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BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA (Dinutuximab Beta) for Patients With High-Risk Neuroblastoma – Business Wire

CAMBRIDGE, Mass. & BEIJING & HEMEL HEMPSTEAD, England--(BUSINESS WIRE)--BeiGene, Ltd. (NASDAQ:BGNE; HKEX:06160) and EUSA Pharma (UK), Ltd. today announced that the China National Medical Products Administration (NMPA) has granted QARZIBA (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory (R/R) neuroblastoma with or without residual disease. Dinutuximab beta is a targeted immunotherapy approved by the European Medicines Agency (EMA).i

Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA, commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene. For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.

We are delighted that the benefit of dinutuximab beta has been recognized in China. This approval represents an important milestone in our mission and collaboration with BeiGene of bringing innovative cancer and rare disease therapies to patients, said Carsten Thiel, Ph.D., Chief Executive Officer of EUSA Pharma.

The approval of dinutuximab beta in China for the treatment of patients with high-risk neuroblastoma was supported by clinical results available from key trials conducted by SIOPEN (The International Society of Paediatric Oncology Europe Neuroblastoma Group) in collaboration with APEIRON Biologics and EUSA Pharma. These randomized controlled trials evaluated the efficacy of dinutuximab beta by comparing the administration of dinutuximab beta with and without interleukin-2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two single-arm studies in the R/R setting. In the SIOPEN trial (HR-NBL1), the five-year event-free survival (EFS) rate in patients treated with dinutuximab beta was 57% vs. 42% of historical controls (p<0.01) and five-year overall survival (OS) rate was 64% vs. 50% (p0.0001).ii The safety of dinutuximab beta has been evaluated in 514 patients. The most common adverse reactions were pyrexia and pain that occurred despite analgesic treatment. Other frequent adverse reactions were hypersensitivity, vomiting, diarrhea, capillary leak syndrome, and hypotension.

About QARZIBA (dinutuximab beta)

QARZIBA is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells. Dinutuximab beta was approved by the European Commission in 2017 (See EMA Summary of Product Characteristics (SmPC)) and is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilized by other suitable measures.

About EUSA Pharma

Founded in March 2015, EUSA Pharma is a world-class biopharmaceutical company focused on oncology and rare disease. The company has extensive commercial operations in the United States and Europe, alongside a direct presence in select other markets across the globe. EUSA Pharma is led by an experienced management team with a strong record of building successful pharmaceutical companies and is supported by significant funding raised from leading life science investor EW Healthcare Partners. For more information please visit http://www.eusapharma.com.

BeiGene Oncology

BeiGene is committed to advancing best and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines to patients across the globe. We have a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 90 clinical trials involving more than 13,000 patients and healthy volunteers. Our expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. The Company currently markets three medicines discovered and developed in our labs: BTK inhibitor BRUKINSA in the United States, China, Canada, and additional international markets; and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab and PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen and Bristol Myers Squibb. We also plan to address greater areas of unmet need globally through our collaborations including with Amgen, Bio-Thera, EUSA Pharma, Mirati Therapeutics, Seagen, and Zymeworks. BeiGene has also entered into a collaboration with Novartis granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

About BeiGene

BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of approximately 7,000 colleagues across five continents. To learn more about BeiGene, please visit http://www.beigene.com and follow us on Twitter at @BeiGeneGlobal

BeiGene Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the planned launch, potential benefits to patients, and opportunity of QARZIBA in China, and other information that is not historical information. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

QARZIBA is a registered trademark of EUSA Pharma (UK), Ltd.

GL-DNB-2100014. August 2021.

__________________

References

i European Medicines Agency, Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta APEIRON Biologics). Accessed: August 2021 via https://www.ema.europa.eu/en/medicines/human/EPAR/qarziba#authorisation-details-section.

ii Ladenstein, R et al. Cancers 2020, 12, 309; doi:10.3390/cancers12020309.

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BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA (Dinutuximab Beta) for Patients With High-Risk Neuroblastoma - Business Wire

Study Calls for COVID-19 Vaccination in Patients With Cancer to Enable Optimal Treatment Delivery During Pandemic – OncLive

When utilizing a validated antibody assay against the SARS-CoV-2 spike protein, investigators revealed a high seroconversion rate of 94% among 200 patients with cancer in New York City who had received a full dose of 1 of the FDA-authorized COVID-19 vaccines.

Patients with solid tumors experienced an impressive seroconversion rate of 98% compared with a rate of 85% in those with hematologic malignancies, 70% in those who had received highly immunosuppressive therapies like anti-CD20 agents, and 73% in those who had previously undergone stem cell transplantation. Notably, patients who received treatment with immune checkpoint inhibitors or hormonal therapies experienced seroconversion rates of 97% and 100%, respectively, following vaccination.

We saw very encouraging [data] showing that most patients with a cancer diagnosis have a really high chance of responding to vaccinationsas long as the vaccinations are done in an appropriate manner, [with] both doses administered, Balazs Halmos, MD, MS, study author, director of Thoracic Oncology, and director of Clinical Cancer Genomics at Montefiore Medical Center, told OncLive in an exclusive interview on the research. This was [true] even for [patients who were receiving] active treatment with chemotherapy, targeted therapy, or immunotherapy.

Halmos and colleagues launched this study to develop a better understanding with regard to the immunogenicity of vaccines in a group of patients with a cancer diagnosis in New York City by examining the rates of anti-spike immunoglobulin G (IgG) antibody positivity after receiving 1 of the 3 authorized COVID-19 vaccines.

A total of 213 patients were enrolled to the study through an informed-consent process. Twenty-nine additional patients with cancer who received the SARS-CoV-2 spike IgG testing were identified through retrospective chart review.

A total of 18 patients did not have this test conducted following consent, and thus, they were excluded from the analysis. Twenty additional patients were excluded because they had their test done before having received full vaccination in accordance with FDA guidance. Four additional patients were excluded for other reasons.

As such, 233 patients with cancer were noted to have received all required doses of their COVID-19 vaccine; all these patients were included in the safety analysis. A subset of 200 patients received the IgG test and were included in the immunogenicity analysis. Serological information from these patients were utilized in association studies between cancer subtypes and therapies.

Investigators also examined the link between the quantitative titer of SARS-CoV-2 spike IgG and cancer subtypes and therapies. If the 200 patients, 185 had available IgG titers that were at least 2 days following the last vaccine dose. A total of 15 patients were excluded from the vaccination cohort with titers; these patients had received the vaccine, but titers were checked less than 1 week from their last dose.

Among those included in the efficacy analysis (n = 200), the median age was 67 years (range, 27-90), 58% were female, and 42% were male. The study population was noted to be representative of the diverse population that resides in the Bronx, New York, with 32% of patients identifying as African American, 39% as Hispanic, 22% as Caucasian, 5% as Asian, and 3% as other ethnicities.

Additionally, 67% of patients had a solid tumor diagnosis and 33% had a hematologic malignancy. Among those with solid tumors, 26% had breast cancer, 14% had gastrointestinal cancer, 9% had genitourinary cancer, 5% had gynecologic cancer, 13% had thoracic or head and neck cancer, 1% had skin or musculoskeletal cancer, and 1% had carcinoma of an unknown primary. Among those with hematologic malignancies, 13% had lymphoid disease, 9% had myeloid disease, and 11% had plasma cell disease.

Seventy-five percent of patients had an active malignancy and 67% were receiving active treatment at the time that they received the COVID-19 vaccine. Fifty-six percent of patients were on active chemotherapy. Moreover, 19% of patients were on active chemotherapy within 48 hours of receiving at least 1 of their COVID-19 vaccine doses.

Fifty-four percent of patients completed vaccination with the Pfizer vaccine, 31% with the Moderna vaccine, and 10% with the Johnson & Johnson vaccine. A total of 3 patients had received a complete mRNA vaccination series but the information regarding the type of vaccine (Pfizer vs Moderna) are not yet available.

Additional findings from the study showed that significantly higher titer values were observed in solid tumors vs hematologic malignancies among a subgroup of 185 patients with available IgG titers longer than 7 days post vaccination, at a median of 7858 AU/mL vs a median of 2528 AU/mL, respectively (P = .013).

When comparing patients who were receiving active cancer treatment vs those who were not, no significant differences in seroconversion were reported, at 96% and 93%, respectively. However, investigators did report lower seropositivity rates in those who were on active cytotoxic chemotherapy versus other treatments, at 92% vs 99%, respectively (P = .04). Moreover, significantly lower seroconversion rates were also noted in those who received immunosuppressive therapies like stem cell transplant (73%; P = .0002), CD20 antibody therapy (70%; P = .0001), or CAR T-cell therapy (all seronegative; P = .0002).

Significantly lower titer levels were observed in patients who received CD20 antibody therapy vs the overall patient population, which underscored the susceptibility of patients receiving these treatments during the pandemic.

No statistically significant associations between age, ethnicity, time since immunosuppressive therapy, steroid use, or treatment within 48 hours of a vaccine dose, and seropositivity were reported.

Although all patients who were receiving CDK4/6 inhibitor treatment demonstrated positive anti-spike IgG test results, notably antibody titers were noted to be very low in this subset (n = 5), at a median of 1242 AU/mL vs a median of 6887 AU/mL in the overall cohort. Given the known involvement of the CDK4/6 pathway in immune activation, this might be biologically plausible and warrants further studies into the impact of CDK4/6 inhibitors on vaccine efficacy, the study authors noted.

A trend to lower titers were also reported among subsets of patients who received BCL-2 or BTK inhibitors.

Among a subset of 22 patients with cancer who had previously been infected with COVID-19, the seroconversion rate was 95%. Notably, antibody titers in those who had prior infection with the virus were found to be significantly higher than those who did not have a known prior infection, at a median of 46,737 AU/mL and a median of 5296 AU/mL, respectively (P < .001).

Our study, along with other emerging data, strongly highlights the continued need to vaccinate patients with a cancer diagnosis urgently and broadly, as vaccinations are likely to be highly effective, the study authors concluded. On the other hand, our study highlights at-risk cohorts of patients, in particular patients with hematologic malignancies following receipt of immunosuppressive therapies such as stem cell transplantation, anti-CD20 therapies, and CAR T-cell treatments. These cohorts of patients could potentially benefit from passive immunization with anti-COVID antibodies in the face of the ongoing pandemic.

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Study Calls for COVID-19 Vaccination in Patients With Cancer to Enable Optimal Treatment Delivery During Pandemic - OncLive

Animal Stem Cell Therapy Market Identify Key Drivers, Trends, Latest Innovations, Business Senario, Demand With Outlook by 2026 – The Market Writeuo -…

According to a new research report titled Animal Stem Cell Therapy Market Global Industry Perspective, Comprehensive Analysis And Forecast by 2021 2026

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The report provides revenue forecasts for global, regional and country levels. It also provides comprehensive coverage on major industry drivers, restraints, and their impact on market growth during the forecast period. For the purpose of research, The Report has segmented global Animal Stem Cell Therapy market on the basis of types, technology and region

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Key Competitors of the Global Animal Stem Cell Therapy Market are: MediVet Biologic, VETSTEM BIOPHARMA, J-ARM, Celavet, Magellan Stem Cells, U.S. Stem Cell, Cells Power Japan, ANIMAL CELL THERAPIES, Animal Care Stem, Cell Therapy Sciences, VetCell Therapeutics, Animacel, Aratana Therapeutics

The Global Animal Stem Cell Therapy Market Research Report is a comprehensive and informative study on the current state of the Global Animal Stem Cell Therapy Market industry with emphasis on the global industry. The report presents key statistics on the market status of the global Animal Stem Cell Therapy market manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry.

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Regional Animal Stem Cell Therapy Market (Regional Output, Demand & Forecast by Countries):- North America (United States, Canada, Mexico) South America ( Brazil, Argentina, Ecuador, Chile) Asia Pacific (China, Japan, India, Korea) Europe (Germany, UK, France, Italy) Middle East Africa (Egypt, Turkey, Saudi Arabia, Iran) And More.

The research report studies the past, present, and future performance of the global market. The report further analyzes the present competitive scenario, prevalent business models, and the likely advancements in offerings by significant players in the coming years.

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Treatment of AML in Older Patients Almost At ‘The Holy Grail’ – Curetoday.com

Recent drug approvals in the acute myeloid leukemia (AML) space have allowed older patients with the disease to continue receiving less intensive therapies than their younger counterparts, while also providing them with improved outcomes.

Were almost at the holy grail where (we) have lower intensity therapy thats well tolerated, but that the response rates are higher, said Dr. Tapan Kadia, an associate professor in the department of leukemia at The University of Texas MD Anderson Cancer Center in Houston, in an interview with CURE. Theyre not reaching (the 85% seen) with intensive chemotherapy, but (theyre) much better.

Kadia recently presented on the topic of treating older patients with AML during CUREs Educated Patient Leukemia Summit and highlighted how its vastly different than treating younger, and more fit patients.

He explained how the incidence of AML increases as a person gets older and that 60% of people who receive a diagnosis are aged older than 60 years.

Because more than half of the patient population with AML is aged older than 60 years, Kadia stressed that treatment approaches should be individualized for each patient. And, he said, factors such as their comorbidities, age, ability to tolerate chemotherapy and whether they could be candidate for a stem cell or bone marrow transplant in the future should play a role in selecting which treatment option they receive.

The major point to get across (is) to characterize a leukemia and to provide a therapy thats best for that patient long term, he said.

Although younger patients with the disease tend to receive more intensive chemotherapy regimens, it is tougher to administer those same regimens to older patients because it can wipe out their blood cell counts and it is difficult to deliver in a safe way, Kadia explained. In addition, older patients are more likely to have comorbidities such as heart disease, diabetes or COPD, which must also be managed in an intensive chemotherapy setting.

In years past, the lower intensity therapies were more tolerable, but they were not associated with great response rates. However, with the recent Food and Drug Administration approval of Venclexta (venetoclax), overall response rates increased to 65% and complete remission rates now are in the range of 35% to 40% when combined with drugs such as decitabine or azacitidine), Kadia explained. He said that these regimens are associated with very, very good outcomes with a median survival of 14.5 months, and the older patients tolerate it better.

Typically, patients who have AML and are in remission undergo a stem cell transplant or bone barrow transplant. However, for an older patient, a transplant is too risky. Regardless, Kadia noted that their remission should be maintained. Maintenance therapy is then used in this population to keep their disease in remission. Maintenance therapy usually consists of low-intensity, long-term treatment that can maintain the diseases response and help prevent the leukemia from returning.

What most people need to realize is that most subsets of AML are incurable, he said. That means (the disease) will typically try to come back if not treated aggressively and long term. And so this maintenance therapy allows people to maintain remission once they have achieved it.

We have really had a revolution in the treatment of AML, where previously we only had two or three drugs to treat AML and many people got intensive chemotherapy, Kadia concluded. We are fortunate now to live in an era where we have nine new drugs approved just in the last five years.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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Treatment of AML in Older Patients Almost At 'The Holy Grail' - Curetoday.com

8 Tips for Coping With a Diagnosis of PPMS – Everyday Health

Primary-progressive multiple sclerosis (PPMS) is known for being an especially debilitating form of multiple sclerosis in which disability steadily worsens over time, sometimes quickly. This form of MS affects about 10 to 15 percent of people diagnosed with multiple sclerosis.

Typically, people with PPMS do not have distinct periods of relapse or remission, as is the case with more common form of MS, relapsing-remitting MS.

While there are approaches for controlling the symptoms of PPMS, treatments for the condition are limited: Only one medication, Ocrevus (ocrelizumab), has been approved by the U.S. Food and Drug Administration (FDA) for slowing the progression of PPMS.

RELATED: 10 Essential Facts About Primary-Progressive MS

Because PPMS is progressive and often disabling, receiving a diagnosis can be traumatic.

Beth Broun, a New York City native who now lives upstate, received her diagnosis 10 years ago. The 56-year-old first noticed something was wrong when her left leg began feeling weak, and she started tripping over her left foot.

My husband and I were walking on the Upper West Side [of Manhattan], and my left foot completely flopped, Broun says.

Her primary care physician sent her to a neurologist who was not an MS expert. He advised her to get physical therapy, but after six months nothing had changed.

It took her two years before she consulted another neurologist who ordered a brain scan and spotted the tell-tale signs of MS.

RELATED: Advances in Diagnosing Multiple Sclerosis

I freaked out, says Broun. She called her husband and told him the news and then did what she thought was the next most essential thing: I went to Bergdorf Goodman, and I bought a $3,500 handbag, because thats what you do when youre diagnosed.

After getting over the initial shock of the diagnosis, Broun started the gradual process of coming to terms her condition and then creating a plan to deal with the symptoms. Here are some tips that can help anyone who is newly diagnosed with PPMS.

As director of the MS Comprehensive Care Center at Stony Brook Neurosciences Institute, Patricia Coyle, MD,treats patients with PPMS and understands why a diagnosis is especially traumatic.

As you learn more about the disease, you realize that primary-progressive MS is a more significant form, where there is basically inevitable disability, says Dr. Coyle.

Coyle stresses, however that an education program is extremely important, because it can reduce fears. The more people know, the better they are able to deal with the illness and to find the information they need about therapies and disease progression that can provide them with the hope to go on and develop coping skills to live with the illness.

Coyle says that people need to know that they can clinically stabilize, sometimes for up to several years, and they can even see some improvement its in the minority, but that can occur.

Because the pace of worsening of PPMS doesnt tend to change over time, people who have it have some idea of what to expect from year to year.

Still, its important to have medical providers who can talk with you about what youre experiencing.

I think you want to have a relationship with your healthcare providers where you are free to ask any questions that you have, Coyle says.

A good starting point to find answers to commonly asked questions about PPMS is the National Multiple Sclerosis Society (NMSS). The website of the Multiple Sclerosis Association of America is another good place to find authoritative information about the various types of MS, including PPMS.

But, Broun cautions, dont automatically trust all of the advice or assertions about MS that you might read online, even when a person appears to write with great authority. Some of what she read along the way convinced her that she might go blind or lose her ability to speak. Having a medical provider who can help you sort out whats real and whats not can be a big help in these situations.

Along these lines, Coyle cautions about internet scams that promise miracle cures and prey on desperate patients.

If you read, We have stem cells that will improve your MS; you only need to pay $20,000, that's not legitimate, she says. There is no documented stem cell treatment for MS at this point in time.

Having PPMS doesnt mean you have to stop working. Its symptoms and progression are unique to each individual, so the illness doesnt have to mean an end to career.

In an article for Roche Pharmaceuticals, professor Jrme de Seze, PhD, the department head of the Neurology and Clinical Investigation Centre at the University of Strasbourg in France, wrote that most PPMS patients he deals with are keen to keep working.

Coyle emphasizes that a diagnosis doesnt have to be a career-ender. While this is a more severe form of MS, people can still continue to function for prolonged periods of time, she says.

Because some of the symptoms such as fatigue and cognitive impairment may limit ones ability to work, an individual may seek to work part-time.

The NMSS provides a legal guide for people with MS that addresses many workplace-related questions, such as "Am I obligated to tell my employer about my MS diagnosis?" and "Can I be let go from my job because of my MS?"

Work situations are always going to vary, but Coyle notes that some employers may be sympathetic.

An informed employer could actually be potentially very helpful with regard to accommodations, she says.

RELATED: 6 Top Tech Recommendations for Primary-Progressive MS

Broun says that the No. 1 thing to do is to put together a trusted health support team. This can include a neurologist, primary care provider, physical therapist, and mental health professional.

In Brouns case, it took some trial and error to find an MS care provider who felt right for her. But after consulting with different specialists, Broun found that Saud Sadiq, MD, a neurologist at the Tisch MS Research Center in New York City, provides guidance and treatment options that she believes in.

RELATED: Shared Decision-Making for MS Treatment

A mental health professional can be a key member of your health support team.

Its not uncommon for patients early in diagnosis to go through an adjustment period of anxiety and some depression when they hear they have MS, says Laura Safar, MD, an assistant professor of psychiatry at Brigham and Womens Hospital in Boston and a psychiatrist who treats psychiatric disorders among people with multiple sclerosis. Patients may have fears about their prognosis, and questions about what their progression will be like.

Individuals cope in different ways, Dr. Safar says. Some of them can be very proactive and want to learn about everything they can do to improve their health. Others can be overwhelmed, and avoid dealing with the diagnosis and treatment.

Safar says that it can be helpful for clinicians to observe how different patients cope with their illness to understand what type of support or interventions they need in addition to educating them about the diagnosis and next steps for treatment.

Doctors and patients alike should be aware that PPMS can affect a persons cognition, or their ability to think, remember, and perform other mental tasks.

Depending on a persons needs, appropriate interventions may include a neuropsychological evaluation to assess cognition, or a referral to a psychotherapist or psychiatrist to focus on emotional and coping issues. Mental health therapy may involve both talking through problems and taking antidepressants.

Rehabilitation therapies including physical, occupational, and cognitive rehabilitation can be tools to sustain and improve a patient's cognitive and functional abilities.

From a psychological perspective, rehabilitationstrategies, when combined with disease-modifyingtreatment, can assist in providing a sense of mastery over their illness, she says.

Ultimately, Safar says that her goal is to help individuals figure out how do I make this illness a part of my life, but not let it define the whole of who I am and what my life is about.

Safar notes that ideally, friends and family members should be part of the support network a person with MS relies on.

I want to make sure that people are not isolated and not alone, she says. So I find out if they have friends or family they can talk to about their diagnosis.

Safar recognizes that for some it can be difficult to even share they are ill. They are concerned that a partner may not be supportive or may turn away. Or they fear that they will cause too much worry among family members. They want to protect their loved ones and their relationships, she says.

Because she firmly believes that patients should not take this on alone, she advises some to take gradual steps toward disclosing information about the diagnosis and see how people close to them react.

RELATED: How MS Contributes to Isolation and What You Can Do to Stay Connected

In addition to seeking emotional support from friends and family, people with PPMS may also want to connect with one or more online groups.

Through participating in several MS groups on Facebook, Broun developed a deep friendship with another person with PPMS who lives in the Netherlands, and they each push the other to be proactive about their illness.

We say were going to fight each other; were going to outrun each other, she jokes. Hes a force.

For those looking for online support and connection, Broun suggests exploring Moodify, the PPMS Facebook page, as well as the International Progressive MS Alliance.

While looking after your mental state is vital to living with PPMS, so is doing your best to maintain good physical health. Broun gets around nowadays with a walker. She says its brutal to see herself in the mirror that way, but without exercise, she might not even be walking.

Exercise is the No. 1 thing to me, says Broun.

When she was first diagnosed, Broun began working with a physical therapist to maintain mobility and function. She started doing leg presses and walking on an uphill treadmill for 10 minutes to build strength in her glutes, hip flexors, and hamstrings.

Currently, she meets with a personal trainer two times a week to work on her muscle strength and balance, which she describes as incredibly weak. Because Broun has foot drop (difficulty lifting the front part of her foot), she does special exercises to keep her foot up.

My favorite exercise, which may not be specific forMS but makes me feel dynamite, is riding a spin bike, says Broun. You would never know I have a MS when Im riding it, but as soon as I get off I can barely walk. I love the confidence and ego boost I get from it.

On her visits with Dr. Sadiq, she also works with a new therapist on a special robotic machine, which helps someone like me use muscles I havent used in years.

To get started with exercise on your own, the Multiple Sclerosis Trustprovides a series of strength, stretching, balance, and breathing exercises for people with MS to explore and use to build their own routine for physical activity.

Following a healthy diet is also important for maintaining strength and stamina. For Broun, this means sticking to a Mediterranean-type diet, primarily consisting of organic proteins and vegetables.

At the end of the day, says Broun, I can control my exercising, and I can control what I can eat to feel good otherwise, MS is an animal, and its going to take over my body.

Coyle recommends exploring the many MS apps on the market for tracking symptoms and other purposes.

Apps have a number of different functions they can help track activities, track what youre doing in terms of exercise, and enable you to easily look up information related to MS, she says.

The Multiple Sclerosis Clinical Care App, for example, contains current information on the diagnosis, classification, and management of multiple sclerosis (MS), concisely presented for use at the point of care.

Several appsare available that allow you to track symptoms, notes TeamScope. My MS Manager, created and supported by the Multiple Sclerosis Association of America, is one such example.

The National MS Society recommends Happy the App, a 24/7 phone-based emotional support service via mobile app that connects individuals experiencing everyday stresses, struggles, anxiety, or loneliness with exceptionally compassionate listeners.

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8 Tips for Coping With a Diagnosis of PPMS - Everyday Health

More positive results from CRISPR trial for sickle cell and thalassaemia – BioNews

16 August 2021

A genome editing-based gene therapy forblood diseasessickle cell disease andbeta-thalassaemia continues to be effective more than two years after treatment.

The clinical trial for the therapy, named CTX001, previously reported good preliminary results (see BioNews 1052) and new data was presented at the European Haematology Association 2021 Virtual Congress.

'We are hearing that it is life-changing.' said Professor Stephan Grupp from the Cell and Gene Therapy Laboratory at Children's Hospital of Philadelphia, one of the researchers collaborating on the trial.

Patients with sickle cell disease or beta-thalassaemia carry a mutation in a single gene that causes problems in an essential blood molecule called haemoglobin. Patients usually require lifelong blood transfusions, sometimes stem cell transplants, as well as ongoing pain management. Sickle cell disease can also impact reproductive health in women (see BioNews 1105).

This clinical trial involved taking a patient's own blood stem cells, called hematopoietic stem cells, and editing them outside the body.CRISPR/Cas9 genome editing wasused to reactivatea different haemoglobin gene that is usually only expressed in the fetus and is switched off at birth. The editedstem cells were transplanted back into thepatients, who started producing fetal haemoglobin,replacingthe nonfunctional haemoglobin that causeddisease symptoms.

'The data presented today in 22 patients are impressive in both the consistency and durability of effect. These results add to the growing body of evidence that CTX001 may hold the promise for a one-time functional cure for sickle cell disease and beta-thalassemia.' said Dr Reshma Kewalramani, CEO of Vertex Pharmaceuticals in Boston, Massachusetts, who developed the treatment in partnership with CRISPR Therapeutics from Zug, Switzerland.

Aftertwo years post-gene therapy, the sickle cell disease patients were reported to be free of vaso-occlusive crises; a painful organ injury occurring when blood cells cause blockages, common to sickle cell disease patients. None of the beta-thalassaemia patients has required any further blood transfusions as all have started producing functional levels of fetal haemoglobin in their blood.

'The evidence so far indicates that it is durable in the time frame we've seen, and we just have to continue to follow the patients' said Professor Grupp.

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More positive results from CRISPR trial for sickle cell and thalassaemia - BioNews