FDA Approves Pfizer’s First Gene Therapy, for Hemophilia B

A Cancer Drug That Impacted My Life For SLL

After reading a book about how Imbruvica was revolutionized for SLL and CLL, I now have a greater appreciation for the drugs I receive.

As a patient diagnosed with small lymphocytic lymphoma (SLL), my interest was piqued when I heard an interview with the author, Nathan Vardi who wrote the book, "For Blood and Money: Billionaires, Biotech, And the Quest for a Blockbuster Drug."Vardi, a finance writer, found the story of how the first BTK inhibitors were developed fascinating. So much so, that he decided to write a book about it. This drug called Imbruvica (Ibrutinib) "revolutionized" the treatment of chronic lymphocytic leukemia (CLL) and SLL. As a patient on Calquence (acalabrutinib), which is a new generation of Imbruvica, I wanted to know more about this pill that revolutionized treatment for patients like me.

Vardi brought different people together to get their input. This included investors because billions are spent on cancer research. It also included physician-scientists, people who work in cubicles in biotechnology offices, as well as patients. No one person could do this. Patients who volunteered for clinical trials were at the very core.

This drug took a unique journey to make it over the finish line. So many contributed to making it available. Most of us who are patients have no idea what goes into these drugs that have helped tens of thousands of people. It also helps all of us who are lucky enough to be able to take this drug understand why these pills are so expensive.

Originally, the drug was developed for rheumatoid arthritis. Then, it was pursued for lymphoma treatment. At that time, they also used blood samples from patients with CLL because it was easier to obtain. Luck definitely played a role. When that showed promise, they changed their focus to pursuing the drug for CLL, not lymphoma, and more money was invested in it.

Vardi explained that biotech engineers wanted to study the biological effects on the human body. He said, initially, "...A covalent bond that irreversibly binds to its target was shunned by the biotech universe." The fact that this drug ended up being used for blood cancer treatment is a crazy story. So many things could have gone wrong, but luck was on their side.

Vardi decided to write his book to explain how this drug became an option for patients. He wanted people to understand the process of drug development. There is the "financial piece, the regulatory piece (like FDA approval), the science piece, and of course, the different personalities that all had to come together." Vardi said in his interview, "How does this happen? Patients should realize what goes into it. Patients can appreciate the insight of how the drug process works." He stated, "Most people are not interested until it is front and center and they are the patient."

Now this drug has led to new-generation drugs like the one I currently take, and I am so grateful for the whole process. If not for the actions that took place, Calquence would not have been developed in time for me to take it and have the benefits of living a quality life because of it. Next on my list of books to read will be "For Blood and Money: Billionaire, Biotech, and the Quest for a Blockbuster Drug." Thank you, Nathan Vardi, for writing a book that I was also told was quite an interesting read!

For more news on cancer updates, research and education, don't forget to subscribe to CURE®'s newsletters here.

What To Know About Aggressive Chronic Lymphocytic Leukemia

Chronic lymphocytic leukemia (CLL) is a blood cancer involving an increased number of white blood cells in the lymphoid tissue.

Over time, the cancerous cells increase, leaving less room for healthy blood cells. Indolent (causing little pain) CLL can remain stable for years without treatment, and aggressive CLL is fast-growing and requires more immediate treatment.

Aggressive CLL is also associated with an onset of symptoms that emerge as the cancerous blood cells leave less room for healthy blood cells. You may feel tired, have difficulty warding off infections, or show other signs of the disease.

This article explains what aggressive CLL is and the symptoms, treatment, and complications.

Aggressive CLL is a fast-growing subtype of CLL. CLL is the most common form of leukemia in adults in the Western world. Because it develops rapidly, you'll likely need immediate treatment following diagnosis.

According to a 2020 study, doctors may use the immunoglobulin heavy-chain variable region gene (IGHV) mutational status to determine whether someone has aggressive CLL. People with a mutated IGHV have a slower-growing form of CLL, while people with unmutated IGHV have a faster-growing, more aggressive form of the disease.

The study also notes that about 50% of people diagnosed with CLL will need to start treatment within 5 years of diagnosis.

What is the most aggressive form of CLL?

The most aggressive form of CLL occurs when CLL transforms into Richter's syndrome.

According to the same 2020 study, people with unmutated CLL have a lower 5-year survival rate than those with mutated CLL. The study reports that the 5-year survival rate is 71% for unmutated (aggressive) CLL and 81% for mutated CLL.

The National Cancer Institute estimates that the overall 5-year survival rate for CLL was about 88% between 2013 and 2019.

However, keep in mind that all survival rates are estimates designed to give you an idea of the overall severity of cancer. Several factors affect your likelihood of survival, including:

overall health

age

response to treatment

aggressiveness of your CLL

stage of the cancer

Your care team can provide the best idea of your overall outlook and help provide treatment that will best suit your needs.

In nonaggressive or indolent cases, your doctor may recommend delaying treatment. This is particularly true if you don't show signs or symptoms of cancer or have typical blood cell counts.

However, they'll monitor your condition and begin treatment once necessary.

If you have a more aggressive form of CLL, you may need more aggressive CLL treatment. Instead of watching and waiting, your doctor may recommend one or more of the following options:

They may also recommend that you join a clinical trial. Clinical trials test new treatment options to find safer and more effective ones.

Aggressive CLL has a higher chance of causing symptoms. You may experience symptoms and complications such as:

anemia, which occurs as a result of a decrease in red blood cells

shortness of breath, which can occur during regular physical activity

enlarged lymph nodes, which you may notice more in your neck

unexplained weight loss that may result from a loss of appetite

increased risk of infections of the lungs, skin, kidneys, or other areas, which may be due to decreased immunoglobulin and neutrophil counts in your blood

fatigue

low grade fevers

night sweats

Your treatment team will be able to provide you with the best information about your overall condition. They'll also be able to provide treatment based on your needs.

When your doctor or healthcare team makes a CLL diagnosis, ask for clarity if you're unsure about the terms they're using. They should be able to tell you whether they believe you have an aggressive form of CLL and which treatment options they recommend based on the diagnosis.

Also, you can seek a second opinion from another healthcare professional. They may run additional tests or find additional information that may help guide you to successful treatment.

Here are some frequently asked questions about aggressive CLL.

What is an aggressive form of CLL?

The most aggressive form of CLL is known as Richter's syndrome.

What is the life expectancy of someone with CLL?

According to the National Cancer Institute, the overall 5-year survival rate for CLL is about 88% between 2013 and 2019.

What are the symptoms of end-stage CLL?

Symptoms include anemia, shortness of breath, enlarged lymph nodes, and unexplained weight loss.

Is CLL considered a terminal illness?

Medical professionals consider CLL to be an incurable illness.

Aggressive CLL is a fast-growing form of CLL that typically requires treatment right away. If you receive a diagnosis of this condition, you'll likely need more aggressive treatment.

Aggressive CLL is associated with an overall worse survival rate and outlook than indolent CLL, but proper and timely treatment can help improve both. Your care team will work closely with you to determine the best treatment plan for your situation.

Taking Care Of Yourself With Chronic Lymphocytic Leukemia

When you live with chronic lymphocytic leukemia, you can feel your best by embracing a healthy lifestyle, getting emotional support, and planning ahead with your cancer doctor or oncologist. These steps can help you take charge of problems that can come up, like pain, stress, and treatment side effects.

Here are some simple ways to boost your physical and mental health.

Get a Survivorship Care Plan

Most people with chronic lymphocytic leukemia live for many years. The cancer can grow slowly. In that case, your doctor might choose to track it with tests rather than treat you for it right away. But once they see signs that the cancer is progressing, you'll probably need treatments on and off for years.

Your doctor can help you stay on top of your ongoing medical care. Ask them to make you a long-term roadmap called a survivorship care plan. The plan could include info like:

A schedule for when you'll get follow-up exams

Other medical tests you might need in the future, like exams that check for long-term effects from your cancer or treatments

A list of lingering side effects your treatment might cause

Recommendations for diet and exercise

Reminders to go to all your appointments with your primary doctor, who will continue to check on your general health 

Speak Up About Treatment Side Effects

Your doctor has several ways to treat this type of leukemia, including chemotherapy, targeted drugs, and monoclonal antibodies. Since nearly any cancer treatment can bring on side effects, it's important to let your care team know as soon as you have any.

Certain side effects, like fatigue and nerve pain, can last for months or years. Other ones can show up years after your treatment has ended. Those are called late side effects. Depending on the treatment you got, they can include high cholesterol, infertility, and heart problems.

Your doctor can prescribe medications that either give you relief from side effects or prevent them from happening in the first place. If you have serious side effects, they could recommend a change in your treatment plan. Ask your doctor about expected side effects and prevention meds before you start a new treatment.

Consider Complementary Therapies

Nonmedical treatment doesn't replace your treatment plan, but you can get these complementary therapies along with your regular care. Some may ease your symptoms and side effects. Just know that they don't help everyone.

Check with your doctor before you try one, so they can make sure it's safe for you. Just a few examples of complementary therapies that may help people with cancer are:

Acupressure. A practitioner rubs or puts pressure on specific parts of your body. It may help control your symptoms.

Acupuncture. A practitioner inserts very thin needles into your body, which may relieve mild pain and certain types of nausea.

Aromatherapy. A practitioner massages essential oils into your body or you breathe them in. The fragrance may ease stress and nausea.

Massage therapy. When a therapist rubs, kneads, and manipulates your muscles and other soft tissues, it can ease stress, anxiety, depression, and pain. It might also make you feel more alert.

Meditation. Studies suggest that when you take time to quietly focus on the present (by paying attention to your breathing, for example), it may ease stress, anxiety, depression, and pain.

Eat a Healthy Diet

It's extra important to eat well while you're getting treatment for chronic lymphocytic leukemia.

In general, fill your plate with fruits and vegetables, lean protein, foods with omega-3 fats, and whole grains. If you don't have the appetite to eat three big meals a day, you can have four or five smaller ones instead.

You can ask your doctor, a nutritionist, or a dietitian to come up with an eating plan that's right for you. The Leukemia & Lymphoma Society can connect you with a registered dietitian for a free one-on-one consultation.

Remember that it's always best to get essential nutrients from food, not pills. Don't take any supplements unless you get your doctor's OK. There's a chance that certain vitamins, minerals, and herbs -- even ones labeled "natural" -- could affect your cancer treatment or bring on side effects.

Get Regular Exercise

Routine workouts can bring you lots of benefits. Among the perks, exercise may:

Give you more energy.

Lift your mood.

Ease stress, anxiety, and depression. 

If you're not active already, ask your doctor to help you get started. They can come up with a fitness program that's safe for you. If you've been working out for a while, pace yourself and set healthy exercise goals. Don't push yourself to the limit or work out as intensely as you did before you got leukemia.

There are lots of great ways to get exercise. Aerobic workouts like walking, jogging, or swimming get your heart pumping. Resistance training exercises like weightlifting build strength, protect your joints, and build bone mass. Even household chores are good for you, since they get you moving.

You could also ask your doctor if mind-body workouts like tai chi and yoga might be right for you. Tai chi combines flowing, balance-building movements with breathwork and meditation. It helps some people with cancer ease pain, fatigue, and sleep problems. Yoga involves holding poses that help you get more flexible while also paying attention to the breath. It may improve anxiety, depression, and stress.

Take Charge of Stress

You might need to juggle all of your usual responsibilities -- like work, school, or parenting -- while you're managing your leukemia. It's important to give yourself some "me time."

Find ways to unwind so you can keep your stress in check. You could:

Take a few minutes each day to relax in a quiet, peaceful place.

Do things you enjoy: Read a book, play with your pet, or listen to music.

Write down what you're feeling in a journal to help process your emotions.

Confide in friends and family, in person, over the phone, or by video chat.

Ask loved ones for help with errands or other chores when you're low on time and energy.

Join a Support Group

This is a good way to meet other people with leukemia who can understand what you're going through. In support groups, people share experiences, exchange info, and encourage one another through stressful times.

You can ask your care team where to find a local support group. You can also search for one in your region through the Leukemia & Lymphoma Society.

Get Help for Depression or Anxiety

It's normal to feel down or overwhelmed at times when you're living with cancer. But if you're sad, depressed, or anxious every day for 2 weeks or more, reach out to your doctor for help.

Ask them to refer you to a mental health professional, like a psychiatrist or a counselor, who can give you treatments that will improve your mood. Options like talk therapy and medication can help you feel like yourself again.

If you need help finding counseling services, call the Leukemia & Lymphoma Society at 800-955-4572.

Other Ways to Give Yourself TLC

Get quality sleep. Chronic lymphocytic leukemia and its treatments can make you feel tired and more tense at times. Enough shut-eye every night can help you recover your energy and ease stress. Talk to your doctor if you're having a hard time falling or staying asleep.

If you smoke, quit. You can ask your doctor to help you kick the habit.

Cut back on alcohol if you drink a lot. In general, experts recommend that you limit yourself to one drink a day if you're a woman and two if you're a man. Ask your doctor what's safe for you.

---