Africa launches a continental drug regulator, the African Medicines Agency (AMA)

Good news!

"Africa has inaugurated its own drug regulator, the African Medicines Agency (AMA), after ten years in the works. The agency aims to make drug development more relevant to African populations, whose genetic diversity is rarely reflected in global research, and harmonize regulation across its nations. The continent also faces rapid population growth and urbanization, and an upheaval in international funding. ..."

"After more than a decade of planning, the launch of the African Medicines Agency (AMA) is being celebrated in Mombasa, Kenya, this week at the Seventh Biennial Scientific Conference on Medical Products Regulation in Africa. The agency’s establishment marks a pivotal moment in Africa’s public health, at a time when the need for biomedical research conducted in Africa, focused on African health problems, has never been greater. ..."

Nature Briefing: Translational Research

Africa finally has its own drug-regulation agency — and it could transform the continent’s health "If it gets things right, the first major regulator of medicines to launch for 30 years could empower Africa to tackle African challenges around health and disease."

Failure is not an option for Africa’s newly launched medicines agency "The inequitable distribution of vaccines during the COVID-19 pandemic was the final proof of the need for more home-grown manufacturing and regulatory capacity across Africa."

African Medicines Agency (AMA)

New legislation in Montana allows any adult to access drugs that have completed Phase I clinical trials

Good news! Broadening the access to experimental treatments.

"The bill, signed into law by Governor Greg Gianforte this week, will permit clinics and doctors to sell and provide nearly any experimental drug to potential patients, so long as it has undergone at least one Phase I clinical trial. Advocates argue that the law will allow Montana to be a hub for medical tourism, especially for potentially life-extending medicine.”"

Weekly Progress Roundup - by Malcolm Cochran - Doomslayer

Montana allows for experimental medical treatments

Good news! Go to Montana for treatment!

"The news: A bill that allows clinics to sell unproven treatments has been passed in Montana. Under the legislation, doctors can apply for a license to open an experimental treatment clinic and recommend and sell therapies not approved by the Food and Drug Administration (FDA) to their patients.

Why it matters: Once it’s signed by the governor, the law will be the most expansive in the country in allowing access to drugs that have not been fully tested. The bill allows for any drug produced in the state to be sold in it, providing it has been through phase I clinical trials—but these trials do not determine if the drug is effective. ..."

The first US hub for experimental medical treatments is coming

Montana Becomes the Right to Try Anything State, Expanding Access to Experimental Drugs "SB 535 formally paves the way for health care clinics to become 'experimental treatment centers' that can provide drugs still in development to willing patients."

Abolish the Food and Drug Administration (FDA)

Will President Trump reform the arcane, complex and byzantine drug approval process?

This is an excellent opportunity for Trump and a Republican dominate US Congress to go down in history as one of the most remarkable US presidents!

Maybe a fresh start is a good option! Bring the FDA into the 21st century!

"It takes 10–15 years and hundreds of millions of dollars for a pharmaceutical company to navigate the Food and Drug Administration (FDA) regulatory process and bring a new drug to market. Many seriously ill people die waiting for the FDA to approve drugs that regulators in other advanced countries have already approved, a phenomenon called “drug lag.” It is impossible to imagine how many drug remedies remain undiscovered and how many people needlessly suffer because pharmaceutical companies must divert excessive research and development dollars to the drug approval process, a phenomenon called “drug loss.”

Congress passed the Food, Drug, and Cosmetic Act (FDCA) in 1938 to ensure drug safety. ..."

Abolish the FDA | Cato Institute "Many seriously ill people die waiting for the FDA to approve drugs that regulators in other advanced countries have already approved."

After 50 Years, the FDA Finds Out Oral Phenylephrine (common nasal decongestant) Doesn't Work

Great headline! We knew for a long time that bureaucracies work like molasses! 😊

I know that pseudoephedrine helps me to reduce nasal congestion when my Spring allergies kick in. But buying it is a hassle!

"On September 12, a Food and Drug Administration advisory panel reported that oral phenylephrine hydrochloride, an ingredient in many popular over‐​the‐​counter decongestants people purchase to treat common cold symptoms, does not reduce nasal congestion. ...

The answer might have something to do with the fact that Congress passed the Combat Methamphetamine Epidemic Act in 2005, which went into effect in March 2006. Designed to prevent the diversion of an effective decongestant, pseudoephedrine, into the black market where meth lab “cooks” converted it into methamphetamine, the Drug Enforcement Administration ordered all oral pseudoephedrine medications to be “behind the counter.”

Pharmacists must record the personal identification of patients wishing to purchase it. The DEA places strict limits on the dose and number of pseudoephedrine‐​containing products patients may obtain within a 30‐​day time frame. ..."

After 50 Years, the FDA Finds Out Oral Phenylephrine Doesn't Work | Cato at Liberty Blog

Organ-on-a-chip system replicates rare neuromuscular disorders, finds new treatment without animal testing

Good news! This could be a breakthrough for organ on a chip systems to develop new drugs!

"Microphysiological systems, also known as organ-on-a-chip systems, are able to replicate key aspects of the human body and its function. These devices can be used with various living human cells to accelerate drug development, disease modeling, and personalize medicine — and the field is growing fast.
Now, researchers in the United States have used an advanced microphysiological system that mimics the pathology of chronic inflammatory demyelinating polyneuropathy and multifocal motor neuropathy, two very rare but very devastating neuromuscular diseases that cannot be replicated in animal models like rodents or primates. ...
There are over 7,000 rare diseases with no effective treatments, and only about 400 are being actively researched due to a variety of reasons, including the lack of animal models. Moreover, about 9 in 10 drug candidates that show promising results in tests on rodents eventually fail in clinical trials on humans. ..."

From the abstract:

"Chronic autoimmune demyelinating neuropathies are a group of rare neuromuscular disorders with complex, poorly characterized etiology. Here the authors describe a phenotypic, human-on-a-chip (HoaC) electrical conduction model of two rare autoimmune demyelinating neuropathies, chronic inflammatory demyelinating polyneuropathy (CIDP) and multifocal motor neuropathy (MMN), and explore the efficacy of TNT005, a monoclonal antibody inhibitor of the classical complement pathway. Patient sera is shown to contain anti-GM1 IgM and IgG antibodies capable of binding to human primary Schwann cells and induced pluripotent stem cell-derived motoneurons (MNs). Patient autoantibody binding is sufficient to activate the classical complement pathway, resulting in detection of C3b and C5b-9. A HoaC model, using a microelectrode array with directed axonal outgrowth over the electrodes treated with patient sera, exhibits reductions in MN action potential frequency and conduction velocity. TNT005 rescued the serum-induced complement deposition and functional deficits while treatment with an isotype control antibody has no rescue effect. These data indicate that complement activation by CIDP and MMN patient serum is sufficient to mimic neurophysiological features of each disease and that complement inhibition with TNT005 is sufficient to rescue these pathological effects and provide efficacy data included in an investigational new drug application, demonstrating the model's translational potential."

Human-on-a-chip system replicates rare neuromuscular disorders, finds new treatment without animal testing The FDA approved the first clinical trial using efficacy data collected from a microphysiological system. Here's why that's a pretty big deal.

Classical Complement Pathway Inhibition in a “Human-On-A-Chip” Model of Autoimmune Demyelinating Neuropathies (open access)

Why Drugs that Work on Mice Don't Work on Humans

Recommendable!

"Neuropsychiatric drug development is “in the midst of a crisis”. This is because, for every 100 neuropsychiatric drugs tested in clinical trials for ailments from autism and depression to Alzheimer’s, only nine end up as approved medications, one of the lowest approval rates across disease categories. ... To understand why this is the case, a team of neuroscientists from the Allen Institute for Brain Science in Seattle used a new procedure, known as a single-nucleus RNA-sequencing analysis, to study the medial temporal gyrus, the area of the brain involved in language processing and deductive reasoning, in both humans and mice ... the researchers worked through almost 16,000 cells to identify 75 different cell types. Although the data set they had from mice matched their human findings almost completely, they found that ... genes that were switched on or off inside those cells, stark differences between human and mouse brains arose ... In particular, the research demonstrated large differences in neurotransmitter receptors between humans and mice, an area that has been very active in drug discovery. Although both species have these receptors, they’re expressed via different types of cell. This means that, although these neurotransmitters exist in both humans and mice, they have very different functions, putting serotonin-based drugs such as antidepressants including Prozac under even more scrutiny ... Other significant differences between mice and human brains were found in genes coding for ion channels, which interconnect neurons by controlling the electrical potential across membranes ... Also an area that has undergone a lot of study for drug discovery for ailments including neuropathic pain and cardiovascular problems"


Why Drugs that Work on Mice Don't Work on Humans | Drug Discovery And Development: Mice are used as the frontline for drug testing for brain disorders. Why? Their brains are supposed to have much of the same functioning as ours. Moreover, | Drug Discovery And Development

A US startup is advertising a genetic therapy designed to lengthen lifespan—at a price

So what! The usual scaremongering!

Why not let someone with an extra $1 million to spare decide how she or he wants to be treated? We should call anyone who participates in these trials a courageous pioneer who may benefit humanity!

To treat telemeres sounds like a sound treatment to me. It has been well known for decades that telemeres are involved in the process of ageing!

If it does not work, we will probably find out very soon! If it does work as promised, the many doubters and invested interests will continue to scare us!

"a controversial $1 million-a-pop genetic treatment for aging"

"The claim: Libella Gene Therapeutics claims it is offering a gene therapy that will lengthen lifespan for $1 million a dose. It says two patients have already paid for the treatment at a clinic in Colombia.


The background: We know that telomeres—molecular timepieces on the ends of chromosomes that shorten each time a cell divides—may have something to do with how our bodies age. In some tissues, an enzyme called telomerase works to rebuild and extend the telomeres so cells can keep dividing. We don’t know whether telomerase protects any cell from aging—or whether it extends the life of entire organisms. Telomere-extending treatments in mice have yielded intriguing results, but nobody has demonstrated it in humans.


The trial: That hasn’t stopped Libella. It proposes injecting patients with viruses carrying the genetic instructions for telomerase reverse transcriptase, which is involved in the manufacture of telomerase.


Any worries?: ... This trial is for an unproven, untested treatment that might even be harmful to your health. It is occurring outside the US because it has not been approved by the Food and Drug Administration.  ..."



A US startup is advertising a genetic therapy designed to lengthen lifespan—at a price

New cystic fibrosis drug Trikafta could make deadly disease manageable

Good news! Well written Washington ComPost article! Perhaps, the Washington ComPost should write more articles about advances in medicine instead of publishing highly distorted political articles to aide and abet in an unprecedented scandalous coup attempt against a sitting president!

What is the kicker here that under President Trump, the FDA is finally approving new drugs much faster!

Trikafta is a great name for a drug! Does it mean cured by triple Franz Kafka? :-)

New cystic fibrosis drug Trikafta could make deadly disease manageable - The Washington Post: Thirty years after scientists discovered the defective gene that causes cystic fibrosis, two new trials show a therapy could help 90 percent of patients. The FDA approved the triple drug, Trikafta, five months ahead of its deadline.

Finally Speed Up And Simplify The Drug Approval Process

Posted: 2/24/2019

I would strongly argue that over the past 10-20 years, we made so enormous progress in our understanding of drugs and biology that the lengthy, expensive drug approval processes could be dramatically shortened and simplified. This would among other things save many lives and make drugs cheaper!

Here is just one most recent example of advances that could help speed up the drug approval process:  Engineered miniature kidneys come of age: Method for growing kidney organoids under flow enhances their vascularization and maturation, increasing their potential for drug testing and regenerative medicine ( dated February 11, 2019)

FDA Drug Approval Process Is Deeply Flawed

Posted: 7/10/2018

Vicious Cycle Of Government Intervention Since 1906

If you need an example of an almost ideal/stylized, but unfortunately, all too real vicious cycle of excessive government intervention just take the official history of drug approval by the U.S. Food And Drug Administration.

A History of the FDA and Drug Regulation in the United States

The only positive entry I could find perusing this history was: “1905 The American Medical Association (AMA) begins a voluntary program of drug approval that would last until 1955. In order to advertise in the AMA and related journals, drug companies must show proof that the drug will treat what they claim”. Without the vicious cycle of government interventions, we would probably have a much better system of private, voluntary drug testing and approval.

Thalidomide 1962

First, I am not a doctor nor am I medically trained etc.

The Thalidomide scandal of 1962 and its aftermath is a great example of a highly irrational, overreaction by our elected representatives that was never corrected. Thalidomide apparently has been an important drug. It is still used today.

In the U.S., the Thalidomide disaster was prevented thanks to “FDA inspector Frances Kelsey, who prevented the drug’s approval within the United States despite pressure from the pharmaceutical company and FDA supervisors. Kelsey felt the application for thalidomide contained incomplete and insufficient data on its safety and effectiveness. Among her concerns was the lack of data indicating whether the drug could cross the placenta, which provides nourishment to a developing fetus.” (emphasis added)

So what actually caused the breakdown of safety tests and approval for this drug? The sources below do not really tell.

It was approved as an over the counter drug in 1957 in Germany on the safety claims of the maker (what?). Did any German health authorities then approve this drug? Why was this drug so quickly promoted and sold as a morning sickness treatment for pregnant women (on the claims of the maker that it was safe for pregnant women as well)?

“Between 1958 and 1960 it was introduced in 46 different countries worldwide”. How was that possible that this drug was so fast approved and marketed worldwide?

“Around this time [1960?], Australian obstetrician Dr. William McBride discovered that the drug also alleviated morning sickness. He started recommending this off-label use of the drug to his pregnant patients, setting a worldwide trend. ” Did nobody verify this claim?

Apparently, highly advanced countries like the UK had inadequate government drug approval procedures in place up to 1962. How come?

“Until Thalidomide tragedy, the drugs were being sold by notification to health authority and NO safety, efficacy or quality data were required to be submitted prior marketing.” This was the sorry, troubling state of affairs in Western Europe.

Since The Thalidomide Scandal Drug Efficacy Needs Approval

Before Thalidomide (1962), the FDA drug approval process was only concerned with drug safety. Then, U.S. legislators rushed to pass the so called the Kefauver Harris Amendment (a.k.a. Drug Efficacy Amendment), which required drug efficacy as well. We still suffer from this incredibly misguided and short-sighted decision! This was certainly not the “finest achievement in consumer protection”, rather the opposite or everything else!

How Many Lives Were Shorted!

We will perhaps never find out how many lives were shortened since the the drug efficacy requirement came into effect in the U.S. However, I bet many more people were prematurely killed or harmed then by Thalidomide, because new and safe drugs were not speedily approved based on safety alone.

Sources:

1. https://en.wikipedia.org/wiki/History_of_the_Food_and_Drug_Administration
2. https://en.wikipedia.org/wiki/Development_of_analogs_of_thalidomide
3. https://en.wikipedia.org/wiki/Thalidomide
4. https://en.wikipedia.org/wiki/Kefauver_Harris_Amendment
5. https://helix.northwestern.edu/article/thalidomide-tragedy-lessons-drug-safety-and-regulation
6. http://www.who.int/medicines/technical_briefing/tbs/Drug_Regulation_History_Present_Future.pdf
7. https://www.omicsonline.org/open-access/historical-overview-of-pharmaceutical-industry-and-drug-regulatory-affairs-2167-7689.S11-002.pdf