How can this be happening?  During the past 10 or so years, I have become increasingly frustrated at my inability to communicate effectively with everyone.  The volume of my speech and my articulation suffered.  I actually took the speech therapy course, LOUD, at St. Joseph's Hospital in Atlanta, but, there was not a lot of carry over for me- laziness on my part.  I worked harder to be understood by friends, but did not work when I talked to Nancy.  My speech was slurred and not loud enough.  Therefore, everything I said was followed by "what?" which was tiresome for both of us and led to more frustration on both our parts.

This past summer at an appointment with my neurologist, he mentioned the program BIG and how successful it was for Parkinson's patients.  Nancy called and they said that they probably would not have an opening until December.  However, they called a few weeks ago and I've begun the program.

First, I have to say that this freaked me out.  I'm very cognizant of my difficulties walking, speaking, thinking.  I have fallen more often than I would like to admit.  I am sometimes reluctant to stand close to another person for fear  that I will fall and take them down with me.  I was very nervous about attending the class.  I've undergone invasive DBS surgery and was less nervous going through that than I was about this. After the first evaluation which included a few exercises, I was ready to quit because I felt so inferior.  Our 19 year old granddaughter said to me, "why do you think you're special?  They see hundreds of patients, probably some worse off than you.  They take you where you are and try to help you. You have to be willing to work."  That did it. I bought in hook, line, and sinker.

It's amazing how my psychological health influences my life.  DUH! HA! I have already learned in two sessions how to get into bed and out of bed without needing Nancy to help.  I'm also much better in getting out of chairs.  I'm practicing the exercises every day and I believe that at the end of the sessions, 4 weeks with 4 days a week, I'll be much better.  I'm actually speaking louder also.  Speaking louder helps my articulation.

Maybe, you can teach an old dog a few new tricks.

I've begun taking RYTARY instead of the 25/100 carbidopa/levadopa I took so long.  It has evened out the on/off time I felt with my old regimen.

In short, I have a lot to be thankful for.  We have talked with and met so many people since all the hoopla following the introduction of Tasigna October a year ago..  I'm happy to say that there are more people every day who get their neurologists to prescribe Tasigna.  Some are seeing huge changes and some are not.  Life is a crap shoot!  The Canadian pharmacies who are making money off of all of us are very happy. HA!

We hope you all have wonderful Thanksgivings with those you love and that life is good for you now and in the future.  Life is improving at the Hoffman's.

I swore that I would never put pen to paper again after I posted my last post.  It's harder now that I'm 75 to get 'psyched up' to write. Nancy stays on my case, though, and your comments persuade me that I still have something to share.

Several things have happened since I last posted.  CBS Evening News with Dr. John LaPook came and interviewed us for about 2 hours.  They plan to run it prior to the next trial which begins in January.  He couldn't have been nicer; his producer, Amy Birnbaum, was a doll and the sound and camera people were great.  It could not have been a nicer experience.  Of course, during the interview, my medication wore off, so my tongue was out of my mouth most of the time.  I'll let you know when we find out when it will be shown.

I had been on requip since my first diagnosis with Parkinson's in 1997.  At one point, I was taking 24 mg. a day- not good because I became an on-line gambler.  I still continued to take it at a reduced dose until a few months ago.  Now, I'm on mirapex ER and doing better.  I also began taking Rytary a few weeks ago.  That is helping a lot- no more on, off times with my medication.

Yes, I'm still on Tasigna and feel that it's helping me.  I'm still taking the original 150 mg that I took in the trial.  While there are days I feel not so good, most days I'm 'up and at em', feeling good.  I'm much more help around the house which Nancy appreciates and thanks me for. I am a retired university professor and am able again to have good conversations with her and with others.  So, I feel useful and needed.

The Michael J. Fox Foundation and the Georgetown University feud became public and, quite frankly, I think the Fox Foundation came out looking like they can't stand not to be the first to make a breakthrough for Parkinson's Disease. It's amazing how politics is in everything!!  So disappointing and also disgusting.

I take medication five times a day.  A few days ago, I moved some of the pills around in my pill container, taking them at different times.  Don't ask me why I did it.  Nancy was furious!  Yesterday, she organized my pills for three weeks as she does every three weeks.  She made me watch and she explained what everything was and why I should take it at a certain time.  She can't die on me, because no one can ever figure out what I take when.  She uses a plastic weekly pill dispenser for each day and labels the hour I'm to take pills.  What a job!

It's astounding to me the trouble that people go through to find our number and make contact with us. That just shows how important it is to have hope.  People have called from all over the world.  Of course, the real question is "How are you doing?"  One recent caller is renting a houseboat on the Potomac and coming up from North Carolina so her husband can hopefully be in the next trial at Georgetown. People say, "oh, thank you, for talking to us." There is no need for thanks.  We are all in this together.  We are the Parkinson's family and we owe each other as much information and help as we can.  We all need listening ears.

The wonderful thing about this upcoming trial is that Novartis has promised in writing to provide Tasigna for all trial participants after the trial ends.  If they had done it for me and my group, it would have been so much easier for all of us.

Right now, I'm pretty 'down' on pharmaceutical companies and insurance companies.  Pharmaceutical companies are in business for the profits they make.  People who need the drugs they manufacture are way down at the bottom of the companies' concerns.  They overcharge, keeping many people from
being able to purchase the drug, and then the insurance companies refuse to pay for many drugs that I take, so we have to go through the appeals process.  It's frustrating and time-consuming.

Life continues to be good at the Hoffman house.  I promise to write when there's anything to say.
Take good care of yourselves and let us hear from you.

In God we trust.  We're having some reservations about trusting drug companies.  HA!!!

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

A few weeks ago, we received information that the results of the Nilotinib study were published in The Journal of Parkinson's Disease. You can go to their website.  It's in the current issue. I picked myself out of the list of participants (#11) and began to look at the results.  The next time I see Dr. Pagan (in August), I intend to get a more detailed and understandable report. In the meantime, I will get Dr. Drew Falconer, our second neurologist to share his opinions about the results and mine, in particular. Dr. Pagan will be beginning another trial with PD patients, I think, in the fall. Also, there will be trials for ALS and Alsheimer's Disease at Georgetown.  We also checked out the Michael J. Fox Foundation and found that they will also begin a trial with Parkinson's patients.  This is highly encouraging.  While the neuroscientists in association with the MJFF were critical of the "hype" they believe came from such a little study with so few participants and no double blind "scientific" components, they still believe it's worth researching.  Only people who have a degenerative, debilitating disease understand the need for HOPE.  That's why I and a number of others are taking Nilotinib and paying a lot of money for it.

By the way, our second neurologist, Dr. Falconer, has recommended that I consider getting Deep Brain Stimulation on the second side. He says that since I'm only being stimulated on one side, the second side would be advantageous.  DBS made a big difference for me when I had the first side done in 2008, so I'm thinking about it.  Eighty to ninety percent of people having DBS note improvement in their symptoms.  The researchers say that about 30% of patients with Parkinson's Disease are good candidates for DBS.

We received a call from the communications person at Georgetown last week.  She said that CBS Evening News was interested in talking with us a year after the trial.  When we know more particulars, we'll share it.

The Study

The Study

We went to Dr. Pagan and over a period of two years, I was involved in a study using Apokyn (a rescue drug) and I received botox in my feet for rigidity.  I had always been eager to be involved in trials and had participated in many in the Atlanta area.  When I saw information about a clinical trial using Nilotinib, a drug now used for Leukemia, I asked about participating.  Dr. Pagan’s only concern was the number of trips we’d need to make to Georgetown for the nine month trial.  We assured him we could do that easily.

I was tested first to see if I was a candidate.  Nilotinib can potentially cause irregular heartbeat, so an EKG was called for.  Bloodwork was also done.  Parkinson’s mental and physical testing was completed and I was told that I qualified.

Over the next six months, we made multiple trips to Georgetown.  Each time an EKG was done to ensure that the QT interval was appropriate.  Bloodwork was done to ensure that I had sufficient minerals and vitamins in my system, and three lumbar punctures were administered. 

The study was labeled as a safety study to see if Parkinson’s patients could tolerate low doses of Nilotinib.  The hope was that it would not only be safe, but also that alpha-synuclein in the spinal fluid and tau in the blood stream would be reduced.  These are plaque forming substances that cause brain cells to die.  Patients were randomly assigned to either a 150mg or 300mg dose of Nilotinib per day.  Strict guidelines for timing the doses were adhered to.  Everyone took medication-there were no placebos given.
                       
The results were significant.  All 11 people who completed the study improved.  We walked, we talked, we thought with greater ease and clarity.

Dr. Pagan videotaped me in the “get up and go” walking test at the beginning of the study and every visit afterward.  Soon, I was walking steadily in half the time of my original walk.  My speech was louder and clearer, I was no longer falling, I was less confused about things in general, and my life was considerably better.  I had always been an avid reader, but had not read a book in a long time.  I just couldn’t concentrate enough.  I first read the David McCullough book on the Wright Brothers and then progressed to others.  I was thrilled!

The study consisted of taking six months of medication.  Three months after the medication ended, I returned to Georgetown for testing again.  I had begun to regress without the medication.  During the trial, the pharmaceutical company, Novartis, made a verbal commitment to continue to provide the medication for no charge to the trial participants.  That did not happen and resulted in much frustration on the part of all of us.  We held out, hoping that Novartis would come though. 

The study was reported at a conference of Neuroscientists on October 17, 2015 in Chicago.

In preparation for that conference, information from the Georgetown communications person was released to the media. (See attachment 1).  Immediately, our phone began to ring asking us to be interviewed. We were interviewed by NPR, NBC, Fox News, Univision, TV Globo-Brazil, and the Daily Mail in London. 

News services picked up the story and our daughter, Jennifer, googled me to tell me that I was mentioned in thousands of articles.

https://www.youtube.com/watch?v=Rifa6LPy60A

Then the phone really began to ring.  Parkinson’s patients and/or their caregivers looked up our phone number and began to call.  There was a great deal of interest in the process and especially in the results.  We spoke to people in twelve states and Canada.  They wanted to know all about my symptoms, duration of the disease, how I was doing, where we got medication, etc. etc. etc. In short, they came looking for hope and found it.  We began to expect return calls from people we had talked with who were in the process of getting the medication, were just beginning it, and wanted us to anticipate when positive changes would occur for them.



When Novartis didn’t come through with their promise of providing medication by the time of the conference, we began to investigate costs.  While Nilotinib is an FDA approved drug for cancer, insurance will not pay for it to be used to treat Parkinson’s disease. We found it at a pharmacy in Canada for $5321 for three months’ medication. While the costs are exhorbitant, we were blessed that my younger brother helped to pay for it.  We ordered it and I began taking the medication again on November 22, four months since my last dose during the trial.  During those four months, I had regressed a lot.  It took about two months of medication to see positive changes.  I believe that I will continue to improve as time passes.

Millions of people worldwide have Parkinson’s and/or Lewy Bodies Disease.  If, in fact, Nilotinib/Tasigna, can continue to decrease the plaque that causes brain cell death, there will still be millions of people who do not have access to the drug
because of cost.

During the time I was off Nilotinib, I wrote a few articles to express myself about pharmaceutical companies. I will be sharing them as a part of this blog.

I am beginning my 9th month of medication.  I have many more good days than bad days.  I am hopeful that this medication will continue to help me feel better and better.
                   
We understand that there is research going on in Israel and England that is focusing on something similar to Nilotinib , phage, that has similar results.  The researchers are hoping to find a cure for Parkinson’s, Alzheimers, ALS.  We pray that they will.  Hopefully, I will still be alive to reap the benefits of the research.  If not, though, I am heartened to think that millions of people may be spared the debilitating effects of a degenerative neurological disease.

Whenever you have a degenerative, debilitating disease, you become a member of another “family”.  All of us are in this together.  I have learned so much during my 19 years with Parkinson’s about finding your purpose in life, about building relationships with people you love, about kindness, about looking everywhere and not being afraid to try medications and trials and surgery and anything else that might help you or others who share the same burdens, about making your life count for SOMETHING.  To paraphrase the educational psychologist Louis Raths, “to make a difference that you lived at all.”

Our move and new PD doctors

The Move to Virginia and New Doctors

During an Easter visit in 2012 with our daughters in the DC area, our son-in-law said that he wanted to have a serious talk with us.

We were puzzled and waited to hear what he would tell us.  We were surprised when he said, “It’s time for you all to move up here.  Laura Jane and Jennifer (our daughters in this area) will be the ones to take care of you.  You should come now and see your grandchildren grow up.”  After telling him we’d infringe on his family’s life, he looked me straight in the eye and said, “Come on.” 

We had never considered moving to the DC area- we always felt it was too expensive with terrible traffic.  Being asked to move here by your son-in-law motivated us to get busy, sell our house in Atlanta where we had lived for 42 years and strike out for an adventure in Virginia.





The first concern we had was finding a neurologist who specialized in movement disorders.  Dr. Michael Okun, Alan’s physician in FL, recommended Dr. Fernando Pagan at Georgetown University.  They had known each other for some years and he felt that Dr. Pagan would take good care of me.