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Dawns A New Era: Regenerative Medicine For Degenerative Disease

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Could breakthrough innovation in ‘Regenerative Medicine’ significantly reduce the need of expensive lifelong medications, or even make the use of some important medical devices less relevant, or even help avoiding expensive and risky surgical interventions? The common answer to these critical questions is now getting clearer, in tandem with the rapid progress of the science of ‘Regenerative Medicine.’

On June 13, 2017, Nature Biomedical Engineering published an interesting an article titled, “3D-printed vascular networks direct therapeutic angiogenesis in ischemia.” In simple words, these 3D-Printed patches are going to usher in a highly innovative way to treat ischemic diseases, in the future. As the researchers highlighted, arterial bypass grafts are currently considered as the gold standard for the treatment of end-stage ischemic disease, though many patients are unable to tolerate the cardiovascular stress of arterial surgery. The researchers found that implantation of 3D-printed grafts containing endothelial-cell-lined lumens, induces spontaneous and geometrically guided generation of collateral circulation in ischemic settings.

In rodent models of hind limb ischemia and myocardial infarction, these scientists successfully demonstrated that the vascular patches rescue perfusion of distal tissues, preventing capillary loss, muscle atrophy and loss of function.

In this article, I shall deliberate on the importance of this discovery, and its overall future implications on a broader perspective.

Regenerative medicine:

Here comes the basic question – What is ‘Regenerative Medicine’?

It is defined as a highly innovative branch of medicine that develops implementable methods to regrow, repair or replace damaged or diseased cells, organs or tissues. According to RegerativeMedicine.net following are illustrations of some conditions or diseases that regenerative medicine has the potential to cure, and what their current state of treatment looks like in in the American perspective:

  • Heart valves- 250,000 patients receive heart valves, at a cost of US$27 billion annually
  • Heart disease and Stroke- 950,00 people die of heart disease or stroke, at a cost of US$ 351 billion annually
  • Diabetes- 17 million patients have diabetes, at a cost of US$ 132 billion annually

I discussed in this blog, the subject of ‘3D Printing in health care’ on January 11, 2016. Hence, won’t dwell on that subject here

Ischemia, and the relevance of the above discovery:

Ischemia, as many would know, is a condition that restricts adequate flow of blood in some parts of our body, which over a period, may narrow, harden or even block the important blood vessels, much often resulting in stroke, heart attack or other related life-threatening vascular disorders.

Currently, ischemic heart conditions are usually treated either with blood thinning drugs, or blood vessel relaxants. In more serious stages of this condition, doctors prefer angioplasty or other surgical interventions, such as coronary artery bypass.

In this broad perspective, the relevance of the above discovery in addressing various debilitating or life- threatening ischemic conditions, is profound. Its novelty lies in the ability of the scientists making a 3D-printed patch that can be infused with cells to help grow healthy new blood vessels.

An emerging medical space:

The science of ‘Regenerative Medicine’ is increasingly being considered as an emerging medical space aimed at the treatment of those diseases that are usually classified as degenerative, incurable and irreversible. As it appears today, this science has the potential to unfold a new paradigm in this space, where patients can expect cure for many serious ailments, such as, spinal injuries, heart disease, Parkinson’s, Alzheimer’s disease and even diabetes, besides many others.

One more recent pursuit in this much uncharted frontier was reported in the British news daily – ‘The Telegraph’ on February 21, 2017, revealing the outcome of a path-breaking medical study for freezing the progression of a crippling ailment called Multiple Sclerosis (MS). This research followed a unique Stem Cell (SC) transplantation process, and is regarded as the largest long-term follow-up of SC transplantation treatment study of MS in regenerative medicine.

This study, spearheaded by Imperial College London, established that 46 per cent of patients who underwent the treatment did not suffer a worsening of their condition for five years. The process works by destroying the immune cells responsible for attacking the nervous system. This is indeed a very significant development in the space of medical research.

The treatment, called autologous hematopoietic stem cell transplantation (AHSCT), was given to patients with advanced forms of MS who had failed to respond to other medications. However, the researchers noted that the nature of the treatment, which involves aggressive chemotherapy, carried “significant risks”.

As many would know, MS is caused by the immune system malfunctioning and mistakenly attacking nerve cells in the brain and spinal cord, leading to problems with movement, vision, balance and speech. It’s a lifelong condition and often causes serious disability, with no cure still in sight. The disease is most commonly diagnosed in people in their 20s and 30s, although it can develop at any age.

A potential game changer:

According to California Institute for Regenerative Medicine (CIRM), this procedure has a game changing potential for successful use:

  • To replace neurons damaged by spinal cord injury, stroke, Alzheimer’s disease, Parkinson’s disease or other neurological problems
  • To produce insulin that could treat people with diabetes, and heart muscle cells that could repair damage after a heart attack, or
  • To replace virtually any tissue or organ that is injured or diseased

Research on “Regenerative Medicine’ signals a new hope:

Following are examples of just a few more promising developments, indicating that research in ‘Regenerative Medicine’ is taking rapid strides, signaling a new hope:

- A cure for Type 1 diabetes:

According to an international report on October 9, 2014, for the first time after 23 years of research, Harvard University has been able to manufacture millions of beta cells required for transplantation. It could mean a cure for diabetes, and the end of daily insulin injections for patients living with Type 1 diabetes. Although, just around 10 per cent of all diabetes is Type 1, it is the most common type of childhood diabetes.

The report indicated, the stem cell-derived beta cells are presently undergoing trials in animal models, including non-human primates, where they are still producing insulin after several months.

Another report of April 2014 indicates that for the first time, scientists have successfully replaced the damaged DNA of a type 1 diabetes sufferer with the healthy genetic material of an infant donor. When these cells are injected back into the diabetic patient, it is expected that they will begin to produce insulin on their own.

- Restoring vision in macular degeneration:

Yet another study published in ‘The Lancet’ in October 2014 stated that scientists in the United States have announced that single transplant of stem cells has helped restore the sight of patients suffering from incurable forms of blindness due to Age-related Macular Degeneration (AMD). Currently no effective treatments exist for this eye disorder, which can cause complete blindness due to the loss of light-receiving photoreceptor cells in the retina.

To recreate a type of cell in the retina that supports those photoreceptors, the new treatment uses stem cells derived from embryos that are only a few days old and have the ability to develop into any kind of tissue in the body. However, the transplants have proved controversial because they use stem cells derived from spare human embryos left over from IVF treatment.

- A cure for heart failure:

One more international report of May 01, 2014 states, by injecting human stem cells into the organs of macaque monkeys, scientists have been able to regenerate their damaged hearts by up to 40 per cent in just a few weeks. Thus, it appears now that a cure for heart failure could be just a few years away and would mean that even people who are “bed-bound” with heart failure could be “up and about” again within a few weeks.

As on date, the heart muscle cannot be repaired, making people with severe heart failure necessarily wait for a heart transplant, provided the patients are willing, and can afford so.

Conclusion:

There is a host of diseases, including several chronic ailments, such as diabetes, heart conditions, rheumatoid arthritis, or some types of cancer, which can’t be reversed, however, could be managed with a lifelong treatment. For most of these diseases, ‘Regenerative Medicine’ has the potential to be a game changer by transforming many lives.

Moreover, ‘Regenerative Medicine’ is expected not just to bring down the cost of health care and the disease burden significantly, but would also help increasing the economic productivity of a nation considerably.

Currently, medical research of the highest order in this area, has mostly been conducted by various academia of global repute, along with a few in the industry. It should soon involve, besides patients, several industries, including pharmaceuticals and biotech sectors, in a big way.

Nevertheless, this emerging trend sends a clear signal that to treat various chronic, incurable, irreversible and seriously debilitating degenerative diseases ‘Regenerative Medicine’ is now poised to take a giant leap in the health care space.  In that process, it would possibly help healing various ailments in a more meaningful, providing a cure for many chronic diseases that was a badly missing piece in the medical science, so far.

Thus, ‘Regenerative Medicines’ to treat many ‘Degenerative Diseases’ signal a great potential to give an altogether new shape and dimension to the future of global health care. It is also expected to ensure lesser lifelong usage of expensive drugs, setting a new normal to bring back the patients’ lives back to the pre-disease state.

By: Tapan J. Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.

 

 

Cancer Cure: Inching Towards The ‘Holy Grail’?

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In a Press Conference on August 20, 2015, the 39th President of the United States, now 90-year-old  Jimmy Carter, revealed (video) that during a liver surgery earlier this month the doctors diagnosed that he has cancer. The type of cancer that he is suffering from is called melanoma, which has already spread to his liver and brain. Medical jargon would term it as deadly metastatic cancer.

Though the surgeons have removed the liver tumor, and well-targeted radiation treatment for four other small tumors in his brain has already been initiated, the original site of the melanoma, the lethal skin cancer, has reportedly not been found, as yet.

Mr. Carter’s medical treatment has started with an infusion of a new class of drug that uses the human immune system to fight cancer cells. The drug has been reported to work not only in advanced metastatic cancer, but also in the old age of patients. The former American President appears optimistic about the treatment outcomes with this new therapy, ‘placing his fate in the hands of God’, though initially he thought that he had just weeks to live.

I shall deliberate in this article, in an easy to understand language, though briefly, the promises offered by two latest options for cancer cure. One of these two, has just become available to patients and the other one, after an initial jaw-dropping success, is undergoing further tests in a renowned research laboratory of the United States.

Two novel pathways for cancer treatment:

Until recently, surgery, radiation and chemotherapy used to be the three common options for cancer treatment. One breakthrough option has just been launched and more in the offing.

In search of a cure for cancer, pathbreaking outcomes of medical research, especially in the following two areas, are significant:

A. Immunotherapy: It is a revolutionary approach to cancer treatment. The first of this novel class of drugs has just come to the market, with which Mr.Jimmy Carter is now reportedly being treated.

B. Re-programming cancer cells back to normal: Success has just been achieved in laboratory studies with this technique. It holds a strong promise to cure cancer, universally.

A. Immunotherapy:

On June 1, 2015, in an article titled, ‘Cure for terminal cancer’ found in game-changing drugs, “The Telegraph” – well-regarded international news daily, reported on anti-cancer immunotherapy drugs, as follows:

“Terminally ill cancer patients have been ‘effectively cured’ by a game-changing new class of drugs. In one trial, more than half of patients who had just months to live saw deadly tumors shrink or completely disappear.”

“In recent days, the results of trials of a number of treatments which harness the body’s immune system have been announced at the American Society of Clinical Oncology’s annual conference in Chicago. They show promise in the fight against skin cancer and lung disease.”

As we know, most of the cancers are deadly. All these grow and spread, as they manage to hide from the immune system, disguising the life-threatening danger. Thus, medical research scientists pondered that the human immune system could play a critical role in the fight against cancer and even cure, by harnessing its ability to fight the deadly disease, effectively and decisively.

To achieve this goal, this class of new cancer drugs work by allowing the body to recognize and attack cancer as any other harmful invader to the body. It effectively blocks a cellular pathway that hinders the ability of the human immune system to attack cancer cells.

At present, to treat different types of cancer, more number of immunotherapy drugs are undergoing clinical investigations.

Brilliant treatment outcomes, but not universal:

It has been reported, about one third of patients taking immunotherapy for the treatment of cancer experienced positive results. Those who responded to this therapy, showed immediate effect with their tumors shrinking or vanishing in a matter of weeks. As a result, the patients who had no more than weeks or months to live, just as former US President Jimmy Carter, have gone into remission for years and continuing with their normal lives.

It has also been reported, otherwise such patients could expect to live just nine months, if given standard treatment of cancer. Researchers said, they were hopeful that half of the patients responded to immunotherapy would end up “living disease-free”.

These drugs are expensive, costing roughly US$150,000 per year, which is a part of a different debate altogether.

Not a ‘magic bullet’:

Besides its high cost and outstanding quality of results, it is worth noting that immunotherapy is not a ‘magic bullet’ for all types of patients and in all cancer. It, therefore, throws a challenge for the oncologists to understand, why immunotherapy benefits only to some cancer patients, and who are those patients?

Moreover, there is a possibility of immunotherapy sending immune system of some patients to overdrive, precipitating auto-immune disorder that may attack also the healthy cells.

Thus, immunotherapy is not the ‘Holy Grail’ for the treatment of cancer, neither it is nowhere near a perfect drug for the treatment of all types of cancers in all patients.

Two key findings:

In this regard, two key findings of the researchers on immunotherapy are as follows:

  • Roughly around 15 to 20 percent of patients could experience shrinkage or remission of cancer
  • Half of the patients who responded found it lasting for at least six months

Thus, immunotherapy can at best be a cure for only some terminally ill cancer patients, mostly for some time, but not for all.

“In the hands of God”:

All these factors on immunotherapy probably would help us to understand, why an erudite person like Mr. Jimmy Carter said, though optimistic about the new treatment, he is placing his fate ‘in the hands of God’.

B. Re-programming cancer cells back to normal: 

The question, therefore, comes up now, if immunotherapy is not the ‘Holy Grail’ for cancer treatment that the research scientists have been intensively searching for, is there anything else coming up for cancer cure?

It appears so. A totally different approach to re-program the cancer cells back to normal has very recently been reported by Mayo Clinic’s Florida Campus in the United States. With this, cancer researchers’ dream of making the tumor cells morphing back to normal cells, they once were, would probably come true.

The research findings, published in Nature Cell Biology on August 24, 2015, represents ‘an unexpected new biology that provides the code, the software for turning off cancer,’ said the senior investigator of this study.

In the normal process, cells in the human body divide constantly to replace themselves and stop dividing when they have replicated sufficiently. However, unlike the normal cells, cancer cells do not stop dividing, they go out of control, leading to huge cell reproduction and tumor growth.

For the ultimate cure of cancer, scientists at Mayo Clinic have now reportedly succeeded in reversing the process responsible for the normal cells from replicating too quickly.

Possible cure now within sight?

This could ultimately lead to a newer class of breakthrough treatment that would be able to reverse cancerous growth in the human body, possibly curing cancer, without the need of surgery, chemotherapy, radiation or even immunotherapy.

Scientists at the Mayo Clinic have said that their initial experiments in some aggressive types of cancer are quite encouraging. They have successfully done this in very aggressive human cell lines from breast and bladder cancer.

Towards the ‘Holy Grail’:

In pursuit of finding a cancer cure, research scientists have been making commendable progress, over a period of time.

In the last few years, spectacular breakthroughs in treatment of cancer have been possible from the increasing genetic and biological understanding of the researchers, especially in ascertaining exact defects in the DNA code of human genes that cause cancer.

Ability to sequencing human genome has offered a key tool to the researchers to compare the DNA codes of cancerous and normal cells and identify the differences.

From within the 20,000 human genes, around 500 cancer genes have been reportedly discovered and are being catalogued. Clear understanding of what happens precisely when the cells divide uncontrollably and cancer spreads in different parts of the patients’ body, is taking place with commendable progress of various research initiatives in this area.

Based on the current knowledge on human genome, a number of new drugs have been and are being developed to target the cancer-causing genes with great accuracy. Such types of drugs are called ‘personalized medicines’, as these act on specific gene abnormality of patients related to certain types of cancer. Sophisticated laboratory tests facilitate treatment with ‘personalized medicines’. These are more effective with lesser side-effects, as compared to generally used anti-cancer drugs, prescribed to all cancer patients.

However, the question keeps lingering, ‘Is the Holy Grail for cancer cure has now come within sight?’

Conclusion:

Medical scientists continue to take rapid strides towards better and more effective treatment for cancer, if not cure, with fewer side-effects.

Claims for long remissions with immunotherapy, are being reported for some patients with even metastatic cancer and also in old age, just like former President of America – Mr. Jimmy Carter.

The success achieved by the scientists of ‘Mayo Clinic’ in re-programming rogue cancer cells back to normal, is stunning.

Being successful in this effort, the researchers have compared cancer with a complex software program of life. When it goes out of control, ‘instead of the code for normal cells, a code for making abnormal cells is executed’. This new study signals a strong possibility of bringing the cancer cells back to normal.

Medical experts keep their fingers crossed. Although, some of them do apprehend that there may never be a single ‘Magic Bullet’ to cure all types of cancer in all patients. This is mainly because cancer involves a large number of different disease areas, such as, breast, lung, bowel, prostate, blood and so on.

But hope refuses to fade out, as science continues to keep unravelling spectacular breakthroughs in this direction, at a fairly brisk pace. All these researches may be cancer types or patient types specifics, but the progress is taking place in the right direction.

Even in the ‘Mayo Clinic study’, scientists have been, so far, successful in re-programming the breast and bladder cancer cells back to normal, though they believe that this success sends a strong signal of an “early and somewhat universal event in cancer.”

Immunotherapy is undoubtedly a path breaking step that ensures cure in some types of cancer and in some categories of patients. However, if re-programming the cancer cells back to normal, eventually becomes an ‘universal event’ in the treatment of this generally frightening disease, no doubt, the medical science is now slowly but surely inching towards the ‘Holy Grail’ for cancer cure…at long last.

By: Tapan J. Ray

Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.