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How can Zolgensma gene therapy help children with spinal muscular atrophy?

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On September 29, 2021

In cellular basis of life, muscular system, nervous system

TeachableMedicalNews article 09302021

Teachable moment in classrooms:

  1. cellular basis of life chapter – role of mRNA in protein synthesis
  2. cellular basis of life chapter – concept of gene mutation leading to protein malfunction
  3. muscular system chapter – role of nerve impulses in directing muscle contraction
  4. nervous system chapter – functions of dendrites and axons of neurons

The news item:  Recently the following reporting was published by BBC news organization:

‘Gene therapy is a game changer for our son’

A five-month-old becomes the first person in England to get a drug with a list price of £1.79m.

The article describes the symptoms, such as loss of mobility in infants, of the spinal muscular atrophy.  The article also mentions the mutant SMN1 gene, and the Zolgensma treatment that replaces the mutated gene. The article states that the frequency of this disorder is 1 in 10,000 births.

So, Why Do I Care??  Most children whose symptoms begin after age 2 have a near normal life expectancy. However, newborns with the “infant-onset” form (about 400 newborns each year) have a very short life expectancy. That is devastating to parents, too, who are helpless while their child lays motionless, and not developing motor skills for movement or speech. Innovative treatments not only raise the hope for a more normal life for the affected infants, but also points to possible treatments of similar disorders.

Plain English, Please!!!  First, let’s talk about what is spinal muscular atrophy (SMA).  This disorder originates from damaged neurons in the spinal cord. The consequence of that damage is that skeletal

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How do we teach the immune system of a cancer patient to attack cancer?

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On July 30, 2021

In cardiovascular system, cellular basis of life, lymphatic and immune system, tissues

TeachableMedicalNews article 07302021

Teachable moment in classrooms:

  1. cellular basis of life chapter– artificial DNA can direct the cell to make a new type of protein
  2. cellular basis of life chapter – receptor proteins are located in the plasma membrane
  3. tissue chapter –the liquid connective tissue nature of blood allows the separation of blood cell types
  4. immune system chapter – role of T cell in immune defense

The news item:  Recently a news report was published about a cancer patient’s fight for her life:

New treatment option for patients battling aggressive form of blood cancer

People battling relapsed or refractory (R/R) Diffuse Large B-cell Lymphoma, an aggressive blood cancer, experience a challenging treatment journey. But a recently approved CAR T cell therapy gives patients a new option.

 

The article stated that the patient had B cell lymphoma that didn’t respond to chemotherapy, and that she started a new treatment where her T cells were removed from her blood, reprogrammed to attack her cancer, and then returned into her body.  The treatment was described as the CAR T treatment where CAR stood for chimeric antigen receptor.

So, Why Do I Care??   Some leukemia type cancers and lymphoma type cancers do not respond to regular chemotherapy. While the total number of the patients is relatively low (a few thousand), the  new approach (CAR T) to cancer treatment now presents a new option for those patients with cancers resisting regular chemotherapy. Knowing how this type of treatment is created allows us to understand treatment regimens such as Kymriah, Yescarta, and Tecartus. In addition, the CAR T treatment type may inspire more out of the box thinking to defeat cancer.

Plain English, Please!!! First, let’s talk about why would we want to train our immune system. In our body only the cells of the immune system are the only ones that can find and destroy specific targets

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Can we teach our immune system to attack cancer by reprogramming white blood cells?

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On June 29, 2021

In cardiovascular system, cellular basis of life, lymphatic and immune system

TeachableMedicalNews article 06282021

Teachable moment in classrooms:

  1. cellular basis of life chapter – some proteins are located in the plasma membrane
  2. blood chapter – hematopoiesis (blood cell formation) in red bone marrow
  3. immune system chapter – role of T-cells in immune defense

The news item:  Recently an article reported on the struggles of a patient suffering with acute lymphoblastic leukemia:

 

Richmond woman benefits from game-changing cancer treatment now offered at VCU

If you knew what Rachel Elliot has been through you might call it a miracle that she is alive and well today, but hers was a miracle over a decade in the making.

The article described a new treatment called Kymriah, a Car-T type treatment, and stated that it involves genetic engineering T-cells.

So, Why Do I Care??  Some cancers resist traditional chemotherapy treatments. Patients suffering from those cancers have very limited life expectancy.  While the total number of ALL cases, close to 6,000 each year, are relatively few, children under the age of 5 are affected mostly.  New cancer treatment approaches that think outside the box are the treatments that bring hope, and eventually cures to those patients.

Plain English, Please!!!

First, let’s talk about what acute lymphoblastic leukemia (ALL) is. This type of leukemia is the cancer of the red bone marrow, and the uncontrolled division of some cells in the bone marrow leads to a large

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Can Alzheimer’s disease be detected early from a blood test?

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On May 31, 2021

In nervous system

TeachableMedicalNews article 05312021

Teachable moment in classrooms:

  1. brain chapter – locations of storing short and long term memory in the brain
  2. nervous system chapter – functioning of neurons
  3. introduction to the human body chapter – basics of PET scan technology

The news item:  Recently the availability of a blood test for Alzheimer’s disease was reported:

First blood test to help diagnose Alzheimer’s goes on sale

How is Alzheimer’s diagnosed? New blood test to help diagnose Alzheimer’s is not approved by the FDA.

 

 

The article states that this test is the first commercially available blood test for Alzheimer’s disease, and that it is a more practical alternative to the PET scan, but the test is not officially approved by the FDA, and the accuracy is still under study. The article also states that the test, developed by the company C2N,  measures amyloid and other protein.

So, Why Do I Care??  There are more than 6 million people afflicted with Alzheimer’s disease in the US. This number will increase as the proportion of older people increases in the US. Early detection of this disease could allow the implementation of preventative strategies such as changes in lifestyle, or even new medications.  Therefore the development of a blood test for early diagnosis is an exciting development, despite the fact that the accuracy of this test needs to be fully proven.

Plain English, Please!!! First, let’s talk about what Alzheimer’s disease is. Dementia type disorders result in the loss of memory, the ability of the patient to recall names or past events. Alzheimer’s disease is a form of dementia disorders; most people with dementia suffer from Alzheimer’s. Because the storage

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Can we decrease people’s cat allergies just by changing the cat food?

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On May 1, 2021

In lymphatic and immune system, respiratory system

TeachableMedicalNews article 04302021

Teachable moment in classrooms:

  1. immune system chapter – IgE antibodies are made in response to allergens
  2. immune system chapter – interactions of antibody with specific targets
  3. immune system chapter – elements of anaphylactic reaction

The news item:  Recently several news outlets reported on a new cat food, Purina’s Pro Plan LiveClear, that reduces people’s allergic reaction to cats:

 

Purina Pro Plan Releasing Cat Food that Can Help Ease Cat Allergies in Humans

Purina Pro Plan LiveClear is designed to safely neutralize Fel d1, the main allergen that causes cat allergies, in your cat’s saliva

The article states that Fel d1 is the major cat allergen, and the new cat food makes cats to carry less of this allergen on their fur.

So, Why Do I Care??

About 10-30% of people are prone to develop allergies to cats. 95% of cat allergies happen because of anaphylactic reaction to the Fel d1 cat allergen. With the popularity of cats in households, any non-invasive method to decrease allergic reactions can have major impact on the quality of life for millions of people in the US alone. In addition, this approach may point the way to similar solution to other allergy problems.

Plain English, Please!!!

First, let’s talk about what is an allergy. Some cells in our immune system are programmed to recognize foreign material we breathe in or we swallow. When immune cells make an IgE antibody in response, then that foreign material is now called an allergen. When the allergen enters the body the second time,

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Can a genetic mutation of cancer cells be exploited to kill them?

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On March 28, 2021

In cellular basis of life, chemical basis of life, digestive system, reproductive system

TeachableMedicalNews article 03282021

Teachable moment in classrooms:

  1. chemical basis of life chapter – DNA structure as double helix
  2. cellular basis of life chapter – DNA replication during mitosis
  3. cellular basis of life chapter – DNA mutation of a gene usually diminishes protein function
  4. digestive system – anatomy of pancreatic duct (origin of most pancreatic cancers)
  5. reproductive system – anatomy of ovary (surface epithelium is the origin of most ovarian cancers)

The news item:  Recently this article appeared online about a new pancreatic cancer treatment:

FDA approves drug for treating pancreatic cancer

Health regulators clear AstraZeneca and Merck treatment for disease projected to kill 46,000 Americans this year.

The article states that Lynparza has been approved for the treatment of ovarian cancers with BRCA mutation, and this approval is now extended to pancreatic cancers with the same mutation. The article also states that the BRCA mutation hinders fixing DNA damage, that Lynparza is a PARP inhibitor, and that 46,000 Americans die yearly because of pancreatic cancer.

So, Why Do I Care??  Pancreatic cancer has a very low survival rate, because the diagnosis usually happens in the late stage of the cancer. A new drug will likely increase survival rates among those 46,000 patients.

Plain English, Please!!!   First, let’s talk about the BRCA mutation. When cells undergo mitosis (cell division) the DNA is copied (replicated), so the new cell can have as much DNA as the starter cell.

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How can a catheter device seal an unwanted open blood vessel near the heart of prematurely born babies?

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On February 26, 2021

In cardiovascular system, respiratory system

TeachableMedicalNews article 02262021

Teachable moment in classrooms:

  1. heart chapter – aorta and pulmonary trunk around the heart
  2. blood vessel anatomy chapter – blood vessels at the start of the pulmonary and systemic circulations
  3. blood vessel anatomy chapter – modified blood circulations in the fetus
  4. respiratory system chapter – blood vessel carrying blood to and from lungs

The news item:  the following report was published recently,

FDA approves new closure device for heart defect in premature babies

One out of 10 of all babies are born premature.

 

The article states that every year about 12,000 very low birthweight premature babies develop a disorder called patent ductus arteriosus (PDA), and that the Amplatzer Piccolo Occluder device seals an opening between two blood vessels.

So, Why Do I Care??  As the article stated 12,000 prematurely born low birthweight babies can be helped by this device. Imagine a tiny newborn whose lungs are not working properly, because not enough blood flows to the lungs. Such respiratory distress is life threatening, so a new device can give those newborns a much better chance for survival.

Plain English, Please!!!

First, let’s talk about what the ductus arteriosus is.  The ductus arteriosus is a quarter-inch long blood vessel that connects the aorta and the pulmonary trunk near the heart. Imagine that the pulmonary

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How can the injection of a non-insulin medication help type 2 diabetes patients?

By

On January 17, 2021

In endocrine system, metabolism

TeachableMedicalNews article 01172021

Teachable moment in classrooms:

  1. endocrine system chapter – insulin production by beta cells
  2. endocrine system chapter – action of hormones through receptors
  3. endocrine system chapter – action of hormones inside target cells
  4. metabolism chapter – effect of insulin on glucose utilization by body cells

The news item:  Recently a new report appeared about the diabetes treatment drug Trulicity:

FDA approves Eli Lilly’s Trulicity to reduce cardiovascular risks in type 2 diabetic patients

Eli Lilly and Co said on Friday the U.S. Food and Drug Administration has approved Trulicity for reducing cardiovascular risk factors in type 2 diabetic patients. The approval makes Trulicity the first and only type 2 diabetes drug approved to reduce heart related risks in adults with and without established cardiovascular disease, the company said in a statement.

The article states that the type 2 diabetes treatment drug was approved for reducing heart disease risk as well.

So, Why Do I Care??  About 30 million people in the US has type 2 diabetes. The disorder causes about 80,000 deaths yearly. In addition, there is a yearly $327 billion cost from lost productivity and medical cost that includes treatment for diabetic vision loss and diabetic nephropathy. Diabetes also causes heart damage. New treatments have a chance to reduce deaths and lost working hours.

Plain English, Please!!!

First, let’s talk about type 2 diabetes. Diabetes, in general, is a disorder where blood sugar (glucose) level is higher than normal for long periods of time. The continuous high blood glucose level damages small

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How can an anti-wrinkle treatment help patients suffering from an overactive bladder?

By

On December 26, 2020

In microbiology, muscular system, nervous system, urinary system

TeachableMedicalNews article 12262020

Teachable moment in classrooms:

  1. muscular tissue chapter – neurotransmitter release at the neuromuscular junction
  2. autonomic nervous system chapter –  parasympathetic division and its neurotransmitters
  3. urinary system chapter – role of detrusor muscle in emptying of the bladder
  4. microbiology – Clostridium botulinum and its toxin

The news item:  Recently an unusual report appeared about the anti-wrinkle treatment, Botox:

https://westnewsmagazine.com/2019/11/13/117802/beyond-wrinkles-botox-offers-effective-treatment-for-common-bladder-condition

The article states that people suffering from an overactive bladder may find help for their symptoms from twice-a-year Botox injection.

So, Why Do I Care??  There are about 50 million people in the US suffering from overactive bladder symptoms. While this affliction is not life threatening, everyday life, ability to hold down a job are disrupted by it. There are several medical interventions to help patients, so Botox is the latest to join the remedies.

Plain English, Please!!!  First, let’s talk about the mechanics of urination. For urination to happen the muscle (called detrusor) inside the wall of the urinary

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How could gene editing fight cancers?

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On December 8, 2020

In cellular basis of life, lymphatic and immune system

TeachableMedicalNews article 12082020

Teachable moment in classrooms:

  1. cellular basis of life chapter – chromosomes contain many genes
  2. cellular basis of life chapter – a gene is a specific stretch of DNA that encodes one protein
  3. cellular basis of life chapter – concept of gene mutation leading to protein

malfunction

  1. immune system chapter – function of T lymphocytes (T cells)

The news item: Recently the gene editing approach to cancer fighting was in the news:

Doctors try CRISPR gene editing for cancer, a 1st in the US

The first attempt in the U.S. to use the gene editing tool CRISPR against cancer seems safe, but it’s too soon to know if it will improve survival.

The report described an experimental treatment of one sarcoma patient and two multiple myeloma patients with the Crispr technology. The article states that this approach deletes 3 genes and adds one gene to the T cells of the patients, and the gene-edited T cells are infused back into the body of the patients.

So, Why Do I Care??

The thing to care about is the potential of the technology of gene editing.  While experimental in humans, and the targets of the technology are relatively rare diseases, if proven useful there are many human genetic disorders that may be managed or cured by it. Any disorder that is caused by mutated genes and malfunctioning proteins may be the future target of gene editing.

Plain English, Please!!!

First, let’s talk about multiple myeloma and sarcoma. Myeloma is a cancer that forms in the red bone marrow. Sarcoma is a cancer that starts from connective

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