Top 14 Medical Innovations for 2022

Expert

Dr Alex McDonald (Board member, Physician’s Weekly) and Dr Rachel Giles (Medical Science Officer, Medicom Medical Publishers)

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Dr Alex McDonald (Board member, Physician’s Weekly) and Dr Rachel Giles (Medical Science Officer, Medicom Medical Publishers) give their perspectives, in no particular order of importance, on our choices for the Top 14 Medical Innovations for 2022. Some of these are already in practice, others are recent breakthroughs not quite ready for “prime time”. All in all, it was a great year for medical science.

1. Next Generation of mRNA Vaccinology

Advancements in the generation, purification and cellular delivery of RNA have enabled the development of mRNA vaccines across a broad array of applications, such as cancer and Zika virus infection. The technology is cost-effective, relatively simple to manufacture, and elicits immunity in a novel way. Furthermore, the emergence of the COVID-19 pandemic demonstrated that the world needed rapid development of a vaccine that was deployable around the globe. Because of previous research that laid the groundwork for this technology, an effective COVID-19 vaccine was developed, produced, approved and deployed in less than a year. This landscape-changing technology has the potential to be used to manage some of healthcare’s most challenging diseases quickly and efficiently.

2. New Treatment for the Reduction of LDL for Primary and Secondary CVD prevention

High levels of blood cholesterol, particularly low-density lipoproteins (LDL-C), are known to be a significant contributor to cardiovascular disease. In 2019, the FDA reviewed the application for inclisiran in treating primary hyperlipidaemia in adults who have elevated LDL-C while on a maximally tolerated dose of statin therapy. Inclisiran is an injectable small interfering RNA that targets the PCSK9 protein. In contrast to statins, it requires infrequent dosing (twice per year) and provides effective and sustained LDL-C reduction in conjunction with statins. Its prolonged effect may help alleviate medication non-compliance, one of the leading causes of failure to lower cholesterol levels. Inclisiran was FDA approved in December 2021 and is widely considered a game-changer for patients with heart disease.

3. Long-lasting monoclonal antibodies for RSV

Respiratory syncytial virus (RSV) is a leading cause of severe respiratory infections in infants and children, but there is currently no vaccine against this. In March 2022, 2 papers in the New England Journal of Medicine, changed all that. Nirsevimab is a monoclonal antibody that targets the F protein on the virus’s surface. It carries a YTE mutation, which extends its half-life; this means that a single dose of the antibody could protect an infant throughout their first RSV season. This is a big improvement over the current monoclonal antibody to RSV, palivizumab, which lasts about a month and therefore is used only for high-risk infants. The final results showed that single injection of nirsevimab administered before the RSV season protected healthy late-preterm and term infants and high-risk babies from medically attended RSV-associated lower respiratory tract infection. This will be interesting to follow because these studies will determine if vaccine-policymaking bodies are willing to approve a monoclonal antibody, rather than a vaccine, as a prophylactic product.

4. Novel Drug for Treatment of Type 2 Diabetes

In the United States, 1 in 10 individuals has diabetes, which affects how the body processes food into energy. One potential therapy is a once-weekly injectable dual glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide receptor agonist (GLP-1) that aims to control blood sugar. Injected under the skin, GLP-1 and GIP receptors cause the pancreas to release insulin and block the hormone glucagon, limiting blood sugar spikes after a meal. Additionally, it slows digestion, resulting in individuals remaining full longer and eating less. Thus far, late phase III clinical trials reveal that the treatment significantly reduces haemoglobin A1C in type 2 diabetes and supports weight loss, making it potentially the most effective therapy for diabetes and obesity yet developed.

5. AI for Early Detection of Sepsis

Sepsis is a severe inflammatory response to infection and a leading cause of hospitalisation and death worldwide. Because septic shock has a very high mortality rate, early diagnosis of sepsis is critical. Diagnosis can be complicated because early symptoms are common across other conditions, and the current standard for diagnosis is non-specific. Artificial intelligence (AI) has surfaced as a new tool that can help rapidly detect sepsis. Using AI algorithms, the Targeted Real-time Early Warning System (TREWS),developed in 2022, detects several key risk factors in real time by monitoring patients’ electronic medical records as physicians input information. Flagging high-risk patients can help facilitate early intervention, which can improve outcomes, lower healthcare costs, and save lives.

6. Predictive Analytics and Hypertension

Often referred to as the “silent killer,” hypertension, or high blood pressure, usually shows no symptoms while increasing risk for serious health problems, including heart disease, heart failure and stroke. Effective treatment options exist; however, many adults remain unaware that they have hypertension until they experience a significant health crisis. Using machine learning, a type of artificial intelligence, physicians are able to better select more effective medications, medication combinations, and dosages to improve control of hypertension. AI also will allow physicians to predict cardiovascular morbidities and enable physicians to focus on interventions before they occur. Predictive analytics equip providers with the key that could open the door to preventing hypertension and many other diseases.

7. Breakthrough Treatment for Postpartum Depression

Experts believe the rate of postpartum depression could be at least twice as high as what current statistics reveal because many cases go undiagnosed. Currently, counseling and anti-depressant medications are the primary treatments, but some women do not respond to these therapies. In 2019, the FDA approved an intravenous infusion treatment designed to treat postpartum depression specifically. This novel therapy, administered around the clock for 60 hours, uses a neurosteroid to control the brain’s response to stress. This treatment design is ground-breaking as it targets the signalling thought to be deficient in hormone-sensitive postpartum depression. Additionally, this treatment appears to show benefits very quickly, while traditional anti-depressants typically take two to four weeks to have a significant effect. This rapid treatment option would be a breakthrough for women with this often overlooked condition.

8. Targeted Medication for Hypertrophic Cardiomyopathy

For decades, clinicians have only been able to treat patients’ hypertrophic cardiomyopathy (HCM) symptoms—using drugs developed to treat other heart conditions—with limited effectiveness. Currently, non-specific medications are prescribed to treat some of the symptoms that HCM shares with other cardiovascular diseases. These therapies include beta-blockers, anti-arrhythmic drugs, calcium channel blockers and anticoagulants. A new treatment, however, works to reduce the root cause of the problem in many patients. Mavacamten, a first-in-class medication, specifically targets heart muscle to reduce abnormal contractions caused by genetic variants that put the heart into overdrive. By acting specifically on this mechanism in HCM patients, this novel treatment not only improves symptoms and quality of life, but potentially could slow progression of the disease. The FDA approved this therapy on April 28, 2022. It is the first medication explicitly dedicated to treating HCM and providing new hope to patients and physicians.

9. Non-Hormonal Alternatives for Menopause

Over 50% of all menopausal women experience hot flashes, which can persist for an average of 7 years. While effective and safe when used appropriately, hormone therapy involves some risk and not all patients are appropriate candidates or ready to try this treatment option. Fortunately, a new group of non-hormonal drugs, called NK3R antagonists, have emerged in 2022 as a viable alternative to hormone therapy. These drugs disrupt a signalling pathway in the brain that has been linked to the development of hot flashes and have shown promise in clinical trials for relieving moderate to severe menopausal hot flashes as effectively as hormones. While additional studies are needed to fully understand the effectiveness and safety of these new drugs, it is clear that the next generation of non-hormonal treatments for menopausal hot flashes is on the horizon.

10. Implantable for Severe Paralysis

Some form of paralysis is a common problem, and most patients experience a significant decline in their overall health. Recently, a team has offered new hope for these patients by leveraging implanted brain-computer interface technology to recover lost motor control and enable patients to control digital devices. The technology uses implanted electrodes to collect movement signals from the brain and decode them into movement commands. It has been shown to restore voluntary motor impulses in patients with severe paralysis due to brain, spinal cord, peripheral nerve or muscle dysfunction. While the interface technology is in its infancy, the FDA has designated the implantable a “breakthrough device,” reinforcing the need to move this technology to the bedside of patients who need it most.

11. CRISPR for amyloidosis

For years, nephrologists have had to watch health decline in patients with transthyretin amyloidosis. Some years ago, gene silencers became available and improved outcomes for patients, which was a very substantial advance in the field. However, patients on gene silencers need lifelong repeated treatments, and although most patients’ disease stabilises, some continue to deteriorate. Preliminary data from a phase 1 trial have shown for the first time that CRISPR-based gene therapies can be administered by intravenous infusion to edit a specific gene in liver cells. Full results revealed in 2022 showed that treatment with the investigational CRISPR-Cas9 gene-editing therapy, NTLA-2001, led to rapid responses in patients with transthyretin (TTR) amyloidosis with cardiomyopathy (ATTR-CM).

Serum levels of the disease-causing TTR protein were reduced by at least 90% at day 28 with a single infusion of NTLA-2001 at two different doses, with reductions sustained across 4 to 6 months' follow-up. This new gene-editing therapy offers patients the prospect of meaningful clinical improvements after a single dose of the drug.

12. Oral therapy for kala-azar

We were excited to see the results of a phase 3 clinical trial looking for a better regimen to treat visceral leishmaniasis, also known as ‘kala-azar’, or sleeping sickness. This trial tests new combination regimens of miltefosine and paromomycin in Ethiopia, Sudan, Kenya and Uganda in eastern Africa. The aim is to replace the toxic sodium stibogluconate component of the current injectable treatment for kala-azar with the orally administered miltefosine. This is a much-needed improvement patients have been waiting for and we have been working toward for years, as oral treatments can be administered at local health centres, closer to affected communities. Patients diagnosed with kala-azar will die if they are not treated. The new, less toxic and more easily administered regimen will also be good for children, who are disproportionately affected by visceral leishmaniasis. Children under the age of 12 represented 60% of clinical-trial patients, and preliminary results show a promisingly high efficacy for them.

13. Psychedelic therapy

Psilocybin, a psychedelic derived from fungi, is showing great promise in the treatment of mental-health conditions. One clinical trial involving participants with treatment-resistant depression reported in Nov 2022 in the New England Journal of Medicine. I am excited about this psilocybin trial for 3 main reasons. First, it tackles the huge problem of depression that is resistant to existing treatments. Second, it’s a multi-centre and multi-country trial, which will let researchers test the psychedelic therapy in a diverse group of people. Finally, it gave some readouts on the dose–effect relationship, as they tested 3 greatly differing doses of psilocybin, at 1 mg, 10 mg and 25 mg. This phase 2 trial showed that only psilocybin at a single dose of 25 mg, but not lower doses, reduced depression scores significantly, but there were adverse effects associated with this intervention. This is clearly a great first step, but also calls for larger and longer trials, including comparison with existing treatments, to determine the efficacy and safety of psilocybin for depression.

14. PSMA-Targeted Therapy in Prostate Cancer

Prostate cancer is the most commonly diagnosed cancer. Accurate imaging is critical for tumour localisation, staging the disease and detecting recurrences. PSMA, an antigen found in high levels on the surface of prostate cancer cells, is a potential biomarker for the disease. PMSA PET scans use a radioactive tracer to attach to PSMA proteins, which are then combined with CT or MRI scans to visualise the location of prostate cancer cells. In 2020, this technology received FDA approval based on phase 3 clinical trials, which showed substantially increased accuracy for detecting prostate cancer metastasis compared to conventional imaging with bone and CT scans. When detected early by PSMA PET scans, recurrent prostate cancer can be treated through a targeted approach with stereotactic body radiation therapy, surgery and/or systemic therapy in a personalised manner.

More reading (partial list):

Goodwin GM, et al. Single-Dose Psilocybin for a Treatment-Resistant Episode of Major Depression. N Engl J Med. 2022 Nov 3;387(18):1637-1648. doi: 10.1056/NEJMoa2206443.
Hammitt LL, et al. Nirsevimab for Prevention of RSV in Healthy Late-Preterm and Term Infants. N Engl J Med. 2022 Mar 3;386(9):837-846. doi: 10.1056/NEJMoa2110275.
Hammitt LL, et al. Nirsevimab for Prevention of RSV in Healthy Late-Preterm and Term Infants. N Engl J Med. 2022 Mar 3;386(9):837-846. doi: 10.1056/NEJMoa2110275.