A key Huntington’s disease trial remedy gets Orphan Drug Designation, as yet another young life is cut short

Ionis Pharmaceuticals, Inc. (formerly Isis Pharmaceuticals) has achieved another milestone in its search for a treatment for Huntington’s disease: on January 5 the company announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for its gene-silencing drug, currently under study in a clinical trial in Europe and Canada.

The FDA designation, intended to facilitate development of the test drug IONIS-HTT_Rx  by offering financial incentives and assistance, could not come at a better time. Huntington’s disease patients – like 18-year-old Terry Leach of San Diego, who died the morning of January 2 – continue to succumb to this devastating, untreatable disorder.

“Although the toxic protein produced from the huntingtin (HTT) gene in HD patients has been a target of interest for many years, IONIS-HTT_Rx is the first therapy to enter clinical development that is designed to treat the underlying cause of this fatal disease,” Frank Bennett, Ph.D., Ionis’s senior vice president of research, said in a company press release. “The granting of Orphan Drug Designation in both the U.S. and Europe highlights the significant need for a drug that could transform the treatment of HD.”

HD-affected Phase I clinical trial volunteers in London received the first dosing of IONIS-HTT_Rx in the October 2015 (click here to read more). IONIS-HTTRx could potentially reduce, partly reverse, and even prevent symptoms.

Likely ending in 2017, Phase I is testing primarily for safety and tolerability. If it is successful, Phase II and III trials measuring the drug efficacy’s would ensue. Together the three phases of a trial typically take at least five years. If the trial is successful, a drug could become available around 2020.

Frank Bennett, Ph.D. (photo by Dr. Ed Wild)

Increased dialogue, helpful benefits

“We were pretty excited to get Orphan Drug Designation,” Kristina Bowyer, Ionis’s executive director of patient advocacy, said in a phone interview on January 5. Ionis is based in Carlsbad, CA.

The designation means that the FDA recognizes “the severity of the disease and the limited population,” she noted.

The designation creates an opportunity for increased dialogue between Ionis and the FDA regarding the IONIS-HTT_Rx clinical trial, Bowyer explained.

Such extra communication can help resolve the unique issues of orphan disease trials. Because an orphan disease like HD involves fewer than 200,000 patients and very specific approaches to treatments, the design of clinical trials is atypical, Bowyer said. The smaller number of potential volunteers also means that the FDA might have to approve a smaller than normal trial, and sometimes perhaps even a faster trial, she added.

“It’s an area where our technology is well-suited,” Bowyer continued, referring to the antisense oligonucleotides that form the backbone of all Ionis drugs. “We have been able to focus on several rare diseases with a known target.”

By law, as outlined in the press release and in an e-mail from Bowyer, the Orphan Drug Designation includes significant financial benefits for Ionis: seven years of market exclusivity in the U.S. if the FDA approves the drug, tax credits related to clinical trial expenses, FDA assistance in clinical trial design, and a waiver of Prescription Drug User Fee Act filing fees – over $1 million per drug as of fiscal year 2009.

“These benefits help manufacturers recover the costs of developing a drug for small numbers of people,” Bowyer wrote. The Orphan Drug Act was signed into law in 1983.

Kristina Bowyer (photo by Gene Veritas)

Trial ‘moving along well’

In IONIS-HTT_Rx, HTT stands for the gene huntingtin, and Rx for medical treatment. The train has just recently begun, so, Ionis has reported no official update at this time.

“Everything is moving along very well,” Bowyer said.

The new name

Ionis has also adapted well to its name change, announced on December 18, 2015, in response to concerns about confusing the name Isis Pharmaceuticals, Inc., with the acronym “ISIS” used in the English-language media for the Middle Eastern terrorist organization, the Islamic State. Isis Pharmaceuticals was founded in 1989.

“We want people when they hear or say our name to think about the incredible drugs we’re developing and not a terrorist group,” Wade Walke, Ph.D., vice president of communications and investor relations, told the press.

Ionis chose its new name based on employee suggestions.

“It seemed to me that everybody came together and decided that Ionis was a nice-sounding, feeling name, as soon as someone hit on it,” said Stanley Crooke, M.D., Ph.D., Ionis’s chairman of the board and CEO. The new moniker is a so-called empty vessel name and has no inherent meaning other than what the company does, he added.

Said Dr. Crooke: “We’re here for the patients. We’re not here for our name.”

(Disclosure: I hold a symbolic amount of Ionis shares.)

The Ionis logo

Too late for Terry, other ‘HD angels’

I remember that Dr. Crooke spoke the same phrase – “We’re here for the patients” – when I met him briefly during one of my first visits to the company in the late 2000s.

I also remember visiting Terry Leach on Labor Day 2015. He was slowly but inexorably slipping away from the ravages of juvenile HD, a particularly devastating form of the disease (click here to read more about my visit).

Nevertheless, I was still shocked by his death on January 2.

What a horrible time to lose a family member. New Year’s will forever remind Terry’s mother Angela and his siblings of his passing.

My immediate reaction that morning was one of intense anger.

No 18-year-old should die!

Having had the privilege of knowing Terry and his family, I felt that I had failed as an advocate to speed the progress towards treatments.

Why hadn’t the Ionis trial come in time to save Terry and the other “Huntington’s disease angels” who have passed in recent weeks?

I know that HD researchers may have similar thoughts, as they work with the specter of this killer disease ever looming.

Terry Leach (family photo)

Keeping the faith

Later in the day, knowing that I needed to transcend my anger and sadness, I recalled that a new year always brings hope. As I took a long, strenuous walk with my dog through our hilly neighborhood, I renewed my resolve to fight HD in 2016.

I spoke to Angela a few times that weekend. Instead of a wake and church service, the family will hold a remembrance for Terry at the family home on January 16. A Christian minister will preside.

At Angela’s request, I wrote some words for the back of the remembrance cards she’s having made: “With his infectious smile and fortitude, Terry set an example for all to follow. His life was short, but full of love and joy. He is now free to walk with the Lord.”

Understandably, Angela was too drained to talk much. She did confirm that Terry – despite his inability to talk, his confinement to a wheelchair, and years of ingesting food through a feeding tube – had achieved his high school diploma. He had attended school through mid-2015, receiving assistance in a program for the disabled.

I asked Angela if she had any words for the HD community.

She said: “Just to keep their faith.”

Angela Leach in 2012 holding artist Lee Ellingson's drawing of her son Terry as "SuperTerry," the superhero who knocks out Huntington's disease (photo by Gene Veritas)

As he lay dying: FDA and Huntington’s disease families meet to ponder potential treatments

On Tuesday, September 22, when the U.S. Food and Drug Administration (FDA) focuses on Huntington’s disease drug development at a meeting with affected individuals and advocates, HD families must drive home the sorrowful truth: people are dying because of the lack of effective treatments.

About two weeks ago, shortly after pouring out my heart about my family’s HD struggles on the FDA’s pre-meeting questionnaire, I received a message from the mother of 18-year-old Terry Leach of San Diego: “It’s Terry’s final days if you wanted to say good-bye.”

A couple days later, on Labor Day, I visited Terry, who suffers from juvenile HD, in his bedroom. As Terry slept, his mother Angela and I looked on. Next to us a home healthcare worker prepared a can of liquid food to be administered via a feeding tube attached to Terry’s abdomen. Hospice workers are also helping.

“He grew a lot,” I said to Angela, amazed at how, despite the particularly cruel toll of juvenile HD, his body had strived to develop. It had been more than a year since I had last seen Terry.

“Yes, he did,” Angela said.

I noticed Terry’s beard and his healthy head of somewhat wiry, dark brown hair.

“You have a very handsome son,” I continued.

“Thank you,” Angela said.

Terry Leach resting at home (family photo)

Unspeakable pain

As Angela nervously shifted her balance from foot to foot, I sensed that she continued to carry the unspeakable burden that comes with juvenile HD: Terry’s first symptoms as an infant, his need for a full-time aide in school after losing his ability to walk and talk, the insertion of the feeding tube in 2010, Botox injections into his arms and legs in recent years to relieve pain, and leg and foot operations, among other procedures and hospitalizations.

Although Terry still attended school last academic year and also the summer session, he has declined considerably in the last few months, Angela said.

The night before my visit, he vomited after receiving liquid food through his tube. On the day I visited, the aide would give him only one can instead of the usual two, noting that his body would not accept anything more than that very small amount.

I asked Angela if I could touch Terry.

“Sure,” she said softly.

I ran my hand along the top of his hair. I remembered Angela and her family’s steadfastness in caring for Terry. I also recalled fondly our collaboration in the cause, starting with an article I wrote about them in 2009. In 2012, Terry emerged as “SuperTerry” in an artist’s comic-book like rendition as a hero defeating HD. In 2014, our families participated in the 2014 Team Hope Walk of the San Diego Chapter of the Huntington’s Disease Society of America.

SuperTerry in San Diego artist Lee Ellingson's rendition (above) and with Gene Veritas (aka Kenneth P. Serbin) at the 2014 Team Hope Walk (below, photo by Misty Oto)

Seeking feedback from the community

As Terry lies dying, the September 22 FDA event, a public meeting on “patient-focused drug development,” will seek feedback from affected individuals and others in the HD community.

The meeting will occur from 9 a.m.-12:30 p.m. at the FDA’s White Oak Campus in Silver Spring, MD. Pre-registration for attending the meeting and viewing via webcast is closed, but the FDA will make available a video of the proceedings shortly thereafter.

The meeting stems from the reauthorization of the Prescription Drug User Fee Act in 2012, in which Congress required the FDA to more systematically solicit input from patient communities with regard to drug development. The FDA hopes this will help its review process.

Huntington’s disease became one of just 20 diseases selected by the FDA for a patient-focused meeting through the end of 2015.

‘Nothing for neurological disorders’

Responding to the preparatory questionnaire, I revealed my situation as a carrier of the HD genetic defect and my mother’s decline and death from HD in 2006.

“I fear that I will become like my mother,” I wrote. “She had mild chorea [involuntary movements associated with HD]. I would not mind having chorea as long as I can continue to be myself, work, and not become a burden on my wife and daughter. My mother became a shadow of herself. I have great anxiety about losing my personality and ability to work as a college professor and writer.”

Like others, I would like to see a treatment that prevented symptoms, I added.

“If I get symptoms, then I would like a medication that allows me to manage the disease just as other diseases such as diabetes are managed without affecting a person’s livelihood, family life, or activities in general,” I continued. “There are lots of advances in cancer treatments, for instance, but really nothing in the field of neurological disorders that prevents, halts, or reverses the condition.”

I uploaded my response to the FDA’s public docket regarding the meeting. Anybody can comment at that link through November 23, 2015.

Speeding up clinical trials

At the meeting, I also plan to urge FDA officials to allow researchers and clinical trial administrators to use new technologies to measure the effect of medicines.

All clinical trials of new drugs taking place in the U.S. must receive the approval of the FDA, considered to have the world’s most rigorous standards. Although the drug industry executives I have met recognize the importance of that rigor in assuring the manufacture of safe and effective drugs, they sometimes have also expressed the need for the FDA to be more flexible and allow for faster clinical trials.

Generally, the FDA still does not accept techniques such as brain scans. HD researchers and other scientists are vigorously searching for biomarkers – signs of disease and drug effectiveness – in the blood, cerebrospinal fluid, and other materials taken from the body that can be measured using the scans and other new techniques.

Instead of waiting for a doctor’s clinical observation of an improvement, these techniques could potentially allow faster and earlier reading of a drug’s effectiveness.

I will stress that the FDA work closely with scientists and the HD community to make clinical trials as efficient and meaningful as possible.

Speed is of the essence for the HD community.

Heartbroken by another loss

As of this writing, Terry is stable, but his prognosis is not positive.

To give me strength as I travel to Maryland on September 21 and take part in the meeting the next day, I will keep fresh my memory of Terry.

I am heartbroken by yet another loss to HD.

As an advocate, I feel I have failed to fulfill the promise of hope presented so often to families such as the Leaches. No 18-year-old should die.

I am comforted to know that Terry is in loving hands – and that he never gave up, always smiling that infectious smile. And I am committed to making sure policymakers know of the people whose lives they could improve and save.

Terry in 2010 as a Hero of the Carlsbad Marathon

SuperTerry destroys the evil monster Huntington’s disease

Fifteen-year-old San Diegan Terry Leach wants to destroy Huntington’s disease, the condition that has devastated him since his toddler years and threatens to take his life very soon.

Terry’s story deeply moved San Diego artist Lee Ellingson to imagine a different outcome. In Lee’s Superman-like comic-book-style rendition (below), Terry has overcome HD by becoming “SuperTerry.” SuperTerry knocks out Huntington’s and saves the world from the ravages of the deadly disease that afflicts an estimated 30,000 Americans and could devastate as many as 250,000 more Americans who live at risk.

SuperTerry is vigorous, powerful, and triumphant. He beams with the joy of restored health and newfound happiness that the real-life Terry – along with every other victim of HD and juvenile HD – hopes for as scientists seek effective treatments and a cure.

“Wouldn’t it be great if this Huntington’s disease was like some kind of monster and Terry had super powers and could defeat the monster?” Lee told me in an interview. “That was my idea. It just kills me that a kid like Terry can have a disease like this at such a young age.”

Lee recently learned of Terry because of his own son Arnold’s struggle to live. Terry and Arnold attend the same after-school care program for disabled children and teens.

Arnold, who turns 13 later this month, was born hydrocephalic, a condition once known as “water on the brain.” He had an emergency operation immediately after birth to insert a shunt that drains fluid from the brain. He has had 23 more operations to adjust the shunt. Arnold also has autism and cerebral palsy.

Although Lee has made drawings for Arnold, SuperTerry is his very first piece illustrating a disease. He was shocked to learn that HD can affect children.

“He’s aware of what’s going on, but his body doesn’t do what it should be doing,” Lee, whose work includes background layout for the 1990s TV series Attack of the Killer Tomatoes and pieces for the San Diego Museum of Natural History, said of Terry. “That’s what’s especially heartbreaking for me. He’s a normal 15-year-old kid inside.”

Lee imagined the monster by thinking of the horrors of HD.

“He’s kind of like a blob,” Lee said. “He’s transparent and green. Slimy! I just wanted him to look real mean.

“I’ve always been pretty religious, but seeing all these kids really tests your faith,” Lee continued. “Kids like Terry and Arnold are the closest things on earth to an angel. Terry will never steal or cheat or rob or hurt anyone.”

Lee made the illustration based on photographs. On August 2 the two met in person for the first time at a fundraising event for the San Diego Chapter of the Huntington’s Disease Society of America (HDSA-San Diego). Lee presented Terry with the illustration.

“Terry was very appreciative of the picture,” his mother Angela told me. “He liked it a lot.”

Lee Ellingson (left) and Terry Leach

A feeding tube, operations, and looming death

SuperTerry HD treatments are needed now.

HD patients constantly struggle to maintain weight because they burn large amounts of calories and suffer from severely hampered swallowing. In 2010, in an end-of-life measure, the real Terry started taking meals and water through a feeding tube connected several times each day to a surgically produced hole in his abdomen. Before the operation, he weighed only 67 pounds. He now weighs 100, enough to help extend his life but still way below the average of 126 pounds for a 15-year-old male.

Terry’s body reacts to the hole as if it were an ulcer, causing him to produce large amounts of saliva that he wipes away with a towel constantly at hand. Doctors will inject botox into his salivary glands to diminish their output. He’s also gotten botox in his arms and legs to relieve pain.

Terry has undergone leg and foot operations to further relieve pain and tightness, correct deformities, and allow him, with assistance, to occasionally leave his wheelchair and walk. (For further background on Terry, please click here and here.)

Terry 

Because he can’t talk, Terry partially communicates through a language program on his iPad, which allows him to interact with people as the device’s speaker pronounces words and phrases that he selects. He takes regular classes at Madison High School, where he is starting tenth grade. Last fall he made the honor roll by carrying a grade-point average of at least 3.5. In middle school he received a number of other awards.

Terry loves computer games. During my visit to the Leach household, he played Club Penguin while I spoke to his mother for 90 minutes.

 “He’s always happy,” said Angela, a single parent who depends on Medi-Cal and other programs for financial assistance and, when she misses work because of Terry’s medical appointments and crises, the generosity of her employer, the San Diego Convention and Visitors Bureau. “He’s always wanting to give me a hug. And he’s so strong. He deals with everything and never quits fighting. He never complains, either. He’s always wanting to help. Everybody that knows Terry sees the light within him.”

However, Angela knows that, unless treatments become available very soon, Terry will die of HD. Recently a cousin of Terry’s with juvenile HD died at the age of 23. Other juvenile HD patients die in their teens or even childhood.

Angela Leach with the original drawing of SuperTerry (photo by Gene Veritas)

Raising awareness, saving the children

Knowing how little time remains for Terry, both Angela and Lee want to use SuperTerry to raise funds for HD research and increase awareness about HD and the difficult issues surrounding it.

Lee plans to expand SuperTerry into a comic strip and perhaps even a graphic novel about HD. Angela hopes to sell SuperTerry t-shirts in collaboration with HDSA.

“I don’t want any fame or glory,” Lee said. “I just want to help raise money. I want Terry to be the star.” As research progresses, science will also find ways to cure other diseases, too, he added. Such research could also benefit Arnold.

Above, San Diego Chargers football stars Philip Rivers (left rear) and Antonio Gates with Arnold Ellingson (left foreground) and Terry at HDSA-San Diego fundraiser in spring 2012. Below, friends Arnold and Terry enjoy Disneyland together.

Angela hopes that her and Lee’s efforts will help inform HD families about the option of genetic testing so that couples can avoid passing the disease onto their children through the use of preimplantation genetic diagnosis (PGD) or in the event of pregnancy – and depending on the couple’s personal and religious beliefs – early termination of the pregnancy. She wants SuperTerry to “save the children” from HD.

“I would never want another mother to go through what I did,” explained Angela, whose husband at the time (now an HD patient living in a nursing home in Indiana) did not tell her about the disease in his family until after Terry was born and the husband himself developed symptoms. HD families need to “get more proactive” about testing and family planning, she added.

“Terry’s life shows you what you face if you take a chance” by conceiving without all of the information, genetic counseling, and other resources available to HD families, she said.

“It would be best to be proactive to minimize that situation,” Angela continued. “It’s a lifelong situation. I’ve watched Terry grow up and deteriorate. There are so many challenges. We’re forever fighting.

“I don’t want his life to be in vain,” she concluded, recalling how an old friend’s son who had been in baby photos with Terry was now an imposing teenage football player. “I was happy for her, but it broke my heart. That could have been Terry. He could have been a football player. Or at least talk.”

Huntington’s disease, loneliness, and love

Imagine how lonely you would feel if you were a young person who knew you might never get married and have children because you faced a cruel and deadly genetic disease.

This is the harsh reality of many young people affected by or at risk for Huntington’s disease, which robs individuals of their abilities to walk, talk, think, and swallow. In the case of an affected male father, the children can inherit a far worse form of the disease, known as juvenile Huntington’s, with onset as early as infancy.

Starting in the teen years and extending into their mid-30s, dating for these people can become an incredibly difficult affair, instantly laden with fear and complex calculations about the future. Even though most standard testing centers won’t allow testing before age 18, those calculations can begin as soon as teens develop an interest in sex and procreation.

When will my symptoms start or worsen?

Should I opt for preimplantation genetic diagnosis to assure my children don’t have HD?

How will I be able to raise my children?

How will my spouse care for me, and for how long?

Why would anybody want to enter a relationship charged with such huge health and emotional challenges?

Will I end up alone?

Will anybody ever love me?

Understanding the pain

These are just some of the many questions that pass through the minds of young people in HD-affected families. The implications can become more serious as a person passes from at-risk to gene-positive status and ultimately on to the early symptoms.

As an HD-positive but currently asymptomatic person, I viscerally understand the emotional pain of young people in this predicament. Even though I have been fortunate to have a supportive wife, I have heard numerous depressing stories of the break-up of marriages and relationships where the mate could no longer handle the responsibility of caregiving.

And, as I await effective treatments to stave off the disease and advocate for the cause, I have experienced my own cascade of difficult emotions.

After writing about this in my last blog article, I received a comment from Nancy Hess, who tested negative for HD, about the deep loneliness we in the HD community can feel: “So often we find ourselves helping others comfort us when talking about HD. This can bring on an unexpected loneliness but then, as one might say to one's self from time to time, ‘Get over yourself!’ The truth is, there are only going to be a few in our lives who get it, and can stick with us through the dark passages.”

Fear of a lonely and love-less life dominate the stories of young people. Watch, for example, the short video “Five Women Speak from the Heart” on the homepage of the San Diego chapter of the Huntington’s Disease Society of America (HDSA-San Diego).

“I felt like having kids was the number one thing in my life,” says Whitney, an at-risk woman whose father developed HD. “I felt like: is my life over?”

“The only thing I fear is just being alone, not having somebody, because I wouldn’t want to put anybody through what I’ve gone through,” says Kristen, whose mother was stricken with the disease. “So I couldn’t expect anybody to love me like my father loved my mother.”

Crying all day long

Recently, Jiyeon Kwon, a 19-year-old man with Huntington’s, wrote in an HD group on Facebook about the social burdens of the disease.

Sometimes I regret telling anyone about my diagnosis. Now I know that some of my friends pretty much define me by it. They probably look at me and see "the guy with Huntington's disease" or "the guy that's going to be dead in ten years." And some people treat me differently and let me get away with stuff they wouldn't let anyone else get away with. It's just frustrating, I guess. It's funny how people who you thought were good friends make a totally different image of you just because of one disease you happen to have. [quoted with Jiyeon’s permission]

We can gain even deeper insight into the emotional impact of HD on relationships by listening to words of the unaffected partner, as in this message that I recently received from a female college student:

I have an amazing and wonderful boyfriend who I plan on spending the rest of my life with. I found out a while back that his mother has Huntington's disease. To be honest, when I found out I didn't research much on what this disease was about. (I have also never met her.) So I didn't realize until today (from my science teacher) that my boyfriend has a 50 percent chance of inheriting this. I've never been so heartbroken and confused in my life. I don't know what to do. I don't know what to think. He's not one to really talk much about this so I don't even know the right way to bring it up. I've done nothing but cry all day long....

I'm so new to all of this, and all I know is all the negative things I've read on the internet. I'm scared for my boyfriend so much but I don't know if it's wrong to show my fear in front of him.

True, enduring love

Indeed, in these circumstances, love must be true to thrive and later endure.

My father and HD-stricken mother’s marriage was far from perfect, but they held on through the fifteen-plus years of her illness. My dad, the “Huntington’s disease warrior,” fed, bathed, and clothed Mom daily until she went into a nursing home in mid-2005. He visited her each day at the home until her death in February 2006.

Because Mom was falling ever more frequently, Dad probably should have relinquished his caregiver role even earlier. But then she may have died even sooner.

In this era of instant hero status, heroism has lost its meaning. Everybody, it seems, can be a hero for the proverbial 15 minutes. But Dad – and the millions of other caregivers in America looking after HD, Alzheimer’s, and other neurologically disabled people – are the real heroes. Nobody knew about him, but he got into the HD trenches every day for 15 years.

In my case, I am greatly blessed with the unbending support of my wife. We learned of my mother’s diagnosis just three years into our marriage and before attempting to have children. As a recent immigrant and still without an established career, she could have responded to the daunting prospect of caring for an HD-stricken husband and having affected children by taking the easy way out: returning to her home country of Brazil and her old job.

But she stuck with me then – and after I tested positive in 1999. The lasting bond we forged was further cemented by our good fortune in having our “miracle baby,” who tested negative in the womb. We know all too well the fear of passing on the defective gene.

In 2009 and 2010 she ran in a half-marathon to benefit HDSA. (Click here to read more about us.)

Support for families

I have gained an important lesson from my parents’ experience and my own family’s situation, as we face my inevitable incapacitation: HD families need greater support from HDSA, physicians, and local social services and clinics. As so many have stated before, HD is a family disease affecting everybody within the nuclear unit and often reaching far into the extended family.

Despite the immense health and social challenges of HD, many families and caregivers steadfastly support young people with the disease. The family of Terry Leach, a San Diego teen who first developed HD as an infant, has shown great love and togetherness in caring for him (click here to read more).

Many spouses and caregivers accompany their loved ones to the local support group. Some caregivers even attend after the patient has gone to a nursing home and can no longer join the group. In other cases, at-risk individuals are unwilling to participate in the group, but their potential caregivers attend anyway.

Doing the right thing

It’s also encouraging to see the young people who are standing by their partners and spouses. I was touched to read the comment to the Facebook HD group by one young woman who was “having the best week. I just got home from Disney World with my amazing boyfriend. We had the best time together. He takes such excellent care of me. I used my wheelchair at Disney World. It worked out great. I love my boyfriend with all my heart. I am so blessed that he is in my life.”

With such stories of commitment and my own years of experience in mind, I wrote back to the young, heartbroken girlfriend of the at-risk college student:

I think the best advice I can give is to be completely honest with yourself and your boyfriend. Do NOT hide or avoid or deny the subject of HD. It's best to meet it openly and head-on, although always with patience, gentleness, and kindness. I am a firm believer that hiding the truth can be convenient and easy in the short run but ends up doing very serious damage in the long run. You're doing the right thing by learning about the disease and all that it could mean for your boyfriend. And it's important for you to stand by him as a girlfriend and as a friend. We cannot predict the future, but even if you end up not being in each other's life down the road, you will have done the right thing by supporting him at this point.