Loss of drug substance and its dramatic consequences in times of CGT

cart-t und loss of drug substance

A medical dream is coming true. The immune system of cancer patients can be conditioned to detect – and fight – tumorous cells. Along with the classic trio of therapies, consisting of radiation, chemotherapy and operation, the CAR-T cell therapy is the next step in bringing medical practitioners closer to their dream of successfully combatting tumors.

A medical dream is coming true. The immune system of cancer patients can be conditioned to detect – and fight – tumorous cells. Along with the classic trio of therapies, consisting of radiation, chemotherapy and operation, the CAR-T cell therapy is the next step in bringing medical practitioners closer to their dream of successfully combatting tumors.

A CAR-T therapy comprises the already established immune therapy and extends the treatment to include cell and genetic therapy. CAR-T is short for “chimeric antigen receptor T-cells”, which are transformed into aggressive tumor killers by means of genetic modification.
T-cells are an element of the human immune system; in normal cases, however, they cannot detect and fight tumorous cells – they rather offer protection from attacks that happen as a result of molecular modifications.

However, when manipulated, T-cells can clearly recognize tumorous cells as a threat to the body and in turn will start to attack them.

What may sound simple is in reality a highly complex and time-consuming developmental process. Every single therapy has to be tailored to the respective patient, which poses enough challenges in itself. But on top of that, for a long time it was an issue to produce the sufficient amount of CAR-T cells necessary for this kind of therapy.

2012: The first successful CAR-T cell therapy – cancer-free for 6 years

In 2012, 6-year-old Emily Whitehead, who was suffering from leukemia, received a CAR-T cell therapy. The specialists had already given up hope, as none of the other applied therapies had proven successful. Emily Whitehead is the first child known to be healed thanks to a CAR-T cell therapy – and she has remained cancer-free since. Her case was a breakthrough and signified the start of a new era in the fight against cancer.

It will take approximately 5 more years to get the first official approvals. In the summer of 2017 the first approvals were given for the treatment of kids and young adults who suffered from severe cases of leukemia.

Importance of drug substance logistics on a new level

It seems logical that someone who up to know was regarded as “terminally ill” is hardly able to travel. However, the labs that are able to manipulate the cells of those patients are more often than not located on the other side of the planet, and so the biopharma logistics industry is facing completely new challenges.
The term “loss of drug substance” all of a sudden takes on a completely new meaning.

“Loss of drug substance” means the loss of biopharmaceutical substances and has taken on a completely new meaning with the introduction of individual CAR-T cell therapies. The same is true for “patient safety” – and you can even take that a step further – after all, the patient waiting for his personalized CAR-T cell therapy has a name.
In case something happens to the cells during shipping and the CAR-T cell therapy is rendered useless, the patient might – in the worst case – pay with his or her life.

 

What are possible reasons for loss of drug substance?

  • Bio contamination caused by improper filling of the container.
  • Faulty bottles or single use bags that can lead to bio contamination.
  • Containers that are unclean or soiled with detergent.
  • Leakage of liquids due to flawed single use bags or bottles.
  • Damage of containers during shipping (shocks, mechanic impacts).
  • Interruption of the cooling chain for the single use container.
  • Bio contamination caused by improper draining of the shipping container.

 

The pharmaceutical industry is challenged to reduce the loss of substances to near 0% in order to save as many lives as possible with the revolutionary CAR-T cell therapy.

As more and more variants of CAR-T cell therapies for different types of cancer are being approved by the FDA on a regular basis, the pharmaceutical industry is facing new logistical challenges – the current issues experienced during the freeze and thaw process and during the filling and draining of substances will now also need to be addressed during shipping.

In short, a safe logistic process as well as safe and compact storage solution are needed.

Will the solution for this problem be an Austrian one?

A young Austrian start-up called Single Use Support has been developing solutions for reducing the loss of drug substance for the past three years. And they were successful. For bigger charges, they were able to develop entire process chains that tackle the issue of contamination caused by manipulation or big fluctuations in temperature.

Currently, Single Use Support GmbH – based in Kufstein, Austria – is busy with the last steps before launching a prototype of the BULK.STREAM and a shell system specifically developed for the shipping of CGT or CAR-T cell therapies – all with the aim to have less loss of drug substance.

 

You can find more information shortly on: www.susupport.com