O2 For Beth Too

Elizabeth was born in 2002 in Flora Clinic, Roodepoort JHB.

From the day she was born she had to battle lung infections and malnutrition that led to frequent hospitalizations to treat pneumonia, prolapses etc.

After what felt like never-ending visits to specialists’ rooms, batteries of tests and enough bloodwork to make the strongest of men cower away from a needle, Elizabeth was diagnosed with Cystic Fibrosis (CF).

She was only 2 years old when we came to learn that monthly visits to specialists and regular extended hospital stays would become the norm, nutritious food would never be enough on its own and each breath Elizabeth would ever take, would be a battle. CF is an inherited genetic disease that affects several organs in the body, primarily the lungs and pancreas by clogging them with thick and sticky mucus. Mucus blocks the tiny pancreatic ducts which supply enzymes required for digestion, consequently food is not properly digested, and nutritional value is lost in the process.

Repeated lung infections and blockages can cause irreversible lung damage and death.

There is no cure for CF.

In the 21 years of her life, she has had to undergo a multitude of surgical procedures (she has 16 theatre hours in total during 2023 alone) – procedures to insert/remove chest ports (a port is an implanted device that provides permanent/long-term venous access for IV antibiotics, nutrition, fluids, and lab work); bronchoscopies; sinus washouts; multiple endoscopic sinus surgeries, etc.

Unfortunately, Elizabeth’s health has reached the point where the less-invasive procedures are no longer providing enough relief.

Elizabeth has just been discharged (Aug 2023) from a 3-week hospital stay during which her frontal sinuses have been completely obliterated.

Elizabeth needs to take a variety of antibiotics daily, she must take pancreatic enzymes with each meal, nebulizing and physiotherapy takes place at least twice daily. She has also been diagnosed with Cystic Fibrosis-related Type I diabetes which has led to the need for continuous measuring of glucose levels.

A sensor is installed just beneath the skin which continuously measures and transmits data to a smart device. Blood glucose levels are then managed by means of an insulin pump or injections.

Despite all the interventions, lung function, sinus health, nutrition etc. remain a challenge.

When Elizabeth was admitted to hospital in July 2023 her FEV1 (forced expiratory volume exhaled) measured 32.7%. During her stay and after weeks of IV antibiotics etc. her FEV1 increased to 42.2%. FEV1 is used to classify the severity of obstructive lung disease. FEV1 < 35 % is classified as very severe. A normal/healthy value would be 80 % or more.

Getting Elizabeth on Trixacar treatment is a matter of urgency. Trikafta/Trixacar are medications used to treat CF. Treatment with

Trikafta/Trixacar can increase estimated lifespan to 82.5 years, 45.5 years longer than what is estimated for CF patients on best supportive care alone. Trikafta treatment costs R5,5 million per year.

Patents assigned to the developers of Trikafta (Vertex Pharmaceuticals) are preventing the distribution of a generic equivalent that will cost roughly R115 000-R120 000 for a month’s supply. Trixacar (a generic version of Trikafta) is manufactured by Gador, a pharmaceutical company based in Argentina and only available to South Africans via Section 21 authorisation (issued by SAHPRA).

A patient/authorised person may the medication during a trip to Argentina (travel costs are roughly R15000/trip).

Doctors have come up with a plan to assist their CF patients. They prescribe alongside Trixacar another medication, Clarithromycin (an antibiotic), which in essence amplifies the potency of Trixacar.

This way it is often possible to use one box over the course of two months.

It seems that Discovery will reimburse to a maximum of R30 000 per month.

A patient will therefore need to pay for a box upfront (R115 000-R120 000) and if used over the course of 2 months a patient will end up having a co-payment of R60 000-R65 000 per box.

Until patents are reconsidered by Vertex we will need to raise enough money to purchase medication and cover travel costs to and from Argentina.

The treatment and travel expenses are high, donations to help make this happen for Elizabeth is most welcome. As treatment is not once-off, monthly contributions would further be of great help. If we can get enough people to make a monthly contribution, treatment for Elizabeth will be possible.

We, Elizabeth and her family need you to take hands with us to help us help BETH BREATHE.