A growing need for a safe and effective treatment

Blood cancers affect over 1.1 million people in the United States annually, with over 156,000 new cases estimated in 2014 alone

After exhausting all options, including chemotherapy, radiation therapy and immunotherapy, a BM/HSC transplant is typically the only remaining treatment option for blood cancer patients.

Hemogenyx Pharmaceuticals seeks to address the following problems that arise with BM/HSC transplants

High failure rate of BM/HSC transplants

Up to 50% of BM/HSC transplants fail due to the body’s rejection of the transplant, complications from the procedure or a relapse of the disease.

Acute shortage of BM/HSC donors

At least 60% of patients who require a BM/HSC transplant are unable to find a match based on data from the Health Resources Services Administration (“HRSA”).

Preparation of patients for BM/HSC

BM/HSC transplantations require the prior conditioning (preparation) of patients for transplantation. Conditioning of a patient for a BM/HSC transplant is a critical element of the procedure, serving two main purposes:

  1. It provides adequate immunosuppression of the patient and clears sufficient niche space in the bone marrow for the transplanted HSC. This allows transplanted cells to engraft in the recipient.
  2. It often helps to eradicate the source of the malignancy

Severe side-effects

Conditioning of patients has traditionally been achieved by administering maximally tolerated doses of a cocktail of chemotherapeutic agents with or without radiation. All preparative regiments that are currently in use are toxic and have severe side effects that can be life threatening due to their off-target activity.

  • High mortality and morbidity rates
  • Radiation damage to the heart or lungs
  • Problems with the thyroid or other hormone-making glands
  • Problems with fertility
  • Damage to bones or problems with bone growth
  • Development of another cancer years later