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The one way downhill path that is Congestive Heart Failure (CHF)

Heart failure is so serious, this government-sponsored article, "More 'malignant' than cancer? Five-year survival following a first admission for heart failure" concludes:

With the notable exception of lung cancer, heart failure is as "malignant" as many common types of cancer and is associated with a comparable number of expected life-years lost.

Another article (here and supported here) advises:

Regardless of treatment, one-fifth of those diagnosed with CHF will die within one year and one-half will be dead within five years. Only about 20 percent survive much longer than 8 to 12 years -- a prognosis worse than most cancers. For those that do survive, quality of life is often severely compromised.

Congestive heart failure (CHF) is a progressive disease. Current FDA approved treatment for earlier stage CHF is only effective in slowing the worsening of heart failure.

The inevitable result is that eventually optimal drug therapy (OMT) becomes insufficient and the native heart needs to be replaced with a donor heart (referred to as the "Gold" standard).

The need for a "Platinum" standard

As described in more detail below, the likelihood of a CHF patient achieving the "Gold" standard, the best available current option, is less than one percent.

What is needed most is a therapy/ies that will halt or reverse the seemingly inevitable downhill journey to the "Gold" standard.

If a way to halt, or a path back, could be found, then surely that would merit the term the "Platinum" standard.

I believe the Sunshine Heart (NASDAQ:SSH) C-Pulse system could become the cornerstone in establishing a therapy/ies that would allow CHF patients to keep their natural heart, with that heart operating adequately.

For the reasons I categorize the C-Pulse as the likely cornerstone "Platinum" therapy, I refer readers to my recent article, "Novartis, NeoStem And Sunshine Heart: Complementary, Not Competing Therapies".

In that article I described how the Novartis and NeoStem therapies were unlikely by themselves to halt or reverse CHF progression and would likely need the C-Pulse therapy to be effective. On the other hand, the C-Pulse alone has been shown to effectively halt and reverse CHF progression in its 20 patient feasibility study.

I expand further below on C-Pulse as the cornerstone of the "Platinum" standard, including the potential role of BioVentrix therapy as a complementary, and not competing, therapy.

More about the "Gold" standard

Donor heart transplantation has become known as the "Gold" standard for addressing end stage heart failure. Today that is where the one-way downhill path of CHF leads to. Due to a scarcity of donor hearts, there are long waiting lists and left ventricular assist devices (LVADs), such as those from Thoratec (NASDAQ:THOR) and HeartWare (NASDAQ:HTWR), have been developed as a bridge to transplant (BTT).

These LVADs carry their own significant risks and so until recently have mostly only been approved and used for BTT in end stage CHF patients. Thoratec has been granted FDA approval for its HeartMate II for destination therapy (DT) in late stage CHF patients.

Newer versions of LVADs have, and promise, improved size, performance, and safety features over earlier models. But HeartWare is still striving to obtain DT approval.

Despite improvements, LVADs are still considered a poorer option to the "Gold" standard of a donor heart transplant.

The dual tragedy of the path to "Gold"

Tragedy 1 - Less than 1 in 300 first time CHF patients (0.33%) will achieve "Gold," based on NHLBI and American Heart Association statistics:

About 2,000 donor hearts are available each year; and

The incidence and prevalence of chronic heart failure (CHF) in the United States is continually rising, with an estimated 670,000 new cases diagnosed per year.

The NHLBI also reports:

Approximately 3,000 people in the United States are on the waiting list for a heart transplant on any given day.

But the official waiting list is just the tip of the iceberg. In an article on total artificial hearts, in the New York Times published on July 13, 2013, Dr. Lynne Stevenson (a professor at Harvard Medical School and director of the cardiomyopathy and heart-failure program at Brigham and Women's Hospital in Boston) is quoted as saying:

It's estimated that if we had enough donor hearts to go around, 100,000 to 150,000 people in the United States with heart failure would benefit. Transplants work best, but we have only 2,000 or so adult hearts that are available each year. It's a huge problem.

Tragedy 2 - Donor hearts available but not able to be used

According to the 2010 Annual Report by the Scientific Registry of Transplant Patients, 4,900 potential donor hearts were not used due to current preservation issues, lack of assessment and time and distance issues. So the availability of donor hearts is 2 to 3 times the donor hearts able to be actually used.

There is much publicity about the need for more organ donors to increase the availability of donor hearts and other donor organs. What I have not seen receive publicity to date are the very significant initiatives by tiny, privately held, Paragonix Technlogies, based in Massachusetts. These initiatives could very significantly increase the number of available donor hearts able to be used.

The Paragonix Technology to increase available donor hearts

Paragonix Technologies' FDA approved technology for donor heart transport (the Sherpa Pak CTS), together with enhancements in the pipeline (the Sherpa Perfusion CTS), (see here) will potentially increase the availability of donor hearts without any increase in the level of donors.

It will do this by keeping donor hearts in better condition and for longer periods than the present "bucket of ice" technology that has been the usual mode of transport.

The opportunity for increased donor hearts using a device such as Paragonix's that eliminates some of the issues that result in unused donor hearts is a substantial one. I believe the impact would be similar in markets outside the US.

All those associated with heart donors and with donor heart recipients should be crying out for an early changeover from the "bucket of ice" mode of transportation of the past to this new and FDA approved technology. To put this in perspective, imagine if the Sherpa Pak CTS was already the accepted means of donor heart transport and someone tried instead to use a "bucket of ice" to transfer a donor heart.

But even with these initiatives by Paragonix Technologies, and other initiatives, there will remain a very large shortfall in available donor hearts. Even if death is avoided along the way, the one-way downhill path referred to above leads to a highly undesirable destination with scarce availability of the "Gold" standard, the best option to get relief.

The answer to this is to find a way to get off the downhill path at a suitable point, and so either halt the downhill progression or actually move back up the hill.

Introducing the "Platinum" standard

I will coin the term the "Platinum" standard for those therapies that provide the potential for the CHF patient to keep their natural heart, with that heart continuing to work adequately in the longer term.

In my recent article referred to above, "Novartis, NeoStem And Sunshine Heart: Complementary, Not Competing Therapies", I discussed various complementary therapies that would likely be used to keep CHF patients alive and provide support that would allow their damaged natural hearts to repair and recover. In that article, I set out to refute various claims that Novartis' drug Serelaxin, and NeoStem's AMR-001 stem cell technology were threats to uptake of the Sunshine Heart C-Pulse technology. I explained instead why the C-Pulse technology would likely be required in conjunction with the use of these therapies for them to be effective in halting or reversing CHF.

I predict the cornerstone of the "Platinum" standard will be the Sunshine Heart C-Pulse System, because of its unique ability to provide additional blood flow to the heart muscle, and to ease the work load of the heart without taking over from the natural heart or contacting the blood stream.

Another supposed "threat" to the C-Pulse system has been raised in the form of BioVentrix. I will explain further below why BioVentrix is also likely complementary to the cornerstone C-Pulse technology and could in fact increase its target market. But first some more detail on C-Pulse for those unfamiliar with this technology.

Sunshine Heart's C-Pulse system:

The Sunshine Heart C-Pulse system is a medical device to treat congestive heart failure by assisting the natural heart's pumping function. To further explain:

The C-Pulse system is not blood-contacting and does not take over from the heart. It employs proven counter-pulsation technology, to reduce the workload of the heart, and to create additional blood flow to the heart muscle. This provides ongoing permanent support and allows a tired heart muscle some opportunity to rest and recover, in a measurable way (NYHA Heart Failure class improvement). For further information see here and here.

Some results from the C-Pulse 20-person feasibility study (Data sources:Prospectus filed with SEC August 10, 2012 and Sunshine Heart Corporate Presentation October 2012):

  • NYHA Heart Failure Class improvement - 12 patients (60%) at 6 months. (Subsequent to completion of the feasibility study 2 patients became asymptomatic and were weaned off the device and another 2 are being considered for weaning);
  • Re-hospitalizations at 6 months 5% (compares to recent trial published control group re-hospitalization rate of over 40% with similar patient population at 6 months);
  • Medication Reduction: Diuretics - discontinued, reduced or unchanged for all patients; Inotropes - 4/4 patents successfully weaned (within 48 hours).

Following on the successful completion of the feasibility trial, the FDA has approved a 388-patient pivotal trial addressing NYHA Class III and ambulatory Class IV CHF patients with targeted marketing approval in early 2017. The C-Pulse already has CE Mark approval for commercial sale in the EU.

BioVentrix - Complementary therapy to Sunshine Heart C-Pulse

1. Why BioVentrix is likely to be a complementary therapy to C-Pulse:

The condition that BioVentrix therapy is applicable to is described in an article from Heart journal as follows:

Following myocardial infarction, one phenomenon that is known to occur is ventricular expansion. With expansion of the ventricle there is dilatation and thinning which can occur without additional necrosis. There is also a distortion in the shape of the heart from an elliptical to a more spherical form. This contributes to substantial mechanical inefficiency and worsening of CHF.

From the BioVentrix website:

The LIVE procedure is based upon a well-defined law of physics called the law of Laplace, which describes the relationship between the radius and pressure of the LV, and its resulting wall tension. Increased wall tension is the underlying cause of LV enlargement, worsening heart failure symptoms and ultimately patient death. Reducing wall tension is key to preventing further LV enlargement and treats the progression of the disease. The Revivent System, placed via the LIVE procedure, is uniquely designed to directly reduce the LV radius, which in turn decreases wall tension and interrupts the ongoing, destructive process of heart failure.

The BioVentrix technology utilizes clamps to create a fold over scar tissue from a heart attack, thus reducing the size of the LV and creating a shape similar to a healthy LV. This results in an immediate improvement in the mechanical pumping efficiency of the LV. But that does not appear to be a therapy that will address the dilatation and thinning of the ventricle that has already occurred.

BioVentrix only claim its therapy will interrupt the ongoing, destructive process of heart failure and appears to make no claim it will prevent subsequent worsening of the condition of the heart after the therapy.

I believe that CHF patients treated with the BioVentrix technology could likely require support from a Sunshine Heart C-Pulse system to prevent subsequent resumption of worsening of the condition of their still compromised heart. In support of this contention, firstly, I would refer readers to the effect on "End-diastolic wall thickness and residual rim of viability" as discussed in this article from the American College of Cardiology. It would seem if the LV reduction is achieved by clamping rather than a return to its original natural dimensions then the LV functioning will likely remain compromised by the changes referred to in the above article.

Secondly, from the same article, there is considerable discussion on Hibernating Myocardium (HM) and its ability to re-activate with re-vascularisation after ischemia or myocardial infarction. Part of the article states:

Moreover, when the reduction in Myocardial Blood Flow (MBF) is large at rest, even a "small" increase of MBF with revascularization may improve patient outcomes but to a greater degree.

The counter-pulsation technology employed by C-Pulse increases myocardial blood flow when the right heart is filling and the left heart is at rest. This provides a greater understanding of how the C-Pulse might have been so effective in reversing CHF in patients in the feasibility trial. C-Pulse increases MBF while the heart is at rest when the degree of benefit is greatest.

2. Why BioVentrix might actually increase Sunshine Heart's C-Pulse target market:

The C-Pulse system reduces the workload of the heart rather than taking over the pumping function of the heart. As CHF progresses, the heart might become so weak that reducing the workload might be insufficient and it might be necessary instead to employ an LVAD to take over the pumping function. This would be the reason for C-Pulse to target only NYHA Class III and ambulatory Class IV and not beyond that in current and past trials.

BioVentrix's website provides an example of a NYHA Class IV patient who was barely ambulatory but improved after reduction of the size of the LV using its procedure. This patient might have been beyond the help of C-Pulse prior to the procedure. But after the procedure C-Pulse might be able to assist by further reducing the workload of a somewhat compromised LV and at the same time assist to re-activate hibernating myocardium by increasing blood flow to the heart muscle while it is at rest.

"2014: The Year Of The Heart"

A paradigm shift is taking place towards new treatments for CHF that promise the possibility of CHF sufferers taking a new path -- the "Platinum" standard -- leading to them keep their own natural heart, with that heart continuing to work adequately in the longer term.

I believe that paradigm shift will gather real momentum in the coming year, leading to 2014 being hailed as "The Year Of The Heart." I further believe that Sunshine Heart will be the cornerstone technology of this paradigm shift and will become referred to as the "Platinum" standard in the same way that donor heart transplantation is referred to as the "Gold" standard. It will be considered the cornerstone because of its ability to work as a standalone therapy or as the key therapy to support other therapies that, as described above, are unlikely to be wholly effective by themselves.

Conclusions, including financial outlook

In time, I believe Sunshine Heart's C-Pulse will likely become the leading therapy for CHF, pushing the boundaries into later stage NYHA Class IV (in conjunction with complementary therapies) and back into NYHA Class II, for treatment for conditions such as unstable angina (refer to transcript from PropThink interview for Dave Rosa, Sunshine Heart CEO's comments on C-Pulse's applicability to ~ 1M NYHA Class II patients in the US alone once a fully implantable option is available).

With wide use of C-Pulse systems, particularly once the fully implantable model is available, the progression of CHF sufferers to late stage NYHA Class IV will likely be greatly reduced.

The financial outlook for Sunshine Heart's C-Pulse, if it is able to tap only a small percentage of the potential target market it addresses, is very positive and could put it in the league of a company the size of Medtronic.

A scenario from one of my earlier articles (Sunshine Heart: Risk & Reward) looked at the potential revenue and operating profits of Sunshine Heart if it were able to achieve market penetration of just 6% of its target market at some future time. Those calculations suggested, at 6% market penetration, Sunshine Heart's profitability would be close to matching Medtronic's 2012 results.

Readers might also like to refer back to my article, "Sunshine Heart: Potential '10 Bagger' In 4 Years; '100 Bagger' In 10 Years", in which I propose, following requests from readers, to update to reflect actual capital raises and other changes that have taken place since that article was written.

Despite any reduction in CHF progression due to "Platinum" standard therapies, and any advances in making increasing numbers of donor hearts available through devices from Paragonix Technologies and others such as TransMedics, the need for donor hearts will likely continue to exceed availability.

The shortfall in donor hearts for those patients still progressing to late stage NYHA Class IV will be increasingly met by LVADs such as those from THOR and HTWR as patient acceptance increases and the technology becomes smaller, fully implantable, and hopefully with lowered device related risks.

Despite any reduction in the number of CHF patients in their target market, the market is likely to remain sufficiently large to allow these LVAD firms to grow profitably.

Caution: The information above is not intended to replace the advice of a doctor. I disclaim any liability for any decisions you might make based on this information.

Additional cautions: As always, please do your own research before any buy or sell decisions. Use of information and research in the article above is at your own risk.

Investing in micro cap companies is not suitable for all investors and can be risky. It's important that investors thoroughly perform their own due diligence and analyze the potential risks. Due to illiquidity, share prices can fall despite strong fundamentals and possible inability to raise sufficient additional cash to continue to fund ongoing operations is always a serious concern. Fuller details of risks associated with Sunshine Heart as identified by the company may be found with their form 10-12B/A registration filing with the SEC and their other SEC filings.

Source: Sunshine Heart's C-Pulse: The 'Platinum' Standard