Why children need Doctornauts

Dear Parents of Children with Diseases and Disabilities: Pay Attention for They Desperately  Need Doctornauts. It’s Time to Get Angry- and Active!

This video from last year features my granddaughter and I speaking about how the Doctornaut Act can help children.

In previous posts I emphasized how physicians or doctornauts who volunteer for clinical studies can rapidly speed up the discovery of medical breakthroughs and cures . In the past, I also emphasized that doctornauts, though adults, can do the same for children. Unfortunately, I wasn’t successful in getting the message across to you, their parents.

Last week I had dinner with my old and kind friend, Joseph Valenzano, the dedicated founder and President of Exceptional Parent (EP), a long –standing, highly respected educational organization which deals with children with diseases and disabilities. During dinner he urged me- to tell the truth, he really gave me hell- not to give up on educating you, the parents, on how doctornauts can dramatically help your children. Joe’s  a very persuasive guy, so here I go again!

There are two categories of childhood maladies, one that are relatively common and the others rare. The latter are oftentimes called orphan diseases. In the former category are conditions such as  cerebral palsy, asthma, diabetes, leukemia, brain tumors, autism, and ADHD. In the latter category it is estimated that there are plus/minus 6000 conditions most of which are of genetic origin. Because of their small populations and , therefore, for economic reasons,  relatively few clinical studies are conducted on them. One source reports that 30% of children with rare diseases will die before their fifth birthday. The encouraging news is that genetic discoveries in more common children’s diseases will be effectively applied to many of them.

Let’s take an example on how adult doctornauts with Parkinson’s disease can help children with Cerebral Palsy. Parkinson’s is caused by the destruction of certain brain cells. There are various ways to treat this disease. One is with pharmaceuticals. But there is often a problem with promising drugs in animal studies because of what is called the blood brain barrier- which prevents them from getting to the brain. Successful research on ways to eliminate the blood brain barrier in doctornauts with Parkinson’s can also be applied to cerebral palsy children. Discoveries made in doctornaut Parkinson disease patients with stem cell implants, insertion of genes into cells and even the use of electric devices to control movement among others are all applicable not only to Cerebral Palsy children but also other central nervous system diseases of children.

Here’s another example: let’s say a brilliant researcher has an out-of-the –box idea on a new therapy for leukemia which FDA and hospital committees would not approve for clinical studies because they believe it’s too risky. But because of the Doctornaut Act a physician with leukemia can and does volunteer, and the therapy proves to be very effective. This same therapy would then be available to children with this disease as well as adults.

( FYI, there are many bright researchers with creative therapeutic ideas which are never tested let alone made public because of the barriers to clinical research. And they remain unknown because there is no place for them to go to have their ideas evaluated in a clinical study. In addition to the establishment, these folks are essential to speed up medical discovery).

Awhile back- before some of you were born!- I brought carnitine into the United States and conducted the first clinical study in adults in patients with hyperthyroidism. Because of its availability it was tested clinically in children with the fatal disease, Primary Carnitine Deficiency, and eventually approved by the FDA saving a number of children’s lives.

You may wonder why there has been little interest in the Doctornaut Act. Despite my personal efforts, with the exception of physician and former Senate Majority Leader, Bill Frist, who had a discussion draft of the Doctornaut Act circulated but failed to attract support, I don’t have a clear answer except the well accepted general rule that there is an establishment system which very rigidly regulated  where bold ideas are not well received. There’s an old saying that progress is disagreeing with the established experts. Also, patients are not aggressive and understandably surrender their lives to the rules of the system. But there has been one dramatic exception to this rule that tells us that mountains can be moved. It’s AIDS patients. In the early days of AIDS there was little support to conduct research on this fatal disease. The angry AIDs patients then got together and launched an extremely effective public relations campaign , strongly supported by mass media, resulting in enormous sums of money poured into AIDS research. And this effort handsomely paid off with the discovery of very effective, life-saving breakthrough therapies.

I am told by a number of my colleagues  that you, the parents, are highly motivated  advocates for your children and willing to take that extra step to help rid them or at least reduce the suffering of their diseases or disabilities. Well, here’s your chance. Sign the petition to Congress for the enactment of the Doctornaut Act by clicking here, and then take the next step. Notify others to join in!

Also, I’d like to hear from you so that I can learn where your heart is and plan next-step strategies.

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