science

Cure on the horizon: Cell and gene therapy

For the first time, the promise of lasting remission for a few cancers such as leukemias, lymphomas and mutiple myeloma, as well as genetic disorders is on the horizon. Cell and gene therapies could provide lasting cure by either rectifying genetic deficiencies that cause disease or, through genetic engineering, modify healthy immune cells to target cancerous cells smartly. Cell and gene therapies could thus restore the body to its disease-free state in the case of simple genetic diseases or enhance the immune system to target abnormal cells as with cancer.

Cellular immunotherapies: Living drugs

The immune system is the body’s natural defense against infections and old or abnormal cells including those that make up cancer. Cellular immunotherapy strategies utilize immune cells from the patient (or donor) that are then genetically altered, outside the body (ex-vivo), and re-infused into the patient. Since live cells are re-engineered to fight the disease, cellular immunotherapies are considered ‘living drugs’. Living drug is not a ‘pill’, it’s a process, personalised to patient, thus intrisically more expensive.

These therapies have the potential to address complex conditions previously considered untreatable.

Lymphocytes, a type of white blood cells, is emerging as the cornerstone of cell-based immunotherapies.

Currently cell-based immunotherapies that are currently being evaluated in the fight against cancer seek to utilize:

  • Tumor-Infiltrating Lymphocytes
  • Engineered T Cells with modified T Cell Receptors (TCR) or Chimeric Antigen Receptor
  • Natural Killer (NK) Cells
  • Monocytes

How living drugs are made

In cell therapies, the patient or donor cells are collected and processed in specialized labs. Here, they are genetically engineered to identify and kill tumor cells.

The cells are grown in number, formulated and re-injected into the patient. The patient now has an army of weaponized T cells with enriched responses that can seek and destroy cancer cells.

Making a living drug (CAR-T)
Source: www.ohcare.com/service/car-t-cell-therapy/

The cell therapy advantage

One shot cure
The promise of a potential cure or lasting remissions without cancer with a one-time short infusion treatment (typically), quite unlike conventional cancer treatments where successive rounds of treatment are needed.
Short treatment, quicker recovery
The absence of aggressive chemotherapy and short treatment time significantly reduces recovery time in patients.
Long term benefits
Since these therapies are living drugs with cells that persist, benefits such as protection against relapse can last for many years.
Acceptance
For autologous therapies, the cells drawn from the patient’s body when re-injected are accepted readily—there is no host vs graft disease (unlike what is observed in bone marrow transplants).
Better quality of life
Without the toxic side-effects typically associated with chemotherapy, or frequent hospital visits for drug infusions, cell therapies provide better quality of life to patients and eases the burden of care for their families.

Immuneel’s R&D roadmap

As we seek to disrupt cancer care in India, our immediate focus is to treat blood cancers. Immuneel’s first investments in the cell therapy platforms are on autologous CAR-T cell therapies which have shown promising results in patients. 

Our first set of development programs are CAR-T cell therapies targeting single and dual receptors for adult lymphomas, adult leukemias and childhood leukemia, in patients who have failed existing therapy.

Our Research & Development center is recognized by DSIR (Department of Scientific and Industrial Research, Government of India).

CAR-T cell therapy: The current frontier of cell therapies

CAR stands for Chimeric Antigen Receptor; the T refers to T cell, a type of killer cell in the immune system.

Normally, T cells have receptors on their surfaces which can detect and attach to any disease cell and kill it. However, in cancer, tumor cells can escape detection by T cells and proliferate.

In autologous CAR-T cell therapy, the patient’s T-cells are extracted, genetically modified and re-injected. These genetically modified cells can be programmed to express new receptors on the cell surface and/or be activated to recognize markers on the cancerous cell allowing them to bind to the cancer cell and kill it.

These genetically modified cells combine both antigen-binding and T cell activating functions through a single receptor.

Collaborating globally to advance our research

Immuneel is committed to continue its development efforts into next generation cellular immunotherapy platforms and technologies aimed at various cancers and other rare diseases.

We are collaborating with our partners globally on targets beyond lymphomas, leukemias and mutiple myeloma, platforms beyond autologous therapies, towards advancing technology in construction design, and building more efficiency in process development, manufacturing and delivery to provide affordable solutions.

Our collaborations will enable our mid to long term pipeline delivery.

Blood cancers to solid tumours

Immuneel is currently focused on treatments for leukemias and lymphomas. It aims to develop cell therapies for other diseases like solid tumours as well.

Autologous to allogeneic

Current treatments like CAR-T are autologous—a personalised treatment, made by weaponizing the patient’s own immune cells. In the future, the aim is to make available a non-personalised, off-the-shelf version of cell and gene medicines. Called allogeneic, they will use human immune cells, but not the patient’s own.

Beyond CAR-T

While CAR-T can revolutionise cancer treatment today, it also opens the door to other cell and gene therapies, which harness the body’s cells to combat disease. Innovation will aim to improve every aspect of the treatment: the cells targeted, the modifications we design and the methods of delivering curative cells.