Rocket Pharmaceuticals Presents First Evidence of Long-Term Improvement and Stabilization in Blood Counts and Durable Mosaicism in RP-L102 Process A…

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today presents updated long-term follow-up from the Phase 1/2 clinical trial of RP-L102 at the European Society of Cell and Gene Therapy (ESGCT) 27th Annual Congress in Barcelona, Spain. RP-L102 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA). The data are included in an oral presentation by Dr. Juan Bueren, Scientific Director of the FA gene therapy program and Head of the Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBERER / IIS-FJD, entitled, Gene Therapy for Patients with Fanconi Anaemia.

Two critical validations for an FA gene therapy product are: 1) stem cell engraftment in the absence of cytotoxic conditioning and 2) evidence of sustained clinical improvement. We are proud to report that the maturing long-term data from the patients treated with RP-L102 meet both of these requirements, said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. In all four patients, bone marrow MMC-resistance, a key measure of phenotypic reversal and engraftment, meets or exceeds the 10% threshold agreed to by the FDA and EMA for the upcoming registration-enabling Phase 2 trial, and all four patients now resemble FA mosaic patients as evaluated by peripheral T-cell chromosomal fragility assay. Remarkably, patients 02002 and 02006, who received what we consider adequate drug product similar to the upcoming Phase 2 trial, now demonstrate durable robust bone marrow MMC-resistance levels of approximately 60% and 32%, respectively, confirming phenotypic correction in long-term bone marrow stem and progenitor cells. Of note, each of the four initial patients continue to show evidence of a proliferative advantage, with ongoing increases in peripheral mononuclear cell VCNs. In addition, improvement or stabilization of peripheral blood counts, which had declined substantially prior to gene therapy, suggests a halt in bone marrow failure progression. In patient 02002, hemoglobin levels are now similar to those in the first year after birth, and all lineages in patients 02002 and 02006 are now stable or improving.

Dr. Shah continued, Preliminary VCN data from three additional patients who were treated with a viable drug product also show engraftment in a dose-dependent manner, consistent with the first four patients. With this progress to date, we look forward to the upcoming results from the first two patients receiving Process B of RP-L102, designed to enable consistent results with commercial-grade product.

The presentation described nine pediatric patients (ages 3-7 years) who received RP-L102 utilizing fresh or cryopreserved mobilized peripheral blood CD34+ cells that were transduced with the therapeutic vector. Four of these patients have been followed for more than 2 years (24-39 months for patients 02002, 02004, 02005, and 02006). The Phase 1/2 study of RP-L102 is an ongoing, open-label, single-center study designed to evaluate the safety and efficacy of Process A RP-L102 without the use of any conditioning regimen conventionally used in allogenic transplant.

Dr. Bueren noted, These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected HSCs in the absence of any conditioning regimen. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer. The ability to treat patients without the use of genotoxic conditioning and to restore blood cell counts is a life-altering advancement for patients and their families, as well as the scientific community which has dedicated over two decades to finding a minimally toxic alternative for FA patients.

Rocket expects initial data from the Phase 1 Process B trial of RP-L102 by year-end. The registration-enabling Phase 2 study in Spain is now enrolling, and additional global sites will follow.

Full results from the ESGCT presentation will be available online at the conclusion of the oral presentation: https://www.rocketpharma.com/esgct-presentations/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Anthony Harries: In the absence of a true test for latent TB we need shorter, safer preventive treatments – The BMJ – The BMJ

Treating latent tuberculosis infection (LTBI) is a key component of the World Health Organizations End TB strategy, which aims to end the epidemic of TB by 2030. This neglected but vital intervention was given further emphasis in September 2018 at the United Nations high level meeting on TB, where world leaders committed to treating at least 30 million people for LTBI by 2022, including 4 million children under 5 years of age, 20 million household contacts of people affected by TB, and 6 million people with HIV.

In this context, Marcel Behr and colleagues continue their thought provoking journey of questioning established dogma. In August 2018, they provided robust evidence to support the assertion that people infected with M tuberculosis tend to progress to disease over an incubation period of two months to two years rather than over their lifetime. In a new Analysis, they go on to question the widely held assumption that M tuberculosis infection is for life.

As the authors point out, there is no perfect way to directly diagnose LTBI. The diagnosis of LTBI is made in people with no evidence of active TB by measuring the immunological response to M tuberculosis antigens using the tuberculin skin test or interferon gamma release assay. Infection is inferred rather than proven.

Behr and colleagues test the assumption that TB immunoreactivity is the same as lifelong infection in two ways: by analysing studies of the natural history of TB immunoreactivity in people given treatment and by assessing the risk of active TB in immunoreactive people who develop severe immunosuppression.

Data from longitudinal studies and clinical trials show that immunoreactivity persists after successful treatment of either LTBI or active TB, with persistence being most prolonged in those with the longest duration of immunoreactivity before starting treatment. Epidemiological studies also show that most people with TB immunoreactivity who become severely immunosuppressed owing to HIV, solid organ transplantation, haematopoietic stem cell transplantation, or tumour necrosis factor inhibition remain free of active TB.

What should we take from this new analysis? In terms of understanding LTBI, Behr and colleagues argument is persuasive that TB immunoreactivity is a sign of having been infected with M tuberculosis and not a marker of continued infection. Their analysis suggests that about 10% of people with TB immunoreactivity harbour viable organisms capable of causing disease in the right circumstances, such as immunosuppression; in the remainder, M tuberculosis bacteria are likely to be permanently dormant or dead.

This perspective reinforces the urgent need for clinically useful and affordable biomarkers that can clearly distinguish between persistent infection and immunological memory of a past infection. The journey of discovery, development, and deployment of a biomarker that can guide clinical decisions is not likely to be quick or easy. But it is a journey that must be taken, and if successful would enable precise identification of those who are truly infected and at most risk of developing active TB.

In the meantime, we have no choice but to continue providing treatment to those with LTBI if we want to honour our promises to end TB. The current preventive policy is to treat those at the highest risk of TB, such as people with HIV or young household contacts of a person with TB, without testing for LTBI. For other high risk groups, including patients with silicosis, patients in renal failure, and certain vulnerable groups, the preventive policy is to systematically test for LTBI before treatment. We need to recognise, however, that we might be treating many people who do not require this intervention because they do not have latent infection. This would be acceptable if the treatment was short and entirely safe. Unfortunately, this is not the case.

Isoniazid is the current drug of choice for treating LTBI, often for six months, but it can cause peripheral neuropathy or drug induced hepatitis, which if unrecognised or identified too late can be fatal. Shorter and safer alternative regimens are now available, the most attractive being a three month course of weekly rifapentine and isoniazid (12 doses). Two important hurdles in making this regimen widely available are country specific regulatory approval and the huge cost of rifapentine. TB programmes need to embrace new shorter treatment durations, and global advocacy and activism are needed to make the drugs affordable in the countries where they are most needed. At the same time, TB programmes need to agitate for an easy to use and inexpensive biomarker that truly identifies LTBI.

Anthony Harries is senior adviser at the International Union Against Tuberculosis and Lung Disease in Paris, France, and honorary professor at the London School of Hygiene and Tropical Medicine, UK.

Competing interests: None declared

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Anthony Harries: In the absence of a true test for latent TB we need shorter, safer preventive treatments - The BMJ - The BMJ

Quality of Life Is Similar in Older Patients Receiving Intensive vs Nonintensive Chemotherapy – Hematology Advisor

No differences in patient-reported quality of life (QOL), anxiety, or depression were found in older patients with acute myeloid leukemia (AML) who were treated with intensive or nonintensive chemotherapy, according to results from a longitudinal study published in Leukemia. Additionally, these patients experienced improved QOL and anxiety while undergoing chemotherapy regardless of regimen intensity.

Frontline chemotherapeutic options for AML include intensive therapy requiring extended hospitalization for 4 to 6 weeks and nonintensive therapy that can frequently be administered in the outpatient setting. For patients with AML older than 60 years, long-term disease-free survival is low, and many of these patients are considered ineligible for intensive chemotherapy, particularly those with comorbidities or poor performance status. Historically, clinicians have considered intensive chemotherapy to be more toxic and difficult to manage compared with nonintensive regimens.

This study assessed 100 patients with newly diagnosed AML who were 60 years or older. Intensive and nonintensive chemotherapy were administered to 50 patients each. Patients completed self-report assessments at baseline (within 72 hours of starting treatment) and study questionnaires at 2, 4, 8, 12, and 24 weeks after diagnosis.

The Functional Assessment of Cancer Therapy-Leukemia tool was used to assess QOL, and the Hospital Anxiety and Depression Scale was used to assess psychological distress at the 6 timepoints.

Patient-reported QOL improved over time (beta level, 0.32; P=.013), with no significant differences in QOL reported between the patient groups at any timepoint. At baseline, approximately one-third of patients in each treatment group reported clinically significant symptoms of depression and anxiety, with no significant differences between the groups. Symptoms of depression remained consistent during treatment, and symptoms of anxiety improved over time (beta level,-0.08; P<.001).

Although rates of depression symptoms remained consistent across the course of therapy, the researchers emphasized that the proportion of patients who experienced depression at baseline was clinically important; in response, they suggested supportive care interventions to decrease distress.

Of note, this study also assessed the psychological symptoms experienced by caregivers from baseline through treatment. To our knowledge, this is also the first study to explore the psychological burden experienced by caregivers of older patients with AML, wrote the researchers.

Nearly half of caregivers reported clinically significant symptoms of anxiety at the time of diagnosis of the patient, and 16.3% reported experiencing symptoms of depression. These rates of psychological distress are higher than the ones often seen in caregivers of patients with hematologic malignancies undergoing hematopoietic stem cell transplantation or those with solid tumors, noted the researchers. However, they cautioned that because of the limited number of caregivers, comparisons between caregivers of patients undergoing intensive and nonintensive chemotherapy could not be made.

Reference

1. El-Jawahri A, Abel GA, Traeger L, et al. Quality of life and mood of older patients with acute myeloid leukemia (AML) receiving intensive and non-intensive chemotherapy. Leukemia. 2019;33:2393-2402.

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Speaking Multiple Sclerosis: A Glossary of Common Terms – Everyday Health

Whether youve been recently diagnosed with multiple sclerosis (MS) or have been living with the condition for a while, chances are youll sometimes hear terms from your healthcare team that are new to you.

The following is a quick, alphabetical guide to the terminology you may need to know as you manage your condition:

Ankle-Foot Orthosis (AFO) A brace designed to support the position of the foot and motion of the ankle to compensate for nerve damage and muscle weakness in the area caused by MS and other movement disorders. An AFO is typically used to stabilize weak limbs or to reposition a limb with contracted muscles into a more normal position.

Autoimmune Disease Your immune system plays a major part of your bodys defense against bacteria and viruses by sending out cells to attack them once they enter your body. However, if you have an autoimmune disease, your immune system mistakenly attacks healthy cells in your body, causing them to weaken or break down. MS is thought to be just one example of an autoimmune disease. It has been suggested that in MS, your immune system may mistakenly attack the cells in your central nervous system.

Axon Long threadlike structures of nerve cells that send impulses to other cells in your body. Research suggests that damage to or loss of these fibers in progressive MS may be linked to worsening disability and more severe progression.

Central Nervous System (CNS) The group of organs in your body that includes the brain, spinal cord, and optic nerves. If you have MS, your bodys immune system may be working against the CNS, producing neurological symptoms such as muscle weakness and vision problems.

Cerebrospinal Fluid (CSF) A clear, colorless liquid that surrounds the brain and spinal cord to protect the CNS and assist in the circulation of nutrients and removal of waste products. In MS, damage to the myelin sheath of nerve cells causes certain types of proteins to be released into the spinal fluid. The presence of these proteins in the CSF, but not in the blood, may point to a diagnosis of MS.

Clinically Isolated Syndrome (CIS) A first episode of neurologic symptoms that lasts at least 24 hours and is caused by inflammation or demyelination (loss of the myelin that covers the nerve cells) in the CNS. People who experience CIS may or may not go on to develop MS. However, when CIS is accompanied by magnetic resonance imaging (MRI)detected brain lesions similar to those found in MS, you have a 60 to 80 percent chance of a second neurologic event and diagnosis of MS within several years, according to the National MS Society.

Cog Fog A commonly used term that refers to the cognitive changes experienced by many people with MS. According to MS Australia, approximately 50 percent of people with the condition will develop some degree of cog fog, or inhibited ability to think, reason, concentrate, or remember. For some, cognitive problems will become severe enough to interfere in a significant way with daily activities.

Corticosteroids (or Steroids) Prescription medication used to treat relapses in relapsing-remitting MS. Your doctor may prescribe intravenous (IV) corticosteroids if the symptoms of your relapse are causing significant problems, like poor vision or difficulty walking. These drugs work by suppressing the immune system and reducing inflammation in the CNS, and they may help relapse symptoms resolve more quickly. But they wont affect your ultimate level of recovery from a relapse or the long-term course of your MS. Methylprednisolone is a commonly used corticosteroid in MS.

Diplopia (or Double Vision) An eye problem in which you see two images of a single object. It may be present when only one eye is open (monocular) or disappear when either eye is closed (binocular). Diplopia is a common symptom of MS, and it occurs because of damage to the optic nerve.

Disease-Modifying Therapies (DMTs) Drugs designed to reduce new relapses, delay progression of disability, and limit new CNS inflammation in people with MS. Although there are multiple DMTs that have been approved by the U.S. Food and Drug Administration (FDA) for use in MS, these drugs generally work by reducing inflammation in nerve cells in theCNS.

Dysarthria A speech disorder caused by neuromuscular impairment and resulting in disturbances in motor control of the muscles used in speech. Its believed the demyelinating lesions in MS may result in spasticity, weakness, slowness, or ataxic incoordination of the lips, tongue, mandible, soft palate, vocal cords, and diaphragm, causing this speech impairment.

Dysphagia (Difficulty Swallowing) A condition that may occur in people with MS, leading to difficulty in eating solid foods or liquids, frequent throat clearing during eating or drinking, a feeling that food is stuck in the throat, or coughing or a choking sensation when eating or drinking. Its the result of nerve damage within the muscles that control swallowing.

Epstein-Barr Virus (EBV) A virus believed to be a possible cause or trigger for MS. Although the exact cause of MS remains unknown, researchers suggest an infectious agent may be involved in its development. Studies have found that antibodies (immune proteins that indicate a person has been exposed to a given virus) to EBV are significantly higher in people who eventually develop MS than in those who dont. Other research has noted that people with a specific immune-related gene and high levels of antibodies to EBV in their blood are 9 times more likely to develop MS than others.

Evoked Potentials A test that measures the speed of nerve messages along sensory nerves to the brain, which can be detected on your scalp using electrodes attached with sticky pads. Its sometimes used in the diagnosis of MS, because nerve damage can slow down the transmission of nerve signals. Evoked potential tests can indicate nerve pathways that are damaged prior to the onset of MS symptoms.

Exacerbation An occurrence of new symptoms or the worsening of old symptoms that may also be referred to as a relapse, attack, or flare-up. Exacerbations can be very mild, or severe enough to interfere with a person's ability to perform day-to-day activities.

Expanded Disability Status Scale (EDSS) A scale used for measuring MS disability and monitoring changes in the level of disability over time. Developed by neurologist John Kurtzke, MD, in 1983, the EDSS scale ranges from 0 to 10 in 0.5-unit increments (scoring is based on a neurological exam) and relies on walking as its main measure of disability. People with an EDSS of 1 have no disability and minimal loss of function, while those with an EDSS of 9.5 are confined to bed and totally dependent on others for functions of daily living.

Foot Drop (or Drop Foot) A symptom of MS caused by weakness in the ankle or disruption in the nerve pathway between the legs and the brain, making it difficult to lift the front of the foot to the correct angle during walking. If you have foot drop, your foot hangs down and may catch or drag along the ground, resulting in trips and falls. Foot drop can be managed with an AFO or other treatments.

Hematopoietic Stem Cell Transplantation (HSCT) A procedure designed to reboot the immune system, the National MS Society says, using hematopoietic (blood cellproducing) stem cells derived from a persons own bone marrow or blood. If your doctor recommends HSCT, youll undergo a chemotherapy regimen before these cells are reintroduced to the body via IV injection, where they will migrate to your bone marrow to rebuild the immune system.

John Cunningham (JC) Virus A common infection completely unrelated to MS that is found in as many as 90 percent of people, according to the UK's MS Trust. JC virus has no symptoms and is normally controlled by the immune system. However, if your immune system is weakened, the JC virus can reactivate, causing potentially fatal inflammation and damage to the brain known as progressive multifocal leukoencephalopathy (PML). Certain MS disease-modifying therapies have been linked with increased risk for PML.

Lhermittes Sign An electric shock-like sensation experienced by some with MS when the neck is moved in a particular way. The sensation can travel down to the spine, arms, and legs.

Lesion (or Plaque) Refers to an area of damage or scarring (sclerosis) in the CNS caused by inflammation in MS. These lesions can be spotted on an MRI scan, with active lesions appearing as white patches. With regular MRIs, a neurologist can tell how active your MS is.

Lumbar Puncture (or Spinal Tap) A procedure used for the collection of cerebrospinal fluid (CSF), sometimes done to help diagnose MS. For this procedure, your doctor will ask you to lie on your side or bend forward while seated, before cleansing an area of your lower back and injecting a local anesthetic. He will then insert a hollow needle and extract a small amount of spinal fluid using a syringe.

Magnetic Resonance Imaging (MRI) The diagnostic tool that currently offers the most sensitive noninvasive way of imaging the brain, spinal cord, or other areas of the body, according to the National MS Society. Its the preferred imaging method for diagnosis of MS and to monitor the course of the disease. MRI uses magnetic fields and radio waves to measure the relative water content in tissues, which is notable in MS because the layer of myelin that protects nerve cell fibers is fatty and repels water. In areas where myelin has been damaged by MS, fat is stripped away and the tissue holds more water. This shows up on an MRI as a bright white spot or darkened area, depending on how the images are made.

McDonald Criteria A guidance used in the diagnosis of MS, authored by an international panel of experts on the condition, originally in 2010. The guidance was updated in 2017. Among the key changes: advising for the use of brain MRI as part of the diagnostic process.

MS Hug A common symptom of MS. If you experience the MS hug, you may feel like you have a tight band around your chest or ribs, or pressure on one side of your torso. Some people find that it is painful to breathe. The MS hug can last for seconds, minutes, hours, or even longer.

Myelin A substance rich in lipids (fatty substances) and proteins that helps form the myelin sheath. In MS, particularly relapsing-remitting MS, an abnormal immune response produces inflammation in the CNS, effectively attacking the myelin in the cells.

Myelin Sheath An insulating layer of fatty substances and proteins that forms around the nerves in body, including those in the CNS. It allows electrical impulses to transmit quickly and efficiently along the nerve cells, but these impulses can be slowed if the sheath is damaged, causing MS.

Neurodegeneration Refers to the process by which the myelin sheath of cells in the CNS is damaged in MS. Its believed to be a major contributor to neurological disability in the condition, and may be the reason immune modulation treatments (disease-modifying therapy) are generally less effective in the progressive MS than in the relapsing-remitting MS.

Neurologist The point person for monitoring your MS treatment and managing MS symptoms. This specialist typically focuses on conditions affecting the CNS.

Neuropathic Pain A type of pain common in MS that results from changes or damage to the myelin sheath and the axons, or nerve fibers, it normally covers. MS-caused neuropathic pain may be chronic, intermittent, or occur only in response to a stimulus.

Neuropsychologist A specialist you may be referred to who helps you manage the cognitive effects of MS. Neuropsychological testing (or testing of the functioning of your brain) involves identifying memory or learning difficulties associated with MS. Cognitive rehabilitation may improve functioning.

Nociceptive Pain Caused by damage to muscles and joints, it can be either acute or chronic, and may not result from MS itself, but be caused by changes in posture or walking or the overuse of assistive devices in those with the condition.

Nystagmus A common eye abnormality in MS, its characterized by involuntary, rhythmic, back-and-forth motion of the eyeball, either horizontally or vertically. For those with nystagmus, the perception of the rhythmic movement of the surrounding stationary world (oscillopsia) can be disorienting and disabling.

Oligoclonal Bands (OCBs) Immunoglobulins, or proteins, that collect in blood plasma or cerebrospinal fluid (CSF). Although not every person with MS has OCBs, their presence can support a diagnosis of MS. Having OCBs is generally associated with a younger age of MS onset and a poorer prognosis.

Optic Neuritis An inflammatory condition that damages the optic nerve, a bundle of nerve fibers that transmits visual information from your eye to your brain, causing pain and temporary vision loss in one eye. Its been linked with nerve damage resulting from MS, and may be among the first symptoms a person with the condition experiences.

Pseudobulbar Affect (PBA) A neurologic effect experienced by roughly 10 percent of people with MS as well as some with Parkinsons disease or amyotrophic lateral sclerosis (ALS), according to the Multiple Sclerosis Association of America (MSAA). Its characterized by sudden, uncontrollable expressions of laughter or crying without an obvious cause, which can be distressing as well as embarrassing to those who experience it. PBA is believed to be a mood disorder related to the disruption of nerve impulses in the CNS, but its different from depression, which is also common in MS.

Pseudoexacerbation A temporary worsening of symptoms without actual myelin inflammation or damage. It is often triggered by other illnesses or infection, exercise, a warm environment, depression, exhaustion, and stress. Urinary tract infection (UTI) is the most common type of infection to cause a pseudoexacerbation.

Sclerosis A general hardening of the body tissue. The term multiple sclerosis refers to the multiple areas of scar tissue often called lesions that develop along affected nerve fibers and that are visible in MRI scans.

Spasticity A symptom of MS that causes your muscles to feel stiff, heavy, or difficult to move. When a muscle spasms, youll experience a sudden stiffening that may cause a limb to jerk. This may be painful.

Trigeminal Neuralgia (or Tic Douloureux) A type of neuropathic pain that occurs on the face (usually on one side only). Its a known symptom of MS, and you may experience it in your cheek; upper or lower jaw; inside the mouth; or in the area around your eyes, ears, or forehead. In MS, its typically caused by damage to the myelin sheath around the trigeminal nerve, which among other functions controls the muscles used in chewing. The condition is triggered by everyday activities, like tensing facial muscles while shaving or when chewing.

Vertigo An intense sensation of the surrounding environment spinning around one. In MS, vertigo is typically caused by growth of an existing lesion or development of a new lesion on the brain stem or cerebellum, the area in the brain that controls balance. It can also be a symptom of a problem with the inner ear, or it can be side effect of medication used to treat MS or other health conditions you may have.

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Speaking Multiple Sclerosis: A Glossary of Common Terms - Everyday Health

Veterans Day: Flotation Therapy Helping Special Operations Officer – Yahoo Finance

Military Float Free at True REST Float Spa 11th Day of Every Month

PHOENIX, AZ / ACCESSWIRE / October 24, 2019 / This Veterans Day, True REST Float Spa, the world's largest float therapy brand, re-ups its commitment to supporting the U.S. military and U.S. veterans with a holistic, 100% natural, scientifically proven pain relief alternative: flotation therapy. On Nov. 11, active duty and retired military are invited to float for free at any True REST Float Spa. Additionally, for every True REST Float Spa float therapy session purchased by a civilian between Oct. 21 and Nov. 11, an active duty military member or veteran will be gifted a free flotation therapy session via their "One for One" Veterans Day Challenge.

Since 2015, True REST Float Spa has given away more than 3,000 complimentary floats to U.S. active duty military and veterans.

After serving 21 years in the United States Army as Green Beret, Special Forces, Communications Specialist, Travis Wilson retired. When Wilson returned home, his body was broken, and the inside of his mind still felt like a battlefield. He endured over 13 surgeries, but nothing brought him relief from the chronic pain he felt. In addition, he couldn't sleep. He knew he needed to find help. He tried stem cell therapy and yoga before trying float therapy in a sensory deprivation pod as an alternative treatment therapy.

Wilson was introduced to flotation therapy thanks to True REST Float Spas' U.S. Military Appreciation Day. Every 11th day of the month, every month, True REST Float Spa offers a free, 60-minute float to any active-duty military member and/or veteran.

Wilson shares how flotation therapy has helped him in a recent True REST Float Spa video.

"I have had 13 surgeries in my military career. A lot of joint pain, just overall body pain. Wasn't sleeping. Wasn't dreaming. I have done stem cell therapy for a TBI and even tried yoga," Wilson explains. "At first floating wasn't for me. I think I only lasted about 30 minutes. The sensory deprivation was just a bit much for my brain. But then, I was calmer after floating. Slept a little bit better. My body just felt better after floating."

When a person floats, they lie absolutely still on top of a specialized Epsom salts water solution with zero stimulation. There are no distractions such as movement, sound, light, taste, touch and smell, so areas of the brain responsible for these activities are essentially turned off. The water temperature is expertly calibrated where the air, water and body match perfectly. Because floating creates a weightless sensation, the spinal cord receives respite as well.

Downtime, rest, stillness and slowing down are essential for healing. The aftereffects of just one float tell it all: increased mental clarity and sensations of peace and neutrality, decreased anxiety and depression, and decreased cortisol.

A scientific study addressing anxiety, "Flotation REST in Applied Psychophysiology" by Thomas H. Fine, M.A., and Roderick Borrie, Ph.D., at the Medical College of Ohio, noted, "Patients reported far more relief from anxiety and stress from flotation than any other modality. For depression, flotation was equal to counseling at near 70%, with relaxation training at 53% and physical therapy and medication at 20%."

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With these types of results, it's no wonder that in May, the U.S. House of Representatives passed H.R. 2359: Whole Veteran Act, which would direct the Department of Veterans Affairs to study alternative health services that it currently offers and determine what it would cost in order to expand those services throughout the Veterans Health Administration. They want veterans to try a combination of old and new treatment methods, and use treatments like yoga, meditation and acupuncture before drugs.

Today Wilson owns and runs Alpha Elite Performance, a sports nutrition company, and encourages his fellow veterans to seek out flotation therapy as an alternative and holistic solution to addressing chronic pain and trauma. He is hoping that by sharing the impact floatation therapy has had on his recovery journey, others will be encouraged to try holistic options like float therapy instead of prescribed medication. "I'm tired of losing friends," he said. "Help is available, and it doesn't come in pill form. This really works."

For more information on True REST Float Spa's U.S. Military Appreciation Day the 11th day of every month and their Veterans Day Challenge, visit http://www.TrueREST.com/Veterans.

About True REST Float Spa

True REST Float Spa is the world's largest float spa brand. With over 79 awarded locations, including 34 open locations and another 15 opening this year across the country, it is on its way to servicing 1 million floats. True REST Float Spa has created a luxury float spa experience in 10 inches of water and 1,000 pounds of Epsom salts. Members float effortlessly in their float suite. Each location is dedicated to providing pain relief, relaxation and better sleep through a 60-minute float session. True REST Float Spa offers monthly memberships, programs and packages. For more information, go to http://www.TrueREST.com. Or visit Facebook: https://www.facebook.com/TrueREST/, Twitter: https://twitter.com/truerest or Instagram: https://www.instagram.com/truerest/. For franchising opportunities, go to https://www.TrueRESTfranchising.com.

MEDIA CONTACT:Jo TrizilaTrizCom PR on behalf of True REST Float Spa and True REST FranchisingOffice: 972-247-1369Cell/Text: 214-232-0078Email: Jo@TrizCom.com

SOURCE: True REST Float Spa

View source version on accesswire.com: https://www.accesswire.com/564081/Veterans-Day-Flotation-Therapy-Helping-Special-Operations-Officer

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Veterans Day: Flotation Therapy Helping Special Operations Officer - Yahoo Finance

Dad who called on the public for stem cells for his son is up for an award – Chronicle Live

Doting dad Stephen Armstrong knows all too well what its like to be waiting for a transplant donor.

His son Jacob was diagnosed at two years old with a rare blood disorder and called on the public to donate stem cells to find him a match.

He then set out to raise as much money as he could for the blood cancer charity Anthony Nolan in a bid to save lives.

And now, after raising over 20,000, his efforts have been recognised by the charity as they honour him at an awards ceremony held at the Tower of London in November.

Stephen, 33, of Wallsend, North Tyneside, has been shortlisted for the Individual Fundraiser of the Year Award at the Anthony Nolan Supporter Awards 2019.

The prestigious awards are back for their seventh year and will recognise the outstanding achievements of the volunteers, fundraisers and campaigners who help the pioneering blood cancer charity save lives.

Stephens nomination is in recognition of his incredible fundraising efforts, leading a group of 19 friends and family in a series of physical challenges, all while his son was undergoing treatment.

After Jacob was diagnosed in 2017, Stephen set out to find a matching stem cell donor, as well as raise awareness of the need for more people on the register.

From here Jacobs Journey was born, and through a series of challenges including the Great North Run, the Great North Bike Ride and climbing Ben Nevis, Stephen has helped raise over 20,000 for the charity.

Jacob, who turns four in November, and his family have been told he does not need a transplant, but Stephen and his family want to continue raising awareness for others who arent so lucky.

When Jacob was diagnosed, we were stunned by how few people were on the stem cell donor register. I couldnt believe how a stranger in the street could potentially save our little boys life, said Stephen, an assistant manager for Dixons Carphone.

Anthony Nolan helped us massively while Jacob was ill and provided a great support network. I feel very proud to be nominated for an award, and I hope it can help build even more awareness for the cause.

Stephen and mum Kirsty, 28, received the news in December 2017 that Jacob was suffering from bone marrow failure, which affects between 30 and 40 children each year.

They first became concerned about his health when they went abroad to get married and noticed he was getting bruised easily. The marks would take weeks to disappear, so when the couple returned to the UK they decided to take Jacob to the doctor for a check up.

After tests he was then diagnosed and was treated at the Great North Childrens Hospital in Newcastle, where he received two blood transfusions.

Stephen added: When we were told Jacob did not need the transplant it was the best news in the world, a total relief. He still needs check ups every three months and his consultants is keeping an eye on him. There are so few people on the stem cell donor register so I just wanted to create a ripple effect with awareness and get more people on it.

Stephen, who has raised a further 8,000 for other smaller charities, has also been nominated for our Chronicle Champions Award in the Champion Fundraiser category.

Henny Braund, Chief Executive of Anthony Nolan, said: It is remarkable to see how many people support our work to find a match for those in need of a stem cell transplant. Without them, none of our lifesaving work would be possible.

Stephen has shown tremendous commitment to Anthony Nolan by continually going above and beyond in his fundraising efforts.

Henny added: We want to extend a huge congratulations to Stephen and look forward to celebrating with him at the awards.

The awards take place on Thursday 28 November at the Tower of London, and all winners will be revealed on the night.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance at life. It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

To see the full shortlist, and find out more about the charity visit http://www.anthonynolan.org/awards

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Dad who called on the public for stem cells for his son is up for an award - Chronicle Live

Progress toward improving detection, monitoring and treatment of metastatic cancers – Brain Tumour Research

24 October 2019

Most cancers kill because tumour cells spread, or metastasise beyond the primary site, for example breast, to invade other organs, brain being one. Now, a University of Southern California (USC), study has found that circulating tumour cells can actually target specific distant organs.

Their study of brain-invading breast cancer reveals that circulating tumour cells have a molecular signature indicating specific organ preferences.

The findings, which appear in Cancer Discovery, explain how tumour cells in the blood target a particular organ and may enable the development of treatments to prevent the spread of these metastatic cancers.

In this study breast cancer cells from the blood of breast cancer patients with metastatic tumours were isolated, expanded and grown in the lab.

Analysis of these cells identified regulator genes and proteins within the cells that apparently directed the cancers spread to the brain. The team were therefore able to predict that a patients breast cancer cells would eventually migrate to the brain.

Assistant professor of stem cell and regenerative medicine at the Keck School of Medicine at USC, Min Yu, also discovered that a protein on the surface of these brain-targeting tumour cells helps them to breech the blood brain barrier and lodge in brain tissue, while another protein inside the cells shield them from the brains immune response, enabling them to grow there.

We can imagine someday using the information carried by circulating tumour cells to improve the detection, monitoring and treatment of the spreading cancers, Yu said.

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Progress toward improving detection, monitoring and treatment of metastatic cancers - Brain Tumour Research

In a first, 26-year-old DMD patient in UP survives with stem cell therapy – India TV News

Image Source : PTI

Children, suffering from DMD, usually die of cardio-respiratory failure. Represtational image

Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

The patient's father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

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In a first, 26-year-old DMD patient in UP survives with stem cell therapy - India TV News

BrainStorm Cell Therapeutics’ President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 – GlobeNewswire

NEW YORK, Oct. 24, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics' President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 - GlobeNewswire