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A Cure for Type 1 Diabetes? For One Man, It Seems to Have Worked.

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.

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.

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.’”

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Business

Time is running out: NHS and their digital evolution journey

By Nej Gakenyi, CEO and Founder of GRM Digital

Many businesses have embarked on their digital evolution journey, transforming their technology offerings to upgrade their digital services in an effective and user-friendly way. Whilst this might be very successful for smaller and newer businesses, but for large corporations with long-standing legacy infrastructure, what does this mean? Recently the UK government pledged £6bn of new funding for the NHS, and the impact this funding and investment could have if executed properly, could revolutionise the UK public healthcare sector.

The NHS has always been a leader in terms of technology for medical purposes but where it has fallen down is in the streamlining of patient data, information and needs, which can lead to a breakdown in trust and the faith that the healthcare system is not a robust one. Therefore, the primary objective of additional funding must be to implement advanced data and digital technologies, to improve the digital health of the NHS and the overall health of the UK population, as well as revitalise both management efficiency and working practices.

Providing digital care

Digitalisation falls into two categories when it comes to the NHS – digitising traditionally ‘physical’ services like offering remote appointments and keeping electronic paper records, and a greater reliance on more innovative approaches driven by advances in technology. It is common knowledge that electronic services differ in GP practices across the country; and to have a drastically good or bad experience which is solely dependent on a geographical lottery contradicts the very purpose of offering an overarching healthcare provision to society at large.

By streamlining services and investing in proper infrastructure, a level playing field can be created which is vital when it comes to patients accessing both the care they need and their own personal history of appointments, GP interactions, diagnoses and medications. Through this approach, the NHS focus on creating world-leading care, provision of that care and potentially see waiting lists decrease due to the effective diagnosis and management enabled by slick and efficient technology.

This is especially important when looking at personalisedhealth support and developing a system that enables patients to receive care wherever they are and helps them monitor and manage long-term health conditions independently. This, alongside ensuring that technology and data collection supports improvements in both individual and population-level patient care, can only serve to streamline NHS efforts and create positive outcomes for both the patient and workforce.

Revolutionising patient experiences

A robust level of trust is critical to guaranteeing the success of any business or provision. If technology fails, so does the faith the customer or consumer has in the technology being designed to improve outcomes for them. An individual will always have some semblance of responsibility and ownership over their lives, well-being and health. Still, all of these key pillars can only stand strong when there is infrastructure in place to help drive positive results. Whilst there may be risks of excluding some groups of individuals with a digital-first approach, technology solutions can empower people to take control of their healthcare enabling the patient and NHS to work together. Tandem efforts between humans and technology

Technology must work in tandem with a workforce for it to be effective. This means the NHS workforce must be digitally savvy and have patient-centred care at the front and centre of all operations. Alongside any digital transformation the NHS adopts to improve patient outcomes, comes the need to assess current and future capability and capacity challenges, and build a workforce with the right skills to help shape an NHS that is fit for purpose.

This is just the beginning. With more invtesement and funding being allocated for the NHS this is the starting point, but for NHS decision-makers to ensure real benefits for patients, more still needs to be done. Effective digital evolution holds the key. Once the NHS has fully harnessed the poer of new and evolving technologies to change patient experiences throught the UK, with consistent communication and care, this will set the UK apart and will mark the NHS has a diriving example for accessible, digital healthcare.

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Pharmaceuticals

Improving Patient Experience: How To Utilise an Interactive Whiteboard in Hospitals

Hospitals and healthcare facilities face a unique set of challenges every day. Patients comprise most of their daily visitors, and no two cases are alike. Yet it’s crucial that medical staff deliver a stellar patient experience for each individual they see.

Of course, long waiting times, confusing information, and other issues can get in the way of this. And when the patient experience suffers, the hospital’s reputation does as well. Bringing in technology such as interactive whiteboards offers a practical solution, and they’re easier to manage than ever.

How Patient Experience Affects Your Bottom Line

Even in facilities featuring top doctors and services, patients will walk out after just one negative interaction. This might include missed or cancelled appointments, faulty or inaccurate diagnoses, and surly customer service staff. Notice the one thing common with these examples? Often, a negative patient experience comes from a problem with communication—or a complete lack of it.

By nature, the medical practice is busy, and miscommunication can be hard to avoid. But it only takes one highly publicised negative experience to undermine a healthcare company’s reputation. Once word spreads about long wait times, cancelled appointments, unresponsive staff, and misunderstood diagnoses, your bottom line starts sinking.

So, how do healthcare organisations improve their communication systems and deliver a better patient experience? Usually, a communication problem requires a communication solution.

Use Technology To Improve Communication and the Patient Experience

To improve communication between your medical professionals and their patients, don’t reinvent the wheel. There are plenty of existing technologies that help bridge communication gaps and increase engagement levels.

For instance, use monitors to post regular updates in the waiting room and let patients know how long they’ll have to wait. Displaying the order in which patients are being treated offers transparency about how long doctors typically meet with their patients. This way, those in the waiting room know what to expect in terms of when they might be seen.

You can also lean on presentations or videos to help patients better understand their diagnosis and treatment plans. This can free up the doctor’s time and give patients another way to process information.

Among the more popular and easy-to-implement ways to improve communication are interactive whiteboards. Through touchscreen technology, these smart devices help make information more accessible for both patients and medical teams.

4 Ways To Use Interactive Whiteboards for Better Patient Experience

Why digital interactive whiteboards? Previous versions such as dry-erase boards and chalkboards were messy prototypes that relied solely on human handwriting. It takes time to write everything on the board. Plus, chalk and markers are both health and environmental hazards. (Besides, doctors have notoriously bad handwriting.)

Interactive whiteboards are a modern way to communicate with patients. You can use your fingers to write on these devices, and many programs will automatically transform handwriting into readable text. Even better, whiteboards can also serve as digital displays that play presentations, slideshows, videos, and images.

If you need to clarify a point, medical staff can annotate the whiteboard’s content directly. Finally, anything displayed on the interactive whiteboard can be saved into a file and printed. This allows both doctors and patients to keep records of their interactions for future reference.

Let’s review some of the other ways interactive whiteboards can improve the patient experience.

Hospital Branding

An interactive whiteboard in the registration area, waiting rooms, consultation areas, and patient rooms signals a healthcare facility’s commitment to communication. Waiting patients can quickly get updates about the current queue lines and estimated waiting times. In between, staff can display hospital information that can help raise brand awareness.

Whiteboards can also play interactive presentations that share the company’s mission, vision, and goals. Then, patients who want to know more can simply interact with the whiteboard to get additional information.

Personalized Care Planning

During consultations, healthcare professionals can easily share their initial findings and discuss the information with patients. Doctors can also use the interactive whiteboard to gather specific information from patients in order to come up with a more accurate diagnosis. Once a treatment is prescribed, patients can turn to the whiteboard to play videos or presentations that help them better understand the treatment process.

Patient Education

Interactive presentations that are catered to individual patients can help educate patients on the nature and treatment of their conditions. By clicking on the presentation via touchscreen controls, they can get more details of how treatment can reverse their condition. Patients can also view simulations of what can happen to them in the absence of treatment.

In addition, patients can use interactive whiteboards to gather more information about their healthcare facility, including its history, specialisations, and public staff data.

(Hospital) Unit-Specific Information

An interactive whiteboard in a hospital room can give doctors and nurses a quick summary of their patient’s current condition. At the same time, they can add changes or instructions so that attending staff can adjust accordingly. Confined patients or their companions can also access information from whiteboards that show updates on administrative matters such as billing, consumption summaries, and timetables.

For the Best Patient Experience, Interactive Whiteboards Need Reliable Device Managers

Interactive whiteboards can improve the patient experience by providing an always-ready communication medium. Because these smart devices are a substantial investment for any healthcare company, be sure to invest in a reliable device management solution to manage, maintain, and secure them.

The right device manager utilises remote cloud technology to provide over-the-air (OTA) updates to all connected devices. It can also control access levels by assigning authority to users based on need. What’s more, a well-designed device manager can secure devices by shutting down units that report unauthorised access.

Device managers can geolocate missing or stolen devices and start the retrieval process. Finally, admins can remotely wipe private data from the devices if there’s a threat of data theft.

As you can see, your choice of device management software can help you get the most out of your interactive whiteboards. Invest in a reliable device manager to maximise their performance for a more efficient medical staff and a better overall patient experience.

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Pharmaceuticals

Strong IP protection is vital to healthcare’s AI-encompassing future

Head of Innovation at InnoScot Health, Robert Rea analyses the challenges and opportunities of integrating artificial intelligence into the NHS

Artificial Intelligence (AI) offers huge possibilities for healthcare improvement, and many are already being realised – but it also presents complexities for innovators looking to protect their ideas.

We’ve seen NHS Scotland and its triple helix partners across industry and academia increasingly leveraging AI with the aim of continuously improving patient care while streamlining operations for smarter, more efficient working practices.

Projects already underway include  the use of AI to help detect lung cancer, assess mammograms and skin lesions, and be used to predict or monitor health conditions, to name just a few examples.

Such applications hold the potential to nothing less than revolutionise diagnosis, treatment, and management of patients.

Thanks to a bank of high-quality data-led insights and forward-thinking, innovation-ready staff, NHS Scotland and its partners are well-placed to take a lead on taking advantage of the benefits of AI transformation.

Those benefits should absolutely be explored fully for improved patient outcomes and predictive potentialities. However, in applying the vast possibilities of AI to Scotland’s healthcare system, we also must be cognisant of broader implications; and the recent legal debate over whether AI can be named as an inventor has generated much interest.

It is an area we at InnoScot Health have been closely following – ensuring protection of the intellectual property (IP) inherent in new ideas that involve AI can be complex, particularly depending on how much of the idea sprang from the AI itself.

Patents are used to protect novel aspects of inventions, stopping others from duplicating them – that much is clear cut – but in December, the UK Supreme Court made headlines when it upheld earlier decisions which rejected a bid to allow AI to be named as an inventor in a patent application.

In 2019, US technologist Dr Stephen Thaler had sought to have his AI recognised as the inventor of a food container and a flashing light beacon after claiming the software was sentient and conscious, but the intellectual property office (IPO) rejected this, saying only a person could be named as an inventor.

The decision was backed by both the High Court and Court of Appeal after they supported the view that only ‘persons’ could hold patent rights.

Dr Thaler was not deterred though, and in December, five Supreme Court judges dismissed a bid to reverse those decisions, concluding similarly that “an inventor must be a person” and that AI could not secure patent rights.

One of the judges Lord Kitchin, said the AI was “a machine with no legal personality” and that Dr Thaler had “no independent right to obtain a patent in respect of any such technical advance”.

The judgement, commentators noted nevertheless, did not specifically deal with the issue of whether the AI did in fact invent the food container and light.

While the IPO welcomed the judgement and the clarification it offered – labelling the dispute a “test case, rather than one which is motivated by any pressing need in the real world” – it added interestingly that the government will “keep this area of law under review to ensure that the UK patent system supports AI innovation and the use of AI in the UK”.

So, would the outcome have been different if the AI had come up with the invention, but Dr Thaler had been identified as the inventor and owner of the patent? That remains unclear, though what is certainly clear is that a lot of organisations would find themselves in a predicament going forward if they were not able to own the patent from AI software-led inventions.

It serves to underline the complexities that now stem from AI use and how its growing prominence and advancement could force law changes in future.

Patents are not the only area of intellectual property protection that are being tested by AI – copyright too is being reassessed in light of fresh developments in the likes of AI-generated works.

Does the copyright from the works belong to the original programmer of the AI, for instance, or does it belong to the user who is instructing it to perform the task?

In future, will companies who use autonomous AI have to declare that their employees were the inventors despite them having little to do with the actual creation?

The IPO recently stated that it acknowledges “there are legitimate questions” around how intellectual property looks on AI creations and decides who owns them.

It believes that any future changes would need to be considered on an international level, not just within the UK.

All Scottish healthcare leaders have been very clear on the deployment of AI – that it should supplement human expertise rather than replace it – and there’s no doubting how effective it appears to be in, for example, enabling review and translation of mammograms with high accuracy alongside clinicians’ experience and insight.

Used well, AI can become an important safeguard in such processes while making them faster and more efficient.

For AI innovators working in healthcare though, the securing of patents and copyrights may become increasingly complex.

Under UK patent law, the NHS, as employer, will usually own the intellectual property created by healthcare professionals in the course of their employment or specifically assigned duties.

Comprehensive AI policies must therefore adapt to encompass both current challenges in delivering its benefits in healthcare settings, while precluding issues from likely future advances.

With a robust, horizon scanning policy in place, AI-related innovation can flourish but with the peace of mind which comes through IP protection.  

Protecting the IP rights of the NHS represents one of the cornerstones of InnoScot Health’s service offering and why the organisation was initially set up.

We assist NHS Scotland in managing this IP, to ensure a return on investment for the health service. Any revenue generated from commercialising ideas and innovations from healthcare professionals is shared with the originator and the health board through a bespoke NHS inventor award scheme as detailed in individual employee contracts and health board IP policies.

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