Advances in medicine are incremental and based on the work of countless researchers responsible for building a foundation of evidence. Every significant advance this year has been years in the making. Let’s take a look at some important and promising drugs, procedures, and diagnostics that made waves in 2019. Smart inhalers for respiratory illnesses Given the omnipresence of the Internet of Things (IoT)—a system of devices connected to the Internet and/or each other with the ability to transfer data without human-to-human or human-to-computer interaction—it was probably just a matter of time before inhalers would become “smart.” In December 2018, the FDA approved the ProAir Digihaler (albuterol sulfate 117 mcg)—the first inhaler equipped with sensors that connect to a mobile app via Bluetooth. The sensors in this inhaler register when the instrument is used to keep track of respiratory flow. These data can help guide physicians on treatment options for patients with respiratory illnesses and facilitate more informed physician-patient dialogue. “One of the challenges physicians are faced with in caring for their asthma and COPD patients is knowing if their patients are using their inhaled medication as they should. That’s what makes a product like this so important to doctor-patient discussions,” said Tushar Shah, MD, global head, Specialty Clinical Development & Medical Affairs, Teva Pharmaceuticals. “Offering a tool that enables doctors to see data on their patients’ inhaler usage will allow them to have more productive conversations about identifying issues and how to manage their illness.” Wireless brain sensors for neurological disorders By detecting brain temperature, intracranial pressure, and brain signaling, wireless brain sensors promise to revolutionize healthcare. In 2019, there was a veritable explosion in this space, with more players than ever before. These sensors are most frequently used in patients with Parkinson disease, traumatic brain injury, sleep disorders, dementia, and other neurological conditions. They help to monitor neurological fluctuations and enhance cognitive function. The wireless component allows these sensors to connect to digital devices for easier accessibility and greater cost-effectiveness. Urine test for cancer detection Diagnosing cancer can be difficult, invasive, and time intensive. Researchers from Massachusetts Institute of Technology (MIT) and Imperial College London, United Kingdom, announced that they developed a urine test to detect cancer. It works by changing the color of urine after injection with ultra-small gold nanoclusters bound to protein carriers. These nanocluster/protein complexes can be made to be sensitive to specific matrix metalloproteinases (MMPs), which are upregulated in some cancers. When the nanoclusters are present in a urine sample, the test turns blue, signaling the presence of MMPs. So far, it has successfully detected colon cancer in mice. Siponimod for multiple sclerosis Typically, multiple sclerosis (MS) first takes a relapsing-remitting course; relapses are followed by remissions. These remissions may not be complete, and patients can be left with residual disability. Moreover, many patients experience progressive disability separate from relapses and remissions, a phenomenon called secondary progressive MS. In 2019, the FDA approved siponimod for the treatment of secondary progressive MS. Siponimod is a sphingosine 1-phosphate receptor modulator that is not only anti-inflammatory, but also neuroprotective, targeting the degenerative aspect of secondary progressive MS. In a high-powered clinical trial, patients with secondary progressive MS experienced lower rates of MS progression compared with those who received placebo. Risankizumab for plaque psoriasis In 2019, the FDA approved risankizumab for the treatment of moderate to severe plaque psoriasis in adults eligible for systemic therapy or phototherapy. This drug is an interleukin (IL)-23 inhibitor; IL-23 is a principal inflammatory protein. In clinical trials, most patients receiving the drug experienced skin clearance, as evidenced by the Psoriasis Area and Severity Index (PASI 90)—a clinically important endpoint that indicates ≥ 90% decrease in PASI scoring. Erdafitinib for bladder cancer The 5-year survival rate for metastatic bladder cancer is low—about 5%. Fortunately, there may be hope for some patients with this deadly disease in the form of erdafitinib. Notably, this drug is the first fibroblast growth factor (FGFR) kinase inhibitor to garner FDA approval. Erdafitinib is used to treat patient with mutations in FGFR3 or FGFR2 that has progressed after treatment with platinum-containing chemotherapy. Patients need to be identified for treatment using a companion diagnostic device, which the FDA also approved. Overall, one of five patients with recurrent or refractory bladder cancer harbors mutations in FGFR. In one clinical trial, the overall response rate among study participants after treatment with erdafitinib was 32.2%, with the response lasting a mean of about 5.5 months. Approximately 25% of study participants had previously received anti–PD-L1/PD-1 therapy, which is a standard first-line treatment for patients with metastatic or locally advanced bladder cancer. Importantly, responses were observed in patients whose disease is refractory to anti–PD-L1/PD-1 therapy. “We’re in an era of more personalized or precision medicine, and the ability to target cancer treatment to a patient’s specific genetic mutation or biomarker is becoming the standard, with advances being made in new disease types. Today’s approval represents the first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer,” noted Richard Pazdur, MD, director, FDA Oncology Center of Excellence. “FGFRs regulate important biological processes including cell growth and division during development and tissue repair. This drug works by targeting genetic alterations in FGFRs.” CRISPR in human trials In 2018, Chinese researcher He Jiankui used CRISPR gene-editing technology to target the HIV-associated gene CCR5 in two babies. This wanton disregard for science and ethics drew reproach from scientists the world round. In 2019, investigators are, again, exploring the possible merits of CRISPR gene-editing technology—but this time, they’re doing things the right way by entering the first sanctioned human trials of CRISPR/Cas9 to treat inherited blindness via intraocular injections. Although promising, this new technology has plenty of unknowns. One potential major issue is CRISPR going to the wrong location and resulting in unintended edits or “off-target” effects, which could inhibit a tumor-suppressor gene or activate a cancer-causing one. As you can see, researchers have been quite busy, and their work has yielded promising drugs, procedures, and diagnostics. Through their research, they have helped make 2019 a year of important and exciting new developments, with many more yet to come. Source
