2019 Was a Year for Advancements in Infectious Disease – Pharmacy Times

Jeannette Y. Wick, RPh, MBA, FASCP

Often, when American pharmacists think about advancements, we think locally or nationally. In the case of infectious disease, however, we must think globally. When it comes to infectious agents, we need to be more concerned about outbreaks, epidemics, and drug resistance all over the world, as these could eventually affect people in the United States.1 Issues including HIV, immunization, malaria, travelers health, and tuberculosis are of tremendous concern,1 and some breakthrough treatments have been approved or are close to approval. Other advancements this year offer hope for people who develop pneumonia.

Lets look at 5 developments.

1. Epidemic forecasting. Infectious disease outbreaks are major contributors to global morbidity and mortality. Real-time epidemic forecasting, which comprises systems that use predictive modeling to help health care providers prepare and respond, continues to advance. These systems rely on epidemic surveillance and consider several factors, such as agent and host mobility, environmental and host susceptibility, health care capacity, pathogen transmissibility, and population density. For predictive modeling to operate effectively, health care providers need standardized case definitions, and they need to share their data quickly.2 Investigators have used epidemic forecasting in food animal herds, which are easier to control and smaller, for years and are now implementing these models in human populations. Investigators have also had good results in modeling viral outbreaks such as dengue, Ebola, and influenza.3 And Japanese investigators used a predictive model to anticipate the likely size of a measles epidemic in real time. This model was used to evaluate public health control measures.4

2. Inhaled antibiotics to treat pneumonia. The leading cause of death from infectious disease is pneumonia.5 The lungs anatomical and physiological features limit antimicrobial access to the infection site, limiting intrapulmonary penetration of most systemically administered antimicrobials (eg, aminoglycosides and -lactams).6 The marrying of drugs and devices has changed outpatient care in a number of conditions, and infectious disease is no exception. Antimicrobials that can be inhaled allow noninvasive, sustained drug concentrations in the pulmonary area, with minimal systemic exposure. Inhaled therapy also raises the possibility of using drugs that have dose-limiting toxicities when given systemically, so patients can receive therapeutic or supratherapeutic doses with few systemic adverse effects.7,8 A number of inhaled medications are available, including:9-12

3. A possible cure for Ebola. Ebola is a viral hemorrhagic fever in humans and other primates. The FDA has granted breakthrough therapy designation for the investigational Ebola treatment mAb114.14 Tested in patients who contracted Ebola during the ongoing outbreak in the Democratic Republic of the Congo (DRC), mAb114 reduced the mortality rate. Investigators isolated this monoclonal antibody from a survivor of the 1995 DRC Ebola outbreak. This survivor had antibodies 11 years after infection, indicative of a strong immune response. In a randomized controlled trial that is continuing in DRC, mAb114-treated patients had an overall mortality rate of 34% compared with 53%, 49%, and 29% for 3 other drugs in the trial. Patients with low viral loads responded best, with a mortality rate of 11%.14

4. Pretomanid for highly drug-resistant tuberculosis (TB). Globally, about 40,000 individuals have highly drug-resistant TB.15 Although only a few cases have been reported in the United States, that is how outbreaks start: with a few cases. Pretomanid is an oral nitroimidazooxazine antimycobacterial. It has been given in the bedaquiline, pretomanid, and linezolid (BPaL) regimen. The Global Alliance for TB Drug Development tested BPaL in a trial of 109 patients. Remarkably, 89% were cured. Previously, best cure rates with other antimicrobials hovered at 34% overall. The BPaL combinations most common adverse effects are peripheral and optic nerve damage, acne, anemia, nausea, vomiting, headache, and abnormal liver tests.15,16 The combination has also been associated with QT prolongation.15

5. Lefamulin for adults with community-acquired bacterial pneumonia (CABP). The FDA approved lefamulin for CABP caused by Chlamydophila pneumoniae, Haemophilus influenzae, Legionella pneumophila, Mycoplasma pneumoniae, Staphylococcus aureus (methicillin-susceptible isolates), and Streptococcus pneumoniae.17 It is available as an injection for intravenous administration or a 600-mg tablet administered orally every 12 hours for 5 days. The drug, a novel pleuromutilin antibiotic, offers an alternative, though a costly one, to moxifloxacin.18,19

CONCLUSIONBreakthrough developments have been few and far between in infectious disease for numerous reasons. This year, investigators have made inroads in important areas, giving reason to hope for more breakthrough therapies in 2020.

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2019 Was a Year for Advancements in Infectious Disease - Pharmacy Times

Where Are They Now? Kira’s quest continues – Kamloops This Week

Danica Crawford undertook a brave initiative in March of 2018.

She asked for donations so her five-year-old daughter Kira, who has cerebral palsy, could undergo potentially life-altering treatments.

The community responded.

In 21 months, donors from Kamloops and beyond have given more than $62,000 to help Kira heal and, with support from her mother, family and the community, Kira has made progress.

Now seven years old, Kira has undergone both of the therapies her mother planned to pursue when launching the campaign.

First up was a procedure in July 2018 called selective percutaneous myofascial lengthening (SPML), a surgical treatment intended to relieve spasticity in her legs.

Cerebral palsy often causes problems with body movement, posture, speech, swallowing and muscle stiffness.

She gained a lot of core strength because that tension was relieved. So the rest of her body relaxed and she gained strength and stability, Crawford said.

Those gains meant Kira could begin using a walker that she could navigate in and propel by herself, requiring only minor course corrections from mom or help getting around obstacles when the two are walking together.

That is a huge gain just in itself, Crawford said.

The surgery has also meant Kira can keep herself upright for longer.

Before, regular therapy over the course of a year allowed Kira to sit independently although with her head hanging down for up to eight seconds.

Now, with better neck strength and control and even further therapy in Toronto earlier this year, Kira can hold her head up by herself. In October, she sat by herself for one minute.

Over the past year, Kira has also improved her finer motor skills and can hold a pencil for up to five minutes, can bring her hand to her mouth and can hold her toys.

Crawford said she is able to speak more than she could before and can spend more time in her classroom at school.

Thats a huge change, to be able to tolerate situations like that, Crawford said. Now shes in the classroom, proving she knows her numbers and showing her cognition and understanding of what the other kids are learning.

Its huge she can express those things now.

In August, Kira underwent her other planned therapy stem cell treatment at Duke University in North Carolina.

The effects of that treatment have not necessarily taken effect just yet. Doctors told Crawford signs of improvement likely wouldnt appear until after January.

But Crawford said Kira has seen the foundations for some greater changes already and the hope is for a 30 per cent overall improvement, not just in motor skills and communication, but in her whole life experience.

The treatment helps with the formation of new neural pathways, meaning Kiras ongoing physical and cognitive therapies are that much more important in the coming months.

When KTW asked Crawford for an update on her daughter, she gathered her thoughts on what it has felt like to receive this much support from the community.

It has been a challenge. As a family who is requesting support, you feel like every purchase youre making you second-guess it, Crawford said. Everything you do, you feel a little funny about. The fact is that Kiras money is Kiras money and it goes toward helping Kira directly.

Its also the most beautiful experience and I almost feel like being shown so much love and so much support it has made life so beautiful for us. I feel like were really lucky to receive that way.

Crawford said she is eager to pay it forward in the small ways she can sharing Kiras experience through social media and connecting with other families in similar situations.

Its been the most beautiful experience of my life and its been the most beneficial experience for Kiras life. She would not be doing these things if it werent for the support people have given, Crawford said.

Updates on Kiras progress can be found on the Facebook page Kira Shines, which can be found by clicking here. Her crowdfunding campaign can also be found on GoFundMe by clicking here.

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Where Are They Now? Kira's quest continues - Kamloops This Week

2019: The year gene therapy came of age – Eyewitness News

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

Picture: Supplied.

WASHINGTON, United States - In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta-thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

CURES

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face gruelling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in the hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

**BIOTERRORISM **

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his ex-communication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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2019: The year gene therapy came of age - Eyewitness News

In case you missed them: Spectrum’s standout stories from 2019 – Spectrum

Spectrum published hundreds of articles this year on a range of topics, from characterization of candidate genes to immune-system connections. We are proud of all of them, but some in particular stand out: They expose harmful therapies such as chiropractic and stem-cell treatments, upend conventional wisdom about autism, expose uncomfortable truths or adroitly explain complex theories about the condition.

Our staff and some of our readers picked the following seven as particular favorites from the year.

Autism, explained

How might a brain-signaling imbalance underlie autism? What is the female protective effect and, conversely, an extreme male brain? We expanded our compendium of autism explainers this year to include some of the most popular theories about the condition.

False hope for autism in the stem-cell underground

Clinics offering stem-cell treatments for autism are proliferating, and desperate parents pay thousands of dollars to have these products injected into their children despite a lack of evidence that they help. Many, in fact, have the potential to cause serious harm, from introducing life-threatening infections to seeding autoimmune disorders. In this story, investigative reporter Brendan Borrell traces the provenance of one childs treatments through a cast of rogue characters and calls attention to the fact that the products are, as one of his sources says, basically afterbirth thrown in a blender.

Can preventing seizures alter the course of autism?

This story sprang to life when reporter Jessica Wright observed experimental brain surgery on a 12-year-old boy named Kevin Lightner. Kevin has dup15q syndrome, a rare genetic condition that often causes seizures and autism, and his case presented a prime opportunity to explore a provocative question: Can epilepsy lead to or at least contribute to autism? Wright followed Kevin and his family through a risky procedure to implant a responsive neurostimulation device into his brain, and over the weeks that followed.

When autistic people commit sexual crimes

Many autistic people become embroiled in the criminal justice system for sexual behaviors, including collecting child pornography, stalking and sexual assault. Some go to prison, and others become registered sex offenders a status that can prevent them from receiving state services for the rest of their lives. But as Melinda Wenner-Moyer explores in this story, autistic people may engage in these behaviors without understanding the implications of their actions or the law. Some experts are calling for a change in how the criminal justice system treats these autistic people, and for more sexual education for autistic teens.

Autism, through the eyes of a computer

Clinicians are the main arbiters of autism traits. They use their expertise to diagnose autism and judge its severity. But a growing cadre of scientists is betting that computers could do some parts of these tasks better. In this story, reporter Nicholette Zeliadt explores the use of wearable sensors and other devices to track autism traits over time as they collect data from autistic people in their homes and schools. These measurements may never replace the judgment clinicians hone through years of experience, but they may ease the workload of experts and the wait time for people who need evaluations.

Large study supports discarding the term high-functioning autism

Autistic people who excel academically are sometimes referred to as high functioning. The problem is, many dont function at all well: They struggle with everyday tasks, from getting dressed to taking the bus. This story underscores the gaping chasm between intelligence and daily living skills, and the crescendo of voices calling to abandon the high functioning label.

Studies of autism treatments lack standard yardsticks

An analysis of 36 years of clinical trials showed that researchers do not use a consistent set of tools to measure the efficacy of autism treatments. Nearly 70 percent of the tools were used in just one study, making it difficult to compare the treatments. And only three validated tools that measure core autism traits were used in more than 5 percent of the studies. But these tools are not designed to measure treatment outcomes, so they may miss subtle signs that a drug, dietary supplement or psychotherapy is working.

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In case you missed them: Spectrum's standout stories from 2019 - Spectrum

Stem Cell Therapy Market Consumer Outlook 2025 | MEDIPOST Co., Ltd., Osiris Therapeutics, Inc. – Market Research Sheets

Stem Cell Therapy Market: Snapshot

Of late, there has been an increasing awareness regarding the therapeutic potential of stem cells for management of diseases which is boosting the growth of the stem cell therapy market. The development of advanced genome based cell analysis techniques, identification of new stem cell lines, increasing investments in research and development as well as infrastructure development for the processing and banking of stem cell are encouraging the growth of the global stem cell therapy market.

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One of the key factors boosting the growth of this market is the limitations of traditional organ transplantation such as the risk of infection, rejection, and immunosuppression risk. Another drawback of conventional organ transplantation is that doctors have to depend on organ donors completely. All these issues can be eliminated, by the application of stem cell therapy. Another factor which is helping the growth in this market is the growing pipeline and development of drugs for emerging applications. Increased research studies aiming to widen the scope of stem cell will also fuel the growth of the market. Scientists are constantly engaged in trying to find out novel methods for creating human stem cells in response to the growing demand for stem cell production to be used for disease management.

It is estimated that the dermatology application will contribute significantly the growth of the global stem cell therapy market. This is because stem cell therapy can help decrease the after effects of general treatments for burns such as infections, scars, and adhesion. The increasing number of patients suffering from diabetes and growing cases of trauma surgery will fuel the adoption of stem cell therapy in the dermatology segment.

Global Stem Cell Therapy Market: Overview

Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.

Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.

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Global Stem Cell Therapy Market: Key Trends

The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.

On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.

Global Stem Cell Therapy Market: Market Potential

A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.

In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.

Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.

Global Stem Cell Therapy Market: Regional Outlook

The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.

Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.

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Global Stem Cell Therapy Market: Competitive Analysis

Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.

Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.

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TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

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Stem Cell Therapy Market Consumer Outlook 2025 | MEDIPOST Co., Ltd., Osiris Therapeutics, Inc. - Market Research Sheets

Kadimastem Announces the Completion of Treatment for Cohort B of Its Phase 1/2a Clinical Trial in ALS Patients – P&T Community

NESS ZIONA, Israel, Dec. 23, 2019 /PRNewswire/ --Kadimastem Ltd.(TASE: KDST), a clinical stage cell therapy company, today announced that treatment with a single dose of 250x10[6] AstroRxcells to all 5 ALS patients in cohort B has been completed.

The primary objective of the trial is to evaluate the safety of injecting AstroRx, an "off-the-shelf" clinical-grade astrocyte cell product, developed and manufactured by the Company, into the cerebro-spinal fluid (CSF) of ALS patients. A secondary objective of the trial includes preliminary efficacy.

The clinical trial is progressing as planned. Cohort B patients will be monitored for 6 months following treatment.In addition, the first patient of cohort C has already been enrolled. Cohort C is designed to assess long term safety and efficacy of a repeated dose administration of AstroRxin 2 consecutive injections separated by an interval of 2-3 months. As previously reported in September 2019, positive interim results of cohort A demonstrated safety and statistically significant preliminary efficacy of a single dose of 100x10[6] AstroRxcell administration. In light of these positive results, the company is taking the necessary steps to obtain approval to a proposed protocol amendment according to which the repeated administration will comprise of the same dose (100x10[6]).

Results of cohort A and B are expected to be reported as planned and as previously announced, by the end of 2019 and during August 2020, accordingly. Results of cohort C are expected in the first half of 2021.

Rami Epstein, CEO of Kadimastem, commented: "Completing treatment for the additional 5 ALS patients in Cohort B, for a total of 10 patients treated with our product in our clinical trial, serves as an additional demonstration of our ability to develop and produce high quality clinical grade cells and takes us a significant step forward in our path to bringing innovative cure to ALS. The expected completion of cohort B 6-months follow-up period will allow us to assess the safety and preliminary efficacy of 250x10[6]cells, compared to that of the lower dose administered in cohort A."

Prof. Michel Revel, Founder and CSO of the Company, added: "The results of the next treatment group, Cohort C, in which each patient will be treated with 2 consecutive injections separated by an interval of 2-3 months,will allow us to assess the possible prolonged efficacy of the repeated dose, compared to the single dose treatment provided in cohorts A and B. The results that will be obtained from the different cohorts, will support us in the process of defining the dose and treatment regimen that will lead to most favorable results for patients over time."

About AstroRx

AstroRxis a clinical grade cell therapy product developed and manufactured by Kadimastem in its GMP-compliant facility, containing functional healthy astrocytes (nervous system support cells) derived from human Embryonic Stem Cells (hESC) that aim to protect diseased motor neurons through several mechanisms of action. The Company's technology enables the injection of AstroRxcells into the spinal cord fluid of patients suffering from Amyotrophic Lateral Sclerosis (ALS) with the goal of supporting the malfunctioning cells in the brain and spinal cord, in order to slow the progression of the disease and improve patients' quality of life and life expectancy. AstroRxhas been shown to be safe and effective in preclinical studies. AstroRxhas been granted orphan drug designation by the FDA.

About ALS

Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive fatal neurodegenerative disease causing disfunction in the upper and lower motor nerves that control muscle function. ALS leads to muscle weakness, loss of motor function, paralysis, breathing problems, and eventually death. The average life expectancy of ALS patients is 2-5 years. According to the ALS Therapy Development Institute, it is estimated that there are approximately 450,000 ALS patients worldwide of which 30,000 reside in the US. According to the ALS Foundation for Life, the annual average healthcare costs of an ALS patient in the US are estimated at US$ 200,000. Thus, the annual healthcare costs of ALS patients in the US alone amount to US$ 6 Billion.

About Kadimastem

Kadimastem is a clinical stage cell therapy company, developing and manufacturing "off-the-shelf" allogeneic proprietary cell products based on its platform technology for the expansion and differentiation of Human Embryonic Stem Cells (hESCs) into clinical grade functional cells. AstroRx, the Company's lead program, is a clinical-grade astrocyte cell therapy for the treatment of ALS, currently undergoing a Phase 1/2a clinical trial. In addition, preclinical trials are ongoing with the Company's IsletRx pancreatic functional islet cells for the treatment of insulin dependent diabetes. Kadimastem was founded by Prof. Michel Revel, CSO of the Company and Professor Emeritus of Molecular Genetics at the Weizmann Institute of Science. Prof. Revel received the Israel Prize for the invention and development of Rebif, a multiple sclerosis blockbuster drug sold worldwide. Kadimastem is traded on the Tel Aviv Stock Exchange (TASE: KDST).

Forward Looking Statement

This document may include forward-looking information as defined in the Securities Law, 5728 1968. Forward-looking information is uncertain and mostly is not under the Company's control and the realization or non-realization of forward-looking information will be affected, among other things, by the risk factors characterizing the Company's activity, as well as developmentsin the general environment and external factors affecting the Company's activity. The Company's results and achievements in the futuremay differ materially from any presented herein and the Company makes no undertaking to update or revise such projectionor estimate and does not undertake to update this document. This document does not constitute a proposal to purchase the Company's securities or an invitation to receive such offers. Investment in securities in general and in the Company in particular bears risks. One should take into account that past performance does not necessarily indicate performance in the future.

Company Contacts:

Yossi Nizhar, CFO y.nizhar@kadimastem.com+972-73-797-1604

Investor Relations and Financial Media Meirav Gomeh-Bauer meirav@bauerg.com+972-54-476-4979

Global Media & Collaborations Dasy Mandel, Director of Business Development d.mandel@kadimastem.com +972-73-797-1613

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SOURCE Kadimastem

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Kadimastem Announces the Completion of Treatment for Cohort B of Its Phase 1/2a Clinical Trial in ALS Patients - P&T Community

Leukaemia and lymphoma have a good survival rate – The Star Online

Of all blood cancers, leukaemia and lymphoma are among the most curable.

However, many people, including doctors, still believe the disease leads to immediate death.

This is no longer true today as they are not fatal.

With optimal treatment, the majority of patients go into remission and are considered cured.

These two cancers have been more extensively studied than other forms of cancer, due to the ease in obtaining samples from blood, bone marrow or lymph nodes, spurring the advent of novel targeted therapies for a cure, says consultant haematologist Dr Ng Soo Chin.

Most blood cancers start in the bone marrow, where blood is produced.

Bone marrow contains stem cells, which mature and develop into red blood cells, white blood cells or platelets.

In most blood cancers, normal cell development is interrupted by the uncontrolled growth of an abnormal type of a particular blood cell.

These abnormal blood cells, which are cancerous, prevent your blood from performing many of its functions, like fighting off infections or preventing serious bleeding.

Leukaemia or white blood is classified into acute and chronic disease, which is then divided further into subtypes: acute lymphocytic leukaemia, acute myeloid leukaemia, chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML).

The presentation between acute and chronic leukaemia differs.

The acute person will tell you he was well a week ago and is now down with symptoms such as lethargy, anaemia and recurrent infection.

Suddenly, he may look pale, so we check his blood count for any abnormalities. A bone marrow exam will further confirm whether it is acute.

With chronic leukaemia, the patient can be unwell for a couple of months.

We are increasingly picking up cases early because of blood test availability.

The survival rate has improved tremendously for acute leukaemia, with more than 50% fully cured because bone marrow transplants are easily available in the country.

For CLL and CML, 95% of patients are alive at the 10-year mark, says Dr Ng.

Generally, chronic leukaemia patients belong to the older age group (50 years and above), but acute leukaemia can occur in all ages.

Leukaemia symptoms are often vague and not specific, so its easy to overlook them as they may resemble symptoms of the flu and other common illnesses.

In fact, chronic leukaemia may initially produce no symptoms and can go unnoticed or undiagnosed for years.

Lymphomas, a type of blood cancer that begins in a subset of white blood cells called lymphocytes, can be classified into Hodgkins and non-Hodgkins.

The main difference between Hodgkins and non-Hodgkins lymphoma is the specific lymphocyte each involves.

Lymphocytes are an integral part of your immune system, which protects you from germs.

Five-year survival rates are high with Hodgkins lymphoma at 86% and non-Hodgkins lymphoma at 70%.

You can beat the disease even if it is detected at a late stage.

Multiple myeloma, which is the third kind of blood cancer, forms in a type of white blood cell called a plasma cell.

Patients often complain of bone pain, and unfortunately, this type of cancer has no cure.

Blood cancers typically involve abnormal white blood cells and can affect paople of all ages, depending on the type of cancer. 123rf.com

Fear of treatment

Chemotherapy is a much dreaded word among cancer patients.

But with advances in medicine, newer chemotherapy-free treatments are now available.

Dr Ng says, Traditionally, cancer is treated via surgery or radiation the layman says we fry and poison them, which is not far from the truth!

Radiation means burning the cancerous area, but a lot of times, the cancer can also be present elsewhere, so there is limitation to this treatment.

With chemotherapy, we use cytotoxic (cell-killing) drugs they go in and knock off both cancer and normal cells.

The short-term effects include vomiting, hair loss, appetite loss and weight loss.

But as doctors, we are looking at a different perspective. We are more worried about white cells dropping (neutropenia) because the patient can pick up an infection that can potentially kill him.

Neutropenia is a condition that results when the body does not have enough neutrophils, a type of white blood cell that is an essential first line of defence against infections.

Thats one risk of chemotherapy, although we can now improve neutropenia by giving a growth factor injection.

But for certain cancers, we need to step up the drugs.

He adds: We are scared of neutropenia, but patients are more concerned about bodily changes.

The older ones get upset over losing hair because they cannot take it when others ask them what has happened to their hair.

Young people are not as concerned with hair loss because it can be trendy.

We understand that chemotherapy is less than pleasant and strong doses can impair fertility in young patients, especially women.

Despite current technology, only one-third of patients are successful in freezing their eggs.

What he is concerned about is that chemotherapy can actually increase the patients risk of getting another cancer, especially blood cancer.

It can happen the day after! says Dr Ng.

Most experts believe chemotherapy damages stem cells, so if youre unlucky, you might get acute myeloid leukaemia after undergoing chemotherapy for breast cancer.

Its just like crossing the road there is always a risk of being knocked down.

All our cells have a biological clock and there is an orderly exchange of old and new cells.

But with blood cancers such as leukaemia, there is a clone of abnormal cells.

Cancer cells have an advantage over normal cells because they can survive longer.

Chemotherapy is still needed to treat most acute blood cancers, although if the mutation is known, targeted therapies can be applied.

For chronic blood cancers, there is no need for chemotherapy. Oral drugs are enough to combat the disease.

Eventually, many patients are able to wean off the drugs.

As we may be aware, immunotherapy is the buzzword in cancer treatment today.

Also called biologic therapy, it is a type of cancer treatment that boosts the bodys natural defences to fight cancer.

It uses substances made by the body or in a laboratory to improve or restore immune system function.

One of the latest treatment modalities is the CAR T-cell therapy, a form of immunotherapy that uses specially altered T cells a part of the immune system to fight cancer.

A sample of a patients T cells are collected from the blood, then modified to produce special structures called chimeric antigen receptors (CARs) on their surface.

When these CAR T-cells are reinfused into the patient, the new receptors enable them to latch onto a specific antigen on the patients tumour cells and kill the cells.

At the moment, this intravenous therapy is available in the United States and hasnt reached our shores yet. It has to be properly regulated first, says Dr Ng.

A volunteer is having his head shaved to donate hair to make wigs for cancer patients in this filepic. Hair loss is one of the side effects of chemotherapy that affect patients the most.

Following natural remedies

The consultant haematologist errs on the side of caution when patients ask about natural cancer remedies, or the dos and donts during treatment.

We always believe there should be a scientific approach to the problem.

If patients are doing okay while undergoing treatment and there is no weight loss, I tell them to go ahead and do what they always do.

However, just be particular about food hygiene, as there is a chance you may get food poisoning.

If youre undergoing chemotherapy, then youll land yourself in hospital, and if your luck is bad, you may even land up in the ICU (intensive care unit).

So make sure the food is cooked and not left overnight to reduce your chances of infection.

Eat a balanced diet, he advises.

When it comes to exercise, he says to work out within your limit.

Instead of pushing the body and running marathons or climbing mountains, go for walks.

Dr Ng says, Life should go on, but be sensible.

Dont go to crowded places because you may pick up an infection, but dont be withdrawn either. All humans need social interaction.

With the billion-dollar dietary supplements industry, companies are constantly trying to lure customers into buying their products.

A lot of supplements are just glorified vitamins in different packaging.

The more expensive they are, the more people will buy them, thinking they are good.

There are people with good intentions, but unfortunately, there are also a lot of scammers out there that is life.

For the amount you spend on supplements, why not keep the money aside and go for a trip once your treatment is over? he suggests.

Often, the late diagnosis is due to preference for alternative treatment.

These alternative treatments are like fashion shows, after some time, they go out of trend.

For me, youre wasting valuable time because cancer is not your friend.

Yes, chemotherapy is tough, but with the latest chemo-free regimen, patients are more willing to come forward.

The earlier it is treated, the higher your chances of recovering, he says.

To share his 30-odd years of knowledge and experience in the field, Dr Ng has written his third book titled Understanding Blood Disorders.

Intended for patients, caregivers and healthcare professionals, proceeds from the sales of the 270-page book will go to the newly set-up Faith Hope Love Hospice Care Malaysia in Petaling Jaya, Selangor.

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Leukaemia and lymphoma have a good survival rate - The Star Online

Pike River widow credits granddaughter with getting her through cancer ‘battle’ – Stuff.co.nz

Pike River widow Anna Osborne is creditingher granddaughter withhelpingher get through invasive cancer treatment.

Osborne, whose husband Milton died West Coastmine disaster just over nine years ago, had stem-cell replacementforHodgkinlymphoma in October.

She was told she had a four weeksto a year to live without the risky surgery, or up to five years with it. Wanting to see 2-year-old grandaughter Amalia start school was a big factor in thedecision to push ahead.

Supplied

Anna Osborne and her two-year-old granddaughter, Amalia, who Osborne says has been her inspiration to prolong her life after fighting cancer.

Her stem-cells wereharvested and frozenin August. The stem cell transplanttook place in Christchurch in October aftersix days of intensive chemotherapy.

READ MORE:*Pike River mine tunnel entry an important moment for widow*Pike River re-entry team breaks through into mine drift*Pike River widow 'full of nerves' for mine drift re-entry*The road to getting back into Pike River

"It kills everything.It was pretty rough going," Osborne told Stuff from herhospital bed in Greymouth.

Joanne Carroll/Stuff

Osborne has undergone a stem cell transplant, but has been in and out of hospital during her recovery.

"It was a long and really difficult process because I became really really ill. Vomiting, sleeping all the time. If I caught an infection it could kill me."

After the successful transplant of her stem-cells back into her body, she stayed in Christchurch'stransplant unit forfive weeks.

She was then transferred to Grey Base Hospital.

Phil Walter/Getty

Osborne greeting Prime Minister Jacinda Ardern when the Pike River re-entry got under way in May.

"I've been home threetimes from hospital, only two days each time. On the second day, I've just gone right down again and got nothing left to give,so I'm backin hospital. It's been a bit of a battle."

She has no appetite, is nauseousand and unable to retain some nutrients.

"My bloods might be normal but then very quickly I can't retain any electrolytes and I go down hill and everything is depleted.

Kevin Stent/Stuff

Osborne, left, with friend and fellow Pike River family member Sonya Rockhouse.

"It's a hard road. You're sick of being sick ...sick of having no energy.

"I know in the end it's going to be worthwhile and I've bought myself another four or five years. Hopefully a lot longer than that."

Osbornewasdiagnosed with the cancer, which attacks theimmune system,in 2002 at age 36.

Stacey Kirk/Stuff

Representatives of some of the Pike River families - Anna Osborne, Sonya Rockhouse and Bernie Monk - talk of their elation at the decision to re-enter the mine. (First published November 2018)

She had radiation for six weeks and went into remission, but the cancercame back just before the Pike River tragedy in November 2010, when 29 men where killed in a series of explosions at the coal mine. Osborne helped campaign for thelegalisation of medicinal cannabiswhile undergoing chemotherapy in 2015.

She is awaiting a full scan to see if the treatment has removed all the tumours in her body. For now, she is taking each day as it comes.

"I'm feeling quite positive. I've got four or five years left in me. You've got to remain positive. It's easy to give up," she said.

At her darkest moments, Amalia was at the forefront of her mind.

"She needs me and I need her. I want to be there when she starts school. She was the person that got me through and made me want to keep going.

"She's magnificent. She's brightened my world ... it's really nice having her to keep going for."

Osborne said she had a special bond with Amalia.

"She will bring her blankie and baby and sit on my lap and just cuddle into me, and my daughter says she doesn't do that for anyone.

"I can't give up on that,it's too beautiful."

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Pike River widow credits granddaughter with getting her through cancer 'battle' - Stuff.co.nz

Mums plea for stem cell donor to treat her cancer after her ex killed teen son who could have been a match – The Sun

A MUM is pleading for a stem cell donor to treat her cancer after her ex killed her teenage son - who may have been her only match.

Tania Morris, 49, was left battered and bruised by former lover Robert Goodwin in a row over fast food last year.

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The monster went on to bludgeon her 19-year-old son Nathan Bates to death while on bail for the attack, before killing himself.

In the latest horror twist, Tania now fears this Christmas could be her last after she was diagnosed with Hodgkin's Lymphoma shortly after burying her only child.

Tania's rare tissue type means there is no one on the entire global register who provides a match.

The pottery worker from Burslem told StokeonTrentLive: "It's heartbreaking. The doctors just keep saying we need a fit and healthy 19-year-old and that breaks my heart as that's how old Nathan was when he was murdered.

"My younger brother could have been a match but he died of a heart attack.

"My other brother Darren was devastated when he was tested and wasn't a match. He wanted so much to help me.

"Dad's only a half match. He's too poorly himself to go ahead but if and when it becomes life or death he could be a last resort. They're worried it would kill both me and him.

"It would really be desperate measures if it comes to that. It would be my last option because it could kill me.

"My mum's not even been tested because she's had a heart bypass.

"We are just hoping that someone comes forward. It's my only chance of beating this."

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Tania was left with horrific injuries in August 2018, when sick Goodwin tried to gauge out her eye before smashing a porcelain doll ornament over her face.

Months later he killed Nathan with a hammer as he slept before killing himself in nearby woodland on October 11 last year.

Tania is still unclear what motivated the murder - that may indirectly lead to her death as well.

The doctors just keep saying we need a fit and healthy 19-year-old and that breaks my heart as that's how old Nathan was when he was murdered.

Goodwin had been due in court the day after Nathans killing to face the assault charge against Tania - but will now never face justice for either offence.

Since her diagnosis in January, Tania has gone through multiple rounds of chemotherapy, but has now been told that the treatment hasn't worked.

She is calling on any young donor to come forward in what she thinks could be her only shot at living.

"I haven't properly grieved for Nathan because of the cancer," she said.

"He just couldn't cope with the thought of going to prison so he killed Nathan, killed himself and if I don't find a donor, he may yet kill me. All the stress he's put our family through also led to my mum's heart attack.

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"He had no reason to do what he did, he just wanted to upset me in the worst possible way.

"He said he loved me to pieces but then he did this."

Anyone who is healthy and aged 16-30 can donate stem cells by signing up with the Anthony Nolan register here.

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Mums plea for stem cell donor to treat her cancer after her ex killed teen son who could have been a match - The Sun

Akari Therapeutics Announces Initiation of Pivotal Phase III Trial of Nomacopan in Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic…

DetailsCategory: Small MoleculesPublished on Monday, 23 December 2019 16:05Hits: 45

NEW YORK, NY, USA and LONDON, UK I December 23, 2019 I Akari Therapeutics, Plc (Nasdaq:AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announces that a U.S. Food and Drug Administration (FDA) investigational new drug application (IND) is open for its multicenter Phase III study for the treatment of pediatric HSCT-TMA with nomacopan, allowing clinical sites to open in the first quarter of 2020.

With the pediatric HSCT-TMA IND now open we look forward to starting the pivotal Phase III study of nomacopan in HSCT-TMA, a potential treatment for a high risk pediatric population that suffer very high death rates and for which there are currently no approved therapies. If successful, we expect HSCT-TMA to be a gateway into a range of other poorly treated orphan TMAs, commented Clive Richardson, CEO of Akari Therapeutics. In addition, following the recent successful completion of our Phase II bullous pemphigoid study, we expect data from our Phase I/II atopic keratoconjunctivitis trial in early 2020 and interim data from our Phase III paroxysmal nocturnal hemoglobinuria trial in the first half of 2020.

HSCT-TMA is an orphan hematological condition that occurs in up to 30% of patients who have received a hematopoietic stem cell transplant (HSCT). There are no approved treatments for pediatric HSCT-TMA, and it has an estimated mortality rate of more than 80% in children with the severe form of the disease1. It is this severe form that is being targeted with nomacopan which is a bifunctional inhibitor of complement C5 and leukotriene B4 (LTB4). Following the recent end-of-Phase II meeting with the FDA, Akari has now opened an IND to initiate its pivotal pediatric HSCT-TMA study based on a single arm responder-based design. Recruitment will be focused on specialist pediatric sites in the U.S. and Europe where treatment tends to be concentrated in specialist centres.

Whilst the role of complement inhibition is understood to play an important role in pediatric HSCT-TMA, the Company believes LTB4 may also be an important target in reducing epithelial activation in both TMA and graft versus-host disease2 (GVHD) which often occur simultaneously. The Company believes daily dosing with nomacopan may also be of particular advantage in facilitating more complete complement suppression, especially in HSCT-TMA patients with high transfusion requirements.

As previously announced, this two-part pivotal Phase III study of nomacopan in pediatric patients with HSCT-TMA is based on guidance from the Companys end-of-Phase II meeting with the FDA. Part A of the trial is a dose confirmation study. Part B of the trial is a single arm responder-based efficacy study that will follow an interim analysis of Part A and a meeting with the FDA. Akari has both FDA fast track and orphan status for this program.

1 Sonata Jodele, et al. New approaches in the diagnosis, pathophysiology, and treatment of pediatric hematopoietic stem cell transplantation associated thrombotic microangiopathy. Transfus Apher Sci . 2016 April; 54(2): 181190

2 Takatsuka, et al. Predicting the severity of intestinal graft-versus-host disease from leukotriene B4 levels after bone marrow transplantation. Transplantation 2000, 26: 1313-1316

About Akari Therapeutics

Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari's lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4). Nomacopan is currently being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal hemoglobinuria (PNH). Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in AKC and BP. Akari is also developing other tick derived proteins, including longer acting versions.

SOURCE: Akari Therapeutics

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Akari Therapeutics Announces Initiation of Pivotal Phase III Trial of Nomacopan in Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic...