Tag Archives: genetic engineering

From Extraordinary Leaders Come Extraordinary Measures

Bookmark and Share     In a few days a dramatic motion picture that could shape politics in one state will be coming to a theatre near you. I have not seen it yet, so it would be premature to declare it to be a cinematic tour de force but I do know it will be inspirational. I also know that it may finally launch the rise of a political force to reckon with from New Jersey.

 The movie, “Extraordinary Measures”, stars Harrison Ford and Brendan Fraser and it is based on the very real plight of John and Aileen Crowley who raced time and battled against all odds.

 John and Aileen were handed their greatest challenge when their children, Megan and Patrick were both born with a fatal neuromuscular disorder called Pompe disease.

Determined to change the deadly fate that doctors predicted, John and Aileen sought the care and treatment that could keep Patrick and Megan alive. But the disorder was so rare that few if any pharmaceutical entities attempted to develop effective treatments  for Pompe.  The disease was so rare that it lacked any widespread study. With so relatively few inflicted with Pompe, there was little if any profit motive for either the medical, scientific or pharmaceutical fields to devote much time, manpower and resources to it. Such a rare disease received barely even a fraction of the attention that more common health problems such as cancer receive.

The unimaginable situation that the Crowleys were dealt, compelled them to settle in Princeton, New Jersey so that they could be nearest to where some of the only doctors who had any experience in Pompe disease were located. But even in the hands of the most experienced experts of the disorder, John and Aileen were consistently forced to face the bleak diagnosis originally prescribed to their children.

Determined, John subsequently took up employment with Bristol-Meyers Squibb in the hope of fostering efforts to expedite a cure or treatment for the disease that was killing little Patrick and Megan.

After two years, with little progress and many roadblocks, he left Bristol-Meyers to break through the barriers and embark on a new venture to increase the pace of research into Pompe and the development of a cure. It was a race against time that was slowly taking the lives of his children.

Crowley joined forces with dedicated professionals and became the CEO of a biotech company called Novazyme. Eventually Novazyme merged with the world’s largest biotech company, Genzyme Corp., and soon after that the combined efforts of the two, brought about by John Crowley, expedited the creation of an enzyme replacement therapy for Pompe disease that is keeping the Crowley kids alive today.

Getting to that point was not easy and there were a great many trials and tribulations, but John Crowley, his wife, and the team he assembled, never gave up. The story is the stuff that books are written about. And before the movie was made about it, a book about the Crowley’s was written by a Pulitzer prize winning author.  It is called “The Cure: How a Father Raised $100 Million – And Bucked the Medical Establishment – In a Quest to Save His Children (ISBN 978-0060734398).

The experience now keeps Crowley hard at work as President and CEO of Cranbury, New Jersey based, Amicus Therapeutics. He believes that increased awareness about rare diseases, in general, is of great importance.

According to Crowley:

“There are 7000 rare diseases that collectively affect over 30 million people. That is more than everyone who suffers from all cancers and AIDS combined. So taken together these diseases are not rare at all – in fact they are remarkably prevalent. While the symptoms and severity of these rare diseases may vary, there are commonalities with regards to a framework of development for novel therapies that can and should be applied to all these disorders.”

Unfortunately, success stories like that of the Crowley family are far and few between. The uniqueness of their plight is one reason why it is so worthy of telling. The extraordinary measures that John Crowley and his wife took and the extraordinary feat of overcoming almost insurmountable odds to achieve a miracle of survival can only be accomplished by extraordinary people. Admittedl, few can be more motivated than a parent fighting for their children’s survival, but few have the faith, innovation and leadership required to make the impossible possible. Such qualities are what true leaders are made of. The type of leaders our nation needs more of. Leader’s who understand that personal freedom, can do more than people shackled by government burdens and blocked by government bureaucracies.

Perhaps that is why John Crowley is a Republican. He is not someone who will allow his fate to be written for him. He is determined to write his own. He is not someone content with being restricted by government but happier to get government out of the way.

It is also perhaps why, in the last few election cycles, John Crowley has been seen as a possible candidate for elected office in New Jersey. In 2008, there were some who hoped that Crowley would seek the US Senate seat currently occupied by the long serving, lackluster Frank Lautenberg.  Crowley failed to enter the race.

Later that year and in the early part of 2009, there was some speculation that John Crowley might run for Governor of New Jersey. Crowley declined to enter that race too.

I am one of those who held out hope for his candidacy.

Crowley is not a typical politician. He is a doer. His children’s lives are a testimony to that fact. He is not a go-along-to-get-a-long type. He is a man who gets what needs to be done, done. The type of person New Jersey and our nation needs in government, and his record is one of accomplishment, education and dedication.

Before his children were born, John was schooled for three semesters at the U.S. Naval Academy, graduated with degrees in foreign service from Georgetown University and went on to obtain his J.D from Notre Dame Law School before working as a litigation associate in the health care industry. He subsequently furthered his education and received an M.B.A from Harvard Business School. From there he went on to work for a San Francisco based management consulting firm.

Now, at 42, in addition to being a proud parent of three, devoted husband, as well as President and CEO of Amicus Therapeutics, Crowley is an intelligence officer in the U.S. Naval Reserve. He is also not shy about his political beliefs. As part of a political action committee called “Building the New Majority”, John assists a grassroots network that helps launch the candidacies of good Republicans on the local, county and statewide levels.

 Whether or not John Crowley will himself ever be one of those candidates is still a question mark. What the future holds is anybody’s guess. At least anybody who is not John Crowley. I have a feeling he knows what he intends to do.

Despite the recent election of a Republican Governor, New Jersey has few figures who are in any great position to win a statewide office in a state that leans so heavily to the left. But John Crowley would be one the few people who could be a strong candidate and who also has experience with uphill battles. The opportunity for statewide office will not come until 2012 when Democrat Senator Bob Menendez will be up for reelection. For that seat, there has recently been some speculation about financial journalist and anchorman Lou Dobbs running for the against Menendez, but if I had my druthers, the next Republican nominee for US Senate would be John Crowley.

Aside from knowing how to run a successful business that saves lives and aside from his having a track record of accomplishment that few can match, few candidates for elected office have a major motion picture made about them. That kind of introduction to the public can not be matched by any 30 second campaign commercial that airs during Desperate Housewives, when you get up to relieve yourself.

So I am looking forward to catching Extraordinary Measures   when it comes out on January 22nd .

Aside from promising to be one of those entertaining, feel good flicks, I look forward to seeing what I believe will be the launch of a new political rising star.

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Science Fiction Becomes Reality

 mikewithdogs Bookmark and Share  Guest editorial by Mike Duminiak

Researchers in Oregon have successfully raised monkeys created from the genetic material of one male and two females. The research is the first major step in using “good genes” from a third party to partially genetically engineer a child from two other main parents. The research was done with the best of intentions.

The germline genetic engineering process was used to replace a bad mitochrondria gene in the mother’s geneticGenetic Engineering material with a good mitochrondria gene from another female. This resulted in children born with nearly all the genetic material and characteristics of the parents, but with the potentially fatal mitochrondrial defect corrected. The applications of this research to ‘editing out’ hereditary genetic ailments while still allowing parents to basically have a child of their own is obvious.

The problem we still face is that scientific progress is advancing at a rate far greater than our legal or ethics systems. We are opening the door to possibilities for which our laws and our society are entirely unprepared. Once you start manipulating genetic material, the problems we face today become multiplied a thousand times.

I don’t advocate the abandonment of this research. I don’t advocate banning its use on humans. Having been born with a congenital heart defect that should have resulted in my death as an infant and would have if not for ground-breaking surgical techniques, I see the good applications of this research as well worth pursuing. However, we need to quit dragging our heals on the legal issues raised by this research and the ethical considerations.

It isn’t just the concern about making designer babies. While the concern that there will be pressure to make every male born over 6 ft with blond hair, blue eyes, heavy musculature and a large penis and every female born with blond hair, blue eyes, a disposition to be thin and large breasts, the real immediate concerns are far different. Long before we reach that level of ability in genetic engineering, we will have already found ourselves faced with serious legal and ethical issues.

For example if homosexuality is genetic, would it be reasonable for potential parents to edit out that genetic trigger to eliminate homosexuality forever? On the other hand, what if a couple with a known risk of sickle cell anemia refused genetic manipulation and their child was born with a “preventable” disorder – would that be child abuse or endangerment?

monkeys_606014aAs a society we have refused to face these kinds of issues. We hail the benefits of scientific research, but we pretend the problems raised by the same will just go away on their own. One of the main reasons why is because we don’t know how to address these issues.

As a conservative constitutionalist Republican, I am at a loss to find any jurisdiction to address these issues beyond the medical care regulations set in various States. I am forced to admit that on this fundamental issue of manipulation of genetic material in creating human life, we need a more uniform and clear set of rules for the whole United States. The only way to do that properly is to adopt a new Constitutional amendment granting the federal government the power to regulate the use of genetic engineering in humans.

The very idea of a government bureaucracy run at the whim of partisanship that has control over the genetic engineering of humans makes my stomach churn. With or without a constitutional amendment, we are likely headed in that direction. That is why taking action now and setting the limits, structure and guidelines for such an entity today is so important. If we wait, this will become yet another partisan football. The time to act is before the crisis comes.

We have time to debate how this regulatory body should be created. I would propose something akin to the Federal Reserve (basically independent from government partisan control) except run by a board representative of the various facets of this issue from medical researchers to doctors to psychiatrists to, yes, members of religious groups representing ethical and moral positions. But that is just one idea and very likely not the best one. That is why we need to start considering this issue now and take the time to get its regulation and oversight right rather than wait and get a political solution that benefits no one.

Science is moving quickly and our window of opportunity to proactively address this issue is rapidly closing. It is time to stop ignoring the issue and start addressing it. While we battle over health care, this issue is being ignored and has the potential to radically change health care in this country forever in ways that are almost unimaginable. Let’s learn the lesson from this current health care fight: waiting until things are starting to fall apart and then engaging in forced partisan fighting is not the smart way to handle an issue.

We need to do better on this matter. We can’t afford to do the same shoddy job with which government has been involved in everything else. Genetic manipulation is the apex of power. In our society, all power is derived from the consent of the people. Genetic engineering changes the people. We, the people, need to get a handle on it before we, the people, literally become the creations of bureaucratic rules and regulations that require or preclude us to be born with certain characteristics. If we fail to act, that will be the result.

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