Where is the Knight on the White Horse?

A few months ago, I wrote this piece trying to talk Novartis into paying for Nilotinib for the clinical trial participants.  We were initially told that after they had agreed to allow us to continue on the drug under "Compassionate care" that the drug would be provided to the participants.   As you may now know, that did not happen.  Since I wrote this, one of the participants has passed away.  We continue to wait for Novartis to do the right thing by all of us who participated in the trial.

WHERE IS THE KNIGHT ON THE WHITE HORSE?

Alan J. Hoffman, Ph.D

Do you remember the joke that went around some years ago about a divorce?  The husband in the couple said that his former wife had “gotten the gold mine” and he had “gotten the shaft.”  That’s the way I and eleven other patients in a clinical trial feel.

Twelve of us participated in a clinical trial at Georgetown University which tested the drug Tasigna (Nilotinib) which is used to treat leukemia to see if it was safe for patients with advanced Parkinson’s Disease.  The idea was formulated by Dr. Charbel Moussa at Georgetown.  He believed that substances from the bloodstream and spinal fluid which cause plaque that increases brain cell death could be reduced by Tasigna.  This would slow down the progress of Parkinson’s Disease.  There was pre-trial testing to make sure the patients would be able to take the medication without side effects of heart disease.  The twelve who were accepted into the program began to take a daily dose of either 150mg or 300 mg capsules.

After about a month, wonderful things began to happen.  The quality of my life began to improve.  I no longer had to have my wife translate what I was saying to our friends.  My articulation and volume improved. I began to do things around the house that I had not done in years-emptying the trash, unloading the dishwasher, setting the table, etc. My attendance at church and at activities increased.  We began to participate in social activities as well. My wife says that it was life-changing for her and for my children and grandchildren.  Moreover, my cognitive skills improved.  I could carry on an intelligent conversation without losing track of the point.  I understand that another patient who was bilingual in Spanish and English and had completely forgotten English began to speak it again after taking Nilotinib.  This made a huge difference for his English-speaking wife.

Then came the rub.  The medication ended for me on August 5.  We dreaded that day because we feared regression without it.  Just then the knight on the white horse appeared and promised Dr. Fernando Pagan, my neurologist, that Novartis, the drug manufacturer, would continue the medication for the twelve trial participants for free.  Excitement was rampant.   We were thrilled beyond measure.  I could continue to take Nilotinib and hopefully would get better and better and at the least, be stable.

Alas, the knight on the white horse has either vanished or is delayed.  Since Nilotinib is not yet approved for treatment of Parkinson’s Disease, insurance won’t pay for it.  The cost is astronomical for us- $5000 in Canada for 112 days worth and over $16000 in the US for the same amount- very serious decisions for non-wealthy people.

I am now on the medication again.  $5321 later, I began the medication on November 22.  Today is December 15.  I am beginning to get better already.  One other individual in the trial is also taking the medication again.  We worry about the 10 others who don’t have a choice.

Dr. Pagan has secured private funding to conduct more trials to see if Nilotinib makes the kind of difference for others that it has made for me.  Years from now, the FDA may approve the medication for Parkinson’s Disease and insurance may pay for it.  I believe that Nilotinib offers the next hope for patients with Parkinson’s Disease.  In the meantime, the twelve of us wait for the knight on the white horse.  When we agreed to be “guinea pigs”, we took the risk that we wouldn’t be harmed by the treatment.  Since the medication helped all of us, wouldn’t it seem fair for the pharmaceutical company, Novartis, that manufactures it and is the richest drug company in the world to pay for the 12 original “guinea pigs” whose quality of life was so poor that they were willing to try anything? Where is the promised compassionate care?

Where is the knight on the white horse?

My Family

Hoffman Brothers Construction

My Uncle John 'driving" the Marek's Grocery truck

My Dad had an 8th grade education, grew up on a farm in a little burg in Wisconsin, and ended up owning a successful construction company in Two Rivers, Wisconsin.  My Mom grew up helping out in her family's grocery store.  She had a high school education and was the state spelling champion.

Mom and Dad when they were courting

A triple wedding!

Nine months and one day later!

They had six children.  I'm the oldest.  Nancy always tells me when I get around my siblings, I revert back to being the oldest and the bossiest.  That's probably true.  It's hard to break those habits from childhood.

A very 1960's Christmas

My siblings were very successful academically and are well respected in their fields of work and life.

We spent a few days last week with the brother who is closest in age to me and his wife.  We had such a good time with them in the little town of Abingdon, VA, and I began to think about all my siblings and their unique skills and talents.

Our niece's wedding and a great opportunity to be together

Tom is 15 months younger than I am.  He was my best friend growing up.  He actually taught me how to study when we were in high school.  He has a Ph.D. in nuclear physics, worked in Oak Ridge, TN, for some years, then became a successful commodities trader.  He and his wife, Sue, have traveled all over the world and are enjoying their four grown children and their six grandchildren.

Roger is probably the genius of us all. He has over 50 patents and has worked in the paper industry for many years.  He is environmentally very active in protecting water, land, and air, and is a generous and giving human being.  He and his wife, Carol, have five children between them and 13 grandchildren.  They are also world travelers.

Ron is a creative genius who was instrumental in designing and building the house he and his wife, Nancy, live in now.  He invented boxes for shipping items of different sizes and shapes, and traveled around getting the US technologically ready for Y2K.  He also authored an incredible book, Hearing Silence, about his experiences as a Marine during the Vietnam war. He is also a prostate cancer survivor and our hero.  Ron and Nancy have five children between them and 5 grandchildren.

My brother, Glen, was killed in a car accident at 41 years old.  There were over 300 people at his funeral.  He was well loved by all of us.  He had a kind and gentle nature that drew people to him.  He participated in the Big Brothers/Big Sisters program and was a great Big Brother to a boy who had a lovely, single mom.  A few years later they married and, together, raised her two children.  Glen and his wife, Darlene, were instrumental in starting a family reunion every Easter that continues to this day, 35 years later.  Darlene has remained a big part of our crazy family and continued planning our family reunions long after Glen's death.  Darlene has two children and 2 grandchildren.

Tough guys heading for a swim: Glen, Ronnie,Roger, Tom, and me

And Mary makes six!

We five brothers were almost stair-steps.  Then, lo and behold, a sister was born.  In truth, they probably saved the best for last.  Mary has two children and four grandchildren.  She lives in Arkansas and has been head of Housing and Urban Development in Conway for many years.  Mary takes care of people; many of whom can't take care of themselves.  She is well loved and has been recognized by many people as a leader in the field of federal housing. She is also the best little sister a guy could ask for.

Enough for today.  Just was thinking about my parents and my siblings and felt like sharing.

From left to right: Ron and Nancy, Roger and Carol, Sue and Tom, my Nancy, Mary, me, and Darlene