Cutting-Edge Care

2018 was a year filled with medical advances, innovations and medications that might someday save your life. 

You don’t have to be a scientist to appreciate how far we’ve come in the world of medicine and health care. That’s definitely a good thing, as many of the treatments used as recently as the last couple of centuries now sound downright maniacal (kerosene to treat head lice, morphine syrup to ease babies’ teething pain and hydroeclectric baths for chronic migraine sufferers, for example).

Although these practices seem bizarre today, they were once accepted treatments. Fortunately, we’ve come a long way. Just this last year has revealed a number of impressive medical advances currently available or on the horizon, as well as new drugs approved by the U.S. Food and Drug Administration (FDA) to treat a wide range of diseases/conditions.

New Way to Treat Obstructive Sleep Apnea

For years, the often cumbersome and uncomfortable CPAP (continuous positive airway pressure sleep mask) device has been the go-to treatment for obstructive sleep apnea, a common sleep disturbance that impacts as many as 21 million Americans. People suffering from the condition but who have difficulty wearing a CPAP device may be interested in a different approach.

Now, new technology utilizes neuromodulation via an implant to open key airway muscles during sleep. The system is controlled by a remote or wearable patch and helps synchronize air intake using a breathing sensor and stimulation lead, which is powered by a small battery. 

According to a journal article on the International Neuromodulation Society website, this technology functions as a “pacemaker” by stimulating the genioglossus muscle in the upper airway and making it contract, which widens the airway and increases airflow. Neurostimulation is reserved for patients who have trouble tolerating the standard obstructive sleep apnea treatment.

Reducing Chemotherapy Hair Loss

A new technology called “scalp cooling” (also known as “scalp hypothermia”) has been highly effective in lessening hair loss in women receiving chemo to treat early-stage breast cancer. 

According to the American Cancer Society (ACS), scalp hypothermia, which was approved by the FDA last year, involves cooling the scalp with ice packs or cooling caps for a period of time before, during and after each chemotherapy treatment. As noted on the ACS website, “Newer versions of these devices use a two-piece cooling cap system that is controlled by a computer, which helps circulate a cooled liquid through a cap a person wears during each chemotherapy treatment. A second cap, made from neoprene, covers the cooling cap to hold it in place and keep the cold from escaping.” 

According to cancer.org, the cooling tightens up or constricts the blood vessels in the scalp. This constriction is believed to reduce the amount of chemo medicine that ultimately reaches the cells of the hair follicles, thereby preventing excess hair loss.

Lab-Grown Organs

It sounds like science fiction, but it’s true. A team at the University of Texas Medical Branch (UTMB) has not only grown lungs in the lab but then successfully transplanted those bioengineered organs into pigs, whose systems are remarkably similar to humans.

As reported in an article in The Telegraph this August, scientists took the lung of one pig and removed the blood and cells, stripping it down to “skeleton” level. They then took nutrients and lung cells from the pig that was to receive the new organ and placed them in a tank with the lung “skeleton.” After 30 days, the bioengineered organ was ready for transplant. Not only were the lab-grown lungs quickly accepted by the pigs after transplants, but within two weeks, they had already developed a network of blood vessels. Two months after transplant, the lungs were still functioning and the pigs had 100 percent oxygen saturation. All of the animals stayed healthy. 

According to Joan Nichols, Professor of Internal Medicine at UTMB, the ultimate goal is to provide new options for the thousands of people on lung transplant waiting lists.

“Somewhere down the line we may be able to take stem cells from a person and produce an organ that is their organ, tissue matched to them, with no immune suppression needed that would function the way their own lung originally did,” Nichols stated.

Researchers anticipate human trials could begin in the next five to 10 years. 

Worried About Alzheimer’s? Get Moving!

Alzheimer’s disease is the sixth leading cause of death in this country, and approximately 5.3 million Americans are living with the disease.

Ocala neurologist Jose A. Gaudier, M.D., P.A., notes that several medicines designed to help prevent Alzheimer’s are currently in the research phase. 

“In medicine, we often talk about ‘prevention,’ which while widely understood in the non-medical circuits as avoidance of getting a disease, in medicine it means decreasing the risk,” explains Gaudier. “Exercise has been widely considered among neurologists and neuroscientists as the best way to delay dementia onset, along with diet.” 

Gaudier points out the results of a two-year controlled study in Finland of adults at risk for dementia. This is the first study to prove that a multi-domain intervention of diet, exercise, cognitive training and vascular risk monitoring versus control can improve or sustain cognition in elderly individuals, something medical treatment studies have failed to do.

Adults over age 65 should aim for at least 150 minutes per week of moderate-intensity aerobic exercise training (brisk walking is a good option), 75 minutes per week of vigorous-intensity aerobic exercise training or a combination of the two supplemented by muscle strengthening activities (such as dynamic resistance training) two days a week or more. 

No Egg Or Sperm Needed

Forget the traditional “ingredients” of conception. Using only stem cells, “embryologists working at the University of Cambridge in the UK have grown realistic-looking mouse embryos,” according to the MIT Technology Review.

The magazine heralded the accomplishment as one of its “10 Breakthrough Technologies of 2018.”

“We know that stem cells are magical in their powerful potential of what they can do. We did not realize they could self-organize so beautifully or perfectly,” said Magdelena Zernicka­-Goetz, who headed the team.

The next step? Creating an artificial embryo from human stem cells. That’s exactly what researchers at the University of Michigan and Rockefeller University are currently working on. Obviously, this raises many ethical questions, but making artificial embryos could allow researchers to study the beginning of human life. 

Fighting Cancer With Immunotherapy 

More strides have been made using immunotherapy techniques, which allow the body’s own immune system to fight back against cancer. Researchers at UCLA’s David Geffen School of Medicine are studying treatments using antibodies that “disrupt” the shield of cancerous tumors, making them more vulnerable to attack. They are also working on treatments in which cells are removed from the patient’s immune system, reengineered and then returned to the body to fight against cancer. 

“Immunotherapy—or using the body’s own immune system to fight cancer—has literally changed the way we treat cancer and given real hope to patients who previously had very few options,” says Lucio Gordan, M.D., with Florida Cancer Specialists—Gainesville. “While immunotherapy is not yet used in treating all types of cancer, there is a vast amount of research being conducted that we anticipate will produce many more viable drugs in the future.”

Therapeutic Cancer Vaccine

According to a news release dated July 10, 2018, Moffitt Cancer Center has recently entered into a licensing agreement with the company MultiVir Inc. to pursue a personalized cancer vaccine. The therapeutic vaccine is comprised of a patient’s dendritic cells (a type of immune cell), which are treated with a genetically engineered adenovirus carrying the human p53 gene (Ad-p53). If successful, this could provide a new option for patients with relapsed/recurrent small cell lung cancer.  

A clinical trial, which is scheduled for completion in April 2020, will evaluate the use of the Ad-p53 Dendritic Cell Vaccine in combination with the immunotherapy drugs Opdivo® (nivolumab) and Yervoy® (ipilimumab). 

“This investigational therapy could provide a new approach for patients with relapsed/recurrent small cell lung cancer. This group of patients, which is about 15 percent of all lung cancer patients, has a low survival rate depending on what stage the cancer is when diagnosed,” says Scott Antonia, M.D., Ph.D., co-inventor of the vaccine and chair of the Thoracic Oncology Department at Moffitt. “We hope this combination therapy will spark the immune system to attack the cancer with minimal side effects for the patient.”

“There is a lot of potential with this investigational therapy. The p53 mutation is seen in almost half of all cancers. If there is success in small cell lung cancer, there could be an opportunity to expand the vaccine’s use for other cancers,” adds Albert A. Chiappori, M.D., co-principal investigator of the clinical trial and senior member of the Thoracic Oncology Department at Moffitt.

Help For Retinal Diseases

FDA approval is expected imminently for a new gene therapy to treat inherited retinal diseases such as retinitis pigmentosa and Leber Congenital amaurosis (LCA). The therapy is innovative in that it “delivers a new ‘normal’ working copy of the gene that results in a functional protein.” The gene is then placed within a modified virus, and that viral vector actually delivers it to retinal cells, resulting in improvement in visual function. 

“Retinal gene therapy has advanced eons in the past 10 years,” wrote Christine Kay, M.D., in the February 2017 issue of Retinal Specialist. “With innovations like optogenetics, we can imagine a future where multiple different diseases can be treated with a larger window of opportunity for therapeutic effect. Although exciting to the clinical community, these advances will be even more attractive to our patients who, until very recently, have been told at yearly follow-ups, ‘There is nothing that can be done.’ We are finally at a point where we can offer realistic hope.”

Telemedicine Technology

Researchers say telemedicine technology, also referred to as “telehealth” or “distant health technology,” is a lifesaving advance in medicine. This process removes barriers and extends the health care environment by equipping the patient with attachable devices that record and report medical information, allowing doctors to monitor a patient’s condition when the patient is at home. The goal is not only to make care more efficient but to ultimately have better outcomes.

“There is now a plethora of tools, including wearables and other devices that are generating physiological data such as respiratory and heart rate and rhythm, oxygen saturation and sleep patterns, which by themselves may be helpful if there is a predisposition toward specific abnormalities,” observes William Dalton, M.D., Ph.D., a medical oncologist at Moffitt Cancer Center in Tampa.

In 2018, over 19 million patients were expected to use such remote monitoring devices. Look for telemedicine technologies to increase in the near future as telehealth programs become more common across the country. 

Medications New To The Market

A number of new drugs were approved this year by the FDA. If you have any of the following conditions, you may want to ask your doctor about these new options. Please note, this is not a complete listing of every recently released medication. 

Aimovig (erenumab-aooe): For preventive treatment of migraine in adults (approved May 2018)

Annovera (segesterone acetate and ethinyl estradiol vaginal system): Vaginal ring to protect against pregnancy for an entire year (approved August 2018)

Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide): For treatment of HIV-1 infection in adults (approved February 2018)

Braftovi (encorafenib): For treatment of unresectable or metastatic melanoma (approved June 2018)

Doptelet (avatrombopag): For treatment of thrombocytopenia in adults with chronic liver disease scheduled to undergo a procedure (approved May 2018)

Epidioloex (cannabidiol): For treatment of rare, severe forms of epilepsy (approved June 2018)

Erleada (apalutamide): For treatment of prostate cancer (approved February 2018)

Galafold (migalastat): For treatment of Fabry disease in adults (approved August 2018)

Ilumya (tildrakizumab-asmn): For treatment of plaque psoriasis (approved March 2018)

Jynarque (tolvaptan): For treatment of autosomal dominant polycystic kidney disease (approved April 2018)

Lokelma (sodium zirconium cyclosilicate): For treatment of hyperkalemia (approved May 2018)

Lucemyra (lofexidine hydrochloride): For non-opioid treatment for management of opioid withdrawal symptoms in adults (approved May 2018)

Lutathera (lutetium Lu 177 dotatate): For treatment of gastroenteropancreatic neuroendocrine tumors (approved January 2018)

Mektovi (binimetinib): For treatment of unresectable or metastatic melanoma (approved June 2018)

Olumiant (baricitinib): For treatment of moderately to severely active rheumatoid arthritis (approved May 2018)

Orilissa (elagolix sodium): For management of moderate to severe pain associated with endometriosis (approved July 2018)

Pifeltro (doravirine): For treatment of HIV-1 infection in adults (approved August 2018)

Poteligeo (mogamulizumab-kpkc): For treatment of two rare types of non-Hodgkin’s lymphoma (approved August 2018)

Symdeko (tezacaftor/ivacaftor): For treatment of cystic fibrosis in patients age 12 years and older (approved February 2018)

Tibsovo (ivosidenib): For treatment of patients with relapsed or refractory acute myeloid leukemia (approved July 2018) 

TPOXX (tecovirimat): For treatment of smallpox (approved July 2018)

Trogarzo (ibalizumabuiyk): For treatment of multi-drug resistant HIV-1 infection (approved March 2018) 

Zemdri (plazomicin): For treatment of adults with complicated urinary tract infections (approved June 2018)  

Sources: fda.gov, clevelandclinic.org, medicalexpress.com, techrepublic.com, centerwatch.com, amprogress.org
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