death, 0.65;P<.001). As the treatment strategy continues to be explored in patients with multiple myeloma, researchers have now begun to investigate an outstanding biological question of whether the alkylating agent causes an increased amount of DNA damage.

An analysis presented during the virtual 2020 American Society of Hematology (ASH) Annual Meeting showed that between the time of diagnosis and relapse, patients treated with high-dose melphalan had an increased number of point mutations. It was unclear from this research how this result translated to treatment selection and sequencing.

In an interview with Targeted Oncology, Mehmet Samur, PhD, senior research scientist, Dana-Farber Cancer Institute, discussed the investigation of high-dose melphalan following ASCT in patients with multiple myeloma and shared insights into how the ongoing questions can be explored in the future.

TARGETED ONCOLOGY: Can you explain what was demonstrated prior with high-dose melphalan followed by ASCT in patients with multiple myeloma?

Sumar: The clinical part of the phase 3 study was published previously. It showed that when you do RVD plus high-dose melphalan following by stem cell treatment, patients do significantly better than patients who get RVD alone. Adding high-dose melphalan increased the PFS benefit by around 12 months.

TARGETED ONCOLOGY: Can you provide background on your analysis of Melphalan for patients with multiple myeloma?

Sumar: Melphalan is an alkylating agent. Because of the way the agent works, we always think that it creates more DNA damage. The study that we presented at ASH was questioning whether this was true or not.

We collected DNA sequencing data from patients who were treated with RVD followed by high-dose Melphalan and a bone marrow transplant. We had a total of 25 patients, and we collected data at the time of diagnosis and the time of relapse. To compare this compilation, we also collected data from 43 patients from the IFM/DFCI 2009 study who only received RVD. We also collected data at diagnosis and relapse in the 43 patients. Genomic alterations were compared at diagnosis and relapse for patients who were injected with high-dose Melphalan and RVD versus patients who were only treated with RVD.

TARGETED ONCOLOGY: What were the findings from this study?

Sumar: We found that patients who got high-dose melphalan plus RVD followed by transplant accumulated more point mutations. To be precise, they accumulated around 10,000 new mutations between diagnosis and relapse at 5 years. For RVD patients, there were around 4500 new point mutations. The study showed that treating patients with high-dose Melphalan is increasing the mutational load by about 2.9-fold at the time of relapse.

TARGETED ONCOLOGY: What are the implications of these findings?

Sumar: There are a couple of things that we see from our study. One point is that we only saw point mutations. We didnt see any large-scale DNA alterations. This suggests that our patients who are treated with high-dose melphalan are more likely to experience changes.

In terms of the pathways that are mutated, the DNA damage repair pathway is more frequently mutated between diagnosis and relapse in patients treated with high-dose melphalan. We think that if we combine inhibitors that can overcome the selection of DNA damage repair pathway mutations, those patients may get additional benefit from the treatment.

We dont have clear data yet on whether this increased mutational load is something that is bad for patients. Even though these patients have more mutations, overall survival times are similar between the 2 arms. C outcomes are not impacted by the increased number of mutation so far.

TARGETED ONCOLOGY: What plan are underway to further this research?

Sumar: We are expanding our study in multiple ways. There is no clear data set we can get answers to our ongoing questions yet. We have reached out to our partners around the world to see if we can come up with a cohort to investigation. Also, we are looking at impact of these mutational load increase on other features like secondary cancer rate.

TARGETED ONCOLOGY: The understanding of gene mutations in myeloma is evolving. Can you discuss the current role of genomic testing?

Sumar: It has been shown in many studies that genetic testing at diagnosis can tell us which patients are high risk and which are low risk. Studies have also shown that patients who have loss of p53 or with deletion 17p will have bad outcomes.

There was a study published last year in the Journal of Clinical Oncology showing which patients with myeloma would have a lower risk. The study also shows that there are certain genomic features prolong survival time in patients.

We have different genomic tools that we can use to look at these different alterations and assess patient risk. Today, I think people are looking at these alterations from all different angles to plan stratification in upcoming clinical trials.

TARGETED ONCOLOGY: In your opinion, what change will we see in the myeloma treatment landscape in the next 5 years?

Sumar: There are a lot of studies looking at new treatment. Everyone is carefully watching out for data on new treatment options like chimeric antigen receptor T-cell therapy, bispecific antibodies, and monoclonal antibodies. It looks like these agents are providing benefit to patients, but they are at the very early stages of research.

Reference:Attal M, Lauwers-Cances V, Hulin C, et al. Lenalidomide, bortezomib, and dexamethasone with transplantation for myeloma. N Engl J Med. 2017; 376(14):1311-1320. doi: 10.1056/NEJMoa1611750

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Exploring Increase of Point Mutations Associated with High-Dose Melphalan in Multiple Myeloma - Targeted Oncology

Straight from the Heart: Cooper’s Story – WBIR.com

By adminDecember 16, 2020Stem Cell Treatment

In just one year, 4-year-old Cooper Stansbury has conquered six rounds of Chemotherapy, two stem cell transplants and 14 days of Proton Therapy.

KNOXVILLE, Tenn. In August, we introduced you to 4-year-old Cooper Stansbury.

Law enforcement from around the area came together to encourage him as he's battling cancer.

In November of 2019, Cooper was diagnosed with neuroblastoma.

"I'll never forget that day. That was the worst day of our lives," said Cooper's father, Sam Stansbury. "My wife collapsed. Obviously, she was falling apart and all I wanted to know is what do we do now, what do we do next."

For more than a year, Cooper has endured treatment after treatment.

Cooper has conquered six rounds of Chemotherapy, two stem cell transplants and 14 days of Proton Therapy.

"The Power of Play" helps Cooper stay positive and decreases anxiety.

"Sometimes they do have to go through hard procedures that might hurt or be scary," said East Tennessee Children's Hospital Child Life Specialist Anna Taylor. "So, I can help distract and help them understand why they are going through it and help them cope and get through the procedure."

Cooper's father said "he's tough as nails."

Just like the toy race cars on his track, nothing slows cooper down.

Cooper is now on cycle 4 of immunotherapy.

"To see him now after everything he's gone through," Cooper's father said. "We're very hopeful."

As Cooper continues to fight, his entire family is looking forward to better days and more play days at home.

"I know he is destined for greatness, without a doubt," he said.

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Straight from the Heart: Cooper's Story - WBIR.com

Early Signs of Activity and Tolerability Found in Allogeneic Product UCART22 for Patients with Relapsed/Refractory CD22+ B-Cell ALL – Cancer Network

By adminDecember 16, 2020Stem Cell Treatment

The allogeneic off-the-shelf CD22-directed T-cell product, UCART22, showed early signs of activity and no evidence of unexpected toxicities at 2 dose levels for adult patients with relapsed/refractory CD22-positive B-cell acute lymphoblastic leukemia, according to the results of a study presented during the 2020 ASH Annual Meeting.1

In the phase 1 BALLI-01 (NCT04150497) dose-escalation and dose-expansion study, 2 patients at the 1 x 105 cells/kg dose achieved a complete remission (CR) with incomplete hematologic recovery on day 28. One of these patients attained a minimal residual disease (MRD)positive CR at day 42 followed by subsequent inotuzumab ozogamicin (Besponsa) and then transplant.

One patient at dose level 2, 1 x 106 cells/kg, experienced a significant bone marrow blast reduction at day 28, followed by disease progression.

No patients experienced dose-limiting toxicities (DLTs), immune effector cellassociated neurotoxicity syndrome (ICANS), graft-versus-host disease (GVHD), adverse effects (AE) of special interest (AESI), a UCART22-related AE that was grade 3 or higher, or a serious AE (SAE).

UCART22 showed no unexpected toxicities at the doses of 1 x 105 cells/kg and 1 x 106 cells/kg with fludarabine and cyclophosphamide lymphodepletion, lead study author Nitin Jain, MD, an assistant professor in the Department of Leukemia, The University of Texas MD Anderson Cancer Center, said in a virtual presentation during the meeting. Host immune recovery was observed early, and the addition of alemtuzumab [Lemtrada] to fludarabine and cyclophosphamide lymphodepletion is currently being explored with the goal to achieve deeper and more sustained T-cell depletion and to promote expansion and persistence of UCART22.

Standard treatment for adult patients with B-cell ALL includes multiagent chemotherapy with or without allogeneic stem cell transplant. However, 30% to 60% of patients with newly diagnosed B-cell ALL who achieve a CR will relapse, and the expected 5-year survival rate for those with relapsed/refractory disease is approximately 10%.

Previously, UCART19, when paired with lymphodepletion using fludarabine, cyclophosphamide, and alemtuzumab, was found to show efficacy in this patient population.2

CD22 is an FDA-approved therapeutic target in B-cell ALL. UCART22 is an immediately available, standardized, manufactured agent with the ability to re-dose, and its CAR expression redirects T cells to tumor antigens, Jain explained.

Moreover, through its mechanism of action, TRAC becomes disrupted using Transcription activator-like effector nucleases (Talen) technology to eliminate TCR from cell surface and reduce the risk of GVHD. CD52 is also disrupted with the use of Talen to eliminate sensitivity to lymphodepletion with alemtuzumab. Finally, there is a CD20 mimotope for rituximab (Rituxan) as a safety switch, Jain added.

UCART22 has also demonstrated in vivo antitumor activity in immune-compromised mice that were engrafted with CD22-positive Burkitt lymphoma cells in a dose-dependent manner.

In the dose-escalation/dose-expansion BALLI-01 study, investigators are enrolling up to 30 patients in a modified Toxicity Probability Interval design. There are 3 cohorts, which have 2 to 4 patients on each cohort: 1 x 105 cells/kg (dose level 1), 1 x 106 cells/kg (dose level 2), and 5 x 106 cells/kg. The focus of the dose-escalation phase of the trial was to determine the maximum-tolerated dose (MTD) and the recommended phase 2 dose (RP2D) before heading into the dose-expansion portion of the trial.

To be eligible for enrollment, patients must have been between 18 and 70 years old, have acceptable organ function, an ECOG performance status of 0 or 1, at least 90% of B-cell ALL blast CD22 expression, and had previously received at least 1 standard chemotherapy regimen and at least 1 salvage regimen.

End points of the trial included safety and tolerability, MTD/R2PD, investigator-assessed response, immune reconstitution, and UCART22 expansion and persistence.

The lymphodepletion regimens were comprised of fludarabine (at 30 mg/m2 x 4 days) plus cyclophosphamide (1 g/m2 x 3 days); the study has since been amended to include the regimen of fludarabine (at 30 mg/m2 x 3 days), cyclophosphamide (500 g/m2 x 3 days), and alemtuzumab (20 mg/day x 3 days) and is currently enrolling patients.

Following screening, lymphodepletion, and UCART22 infusion, patients underwent an observation period for DLTs with a primary disease evaluation at 28 days; additional efficacy evaluations occurred at 56 days and 84 days. Patients were followed for 2 years and continued to be assessed for long-term follow-up.

As of July 1, 2020, 7 patients were screened, of which 1 patient failed and 6 were therefore enrolled on the study. One patient discontinued therapy before receiving UCART22 due to hypoxia from pneumonitis that was linked with lymphodepletion. Five patients were treated with UCART22 at dose level 1 (n = 3) and dose level 2 (n = 2).

The median age of participants was 24 years (range, 22-52), 3 of the 5 patients were male, and 3 had an ECOG performance status of 0. The median number of prior therapies was 3 (range, 2-6), and there were a median 35% bone marrow blasts (range, 10%-78%) prior to lymphodepletion.

Three patients had complex karyotype and 2 had diploid cytogenetics. One patient each had the following molecular abnormalities: CRLF2, CRLF2 and JAK2, CDKN2A loss, KRAS and PTPN11, and IKZF1. Only 1 patient had undergone haploidentical transplant. Four patients previously received prior CD19- or CD22-directed therapy, including blinatumomab (Blincyto), inotuzumab ozogamicin (Besponsa), and CD19-directed CAR T-cell therapy. At study entry, 3 patients had refractory disease and 2 patients had relapsed disease.

Grade 3 or higher treatment-emergent AEs (TEAEs), which were unrelated to study treatment, included hypokalemia, anemia, increased bilirubin, and acute hypoxic respiratory failure. Also not related to UCART22, 3 patients experienced 4 treatment-emergent SAEs: porta-hepatis hematoma, sepsis, bleeding, and sepsis in the context of disease progression. No treatment discontinuations due to a treatment-related TEAE were reported.

The patient who achieved a CR followed by transplant was a 22-year-old male who had undergone 2 prior treatments for B-cell ALL and received UCART22 at a dose of 1 x 105 cells/kg. He did not experience CRS, ICANS, GVHD, nor a SAE, and all TEAEs were grade 1.

Jain also noted that host T-cell constitution was observed in all patients within the DLT observation period. UCART22 was also not detectable through flow cytometry or molecular analysis, the latter of which was at dose level 1 only.

References:

1. Jain N, Roboz GJ, Konopleva M, et al. Preliminary results of BALLI-O1: a phase I study of UCART22 (allogeneic engineered T cells expressing anti-CD22 chimeric antigen receptor) in adult patients with relapsed/refractory anti-CD22+ B-cell acute lymphoblastic leukemia (NCT04150497). Presented at: 2020 ASH Annual Meeting and Exposition; December 4-8, 2020; Virtual. Abstract 163.

2. Benjamin R, Graham C, Yallop D, et al. Preliminary data on safety, cellular kinetics and anti-leukemic activity of UCART19, an allogeneic anti-CD19 CAR T-cell product, in a pool of adult and pediatric patients with high-risk CD19+ relapsed/refractory b-cell acute lymphoblastic leukemia. Blood. 2018;132(suppl 1):896. doi:10.1182/blood-2018-99-111356.

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Early Signs of Activity and Tolerability Found in Allogeneic Product UCART22 for Patients with Relapsed/Refractory CD22+ B-Cell ALL - Cancer Network

Epigenetic Changes Implicated in Age-related Diminution in Vision and Its Possible Reversal – JD Supra

By adminDecember 16, 2020Stem Cell Treatment

The Fountain of Youth -- an enduring aspiration, particularly as the ravages of age reduce human faculties prior to leading inexorably to death. Reduction in sight is the human faculty that can have the greatest effect on quality of life in the aged -- a faculty that begins to decline in the 4th or 5th decade of life and doesn't get better (when it does) without medical intervention.

But what if there were a way to rejuvenate sight? That prospect is the tantalizing suggestion in a paper published on December 2nd entitled "Reprogramming to recover youthful epigenetic information and restore vision," Nature 588: 124-29*. The basis of the report is the recognition that many of the age-related effects on vision are an example of gene expression differences associated with epigenetic changes in chromosomal DNA. Epigenetics is a phenomenon of gene structure and expression involving small differences in nucleotide bases, typically methylation of cytosine residues at specific (CpG) sites. These changes have been studied in normal development, where gene expression changes arise as different cell types properly differentiate and act as a molecular "clock" reflecting age. The ability to turn back cellular time has been demonstrated by the development of induced pluripotent stem cells (iPSCs), wherein terminally differentiated somatic cells (typically fibroblasts) can be turned into pluripotent cells. Pluripotent cells are capable of differentiating into cells of each embryonic germinal layer (ectoderm, mesoderm, endoderm), and iPSCs can be produced by expressing four specific genes: OCT4, SOX2, KLF4 and MYC. All of these genes encode transcription factors capable of affecting (and effecting) developmentally relevant gene expression. Consequent to this "de-differentiation" occasioned by expression of these genes is a "resetting" of the epigenetic patterns associated with development. In this paper the researchers hypothesized that resetting these epigenetic patterns could also rejuvenate neuroretinal cells to reinvigorate and overcome the ocular nerve damages by glaucoma in an animal model.

Because one of these genes (MYC) is also associated with cancer development (i.e., it is an oncogene) the researchers developed an inducible expression construct that expressed only the OCT4, SOX2, and KLF4 members of the quartet (OSK). (This decision was also informed by the experience of other researchers that continuous expression of all four genes in animal models resulted in teratomas or was fatal within days of introduction.) Their system used a polycistronic (i.e., all the genes in one linear array) construct of all three genes regulated by a tetracycline response element (TRE) promoter in a adeno-associated viral vector. This construct was tested by introduction into fibroblasts from aged (20 month old) mice and gene expression related to aging (i.e., that showed differential expression with age) was evaluated. These studies showed that OSK expression for 5 days resulted in a "youthful" mRNA expression pattern in these genes (without any effect on the terminal differentiation state of the fibroblasts).

The TRE promoter enabled selection for or against expression of the OSK gene cassette; as the authors explain "[t]he TRE promoter can be activated either by reverse tetracycline-controlled transactivator (rtTA) in the presence of the tetracycline derivative doxycycline (DOX) ('Tet-On') or by tetracycline-controlled transactivator (tTA) in the absence of DOX ('Tet-Off')." Simply put, the presence of absence of DOX in the animal's drinking water determined whether the expression cassette is "on" or "off," as illustrated in this figure:

Long-term (10-18 months) expression of this cassette was achieved in both young (5 months-old) and aged mice with no tumorigenesis or other negative side effects being observed.

To test the ability of induced OSK expression to rejuvenate optical nerve cells the researchers examined retinal ganglion cells (RGC, which project axons away from the retina informing the optic nerve) in an optic nerve crush injury model (which mimics the effects of optic nerve injury due to inter alia glaucoma). The construct was delivered by injection into the vitreous humor and resulted in about 37% of the RGCs taking up and expressing the OSK genes in response to DOX administration. A separate cohort of mice were administered versions of the construct where DOX inhibited OSK expression. In these experiments, "the greatest extent of axon regeneration and RGC survival occurred when all three genes were delivered and expressed as a polycistron within the same AAV particle" according to the researchers. In contrast, inhibition of OSK expression in the "Tet-Off" mice showed no axonal growth. Moreover, delivery of the OSK genes individually in separate viral vectors or in pairs also did not show axonal growth, indicating the need for these genes to be expressed together in proper relative amounts provided by the polycistronic construct. The researchers also found OSK expression induced expression of Stat3, a gene know to encourage regeneration. These results were obtained in using 12-month-old mice as well as 1- and 3-month-old mice, which indicated, as the authors note, that "ageing does not greatly diminish the ability of OSK transcription factors to induce axon regeneration." Increased axonal growth from RGCs was found even after crush injury, an effect found with no other treatment modalities.

The researchers then determined whether these reinvigorated RGCs showed changes in DNA methylation patterns. In the absence of DOX-induced OSK expression injury in this model caused an "accelerated" aging pattern, whereas in the presence of DOX-induced OSK expression counteracted this effect according to the results reported in this paper. Interestingly, this preservation of a "youthful" pattern of DNA methylation was found to be enriched at genes "associated with light detection and synaptic transmission." Having shown this association the researchers then investigated whether axonal regeneration required youthful changes in DNA methylation. These experiments were performed by reducing expression of genes that caused DNA demethylation in RGCs (and whose expression was known to be increased in cells expressing OSK) and detecting that axonal regeneration did not occur in these mice even in the presence of DOX-induced OSK expression.

Whether these effects of OSK expression would also be seen in human neurons was investigated using differentiated human neurons in vitro. Neurons harboring an OSK-encoding construct were treated with vincristine (a drug that occasions axon injury) and DOX-induced OSK expression was shown to "counteract[] axonal loss and the advancement of DNA methylation age," showing a 15-fold greater area of proliferation in OSK-expressing cells than control vincristine-treated neural cells. These cells also showed the demethylation-dependent characteristics that were shown in RGCs in the mouse optic nerve crush injury model.

The most clinically significant result disclosed in this paper involved the effect of OSK expression in a glaucoma model in vivo. Intraocular pressure was increased to pathological levels by injecting microbeads unilaterally into the anterior chamber of mouse eye for 21 days. At 4 weeks, after these animals showed correspondingly unilateral decreases in axonal density and the number of RGCs present in the treated eye. The viral vector encoding inducible OSK expression thereafter was introduced by intravitreal injection followed by DOX-induced OSK expression for 4 weeks. Compared with control (introduction of saline or viral vectors not encoding OSK into the microbead-treated eyes) the OSK vector-treated eyes showed "restored axon density equivalent to that in the non-glaucomatous eyes, with no evidence of RGC proliferation." These mice also showed a reversal of vision loss caused by the glaucomatous injury. Together these results indicated that OSK expression could be a therapy for glaucoma in humans.

Finally, the paper reports efforts to determine whether OSK expression could improve age-related (as opposed to injury- or pathology-related) vision problems. In these experiments, 3-and 11-month-old mice were treated by intravitreal injection of DOX-inducible OSK encoding constructs and OSK expression induced for 4 weeks. Twelve-month-old mice showed age-related visual acuity and RGS electrical activity diminution which was reversed by DOX-induced OSK expression. However, these phenotypic changes were not observed to be associated with an increased number of RGCs or axon density, which prompted these researchers to hypothesize that the effect were dependent on changes in gene expression ("transcriptomic changes" as these were termed in the paper). RGCs from treated or untreated 12-month-old mice were isolated and compared with RGCs from 5-month-old mice and expression of 464 genes were found to be altered: expression of almost all (90%) of these genes were found to be restored to youthful levels in OSK-expressing RGCs. The participation of DNA methylation changes in aged RGCs in producing a youthful pattern of gene expression was further assessed and validated using artificial intelligence/machine learning approaches.

The results reported in this paper suggest therapeutic interventions that could improve vision in the aged human population even in the absence of vision-impairing pathologies such as glaucoma. Although cautious to mention that "we do not wish to imply that DNA methylation is the only epigenetic mark involved in this process" and "[i]t is likely to involve other transcription factors and epigenetic modifications," the authors are not blind to the implication that:

[W]e show that it is possible to safely reverse the age of a complex tissue and restore its biological function in vivo. Using the eye as a model system, we present evidence that the ectopic expression of OSK transcription factors safely induces in vivo epigenetic restoration of aged CNS neurons, without causing a loss of cell identity or pluripotency. Instead, OSK promotes a youthful epigenetic signature and gene-expression pattern that causes the neurons to function as though they were young again. The requirement for active demethylation in this process supports the idea that changes in DNA methylation patterns are involved in the ageing process and its functional reversal.

* By researchers from Harvard Medical School, Yale University School of Medicine, Massachusetts General Hospital, UCLA Geffen School of Medicine, and The University of New South Wales Medical School.

California’s Proposition 14: short in the arm for stem cell research – BioNews

By adminDecember 16, 2020Stem Cell Clinics

14 December 2020

The recent close-call US presidentialelection grabbed headlines, but no less closely-fought was California's Proposition 14, also on the ballot in October, which will have a huge impact on the future of stem cell research in the state.

The Stem Cell Research Institute Bond Initiative (Prop 14),whichwas on theballotinthe initiated state statutewill allow the state to issue billions of dollars in bonds for its stem cellresearch programme. The vote could not have been closer, with 51 percent of ballots for and 49 percent against.

This initiative will enable financiers to lend US $5.5 billion to a stem cell agency, the California Institute for Regenerative Medicine (CIRM) whichthe state's taxpayers will repay with interest over the next three decades. The sizeable bond fund will be allocated to research, human clinical trials and programmes and also for start-up costs for facilities in the stem cell field. About US $1.5 billion of the money will be spent researching neurodegenerative conditions, including Alzheimer's, Parkinson's and dementia. Some of the funds will be allocated to the shared labs programme:state-funded facilities dedicated to conducting studies on human embryonic stem cells. A working groupfocused on improving access to medical treatments will be established, as well astraining and fellowship programmes for undergraduate and postgraduate students.

Stem cell research is in its infancy, with some treatments that have worked in animals now progressing to humantrials. The research is promising, as the ability to generate specialised cells could eventually be used torepair or replace damaged tissues and organs. However, there are ethical controversies surrounding stem cell research, especially regarding embryonic stem cells derived from early human embryos.

In 2001, the conservative Bush administration prohibited federal funding for stem cell research owing to the ethical concerns. This promptedreal estate developer and investor Robert Kleinto initiate the original Proposition 71 which aimed to establish a state constitutional right to conduct stem cell research in California in 2004.It was believed that Prop 71 would propel California as a world leader in stem cell research and 59 percent of Californians voted in favour.

The proposition also established CIRM the only state-funded stem cell research agency in the USA in the California Constitution. Since its inception, it has granted research funds to various institutions and companies including Stanford University and the University of California. So far, it has funded 68 clinical trials (phases 1-3) for a wide range of diseases and conditions, includingcancer, diabetes and many rare disorders.

In 2019, CIRMstarted to run out of funds, and by2020 hadsuspended applications for new projects except US $5 million in emergency funding for COVID-19 research. The Californians for Stem Cell Research, Treatments and Cures political action committee (PAC) responded with a campaign to boost funding via Prop 14. SupportersincludedUS House speaker Nancy Pelosi, California governorGavinNewsom, LA mayor EricGarcetti, the California Democratic Party and several organisations such as ALS Association and the Michael J Fox Foundation for Parkinson's research and Huntington's Disease Society of America.

There is also resistance to Prop 14:dissenters feel thattheinitiative has notdelivered its promises after 16 years. Furthermore the original rationale no longer applies asfederal funding is no longer blocked. Former President Barack Obama then removed the restrictions on embryonic stem cell research (a position unchanged by the Trump administration) which means California scientists can apply for (highly competitive) federal research grants.

In the current climate, Prop 14 will exacerbate California's already colossal budget deficits. The coronavirus pandemic has worsened housing crisesand unemployment rates, highly visible in cities like San Francisco and Los Angeles.

As Marcy Darnovsky, executive director of the Center for Genetics and Society, said:'It does nothing to address CIRM's built-in conflicts of interest, or its lack of legislative oversight despite it being an agency supported wholly by public funds. The new proposition makes some things worse; for example, it outsources critically important decisions about ethical standards to an unaccountable national committee... In the meantime, that campaign's shameless over-promising and hype set the stage for the hundreds of under-regulated commercial stem cell clinics now offering unapproved 'treatments' that have caused tumours and blindness...'

It is not surprising that Californians were so torn. Perhaps it's wiser to allocate the pot to more pressing matters like job creation, housing and other urgent needs?

Link:
California's Proposition 14: short in the arm for stem cell research - BioNews

What Patients With Cancer, Survivors Need to Know About the Emergency Use Authorization of COVID-19 Vaccine – Curetoday.com

By adminDecember 16, 2020Stem Cell Medical Center

Following the Food and Drug Administration (FDA)s emergency use authorization of the Pfizer-BioNTech COVID-19 Vaccine on Friday, many patients with cancer who are actively receiving treatment, and those who no longer have signs of active disease, are sure to have questions as to what they should know about the vaccine.

In fact, Dr. Debu Tripathy, chair of Breast Medical Oncology at The University of Texas MD Anderson Cancer Center and editor in chief of CURE, said he and his colleagues were getting questions about the distribution of the vaccine prior to its authorization by the FDA and Centers for Disease Control and Prevention.

We have been getting questions more and more frequently; all our patients want to know what the schedule is for when they might get a vaccine, said Tripathy in an interview with CURE.

To address any questions patients with cancer and survivors may have regarding the vaccine, CURE recently spoke with Drs. Debu Tripathy and Roy Chemaly, chief infection control officer and a professor in the department of infectious diseases, infection control and employee health at The University of Texas MD Anderson Cancer Center.

On Monday, the first of many high-risk health care workers started receiving the vaccine across the United States. Many frontline workers will continue to receive it over the next several weeks, including those who work directly with patients with cancer who are at a high risk for infection.

After those frontline workers, there is a process for which patients will begin to receive the vaccine, according to Tripathy.

Patients with underlying conditions as well as patients with cancer who are considered the most vulnerable for contracting the infection will likely be a top priority to receive the vaccine. Those patients would include someone with a hematologic malignancy who has had a stem cell transplant and/or are immunosuppressed. However, there are still some details that need to be ironed out.

We haven't gotten into the nitty gritty in terms of how we're going to divide (the vaccine) to some extent, said Tripathy. We're going to have the physicians be involved in prioritizing this based on their knowledge because they're the ones who know the patients the best.

Chemaly also noted that the vaccine will likely be administered to patients on a case-by-case basis.

Now for cancer patients who are still under active treatment with chemo or radiation, or early after stem cell transplantation, there is no data on how effective the vaccine is, and should it be used, he said. So we're going to be a little bit more cautious and take it case by case to recommend these vaccines to our cancer patients, as we wait for more data to come out from the general population, then see how safe it is and how effective (it is) in order to really extrapolate to our cancer patients.

If a patient is no longer receiving active treatment and there are no signs of active cancer, Chemaly said, they should have a good response to the vaccine, and it will likely be safe for them to receive it as well.

Now, for other patients who (are) still in the follow-up period, not really called survivors of cancer, we're going to probably provide some guidance, for example, for recipients of a stem cell transplantation. If it's been six months from allogeneic transplantation, they're stable and recovering well from after transplant, then it is probably be safe to give it to these patients, he said. Autologous transplant could be three months from the transplantation if they have no active issues, if they are still in remission and they're stable enough to receive a vaccine.

As with any vaccine, Tripathy said, some people will have reactions, but at least there are data from healthy individuals that can be shared with patients with cancer. When those data are shared with patients with cancer, however, there will be some unknowns. For instance, will patients with cancer be able to generate antibodies and develop the same protection, and might there be unique side effects that this patient population will experience.

These are things that we will have to learn as we go, and we will, Tripathy said. As the cancer centers and practices start immunizing their patients, were going to be tracking outcomes on it.

In fact, just like with any drug that receives FDA approval, there will be a process for reporting any side effects that occur when a patient receives the vaccine.

As for the individuals who developed severe allergic reactions to the vaccine in the United Kingdom, Chemaly noted that those individuals had a history of anaphylaxis, or severe allergic reactions to different antigens. And two out of those three individuals who experienced the severe reactions were already carrying an EpiPen (epinephrine), which helps to combat serious allergic reactions.

And we're prepared to intervene if someone develop(s) this kind of reaction when we give the vaccine, Chemaly said.

Everyone not just patients with cancer should expect to follow all the public health measures from wearing a mask to social distancing and frequent hand hygiene for at least another six months to one year even if vaccinated, according to Chemaly.

We need to create herd immunity (because) without herd immunity, we're not going to eliminate this virus, he said. Second, even if (you) get (a) vaccine, (it) doesn't mean (youre) not going to be exposed to the virus in the community or in your workplace. At that point, you may carry the virus and not getting sick from it or get admitted to the hospital but (you) can still transmit the virus to other people. This why masking is still so important.

Chemaly said hes received questions from patients and employees every day about their worry of receiving the vaccine. And while he said its understandable, he assures the public that the trials have been conducted under a microscope, meaning so many eyes have been watching everything that has happened.

No one is hiding anything, he said. Based on that, I advise my patients, my colleagues (and) other health care workers in the health care setting, that, what we know is (the vaccine is) safe and is effective there is no long-term side effect up to two or three months from receiving the vaccine. I, myself, feel very comfortable taking it, and I'm going to be lining up to get the vaccine as soon as it is available.

I think that we are witnessing an incredible moment in history where we rallied to do something that had never been done, and that is to get a vaccine from scratch in less than one year, Tripathy said. That is a pretty astounding technologic feat that not many people would have believed it was possible when all this started that in this short period of time; we did it.

Now, its up to patients to make an informed decision as to whether to get the vaccine, although the available data point to its potential effectiveness.

Nothing works unless you get the vaccine, he said. If you don't get the vaccine, all of this was for nothing.

However, Tripathy acknowledged why some people may be concerned and reluctant to receive the vaccine.

Things have happened in medical history where that might give some people pause, he said. There's a lot of concern about people that are underserved and minorities because there is a history of them not receiving fair treatment when it comes to medicine and clinical trials. And so, we have to go the extra mile to reassure patients. But we can't pretend that we can reassure people 100%. Just like many other decisions you make in life, you take the best information you have and you make a recommendation for other people or for yourself. All we can do is be truthful, present our recommendations and hope that a majority of people do get vaccinated.

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What Patients With Cancer, Survivors Need to Know About the Emergency Use Authorization of COVID-19 Vaccine - Curetoday.com

2020 at the U: The year in review – University of Miami

By adminDecember 16, 2020Stem Cell Medical Center

Take a look at a month-by-month recap of the biggest stories at the University of Miami during the past year.

From a pandemic that forced the migration of spring semester classes to an online environment, to a new dean for the College of Engineering, to a Super Bowl halftime performance by the Band of the Hour, to a record-breaking gift for the Sylvester Comprehensive Cancer Center, 2020 proved to be a very unusual year for the University of Miami.

January

The Planet Kreyol student organization and the Office of Multicultural Student Affairs commemorate victims of the 2010 Haiti earthquake with dance, song, poetry, and more on January 15.

With evidence mounting that climate change is worsening everything from wildfires to hurricanes, the University of Miamis Rosenstiel School of Marine and Atmospheric Science hosts the three-day Miami Climate Symposium 2020: Predicting and Living with Extremes. The summitheld Jan. 22-24explored how sea level rise, saltwater intrusion, extreme heat waves, and other conditions exacerbate tropical cyclones, storm surge, and coastal flooding, as well as analyzed adaptation policies and strategies.

A professor of jazz trumpet at the Frost School of Music and four alumni of the University of Miami receive Grammy Awards during the 62nd annual ceremony held Jan. 26 in Los Angeles. Brian Lynch, jazz trumpet professor, is honored in the category of Best Large Jazz Ensemble. Cristian Macelaru, B.M. 03; Julio Reyes Copello, M.M. 00; Carlos Fernando Lopez, B.M. 12; and Natalia Ramirez, M.A. 17, also win Grammy Awards.

Miami baseball alumnus Tommy Adams makes the largest gift in support of the baseball program by a former University baseball student-athlete, donating $1 million toward the University of Miamis Baseball Facility Enhancement Campaign.

February

Five months after Hurricane Dorian devastated the Bahamas, students enrolled in the semester-long action project interdisciplinary class offered by the Miami Herbert Business School travel to Freeport for a three-day visit, meeting with Bahamian students and local businesses to share ideas and inspire hope.

Building on the immense resources and expanding the vision of the Center for Computational Science, the University establishes the Miami Institute for Data Science and Computing to catalyze data-intensive research that will solve real-world problems and enhance the understanding of data science among students and the public.

The Lancet, one of the worlds leading medical journals, announces that Felicia Marie Knaul, director of the University of Miami Institute for Advanced Study of the Americas, will lead a new Lancet Commission examining gender-based violence and maltreatment of young people, two areas with a dearth of study and understanding.

Students from the University of Miamis Frost Band of the Hour and the color guard, as well as the Hurricanettes dancers, perform in the Pepsi Super Bowl LIV Halftime Show at Hard Rock Stadium on Feb. 2.

Redshirt senior David Dinsmore wins his fourth straight gold medal in the mens platform on Feb. 21, capturing top honors at the 2020 ACC Swimming and Diving Championships.

At the Dolphins Cancer Challenge, Team Hurricaneswith 1,250 strongjoined thousands of others on Feb. 29 to run, walk, and ride to fight cancer and support the Sylvester Comprehensive Cancer Center.

March

For the health and well-being of the campus community, the University extends spring break for students through March 22, announcing that classes will resume on March 23 but strictly in online environments through at least April 4. Shortly thereafter, with COVID-19 cases surging across the nation, the University, in accordance with public health guidance to reduce density on campus, extends online instruction through the remainder of the spring semester and implements partial closing of on-campus housing.

April

An international team of scientists led by Dr. Camillo Ricordi, director of the Diabetes Research Institute and Cell Transplant Center at the University of Miami Miller School of Medicine, is granted immediate FDA authorization for a 24-patient clinical trial to test the safety and exploratory efficacy of umbilical cord-derived mesenchymal stem cells to block the life-threatening lung inflammation that accompanies severe cases of COVID-19.

Joined by the Rapid Defense Network in New York, the Southern Poverty Law Center, and others, the School of Laws Immigration Clinic files a lawsuit on April 13 accusing U.S. Immigration and Customs Enforcement authorities of ignoring COVID-19 guidelines in three Florida detention centers.

Debbie Ajagbe is named the 2020 ACC Indoor Track Scholar-Athlete of the Year on April 17, with five other Miami women joining her on the All-ACC Academic Team for Indoor Track and Field. Earlier in the year, Ajagbe, a mechanical engineering major, earned both ACC Womens Field Performer of the Year and ACC Championship Field MVP honors, winning both the weight throw and shot put at the conference championships.

May

The 180 graduates of the Miller School of Medicines Class of 2020 celebrate their newly minted degrees during a virtual commencement on May 9.

Arva Moore Parks, a prominent historian and preservationist, who served on the University of Miami Board of Trustees for 26 years and wrote several books on Greater Miami, Coral Gables, and University history, passes away on May 10.

Nine University of Miami Athletics programsmens basketball, mens and womens cross country, mens diving, golf, rowing, mens and womens tennis, and womens track and fieldare recognized for perfect single-year scores of 1,000 in the 2018-19 Academic Progress Report released May 19 by the NCAA.

UMTVs first Black show, The Culture, is nominated by the Suncoast Chapter of the National Academy of Television Arts & Sciences for a student production award in the magazine program category.

Brian Van Belle and Chris McMahon, two of the Miami Hurricanes best pitchers, are named All-Americans by Collegiate Baseball Newspaper on May 26. Both Van Belle and McMahon earned spots on the second team after posting brilliant performances in the abbreviated 2020 season that was halted due to the COVID-19 pandemic.

June

Pratim Biswas, the Lucy and Stanley Lopata Professor in the McKelvey School of Engineering at Washington University in St. Louis and a pioneer in his field recognized for applying aerosol science and engineering to multiple areas, is named dean of the University of Miami College of Engineering.

The University of Miami Board of Trustees elects six new members to its ranks with expertise in business, finance, law, technology, and strategic planning. Patricia Menendez-Cambo, Adam E. Carlin, Jose R. Mas, Alice S. Vilma, Carolyn B. Lamm, and Jordan Rhodes were elected to the Board on June 19.

Dr. Judy Schaechter, chair of the Miller School of Medicine Department of Pediatrics, is named a 2020-21 Health Policy Fellow by the Robert Wood Johnson Foundation and the National Academy of Medicine. The prestigious one-year fellowship in Washington, D.C., will enable her to expand her longtime involvement in health policy and child policy at the local and state levels to the federal level.

July

The University becomes one of 89 locations around the nation, and one of only six in Florida, to enroll volunteers for the first Phase 3 clinical trial of a COVID-19 vaccine. The trial, part of the National Institutes of Health COVID-19 Prevention Trials Network, tested a vaccine developed by scientists at the NIHs National Institute of Allergy and Infectious Diseases and collaborators at biotechnology company Moderna, Inc. Vice President Mike Pence visited the Miller School of Medicine along with Florida Gov. Ron DeSantis on July 27 to thank the University for its participation in the trial.

The National Oceanic and Atmospheric Administration selects the Rosenstiel School of Marine and Atmospheric Science to host the Cooperative Institute for Marine and Atmospheric Studies, which will bring together the research and educational resources of 11 partner universities to increase scientific understanding of the Earths oceans and atmosphere within the context of NOAAs mission. The selectionmade through an open, competitive evaluationcomes with an award of up to $310 million over the course of five years, with the potential for renewal for another five years based on successful performance.

Amid ongoing nationwide protests against police brutality sparked by the tragic death of Minneapolis resident George Floyd in May, President Julio Frenk reaffirms his commitment to racial and ethnic equality, outlining in a letter sent to all students, faculty, and staff a 15-point plan the University will implement to support racial equality, inclusion, and justice across the institution and in the greater South Florida community.

Patti Herberta longtime University of Miami alumna and benefactor who, along with her husband Allan, donated millions of dollars to the institution, helping to transform academics and student lifepassed away on Monday, July 27. She was 84.

University of Miami Libraries launches Documenting COVID-19: South Floridas Pandemic Experience. Through community-generated and community-contributed content that will be made available through digital collections and by visiting the library, the initiative will chronicle how local communities are dealing with the crisis.

August

In a move to support a safe learning and working environment for students, faculty, and employees, the Butler Center for Service and Leadership establishes a new team of public health ambassadors to support the Universitys COVID-19 reopening and operating plan. The 75 students who made up the new Public Health Ambassadors Program during the fall semester enforced guidelines on the Coral Gables Campus by offering support and utilizing peer-to-peer influence to encourage members of the campus community to engage in the healthy behaviors of physical distancing, wearing face coverings, and hand washing/sanitizing.

Lakeside Village, a 12-acre facility on the shores of Lake Osceola in the heart of the Coral Gables Campus, officially opens its doors on Aug. 13 to greet the inaugural class of students to the transformative housing complex.

Featuring a mix of virtual and in-person instruction, the first day of fall semester classes begins on Aug. 17. On the Coral Gables Campus, a number of safety protocolsfrom mandatory mask-wearing to social distancinghelp ensure the well-being of students, faculty, and staff.

The Miller School of Medicine Class of 2024 launches the innovative NextGenMD Curriculum, which focuses on health system science and features an enhanced emphasis on mentorships. The students will be significantly better prepared to respond to COVID-19 and to the public health challenges that will follow.

The Miller School of Medicine becomes one of a few medical schools across the nation selected by the National Institutes of Health to test the effectiveness of treating COVID-19 patients with convalescent plasma.

Following a limited opening in April 2020, Canes Central, a new student-centered, service-oriented department, fully opens. It offers both in-person and online undergraduate and graduate students assistance on matters relating to registration and records, billing and payment, financial aid, and Cane Cards.

Legendary Miami Hurricanes baseball coach Jim Morriswho in his 25 seasons at the University of Miami won 1,090 games, made the NCAA postseason 23 straight years, reached the College World Series 13 times, and won national championships in 1999 and 2001is voted into the 2020 induction class of the National College Baseball Hall of Fame.

September

Sylvester Comprehensive Cancer Center at the University of Miami Leonard M. Miller School of Medicine receives a landmark gift of $126 million. The groundbreaking donationthe single largest in the University of Miamis 95-year historywill accelerate breakthrough advances in finding cures for cancer and expand innovative treatment options for cancer patients.

The University of Miami jumps eight spots to No. 49 in U.S. News & World Reports 2021 Best Colleges issue, placing the institution back among the 50 top-tier colleges and universities. This jump in the rankings reflects our commitmenteven in these unprecedented timesto comprehensive excellence and selective preeminence, said President Julio Frenk.

Sylvester Comprehensive Cancer Center, part of the University of Miami Health System, opens the Dwoskin Proton Therapy Center on Sept. 15. The new state-of-the-art facility treats patients with proton therapy, an advanced type of low-dose radiation that is extremely precise and two-thirds the speed of light.

Physician-researchers with the Miller School of Medicine begin a new Phase 3 clinical trial to test another investigational vaccine for COVID-19. Part of a large-scale international trial in partnership with Janssen Pharmaceuticals, the clinical trial to test the Janssen vaccine is the Miller Schools second human study of its kind.

October

The University of Miami becomes the first college testing site for a quick, easy, and cost-effective Israeli-produced COVID-19 breath analyzer that could revolutionize coronavirus testing if approved by the FDA.

In a Miami Herbert Business School webinar held Oct. 8, U.S. Secretary of Health and Human Services Alex Azar highlights the administrations effort to restructure the health care system to combat the COVID-19 pandemic and support countries in Latin America and the Caribbean.

November

Taking their dedication to fighting cancer to a new level, the Miami Dolphins pledge a transformational $75 million gift to Sylvester Comprehensive Cancer Center at the University of Miami Leonard M. Miller School of Medicine, South Floridas only NCI-designated cancer center.

Musicians from the Frost School of Music join Dean Shelly Berg and celebrity musicians to participate in a benefit concert on Thanksgiving Day in support of nurses. The livestreamed Nurse Heroes Live! concert raises funds for the Nurse Heroes Foundation, an initiative working to support and honor nurses.

December

Four extraordinary University of Miami alumniJose R. Mas, Jackie Nespral, Hilarie Bass, and Jaret L. Davisshare their advice with more than 5,000 students at four virtual commencement ceremonies held Dec. 10 and 11.

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Five Mobile County hospitals to get Pfizer vaccine this week – AL.com

By adminDecember 16, 2020Stem Cell Medical Center

Five hospitals in Mobile County will be part of the Alabama launch of the Pfizer COVID-19 vaccine, according to an official with the Mobile County Health Department.

The hospitals will give the initial doses to frontline healthcare workers

Dr. Scott Chavers, an epidemiologist with the Mobile County Health Department, told AL.com on Friday that Mobile Infirmary, University Hospital, USA Childrens and Womens Hospital, Providence Hospital and Springhill Medical Center are on the states initial distribution lists.

He said the first phase of distribution, called Phase 1a, will include 5,800 initial vaccine doses that will be distributed to the Mobile County area and will be specifically given to healthcare workers.

The Alabama Department of Public Health, last week, confirmed the state is expected to receive about 40,000 initial doses overall.

They were chosen because of their capability of storing the vaccine at the ultralow temperatures required for the Pfizer vaccine at a negative 80 degrees Celsius, Chavers said. Storage at that temperature is not commonly used (at hospitals). They are typically used for stem cell and tissue research, and only research institutions have (storage units at that low of a temperature).

Mobile Infirmary is expected to received 1,000 doses this week, and will begin providing them to healthcare workers early Wednesday morning. Infirmary Health, which manages the Mobile hospital, will also provide the vaccine to frontline health care workers at Thomas Hospital in Fairhope and North Baldwin Infirmary in Bay Minette.

Hannah Peterson, spokeswoman with Infirmary Health, said the goal this week is to provide approximately 1,000 vaccines this week to Infirmary Health employees, EMS personnel and other local providers and adopted hospitals.

It was unclear on Monday morning when USA Health would begin providing its initial dosages. CEO Owen Bailey, late last week, said he believe a plan would be finalized after the Food and Drug Administration officially green-lighted the vaccine, which occurred Friday.

Its given folks a lot of hope that weve reached this point, said Bailey, following a ribbon cutting ceremony on a new $20 million trauma center that will be operational by mid-January.

Chavers said the second distribution channel, which is also part of the countys Phase 1a rollout, will include pharmaceutical chains Walgreens and CVS. The companies struck deals with the federal government to vaccinate staff and residents at long-term care facilities around the country. Most states, including Alabama, have put the facilities at the top of the priority list for vaccines.

They are in the first wave so that when the Pfizer vaccine shows up for the hospitals, within a couple of days, it will go through CVS and Walgreens (and be administered at the long-term care facilities), Chavers said.

The Pfizer vaccine requires a second dose 21 days after the initial shot. Chavers said the rollout for the second dose will occur next month and will be similar to what is occurring this week: Hospitals and long-term care providers are the priority.

Related content: First vaccines in Birmingham area slated to reach UAB next week

The next phase of vaccine distribution will include the Moderna vaccine once its given the go-ahead from the FDA. An FDA advisory panel is expected to determine whether to authorize the vaccine later this week. Clinical trials involving 30,000 participants found the Moderna vaccine to be 94.1 percent effective.

Chavers said the Moderna vaccine will not require extreme cold for storage, and will be easier to distribute.

It needs to be held at a negative 10 degrees Celsius so its not as stringent, he said. With (the) Pfizer vaccine, you have six hours to use it whereas the Moderna vaccine, you have 30 days. The Moderna vaccine is much more aligned with traditional vaccine delivery.

Healthcare workers at outlying hospitals across the state will be among those receiving the limited numbers of the Moderna vaccine, said Chavers, as they are not part of the initial distribution of the Pfizer vaccine.

But he said the exact distribution of the Moderna vaccine has not been laid out.

Well have more information (later this week) on how that will be distributed, Chavers said. One of the critical messages is that this will not be a fast process

The general public is likely not going to see vaccines until late spring. Im talking about March or April. You cannot vaccinate everyone on the same day. It will be a months-long process.

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Five Mobile County hospitals to get Pfizer vaccine this week - AL.com

Israeli biotech firm’s ALS treatment shows safety of use in trials – The Jerusalem Post

By adminDecember 16, 2020Embryonic Stem Cells

Ness Ziona-based biotech firm Kadimastem has shown encouraging results of Cohort B of its Phase 1/2a clinical trial for AstroRx, its Amyotrophic Lateral Sclerosis (ALS) treatment trial. The objective of this trial was to evaluate the safety of their treatment, with a secondary objective of the trial of estimating its preliminary efficacy. The treatment was developed by Kadimastem and contains functional, healthy astrocytes (nervous system support cells) derived from Human Embryonic Stem Cells (hESC) that aim to protect diseased motor neurons. The company's technology allows injecting AstroRx into the spine of the patient, to slow down the progression of the disease. The treatment has been granted orphan drug designation by the FDA for the treatment of ALS. The five patients included in this part of the trial showed no serious adverse effects during the half a year follow up after the treatment was given. The rate in which it slows down the disease was also tested for, using the ALS Functional Rating Scale-Revised (ALSFRS-R), which tracks ALS progression, and has shown that after the treatment was given, there was a 45% decline in the disease's progression rate. At the end of the 6-month post-treatment period, the rate of ALSFRS-R progression was similar to the rate that was measured before treatment. "The results after 6 months of follow up are encouraging, as they suggest a clinically meaningful signal of effect for 3 months by a single administration of AstroRx and confirm the safety of AstroRx," said Dr. Marc Gotkine, Head of the ALS Clinic at the Department of Neurology at Hadassah Medical Center in Jerusalem, and the Principal Investigator of the trial.

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Israeli biotech firm's ALS treatment shows safety of use in trials - The Jerusalem Post

Avadel Pharmaceuticals Announces Submission of New Drug Application for FT218 to the FDA

By adminDecember 16, 2020Global News Feed

DUBLIN, Ireland, Dec. 16, 2020 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL) today announced the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for FT218, an investigational, once-nightly formulation of sodium oxybate designed to treat excessive daytime sleepiness and cataplexy in adults with narcolepsy.

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Avadel Pharmaceuticals Announces Submission of New Drug Application for FT218 to the FDA