A New Treatment for leukemia
Chronic Lymphocytic Leukemia (CLL) is the
most common form of leukemia in the Western world. In the USA, 20,700
new cases are diagnosed each year, predominantly in older men. The
disease is characterized by the overproduction of white blood cells
called B lymphocytes, which normally play a role in protecting the body
from infections. Over time, this overproduction leads to the
accumulation of these cells in the bone marrow, lymph nodes, and spleen.
Many patients experience no symptoms for
years, with the disease often discovered during routine blood tests that
reveal elevated lymphocyte counts. However, as the disease progresses,
it can cause symptoms such as swollen lymph nodes, an enlarged spleen,
reduced bone marrow function (leading to decreased hemoglobin and
platelets), recurrent infections, significant weight loss, and night
sweats.
The course of the disease varies widely, ranging from cases requiring
only long-term observation to more aggressive forms necessitating
immediate treatment.
The Era of Chemotherapy is Over
In recent years, a deeper understanding of the biology of CLL has
revolutionized its treatment. Chemotherapy, once the standard, has been
replaced by targeted biological therapies that selectively attack
cancerous cells without harming healthy ones. These treatments are not
only more effective but also have significantly fewer side effects,
allowing patients to enjoy prolonged remission and a better quality of
life.
Two Main Groups of Treatments
Until recently, CLL treatments primarily fell into two categories:
BTK Inhibitors: These target the BTK enzyme, essential for the
survival and proliferation of CLL cells. Current drugs irreversibly bind
to the enzyme, but resistance often develops, rendering the treatment
ineffective as the disease progresses.
BCL-2 Inhibitors: By inhibiting this protein, these drugs induce the
death of CLL cells. In some cases, they are combined with antibodies
targeting specific markers on CLL cells, enhancing the treatment's
effectiveness. When one type of treatment fails, patients often
transition to the other.
Progress in Advanced Disease
Until recently, patients whose disease advanced after these two lines of
treatment faced poor prognoses, with life expectancy of less than six
months and no viable options. This has now changed thanks to
advancements in treatment.
One illustrative case involved a man with resistant CLL, severe lymph
node swelling, and debilitating weakness. Despite previous treatments
ceasing to work, a new therapy provided through his private insurance
led to remarkable improvement. Within three weeks, he regained mobility,
resumed daily activities, and no longer required blood transfusions.
His blood counts normalized, and his quality of life significantly
improved.
Overcoming Drug Resistance
Clinical trials have introduced promising new BTK inhibitors that
address resistance caused by mutations. Approved by the FDA, these drugs
offer hope to patients who previously had no options. They provide a
breakthrough by overcoming resistance mechanisms, reducing side effects,
and maintaining a high quality of life.
Expanding Access to New Treatments
These innovative therapies for advanced CLL are currently under review
for inclusion in Israel's healthcare basket. Efforts must focus on
making these treatments accessible to patients who have exhausted other
options. By doing so, we can help extend their lives and allow them to
create meaningful memories with loved ones while preserving their
quality of life.