Parents of Boy with Brain Disorder That’s ‘100 Percent Fatal’ Raise Money for Gene Therapy Clinical Trial

gofundme Connor

The parents of a boy with a rare, fatal brain disorder are raising money so he can participate in a gene therapy trial that has potential to stop the disease.

Marisa DiChiacchio and Mike Dobbyn’s 12-year-old son Connor has Sanfilippo Syndrome Type C, which is a rare degenerative brain disorder that is “like Alzheimer’s, but in children,” according to a GoFundMe campaign created earlier this month.

According to the campaign, the syndrome is “100 percent fatal” and has no cure.

“As the degeneration rapidly progresses, Connor will stop walking, stop talking, stop being able to feed himself,” the GoFundMe campaign says. “He’ll develop movement disorders and seizures, suffer severe dementia, endure a lot of pain and suffering, and then he’ll die.”

But Connor’s family is raising money for the Cure Sanfilippo Foundation and a gene therapy clinical trial that needs funding at UT Southwestern in Dallas, Texas.

gofundme Marisa DiChiacchio, Mike Dobbyn and their sons Keenan and Connor

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“Research for this gene therapy has shown in pre-clinical models that it can stop the disease,” the campaign says. “This clinical trial is the crucial step of determining whether earlier research is able to show the same therapeutic benefits in children.”

“It was like a bomb was dropped on us in the geneticist office,” Connor’s dad told CBS Philadelphia of the moment they learned of his diagnosis. “I just remember it was a life-shortening condition, the geneticist actually told us at the time, ‘Don’t Google this yet because we don’t have a 100 percent conviction on this diagnosis yet.’ “

gofundme Marisa DiChiacchio, Mike Dobbyn, Connor

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“We knew Connor obviously was special and loved and adored by so many people, but sometimes you just don’t realize until something devastating like this comes out,” Connor’s mother added. “And just everybody comes out of the woodwork trying to help and wanting to help. So he’s like our local community rock star.”

As of Wednesday, the campaign had raised $172,210 of its $3,000,000 goal. 

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Affimed and NKMax America to Study the Combination of AFM24, an EGFR-Targeted Innate Cell Engager, with SNK01 Natural Killer Cell Therapy

  • Proof of Concept study to establish safety and recommended dose of Affimed’s innate cell engager (ICE®) AFM24 in combination with NKMax America’s Natural Killer (NK) cells in solid tumors

  • Pre-clinical data substantiates synergy between Affimed’s ICE® molecules and both NKMax America’s autologous and cryopreserved allogeneic NK cell therapy products

Heidelberg, Germany, and Santa Ana, California, October 20, 2020 – Affimed N.V. (NASDAQ: AFMD) and NKMax America Inc., both clinical stage biotech companies focused on harnessing the power of the body’s innate immune system, announced today that they entered into a clinical collaboration agreement to investigate the combination of AFM24, a CD16A/EGFR-targeted ICE®, with the autologous NK cell product SNK01. Pursuant to the collaboration, the companies plan to explore the combination in a first-in-human proof-of-concept (POC) trial in patients with EGFR-expressing tumors. The agreement follows a previous collaboration between the two companies in the preclinical setting to better understand the combined activity of their respective platforms. The results of the preclinical collaboration have shown substantive synergy between Affimed’s ICE® molecules and NKMax America’s autologous and cryopreserved allogeneic natural killer cell products.

Under the agreement, the companies will contribute their respective product candidates and resources towards submitting an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) and a subsequent clinical trial. The clinical trial will combine NKMax America’s SNK01 (enhanced natural killer cells) with AFM24 in the autologous setting with the option to expand the clinical trial to the allogeneic setting. The cost of the clinical study will be shared by Affimed and NKMax America. The agreement also provides for the opportunity to pursue further clinical study combinations with additional product candidates from both parties.

NKMax America has developed a proprietary NK cell expansion and activation technology platform which allows it to produce unprecedented commercial amounts of autologous and allogeneic NK cells from numerous donors that have near total expression of activating receptors like CD16A, NKG2D, NKp30 and NKp46. In addition, its unique technology increases the cytotoxicity of the expanded NK cells by nearly 8000 percent. In addition, the SNK01 product does not require lymphodepletion or cytokine support.

Using its ROCK® (Redirected Optimized Cell Killing) platform, Affimed has developed a novel pipeline of ICE® products. AFM24, a tetravalent, bispecific epidermal growth factor receptor (EGFR)- and CD16A-binding ICE®, is unique due to its activation of innate immunity to kill solid tumors, inducing both antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), whereas other EGFR-directed therapies rely heavily on signal transduction inhibition. A first-in-human Phase 1/2a open-label, non-randomized, multi-center, multiple ascending dose escalation/expansion study is underway evaluating AFM24 as monotherapy in patients with advanced solid EGFR-expressing malignancies whose disease has progressed after treatment with previous anticancer therapies.

“We believe combining ICE® molecules generated from our ROCK® platform with adoptive NK cell transfer can improve patient outcomes by ensuring patients have active and viable innate cells to be directed to the tumor and induce cytotoxic killing. In addition,

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Testosterone Replacement Therapy Market | Market to Witness Increased Demand for Injectables Through 2024

The testosterone replacement therapy market size is set to grow by USD 113.54 million during the period spanning over 2020-2024. One of the key factors driving growth is the rising initiatives to increase awareness about the disease. The rising incidence of hypogonadism has led many organizations across the world to introduce awareness campaigns and encourage early diagnosis of the condition. The rise in novel therapeutic approaches is a significant trend that will further stimulate market growth. Vendors in the market are increasing their R&D efforts to develop new and alternative treatment options that are safer for patients.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201019005262/en/

Technavio has announced its latest market research report titled Global Testosterone Replacement Therapy Market 2020-2024 (Graphic: Business Wire)

To learn more about the global trends impacting the future of market research, download a free sample now

Parent Market Analysis

Technavio categorizes the global testosterone replacement therapy market as a part of the pharmaceutical market within the overall healthcare industry. The parent pharmaceuticals market covers products and companies engaged in research and development (R&D) or production of generic drugs, non-generic drugs, and veterinary drugs. Growth in the healthcare market will be driven by the increasing life expectancy of individuals worldwide.

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Testosterone Replacement Therapy Market: Geographic Segmentation

The report segments the market by geography: Asia, Europe, North America, and ROW. About 53% of the market’s growth will originate from North America during the forecast period. This is due to the rising prevalence and awareness about the treatment for conditions such as hypogonadism, testosterone deficiency, and erectile dysfunction. The US is the key market for testosterone replacement therapy in North America.

Testosterone Replacement Therapy Market: Segmentation by Product

The injectables segment was leading the market in 2019. The segment is mainly driven by the availability of patient assistance programs and direct administration benefits. Also, the increasing prevalence of conditions such as obesity, diabetes, dyslipidemia, and metabolic syndrome is fueling the growth of the segment. This report provides an accurate prediction of the contribution of all the segments to the growth of the testosterone replacement therapy market size.

Testosterone Replacement Therapy Market: Growth Drivers

The market is driven by the rising initiatives to increase awareness about diseases among the population. The increasing prevalence of conditions such as hypogonadism has led many organizations across the world to undertake initiatives to educate people about available treatment options. For instance, the Endocrine Society undertakes initiatives to increase awareness about hypogonadism among medical professionals by releasing clinical practice guidelines on testosterone therapy. Similarly, organizations such as the Testicular Cancer Awareness Foundation, Endocrine Awareness Center for health, and the American Urological Association are educating people about the diagnosis and treatment for hypogonadism. This is driving a significant number of people to proactively approach fertility care centers and medical practitioners

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Experimental therapy could remove need for insulin

Scientists have proposed a new therapy for type 2 diabetes. If proven effective, the therapy could help some people discontinue insulin treatment.

Scientists have proposed a new therapy for the treatment of type 2 diabetes, with a proof-of-concept study showing positive initial results. If effective, the therapy may mean that some people can stop taking insulin treatment.

The authors of the research presented their findings at UEG Week Virtual 2020, a conference organized by United European Gastroenterology, a professional nonprofit organization for specialists in digestive health.

According to the National Institute of Diabetes and Digestive and Kidney Diseases, a person may have type 2 diabetes when their blood sugar is too high.

People gain blood sugar, or blood glucose, mainly from the food they eat. Insulin helps cells access this glucose to use as energy. However, for a person with type 2 diabetes, either their body does not make enough insulin or their cells do not respond to insulin correctly.

This then means that the glucose in their blood increases, which can lead to complications of diabetes, such as heart and kidney disease, visual impairment, and loss of sensation in the limbs. The higher the blood glucose over time, the higher the risk of these complications.

According to the Centers for Disease Control and Prevention (CDC), about 1 in 10 adults in the United States have diabetes, and 90–95% of these individuals have type 2 diabetes.

Doctors typically recommend lifestyle changes, such as being more physically active and eating a more healthful diet, to treat type 2 diabetes, as well as medications to manage a person’s blood pressure and blood glucose levels.

Insulin treatment may be necessary if a person is unable to maintain their blood sugar at normal levels. This treatment can take the form of injections, pens, pumps, or inhalers. It encourages the cells in a person’s body to absorb more blood sugar.

However, people’s perception of the side effects of insulin treatment can be quite pronounced. As a result, doctors may be less likely to prescribe insulin, and, when they do, people may not take it regularly.

Consequently, therapies that can avoid these perceived side effects may be valuable in ensuring that people keep up with their prescribed treatment and avoid risking serious health issues.

In this context, the researchers behind the present study used a novel technique that scientists first reported using in humans in 2016. Based on those preliminary results, it seemed promising.

The technique is called duodenal mucosal resurfacing (DMR). The duodenum is the first part of a person’s small intestine. DMR involves lifting the mucosal layer of the duodenal to allow the ablation of the revealed area using heated water — a process that removes the cells in the targeted area.

The researchers who developed the DMR technique were trying to replicate the positive impact that bariatric surgery (gastric bypass) has on blood sugar levels with a less invasive technique.

Studies of how bariatric surgery improves blood sugar control have concluded that there

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EMA OKs First CAR T-Cell Therapy for Mantle Cell Lymphoma

The European Medicines Agency (EMA) today recommended granting conditional marketing authorization to brexucabtagene autoleucel (Tecartus), making it the first approved chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) in the European Union.

Brexucabtagene autoleucel is the third CAR T-cell therapy to be recommended for approval in Europe, but the only one for this indication.

The agent was approved for the same use in the United States earlier this year and was described by one expert as representing a “new frontier” in the treatment of MCL.

The new agent addresses an unmet need in MCL for patients who relapse or progress despite available therapies.

The current standard of care for this cancer includes stem cell transplantation and various therapy regimens, including Bruton’s tyrosine kinase (BTK) inhibitors, all of which are often initially effective. However, patients commonly relapse or stop responding to treatment, according to the EMA.

“This opinion is an important milestone for patients in Europe living with relapsed or refractory mantle cell lymphoma,” said Ken Takeshita, MD, global head of clinical development at Kite, the agent’s manufacturer, in a press statement.

It is based on safety and efficacy results from the multicenter, single-arm ZUMA-2 trial in 74 adult patients with refractory or relapsed MCL who had received at least two prior therapies.

During the study’s 12-month follow-up period, 84% of patients had a partial response and 59% had a complete response.

The most common side effects are cytokine release syndrome, infections, and encephalopathy. Monitoring and mitigation strategies for these side effects are described in the product information and the agent’s risk management plan.

Further efficacy and safety data are being collected as part of long-term follow-up from the pivotal study and an additional registry-based study.

Brexucabtagene autoleucel was supported through EMA’s Priority Medicines (PRIME) initiative, which provides early and enhanced scientific and regulatory support to medicines that have the potential to address unmet needs.

Nick Mulcahy is an award-winning senior journalist for Medscape. He previously freelanced for HealthDay and MedPageToday, and had bylines in WashingtonPost.com, MSNBC, and Yahoo. Reach him by email and follow him on Twitter.

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Physical Therapy and Sports Medicine – Know the Differences

It isn’t unusual to use the terms sports medicine and physical therapy interchangeably. However the two aren’t exactly the same things even though you may find yourself the patient of both fields at the same time.

While compatible, the difference between the two branches of medical discipline is clear. Both are effective for treating injuries, diseases and disorders of the muscle and skeletal systems of the body and both are useful for the prevention of future injury or disease symptom reoccurence. By taking an individual look at both types of medical assistance, you will soon see the differences between physical therapy and sports medicine.

Let’s take a look at the field of physical therapy first. Unlike sports medicine, physical therapy as a whole is devoted to correcting any injury, disease, or disorder of the bones and muscles that can be treated with non invasive techniques. The focus is to provide patients with relief from pain, improve their muscle, joint, and bone function, while providing techniques the patient can use on their own for additional healing. The primary tools of a therapist are good diagnosing and evaluating skills, knowledge of the musculoskeletal system, and knowledge of which therapy is effective for each situation.

One large difference between the two is that physical therapy doesn’t only deal with sports related injuries or problems. Physical therapists can choose to specialize in their careers with emphasis on things like pediatrics, geriatrics, and neurology. Other areas of expertise included in physical therapy are sports, cardiovascular sciences, and occupational therapy.

When you are looking at the differences between these two healing arts you certainly can’t overlook sports medicine. The type of medical discipline is solely used for sports related injuries and disorders. While sports physical therapy techniques are commonly used in addition to sports medicine, sports medicine may involve surgeries, procedures, and medications not used in physical therapy. Sports medicine practitioners also can have specialty fields as well. Orthopedics and skeletal emphasis are common. Advanced study of how long term sporting activities and treating sports related injuries are always a part of this particular discipline.

Understanding the difference between physical therapy and sports medicine may be key to determining how to best treat your physical problem. While they can and are commonly used together, each one has its own distinct benefit. Your medical care team can further help distinguish the two and point you in the right direction in terms of your health care needs. The important thing is take care of any injury, sports related or not, to ensure your body’s function in the years to come.

The difference between these two medical approaches is obvious. Although the two are often used together, each one has its own purpose and uses. Your doctor can give you advice on which specialist is right for your needs.

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