Another Cancer Breakthrough !

Scientific research is ever closing the gap on the treatment of Cancer and now the Therapeutic Goods Administration (TGA) has approved what is known as CAR-T therapy for use in paediatric and young adult patients with B-cell precursor acute lymphoblastic leukaemia (ALL) and adult patients with DLBCL who have failed to respond to other treatments , including chemotherapy.

This has been available overseas for some time and global trials have delivered amazing results. These clinical trials delivered an incredible sixty-two percent relapse free survival rate at twenty-four months in paediatric patients suffering ALL  and a forty-three percent probability of overall survival for adult DLBCL patients. It delivers hope where previously a diagnosis of this type of Cancer was virtually a death sentence.

Car-T therapy involves extracting a patient's own immune cells and genetically re-engineering them before infusing them back into the body.  The treatment is known commercially as Kymriah and is owned by the Pharmaceutical giant Novartis.  Similar approval has been granted in the European Union, Canada and Switzerland.

Unfortunately, medical breakthroughs of this nature are hideously expensive for the laboratory involved.   They often take years to perfect and involve medical trials designed to reveal side effects that may take ages to appear.  The owners have just the remaining period of patent protection to recoup these development costs and so the treatment is expensive.

At this stage, Kymriah will not be available here on the Pharmaceutical Benefits scheme.  It is so new that it doesn't fit into existing categories such as " drugs " and is therefore ineligible for funding.  It's approval means it will become available at three treatment centres and  patients may face a potential cost of $ 598,000.

It all depends on how quickly the government can find the money to include such new innovations into the PBS.  We recently had several inclusions that took the treatment cost from thousands of dollars to just six dollars and forty cents for a pensioner patient.  Obviously, Kymriah will have priority for similar listing.

Unfortunately, many big pharmaceutical firms are losing their appetite for research to develop new treatments because of the cost factor.  By rule of thumb, it is now estimated that bringing a new major drug to market will involve the outlay of a billion dollars and it will be copied by countries that do not respect the intellectual rights of others.   Recouping costs must be made during the patent protection period and then it may be copied freely as a generic version.

Development may take many years of costly research and often reasons emerge that mean the project is abandoned.   Research firms need deep pockets and infinite patience because only a meagre amount of research delivers a successful bonanza.

Let us hope the investors who fund these chemical firms hold their nerve and allow the research to continue.  It is the success stories like this one that keeps the research going to deliver the impossible.