Source: The New York Times
A new treatment using stem cells that produce insulin has surprised experts and given them hope for the 1.5 million Americans living with the disease.
Brian Shelton’s life was ruled by Type 1 diabetes.
When his blood sugar plummeted, he would lose consciousness without warning. He crashed his motorcycle into a wall. He passed out in a customer’s yard while delivering mail. Following that episode, his supervisor told him to retire, after a quarter century in the Postal Service. He was 57.
His ex-wife, Cindy Shelton, took him into her home in Elyria, Ohio. “I was afraid to leave him alone all day,” she said.
Early this year, she spotted a call for people with Type 1 diabetes to participate in a clinical trial by Vertex Pharmaceuticals. The company was testing a treatment developed over decades by a scientist who vowed to find a cure after his baby son and then his teenage daughter got the devastating disease.
Mr. Shelton was the first patient. On June 29, he got an infusion of cells, grown from stem cells but just like the insulin-producing pancreas cells his body lacked.
Now his body automatically controls its insulin and blood sugar levels.
Mr. Shelton, now 64, may be the first person cured of the disease with a new treatment that has experts daring to hope that help may be coming for many of the 1.5 million Americans suffering from Type 1 diabetes.
“It’s a whole new life,” Mr. Shelton said. “It’s like a miracle.”
Diabetes experts were astonished but urged caution. The study is continuing and will take five years, involving 17 people with severe cases of Type 1 diabetes. It is not intended as a treatment for the more common Type 2 diabetes.
“We’ve been looking for something like this to happen literally for decades,” said Dr. Irl Hirsch, a diabetes expert at the University of Washington who was not involved in the research. He wants to see the result, not yet published in a peer-reviewed journal, replicated in many more people. He also wants to know if there will be unanticipated adverse effects and if the cells will last for a lifetime or if the treatment would have to be repeated.
But, he said, “bottom line, it is an amazing result.”
Dr. Peter Butler, a diabetes expert at U.C.L.A. who also was not involved with the research, agreed while offering the same caveats.
“It is a remarkable result,” Dr. Butler said. “To be able to reverse diabetes by giving them back the cells they are missing is comparable to the miracle when insulin was first available 100 years ago.”
And it all started with the 30-year quest of a Harvard University biologist, Doug Melton.
‘A Terrible, Terrible Disease’
Dr. Melton had never thought much about diabetes until 1991 when his 6-month-old baby boy, Sam, began shaking, vomiting and panting.
“He was so sick, and the pediatrician didn’t know what it was,” Dr. Melton said. He and his wife Gail O’Keefe rushed their baby to Boston Children’s Hospital. Sam’s urine was brimming with sugar — a sign of diabetes.
The disease, which occurs when the body’s immune system destroys the insulin-secreting islet cells of the pancreas, often starts around age 13 or 14. Unlike the more common and milder Type 2 diabetes, Type 1 is quickly lethal unless patients get injections of insulin. No one spontaneously gets better.
“It’s a terrible, terrible disease,” said Dr. Butler at U.C.L.A.
Patients are at risk of going blind — diabetes is the leading cause of blindness in this country. It is also the leading cause of kidney failure. People with Type 1 diabetes are at risk of having their legs amputated and of death in the night because their blood sugar plummets during sleep. Diabetes greatly increases their likelihood of having a heart attack or stroke. It weakens the immune system — one of Dr. Butler’s fully vaccinated diabetes patients recently died from Covid-19.
Added to the burden of the disease is the high cost of insulin, whose price has risen each year.
The only cure that has ever worked is a pancreas transplant or a transplant of the insulin-producing cell clusters of the pancreas, known as islet cells, from an organ donor’s pancreas. But a shortage of organs makes such an approach an impossibility for the vast majority with the disease.
“Even if we were in utopia, we would never have enough pancreases,” said Dr. Ali Naji, a transplant surgeon at the University of Pennsylvania who pioneered islet cell transplants and is now a principal investigator for the trial that treated Mr. Shelton.
For Dr. Melton and Ms. O’Keefe, caring for an infant with the disease was terrifying. Ms. O’Keefe had to prick Sam’s fingers and feet to check his blood sugar four times a day. Then she had to inject him with insulin. For a baby that young, insulin was not even sold in the proper dose. His parents had to dilute it.
“Gail said to me, ‘If I’m doing this you have to figure out this damn disease,’” Dr. Melton recalled. In time, their daughter Emma, four years older than Sam, would develop the disease too, when she was 14.
Dr. Melton had been studying frog development but abandoned that work, determined to find a cure for diabetes. He turned to embryonic stem cells, which have the potential to become any cell in the body. His goal was to turn them into islet cells to treat patients.
One problem was the source of the cells — they came from unused fertilized eggs from a fertility clinic. But in August 2001, President George W. Bush barred using federal money for research with human embryos. Dr. Melton had to sever his stem cell lab from everything else at Harvard. He got private funding from the Howard Hughes Medical Institute, Harvard and philanthropists to set up a completely separate lab with an accountant who kept all its expenses separate, down to the light bulbs.
Over the 20 years it took the lab of 15 or so people to successfully convert stem cells into islet cells, Dr. Melton estimates the project cost about $50 million.
The challenge was to figure out what sequence of chemical messages would turn stem cells into insulin-secreting islet cells. The work involved unraveling normal pancreatic development, figuring out how islets are made in the pancreas and conducting endless experiments to steer embryonic stem cells to becoming islets. It was slow going.
After years when nothing worked, a small team of researchers, including Felicia Pagliuca, a postdoctoral researcher, was in the lab one night in 2014, doing one more experiment.
“We weren’t very optimistic,” she said. They had put a dye into the liquid where the stem cells were growing. The liquid would turn blue if the cells made insulin.
Her husband had already called asking when was she coming home. Then she saw a faint blue tinge that got darker and darker. She and the others were ecstatic. For the first time, they had made functioning pancreatic islet cells from embryonic stem cells.
The lab celebrated with a little party and a cake. Then they had bright blue wool caps made for themselves with five circles colored red, yellow, green, blue and purple to represent the stages the stem cells had to pass through to become functioning islet cells. They’d always hoped for purple but had until then kept getting stuck at green.
The next step for Dr. Melton, knowing he’d need more resources to make a drug that could get to market, was starting a company.
Moments of Truth
His company Semma was founded in 2014, a mix of Sam and Emma’s names.
One challenge was to figure out how to grow islet cells in large quantities with a method others could repeat. That took five years.
The company, led by Bastiano Sanna, a cell and gene therapy expert, tested its cells in mice and rats, showing they functioned well and cured diabetes in rodents.
At that point, the next step — a clinical trial in patients — needed a large, well financed and experienced company with hundreds of employees. Everything had to be done to the exacting standards of the Food and Drug Administration — thousands of pages of documents prepared, and clinical trials planned.
Chance intervened. In April 2019, at a meeting at Massachusetts General Hospital, Dr. Melton ran into a former colleague, Dr. David Altshuler, who had been a professor of genetics and medicine at Harvard and the deputy director of the Broad Institute. Over lunch, Dr. Altshuler, who had become the chief scientific officer at Vertex Pharmaceuticals, asked Dr. Melton what was new.
Dr. Melton took out a small glass vial with a bright purple pellet at the bottom.
“These are islet cells that we made at Semma,” he told Dr. Altshuler.
Vertex focuses on human diseases whose biology is understood. “I think there might be an opportunity,” Dr. Altshuler told him.
Meetings followed and eight weeks later, Vertex acquired Semma for $950 million. With the acquisition, Dr. Sanna became an executive vice president at Vertex.
The company will not announce a price for its diabetes treatment until it is approved. But it is likely to be expensive. Like other companies, Vertex has enraged patients with high prices for drugs that are difficult and expensive to make.
Vertex’s challenge was to make sure the production process worked every time and that the cells would be safe if injected into patients. Employees working under scrupulously sterile conditions monitored vessels of solutions containing nutrients and biochemical signals where stem cells were turning into islet cells.
Less than two years after Semma was acquired, the F.D.A. allowed Vertex to begin a clinical trial with Mr. Shelton as its initial patient.
Like patients who get pancreas transplants, Mr. Shelton has to take drugs that suppress his immune system. He says they cause him no side effects, and he finds them far less onerous or risky than constantly monitoring his blood sugar and taking insulin. He will have to continue taking them to prevent his body from rejecting the infused cells.
But Dr. John Buse, a diabetes expert at the University of North Carolina who has no connection to Vertex, said the immunosuppression gives him pause. “We need to carefully evaluate the trade-off between the burdens of diabetes and the potential complications from immunosuppressive medications.”
Mr. Shelton’s treatment, known as an early phase safety trial, called for careful follow-up and required starting with half the dose that would be used later in the trial, noted Dr. James Markmann, Mr. Shelton’s surgeon at Mass General who is working with Vertex on the trial. No one expected the cells to function so well, he said.
“The result is so striking,” Dr. Markmann said, “It’s a real leap forward for the field.”
Last month, Vertex was ready to reveal the results to Dr. Melton. He did not expect much.
“I was prepared to give them a pep talk,” he said.
Dr. Melton, normally a calm man, was jittery during what felt like a moment of truth. He had spent decades and all of his passion on this project. By the end of the Vertex team’s presentation, a huge smile broke out on his face; the data were for real.
He left Vertex and went home for dinner with Sam, Emma and Ms. O’Keefe. When they sat down to eat, Dr. Melton told them the results.
“Let’s just say there were a lot of tears and hugs.”
For Mr. Shelton the moment of truth came a few days after the procedure, when he left the hospital. He measured his blood sugar. It was perfect. He and Ms. Shelton had a meal. His blood sugar remained in the normal range.
Mr. Shelton wept when he saw the measurement.
“The only thing I can say is ‘thank you.’”
HALF OF ADULTS SUFFER ANXIETY, REVEALS NEW RESEARCH
Leading Experts Identify ‘Epidemic Of Anxiety’ and Call for Empowerment Tools
New research reveals that half (48%) of all adults in the UK experience anxiety, equating to nearly 26 million people.[i] Given the widespread prevalence of this mental health condition, leading health experts have identified an ‘epidemic of anxiety’.
Commenting in a new ‘Empowerment in the Epidemic of Anxiety Report’,[ii] the panel of community and industry health experts highlighted the growing burden of mental health issues on an already overstretched NHS. They conclude that the way forward must include empowering and supporting those with mild anxiety to develop coping strategies.
Surge of Anxiety
The UK has seen a marked rise in generalised anxiety over the past decade, especially among younger people and women.[iii] Over 8 million people (around 1 in 10) are living with a diagnosable anxiety disorder at any one time,[iv] but this may not paint the whole picture. New research commissioned by Kalms Herbal Remedies shows that half (48%) the adult population often suffers from anxiety at a level that impacts their day-to-day living.i
Dr Sarah Jarvis, general practitioner, clinical consultant and expert panellist, comments: “We have a perfect storm of issues in relation to anxiety. The figures for people suffering with anxiety have increased considerably during the pandemic. To add to the problem, waiting lists for mental health services have increased further. This lack of availability of service inevitably has a huge impact on general practice as well – people with anxiety consult their GP more often, and I have found it increasingly difficult to be able to offer services for people at the milder end of the anxiety and depression spectrum.”
To help prevent mild anxiety from progressing, and to improve the quality of people’s lives sooner, the expert panel recommend that people utilise a ‘mild anxiety toolbox’ – a series of powerful, evidence-based tools that can be employed into everyday life.
Talking therapies are a mainstay for many mental health concerns, including anxiety. Several of the methods used in formal talking therapies can be adapted and learned as a form of self-help therapy without the assistance of a professional.[v] For example, CBT-inspired self-help strategies and techniques such as reframing helpful thoughts, tackling worries and facing fears can be practiced at home.
Complementary therapies or practices can be integrated into everyday life to either help ease mild anxiety symptoms or to help prevent anxiety altogether. For example, mindfulness can work well alongside yoga and hypnotherapy. Developing an awareness of one’s thoughts, feelings and behaviours can help to break negative habits and improve self-esteem. vi,[vi]i
Lifestyle changes such as getting more sleep, eating a balanced diet, limiting caffeine and alcohol, and exercising can go a long way toward easing anxiety. For example, just 30 minutes or more of exercise a day for three to five days a week can significantly improve anxiety symptoms.[vii]i
Numerous de-stressing and mood-boosting apps are available to help reduce anxiety. A good place to start is the ‘Hub of Hope’ app – the UK’s leading mental health support database, which brings local, national, peer, community, charity, private and NHS mental health services together in one place. Some mild anxiety sufferers may also benefit from group discussions; organisations offering a range of resources include Anxiety UK, SANE and Mind.
Many people rely on herbs to help with mild to moderate mental health conditions, including anxiety, whilst avoiding the unwanted and negative side effects of medications. For example, studies have shown reductions in cortisol levels and anxiety following the use of ashwagandha.ix Lavender oil contains terpenes, such as linalool and linalyl acetate, which may have a calming effect on chemical receptors in the brain.ix,x Studies have shown that a daily capsule of pharmaceutical-quality lavender oil, found only in Kalms Lavender, is as effective as commonly prescribed anti-anxiety medications such as lorazepam and paroxetine.xi, xii
If mild anxiety worsens or becomes disabling, the next step is seeking professional help in order to receive an accurate diagnosis and effective treatment.
[i] Survey by Census Wide, commissioned by Lanes Health. Conducted on 3,726 Respondents – including 2,000 nationally representative respondents. October 2023.
[ii] Lanes Health. Empowerment in the Epidemic of Anxiety: Equipping patients and healthcare providers with the necessary tools and skills to manage mild anxiety. 2023.
[iii] The Guardian. UK has experienced ‘explosion’ in anxiety since 2008, study finds. September 2020. Available at: https://www.theguardian.com/society/2020/sep/14/uk-has-experienced-explosion-in-anxiety-since-2008-study-finds (Accessed: April 2023).
[iv] Mental Health UK. What is anxiety? Available at: https://mentalhealth-uk.org/help-and-information/conditions/anxiety-disorders/what-is-anxiety/ (Accessed: April 2023).
[v] Mental Health UK. Treatments for anxiety disorders. Available at: https://mentalhealth-uk.org/help-and-information/conditions/anxiety-disorders/treatment/ (Accessed: May 2023).
[vi] Royal College of Psychiatrists. Hidden waits force more than three quarters of mental health patients to seek help from emergency services. October 2022. Available at: https://www.rcpsych.ac.uk/news-and-features/latest-news/detail/2022/10/10/hidden-waits-force-more-than-three-quarters-of-mental-health-patients-to-seek-help-from-emergency-services (Accessed: April 2023).
[vi]i Mind. Types of complementary and alternative therapies. Available at: https://www.mind.org.uk/information-support/drugs-and-treatments/complementary-and-alternative-therapies/types-of-complementary-and-alternative-therapies/#Hypnotherapy/ (Accessed: May 2023).
[vii]i Mayo Clinic. Depression and anxiety: Exercise eases symptoms. September 2017. Available at: https://www.mayoclinic.org/diseases-conditions/depression/in-depth/depression-and-exercise/art-20046495 (Accessed: May 2023).
ix Salve J, et al. Cureus. 2019;11(12):e6466.
x Malcolm BJ and Tallian K. Ment Health Clin. 2018;7(4):147–155.
xi Woelk H and Schläfke S. Phytomedicine. 2010;17(2):94–99.
xii Kasper S, et al. Int J Neuropsychopharmacol. 2014;17(6):859–869.
How Gen AI Opens a Whole New World for Dyslexic Individuals
Boris Krumrey, Global VP of Automations at UiPath
Growing up in 1970s West Berlin, I experienced a lack of attention from primary school teachers who were not equipped to address special needs for dyslexic children. While teachers were somewhat aware of conditions like dyslexia, they lacked the necessary training to support students like me. I vividly remember the disheartening moments of reading aloud, as other children would complain about my struggles, with the teachers choosing not to intervene. Writing assignments was even more demoralising, as the teacher looked at me with disappointment, regardless of the pressure I faced, as my spelling and handwriting showed no improvement.
Living with dyslexia can pose significant challenges in reading and writing, making self-expression daunting. However, new tools and technological developments are presenting exciting opportunities for workers who are neurodiverse or are living with learning difficulties.
Any traumatic experiences faced by dyslexic individuals often lead to deep disbelief in one’s abilities. Often simple tests such as identifying a series of numbers or words can frustrate people and even lead to misdiagnosis or failure to receive new opportunities. However, a new horizon of possibilities has emerged with the advent of generative artificial intelligence (Gen AI). Thanks to the content writing capabilities, data analysis and automation, Gen AI is poised to be an ideal tool for dyslexic individuals, empowering them to overcome writing obstacles and unlock their full creative potential.
Understanding the use cases
Gen AI can act as an important catalyst for a business on its automation journey, unlocking the door to a wealth of new opportunities. Technology, such as AI, can seem intimidating at first, but taking the first step to an intelligently automated business truly can improve efficiency and workplace experience dramatically for individuals.
Of course, before implementing AI solutions, it is important to understand the exact use cases and where they can be applied for many tasks. Looking first at enhancing writing efficiency, generative AI provides invaluable assistance in improving writing efficiency for dyslexic individuals. The technology offers real-time suggestions, corrections, and alternative phrasing as a reliable companion during the writing process. Dyslexic writers can focus on their ideas and thoughts while the AI refines the expression, eliminating the frustration caused by dyslexia-related writing challenges.
Predictive capabilities are perhaps one of the remarkable features of Gen AI. The ability to anticipate words and phrases, often aligning perfectly with the writers’ intentions has proven to be a real game changer. It significantly reduces the time and effort required to produce coherent and correctly written content, enhancing both speed and accuracy in the writing process.
Gen AI understands the unique challenges faced by dyslexic individuals, particularly in terms of visual perception. Dyslexia commonly involves difficulties accurately reading letters or words, resulting in visual confusion. The customisable features of AI can address this, for example, tailoring the text presentation to suit individual needs. It can also make the writing experience more accessible and enjoyable.
This all draws back to the essential principles of boosting confidence and self-expression among workers. The stigma surrounding learning difficulties, such as dyslexia, can negatively impact self-confidence, but Gen AI is the equivalent of a supportive partner, encouraging dyslexic writers to express themselves freely without the fear of judgement or misunderstanding. Providing real-time feedback and assistance instils an important sense of assurance, empowering individuals to embrace their unique voices and share their ideas with the world.
Spotlighting the human impact and AI limitations
The ways in which Gen AI can overhaul work should not be conflated with a testament to the decline of human intelligence and value in the workplace. In fact, it is quite the opposite. Workers with learning disabilities often let self-doubt block potential due to mistakes that they do not see among the wider pool of workers. This idea of being ‘other’ can distance workers for the wrong reasons. If AI can free workers of tasks that fuel self-doubt, they can apply their specialist skills and stop feeling as though they are being dragged down by perceived weaknesses. Gen AI is bringing out the human value to work more than some individuals might have ever believed.
However, as with every technology and human relationship, it is essential to analyse and limit possible negative impacts. Starting with language formulation, Gen AI’s predictive capabilities and real-time suggestions can influence the language formulation process. While this can be beneficial for dyslexic individuals who struggle with word recall or spelling, there is a possibility AI’s suggestions may steer the writing towards a more standardised or conventional form. This may inadvertently dilute the writer’s authentic expression, altering their unique style or creative choices.
To mitigate this, AI usage should be selective and applied only to areas of struggle, such as sentence structure and spelling, letting creative flair do the rest. It’s also recommended that teams and dyslexic individuals retain manual reviewing and editing. This ensures they maintain control over the final product, making deliberate choices that align with their authentic voice and personal style.
Authenticity lies in embracing imperfections and unique qualities. Dyslexic learners can celebrate their distinct perspectives, creative approaches, and personal growth throughout their writing journey. Acknowledging and highlighting their individuality can create a genuine connection with their readers which is the core goal of any copy. Once the concerns about authenticity have been addressed, it is key to strike a balance that allows the AI to support and amplify their writing while maintaining the authenticity and genuine expression that make their work truly remarkable.
A powerful AI and human partnership
I once met a friend who struggled with dyslexia but had a talent for working with computers. He helped run his father’s real estate business, but when I asked him why he didn’t study computer science, he explained that his dyslexia made him worry about the amount of time he would have to spend debugging his code due to syntax errors.
As someone who studied computer science, I never fully appreciated the challenge dyslexic individuals face when it comes to coding. Computers are patient and tireless, always correcting mistakes as long as the user persists. However, with Gen AI, coding challenges for people with dyslexia can be immediately filtered out before compilation.
In the future, automation platforms like UiPath will integrate Gen AI into all tools that support intelligent automation for daily knowledge work. Dyslexia will no longer be a barrier to unleashing creativity.
Gen AI emerges as a transformative tool for dyslexic individuals, revolutionising the writing and coding experience. It empowers writers and developers to overcome the barriers imposed by dyslexia and unlock their full creative potential. By providing tailored support, boosting confidence, and facilitating effective communication, Gen AI ensures the written word becomes a playground for self-expression rather than a source of frustration. We should celebrate the union of technology and humanity as dyslexic individuals triumph over their writing challenges and share their remarkable stories with the world.
Testing, testing…the key to health tech triumph
By Chris Bradshaw, Strategy Director at Infinum
Musicians prepare rigorously for successful performances through hours of practice and refinement. In the realm of direct-to-consumer health tech, achieving innovation also requires meticulous groundwork.
When executed effectively, direct-to-consumer solutions empower patients and caregivers, providing them with informed health choices and rapid access to medical insights. Nevertheless, growth brings its own set of challenges. When health tech products lack rigorous testing and design, they can result in user confusion, unreliable data, and even jeopardise patient safety.
Amidst the whirlwind of innovation in this field, a crucial aspect often gets overlooked – preparing for worst-case scenarios. To ensure both success and safety, it’s imperative to thoroughly investigate potential user issues prior to launch.
To excel in this field of innovation, keep these three essential components in mind: comprehensive testing, a user-centric approach, and strong privacy measures.
Testing, your products best friend
Just as rehearsals are the heart of a polished musical performance, comprehensive testing serves as the foundation for identifying and addressing potential issues in the early stages.
By simulating various scenarios and user interactions, companies can understand how their products perform under different circumstances. A method that’s getting attention is sprint testing. It pushes the product’s long-term goals and ethical aspects to make sure the final product is strong, reliable, and safe.
Running discovery sessions for any product is also critical to help predict and navigate potential problems, but it’s even more important in the health tech industry. Even more so when you’re working on direct business to consumer products, where the risks can increase significantly.
In short, the lesson is test, test and test again. By investing time and resources into comprehensive testing, companies can avoid costly and potentially harmful issues after product launch.
It’s also important to remember that testing doesn’t stop when the product or app is launched; it’s crucial to gather consistent feedback from users to improve the solution continuously.
Strike the right cord with users
Putting your end-users first is not just a catchphrase; it’s a fundamental principle to launch any product or service. At the same time, if you’ve engaged in rigorous testing and gathered feedback from users before launch, you’ll already be set up for success.
For example, at Infinum, we worked to develop Cormeum, the smart heart-failure app. To validate some of our initial assumptions on what would work best, we trialed the prototype with real cardiac arrest patients and discovered they preferred larger visuals, a straightforward tone of voice, and a streamlined morning and evening tracking routine.
This allowed us to design a product that is easy and seamless to use, and empathises with users, understanding their needs, expectations and fears. This is critical in medical situations and helps ensure your product isn’t just meeting requirements, it instills confidence.
User-centric design goes beyond aesthetics—it’s about enhancing the user experience across the journey and customising it for specific needs. By integrating user feedback early on, companies can build products that deeply connect with their target audience.
The importance of data privacy
It’s also important to remember that health tech should value patient data as much as a doctor does. As these technologies collect and manage personal health information extensively, the ethical responsibility to safeguard user data becomes even more crucial. A significant data breach can result not only in substantial financial losses but also harm to a company’s reputation.
Openness and clear communication about data usage and security measures can significantly reassure users about their information’s safety. Staying up to date with data protection regulations and implementing strong cybersecurity measures are essential for thwarting potential threats. A single breach can undo years of hard work and innovation.
An excellent case of emphasising data privacy from the beginning is Bloom Diagnostics, a health-tech company focused on at-home diagnostic tests. With Infinum’s assistance, they used strong encryption and secure storage methods to protect user health data, building a foundation of trust with their users.
How to create a masterpiece
Health tech success requires a huge amount of planning and an unwavering commitment to testing and your end users, just as composing a masterpiece takes time and dedication.
Done right, direct-to-consumer health tech has the potential to transform the entire healthcare industry, and in many ways, already is.
Businesses venturing on this journey aren’t alone; tech consultants can help get the process right. Together, real change is possible, transforming the lives of patients and healthcare staff for years to come.