Dr. Moses Shieh, who is a bariatric surgeon, tells Consumers Digest that he had 15 patients who were scheduled to swallow the Obalon Balloon System on Jan. 10, 2017, which was the first day that the minimally invasive obesity-treatment device was available in the United States.
Obalon is the first and only weight-loss balloon system that’s designed to be swallowed and that’s been approved by Food and Drug Administration. However, FDA approved three other minimally invasive obesity-treatment devices in the past 2 years.
Experts tell us that these devices are eagerly anticipated alternatives to more-invasive treatments that are aimed at obesity, such as bariatric surgery, particularly for patients who have obesity class I—a body mass index (BMI) of 30.0–34.9 kilograms per meter squared (kg/m2)—or obesity class II—a BMI of 35–39.9 kg/m2.
Minimally invasive obesity devices represent just one of several medical breakthroughs in the past 2 years that add treatment options or expand existing ones.
Several of the medical breakthroughs that are discussed in this article received expedited approval through FDA programs that are intended to speed the approval and delivery of new medications and devices to patients.
The recently passed 21st Century Cures Act is intended to build on FDA’s efforts to expedite medical-product innovation by creating the breakthrough-device pathway, which builds on the expedited-access pathway. It also establishes the limited-population pathway, which will help to streamline the development programs for certain antibacterials and antifungals that are intended to treat targeted groups of patients who have serious or life-threatening infections, as well as a new program for the development of regenerative-medicine products.
LOSING WEIGHT. Centers for Disease Control and Prevention (CDC) says at least one-third of adults are obese (a BMI of 30 or more) and that the estimated annual medical cost of obesity is $147 billion.
Obesity treatments include exercise, healthy diet, prescription medicine, surgery and four types of FDA-regulated medical devices: electronic stimulation systems, gastric bands, gastric-emptying systems and gastric-balloon systems.
Obalon, which is the latest FDA-approved gastric-balloon system, is a nonsurgical weight-loss treatment for adults who have a BMI of 30–40 kg/m2 and who failed to lose weight through diet and exercise. Obalon consists of a balloon that’s folded inside of a capsule, which is swallowed by a patient who isn’t sedated. After the balloon reaches the stomach, it’s inflated with gas through a microcatheter. The microcatheter then is removed, which leaves the buoyant balloon in the stomach. The stomach now has less room for food, so the patient feels fuller more quickly during a meal. Typically, three balloons are placed in a stomach over 3 months to facilitate weight loss over a 6-month treatment period. At the end of the 6 months, the three balloons are removed through an outpatient endoscopy in which the patient is conscious and lightly sedated.
A large clinical study of Obalon showed that people who were treated with the balloon system lost 14.4 pounds on average during the 6 months that they had the balloons in their stomach and that 89.5 percent of patients maintained their weight loss for at least 1 year. Shieh, who has treated obesity since 2004, considers Obalon to be a medical breakthrough because of its customizability, ease of use and reduced side effects.
“It’s not a one-size-fits-all [treatment],” Shieh says. “With three balloons, you really help patients to lose a [maximum amount] of weight.”
Andrew Rasdal, who is the president of Obalon Therapeutics, says Obalon will be available nationwide by the end of 2017. According to Bariatric Surgery Source, which is an online educational resource for weight-loss patients, the total cost of the procedure is $8,150 on average. Obalon isn’t covered by insurance, but many doctors provide financing options to help patients to achieve their weight-loss goals, Obalon Therapeutics says.
The Orbera Intragastric Balloon System and the ReShape Integrated Dual Balloon System, which are gastric-balloon systems that FDA approved in 2015, are placed in the stomach through the mouth via a minimally invasive endoscopic procedure while the patient is under mild sedation. After the balloon is in place, the Orbera and ReShape balloons are filled with saline. Experts say Obalon’s use of gas creates fewer adverse reactions, such as discomfort, nausea, ulceration and vomiting, than do the saline-based systems. We also were told that the cost of either procedure compares with the cost of Obalon.
FINDING NEW TREATMENTS. The recently passed 21st Century Cures Act is intended to build on FDA’s efforts to expedite medical-product innovation by creating the breakthrough device pathway, which builds on the expedited-access pathway.
A fourth new minimally invasive obesity device, Aspire Bariatrics’ AspireAssist, is a gastric-emptying system that gained FDA approval in June 2016. The device helps patients to lose weight through a surgically placed tube that drains a portion of the stomach’s contents after every meal. AspireAssist requires frequent monitoring by a health-care provider, who shortens the tube as a patient loses weight. Katherine D. Crothall of Aspire Bariatrics says the device benefits patients who have a BMI of 35–55 kg/m2 and don’t want to undergo bariatric surgery. The treatment costs $8,000–$9,000, or one-third of the cost of bariatric surgery, Crothall says. She tells us that AspireAssist isn’t covered by insurance plans, but the company is speaking with several insurance companies in hopes that they’ll cover the device.
David Filmore of Medtech Insight, which is an industry publication, says minimally invasive obesity devices won’t make existing devices and obesity surgeries obsolete. He says bariatric surgery has a more pronounced effect on weight loss for those who have a BMI of at least 35 kg/m2. However, Filmore says gastric-balloon and gastric-emptying systems are beneficial, because they expand obesity-treatment to patients who don’t qualify for bariatric surgery.
ARTIFICIAL PANCREAS. Device manufacturers are developing diabetes treatments, too. Medtronic gained FDA approval in September 2016 for its Mini-Med 670G hybrid closed-loop system, which is the first FDA-approved device that measures and delivers basal insulin 24 hours per day in type 1 diabetes patients who are age 14 and older.
CDC estimates that about 5 percent of diabetes patients have type 1 diabetes, or juvenile diabetes, which typically is diagnosed in children and young adults.
Often referred to as an “artificial pancreas,” the MiniMed 670G adjusts insulin levels by measuring glucose levels every 5 minutes and delivering or withholding insulin automatically. Patients only have to enter the amount of carbohydrates that they consume into the system and have to calibrate the system’s sensor periodically.
ARTIFICIAL PANCREAS. The MiniMed 670G is the first FDA-approved device that measures and delivers basal insulin 24 hours per day.
“[It’s] designed to learn what an individual’s insulin needs are and to take action to minimize both high and low glucose levels,” says Alejandro Galindo of Medtronic. “The feedback we’ve heard from patients and caregivers in the clinical trial is that patients are able to trust the system and rest uninterrupted throughout the night. This has significant quality-of-life benefits, particularly for those who have struggled with the nighttime routine, which can be stressful and burdensome.”
American Diabetes Association says the MiniMed 670G breakthrough ultimately will lead to a fully automated closed-loop system that won’t require patients to enter the amount of carbs that they consume into the system.
Aaron Kowalski of Juvenile Diabetes Research Foundation says that, even though the United States is one of the more advanced countries in diabetes treatment, it still is difficult for people who have type 1 diabetes to manage their glucose levels. That puts them at risk for long-term complications, which include blindness and kidney disease, he says.
“Taking the burden off the shoulders of people, I think it really is going to be a big step forward,” he says.
Galindo says the MiniMed 670G system will be available in spring 2017. Medtronic expects that the system will cost the same—$6,000–$9,000—as its current pump systems do. Medtronic says the system will be covered by insurance, and the company will have more information about coverage when the device is available.
DISAPPEARING ACT. Coronary heart disease develops when cholesterol-containing deposits accumulate in coronary arteries and narrow those arteries, which decreases blood flow to the heart. The disease is responsible for about 370,000 deaths each year in the United States, according to National Heart, Lung and Blood Institute, which is part of National Institutes of Health (NIH).
Doctors typically use angioplasty, which widens an artery through the use of a metal stent, to treat the disease. However, the stents often cause scar tissue to form, which results in the artery narrowing again.
VANISHING. Abbott Vascular’s Absorb GT1 Bioresorbable Vascular Scaffold (BVS) System is the first fully absorbable stent.
Drug-eluting stents release a medication for a few months after they’re inserted into an artery to combat the formation of scar tissue. In July 2016, FDA approved Abbott Vascular’s Absorb GT1 Bioresorbable Vascular Scaffold (BVS) System, which is the first fully absorbable drug-eluting stent. The stent, which is made of a biodegradable polymer that’s similar to the material that’s used to dissolve sutures, widens the artery and releases the medication everolimus for 6–12 months to limit scar tissue. The stent is absorbed by the body in 3 years. All that remains are four tiny platinum markers that are embedded in the walls of the artery to help cardiologists to identify where the device was placed.
Dr. James C. Blankenship, who is the past president of Society for Cardiovascular Angiography and Interventions, which is a professional association, calls the Absorb GT1 BVS System a “paradigm shift.” (Researchers tried to develop an absorbable stent for the past 20 years, he says.) However, he says, 10–20 years of data will be required to determine whether the Absorb GT1 BVS System yields any clinical benefits, such as whether it helps patients to live longer or whether it prevents heart attacks.
In addition, he says, “there may be a psychological benefit in that people feel better [when] they don’t have a piece of metal in their body.”
Blankenship tells us that the Absorb GT1 BVS System is thicker and more difficult to insert into the artery than are conventional metal stents. Future bioresorbable stents will be thinner, he says. Filmore tells us that other absorbable stents that are in development are made of polymers that dissolve faster than does the Absorb GT1 BVS System.
Abbott launched the Absorb GT1 BVS System in late 2016 in the United States, and the company is training doctors on how to implant it in patients. (You can find a map of hospitals where it’s available at dissolvingstent.com.) Blankenship says the GT1 BVS System costs no more than does a conventional stent, which typically costs $1,200, according to ECRI Institute, which is a medical-research organization. The Absorb GT1 BVS System typically is covered by insurance, he says.
MAKING WAY. V-Wave Medical’s V-Wave Shunt treats heart failure with preserved ejection fraction.
REDUCING PRESSURE. New minimally invasive interatrial shunts show a potential to treat heart failure with preserved ejection fraction (HFpEF), which is a difficult-to-treat condition that causes pulmonary congestion. About half of the estimated 500,000 new heart-failure cases that are diagnosed annually in the United States are classified as HFpEF.
ECRI Institute says two interatrial shunts are in development: Corvia Medical’s InterAtrial Shunt Device and V-Wave Medical’s V-Wave Shunt. Neither device is approved for sale in the United States, but ECRI Institute expects an early adoption of interatrial shunts to take place in a few years.
Interatrial shunts are implanted in patients to create a permanent opening in the wall that separates left and right atrial chambers of the heart. The shunts help to relieve elevated left-atrial pressure by allowing blood to flow from the higher pressure left atrium into the lower pressure right atrium. The idea is to equalize pressure and relieve pulmonary congestion, ECRI Institute says.
The shunts are considered to be a breakthrough, because no other therapy “has proven to work to improve clinical outcomes for this population,” says Dr. Christopher O’Connor, who is the editor-in-chief of Journal of American College of Cardiology: Heart Failure. “The landscape is not crowded for new therapies in this place.”
Early studies that were published in March 2016 in The Lancet, which is a British medical journal, showed that after the InterAtrial Shunt Device and V-Wave Shunt Device were implanted, patients had fewer heart-failure symptoms and were able to exercise for longer periods.
No one could tell us how much that interatrial shunts will cost. ECRI Institute says they’ll cost somewhere between the price of a cardiac stent, which starts at $2,500, and a prosthetic cardiac implant valve, which starts at $20,000.
MANAGING MS. Experts say they expect that in late March 2017, FDA will approve Genentech’s Ocrevus (ocrelizumab), which is the first medication that shows results in the treatment of relapsing-remitting multiple sclerosis (RRMS) and primary progressive multiple sclerosis (PPMS).
RRMS is the most common form of MS, according to National Multiple Sclerosis Society (NMSS). Patients typically experience the initial symptoms, such as episodes of double vision, numbness and decreased neurological function, in their 20s and 30s. For those who have RRMS, the symptoms will disappear and relapse until the disease progresses 10–20 years later into secondary progressive multiple sclerosis (SPMS), which is characterized by symptoms that worsen.
PPMS is the most progressive form of MS. The symptoms don’t disappear and relapse. Instead, the symptoms accumulate and get worse. Experts say Ocrevus would be the first approved treatment for PPMS.
Ocrevus is designed to target CD20-positive B cells, which are immune-system cells that are believed to be a key contributor to nerve-cell damage. This nerve-cell damage leads to disabilities for people who have MS. Based on preclinical studies, Ocrevus binds to CD20-positive surface proteins that are on B cells but aren’t on plasma cells or stem cells, which preserves and doesn’t block important functions of the immune system.
“To have anything is a big deal, and this treatment opens up the door to primary progressive multiple sclerosis to being a version of the disease you can treat,” says Tim Coetzee of NMSS. “It gives people with primary progressive multiple sclerosis significant hope.”
FDA granted to Ocrevus a priority-review designation, which is granted to medications that FDA determines to have the potential to improve the safe and effective treatment of a serious disease. Ocrevus also is the first MS medication that FDA granted a breakthrough-therapy designation.
In December 2016, New England Journal of Medicine published positive results from three Ocrevus Phase III
studies on RRMS and PPMS. The data from the studies showed consistent reductions in major markers of the disease’s activity and progression. Genentech says Ocrevus showed the capability to reduce the relapse rate of RRMS and to reduce the progression of symptoms for at least 12 weeks in PPMS.
“This is quite a remarkable result in robust clinical trials,” Peter Chin of Genentech says.
Genentech says Ocrevus will be available as soon as it’s approved, but the company tells us that it doesn’t comment on the price or insurance coverage of preapproved medications.
PAINFUL PROCESS. The 40 percent–50 percent of postmenopausal women who experience dyspareunia, which is moderate-to-severe pain when they have sex, can find relief through the use of the medication Intrarosa, which FDA approved in November 2016.
Manufactured by Endoceutics, Intrarosa is the first FDA-approved product that contains the active ingredient prasterone, which is a synthetic version of the steroid hormone dehydroepiandrosterone (DHEA) and is included in certain dietary supplements for which the efficacy and safety haven’t been established.
Dyspareunia is a symptom of vulvar and vaginal atrophy that results from menopause, when the sex hormones androgen and estrogen decrease. This hormone deficiency causes the vaginal wall to thin and lubrication to decrease.
Endoceutics researchers discovered that prasterone can create androgen and estrogen in the peripheral tissues of postmenopausal women. These androgen and estrogen hormones repair the hormone deficiency in postmenopausal women and don’t release active sex steroids elsewhere in the body. In other words, no risk exists of adverse effects in other tissues, such as those that are in the uterus.
“I think this is a new paradigm of treatment of menopause symptoms,” says Dr. Fernand Labrie of Endoceutics. “What this treatment does is give back to women what’s missing in them.”
Typically, dyspareunia is treated with estrogen that’s taken orally or applied locally in the vagina. However, despite the painful symptoms of dyspareunia, only about 3 percent of affected women seek treatment for it, because they fear estrogen-related side effects, such as heart attacks, breast cancer or stroke, according to Dr. Irwin Goldstein, who is the director of sexual medicine at Alvarado Hospital.
The safety and efficacy of Intrarosa, which is a once-daily vaginal insert, were established in two 12-week trials. Compared with a placebo, Intrarosa was shown to reduce the severity of pain that women experienced during sexual intercourse. The safety of the medication also was established in a 52-week trial. The most common adverse reactions are vaginal discharge and an abnormal Pap smear.
Intrarosa is considered to be a medical breakthrough, because Endoceutics took a medication that was available as a dietary supplement—although it wasn’t approved by FDA—and made it available in an approved suppository form, Goldstein says.
“The breakthrough is to make medical use out of something already out there for years, explain how things are happening endocrinologically and really help women who have menopausal symptoms,” Goldstein says.
DECREASING DELUSIONS. An estimated 50,000 Americans are diagnosed with Parkinson’s disease each year, and about 1 million Americans have the condition, according to FDA. Delusions or hallucinations occur in as many as 50 percent of patients who have Parkinson’s disease, FDA says.
“It’s often very frightening for patients and always disturbing for caregivers and family members,” Peter Schmidt of National Parkinson Foundation tells us.
That’s why Schmidt says FDA’s April 2016 approval of Acadia Pharmaceuticals’ Nuplazid (pimavanserin), which is the first antipsychotic medication that’s approved to treat the delusions and hallucinations that are associated with Parkinson’s disease, is an important breakthrough.
FRIGHTENING. Delusions or hallucinations occur in as many as 50 percent of patients who have Parkinson’s disease, FDA says.
Parkinson’s disease typically occurs in people who are at least age 60 when brain cells that produce dopamine become impaired or die. Parkinson’s medications are designed to relieve the disease’s motor symptoms (such as stiffness and trouble walking) by increasing the brain’s dopamine levels. Unfortunately, increased dopamine levels cause delusions and hallucinations. Before Nuplazid was approved, doctors typically prescribed FDA-approved antipsychotics for bipolar disorder and schizophrenia to treat Parkinson’s-related delusions and hallucinations.
Nuplazid, which a patient takes as a tablet once per day, binds to serotonin receptors and reduces their stimulation. Other antipsychotic treatments for Parkinson’s disease, such as Haldol and olanzapine, bind to and stimulate serotonin receptors. In other words, they might reduce hallucinations, but they also worsen the primary motor symptoms of Parkinson’s disease, such as difficulty walking.
In a clinical trial, Nuplazid was found to decrease the frequency and severity of delusions and hallucinations but not to worsen the primary motor symptoms of Parkinson’s disease. The most common side effects that were reported are confusion, nausea, and swollen ankles, legs and feet because of the accumulation of excessive tissue fluid.
Nuplazid isn’t approved to treat patients who have dementia-related psychosis that’s unrelated to Parkinson’s disease psychosis. Nuplazid costs $1,950 for a 30-day supply and generally is covered by insurance, Acadia Pharmaceuticals says.
TREAT CANCER. Researchers at Dana-Farber Cancer Institute announced in November 2016 that they identified genomic changes that might predict how testicular cancer develops. The changes might help researchers to understand why the majority of testicular-cancer tumors are highly curable through chemotherapy, unlike most solid tumors. Researchers believe that this will help to explain the chemosensitivity of other types of cancerous tumors and the evolution of chemoresistance in other germ-cell tumors. In other words, the findings might unlock new ways to treat all cancer patients.
“[This study] is a good lesson in doing a deep dive and a large genomic dissection of rare cancers in a whole subset of patients who don’t do well and still need help,” says Dr. Eliezer Van Allen of the Dana-Farber research team. “The real breakthrough is setting the stage for what will be a big breakthrough of truly understanding how this process occurs.”
American Cancer Society (ACS) estimates that in 2017, 8,850 new cases of testicular cancer that have germ-cell tumors will occur in the United States. Of those cases, 410 men will die, ACS says. Most of the tumors are highly sensitive to chemotherapy, so at least 80 percent of patients who have germ-cell tumors are cured. However, a significant number of tumors become chemotherapy-resistant, and about 10 percent of patients who have chemotherapy-resistant germ-cell tumors that spread to other parts of their body die as a result.
Previous studies of testicular-tumor genomes revealed mutations and chromosomal damage but didn’t identify specific alterations that were linked to chemosensitivity or chemoresistance. Dana-Farber researchers found a type of chromosomal damage that might be linked to the development of chemosensitive germ-cell tumors. Those tumors have a gene that directs cells to make a tumor-suppressing protein. In most types of cancer, this gene is mutated or lost.
The researchers also found that, unlike most types of cancer, testicular tumor cells are poised to self-destruct by apoptosis, which is the body’s quality-control process that eliminates abnormal and unneeded cells. Many cancers block apoptosis.
“It’s a little ways away,” Van Allen says of finding a treatment for these cancers.
Memorial Sloan Kettering Cancer Center researchers say the genomic profiling of patients who have advanced germ-cell tumors could help researchers to discover cancer treatments.
“It’s up to us now to figure out how to better identify more-aggressive tumors and work on therapies to treat them,” says Dr. Joshua Meeks, who is an assistant professor of urology and obstetrics at Northwestern University Feinberg School of Medicine.
Lea Radick has reported on health-care issues for 11 years. Her work has appeared in American Health & Drug Benefits, American Libraries magazine and The Washington Post’s Express.