The 18th Annual Medical Innovation Summit led by Cleveland medical center was held online. One of the hot spots was to announce the top ten medical innovation products in 2021. These ten breakthrough medical devices and treatment methods that are expected to have an impact next year involve hemoglobin disease, primary multiple sclerosis, cystic fibrosis, hepatitis C, prostate cancer, migraine and other diseases.
Gene therapy for hemoglobinopathies
Hemoglobinopathy is a genetic disease that affects hemoglobin molecules. Hemoglobin is responsible for transporting oxygen in the blood. The most common hemoglobin diseases include sickle cell disease and thalassemia, which together affect more than 330000 infants worldwide every year. Both can lead to insufficient hemoglobin and oxygen in the human body, which can damage nerves and organs, including kidneys, liver and spleen, and can be fatal. At present, the only treatment is blood and bone marrow transplantation. The latest research in the field of hemoglobinopathy has brought an experimental gene therapy. Through the most accurate science, it is hopeful to help the patient’s body generate hemoglobin molecules with normal functions.
Novel drug for primary progressive multiple sclerosis
Multiple sclerosis (MS) is a chronic disease caused by the abnormal attack of the human immune system on the myelin sheath of nerve cells in the brain, spinal cord and optic nerve. It is characterized by muscle weakness, fatigue and visual difficulty, which eventually leads to disability. There are about 2.3 million MS patients worldwide, which can not be completely cured at present.
Primary multiple sclerosis is a neurodegenerative disease of spinal cord, which is called primary lateral sclerosis. FDA approved a new therapeutic monoclonal antibody with new targets. Roche Group’s drug ocrevus ® （ Ocrelizumab) is used to treat active recurrent multiple sclerosis defined by clinical and imaging features and early primary progressive multiple sclerosis, so as to improve the imaging features of disease progression, disability and inflammatory activity. It is reported that ocrelizumab is the first and only therapeutic drug for patients with primary progressive MS.
Smartphone connected pacemaker device
For the first time, the cardiac pacemaker is connected with the smartphone / tablet used by patients every day, seamlessly entering the patients’ lives for safe and direct communication, which is easy to track, understand and pay attention to the patients’ heart health, so as to make the interaction between patients and doctors more close and efficient. Especially during covid, the need to visit in person is reduced. The most advanced technology provides the most advanced care.
New medicine for cystic fibrosis
Cystic fibrosis is a hereditary lung disease characterized by viscous mucus that blocks the airway and accumulates bacteria, leading to infection, inflammation and other complications. The disease is caused by a defect in CFTR protein. Previously developed drugs were only effective for people with highly specific mutations.
Elexacaftor / tezacaftor / ivacaftor is a fixed dose combination of CFTR regulator and chlorine channel opener. It has been approved by FDA in October 2019 for CF patients with at least one f508del mutation in CFTR gene to alleviate the disease. It is estimated that patients with f508del mutation account for 90% of patients with the disease.
Universal hepatitis C treatment
Hepatitis C is listed as a “silent epidemic” by the U.S. Centers for Disease Control and prevention, and has become a major public health problem in the United States. Infection with hepatitis C virus can lead to serious life-threatening health problems, such as liver failure, liver cirrhosis and liver cancer. Because there is no vaccine against the virus, patients can only use drug treatment, but many treatments are accompanied by adverse side effects or only effective for some genotypes.
Sofosbuvir / velpatasvir was approved as the optimal regimen for the treatment of adult hepatitis C. The drug can effectively inhibit proteins that play a key role in HCV RNA replication. Most patients only need to take one pill a day for 12 weeks. It covers six hepatitis C genotypes 1-6, with an effective rate of more than 90%, providing a more effective treatment option for a wider range of patients.
Bubble CPAP for increased lung function in premature infants
Premature infants who are underweight and frail usually need special care. For patients with infant respiratory distress syndrome (IRDS), surfactants are usually given to infants during mechanical ventilation, which may cause long-term lung injury and lead to the development of chronic lung disease. Different from mechanical ventilation, B-CPAP is a non-invasive ventilation strategy, which can provide newborns with continuous positive airway pressure and maintain vital capacity during exhalation. Oscillation rather than constant pressure enhances safety and effectiveness. When used for a long time, it can minimize physical damage and stimulate lung growth.
Increased access to telemedicine through new practice & policy changes
During covid-19, we saw more and more online diagnosis and telemedicine. Since March, U.S. state and federal regulators have taken rapid action to break down barriers and remove obstacles. These fundamental changes in government and medical provider policies have enabled more and more virtual nursing models and telemedicine to be realized. It not only speeds up patients’ access to medical services, but also protects medical workers and community members. These measures have opened the gate for telemedicine and spawned many new networks and projects.
Vacuum induced uterine tamponade device for postpartum hemorrhage
Postpartum hemorrhage (PPH) is a terrible complication after delivery. Pregnant women with postpartum hemorrhage may need blood transfusion, use drugs with dangerous side effects, long-term surgery, and even have to urgently remove the uterus, and eventually lose their fertility.
It is a common hemostatic method to compress the bleeding site in the uterus with balloon in a non-surgical way. The latest progress in this field is remarkable. The vacuum induced uterine tamponade uses the negative pressure generated in the uterus to collapse the bleeding cavity, which is more physiological than balloon compression. This device provides another minimally invasive means for clinicians to deal with postpartum complications. In addition, this low-tech solution is also easier to benefit resource poor developing countries.
PARP inhibitors for prostate cancer
Prostate cancer is a common male cancer. Both rucaparib and olaparib were approved by FDA for the treatment of prostate cancer in May 2020, which broadened the treatment options for men all over the world and opened the door to improve the survival rate.
Rubraca (rucaparib) is a poly ADP ribose polymerase (PARP) inhibitor, which can block the repair of damaged DNA in cancer cells, resulting in cancer cell death. On May 15, 2020, FDA accelerated the approval of rubraca for the treatment of adult patients with BRCA gene mutation metastatic castration resistant prostate cancer (mcrpc) who had previously received androgen receptor targeted therapy and taxane chemotherapy. This is the first PARP inhibitor approved for the treatment of prostate cancer. Olaparib is a drug that lacks the side effects of chemotherapy. It can target Achilles tendon in prostate cancer, but its ability to repair damaged DNA is weak. Now, this drug may be approved as the first gene targeted therapy for prostate cancer.
Immunologics for migraine prophylaxis
Migraine is a very common chronic disease. According to the statistics of the World Health Organization (who), one in ten people in the world is suffering from migraine, and the number of women is three times that of men. Patients often have disease attacks. Symptoms include headache, photophobia, fear of sound, hallucinations and nausea. At present, the common drugs for acute migraine are traptans. However, these drugs have side effects that cause vasoconstriction, so patients at risk of cardiovascular disease cannot use them.
On February 22, lundbeck, Denmark, announced that vyepti (eptinezumab jjmr) was approved by the US FDA for the preventive treatment of migraine in adults. The recommended dose is 100 mg every 3 months; Some patients may benefit from a dose of 300 mg. Vyepti is the first FDA approved intravenous (IV) drug for the prevention of migraine.