Health care wants YOU: 9 opportunities, 4 pearls for digital health startups

digital health startups - reverse pitches
A visual summary of reverse digital health pitches (illustration: CollectiveNext).

Boston’s digital health world is humming with tech talent, idealistic health care professionals and business-savvy accelerator organizations. The passion was palpable last week as 300-plus people gathered at MassChallenge’s latest Pulse@Check digital health meetup, hoping to turn their health care ideas into reality.

The event, hosted by Boston Children’s Hospital’s Innovation and Digital Health Accelerator (IDHA) and Cerner, a lead developer of health care IT systems, presented numerous opportunities and tips for digital health startups.

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Smart pad would provide biofeedback for Kegel incontinence exercises

biofeedback Kegel exercises urinary incontinence

If you’ve ever been given Kegel exercises to strengthen your pelvic floor, you may have wondered if you’re doing them right or if you’re getting better. Two physicians at Boston Children’s Hospital have developed a stick-on pad that could someday tell you.

Carlos Estrada, MD, director of the Spina Bifida Center and co-director of Urodynamics and Neurourology, and Jeanne (Mei Mei) Chow, MD, director of Uroradiology at Boston Children’s both work with children who have urinary incontinence. In the clinic, Estrada has equipment that provides biofeedback as kids practice squeezing their pelvic floor muscles. But parents had been asking for a home solution. “They say, ‘it’s hard to do it at home without getting any feedback,’” says Estrada.

Done right, Kegels can have an 85 percent success rate, he says. But lacking feedback, most people give up on them, including adults. “Adults can get monitoring, but it’s done in specialized clinics with intrarectal and intravaginal probes,” Estrada says.

Most people take a pass on that.

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Impaired recycling of mitochondria in autism?

mitochondria in autism tuberous sclerosis

A study of tuberous sclerosis, a syndrome associated with autism, suggests a new treatment approach that could extend to other forms of autism.

The genetic disorder tuberous sclerosis complex (TSC) causes autism in about half of the children affected. Because its genetics are well defined, TSC offers a window into the cellular and network-level perturbations in the brain that lead to autism. A study published today by Cell Reports cracks the window open further, in an intriguing new way. It documents a defect in a basic housekeeping system cells use to recycle and renew their mitochondria.

Mitochondria are the organelles responsible for energy production and metabolism in cells. As they age or get damaged, cells digest them through a process known as autophagy (“self-eating”), clearing the way for healthy replacements. (Just this month, research on autophagy earned the Nobel Prize in Physiology or Medicine.)

Mustafa Sahin, MD, PhD, Darius Ebrahimi-Fakhari, MD, PhD, and Afshin Saffari, in Boston Children’s Hospital’s F.M. Kirby Neurobiology Center now report that autophagy goes awry in brain cells affected by TSC. But they also found that two existing medications restored autophagy: the epilepsy drug carbamazepine and drugs known as mTOR inhibitors. The findings may hold relevance not just for TSC but possibly for other forms of autism and some other neurologic disorders.

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Muscular dystrophy study suggests new therapeutic approaches to autism

muscular dystrophy autism social interaction
In addition to weakening muscle cells, loss of dystrophin also impairs Purkinje cells in the cerebellum.

Robin Kleiman, PhD, is Director of Preclinical Research at Boston Children’s Hospital’s Translational Neuroscience Center.

One of the hardest parts of developing new treatments for autism spectrum disorder (ASD) is that almost every patient has a different combination of environmental and genetic risk factors. This suggests that every patient could take a unique path to their diagnosis. It is hard to come up with a single treatment that will help patients with fundamentally different root causes of ASD.

One way to approach this problem is to look for ways to cluster sub-types of autism for clinical trials, based on genetic risk factors or the types of neural circuits that are affected. If circuit dysfunction could be monitored and diagnosed easily in patients, it might be possible to develop treatments to reverse the dysfunction that cut across genetic and environmental causes of ASD. That is the hope of research on well-defined “syndromic” causes of autism such as tuberous sclerosis complex, Fragile X syndrome and Rett syndrome.

Accelerating research collaborations to design clinical trials for children with brain disorders, including ASD, is a major mission of Boston Children’s Hospital’s Translational Neuroscience Center (TNC). A recent study in Translational Psychiatry, led by Mathew Alexander, PhD, in the Boston Children’s lab of Lou Kunkel, PhD, in collaboration with the TNC and Pfizer, is a prime example. It suggests that patients with Duchenne muscular dystrophy (DMD) may constitute another subset of ASD patients — one that could benefit from phosphodiesterase (PDE) inhibitors, a family of drugs including Viagra.

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Potential cure for diabetes may be in the stomach

diabetes stomach beta cells
The yellow-green cells in this “mini-stomach” are capable of making insulin. The mini-organ was made from biopsied cells from mouse stomachs, with reprogramming factors added.

If only there were a cure. David Breault, MD, PhD, associate chief of the Division of Endocrinology at Boston Children’s Hospital, was seeing patient after patient with Type I diabetes. Children facing lifetimes of insulin injections, special diets and the threat of long-term complications including blindness, heart disease and kidney failure.

Breault knew that patients with type I diabetes mysteriously destroy their own insulin-producing beta cells. He had read reports of researchers transplanting beta cells to supplement insulin. These transplants, even when successful, required powerful immunosuppressant medications to prevent patients’ immune systems from attacking the donor cells.

But Breault was also aware that investigators had, for a decade, been looking to stem cells as the source of a constantly renewing supply of beta cells. Advancing that promise, he has now found a way to convert patients’ own cells — from the stomach and intestine — into beta cells that produce insulin.

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Post-millennial hackers build hospitals of the future in Minecraft

From the long line stretching down the hall at the Kids-Only Minecraft Hackathon, a 7-year-old could be heard shouting, “Would it be helpful if I gave access to others on my server?”

Parents perked up with curiosity. Many of the kids, waiting to sign in to create the hospital of the future, looked even more excited. With a ton of enthusiasm and some impressive design skills, the kids got to work on their laptops and tablets re-designing the hospitals of our future, one digital brick at a time. The first-ever Kids-Only Minecraft Hackathon was well underway.

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Inspired research in newborn lung disease: Stella Kourembanas, MD

Stella Kourembanas
Stella Kourembanas in the NICU with Julian (photos: Katherine Cohen)

During the NICU rotation of her clinical training, Stella Kourembanas, MD, sat at the bedside of newborn babies with hypoxia. The newborns weren’t getting enough oxygen and were suffering from pulmonary hypertension — abnormally elevated blood pressure in the lung’s blood vessels. What was triggering these patients’ disease?

Kourembanas decided her fellowship research would focus on determining how hypoxia triggers the blood vessels to become abnormal. She built her career, showing how hypoxia affects the endothelial cells that line blood vessels: what genes are turned on, how they regulate interaction between cells and how that affects the lung vasculature.

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Harnessing MRI to steer drugs to hard-to-reach targets

drug delivery propelled by MRI

Once a drug is injected systemically, can you steer it to where you want it under MRI guidance? Pierre Dupont, PhD, and colleagues saw this as an engineering problem. Solving it could enable concentrated drug delivery to, say, a deep tumor in the lungs while simultaneously taking images.

Labeling drugs with magnetized particles is the first step, allowing the MRI scanner’s magnetic pulses to propel them. The next step is to be able to actively steer the particles through a series of branching vessels to a desired location. But getting a scanner to both image and propel particles forcefully enough to overcome the force of the blood flow is easier said than done.

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Entry door for deadly C. difficile toxin suggests new mode of protection

Clostridium difficile
C. difficile (Wikimedia Commons)

Clostridium difficile, also called “C. diff,” tops the CDC’s list of urgent drug-resistant threats. Marked by severe diarrhea and intestinal inflammation, C. diff has become a leading cause of death from gastrointestinal illness, causing half a million infections a year in the U.S. alone.

C. diff flourishes best in hospitals and long-term care facilities where people are on long-term antibiotic treatment. “Antibiotics clear out the normal intestinal bacteria and create a space for C. diff to colonize and grow in the colon,” says Min Dong, PhD, who researches bacterial toxins in the Department of Urology at Boston Children’s Hospital.

In today’s Nature, Dong and postdoctoral fellow Liang Tao, PhD, together with researchers at University of Massachusetts Medical School, reveal how C. diff’s most potent toxin gets into cells. The toxin’s entryway, a receptor called Frizzled, provides an important and interesting clue to fighting the hard-to-eradicate infection.

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Startup uses Uber to get patients to their medical appointments

Uber medical transportation Circulation

Getting to the doctor will soon get easier for some struggling patients. Boston Children’s Hospital has joined forces with the ride-hailing service Uber to pilot a non-emergency medical transportation platform.

The online, HIPAA-compliant tool, called Circulation, connects with health care information systems, enabling hospitals to schedule Uber rides for patients. The pilot will serve Boston Children’s, Mercy Health System in Pennsylvania and Nemours Children’s Health System in Wilmington, Delaware.

A 2005 study estimated that 3.6 million people miss medical appointments because they don’t have access to transportation. While Medicare and Medicaid and other payers provide non-emergency transportation benefits, such as taxi vouchers, patients may be unaware of the programs or have trouble navigating reimbursement rules for the rides. Frequently, the taxi or car service arrives late or doesn’t show up at all.

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