Top 10 Medical Innovations in 2021: Gene Therapy, CD20 Monoantinance, PARP Inhibitors, etc.

The Cleveland Clinic recently named the Top 10 Medical Innovations of 2021.
selected innovative technologies/therapies, including CD20 targeted therapy ocrelizumab, a new drug for cystic fibrosis, Trikafta, hepatitis C pangene therapy, PARP inhibitors and many other innovative drugs.
in the future to improve treatment effectiveness and change healthcare," said a team of experts at the Cleveland Clinic.
Today, we're going to introduce you to these top ten technologies and drugs related to medical innovation.
1. The gene therapy for hemoglobin disease is a group of hereditary blood diseases caused by abnormal hemoglobin molecular structure, or abnormal rate of globin peptide chain synthesis.
most common diseases are sickle cell anemia and thalassemia, which affect more than 330,000 children worldwide each year.
New research based on the disease has led to an experimental gene therapy that gives patients the potential to make functional hemoglobin molecules, which can reduce the presence of sickle cells or ineffective red blood cells in thalassemia and thus avoid complications.
Clinically, the gene therapy CTX001 from CRISPR Therapeutics has obtained positive interim data in phase 1/2 clinical trials, with one patient with blood transfusion dependence β
This is an innovative gene-edited hematopoietic stem cell that uses CRISPR gene-editing technology to increase the ability of hematopoietic stem cells obtained from the patient to express fetal hemoglobin (HbF) and then return the modified hematopoietic stem cells to the patient.
HbF can compensate for functional deficiencies in the patient's adult hemoglobin, thereby alleviating the patient's symptoms.
2. The new drug for the treatment of primary multiple sclerosis (MS) is a chronic disease of the central nervous system that disrupts the normal functioning of the brain, optic nerve and spinal cord through inflammation and tissue damage.
progression of MS leads to increased loss of physical function (e.g. walking) and cognitive function (e.g. memory).
about 15% of MS patients are primary progressive, characterized by gradual onset and steady development of symptoms and symptoms.
to slow the progression of MS disease, selective targeting of CD20-positive B cells is a viable pathway - a type of cell that is thought to be the key immune cell that causes damage to myelin and axons (nerve cells).
The single-anti-drug ocrelizumab, developed by Roche to target CD20-positive B cells, is the first innovative FDA-approved treatment for patients with primary progressive MS, according to the Cleveland Clinic's official website.
It has been recognized and prioritized by the FDA for breakthrough therapies, which can be machine-binding to certain B cells that express CD20 antigens to inhibit their function, but not stem cells or plasma cells, so that important functions of the immune system are preserved.
, ocrelizumab is designed as an injectable drug that requires only two infusions per year and is expected to significantly improve compliance in patients with multiple sclerosis.
devices such as pacemakers and defibrillators that connect smartphones transmit electrical impulses to the heart muscle cavity to stimulate heart beats.
can be used to prevent or correct arrhythmic arrhythmics, such as slow or excessive heart rhythms.
monitoring of these devices is an important part of care.
in traditional treatments, the device's remote monitoring is carried out through the bedside console and the data is transmitted to the doctor.
the clinical use of pacemakers is already high, many people lack understanding of the device and its function.
, remote monitoring is less compliance.
bluetooth-enabled pacemaker device corrects disconnections between patients and their heart treatment.
these devices, in conjunction with mobile applications, enable patients to gain a deeper understanding of their health data, which can also be passed on to doctors in a timely manner, allowing patients and doctors to interact more closely and efficiently.
4. Cystic fibrosis therapy for the new drug cystic fibrosis (CF) is due to the coded cystic fibrosis transfilm conductivity regulatory factor (CFTR) gene mutation, resulting in CFTR protein deficiency or functional defects caused by genetic diseases.
CFTR protein functional defects or deficiencies lead to uneven inflow and outflow of salt and water in many organ cells, an abnormally viscous build-up of mucus in the lungs, chronic lung infections and aggressive lung damage, and ultimately death.
about 2,000 known mutations in the CFTR gene, while previously developed drugs have only been effective in patients with certain mutations.
2019, the FDA approved the launch of Vertex Pharmaceuticals' combination therapy Trikafta (elexacaftor/tezacaftor/ivacaftor), which is expected to treat 90 percent of patients with cystic fibrosis.
, elexacaftor is a new generation of CFTR protein correctors that restore the function of CFTR proteins carrying F508del mutations, thereby improving respiratory function in CF patients;
approval of the breakthrough therapy trikafta gives thousands of patients with an F508del gene mutation and a minimal functional mutation access to innovative therapies that target cystic fibrosis pathogenic mechanisms.
5. Hepatitis C (HCV), a universal treatment for hepatitis C, is known as the "silent killer", one of the most common liver diseases and a common public health problem for all mankind.
estimates that about 3 per cent of the world's population (about 150 million people) is affected by hepatitis C.
treatment, hepatitis C can lead to serious consequences such as liver fibrosis, cirrhosis, and even hepatocellular carcinoma.
, however, the discovery of the hepatitis C virus and the development of related therapies have made hepatitis C one of the few curable chronic diseases in medical history.
, this year's Nobel Prize in Physiology or Medicine awarded three scientists who discovered the hepatitis C virus (congratulations!). Hepatitis C virus discoverer won the 2020 Nobel Prize in Physiology or Medicine. Attached: Winners in the last 10 years).
hepatitis C can be divided into 1 to 6 types according to genotype, the initial birth of several therapies only for some of the subtypes of hepatitis C patients, such as NS5B polyenzyme nucleoside inhibitors, NS3/4a protease inhibitors.
in 2016, the oral cocktail therapy epclusa (C-tonsa) was born that can treat all hepatitis C subtypes.
, generic genotype hepatitis C therapy has been born, such as Vosevi, Afyi Noquan (Gkarivir/Pyrenthavir) and so on.
the emergence of universal hepatitis C therapy has greatly simplified the treatment of patients with hepatitis C, and is expected to reduce the enormous burden of HCV at the public health level.
Expand Reading: From "Silent Killer" to 100% Cure, Review China's 3-Year Path to Conquering Hepatitis C 6. Baby CPAP, which improves lung function in premature babies, often requires specialized care, including ventilation for premature babies with neonatal respiratory distress syndrome (IRDS).
For premature babies, surfactants are usually given to babies during mechanical aeration, although this practice can cause lasting lung damage and lead to the development of chronic lung disease.
unlike mechanical ventilation, the Baby Positive Pressure Respiratory Therapy System (Bubble CPAP) is a non-invasive ventilation strategy that provides continuous positive pressure ventilation support to newborns as they breathe and maintains lung capacity.
when used for long periods of time, it minimizes physical damage and stimulates lung growth.
7. Innovative practices and policy changes have increased access to telemedicme during the COVID-19 (disease caused by the new coronavirus), which is increasingly being used due to the need for doctors to consult patients online.
thanks to innovative practices and policy changes, telemedic services such as virtual care models have been developed that not only enable patients to receive rapid treatment, but also protect health care providers and community members.
these changes have also opened the door to telemedicies, driving the development of existing Internet medical services while also spawning new projects.
. Vacuum-induced postpartum hemorrhage, which controls postpartum bleeding, is a serious postnatal complication, with about 1 to 5 per cent of mothers reporting postpartum haemorrhage.
who bleed after childbirth may need blood transfusions, medications that can cause dangerous side effects, undergo lengthy and uncomfortable operations, or even have their uterus removed urgently and lose fertility.
is a common non-surgical method of hemorrhage by using the balloon to compress the bleeding area in the womb, but it also causes the uterus to expand.
recent advances in this field have provided a new treatment for mothers who bleed after child-rearing.
Vacuum-induced uterine filler is a method of using negative pressure generated in the uterus to collapse the bleeding chamber, which is more in line with the physiological structure of women than balloon compression and avoids the pain caused by invasive treatment.
induction device provides a new minimally invasive tool for clinicians to treat postparto complications.
and the device is low-tech and can benefit more patients.
9. PARP inhibitors treat prostate cancer PARP (poly-adenosine phosphate keruclease) inhibitors are a treatment that kills cancer cells using the principle of "synthetic death".
"synthetic death" refers to the phenomenon of two non-lethal genes being suppressed at the same time, leading to cell death.
used this mechanism to find specific mutations in tumors, and then to find its "synthetic lethal partner", which in turn specifically kills cancer cells.
PARP is an important DNA repair protein in cells.
PARP inhibitors can cause cancer cell death by inhibiting PARP-mediated DNA damage repair mechanisms that lead to the accumulation of excessive DNA damage in tumors carrying BRCA gene mutations.
the efficacy of PARP inhibitors has been proven in both breast and ovarian cancers.
but studies have shown that PARP inhibitors can also be used to treat prostate cancer in men.
May 2020, the FDA approved two PARP inhibitors to treat prostate cancer: one for Clovis Oncology's rucaparib, which was approved to treat metastatic despotic resistance to prostates carrying BRCA gene mutations Adult patients with cancer (mCRPC); another, Olapali, developed jointly by AstraZeneca and MSD, has been approved for the treatment of mCRPC patients with mutations in the HRR gene.
cancer is the second most common cancer among men.
mCRPC is a serious type of prostate cancer that has spread to other parts of the body, and tumors continue to multiply even though the body's androgens have been reduced to very low levels.
the number of treatments available to men for mCRPC increased, the five-year survival rate was still low.
approval of PARP inhibitors has opened up new treatment options for mCRPC patients.
10. The immunotherapy drug CGRP for migraine prevention is called calcitonin gene-related peptides.
the 1990s, scientists first discovered elevated levels of CGRP in the blood during migraine attacks, and found that CGRP released by the Trident nervous system was the "switch" that triggered migraine attacks.
findings make the CGRP signaling pathrapy a popular target for the treatment of migraines.
several antibody therapies for migraine prevention, including erenumab-aooe, fremanezumab-vfrm, galcanezumab-gnlm, etc., have been approved worldwide for antibody therapy of CGRP subjects or CGRP itself.
recent year, a new breakthrough has been made in migraine treatment.
CGRP-influenced antagonist ubrogepant developed by Allergan and Biohaven's CGRP-inhibitor Rimegepant have been approved by the FDA for the treatment of acute migraines.
addition, in February 2020, the FDA approved Lundbeck's Vyepti (eptinezumab-jjmr) for preventive treatment of migraines in adults.
, Vyepti is the first intravenous drug approved by the FDA to prevent migraines, according to a press release.
migraine is a very common chronic disease.
one in ten people in the world suffer from migraines, three times as many as men, according to the World Health Organization.
often have episodes of the disease, which include headache, fear of light, fear of sound, hallucinations and nausea.
the arrival of CGRP-targeted drugs has entered a new era in the treatment of migraine patients.
: 1 . . . Cleveland Clinic Top 10 Medical Innovations For 2021. Retrieved Oct 6, 2020, from CRISPR Therapeutics and Vertex Announce Positive Safety and Efficacy Data From From First Two Patients Tr.