To the extended Multiple Sclerosis (MS) community,
There seems to be a lot of dialogue between patients and organizations alike regarding the most recently published trials conducted by Dr Richard Burt from Northwestern University in America. For those of you who are not familiar, this clinical trial looked to ascertain whether Multiple Sclerosis (MS) patients could improve their scoring on the standard Expanded Disability Status Scale (EDSS), which is simply an incremental measuring scale ranging from 1 through to 10. The trial used the treatment type known as Hematopoietic stem cell (HSC) transplantation, also known as (HSCT). This type of treatment is primarily a combination of chemotherapy and stem cell therapy. My fellow colleague has produced a well-designed and well conducted trial which is notably the most influential study to date in HSCT. It is important to understand the results beyond the statistics to see the actual impact for MS patients.
The trial was officially published in the medical journal JAMA on January 20, 2015. There were a total of 151 patients enrolled for the study with the target of tracking patient progress for 5 years. For the readers who may not be familiar with the disease, there are three types of MS noted in order of severity, relapsing-remitting (RR), secondary-progressive (SP) and primary-progressive (PP).
Out of the 151 total patients, 123 were RR, only 28 were SP and zero PP. The average patient age in the trial was 36 years; an age range between 18-60 years. This equates to a focus of 81% of the patients for the entire trial being the one type, relapsing-remitting MS. It is important to note that this type of MS patient has historically been the only group to show any response to treatments as demonstrated in other trials using medications for MS. Therefore it is essential to understand that the trial excluded the least responsive patients.
The chemotherapeutic agents used in a HSCT treatment can be either ablative or non-ablative. The most common form and also the form used in Dr Burt’s trial is non-ablative, meaning a milder version of chemotherapeutic agents were used (ablative being the harsher of the two). The non-ablative HSCT treatment still carries inherent risk of death as can be evidenced by the recent loss of an Australian patient who courageously flew across to Russia to undergo the same treatment. This fact should not be marginalised when assessing potential treatment options.
The final results were only available from 145 patients out of the original 151. Although the target of the trial was to track the progress of patients across a time period of 5 years; the published records state that they were only able to track patient results for a maximum average of 2.5 years.
ONLY Relapsing Remitting patients with less than 10 years of having the disease showed a positive improvement. This select group equated to an overall 56% of the total assessable patients. The positive improvement noted came from an average recording which did not exceed more than one shift on the EDSS measurement scale. As previously stated in the opening paragraph the scale is a range of 1 to 10 therefore one shift equates to a maximum of 10% improvement. Albeit this is considered statistically significant, however on a practical level the individual impact may not be so notable. To put this into perspective, the majority of MS medication trials achieved similar or better results than one shift with RR patients. The more concerning component of these results is that not one of the few patients with secondary-progressive had any improvement.
As a straightforward conclusion, this trial has clinically indicated HSCT to only be effective to patients in the early stages of relapsing remitting MS.
As some of you may well be aware clinical trials for any form of medication or new treatment has typically been strenuous, time-consuming and tremendously expensive. This is why traditionally the only parties who would undertake the process of coordinating and funding clinical trials have been the multinational pharmaceutical companies. Quite simply, it is seen as an investment which is assigned a high probability of commercial value to recoup capital investment plus profit.
It is very important to note this fact as we need to understand the dynamic of the value system for each individual scenario. An example scenario which uses the same target population are the trials recently concluded by Dr Burt in America evaluating the clinical outcomes of Hematopoietic stem cell (HSC) transplantation, also known as (HSCT) with patients suffering from Multiple Sclerosis (MS). Unbeknownst to the wider public is the fact that the Chemotherapy conglomerates are trying to seize the opportunity to create a new market segment. These companies can see the positive dollar value of backing clinical studies whereby the outcome is a new group of patients outside of cancer patients to purchase Chemotherapy medication. As recorded by Dr Burt’s report each individual patient costed the trial in excess of $150,000 USD which is sizable when considering the study consisted of 156 subjects. To put this in perspective, it equates to $30,638,297 in current Australian dollars.
Another example scenario is headed by the Canadian MS Society. Statistically, more Canadians suffer from MS than in any other country so the Canadian MS foundation is extremely supportive and proactive in supporting Stem Cell. A total amount of 4.2 million has been announced as the opening budget to commence wide scale clinical trials comparable to the trials we are scheduled to complete here in Australia.
In Australia we have been achieving clinical outcomes with MS patients which have astonished the wider medical and scientific community. The time has come to put Australia on the map and in the history books as the first group of experts to truly develop a worthy cause of remedy for MS sufferers. Unfortunately, from a funding perspective, the innovative protocols to be verified under trial conditions have no pharmaceutical dollar value. The key element in this scheduled trial is the actual stem cells which everyone already has a steady supply of. Unlike Canada, at this stage, we in Australia do not have any financial support. Also it is our plan to conclude 300 patient outcomes so our results will present irrefutable data which can later be used to rally support to achieve Medicare subsidy.
Therefore this trial in Australia is being funded by a collaborative effort of private medical practice, a handful of investors and the subject participants themselves. There are a number of parties contributing other forms of resources and/or donating their expert time to the successful outcomes of our subject patients. The patient contribution will not exceed a maximum of 40% of their individual treatment cost, however the patient receives 100% of the benefit. Each patient will be evaluated on a case by case basis as different patients will need to be matched with slightly differing protocols to ensure suitability.