Written by Nasma - 4 Minutes reading time

How biotech can mend a broken heart

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CellProthera has been working to do the impossible – mend broken hearts. The biotech company’s ProtheraCytes cell therapy regenerates damaged tissue following a heart attack, thus restoring cardiac function.

While regenerative cell therapy is dependent on the patient’s own cells, and reliant on the patient’s ability to provide cells, repairing the original organ is a great alternative to the option of a heart transplant.

A total of 33 patients have received CellProthera’s ProtheraCytes in the latest trial, named EXCELLENT, which concluded in September, and the company is now preparing for an expanded Phase 3 trial.

The cellular product is made by expanding each patient’s CD34+ stem cells, known for their ability to promote the formation of new blood vessels, and have previously been shown in a pilot study to prevent the need for a heart transplant in patients with heart failure following severe acute myocardial infarction (AMI).

How do regenerative therapies help after heart attacks?

According to de Kalbermatten, CD34+ stem cells are not only able to regenerate blood cells, but also they are progenitors of different other tissues.

“One of them, which is the endothelial cells, progenitor cells, are actually the main component of any blood vessel that you have in your body,” he explained.

After heart attacks, the current standard of care is to take medication to reduce the speed of degradation of the heart function, but there is no way to repair heart tissue because the heart has lost its regenerative capacity.

“We address those patients with our product that is actually based on stem cells that we have produced ex vivo with our specific technology. And we re-inject those stem cells directly into the heart to repair the tissue so that those patients, instead of going into this very bad disease called chronic heart failure, actually recover quite early after a heart attack, and just live a normal life.”

What are ProtheraCytes?

ProtheraCytes start from the blood sample of a patient.

“We go through an expansion process, ex vivo, cell selection, but also cell expansion. That’s multiple steps of production that then leads to this final product, which is a condensed large number of CD34+ stem cells,” de Kalbermatten said.

The ProtheraCytes are then injected straight into the heart lesion.

“Once there, it receives signals from the lesion. And that’s why it’s very important to treat those patients early after the heart attack and not too late. We inject those cells within a month after the heart attack. They receive a few proteins, signals called chemokines and they start to go into two different directions.

“One direction is they differentiate into daughter cells because they are stem cells, so they get more and more mature, transform into an endogenous cells, which is the main component of blood vessels. So you regenerate the heart on one side with the differentiation of those stem cells, but also they are secreting a lot of different factors inside small vesicles called exosomes. And those exosomes then release proteins and microRNAs that are able to boost the regeneration of small micro vessels, but also microRNA that are anti-inflammatory, so that you can preserve as many muscle tissue as you can.”

While the company isn’t claiming to repair 100% of the damaged tissue, de Kalbermatten said even reducing the amount of tissue destroyed by a third is a “big, big win.”

He explained that this is a one-time procedure: the patient gives blood once, returns to the hospital for a ‘minimally invasive’ injection, and then goes back home.

Are there other diseases and conditions where regenerative therapy is or would be useful?

The beauty of the technology, de Kalbermatten said, is that as the stem cells are not pre-defined, they can be used in different ways.

He said they are working with a U.S. company on repairing the brain after severe strokes, as well as for diabetic patients.

Is regenerative therapy expensive?

“This is the main challenge of autologous therapy, you’re really producing one batch or one product per patient,” de Kalbermatten admitted.

However, to keep costs down, he said automation of stages has been critical.

He explained that about 30% of the overall production cost is directly related to the manual operation, which not only affects cost, but also can create quality issues.

“You can’t really afford to fail in the production of a personalized medicine, considering that you can’t really redo it,” de Kalbermatten noted.

“You lose your windows of opportunity and could not treat the patient. So, we have to go into that direction of automation to standardize the process, to reduce the risk of errors. And we have a first technology platform that we’ve developed in the last five years, which allows to produce five patients at a time.

“This allows us to see comfortably treatment of thousands of patients per year in different countries. But there is already a project internally that we are pushing forward to get into the next stage, the next generation of a technology platform that would be able to really completely scale out our production process so that we’ll be able to produce, in parallel, a lot of batches at a time.”

By Jim Cornall

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Also published on Labiotech.eu

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